Clinical Trial Updates

The Latest Updates (posted May 8, 2013)...

Dosing Begins in Phase I Study of Marburg Virus Treatment

Sarepta Therapeutics, Inc. in May announced that it has initiated dosing in a Phase I multiple-ascending dose clinical trial of AVI-7288, the company's lead drug candidate for the treatment of Marburg virus infection. The study is designed to characterize the safety, tolerability, and pharmacokinetics of AVI-7288 after repeat dosing in healthy adult volunteers. The initiation of this study follows the successfully completed Phase I single-ascending dose study, which showed AVI-7288 was well tolerated in healthy volunteers. Sarepta is developing AVI-7288 under a contract from the U.S. Department of Defense through the Joint Project Manager Transformational Medical Technologies Project Management Office. AVI-7288 utilizes Sarepta's advanced and proprietary PMOplus® chemistry. The randomized, double-blind, placebo-controlled study will be overseen by an independent data and safety monitoring board, which will review safety and clinical laboratory data after each dose cohort prior to enrolling the next highest dose cohort. Thirty-two volunteers will be enrolled in one of four cohorts made up of eight subjects each. The cohorts will include six subjects who receive the therapeutic, and two who will receive a placebo. Source: Marketwired 5/7/13

Trial Opens in South Korea for Newly Diagnosed Glioblastoma

Novocure announced in May that the EF-14 pivotal clinical trial for patients with newly diagnosed glioblastoma (GBM) brain tumors has enrolled its first patient in South Korea. This randomized, controlled trial is designed to enroll approximately 700 patients and will test the efficacy and safety of NovoTTF™ therapy when added to the current best standard of care for newly diagnosed GBM. NovoTTF therapy is delivered by a portable, noninvasive medical device designed for continuous use throughout the day by the patient. The device, the NovoTTF-100A system, has been shown in in vitro and in vivo studies to slow and reverse tumor growth by inhibiting mitosis, the process by which cells divide and replicate. The device inhibits mitosis by creating a low intensity, alternating electric field within the tumor that exerts physical forces on electrically charged cellular components, preventing the normal mitotic process and causing cancer cell death prior to division. Newly diagnosed GBM patients in the United States, Europe, and Asia may now enroll in the trial, which is enrolling at more than 70 hospitals around the world, and will include nine centers in South Korea. The opening of the trial in South Korea followed a full review of the clinical trial protocol and device by the Korean Food and Drug Administration. Source: PR Newswire 5/7/13

IND Filed for Phase II Osteoarthritis Trial

RNL BIO has announced that it has filed an Investigational New Drug (IND) application with the Food and Drug Administration (FDA) to begin clinical trials with its adipose-derived stem cell product, termed RNL-JointStem, for the treatment of osteoarthritis (OA) in the U.S. Assuming approval of the IND by the Center for Biologics Evaluation and Research at the FDA, RNL BIO plans to initiate its double-blinded, randomized, positive-control Phase II clinical trial during the third quarter of 2013 in Sugarland, Texas. Phase I and II trials of RNL-JointStem have already been completed under the authority of the Korean Food and Drug Administration. If the U.S. trial is successful, the company hopes it can lead to a paradigm shift in the treatment of OA by using stem cell products derived from a patient's own fat tissue and administered in a single injection, unlike the many invasive treatments currently used in the treatment of OA. The study aims to confirm the efficacy of RNL-JointStem for cartilage regeneration, pain reduction, and joint function improvement for OA patients. The clinical trial will compare RNL-JointStem to existing modalities utilizing hyaluronic acid. The intended market for RNL-JointStem is patients under 60 years of age for whom there is no efficacious therapy at present. Source: PR Newswire 5/2/13

Study Shows Improvement in Mood, Cortisol Levels with Botanical Blend

Dr. Shawn Talbott presented a new study on Relora® at the Experimental Biology 2013 Meeting. The study, conducted by Supplement Watch/GLH Nutrition, found that a proprietary blend of Magnolia officinalis and Phellodendron amurense significantly improved mood and other psychological states in moderately stressed subjects. The study randomly assigned 60 subjects (56 of whom successfully completed the study) experiencing moderate levels of perceived psychological stress to take Relora or a placebo twice a day for four weeks, a time period selected to minimize the influence of short-term mood changes that result from daily stressors. All subjects submitted three saliva samples (morning, mid-day, and evening) for cortisol testing and completed a baseline Profile of Mood States questionnaire on the first and last day of the study. The results indicated that daily use of Relora reduced cortisol exposure and perceived stress while improving a variety of mood parameters--most significantly, anger (reduced by 42 percent) and fatigue (reduced by 31 percent). The botanicals in Relora have been used in traditional Chinese medicine for thousands of years, and have been recognized in the West for treating stress. Source: Marketwired 5/2/13

Trial in Previously Treated Metastatic Bladder Cancer Now Open

OncoGenex Pharmaceuticals, Inc. in late April announced initiation of the Borealis-2™ clinical trial, an investigator-sponsored, randomized Phase II trial evaluating OGX-427 in combination with docetaxel in patients with advanced or metastatic bladder cancer who have disease progression following first-line, platinum-based chemotherapy. Borealis-2 will randomize approximately 200 patients to receive either OGX-427 plus docetaxel treatment or docetaxel treatment alone. Patients may also continue weekly OGX-427 infusions as maintenance treatment until disease progression or unacceptable toxicity, if they complete all 10 planned cycles of docetaxel or are discontinued from docetaxel due to docetaxel toxicity. The primary endpoint of the trial is overall survival, with secondary objectives to evaluate safety, tolerability, tumor response rates, and the effect of therapy on heat shock protein (Hsp27) levels and circulating tumor cells. Borealis-2 is the second randomized, controlled clinical trial of OGX-427 in advanced bladder cancer. Borealis-1™ is a company-sponsored, randomized, placebo-controlled Phase II trial of OGX-427 in combination with first-line gemcitabine and cisplatin in patients with metastatic bladder cancer. If either Borealis trial shows a survival advantage, OncoGenex plans to initiate conversations with the Food and Drug Administration about the possibility of a Phase III trial of OGX-427 in bladder cancer as part of the ORCA™ program. Borealis-2 is being conducted at approximately 30 sites in the U.S. and is sponsored by the Hoosier Oncology Group. Source: PR Newswire 4/30/13

Phase I Trial Initiated for Anticancer Compound

Lixte Biotechnology Holdings, Inc. has reported the start of a Phase I trial to determine the appropriate dose of its potentially first-in-class, anticancer compound, LB-100, when given alone and then when given in combination with docetaxel (Taxotere®). Details of the trial under way at an National Cancer Institute-designated Comprehensive Cancer Center are available on ClinicalTrials.gov. LB-100 inhibits an enzyme necessary for cancer cells to recover from damage to DNA. In mouse model systems of human cancers, LB-100 potentiates the activity of widely used anticancer regimens that exert their therapeutic benefit by damaging DNA, without apparent increases in their toxicity. Source: PR Newswire 4/25/13

Earlier Updates...

Foundations Fund Phase I Study of Cancer Drug in Alzheimer's Patients

The Alzheimer's Drug Discovery Foundation and BrightFocus Foundation in April announced a funding collaboration that will support a Phase I human clinical trial to evaluate the cancer drug bexarotene as a treatment for Alzheimer's disease. Through this partnership, the organizations are working to address a gap in funding that exists for these types of early stage studies. The trial will be conducted by ReXceptor Inc., a biotechnology company set up by researchers at Case Western Reserve University, and will examine the efficacy of bexarotene on key Alzheimer's disease indicators, including brain amyloid beta and apolipoprotein E. Bexarotene has been commercially available in the US for more than a decade as an orally administered treatment for T-cell lymphoma. Recently, researchers demonstrated that bexarotene might also be useful in the treatment of Alzheimer's disease. The trial, set to commence later this year, will evaluate at least 12 healthy human subjects. This study will be conducted by C2N Diagnostics and Compass Research, Inc. Source: PR Newswire 4/24/13

Positive Results Seen in Refractory RA with Allogeneic Stem Cell Product

TiGenix in April announced positive six-month safety data of its Phase IIa study of Cx611 in rheumatoid arthritis (RA), as well as a first indication of therapeutic activity on standard outcome measures and biologic markers of inflammation for at least three months after dosing. The multicenter, randomized, double-blind, placebo-controlled trial enrolled 53 patients with active refractory RA (mean time since diagnosis 15 years), who failed to respond to at least two biologics (mean previous treatment with three or more disease-modifying antirheumatic drugs and three or more biologics). The study design was based on a three-cohort, dose-escalating protocol. For both the low- and medium-dose regimens, 20 patients received active treatment versus three patients on placebo; for the high-dose regimen, six patients received active treatment versus on on placebo. Patients were dosed at day 1, 8, and 15 and were followed up monthly over a six-month period. Follow-up consisted of a detailed monthly workup of all patients measuring all predefined parameters. The aim was to evaluate the safety, tolerability, and optimal dosing over the full six months of the trial, as well as exploring therapeutic activity. Only one patient suffered serious adverse events that led to discontinuation of the treatment. All other side effects were mild and transient. Importantly, the first results show no signs of hematological side effects or thrombosis. Source: Marketwired 4/22/13

Phase II Interferon-Free Trial Initiated in HCV

Achillion Pharmaceuticals, Inc. in April announced that it has initiated an international Phase II clinical trial with sovaprevir and ACH-3102 for the treatment of genotype 1 chronic hepatitis C (HCV). The trial will evaluate an all-oral, 12-week, interferon-free regimen consisting of sovaprevir, ACH-3102, and ribavirin in patients with chronic HCV who have not received prior therapy. In previous trials, both sovaprevir and ACH-3102 were shown to be efficacious, safe, and well tolerated, and had demonstrated a high barrier to resistance. This trial which will evaluate safety, tolerability, and efficacy in up to 50 treatment-naive patients. Initially, 30 patients will be enrolled and randomized to receive either a combination of sovaprevir, ACH-3102, and ribavirin or matching placebos. Once-daily 200 mg or 400 mg doses of sovaprevir with ACH-3102, given as a 150 mg loading dose followed by a 50 mg once-daily dose, in combination with ribavirin, will be evaluated in this trial. Primary endpoints include safety, tolerability, and sustained virologic response four weeks after the completion of dosing. The trial is expected to enroll patients at sites in the United States, Canada, New Zealand, and Australia. Source: Globe Newswire 4/16/13

Trial Launched to Evaluate New Pacing System in MRI Environment

The first patient in the United States has been implanted with the Boston Scientific Corporation next-generation ImageReady™ MR Conditional pacing system in the SAMURAI clinical trial. The study is designed to confirm the safety and effectiveness of the system in the magnetic resonance imaging (MRI) environment. Pacing systems are designed to treat bradycardia, a condition in which the heart beats too slowly depriving the body of sufficient oxygen. MRI provides detailed images of organs and tissues without the use of radiation. While those images can help clinicians make informed decisions about treatment and care, most pacemakers are not compatible with MRI technology and therefore patients may not have access to the sophisticated scanning capabilities of the diagnostic system. The next-generation ImageReady pacing system is comprised of the Boston Scientific Ingenio™ MRI pacemaker family and the new INGEVITY™ MRI pacing leads. The proprietary technology is aimed at reducing MRI interference with device performance. In addition, the INGEVITY pacing lead platform is designed to provide key enhancements in handling and fixation compared to standard leads available today, and is specifically engineered to function in the MRI environment. The SAMURAI trial is a prospective, open-label, two-group randomized, multicenter, global clinical study designed to support U.S. Food and Drug Administration regulatory approval. The trial is expected to enroll approximately 363 patients at 45 centers in seven countries. The first patient implant in the United States occurred at OhioHealth's Riverside Methodist Hospital in Columbus, Ohio. The first study implant in the world occurred in Malaysia at the Institut Jantung Negara (Kuala Lumpur). Source: PR Newswire 4/15/13

Study Investigating Treatment for Noninfectious Anterior Scleritis Opens

XOMA Corporation in April announced the National Eye Institute, one of the U.S. National Institutes of Health, has opened its noninfectious, active, anterior scleritis trial for patient enrollment. The open-label, single-arm, Phase I/II study is designed to assess the safety and potential efficacy of gevokizumab in patients experiencing noninfectious, active, anterior scleritis, which is the inflammation of the sclera (the fibrous white membrane surrounding the eyeball excluding the cornea). This is XOMA's third indication in a program of three proof-of-concept studies for gevokizumab. The institute is expected to enroll 10 patients with scleral inflammatory grade of greater than or equal to 1-plus in at least one eye using a standardized photographic scleritis grading system. All patients will receive 60 mg of gevokizumab dosed every four weeks for a 16-week period. Patients who respond will have the option to continue treatment for an additional 20 weeks. Responders in the study are defined as patients who experience a two-step reduction on a five-point scale from 0 to 4 or reach Grade 0 in scleral inflammation in the study eye. Secondary measurements will include changes in visual acuity, changes in intraocular pressure, and changes in scleral grading. Safety will be monitored throughout the trial. Source: Globe Newswire 4/10/13

Sites Announced for First Trial in Fatty Liver Disease with Advanced Fibrosis

Galectin Therapeutics announced details in April on its first-in-human Phase I clinical trial supporting a proposed indication of GR-MD-02 for treatment of nonalcoholic steatohepatitis (NASH, or fatty liver disease) with advanced fibrosis. The trial will be conducted in six centers in the United States that have extensive experience in conducting studies in liver disease. Patients will receive four weekly doses of GR-MD-02 and, while evaluation for safety is the primary objective, a series of biomarkers will be evaluated to assess for early signs of efficacy. The clinical trial investigator kick-off meeting was held in Atlanta, Ga., on April 5, led by Galectin and CTI Clinical Trials & Consulting Services Inc. of Cincinnati, Ohio, a clinical research organization with extensive experience in liver-related clinical trials. The study sites include the Brooke Army Medical Center at Fort Sam Houston in San Antonio, Texas; Indiana University School of Medicine in Indianapolis; St. Louis University School of Medicine in Missouri; Virginia Commonwealth University School of Medicine in Richmond; Emory University School of Medicine in Atlanta, Ga.; and Icahn School of Medicine at Mount Sinai in New York City. It is anticipated that the enrollment and infusion of the first cohort will be completed by the end of the second quarter of 2013. The double-blinded study is planned to enroll the first cohort followed by potential sequential cohorts receiving increasing doses of GR-MD-02 with eight patients in each cohort randomized 6:2 (study drug:placebo). After the safety of the first dose is assessed, the patients will receive three additional doses weekly doses of GR-MD-02. The dose will be increased after assessment of each cohort to presumptive target therapeutic levels, which is currently projected to take one year from the start of the study. Source: PR Newswire 4/9/13

Positive Results Reported from Phase I/II Cancer Vaccine Test in Multiple Myeloma

Vaxil Bio announced in April that its wholly owned subsidiary Vaxil BioTherapeutics has reported that a Phase I/II clinical trial (VAXIL-001) of the company's therapeutic vaccine ImMucin, in 15 patients with multiple myeloma (a hematological cancer), has met all endpoints with considerable success. All patients enrolled in this study were experiencing a gradual re-emergence of the disease after a period of remission that had been attained following an autologous stem cell transplantation. The results indicate a high safety profile for ImMucin. In addition, all patients demonstrated a strong immunological response to ImMucin. Furthermore, nine of the 15 patients demonstrated a clinical response. Of these, five patients ended the study in a state of complete response and a further four ended the study with stable disease, which requires no further treatment. The VAXIL-001 trial was carried out in two Israeli medical centers: Hadassah Ein Kerem University Hospital in Jerusalem and Rambam Medical Center in Haifa. Patients received either six or 12 ImMucin injections at a dose of 100 or 250 micrograms, along with GM-CSF (Leukine) at a dose of 250 micrograms. The ImMucin vaccine is a unique 21 amino acid sequence (peptide) taken from the MUC1 antigen (cancer marker), which is expressed on the myeloma cancer cells. ImMucin educates the patient's immune system to identify and destroy cells that express this particular antigen. The company intends to publish the full results of this clinical trial in an appropriate scientific journal as soon as possible. Source: Globe Newswire 4/9/13

Company Receives ISP Approval of Protocol for Phase II Trial in Chronic Migraine

Trigemina, Inc. in April announced that the Instituto de Salud Publica (ISP) of Chile has approved the company's protocol for its Phase II clinical study of TI-001, intranasal oxytocin, for the treatment of chronic migraine. The TRIG-05 study is expected to begin enrollment in April, with a primary clinical endpoint of demonstrating efficacy, tolerability, and safety of TI-001. In preclinical and human pilot studies, treatment with TI-001 has shown promising results for use as a safe and effective therapy for all forms of chronic and subacute head pain. The new study is a placebo-controlled, double-blind, randomized-withdrawal, and enrollment-enriched trial of TI-001 in chronic migraine patients. A total of 96 patients will be enrolled in four trial sites in Chile and another trial site in Argentina to begin in June. Additional objectives will include determining the appropriate dose for a future Phase III trial and to explore baseline IL-6 plasma levels as a potential biomarker for efficacy response. Source: PR Newswire 4/8/13

First Patient Enrolled in Phase III Trial of System for Alcohol-Induced Liver Decompensation

Vital Therapies, Inc. in April announced enrollment of the first patient in a Phase III trial designated VTI-208. The patient was enrolled at Emory University Hospital in Atlanta, Ga., last month. VTI-208 is a randomized, open-label, multicenter, controlled trial comparing the company's investigational bio-artificial liver support system, ELAD®, plus standard-of-care versus standard-of-care alone in 200 patients with alcohol-induced liver decompensation. The primary endpoint is 90-day overall survival and the safety and tolerability of the two treatment groups will also be compared. Patients will also be followed for five years in an extension protocol that will gather further survival, transplant, and other health data. Several other centers that will participate in the study are also already open for patient enrollment, and the company anticipates the study to open up 25 clinical sites by year-end. The company received scientific advice from the European Medicines Agency and U.S Food and Drug Administration on the design and planned analysis of the VTI-208 trial, which will be conducted at sites in the United States, Europe, and Australia. Later this year, Vital Therapies plans to initiate two other randomized, controlled Phase III clinical trials: VTI-210, in patients with biopsy-proven acute alcoholic hepatitis; and VTI-212, a trial in patients with fulminant hepatic failure. Source: Marketwire 4/4/13

MPA Approves CTA for Neo-Kidney Augment Phase I Trial in Sweden

Tengion, Inc. in early April announced the acceptance of the company's Clinical Trial Application (CTA) filed with the Medical Products Agency (MPA) in Sweden to initiate a Phase I clinical trial to evaluate the safety and delivery of its Neo-Kidney Augment™ product in up to five patients with advanced chronic kidney disease (CKD). "We are excited to have received MPA approval for [the trial] in Sweden and expect to initiate the trial this quarter. We will use the data from this trial to establish safety and a delivery approach for our planned U.S. Phase I trial, which we expect to start in the fourth quarter of 2013," said John L. Miclot, president and chief executive officer of Tengion. The Neo-Kidney Augment is intended to prevent or delay the need for dialysis or kidney transplant by catalyzing the regeneration of functional kidney tissue in CKD patients. The trial will involve delivery of an active regenerative dose of Neo-Kidney Augment in patients with CKD, and is expected to enroll up to five patients in 2013, with each patient being followed for up to two years. This CTA acceptance follows Tengion's inspection by an Official Qualified Person for regulatory compliance with European Medicines Agency standards for clinical manufacturing, processes, management, and quality programs. Following the successful inspection, the company was issued a Declaration of Compliance to enable clinical production of the Neo-Kidney Augment at its cGMP manufacturing facility in Winston-Salem, N.C. Source: PR Newswire 4/1/13

Enrollment Completed for Phase II in Insulin Candidate for Diabetes

Biodel Inc. in late March announced the completion of patient enrollment in a Phase II clinical study of ultra-rapid-acting prandial insulin candidate BIOD-123 for the treatment of diabetes. The trial will evaluate the use of BIOD-123 versus Humalog® on measures of HbA1c, postprandial glucose excursions, glycemic variability, hypoglycemic event rates, and weight changes. Dr. Errol De Souza, president and chief executive officer of Biodel,stated: "Completing enrollment of the BIOD-123 Phase II clinical trial is a significant milestone for Biodel, having nearly doubled the number of subjects in our original trial design without changing the timelines. This [larger trial] will allow us to more effectively test the benefits of a product candidate that has a more rapid absorption profile than currently marketed insulins. We remain on track to report top-line data from this trial in the third calendar quarter of 2013." The trial is a randomized, open-label, parallel-group study being conducted at approximately 30 investigative centers in the United States. In the trial, approximately 130 patients with type 1 diabetes were randomized to receive either BIOD-123 or Humalog to use as their mealtime insulin during an 18-week treatment period. Both arms of the study use insulin glargine, sold as Lantus®, as the basal insulin. Source: Globe Newswire 3/28/13

Phase IIb Study Launched for Treatment of Uterine Fibroids

Neurocrine Biosciences, Inc. in March announced that a Phase IIb clinical trial to evaluate elagolix for the treatment of uterine fibroids has been initiated. Elagolix is an oral gonadotropin-releasing hormone antagonist in development by AbbVie for the treatment of uterine fibroids and endometriosis. Neurocrine and AbbVie entered into a collaboration and license agreement for elagolix during 2010. The new study is a randomized, parallel, double-blind, placebo-controlled clinical trial evaluating elagolix in women with heavy uterine bleeding associated with uterine fibroids. This study will evaluate 280 subjects over a six-month dosing period. The primary efficacy endpoint of the study is an assessment of the change in menstrual blood loss utilizing the alkaline hematin method comparing baseline to month six. Additional secondary efficacy endpoints will be evaluated including assessing the change in fibroid volume and hemoglobin. Bone mineral density will be assessed via DXA scan at baseline, the conclusion of dosing, and six months post-dosing. Source: PR Newswire 3/27/13

Positive Top-Line Data Announced for Drug for Frozen Shoulder

BioSpecifics Technologies Corp. in March announced positive, statistically significant top-line data from the Phase IIa study of Xiaflex® for the potential treatment of frozen shoulder (adhesive capsulitis). The open-label, controlled, dose-ranging Phase IIa study was conducted by BioSpecifics' strategic partner, Auxilium Pharmaceuticals, Inc., and was designed to assess the safety and efficacy of Xiaflex for the treatment of stage 2 unilateral idiopathic frozen shoulder in comparison to an exercise-only control group. The study involved 50 adult men and women at 11 U.S. sites. Four cohorts of 10 patients each received up to three ultrasound-guided extraarticular injections of varying doses of Xiaflex (ranging from 0.29 mg to 0.58 mg in three different volumes; 0.5, 1.0, or 2.0 mL), separated by a minimum of 21 days. All patients were instructed to perform home shoulder exercises. The fifth cohort of 10 patients received no Xiaflex injections and only performed home shoulder exercises. The study's primary endpoint was the change (in degrees) from baseline to the day 92 follow-up in active forward flexion in the affected shoulder compared to the exercise-only cohort. Safety assessments were made during all study visits and immunogenicity testing was performed at screening and day 92. Source: PR Newswire 3/26/13

Results from Phase IIb Lung Cancer Trial Disappoint

Cerulean Pharma Inc. in March announced that a randomized Phase IIb study of its experimental cancer treatment, CRLX101, in patients with advanced non-small cell lung cancer (NSCLC) did not meet its primary efficacy endpoint, overall survival benefit. Consistent with earlier clinical trials, CRLX101 showed signals of activity, including tumor reductions that meet RECIST criteria, and a favorable safety profile. CRLX101 is being studied in clinical trials in renal cell carcinoma in combination with Avastin®, and as a monotherapy in ovarian cancer, gastric cancer, and small cell lung cancer at leading U.S. academic institutions. Combination data with Avastin will be presented at the American Association of Cancer Research Annual Meeting in April. The Phase IIb NSCLC trial enrolled 157 patients in Russia and Ukraine who were randomized in a two-to-one ratio to two treatment arms, CRLX101 plus best supportive care (BSC) compared to BSC alone. The trial's primary endpoints were median overall survival and safety and tolerability. Study participants were additionally assessed for progression-free survival, objective tumor response, and pharmacokinetic parameters. The company will analyze the data from this trial to inform the further clinical development of CRLX101. Cerulean would like to extend its thanks to the patients and investigators who participated in this trial and the families of the patients. Source: PR Newswire 3/22/12

First Patients Enrolled in Global Phase III Program in Type 2 Diabetes

Intarcia Therapeutics, Inc. announced in March the enrollment of the first patients in FREEDOM-1, the first of four planned Phase III clinical trials studying the safety and efficacy of ITCA 650 (continuous subcutaneous delivery of exenatide) for the treatment of type 2 diabetes (T2D). Intarcia is conducting the FREEDOM clinical program with strategic partner Quintiles, Inc., the world's leading clinical research organization, which will be responsible for the global implementation of all four trials. This collaboration, along with strategic input from a world-class group of renowned diabetes experts, is designed to achieve the highest standards of quality, safety, and operational excellence. The FREEDOM clinical trial program aims to demonstrate that optimizing GLP-1 therapy with once- or twice-yearly dosing of ITCA 650 results in effective, well-tolerated glycemic control that virtually ensures longer-term compliance--a transformational approach to controlling T2D that aims to free patients from the frequent self-injections that all too often result in poor therapeutic compliance and control. The FREEDOM-1 study is a placebo-controlled, double-blind study involving T2D patients whose HbA1c is not controlled on zero to three oral antidiabetes drugs and whose HbA1c measures between 7.5 percent and 10 percent. The 450 patients will be randomized into three groups in a 1:1:1 ratio, evaluating ITCA 650 40 mcg/d and ITCA 650 60 mcg/d versus placebo. Subjects in the active arms will be treated with three-month devices for the first 13 weeks that deliver an initial dose of 20 mcg/d, and then treated with six-month ITCA 650 at doses of 40 or 60 mcg/d. The primary endpoint will be change in HbA1c at nine months; secondary endpoints include changes in weight, tolerability, and other measures of safety and efficacy. Source: PR Newswire 3/21/13

Phase I Clinical Trial Under Way for Advanced Ovarian Cancer

EGEN, Inc. in March announced that it has initiated a Phase I clinical trial of its novel immunotherapy agent, EGEN-001, in combination with PEGylated liposomal Doxorubicin (Lipodox) for the treatment of recurrent ovarian cancer. The EGEN-sponsored trial is conducted by a network of researchers led by Gynecologic Oncology Group (GOG) at member institutions under an agreement between the GOG and EGEN. Dr. Premal Thaker, of Washington University School of Medicine, is the study chair for the trial. "We are pleased to have this trial begin, as it advances the testing of our novel EGEN-001 product with an approved second-line treatment for platinum-resistant ovarian cancer," commented Dr. Khursheed Anwer, president and CSO of EGEN. "Long-term treatment with an immune modulating agent in conjunction with cytotoxic agents is a promising approach to combatting difficult to treat cancers." The product utilizes the company's proprietary TheraPlas® delivery technology, and is composed of interleukin-12 (IL-12) gene formulated with a biocompatible delivery polymer. IL-12 is a potent cytokine which works by enhancing the body's immune system against cancer and inhibiting tumor blood supply. EGEN-001 product is currently being examined as a single agent in a Phase II clinical trial for the treatment of platinum-resistant ovarian cancer and in combination with chemotherapeutic agents following cytoreductive surgery and HIPEC therapy in colorectal cancer. Source: PR Newswire 3/19/13

Partners Initiate Phase I Trial of Oral Treatment for Psoriasis

Virobay, Inc. and LEO Pharma A/S in March announced that their collaboration on the development of an oral treatment for psoriasis has reached an important milestone as Virobay has initiated a Phase I clinical trial of VBY-891--a selective cathepsin S inhibitor. The first trial of VBY-891 is a double-blind, randomized, placebo-controlled study designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single and multiple escalating doses in healthy adults, to guide Phase II clinical development plans for the product. Source: PR Newswire 3/18/13

Vaccine Candidate Studied Against Hand, Foot, and Mouth Disease

Sinovac Biotech Ltd. announced in March preliminary top-line data from its Phase III clinical trial assessing the efficacy, immunogenicity, and safety of the company's proprietary Enterovirus 71 (EV71) vaccine against hand, foot, and mouth disease (HFMD). The primary objective of the study was to evaluate the efficacy of the EV71 vaccine in the prevention of HFMD caused by EV71 in infants of 6 to 35 months old. The preliminary data showed that the vaccine was 95.4 percent efficacious against HFMD caused by EV71, and found good immunogenicity and safety for the vaccine. The double-blinded, randomized, placebo-controlled trial was conducted at three sites across China's Jiangsu province. Approximately 10,000 healthy infants completed the two-dose vaccination schedule (at 0 and 28 days) in the first quarter of 2012, prior to the HFMD epidemic season in China, followed by active monitoring period. In parallel, Sinovac conducted another clinical study that was comprised of 1,400 volunteers and designed to evaluate the consistency of three consecutive lots of EV71 vaccine manufactured by the company. The trial was conducted in children from 6 months to 5 years old. After receiving the vaccine, the ratios of neutralizing antibody GMTs on the 56th day of any two groups were calculated and the 95 percent confidence intervals of the ratios are all between 0.67 and 1.5, which indicates the immunogenicity of the three vaccine lots is equivalent. The study results showed consistent immune response for all three lots and a good safety profile. Source: PR Newswire 3/14/13

Enrollment Completed for Phase II Alzheimer's Trial

Ceregene, Inc. in March announced the completion of enrollment in its double-blind Phase II clinical study of CERE-110 (AAV-NGF), a gene therapy product designed to deliver nerve growth factor (NGF) for the treatment of Alzheimer's disease. The clinical study was carried out in collaboration with the Alzheimer's Disease Cooperative Study based at the University of California San Diego and funded by a grant from the National Institute on Aging, part of the National Institutes of Health. Forty-nine patients with mild-to-moderate Alzheimer's disease were treated with CERE-110 at 10 clinical sites throughout the U.S. Approximately half of the patients received CERE-110 while the other half received an appropriate sham (placebo) surgery control treatment. Patients will be followed for a minimum of two years with respect to safety, brain imaging, and measures of cognitive function and quality of life. The company also has completed enrollment in two controlled Phase II clinical trials of CERE-120 for Parkinson's disease. Source: PR Newswire 3/13/13

Phase III Trial in Multiple Myeloma to be Discontinued

Aeterna Zentaris Inc. in March announced that an independent data safety monitoring board (DSMB) has recommended discontinuing the ongoing Phase III study comparing the efficacy and safety of perifosine to placebo when combined with bortezomib (Velcade®) and dexamethasone in patients with relapsed or relapsed/refractory multiple myeloma. Based on the outcome of its preplanned interim analysis of efficacy and safety, the DSMB recommended that patient enrollment be stopped and the study discontinued. The DSMB reported that it was highly unlikely the study would achieve a significant difference in its primary endpoint of progression-free survival; no safety concerns were raised. Source: PR Newswire 3/11/13

Patient Enrollment Completed in Mitral Regurgitation Trial

Cardiac Dimensions®, Inc. in March announced that it has completed enrollment in its TITAN II clinical trial, a follow-up to the landmark TITAN clinical trial of the company's Carillon® Mitral Contour System®. TITAN II is a prospective, single-arm, European, multicenter clinical trial initiated to further evaluate an enhanced version of the system, which is a novel, minimally invasive therapy designed to treat heart failure patients suffering from functional mitral regurgitation (FMR), a condition in which blood flow to the body is reduced due to a poorly functioning heart valve. A total of 30 patients across five sites have been implanted with the Carillon system, and will be followed for a one-year period as part of the trial. Patients will be evaluated for improvements in mitral regurgitation, functional capacity, quality of life, and reverse cardiac remodeling. Published in the European Journal of Heart Failure, data from TITAN suggest that patients implanted with the system experienced significant relief from FMR and improvements in functional capacity and quality of life compared to a contemporaneously enrolled comparison group. Source: PR Newswire 3/8/13

Company Expects Impressive Phase III Enrollment in Brain Cancer Trial

Northwest Biotherapeutics, which is developing DCVax® personalized immune therapies for solid tumor cancers, announced in March that it expects to complete enrollment in its 312-patient Phase III clinical trial for glioblastoma multiforme brain cancer within a period that is faster or more efficient than relevant comparison trials with immune therapies for the same brain cancer. The company anticipates completing enrollment by the first quarter or early second quarter of next year, and expects to reach its first interim analysis for efficacy by approximately the third quarter of this year. The company undertook a limited period of enrollment in 2008, and then kept the trial going with the patients already enrolled, but suspended new enrollment due to resource constraints during the worst of the economic downturn, through the end of 2010. The company began the process of reactivating clinical trial sites for new enrollment in the first quarter of 2011, and resumed screening in the second quarter that year. "There has been widespread confusion in the investment community about the size and pace of clinical trials being conducted with various immune therapies for brain cancer," commented Linda F. Powers, CEO of Northwest Bio. "It is basic to clinical trials that [sponsors] must screen more patients than they enroll. Normally, there is no confusion about the fundamental difference between these: 'enrollment' means only the patients actually being treated (with drug or placebo) in the trial. This is a key metric for investors: it is the measure of the size and the pace of a trial." Source: PR Newswire 3/7/13

Treatment Trial Launched for Crohn's Disease

Canadian participants are being sought for a new Health Canada-approved clinical trial for the treatment of Crohn's disease, the Vancouver-based biopharmaceutical company Qu Biologics announced in March. Qu Biologics develops Site Specific Immunomodulators (SSIs), a novel class of immunotherapies that aim to restore balance to the body's natural defense system by potentially "rebooting" the affected organ's innate immune response. Health Canada has determined that outcomes from an SSI treatment candidate called QBECO, previously provided to a small number of Crohn's patients through a compassionate use program, support further study in controlled clinical trials. The company's Phase I/II trial will recruit 60 adults with active, uncontrolled, moderate-to-severe Crohn's disease and evaluate the potential clinical response and disease remission among participants treated with QBECO. The main purpose of this study will be to test whether this investigational treatment is safe and potentially effective for the treatment of Crohn's disease. Results could be expected as early as the second quarter of 2014, depending on recruitment. The randomized, placebo-controlled, double-blind study will be conducted in Vancouver. Currently, only Canadian residents are eligible to participate in the trial. Source: Marketwire 3/7/13

Seizure Treatment Generates Positive Results in Phase III Monotherapy Study

UCB in March announced positive results of a Phase III study designed to evaluate the efficacy and safety of conversion to lacosamide monotherapy in adult patients with partial-onset seizures with or without secondary generalization compared to a historical control. The study met its primary endpoint, demonstrating that the exit rate for patients on lacosamide (400 mg/day) was significantly lower than the historical control. UCB plans to submit these data as part of its supplemental New Drug Application for lacosamide to the U.S. Food and Drug Administration, which is planned in the second half of 2013. Lacosamide is currently approved in 36 countries as adjunctive therapy in the treatment of partial-onset seizures in adults with epilepsy. The international, historical-controlled, multicenter, double-blind, randomized trial evaluated lacosamide (400 mg/day) for conversion to monotherapy in 427 patients taking one to two other antiepileptic drugs. A lacosamide 300 mg/day arm was added to blind the treatment group and to ensure a study design consistent with the historical control studies on which the conversion to lacosamide monotherapy study was based. The topline results will be followed by full efficacy and safety analyses, which will be submitted for presentation at an upcoming epilepsy meeting. Lacosamide is not currently approved for use as monotherapy. Source: PR Newswire 3/5/13

First Patients Enrolled in Ireland for Hypertension Trial

ROX Medical in March announced enrollment of the first two Irish patients in the CONTROL-HTN randomized controlled trial of the ROX FLOW procedure for the treatment of resistant hypertension. Both patients were evaluated by hypertension specialists, and one of the two was randomized to receive the ROX FLOW procedure, which was performed by Joseph Galvin , MD, at the Mater Private Hospital, Dublin, Ireland. The minimally invasive procedure was performed in the cardiac catheter lab in less than one hour. The ROX procedure is a reversible therapy option for the management of resistant hypertension and may offer an alternative and/or concomitant therapy option to another increasingly popular hypertension procedure, renal denervation. ROX Medical's FLOW procedure is a minimally invasive, catheter procedure whereby a small nitinol coupler is inserted between the artery and vein in the upper leg. The procedure reduces peripheral vascular resistance, thereby holding the promise of a meaningful long-term reduction in hypertension. Uniquely, the FLOW procedure only involves the vascular structure, has an immediate effect, and is fully reversible. The ROX FLOW procedure for hypertension is not approved for use in the U.S. Source: PR Newswire 3/4/13

Positive Results Announced from Phase I Parkinson's Disease Study

NeuroDerm, Ltd. in March announced the results of a Phase I safety and pharmacokinetic trial of ND0612, a novel drug formulation for the treatment of Parkinson's disease. ND0612 is a proprietary levodopa/carbidopa liquid formula administered continuously subcutaneously through a patch pump. It is designed to provide steady levodopa blood levels for the reduction of motor complications in Parkinson's disease. Results of this study support the continued development of ND0612 for the treatment of Parkinson's disease. In this double-blind, placebo controlled, dose-escalation trial in young, healthy volunteers, ND0612 was shown to be safe and tolerable in all of the tested doses. Furthermore, clinically meaningful levodopa concentrations were reached and, for the first time in humans, steady state levodopa concentrations were maintained in a practical manner both day and night. The full results of this study will be presented at a future scientific meeting. ND0612 bypasses the gastrointestinal tract, so the company expects that steady state levodopa levels should be little influenced by intestinal absorption or oral ingestion of food or drugs. Source: PR Newswire 3/4/13

Enrollment Exceeds 4,000 in Preterm Birth Study

Sera Prognostics, Inc., in late February announced that its Proteomic Assessment of Preterm Risk (PAPR) clinical study has now exceeded enrollment of 4,000 subjects, making it the largest broad prospective cohort trial focused on proteomic preterm birth prediction. The company initiated the PAPR study in 2011 to establish a large proprietary biobank to support the development and clinical validation of its maternal-fetal medicine diagnostic testing programs. PAPR is a validation study that builds upon the original National Insitute of Child Health and Human Development Maternal-Fetal Medicine Units Network Preterm Prediction Study, which reported the initial discovery of a novel, high-performing preterm birth predictor in the May 2011 issue of American Journal of Obstetrics & Gynecology. The combined number of patients forming the basis for Sera's discovery/verification and clinical validation of its preterm birth test now totals more than 7,000--making it the broadest combined population sample set dedicated to the question of preterm birth prediction in the U.S. "With the tools available to us today, physicians are unable to predict the large majority of women who will eventually deliver preterm," said Kim Boggess, MD, professor of obstetrics and gynecology at the University of North Carolina at Chapel Hill, a principle investigator in the PAPR study. "While new advances in molecular diagnostics offer possible strategies to improve risk assessment, it is critically important that the validity of new prediction tools be supported by solid data from large, well-designed clinical studies that are representative of the intended patient populations." Sera's preterm birth test is designed to use a routine blood sample obtained during the second trimester of pregnancy to provide an assessment of a woman's risk of preterm birth. Upon launch, commercial testing will be performed at Sera's state-of-the-art CLIA laboratory and results returned to referring physicians. Physicians may then use this information to help guide clinical decisions with respect to personalized interventions intended to improve clinical care and potentially reduce the costs of prolonged stays in neonatal intensive care units. PAPR includes participating sites in Massachusetts, Delaware, North Carolina, South Carolina, Ohio, Tennessee, Utah, Texas, Arizona, California, and Oregon. Blood samples are being prospectively collected from pregnant women between 17 and 28 weeks of gestation and are tracked by delivery outcome and both maternal and neonatal health. Sera's test measures concentrations of multiple protein biomarkers across several functional pathways of relevance to preterm birth. Study completion is expected in the second half of 2013 when results will also be announced. Source: Globe Newswire 2/28/13

Insulin Delivery Device Shows Promise in Type 2 Diabetes

CeQur SA, in late February announced that initial data from a study of the company's PaQ® Insulin Delivery Device were presented at the 6th International Conference on Advanced Technologies & Treatments for Diabetes (ATTD) in Paris, France. The data suggest that PaQ is a safe, highly satisfying alternative to multiple daily insulin injections (MDI) for people with type 2 diabetes--many of whom need new tools to help them achieve better glycemic control. Half of all patients requiring MDIs report that they intentionally skip doses because they consider the injections embarrassing, inconvenient, painful, and/or disruptive to their daily activities. PaQ is a discreet, wearable device that provides three days of consistent, basal insulin delivery along with easy, on-demand bolus insulin. The small device comprises a disposable insulin infuser reservoir attached to a reusable insulin monitor. The 20-patient PaQ study evaluated the ability of people with type 2 diabetes who were on a stable MDI regimen to use PaQ in replacing the daily insulin injections required to control their blood sugar. Study endpoints included glycemic control, patient satisfaction, and safety. The study was led by Professor Thomas Pieber, head of the Division of Endocrinology and Metabolism at the University Hospital in Graz, Austria. Source: PR Newswire 2/28/13

Results Presented for Anti-GM-CSF Antibody in Persistent Asthma

KaloBios Pharmaceuticals, Inc. presented data from its Phase I/II study in persistent asthma for KB002 (precursor chimeric anti-GM-CSF monoclonal antibody to KB003) at the American Academy of Allergy, Asthma, and Immunology Annual Meeting in Austin, Texas, in February. The findings demonstrated preliminary safety, tolerability, and signs of activity of anti-GM-CSF antibodies in asthma and support continued development of the company's Humaneered® KB003 anti-GM-CSF monoclonal antibody in severe asthma. KB003 is currently in a Phase II trial in severe asthma patients inadequately controlled by corticosteroids. When KB002 was added to patients' standard of care treatment, the study showed a reduction in airway inflammation (eosinophils) and improvements in FEV1 (a measure of lung function). Moreover, asthmatics with reversible FEV1 at baseline showed greater FEV1 responses than nonreversible patients. As a result, the company is targeting this patient population in its ongoing Phase II KB003 study. The KB002 Phase I/II asthma study, which screened more than 50 patients to enroll both atopic (eosinophilic) and nonatopic (neutrophilic) asthma subjects, randomized 24 subjects (2:1, active treatment versus placebo). The objectives of the study primarily were to evaluate safety and tolerability, effects on sputum inflammatory markers, and lung function after a single dose of KB002. KB002 and KB003 are recombinant monoclonal antibodies designed to target and neutralize human granulocyte macrophage colony-stimulating factor (GM-CSF), with potential for use in inflammatory and autoimmune indications. KB003 is a Humaneered® version of the chimeric KB002 antibody, with the same epitope target and therefore the same mechanism of action. The company plans to use KB003 for all future clinical studies in this program. Source: PR Newswire 2/26/13

New Bioterrorism Vaccine Gets First Test in Humans

Integrated BioTherapeutics (IBT) in February announced the initiation of a Phase I clinical trial testing the safety and immunogenicity of its staphylococcal enterotoxin B vaccine (STEBVax) in healthy adults. This trial, marking the first time a superantigen vaccine has been administered to humans, is designed to enroll 28 individuals. STEBVax is a proprietary, rationally designed and attenuated form of staphylococcal enterotoxin B (SEB), a member of a group of toxins called superantigens due to the ability to cause a massive inflammatory response leading to toxic shock. "SEB is a biowarfare threat to the U.S., and the superantigens can be critical factors affecting the outcome of Staphylococcus aureus infections," said Dr. M. Javad Aman, president and chief scientific officer for IBT. "This clinical study advances our vaccine programs designed to protect military and civilian populations against the threat of SEB and our long-term goal of developing vaccines and therapeutics for Staphylococcus aureus." The National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health, have sponsored the trial, which is being conducted at the Center for Vaccine Development at the University of Maryland in Baltimore. Source: PR Newswire 2/26/13

First Patient Enrolled in Phase II for Prevention of Post-Surgical Pain

Adynxx in February announced that the first patient was dosed in a Phase II study of its lead investigational drug candidate for the prevention of post-surgical pain, AYX1. The 90-patient, placebo-controlled study will evaluate the safety and efficacy of a single administration of AYX1 given prior to unilateral total knee arthroplasty to reduce acute pain and to prevent the transition to persistent pain. The study will evaluate AYX1's ability to reduce acute pain and prevent persistent pain, especially pain associated with movement, enabling patients to begin rehabilitation and resume other activities earlier. It will follow patients for 42 days, with a primary endpoint of pain with walking. Secondary and exploratory endpoints will include pain at rest, pain with knee range of motion, rate and extent of functional recovery, opioid consumption, and safety assessments. Funded exclusively by Domain Associates, Adynxx completed a Phase I safety study in healthy volunteers in September 2012 and progressed rapidly to the initiation of its Phase II clinical study. Source: PR Newswire 2/25/13

Study Assesses Treatment's Impact on Renal Function in Myeloma Patients

Onyx Pharmaceuticals, Inc. announced in February that Leukemia has published results from the Phase II trial known as PX-171-005 (NCT00721734), an open-label, multicenter clinical trial evaluating Kyprolis® (carfilzomib) for injection in patients with relapsed and refractory multiple myeloma and varying degrees of renal insufficiency. Renal impairment is a frequent and severe complication in patients with multiple myeloma, and presents challenges in the utilization of some antimyeloma therapies. Fifty patients with relapsed, refractory, and/or progressive multiple myeloma who had received at least two prior therapeutic regimens were enrolled in the open-label, single-agent, multicenter trial, and 47 were evaluable for response. The authors reported on the primary and secondary endpoints of the trial. The primary endpoint was the influence of renal impairment on the pharmacokinetics (PK) of carfilzomib. Statistical comparisons showed no differences between renal function status and the dose-adjusted PK parameters, including apparent plasma clearance, dose-normalized AUCinf (total exposure), and the dose-normalized Cmax. Secondary outcomes included safety, tolerability, pharmacodynamic measures, and efficacy. Data from the trial were initially presented at the 2010 American Society of Clinical Oncology Annual Meeting and at subsequent international scientific meetings. The trial was conducted at five sites in the United States. Source: Marketwire 2/20/13

NIH-Funded Researchers Begin Trial of Shigella Vaccine Candidates

Researchers have launched an early-stage human clinical trial of two related candidate vaccines to prevent infection with Shigella, bacteria that are a significant cause of diarrheal illness, particularly among children. The Phase I clinical trial, funded by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), will evaluate the vaccines for safety and their ability to induce immune responses among 90 healthy adults ages 18 to 45 years. The trial is being conducted at the Cincinnati Children's Hospital Medical Center, one of the eight NIAID-funded Vaccine and Treatment Evaluation Units in the United States. Shigella infection, called shigellosis, is an intestinal disease spread via contact with infected feces, by consumption of contaminated food or water, or by contact with a contaminated surface. Symptoms include diarrhea, abdominal pain, fever, nausea, and vomiting. In healthy adults, the infection generally clears on its own in five to seven days, but if left untreated, can lead to hospitalization or death, especially among young children and adults with weakened immune systems. Antibiotics are the standard treatment for patients with shigellosis, but drug-resistant strains of the bacterium are becoming more common. Led by principal investigator Robert W. Frenck, Jr., MD, director of clinical medicine at Cincinnati Children's, the new clinical trial will evaluate two related candidate vaccines, known as WRSs2 and WRSs3, which have been found to be safe and effective when tested in guinea pigs and nonhuman primates. Both target Shigella sonnei, one of the bacteria's four subtypes and the cause of most shigellosis outbreaks in developed and newly industrialized countries. Though neither candidate vaccine has been tested in humans, a precursor to both, known as WRSs1, was found to be safe and generated an immune response in small human trials in the United States and Israel. This early work was supported by NIAID, the U.S. Department of Defense, and the Walter Reed Army Institute of Research. All three versions of the vaccine were developed by researchers at the Walter Reed institute. After undergoing informed consent, study participants will be split into 10 groups of eight participants each, with each group receiving an increasing dose of WRSs2 or WRSs3. The remaining 10 participants will receive placebo. Immediately after vaccination, participants will be admitted to inpatient care. Eight days later, or sooner if serious shigellosis symptoms occur, participants will begin a course of antibiotics until they pass two consecutive stools that test negative for S. sonnei. During the hospital stay, which can last up to 13 days, participants will be closely monitored and receive physical exams several times daily. Once discharged, participants are expected to collect and supply a stool sample at follow-up physical exams on study days 14, 28, and 56. Source: EurekAlert! 2/20/13

Top-Line Data Updated from Phase II in Second-Line Non-Small Cell Lung Cancer

Peregrine Pharmaceuticals, Inc. in February reported data from its randomized, double-blind, placebo-controlled Phase II trial of bavituximab in patients with second-line non-small cell lung cancer (NSCLC). Data from the trial have been updated based on completion of an earlier review of discrepancies in the trial and the most current survival data from the trial. Updated results from this trial indicate a meaningful improvement in median overall survival of 11.7 months in the 3 mg/kg bavituximab + docetaxel arm compared to 7.3 months in the control arm. Persistent separation in the survival curves was observed with response rates and progression-free survival also favoring the 3 mg/kg bavituximab + docetaxel arm in this difficult-to-treat second-line NSCLC. The results also demonstrated that bavituximab was well-tolerated with no significant differences in adverse events between the trial arms. Peregrine plans to report additional data from the trial, including updated subgroup analysis and safety data, at an upcoming scientific meeting. There are seven other ongoing bavituximab trials across many different oncology indications. This trial enrolled 121 patients with previously treated locally advanced or metastatic NSCLC. Patients enrolled in the trial were not selected based on genetic or other biomarkers. All patients had confirmed Stage IIIb or IV nonsquamous NSCLC and had progressed following one prior chemotherapy regimen. The trial was designed to evaluate overall response rate measured in accordance with RECIST criteria, progression-free survival, duration of response, overall survival, and safety. "From a regulatory standpoint, this trial achieved its three main goals in preparing for a Phase III trial by identifying a dose, augmenting the existing set of favorable bavituximab safety data, and demonstrating good signs of survival effect in patients," said Robert L. Garnick, PhD, head of regulatory affairs at Peregrine. "With these data in hand, we are now preparing for additional discussions with regulatory bodies, including an end-of-Phase II meeting with the [Food and Drug Administration (FDA)] by mid-year with an overall goal of being in a position to initiate a pivotal trial near year-end." The review was prompted by the discovery of vial coding discrepancies while preparing for an end-of-Phase II meeting with the FDA, as announced on September 24, 2012. The internal review included a thorough operational review of multiple third-party vendor operations at sites worldwide, testing of investigational product used in the trial, additional patient sample testing to determine drug levels, and a review of immunogenicity testing results from the trial. The initial results of the extensive internal review were announced on January 7, 2013 and indicated that discrepancies were isolated to the placebo and 1 mg/kg treatment arms of the trial and that there was no evidence of discrepancies in the 3 mg/kg treatment arm of the trial. Based on the results of the internal review, Peregrine has taken a conservative approach toward analyzing the results from the trial, which included combining the placebo and 1 mg/kg arms into one treatment arm (control arm), and comparing those results to the 3 mg/kg arm. Source: Marketwire 2/19/13

Company Receives FDA Go-Ahead for Phase III Essential Tremor Trial

InSightec Ltd announced that it has received Food and Drug Administration (FDA) approval to begin its pivotal Phase III clinical trial for treatment of essential tremor, a common movement disorder, using ExAblate® Neuro. This trial is intended to provide the safety and effectiveness data about the use of the device in order to support FDA pre-marketing approval. ExAblate Neuro uses MR guided focused ultrasound (MRgFUS) therapy to provide an incisionless treatment, through the intact skull, with no ionizing radiation. ExAblate MRgFUS uses high-intensity ultrasound waves to destroy target tissue in the brain while the patient lies in an MRI, which provides continuous visualization, plan, guidance, monitoring, and control of the treatment. The study will be a multicenter, double-blinded, randomized control trial, with one-year followup. Patients who enroll in the trial will be randomized to either ExAblate treatment or no treatment. The first patients are expected to be enrolled in mid-2013. Results from early studies showed that patients experienced immediate and durable symptom improvement. The trial is based on the safety and initial effectiveness results from 15 patients treated in an FDA feasibility trial sponsored by the Focused Ultrasound Foundation. In December 2012, ExAblate Neuro was granted the European CE marking for essential tremor, tremor dominant Parkinson's disease, and neuropathic pain. Source: PR Newswire 2/19/13

$27.3 Million Investment Will Fund Trial for Weight-Loss Device

BAROnova, Inc. announced in February the closing of its Series C financing of $27.3 million, led by investments from Sante Ventures and Boston Scientific Corp. Participants included Series B investors ONSET Ventures, Highland Capital Partners, and Arboretum Ventures, along with new investors Sante Ventures, Boston Scientific, and Lumira Capital. Proceeds will be used to fund a pivotal study of the TransPyloric Shuttle® weight-loss technology to gain U.S. and European regulatory approvals. Financial terms were not disclosed. The TransPyloric Shuttle is an innovative mechanical device that is expected to slow the digestion process and create the sensation of fullness, which should slow or stop patients from overeating. The device is placed into the stomach endoscopically through the mouth in an approximately 10-minute outpatient procedure, and may be removed as needed in a similar fashion and time frame. Source: PR Newswire 2/15/13

Nanobody for Rheumatoid Arthritis Shows Excellent Safety and Efficacy in Phase II

Ablynx in February announced efficacy and safety data for its anti-IL-6R nanobody, ALX-0061, at the 24-week final analysis of the Phase II part of a combined Phase I/II study in patients with moderately to severely active rheumatoid arthritis (RA) on a stable background of methotrexate. In this part of the study, 37 RA patients were recruited and were randomized to three dose groups of intravenously administered ALX-0061 (1 mg/kg Q4W, 3 mg/kg Q4W, and 6 mg/kg Q8W1) or to placebo. A total of 34 patients were eligible for determination of efficacy parameters at the 12-week interim period, and all these patients continued the study until week 24. Depending on the patient's disease status at week 10, the monthly dose was increased (from 1 mg/kg to 3 mg/kg; or from 3 mg/kg to 6 mg/kg) or the dosing regimen intensified (from 6 mg/kg Q8W to 6 mg/kg Q4W), and patients on placebo could start monthly ALX-0061 treatment at 3 mg/kg. At all doses tested, ALX-0061 was well-tolerated and the safety profile compared favorably to data reported for other biological disease modifying antirheumatoid arthritis drugs. Source: Marketwire 2/13/13

Institute Recruiting Patients for Trial of Medication to Treat NF2 Tumors

House Research Institute is recruiting patients for a Phase II clinical trial to test a medication that may slow the progression of neurofibromatosis type-2 (NF2), a genetic disease characterized by noncancerous tumors in the central nervous system. A diagnosis of NF2 is made when tumors, called vestibular schwannomas or acoustic neuromas, are found on both auditory nerves. The growth of the tumors can lead to hearing loss, facial paralysis, and balance difficulties because the tumors are located on the auditory, balance, and facial nerves. When the tumors are surgically removed, patients are often left completely deaf. The clinical trial is investigating the medication RAD001 (everolimus), an analog of Rapamycin, to see if the drug is effective in slowing the growth of the vestibular schwannomas in NF2 patients. The preclinical research results are promising. Up to 25 patients will be enrolled in the clinical trial. The study consists of taking RAD001 orally for 12 months or until the tumor shows growth. Tumor growth will be monitored with several MRI scans throughout the 12 months of the study. In addition to the primary objective of the trial, the secondary objective is to see if RAD001 slows the growth of other central nervous system tumors the patients may have, and to see if the patients experience any changes in their hearing. Support for the trial is provided by Novartis Pharmaceuticals Corp., the manufacturer of the Rapamycin analog RAD001 used for this study, Advocure NF2, and by a grant from the Department of Defense. Source: Newswise 2/12/13

Bevacizumab Improves Survival in Recurrent and Metastatic Cervical Cancer

Patients with advanced, recurrent, or persistent cervical cancer that was not curable with standard treatment who received the drug bevacizumab (Avastin) lived 3.7 months longer than patients who did not receive the drug, according to an interim analysis of a large, randomized clinical trial. The trial, known as GOG240, was sponsored by the National Cancer Institute (NCI), part of the National Institutes of Health, and conducted by a network of researchers led by the Gynecological Oncology Group. Genentech, Inc., the drug manufacturer, provided support for the trial under the Cooperative Research and Development Agreement with NCI for the clinical development of bevacizumab. The data safety monitoring committee overseeing the trial recommended that the results of a recent interim analysis be made public because the study had met its primary endpoint of demonstrating improved overall survival in patients who received bevacizumab, which also means that it delayed the chance of dying from the disease. Patients who received bevacizumab got a dose of 15 mg/kg of body weight administered in the vein with their chemotherapy treatment and continued with this dose one day every three weeks until disease progression or unacceptable toxicity occurred. Those patients lived a median 3.7 months longer than those who did not receive bevacizumab. Patients treated with chemotherapy alone had a median survival of 13.3 months while those who received chemotherapy and bevacizumab had a median survival of 17 months. This survival difference was highly statistically significant. However, patients receiving bevacizumab experienced more side effects than those who did not. These side effects were consistent with side effects previously known to be associated with bevacizumab. A total of 452 patients in the United States and Spain with metastatic, recurrent, or persistent cervical cancer not curable with standard treatment were enrolled between 2009 and 2012. Full data have been submitted to the American Society of Clinical Oncology 2013 Annual Meeting. Source: Newswise 2/8/13

FDA Approves Clinical Trial in ALS Patients

The ALS Therapy Development Institute (ALS TDI) announced in February that it has received U.S. Food and Drug Administration (FDA) approval to conduct a clinical trial of TDI-132 (fingolimod) in amyotrophic lateral sclerosis (ALS). Fingolimod is currently being marketed by Novartis AG as Gilenya™ as a treatment for some forms of multiple sclerosis. This clinical trial is being launched as a Phase IIa with the primary purpose of determining the safety and tolerability of TDI-132/Gilenya in people with ALS. ALS TDI, a nonprofit biotech, is the sole funding sponsor of this clinical trial, thanks to the support it received from the ALS community. Current enrollment sites include Massachusetts General Hospital in Boston; the University of California, Irvine; Georgia Health Sciences University in Augusta; and Methodist Neurological Institute in Houston, Texas. ALS TDI researchers first began preclinical testing of TDI-132 in 2011 for its ability to block certain immune cells from entering the brain and spinal cord, where they can cause activities that result in damage to motor neurons. The institute has confirmed that TDI-132 significantly reduces the circulation of these immune cells through the bloodstream, resulting in fewer of them infiltrating into the central nervous system. Further experiments at ALS TDI showed treatment with TDI-132 resulted in positive outcome based on several disease measures in preclinical studies. In February 2012, the institute officially announced TDI-132 as a clinical candidate. ALS TDI worked with the Northeast ALS Consortium (NEALS) on the clinical trial design. NEALS will oversee the execution of the clinical trial. Source: PR Newswire 2/11/13

Weight Loss Trial Does Not Meet Predefined Efficacy Endpoints

EnteroMedics Inc. in February announced results from its randomized ReCharge pivotal trial of VBLOC® vagal blocking therapy for the treatment of obesity. The trial demonstrated a clinically meaningful and statistically significant excess weight loss (EWL) of 24.4 percent for VBLOC therapy-treated patients, with 52.5 percent of patients achieving at least 20 percent EWL. The trial met its primary safety endpoint, though it did not meet its predefined primary efficacy measures. EnteroMedics plans to move forward with a Pre-Market Approval application with the U.S. Food and Drug Administration in the second quarter of 2013. The trial was a prospective double-blind, sham-controlled study involving 239 randomized patients (233 implanted) at 10 sites in the United States and Australia. Patients were surgically implanted with either a fully functional device with leads to the vagus nerve (treated) or a device without leads to the vagus nerve (sham control). Source: Marketwire 2/7/13

Phase III Rheumatoid Arthritis Program Discontinued

Eli Lilly and Co. announced in February that it will discontinue the Phase III rheumatoid arthritis (RA) program for tabalumab, an anti-BAFF (B cell activating factor) monoclonal antibody, due to lack of efficacy. The decision was not based on safety concerns. The tabalumab Phase III program for systemic lupus erythematosus, ILLUMINATE, is ongoing and will continue as planned. In December 2012, Lilly discontinued the Phase III RA registration study FLEX-M for lack of treatment effect. FLEX-M was investigating tabalumab in patients with moderate-to-severe RA who had an inadequate response to methotrexate therapy. Based on FLEX-M findings, an interim futility analysis was conducted of the FLEX-V study, which was investigating tabalumab for the treatment of patients with moderate-to-severe RA who had an inadequate response to one or more tumor necrosis factor (TNF) inhibitors. Based on the outcomes of these two separate interim futility analyses, Lilly has decided to discontinue development of tabalumab in the current RA program. All ongoing Phase II and Phase III RA studies will be stopped. Source: PR Newswire 2/7/13

First Patient Treated in Phase I Trial for Thoracic Malignancies

Genelux Corp. in February announced that researchers at Memorial Sloan Kettering Cancer Center in New York treated the first patient in a Phase I clinical trial of GL-ONC1 in people with malignant pleural effusion, a complication that occurs in about 30 percent of lung cancers. The safety and dose-escalation study will, for the first time, evaluate GL-ONC1 administered through the lung cavity (intrapleurally) as a single agent therapy. Patients enrolled in the trial may have one of a number of possible cancer types, such as malignant pleural mesothelioma or non-small cell lung cancer. GL-ONC1 delivers therapeutic and diagnostic capabilities (via green fluorescent proteins) simultaneously and directly to tumors without harming healthy tissues or cells. It has been well-tolerated and shown encouraging results in early human trials against a number of solid tumor cancers. The primary goal of the trial is to establish a recommended dose of GL-ONC1, when administered intrapleurally to patients with malignant pleural effusion, which is primarily attributable to, malignant pleural mesothelioma, and other cancers. Secondary objectives include the feasibility, safety, and tolerability of intrapleural vaccinia virus; the detection of virus in body fluids; evaluation of viral appearance in tumors; evaluation of antivaccinia virus immune response (e.g., antibody responses) and evidence of antitumor activity. The total number of patients studied will depend on the number of dose levels tested, with a possibility of up to 54 to be enrolled. Source: Marketwire 2/6/13

Phase IIb Impetigo Trial Enrollment Expanded to South Africa

NovaBay® Pharmaceuticals, Inc. in February announced that partner Galderma S.A. has initiated the South African arm of its Phase IIb clinical study of a proprietary topical formulation of NVC-422 (CD07223) for the treatment of impetigo, a highly contagious skin infection. Designed to confirm efficacy and evaluate two different dosage regimens, the study is expected to enroll more than 300 patients at 24 clinical sites in four countries worldwide. The first patients were enrolled in the U.S. arm of the study in September 2012. Galderma previously established preclinical and clinical safety for topical NVC-422, including the successful completion of safety studies involving more than 300 healthy volunteers, as well as the feasibility of manufacturing scale-up and long-term shelf-life. In a previous Phase II clinical study, NVC-422 was shown to be safe and well tolerated. NVC-422 gel also demonstrated a clinical response rate of 92 percent and a microbiological response rate of 95 percent at the same dosage used in the current Phase IIb study. Furthermore, in a subset of patients who enrolled in the study with MRSA infections, NVC-422 gel demonstrated clinical and microbiological response rates of 100 percent. Additionally, NVC-422 was rapidly bactericidal against 55 clinical isolates of MRSA acquired from a database of patients in the U.S. and Europe. Source: Globe Newswire 2/5/13

Three NIH-Sponsored Clinical Trials Test Influenza Treatments

Three clinical trials that seek to find more effective treatments for influenza are enrolling volunteers with influenza at the National Institutes of Health’s (NIH's) Clinical Center in Bethesda, Md., and at several dozen other domestic and international sites. One study examines whether treatment with a licensed influenza drug, oseltamivir, reduces the time that infected people continue to produce virus in the upper airway. A second tests whether a combination of three licensed flu antiviral drugs works better than oseltamivir alone in people with influenza who have chronic health conditions, such as heart or lung disease, that put them at greater risk of severe illness. The third tests whether treatment with plasma enriched with anti-influenza antibodies improves the condition of hospitalized influenza patients compared to standard antiviral treatment alone. The oseltamivir trial will enroll a total of approximately 560 people at 31 locations in the United States, Argentina, and Thailand. The trial comparing oral oseltamivir alone to treatment with oseltamivir plus two other licensed antiviral drugs is enrolling a total of up to 720 adults at sites in the United States, Argentina, Australia, Mexico, and Thailand. The third trial is enrolling children as well as adults, including pregnant women, hospitalized with severe influenza. This trial aims to enroll a total of approximately 100 people at approximately 20 sites in the United States. Source: NIH News 2/5/13

Company Receives FDA Approval to Initiate Cellular Therapy Trial for Thermal Burns

Arteriocyte announced in late January approval from the Food and Drug Administration (FDA) to initiate a Phase I clinical trial using its Magellan® System technology in the treatment of thermal burn wounds. The FDA Investigational Device Exemption (IDE-15140) allows Arteriocyte and its clinical partners to initiate evaluation of autologous platelet gel as an adjunctive therapy for autologous skin grafting in patients with thermal injuries. This treatment has been developed in partnership with the United States Telemedicine and Advanced Technology Research Center and the University of Utah Health Science Center and Intermountain Burn Center. Arteriocyte is initiating a series of investigations employing its Magellan therapies for burn wounds as part of its Cellular Therapies for Battlefield Wounds Program. The Magellan System is an FDA 510(k) cleared medical device for the rapid production of platelet rich plasma from blood and bone marrow that can be applied to a surgical site as surgeons deem necessary for their clinical use requirements. Autologous platelet gel is used to improve adherence of the autologous skin graft, while providing beneficial growth factors and antimicrobial protection ensuring graft survival. Enrollment into the trial is anticipated to begin in early 2013 at the University of Utah. Source: PR Newswire 1/31/13

Phase II Study Initiated for Treatment of Malaria

Dilaforette, a Karolinska Development portfolio company, in late January announced that the company has approval from the regulatory authority in India to start a Phase II study in that country with sevuparin in patients with moderate to severe malaria. Dilaforette and its collaborator, the Mahidol Oxford Tropical Medicine Research Unit, plan to enroll 50 patients in India, where severe malaria remains an important problem. The primary objective of the study is to evaluate safety along with several efficacy parameters. The first study site is open for enrollment and the first patients are expected in connection with the coming rainy season. A separate clinical trial in uncomplicated falciparum malaria is already ongoing in Thailand since 2011. Sevuparin interferes with a pivotal culprit in malaria pathophysiology: blockage of the smallest blood vessels in vital organs. It can potentially help to reduce mortality in cases of severe malaria where antimalarial treatment alone is not sufficient, by preventing and reversing the infected cells' ability to block blood vessels. Source: Cisionwire 1/30/13

Enrollment Begins for Phase III Pain Trial

Charleston Laboratories, Inc. announced in January that patient enrollment has begun for the company's pivotal Phase III clinical trial for its first product, CL-108, for the treatment of moderate to severe pain. "The initiation of this study marks the culmination of work by many people," said Dr. Bernard P. Schachtel, chief scientific officer of Charleston. "As exemplified by the enthusiasm and dedication of the many doctors, nurses, study coordinators, and other researchers at our recent investigators meeting, this study offers the rare and exciting opportunity to research a truly novel indication--the treatment of both moderate-to-severe pain and the nausea and vomiting commonly associated with its treatment." CL-108 combines an antiemetic with an opioid to treat moderate-to-severe pain while significantly reducing and, in many cases, eliminating opioid-induced nausea and vomiting (OINV). Nausea and vomiting are the most common and burdensome side effects of opioid medications, and have a significant effect on patients' lives and the physicians treating these patients. Demonstrating the values of an opioid medication with less OINV in this study will not only advance the entry of CL-108 into the marketplace, but further the development of other Charleston products now in the lab. Source: PR Newswire 1/28/13

First Patient Treated in Chronic Kidney Disease Trial

La Jolla Pharmaceutical Co. announced in January that it has administered the first dose for its Phase I/II clinical trial of GCS-100 in patients with chronic kidney disease (CKD). The study is an open-label, multicenter test of GCS-100 in patients with Stage 3b and 4 CKD. The primary objectives of the study include evaluating the safety of a single dose (Part A) and repeat doses (Part B) of GCS-100. Secondary study objectives include evaluating galectin-3 serum levels, renal function, and other markers of disease activity in CKD. The study is open to patients at least 18 years of age with moderately severe to severe renal impairment. While not currently anticipated, the study design may be amended at times to comply with requests from the Food and Drug Administration, the governing institutional review board, study investigators, or at the discretion of the company. "We are very pleased with the quick progress made to start the Phase I portion of the trial. We hope to see safety data the first half of this year and a streamlined path to the extended dosing part of the study," said George Tidmarsh, MD, PhD, president and CEO of La Jolla. Source: Marketwire 1/28/13

Phase III Study Initiated in Pancreatic Adenocarcinoma Patients

Threshold Pharmaceuticals, Inc. in January announced that its partner Merck KGaA, Darmstadt, Germany, through its division Merck Serono, initiated the global Phase III MAESTRO study assessing the efficacy and safety of investigational hypoxia-targeted drug TH-302 in combination with gemcitabine in patients with previously untreated, locally advanced unresectable or metastatic pancreatic adenocarcinoma. The initiation of the study resulted in Threshold earning a $30 million milestone payment from Merck KGaA pursuant to the terms of Threshold's license and codevelopment agreement with Merck KGaA. MAESTRO is a randomized, placebo-controlled, international, multicenter, double-blind trial of TH-302 plus gemcitabine compared to placebo plus gemcitabine, and is expected to enroll 660 patients. The primary efficacy endpoint is overall survival; the secondary endpoints include efficacy measured by progression-free survival, overall response rate, and disease control rate, as well as assessments of safety and tolerability, pharmacokinetics, and biomarkers. The study is being conducted under a Special Protocol Assessment with the U.S. Food and Drug Administration. TH-302 is an investigational hypoxia-targeted drug that is designed to be activated under severe tumor hypoxic conditions, a hallmark of many cancers. Areas of low oxygen levels (hypoxia) in solid tumors are due to insufficient blood supply as a result of aberrant vasculature. This marks the second Phase III study of TH-302 in addition to the ongoing Phase III pivotal trial in patients with soft tissue sarcoma. In addition, the company continues to explore the potential breadth and activity of TH-302 against a variety of solid tumors and hematological malignancies in multiple ongoing earlier stage clinical trials. Source: Marketwire 1/25/13

Final Phase I Safety Study Completed for Chronic Neuropathic Pain

VistaGen Therapeutics, Inc. in January announced the successful completion of its final Phase I safety study of AV-101, a novel orally available prodrug candidate being developed for treatment of multiple conditions involving chronic neuropathic pain. The study results indicate that AV-101 is safe and well tolerated, with favorable bioavailability and pharmacokinetics. The study was a randomized, double-blind, placebo-controlled, dose-escalation trial conducted at the University of California, San Diego, and involving three cohorts of healthy volunteers, each receiving multiple daily treatments of one of three dose levels of orally administered AV-101 over a 14-day period. The primary objectives of the study were to evaluate the safety, tolerability, and pharmacokinetics (PK) of three different daily doses of AV-101 compared to placebo controls. A total of 46 healthy volunteers completed the study. The oral administration of AV-101 was safe and well tolerated by all subjects at all three dose levels tested. In addition, the PK of AV-101 was fully characterized across the range of three dose levels in the study. AV-101 (L-4-chlorokynurenine), is a novel, orally available prodrug that is converted in the brain into an active metabolite, 7-chlorokynurenic acid (7-Cl-KYNA), which regulates an important neurotransmitter in the brain called the N-methyl-D-aspartate (or NMDA) receptor. A synthetic analogue of kynurenic acid, a naturally occurring neural regulatory compound, 7-Cl-KYNA is one of the most potent and selective blockers of the regulatory GlyB-site of the NMDA receptor. VistaGen's AV-101 Investigational New Drug application covers clinical development for neuropathic pain. In addition to neuropathic pain, VistaGen expects the results of its Phase I clinical program to be useful for supporting the development of AV-101 for other neurological disorders, including depression and epilepsy. Source: Marketwire 1/23/13

NIH Trial Begins for Treatment of Rare, Fatal Neurological Disorder

A clinical trial to evaluate a drug candidate called cyclodextrin as a possible treatment for Niemann-Pick disease type C1 (NPC), a rare and fatal genetic disease, started in January. Scientists from the National Institutes of Health's (NIH’s) National Center for Advancing Translational Sciences (NCATS) and the Eunice Kennedy Shriver National Institute of Child Health and Human Development will conduct the clinical trial at the NIH Clinical Center. Reaching this trial stage required collaboration among government, industry, patient advocacy groups, and academic researchers. No therapies approved by the U.S Food and Drug Administration (FDA) are available to treat NPC. The disease is characterized by the inability of cells to metabolize and dispose of cholesterol and lipids. It causes excessive amounts of cholesterol to accumulate within the liver, spleen, and brain. NPC leads to progressive impairment of motor and intellectual function in early childhood. In childhood onset cases, life expectancy does not normally exceed a patient’s teenage years. “A crucial part of the NCATS mission is to collaborate within and beyond the NIH on projects to improve and accelerate the translational research process and deliver tangible improvements in human health,” said NCATS Director Christopher P. Austin, MD. “The cyclodextrin project is an important step in the development of both a potential treatment for a devastating disease that ravages the bodies and minds of its victims and a more efficient way to do translational projects.” In 2009, the NIH Therapeutics for Rare and Neglected Diseases (TRND) program, which is now led by NCATS, selected NPC cyclodextrin as one of its initial pilot projects to repurpose cyclodextrin from its conventional use as an ingredient in other drugs to a therapeutic for this rare disorder. TRND researchers work with project collaborators to conduct preclinical studies advancing potential treatments for rare and neglected diseases to human clinical trials. TRND researchers and collaborators submitted the data in an Investigational New Drug application, filed November 14, 2012, that the FDA has now agreed is sufficient to start a Phase I clinical trial involving the testing of multiple doses of cyclodextrin in nine patients to determine a safe dose of cyclodextrin that will support an expanded Phase II trial to begin to evaluate the effectiveness of the drug. The team already is in the initial stages of collaborating with the Network for Excellence in Neuroscience Clinical Trials (NeuroNEXT), which is administered by the NIH’s National Institute of Neurological Disorders and Stroke, to plan a Phase II multicenter trial. The NPC clinical trial is the fourth TRND project to advance to human clinical trials in the last 15 months. The three other clinical trials are evaluating treatments for sickle cell disease, chronic lymphocytic leukemia, and hereditary inclusion body myopathy. TRND has a portfolio of 14 projects, which focus on rare and neglected tropical diseases. Source: National Institutes of Health 1/23/13

First Patients Enrolled in Pivotal Phase III Hepatitis C Trial Program

In January, Boehringer Ingelheim Pharmaceuticals, Inc. announced that the first patients have been enrolled in the company's pivotal Phase III interferon (IFN)-free hepatitis C (HCV) clinical trial program, HCVerso™. Boehringer Ingelheim's investigational IFN-free regimen combines the compounds faldaprevir (BI 201335), a protease inhibitor administered once-daily, and BI 207127, a non-nucleoside polymerase inhibitor administered twice-daily, plus ribavirin. Phase III clinical trial sites are established in more than 25 states in the U.S. The HCVerso clinical trial program includes two pivotal Phase III IFN-free studies that will enroll approximately 1,000 treatment-naive HCV genotype-1b (GT-1b) patients, including those who are interferon eligible or ineligible. The decision to enroll GT-1b patients in the Phase III trials follows results from the Phase IIb SOUND-C studies, where Boehringer Ingelheim's investigational, IFN-free regimen showed higher viral cure rates in patients with the 1b HCV genotype, the most prevalent type of HCV globally. New preliminary results from the SOUND-C3 trial show that 100 percent (n=20) of patients with HCV GT-1b achieved sustained virologic response four weeks after completing a 16-week course of treatment. These data further support the trial design for Boehringer Ingelheim's pivotal HCVerso study. Full results from SOUND-C3 are expected in 2013. Results from the SOUND-C2 study, which were presented in November 2012 at the 63rd Annual Meeting of the American Association for the Study of Liver Diseases, found that up to 85 percent of patients infected with HCV GT-1b treated with Boehringer Ingelheim's investigational IFN-free regimen of faldaprevir, BI 207127, and ribavirin achieved viral cure at 12 and 24 weeks following treatment completion. Source: PR Newswire 1/17/13

First Patient Treated in Trial Evaluating Catheter Ablation

The first patient has been treated in the Boston Scientific Corp. ZERO AF clinical trial to evaluate the safety and effectiveness of the Blazer® Open-Irrigated Temperature Ablation Catheter in patients with symptomatic, drug refractory paroxysmal atrial fibrillation. This international, multicenter study will include up to 33 sites in the United States, Europe, and Asia-Pacific, and as many as 472 patients. The results of the ZERO AF trial are expected to be used to support a U.S. Food and Drug Administration regulatory submission for a paroxysmal atrial fibrillation indication. Paroxysmal atrial fibrillation is a type of atrial fibrillation in which the irregular heartbeat starts up very quickly, stops spontaneously, and abruptly returns to the normal rhythm, resulting in patients feeling symptomatic. Catheter ablation, a procedure in which localized electrical energy is delivered into the heart tissue aimed at restoring the continuous normal rhythm, is a common treatment for many heart rhythm disorders, including paroxysmal atrial fibrillation. The Blazer Open-Irrigated Catheter is the latest addition to the extended families of Boston Scientific Blazer catheters. It is the company's first entry into the open-irrigated catheter segment, and is approved for use in CE Mark countries and Canada. The Blazer Open-Irrigated Catheter offers the Total Tip Cooling™ design, engineered to consistently cool the entire tip of the electrode during radiofrequency energy delivery. The first U.S. procedure was performed at St. David's Medical Center in Austin, Texas, by J. David Burkhardt, MD, also part of Texas Cardiac Arrhythmia. "As expected, the [catheter] handled well, consistent with my previous experience using other Boston Scientific Blazer catheters," said Dr. Burkhardt. In the United States, the Blazer Open-Irrigated Catheter is an investigational device and is not available for sale. Source: PR Newswire 1/16/13

Company Provides Update of Phase IIb Allergic Asthma Trial

Cytos Biotechnology Ltd. in January announced an update on the Phase IIb clinical trial with its lead product candidate, CYT003, in allergic asthma. The clinical trial will recruit approximately 360 adult patients with moderate-to-severe allergic asthma not sufficiently controlled on current standard therapy. The study will be conducted at more than 90 centers in North America and Europe, and is expected to read out top-line data in the first half of 2014 and have full data toward the end of that year. Regulatory approvals to conduct the trial have been obtained in various countries in Europe and North America, including the United States, where an Investigational New Drug application has been approved by the U.S. Food and Drug Administration. In the course of periodic drug quality measurements, a technical issue was identified with the rubber stoppers used to close the injection vials containing the clinical trial material. Cytos will replace this material and have new material available at clinical trial sites in the second quarter of 2013. Overall guidance for the clinical trial remains unchanged. Source: PR Newswire 1/16/13

Company Announces Completion of Stroke-Aphasia Trial

Soterix Medical, Inc. announced in January the completion of its Phase I stroke-aphasia trial using its revolutionary and proprietary noninvasive brain targeting platform (High Definition-Transcranial Direct Current Stimulation [HD-tDCS]). "We are proud to complete our Phase I trial. This is a major milestone for Soterix Medical and enhances our efforts to transform the way stroke rehabilitation is managed. I wish to thank the National Institutes of Health for [its funding and] the partnering clinical site of University of South Carolina, and recognize each and every patient in our clinical trial," said Dr. Abhishek Datta, chief technology officer and cofounder of Soterix Medical, and coinventor of HD-tDCS. "We are thrilled to see the fruition of our vision of using small electrode arrays to deliver targeted stimulation to desired brain targets. Our patented approach allows individualized tDCS therapy and initial studies indicate more tolerable stimulation than conventional tDCS using sponges." Soterix Medical has secured an investigational device exemption from the U.S. Food and Drug Administration (FDA) in anticipation of a mutisite Phase II trial. In a recent landmark study from the group that introduced tDCS in its current form, Dr. Michael Nitsche and Dr. Walter Paulus found that HD-tDCS produced larger and longer-lasting brain excitability changes than conventional tDCS. At City University of New York (CUNY), Dr. Abhishek Datta spearheaded the modeling approach for HD-tDCS as part of his doctoral study at the CUNY laboratory of Dr. Marom Bikson and Dr. Lucas C. Parra. HD-tDCS was developed to combine the benefits of tDCS (namely, low-intensity currents, safety profile, and neuromodulation plasticity) with targeting control matching transcranial magnetic stimulation, an FDA-approved therapy. HD-tDCS dose (electrode placement and individual electrode currents) is determined using individualized brain current flow modeling in combination with neurotargeting software (HDTargets and HDExplore). Source: PR Newswire 1/15/13

FDA Approves Spinal Cord Injury Cell Therapy Trial

Neuralstem, Inc. announced in January that it received approval from the U.S. Food and Drug Administration (FDA) to commence a Phase I safety trial of its lead cell therapy candidate, NSI-566, in chronic spinal cord injury patients. This open-label, multisite study will enroll up to eight patients with thoracic spinal cord injuries (T2-T12), who have an American Spinal Injury Association (AIS) A level of impairment, between one and two years after injury. AIS A impairment refers to a patient with no motor or sensory function in the relevant segments at and below the injury, and is considered to be complete paralysis. The primary objective of the study is to determine the safety and toxicity of human spinal stem cell transplantation for the treatment of paralysis and related symptoms due to chronic spinal cord injury (SCI). The secondary objectives of the study are to evaluate graft survival in the transplant site by MRI, as well as the effectiveness of transient immunosuppression. Additionally, the study will look at a variety of exploratory objectives to evaluate the ability of human spinal cord stem cell transplantation to positively affect AIS level, motor and sensory index scores, bowel and bladder function, pain, and other factors. All patients in the study will receive six injections in, or around, the injury site. The first four patients will receive 100,000 cells per injection, the second four patients will receive 200,000 cells per injection. All patients will also receive physical therapy postsurgery, as well as immunosuppressive therapy, which will be for three months, as tolerated. The trial study period will end six months postsurgery for each patient. Source: PR Newswire 1/14/13

Phase I/II Trial Enrollment Complete for HIV/AIDS Therapeutic Vaccine

GeoVax Labs, Inc. announced in January it has completed enrollment in a nine-patient Phase I/II clinical trial testing the safety, immunogenicity, and ability of its DNA/MVA vaccine to elicit protective immune responses in HIV-infected individuals. The primary goal of this study is to document the safety and immunogenicity of GeoVax's vaccine in HIV-positive patients with well-controlled infections using oral HIV drug medication. Following vaccination, the trial includes a short period of drug interruption to evaluate the ability of the vaccine to control the infection in the absence of continuing drug therapy. The trial (designated GV-TH-01) consists of priming with a recombinant DNA vaccine followed by boosting with a recombinant modified vaccinia Ankara vaccine. The vaccine regimen elicits both antiviral antibody that can block infection and antiviral T cells that can recognize and kill infected cells. The trial is being conducted at the AIDS Research Consortium of Atlanta, the Alabama Vaccine Research Center at the University of Alabama, Birmingham, and the AIDS Research Alliance of Los Angeles. Robert McNally, PhD, president and CEO of GeoVax, stated, "This pilot study is our first trial investigating use of a therapeutic vaccine to address the need for a treatment that is better tolerated and less costly than the HIV oral medications currently available. We anticipate having meaningful data from the program later this year." The next step planned for the vaccine's development program is a Phase I clinical trial to investigate its use in combination with standard-of-care drug therapy in young adults. This trial will likely be conducted by the International Maternal Pediatric Adolescent AIDS Clinical Trial Group. One of the hopes for therapeutic vaccination is that combining a vaccine with drugs will allow the eradication of virus from an infected individual. Source: Marketwire 1/10/13

Enrollment Begins for Phase III Myelofibrosis Trial

Cell Therapeutics, Inc. in January announced that the company has initiated clinical trial sites and began enrolling patients in a Phase III clinical trial, known as PERSIST-1 or PAC325, for pacritinib, the company's investigational JAK2 inhibitor, which is being evaluated for the treatment of patients with myelofibrosis. Pacritinib is a selective oral JAK2 inhibitor that demonstrated meaningful clinical benefits and good tolerability in myelofibrosis patients in Phase II clinical trials, without apparent drug-related thrombocytopenia or anemia. The goal is to enroll 270 myelofibrosis patients without exclusion for low platelet counts. PERSIST-1 is a multicenter, randomized, controlled trial comparing the efficacy and safety of pacritinib with that of best available therapy in patients with primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis. Eligible patients will be randomized 2:1 to receive either pacritinib 400 mg taken orally once daily or the best available therapy, excluding JAK inhibitors. The primary endpoint will be the percentage of patients achieving at least a 35 percent reduction in spleen volume measured by MRI or CT at 24 weeks of treatment. The trial is expected to enroll patients at clinical sites in Europe, Australia, and the United States. A second Phase III trial is planned to evaluate pacritinib compared to best available therapy, including JAK inhibitors, in patients with myelofibrosis whose platelet counts are < 100,000/µL. Pacritinib is an oral, once-a-day, tyrosine kinase inhibitor with dual activity against JAK2 and FLT3, and has orphan drug designation in the U.S. and Europe. Source: PR Newswire 1/9/13

Company Initiates Trial for Drug-Eluting Bioresorbable Vascular Scaffold

Abbott announced in January the initiation of the ABSORB III clinical trial in patients in the United States. This randomized, controlled trial is designed to enroll approximately 2,250 patients, the majority in the United States, and compare the performance of Abbott's drug-eluting Absorb™ bioresorbable vascular scaffold device to the company's Xience™ family of drug-eluting stents. The start of this trial in the United States follows the recent international commercial launch of Absorb in Europe and parts of Latin America and Asia, including the recent regulatory approval and launch in India. Data from the ABSORB III trial will support U.S. regulatory filings for Absorb. Absorb is intended to treat coronary artery disease by opening a clogged vessel and restoring blood flow to the heart, similar to a drug-eluting metallic stent, the current standard of care. Absorb then dissolves into the blood vessel, leaving behind a treated vessel that may resume more natural function and movement because it is free of a permanent metallic implant. Unlike a permanent metallic stent, preliminary evidence of natural vessel function suggests that treatment with Absorb could provide important clinical benefits. The primary endpoint of ABSORB III is target lesion failure, a combined measure of safety and efficacy, at one year. In addition, a subset of patients within the trial will be evaluated for novel endpoints such as vasomotion, a measure of how much natural motion returns to the vessel as Absorb dissolves into the arterial tissue. The scaffold delivers everolimus, an antiproliferative drug used in Abbott's Xience coronary stent systems. Absorb is an investigational device, limited by United States law to investigational use and is not approved or available for sale in the United States. Absorb is authorized for sale in CE Mark countries. Absorb is available in Europe, the Middle East, parts of Latin America, and parts of Asia Pacific, including India, Hong Kong, Malaysia, and New Zealand. Source: PR Newswire 1/8/13

Trial of 500 Breast Cancer Patients Completed

MetaStat, Inc. announced in January that a 500-patient trial in breast cancer has been completed as the company continues to develop its metastasis diagnostics technology. The trial was completed on time and on budget in line with previously disclosed expectations, and the company expects the full analysis and publication of the data later in the year. Currently available genome-wide studies attempt to provide prognostic information on the probability of cancer recurrence. However, these studies are less than optimal in predicting metastatic outcome because they genetically profile whole tumor tissue without respect to the fact that only a small subpopulation of metastatic cells inside the primary tumor is responsible for escaping and initiating dissemination and metastasis. When whole tumor tissue is used for genetic profiling, the expression pattern of these metastatic cells is masked by the presence of a vast majority of noninvasive tumor cells and undefined stromal cells. Therefore, little information is available about the crucial early steps of metastasis. MetaStat intends to develop next-generation metastasis diagnostic products based on this and its other proprietary technologies. Source: PR Newswire 1/7/13

Phase II Trial Gets Under Way for Obesity

Rhythm announced in January the initiation of a Phase II clinical trial with RM-493, the company's novel melanocortin 4 receptor (MC4R) agonist, for the treatment of obesity. Based on the data from preclinical studies and early clinical trials, the company believes RM-493 has substantial therapeutic potential for reducing body weight and insulin resistance in obese and diabetic patients. The trial is designed to evaluate the effect of RM-493 on weight loss and safety in obese subjects treated for three months. The trial will also assess the drug's effects on glucose and insulin resistance. In 2012, Rhythm completed Phase I clinical trials with RM-493, with obese subjects being treated for up to 28 days. These studies evaluated RM-493's safety, pharmacokinetics, and effect on body weight. Extensive preclinical studies in obese primates preceded these human clinical trials; primates treated for eight weeks lost an average of 13.5 percent of their body weight, with significant improvements in both insulin sensitivity and cardiovascular function. RM-493 is a small-peptide MC4R agonist that mediates a key pathway in humans that regulates energy homeostasis and food intake. The MC4 pathway is well validated in humans; mutations of MC4R are associated with obesity and are estimated to be responsible for 4 to 6 percent of all severe obesity. Source: PR Newswire 1/4/13

Enrollment Completed for Phase II Patch Study in Postmenopausal Osteoporosis

Radius Health, Inc. Announced in early January that it had completed enrollment for its randomized Phase II study of BA058-transdermal (BA058-TD) in healthy postmenopausal women with osteoporosis. The last of 250 patients completed her randomization visit for the study of this short wear-time transdermal patch based on 3M's patented Microstructured Transdermal System technology to administer BA058 without the need for subcutaneous injection. Radius is developing BA058, a novel synthetic peptide analog of human parathyroid hormone-related protein (hPTHrP) that is a bone anabolic compound with the potential to treat severe osteoporosis. Currently, BA058 is also being studied as a daily subcutaneous injection (BA058-SC) in a Phase III study with 2,400 patients for fracture prevention in women with severe postmenopausal osteoporosis, in addition to the Phase II study of the transdermal patch, which is designed to improve patient compliance. Radius and 3M Drug Delivery Systems announced in December 2012 an exclusive agreement for the development and commercialization of the BA058-TD short wear-time patch. The BA058-TD trial is a randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of BA058-TD in otherwise healthy postmenopausal women with osteoporosis, as assessed by changes in bone mineral density. In addition, pharmacokinetic parameters and serum markers of bone metabolism for BA058-TD will be compared to both transdermal placebo and BA058-SC. The company says the patch has the potential to provide patients with an effective bone-building alternative to a daily subcutaneous injection, and hopes patient compliance will increase with this novel mode of drug delivery. Radius previously announced positive results from a series of BA058-TD Phase I clinical trials, which successfully demonstrated the ability of the transdermal patch to safely and rapidly deliver BA058 after a short wear-time, with no increased delivery resulting from longer wear, as well as supportive biochemical evidence of bone-building activity following seven days of dosing. Source: Marketwire 1/3/13

Company Granted Special Protocol Assessment for Phase III Trial in Endometrial Cancer

Aeterna Zentaris Inc. in December announced that it has reached an agreement with the U.S. Food and Drug Administration (FDA) on a Special Protocol Assessment (SPA) for an upcoming Phase III registration trial in endometrial cancer with its doxorubicin peptide conjugate, AEZS-108. The SPA agreement states that the proposed trial protocol design, clinical endpoints, and planned analyses are acceptable to the FDA to support a regulatory submission. "We are pleased with the agreement with the FDA, which provides us with a clearly defined development and regulatory pathway for AEZS-108 in endometrial cancer," stated Juergen Engel , PhD, president and CEO at Aeterna Zentaris. This will be an open-label, randomized, multicenter trial conducted in North America and Europe, comparing AEZS-108 to doxorubicin as second-line therapy for locally advanced, recurrent, or metastatic endometrial cancer. The trial will involve approximately 500 patients and the primary efficacy endpoint is improvement in median overall survival. Source: PR Newswire 12/28/12

Phase III Results Mixed for Anti-Inflammatory Drug for Meniscectomy Surgery

Omeros Corp. in December reported results from its first pivotal Phase III clinical trial evaluating OMS103HP in patients undergoing arthroscopic partial meniscectomy surgery. In this multicenter, double-blind trial comparing OMS103HP to vehicle control in 344 subjects, the prespecified primary endpoint was the Symptoms Subscale of the Knee Injury and Osteoarthritis Outcome Score (KOOS)--a patient-reported measure that is comprised of questions about knee swelling, clicking, catching, and stiffness. In addition, pain measured in the early postoperative period was a prespecified secondary endpoint. Although the Symptoms Subscale of the KOOS did not reach statistical significance, OMS103HP achieved statistically significant reduction of postoperative pain. The pain reduction data were similar in magnitude to those in the Phase II clinical trial. OMS103HP also demonstrated improvement across a series of assessments, including postoperative narcotic usage (with more than twice as many OMS103HP-treated subjects taking no postoperative narcotics), incidence of inflammatory adverse events, tourniquet use, crutch use, time to discontinuation of crutches, and time to return to work, a number of which also achieved statistical significance. In this study, as in the earlier clinical trials, OMS103HP was well tolerated. Given the strength and consistency of the data in this trial, Omeros' second OMS103HP Phase III trial remains on track, and will begin in the first half of 2013. Delivered directly to the joint in arthroscopic irrigation solution, use of OMS103HP avoids the frequently reported damage to cartilage cells due to intraarticular delivery of local anesthetics as well as the detrimental effects of systemically delivered analgesics. Source: PR Newswire 12/27/12

Clinical Trial Agreement Set for Further Development of Influenza Drug

Sarepta Therapeutics, Inc. announced in December that it has entered into a Clinical Trial Agreement (CTA) with the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), to conduct a Phase I study with AVI-7100, the company's lead drug candidate with a novel mechanism of action and potentially broad-spectrum activity against influenza viruses, including Tamiflu-resistant virus strains. "We are very excited and honored to work with the NIH, which has a long-standing commitment to fighting pandemic and seasonal influenza," said Chris Garabedian, president and CEO. "Our agreement signifies the importance of developing new treatment options for influenza with novel mechanisms of action given the limitations of currently available influenza antivirals. Our combined effort with the NIH will further increase our understanding of AVI-7100 and its underlying platform chemistry, which we are also applying to other infectious disease targets." The agreement establishes a formal collaboration between NIAID and Sarepta to allow NIAID researchers to proceed with a Phase I, double-blind, placebo-controlled, dose-escalating study to assess the safety, tolerability, and pharmacokinetics of single and multiple doses of an intravenous formulation of AVI-7100 in healthy volunteers. The trial is being conducted at the NIH Clinical Center in Bethesda, Md., under the direction of Richard Davey, MD, of NIAID's Division of Intramural Research (ClinicalTrials.gov Identifier: NCT01747148). Per the terms of the agreement, Sarepta will provide AVI-7100 to NIAID. In return, Sarepta will have the right to use the data from this clinical study to support future development of AVI-7100. Source: Marketwire 12/21/12

Enrollment Completed for Phase II Trial of VVC Treatment

MethylGene Inc. in December announced that it has completed enrollment in its multicenter, randomized, double-blind, placebo-controlled trial (Trial 290-005) evaluating MGCD290 plus fluconazole versus fluconazole alone in patients with moderate-to-severe vulvovaginal candidiasis (VVC). MGCD290 is a novel antifungal agent with the potential to significantly improve outcomes for patients suffering from fungal infections, including VVC. Trial 290-005 began enrolling patients with moderate to severe VVC in the first quarter of 2012, and more than 200 patients were enrolled at 19 sites in North America. The primary endpoint for the study is therapeutic cure at day 28, which is a composite endpoint of clinical cure (resolution of signs and symptoms of the infection) and mycological cure (an absence of yeast in culture). The primary endpoint will be based on the modified intent-to-treat population, which includes those patients who received treatment and were culture positive for yeast at baseline. Secondary endpoints include the clinical and mycological cure rates at days 14 and 28, therapeutic, clinical, and mycological cure rates at day 14, recurrence rate at day 28, and time to recurrence. Top-line data are expected in March of 2013. Source: Marketwire 12/19/12

Recruitment Success Reached in Huntington Trial

Prana Biotechnology in December announced that it has completed recruitment in the Reach2HD Phase IIa clinical trial using PBT2 in patients with Huntington disease (HD). The six-month double-blind, placebo-controlled trial in patients with early- to mid-stage HD met its enrolment target of 100 patients ahead of schedule. Additional patients may be included in the trial, subject to final procedures. PBT2 offers a novel mechanistic approach to the treatment of HD, and the trial was coordinated in conjunction with the Huntington Study Group across 20 clinical sites in the U.S. and Australia. In this trial, Prana is studying the safety and tolerability of PBT2 in Huntington patients and investigating potential benefits in cognition, motor coordination, behavioral, functional, and psychiatric effects. In addition, the trial will pilot biomarker and imaging assessments. Source: Marketwire 12/19/12

Results from Progressive Supranuclear Palsy Trial Disappoint

Allon Therapeutics Inc. announced in December that its pivotal clinical trial evaluating its lead product candidate davunetide as a treatment for progressive supranuclear palsy (PSP) failed to demonstrate efficacy in this population. The study had co-primary outcome measures: the Progressive Supranuclear Palsy Rating Scale (PSPRS) and the Schwab and England Activities of Daily Living (SEADL). Data analysis failed to detect an effect on either the PSPRS or the SEADL. The study also examined a series of secondary and exploratory endpoints. There was no evidence of a drug effect on these secondary or exploratory endpoints in the prespecified analysis. The company will undertake further analysis to determine if there is any evidence of an effect or explanation for the absence of an effect. Allon President and CEO Gordon McCauley said, "This is a very sad day for patients, family members, and caregivers living with PSP, because so many of them held out great hope that these results would define a drug that has an impact on their disease. Sadly, these results have not fulfilled these hopes, but we are deeply grateful to them for their unrelenting support of this study. While this outcome is not at all what we anticipated, we do believe that we designed the correct study and executed that study well." Allon said the multinational Phase II/III randomized, double-blind, placebo-controlled trial enrolled a total of 313 subjects definitively diagnosed with PSP. Subjects were randomized into two groups, and treated with 30 mg of davunetide or placebo twice per day for 52 weeks. The study was carried out under a Special Protocol Assessment with the U.S. Food and Drug Administration at 47 sites in the United States, Canada, United Kingdom, France, Germany, and Australia. The study was designed to enroll a homogeneous PSP population and data generated by the study confirm this finding. The company said it will evaluate its strategic options going forward, but it will not allocate any additional capital to research and development activities for davunetide at this time. Source: PR Newswire 12/18/12

Last Patient Enrolled in Pivotal Phase III Anterior Uveitis Study

EyeGate Pharma announced in December that it has enrolled the last patient in the pivotal Phase III study of EGP-437 in patients with anterior uveitis. The study's objective is to evaluate the safety and efficacy of ocular iontophoresis with dexamethasone phosphate ophthalmic solution (EGP-437) as compared to treatment with topically applied prednisolone acetate (1 percent) ophthalmic suspension eyedrops. In order to be enrolled into this multicenter, randomized, double-masked study, subjects need to be between 12 and 85 years of age with a diagnosis of noninfectious anterior uveitis, defined as an anterior chamber cell count (ACC) of = 11 cells. About 200 patients were randomly assigned into one of two treatment arms in a 1:1 ratio. The primary efficacy endpoint evaluates the proportion of patients with ACC of zero at day 14. Safety will be assessed by the incidence and severity of adverse effects, and measures of intraocular pressure and best-corrected visual acuity. "Last patient, last visit" is anticipated in mid-February 2013. The company expects to have top-line data in the early spring of 2013. Source: Marketwire 12/18/12

Phase II/III Metastatic Breast Cancer Trial Registered on ClinicalTrials.gov

Immunovative, Inc. announced in December that Immunovative Therapies, Ltd. (ITL) has registered its Phase II/III, randomized, placebo-controlled study in anthracycline/taxane and capecitabine pretreated metastatic breast cancer on the ClinicalTrials.gov website. ITL has previously released that it has been granted regulatory clearance from Thailand authorities to advance its lead AlloStim™ immunotherapy product candidate to Phase II/III development. The details of the study design are now available to the public on the ClinicalTrials.gov website. The primary endpoint of the study is overall survival. The study has been powered to detect a difference of 50 percent or greater in overall survival between the treatment and control arms. If successfully executed and the predefined overall survival endpoint reaches statistical significance, ITL expects that the clinical data from this trial can be used to support possible marketing applications for AlloStim in the U.S., European Union, and other jurisdictions. The clinical trial is scheduled for launch in April 2013. The approved AlloStim Phase II/III clinical trial will be conducted at the National Cancer Institute of Thailand in Bangkok. Source: Marketwire 12/17/12

Diabetes Trial Stopped for Futility Following Phase IIb Results

Tranzyme Pharma in December announced it is discontinuing and immediately ending patient enrollment in DIGEST, a Phase IIb trial in diabetic patients receiving TZP-102 for the management of symptoms of gastroparesis, due to insufficient efficacy. The decision followed a planned interim futility analysis, which examined patients'responsiveness to thrice-daily oral dosing of 10 mg of TZP-102 or placebo at the end of weeks 4 and 8 of a 12-week trial. The results are consistent with the findings of a prior Phase IIb trial, in that there was a very large placebo effect and no treatment effect. Source: Globe Newswire 12/17/12

Phase II Trial Initiated in Patients with Prosthetic Joint Infections

Cempra, Inc. in December announced the initiation of its Phase II clinical trial of CEM-102 in patients with prosthetic joint infections (PJIs). CEM-102 is the company's loading dose formulation of fusidic acid, an orally active antistaphylococcal agent with a long history of safety and efficacy outside the U.S. The Phase II study is an open-label clinical trial in which 50 patients with PJIs will be randomized to receive either oral CEM-102 plus rifampin or current standard of care, which is intravenous antibiotic therapy with antibiotics such as vancomycin, nafcillin, or cefazolin. The primary outcome measure is demonstration of infection-free status at 12 weeks following initiation of therapy. Long-term monitoring for infection relapse or recurrence will continue for the subsequent two years. "The availability of an all-oral antibiotic regimen for the treatment of PJI offers the promise of convenient drug administration without the expense and morbidity of maintaining long-term venous access," said David Oldach, MD, FIDSA, senior vice president of clinical research. "We hope that the results of this study will lead to a new option for these patients. We expect to obtain top-line results from this study during the fourth quarter of 2013." PJIs occur in about 1 percent of hip replacements and 2 percent of knee replacements, with an overall incidence rate of about 10,000 per year in the U.S. at current hip and knee arthroplasty rates. Source: PR Newswire 12/14/12

Top-Line Data Announced from Phase Ib Study for Treatment of Cellulite

BioSpecifics Technologies Corp. in December announced top-line 30-day data from the Phase Ib study of Xiaflex in a new indication for the potential treatment of adult patients with edematous fibrosclerotic panniculopathy, commonly known as cellulite, which is being conducted by BioSpecifics' partner Auxilium Pharmaceuticals, Inc. All doses of Xiaflex were generally well tolerated, and these data support the progression into a Phase IIa clinical trial in cellulite, which Auxilium plans to initiate in the second half of 2013. The Phase Ib study is a single-site, open-label, dose-escalation trial that enrolled 99 women between 21 and 60 years of age. Study participants were assigned to one of 11 arms, each of which varied in treatment dose, injection concentration, and volume, to receive a single injection of Xiaflex, divided into 10 aliquots over a predefined 8 x 10 cm template around a target dimple. The objectives of the study are to assess the safety and effectiveness of a single injection at 30, 60, and 90 days across multiple dosing arms. Pharmacokinetic evaluations were made, as well. Across all dosing arms, 60 patients (63 percent) who were treated experienced some improvement in the volume of their target cellulite dimple at day 30. Overall, 17 percent of patients had a greater than or equal to 30 percent improvement in their target dimple at day 30; however, multiple Xiaflex dosing arms had more than 40 percent of patients experience an improvement greater than or equal to 30 percent in their target dimple at day 30. Source: PR Newswire 12/13/12

Lilly Discontinues a Phase III Rheumatoid Arthritis Registration Study

Eli Lilly and Co. announced in December that it will stop one of three Phase III rheumatoid arthritis (RA) registration studies of tabalumab, an anti-BAFF monoclonal antibody, due to insufficient efficacy. The decision followed a planned interim futility analysis of the FLEX-M study investigating tabalumab, also known as LY2127399, for the treatment of patients with moderate-to-severe RA who had an adequate response to methotrexate therapy. The decision was not based on safety concerns, and patients currently enrolled in other tabalumab RA studies will continue treatment. The FLEX-M study was designed to support registration of tabalumab as a potential treatment for RA, including an assessment of its effect on structural progression. Lilly continues to evaluate tabalumab in two other Phase III RA registration studies as well as an open-label extension study and several other smaller studies. Lilly is suspending enrollment of new patients in the RA program until additional analysis from other ongoing RA studies is completed in early 2013. These other studies are in different patient populations. Phase III studies for systemic lupus erythematosus are ongoing and will continue to enroll new patients. Currently, there is no evidence to suggest that efficacy results from the FLEX-M study in RA are indicative of potential efficacy in the lupus population. Lilly remains committed to the ongoing Phase III lupus program. "The results of this study were unexpected given the data generated in earlier Phase II clinical studies of tabalumab," said Eiry Roberts, MD, vice president of autoimmune product development at Lilly. "We remain committed to patients with rheumatoid arthritis and lupus, and will move rapidly to evaluate the impact of these data on the overall tabalumab clinical development program. Beyond tabalumab, Lilly will continue to develop additional treatment options for patients with autoimmune diseases." Source: PR Newswire 12/13/12

Experimental Agent Briefly Eases Depression Rapidly in Test

A drug that works through the same brain mechanism as the fast-acting antidepressant ketamine briefly improved treatment-resistant patients' depression symptoms in minutes, with minimal untoward side effects, in a clinical trial conducted by the National Institutes of Health (NIH). The experimental agent, called AZD6765, acts through the brain's glutamate chemical messenger system. Existing antidepressants available through prescription, which work through the brain’s serotonin system, take a few weeks to work, imperiling severely depressed patients, who can be at high risk for suicide. Ketamine also works in hours, but its usefulness is limited by its potential for dissociative side effects, including hallucinations. "Our findings serve as a proof of concept that we can tap into an important component of the glutamate pathway to develop a new generation of safe, rapid-acting, practical treatments for depression," said Carlos Zarate, MD, of the NIH’s National Institute of Mental Health, which conducted the research. Zarate, and colleagues, reported on their results online December 1 in the journal Biological Psychiatry. About 32 percent of 22 treatment-resistant depressed patients infused with ASD6765 showed a clinically meaningful antidepressant response at 80 minutes after infusion that lasted for about half an hour--with residual antidepressant effects lasting two days for some. By contrast, 52 percent of patients receiving ketamine show a comparable response, with effects still detectable at seven days. So a single infusion of ketamine produces more robust and sustained improvement, but most patients continue to experience some symptoms with both drugs. However, depression rating scores were significantly better among patients who received AZD6765 than in those who received placebos. The researchers deemed this noteworthy, since, on average, these patients had failed to improve in seven past antidepressant trials, and nearly half failed to respond to electroconvulsive therapy. Zarate and colleagues say their results warrant further trials with AZD6765, testing whether repeated infusions a few times per week or higher doses might produce longer-lasting results. Source: NIH News 12/10/12

First Patient Enrolled in Study of Removable Stent for Chronic Pancreatitis

The first patient has been enrolled in a Boston Scientific Corporation study comparing the WallFlex®  Biliary RX Fully Covered self-expanding metal stent (SEMS) to plastic stents for the treatment of benign bile duct strictures caused by chronic pancreatitis. This multi-center, prospective, randomized study will enroll 164 patients at leading hospitals in Australia, Austria, Belgium, Canada, France, Germany, Hong Kong, India, Italy, and the Netherlands. In a separate single-arm study, 187 patients were treated with the WallFlex Stent for multiple types of benign biliary strictures, including those caused by chronic pancreatitis. The stents implanted in that study were removed up to one year after being placed in the body. Five-year follow-up post stent removal is ongoing. Preliminary data were presented this summer at Digestive Disease Week 2012 by Professor Jacques Deviere of Erasme Hospital in Brussels. The data indicate that a SEMS can be removed safely any time up to one year post placement, and that short-term stricture resolution rates compare favorably to the results reported with plastic stents in chronic pancreatitis-related benign biliary strictures. Source: PR Newswire 12/4/12

300th Patient Enrolled in Spinal Implant Trial

Intrinsic Therapeutics in early December announced a key milestone in a clinical study of its Barricaid® Prosthesis: 300 patients are now enrolled in a prospective, randomized, multicenter trial to definitively establish the safety and effectiveness of the implant. This Level 1 controlled study is being conducted across five countries in Europe and is expected to include 400 to 650 patients. The goal of the project is to confirm fewer disc reherniations and improved clinical and radiographic outcomes in patients treated with the prosthesis compared to the gold-standard of limited discectomy. For patients who present with primary disc herniation with large anular defects, the Barricaid device is engineered to close the hole in the anulus in order to prevent future leaks and to reestablish pressure within the disc. o-discectomy. The act of closing the anular defect reduces the risk of reherniation, preserves the internal pressure, and provides the opportunity to maintain disc height. Source: PR Newswire 12/3/12

Company Cleared to Conduct Phase II/III Trial in Advanced Metastatic Breast Cancer

Immunovative, Inc. announced in late November that Immunovative Therapies, Ltd. has been granted regulatory clearance from Thailand authorities to advance its lead AlloStim™ immunotherapy product candidate to the Phase II/III clinical development stage in advanced metastatic breast cancer. If successfully executed and the predefined overall survival endpoint reaches statistical significance, the company expects that clinical data from this trial can be used to support marketing applications for AlloStim in the U.S., European Union, and other jurisdictions. The clinical trial is scheduled for launch in April 2013. The approved trial will be conducted at the National Cancer Institute of Thailand in Bangkok as a dual-arm, randomized, placebo-controlled, double-blind study in patients with metastatic breast cancer previously treated with an anthracycline, taxane, and capecitabine (xeloda). Her2+ patients must have additionally failed a trastuzumab (Herceptin)-containing regimen and premenopausal ER+ and/or PR+ patients must be resistant to endocrine therapy (including an aromatase-inhibitor). A total of 208 subjects will be accrued (104 in each arm). The primary endpoint of the trial is overall survival. Secondary endpoints include extensive safety analysis and evaluation of RECIST in correlation with pathological examination of longitudinal biopsy samples. Tertiary endpoints include exploratory immunological response monitoring and determination of whether survival or response correlates to neo-antigen and/or recall antigen immune competence. Source: Marketwire 11/29/12

Recruitment Completed for Alzheimer's Trial

Prana Biotechnology in November announced that it has completed recruitment in the IMAGINE trial, a 12-month Phase II trial testing PBT2, the company's drug in development for Alzheimer's disease. The double-blind, placebo-controlled trial has enrolled 41 patients with prodromal or mild Alzheimer's disease in Melbourne, Australia. One additional patient may be included in the trial, subject to final screening procedures. All trial participants are undergoing brain scans to measure PBT2's effect on amyloid deposits in the brain (using PiB-PET scanning) and effects on increasing brain activity (F-FDG PET). Cognition effects are being measured by the Neuropsychological Test Battery, a test that measures the type of cognitive problems experienced by prodromal and early Alzheimer's patients. In an earlier 12-week trial, PBT2 significantly reduced the level of Abeta protein in the spinal fluid of treated patients as well as significantly improving their cognitive Executive Function. PBT2 restores neuronal health by selectively binding and redistributing brain metals (copper, zinc) that have become imbalanced due to disease or the aging process. Furthermore PBT2 is able to prevent Abeta protein-induced toxicity and promote its disaggregation in the brain. Source: Marketwire 11/27/12

Phase III Results Published for Nail Infection Treatment

Valeant Pharmaceuticals International, Inc. announced in November that the Journal of the American Academy of Dermatology has published the positive results from two pivotal, international,  multicenter, randomized, double-blind, vehicle-controlled studies that were conducted in 1,655 subjects with onychomycosis, a common nail infection caused predominantly by dermatophyte fungi, resulting in nail destruction and deformity. The only approved topical treatments are lacquers with very limited efficacy. Oral treatments, while more efficacious, are limited by drug interactions and numerous safety concerns including the potential for acute liver injury. Valeant investigated the safety and efficacy of efinaconazole 10 percent topical solution, the first triazole antifungal agent developed for distal lateral subungual onychomycosis. The study's primary endpoint was stringently defined as the complete cure rate at week 52, which means that the target nail showed no clinical involvement and no evidence of fungus present by both KOH testing and a negative fungal culture. In Study 1, 17.8 percent of subjects treated with efinaconazole were completely cured, as compared to only 3.3 percent of subjects treated with vehicle and in Study 2, 15.2 percent of subjects treated with efinaconazole were completely cured, as compared to only 5.5 percent of subjects treated with vehicle. Using the secondary endpoint of complete or almost complete cure, defined as less than 5 percent clinical involvement and 100 percent mycologic cure, the success rates for efinaconazole increased to 26.4 percent and 23.4 percent, respectively. Source: PR Newswire 11/26/12

FDA Gives Go-Ahead for Phase III Trial to Decrease Kidney Transplant Rejection

Isotechnika Inc. announced in November that it has received permission from the U.S. Food and Drug Administration (FDA) to commence the first of two planned Phase III kidney transplant trials for its lead product candidate, voclosporin. As previously disclosed, Isotechnika reached an agreement with the FDA on a Special Protocol Assessment (SPA) for this study in March 2012. The SPA is a written agreement with the FDA that the Phase III clinical study design, endpoints, statistical analyses, and other aspects of the planned study are acceptable to support marketing approval. The SPA was received after the FDA reviewed Isotechnika's Phase IIb data and evaluated the proposed Phase III trial design and endpoints. The planned randomized, multicenter trial will include approximately 600 newly transplanted kidney patients from clinical trial sites across North America. The primary endpoint of the trial will be defined as noninferiority in biopsy proven acute rejection episodes with patients receiving voclosporin for six months as compared to tacrolimus. A key secondary endpoint will be the incidence of new onset diabetes after transplantation, as well as the overall safety and tolerability of voclosporin relative to tacrolimus. Source: Globe Newswire 11/26/12

Belgian Authorities Approve World's First Phase III Trial in Regenerative Medicine for Heart Failure

The Belgian biotechnology company, Cardio3 BioSciences (C3BS), in November announced it has received authorization from the Belgian Federal Agency for Medicines and Health Products to begin its Congestive Heart failure Cardiopoietic Regenerative Therapy (CHART-1) European Phase III trial for C3BS-CQR-1 in Belgium. This represents a world premiere for a regenerative medicine product targeting heart failure to be tested in the context of a Phase III trial. C3BS-CQR-1 is an autologous stem cell therapy for heart failure. The trial is a prospective, multicenter, randomized, sham-controlled, patient- and evaluator-blinded study comparing treatment with C3BS-CQR-1 to a sham treatment. The trial will recruit a minimum of 240 patients with chronic advanced symptomatic heart failure. The primary endpoint of the trial is a composite endpoint including mortality, morbidity, quality of life, Six Minute Walk Test, and left ventricular structure and function at nine months post-procedure. Studies in additional countries will commence once national regulatory approvals have been received. The Cardio3 BioSciences therapy, called C3BS-CQR-1, involves taking stem cells from a patient's own bone marrow and, through a proprietary process called Cardiopoiesis, reprogramming those cells so that they go on to become heart cells. The cells, known as cardiopoietic cells, are then injected back into the patient's heart through a minimally invasive procedure using a catheter called C-Cathez®, with the aim of repairing damaged tissue and improving heart function and patient clinical outcomes. This Phase III trial builds on the successful outcome of the Phase II trial conducted between 2009 and 2010 in multiple clinical sites in Belgium, Serbia, and Switzerland. Source: PR Newswire 11/22/12

Enrollment Under Way for Phase I Study of Parkinson's Disease Treatment

NeuroDerm, Ltd. announced in November that enrollment of healthy subjects is ongoing in its Phase I clinical trial of ND0612, a novel and proprietary levodopa/carbidopa liquid drug formulation for the treatment of Parkinson's disease being developed for continuous administration as an adjunct to oral levodopa via a subcutaneous delivery patch. It is designed to bypass the digestive tract and provide steady levodopa blood levels and enhanced bioavailability of oral levodopa for the reduction of motor complications in Parkinson's disease. In preclinical studies of ND0612, plasma concentrations of levodopa reached straight-line steady state levels. The current Phase I double-blind, dose-escalation trial in young, healthy volunteers will assess ND0612 for safety and tolerability, as well as for levodopa and carbidopa steady state plasma levels. Source: PR Newswire 11/20/12

Hypertension Drug Combination Meets Primary Endpoint in Phase III Trial

XOMA Corp. in November announced the 837-patient Phase III PATH trial (Perindopril Amlodipine for the Treatment of Hypertension) has demonstrated the fixed-dose combination of perindopril arginine combined with amlodipine besylate is statistically significantly superior to either compound alone in reducing both sitting diastolic and sitting systolic blood pressure after six weeks of treatment. Servier markets the product as COVERAM(R) in 91 countries outside the U.S., and as holder of its U.S. rights, XOMA intends to sublicense the product to a third-party organization that is dedicated to commercializing products for the cardiovascular marketplace. Source: Globe Newswire 11/20/12

Phase III Study in Pancreatic Cancer Shows Improvement in Overall Survival

A new cancer drug combination demonstrated significant improvement in overall survival of late-stage pancreatic cancer patients compared to those receiving standard treatment, according to results of a Phase III clinical trial led by physicians from Scottsdale Healthcare's Virginia G. Cancer Center Clinical Trials, a partnership with the Translational Genomics Research Institute (TGen). Physicians at the Virginia G. Piper Cancer Center at Scottsdale Healthcare were first to design a clinical trial to determine the safety, tolerability, and effectiveness of nab-paclitaxel (Abraxane) in combination with the standard drug gemcitabine in patients with advanced pancreatic cancer. Results of that multicenter study chaired by Dr. Daniel Von Hoff were encouraging enough that it led to one of the largest international studies ever done in pancreatic cancer, with 861 patients. Full results are expected to be presented at the American Society of Clinical Oncology (ASCO) 2013 Gastrointestinal Cancers Symposium in January in San Francisco, Calif. Nab-paclitaxel is an albumin-bound formulation of paclitaxel, produced by Celgene Corp. Dr. Von Hoff said that results of the MPACT (Metastatic Pancreatic Adenocarcinoma Clinical Trial) study will lead Celgene to submit for Food and Drug Administration approval. TGen and International Genomics Consortium scientists in collaboration with scientists from Abraxis Bioscience found that in pancreatic cancer, an albumin-binding protein called SPARC was present at high levels in cells within the pancreatic tumor microenvironment. They hypothesized that the albumin formulation of nab-paclitaxel may be taken up by tumor and surrounding cells with high SPARC expression. Source: PR Newswire 11/16/12