Brainstorm Cell Therapeutics Ltd., of New York, said the University of Massachusetts Memorial Medical Center in Worcester, Mass., will begin enrolling patients in the company's randomized, double-blind, placebo-controlled phase II trial testing adult stem cell therapy Nurown in amyotrophic lateral sclerosis (ALS). The primary endpoint is the safety of intrathecal and intramuscular administration of Nurown, while secondary endpoints include change in the ALS Functional Rating Scale and change in slow vital capacity.
Clementia Pharmaceuticals Inc., of Montreal, said it started a phase II trial of palovarotene, a retinoic acid receptor gamma agonist, for the treatment of fibrodysplasia ossificans progressive (FOP), a rare, genetic disease characterized by painful, recurrent episodes of soft tissue swelling and abnormal bone formation in muscles, tendons and ligaments. The double-blind, placebo-controlled trial will evaluate the effect of different doses of palovarotene on new bone formation during and after a flare-up in 24 patients with FOP who are 15 or older. Treatment will be initiated within seven days from flare-up onset and will continue for six weeks, with an additional six weeks of follow-up. A number of clinical endpoints will evaluate the efficacy and safety of palovarotene, including imaging endpoints for new bone formation and clinical assessments of physical function and patient-reported outcomes. The trial is expected to be completed in 2015. Clementia in-licensed palovarotene from Basel, Switzerland-based Roche AG in 2013.
Egalet Corp., of Wayne, Pa., reported results from the category 1 abuse deterrence study for Egalet-001, an abuse-deterrent, extended-release, oral morphine-based product in development for the management of pain severe enough to require daily, around-the-clock opioid treatment and for which alternative treatments are inadequate. In the study, Egalet-001 demonstrated resistance to common forms of physical and chemical manipulation as compared to MS Contin (morphine sulfate controlled-release) and resisted crushing in order to swallow, snort or smoke, and dissolving in order to inject. Egalet-001 also displayed extreme resistance to crushing and grinding with all household tools used in the study, whereas the comparator was easily converted to a fine powder.
Elite Pharmaceuticals Inc., of Northvale, N.J., said it started dosing in a pivotal bioequivalence study in healthy volunteers testing ELI-201, its twice-daily abuse-deterrent oxycodone/naltrexone product, which uses Elite's pharmacological abuse-deterrent technology. The open-label, single-dose, partially randomized crossover study is designed to compare the bioavailability and establish bioequivalence of ELI-201 controlled-release capsules to Oxycontin controlled-release tablets. The secondary objectives include determining the bioavailability of oxycodone from ELI-201 under fasted and fed conditions and evaluating safety and tolerability.
Genspera Inc., of San Antonio, presented a clinical update for G-202 in hepatocellular carcinoma (HCC) at the Asia-Pacific Primary Liver Cancer Expert meeting in Taipei, Taiwan. Interim results from the phase Ib and ongoing phase II study in HCC patients who had previously progressed on or who were intolerant to Nexavar (sorafenib, Amgen Inc. and Bayer AG) showed that 80 percent of those treated with G-202 had stable disease at two months, and 50 percent exhibited stable disease at four months on study. G-202, which emerged from Genspera's platform combining plant-derived cytotoxin thapsigargin with a prodrug delivery system, is activated by the prostate-specific membrane antigen enzyme.
Helix Biopharma Corp., of Aurora, Ontario, said it opened patient screening for the ninth dose level cohort in its ongoing phase I/II study of L-DOS47 in Poland, following completion of the first treatment cycle in the three patients enrolled in the eighth dose level cohort, in which the therapy was well tolerated. L-DOS47 is the first immunoconjugate-based drug candidate in development based on the firm's DOS47 technology, which is designed to modify the microenvironmental conditions of cancer cells in a manner that leads to their destruction. The drug is in a phase I study in the U.S. in non-small-cell lung cancer.
Neos Therapeutics Inc., of Grand Prairie, Texas, said it completed a phase III study of its methylphenidate XR-ODT candidate, NT-0102, in children with attention deficit hyperactivity disorder (ADHD). The trial, which randomized 87 children with a diagnosis of ADHD, showed that NT-0102 met primary and secondary efficacy endpoints, demonstrating statistically significant improvements on both the Swanson, Kotkin, Agler, M-Flynn, and Pelham Scale and the Permanent Product Measure of Performance scale, through 12 hours post-dose. No serious adverse events were reported during the study, and the adverse event profile was consistent with the drug's mechanism of action. Neos said it plans to file a new drug application for NT-0102 in the "near future."
Nymox Pharmaceutical Corp., of Hasbrouck Heights, N.J., said it closed enrollment for Study NX02-0022, its second re-injection phase III trial of NX-1207 in benign prostatic hyperplasia (BPH). The study, which enrolled patients who previously participated in an earlier study, used a lyophilized formulation of NX-1207 that has longer stability when stored at room temperature. An interim analysis from the trial will be reported within the next few weeks. In the first reported NX-1207 re-injection study (NX02-0020), mean overall symptomatic improvement in the AUA BPH Symptom Score in treated patients was 7.6 points (p = 001).
Targacept Inc., of Winston-Salem, N.C., reported top-line data from a phase IIb monotherapy study testing TC-1734 in mild to moderate Alzheimer's disease, showing that the study did not meet the objective of demonstrating superiority over donepezil after 52 weeks of treatment. The trial did not include a placebo arm and was not designed to determine whether TC-1734 is equivalent to donepezil. The co-primary endpoints for the study were measures of cognitive function and global function. Consistent with previous clinical results, TC-1734 exhibited a benign safety and tolerability profile. Based on the results, Targacept said it does not intend to invest in further development of TC-1734, an NNR therapeutic. Shares of Targacept (NASDAQ:TRGT) fell 38 cents to close Tuesday at $4.03.
Taurx Therapeutics Ltd., of Aberdeen, UK, said it achieved its target enrollment of 833 subjects into one of its two ongoing phase III trials of LMTX, a tau aggregation inhibitor, for the treatment of Alzheimer's disease. The placebo-controlled study is designed to assess the efficacy of LMTX in slowing the progression of Alzheimer's in people diagnosed with mild to moderate disease and also is evaluating the safety and pharmacokinetic profile of LMTX, as well as examining the effect on imaging endpoints in a subset of subjects. The company's other phase III trial, with a target enrollment of 700 mild Alzheimer's patients, is expected to complete enrollment in the next two to three months.
ZS Pharma Inc., of Coppell, Texas, said it completed enrollment in ZS004, its second phase III trial testing ZS-9 (sodium zirconium cyclosilicate), an investigational treatment for hyperkalemia. The randomized, double-blind, placebo-controlled study is designed to confirm, over a longer treatment period, the positive results previously reported for ZS003, a phase III trial in which once-daily doses of ZS-9 maintained potassium levels within the normal range with safety and tolerability similar to placebo. A total of 258 patients have been enrolled. Top-line data are expected late in the third quarter or early in the fourth quarter.