Aerie Pharmaceuticals Inc., of Bedminster, N.J., said dosing has begun in its phase III registration trial in Canada testing Rhopressa, a once-daily, triple-action eye drop designed to lower intraocular pressure (IOP) in patients with glaucoma or ocular hypertension. The study, dubbed Rocket 3, aims to evaluate safety and tolerability of Rhopressa in patients over a 12-month period. The three-study phase III program also includes Rocket 1 and Rocket 2, both of which are designed to demonstrate noninferiority of IOP lowering compared to timolol. Total enrollment in the phase III program is expected to reach about 1,300 patients. Results are expected in mid-2015 and, if positive, should lead to a new drug application filing by mid-2016.
Aesculap Biologics LLC, of Center Valley, Pa., said the first patient was implanted with the firm's Novocart 3D product as part of an FDA investigational new drug application for a phase III randomized, partially blinded multicenter study to test the matrix-induced autologous chondrocyte product in repairing articular cartilage defects of the knee. The first implantation occurred during knee surgery to repair disease articular cartilage.
Alkermes plc, of Dublin, said it completed patient enrollment in a phase II study of ALKS 3831, an investigational, oral, broad-spectrum atypical antipsychotic medicine in development for the treatment of schizophrenia. ALKS 3831 is composed of samidorphan, a mu-opioid antagonist, in combination with established antipsychotic drug olanzapine. The double-blind, active-controlled, dose-ranging study will assess ALKS 3831's efficacy, safety and tolerability in the treatment of schizophrenia and its attenuation of weight gain, compared with olanzapine. Alkermes expects top-line results from the 12-week, double-blind treatment period of the study in early 2015. The double-blind treatment period will be followed by an additional 12-week period in which all patients will receive ALKS 3831.
Antisense Therapeutics Ltd., of Victoria, Australia, reported data from its phase II study of ATL1103 in acromegaly, showing that the drug met its primary endpoint, producing a statistically significant average reduction in the serum insulin-like growth factor-1 (sIGF-1) levels of 26 percent from baseline (p < 0.0001) at week 14 at the 400-mg per-week dose. All patients treated with 400 mg per week had reductions in sIGF-I levels from baseline, and greater reductions in sIGF-I were observed in patients who had lower body weights. Those data position ATL1103 to move into phase III development, and the company plans to accelerate out-licensing activities to secure a pharmaceutical development partner for further work.
Biolinerx Ltd., of Jerusalem, said it dosed the first patient in a phase I trial for a second indication of its BL-8040 cancer therapy platform, as a treatment for the mobilization of stem cells from the bone marrow to the peripheral blood circulation, where they can be harvested for transplant supporting the treatment of hematological indications. The study will consist of two parts: Part one will be a randomized, double-blind, placebo-controlled dose-escalation study exploring the safety and tolerability of escalating repeated doses of BL-8040 in healthy volunteers, along with secondary objectives assessing the efficacy in mobilizing stem cells as a standalone therapy and pharmacokinetics; the second part will be an open-label study to assess BL-8040's stem cell mobilization capacity, as well as the yield of cells collected by apheresis. The first part will enroll up to four cohorts, with eight healthy volunteers in each cohort, while part two will involve a single cohort of eight healthy volunteers.
Edison Pharmaceuticals Inc., of Mountain View, Calif., reported results from a phase II placebo-controlled trial in pediatric patients with Rett syndrome, showing that those treated with EPI-743 demonstrated a statistically significant increase in head circumference. Decreased brain growth and head circumference are hallmarks of the disease. Specifically, head growth in the treatment group was 0.75 cm from baseline, compared to 0.29 cm in the placebo group (p = 0.05). In a subgroup analysis of the children with the largest head growth, improvements in oxygenation, hand function and disease-relevant biomarkers also were recorded. The six-month study, which enrolled a total of 24 subjects, ages 2.5 to 8 years, however, did not meet the primary endpoint of improving the subjective Rett syndrome disease severity score. Edison said it plans to meet with regulatory authorities in the coming months to determine the next steps.
F2G Ltd., of Manchester, UK, said it started a phase I single ascending-dose study of its intravenous (I.V.)/oral systemic antifungal candidate, F901318, in development for invasive aspergillosis and other life-threatening fungal infections. The phase I study will test the I.V. formulation, with an oral formulation to be tested in the future.
Geneuro SA, of Geneva, reported results from a one-year, open-label extension of a phase IIa study testing monoclonal antibody GNbAC1 in 10 multiple sclerosis (MS) patients, nine of which had primary or secondary progressive disease. Results confirmed the long-term safety profile and, with respect to clinical endpoints, the average Expanded Disability Status Score, which measures disease progression, remained stable over one year. GNbAC1 targets the MSRV-Env protein expressed by genes of endogenous retroviral origin, which Geneuro said appears to play a critical role in the pathogenesis of MS. A proof-of-concept study to test the efficacy of GNbAC1 in MS will follow in 2015.
Neuroderm Ltd., of Rehovot, Israel, said the first patients with severe Parkinson's disease have been enrolled and dosed in a phase IIa trial of ND0612H, a high-dose form of the firm's liquid levodopa/carbidopa (LD/CD) drug formulation, delivered continuously through subcutaneous administration by a belt-pump. The objective of the study is to evaluate the pharmacokinetic profile and the steady state levodopa blood levels of different doses of ND0612H, as well as of ND0612L, a low-dose form of liquid LD/CD for moderate-stage Parkinson's disease patients delivered through a similar belt-pump device.
Nuvo Research Inc., of Mississauga, Ontario, said it completed enrollment in a phase II trial of WF10 in patients with refractory allergic rhinitis. The 16-week, randomized, double-blind, placebo-controlled study will assess the efficacy, safety and tolerability of a regimen of five infusions of either WF10 or its main constituents (sodium chlorite and sodium chlorate) relative to saline control in patients with multiple airborne allergies who suffer from moderate to severe allergic rhinitis. The trial will measure total nasal symptom score and other secondary endpoints. Nuvo expects the trial to be completed in late 2014 with top-line results anticipated in the first quarter of 2015.
Oncolytics Biotech Inc., of Calgary, Alberta, said it completed patient enrollment in a randomized phase II study of paclitaxel plus its oncolytic virus candidate, Reolysin, vs. paclitaxel alone in patients with persistent or recurrent ovarian, fallopian tube or primary peritoneal cancer. The primary objectives of the trial are to estimate the progression-free survival hazard ratio and to determine the frequency and severity of adverse events associated with treatment of weekly paclitaxel alone and weekly paclitaxel with Reolysin. About 110 patients were enrolled in the study, which is sponsored by the National Cancer Institute.
Prometic Life Sciences Inc., of Laval, Quebec, said it had a successful pre-investigational new drug (IND) meeting with the FDA for its antifibrotic lead drug candidate, PBI-4050. That meeting focused on the company's proposed phase II program for PBI-4050 in patients with diabetic kidney disease (DKD) and other rare diseases as well as the manufacturing and preclinical package that it intends to include in the IND submission. The multicenter study for DKD will be three-arm, double-blind, placebo-controlled involving two different doses of PBI-4050.
Redhill Biopharma Ltd., of Tel Aviv, Israel, said it is starting a phase III trial designed to to evaluate the safety and efficacy of RHB-102, an oral, extended-release, once-daily formulation of antiemetic drug ondansetron, in patients suffering from acute gastroenteritis. The randomized, double-blind, placebo-controlled, parallel group study is expected to enroll 320 adults and children older than 12 to be randomized to receive either RHB-102 or placebo. The primary endpoint is the absence of vomiting from 30 minutes after the first dose through the discharge from the emergency department. Secondary endpoints include frequency of vomiting, severity and time to resolution of nausea and time to resumption of normal activities. Top-line results from the RHB-102 trial, dubbed GUARD, are expected during the second half of 2015.
Ultragenyx Pharmaceutical Inc., of Novato, Calif., reported positive interim data from the phase I/II study of recombinant human beta-glucuronidase (rhGUS, UX003), an investigational therapy for the treatment of mucopolysaccharidosis 7 (Sly syndrome). Data from the primary 12-week analysis from the open-label study show evidence of clearance of lysosomal storage as indicated by the decline in urinary glycosaminoglycan (GAG) excretion and the reduction in liver size. The change in urinary GAG excretion was observed by two weeks after the first dose of rhGUS and declined by approximately 40 percent to 50 percent from baseline after 12 weeks of treatment. Decreases in liver size were observed in the two patients who had enlarged livers at baseline. No adverse events have been observed through up to 28 total weeks of treatment. Those results were presented at the Society for the Study of Inborn Errors of Metabolism symposium in Innsbruck, Austria.