Alder Biopharmaceuticals Inc., of Bothell, Wash., initiated PROMISE 2 (PRevention Of Migraine via Intravenous ALD403 Safety and Efficacy 2), the second pivotal phase III trial evaluating eptinezumab (formerly ALD403) to prevent migraines. The monoclonal antibody is designed to inhibit calcitonin gene-related peptide, or CGRP. The double-blind, randomized, placebo-controlled trial will evaluate the safety and efficacy of two doses of eptinezumab, administered by infusion once a week for 12 weeks, in approximately 1,050 individuals with chronic migraine. The primary endpoint is mean reduction in migraine days from baseline over weeks one to 12. Key secondary endpoints are the 75 percent responder rate over weeks one to 12 and over weeks one to four, as determined by change in migraine days between eptinezumab and placebo. Top-line data are expected in the first half of 2018. The eptinezumab pivotal program also consists of PROMISE 1 (PRevention Of Migraine via Intravenous ALD403 Safety and Efficacy 1), an ongoing study evaluating eptinezumab in approximately 800 patients with frequent episodic migraine, and a planned open-label study to confirm the candidate's long-term safety and tolerability, expected to begin in the first quarter of 2017. (See BioWorld Today, March 29, 2016.)
Eyenovia Inc., of New York, said findings from a randomized, controlled phase II study of more than 100 subjects who received its high-precision micro-formulation of 6 µL of a dilatory agent compared to standard eye dropper dosing of 40 µL to 50 µL were reported in the peer-reviewed journal Therapeutic Delivery. The study compared the pharmacodynamic outcome of pupillary dilation measured by digital pupilometry, showing that Eyenovia's micro-formulation achieved equivalent or greater biologic effect compared to standard eye drop therapy. Separately, participants reported better head-positioning comfort, reduced tearing/overflow and increased likelihood of adhering to ocular medication regimens with the Eyenovia regimen compared to standard eye dropper dosing.
Foamix Pharmaceuticals Ltd., of Rehovot, Israel, said enrollment was completed in two phase III trials evaluating the efficacy and safety of FMX101, its topical 4 percent minocycline foam, to treat moderate to severe acne. Top-line results from both trials are expected in the first half of next year.
Galapagos NV, of Mechelen, Belgium, said it initiated a phase I study of GLPG2737, its C2 corrector for cystic fibrosis, in healthy volunteers, triggering a $10 million milestone payment from Abbvie Inc., of North Chicago. The randomized, double-blind, placebo-controlled, single-center study is evaluating the safety, tolerability and pharmacokinetics of oral single and multiple ascending doses of GLPG2737 in at least 64 healthy volunteers in the Netherlands. Top-line results are expected in the second quarter of 2017.
Gemphire Therapeutics Inc., of Livonia, Mich., said the first patients were enrolled in the phase IIb ROYAL-1 (A 12-Week, Phase 2 Study of Gemcabene in Hypercholesterolemia Patients on Stable Moderate and High-Intensity Statins) trial to investigate gemcabene in patients with hypercholesterolemia not adequately controlled on high-intensity or moderate-intensity stable statin therapy. The randomized, placebo-controlled, double-blind trial was designed to enroll a broad patient population, including patients with heterozygous familial hypercholesterolemia and atherosclerotic cardiovascular disease, who have baseline LDL-C values ≥ 100 mg/dL. The trial is expected to enroll up to 104 adult patients at U.S. sites, who will be randomized 1:1 to 600 mg of gemcabene or placebo. The primary endpoint is the percent change from baseline of LDL-C at 12 weeks. Secondary endpoints include change from baseline in non-HDL-C, total cholesterol, triglycerides, apolipoprotein B and high-sensitivity C-reactive protein at the 12-week time point. Earlier this year, Gemphire raised $30 million in an IPO to help fund the development program. (See BioWorld Today, April 20, 2016.)
Global Blood Therapeutics Inc. (GBT), of South San Francisco, said the first patient was enrolled in ZEPHYR, the phase IIa trial evaluating the safety and efficacy of GBT440 to treat hypoxemia in patients with idiopathic pulmonary fibrosis (IPF) who are on supplemental oxygen at rest. The open label study is expected to enroll 16 IPF patients, ages 45 to 80 years, to evaluate the safety and efficacy of 900 mg of oral GBT440 daily. The primary endpoint is the effect of GBT440 on oxygen saturation at rest over a 90-day period. Key secondary endpoints include the effect of GBT440 on the requirement for supplemental oxygen, IPF-related symptoms, and quality of life using patient reported outcomes and the six-minute walk distance. The study also will assess safety, tolerability and the pharmacokinetic profile of GBT440 in IPF patients. Last month, GBT also reached an agreement with the FDA on the design of a pivotal phase III trial for GBT440 to treat adults and adolescents with sickle cell disease. (See BioWorld Today, Oct. 26, 2016.)
Gtx Inc., of Memphis, said enobosarm (previously GTX-024) achieved the pre-specified primary efficacy endpoint in the 9-mg dose cohort of patients in both stage 1 and the ongoing stage 2 portion of its phase II trial in women with advanced, estrogen receptor-positive, androgen receptor-positive breast cancer. The primary efficacy endpoint required at least nine of 44 evaluable patients to achieve clinical benefit, defined as complete response, partial response or stable disease, as measured by Response Evaluation Criteria in Solid Tumors (RECIST) at 24 weeks of treatment. In the ongoing trial, the efficacy endpoint was achieved in the first 22 confirmed evaluable patients. The company plans to report top-line findings from these patients in December. Gtx said the trial will continue enrolling and treating eligible patients with enobosarm until 44 evaluable patients have completed the trial, with top-line results from the full study expected by mid-2017. Enobosarm was well tolerated among patients treated to date in the 9-mg dose cohort, according to Gtx, which said most adverse events were either grade 1 or 2.
Jazz Pharmaceuticals plc, of Dublin, said patient enrollment was completed in its phase III study evaluating JZP-110 in excessive sleepiness associated with narcolepsy. (See BioWorld Today, Jan. 14, 2014.)
Mabvax Therapeutics Holdings Inc., of San Diego, said the first patients were treated with its therapeutic antibody, MVT-5873, in combination with standard of care chemotherapy in previously untreated pancreatic cancer. Earlier this month, the company reported that the safety of MVT-5873 was established during administration as monotherapy at three incremental dose levels in relapsed or refractory locally advanced or metastatic pancreatic cancer. The expansion cohort is designed to access the candidate's safety and tolerability in combination with chemotherapy as well as a recommended phase II dose.
Medivir AB, of Stockholm, Sweden, said Janssen Research & Development LLC, a unit of Johnson & Johnson, of New Brunswick, N.J., initiated an open-label phase IIb study of JNJ-4178, a combination of simeprevir, odalasvir and AL-335, in treatment-naïve and treatment-experienced subjects with chronic hepatitis C virus (HCV) without cirrhosis. The global, multicenter study is expected to enroll patients with HCV genotype 1, 2, 4, 5 and 6 at sites in North America, Europe and Asia to evaluate the efficacy, safety and pharmacokinetics of JNJ-4178/AL-335 (800 mg), odalasvir (25 mg), and simeprevir (75 mg). Patients will receive the combination for either six or eight weeks. The primary efficacy endpoint is the percentage of patients with a sustained virological response 12 weeks after the end of treatment, or SVR12.
Oncopep Inc., of Boston, started a phase Ib trial evaluating PVX-410, a multi-peptide therapeutic cancer vaccine, in patients with moderate or high-risk for progression smoldering multiple myeloma, an asymptomatic precursor to multiple myeloma. The study will assess the safety and tolerability of PVX-410 in combination with London-based Astrazeneca plc's durvalumab with or without lenalidomide. PVX-410 consists of four peptides from unique regions of three multiple myeloma-associated antigens and is designed to elicit an immune response to the targeted tumor antigens. The compound was granted orphan drug designation from the FDA in 2013.
Pharmamar SA, of Madrid, disclosed the start of an open-label, multicenter, phase I study in Japan to evaluate the recommended dose of RNA polymerase II inhibitor PM1183 (lurbinectedin) in Japanese patients with certain types of unresectable/advanced solid tumors. The secondary objective will be to evaluate the safety profile, to obtain preliminary information on the antitumor activity along with pharmacokinetic characteristics of PM1183 in Japanese patients when compared to Caucasians.
Recro Pharma Inc., of Malvern, Pa., disclosed positive results from its second of two phase III trials evaluating intravenous meloxicam for the treatment of acute postoperative pain. Meloxicam achieved the primary endpoint of a statistically significant difference in Summed Pain Intensity Difference over the first 24 hours (SPID24) compared to placebo in patients following abdominoplasty surgery. With the positive data from this study, company execs said this completes the efficacy program for the NDA. In the multicenter, randomized, double-blind, placebo-controlled clinical trial, 219 patients were enrolled and randomly assigned to receive a postoperative regimen of meloxicam (30-mg bolus injection) or placebo in a 1:1 ratio, once every 24 hours. The meloxicam treatment arm demonstrated a statistically significant reduction in SPID24 (p=0.0145) compared to the placebo arm. The study also achieved statistical significance for 10 of the secondary endpoints, including statistically significant differences in SPID12 (p=0.0434), time to perceptible pain relief (p=0.0050), subjects with ≥30 percent improvement at 24 hours (p=0.0178), number of times patients required rescue in the first 24 hours after randomization (p=0.0275), as well as number of times rescued from 24 to 48 hours (p=0.0009), and several other pain relief metrics, compared to placebo. Shares of Recro (NASDAQ:REPH) closed Monday at $9.30, up $1.54, or 20 percent.
Santen Pharmaceutical Co. Ltd., of Osaka, Japan, disclosed topline results of its SAKURA (Sirolimus study Assessing double-masKed Uveitis tReAtment) global clinical development program designed to confirm the efficacy, safety, and optimal dose between three active doses of sirolimus intravitreal injection as monotherapy for patients with non-infectious uveitis of the posterior segment. The SAKURA Program met its objective, demonstrating that Opsiria (sirolimus) can effectively and safely reduce intraocular inflammation (as measured by vitreous haze). Opsiria regulates the immune system through the inhibition of mTOR which acts by interrupting the inflammatory cascade that leads to T-cell activation, differentiation and proliferation, and production of interleukin-2, as well as other pro-inflammatory cytokines and, also, promoting immune tolerance by inducing T regulatory cells. The company plans to file an NDA based on the findings.
Windtree Therapeutics Inc., of Warrington, Pa., said phase IIa data on Aerosurf (lucinactant for inhalation) were presented at the International Congress of European Neonatal and Perinatal Societies in Valencia, Spain. The previously disclosed data showed that Aerosurf, administered non-invasively through nasal continuous positive airway pressure (nCPAP) to premature infants of 29 to 34 weeks gestational age with respiratory distress syndrome, was generally safe and well tolerated and may reduce the incidence of nCPAP failure.