4SC AG, of Planegg-Martinsried, Germany, said Japanese partner Yakult Honsha Co. Ltd., of Tokyo, completed a Japan-based phase I study of resminostat in patients with solid tumors. The study demonstrated a clean safety profile for the oral histone deacetylase inhibitor and triggered an undisclosed milestone payment from Yakult to 4SC.

Ablynx NV, of Ghent, Belgium, said it obtained positive results from two additional phase I studies, using its anti-RSV inhaled Nanobody (ALX-0171) designed for treating respiratory syncytial viral infections, following data from a first successful phase I trial reported in September 2012. Results from the 24-patient safety study showed that the administration of the Nanobody was well tolerated, while a 41-subject pharmacokinetic study showed that ALX-0171 levels in the lung demonstrated very rapid attainment of clinically relevant levels after once-daily administration by inhalation. Ablynx expects to move into a proof-of-concept study in the target pediatric population in the fourth quarter of this year, with data due in 2015.

Alexion Pharmaceuticals Inc., of Cheshire, Conn., said researchers presented data from a large, retrospective, natural history study of patients with severe perinatal and infantile hypophosphatasia, an inherited, rare metabolic disorder that can lead to progressive damage to multiple vital organs, destruction and deformity of bones and death. Early and sustained improvements were observed in infants, children and juveniles receiving asfotase alfa, according to phase II data presented at the Pediatric Academic Societies and the Asian Society for Pediatric Research meeting in Vancouver, British Columbia. Last year, the FDA granted breakthrough therapy designation to asfotase alfa for perinatal-, infantile- and juvenile-onset hypophosphatasia.

Biodelivery Sciences International Inc., of Raleigh, N.C., said it has randomized more than half of the planned number of patients required for its ongoing phase III study of Clonidine topical gel, its proposed treatment for painful diabetic neuropathy. An interim analysis is expected in the third quarter.

Circadian Technologies Ltd., of Melbourne, Australia, reported data at the Association for Research in Vision and Ophthalmology meeting in Orlando, Fla., showing that OPT-302 (formerly VGX-300), an inhibitor of VEGF-C, can prevent the formation of wet age-related macular degeneration (AMD) lesions and regress established lesions in the laser-induced mouse model of wet AMD. The drug reduced wet AMD lesion size and vessel leakage to a comparable extent as marketed agent Eylea (aflibercept, Regeneron Pharmaceuticals Inc.). Data also showed that VEGF-C levels are elevated in wet AMD and that OPT-302 can also reduce the expression of genes involved in blood vessel growth and inflammation in the mouse model.

Colucid Pharmaceuticals Inc., of Durham, N.C., said it reached agreement with the FDA for the planned SAMURAI study to evaluate the safety and efficacy of two doses of Lasmiditan in comparison to placebo for the treatment of acute migraine. The special protocol assessment includes two endpoints: a primary endpoint of the proportion of patients who are free of headache pain at two hours and a key secondary endpoint defined as the proportion of patients who no longer suffer from their most bothersome associated symptom of migraine (nausea, photophobia, phonophobia) at two hours. The trial will include patients with risk factors for cardiovascular disease. Lasmiditan is designed to work by selectively targeting 5-HT1F receptors expressed in the trigeminal pathway and aims to deliver efficacy without the vasoconstrictor activity associated with earlier generations of migraine therapies.

Cytomedix Inc., of Gaithersburg, Md., and wholly owned subsidiary Aldagen Inc. reported preliminary results at 90 days from the RECOVER-Stroke phase II study in patients with neurological damage arising from an ischemic stroke and treated with ALD-401, with observed improvements in the primary endpoint, measured via modified Rankin Score, failing to demonstrate clinical or statistical significance. Cytomedix said it plans to discontinue further funding of the ALDH Bright Cell development program and close its R&D facility in Durham, N.C. In the meantime, the firm remains focused on the commercial launch of Autologel, which was cleared by the FDA, with the goal of driving sales in the estimated $3.4 billion U.S. chronic wound care market. Shares of Cytomedix (OTCQX:CMXI) closed Tuesday at 51 cents, down 6 cents.

Erytech Pharma SA, of Lyon, France, said ANSM, the French health care agency, cleared the firm to start a phase II study testing ERY-ASP in second-line patients with pancreatic cancer. The trial will enroll about 100 patients and test ERY-ASP in addition to best standard of care vs. best standard of care alone, with a primary endpoint of progression-free survival at four months. Patients will be stratified according to the expression of asparaginase synthetase (ASNS) of their primary tumors. Erytech estimates that about 70 percent of patients have tumors low in ASNS and could be responders to ERY-ASP. Patient enrollment is slated to start this quarter.

Evoke Pharma Inc., of Solana Beach, Calif., presented data at Digestive Disease Week 2014 in Chicago from its phase IIb trial of EVK-001, an intranasal formulation and delivery method of metoclopramide for diabetic female patients with symptoms associated with gastroparesis. Results from the 287-patient study showed that EVK-001 was well tolerated and that patients exhibited statistically significant symptomatic improvement.

Icon Bioscience Inc., of Sunnyvale, Calif., said IBI-10090, its lead product, is in a pivotal phase III trial as a sustained-release therapy for inflammation associated with cataract surgery. The double-blind, placebo-controlled study is expected to enroll 390 patients and will employ the two lower doses used in a prior phase II/III study. The primary endpoint is the percent of patients with anterior chamber cell clearing at day eight. Patient enrollment is expected to complete later this year. IBI-10090 is a version of dexamethasone formulated using the company's Verisome technology.

Intra-Cellular Therapies Inc., of New York, presented phase II data at the American Psychiatric Association meeting in New York, showing that schizophrenia candidate ITI-007, at a dose of 60 mg, demonstrated a statistically significant improvement in psychosis, according to the study's pre-specified primary endpoint, defined as a change from baseline on the PANSS total score vs. placebo on day 28 (p = 0.017). ITI-007 also was well tolerated, with the most frequent adverse events being sedation and somnolence.

Marina Biotech Inc., of Boston, presented data from 50 patients to date at the Therapeutics Discovery Symposia in Waltham, Mass., on the firm's SMARTICLES-delivered nucleic acid drug candidates, Results demonstrated delivery to tumor in phase I and phase II trials; statistically significant dose-dependent and specific knockdown of a gene target in phase I; and single-agent antitumor activity in patients with recurrent or refractory non-Hodgkin's lymphoma in a phase II trial. The firm also presented preclinical data showing antitumor efficacy with both single- and double-stranded oligonucleotides in rodent models.

Naurex Inc., of Evanston, Ill., reported top-line results from a phase IIb study of GLYX-13, showing that its first-generation NMDA receptor modulator was well tolerated and that the rapid, robust and sustained antidepressant effects observed in an earlier single-dose study were achieved and maintained with repeat dosing in subjects with difficult-to-treat depression. The company also said it started a phase IIa study of its second-generation, orally active agent, NRX-1074, in development for major depressive disorder. Naurex additionally disclosed that its existing shareholders, which include multiple venture funds and pharmaceutical companies, have invested another $25 million in the company.

Nymox Pharmaceutical Corp., of Hasbrouck Heights, N.J, reported data from an ongoing phase II trial showing that, after up to 22 months for NX-1207 single-injection-treated low-grade localized prostate cancer patients, there was an 85 percent reduction compared to controls in the proportion of patients who had upgraded blinded biopsy results in the treated areas and went on to require and receive radiation therapy and/or prostatectomy. The study, NX03-0040, enrolled 146 patients randomized to one of two doses of NX-1207 (2.5 mg or 15 mg) or to active surveillance (no treatment). Follow-up studies are being conducted on all patients to monitor outcome and safety data.

Opko Health Inc., of Miami, said it completed enrollment in the third phase III trial of Rayaldee, designed to evaluate the oral vitamin D prohormone's long-term safety and efficacy in treating secondary hyperparathyroidism in patients with stage III or stage IV chronic kidney disease and vitamin D insufficiency. The study is a six-month, open-label extension of the two pivotal trials involving about 430 patients altogether. Top-line data from the two pivotal trials are expected during the third quarter of this year.

Rhythm Pharmaceuticals Inc., of Boston, presented phase II results at the Digestive Disease Week conference in Chicago showing that relamorelin, administered twice daily for four weeks in patients with moderate to severe gastroparesis, significantly improved gastric emptying, significantly reduced vomiting and, in a large subgroup (about 60 percent of patients) with vomiting at baseline, significantly improved a composite symptom score comprising nausea, abdominal pain, bloating and early satiety. Relamorelin is a small-peptide analogue of ghrelin.

Sucampo Pharmaceuticals Inc., of Bethesda, Md., said a study of Amitiza (lubiprostone) for opioid-induced constipation in patients with chronic noncancer pain published online in Pain Medicine showed that patients treated with the drug demonstrated significant overall improvement for abdominal discomfort, straining and constipation severity and stool consistency when compared to placebo. Patients rated the effectiveness of lubiprostone as significantly better than placebo for 11 of 12 weeks.