Alder Biopharmaceuticals Inc., of Bothell Wash., presented data from its phase IIb trial of CGRP-inhibiting monoclonal antibody eptinezumab (formerly ALD-403) for the prevention of migraine at the American Academy of Neurology meeting in Boston. Findings showed that chronic migraine patients treated with a single infusion of eptinezumab experienced a significant reduction in migraine days as measured by 75 percent responder rates, with maximum efficacy in one to four weeks that was maintained through 12 weeks. Post-hoc analysis of the data also suggested that a single administration led to a clinically meaningful reduction in the percentage of patients experiencing migraine within 24 hours to 48 hours post infusion. Top-line results from the first phase III pivotal trial are expected later this year, and a possible BLA filing could come next year.

Aspyrian Therapeutics Inc., of San Diego, said findings from an ongoing trial of RM-1929, a candidate from its photoimmunotherapy, or PIT, platform were presented at the American Head & Neck Society meeting in San Diego. Results from seven patients with recurrent head and neck squamous cell carcinoma who cannot be satisfactorily treated with surgery, radiation or platinum chemotherapy, showed that all seven treated with repeated cycles of PIT with RM-1929 had evidence of tumor necrosis at the treatment site. No skin photosensitivity or normal tissue toxicity was noted. Four out of seven (57 percent) patients had durable clinical response to the treatment without progression of disease. The targeted photoimmunotherapy used a conjugate of cetuximab and IR700 dye.

Celyad SA, of Mont-Saint-Guibert, Belgium, said it dosed the first patient of the second dose in the solid tumor arm of its THINK (THerapeutic Immunotherapy with NKR-2) trial. At the first solid tumor dose level, one pancreatic and two colorectal cancer patients were successfully dosed. None of these patients experienced dose-limiting adverse events. The latest patient to be dosed has ovarian cancer. The open-label study is assessing the safety and clinical activity of multiple administrations of autologous NKR-2 T cells in seven refractory cancers, including five solid tumors and two hematological tumors.

Genmab A/S, of Copenhagen, Denmark, and partner Janssen Research & Development LLC, a unit of New Brunswick, N.J.-based Johnson & Johnson, in collaboration with the European Myeloma Network and Stichting Hemato-Oncologie voor Volwassenen Nederland, plan to start a phase III study of daratumumab in relapsed and refractory multiple myeloma. The randomized study (MMY3013, APOLLO) will compare daratumumab, a human IgG1k monoclonal antibody, in combination with Pomalyst (pomalidomide, Celgene Corp.) and dexamethasone vs. pomalidomide and dexamethasone in patients who have previously been treated with an immunomodulatory drug and a proteasome inhibitor. The study is expected to start this quarter and is designed to confirm results from the MMY1001 (EQUULEUS) phase I study.

Ignyta Inc., of San Diego, in an update on its entrectinib program, reported interim data from 32 patients with ROS1 fusion-positive non-small-cell lung cancer (NSCLC), including new patients from the STARTRK-2 trial enrolled as of Dec. 16, 2016. Data showed 24 had confirmed RECIST responses, for an objective response rate (ORR) of 75 percent. The median duration of response was 17.2 months among the responder patients, and the median progression-free survival was 19.1 months among all patients. Eleven of 32 patients (34 percent) had CNS metastases at baseline and, of those, seven had confirmed RECIST responses, for an ORR of 64 percent. Entrectinib is an oral, CNS-active tyrosine kinase inhibitor targeting tumors that harbor TRK, ROS1 or ALK fusions. It is being studied in the phase II STARTRK-2 study.

Mallinckrodt plc, of Staines-upon-Thames, U.K., reported results at the American Academy of Neurology meeting in Boston from a study of H.P. Acthar Gel (repository corticotropin injection), showing that treatment may be associated with a decline in medical resource utilization in the three months following treatment completion, including the number of emergency room visits, hospitalizations and days hospitalized in patients with infantile spasms (IS). H.P. Acthar Gel was approved by the FDA as a first-line monotherapy treatment for IS in 2010.

Merck KGaA, of Darmstadt, Germany, presented data at the American Academy of Neurology meeting in Boston from a retrospective subgroup analysis of the phase III CLARITY trial in 289 patients with high disease activity, demonstrating statistically significant reduction in the risk of disability progression and relapse with cladribine tablets at a dose of 3.5 mg/kg (n=140) compared with placebo (n=149) in relapsing multiple sclerosis (MS) patients who were either treatment-naïve or had prior disease modifying drug exposure. Findings showed that treatment with cladribine was associated with a larger reduction in the risk of six-month confirmed EDSS progression in patients with high disease activity (82 percent; p=0.0001) than observed in the overall CLARITY population (47 percent; p=0.0016) vs. placebo. Additionally, data showed that cladribine tablets reduced the relative risk of annualized relapse rate in patients with high disease activity (67 percent; p<0.0001) compared with the overall CLARITY population (58 percent; p=<0.0001).

Miragen Therapeutics Inc., of Boulder, Colo., presented interim results at the Society for Investigative Dermatology meeting in Portland, Ore., from a phase I study testing MRG-201, a drug designed to mimic the activity of microRNA-29, which has been shown to decrease the expression of certain genes that are involved in scar formation. As of mid-March, 54 volunteers had participated in the study, which evaluated expression of microRNA-29 and its pharmacodynamic biomarkers in untreated skin incisions following single or multiple administrations of MRG-201. Data showed that where volunteers' skin was incised without receiving MRG-201, microRNA-29 expression was decreased and direct target genes were up-regulated as compared to unincised skin; where volunteers received intradermal injections of MRG-201, pharmacokinetic analysis of the volunteers' plasma revealed that very little drug (less than 150 ng/mL) was generally detectable in the volunteers' blood. The product was generally well-tolerated. Pharmacodynamic data showed that single and multiple doses of MRG-201 were generally accompanied by reduced expression of certain genes associated with fibrosis as compared to a placebo injected incision in the same volunteer, while multiple administrations were generally accompanied by reduced fibroplasia, a marker of scar formation, as assessed by histopathology (p<0.01).

Oncoceutics Inc., of Philadelphia, said the first patient was treated in a phase I/II trial testing ONC-201 in combination with dexamethasone in adults with relapsed/refractory multiple myeloma. The study, supported by a $1.7 million grant from the FDA's Office of Orphan Products Development, is expected to enroll 42 patients. In preclinical studies, ONC-201 was shown to engage the dopamine receptor D2, activating the integrated stress response, which up-regulates a host of proteins that induce tumor cell death and inhibit the synthesis of proteins that are key for cancer growth.

TG Therapeutics Inc., of New York, reported preliminary results from its ongoing phase II study of TG-1101 (ublituximab), its glycoengineered anti-CD20 monoclonal antibody, in patients with relapsing forms of multiple sclerosis at the American Academy of Neurology meeting in Boston. Data showed that TG-1101 was well-tolerated, with no grade 3/4 adverse events observed, and all doses were fully delivered to all subjects to date. All patients met the primary endpoint of 95 percent or greater B-cell depletion by four weeks. The median B-cell depletion at week four was 99 percent after two infusions (day one and day 15) with a cumulative dose of 600 mg, which the company said compares favorably to other anti-CD20 monoclonal antibodies.