Aerie Pharmaceuticals Inc., of Irvine, Calif., reported 12-month safety results of the Mercury 1 phase III registrational trial of fixed-dose combination candidate Roclatan, which the company said were consistent with those observed for the 90-day efficacy period in the trial. There were no new adverse events that developed following the initial 90-day period, and there were no drug-related serious or systemic adverse events. The most common adverse event for Roclatan was conjunctival hyperemia, or eye redness, which was observed in about 60 percent of patients, of which about 70 percent was determined to be mild by biomicroscopy. As observed in previous trials, hyperemia was sporadic, with only about 10 percent of patients with hyperemia across each physician visit during the 12-month period. In addition, intraocular pressure (IOP) measurements taken at 8 a.m., 10 a.m. and 4 p.m. at months six, nine and 12, showed greater IOP lowering in the Roclatan group compared to both latanoprost and Rhopressa (netarsudil ophthalmic solution) in a range from 1 mmHg to 3 mmHg. Both Mercury 1 and Mercury 2 previously achieved their 90-day primary efficacy endpoints, with Roclatan demonstrating statistical superiority over each of its components at all measured time points, including Aerie product candidate Rhopressa 0.02 percent and market-leading prostaglandin analogue latanoprost, all of which were dosed once daily in the evening. An NDA is expected to be submitted in the first half of 2018. (See BioWorld Today, Sept. 16, 2016.)

Axim Biotechnologies Inc., of New York, said it entered a services agreement with an Israel-based CRO for a phase II proof-of-concept study testing its cannabidiol and gabapentin chewing gum product to treat restless legs syndrome. The pharmacokinetic and double-blind, randomized, single-center trial will enroll about 30 patients.

Biondvax Pharmaceuticals Ltd., of Ness Ziona, Israel, reported statistically significant positive results from the phase IIb trial of M-001, its universal flu vaccine candidate, meeting both the safety and influenza-specific cellular immune response endpoints. The 219-participant study showed that the M-001 vaccine was well-tolerated. T-cell immunity measured at baseline (prior to immunization) and after immunization with M-001 showed statistically significant elevated T-cell-dependent immune responses in both dosage forms vs. placebo. The response was more notable in the 1-mg dose group vs. the 0.5-mg dose group. The study's secondary endpoint, evaluating antibody (HAI) response to avian H5N1 pandemic vaccination after M-001 or placebo administration, showed that in one of the four H5N1 strains tested, a statistically significant HAI elevation was observed in participants who had received M-001. Based on those results, Biondvax said it is in discussions with regulatory authorities about proceeding directly to a pivotal phase III trial.

Celyad SA, of Mont-Saint-Guibert, Belgium, said it started the SHRINK trial, a third study testing lead candidate CYAD-10 (CAR T NKG2D), targeting metastatic colorectal cancer patients. The open-label phase I study will evaluate safety and clinical activity of multiple doses of CYAD-01, administered concurrently with the neoadjuvant FOLFOX treatment in patients with potentially resectable liver metastases from colorectal cancer. The dose-escalation part of the study will enroll up to 18 patients while the extension phase would enroll 21 additional patients.

Diffusion Pharmaceuticals Inc., of Charlottesville, Va., reported the completion of a major production run of its lead drug candidate, trans sodium crocetinate (TSC), for use in its planned phase III trial, which will test TSC in the treatment of newly diagnosed inoperable glioblastoma brain cancer patients. It is of sufficient quantity to support the entire trial, the company said. Diffusion plans to initiate testing by the end of 2017.

Incyte Corp., of Wilmington, Del., said the first patient was treated in GRAVITAS-301, a pivotal phase III trial testing itacitinib, a selective JAK1 inhibitor, in combination with corticosteroids compared to placebo plus corticosteroids as a first-line treatment in patients with acute graft-vs.-host disease. The randomized, double-blind, placebo-controlled study will enroll about 430 patients, 18 or older, who have undergone one allogeneic transplant from any donor and any donor source for a hematologic malignancy or disorder. The primary endpoint is overall response rate (ORR) at day 28, defined as the proportion of subjects demonstrating a complete response, very good partial response or partial response. Key secondary endpoints include non-relapse mortality at month six, defined as the proportion of subjects who died due to causes other than malignancy relapse, as well as duration of response, and ORR at days 14, 56 and 100.

Ironwood Pharmaceuticals Inc., of Cambridge, Mass., reported top-line data from a phase IIb trial of IW-3718 in adult patients with uncontrolled gastroesophageal reflux disease (GERD), showing it met its primary endpoint, indicating that twice-daily, oral dosing of IW-3718 1,500 mg plus a proton pump inhibitor (PPI) significantly reduced heartburn severity in patients with uncontrolled GERD compared to patients treated with a PPI alone. Patients in the IW-3718/PPI group showed a mean decrease of 58 percent from baseline in heartburn severity compared to 46 percent in patients treated with a PPI alone (p = 0.04). Further, a 45 percent reduction in weekly heartburn severity was determined to be clinically meaningful for patients in that study based on patient-reported outcome measures. IW-3718 1,500 mg was well-tolerated in the trial. Ironwood said it plans to have end-of-phase II meetings with the FDA, after which the company expects to advance the drug, a gastric retentive formulation of a bile acid sequestrant, into phase III in the second half of 2018.

Kamada Ltd., of Rehovot, Israel, said it submitted to the FDA for review a proposed pivotal phase III protocol for inhaled alpha-1 antitrypsin (AAT) therapy for the treatment of AAT deficiency (AATD). The proposed study is intended to treat AATD subjects with inhaled AAT at a dose of 80 mg once daily for a period of two years, with a placebo arm at a 2-to-1 ratio with crossover to the treatment arm following a period of 12 months. In parallel, a concurrent intravenous AAT arm will be evaluated for two years. The study is planned to include about 200 to 300 patients, and is expected to measure lung function as a primary endpoint and lung density as a secondary endpoint.

Kite Pharma Inc., of Santa Monica, Calif., said data were published in Molecular Therapy from a National Cancer Institute study of its anti-CD19 CAR T-cell therapy in patients with aggressive non-Hodgkin lymphoma (NHL), including diffuse large B-cell lymphoma. The work follows data published in February, in which nine patients with chemorefractory aggressive NHL were treated with a single dose of anti-CD19 CAR T cells with a CD28 co-stimulatory domain. Seven of the nine patients were evaluable for response, with complete remissions (CR) observed in five. Four of the five CRs are ongoing from 38 months to 56-plus months after treatment. There were no chronic toxicities attributable to CAR T cells except B-cell aplasia and hypogammaglobulinemia.

Theravance Biopharma Inc., of Dublin, and Mylan NV, of Hertfordshire, U.K., reported results from a 12-month phase III safety study of revefenacin (TD-4208), a long-acting muscarinic antagonist and a proposed once-daily, nebulized bronchodilator in development for the treatment of chronic obstructive pulmonary disease (COPD). The study of 1,055 patients with COPD demonstrated that revefenacin was generally well-tolerated, and no new safety issues were identified. Rates of adverse events and serious adverse events were low and comparable to those seen in the standard-of-care treatment arm. An NDA is expected in the fourth quarter.

Trevena Inc., of King of Prussia, Pa., said during its analyst day that it has completed enrollment of patients in the ATHENA study testing Olinvo (oliceridine injection) to manage pain associated with a wide range of procedures and diagnoses. A total of 772 patients were administered the drug. The study is expected to support a planned NDA submission in September or October of this year.