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Ovid Therapeutics announced plans to move ahead with a single pivotal Phase 3 trial of OV101 in pediatric patients with Angelman syndrome based on its End-of-Phase 2 Meeting with the U.S. Food and Drug Administration. If successful, the Phase 3 efficacy and safety trial called NEPTUNE is intended to support a New Drug Application for OV101 in Angelman syndrome. OV101 is a novel delta-selective GABAA receptor agonist. Based upon review and discussion of the Phase 2 STARS trial results, the clinical trial design, and the applicability of Clinical Global Impressions of Improvement (CGI-I) as a primary endpoint, the FDA and Ovid are in general agreement that the pivotal Phase 3 NEPTUNE clinical trial to be completed prior to an NDA submission, will include the following elements: Single 12-week, two-arm, randomized, double-blind, placebo-controlled trial; Once-daily dose; Approximately 50-60 pediatric patients aged 4 to 12 years, diagnosed with Angelman syndrome randomized to either placebo or OV101; Primary endpoint of change in overall CGI-I score. CGI-I will be used as a single primary endpoint, mainly due to the rare nature of Angelman syndrome, the lack of treatment options, the nonexistence of assessment instruments specific to Angelman syndrome, the heterogeneity of the disorder, and the lack of sensitivity or appropriateness of other validated measures. Based on feedback from the FDA, Ovid will develop a framework for study investigators to ensure uniform use of the validated CGI-I scale by focusing on specific symptoms that are relevant and important to patients with Angelman syndrome and their caregivers. Ovid expects to begin enrollment of the NEPTUNE trial in the second half of 2019, pending FDA concurrence on the study protocol and supporting framework and materials. Updates and further details of this trial, including secondary endpoints, will be provided upon trial initiation. In addition to the planned Phase 3 NEPTUNE trial, Ovid is initiating the ELARA clinical trial, an open-label extension study for individuals with Angelman syndrome who have completed any prior OV101 study. ELARA will use once-daily dosing and will assess long term safety and tolerability in addition to efficacy measures. Trial initiation activities are underway. The company is also exploring options for patients under four years of age.

BeyondSpring announced that the Phase 3 portion of its pivotal Study 105, evaluating its lead asset, Plinabulin, in the 105 enrolled patients treated with docetaxel chemotherapy, has met its primary endpoint of non-inferiority versus Neulasta for the duration of severe neutropenia, or DSN, of the first cycle, with statistical significance in a pre-specified interim analysis. In this trial, Plinabulin as a single agent was compared head-to-head with Neulasta as a single agent. Based on the results from this Phase 3 trial, as well as the top line data from the Phase 2 portion of Study 106 released at the recent American Society of Hematology, or ASH, BeyondSpring now has the necessary data to submit a new drug application, or NDA, to the China FDA, or National Medical Products Administration, or NMPA, for the use of Plinabulin for the treatment of chemotherapy-induced neutropenia, or CIN. The company is also on track to submit an NDA to the U.S. FDA for the treatment of CIN. Both submissions will be for broad claims for the treatment of CIN in terms of type of chemotherapy as well as cancer type. The double-blind, randomized Phase 3 portion of Study 105 was to enroll approximately 150 patients in the U.S., Europe and China with advanced breast cancer, hormone refractory prostate cancer and advanced non-small cell lung cancer, or NSCLC, who were randomized to receive docetaxel, with either Plinabulin at a fixed dose of 40 mg on Day 1, or pegfilgrastim at 6 mg on Day 2 of each 21-day treatment cycle. Plinabulin is administered on the same day of chemotherapy, 30 minutes after chemo administration. Neutrophil count and other hematological parameters were evaluated by Covance Central Laboratory. Covance is also the global clinical CRO for the study for patient enrollment. The primary endpoint of the study was the DSN in the first cycle. A pre-specified interim analysis following approximately 100 patients enrolled was built into the protocol study design to evaluate the primary endpoint of non-inferiority for DSN versus Neulasta.

Intec Pharma (NTEC) announced that the Accordion Pill developed for a proprietary Novartis (NVS) compound is continuing into a clinical PK study under the previously announced Feasibility and Option agreement with Novartis Pharmaceuticals. Following achievement of the required in-vitro specifications, the companies are continuing the program into a clinical PK study during 1H19. The details of the therapeutic area or specific compound were not released.