Zynerba Pharmaceuticals Inc.’s stock was slashed nearly in half June 30 as the company’s pivotal Connect-FX study for treating fragile X syndrome behavioral symptoms failed to achieve statistical significance in its primary and three key secondary endpoints.
Two separate groups have recently shown that in mouse models, inactivation of a single gene was enough to directly convert other cell types in the brain into neurons.
PERTH, Australia – Melbourne-based Neuren Pharmaceuticals Ltd. will be able to progress NNZ-2591 to the clinic for three orphan indications after raising AU$20 million (US$13.75 million) in a placement to institutional and sophisticated investors in Australia, New Zealand, Hong Kong and the U.K.
Shares of Heron Therapeutics Inc. sank 27% in morning trading, after the San Diego-based firm disclosed a second complete response letter (CRL) for HTX-011 in postoperative pain. The CRL, received from the FDA on June 26, the anticipated PDUFA date, stated the agency was unable to approve the NDA in its present form and called for additional nonclinical information.
While the FDA approved Zogenix Inc.’s Fintepla (fenfluramine), an oral treatment for patients ages 2 and older with seizures associated with Dravet syndrome, a rare, pediatric-onset form of epilepsy, the company stock continued its months-long struggle.
While Biohaven Pharmaceutical Holding Co. Ltd.’s phase II/III proof-of-concept study of troriluzole for treating obsessive-compulsive disorder (OCD) failed to meet its primary outcome measure at week 12, the company found enough silver in the study’s lining to press on for a larger phase III trial.
Yumanity Therapeutics Inc. said that Merck & Co. Inc. is making a big bet on its nascent neurodegenerative disease pipeline, licensing exclusive rights to programs addressing amyotrophic lateral sclerosis (ALS) and a rare type of dementia in a deal with up to $500 million in potential milestone payments. The collaboration also includes an up-front payment, money toward a series C financing and potential royalties on net sales of the licensed programs, though the value of those elements wasn't disclosed.
Zynerba Pharmaceuticals Inc. in late May popped the lid off top-line data from the open-label phase II study called Bright with ZYN-002 in children and adolescents with autism spectrum disorder, and the findings drew adjectives from Wall Street such as “provocative” and “encouraging.”
Nan Fung Life Sciences is “looking to make a significant presence in the field,” said Engrail Therapeutics Inc. CEO Vikram Sudarsan, whose firm bagged a $32 million series A round led by the global investment platform of Hong Kong’s Nan Fung Group, and its support represents “a clear statement in that direction.”
Neurocrine Biosciences Inc.’s chief business development and strategy officer, Kyle Gano, said “there was really no playbook” for the deal in which his firm is paying Takeda Pharmaceutical Co. Ltd. $120 million up front for an exclusive license to seven programs, including three clinical-stage assets targeting the notoriously difficult indications of schizophrenia and treatment-resistant depression (TRD), as well as depression-related anhedonia.
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