Not long after a morning earnings call on March 27 in which Intelgenx Corp. CEO Horst Zerbe said his team was still awaiting word from the FDA on its resubmitted 505(b)(2) application for its acute migraine candidate, Rizaport Versafilm, that news arrived in the form of a complete response letter (CRL), its third following earlier CRLs in February 2014 and April 2019.
Regenacy Pharmaceuticals Inc., of Waltham, Mass., closed on a $30 million series A financing designed to initiate a phase II proof-of-concept trial for ricolinostat, an oral, selective histone deacetylase (HDAC) 6 inhibitor, in diabetic peripheral neuropathy.
Redpin Therapeutics Inc., of New York, plans to take its newly secured $15.5 million series A financing to continue developing its ion-channel based chemogenetics platform for addressing neural circuit dysfunctions such as epilepsy, neuropathic pain and Parkinson’s disease.
Stoke Therapeutics Inc. is marching ahead in the second half of this year with its phase I/IIa study with STK-001 in Dravet syndrome (DS), one of the more abysmal forms of epilepsy, although the FDA has temporarily hobbled part B of the test, pending preclinical data that will more fully characterize the safety profile of the antisense oligonucleotide (ASO).
Design Therapeutics Inc., a San Diego startup developing new therapies for degenerative disorders caused by nucleotide repeat expansions, has raised $45 million in series A financing.
Nineteengale Therapeutics is a new joint venture that was launched to fill a large void. Cyclica Inc., along with its partner, Neurotheryx Canada Ltd., both of Toronto, created Nineteengale to find and develop cannabinoid-inspired drugs for bipolar disorder, anxiety and pain management.
Tony McKinney, the founder and CEO of Ethismos Research Inc., has worked with the small molecule he plans to take into the clinic this year for the past 10 years. All the while he’s used the same support group of personnel to develop the molecule. Sometimes that group works and sometimes it’s on hiatus.
PERTH, Australia – The FDA granted Melbourne-based Neuren Pharmaceuticals Ltd. and San Diego, California-based Acadia Pharmaceuticals Inc. a rare pediatric disease designation for trofinetide for treatment of Rett syndrome, a rare neurodevelopmental congenital central nervous system disorder.
Lowering levels of tau protein improved multiple symptoms of autism spectrum disorders (ASD) in two different mouse models of the disease, both of which are driven by hyperactivity of the mTOR PI3 kinase pathway.
Launching a broad new front in its long-running battle against dementia and other neurological diseases, Biogen Inc. has moved to license multiple Sangamo Therapeutics Inc. programs for $350 million up front plus up to $2.37 billion in development, regulatory and commercial milestone payments.
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