Why U.S. FDA-approved Wakix (pitolisant) has worked to treat excessive daytime sleepiness (EDS) in narcolepsy but not in idiopathic hypersomnia (IH) patients is the question that lies before Harmony Biosciences Holdings Inc., and one that the U.S. FDA will take up at a future sit-down.
In a race with Karuna Therapeutics Inc. to get its schizophrenia drug to market, Cerevel Therapeutics Holdings Inc. has priced an underwritten public offering at $22.81 per share. The offering is worth of about $450 million, with the company estimating sales of the 19.7 million shares will yield net proceeds of about $433.6 million.
For most psychiatric illnesses, the precipitating event is mysterious. Many conditions are thought to result from a mix of genetic risk and environmental factors, but the specific trigger remains unknown. In post-traumatic stress disorder (PTSD), the environmental trigger is usually clear. In many cases, it is all the affected individuals can think about. “Intrusive reliving” of the triggering situation is one of the core features of PTSD.
After rising significantly pre-market on Oct. 5 – when positive phase IIa results with Lomecel-B in Alzheimer’s disease (AD) were made public – shares of Longeveron Inc. (NASDAQ:LGVN) sank during the day and again Oct. 6 to close at $1.75, down 34 cents, or 16%.
A discovery-stage company founded 10 years ago and focused on developing a PINK1 activator for Parkinson’s disease and other indications is now under the umbrella of Abbvie Inc. through an acquisition potentially worth $655 million. North Chicago-based Abbvie paid $110 million at closing for San Francisco-based Mitokinin Inc., but the deal also includes up to $545 million in potential payments upon hitting development and commercial milestones related to the PINK1 (PTEN-induced kinase 1) program. On top of that, Mitokinin shareholders are entitled to tiered royalties based on net sales.
As pricing negotiations for Biogen Inc./Eisai Co. Ltd.’s newly approved Leqembi (lecanemab) for Alzheimer’s disease get underway at Japan’s Central Social Insurance Medical Council (Chuikyo), industry watchers see opportunity for potential drug price reform.
It has been a long time coming, but Fabre-Kramer Pharmaceuticals Inc. finally received U.S. FDA approval for its major depressive disorder candidate, Exxua (gepirone hydrochloride extended-release tablets). The approval comes three months after the June 23 PDUFA date (as a result of amendments filed by the company in April and May), but 24 years after the original NDA was filed in 1999.
Positive top-line data from the phase IIb study of BNC-210 to treat post-traumatic stress disorder (PTSD) supercharged Bionomics Ltd.’s stock. Shares (NASDAQ:BNOX) closed trading a dramatic 243.8% higher at $3.37 each on Sept. 28. The oral, selective negative allosteric modulator of the alpha7 nicotinic acetylcholine receptor stumbled last December in a phase II trial for treating social anxiety disorder but now has regained momentum.
Brainstorm Cell Therapeutics Inc. said it’s exploring all its options in the wake of a Sept. 27 U.S. FDA advisory committee vote, in which the committee overwhelmingly disagreed with the company that the data it presented supported the effectiveness of Nurown (debamestrocel) for the treatment of mild to moderate amyotrophic lateral sclerosis.
A new gene editing method uses the CRISPR technique to modify the cells of an organ in vivo, creating a mosaic used to identify the effects of each altered gene. Scientists from the Swiss Federal Institute of Technology (ETH) in Zürich developed this technology called AAV-Perturb-seq, based on adeno-associated virus (AAV) to target, edit and analyze single-cell genetic perturbations.
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