Bionomics Ltd.’s U.S. debut last December, when the firm raised about $23 million through the sale of American depositary shares, called new attention to the Australian firm and its prospects in social anxiety disorder (SAD) and post-traumatic stress disorder (PTSD).
In one of its familiar U-turns, the U.K. National Institute for Health and Care Excellence (NICE) has recommended NHS England should fund a rare disease gene therapy from Orchard Therapeutics plc, considered to be the world’s most expensive drug. The list price for Libmeldy (atidarsagene autotemcel) in England and Wales is £2,875,000 (US$3.9 million), making it the most expensive drug that NICE has ever evaluated.
Sio Gene Therapies Inc. is retrenching the business as it terminated the company’s AXO-Lenti-PD license agreement with Oxford Biomedica plc for treating Parkinson’s disease. Also, Sio’s CEO, Pavan Cheruvu, is leaving the company. Sio said it is deprioritizing its Parkinson’s disease program due to several factors, including resource requirements and development timelines “to reach meaningful value inflection for the program and an increasingly challenging market and regulatory environment” for the indication.
Anavex Life Sciences Corp. reported top-line data from the 33-subject phase III study with Anavex 2-73 (blarcamesine) in adult females with Rett syndrome, turning up a statistically significant improvement over placebo for the primary efficacy endpoint as well as for all the secondary efficacy endpoints. The sigma-1 receptor activator, given as a once-daily, oral liquid, proved well-tolerated, too, with good compliance by patients, Anavex said.
While comments continue to pour in, both in opposition and support, regarding the U.S. Centers for Medicare & Medicaid Services’ (CMS) proposed national coverage decision that would restrict Medicare coverage of monoclonal antibodies intended to treat Alzheimer’s to those used in CMS- or NIH-approved clinical trials, some groups also are appealing to Health and Human Services (HHS) Secretary Xavier Becerra to step into an HHS agency turf war.
Several pharma companies think that targeting a rogue protein known as alpha-synuclein could be the key to halting or reversing neurodegeneration in Parkinson’s disease – but has the industry learned from a string of failures in Alzheimer’s? In the space of a few weeks, Novartis AG and Sanofi AS have signed major deals for molecules targeting misfolded alpha-synuclein, the rogue protein thought to be the root cause of Parkinson’s disease.
Destroying senescent cells in the aging stem cell niche, either genetically or pharmacologically using the small-molecule senolytic ABT-263 (navitoclax; Abbvie Inc.), enhanced hippocampal neurogenesis and cognitive function in mice, a Canadian study led by scientists at the University of Toronto has found.
UCB SA has unveiled plans to acquire Zogenix Inc. in a deal worth up to $1.9 billion (€1.7 billion), adding to its portfolio an approved drug aimed at rare forms of epilepsy. Brussels-based UCB will pay $26 per share in cash for the Emeryville, Calif.-based biotech, plus a contingent value right of $2 per share, which would pay out upon approval of the oral drug Fintepla (fenfluramine) for Lennox-Gastaut syndrome.
Not heeding earlier FDA advice has earned Levo Therapeutics Inc. a complete response letter (CRL) for its NDA seeking approval for intranasal carbetocin (LV-101) in hyperphagia associated with Prader-Willi syndrome (PWS). The CRL followed a 12-1 negative advisory committee vote in November.
PERTH, Australia – Australia has recently established new research and discovery centers to study psychedelic treatments for mental health disorders. Headquartered Melbourne, the Psychae Institute is a global research collaboration supported by a AU$40 million (US$29 million) investment from a North American biotechnology company.
RSS
