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.
The U.S. Center for Medicare & Medicaid Services (CMS) opened a 30-day comment window on its proposed national coverage determination (NCD) to limit Medicare access to monoclonal antibodies targeting amyloid beta in Alzheimer’s disease (AD) only in clinical trials. Biogen Inc., clearly the target, along with its AD treatment Aduhelm (aducanumab), was among the first to respond.
In proposing a national coverage determination to limit coverage of a class of Alzheimer’s drugs to those being used in clinical trials approved by the U.S. Center for Medicare & Medicaid Services or the NIH, CMS appears to be treading in FDA territory. “For the first time ever, Medicare is second-guessing FDA – and not only on work that it has already done, but on work FDA will do in the future,” said George Vradenburg, chair and co-founder of Us Against Alzheimer’s, a patient advocacy group. “HHS [the Department of Health and Human Services] is clearly at war with itself, with one agency approving this class of drugs and another slamming the door shut on treatment. . .. Does CMS no longer trust the FDA’s work?”
Targeting the toxic alpha-synuclein protein found in the brains of people with Parkinson’s is one of the most promising approaches to treat the disease in the clinic – but getting any drug into the brain is a challenge. Sanofi SA has joined with ABL Bio Inc. to solve this problem, in-licensing ABL-301, a preclinical bispecific antibody that locks on to misfolded alpha-synuclein but also includes a molecular “shuttle” that allows it to penetrate the blood-brain barrier.
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