By creating a new mouse model of Alzheimer’s disease that better recapitulated how the disease plays out in humans, investigators at KU Leuven have gained new insights into how amyloid plaques, tau tangles and neuronal death are related at the molecular level.
“From one to many” is how Actio Biosciences Inc. describes its approach to drug development. The firm emerged with a $55 million series A financing and an eye for biological targets found in both rare and common diseases, starting with TRPV4, a target associated with Charcot-Marie-Tooth disease type 2C and other bone diseases.
New and updated clinical data presented by biopharma firms at the International Association for the Study of Lung Cancer, 2023 World Conference on Lung Cancer, including: Amgen, Astrazeneca, BMS, Daiichi, Gilead, Janssen.
Verge Genomics Inc. has entered a second big AI deal with a large drug company. Privately held Verge will receive up to $42 million, including up-front, equity and near-term payments from Alexion, Astrazeneca Rare Disease, to identify multiple targets for rare neurodegenerative and neuromuscular diseases. The deal could top out at $840 million. There also is potential for downstream royalties.
While the U.S. FDA didn’t ask for more study data or have safety or efficacy concerns, it does want modifications to Alexion, Astrazeneca Rare Disease’s sBLA for Ultomiris (ravulizumab-cwvz) to treat adults with the rare central nervous system disease neuromyelitis optica spectrum disorder. The agency has issued a complete response letter (CRL) requesting changes to Ultomiris’ Risk Evaluation and Mitigation Strategy (REMS) to better validate patients’ meningococcal vaccination status or prophylactic administration of antibiotics before being treated.
Otsuka Pharmaceutical Co. Ltd. announced plans to acquire Mindset Pharma Inc. for C$80 million (US$50.76 million) in an all-cash deal that will see Otsuka strengthen its neurology and psychiatric pipeline. Mindset, of Toronto, is developing next-generation psychedelic therapeutics to treat psychiatric and neurological disorders with high unmet needs.
Sage Therapeutics Inc. is reducing its workforce by 40% and pausing its earlier-stage programs, just a few weeks after receiving mixed news of both a U.S. FDA approval and a complete response letter for its depression drug Zurzuvae (zuranolone). The move offers the Cambridge, Mass.-based company an annualized net savings of approximately $240 million, 60% of which is related to R&D, and will extend its runway into 2026.
“I am not a fortune teller, nor am I a gambler. I will make no bets,” Lorraine Kalia told the audience at the 2023 International Congress of Parkinson’s Disease and Movement Disorders. “But I am optimistic.” At the meeting, which is being held in Copenhagen this week, Kalia, who is a scientist at Toronto Western Hospital’s Krembil Brain Institute and at the University of Toronto’s Tanz Centre for Research in Neurodegenerative Diseases, was giving an overview of “Emerging targets in the clinic” in a plenary session on “Therapeutic strategies for the future.”
The first special purpose acquisition company (SPAC) merger announced in 2023 has fallen apart. Aprinoia Therapeutics Inc. and Ross Acquisition Corp. II mutually agreed to call off the merger that had been valued at $280 million. The failed deal is part of a larger trend that has gained momentum in the past year as a struggling economy and tighter U.S. SEC restrictions dampened SPAC deals.
Rapport Therapeutics Inc. raised $150 million in a series B round, less than six months after unveiling its $100 million series A investment. It’s further evidence that its distinctive precision neuromedicine approach has gained traction with some deep-pocketed investors and adds further momentum both to its clinical development and its discovery research efforts.