By the end of November 2024, the BioWorld Neurological Diseases Index had dropped 13.4%, deepening its decline of 9.4% at the end of August. Meanwhile, the Dow Jones Industrial Average surged 19.16% in the first 11 months of the year, building on a 10.81% gain reported at the end of October. The Nasdaq Biotechnology Index posted a year-to-date increase of 6.27% through November, retreating from its peak gain of 11.7% in August.
News that Trevi Therapeutics Inc. got the size just right of the phase IIb Coral trial with Haduvio (oral nalbuphine extended release) led the company’s shares (NASDAQ:TRVI) to close Dec. 12 at $3.60, up $1.11, or 45%, after trading as high as $4.60.
CNS specialist Noema Pharma AG has added a further $44 million to its series B, bringing the total for the round to $147 million. The new financing bolsters the balance sheet as the Basel, Switzerland-based company progresses four phase II trials, with key data readouts expected in 2025.
Researchers from the University of California San Francisco (UCSF) have successfully replicated the design of regulatory T cells, achieving local targeted immune suppression and protection from CAR T-cell cytotoxicity. Many of the treatments used so far in the context of inflammatory and autoimmune disorders lead to systemic immunosuppression. In this sense, limiting immunosuppression locally to targeted tissues may help overcome systemic toxicity.
Bionomics is progressing BNC-210 to phase III trials in post-traumatic stress disorder (PTSD) following positive feedback from the U.S. FDA. Final data from the phase II Attune study showed that BNC-210 improved PTSD symptom severity at week 12 with efficacy observed as early as week four.
Cervomed Inc. executives said they intend to scrutinize low plasma drug concentrations that appeared to spoil results from the phase IIb Rewind-LB trial testing neflamapimod in patients with dementia with Lewy bodies.
The U.S. FDA has greenlit the first steps of Uniqure NV’s accelerated approval pathway for gene therapy AMT-130 to treat Huntington’s disease. The agency said data from the ongoing phase I/II studies compared to natural history external control are muscular enough to get the process going without having to dive into additional studies.
Dewpoint Therapeutics Inc. and Mitsubishi Tanabe Pharma Corp. have entered a research collaboration worth up to $480 million to advance Dewpoint’s novel TDP-43 small-molecule condensate modulator for amyotrophic lateral sclerosis. Under terms of the deal, Boston-based Dewpoint will receive an undisclosed up-front payment and is eligible to receive R&D-based milestone payments up to $480 million. Upon reaching those milestones, Osaka, Japan-based MTPC will have an exclusive option to license the program and assume responsibility for global clinical development and commercialization. Dewpoint will also receive tiered royalties on net sales.
Voyager Therapeutics Inc.’s recent selection of a lead development candidate, VY-1706, for its tau silencing gene therapy program in Alzheimer’s disease brought renewed attention to the target, which continues to intrigue a substantial lineup of developers. Bellwether data rolled out this fall from UCB SA and Roche AG at the Clinical Trials in Alzheimer’s Disease meeting in Madrid.
A ketone body, a molecule derived from the metabolism of acids to obtain energy when glucose is not available, could become an effective ally in treating Alzheimer’s or preventing the effects of aging on the brain. A group of scientists at the Buck Institute for Research on Aging have studied the role of β-hydroxybutyrate as a signaling metabolite of misfolded proteins by interacting with them and altering their solubility, a mechanism that allows their elimination, as observed in preclinical models.
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