The third day of the AD/PD 2024 conference in Lisbon started with a plenary lecture given by Professor Howard Fillit entitled, “Translating the biology of aging into new therapeutics for Alzheimer’s disease.” Fillit, a recognized neuroscientist and geriatrician, and co-founder of the Alzheimer’s Drug Discovery Foundation (ADDF), pointed to the geroscience hypothesis which postulates that targeting aging processes may result in preventive and therapeutic options for diseases of old age, including Alzheimer’s disease (AD).
A first-quarter 2024 launch for Alzheimer’s drug donanemab appears to be off the table as Eli Lilly and Co. disclosed a last-minute decision by the U.S. FDA to convene an advisory committee to review data from the phase III Trailblazer-ALZ 2 trial.
Tweaks made to the design of the phase III trial called Phoenix (vs. the narrowly positive phase II Centaur study) with Amylyx Pharmaceuticals Inc.’s amyotrophic lateral sclerosis (ALS) drug Relyvrio (sodium phenylbutyrate plus taurursodiol) didn’t work. Now, the Cambridge, Mass.-based firm is facing possible withdrawal of the treatment from the U.S. and Canada, where it’s known as Albrioza. Shares of Amylyx (NASDAQ: AMLX) closed March 8 at $3.36, down $15.61, or 82.3%, after the firm disclosed top-line results from Phoenix, a global, 48-week, randomized, placebo-controlled phase III effort with Relyvrio, also known as AMX-0035.
On March 4, 2024, several groups of scientists discussed the challenges of investigating the effects of HIV in the central nervous system (CNS) at the oral abstract session on neuropathogenesis of HIV held during the 31st Conference on Retroviruses and Opportunistic Infections (CROI), in Denver. A cure for HIV will require eliminating the virus in all its reservoirs, those tissues where HIV remains latent but retains the capacity for reactivation and replication. However, despite antiretroviral therapy (ART), the virus could continue to replicate continuously at a low level in some reservoirs, including the CNS.
Vivoryon Therapeutics NV said it will conduct an in-depth analysis after reporting top-line data showing the phase IIb European Viviad study testing its oral small molecule, varoglutamstat, in patients with early Alzheimer’s disease missed statistical significance on the primary endpoint measuring changes in cognition over time. Investors, however, opted for a more immediate reaction, sending company shares (Euronext Amsterdam:VVY) plunging 90% March 4, to close at €0.80 (US87 cents)
Biovie Inc.’s double-barrelled blast of positive data with insulin sensitizer NE-3107 in Alzheimer’s disease (AD) and Parkinson’s disease (PD) launched shares (NASDAQ:BIVI) on a wild ride to close March 1 at $2.23, up 99 cents, or almost 80%, after trading as high as $3.31.
San Diego-based Kenai Therapeutics Inc. raised $82 million in a series A round to move its disease-modifying cell therapy for Parkinson’s disease into the clinic. The company, which leverages induced pluripotent stem cell (iPSC) technology, will advance its next-generation allogeneic neuron replacement cell therapies for neurological diseases, specifically completing a clinical proof-of-concept trial for its lead candidate, RNDP-001.
Pharmaust Ltd.’s monepantel met its primary safety endpoints and showed positive signals of potential efficacy in a phase I trial in patients with motor neuron disease (MND)/amyotrophic lateral sclerosis (ALS). With these results, the company will now progress to a pivotal phase II/III trial by midyear, Pharmaust CEO Michael Thurn told BioWorld.
Separate teams of investigators have reported new insights into how the brain disposes of metabolic waste via the glia-based lymphatic system, or glymph system. In two papers published in Nature on Feb. 28, 2024, scientists from Washington University in St. Louis described how in sleeping animals, the synchronized activity of neurons drove ionic gradients that facilitated the movement of fluid through brain tissue. And researchers from the Massachusetts Institute of Technology showed that, in a mouse model of Alzheimer’s disease (AD), the glymphatic system mediated clearance of amyloid-β after sensory stimulation at a 40-Hertz rhythm.
In the end, Minerva Neurosciences Inc. NDA submission wasn’t able to overcome the U.S. FDA’s concerns regarding data for dual 5-HT2A/sigma 2 antagonist roluperidone. The agency issued a complete response letter (CRL) for the application, which had been seeking approval as the first treatment specifically targeting negative symptoms of schizophrenia.