The small-molecule steroid sulfatase (STS) inhibitor STX-64 (ONESTX-1, irosustat) previously showed a good safety and tolerability profile in several phase I and II clinical trials that evaluated the candidate for oncology indications.
Recent findings have suggested GAL-201 from Galimedix Therapeutics Inc. is a robust oral candidate to treat Alzheimer’s disease (AD). GAL-201 binds to the misfolded form of amyloid-β (Aβ) monomers, thus preventing its aggregation and formation of neurotoxic oligomers and protofibrils.
Reflecting the evolving scientific understanding of Alzheimer’s disease, the U.S. FDA revised its 2018 draft guidance on developing drugs to treat early Alzheimer’s.
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.
Since microglia play a critical role in resolving amyloid pathology and it has been previously demonstrated that microglia function can be induced by TREM2 activation, TREM2 agonism has been considered a promising novel therapeutic approach to delay or prevent the progression of Alzheimer’s disease.
At the 2024 Alzheimer’s & Parkinson’s Diseases Conference this week, Michel Goedert from the MRC Laboratory of Molecular Biology in Cambridge gave the Donald L. Price Memorial Plenary Lecture entitled, “Cryo-EM structures of disease filaments from human brains.” Donald Price was particularly well-known for his work on plaques and tangles in nonhuman aged primate brains and for the development of several animal models of neurodegenerative diseases.
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)
Neurocode USA Inc. added Alzpath Dx, a blood test for Alzheimer’s disease screening, to its lab developed tests, making it the first available for commercial use in the U.S. The move stands to make screening for the neurodegenerative disease much more accessible, as recent study results indicate Alzpath’s accuracy rivals PET scans and cerebrospinal fluid tests, the current gold standards, in detecting the signature biomarkers of the disease.
