Subtyping is what made precision medicine in cancer a reality. And for successful drug discovery in all its stages, finding subtypes in Alzheimer’s disease is all but imperative. Prior to the approval of the modestly effective Lequembi (lecanemab, Biogen Inc./Eisai Co. Ltd.) Kisunla (donanemab, Eli Lilly and Co.), and the since-withdrawn Aduhelm (aducanumab, Biogen Inc./Eisai Co. Ltd.), more than a dozen failed phase III clinical trials were all that amyloid-targeting drugs had to show for themselves for decades of effort.
Praxis Precision Medicines Inc. is making a change to its upcoming registrational, 400-patient Power2 study – adding a dose arm plus a depression/mood endpoint – based on encouraging phase II data from the Radiant study with vormatrigine in focal onset seizure (FOS) patients over an eight-week period. Specifically, Power2 will test 20 mg, 30 mg, and 40 mg of the drug against placebo for 12 weeks.
Spine Biopharma Inc. cited an “inconsistent sham control response” among some of the sites in its phase III study testing SB-01 as an intradiscal treatment for patients with chronic low back pain associated with degenerative disc disease. The study, dubbed MODEL, fell short of statistical significance on pain intensity and pain-related function, despite showing numerical and clinically meaningful improvements.
Wavebreak Therapeutics LLC has identified thiazole compounds acting as α-synuclein (SNCA) aggregation inhibitors reported to be useful for the treatment of amyloidosis, Creutzfeldt-Jakob disease, frontotemporal dementia, Gaucher disease, Lewy body dementia, multiple system atrophy, progressive supranuclear palsy and Parkinson’s disease, among others.
Researchers from Suven Life Sciences Ltd. have presented results on the evaluation of the pharmacological properties of SUVN-I6107 in various animal models of cognitive deficits at the Alzheimer's Association International Conference (AAIC) 2025.
Cassava Sciences Inc. a has reported positive preclinical results of a study evaluating simufilam in a mouse model of tuberous sclerosis complex (TSC)-related epilepsy. TSC is a rare genetic disorder resulting from a mutation in the TSC1 or TSC2 gene.
Neuroinflammation is a hallmark of Alzheimer’s disease (AD), driven in part by chronic microglial activation and elevated pro-inflammatory cytokines, which contribute to neuronal damage and cognitive decline. Targeting microglial activity has emerged as a promising therapeutic approach.
Deep learning tools for protein design can also be used to create molecules that bind to them. Certain peptides, such as intrinsically disordered proteins (IDPs), are challenging to target due to their variable nature. However, scientists from the lab of Nobel laureate David Baker have developed a method to generate binders for IDPs by searching the world’s largest protein database with their AI-powered tool RFdiffusion.
In the search for effective and safe Kv7 activators, a collaboration spanning Belgium, Germany and Italy has identified JNJ-37822681 as a promising lead.
GSK plc scientists have presented findings from studies on the therapeutic potential of GSK-5862611, an anti-Sortilin antibody, in addressing neurodegenerative disease mechanisms linked to the TDP43 G298S mutation.
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