Biogen presented new data for its Ionis Pharmaceuticals–partnered anti-tau candidate diranersen at the Alzheimer’s Association International Conference, which began in London on July 15. The Phase 2 trial did not meet the dose-related endpoint but showed “unprecedented” reductions in total tau in cerebrospinal fluid and brain tau pathology, according to Szofia Bullain, vice president of Alzheimer’s Disease and Dementia Clinical Development at Biogen.
Biogen also announced the U.S. Food and Drug Administration's approval of a subcutaneous formulation of Leqembi, developed with Eisai, for initial doses. This means that the anti-amyloid antibody can now be administered fully at home, distinguishing it from Eli Lilly’s Kisunla.
In developments related to psychedelics, Compass Pathways released positive results for its psilocybin-based asset COMP360. The treatment led to a clinically meaningful reduction in depression severity by six weeks during a late-stage trial. These results will support Compass Pathways’ ongoing rolling submission for regulatory approval. Definium Therapeutics also reported promising outcomes for its LSD-based treatment targeting major depressive disorder. Additionally, the Food and Drug Administration issued final guidance supporting developers working with psychedelic drugs.
In transthyretin amyloid cardiomyopathy (ATTR-CM), Ionis and AstraZeneca announced that their antisense therapeutic Wainua failed in a Phase 3 study. This outcome had implications across the ATTR-CM field, affecting companies such as Alnylam while potentially benefiting BridgeBio and Intellia Therapeutics due to more favorable readthroughs.
Roche ended two trials partnered with Ionis involving an antisense oligonucleotide (ASO) targeting Huntington’s disease. This decision may favor Wave Life Sciences, which is developing a more selective ASO designed to target only mutant huntingtin while preserving the neuroprotective wild-type form.
