Articles related to DRUG PIPELINE

The FDA granted Breakthrough Therapy designation to DT120, an oral lysergide formulation under investigation for major depressive disorder, based on phase 3 findings suggesting rapid and sustained antidepressant effects, though the treatment remains investigational and unapproved.

An experimental cell therapy that couples a patient’s red blood cells with myelin protein fragments was well tolerated in a 10-patient Phase 1b study and was associated with increases in regulatory T-cells and reductions in neurofilament light chain, a biomarker linked to nerve injury.

The FDA accepted Belite Bio’s NDA for tinlarebant in Stargardt disease type 1 and granted Priority Review. In the phase 3 DRAGON trial, treatment reduced atrophic retinal lesion growth by 35.7% compared with placebo, with regulatory review ongoing.

The FDA approved olezarsen (Tryngolza), an apoC-III antisense inhibitor, as an adjunct to diet for reducing triglycerides and acute pancreatitis risk in adults with severe hypertriglyceridemia, based on phase 3 data showing an 85% reduction in pancreatitis events compared with placebo.

Ocular Therapeutix announced FDA alignment on a planned fourth-quarter 2026 NDA submission for Axpaxli in wet AMD, based on SOL-1 phase 3 efficacy data and interim safety results from the ongoing SOL-R trial.

The LRRK2 inhibitor BIIB122 failed to slow disease progression versus placebo in 648 early-stage idiopathic Parkinson’s patients in the Phase 2b LUMA trial, leading Biogen and Denali to discontinue joint development.