Articles related to DRUG PIPELINE

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.

The FDA granted Fast Track designation to elunetirom, an oral CNS thyroid hormone receptor agonist, for adjunctive treatment of bipolar depression, while the agent remains in a 30-participant open-label phase 2 trial with no human efficacy data yet available.