Tag: oral therapy

STOCKHOLM, Sweden, June 25, 2026 – AnaCardio, a clinical stage biopharmaceutical company developing novel drugs to treat heart failure, today announced the publication in The Lancet of results from its Phase 1b/2a GOAL-HF1 study evaluating AC01, a first-in-class, oral calcium-sensitising contractile agent, in patients with heart failure with reduced ejection fraction (HFrEF). The results showed a favourable safety and tolerability profile, dose-proportional pharmacokinetics with confirmed target engagement. In exploratory efficacy analyses, numerical rapid and sustained improvements in haemodynamics and cardiac structure and function were observed on top of optimised guideline-directed medical therapy (GDMT).

Data show investigational atumelnant drove sustained androgen reductions while enabling lowering of glucocorticoid supplementation to physiologically normal levels in adults with classic CAH

Data from up to two years of treatment in PATHFNDR-1 and PATHFNDR-2 open-label extension studies show PALSONIFY maintained lower IGF-1 levels and stable symptoms

SAN DIEGO, June 12, 2026 (GLOBE NEWSWIRE) -- Belite Bio, Inc (NASDAQ: BLTE) (“Belite Bio^®” or the “Company”), a clinical-stage drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, today announced the completion of its rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for tinlarebant. Tinlarebant is an investigational, once-daily oral therapy for the treatment of Stargardt disease type 1 (STGD1), a rare, inherited retinal disease caused by mutations in the ABCA4 gene that leads to progressive and irreversible vision loss. STGD1 affects an estimated 53,000 people in the U.S. alone, and there are currently no approved treatment options for the disease.

- CMT-SORD program advances; meeting scheduled with the FDA in Q3 2025 to discuss govorestat for the treatment of CMT-SORD

PALI-2108 demonstrated to be safe and well tolerated with no serious adverse events (SAEs)

-  500 subjects enrolled across the United States, the United Kingdom, France, Czech Republic, Switzerland, China, Taiwan, and Australia