ALSYMO

Alsymo is committed to fighting Pulmonary Arterial Hypertension, PAH: high blood pressure in the lung caused by the vascular remodeling of small arteries. PAH is a rare disease affecting 40.000 patients worldwide. Despite 14 drugs and a 4 billion USD market, still 1 out of 10 patients dies every year. Two to 5% of PAH patients have Pulmonary Veno-Occlusive Disease, PVOD, in which the venules are also remodeled. Two out of 3 PVOD patients die within 3 years after diagnosis.

The NMDA receptor was known to mediate cognitive functions in the central nervous system. It is also present in the lung as a molecular hub at the crossroads of the pathways leading to vascular remodeling. New peripheral NMDA receptor antagonists that do not cross the blood-brain barrier have been designed to avoid central side-effects. Alsymo has acquired the exclusive worldwide license of their patents from the Paris-Saclay SATT.

Alsymo’s drug candidate inhibits lung vascular remodeling in the FDA-approved animal model of monocrotaline poisoning mimicking PAH. Alsymo now wants to raise 9 M€ to perform preclinical regulatory development and Phase 1 clinical trial.

Then, two Phase 2 clinical trials are planned with world-renowned KOLs: Prof. Humbert for PAH and Prof. Montani for PVOD, both from Bicêtre Hospital, where Alsymo is also located with a privileged access to PAH clinicians, researchers, and patients. It is a key success factor, together with the breakthrough profile of its drug candidate.