Butterfly schreef op 8 januari 2025 14:11:
youtu.be/qny8N5vhaJI?feature=sharedKL1333: Innovative therapy in late-stage development
Project status: Positive interim analysis of data from patients in Wave 1 of the FALCON study
FALCON is a Phase 2, global, randomized, placebo-controlled, potentially registrational study evaluating the safety and efficacy of KL1333 in adult patients with primary mitochondrial disease who experience consistent, debilitating fatigue and myopathy (muscle weakness), the most common and impairing symptoms.
A total of 180 patients with mitochondrial DNA mutations who meet the eligibility criteria are randomized 3:2 to receive KL1333 (50mg-100mg) or placebo twice daily for 48 weeks. The two alternative primary endpoints assess consistent fatigue (using the PROMIS Fatigue Mitochondrial Disease Short Form) and myopathy (using the 30 second Sit-to-Stand test), only one of which must be positive to file for marketing approval.
In June 2023, the first patient was dosed. In July 2024, an interim analysis evaluating 24-week data from the first wave of patients confirmed the strong safety profile of KL1333, and both primary endpoints passed futility, meaning that both have the potential to demonstrate benefit in the final analysis of the study.
KL1333 has Orphan Drug Designation (ODD) in both Europe and the U.S. as well as Fast Track designation in the U.S.