Abliva AB (Nasdaq Stockholm: ABLI), a clinical-stage biotech company developing medicines for the treatment of rare and severe mitochondrial diseases, today announced the start of the company’s global, potentially registrational Phase 2 study with KL1333 (the FALCON study).
Abliva’s lead program, KL1333, under development for the treatment of mitochondrial DNA-related primary mitochondrial diseases in adult patients suffering from debilitating fatigue and muscle weakness, has now entered into late-stage clinical development with the activation of the first site in the FALCON study. The global team will now begin screening patients into the study, a potentially registrational, Phase 2 study to evaluate the safety and efficacy of KL1333. Given a screening period of 8-12 weeks, the first patient is expected to be dosed in the first quarter of 2023.
As previously communicated, it is intended that an interim analysis of the first 40 patients will occur in late 2023/early 2024, which will provide insight into the relative chance of overall success of the study as well as inform as to the number of patients required for the remainder of the trial.
“We are thrilled today to announce the start of the FALCON study, the design of which is supported by positive Phase 1b study results, natural history data, and valuable patient input,” said Magnus Hansson, CMO. “This is an important milestone for both Abliva as well as the patients, who desperately need therapies for their disease.”