Abliva Reaches Patient Enrollment Goal for Wave 1 of the FALCON Study

Abliva AB (Nasdaq Stockholm: ABLI) a clinical-stage company developing drugs for the treatment of rare and severe primary mitochondrial disease, today announced that the goal of enrolling 40 patients for Wave 1 of the ongoing, potentially registrational, Phase 2 FALCON study has been met. The interim analysis remains on track for summer 2024.

The FALCON study is a global, randomized, placebo-controlled, and potentially registrational clinical Phase 2 study evaluating the efficacy of the company’s lead candidate KL1333 in adult mitochondrial disease patients experiencing fatigue and myopathy. The patients are recruited in two waves, Wave 1 and Wave 2, with an interim analysis separating the two.

Forty (40) patients have now been enrolled (randomized). The interim analysis, on track for mid-2024, will include data from the Wave 1 patients’ first six months of dosing. This analysis will include a futility analysis as well as a sample size calculation for Wave 2 and will determine the final number of patients to be recruited into the full, potentially registrational, study.

“We are extremely pleased to have achieved our enrollment target for Wave 1 of the study. The rapid identification of patients by our study sites speaks to the high interest amongst patients in the FALCON study and confirms the substantial unmet medical need in primary mitochondrial disease. Our clinical sites across Europe and the US are extremely committed to the study and we all look forward to working together over the next six months to ensure delivery and quality data at the time of the interim analysis”, said Dag Nesse, VP Clinical Operations.