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 first patient has been dosed in the company's global Phase 2 clinical study, the FALCON study. The study will evaluate the effects of Abliva's lead drug candidate KL1333 in patients with primary mitochondrial disease.
The FALCON study, a global, Phase 2, placebo-controlled and potentially pivotal clinical study is evaluating the effects of the company’s lead drug candidate, KL1333, in adult patients with mitochondrial disease suffering from chronic fatigue and muscle weakness.
Today, Dr. Rita Horvath and her team at the Department of Clinical Neurosciences, Addenbrooke's Hospital in Cambridge, UK, announced that the first patient has been dosed in the FALCON study. The study will now continue to the interim analysis which will include data from approximately 40 patients treated for up to six months. The interim analysis, a blinded analysis that will be conducted by an independent data monitoring committee (IDMC), will review the data for futility and will also provide a sample size estimation, based on the power of the two primary endpoints, that will inform the final size of the study. This informative interim analysis is expected in the first half of 2024.
The company also announced that all countries in the first wave of the study (US, UK, France, Spain, Belgium, and Denmark) have approved the study, and patients will be recruited from over 15 sites across these six countries.
“This is an important milestone for the company and for mitochondrial disease patients who suffer daily from a debilitating disease with no approved medicines. We are thankful for the strong participation from our sites and our investigators in identifying patients and we remain on track to enroll Wave 1 of the study this year with the interim analysis next year”, said Ellen K. Donnelly, CEO.