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 company has now included the target number of patients required for screening in Wave 1 of the FALCON study. The study therefore remains on track to have all Wave 1 patients commence dosing by the end of the year with the interim analysis towards the middle of 2024.
Abliva is currently running the FALCON study, a global, randomized, placebo-controlled, and potentially registrational clinical Phase 2 study evaluating the efficacy of KL1333 in adult mitochondrial disease patients experiencing fatigue and myopathy.
The target number of patients required for screening in Wave 1 of the study has now been met. Screened patients have been, and will continue to be, evaluated for initiation of dosing in the study, with the goal that all eligible patients will have initiated dosing by the end of 2023. In the FALCON study, patients who meet the initial criteria are evaluated during a screening period, during which time their genetic background and a baseline of consistent fatigue and myopathy is confirmed. Patients that satisfy the necessary criteria commence dosing at the end of their screening period.
“The fact that we were able to quickly identify over 90 rare disease patients for screening in this study, with an intention to dose at least 40, speaks to both the commitment of the sites to this study and to the significant interest by patients to participate in a study that offers a drug that may address both their fatigue and their myopathy”, said Dag Nesse, VP Clinical Operations.
The FALCON trial has an adaptive study design with an interim analysis that will determine the final number of patients to be recruited in the full study (Wave 1+ Wave 2) to support a potentially registrational dataset.
For more information, please contact:
Catharina Johansson, Deputy CEO, CFO & VP Investor Relations
+46 (0)46-275 62 21, [email protected]
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About the FALCON study
The FALCON study is a global, randomized, placebo-controlled, potentially registrational,
clinical Phase 2 study with KL1333. Through the study, the company evaluates the safety and
efficacy of KL1333 on primary mitochondrial disease in adult patients with mitochondrial DNA
mutations, with a focus on chronic fatigue and muscle weakness, which are the most common
and debilitating disease expressions in these patients. The company will recruit 120 – 180
patients, in two waves, who will be given KL1333 or placebo twice daily for 12 months. An
interim analysis will take place after the completion of Wave 1 and will give important statistical
information on safety and powering in Wave 2.