Abliva Reports Positive Interim Results for FALCON Study on KL1333 in Primary Mitochondrial Disease

Abliva AB, a clinical-stage biopharmaceutical company listed on Nasdaq Stockholm, announced a favorable interim analysis outcome for their FALCON study, which evaluates the safety and efficacy of KL1333 in patients with primary mitochondrial disease. The FALCON study aims to assess two primary endpoints: fatigue and myopathy. The independent Data Monitoring Committee (DMC) reviewed the 24-week interim data and recommended continuing the study without modifications, confirming the strong safety profile of KL1333.

The DMC's interim review involved analyzing the conditional power of the two primary endpoints after 24 weeks of treatment. Both endpoints passed futility, leading the DMC to recommend continuing the study with a total of 180 patients, thus enhancing the probability of achieving a positive outcome upon the study's completion. Importantly, no safety concerns or drug-related serious adverse events were reported, further validating KL1333's safety profile.

Ellen K. Donnelly, Chief Executive Officer of Abliva, expressed her satisfaction with the interim analysis results. She highlighted that the positive outcome not only underscores the risk-benefit profile of KL1333 but also strengthens the company's confidence in the novel mechanism of action and the overall study design. Donnelly emphasized the high unmet medical need for treatments targeting mitochondrial diseases, noting that over 85,000 people in the US and Europe are affected by the condition. KL1333, she believes, holds the potential to meet this need and achieve significant market success in key regions.

The FALCON study is a Phase 2, global, randomized, placebo-controlled trial. It evaluates KL1333 in adult patients with primary mitochondrial disease who suffer from debilitating fatigue and myopathy. Patients meeting the eligibility criteria, specifically those with mitochondrial DNA mutations, are randomized in a 3:2 ratio to receive either KL1333 or a placebo. The treatment encompasses doses of 50mg-100mg administered twice daily over a period of 48 weeks. The primary endpoints are designed to measure chronic fatigue and muscle weakness, the most common and severe symptoms of mitochondrial disease, using the PROMIS Fatigue Mitochondrial Disease Short Form and the 30-second Sit-to-Stand test, respectively.

Primary mitochondrial disease is characterized by the cells' impaired ability to convert energy, with symptoms varying significantly depending on the affected organs and the extent of mitochondrial dysfunction. Often presenting in early childhood, the disease can lead to severe manifestations such as mental retardation, extreme fatigue, muscle weakness, heart issues, diabetes, movement disorders, stroke-like episodes, and epileptic seizures. It is estimated that 125 people per million are affected by this condition.

Abliva's lead candidate, KL1333, is designed for long-term oral treatment in genetically confirmed adult patients exhibiting chronic fatigue and myopathy due to primary mitochondrial disease. The drug targets conditions such as MELAS-MIDD, KSS-CPEO spectrum disorders, and MERRF syndrome. KL1333 works by restoring the balance of NAD+ and NADH, promoting the formation of new mitochondria and improving energy levels. Previous Phase 1a/b studies have shown that patients treated with KL1333 experienced improvements in both fatigue symptoms and functional abilities.

KL1333 is currently in a global Phase 2 study, the FALCON study, and has received orphan drug designations in the USA and Europe, as well as Fast Track designation in the USA. Abliva, based in Lund, Sweden, continues to focus on developing treatments for mitochondrial diseases, with KL1333 in late-stage development and NV354, an energy replacement therapy, having completed preclinical development.

How to obtain the latest research advancements in the field of biopharmaceuticals?

In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!