From mutation to solution – Abliva gains ground
Abliva continues to solidify its position as a leader in developing treatments for mitochondrial disease. During Q3 2024, the biotech company reached a milestone with the positive interim analysis of its FALCON trial, evaluating the drug candidate KL1333 as a potential treatment for primary mitochondrial disease.
Based in Lund, Abliva is dedicated to improving the lives of patients with primary mitochondrial disease. These diseases impair the ability of cells to produce energy, leading to debilitating symptoms such as fatigue and muscle weakness. Mitochondrial disease profoundly impacts patients, severely diminishing their quality of life and, in some cases, becoming life-threatening. Currently, no approved treatments exist for systemic mitochondrial diseases, presenting Abliva’s leading drug candidate, KL1333, with a significant opportunity in this underserved area.
Positive interim results drive momentum for KL1333
Abliva made notable progress in Q3 2024 with the interim results from its phase II FALCON study evaluating KL1333. The FALCON study has been designed as a pivotal trial, meaning that the data, if positive, should be sufficient to support a marketing approval application based on the two primary endpoints, with just one endpoint required for regulatory registration. The study targets adult patients with multisystemic mitochondrial disease caused by mutations in mitochondrial DNA (mtDNA). These patients often experience severe and persistent fatigue as well as muscle weakness, which are the trial’s two primary endpoints. The interim analysis reviewed data from patients who had received treatment for at least 24 weeks. The results demonstrated that both primary endpoints met the thresholds for futility — a critical indicator for continued development.
The independent Data Monitoring Committee also confirmed the safety profile of KL1333 and approved the expansion of the study to a total of 180 patients. The company reports a robust safety profile even after long-term treatment, with no drug-related serious adverse events yet recorded.
The next step will be to secure funding and prepare for the second and final stage of the study, involving approximately 40 clinical sites across 10 countries. This progress is particularly noteworthy in a field where other players, such as Reneo and Astellas, recently have faced setbacks and exited the space.
Focused on long-term success
Armed with the interim results, Abliva has not only reinforced its position but also attracted growing interest from strategic partners and investors. KL1333 is the most advanced program targeting patients with mtDNA mutations — a group estimated to comprise approximately 80 per cent of the adult population with mitochondrial diseases. The company estimates a market potential exceeding USD 1 billion in peak sales and has built an infrastructure to reach patients through specialised medical centers.
Advancing toward market approval
Abliva is now exploring multiple pathways to bring KL1333 to market and make the treatment available for patients. Strategic discussions are progressing alongside its patient-centered scientific advancements, with the company reporting strong ongoing interest in its work from researchers, clinicians, and investors. Thus, Abliva is driving progress in a field that has long lacked effective solutions.
Financial overview
For the third quarter of 2024, Abliva reported an operating loss of SEK -19.9 million (-32.9) and cash flow from operating activities of SEK -37.1 million (-4.9). Cash and cash equivalents totalled SEK 63.8 million (58.6) including short term receivables. The company is actively preparing for the second and final stage of the FALCON study, which is set to commence pending secured funding.
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