Abliva’s CEO: “One step closer to a marketed medicine”
Abliva has reported positive interim analysis results for the potentially registrational FALCON study, assessing KL1333 in patients with primary mitochondrial disease. While validating the study design and confirming KL1333’s strong safety profile, it also triggers a convertible loan conversion, providing Abliva with proceeds of 42 million SEK before transaction costs.
– The interim analysis is an important de-risking event for the program, says Ellen K. Donnelly, CEO of Abliva.
Abliva is developing therapies with the goal of improving mitochondrial function. The lead candidate KL1333 is a drug candidate being evaluated in the phase II FALCON study for the treatment of mitochondrial DNA (mtDNA)-related mitochondrial disease in adult patients suffering from debilitating fatigue and muscle weakness.
Positive interim analysis
Abliva achieved full enrollment in the Wave 1 part of the FALCON trial in Q4 2023, and on May 30th 2024, the last patient had completed its 24-week visit. The Independent Data Monitoring Committee (DMC) has now reviewed the 24-week interim data from the Wave 1 patient cohort, in which the independent group reviewed a pre-specified analysis of the conditional power of the two primary endpoints after 24 weeks of dosing.
Both endpoints passed futility, and the DMC recommended including a total of 180 patients, increasing the probability of a positive readout upon completion of the full study. The DMC concluded that KL1333 has a strong safety profile and that no safety concerns or drug-related serious adverse events (SAEs) were seen after 24 weeks of dosing.
Abliva aims to start the final wave of FALCON study in the second half of 2024.
Abliva adds 39 million SEK to its coffers
Meanwhile, Abliva has also announced that the convertible loan from the capital raise earlier this year will be converted into company shares. This conversion, prompted by the positive interim analysis outcome, will provide Abliva with an additional 42 million SEK in proceeds before transaction costs of approximately 2.9 million SEK.
Comments from the CEO
To learn more about the significance of the positive interim analysis results, BioStock reached out to Abliva’s CEO Ellen K. Donnelly.
Could you begin by discussing the positive interim analysis results and the significance of the 24-week interim data for KL1333?
– The interim readout is great news as it not only confirms that the program has passed futility, but it also provides an important de-risking step for the program as we now have more information regarding the potential efficacy of the two endpoints and the safety profile of KL1333. When we started the FALCON study we had only dosed 6 patients with KL1333 (2 on placebo) for 10 days with 50 mg KL1333/day. The interim analysis reviewed data from 35 patients dosed with 50 – 100mg/ day for 24 weeks. So knowing that KL1333 continues to have a good safety profile at a higher dose over six months of treatment is very important. To know that both of our primary endpoints, fatigue and myopathy, are still ‘in play’ is very encouraging and takes us one step closer to the market.
Can you elaborate on the independent primary endpoints in the FALCON study?
– Our study focuses on the two symptoms that patients with primary mitochondrial disease cite as the most debilitating – fatigue and myopathy. We are evaluating fatigue with a mitochondrial-disease-specific patient-reported questionnaire that we developed with patients, and myopathy with a simple test where you count the number of times you can stand up and sit down from a chair in 30 seconds (30 Second sit-to-stand test). The FALCON study is designed so that only one of these independent (alternative) endpoints needs to show a statistically significant improvement in the patients for the study to be positive. And now, after this positive interim readout, we are even more confident with this primary endpoint design, knowing that we still have two chances to be successful.
How does KL1333’s novel mechanism of action contribute to its potential success?
– KL1333 specifically targets an underlying cause of mitochondrial disease – a decreased NAD+/NADH ratio, crucial for energy conversion in the mitochondria. By converting NADH to NAD+, KL1333 restores the ratio and has the potential to normalize cellular energy production.
Can you explain the expedited path to market for KL1333?
– The regulatory agency in the US that approves new medicines, the Food & Drug Administration (FDA), has a number of programs that facilitate the development of medicines for rare diseases with high unmet medical need. One example is the requirement for only one (versus two) late-stage study for approval. Another example is Fast Track Designation, a program that helps accelerate the development and review of programs with serious conditions with a high unmet need. We have already taken advantage of the opportunity to meet with the FDA frequently to discuss key aspects of the program, and we look forward to taking advantage of the rolling review of our new drug application when, if the study is successful, we seek approval of our medicine upon completion of this study.
Finally, the positive interim analysis has triggered a convertible loan conversion, providing additional proceeds of 42 million SEK before transaction costs. How will the money be used?
– The convertible loan conversion completes the financing round of SEK 80M announced earlier this year. The proceeds from this conversion will support completion of the first wave of the study, continue the preparations of the new countries for the final wave of the study, and elongate our runway, providing us time now after the positive interim readout to continue discussions with investors and potential partners.
The content of BioStock’s news and analyses is independent but the work of BioStock is to a certain degree financed by life science companies. The above article concerns a company from which BioStock has received financing.
