-Independent committee confirms FALCON study is powered for potential success with both alternative endpoints passing futility-

-Committee confirms strong safety profile of KL1333-
 
-Abliva to host conference call and webcast on Friday 19 July at 4:30 pm CET / 10:30 am ET–
 
Lund, Sweden, July 18, 2024 — Abliva AB (Nasdaq Stockholm: ABLI), a clinical-stage company developing drugs for the treatment of rare and severe primary mitochondrial disease, today announced a positive outcome of the interim analysis for FALCON, the potentially registrational study evaluating KL1333 in patients with primary mitochondrial disease. The study evaluates fatigue and myopathy as alternative independent primary endpoints, only one of which is required for a successful study readout. The independent Data Monitoring Committee (DMC) recommended continuing the study without modification. The favorable recommendation by the DMC to continue the FALCON study with a total of 180 patients validates the overall study design and confirms the strong safety profile of KL1333.
 
The DMC has reviewed the 24-week interim data from the Wave 1 patient cohort. In their analysis, the independent group conducted 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 to include a total of 180 patients, increasing the probability of a positive readout upon completion of the full study. The DMC emphasized that KL1333 has a strong safety profile. No safety concerns or drug-related serious adverse events (SAEs) were seen after 24 weeks of dosing. 
 
The interim analysis was conducted by an independent committee to maintain the rigor of a blinded study. Statistical significance across the two primary endpoints will be evaluated at the conclusion of the full study. 
 
“We are pleased with the positive outcome of the interim analysis as it highlights the positive risk-benefit of KL1333, de-risking the full study, increasing our confidence in KL1333’s novel mechanism of action and the FALCON study design” said Ellen K. Donnelly, Chief Executive Officer of Abliva. “There are currently no approved therapies for mitochondrial disease and with over 85,000 people affected across the US and Europe, we believe KL1333 is well-positioned to address this high unmet medical need and reach blockbuster potential in key geographies. KL1333 has the potential for an expedited path to market and we are committed to rapidly advancing this important medicine towards approval, with plans to start the final wave of FALCON study recruitment in the second half of 2024.” 
 
Conference call
Abliva will host an investor call on Friday, July 19, 2024, at 10:30 AM ET / 4:30 PM CET, to discuss the positive outcome of the interim analysis for FALCON. A live question and answer session will follow the formal presentation. To register, click here.

If you would like to ask a question during the live Q&A, please submit your request to questions@lifesciadvisors.com.