Swedish biotech company Abliva has successfully met the target number of patients for screening in the first wave of the global phase II FALCON study, demonstrating their commitment to maintaining the project’s timeline. BioStock spoke with VP Clinical Operations, Dag Nesse, about this achievement in the study.
Abliva is developing therapies for systemic mitochondrial diseases, an area where treatments are lacking today. Currently underway, the company’s FALCON is a global phase II trial, employing randomisation, a placebo-control, and potentially registrational design. It aims to assess the effectiveness of the main candidate KL1333 in adult mitochondrial disease patients experiencing fatigue and myopathy.
Achieves important milestone
In June this year, Abliva initiated dosing in patients in the FALCON study. Last week, they announced that the screening goals for the first wave in the study had been achieved and that the evaluation process for dosing initiation is ongoing, thus maintaining the study’s timeline. This underscores the company’s dedication to adhering to the project’s schedule, and Abliva is steadfast in its goal to have all eligible patients commence dosing by the close of 2023.
During the screening phase of the FALCON study, patients meeting the initial criteria undergo an assessment to confirm their genetic background and to establish a baseline for consistent fatigue and myopathy. Dosing begins at the conclusion of this screening period for those who meet the necessary criteria.
The FALCON trial utilises an adaptive study design, including an interim analysis that will dictate the ultimate patient enrolment for the complete study. The total enrolment will be made in two waves, with the intention to compile a dataset that will support registration.
Vice President of Clinical Operations tells us more
BioStock spoke with Abliva’s VP Clinical Operations, Dag Nesse, to learn more about the achievement.
Could you elaborate on the significance of meeting the screening goal for wave 1 in relation to the overall success of the study?
– Reaching the target number of patients required for screening in wave 1 of the study is a very important milestone as it makes us confident that we remain on track to initiate dosing in all patients by the end of the year, and for the interim analysis in the middle of next year.
– We have also been happy to see the significant interest by patients to participate in the study and are thankful for the work done by the clinical sites to get this large number of patients screened and further evaluated.
What will affect the size and design of wave 2 and when will you start that wave?
– A great advantage with the study’s adaptive design is that we can analyse data from a smaller group of patients in an interim analysis, performed by an external committee. From that, we get an indication on how many more patients we need to include to increase our chances of getting a reliable and statistically significant result once the study is concluded.
– In the study, we have two primary endpoints, fatigue and muscle weakness, and thus two opportunities for us to have a successful study result. In the interim analysis, the external committee will evaluate data for both of these endpoints and give us an indication on the required size of the full study, between 120 and 180 patients, based on their findings.
– The interim analysis is planned to be conducted towards the middle of 2024, and we are currently preparing sites and documentation for Wave 2.
What are the key takeaways from the patient interest and commitment displayed in this study?
– Primary mitochondrial disease has a significant unmet medical need as there are no treatments for systemic mitochondrial disease. In the planning of this study, we have been working closely with patient advocacy organisations in the US and Europe.
– The feedback from these groups is that patients are eagerly awaiting a treatment that can help with their fatigue and myopathy. This was confirmed by fact that we were quickly able to screen over 90 patients in a rare disease study, and is a testament to the patient’s interest in a new medicine for mitochondrial disease.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.