Abliva reaches enrollment goal
For biotech Abliva, the main focus for 2023 has been the FALCON study, a potentially registrational, global, phase II study with lead candidate KL1333. In June, Abliva began dosing patients, and yesterday the company announced that the goal of enrolling 40 patients for Wave 1 has been met. BioStock spoke to Dag Nesse, Vice President of Clinical Operations, to learn more.
Lund-based Abliva is on a mission to improve the lives of patients suffering from primary mitochondrial disease. The disease disturbs cellular energy conversion and causes severe symptoms such as stroke-like episodes, muscle weakness, and heart failure. Primary mitochondrial disease has a profound effect on patients as it severely lowers the quality of life and can be fatal. There are no approved therapies for the treatment of systemic mitochondrial disease, and the competitive landscape is thus limited, providing opportunity for Abliva’s lead candidate KL1333.
Phase II study underway
Abliva has successfully taken KL1333 into a global – and potentially registrational – phase II study called FALCON, which is currently underway and active in six countries. The study objective is to evaluate the efficacy of KL1333 in adult patients with mitochondrial disease who experience fatigue and muscle weakness. Patients will be recruited and dosed in two waves with an interim analysis separating the two waves. The first wave includes 40 patients who will receive KL1333 or placebo twice daily for 48 weeks.
Hitting another milestone
During the year, Abliva has announced several study related milestones, starting with the first patient dosed in June and meeting the target number of patients for screening in Wave 1. Yesterday, roughly a year after the first site was initiated, Abliva announced that the goal of enrolling 40 patients for Wave 1 has been met.
This means that Abliva has fulfilled its communicated timeline goal for the study, despite the challenges inherent in rare disease development and the harsh market climate.
VP of Clinical Operations comments
BioStock reached out to Dag Nesse, Abliva’s Vice President of Clinical Operations, to find out more about this latest milestone in the phase II study.
Dag, can you start by telling us more about what implications this news has for the FALCON study?
– We are extremely proud to have met this milestone as we have set a high bar for recruitment (confirmation of genetic background with predefined levels of both myopathy and fatigue) to ensure a homogeneous population of patients. Having never recruited this patient population before, it was unclear how difficult it would be to find these patients. In addition, we have a screening period in the study of 8-12 weeks where the patients are evaluated and, at the end, many did not satisfy the criteria. Thus, we are very happy that we completed the recruitment in this challenging rare disease area on time.
Please elaborate on the overall design of the FALCON-study and how the two waves relate to each other.
– That is a great question. The FALCON Wave 1 patients are now receiving KL1333 or placebo twice daily for a total of 48 weeks. When all Wave 1 patients have been dosed for their first six months of the study, there will be an interim analysis. At the end of the full study, the data from the Wave 1 patients and the data from the Wave 2 patients will be combined and evaluated to see if KL1333 had a statistically significant effect on muscle weakness or fatigue.
– The Wave 1 patients, currently in the dosing stage, will later be joined by additional patients from Wave 2, which will start after the interim analysis this coming summer. The interim analysis has a futility readout as well as a sample size adjustment which will tell us how many more patients are required to be enrolled in the study. We expect to enroll between 120 and 180 patients in the full study.
With the enrollment goal for Wave 1 achieved, could you be more detailed on the timeline for the readout of Wave 1 results?
– The interim analysis is still on track for summer 2024. I would like to highlight, though, that the data available to all of us at the interim will be quite slim as the interim analysis is a blinded analysis conducted by an independent data monitoring committee. When we receive notice of the outcome, we will only know one thing – the final number of patients to be recruited into the study (or that the study was futile).
– This independent, blinded assessment is required due to the pivotal nature of the study. Once we have the final number of patients, we will start recruiting patients for Wave 2 and the final chapter of the study.
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