First patient dosed in Abliva’s Falcon study
In December Abliva started its phase II study to evaluate KL1333 in patients with primary mitochondrial disease. Earlier this week the company announced that the first patient in the study has been dosed. BioStock contacted Abliva CEO Ellen K. Donnelly to ask what this milestone means for the company.
Lund-based Abliva specialises in developing therapies for patients affected by mitochondrial diseases. Currently, there are no available treatments for systemic mitochondrial disease, underscoring the importance of the company´s efforts in this field.
Abliva’s development pipeline consists of drug candidates designed to modulate mitochondrial function and restore cellular energy production. Last December, the company embarked on a potentially registrational phase II study with its main candidate, KL1333.
First patient dosed
The Falcon study is a global, phase II, placebo-controlled, and potentially pivotal clinical trial. It targets adult patients suffering from chronic fatigue and muscle weakness due to mitochondrial disease. Dr. Rita Horvath and her team at the Department of Clinical Neurosciences, Addenbrooke’s Hospital in Cambridge, UK, have announced that the first patient has now been dosed in the study.
The initial phase will involve the recruitment of approximately 40 patients to contribute to an interim analysis slated for the first half of 2024. This interim analysis will be a blinded analysis conducted by an independent data monitoring committee. The review will assess the program for futility and will provide guidance as to the final size of the study.
Abliva has also confirmed that all countries involved in the first wave of the study – namely the US, UK, France, Spain, Belgium, and Denmark – have approved the study. Patients will be recruited from over 15 sites across these six nations.
Comments from the CEO
BioStock talked to Abliva CEO Ellen K. Donnelly to learn more about what the latest news means for the company.
The first patient was recently dosed in your Falcon study. What is your comment to Abliva reaching this important milestone?
– The first patient dosed in a study is an important milestone for patients, physicians, and the company. This first patient is not just the ‘first patient dosed’, but your friend/parent/sibling who is leading the way for many patients behind them, trusting us, believing in our medicine and hoping that it will make them feel better. Patients make a huge commitment when they enter a clinical study, and we appreciate their willingness to help test new medicines with the hope they will soon have approved therapies to address their disease.
– This milestone marks the culmination of ‘study start up’, an operationally intense period in a study when the team is focused on document review, contracting, user testing of software, regulatory and ethics discussions. Each country and site in this study has a different set of requirements and the team has been skilfully addressing each requirement to ensure that everything is in place to support this milestone. With all of our wave 1 countries active, patients will begin to flow into the study with a goal to have the first group recruited by the end of the year.
You have chosen a patient-focused design for the trial. Can you tell us more about that?
– We make medicines for patients. They sit at the forefront of everything we do. This study has a number of design elements that were included due to patient feedback. First, in early patient interviews we heard, consistently, that the symptom that bothered them the most was fatigue as it greatly impacted their daily life (a fact that is reiterated by several studies in different countries.
– Given the patient focus on fatigue, we conducted a study to validate a new fatigue endpoint for mito disease, the PROMIS Fatigue Mitochondrial Disease Short Form, and worked with the FDA to get their backing on the new endpoint. We have received feedback from patients and physicians that they have been waiting for a study focused on fatigue, so hopefully the time spent validating this new endpoint will pay off with faster recruitment and a positive outcome for the patients.
– We have also incorporated several things in the study to make it easier for the patients to participate. For example, we have chosen a myopathy endpoint, the 30 second Sit to Stand test, that can be done at home, eliminating many visits to the hospital. And, on the rare occasion when the patients do need to visit the site, we have travel coordinators that help them book the tickets and arrange the travel.
What are you hoping to see in the interim analysis of this initial phase of the study?
– Once all wave 1 patients have completed six months of treatment there will be a blinded analysis of the data. This analysis will first evaluate the study for futility, which means that the study will not continue if it is unlikely to show a significant effect in the KL1333 patients versus the patients treated with placebo.
– The committee will also look at the power of the two endpoints and will determine, based on the interim data, the final size for the study. Given that this is a rare disease where there is a lot of work required to find and recruit patients, the interim analysis is important both for the patients, providing them opportunity to join promising studies, and for our successful conclusion of the study.
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