Abliva, headquartered in Lund, is a biotech company specialising in the development of treatments for rare and severe mitochondrial diseases. The company’s leading candidate, KL1333, is undergoing a global, randomised, placebo-controlled phase II study, FALCON. The study aims to evaluate the safety and efficacy of KL1333 in treating primary mitochondrial disease—a condition for which there are currently no available treatments.

The FDA recently granted Fast Track designation to KL1333, highlighting the urgent need for effective treatments for this patient group. Learn more.

World Mitochondrial Disease Week raises awareness

From September 18th to 24th, World Mitochondrial Disease Week raises global awareness about mitochondrial diseases, through educational, fundraising, and advocacy activities. This year, the focus is on fatigue, a common symptom affecting patients affected by more than 350 genetic mutations that fall under the umbrella of mitochondrial diseases.

Fatigue is not just physical tiredness; it’s a complex symptom that can also manifest as mental exhaustion and diminished endurance. Despite its prevalence, the etiology of fatigue remains poorly understood, leading to its underdiagnosis and poor management.

Patient-centric approach

Abliva has gone to great lengths to understand the experiences of patients with mitochondrial diseases. In collaboration with patient organisations, the company has conducted extensive research to select relevant outcome measures for its FALCON study.

In 2021, Abliva carried out a large interview study in partnership with the United Mitochondrial Disease Foundation in the United States. The study has provided Abliva with valuable insights into the challenges faced by patients, particularly the debilitating symptom of chronic fatigue.

Addressing chronic fatigue

Fatigue is a persistent and debilitating condition for many mitochondrial disease patients and this fatigue affects the patient both physically and mentally. Not only is a fatigue common amongst the patient group, fatigue worsens as the disease progresses and is the number one symptom that patients wish the pharmaceutical industry would address.

BioStock contacted CMO Magnus Hansson to learn more about the company’s work in close cooperation with patient organisations.

Why is Mitochondrial Disease Week important?

– The week is important to increase awareness and understanding of the disease. Individuals and local patient advocacy groups across the world are engaged in spreading knowledge about the disease through advocacy and fundraising activities. It is a good opportunity for the entire community to work together on activities that directly and indirectly help patients and show an outpouring of support for the patients.

What do you think about this year’s theme, fatigue?

– This year’s focus on fatigue is very well aligned with Abliva’s work to develop treatments that address the symptoms that are most important to the patient. Insufficient mitochondrial energy production is directly felt by the patient, impacting their ability to do normal daily activities or even think clearly. Our aim is to improve this very important quality of life aspect by boosting mitochondrial energy production through the administration of KL1333.

Abliva is known to work closely with patient organisations in the development. Could you elaborate a bit on how you are working together with them?

– Our ongoing collaborations with patient organisations across the world have been instrumental in helping us design a development program that addresses the patients’ chief concerns in a ‘patient-friendly’ form. Patient input in the early days confirmed the importance of fatigue. Patients later provided input into the development of our mitochondrial-disease specific fatigue questionnaire, and then, more recently, they reviewed our clinical protocol to ensure that the endpoints were not too burdensome for a mitochondrial disease patient.

– The patient organisations have also been instrumental in helping the regulatory authorities better understand the disease and the complications. Their participation in our meeting with the FDA, as an example, facilitated our interactions with the regulators and helped us align on the FALCON study design. Finally, we are currently working with several patient organisations to spread the word about our ongoing study and help bring patients to our study.    

Returning to Mitochondrial Disease Week, how will Abliva work to promote disease awareness during this week?  

– During the pandemic we shifted the format of our participation in World Mitochondrial Disease Week from a physical meeting to a video project and the feedback was fantastic as our reach was much broader and people from across the globe could watch our videos at their convenience.

– Over the course of the week, we will publish seven videos focused on mitochondrial disease, the patients, and our own efforts to treat the disease. This year, a large part of our team has participated in recorded interviews and presentations with a focus on different aspects of our FALCON study.

– We have also included an interview from last year with a patient, telling her story about how she was diagnosed and her experiences with different symptoms. The videos will be accessible at abliva.com and on our YouTube channel. You can also follow us on LinkedIn to get live updates!

Read more about Abliva on the company’s website.