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Abliva’s mission to become mitochondrial medicine powerhouse

Abliva’s mission to become mitochondrial medicine powerhouse

Abliva’s mission to become mitochondrial medicine powerhouse

8 April, 2024

Abliva’s CMO Magnus Hansson attended the 8th annual meeting of Mitochondrial Medicine – Therapeutic Development in March. The conference brings together leaders in translational mitochondrial medicine to discuss recent advances in the area of mitochondrial research and clinical development. BioStock spoke with Hansson to better understand how scientific meetings like this one fit into the company’s overall mission.

The mitochondria, the ‘powerhouse of the cell’ are cellular organelles responsible for energy production inside the body. In rare instances, genetic mutations cause damage to the mitochondria causing them to stop working properly, leading to diseases called primary mitochondrial disease, or ‘mitochondrial disease’ for short.

Despite being rare – roughly 1 in 5,000 people suffer from mitochondrial disease – the consequences can be serious. Depending on the location and prevalence of the mutation, patients can experience a range of symptoms from cognitive issues, difficulties breathing, deafness, extreme fatigue and difficulties moving. There are currently no treatments available to patients with mitochondrial disease affecting multiple organ systems.

Abliva is developing a new medicine for patients with mitochondrial disease

Abliva, a biotech based in Lund, is in the business of developing therapies that restore mitochondrial function. The company has a broad range of pipeline projects in development. Its lead candidate is KL1333, a drug being evaluated in the phase II FALCON study for the treatment of mitochondrial DNA (mtDNA)-related mitochondrial disease in adult patients suffering from debilitating fatigue and muscle weakness.

Last December, Abliva achieved its enrolment target for Wave 1 of the study, and KL1333 dosing is ongoing. Interim data are expected in mid-2024. Read more.

The significance of attending scientific conferences

According to Abliva’s latest financial statement, the company’s long-term goal is “to become the leading global company focused on the discovery of therapeutics for mitochondrial disease.” Part of Abliva’s strategy to reach that goal is to attend scientific conferences aimed at bringing together experts, both academic and clinical, in the field of mitochondrial medicine.

The company recently attended the 8th annual meeting of Mitochondrial Medicine – Therapeutic Development, which took place from March 18 -20 in Hinxton, just outside Cambridge, UK. With a focus on mitochondrial disorders and the search for novel effective therapies for such disorders, the conference aims to create a ripe environment for forging collaborations between clinicians, laboratory scientists and the life sciences industry in this field.

This year’s programme highlighted recent discoveries on mitochondrial disorders and strategies to treat these, and included a variety of panel discussions, including a panel focused on rallying the international community to address the current opportunities and challenges in clinical trials. Read more about the meeting here.

CMO insights

Abliva’s CMO Magnus Hansson took part in one of the panel discussions at the meeting in Hinxton. BioStock reached out to him to learn more about the company’s mission to become a leader in the field of mitochondrial medicine, and how conferences like this can be part of the journey.

Magnus, we’d love to hear more about the recent meeting you attended and the value it brings to the company.

– It was a pleasure meeting several of the patient advocacy groups, investigators in our ongoing FALCON study, and researchers at the meeting. The theme of the conference – advancing new therapies for mitochondrial disease – is at the core of what we do at Abliva, and we had the opportunity to discuss our learnings from previous clinical trial experiences and how we have implemented these learnings into clinical trial designs to improve the evaluation of new therapies with the aim to increase the success rate in our field.

– Unlike in a large medical field such as oncology, our community is relatively small, and we all know each other. There is a great collaborative spirit among different stakeholders in the mito community, all working together to develop medicines, learning from all of the gathered experience in mitochondrial therapy research and development.

– Typically, we only have a few moderately sized international meetings every few years, for example the Euromit meeting in Bologna last year, and some smaller annual meetings, so we take every opportunity to reconnect at conferences like this. One reason we attend is to make sure we are aware of ongoing advancements in the area. The annual meetings hosted by Wellcome Genome Campus in Hinxton focus on therapeutic developments, and it is clear that Abliva is at the forefront in this area, both scientifically as well as how we approach the clinical development.

Magnus Hansson, CMO, Abliva
Magnus Hansson, CMO Abliva

What is some of the feedback you’ve received from other experts in the field regarding your development programme?

 You are on to something here. A significant benefit of these meetings is that they are good opportunities to meet up with investigators participating in our FALCON study. From those discussions, I learned that many patients really appreciate the focus we have on fatigue in our study and that this approach has been long awaited. There was also an enthusiasm for the fact that we have developed a primary endpoint for fatigue that is specific to mitochondrial disease – the PROMIS Fatigue Primary Mitochondrial Disease Short Form – that is currently in use in the FALCON study and is available to the mitochondrial disease community to use in their research and clinical trials.

– We also heard that our efforts in designing the trial to be as patient-friendly as possible (to facilitate recruitment and participation) was appreciated, and we received positive feedback from the investigators on the daily, operational aspects of the study.

What meetings are next on the agenda and how will they help Abliva deliver on your strategy to become a global leader?

– We have a busy schedule this spring, meeting with many of our key stakeholder groups. Over the next few months we are excited to introduce, or, in many cases update, investors, potential partners, investigators and patients on our strategy and the progress we’ve made with KL1333. Awareness of mitochondrial disease continues to grow, but we work hard to help spread the word and bring attention to this area of high unmet need.

– I will join our CEO, Ellen Donnelly, at Bio€quity in San Sebastián in May. We will also present the company at the Kempen Life Sciences Conference in Amsterdam this month and at BIO International – the biggest biotechnology conference of the year – in San Diego in early June. And we will of course join the BioStock Global Forum in Lund in late May where I will present our programmes.

– The next mito conference will be in late June when the Abliva team will attend the annual UMDF Mitochondrial Medicine Conference – the biggest US meeting on mitochondrial disease with a clear patient focus.

Abliva has quite an ambitious goal to become a leading company in mitochondrial therapeutics. What are you doing to make this a reality?

– The feedback from our interactions at the mitochondrial therapeutic development conference confirms that we are making good progress towards this goal. Our projects rest on a strong foundation of research and development in the area of mitochondrial dysfunction. Our CSO Eskil Elmér’s research related to mitochondrial biology began over three decades ago, and my own interests in mitochondria and therapeutic development began over two decades ago. We have used this knowledge to develop a pipeline of products, one of which, KL1333, is being evaluated in a clinical study that has been designed to support marketing approval of KL1333.

– As a ‘first-in-class’ medicine in a therapeutic area with no approved medicines, we’ve had to use all of our knowledge and experience, as well as the knowledge and experience of the mito community, physicians, investigators, scientists and patients,  to establish a strong development plan for the KL1333 programme. The fact that this development programme has resonated well with both the patient community, the regulatory authorities at our advice meetings, and with payers when interviewed, is another confirmation of our strong position in this area.

– Drug development is a complex process, and we are focused on a number of parallel tracks that are needed to bring new treatments forward. Biology is inherently difficult to predict with certainty, and that’s part of the challenge and opportunity in drug development. The upcoming interim analysis of our KL1333 FALCON trial will therefore be a key milestone to trigger the final stage of the KL1333 development programme.

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.

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