Abliva leads the fight against mitochondrial disease
This week, mitochondrial disease is in focus during Mitochondrial Disease Week, highlighting a condition that, despite its often severe impact, remains relatively unknown. While research has progressed, there are still no approved treatments. Swedish biotech Abliva is at the forefront of changing this, with its phase II study of drug candidate KL1333 recently reaching a significant milestone.
World Mitochondrial Disease Week aims to raise awareness of mitochondrial disease. The disease occurs when the cells’ energy producers don’t function properly, resulting in impaired function in affected organs, often leading to severe disabilities. The most prominent symptoms experienced by these patients are fatigue — both mental and physical — as well as muscle weakness, and in some cases, the disease leads to premature death. Despite affecting 1 in 5,000 people, mitochondrial disease is still relatively unknown to the public.
Increased focus on mitochondrial disease
In addition to the severity of the illness, understanding of mitochondrial disease has historically been limited, leaving both patients and healthcare providers at a loss. While knowledge of the condition has gradually improved, there are still no approved treatments for systemic mitochondrial disease.
This week’s goal is thus to raise awareness, increase visibility, and fund the development of effective treatments. This year, the theme is “Illuminate Tomorrow: Revitalise your energy”.
Abliva – a pioneering force
This is precisely what Swedish biotech Abliva is striving to achieve through the development of its drug candidates KL1333, which has orphan drug designation (ODD) in both Europe and the U.S., as well as Fast Track status in the U.S., and NV354, which also has obtained ODD in Europe and the US.
KL1333, the company’s most advanced candidate, is currently being evaluated in the phase II study FALCON. This summer, Abliva announced what may be the most important milestone in the company’s history – the positive interim analysis of 24-week data from 35 patients in the first part of the study.
This analysis not only confirmed that KL1333 continues to demonstrate a good safety profile, but it also showed promising results for the two alternative primary endpoints, fatigue and myopathy. Both endpoints passed a futility test, meaning that both have the potential to readout positive at the end of the 48 week study. According to Abliva, the results are highly encouraging and represent a significant de-risking of the program. Read more here.
From a patient perspective, this brings hope as these symptoms are the most devastating and worsen as the disease progresses. In total, the study will include 180 patients, and the company aims to begin the second and final wave of the FALCON study in the second half of 2024.
As a leading player in the development of treatments for mitochondrial diseases, Abliva is participating in Mitochondrial Disease Week by sharing daily videos highlighting the company’s ongoing activities. However, their work continues year-round, with the hope of illuminating tomorrow by revitalising the energy of these patients.
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.
