World Mitochondrial Disease Week aims to raise awareness of mitochondrial disease. This disease means that the cells' energy producers do not function properly, which leads to impaired function in the affected organs, which is often very disabling. The most common symptoms these patients experience are severe fatigue - which can be both mental and physical, as well as muscle weakness, and in some cases the disease leads to premature death. Despite the fact that 1 in 5000 people are affected by mitochondrial disease, it is still relatively unknown to the public.
Increased focus on mitochondrial diseases
In addition to the severe illness of affected patients, understanding of the disease has historically been low, leaving both patients and healthcare providers helpless. Although knowledge of mitochondrial disease has gradually increased, there are still no approved treatments for systemic mitochondrial disease.
This week therefore aims to increase awareness, attention and funding for effective treatments. This year's theme is "Illuminate Tomorrow: Revitalize your energy".
Abliva – a pioneering player
This is exactly what the Swedish biotechnology company Abliva wants to achieve with the development of its drug candidates. KL1333, which has orphan drug designation in both Europe and the US and Fast Track status in the US, and NV354, which has also received orphan drug designation in both Europe and the United States.
KL1333, the company's most advanced candidate, is currently being evaluated in the Phase II study FALCON. This summer, Abliva announced perhaps the most important milestone in the company's history with the positive interim analysis of 24-week data from 35 patients in the first part of the study. This analysis not only showed that KL1333 has a continued good safety profile, but also that the two alternative primary endpoints – fatigue and myopathy – had passed a futility test. This meant that both have the potential for a positive readout at the end of the 48-week study. According to Abliva, the results are very motivating, and an important risk reduction for the program. Read more here. From a patient perspective, this is hopeful as these symptoms are the most devastating and also worsen as the disease progresses. The study will include a total of 180 patients and the company aims to start the second and final wave of the FALCON study in the second half of 2024.
As a leading player in drug development in mitochondrial diseases, Abliva is participating in Mitochondrial disease week by sharing daily this week videos with highlights from the company's ongoing activities. However, the work continues year-round with the hope of bringing light to tomorrow by restoring these patients' energy.