Our strategy
Abliva is focused on becoming the leading company in mitochondrial medicine, developing therapeutics for mitochondrial disease, orphan indications of high unmet medical need. The company intends to build a fully integrated research, development, and commercial organization, developing innovative therapeutics and taking them directly to the patients.
Addressing Primary Mitochondrial Diseases
Mitochondria function as the powerhouses of our cells and are crucial for the cells’ energy metabolism. Primary mitochondrial disease is a rare orphan disease where the energy metabolism in the cells is impaired, causing deterioration that leads to multifaceted disorders and great suffering for patients. The symptoms worsen over time and, in many cases, the disease leads to premature mortality. Mitochondrial medicine has become an area of ever increasing focus for the pharmaceutical industry as there are currently no effective treatment options. Through Abliva’s research and development, we have an opportunity to improve the health and quality of life of these patients.
Delivering a Portfolio of First-in-Class Therapies
Abliva’s in-house R&D capabilities have been instrumental in creating and delivering a portfolio that includes several projects with mechanisms of action suitable for a wide range of different types of mitochondrial disease.
KL1333 restores the balance of the coenzymes NAD+ and NADH, creating new mitochondria and improved energy levels. KL1333 has completed a number of key Phase 1 studies that enabled the start of a potentially registrational Phase 2 study in 2022. KL1333 is protected by both a composition of matter patent as well as Orphan Drug Designation (ODD) in the U.S. and in Europe. The commercial opportunity is significant with even conservative estimates exceeding $1B/year in annual sales1).
NV354, an energy replacement therapy, is a pro-drug of succinate. The drug candidate was invented by Abliva scientists at Lund University and is supported by a strong group of patents as well as ODD in the U.S. NV354 is being developed for the mitochondrial disease Leigh syndrome initially with potential to expand to other indications that have a dysfunctional complex I in the electron transport chain.
Further, Abliva has efforts ongoing to identify additional portfolio opportunities focused on the regulation and stabilization of cellular energy production.
Leveraging Opportunities in Rare Diseases
Abliva is continually working to take advantage of the opportunities afforded to companies working in the rare disease space. The company requested and was granted, orphan drug designation (ODD) for both KL1333 and NV354. ODD is a regulatory designation that provides sponsors with several advantages including more regulatory assistance and scientific advice during the development process, lower development costs, attractive pricing, and market exclusivity (10 years in the EU and 7 years in the US). The outlook for reaching the market is also better than for traditional medicines2,3).
In addition, we have sought scientific advice for KL1333 from pharmaceutical regulators across the U.S., U.K., and Europe. This advice has been extremely important to the company, as is clearly demonstrated with the advice from the FDA that led us to move to a single, potentially registrational Phase 2 study, allowing us to get to market more quickly. We have also received valuable and positive feedback from the U.K. regulatory agency on our NV354 program, validating its potential to move into studies in humans.
1) Gorman et al., Prevalence of Nuclear and Mitochondrial DNA Mutations Related to Adult Mitochondrial Disease, 2015.
2) Jayasundra et al. Orphanet J of Rare Dis. Estimating the clinical cost of drug development for orphan versus non-orphan drugs. 2019.
3) EvaluatePharma, Orphan Drug Report 2019.