Abliva

Abliva develops medicines for the treatment of primary mitochondrial diseases. These congenital, rare and often very severe diseases occur when the cell's energy provider, the mitochondria, do not function properly. The portfolio includes projects at different stages and ranges from early discovery phase to clinical phase.

Our strategy

Abliva is focused on becoming the leading biopharmaceutical company in mitochondrial medicine, developing therapeutics for primary mitochondrial diseases, orphan indications of high unmet medical need. The company will build a fully integrated research, development, and commercial organization, developing innovative therapeutics and taking them directly to the patients.

Addressing Primary Mitochondrial Diseases (PMD)

Mitochondria function as the powerhouses of our cells and are crucial for the cells’ energy metabolism. PMD are rare orphan diseases 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 diseases lead to premature mortality. Mitochondrial medicine has become an area of increasing focus for the pharmaceutical industry as there are currently no effective treatment options for patients. Through Abliva’s research and development, we have an opportunity to improve the quality of life for 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 PMDs.

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 to prepare the asset for registrational Phase 2/3 study start in late 2021. 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 sales. 

NV354, an energy replacement therapy, is a pro-drug of succinate. The drug was invented in the Abliva laboratories at Lund University and is supported by a strong group of patents. NV354 is being developed for the PMD Leigh Syndrome initially with potential to expand to other PMD indications that have a dysfunctional complex I in the electron transport chain. 

Additionally, Abliva has additional efforts ongoing in discovery that are focused on the regulation and stabilization of the mitochondrion’s 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 KL1333 in both the US and EU. ODD is a regulatory designation that provides sponsors with a number of 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 medicines1,2.

In addition, the company has sought advice from pharmaceutical regulators in the US, UK and Europe. This advice has been extremely important, as is clearly demonstrated with the 2020 advice from the FDA that led the company to move to a single, registrational Phase 2/3 study (versus the traditional sequential Phase 2 followed by Phase 3 design), allowing Abliva to get to market more quickly. 

1) Jayasundra et al. Orphanet J of Rare Dis. Estimating the clinical cost of drug development for orphan versus non-orphan drugs. 2019.
2) EvaluatePharma, Orphan Drug Report 2019.