Abliva develops medicines for the treatment of mitochondrial disease. This congenital, rare and often very severe disease occurs 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.

Strategic Focus: Mitochondrial Disease

At Abliva, we are focused on becoming the leading company in mitochondrial medicine, developing therapeutics for mitochondrial disease, orphan indications of high unmet medical need. We intend to build a fully integrated research, development, and commercial organization, developing innovative therapeutics and taking them directly to the patients.

Building the Premier Mitochondrial Medicine Company

Abliva’s long-term goal is to become the leading global company focused on the discovery of therapeutics for mitochondrial disease. We will do this with our clear strategy, strong portfolio of assets, research and development organization, and team with decades of experience in mitochondrial medicine and drug development.

Over the next few years, we will focus on the delivery of our portfolio to the market. We aim to:

  • Augment our strong research and development capabilities and build a commercial organization.
  • Bring new innovative therapeutics to the patients and fuel our pipeline with new candidates from discovery.
  • Attract and retain talented colleagues with a passion for drug development.
  • Build a strong network of experts that will complement, enhance, and support our efforts across development that will include patients, physicians, researchers, regulators, payers, and technical experts.
  • Generate future revenues through two paths: sales revenue for the drugs we intend to bring to market, and revenue from out-licensing assets (through milestone payments and royalties).

Addressing Primary Mitochondrial Disease

Primary mitochondrial disease is a rare orphan disease where the energy metabolism in the cells, by the powerhouses of our cells – the mitochondria – is impaired. This causes deterioration that leads to multifaceted disorders and great suffering for patients. 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 is being developed as a treatment for primary mitochondrial disease patients suffering from multiple debilitating symptoms, including chronic fatigue and myopathy. KL1333 has completed several key Phase 1 studies that enabled the start of a potentially registrational Phase 2 study in 2022. KL1333 is protected by a composition of matter patent and Orphan Drug Designation (ODD) in the US and in Europe. It has also received Fast Track Designation in the US. The commercial opportunity is significant with even conservative estimates exceeding USD 1 billion per year in annual sales1).

NV354 is being developed for mitochondrial disease with neurologic complications, including Leigh syndrome, MELAS (Mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes), and LHON (Leber’s hereditary optic neuropathy). NV354 has completed preclinical development and is supported by a strong group of patents as well as ODD in the US and Europe.

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 committed to taking advantage of rare disease opportunities, successfully attaining ODD for both KL1333 and NV354. ODD provides significant benefits, including regulatory assistance, cost reduction, attractive pricing, and an additional layer of market exclusivity (10 years in the EU, 7 in the US). The outlook for reaching the market is also better than for traditional medicines.2,3) KL1333 has also secured Fast Track designation in the US, streamlining development and marketing application reviews.

Seeking scientific advice from regulators in the US, UK, and Europe has been invaluable, resulting in a shift toward a single, potentially registrational, Phase 2 study for KL1333, expediting its path to market.

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.