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 primary mitochondrial diseases, which are orphan indications. We aim to take our projects to market authorization and commercialize them either on our own or together with a partner.

Focus on KL1333 and NV354

Abliva’s focus on primary mitochondrial diseases means that the company is concentrating financial and personnel resources on the KL1333 and NV354 drug candidates.

  • KL1333, a powerful NADregulator,  is in clinical phase and we are planning the start of a registrational Phase 2/3 study during 2021. 
  • NV354, an energy replacement (succinate) therapy, is being prepared for clinical trials, aiming at entering the clinical phase during 2022.

High unmet medical need in primary mitochondrial diseases

Primary mitochondrial diseases are congenital, rare, and often very severe diseases that occur when the cell’s energy provider, the mitochondria, do not function properly. The disorders can manifest differently depending on the organs affected and have historically been viewed as clinical syndromes, and more recently as disease spectra caused by genetic defects affecting mitochondrial function. An estimated 125 in every 1,000,000 people suffer from a primary mitochondrial disease. The diseases often present in early childhood and lead to severe symptoms, such as mental retardation, fatigue, myopathy, heart failure and rhythm disturbances, diabetes, movement disorders, stroke-like episodes, and epileptic seizures.

These diseases often cause great suffering for both patients and family members. The symptoms worsen over time and, in many cases, the diseases lead to a far too early death. Today, a very limited number of treatment options are available, which means there are major unmet medical needs.

The objective is to improve life for patients suffering from primary mitochondrial diseases. Our portfolio includes several projects with mechanisms of action suitable for different target patient populations. 

Significant advantages with developing orphan drugs

Medicines developed for rare diseases are well placed to obtain orphan drug designation. The authorities’ objective with orphan drug designation is to promote the development of treatments specifically for rare diseases by providing better support during the development process. The outlook for reaching the market is also better than for traditional medicines. Medicines that receive orphan drug status after market authorization are given market exclusivity for 10 years in the EU and 7 years in the US.. 

KL1333 has obtained orphan drug designation and NV354 also has the potential to receive orphan drug designation. An orphan drug designation generally offers several positive benefits, including:

  • regulatory assistance and scientific advice from pharmaceutical regulators
  • efficient development
  • lower development costs
  • greater chance of regulatory approval compared with drug candidates that lack orphan drug designation
  • attractive pricing compared with drug candidates that lack orphan drug designation1)2)

Abliva collaborates continuously with world-class consultants in the field of orphan drugs, who assist the company in its dialogue with regulators. Abliva has also established partnerships and a continuous dialogue with some of the world’s leading clinical centers for the treatment of primary mitochondrial diseases.