Research & Development

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 primary mitochondrial diseases

Our portfolio

KL1333: Innovative therapy in late-stage development

Project status: Dosing in patients is ongoing in the FALCON study

KL1333 has been evaluated in both healthy volunteers and patients and has been granted orphan drug designation in both the United States and Europe. The global FALCON study with KL1333 was initiated in late 2022. The company will recruit patients in two waves, and an interim analysis will take place after the completion of Wave 1 towards the middle of 2024. KL1333 has Orphan Drug Designation (ODD) in both Europe and the U.S. as well as Fast Track designation in the U.S.

Mitochondrial disease target population: MELAS-MIDD, KSS-CPEO and MERRF

KL1333 is being developed as a treatment for a subset of adult primary mitochondrial disease patients suffering from multiple debilitating symptoms, including chronic fatigue and myopathy. Diagnoses can include MELAS-MIDD and KSS-CPEO spectrum disorders as well as MERRF syndrome.

Mechanism of action: NAD⁺/NADH modulation

KL1333 is a potent modulator of the cellular levels of NAD+ and NADH, central co-enzymes in the cell’s energy metabolism. KL1333 has in preclinical models demonstrated to increase mitochondrial energy output, have long-term beneficial effects on energy metabolism, strengthen muscle function and improve biomarkers of mitochondrial disease.

NV354: First-in-class therapeutic targeting high unmet need

Project status: Preclinical development completed

The program has completed preclinical development and has been granted Orphan (Drug) Designation both in the EU and the U.S. Given the prioritization of KL1333, no significant cost-intensive operational activities are planned for NV354 at this time.

Mitochondrial disease target population: Neurology

NV354 is being developed for mitochondrial disease with neurologic complications, in particular at insufficient activity in the mitochondrial protein complex I. The resulting deficiency in energy conversion contributes to clinical signs and symptoms in many types of mitochondrial disease, including neurologic complications seen in Leigh syndrome, MELAS, or LHON. There are also additional expansion opportunities outside of mitochondrial disease.

The drug candidate was discovered due to its ability to increase mitochondrial function in cells from mitochondrial Leigh syndrome patients. Leigh syndrome usually debuts at one to two years of age and includes psychomotor regression, hypotonia and developmental delays. The disease is fatal, and children with early-onset Leigh syndrome usually die before adulthood.

Mechanism of action: Energy replacement

Brain-penetrable NV354 is based on an innovation in which the body’s own energy substrate, succinate, is made available in the cell via a prodrug technology. A prodrug is an inactive drug that is activated first when it enters the body by the transformation of its chemical structure.

Early programs

Abliva has efforts ongoing to identify additional portfolio opportunities focused on the regulation and stabilization of cellular energy production.