The rare disease therapy was developed by Modis Therapeutics. This company is based on Columbia University research. Modis Therapeutics was formed through a collaboration with academic experts in mitochondrial biology to develop disease-modifying therapies for rare genetic diseases. The company completed a series A financing with investments from F-Prime Capital Partners, OrbiMed, Aceras Life Sciences, and Osage University Partners. The company has assembled a management team with deep experience in the development of high-impact products for serious diseases at leading biotech and pharmaceutical companies. Modis is a dedicated team, passionate about bringing novel therapies to patients with uncommon diseases.

Their lead product, MT1621, a deoxynucleoside combination therapy, is in development for an inherited mitochondrial disorder, thymidine kinase 2 deficiency (TK2d). When thymidine kinase 2 (TK2) activity is deficient, the result is an imbalance of the nucleotide pool inside the mitochondria that are used to replicate mtDNA. With mtDNA unable to replicate properly, cells cannot produce adequate energy. Production of energy in cells is decreased in tissues throughout the body — especially those that have greater energy requirements, predominantly skeletal muscle in patients with TK2 deficiency.

TK2 deficiency presents primarily as progressive and severe muscle weakness (lat. Musculus infirmitatem) that profoundly impairs movement, breathing, eating/nutrition and other normal functions. Symptom onset can start as early as the first year of life or as late as adulthood. Like other inborn errors of metabolism, generally, onset early in life will predict faster progression of a disease. Patients with TK2d most often die from respiratory failure. TK2 deficiency may be misdiagnosed as other diseases such as Spinal Muscular Atrophy or Muscular Dystrophy. There are currently no approved therapies for TK2 deficiency.

Insights and knowledge gleaned during the development of MT1621 position Modis to leverage the increasing understanding and genetic characterization of mitochondrial diseases to develop targeted high impact therapies for these underserved diseases. Pre-clinical and initial clinical data from studies with deoxynucleoside combination therapy suggest that it may meaningfully alter the course of disease in patients with TK2 deficiency. Modis has exclusively licensed worldwide rights to data and intellectual property related to a broad range of mtDNA depletion disorders from its academic collaborators. Modis is advancing discussions with the FDA and EMA on the regulatory path to approval for MT1621. The company is planning to conduct additional clinical studies with the goal of obtaining regulatory approval to make MT1621 available to patients globally. If approved, MT1621 would represent the first commercial therapy for TK2d or any MDD

MT1621 is a therapy that targets the underlying pathophysiology of TK2d by restoring mitochondrial DNA replication fidelity. The drug candidate consists of a combination of deoxynucleosides (the building blocks of mtDNA) given orally as a dissolved solution. Deoxynucleoside combination therapy improves nucleotide balance, increases mtDNA copy number, improves cell function, and prolongs life in preclinical models of TK2d.

Insights and knowledge gleaned from the development of MT1621 position Modis to leverage the increasing understanding and genetic characterization of mitochondrial diseases to develop targeted high impact therapies for these underserved diseases. Modis is actively developing a pipeline of next-generation therapeutic products for TK2d and other MDDs.