Modis Therapeutics, Inc., a company developing medicines for patients with rare genetic diseases has raised $30 million in Series A funding led by F-Prime Capital Partners and OrbiMed. Joshua Grass, former F-Prime Entrepreneur-in-Residence and head of Corporate Development at BioMarin Pharmaceutical, has joined as President and Chief Executive Officer. Founding investor Aceras Life Sciences, LLC and new investor Osage University Partners also participated in the financing.
Founded in 2016, Modis is advancing its lead experimental therapy, MT1621, as a treatment for thymidine kinase 2 deficiency (TK2d), a rare, genetically defined mitochondrial DNA (mtDNA) depletion disorder. TK2d results from inherited mutations in the nuclear TK2 gene and leads to severe muscle weakness and premature death. TK2d symptom onset varies in age from infants to adults and there are currently no approved treatments.
The development of MT1621 is based on research from the lab of Dr. Michio Hirano, the chief of the Division of Neuromuscular Medicine at Columbia University Irving Medical Center, the lab of Dr. Ramon Martí, the head of the Neuromuscular and Mitochondrial Pathology Research Group at Vall d’Hebron Research Institute in Barcelona, and other academic collaborators.
Modis has exclusively licensed worldwide rights to data and intellectual property related to a broad range of mtDNA depletion disorders from Columbia University Irving Medical Center and Vall d’Hebron Research Institute.
Dr. Hirano’s lab was able to first show that a combination of nucleosides, the active ingredients in MT1621, addresses the direct cause of mitochondrial dysfunction and prolongs survival in a genetic animal model of TK2 deficiency. Dr. Hirano, Dr. Martí and other collaborators in Spain translated this work to the treatment of TK2d patients with encouraging results.
Modis is planning additional clinical studies and will be meeting with the FDA and EMA to discuss the registration path for MT1621 with the goal of enabling broad global access for patients with TK2d. Modis has orphan drug designation for MT1621 in both the United States and Europe and has rare pediatric disease designation in the US.
Beyond MT1621, Modis is developing other molecules and therapeutic modalities to treat TK2 deficiency and other mitochondrial diseases with defined genetic causes.
“This is a compelling orphan drug development opportunity for a potentially high impact therapeutic for a very serious disease that affects both children and adults. We are fortunate to have encouraging clinical data, committed physician-scientist collaborators, and two recently published natural history studies of TK2d to help interpret the effect of treatment,” said Joshua Grass, President and Chief Executive Officer of Modis Therapeutics.
