Rare Daily Staff

Mitochondrial disease drug developer Modis Therapeutics completed a $30 million series A financing round and named Joshua Grass, former head of corporate development at BioMarin Pharmaceutical, as its president and CEO.

F-Prime Capital Partners and OrbiMed led the investment. Grass most recently had been an Entrepreneur-in-Residence at F-Prime. With the financing, F-Prime’s Ben Auspitz joined the board of Modis as chairman and OrbiMed’s Rishi Guptahave also joined the board. Founding investor Aceras Life Sciences and new investor Osage University Partners also participated in the financing.

Modis’ lead experimental therapy, MT1621, is in development as a treatment for TK2d, a rare mitochondrial DNA depletion disorder. TK2d is a genetic disorder that results in mitochondrial dysfunction, leading to inadequate energy production in cells. TK2d may present at all ages and causes progressive and severe muscle weakness, respiratory insufficiency, and is often fatal. There are currently no approved therapies for this disease.

Modis has an exclusive worldwide license to data and intellectual property covering mtDNA depletion disorders from Columbia University Irving Medical Center and Vall d’Hebron Research Institute. The development of MT1621 is based on this work. The researchers were able to 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. Collaborators translated this work to the treatment of TK2d patients with encouraging results.

Modis is planning additional clinical studies and will be meeting with the U.S. Food and Drug Administration and the European Medicines Agency 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 United States.

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,” said Joshua Grass, president and CEO of Modis Therapeutics. “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.”

October 2, 2018
Photo: Joshua Grass, president and CEO of Modis Therapeutics