Designation for the Treatment of TK2 Deficiency Based on Compelling Data from Initial Clinical Studies and High Unmet Need in Thymidine Kinase 2 Deficiency
On February 19, 2019, Modis Therapeutics announced that the FDA has granted Breakthrough Therapy designation to MT1621, Modis’ investigational treatment for patients with thymidine kinase 2 deficiency (TK2d). Modis Therapeutics is a biopharmaceutical company based in Oakland that is focused on developing disease-modifying therapies for rare genetic diseases with high unmet medical need.
Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious or life-threatening condition, and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s). A drug that receives Breakthrough Therapy designation is eligible for intensive guidance on an efficient drug development program, organizational commitment involving senior managers from the FDA, and all Fast Track designation features, including eligibility for Accelerated Approval and Priority Review, if relevant criteria are met.
Thymidine kinase 2 deficiency (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.
In late 2018, Modis received $30 million in a Series A round led by F-Prime Capital Partners and OrbiMed. Founding investor Aceras Life Sciences, LLC and new investor Osage University Partners also participated in the round.