According to a story from BioSpace, the biopharmaceutical company Modis Therapeutics recently announced that the US Food and Drug Administration (FDA) has granted Breakthough Therapy designation for the company’s experimental product candidate MT1621. This investigational therapy is in development as a treatment for thymidine kinase 2 (TK2) deficiency. Modis focuses in developing drugs for rare diseases with high unmet medical need.
About TK2 Deficiency and Mitochondrial Disease
TK2 deficiency is a type of mitochondrial disease. Mitochondrial diseases are a group of genetic disorders that causes the mitochondria not to function properly. The mitochondria are an essential organelle that is found in most types of cells in the body, with red blood cells being the only exception. They are responsible for generating energy for the cell. Mitochondrial diseases are usually caused by mutations of the mitochondrial DNA or the nuclear DNA. Symptoms tend to be the worst when the issue affects cells that use a lot of energy, such as the muscles or parts of the brain. These symptoms affect many aspects of bodily function and include poor growth, poor muscle coordination, dementia, neurological issues, muscle weakness, breathing disorders, vision problems, digestive disorders, hearing problems, disease of the kidney, liver, and heart, and learning disabilities. Treatment options are limited in number and in their effectiveness. To learn more about mitochondrial diseases, click here.
About Breakthrough Therapy Designation
Breakthrough Therapy designation is intended for therapies in development that have the potential to treat life-threatening diseases and disorders. The designation is intended to speed up the review and development process for the drug. To qualify, a therapy must demonstrate the potential to treat a life-threatening disease that has no available therapies or must offer significant advantages over current therapies. Breakthrough Therapy designation confers significant benefits to the recipient, such as close collaboration on development with the FDA, Fast Track designation, and potential eligibility for Priority Review and Accelerated Approval if other criteria are satisfied.
Early data suggests that MT1621 could have substantial positive impacts on patients with this type of mitochondrial disease. The drug is a combination deoxynucleoside therapy that is intended to treat the underlying cause of TK2 deficiency; in preclinical models, the drug was capable of improving cellular function.