Zogenix Completes Acquisition of Modis Therapeutics, Inc.
“We welcome Modis to our growing
About MT1621 and TK2 Deficiency
MT1621 is an investigational deoxynucleoside substrate enhancement therapy in late-stage development for the treatment of TK2 deficiency (TK2d), a genetic disorder that results in mitochondrial dysfunction, leading in turn to inadequate energy production in cells. The disease presents as progressive and severe muscle weakness that profoundly impairs movement, breathing, eating, and other normal functions, and is often fatal. Believed to be significantly underdiagnosed, TK2d affects up to 2,500 patients in the U.S., primarily infants and young children. There are currently no approved therapies for this disease.
MT1621 is designed to restore mitochondrial function by targeting the underlying pathophysiology of TK2d. Deoxynucleoside substrate enhancement therapy has been shown to improve cell function and prolong life in preclinical models of TK2d. Data from a Phase 2 retrospective treatment clinical study called RETRO demonstrated increased survival probability and improved functional abilities for patients treated with MT1621 compared with untreated natural history control patients, and suggest that MT1621 may meaningfully alter the course of the disease. With Breakthrough Therapy and PRIME designations already received, MT1621 may be eligible for an accelerated regulatory path in both the U.S. and
Zogenix is a global pharmaceutical company whose mission is to develop and commercialize therapies that transform the lives of patients and their families living with rare diseases. The company’s lead compound is FINTEPLA® (ZX008, fenfluramine) for the treatment of seizures associated with Dravet and Lennox-Gastaut syndromes, two rare and often-catastrophic childhood-onset epilepsies often marked by frequent and prolonged seizures and significant intellectual, behavioral, and motor disabilities.
Zogenix cautions you that statements included in this press release that are not a description of historical facts are forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “indicates,” “will,” “intends,” “potential,” “suggests,” “assuming,” “designed,” and similar expressions are intended to identify forward-looking statements. These statements include: the size of the patient population of TK2d; the potential for MT1621 to significantly improve outcomes in patients with TK2d; the potential of MT1621 to receive for accelerated regulatory review in the U.S. or
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Source: Zogenix, Inc.