Pretzel Therapeutics Receives FDA Fast Track Designation for PX578 for the Treatment of POLG-mediated Primary Mitochondrial Disorders
Pretzel Therapeutics, Inc., a clinical-stage biotechnology company advancing a new class of medicines designed to restore mitochondrial function, improve cellular energetics and impede disease progression across a range of neurological and rare diseases, today announced that the U.S. Food and Drug Administration has granted Fast Track Designation to PX578 for the treatment of POLG-mediated primary mitochondrial disorders (POLG disease), a rare, progressive genetic condition for which there are currently no approved disease-modifying treatments.
Pretzel Therapeutics Advances PX578 into Phase 2 Study in Individuals Living with POLG-mediated Primary Mitochondrial Disease Following FDA Clearance of IND
Pretzel Therapeutics, Inc., a clinical-stage biotechnology company advancing a new class of medicines designed to restore mitochondrial function, improve cellular energetics and impede disease progression across a range of neurological and rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application to initiate a first-in-patient clinical trial of PX578, the Company’s lead therapeutic candidate. The Phase 2 study, POLARIS (POLg Activation and Recovery In Subjects), will evaluate PX578 in adult patients with POLG-mediated primary mitochondrial disease (POLG disease), a rare, progressive genetic disorder caused by mutations in the POLG gene causing a depletion of mitochondrial DNA. This mitochondrial DNA depletion syndrome results in mitochondrial dysfunction and a range of debilitating neurological and systemic manifestations. There are currently no approved disease-modifying treatments for this condition. POLARIS will build on recent success with a Phase 1 healthy volunteer study with PX578 conducted in New Zealand.
