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.

“Receiving Fast Track Designation is an important regulatory milestone for PX578 and further underscores the urgent need for new treatment options for people living with POLG disease,” said Ashish Dugar, Chief Development Officer of Pretzel Therapeutics. “As we prepare to initiate the POLARIS study in patients, we remain focused on generating the clinical evidence needed to advance PX578 and bring a potentially disease-modifying therapy to individuals impacted by this progressive and debilitating disease.”

This designation follows recently announced FDA clearance to initiate a first-in-patient clinical trial of PX578, the Company’s lead therapeutic candidate. POLARIS (POLg Activation and Recovery In Subjects) will evaluate PX578 in adult patients with POLG disease building on recent success with a Phase 1 healthy volunteer study with PX578 conducted in New Zealand.

FDA Fast Track designation reflects the significant unmet medical need in POLG disease and supports ongoing engagement with FDA as Pretzel advances PX578 toward Phase 2 study initiation. The designation provides opportunities for more frequent FDA interactions during development and potentially expedites review of PX578.