Waltham, Mass. — Pretzel Therapeutics Inc. said the U.S. Food and Drug Administration has granted Fast Track Designation to PX578 for the treatment of POLG-mediated primary mitochondrial disorders, a rare and progressive genetic disease with no approved disease-modifying treatments.
Pretzel Therapeutics is a clinical-stage biotechnology company developing therapies aimed at restoring mitochondrial function and improving cellular energy production.
“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,” Chief Development Officer Ashish Dugar said.
“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,” he added.
The designation follows the FDA’s recent clearance for Pretzel Therapeutics to begin a first-in-patient clinical trial of PX578, the company’s lead therapeutic candidate.
The planned Phase 2 POLARIS study, which stands for POLG Activation and Recovery In Subjects, will evaluate PX578 in adults with POLG disease. The company plans to begin the study in late 2026.
Pretzel said the trial will build on results from a Phase 1 study of PX578 in healthy volunteers conducted in New Zealand.
FDA Fast Track Designation is intended to facilitate development and review of treatments for serious conditions with unmet medical needs. The designation allows for more frequent interactions with the FDA during development and may help expedite review of PX578.
