Hepcidin Mimetic Candidate Receives Fast Track Designation for Polycythemia Vera

Protagonist Therapeutics announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to PTG-300 in the treatment of polycythemia vera, a rare disease characterized by the excessive production of red blood cells. PTG-300 is an injectable synthetic peptide mimetic of the natural hormone hepcidin that has demonstrated the ability to dramatically decrease the requirement for phlebotomy in an ongoing Phase 2 study in polycythemia vera patients. PTG-300 has previously received orphan drug designation for the treatment of polycythemia vera from the FDA.

"Fast Track designation reflects the potential for PTG-300 to improve upon the treatments that are currently available for patients with polycythemia vera, and provides opportunities to substantially accelerate clinical development," said Dinesh V. Patel, Ph.D., Protagonist President and Chief Executive Officer. "We continue to enroll and treat patients in our ongoing Phase 2 study, with complete enrollment expected in mid-2021. We look forward to working closely with the FDA to discuss and finalize a pivotal program in the first half of 2021."

The FDA Fast Track Program is designed to facilitate the development and expedite the review of new therapeutics that are intended to treat serious conditions and that demonstrate the potential to address unmet medical needs. Drugs that receive this designation benefit from more frequent interactions and meetings with the FDA and potential pathways for expedited approval.

Polycythemia vera is a myeloproliferative neoplasm characterized primarily by the increased production of red blood cells. Well-established treatment guidelines focus on maintaining hematocrit levels continuously below 45 percent to reduce the risk of thrombotic events. Unfortunately, current treatment options are unable to maintain hematocrit to below the 45 percent target for many patients and may be associated with serious side effects. There are an estimated 100,000 patients with polycythemia vera in the U.S. and approximately 100,000 patients in major EU countries.