Protagonist Therapeutics
Protagonist Therapeutics works on 2 rare diseases tracked on Trial Friend, including Polycythemia Vera, Thalassemia, with 2 active clinical trials.
Protagonist Therapeutics develops peptide-based therapeutics for rare hematologic diseases. The company's lead candidate rusfertide, partnered with Takeda for ex-US commercialization, is a hepcidin mimetic under FDA Priority Review (NDA accepted, PDUFA date Q3 2026) for polycythemia vera, a rare myeloproliferative disorder of excessive red blood cell production.
Focus areas at Protagonist Therapeutics
Within its broader pharmaceutical portfolio, Protagonist Therapeutics has active clinical trial programs and drug development efforts across 2 rare diseases, including Polycythemia Vera, Thalassemia. These programs may span orphan drug designation, novel therapeutic mechanisms, and precision medicine approaches targeting the underlying causes of each disease.
The clinical trials section below shows active and recruiting rare disease studies sponsored by Protagonist Therapeutics, sourced live from ClinicalTrials.gov. Each trial includes its current recruitment status, study phase (Phase 1 through Phase 4), conditions under investigation, and the number of active trial sites. The FDA-approved drugs section lists treatments that have received U.S. Food and Drug Administration approval, with brand names, generic names, approval dates, and matched rare disease indications from the openFDA database.
Protagonist Therapeutics is headquartered in Newark, United States, founded in 2006, publicly traded under the ticker symbol PTGX. The company maintains a dedicated rare disease division alongside its broader therapeutic portfolio, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.
Protagonist Therapeutics Drug Pipeline
Protagonist Therapeutics has 2 active clinical trials across 1 development stage. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline counts the active and recruiting trials listed on this page, from ClinicalTrials.gov. For companies that work outside rare disease, we list only trials for rare diseases we track, so the company's full pipeline may be larger.
Protagonist Therapeutics Clinical Trials (2)
Active and recruiting clinical trials sponsored by Protagonist Therapeutics, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
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FDA-Approved Drugs
Protagonist Therapeutics Trial Locations
Protagonist Therapeutics clinical trials are running at 185 sites in 20 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
Rare Disease Focus Areas (2)
Diseases targeted by Protagonist Therapeutics's clinical trial and drug development programs
Polycythemia Vera is a myeloproliferative neoplasm in which bone marrow produces too many red blood cells, leading to thick blood that carries oxygen less effectively and increases clot risk. Most pat...
Thalassemia is an inherited blood disorder where reduced or absent production of one type of hemoglobin chain causes severe anemia, organ damage from iron overload, and bone abnormalities. Severe form...
Patient Resources
Organizations and resources related to Protagonist Therapeutics's rare disease focus areas
Frequently Asked Questions About Protagonist Therapeutics
Common questions about Protagonist Therapeutics's rare disease programs, clinical trials, and treatments.