PTC844 / PTC Therapeutics - LARVOL DELTA

Clinical Development of the Novel Dihydroorotate Dehydrogenase Inhibitor PTC844: Preclinical Studies, Phase 1 Study in Healthy Volunteers, and Design of a Phase 2 Study in Adults with Active Rheumatoid Arthritis (ACR Convergence 2026) - No abstract available

P1 data • P2 data • Preclinical • Immunology • Inflammatory Arthritis • Rheumatoid Arthritis • Rheumatology

Study of PTC844 in Participants With Active Rheumatoid Arthritis (clinicaltrials.gov) - P2 | N=75 | Not yet recruiting | Sponsor: PTC Therapeutics

New P2 trial • Immunology • Inflammatory Arthritis • Rheumatoid Arthritis • Rheumatology

Evaluation of the potent and selective dihydroorotate dehydrogenase inhibitor PTC844 for use in immune-mediated diseases (EULAR 2026) - "PTC844 is a novel, orally administered, small molecule that has demonstrated uniquely potent and selective inhibition of DHODH compared with in-class compounds both approved and under development to treat immune-mediated disease (teriflunomide, the teriflunomide prodrug leflunomide and vidofludimus)...In the xGvHD mouse model, the median survival time (MST) with PTC844 was 76 days compared with 36 days without treatment, demonstrating better survival than cyclosporine, Janus kinase inhibitors or prednisolone...In a rabbit model of arthritis, PTC844 reduced swelling by 58% at Day 40 compared with the group without treatment, a similar reduction to that observed with methotrexate...Inhibition of DHODH in vivo resulted in elevations in DHO levels, which allowed identification of DHO levels associated with efficacy in preclinical studies. Conclusions Preclinical findings support further testing of PTC844 in immune-mediated disease."

CNS Disorders • Graft versus Host Disease • Immunology • Inflammatory Arthritis • Lupus • Multiple Sclerosis • Solid Tumor