Verismo Therapeutics, a clinical-stage CAR-T company and Penn spinout, and pioneer of the novel KIR-CAR platform technology, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to SynKIR-110 for the treatment of patients with mesothelin-expressing mesotheliomas.
SynKIR-110 is a next generation approach to cell therapy targeting solid tumors, and the first product to use the novel KIR-CAR platform, a modified NK-like receptor designed to improve persistence and efficacy against aggressive solid tumors. Phase 1 clinical trials of SynKIR-110 in patients with mesothelin-expressing ovarian cancer, mesothelioma and cholangiocarcinoma will commence in Q1 2023.
“Orphan Drug Designation is another major milestone for Verismo following the acceptance of our SynKIR-110 IND,” said Dr. Bryan Kim, CEO of Verismo. “Mesothelioma is a rare and deadly disease for which few other treatment options currently exist. We look forward to working with the mesothelioma community to advance SynKIR-110 as a potential treatment while we continue to expand clinical investigation of this novel platform in other cancers in the solid tumor space.”
Orphan Drug Designation is a status given to drugs intended to treat a rare disease that affects fewer than 200,000 people in the United States and show promise in the treatment, prevention, or diagnosis of that disease. Benefits to the study sponsor include seven years of marketing exclusivity, federal tax credits, waiver of Prescription Drug User Fee Act (PDUFA) fees and increased regulatory assistance from the FDA.
The Phase 1 trial for SynKIR-110, STAR-101 (SynKIR T cell Advanced Research), will begin enrolling patients in the first quarter of 2023 at the initial clinical site, the Hospital of the University of Pennsylvania.