On September 28, 2022 Verismo Therapeutics, a clinical-stage CAR-T company and Penn spinout, and pioneer of the novel KIR-CAR platform technology, reported 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 (Press release, Verismo Therapeutics, SEP 28, 2022, View Source [SID1234621530]).
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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.
About the KIR-CAR Platform
The KIR-CAR platform is a dual-chain CAR T cell therapy and has been shown in preclinical models to be capable of maintaining antitumor T cell activity even in challenging solid tumor environments. DAP12 costimulatory chains aid additional T cell stimulating pathways, further improving cell persistence. This continued function and persistence can lead to ongoing regression of solid tumors in preclinical models, including those refractory to traditional CAR T cell therapies. Furthermore, the KIR-CAR platform can be combined with many additional emerging technologies, such as in vivo gene editing, advanced T cell selection and modulation, combination therapies, and even allogeneic cellular therapies to provide an adaptable targeted anti-tumor therapy for patients in need.
About Mesothelioma
Mesothelioma is a rare type of cancer that affects the lining of the chest or abdominal cavities. More than 2,000 cases of this aggressive cancer are diagnosed in the U.S. each year. While life expectancy can differ depending on a variety of factors, such as type and location of the disease, stage at diagnosis, and general patient health, overall, patients usually survive only four to eighteen months after diagnosis.