FDA Grants Orphan Drug Designation to SynKIR-110 for Mesothelin-Expressing Mesothelioma

FDA

The FDA has granted an orphan drug designation (ODD) to SynKIR-110, a first-in-class KIR-CAR T-cell immunotherapy candidate, for the treatment of patients with mesothelin-expressing mesothelioma.¹

SynKIR-110 is a next-generation cell therapy designed to target solid tumors. It is the first product to use the novel KIR-CAR platform, which is a modified natural killer–like receptor designed to improve persistence and efficacy against aggressive solid tumors. The KIR-CAR platform utilizes a dual-chain CAR T-cell therapy.

“ODD is another major milestone for Verismo following the acceptance of our SynKIR-110 [investigsational new drug application],” Bryan Kim, DMD, chief executive officer of Verismo, stated in a press release. “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.”

Preclinical models demonstrated that the KIR-CAR platform maintained antitumor T-cell activity in challenging solid tumor environments. Cell persistence was improved by DAP12 costimulatory chains aiding additional T-cell stimulating pathways.

Additionally, the KIR-CAR platform can be combined with other additional emerging technologies, such as in vivo gene editing, advanced T-cell selection and modulation, combination therapies, and allogeneic cellular therapies.

SynKIR-110 will be investigated in the phase 1 STAR-101 trial in patients with mesothelin-expressing ovarian cancer, mesothelioma, and cholangiocarcinoma. The trial is expected to begin enrolling patients in the first quarter of 2023.

This first-in-human trial will assess the safety, tolerability, and preliminary efficacy of SynKIR-110 in these patient populations to address a pertinent unmet need.² For patients with mesothelioma, life expectancy can vary based on the type and location of the disease, stage at diagnosis, and general patient health, but survival typically ranges from 4 to 18 months after diagnosis.

Earlier in September 2022, the FDA granted clearance to initiate the first-in-human trial.²

“Pre-clinical data have shown enhanced efficacy in murine models with no additional safety concerns. The FDA’s clearance of our SynKIR-110 IND represents a new chapter for Verismo as we initiate the first-ever clinical trial for KIR-CAR T cells. It also validates the years of innovative research and hard work by industry pioneers at Penn and the Verismo team,” Kim stated in a press release.²

References

1. Verismo Therapeutics receives U.S. FDA orphan drug designation for SynKIR-110™, a first-in-class KIR-CAR T cell immunotherapy candidate, for the treatment of mesothelioma. News release. Verismo. September 28, 2022. Accessed September 28, 2022. https://prn.to/3fgvuSK
2. Verismo Therapeutics announces FDA clearance of IND application for SynKIR-110™, a KIR-CAR T cell immunotherapy candidate. News release. Verismo. September 20, 2022. Accessed September 28, 2022. https://prn.to/3UP717i