FDA grants orphan drug status to CAR-T cell immunotherapy candidate

By Jane Byrne contact

- Last updated on GMT

© GettyImages/Meletios Verras
© GettyImages/Meletios Verras

Related tags: solid tumor, CAR-T, Fda

Penn spinout, Verismo Therapeutics, says the FDA has granted Orphan Drug Designation (ODD) to its KIR-CAR T-cell immunotherapy drug, SynKIR-110, for the treatment of patients with mesothelin-expressing mesotheliomas.

ODD is a status given to drugs intended to treat a rare disease that affects fewer than 200,000 people in the US 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.

"Orphan Drug Designation is another major milestone for Verismo following the acceptance of our SynKIR-110 IND,"​ said Dr Bryan Kim, CEO of Verismo.

A clinical-stage CAR-T company, and pioneer of the KIR-CAR platform technology, Verismo saw its Investigational New Drug (IND) application to the US Food and Drug Administration (FDA) approved earlier this month, allowing it to initiate its first-in-human Phase 1 clinical trial of SynKIR-110.

Verismo plans to begin enrolling patients for that study, SynKIR T cell Advanced Research (STAR-101), in the first quarter of 2023 at the initial clinical site, the Hospital of the University of Pennsylvania. The trial will assess the safety, tolerability, and preliminary efficacy of the treatment in patients with mesothelin expressing ovarian cancer, cholangiocarcinoma and mesothelioma, diseases which have low survival rates and an urgent need for effective treatment options.

Tackling solid tumors

It will be the first-in-human study for SynKIR T cells, a next generation approach to cell therapy that targets solid tumors powered by the KIR-CAR platform. 

SynKIR-110 is the first product to use the novel KIR-CAR platform. Our technology incorporates a natural on/off switch that allows KIR-CAR T cells to rest when not exposed to tumor antigens, as well as providing an enhanced cell-surface stability of the KIR-CAR,”​ said Dr Laura Johnson, CSO of Verismo. “These enhancements will allow KIR-CAR T cells to better cope with the harsh tumor microenvironment of solid tumors and, potentially, lead to better outcomes for patients.”

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, reported the developer.

"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,”​ added Verismo's CEO.

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