FDA grants Orphan drug designation to KIR-CAR T cell therapy for mesothelioma patients

October 2022 Pharma News Nalinee Pandey
Example of asbestiform riebeckite ore, also known as crocidolite (or "blue asbestos"), one of six mineral types currently regulated as asbestos.

The Food and Drug Administration (FDA) has awarded orphan drug designation to novel immunotherapy, SynKIR-110TM for mesothelioma patients. The announcement was made on 28th September 2022.

Mesothelioma is a rare disease caused mainly due to exposure to asbestos. Despite available treatment modalities, the clinical outcomes remain poor for this aggressive disease. Verismo therapeutics has used their novel novel-KIR-CAR technology platform for developing an advanced treatment option for mesotheliomas.

Potential treatment option

Verismo therapeutics, a clinical-stage CAR-T cell company has developed advanced SynKIR-110 as a potential treatment option for aggressive mesotheliomas. This company is a novel-KIR-CAR technology platform which uses a modified NK-like receptor designed to improve persistence and efficacy against aggressive solid tumours. It is a dual-chain CRA-T cell therapy platform and its efficacy has been well-established in preclinical models of solid tumours. The platform is capable of maintaining antitumor T cell activity and having persistent activity against tumours even for those refractory to traditional CAR T cell therapy. Furthermore, this platform can be used in combination with other technologies such as in vivo gene editing, advanced T cell selection, combination therapies and allogeneic cellular therapies.

Phase-I trial

SynKIR-110 is the first product that has used the KIR-CAR platform and uses a modified NK-like receptor designed to improve persistence and efficacy against aggressive solid tumours. The Phase-I clinical trial for SynKIR-110 will commence in early 2023 at the initial clinical site, the Hospital of the University of Pennsylvania.

This technology was specifically developed for solid tumours. It will be interesting to see its efficacy in the planned clinical trial.