IASO Bio and Innovent’s BCMA CAR-T cell therapy gains Orphan Drug Designation

15-Feb-2022 - Last updated on 15-Feb-2022 at 12:47 GMT

Pic:getty/marianvejcik

IASO Biotherapeutics and Innovent Biologics have announced that the FDA has granted Orphan Drug Designation (ODD) to their fully human anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T-cell therapy for the treatment of relapsed and/or refractory multiple myeloma (R/R MM).

This ODD will accelerate drug development and registration action of the therapy, CT103A, in the US. It will be eligible for certain development incentives, including FDA support for clinical studies, a waiver or reduction of registration application fee, and a seven-year US market exclusivity granted upon product approval.

In February 2021, CT103A was granted Breakthrough Therapy Designation by China's National Medical Products Administration (NMPA) for the treatment of R/R MM.

Advancing clinical development

Multiple myeloma is a deadly blood cancer that often infiltrates the bone marrow causing anemia, kidney failure, immune problems, and bone fractures. For multiple myeloma patients, common first-line drug treatments include proteasome inhibitors, immunomodulatory drugs, and alkylating agents.

While treatment may result in remission, most patients will inevitably enter the relapsed or refractory stage as there's currently no cure. As a result, there is a significant unmet need for patients with relapsed/refractory multiple myeloma.

In 2020 there were an estimated 32,300 new MM cases in the US; and 21,100 in China. This is set to rise to around 37,800 in the US by 2025 and 24,500 by 2025.

Previous studies indicate subjects with R/R MM who received high-dose BCMA-targeting CAR-T cells may achieve better remission but have worse adverse events. Moreover, once the disease progresses again, the re-infusion of CAR-T cells will not be effective.

In response, IASO Bio and Innovent have co-developed CT103A: a lentiviral vector containing a CAR structure with a fully human scFv, CD8a hinger and transmembrane, 4-1BB co-stimulatory and CD3ζ activation domains. Based on strict selection and screening, utilizing a proprietary in-house optimization platform, the construct of the BCMA CAR-T is potent and persistent.

"FDA approval of ODD to CT103A is of great significance to patients with multiple myeloma and represents the FDA's recognition of CT103A and the clinical data provided by IASO Bio", said Dr. Wen Wang, Chief Executive Officer and Chief Medical Officer of IASO Bio. "Currently, our team is advancing the clinical development of CT103A to the four dimensions of strategy including frontline therapy, combination therapies, indication expansion, and ex-China development. We are looking forward to the launch of CT103A both in China and US as soon as possible to offer living-saving treatment option to more patients."