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FDA Grants BCMA CAR-T Cell Therapy for the Treatment of R/R Multiple Myeloma Orphan Drug Designation

Anna Osborne
17 February 2022
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Biopharmaceutical companies Innovent and IASO Bio have announced that their co-developed human B-cell maturation antigen (BCMA) – targeted CAR-T cell therapy, has been formally granted Orphan Drug Designation by the FDA for the treatment of r/r multiple myeloma (MM).

This new designation comes following a year in development since the BCMA CAR-T cell therapeutic – referred to as IBI8326 and CT103A by Innovent and IASO Bio respectively – received Breakthrough Therapy Designation from the NMPA in February 2021.

Dr. Hui Zhou the Senior Vice President of Innovent comments, “this ODD of IBI326 is a milestone recognising our effort to develop a novel anti-BCMA CAR with better efficacy and persistence.”

Innovent is a global biopharmaceutical company that develops, manufactures and transitions to market innovative therapeutics for the treatment of cancer and other major diseases. IBI326, is the latest product in their robust clinical pipeline that has seen 5 treatments gain approval for commercialisation in China.

Central to Innovent’s integrated approach is their history of strategic collaborations. They partnered with clinical stage bio-pharmaceutical company IASO Bio in November 2017 for the co-development of this innovative treatment for r/r multiple myeloma.

IASO Bio specialises in discovering and developing novel therapies for cancer and autoimmune diseases, using their proprietary manufacturing processes and high-throughput CAR-T drug platform. CT103A joins IASO’s diverse pipeline portfolio, as their leading asset in light of its Phase II trial results.

Dr. Wen Wang, CEO and CMO of IASO Bio notes that the “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.”

The clinical progression of IBI8326/CT103A will bring hope to patients with r/r multiple myeloma, to which there is currently no cure.

Presently, treatment options for this deadly blood cancer have potential to lead to temporary remission, but the majority of patients will inevitably fall back into relapsed or refectory MM. The number of patients diagnosed with MM is forecast to increase to 162,300 in the US and 182,200 In China by 2025. This presents a significant unmet need in the treatment of MM.

Clinical data from previous BCMA-targeting CAR-T cells treatments for r/r MM have shown a high level of adverse events and re-infusion ineffectiveness upon disease progression. IBI8326/CT103A was developed as a potent and persistent BCMA-targeting CAR containing lentiviral construct, using a proprietary optimisation platform for specific selection and screening, to overcome these issues.

The ODD of IBI8326/CT103A will support in accelerating its development and potential approval in the US, through access to developmental incentives; FDA support of its impending future clinical trials, a reduced registration application fee and 7 years of US market exclusivity.

“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,” comments IASO Bio’s Dr. Wen Wang.

This sentiment is reinforced by Innovent, as Dr. Hui Zhou concludes “[the ODD] strongly motivates us to expedite the clinical development of IBI326. We hope to launch IBI326 as soon as possible which will bring hope to patients with multiple myeloma.”

Sources: Innovent Press Release and IASO Bio Press Release