US FDA Grants Orphan Drug Status To Gracells FasTCAR-enabled BCMA/CD19 Dual-targeting CAR-T Cell Therapy GC012F To Treat Multiple Myeloma

Gracell Biotechnologies Inc. announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation for GC012F, Gracell's FasTCAR-enabled BCMA/CD19 dual-targeting CAR-T cell therapy for the treatment of multiple myeloma.

"As our lead candidate currently being developed on Gracell's FasTCAR next-day manufacturing technology platform, GC012F is a unique BCMA and CD19 dual-targeting CAR-T cell therapy," commented Dr. Martina Sersch, chief medical officer of Gracell. "GC012F has demonstrated fast, deep and durable responses in patients with relapsed/refractory multiple myeloma in an ongoing IIT study in China with most patients on study being high risk according to mSMART 3.0 criteria, a difficult-to-treat patient population. We are very excited about being granted Orphan Drug Designation for the treatment of Multiple Myeloma by the US FDA, another key milestone in advancing our program globally. Multiple Myeloma patients are in need of more efficacious and tolerable therapies providing deep and durable responses and ultimately extending progression free and overall survival."

The long-term follow-up data for GC012F was presented in June at the ASCO 2021 Annual Meeting and the EHA 2021 Congress. GC012F is currently being evaluated in investigator-initiated trials in China, including in newly diagnosed multiple myeloma patients. The tech transfer to Lonza to support manufacturing of GC012F in the US is currently ongoing, with US IND filing targeting the first half of 2022.

Granted by the US FDA, Orphan Drug Designation incentivizes the development of innovative drugs and biologics for the safe and effective treatment of rare diseases and conditions that affect fewer than 200,000 people in the US Orphan Drug Designation qualifies the sponsor of the therapy for certain development incentives, including up to seven years of market exclusivity upon regulatory approval, as well as tax credits for clinical testing and reduction of or exemption from prescription drug user fees.

GC012F is a FasTCAR-enabled dual-targeting CAR-T product candidate that is currently being studied in an ongoing investigator-initiated Phase 1 trial across multiple centers in China for the treatment of MM. GC012F tackles MM by simultaneously targeting both malignant plasma cells expressing BCMA and early progenitor cells expressing CD19 in order to drive fast, deep and durable responses in MM patients.

CAR-T cells manufactured on Gracell's proprietary FasTCAR platform appear younger, less exhausted and show enhanced proliferation, persistence, bone marrow migration and tumor cell clearance activities as demonstrated in preclinical studies. With next day manufacturing, FasTCAR is able to significantly improve cell production efficiency which may result in meaningful cost savings, increasing the accessibility of cell therapies for cancer patients.

Gracell Biotechnologies Inc. ("Gracell") is a global clinical-stage biopharmaceutical company dedicated to discovering and developing breakthrough cell therapies.