- The FDA granted orphan drug designation to GC012F, a novel chimeric antigen receptor T-cell therapy for the treatment of relapsed or refractory multiple myeloma.
- GC012F (Gracell Biotechnologies) is an autologous, gene-edited, bispecific CAR-T that targets the B-cell maturation antigen (BCMA) and CD19 proteins on the surface of cancer cells.
- Among the benefits of Orphan Drug status in the U.S. is a seven-year period of market exclusivity for the indication, if approved.
- “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,” commented Dr. Martina Sersch, Chief Medical Officer of Gracell.
- Multiple myeloma (MM) is the third most common type of blood cancer in the United States, originating from plasma cells, a type of immune cell that is typically responsible for secreting antibodies to fight infection.
Gracell Biotechnologies Inc, a global clinical-stage biopharmaceutical company dedicated to developing highly efficacious and affordable cell therapies for the treatment of cancer, today announced that the U.S. 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 U.S. 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 U.S. is currently ongoing, with U.S. IND filing targeting the first half of 2022.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.
What is Multiple myeloma?
Multiple myeloma is a cancer that forms in a type of white blood cell called a plasma cell. Healthy plasma cells help you fight infections by making antibodies that recognize and attack germs. In multiple myeloma, cancerous plasma cells accumulate in the bone marrow and crowd out healthy blood cells.
Multiple myeloma (MM) is the third most common type of blood cancer in the United States, originating from plasma cells, a type of immune cell that is typically responsible for secreting antibodies to fight infection. Globally, approximately 160,000 patients are diagnosed with MM every year with over 32,000 expected to be diagnosed in the United States in 2020. In recent years, many advances have been made to treat MM, however, the disease is still considered incurable.
Multiple myeloma patients with certain cytogenetic and other abnormalities are classified by the International Myeloma Working Group (IMWG) and Mayo Stratification for Myeloma and Risk-Adapted Therapy (mSMART), criteria as high-risk patients. They represent 20-30% of the overall MM patient population. High-risk patients have a much higher risk of early relapse and shorter progression free and overall survival. These patients are considered the most difficult to treat MM patients, typically with a poor prognosis.
What is CD19 directed therapy?
Chimeric antigen receptor T cells (CAR-Ts) are used to treat B cell malignancies by targeting CD19, a surface marker specific for B cells. While CD19 is expressed on malignant B cells, it is also expressed on normal B cells
What is GC012F?
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.
Safety study data:
This is a single arm, open label, single-center prospective study to evaluate the safety and efficacy of GC012F as frontline therapy for transplant eligible multiple myeloma(MM) patients with high-risk profile. Patients enrolled will be newly diagnosed treatment naïve patients who are eligible for transplant.
Patients will be leukapheresed prior to receiving up to 2 cycles of standard induction therapy optional at physicians’ choice. After the optional induction therapy, patients will receive standard lymphodepleting therapy with fludarabine and cyclophosphamide prior to GC012F infusion. After a single infusion of GC012F, patients will be assessed for Response Rate and depth of response at month 1. In case CR/sCR not achieved, patients will be re-asssesd for depth of Response at month 3 post CART infusion. If at month 3 response is less than CR/sCR, patients can proceed to transplant/anti-tumor therapy according to clinical practice. In case CR/sCR is achieved at either month 1 or month 3, MRD will be assessed. In case MRD positive, patient may receive a hematological stem cell transplant (HSCT) optional or other anti-tumor therapy. Patients who obtain a CR/sCR at month 1 or month 3 according to the IMWG criteria and are assessed MRD negative response will be confirmed by PET-CT scan and they will continue to be observed. If patients obtain a MRD negative CR/sCR at month 1 then additional assessment at month 3 is not necessary. MRD will be tested when CR/sCR is achieved. At month 6 an additional response assessment will be conducted-maintenance therapy can be given post month 6 at physicians’ choice. Patients will continue to be followed and assessed for duration of response at fixed timepoints at months 12, 18 and 24. Safety and efficacy will be analyzed throughout the study at timepoints as per schedule of assessment until the end of study.
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. Leveraging its pioneering FasTCAR and TruUCAR technology platforms, Gracell is developing a rich clinical-stage pipeline of multiple autologous and allogeneic product candidates with the potential to overcome major industry challenges that persist with conventional CAR-T therapies, including lengthy manufacturing time, suboptimal production quality, high therapy cost and lack of effective CAR-T therapies for solid tumors.
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