Gracell Biotechnologies Announces FDA Clearance of IND Application for Phase 1 Clinical Trial of FasTCAR-T GC012F as Early-Line Treatment of Multiple Myeloma

Rhea-AI Summary

Gracell Biotechnologies Inc. announced that the U.S. FDA has cleared its Investigational New Drug (IND) application, allowing the Company to initiate a Phase 1 clinical trial of GC012F in the United States for the early-line treatment of multiple myeloma (ELMM). GC012F is an autologous CAR-T therapeutic candidate dual-targeting B cell maturation antigen (BCMA) and CD19 and utilizes Gracell’s proprietary FasTCAR next-day manufacturing platform. In addition to the ELMM study, GC012F is being evaluated in an ongoing Phase 1b/2 U.S. IND study for the treatment of relapsed/refractory multiple myeloma (RRMM) and in a Phase 1/2 clinical trial for the treatment of refractory systemic lupus erythematosus (rSLE) that will initiate in 2024. GC012F is also being evaluated in four investigator-initiated trials (IIT) for the treatment of rSLE, RRMM, newly-diagnosed multiple myeloma (NDMM) and B-cell non-Hodgkin lymphoma (B-NHL). Updated clinical results from the NDMM IIT presented at the 65th American Society of Hematology Annual Meeting & Exposition in December 2023, showed GC012F demonstrated an overall response rate of 100% and minimum residual disease negative stringent complete response rate of 95.5%.

Positive

• U.S. FDA has cleared Gracell’s Investigational New Drug (IND) application for GC012F
• GC012F is being evaluated in multiple clinical trials for the treatment of various diseases
• Updated clinical results from the NDMM IIT showed promising response rates for GC012F

Negative

• None.

Oncology Doctor

The FDA's clearance of Gracell's IND for GC012F represents a significant step in the search for more effective treatments for multiple myeloma, a hematologic cancer that remains challenging to treat, particularly in its early stages. The dual-targeting approach of GC012F, which focuses on both BCMA and CD19, could potentially offer a more comprehensive treatment strategy by addressing multiple pathways of the disease's progression.

From a clinical perspective, the ability to manufacture CAR-T cells using Gracell's FasTCAR platform within a day could reduce the time patients wait for therapy, which is crucial in fast-progressing diseases. This expedited process may also lead to cost reductions, an important consideration given the typically high expenses associated with CAR-T therapies. The reported overall response rate and stringent complete response rate from the NDMM IIT are particularly encouraging, suggesting that GC012F may induce deep remissions in patients.

Medical Research Analyst

Gracell's IND clearance for GC012F in early-line multiple myeloma treatment is a pivotal development for stakeholders, as it signifies progression into a new phase of research that could broaden the therapeutic's applicability. The updated clinical results indicating high efficacy rates are promising, but it is important to note that these results are from an investigator-initiated trial, which may differ in scale and control compared to company-sponsored studies.

Long-term safety and efficacy data will be crucial for evaluating GC012F's market potential and competitive positioning. As the therapy advances through clinical trials, the data generated will be critical for investors assessing Gracell's value proposition, particularly in an industry where regulatory milestones can significantly impact a company's stock performance and perceived long-term viability.

Biotech Financial Analyst

The FDA's approval for Phase 1 trials of GC012F in the United States is a noteworthy milestone for Gracell, potentially impacting investor confidence and the company's market valuation. The progression into clinical trials for early-line treatment suggests confidence in the therapy's potential and an attempt to capture a larger share of the treatment landscape for multiple myeloma.

Investors should monitor enrollment rates and interim data releases, as these will provide insights into the trial's progress and potential for success. Furthermore, the proprietary nature of Gracell’s FasTCAR platform could present a competitive edge and if successful, may lead to licensing opportunities or partnerships, thus affecting the company's revenue streams and strategic positioning within the biotech sector.

Further expands clinical development of FasTCAR-T GC012F in multiple myeloma amid ongoing U.S. trial evaluating therapy for treatment of relapsed/refractory multiple myeloma

SAN DIEGO and SUZHOU, China and SHANGHAI, China, Jan. 29, 2024 (GLOBE NEWSWIRE) -- Gracell Biotechnologies Inc. (“Gracell” or the “Company”, NASDAQ: GRCL), a global clinical-stage biopharmaceutical company dedicated to developing innovative and highly efficacious cell therapies for the treatment of cancer and autoimmune disease, today announced that the U.S. Food and Drug Administration (FDA) has cleared Gracell’s Investigational New Drug (IND) application, allowing the Company to initiate a Phase 1 clinical trial of GC012F in the United States for the early-line treatment of multiple myeloma (ELMM).

“We are extremely pleased to receive our third U.S. IND clearance for our lead FasTCAR candidate GC012F, now expanding company-sponsored investigation into early-line multiple myeloma,” said Dr. William Cao, founder, Chairman and Chief Executive Officer of Gracell. “This IND marks an exciting milestone as we extend efforts to address significant unmet needs earlier in multiple myeloma treatment through GC012F’s unique dual-targeting approach and FasTCAR manufacturing, which have already shown great promise for deeper, durable responses. It also clears the path toward generating safety and efficacy data specifically in ELMM patients, providing us with the opportunity to better explore and define GC012F’s potential role across this initial treatment setting where there is a deep need to drive improved, sustained outcomes.”

GC012F is an autologous CAR-T therapeutic candidate dual-targeting B cell maturation antigen (BCMA) and CD19 and utilizes Gracell’s proprietary FasTCAR next-day manufacturing platform. This is the third U.S. IND clearance for GC012F. In addition to the ELMM study, GC012F is being evaluated in an ongoing Phase 1b/2 U.S. IND study for the treatment of relapsed/refractory multiple myeloma (RRMM) and in a Phase 1/2 clinical trial for the treatment of refractory systemic lupus erythematosus (rSLE) that will initiate in 2024. GC012F is also being evaluated in four investigator-initiated trials (IIT) for the treatment of rSLE, RRMM, newly-diagnosed multiple myeloma (NDMM) and B-cell non-Hodgkin lymphoma (B-NHL).

In updated clinical results from the NDMM IIT presented at the 65^th American Society of Hematology Annual Meeting & Exposition in December 2023, GC012F demonstrated an overall response rate of 100% and minimum residual disease negative stringent complete response rate of 95.5%.

About GC012F
GC012F is Gracell’s FasTCAR-enabled BCMA/CD19 dual-targeting autologous CAR-T cell therapy, which aims to transform cancer and autoimmune disease treatment by seeking to drive deep and durable responses with an improved safety profile. GC012F is currently being evaluated in clinical studies in multiple hematological cancers as well as autoimmune diseases and has demonstrated a consistently strong efficacy and safety profile. Gracell has initiated a Phase 1b/2 trial evaluating GC012F for the treatment of RRMM in the United States and a Phase 1/2 clinical trial in China is to be commenced imminently. An IIT has also been launched to evaluate GC012F for the treatment of rSLE and the IND applications to study GC012F in rSLE have been cleared by the U.S. FDA and China’s National Medical Products Administration, respectively. Additionally, a new IND application for GC012F in ELMM has also been cleared by the U.S. FDA.

About FasTCAR
Introduced in 2017, FasTCAR is Gracell’s revolutionary next-day autologous CAR-T cell manufacturing platform. FasTCAR is designed to lead the next generation of therapy for cancer and autoimmune diseases, and improve outcomes for patients by enhancing effect, reducing costs, and enabling more patients to access critical CAR-T treatment. FasTCAR drastically shortens cell production from weeks to overnight, potentially reducing patient wait times and probability for their disease to progress. Furthermore, FasTCAR T-cells appear younger than traditional CAR-T cells, making them more proliferative and effective at killing cancer cells. In 2022 and 2023, FasTCAR was named the winner of the Biotech Innovation category of the 2022 Fierce Life Sciences Innovation Awards and the Overall Immunology Solution of 2023 by BioTech Breakthrough Awards, for its ability to address major industry obstacles.

About Gracell
Gracell Biotechnologies Inc. (“Gracell”) is a global clinical-stage biopharmaceutical company dedicated to discovering and developing breakthrough cell therapies for the treatment of cancers and autoimmune diseases. Leveraging its innovative FasTCAR and TruUCAR technology platforms and SMART CART™ technology module, 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 cell quality, high therapy cost, and lack of effective CAR-T therapies for solid tumors and autoimmune diseases. The lead candidate BCMA/CD19 dual-targeting FasTCAR-T GC012F is currently being evaluated in clinical studies for the treatment of multiple myeloma, B-NHL and SLE. For more information on Gracell, please visit www.gracellbio.com. Follow @GracellBio on LinkedIn.

What is the purpose of Gracell's IND application clearance by the U.S. FDA?

The clearance allows Gracell to initiate a Phase 1 clinical trial of GC012F in the United States for the early-line treatment of multiple myeloma (ELMM).

What is GC012F?

GC012F is an autologous CAR-T therapeutic candidate dual-targeting B cell maturation antigen (BCMA) and CD19 and utilizes Gracell’s proprietary FasTCAR next-day manufacturing platform.

In which clinical trials is GC012F being evaluated?

GC012F is being evaluated in an ongoing Phase 1b/2 U.S. IND study for the treatment of relapsed/refractory multiple myeloma (RRMM) and in a Phase 1/2 clinical trial for the treatment of refractory systemic lupus erythematosus (rSLE) that will initiate in 2024. GC012F is also being evaluated in four investigator-initiated trials (IIT) for the treatment of rSLE, RRMM, newly-diagnosed multiple myeloma (NDMM) and B-cell non-Hodgkin lymphoma (B-NHL).

What were the clinical results from the NDMM IIT presented at the 65th American Society of Hematology Annual Meeting & Exposition in December 2023?

Updated clinical results from the NDMM IIT showed GC012F demonstrated an overall response rate of 100% and minimum residual disease negative stringent complete response rate of 95.5%.