IASO Bio’s CAR-T Eque-Cel Cleared for US Trial in Generalized Myasthenia Gravis
Eque-cel was previously evaluated in antibody-mediated idiopathic inflammatory disorders of the nervous system in an investigator-initiated trial.
Jinhua Zhang, ML
IASO Biotechnology (IASO Bio)’s equecabtagene autoleucel (eque-cel, also known as CT103A), an investigational autologous BCMA-directed chimeric antigen receptor T-cell (CAR-T) therapy, has received clearance of an investigational new drug (IND) application from the FDA for a trial in generalized myasthenia gravis (gMG).1
Eque-cel was previously evaluated in 2 patients with antibody-mediated idiopathic inflammatory disorders of the nervous system in the context of an investigator-initiated trial (NCT04561557). In these patients, clinical symptoms were reported to continue to improve over the 18 months following infusion of the CAR-T product. Furthermore, significant improvements in limb strength and vital capacity were observed at 3 months posttreatment, as were sustained improvements on instruments including the Myasthenia Gravis-Activities of Daily Living Score, Quantitative Myasthenia Gravis Score, Myasthenia Gravis-Quality of Life Score, and Modified Rankin Score.
In terms of safety, 1 of the patients experienced a grade 1 case of cytokine release syndrome (CRS). There were no other CRS cases or any cases of immune effector cell-associated neurotoxicity syndrome in the 2 patients. Within 4 weeks of receiving the CAR-T, all grade 3 or higher cases of hemocytopenia were recovered from.
One of the 2 patients was a 33-year-old woman who was positive for AChR-IgG and Titin-IgG and had been treated with a thymectomy 21 months before enrollment. She had also previously received cholinesterase inhibitors, glucocorticoids, immunosuppressants, and an antiCD20 monoclonal antibody and had not experienced clinical remission after any. The other patient was a 60-year-old woman who was positive for MuSK-IgG4. This patient, who had a 20 year disease history, had not experienced treatment success with prior use of hormones, immunosuppressants, and an antiCD20 monoclonal antibody
"We are very pleased that eque-cel has been approved for clinical trial for gMG in the United States,” Jinhua Zhang, ML, the founder and chairman of IASO Bio, said in a statement.1 “This is IASO’s first IND approval in the US to apply CAR-T therapies in autoimmune diseases. This not only represents the international recognition of our R&D strength and the potential value of our cell therapy product in the field of autoimmunity, but also an important milestone for IASO to start a new journey in the global autoimmune market. I would like to thank the clinicians who helped us conduct the clinical trial, and the patients and their families who participated in the clinical trial for their trust in us. Seeing patients with severe autoimmune diseases who have been repeatedly tormented by the disease and cannot take care of themselves return to normal life after treatment with eque-cel is the biggest motivation for our R&D effort.”
Eque-cel is also being evaluated for the treatment of relapsed/refractory (r/r) multiple myeloma (MM) and neuromyelitis optica spectrum disorder.2,3 The FDA accepted the therapy’s IND for r/r MM in December 2022 and granted it orphan drug designation for this indication in February 2022.2,4 In February 2023, the FDA additionally granted regenerative medicine advanced therapy and fast track designations to eque-cel for r/r MM.
Data from the phase 1b/2 FUMANBA-1 clinical trial (NCT05066646; ChiCTR1800018137) evaluating eque-cel in r/rMM in China were presented at the American Society of Clinical Oncology (ASCO) 2023 Annual Meeting, held June 2-6, in Chicago, Illinois.5 Among a subset of 89 patients who had not previously received treatment with a CAR-T therapy for their disease, the overall response rate was was 98.9% and the complete response (CR)/stringent CR rate was 78.7%.
“We will actively promote the clinical development of eque-cel in the US to provide a more effective and longer-lasting treatment option for MG patients around the world,” Zhang continued.1 “At the same time, we are committed to expanding breakthrough cell therapies to a broader range of autoimmune diseases, and strive to improve the global treatment landscape of such diseases so that more patients suffering from debilitating autoimmune diseases can return to a normal, healthy life.”
REFERENCES
1. IASO Bio announces U.S. FDA approval of investigational new drug application for BCMA CAR-T equecabtageneautoleucel for generalized myasthenia gravis. News release. IASO Biotechnology. April 5, 2024. Accessed April 7, 2024. https://www.iasobio.com/info.php?id=234
2. IASO Bio announces CT103A granted regenerative medicine advanced therapy (RMAT) and fast track (FT) Designations by the FDA. News release. IASO Bio. February 12, 2023. Accessed April 7, 2024. https://www.prnewswire.com/news-releases/iaso-bio-announces-ct103a-granted-regenerative-medicine-advanced-therapy-rmat-and-fast-track-ft-designations-by-the-fda-301744775.html
3.World's first CAR-T for NMOSD treatment, IASO Biotherapeutics' equecabtageneautoleucel, receives IND approval by NMPA. News release. IASO Biotherapeutics. August 19, 2022. Accessed April 7, 2024. http://www.iasobio.com/info.php?id=198
4. U.S. FDA grants orphan drug designation to BCMA CAR-T cell therapy co-developed by IASO Bio and Innovent. News Release. IASO Biotherapeutics and Innovent Biologics, Inc.; February 14, 2022. Accessed February 17, 2022. Accessed April 7, 2024. https://bit.ly/3gXGmT8
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