BCMA CD19 Targeting CAR Yields Complete Lupus Remission
Eleven of 12 efficacy-evaluable patients with SLE and LN have met the DORIS criteria for complete remission.
BCMA CD19 cCAR therapy (iCell Gene Therapeutics) yielded durable, complete remission in people with systemic lupus erythematosus (SLE) with lupus nephritis (LN) in new follow-up data from an investigator-initiated phase 1 clinical trial (NCT05474885).1
The data were presented at the European Alliance of Associations for Rheumatology (EULAR) 2024 Congress, held June 12-15 in Vienna, Austria.
“It is exciting to see the continued progress over time in patients with lupus nephritis treated with iCell’s BCMA CD19 compound CAR therapy,” Yupo Ma, MD, PhD, Founder and Chief Scientific Officer,iCell Gene Therapeutics, said in a statement.1 “Humoral immunity is now recovered in all LN patients. Eleven patients are in complete remission, with all elevated autoantibodies and lupus symptoms eliminated, and the ongoing improvement in kidney function and recovery from lupus renal damage in these patients is encouraging. We are excited to progress this therapy in clinical development and plan to file investigational new drug applications (INDs) in the United States and China soon.”
The new data include mean follow-up duration for up to 20 months in patients with SLE patients, with a mean 16 months of follow-up in patients with LN. Twelve of 13 participants received the target dose of 3x106 cCAR cells/kg, including 10 with LN. Investigators found that elevated autoantibodies were eliminated in all participants that received the target dose (n = 12; efficacy population). These 12 patients scored 0 on Physician Global Assessment and have had no relapses while taking no lupus medications after BCMA CD19 cCAR treatment. All but 1 of these participants (92%) meet the DORIS criteria for complete remission.1
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All patients with LN diagnosed less than 10 years ago had a complete renal response with a mean normal 24-hour protein with over a 90% decline from screening. Two of 4 patients that were diagnosed with chronic kidney disease over 10 years had improved renal function, and 2 performed renal biopsies showed no active disease.1
BCMA CD19 cCAR treatment was well-tolerated with minimal infections including COVID-19 and 1 grade 1 case of urinary tract infection. There were no cases of cytokine release syndrome greater than grade 1, no immune effector cell-associated neurotoxicity syndrome (ICANS), and no CAR-T-cell-related encephalopathy syndrome (CRES). All patients with LN have recovered humoral immunity.1
“iCell’s BCMA CD19 compound CAR therapy is a differentiated approach and has the most mature safety database among autoimmune cell therapy candidates,” Greg Deener, Chief Executive Officer, iCell, added.1 “Our BCMA CD19 targeted cell therapy proof-of-concept study is the only trial of which we are aware that has demonstrated the safe elimination of all autoantibodies, delivering complete remission in the vast majority of patients. We are hopeful these results will translate into the breakthrough needed for patients with lupus and other autoimmune diseases.”
iCell's therapy is a dual-targeting BCMA CD19 compound chimeric antigen receptor (cCAR) T-cell therapy. The clinical trial was a single arm trial conducted in Zhongshan People’s Hospital and Peking University Shenzhen Hospital in China.
“This study demonstrates that the complete reset of humoral immunity results in elimination of all elevated autoantibodies, that long term medication free remission is achievable, and that treatment with a BCMA-CD19 compound CAR is well-tolerated in patients suffering from lupus nephritis,” Ma said in an earlier statement when initial data from the trial were published in May 2024.2 “iCell now has the largest clinical dataset of autoimmune patients receiving CAR therapy, and we are excited to continue the advancement of our BCMA-CD19 cCAR.”
REFERENCES
1. iCell Presents Positive New Follow-up Clinical Data Confirming BCMA CD19 cCAR Safely Delivers Long-term Medication Free Complete Remission from Systemic Lupus Erythematosus & Lupus Nephritis at EULAR 2024. News release. iCell Gene Therapeutics. June 12, 2024. https://www.businesswire.com/news/home/20240612055623/en/iCell-Presents-Positive-New-Follow-up-Clinical-Data-Confirming-BCMA-CD19-cCAR-Safely-Delivers-Long-term-Medication-Free-Complete-Remission-from-Systemic-Lupus-Erythematosus-Lupus-Nephritis-at-EULAR-2024
2. iCell Gene Therapeutics Announces Positive Clinical Data from Investigator Initiated Phase 1 Trial Evaluating BCMA-CD19 Compound CAR in Patients with Systemic Lupus Erythematosus/Lupus Nephritis Published in Annals of the Rheumatic Diseases. News release. iCell Gene Therapeutics. May 23, 2024https://www.businesswire.com/news/home/20240523089601/en/iCell-Gene-Therapeutics-Announces-Positive-Clinical-Data-from-Investigator-Initiated-Phase-1-Trial-Evaluating-BCMA-CD19-Compound-CAR-in-Patients-with-Systemic-Lupus-ErythematosusLupus-Nephritis-Published-in-Annals-of-the-Rheumatic-Diseases
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