Anitocabtagene autoleucel

A new FDA approval was based on phase 3 results showing Darzalex Faspro significantly delayed disease progression to multiple myeloma or death by 51% versus monitoring. For the first time, patients with abnormal cells detected in their bone marrow have a treatment option before the condition develops into multiple myeloma.The FDA has approved Johnson & Johnson’s Darzalex subcutaneous formulation to treat patients with smoldering multiple myeloma (SMM) that is at a high risk of developing into multiple myeloma.Before Thursday’s approval, doctors could only monitor patients with SMM, which is an asymptomatic intermediate disease state, in hopes of catching early signs of the condition’s development into cancerous MM.More than 35,000 people were estimated to be diagnosed with myeloma in the U.S. in 2024, with about 15% of new diagnoses classified as SMM, according to J&J. About 50% of high-risk SMM cases are likely to progress into active MM within two to three years.The approval was based on results from a phase 3 trial called Aquila, which showed that Darzalex Faspro significantly delayed disease progression to MM or death by 51% versus basic monitoring. The median progression-free survival time was not reached in the Darzalex arm, versus a median result of 41.5 months in the control group.The FDA initially had hesitations about the company’s application, raising doubts about whether more mature patient survival data were needed to determine Darzalex’s benefit-risk profile for an asymptomatic precursor condition, especially since the CD38 antibody is well established as a powerful treatment for multiple myeloma.In approving Darzalex, the FDA followed the recommendation of an external advisory committee. Advisors to the agency said they were convinced by results from the Aquila trial, including a preliminary but clear sign of a trend toward improved survival, although they still urged J&J to follow the study to further characterize Darzalex’s profile in SMM.A step ahead of the FDA, European regulators had already approved Darzalex in SMM in July. The approval of Darzalex in SMM offers drugmakers some relief, because the drug’s proposed use had previously drawn criticism from Vinay Prasad, M.D., before he became the FDA’s chief medical and scientific officer as well as director of the Center for Biologics Evaluation and Research. Prasad has been known as a proponent of overall survival serving as the most important endpoint to determine a drug’s approvability. In a December 2024 post, he criticized the lack of patient exposure to Darzalex as a subsequent treatment for patients in Aquila’s control arm.Although Darzalex’s review fell within the purview of Richard Pazdur, M.D., who leads the FDA’s Oncology Center of Excellence, Prasad’s chief medical and scientific officer title at the agency theoretically could give him power to intervene.The Darzalex approval in SMM serves as proof that Prasad is “taking a more holistic approach to some approvals,” Everecore ISI analyst Cory Kasimov wrote in a Thursday note.In the MM field, Kasimov viewed the nod as a positive sign for Arcellx and Gilead Sciences’ upcoming accelerated approval bid for their BCMA CAR-T therapy anito-cel. Since the partners plan to seek accelerated approval based on the phase 2 IMMagine-1 trial, the drug won’t have randomized OS data to back its case. As a cell therapy, anito-cel lands with Prasad’s department, with expected meaningful input from Pazdur’s team as well. 

REDWOOD CITY, Calif.--(BUSINESS WIRE)--Arcellx, Inc. (NASDAQ: ACLX), a biotechnology company reimagining cell therapy through the development of innovative immunotherapies for patients with cancer and other incurable diseases, today announced it will share two presentations at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition taking place December 6-9, 2025, in Orlando, Florida, including updated clinical data from iMMagine-1, its Phase 2 pivotal study (publication #256) of anitocabtagene autoleucel (anito-cel) in patients with relapsed and/or refractory multiple myeloma (RRMM). Additionally, an abstract (publication #7644) describing the fast off-rate of anito-cel’s D-Domain binder will be published in a supplemental issue of Blood in November 2025. The company will also have a medical affairs booth (#1363) at the Orange County Convention Center. “We’re pleased to share that, along with our partners at Kite, we conducted our pre-BLA meeting with the FDA and remain confident in our planned 2026 commercial launch of anito-cel,” said Rami Elghandour, Arcellx’s Chairman and Chief Executive Officer. “This milestone marks a significant step toward bringing our innovative therapy to the multiple myeloma community. We look forward to sharing updated clinical data from our iMMagine-1 study in an oral presentation at ASH. These are exciting times at Arcellx!” ASH Presentation Details: Title: Phase 2 registrational study of anitocabtagene autoleucel for the treatment of patients with relapsed and/or refractory multiple myeloma: Updated results from iMMagine-1 Speaker: Krina K. Patel, MD, MSc, MD Anderson Cancer Center Session Name: 655. Multiple Myeloma: Cellular Therapies: Clinical Trial Advances in CAR T-Cell Therapy for Multiple Myeloma Session Date: Saturday, December 6, 2025 Session Time: 2:00 p.m. – 3:30 p.m. ET Presentation Time: 2:45 p.m. ET Location: OCCC – West Hall E1 Publication Number: 256 Submission ID: abs25-4541 Title: Visualizing geographic variation and systemic inequities of disease burden and CAR T-cell therapy access in multiple myeloma in the US Speaker: Brandon Blue, MD, Moffitt Cancer Center Session Name: 907. Outcomes Research: Plasma Cell Disorders: Poster III Session Date: Monday, December 8, 2025 Session Time: 6:00 p.m. – 8:00 p.m. ET Location: OCCC – West Halls B3-B4 Publication Number: 6344 Submission ID: abs25-2093 Title: The fast off-rate of anito-cel’s D-Domain binder contributes to its distinctive pharmacology profile in preclinical models of multiple myeloma Disposition: Online Publication Publication Number: 7644 Submission ID: abs25-1695 About Multiple Myeloma Multiple Myeloma (MM) is a type of hematological cancer in which diseased plasma cells proliferate and accumulate in the bone marrow, crowding out healthy blood cells and causing bone lesions, loss of bone density, and bone fractures. These abnormal plasma cells also produce excessive quantities of an abnormal immunoglobulin fragment, called a myeloma protein (M protein), causing kidney damage and impairing the patient’s immune function. MM is the third most common hematological malignancy in the United States and Europe, representing approximately 10% of all hematological cancer cases and 20% of deaths due to hematological malignancies. The median age of patients at diagnosis is 69 years with one-third of patients diagnosed at an age of at least 75 years. Because MM tends to afflict patients at an advanced stage of life, patients often have multiple co-morbidities and toxicities that can quickly escalate and become life-endangering. About Anitocabtagene Autoleucel (anito-cel) Anitocabtagene autoleucel (anito-cel, previously CART-ddBCMA) is the first BCMA-directed CAR T-cell therapy to be investigated in multiple myeloma that utilizes Arcellx’s novel and compact binder known as the D-Domain. The small, stable D-Domain binder enables high CAR expression without tonic signaling and is designed to quickly release from the BCMA target. This combination may allow for the effective elimination of multiple myeloma cells without severe immunotoxicity. Anito-cel has been granted Fast Track, Orphan Drug, and Regenerative Medicine Advanced Therapy Designations by the U.S. Food and Drug Administration. About Arcellx and Kite Collaboration Arcellx and Kite, a Gilead Company, formed a global strategic collaboration and license agreement to co-develop and co-commercialize anito-cel for patients with multiple myeloma. Anito-cel is currently being developed in a Phase 2 registrational pivotal study and a global Phase 3 randomized controlled study for relapsed and/or refractory multiple myeloma (RRMM). Kite and Arcellx will jointly commercialize the anito-cel asset in the United States, and Kite will commercialize the product outside the United States. About Arcellx, Inc. Arcellx, Inc. is a clinical-stage biotechnology company reimagining cell therapy by engineering innovative immunotherapies for patients with cancer and other incurable diseases. Arcellx believes that cell therapies are one of the forward pillars of medicine and Arcellx's mission is to advance humanity by developing cell therapies that are safer, more effective, and more broadly accessible. For more information on Arcellx, please visit www.arcellx.com. Follow Arcellx on X @arcellx and LinkedIn. Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. All statements in this press release that are not purely historical are forward-looking statements, including, without limitation, statements regarding: anticipated announcements of additional data; anito-cel pharmacology profile, tolerability and toxicity trends; the potential of anito-cel for providing meaningful benefit in patients suffering from multiple myeloma; the potential commercial launch of anito-cel, subject to FDA approval, in partnership with Kite; and the potential impact of Arcellx’s product candidates and platforms on patients and cell therapy. The forward-looking statements contained herein are based upon Arcellx’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. These forward-looking statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, including risks that may be found in the section entitled Part II, Item 1A (Risk Factors) in the Quarterly Report on Form 10-Q for the fiscal quarter ended June 30, 2025, filed with the Securities and Exchange Commission (SEC) on August 7, 2025, and the other documents that Arcellx may file from time to time with the SEC. These forward-looking statements are made as of the date of this press release, and Arcellx assumes no obligation to update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise, except as required by law.