On February 27, 2025 Arcellx, Inc. (NASDAQ: ACLX), a biotechnology company reimagining cell therapy through the development of innovative immunotherapies for patients with cancer and other incurable diseases, reported business highlights and financial results for the fourth quarter and year ended December 31, 2024 (Press release, Arcellx, FEB 27, 2025, View Source [SID1234650687]).
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"2024 was a transformational year for Arcellx as our ASH (Free ASH Whitepaper) data presentations for anito-cel, in partnership with Kite, continued to demonstrate anito-cel’s differentiated clinical profile for the potential treatment of patients with relapsed or refractory multiple myeloma," said Rami Elghandour, Arcellx’s Chairman and Chief Executive Officer. "In our Phase 1 and iMMagine-1 studies, data demonstrate that anito-cel has the potential to provide a meaningful benefit to a broad population of patients. Our Phase 1 study demonstrated that the deep responses observed with anito-cel also translated to durable benefit for patients with a 30.2 month median progression-free survival in a challenging patient cohort and the preliminary data from our pivotal iMMagine-1 study demonstrated anito-cel also delivered deep responses for late line myeloma patients with a 97% overall response rate and 62% complete response rate at a median follow-up of 9.5 months. Most notably, as presented during ASH (Free ASH Whitepaper), the safety profile for anito-cel continues to be manageable with no delayed neurotoxicities, including no Parkinsonism, no cranial nerve palsies, and no Guillain-Barré syndrome in the155 patients dosed across the Phase 1 and iMMagine-1 studies. Physician engagement and enthusiasm for anito-cel continues to build following these data presentations and as more sites gain experience with anito-cel through iMMagine-3, our earlier-line study. Kite is manufacturing for iMMagine-3, and turnaround times are consistent with Kite commercial products. This year, we look forward to presenting updated data from our iMMagine-1 study mid-year, preparing for the commercial launch of anito-cel in multiple myeloma in 2026, enrolling our program in generalized myasthenia gravis, and expanding our ARC-SparX program in acute myeloid leukemia to include an additional SparX antigen target. Thank you to our Arcellx team who are willing to embrace challenges and think creatively to make an impact for the patients we serve."
Recent Business Progress
Presented positive preliminary data for the Phase 2 pivotal iMMagine-1 study of anito-cel in patients with relapsed or refractory multiple myeloma (RRMM) at the 66th ASH (Free ASH Whitepaper) Annual Meeting and Exposition. The data presented on December 9, 2024 demonstrated deep and durable responses with a predictable and manageable safety profile in a high-risk fourth-line or higher (4L+) RRMM population, including triple- and penta-class refractory disease. The data were from an October 31, 2024 data cutoff date, with a median follow-up of 9.5 months for the 86 patients who were evaluable for efficacy based on a follow-up of at least two months after treatment with anito-cel, and 98 patients were evaluable for safety based on a follow-up of at least one month after treatment with anito-cel.
Overall response rate was 97% (83/86) with a complete response/stringent complete response rate of 62% (53/86) and a very good partial response or higher rate of 81% (70/86), per International Myeloma Working Group (IMWG) criteria as investigator-assessed. Of those evaluable for minimal residual disease (MRD) testing, 93.1% (54/58) achieved MRD negativity at a minimum of 10 -5 sensitivity. Median progression-free survival (mPFS) and overall survival (OS) were not reached; 6-month PFS and OS rates were 93.3% and 96.5%, respectively, and 12-month PFS and OS rates were 78.5% and 96.5%, respectively. No delayed or non-ICANS neurotoxicities, including no Parkinsonism, no cranial nerve palsies, and no Guillain-Barré syndrome, were observed in the 155 patients dosed with anito-cel.
Fourth Quarter and Full Year 2024 Financial Highlights
Cash, cash equivalents, and marketable securities:
As of December 31, 2024, Arcellx had cash, cash equivalents, and marketable securities of $625.7 million. Arcellx anticipates that its cash, cash equivalents, and marketable securities will fund its operations into 2027.
Collaboration revenue:
Collaboration revenue were $15.3 million and $63.1 million for the quarters ended December 31, 2024 and 2023, respectively, a decrease of $47.8 million. This decrease was primarily driven by the December 2023 expansion to the license and collaboration agreement with Kite Pharma, Inc. Collaboration revenue were $107.9 million and $110.3 million for the twelve months ended December 31, 2024 and 2023, respectively, a decrease of $2.4 million.
R&D expenses:
Research and development expenses were $44.6 million and $28.8 million for the quarters ended December 31, 2024 and 2023, respectively, an increase of $15.8 million. Research and development expenses were $157.1 million and $133.8 million for the twelve months ended December 31, 2024 and 2023, respectively, an increase of $23.3 million. The increases were primarily driven by increased personnel costs, which include non-cash stock-based compensation expense, and increased costs relating to anito-cel and other pipeline programs.
G&A expenses:
General and administrative expenses were $23.8 million and $19.4 million for the quarters ended December 31, 2024 and 2023, respectively, an increase of $4.4 million. General and administrative expenses were $88.4 million and $66.4 million for the twelve months ended December 31, 2024 and 2023, respectively, an increase of $22.0 million. The increases were primarily driven by increased personnel costs, which include non-cash stock-based compensation expense, increased costs relating to commercial readiness, and increased depreciation expense.
Net losses:
Net losses were $47.1 million and $19.8 million for the quarters ended December 31, 2024 and 2023, respectively. Net losses were $107.3 million and $70.7 million for the twelve months ended December 31, 2024 and 2023, respectively.
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. Multiple myeloma 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.