On March 13, 2025 Cartesian Therapeutics, Inc. (NASDAQ: RNAC) (the "Company"), a clinical-stage biotechnology company pioneering mRNA cell therapy for autoimmune diseases, reported financial results for the full year ended December 31, 2024, and outlined recent corporate updates (Press release, Cartesian Therapeutics, MAR 13, 2025, View Source [SID1234651131]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Following a year marked by tremendous progress, we remain committed to advancing our pipeline of mRNA cell therapies designed to be dosed in the convenient outpatient setting, without the need for preconditioning chemotherapy," said Carsten Brunn, Ph.D., President and Chief Executive Officer of Cartesian. "Notably, we remain on track to commence our planned Phase 3 AURORA trial of Descartes-08 in patients with myasthenia gravis (MG) in the first half of this year. Supported by positive results from our Phase 2b trial demonstrating deep and durable improvements for Descartes-08-treated participants, along with our Special Protocol Assessment (SPA) agreement with U.S. Food and Drug Administration (FDA), we are confident that we have a clear path toward potential approval of this promising new therapy."

Dr. Brunn continued, "Beyond MG, we remain on track to report preliminary data from our ongoing Phase 2 open-label trial of Descartes-08 in patients with systemic lupus erythematosus (SLE) and expect to initiate our Phase 2 pediatric basket trial of Descartes-08 in select autoimmune diseases in the second half of this year. With these anticipated milestones, along with our strong balance sheet, we believe we are well-positioned to deliver on our mission to expand the reach of cell therapy to autoimmunity."

Recent Pipeline Progress and Anticipated Milestones

•Phase 3 AURORA Trial of Descartes-08 in MG on Track to Commence in the First Half of 2025. The randomized, double-blind, placebo-controlled Phase 3 AURORA trial is designed to assess Descartes-08 versus placebo (1:1 randomization) administered as six once weekly outpatient infusions without preconditioning chemotherapy in approximately 100 participants with acetylcholine receptor autoantibody positive (AChR Ab+) MG. The primary endpoint will assess the proportion of Descartes-08 participants with an improvement in MG Activities of Daily Living (MG-ADL) score of three points or more at Month 4 compared to placebo. Descartes-08, Cartesian’s lead product candidate, is an autologous anti-B cell maturation antigen (BCMA) mRNA-engineered chimeric antigen receptor T-cell therapy (mRNA CAR-T).
In January 2025, Cartesian announced that it has received written agreement from the FDA under the SPA process on the overall design of the planned Phase 3 AURORA trial. The SPA agreement indicates that the FDA has determined that the proposed trial design is acceptable to support a future Biologics License Application for Descartes-08 in MG, subject to the ultimate outcome of the trial.

•Announced Positive Updated Results from Phase 2b Trial of Descartes-08 in Participants with MG, with Deepening Response Observed Over Time, Durable Through Month 12. In December 2024, the Company announced updated efficacy and safety data from the Phase 2b trial of Descartes-08 in participants with MG.

Participants included in the primary efficacy dataset (n=12)1 experienced an average MG-ADL reduction of 5.5 (±1.1) at Month 4. Responses were observed to be durable through Month 12, with 80% (4/5) of evaluable participants from the primary efficacy dataset maintaining a clinically meaningful response, defined as a reduction in MG-ADL score of at least 2 points. Descartes-08 continues to be observed as well-tolerated, supporting outpatient administration without the need for lymphodepleting chemotherapy.

•Dr. Tuan Vu, one of Cartesian’s Clinical Advisors, will Present at the American Academy of Neurology Annual Meeting on April 9, 2025 at 1:12 pm PT. The presentation and abstract are titled, "The Efficacy and Safety of Autologous BCMA-directed mRNA CAR T-Cell Therapy in Generalized Myasthenia Gravis: Results from a Phase 2b Randomized Placebo-controlled Trial."

•Preliminary Data from Ongoing Phase 2 Open-Label Trial of Descartes-08 in Patients with SLE Expected in the Second Half of 2025. The trial is designed to assess the safety, tolerability and clinical activity of outpatient Descartes-08 administration without preconditioning chemotherapy in patients with SLE. SLE is an incurable autoimmune disease marked by systemic inflammation that affects multiple organ systems and impacts approximately 1.5 million people in the United States.

•Phase 2 Pediatric Basket Trial of Descartes-08 in Select Autoimmune Diseases Expected to Initiate in the Second Half of 2025. This pediatric basket trial will target juvenile SLE, juvenile MG, juvenile dermatomyositis (JDM) and anti-neutrophil cytoplasmic antibody (ANCA) associated vasculitis. The FDA previously granted Rare Pediatric Disease Designation to Descartes-08 for the treatment of JDM, which is a rare pediatric autoimmune disorder.

•Dosing Ongoing in First-in-Human Phase 1 Clinical Trial of Descartes-15. The Phase 1 dose escalation trial of Cartesian’s next-generation, autologous anti-BCMA mRNA CAR-T cell therapy, is designed to assess the safety and tolerability of outpatient Descartes-15 administration in patients with multiple myeloma. Following the Phase 1 dose escalation trial, the Company expects to subsequently assess Descartes-15 in autoimmune indications.

Corporate Updates

•Emily English Promoted to Chief Operations Officer. Emily English, formerly Cartesian’s Senior Vice President and Head of Manufacturing Operations, was promoted to Chief Operations Officer in January 2025. Emily’s significant contributions, including her leadership in the expansion of the new, state-of-the-art current good manufacturing practice (cGMP) facility in Frederick, Maryland, have been instrumental in Cartesian’s progress as the Company continues to advance its pipeline.

Full Year 2024 Financial Results

•Cash, cash equivalents and restricted cash as of December 31, 2024 was $214.3 million and is expected to support planned operations, including completion of planned Phase 3 AURORA trial for Descartes-08 in MG, into mid-2027.

•Research and development expenses were $45.1 million for the year ended December 31, 2024, compared to $71.3 million for the year ended December 31, 2023. The decrease was primarily a result of the Company’s restructuring in 2023 prior to the merger between Cartesian and Selecta Biosciences, including reductions in expenses for preclinical and clinical programs due to the strategic reprioritization, stock compensation in connection with the Selecta Biosciences/ Cartesian Therapeutics merger, and higher expenses in 2023 compared to 2024 due to one-time cash charges related to salaries and benefits.

•General and administrative expenses were $30.1 million for the year ended December 31, 2024, compared to $40.5 million for the year ended December 31, 2023. The decrease in expense for the year ended December 31, 2024 was primarily the result of reductions in expenses incurred for stock compensation and professional fees in connection with the merger between Cartesian and Selecta Biosciences.

•Net loss was $(77.4) million, or $(4.48) net loss per share (basic), for the year ended December 31, 2024, compared to net loss of $(219.7) million, or $(49.76) net loss per share (basic), for the year ended December 31, 2023.

About Descartes-08

Descartes-08, Cartesian’s lead mRNA cell therapy candidate, is an autologous mRNA-engineered chimeric antigen receptor T-cell therapy (mRNA CAR-T) product targeting B-cell maturation antigen (BCMA) in clinical development for generalized myasthenia gravis (MG) and systemic lupus erythematosus. In contrast to conventional DNA-based CAR T-cell therapies, mRNA CAR-T administration is designed to not require preconditioning chemotherapy, can be administered in the outpatient setting, and does not carry the risk of genomic integration associated with cancerous transformation. Descartes-08 has been granted Orphan Drug Designation and Regenerative Medicine Advanced Therapy Designation by the U.S. Food and Drug Administration for the treatment of MG, and Rare Pediatric Disease Designation for the treatment of juvenile dermatomyositis.

About Descartes-15

Descartes-15 is a next-generation, autologous anti-BCMA mRNA CAR-T cell therapy. In preclinical studies, Descartes-15 has been observed to achieve an approximately ten-fold increase in CAR expression and selective target-specific killing, relative to Descartes-08. Similar to Descartes-08, Descartes-15 is designed to be administered without preconditioning chemotherapy and does not use integrating vectors.