On March 7, 2024 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, 2023, and recent corporate updates (Press release, Cartesian Therapeutics, MAR 7, 2024, View Source [SID1234640917]).

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"With several potentially value-creating milestones anticipated throughout the year ahead, we are making strong progress in our mission to deliver innovative cell therapies to patients suffering from autoimmune diseases," said Carsten Brunn, Ph.D., President and Chief Executive Officer of Cartesian. "For our lead product candidate, Descartes-08, we continue to expect to report topline data from the ongoing Phase 2b trial in patients with myasthenia gravis (MG) mid-year. We believe this represents the most advanced and only randomized, controlled Phase 2 trial of a chimeric antigen receptor (CAR) T-cell therapy for autoimmune diseases."

Dr. Brunn continued, "Beyond MG, we continue to expect to initiate a Phase 2 study in patients with systemic lupus erythematosus (SLE) in the first half of the year. Supported by the clinical dataset from the previously completed Phase 2a study in patients with MG, we believe that Descartes-08, which we engineer with our novel mRNA engineered CAR-T (mRNA CAR-T) technology, does not require preconditioning chemotherapy, and is expected to be administered in an outpatient setting, could serve as the first CAR-T cell therapy to reach patients with autoimmune diseases."

Recent Pipeline Progress and Anticipated Milestones

Topline Data from Randomized Phase 2b Study of Descartes-08 in Patients with MG Expected for Mid-2024. Enrollment remains ongoing in the Company’s Phase 2b randomized, double-blind, placebo-controlled trial of Descartes-08 in patients with MG (NCT04146051), with topline results expected in mid-2024.
Announced Positive Long-Term Follow-Up Data from Phase 2a Study of Descartes-08 in Patients with MG. In January 2024, Cartesian announced positive twelve-month follow-up data from its Phase 2a study of Descartes-08, the Company’s autologous anti-B cell maturation antigen (BCMA) mRNA CAR-T cell therapy product candidate, in patients with generalized MG, a chronic autoimmune disorder that causes disabling muscle weakness and fatigue. In this study, Descartes-08 was administered in an outpatient setting without integrating vectors or preconditioning chemotherapy, and durable depletion of autoantibodies and clinically meaningful improvements in MG severity scores during the one-year follow-up period were observed. Descartes-08 was observed to be well-tolerated, with no dose-limiting toxicities, cytokine release syndrome, or neurotoxicity.
Descartes-08 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of MG.

Initiation of Phase 2 Study of Descartes-08 in Patients with SLE Expected for First Half of 2024. The Company expects to initiate a Phase 2 study of Descartes-08 in patients with SLE (NCT06038474) in the first half of 2024. The Phase 2 study, for which the Company has received investigational new drug (IND) clearance, is designed to assess the safety and tolerability of outpatient Descartes-08 administration without preconditioning chemotherapy. SLE is an incurable autoimmune disease marked by systemic inflammation that affects multiple organ systems. It is estimated to impact approximately 1.5 million people in the United States.
IND Application Cleared for Descartes-15. In January 2024, Cartesian announced that the FDA cleared its IND application for Descartes-15, a next-generation, autologous anti-BCMA mRNA CAR-T product candidate. As with Descartes-08, Descartes-15 is designed not to require preconditioning chemotherapy, has been observed to have predictable and controllable pharmacokinetics and is designed to avoid the risk of genomic integration. Planning for the first-in-human Phase 1 dose escalation study is underway. The study is designed to assess the safety and tolerability of outpatient Descartes-15 administration in patients with multiple myeloma. The Company expects to subsequently assess Descartes-15 in autoimmune indications.
Corporate Update

Upcoming Special Meeting of Stockholders Seeking to Approve Preferred Stock Conversion and Reverse Stock Split. Cartesian plans to hold a special meeting of stockholders on March 27, 2024 to seek stockholder approval for the conversion of the Company’s Series A Non-Voting Convertible Preferred Stock into the Company’s common stock and a reverse stock split at a ratio in the range of 1-for-20 and 1-for-30. The reverse stock split would impact all holders of Cartesian common stock proportionally and would not impact any stockholder’s percentage ownership of common stock. Holders of common stock of record as of February 13, 2024 are eligible to vote at the meeting.
Announced Plans to Transition Corporate Headquarters to Frederick, Maryland. The Company recently announced plans to transition its corporate headquarters to Frederick, Maryland. The approximately 20,000 square foot state-of-the-art current good manufacturing practice (cGMP) compliant facility has clinical and commercial manufacturing scale capabilities designed to support the Company’s maturing pipeline.
Completed Merger with Selecta Biosciences, Inc. and Concurrent $60.25 Million Private Financing. In November 2023, Cartesian announced its merger with Selecta Biosciences, Inc., creating a fully integrated, publicly traded company pioneering mRNA cell therapy for the treatment of autoimmune diseases. In connection with the merger, Cartesian announced a $60.25 million private financing led by Timothy A. Springer, Ph.D.
Full Year 2023 Financial Results

Pro forma cash, cash equivalents, and restricted cash of approximately $118.3 million as of December 31, 2023, which reflects net proceeds received in the first quarter of 2024 from the November 2023 financing. The Company’s pro forma cash and cash equivalents as of December 31, 2023, is expected to support planned operations and the development of Cartesian’s pipeline into the second half of 2026, through the Phase 3 study of lead candidate, Descartes-08.

Research and development expenses were $71.8 million for the year ended December 31, 2023, compared to $72.4 million for the year ended December 31, 2022. The decrease in expense of $0.6 million for the year ended December 31, 2023 was primarily due to reductions in expenses incurred for preclinical and clinical programs due to the strategic reprioritization partially offset by expenses incurred for stock compensation and personnel expenses.
General and administrative expenses were $40.6 million for the year ended December 31, 2023, compared to $23.9 million for the year ended December 31, 2022. The increase in expense of $16.7 million for the year ended December 31, 2023 was primarily due to expenses incurred for stock compensation, personnel expenses, and professional fees incurred in connection with the merger.
Net loss was $(219.7) million, or $(1.66) net loss per share (basic/diluted), for the year ended December 31, 2023, compared to net income of $35.4 million, or $0.24 net income per share (basic), for the year ended December 31, 2022.