Cartesian Therapeutics, Inc. (United States)

Clarivate's Drugs to Watch list highlights 11 promising drugs, including Novo Nordisk's Awiqli and CagriSema, Bristol Myers Squibb's Cobenfy, Eli Lilly and Almirall's Ebglyss, Sanofi and Alnylam's fitusiran, GSK's GSK-3536819, Amgen's Imdelltra, Moderna's mResvia, Sobi and Cartesian Therapeutics/Selecta Biosciences' SEL-212, Arvinas and Pfizer's vepdegestrant, and Exelexis' zanzalintinib, with potential to transform treatments and achieve significant sales by 2030.

December 2024 saw significant updates in cell and gene therapies: Cilta-cel showed higher MRD negativity in multiple myeloma; Descartes-08 produced durable responses in myasthenia gravis; Bendamustine was effective with fewer AEs in LBCL; Beti-cel demonstrated long-term curative potential in β-thalassemia; AURN001 showed efficacy and safety in corneal edema treatment.

Cell therapy, particularly CAR-T, is advancing in treating autoimmune diseases, showing promise in early trials for conditions like lupus. Despite no approvals yet, its potential for curative effects excites the field. Companies like Kyverna, Cabaletta, and Cartesian are leading research, with some patients experiencing life-changing results. However, experts caution about overhyping, emphasizing the need for long-term data and recognizing that cell therapy won't work for all patients.

The Myasthenia Gravis Treatment Market is set to grow significantly by 2034, driven by improved diagnostics, emerging therapies, and increased awareness. Key companies like Horizon Therapeutics and Amgen are developing novel treatments. The market size reached USD 4,950 million in 2023, with the U.S. seeing 129 thousand cases.

RNA therapy offers precision targeting, rapid development, broad therapeutic potential, non-permanent effects, and customization for personalized medicine. Clinical trials highlight RNA therapy's promise in treating genetic disorders, cancers, and infectious diseases, with mRNA vaccines demonstrating agility during COVID-19. Advances in RNA delivery systems and platform technology enhance scalability and versatility, paving the way for effective treatments.

Cartesian Therapeutics’ Descartes-08 CAR-T therapy for MG showed durable responses in a phase 2b trial, with significant reductions in MG-ADL scores. The therapy was well-tolerated, with no severe adverse events. A phase 3 trial, AURORA, is planned for next year, aiming to confirm these findings in a larger patient population.

Cartesian Therapeutics reports Phase IIb trial data for Descartes-08 in MG, showing durable responses at month 12. The trial included 36 pre-treated patients, with Descartes-08-treated patients experiencing deepening responses over time. Cartesian plans to start Phase III study AURORA in 2025.

Cartesian Therapeutics announces updated efficacy and safety data from Phase 2b trial of Descartes-08 for myasthenia gravis, with deepening responses observed over time and a durable safety profile. The company plans to commence Phase 3 AURORA trial in 1H2025, aiming to assess improvements in MG-ADL scores at Month 4.

Cartesian Therapeutics maintains strong short-term liquidity with a current ratio of 10.7, though analysts do not anticipate profitability this year. Descartes-08, an mRNA CAR-T therapy, showed a 5.5-point reduction in MG-ADL score at Month 4 in Phase 2b trial, with no safety issues reported. The Phase 3 AURORA trial will assess a three-point improvement in MG-ADL score at Month 4. Cartesian also converted Series B Non-Voting Convertible Preferred Stock into common stock, resulting in 23,893,525 shares of Common Stock. Mizuho reaffirmed its Outperform rating on Cartesian, highlighting Descartes-08's competitive edge.