Cartesian Therapeutics’ mRNA CAR-T therapy met its primary endpoint in a mid-stage trial for the chronic autoimmune disorder and expects to raise $130 million via private placement equity financing.
Cartesian Therapeutics announced Tuesday its lead mRNA CAR-T therapy candidate met its primary endpoint in a Phase IIb trial for the rare, muscle-weakening disorder myasthenia gravis, and separately that it has entered into an agreement for a private investment in public equity financing expected to gross $130 million.
In the trial, Cartesian’s Descartes-08 CAR-T therapy proved effective in lessening symptoms in 71% of trial patients with myasthenia gravis (MG) compared to 25% on placebo. In the study, 10 out of 14 patients from the pre-specified efficacy population showed an improvement of at least five points in their MG composite (MGC) score, which measures disease severity and impact. A three-point improvement is generally considered clinically meaningful, according to the company.
In addition, 11 out of 16 (69%) in the pre-protocol population met the primary endpoint compared to 33% in the placebo group, with “deep improvements” across the MG severity scales reported at month three.
Descartes-08 also showed a favorable safety profile. A significant benefit of the therapy compared to most cell therapy methods is that Cartesian’s candidate does not require lymphodepleting chemotherapy before treatment, according to the company. The feature also allows for outpatient administration.
There were no cases of cytokine release syndrome, an inflammatory response that occurs with between 37% and 93% frequency following CAR-T cell therapy. The FDA granted Regenerative Medicine Advanced Therapy designation to Descartes-08 for MG in May 2024.
“We believe the positive data presented today demonstrate clinical proof-of-concept of our novel mRNA platform and highlight the potential of Descartes-08 to provide deep and durable improvements for patients with MG,” CEO Carsten Brunn said in a statement, adding the biotech has plans to hold a meeting with the FDA by the end of the year.
The current standard of care for MG includes chronic use of steroids and immunosuppressants with often limited efficacy. The rare autoimmune disease destroys the communication between nerves and muscles, weakening voluntary muscle control.
Johnson & Johnson picked up a monoclonal antibody for autoimmune disease in its 2020 $6.5 billion buy of Momenta Therapeutics. The company touted positive Phase III results in generalized MG just days ago and shared plans to submit to regulatory authorities later this year.
Cartesian’s mRNA cell therapy is being tested in additional autoimmune indications. The company announced Tuesday it dosed its first systematic lupus erythematosus patient in a Phase II trial of Descartes-08.
Separately, the biotech also announced $130 million in private investment in public equity (PIPE) financing from both new and existing investors. The proceeds will fund its pipeline programs and be used for general corporate purposes. Cartesian was listed on the Nasdaq in November 2023 via a reverse merger with Selecta Biosciences.
