Cartesian Secures Phase II Success in Myasthenia Gravis, Seeks $130M Funding

Cartesian Therapeutics revealed that their lead mRNA CAR-T therapy candidate, Descartes-08, achieved its primary goal in a Phase IIb trial for myasthenia gravis, a disorder characterized by muscle weakness. The company also secured a $130 million private investment in public equity financing.

In the clinical trial, Descartes-08 demonstrated effectiveness in reducing symptoms in 71% of patients with myasthenia gravis compared to 25% in the placebo group. Specifically, 10 out of 14 patients in the pre-specified efficacy group showed an improvement of at least five points in their MG composite (MGC) score, a measure of disease severity. A three-point improvement is generally deemed clinically meaningful. Additionally, 11 out of 16 patients in the pre-protocol group met the primary endpoint, compared to 33% in the placebo group, with significant improvements noted in the MG severity scales at the three-month mark.

Descartes-08 also demonstrated a favorable safety profile. Unlike most cell therapies, Cartesian’s candidate does not require lymphodepleting chemotherapy prior to treatment, which allows for outpatient administration. Notably, there were no instances of cytokine release syndrome, an inflammatory response that occurs frequently with CAR-T cell therapies. The FDA granted Regenerative Medicine Advanced Therapy designation to Descartes-08 for myasthenia gravis in May 2024.

CEO Carsten Brunn stated the positive data indicate clinical proof-of-concept for their novel mRNA platform and underscore the potential of Descartes-08 to offer significant and lasting improvements for patients with myasthenia gravis. The company plans to meet with the FDA by year-end.

The current standard treatments for myasthenia gravis involve chronic use of steroids and immunosuppressants, which often have limited effectiveness. This rare autoimmune disease disrupts communication between nerves and muscles, leading to weakened muscle control.

In related news, Johnson & Johnson acquired a monoclonal antibody for autoimmune disease in a $6.5 billion purchase of Momenta Therapeutics in 2020. The company recently reported positive Phase III results for generalized myasthenia gravis and plans to seek regulatory approval later this year.

Cartesian’s mRNA cell therapy is also being evaluated for other autoimmune conditions. The company announced it has dosed its first patient with systematic lupus erythematosus in a Phase II trial of Descartes-08.

Additionally, Cartesian announced $130 million in private investment in public equity (PIPE) financing from both new and existing investors. These funds will support its pipeline programs and general corporate purposes. Cartesian was listed on the Nasdaq in November 2023 through a reverse merger with Selecta Biosciences.

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