Data from trial of experimental CAR T-cell therapy for gMG due in early 2027
Developer plans to seek FDA approval should results be positive
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- Global Phase 3 clinical trial data for Descartes-08, an experimental CAR T-cell therapy for gMG, are expected in early 2027.
- Descartes-08 targets antibody-producing B-cells as an outpatient treatment without requiring preconditioning chemotherapy.
- Should results be positive, its developer expects to submit a biologics license application to the FDA by mid-2027.
Results from a global clinical trial testing the experimental CAR T-cell therapy Descartes-08 in people with generalized myasthenia gravis (gMG) are expected in the first few months of next year, according to its developer, Cartesian Therapeutics.
The Phase 3 AURORA trial (NCT06799247), which began enrolling last year, is comparing Descartes-08 with a placebo in up to 100 adults with gMG who are positive for self-reactive antibodies targeting the acetylcholine receptor (AChR) protein, the most common type of gMG-driving antibody.
Should results be positive, Cartesian expects to submit, by the middle of next year, a biologics license application (BLA) to the U.S. Food and Drug Administration seeking Descartes-08’s approval for gMG. Meanwhile, the company is preparing for the therapy’s potential launch on the market.
“With Phase 3 AURORA data expected [between January and March] of 2027, our focus … remains on execution, both in the clinic and in preparing the organization for a commercial launch,” Carsten Brunn, PhD, president and CEO of Cartesian, said in a company press release. “Ahead of our anticipated BLA submission in mid-2027, we have expanded our medical affairs and commercial teams to drive launch readiness.”
No preconditioning chemotherapy required
In gMG, self-reactive antibodies, most commonly those targeting AChR, impair communication between nerves and muscles, leading to symptoms such as muscle weakness and fatigue.
Descartes-08 is designed to eliminate B-cells, the immune cells responsible for producing antibodies, including those that drive gMG. Treatment involves collecting a patient’s T-cells, another type of immune cell, and modifying them in the lab to carry a chimeric antigen receptor (CAR) protein that recognizes BCMA, a protein found at the surface of B-cells. Once infused back into the patient, these modified T-cells seek out and destroy B-cells.
Unlike conventional CAR T-cell therapies, Descartes-08 does not require patients to undergo preconditioning chemotherapy, which is typically given before treatment to reduce existing immune cells and make room for the therapeutic cells. This allows the infusion to be administered in an outpatient setting, without hospitalization.
In AURORA, participants are randomly assigned to receive Descartes-08 or a placebo as six once-weekly outpatient infusions. Neither participants nor researchers know who receives which treatment.
The trial’s main goal is to determine whether significantly more Descartes-08-treated participants achieve a clinically meaningful reduction of at least three points on the MG Activities of Daily Living (MG-ADL) scale after four months, compared with those given the placebo. The MG-ADL scale assesses the disease’s impact on everyday activities, with lower scores indicating a lower impact.
Earlier trial showed encouraging findings
AURORA follows encouraging findings from the Phase 2b MG-001 trial (NCT04146051), which tested Descartes-08 against a placebo in 36 gMG patients, most of whom tested positive for anti-AChR antibodies.
At three months, significantly more Descartes-08-treated participants achieved a reduction of at least five points on the Myasthenia Gravis Composite (MGC), a measure of disease severity, than those given the placebo (66.7% vs. 27.3%). The difference was statistically significant, meeting the trial’s main goal. Group differences were maintained for up to one year.
A subsequent analysis involving the 19 participants with anti-AChR antibodies showed that 63% of those receiving Descartes-08 achieved a five-point drop in the MGC score within three months, compared with 12.5% of those on the placebo. This difference was also statistically significant.
Descartes-08 was also associated with clinically meaningful score reductions in other measures of disease severity, including the MG-ADL scale, which were maintained for up to a year.
We believe this profile has the potential to meaningfully change how MG is treated.
The most common side effects were infusion-related reactions, which generally resolved within two days. No adverse events were reported from month three onward.
In addition, data from five trial participants who received a second treatment course due to symptom worsening showed that, one year after retreatment, MGC scores fell an average of 15 points and MG-ADL scores dropped by 6.6 points. Four of these patients maintained clinically meaningful MG-ADL score reductions through that year. No new safety concerns were identified with retreatment.
“With larger teams on the ground now, we are excited by the opportunity to expand awareness of Descartes-08’s differentiation, particularly on the back of the retreatment data … which continued to demonstrate deep and durable responses with a consistent safety profile, all with outpatient administration and no preconditioning chemotherapy,” Brunn said. “We believe this profile has the potential to meaningfully change how MG is treated.”
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