Data from late-stage gMG cell therapy trial expected in early 2027
Cartesian expects to seek FDA approval for Descartes-08 by midyear
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- Descartes-08, a CAR T-cell therapy, treats generalized myasthenia gravis (gMG).
- It targets B-cells to reduce self-reactive antibodies causing muscle weakness.
- Phase 3 trial data are expected in early 2027, with FDA approval sought in mid-2027.
Top-line results from a global, late-stage clinical trial testing the experimental cell therapy Descartes-08 against a placebo in adults with generalized myasthenia gravis (gMG) are expected in the first months of 2027.
The Phase 3 study, AURORA (NCT06799247), aims to enroll about 100 adults with gMG who test positive for self-reactive antibodies targeting the acetylcholine receptor (AChR) protein, the most common type of gMG-driving antibody. Recruitment started last year and is continuing at sites across the U.S., Canada, Europe, and Turkey.
Cartesian Therapeutics, Descartes-08’s developer, said it received a credit facility of up to $150 million from K2 HealthVentures that will help fund ongoing preparations for the therapy’s potential commercial launch.
“With this additional financing, we believe we are now fully funded beyond anticipated timelines for achievement of three near-term clinical catalysts, including topline data from our Phase 3 AURORA trial in MG in the first [three months] of 2027,” Carsten Brunn, PhD, president and CEO of Cartesian, said in a company press release “We look forward to advancing Descartes-08 toward registration and commercial launch in MG.”
Assuming that AURORA’s results are positive, Cartesian expects to file for Descartes-08’s approval in the U.S. by mid-2027.
CAR T-cell therapy doesn’t require hospitalization
In gMG, the immune system wrongly produces self-reactive antibodies that block signals from nerve to muscle cells throughout the body, leading to symptoms such as muscle weakness and fatigue. The most common gMG-causing antibodies target AChR, a protein involved in nerve-to-muscle communication.
Descartes-08 is an autologous CAR T-cell therapy. This kind of treatment involves collecting T-cells (a type of immune cell) from patients, then engineering them in a lab to equip them with a human-made protein called a chimeric antigen receptor (CAR).
The therapy equips T-cells with a CAR that targets BCMA, a protein found on the surface of B-cells, the immune cells that make antibodies. The modified T-cells are then infused back into the patient, and the CAR directs them to attack and kill B-cells, with the aim of reducing levels of disease-driving antibodies to ease symptoms.
Many CAR T-cell therapies require patients to first undergo preconditioning, in which intensive treatments like chemotherapy are used to wipe out existing immune cells to make room for the therapeutic cells. Descartes-08 is designed not to require preconditioning, allowing the therapy to be given in an outpatient setting.
In an earlier Phase 2b trial, MG-001 (NCT04146051), six once-weekly Descartes-08 infusions were shown to be superior to a placebo at easing symptoms in a small number of gMG patients, including those with anti-AChR antibodies.
After a year, the single-therapy course resulted in an average 4.8-point reduction in the Myasthenia Gravis Activities of Daily Living (MG-ADL) score, a standardized scale that measures the impact of the disease on daily life. A 2-point MG-ADL score reduction is considered clinically meaningful.
“Descartes-08 remains the only CAR-T in autoimmune disease that we are aware of that is designed for outpatient administration without preconditioning chemotherapy, and our prior data demonstrate deep and durable responses after a single course of therapy,” Brunn said.
In AURORA, participants are randomly assigned to receive six once-weekly infusions of either Descartes-08 or a placebo. The study’s main goal is to assess the proportion of participants who see a score reduction of at least 3 points on the MG-ADL after four months.
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