Rese-cel clinical trial results for myasthenia gravis expected in 2026
Study testing B-cell–targeting therapy as potential MG treatment
Results from an ongoing Phase 1/2 clinical trial testing Cabaletta Bio‘s experimental cell therapy rese-cel (resecabtagene autoleucel) in adults with myasthenia gravis (MG) are expected in the second half of 2026.
That’s according to a press release from Cabaletta, where the company reported its latest financial and business updates.
The study, called RESET-MG (NCT06359041), has fully enrolled about a dozen adults with generalized MG, ages 18 to 70. All participants will receive rese-cel after undergoing a preconditioning chemotherapy regimen that clears existing immune cells to make room for the therapeutic cells.
Next steps toward larger regulatory trials
In the meantime, Cabaletta is working with the U.S. Food and Drug Administration (FDA) to align on key details for potential registrational clinical trials that could support regulatory approvals of rese-cel for MG and other autoimmune conditions.
The company hopes to have an agreement with the FDA in the first half of 2026. If successful, enrollment for a registrational MG trial could begin later that year.
MG is an autoimmune disease caused by antibodies that interfere with the communication between nerve and muscle cells. This results in symptoms like muscle weakness and fatigue.
Rese-cel, formerly known as CABA-201, is designed to kill B-cells, the immune cells that produce antibodies, including the self-reactive antibodies that drive MG and other autoimmune diseases. The therapy is expected to reduce levels of these disease-driving antibodies, ultimately easing symptoms.
The therapy works by collecting T-cells (another type of immune cell) from a patient. These cells are then engineered in a lab to harbor a chimeric antigen receptor (CAR), which is essentially a molecular weapon that targets a specific protein.
In rese-cel, the engineered CAR targets CD19, a protein found at the surface of B-cells. The modified T-cells are then infused back into the patient to seek out and destroy their B-cells.
The FDA has granted the therapy fast track designation for MG. The FDA gives this designation to therapies that have the potential to improve care for serious illnesses. The designation is meant to help speed their development and entitles their makers to more frequent communication with the FDA during the development process.
The RESET-MG study, which was cleared by the FDA in late 2023, is testing rese-cel in MG patients with some of the most common types of MG-causing antibodies, and MG patients without such antibodies.
The most common MG-causing antibodies include those targeting the acetylcholine receptor (AChR), muscle-specific kinase (MuSK), and low-density lipoprotein receptor-related protein 4 (LRP4).
Study testing single-dose treatment
Participants will receive a single weight-based dose of rese-cel following preconditioning and will be monitored for up to about three years (156 weeks).
The trial’s main goal is to evaluate the therapy’s safety up to about a month after treatment. Secondary goals include other safety measures for the remaining study period, and assessing the therapy’s pharmacological properties, as well as changes in disease-related biomarkers and standard measures of disease severity and its impact on daily life.
Cabaletta’s RESET clinical development program includes other Phase 1/2 trials testing rese-cel in other autoimmune conditions. Preliminary data from some of these studies have been promising for both safety and efficacy, suggesting rese-cel has the potential to induce durable responses without the need of other immunosuppressants.
“Our team continued to execute with discipline and precision to extend our leadership through the RESET clinical development program,” said Steven Nichtberger, MD, CEO of Cabaletta. “Rapid enrollment has resulted in multiple clinical data presentations highlighting rese-cel’s ability to deliver drug-free, transformative clinical responses for patients across multiple autoimmune diseases.”
Cabaletta is also testing rese-cel in studies that omit the preconditioning chemotherapy, since that step can often cause difficult side effects.
“The early no preconditioning data from our initial dose [group] support our plan to evaluate the efficacy and durability of rese-cel in lupus and other autoimmune patients using a single, weight-based dose without preconditioning,” Nichtberger said.
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