Cartesian Therapeutics has launched a Phase I/II clinical trial to evaluate its CAR-T candidate Descartes-08 to treat patients suffering from generalised myasthenia gravis (GMG).
GMG is a chronic, rare autoimmune disease that develops when auto-antibodies attack certain proteins at the neuro-muscular junction. The condition hinders the normal communication of nerves with muscles and leads to muscle weakness and fatigue.
Descartes-08 is a CD8+ CAR-T therapy designed to target plasma cells that express B-cell Maturation Antigen (BCMA) protein. It is engineered to have a predictable half-life.
The drug candidate is also said to allow repeat dosing to maximise its potency potential and reduce toxicity risk.
Cartesian Therapeutics chief medical officer Metin Kurtoglu said: “Descartes-08’s enhanced safety features enable use for of a wide range of diseases, from multiple myeloma and other cancers to autoimmune diseases.”
The newly initiated non-randomised, open-label, single-group Phase I/II trial will assess the safety and preliminary efficacy of Descartes-08 in around 18 patients.
Researchers at the University of Miami Health System and the Miller School of Medicine will lead the study, which is expected to be completed in 2022.
The primary outcome is the maximum tolerated dose, while secondary measures include the change in Myasthenia Gravis of Daily Living Score.
University of Miami Miller School of Medicine neurology assistant professor and principal investigator Volkan Granit said: “Patients with severe GMG have limited treatment options and are often dependent on nonselective, chronic immunosuppressive therapies (ISTs) that have long-term toxicities.
“Cartesian’s CAR-T technology selectively targets the primary culprit in the disease, antibody-producing plasma cells. Such selective targeting would be a first in GMG and could help patients discontinue use of chronic ISTs.”
In addition to GMG, Descartes-08 is in Phase I/II development to treat multiple myeloma.