Galapagos pauses CAR-T cell therapy trial over Parkinsonism case

Galapagos has paused enrollment in a trial of a BCMA-directed CAR-T cell therapy, pumping the brakes in response to an adverse event also seen in recipients of Bristol Myers Squibb and Johnson & Johnson’s rival drugs.

Belgium’s Galapagos began the phase 1/2 trial late last year to assess BCMA CAR-T candidate GLPG5301 in adults with relapsed or refractory multiple myeloma. The study is a test of both the safety and efficacy of the BCMA-directed CAR-T and the feasibility of making the autologous cell therapy at the point of care under the biotech’s seven-day vein-to-vein process.

Galapagos reported the setback as part of second-quarter results issued Thursday afternoon. The biotech put enrollment on hold after one case of Parkinsonism, movement symptoms associated with Parkinson's disease. Galapagos has filed a protocol amendment with the European Medicines Agency and expects to resume enrollment in the coming months.

Physicians have seen Parkinsonism in recipients of other BCMA-directed CAR-T cell therapies. J&J saw cases during the development of Carvykti, leading to the inclusion (PDF) of Parkinsonism as a risk in the cell therapy’s black box warning. The label for BMS’ rival therapy Abecma lacks the warning but does mention (PDF) a grade 3 Parkinsonism adverse event.

Talking on an earnings call Friday, Jeevan Shetty, M.D., Galapagos’ head of clinical development oncology, said the biotech hasn’t “seen anything in this particular patient, which was an atypical patient presentation, that is different from what is out there in the limited literature.”

Shetty said Galapagos chose to pause the study “in an abundance of caution” to enable its team to “really interrogate this particular patient history.” The interrogation included an internal review of all the patient’s characteristics and an assessment of external guidance and advice. The process has informed the establishment of “additional specific safety measures,” Shetty said.

“Moving forward, we feel very comfortable with the continuation of the study and in fact have submitted the protocol to the EMA in June, and we anticipate resuming the recruitment imminently,” the executive said.

Through the protocol changes, the “neurological component of monitoring has been further fortified,” Shetty said, and Galapagos will “more closely follow the history of patients.” The biotech plans to share data from the study in 2025.