FDA Places MELANI-01 Study for Multiple Myeloma on Hold After Safety Report Submission

The FDA placed the MELANI-01 trial on hold following the submission of a safety report, according to a Cellectis press release.

The safety report showed that a patient, who enrolled in the study at dose level 2, with relapsed and refractory multiple myeloma, experienced a fatal treatment-emergent adverse event of cardiac arrest, resulting in the trial’s clinical hold. The patient also underwent numerous prior therapies including autologous CAR T-cells.

“We share the FDA’s commitment to patient safety and are working collaboratively with the agency and the investigators to resolve this clinical hold,” said Carrie Brownstein, MD, Chief Medical Officer, Cellectis, in a press release. “The safety of patients enrolled in our clinical trials is our utmost priority and we at Cellectis remain committed to safely resuming the clinical development of UCART product candidate targeting CS1 for patients with multiple myeloma and unmet medical need.”

Before the FDA enacted the clinical hold, Cellectis expanded enrollment on the trial for dose level 1. This was thought to be the appropriate dose level for further evaluation, specifically in the expansion portion of the trial and the recommended phase 2 dose based on preliminary assessments of the data. With this, Cellectis had begun updating its clinical protocol to reflect the change and to also monitor and mitigate any other potential risks.

As it stands, Cellectis is working closely with the FDA to adhere to the agency’s requests, specifically by changing the MELANI-01 trial’s clinical protocol to ultimately enhance patient safety. More, Cellectis expects to submit “requested information including an amended protocol in due course.”

MELANI-01 is a phase 1 open-label dose escalation trial that examines UCARTCS1A to treat patients with relapsed or refractory multiple myeloma. The product candidate, UCARTCS1A, is an allogenic, edited T-cell product designed to treat CS1/SLAMF7-expressing hematologic malignancies.

As for the 2 other phase 1 dose escalation trials under Cellectis, patient enrollment is ongoing. The AMELI-01 trial is evaluating UCART123 in relapsed and refractory acute myeloid leukemia, while the BALLI-01 trial will evaluate UCART22 in relapsed and refractory B-cell acute lymphoblastic leukemia.

The American Cancer Society estimates that over 32,000 new cases of multiple myeloma will be diagnosed, with almost 13,000 new deaths in the United States because of the disease expected to occur in 2019.

Reference:

Cellectis Reports Clinical Hold Placed on MELANI-01 Study [news release]. New York, New York. Published July 6, 2020. https://www.cellectis.com/en/press/cellectis-reports-clinical-hold-placed-on-melani-01-study/. Accessed July 7, 2020.