Updated: FDA gives Cellectis all clear after patient death halted off-the-shelf CAR-T trial – Endpoints News

Four months after a patient death forced Cellectis to halt one of their off-the-shelf CAR-T trials, the FDA has given them the OK to start dosing patients again.

The Paris-based biotech said they changed the studys protocol to accommodate the agencys concerns, although they will still have to work with investigators to obtain local approval to restart the trial and start recruiting patients again. Prior its halt, the Phase I had sites in New York, New Jersey and Texas.

Cellectis did not disclose what the changes were, but after the initial hold, analysts suggested the company might monitor patient cytokine levels more closely, swap out of one of the chemotherapy drugs used or prevent patients who have previously received an experimental multiple myeloma CAR-T from enrolling.

The companys stock {CLLS} rose 5.6% on the news, from $19.70 to $20.80.

Though partnered with the new upstarts such as Arie Belldegruns Allogene, Cellectis, a 21-year-old company, has been working on off-the-shelf versions to the first CAR-Ts since before those CAR-Ts were approved.

One of three different solo projects now in the clinic, the halted study, MELANI-01, is testing a cell therapy for multiple myeloma. If effective, it could serve as a potentially easier way to manufacture and administer an alternative to the multiple myeloma CAR-Ts now nearing approval from J&J and Legend Biotech and from bluebird bio and Bristol Myers Squibb.

When they announced the hold, Cellectis CMO Carrie Brownstein said that the company had already independently decided to move forward with dose level 1 in the study. The patient who died had been the first to receive dose level 2, an infusion of 3 million cells per kilogram. They developed cytokine release syndrome, the hallmark side effect of CAR-T therapy, and died of cardiac arrest 25 days later.

The dose 1 level involves 1 million cells per kilogram.

The FDA has been on high alert for safety issues in cell therapy trials, and this wasnt the first time theyve paused a Cellectis study. Back in 2017, another patient death paused development of UCART123.

After some protocol adjustments, the therapy was allowed to go back into patients. It is now in a Phase I for acute myeloid leukemia.

Updated: FDA gives Cellectis all clear after patient death halted off-the-shelf CAR-T trial - Endpoints News

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