CAR T–focused biotech Cargo Therapeutics surprised and disappointed analysts when it announced that it would discontinue a mid-stage trial of its lead program, firi-cel.
Cargo Therapeutics is a little lighter—on both the pipeline and workforce front—as the CAR T–focused biotech announced late Wednesday that it is discontinuing a mid-stage study of its lead candidate and parting with approximately half of its employees.
Jefferies analysts in an investor note Thursday called the news a “surprise disappointment” as the Phase I study had showed a 50% complete response rate and long multi-year durability of firicabtagene autoleucel (firi-cel)—an autologous CD22 CAR T cell therapy.
The San Carlos, Calif.–based company had been studying firi-cel in a Phase II trial for patients with large B cell lymphoma (LBCL) whose disease is relapsed or was refractory to CD19 CAR T cell therapy. In a press release, Cargo revealed it had conducted an ad hoc analysis of the FIRCE-1 study “prompted by recent safety events” and ultimately concluded that the results did not support a competitive benefit-risk profile in this patient population.
While treatment with firi-cel led to an overall response rate of 77% and complete response rate (CR) of 43%, durability of the CR at three months was just 18%, according to Cargo. This was “considerably lower” than in the Phase I study, according to a William Blair investor note issued Thursday.
“We are disappointed the efficacy of firi-cel in the Phase II trial was unable to replicate what was observed in the Phase I Stanford study,” the analysts wrote. “We look forward to additional details on baseline characteristics and CD22 expression in these patients to better understand why their responses were not durable.”
Additionally, 18% of participants developed immune effector cell–associated hemophagocytic lymphohistiocytosis-like syndrome—a toxicity associated with CAR T cell therapy—at grade 3 or higher, according to Cargo.
While Cargo didn’t specifically say it was shelving firi-cel, it mentioned no further development plans.
Cargo’s pipeline also includes a tri-specific CAR T named CRG-023, which the company plans to advance into a Phase I dose escalation study. Enrollment for this study is on track to begin the second quarter of this year, according to Cargo.
The biotech, which raised $200 million in an oversubscribed series A round in March 2023, will also progress its allogeneic platform to lead vector candidate selection while “evaluating its strategic options.”
Cargo had preliminary cash, cash equivalents and marketable securities of $368.1 million as of Dec. 31, 2024. With the newly announced workforce reduction, the company expects to extend its cash runway into mid-2028.
