Allogene Therapeutics Gains on FDA Fast Track, RMAT Status for Lymphoma Drug

Shares of Allogene Therapeutics Inc. (NASDAQ:ALLO) rose 2.7% on Wednesday after the U.S. Food and Drug Administration (FDA) granted two significant regulatory designations to its experimental CAR T therapy, cemacabtagene ansegedleucel (cema-cel).

Regulatory Milestone and Trial Data

The FDA granted both Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations for cema-cel. These designations are intended to expedite the development and review of therapies for serious conditions and are based on the potential to address unmet medical needs. They allow for more frequent engagement with the FDA throughout the clinical development process.

The agency's decision was supported by a review of interim data from Allogene's ongoing ALPHA3 trial. The study evaluates cema-cel in adult patients with large B-cell lymphoma (LBCL) who show minimal residual disease (MRD) after first-line therapy. Key findings from the data cutoff include:

• MRD Clearance: 58.3% of patients in the cema-cel arm achieved MRD negativity, compared to just 16.7% in the observation arm, marking a 41.6% absolute difference.
• ctDNA Reduction: Patients receiving cema-cel showed a 97.7% median decrease in plasma ctDNA, a measure of tumor fragments, versus a 26.6% median increase in the observation arm.

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Favorable Safety Profile

Allogene reported a favorable safety profile for cema-cel as of the data cutoff. The company stated there were no treatment-related serious adverse events. Notably, there were no reported cases of common CAR T-related toxicities such as cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), or graft-versus-host disease.

Furthermore, no patients required hospitalization for treatment-related adverse events, with the therapy being managed primarily in an outpatient setting. This safety profile is a key differentiator for allogeneic, or "off-the-shelf," CAR T therapies compared to autologous treatments that require using a patient's own cells.