New CAR T Granted FDA RMAT Designation for Anaplastic Thyroid Cancer

• The FDA has granted AIC100 regenerative medicine advanced therapy (RMAT) designation for the treatment of recurrent anaplastic thyroid cancer.
• AIC100 is a chimeric antigen receptor (CAR) T-cell product.
• RMAT designation is intended to expedite the development and review of regenerative medicines that treat, modify, reverse, or cure serious conditions.

AIC100, a novel CAR T-cell therapy, has been granted a RMAT designation from the FDA in anaplastic thyroid cancer.¹

RMAT designation is designed to expedite the development and review of regenerative medicines that are intended to treat serious conditions. RMAT designation can provide more interaction with the FDA.

“We believe this important recognition from the FDA further supports the therapeutic potential of AIC100 to change the current treatment paradigm in advanced thyroid cancer and potentially other forms of aggressive solid tumors,” said Daniel Janse, PhD, chief executive officer at AffyImmune, in a press release. “RMAT designation was granted following the FDA’s review of safety and efficacy data from the first 10 patients dosed with AIC100 in our phase 1 study [NCT04420754]. We believe the RMAT designation reinforces the potential ability of AIC100 to meet the high unmet medical need in recurrent [anaplastic thyroid cancer], an aggressive disease where a standard of care is currently not available.”

About the Phase 1 Study of AIC100

Findings from the phase 1 study were presented at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting.² Ten patients were treated with AIC100 at 3 dose levels. One patient with anaplastic thyroid cancer who received 500 million CAR T cells achieved a metabolic complete response. The overall response rate for patients at dose levels 2 and 3 receiving 100 million or 500 million CAR T cells, respectively, was 33%, and the disease control rate was 67%. One patient receiving 100 million CAR T cells achieved a durable partial response.

There were no dose-limiting toxicities reported, and the maximum tolerated dose was not reached. Six patients developed grade 1/2 cytokine release syndrome. There were no reports of immune effector cell-associated neurotoxicity syndrome or serious adverse events.

“These findings represent a significant milestone in the development of AIC100 as a potential treatment option for patients with advanced thyroid cancers. The safety profile and promising antitumor activity observed in this phase 1 study provide a strong foundation to advance this candidate further in development,” said Samer A. Srour, MD, the trial’s lead investigator and assistant professor at The University of Texas MD Anderson Cancer Center, in the press release.1 “We look forward to improve on these results and to further evaluate the safety and efficacy of AIC100 at higher dose levels and to potentially expand its application to other ICAM-1-positive cancers.”

REFERENCES:

1. AffyImmune receives FDA regenerative medicine advanced therapy (RMAT) designation for AIC100 in recurrent anaplastic thyroid cancer. News release. AffyImmune. July 23, 2024. Accessed July 23, 2024. https://tinyurl.com/yztpyv29

2. AffyImmune announces confirmed complete response in patient with anaplastic thyroid cancer. News release. AffyImmune. May 23, 2024. Accessed July 23, 2024. https://tinyurl.com/yckjwtts