AffyImmune Gets FDA RMAT Designation for AIC100 in Recurrent Anaplastic Thyroid Cancer

AffyImmune, a biotechnology company in the clinical-stage focusing on pioneering chimeric antigen receptor (CAR) T cell therapies, has achieved a significant milestone. The U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to AffyImmune’s CAR T-cell product candidate, AIC100. This treatment shows promise for patients with recurrent anaplastic thyroid cancer (ATC), a notably aggressive form of the disease.

Daniel Janse, Ph.D., CEO of AffyImmune, highlighted the importance of this recognition. He stated that the FDA’s acknowledgment underscores AIC100’s potential to revolutionize the treatment landscape for advanced thyroid cancer and possibly other aggressive solid tumors. The RMAT designation was awarded based on safety and efficacy data from the first ten patients treated with AIC100 in a Phase 1 study. This status supports the belief that AIC100 could fulfill the unmet medical needs in recurrent ATC, a condition lacking a current standard of care.

The RMAT designation is aimed at speeding up the development and review processes for regenerative medicine therapies. A therapy qualifies for this designation if it is intended to treat, modify, reverse, or cure a serious condition and if preliminary clinical evidence suggests it can address the unmet medical needs for the disease. Companies with RMAT status gain advantages such as increased interaction with the FDA, including access to senior managers, to hasten drug development.

Sonal Gupta, M.D., Ph.D., Senior Vice President and Head of Clinical Development at AffyImmune, emphasized their commitment to advancing AIC100 for those affected by this severe cancer. The RMAT designation enables enhanced dialogue with the FDA, facilitating quicker development plans for their carefully tuned CAR T therapy. Gupta stressed their eagerness to collaborate closely with the FDA and other regulatory bodies as they move forward with the program.

At the ASCO 2024 conference, AffyImmune presented interim results from their Phase 1 study, which evaluates the safety and efficacy of AIC100. This therapy targets ICAM-1 and binds with affinity to LFA-1 in patients with advanced thyroid cancer. A notable achievement from this study is the metabolic complete response observed in a patient with ATC, the most aggressive form of thyroid cancer.

AffyImmune is a clinical-stage biopharmaceutical company based in Natick, Massachusetts, dedicated to developing a pioneering ICAM-1 targeting and affinity-tuned LFA-1 binder CAR T therapy. Their aim is to transform the lives of patients with advanced solid cancers. The company’s proprietary technology leverages ICAM-1/LFA-1 interactions to potentially overcome critical CAR T challenges such as toxicity, antigen loss, T-cell trafficking, and exhaustion. This technology also allows in vivo tracking of CAR T cells. The leading product, AIC100, is currently in Phase 1 trials to treat advanced thyroid cancers, including advanced differentiated thyroid cancer and ATC. AffyImmune is focused on creating a specialized pipeline targeting ICAM-1-positive cancers with significant unmet medical needs.