FDA Grants Regenerative Medicine Advanced Therapy Designation for AIC100 to Treat Thyroid Cancer

AIC100 could meet an unmet medical need to effectively treat individuals with anaplastic thyroid cancer — the most aggressive form of the disease.

The FDA has granted regenerative medicine advanced therapy (RMAT) designation for AIC100 (Affylmmune Therapeutics), an ICAM-1 targeting and affinity-tuned LFA-1 binder chimeric antigen receptor (CAR) T-cell therapy product candidate, for the treatment of recurrent anaplastic thyroid cancer (ATC).¹ This form of thyroid cancer is reported to be a rare and highly aggressive malignant tumor that impacts 2% to 3% of all thyroid gland neoplasms. Study authors noted that ATC remains as the deadliest disease worldwide with few effective treatment options, with a median survival rate of less than 6 months.^2,3,4

Doctor palpates of fat mature woman's neck for diagnostics of thyroid diseases and hypothyroidism at medical clinic. Thyroid treatment - Image credit: Peakstock | stock.adobe.com

Image credit: Peakstock | stock.adobe.com

“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, CEO at AffyImmune, in a news 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. We believe the RMAT designation reinforces the potential ability of AIC100 to meet the high unmet medical need in recurrent ATC, an aggressive disease where a standard of care is currently not available.”¹

About the Trial

Trial Name: Study of AIC100 CAR T Cells in Relapsed/Refractory Thyroid Cancer

ClinicalTrials.gov ID: NCT04420754

Sponsor: AffyImmune Therapeutics, Inc.

Completion Date (Estimated): August 2030

The phase 1 study assessed effective treatment options among individuals with advanced differentiated thyroid cancer (ADTC) and ATC, evaluating the safety and efficacy of AIC100. The data was presented at the American Society of Clinical Oncology (ASCO) 2024 Annual Meeting.⁴

“We are excited to be the first to demonstrate the potential of autologous CAR T to induce a complete response in a patient with a solid tumor cancer. The confirmed mCR in the AIC100 phase 1 study is the first known complete response following a single IV dose of autologous CAR T cells observed in any solid tumor cancer type,” said Matt Britz, CEO, AffyImmune, in a news release.⁴

The study included 10 individuals—6 with ATC and 4 with poorly differentiated thyroid cancer, that were infused with 3 dose levels (DLs) of AIC100. The study authors noted that among all DLs, no dose limiting toxicities were displayed and the maximum tolerated dose was not reached. Among individuals in DL2 and DL3, the overall response rate was 33%. However, 1 individual in DR2 displayed durable partial response as another individual in DL3 displayed an ongoing durable metabolic complete response.⁴

Further results displayed that 60% of individuals developed grade 1/2 cytokine release syndrome, but no occurrence of immune effector cell-associated neurotoxicity syndrome or other serious adverse events were reported with the use of AIC100.⁴

“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 anti-tumor activity observed in this phase 1 study provide a strong foundation to advance this candidate further in development,” said Samer A. Srour, MB ChB, MS, the trial’s lead investigator and assistant professor at The University of Texas MD Anderson Cancer Center, in a news release.⁴

The findings suggest that AIC100 could meet an unmet medical need to effectively treat individuals with ATC—the most aggressive form of the disease.^1,4

“We remain focused on advancing the development of AIC100 for patients and families living with this devastating cancer,” said Sonal Gupta, MD, PhD, senior vice president and head of clinical development, in a news release. “Receiving RMAT designation helps facilitate this goal by enabling increased dialogue with the FDA to expedite our development plan for our affinity-tuned CAR T therapy. We look forward to working closely with the FDA and other regulatory agencies as we continue to advance this program.”¹

REFERENCES

1. AffyImmune Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for AIC100 in Recurrent Anaplastic Thyroid Cancer. Business Wire. News release. July 23, 2024. Accessed July 24, 2024. https://www.businesswire.com/news/home/20240723305679/en.

2. Anaplastic Thyroid Cancer. StatPearls. News release. January 1, 2023. Accessed July 24, 2024. https://www.ncbi.nlm.nih.gov/books/NBK538179/.

3. Recurrent thyroid cancer with changing histologic features. National Library of Medicine. News release. July 23, 2010. Accessed July 24, 2024. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2900987/.

4. AffyImmune Announces Confirmed Complete Response in Patient with Anaplastic Thyroid Cancer. Business Wire. News release. May 23, 2024. Accessed July 24, 2024. https://www.businesswire.com/news/home/20240523653892/en/.