FDA Grants Fast Track Status to CAR T-Cell Product AIC100 for Thyroid Cancer

The FDA has granted a fast track designation to the CAR T-cell product AIC100 for the treatment of patients with anaplastic thyroid cancer and refractory poorly differentiated thyroid cancer.

The FDA has granted a fast track designation to the CAR T-cell product AIC100 for the treatment of patients with anaplastic thyroid cancer and refractory poorly differentiated thyroid cancer, according to an announcement from AffyImmune Therapeutics, Inc., the drug developer.1

“We are pleased to have received fast track designation for our first-in-human CAR T-cell product currently being tested in patients with refractory thyroid cancer,” Eric von Hofe, president and chief operating officer of AffyImmune, stated in a press release. “It highlights the unmet need in treating refractory solid tumors and points to the potential of AIC100 to address that need. We look forward to a close relationship with the FDA to expedite development and future approvals.”

The third-generation CAR T-cell therapy has an affinity to ICAM-1 that is comparable to the affinity noted between naturally-occurring T cells and their targets, which is much lower than the majority of the CARs that have been used to date.2

Because of this, the product may result in less off-target adverse effects. Moreover, the therapy also expresses SSTR2, which allows for real-time monitoring of CAR T-cell distribution and activity; this could also serve as a safety and activation switch with the incorporation of certain FDA-approved agents, according to the biopharmaceutical company.

The product is currently under evaluation in a phase 1 trial (NCT04420754), which is being conducted at Weill Cornell Medicine and other centers. Here, investigators are working to identify the safety and recommended dose of the product in patients with advanced thyroid cancer or anaplastic thyroid cancer.3

To be eligible for enrollment, patients need to be at least 18 years of age and have either anaplastic thyroid cancer that is BRAF wild-type at any stage, including newly diagnosed; anaplastic thyroid cancer that is BRAF mutated following failure of BRAF-specific therapy; or poorly differentiated thyroid cancer in which surgery, radioactive iodine, chemotherapy, radiation therapy, and targeted therapies have failed.

Moreover, patients need to have measurable disease, an ECOG performance stats of 0 to 2, and a life expectancy of greater than 8 weeks.

If patients are pregnant or breastfeeding; have active, uncontrolled systemic infections; previously received investigational gene therapy or CAR T-cell therapy; have active or clinically relevant central nervous system disorder; evidence of another malignancy within 2 years before screening; or seropositive response of human immunodeficiency virus or uncontrolled hepatitis B or C virus, they will be excluded.

The primary outcome measures for the trial include incidence of overall grade 3 to 5 toxicities and incidence of CAR T–related toxicities.4 Secondary outcome measures include detection, expansion, and persistence of AIC100 cells following infusion; analysis of CAR T subsets via flow cytometry in peripheral blood; assessment of CAR T-cell infiltrate in the tumor per biopsy at treatment completion and/or the time of disease progression; cytokine levels in plasma samples; and CAR T antibodies in the peripheral blood.

The first patient was enrolled to the trial in October 2020, after overcoming COVID-19–related delays.5

The product is also slated to be evaluated in gastric cancer and triple-negative breast cancer. Investigators also hope to further examine AIC100 plus PD-1/cytokines for potential use in solid tumors.


  1. AffyImmune Therapeutics’ AIC100 granted fast track designation for treating thyroid cancer. News release. AffyImmune Therapeutics, Inc. May 17, 2021. Accessed May 19, 2021. https://prn.to/3yrEWrx
  2. AffyImmune is developing a leading pipeline of affinity-tuned CAR T-cell therapies to treat solid tumors. AffyImmune Therapeutics, Inc website. Accessed May 19, 2021. https://bit.ly/3eZddXG
  3. A multi center phase I study of AIC100 in relapsed and/or refractory advanced thyroid cancer and anaplastic thyroid cancer. Weill Cornell Medicine website. Accessed May 19, 2021. https://bit.ly/3yopHjf
  4. Study of AIC100 in relapsed/refractory thyroid cancer. ClinicalTrials.gov. Updated January 12, 2021. Accessed May 19, 2021. https://clinicaltrials.gov/ct2/show/NCT04420754
  5. AffyImmune Therapeutics announces first patient enrolled in phase I clinical trial of AIC100 CAR T cells for the treatment of advanced, refractory thyroid cancer. News release. AffyImmune Therapeutics, Inc. October 29, 2020. Accessed May 19, 2021. https://prn.to/2RvDrbk