The US Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to AffyImmune Therapeutics’ CAR T-cell therapy, AIC100, for the treatment of recurrent anaplastic thyroid cancer (ATC). This designation follows the FDA’s thorough review of the available initial clinical data, which showcases AIC100’s potential to treat this aggressive form of thyroid cancer effectively.
The RMAT designation is a significant regulatory milestone aimed at expediting the development and review of regenerative medicine therapies that exhibit the potential to fulfill unmet medical needs. To qualify for this designation, a regenerative medicine therapy must be intended to treat, modify, reverse, or cure a serious condition. Moreover, preliminary clinical evidence must indicate that the therapy could address unmet medical needs specific to the targeted disease.
AffyImmune’s AIC100, an affinity-tuned LFA-1 binder CAR T-cell therapy, has shown promising outcomes in interim results from their Phase I study. This therapy specifically targets ICAM-1, a molecule involved in the aggressive behavior of certain cancer cells. The initial data from this trial have been encouraging, with one patient achieving a metabolic complete response, indicating the therapy’s potential efficacy.
AffyImmune’s AIC100 Receives RMAT Designation, Promising Advances in Thyroid Cancer Treatment
The RMAT designation provides several benefits to AffyImmune, including increased opportunities for interaction with the FDA. These interactions are designed to streamline the drug development and review process, ensuring that promising therapies can reach patients more quickly. The FDA’s commitment to expediting these interactions underscores the importance of advancing innovative treatments that address critical unmet needs.
AffyImmune’s CEO, Daniel Janse, expressed his enthusiasm about the designation, stating, “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. RMAT designation was granted following the FDA’s review of safety and efficacy data from the first ten patients dosed with AIC100 in our Phase I study. 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.”
The interim data from the Phase I clinical trial assessing the safety and efficacy of AIC100 highlight its potential. The trial’s focus on comprehensive endpoints, including safety, efficacy, and patient outcomes, has provided valuable insights into the therapy’s impact. Particular attention has been given to the operationalization of severe and life-threatening bleeding events, joint function, and patient-reported outcomes (PROs). The need for standardized definitions and appropriate tolerance windows to prevent missing data has been emphasized in the study protocol (SP) and statistical analysis plan (SAP).
Ensuring Comprehensive Safety and Data Integrity in AffyImmune’s AIC100 Thyroid Cancer Treatment
The collection of adverse events (AEs), including serious adverse events (SAEs), is crucial to ensure a comprehensive safety profile for AIC100. The current SP and SAP need to include all events leading to hospitalization or death, not just those directly related to Hemophilia treatment. This comprehensive approach will provide a clearer picture of the treatment’s safety profile. To ensure the completeness and reliability of the data, AffyImmune must implement robust training programs for healthcare providers involved in the study. This includes regular site monitoring visits and mandatory data fields in the German Hemophilia Registry (DHR). The SP must clearly outline these measures to ensure consistent data collection across all study centers.
The statistical methods used in the study must be clearly defined and justified. The G-BA has recommended using a shifted hypothesis threshold to account for the non-randomized study design. The pU must also conduct sensitivity analyses to address potential biases and ensure the robustness of the study results. A critical aspect of the study is the collection of baseline data. The SP must specify that all inclusion and exclusion criteria are operationalized at the index date. For patients transitioning to Valoctocogen Roxaparvovec during the study, baseline data must be re-collected to maintain data integrity.
Additionally, the identification and management of confounders require a systematic literature review and expert consultation. This process must be thoroughly documented in the SP to ensure all relevant confounders are considered in the analysis.
The FDA’s RMAT designation is a testament to the promising potential of AffyImmune’s AIC100 in addressing the unmet needs of patients with recurrent anaplastic thyroid cancer. This recognition underscores the significance of AffyImmune’s innovative approach and provides a pathway to bring this potentially life-saving treatment to patients more efficiently. With a continued commitment to rigorous evaluation and data integrity, AffyImmune is poised to make a substantial impact on the treatment landscape for advanced thyroid cancer and potentially other aggressive solid tumors.
Resource: Pharmaceutical Business Review, July 24, 2024
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