AbelZeta reveals early anti-tumor activity of C-CAR031 in advanced HCC at ASCO 2024

AbelZeta Pharma, Inc., a global biopharmaceutical company focused on innovative cell-based therapies, recently revealed promising preliminary results from its first human trial of C-CAR031. This trial, conducted in collaboration with AstraZeneca, targeted patients with advanced hepatocellular carcinoma (HCC). The findings were presented at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting.

C-CAR031, an autologous CAR-T therapy, is designed to target GPC3, a protein highly expressed in HCC. Developed by AstraZeneca and manufactured by AbelZeta, this therapy aims to offer new hope for patients with limited treatment options. The trial's primary goal was to assess both safety and anti-tumor efficacy in patients who had undergone multiple prior therapies.

Tony (Bizuo) Liu, CEO of AbelZeta, expressed optimism about the trial's initial outcomes. He highlighted the potential of C-CAR031 to redefine treatment approaches for HCC and other GPC3-positive tumors. Principal Investigator Professor Tingbo Liang from Zhejiang University echoed this sentiment, noting the significant tumor reduction observed in the majority of patients, which suggests substantial clinical value.

As of March 14, 2024, 24 patients had received C-CAR031 infusions across four dose levels (DLs). The median follow-up period was 9.03 months. Among these patients, 91.3% saw tumor reductions, with a median reduction of 42.2%. The overall disease control rate (DCR) was 91.3%, and the objective response rate (ORR) was 56.5%. Notably, the ORR at the highest dose level (DL4) reached 75.0%. Median overall survival (mOS) was estimated at 11.14 months.

The safety profile of C-CAR031 was also encouraging. No dose-limiting toxicities or immune effector cell-associated neurotoxicity syndromes were observed. Cytokine release syndrome (CRS) occurred in 91.7% of patients, but the majority were grade 1 or 2, with only one patient experiencing grade 3 CRS.

The Phase I study (NCT05155189) was designed to evaluate the safety and anti-tumor activity of C-CAR031 in advanced or unresectable HCC. Of the 24 patients treated, 83.3% had extrahepatic metastasis, and the median number of prior systemic therapies was 3.5.

AbelZeta Pharma, headquartered in Rockville, Maryland, and Shanghai, China, is dedicated to developing proprietary cell-based therapies targeting both hematological malignancies and solid tumors. Their pipeline includes CAR-T and TIL therapies, with research and development conducted in their own GMP facilities.

While these early results are promising, AbelZeta acknowledges that future outcomes are uncertain and dependent on further research and clinical trials. The company remains committed to advancing innovative therapies that harness the immune system to combat cancer and other diseases.