Cure Genetics Reports Promising CAR-NKT CGC729 Data for RCC at ASGCT 2024

SUZHOU, China, May 23, 2024 — Cure Genetics recently presented promising safety and efficacy data for their CAR-NKT product, CGC729, targeting relapsed and refractory metastatic renal cell carcinoma (R/R mRCC) at the 27th Annual Meeting of the American Society for Gene & Cell Therapy (ASGCT) in Baltimore. This first-in-human trial highlights CGC729's potential in treating heavily pretreated R/R mRCC patients, demonstrating a favorable safety profile and potential anti-tumor activity.

Phase I Clinical Trial Overview

The phase I dose-escalation clinical trial was carried out at Fudan University Shanghai Cancer Center. It employed a single-arm, 3+3 design to assess the safety and efficacy of CGC729 across three dosage levels (DL1: 5 x 10^6/m^2; DL2: 1.5 x 10^7/m^2; DL3: 4.5 x 10^7/m^2). Participants included five patients with relapsed and refractory metastatic renal cell carcinoma who had undergone at least two prior therapy lines. Each patient received a single infusion of CGC729 after undergoing lymphodepletion.

Safety Profile

As of April 11, 2024, five patients had been treated with CGC729 — three at DL1 and two at DL2. No dose-limiting toxicities were observed. Common adverse events included lymphodepletion-associated neutropenia, thrombocytopenia, and leukopenia. One patient in the lowest dose group (DL1) experienced grade 2 cytokine release syndrome (CRS) and grade 2 immune effector cell-associated neurotoxicity syndrome (ICANS). Both conditions were managed effectively and resolved rapidly.

Efficacy Results

Of the four evaluable patients, the overall response rate (ORR) was 50% (2 out of 4). For patients with CD70 positive tumors (n=3), the ORR was 66.7% (2 out of 3), with two achieving partial responses. CGC729 showed significant anti-tumor activity, with the longest duration of response ongoing at nine months post-treatment. Pharmacokinetic analyses indicated robust expansion and prolonged persistence of CGC729 in the blood for up to 20 weeks, irrespective of CD70 expression levels in tumors.

Conclusion and Future Prospects

This interim analysis from the first-in-human trial underscores the potential of CGC729 as a treatment for R/R mRCC, showcasing a compelling safety profile and robust anti-tumor activity. The data suggest that CGC729 could become a future therapeutic option for patients with this challenging cancer.

Innovative Therapeutic Platform

The treatment of solid tumors, including R/R mRCC, is complicated by tumor heterogeneity and a suppressive tumor microenvironment (TME). Cure Genetics' AIMS CAR-NKT platform leverages the unique properties of Natural Killer T (NKT) cells, known for their multifactorial anti-tumor abilities and TME modulation. This platform is designed to enhance tumor infiltration, modulate the TME, and sustain cell expansion. The encouraging results from CGC729 are being used to develop further autologous and allogeneic products.

About CGC729

CGC729 is an autologous anti-CD70 CAR-NKT product with a unique mechanism of action. It combines adaptive and innate immunity, targeting CD70, CD1d, and stress ligands commonly found in renal cell carcinoma. Additionally, CGC729 can modulate the TME by influencing macrophage differentiation and resisting TGF-β inhibition.

About Cure Genetics

Founded in Suzhou in 2016, Cure Genetics is a clinical-stage company focused on developing innovative cell and gene therapies for solid tumors and genetic diseases. Through its AIMS CAR-NKT and VELP™ AAV platforms, the company is advancing a diverse pipeline of treatments. By establishing global collaborations and licensing agreements, Cure Genetics aims to deliver novel therapies to patients with unmet medical needs while setting a benchmark in the global cell and gene therapy landscape.

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