On May 10, 2022 CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors, reported that the phase I trial interim results of an investigator-initiated trial of CT041 have been published in Nature Medicine (View Source), which is one of the top international medical journals in the Nature Portfolio (2-year Impact Factor of 53.44) (Press release, Carsgen Therapeutics, MAY 10, 2022, View Source [SID1234614148]).
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The publication, titled "Claudin18.2-specific CAR T cells in gastrointestinal cancers: phase I trial interim results" presented results from a multi-center, open-label Phase I clinical trial conducted in China to explore the safety, efficacy, and cellular pharmacokinetics of CT041 in patients with advanced CLDN18.2-positive gastrointestinal cancers. The interim trial results showed that among 37 patients with advanced gastrointestinal cancers, CT041 was well tolerated with a manageable safety profile including no dose-limiting toxicity in 28 days post infusion, no grade ≥3 cytokine release syndrome, no neurotoxicity, and no treatment-related death. The objective response rate (ORR) and disease control rate (DCR) in patients with gastrointestinal cancers were 48.6% and 73.0%, respectively. In patients with gastric cancer or gastroesophageal junction cancer (GC/GEJ), the ORR and DCR reached 57.1% and 75.0%, respectively.
As of the date of this announcement, CT041 is the world’s first and only CAR T-cell candidate for the treatment of solid tumors entering a confirmatory Phase II clinical trial.
The corresponding author for the article, Professor Lin Shen of Beijing Cancer Hospital, said, "CAR T-cell therapy has successfully treated a variety of hematologic malignancies, while few breakthroughs have been made in solid tumors. CT041 is the first-in-class CAR T-cell product candidate against Claudin18.2. In this clinical trial, CT041 showed promising efficacy and manageable safety for patients with gastrointestinal cancers, particularly gastric cancer, who have failed prior lines of conventional therapies. As the largest clinical trial for solid tumors to date, the CAR T-cell therapy showed unprecedented efficacy against solid tumors. The publication of the interim trial results in Nature Medicine enables us to better share the results with our peers and advance CAR T-cell therapy in solid tumors. It also demonstrates the potential for more innovative, China-developed medicines to reach and benefit patients in global markets."
"This study is made possible by the joint efforts of CARsgen’s research and development team and our investigators, as well as the support and trust from the patients and their families. For CT041, the world’s first solid tumor CAR T-cell product candidate that has entered a confirmatory Phase II clinical trial, we will spare no efforts to advance the clinical trials and benefit patients as soon as possible," added Shen.
Dr. Zonghai Li, Founder, Chairman of the Board, Chief Executive Officer, and Chief Scientific Officer of CARsgen Therapeutics Holdings Limited, said, "Congratulations to the research team led by Prof. Shen. We thank the patients participating in the trial for their trust and thank all the researchers for their dedication. Publication of the interim CT041 trial results in Nature Medicine demonstrates the high quality and value of this trial. The development of innovative therapies has always been an arduous journey, particularly for effective CAR T-cell therapies against solid tumors. Despite these challenges, we started to collaborate with our investigators in 2015 for the treatment of hepatocellular carcinoma and glioblastoma. Since then, we have continued to explore innovative technologies and products. I am very glad to see the breakthrough interim results of CT041 in the research led by Prof. Shen. We will continue the joint efforts with our investigators in the global CT041 clinical development."
About CT041
CT041 is an autologous CAR T-cell product candidate against the protein CLDN18.2 that has the potential to be the first-in-class globally. CT041 targets the treatment of CLDN18.2-positive solid tumors with a primary focus on GC/GEJ and pancreatic cancer (PC).
In addition to the investigator-initiated trials in China, CARsgen has initiated a Phase Ib clinical trial for advanced GC/GEJ and PC, confirmatory Phase II clinical trial for advanced GC/GEJ in China and initiated a Phase 1b clinical trial for advanced gastric or pancreatic adenocarcinoma in North America. CARsgen also intends to initiate a pivotal Phase 2 clinical trial in North America in 2022.
CARsgen plans to submit an NDA to the NMPA in China in the first half of 2024 and to submit the BLA to the U.S. FDA in 2024.
In 2020 and 2021, CT041 received Orphan Drug designation from the U.S. FDA for the treatment of GC/GEJ and Orphan Medicinal Product designation from the EMA for the treatment of advanced gastric cancer. In November 2021, CT041 was granted PRIME Eligibility by the EMA for the treatment of advanced gastric cancer. In January 2022, CT041 was granted Regenerative Medicine Advanced Therapy (RMAT) Designation by the U.S. FDA for the treatment of advanced gastric or gastroesophageal junction adenocarcinoma with CLDN18.2-positive tumors.
As of the date of the announcement, CT041 is the only CLDN18.2-targeted CAR T-cell product candidate globally that is being studied in clinical trials with IND/CTA approvals from the FDA, the NMPA, and Health Canada.