On September 29, 2015 CARsgen Therapeutics, a private and venture backed company focused on the development of CAR-T-based autologous immunotherapy to treat solid tumors, reported a business and clinical update on its immunotherapy programs (Press release, Carsgen Therapeutics, SEP 29, 2015, View Source [SID:1234514784]).

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New Partnership Agreements
In September 2015, CARsgen and Shanghai Cancer Institute entered into a five year research and development agreement for the advancement of CARsgen’s CAR-T cancer immunotherapeutics. The partners will work together to identify and develop novel CAR-T candidates as well as next-generation CAR-T technologies. Under terms of the agreement, CARsgen will own commercial rights of any intellectual assets generated from the collaboration. Financial terms of the agreement were not disclosed.

In May 2015, CARsgen entered an agreement with Shanghai Jiaotong University (affiliated with Renji Hospital) to conduct development of CARsgen’s glypican-3 (GPC3) CAR-T therapeutic designed to treat relapsed or refractory hepatocellular carcinoma, or advanced liver cancer, in addition to other CARsgen immunotherapeutics.

Progress in Immunotherapy Program for Advanced Liver Cancer
In March 2015, CARsgen and Renji Hospital initiated a Phase 1 clinical study to evaluate the safety and early-stage efficacy of CARsgen’s GPC3 chimeric antigen receptor T-cell (CAR-T) therapy. To date, six patients with relapsed and/or refractory hepatocellular carcinoma (HCC) have been enrolled and treated with escalating doses of GPC3-CAR-T infusions (up to ~1.1×109 cells). Two of the six patients (33%) demonstrated more than a 90 percent decline in levels of alpha-fetoprotein (AFP), a liver biomarker indicating the activity of liver cancer. Glypican 3 (GPC3) is expressed in a variety of tumors, with a high frequency in HCC. In in vivo and in vitro preclinical studies, GPC3-CAR-T effectively killed GPC3 positive HCC cells.

GPC3-CAR-T was safe and well tolerated in all six patients treated to date, with no serious adverse events observed. The most frequent treatment-related adverse events were moderate temperature elevations, which CARsgen believes resulted from the expected cytokine storm. GPC3-CAR-T cells persisted in the peripheral blood circulation throughout the treatment period. Αlpha-fetoprotein (AFP), a surrogate HCC biomarker for liver, was found having 90% decline in two patients. In one of these patients, just two months following treatment, AFP levels were determined to be in the normal range.

Dr. Zonghai Li, president & CEO of CARsgen, commented, "The preliminary clinical data accumulated from the first six patients is very encouraging. It helps us to identify additional parameters including on-target off-tumor toxicity, effective dose regime, and pre-infusion operations. We plan to continue our clinical test and to enroll up to 20 patients in the ongoing Phase 1 study, in support of our commitment to bring effective treatments to HCC patients through our unique and proprietary immunotherapy."
Future Development Programs

In addition to its GPC3-CAR-T program, CARsgen has a broad range of CAR-T candidates for lung, brain and stomach cancers. CARsgen and RenJi Hospital began enrolling up to 10 patients in a Phase 1 clinical study of an anti-EGFR CAR-T therapy designed to treat glioblastoma multiforme (GBM or brain cancer).

About GPC3-CAR/CSG-GPC3
GPC3-CAR/CSG-GPC3 T cell therapy is the world’s first CAR-T solution indicated for late-stage HCC. CARsgen is enrolling patients in a Phase 1 clinical study in China, with results expected in [Mid-2016]. For more information or to find a study site, please visit www.clinicaltrials.gov, identifier NCT02331693.