Announcement on submission of the Clinical Trial Plan Notification for phase I/II clinical trial of NY-ESO-1・siTCR gene therapy in Japan

Kusatsu/Shiga, Japan — January 24, 2017 — Takara Bio Inc. (Takara Bio), today announced that it has submitted the Clinical Trial Notification for regenerative medicines to PMDA (Pharmaceuticals and Medical Devices Agency), Japanese regulatory agency, to conduct phase I/II clinical trial for siTCR gene therapy (NY-ESO-1・siTCR gene therapy; Development code: TBI-1301) targeting synovial sarcoma in Japan.
In the clinical trial, TCR which recongnizes NY-ESO-1 antigen, a tumor antigen, is transferred ex-vivo to lymphocytes of patients, and the gene-modified lymphocytes are infused back to the patients. The patients will be monitored for safety and efficacy. The Takara Bio’s method of gene transduction and T-cell expansion using RetroNectin®, and Takara Bio’s original retroviral vectors for siTCR transduction will be used during the cell processing for the clinical trial.
After the clearance, the protocol will be reviewed by each IRB (Institutional Review Board) and the study will be initiated.
Takara Bio is also conducting Phase Ib clinical trial of NY-ESO-1・siTCR gene therapy targeting solid tumor in Canada. With the safety and efficacy data obtained from the clinical trials in Japan and Canada, Takara Bio attempts to achieve the early-approval utilizing the conditional and term-limited approval system for regenerative medicine under The Law on Securing Quality, Efficacy and Safety of Products including Pharmaceuticals and Medical devices. Takara Bio aims to commercialize the NY-ESO-1・siTCR gene therapy in the fiscal year 2020.
【Overview of the Clinical Trial】

Study name Multicenter Phase I/II Clinical Trial of TBI-1301 for synovial sarcoma with pretreatment of chemotherapeutic administration
Condition Patients with unresectable progressive or recurrent synovial sarcoma
Main Endpoint Phase I Clinical trial: Safety
Phase II Clinical trial: Efficacy
Estimated Enrollment 8
Duration Mar 2017 – Jan 2020
Site National Cancer Center Hospital, Osaka National Hospital and others, total 5 sites


For more information:Corporate Communications, Takara Bio Inc. E-mail: bio-ir@takara-bio.co.jp

This article is translated from press release in Japanese for your convenience. Forward-Looking Statements Statements in this news release, other than those based on historical fact, concerning the current plans, prospects, strategies and expectations of the Company and its Group represent forecasts of future results. While such statements are based on the conclusions of management according to information available at the time of writing, they reflect many assumptions and opinions derived from information that includes major risks and uncertainties. Actual results may vary significantly from these forecasts due to various factors. Factors that could influence actual results include, but are not limited to, economic conditions, especially trends in consumer spending, as well as exchange rate fluctuations, changes in laws and government systems, pressure from competitors’ prices and product strategies, decline in selling power of the Company’s existing and new products, disruptions to production, violations of our intellectual property rights, rapid advances in technology and unfavorable verdicts in major litigation.

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