Designation Description Change of NY-ESO-1 gene therapy product under "SAKIGAKE Designation System"

Takara Bio Inc. (Takara Bio), announced today that a NY-ESO-1・siTCRTM gene therapy product (Development code: TBI-1301) for synovial sarcoma co-developed by Takara Bio and Otsuka Pharmaceutical Co., Ltd, (Otsuka) in Japan has been approved for the description change of product under SAKIGAKE Designation System (hereinafter, "SAKIGAKE") by the Ministry of Health, Labour and Welfare as of February 14, 2019.

Takara Bio is conducting domestic Phase I/II clinical trial of TBI-1301 targeting synovial sarcoma since January 2017, and it has been designated as a SAKIGAKE product in March 2018. This designation change is based on the agreement that Takara Bio has concluded with Otsuka for domestic co-development and exclusive sales in April 2018.

Both companies will aim cooperatively for co-development of this product.

Designation Time Number Designation Date Product Name Designated

Applicant Designated

Efficacy of NY-ESO-1・siTCRTM gene therapy product
Before 3rd time 1 March 27, 2018 TBI-1301 Takara Bio Anti-tumor therapy for synovial sarcoma, which a receptor gene recognizing tumor antigen, is transduced ex-vivo to lymphocytes of patients, and the gene-modified lymphocytes are infused back to the patients
After Same as above Same as above Same as above Same as above Otsuka

Same as above

Announcement on product designation of NY-ESO-1・siTCR gene therapy product for synovial sarcoma under "SAKIGAKE Designation System" by the Ministry of Health, Labour and Welfare (Released on March 27, 2018)
http://ir.takara-bio.co.jp/en/news_all/news_Release/news_Release04062404159330868269856.html

Otsuka enters agreement with Takara Bio for co-development and sales rights in Japan to NY-ESO-1 siTCRTM and CD19 CAR gene therapies (Released on April 9, 2018)
https://www.otsuka.co.jp/en/company/newsreleases/2018/20180409_1.html

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