Company Profile. February 2018

Transcription

1 Company Profile February 2018

2 We pioneer immunotherapy, to enable a world where cancer patients can defeat cancer on their own. From GreenPeptide to BrightPath on July 1,

3 Company Profile 2

4 BrightPath Biotherapeutics A clinical-stage biopharmaceutical company focused on immuno-oncology Our pipeline covers cancer peptide vaccine, cell therapy, and immuno-modulator antibodies to address the most exciting and fast growing I/O market Cell Therapy ips-t Peptide ITK-1 GRN-1201 GRN-1301 Neoantigen Fully-personalized neoantigen vaccine Antibody Immuno-modulator 3

5 Our Business Model We have established a systematic and consistent development process, from the basic research and preclinical studies to obtaining a POC, in cooperation with academia and our partner companies Basic Research Pre-Clinical Study Early-stage Clinical Study Late-stage Clinical Study Launch BP considers conduct of P3 study and sales of regenerative/cell medicine subject to accelerated approval in JP. Partnering / License-out Collaboration with Academia / Biotech Seeds Strong research skill and experience in our own research laboratory Strong development skills/know-how accumulated based on the development of ITK-1 in Japan/GRN-1201 in US : Partnering/License-out Timing: Partnering at earlier stage would be possible. 4

6 Our Advantage -Project Driving- BrightPath has expertise in drug project management from drug discovery through clinical supported by our experience in late-stage clinical trials Drug discovery Kawasaki Research Laboratories Functions Clinical Development Phase I-III Advanced Immuno Therapy Pre-clinical Regulatory Monitoring Data management GCP inspection Biostatics CMC Kurume(Testing Technology) Investigational drug management Licensing / M&A / Collaborative research Collaboration External company / academia (CRO/ CMO etc.) 5

7 Company Overview Capital JPY 3,774 million (as of March 2017) Management Team Chief Executive Officer Chief Operative Officer Chief Financial Officer Director External Director External Auditor Kenichi Nagai Yutaka Waki, D.V.M. Teruhiko Sakai, CPA Akira Yamada, Ph.D. Professor, Kurume University Hirotaka Takeuchi, Ph.D. Professor, Harvard Business School Honorary Professor, Hitotsubashi University Yoshihiro Imai Taketoshi Abe Yoshiyasu Yamaguchi Employees 40 (as of June 2017) 6

8 Company Overview Location Headquarters Fukuoka Bio Incubation Center, 1-1 Hyakunenkoen, Kurume-shi, Fukuoka Tokyo Branch Kojimachi Central Building 7F, Kojimachi, Chiyoda-ku, Tokyo Kawasaki Research Laboratory Life Innovation Center 412, Tonomachi, Kawasaki-ku, Kawasaki-shi, Kanagawa 7

9 Company History :Corporate history :Pipeline (May) Established at Fukuoka Prefecture as GreenPeptide (Oct.) Listed on the Tokyo stock exchange (code: 4594) (Oct.) Start of phase I clinical trials of GRN-1201 for malignant melanoma in the US (Jul.) GreenPeptide changed its name to BrightPath Biotherapeutics Co., Ltd. (Jan.) Start of phase II clinical trials of GRN-1201 in combination with an immune checkpoint antibody for non-small cell lung cancer in the US (Nov.) An exclusive license contract related to ITK-1 commenced with FUJIFILM Corporation (Jun.) Start of phase III clinical trials of ITK-1 in patients with castrationresistant prostate cancer (Aug.) Opened Kawasaki Research Laboratories at Kanagawa Prefecture 8

10 Research and Licensing Collaborations Partner Product Collaboration Territory Year Fujifilm ITK-1 Out-licensing WW 2011 Kanagawa Prefecture Cancer Center GRN-1301 In-licensing WW 2016 Kanagawa Prefecture Cancer Center Novel neoantigen Research Collaboration 2016 National Cancer Center Japan Personalized neoantigen vaccine Research Collaboration 2017 Kanagawa Prefecture Cancer Center / Tokyo university Personalized neoantigen vaccine Research Collaboration 2018 Mie University Personalized neoantigen vaccine Research Collaboration 2018 Advanced Immune Therapy (Originated from Tokyo University) ips-t In-licensing JP 2016 Juntendo University ips-t Research Collaboration

11 Kawasaki Research Laboratories (KRL) KRL in the Life Innovation Center in Kawasaki City is located across the Tama River from Tokyo International Airport (Haneda Airport) KRL focuses on the research of neoantigens and immuno-modulators Haneda Airport KRL 神奈川県川崎市川崎区殿町 3 丁目 25 番 22 ライフイノベーションセンター 412 電話 FAX

12 Financial Highlight Cash Balance JPY 3.9 billion (December 31, 2017) Achieved money raising (JPY3.2B) in January, 2018 No Debt Cash Expenditures JPY 2 billion (Forecast for FY 2017) Market Capitalization JPY 33.9 billion (January 31, 2017) TSE Mothers (Tokyo)

13 R&D Pipeline 12

14 Pipeline Project Indication Region Basic Research Preclinical Phase I II III Launch Partners ITK-1 Semi-personalized cancer peptide vaccine Prostate cancer JP FUJIFILM Corporation GRN-1201 NSCLC US Cancer peptide vaccine In combination with an immune checkpoint inhibitor (for NSCLC) Melanoma US Out-licensing Candidate GRN-1301 Neoantigen T790M point mutation antigen vaccine NSCLC TBD Out-licensing Candidate ips-t Induced pluripotent stem cell (ipsc) derived rejuvenated T cell therapy Lymphoma JP Joint development project with: University of Tokyo Juntendo University 13

15 ITK-1 Semi-personalized cancer peptide vaccine HLA-A24 restricted peptide (Common in Japanese patients) Out-licensed to FUJIFILM Corporation in 2011 A Phase III clinical study is ongoing in Japan (currently following-up) Indication : Castration-resistant prostate cancer Cell Therapy ips-t Peptide ITK-1 GRN-1201 GRN-1301 Neoantigen Fully-personalized neoantigen vaccine Antibody Immuno-modulator 14

16 ITK-1: Clinical Data The efficacy of ITK-1 was suggested by prior clinical studies and institutional clinical investigations Castration-resistant prostate cancer Overall Survival (OS) ITK-1 (Ph1+Extension study) n=15, Open label 23.8 *1 Historical Control ITK-1 (Kurume University) n=12, Open label Decetaxel (Ph3 TAX327) n=335, Approved in * *2 Mitoxantrone (Ph3 TAX327) n= * (Reference) *1 BrightPath Biotherapeutics/ *2 Noguchi M, et al. The Prostate 2011; 71: *3 Berthold DR, et al. J Clin Oncol 2008; 26: month Most of AEs are under CTCAE Grade 2 (Injection site reaction) 15

17 GRN-1201 Cancer peptide vaccine consisting of four peptide antigens HLA-A2 restricted peptide (Common in American and European patients) Clinical studies are ongoing in the US Phase 1 study for melanoma (n=18) Phase 2 study for NSCLC (Combination with immune checkpoint antibodies) Cell Therapy ips-t Peptide ITK-1 GRN-1201 GRN-1301 Neoantigen Fully-personalized neoantigen vaccine Antibody Immuno-modulator 16

18 GRN-1201: Antigen Expression GRN-1201 targeting TAAs are expressed in many types of cancer Melanoma: >85% NSCLC: >95% Majority of tumor samples expressed all four target TAAs MELANOMA NSCLC Adeno Ca CRC PROSTATE Adeno Ca OVARIAN All types PANCREAS Adeno Ca SCCHN Case Number % 31/ % 74/ % 93/ % 28/ % 83/ % 26/ % 76/ % BrightPath Biotherapeutics, In-house data More than 1,000 patients have been administered with GRN-1201 homologue/related peptide in institutional clinical investigations at Kurume University Highly favorable immunogenicity and safety profiles 17

19 GRN-1201: Pharmacology GRN-1201 peptides demonstrated capability of inducing immune response Induction of immune responses by each GRN 1201 homologous peptide in HLA A*02+ Japanese patients with advanced cancer (Data from institutional clinical investigations at Kurume University) CTL IgG CTL & IgG DTH* Peptide Total A2+ pts Pts analyzed Pts induced % Pts analyzed Pts induced % Pts analyzed Pts induced % Pts analyzed Pts induced % A % % % % B % % % % C % % % % D % % % % *DTH: Delayed-type hypersensitivity BrightPath Biotherapeutics In-house data 18

20 GRN-1301 Cancer peptide vaccine targeting a drug-resistance-derived gene mutation (EGFR T790M point mutation) caused by primary treatment for NSCLC with EGFR-TKI HLA-A2 restricted peptide (Common in American and European patients) Confirmed healthy donors and EGFR-TKI treated patients immunogenic response to the candidate peptide Basic research Indication:Non-small cell lung cancer(nsclc) Cell Therapy ips-t Peptide ITK-1 GRN-1201 GRN-1301 Neoantigen Fully-personalized neoantigen vaccine Antibody Immuno-modulator 19

21 GRN-1301: Background Cancer immunotherapy for a drug-resistant gene mutation (EGFR T790M point mutation) involved in resistance to a primary treatment for lung cancer, EGFR-TKI Annual number of newly diagnosed patients: 500,000 patients Approximately 80% of these had NSCLC (United States: 210,000 patients, EU (Top 5 countries): 200,000 patients, Japan: 100,000 patients) 1 Primary EGFR mutation rate 10-15% T790M mutation (secondary EGFR mutation) rate in EGFR-TKI-resistant patients EGFR tyrosine kinase inhibitors (EGFR-TKI) 2 are used as standard therapy 60% 1 Source: GLOBOCAN Tarceva (erlotinib, sales amount in 2015: $1.59 bn), Iressa (gefitinib), and Giotrif (afatinib) were approved by 3 bureaus. 20

22 GRN-1301: Background Neoantigen T790M gene mutation is a therapeutic target for cancer immunotherapy Immunogenicity of antigen epitope (peptide) has been demonstrated A patent was granted in the US Only appear in cancer cells Escape (antigen loss) from immune surveillance mechanisms may not occur Driver mutations are essential for cancer malignancy, occuring against a challenge by EGFR-TKI (primary treatment) Relatively high incidence 21

23 ips-t Induced pluripotent stem cell (ipsc) derived rejuvenated T cell therapy The world s first clinical application of rejuvenated medicine in cancer immunotherapy Joint development project with the Institute of Medical Science of University of Tokyo and Juntendo University Indication: Lymphoma Cell Therapy ips-t Peptide ITK-1 GRN-1201 GRN-1301 Neoantigen Fully-personalized neoantigen vaccine Antibody Immuno-modulator 22

24 ips-t For T cell therapy, securing the sufficient number of T cells with anti-cancer activity has been challenging. Through the use of ips technology, an unlimited number of rejuvenated (high-activity) T cells can be supplied 23

25 Fully-personalized Cancer Neoantigen Vaccine A neoantigen is defined as a tumor antigen generated from gene mutations present in cancer cells. The human immune system recognizes neoantigens as non-self, which induce immune responses. Since tumor neoantigens are unique to each patient s tumor, they are considered as ideal targets of personalized treatment BrightPath is conducting collaborative researches to develop a methodology of fullypersonalized cancer neoantigen vaccine with Japanese academia Cell Therapy ips-t Peptide ITK-1 GRN-1201 GRN-1301 Neoantigen Fully-personalized neoantigen vaccine Antibody Immuno-modulator 24

26 Fully-personalized Cancer Neoantigen Vaccine Development of Fully-personalized Cancer Neoantigen Vaccine Cancer Patient Mutation Identification Vaccine Design Fullypersonalized Vaccine Tissues Biopsy NGS MS analysis Computing Modalities Peptide RNA/DNA Adjuvants Formulation 25

27 Fully-personalized Cancer Neoantigen Vaccine Research collaborations with Japanese leading academia/institute realize fullypersonalized cancer neoantigen vaccine therapy 26

28 We pioneer immunotherapy, to enable a world where cancer patients can defeat cancer on their own.