Jcr Pharmaceuticals Co., Ltd. · TSE:4552
Jcr Pharmaceuticals — FY2021 Earnings Presentation
30 slides from Jcr Pharmaceuticals's earnings presentation, May 12, 2022
Slide 1: FY2021 Second-Half Results Briefing Session - Business Highlights
FY2021 Second-Half Results Briefing Session - Business Highlights - May 12, 2022 JCR Pharmaceuticals Co., Ltd 【Securities code】 4552, PRIME. TSE Copyright © 2022 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Slide 2: Agenda
Agenda ❚ R&D Highlights ❚ R&D Updates ❚ Vaccine Stock Production Business ❚ Interim Summary of Midterm Business Plan “REVOLUTION” Copyright © 2022 JCR Pharmaceuticals Co., Ltd. All rights reserved. Business 1
Slide 3: Research and Development Highlights (Sep 2021 – Apr 2022)
Research and Development Highlights (Sep 2021 – Apr 2022) 2021 ♦Sep. Entered into an exclusive collaboration and commercialization agreement for JR-141 (pabinafusp alfa) with Takeda in certain regions ♦Oct. EMA grants PRIME DESIGNATION to JR-141 (pabinafusp alfa). 2022 ♦Jan. EMA grants Orphan Design
Slide 4: Development Portfolio Overview
Development Portfolio Overview ：progress in development stage since November 2021 Indication Preclinical Clinical Trials Filed Approved Remarks/ Time to next value inflection point Approved MPS type II • J-Brain Cargo® JR-141 Filed • FY2025～ (Approval in US, EU) (Hunter Syndrome) Phase 3 MPS type I
Slide 5: Progress on JR-141 (JR-141-GS31)
Progress on JR-141 (JR-141-GS31) IZCARGO®（Brand name in Japan） JR-141 pabinafusp alfa: BBB-penetrating iduronate-2-sulfatase (rDNA origin) ◆ Sep. 2021: Entered into an exclusive collaboration and commercialization agreement for JR-141 with Takeda in certain regions ◆ Oct. 2021: PRIME Designation fro
Slide 6: Progress on JR-171
Progress on JR-171 JR-171 lepunafusp alfa: BBB-penetrating α-L-iduronidase (rDNA origin) Summary of Global Phase 1/2 Clinical Trial（JR-171-101) ◆ May 2022: Completed patient recruitment of Part 2 Part 1 N=4 Part 2 N=14 or more ✓ 18 years or older ✓ 0 years or older ✓ No or mild intellectual disabili
Slide 7: Progress on JR-171: CSF Heparan Sulfate Concentrations
Progress on JR-171 JR-171 lepunafusp alfa: BBB-penetrating α-L-iduronidase (rDNA origin) Phase 1/2 Global Clinical Trial（JR-171-101) ： Change in CSF Heparan sulfate (HS) Concentrations as surrogate for substrate reduction in the CNS 8000 HS concentration (ng/mL) 7000 Part 1 Part 2 6000 5000 4000 300
Slide 8: Initiation of Development of JR-479
Initiation of Development of JR-479 JR-479 (BBB)-penetrating β-Hexosaminidase A Indication : GM2 gangliosidosis Tay-Sachs disease：Deficiency in α-subunit of β-Hexosaminidase Sandhoff disease：Deficiency in β-subunit of β-Hexosaminidase Frequency*¹ : Tay-Sachs：1/100,000～300,000 life births Sandhoff：Le
Slide 9: JCR's LSD Pipeline comprises 18 Programs
JCR's LSD Pipeline comprises 18 Programs ：progress in development stage since November 2021 JR-141 MPS II Approved Japan （Hunter) Fabry disease JR-141 MPS II Filed Brazil （Hunter) JR-141 MPS II JR-171 MPS I Clinical Global （Hunter) Global （Hurler) MPS IIIA MPS IIIB Non-clinical JR-162 Pompe JR-441 J
Slide 10: Gene Therapy Update
Gene Therapy Update Mar. 28, 2022: JCR enters into an exclusive license and collaboration agreement with Takeda to develop gene therapies using J-Brain Cargo® platform. －Initial focus: Lysosomal Storage Diseases －Option: Expansion into additional rare diseases and other indications J-Brain CargoⓇ Ge
Slide 11: Update on Vaccine Production Business
Update on Vaccine Production Business All orders under the Dec. 30, 2020 contract have been fulfilled. ・Sales recorded based on shipments. (Partial sales will be recorded in FY2022). ・No lot failures demonstrates strength in the manufacturing field. A new plant is under construction in the Kobe Scie
Slide 12: Agenda
Agenda ❚ R&D Highlights ❚ R&D Updates ❚ Vaccine Stock Production Business ❚ Interim Summary of Midterm Business Plan “REVOLUTION” Copyright © 2022 JCR Pharmaceuticals Co., Ltd. All rights reserved. Business 11
Slide 13: REVOLUTION: Important Business Challenges
“REVOLUTION”：Important Business Challenges JCR will address 6 important business challenges in anticipation of our full-fledged globalization. Top priority business challenge in anticipation of growing presence of JCR in the rare disease area [1] Qualitative and quantitative reorganization of the qu
Slide 14: Midterm Business Plan REVOLUTION: Interim Summary
Midterm Business Plan “REVOLUTION” : Interim Summary Qualitative and quantitative Quality assurance system updated reorganization ーNewly established the Analytical R&D Center of the quality assurance system ーStarted construction of a quality testing building (scheduled for completion in FY2022) Stre
Slide 15: Midterm Business Plan REVOLUTION: Guidance
Midterm Business Plan “REVOLUTION” : GUIDANCE Although the quantitative guidance set forth in the “REVOLUTION” was achieved in FY2021, we will continue to work on the Six Important Business Challenges and accelerate REVOLUTION with the aim of maximizing value in our global business. Currency: JPY Fi
Slide 16: Vision for Achieving the REVOLUTION
Vision for Achieving the ”REVOLUTION” Partnerships are at the core of JCR’s growth and acceleration strategy Internal Expand/build footprint in USA and Europe Development LSD Portfolio Partner focus: Partnerships Synergy in capacity and commercialization Lysosomal Storage Muscle delivery, Internal R
Slide 17: Vision for Achieving the REVOLUTION
Vision for Achieving the ”REVOLUTION” Partnerships are at the core of JCR’s growth and acceleration strategy Internal Expand/build footprint in USA and Europe Development LSD Portfolio Partner focus: Partnerships Synergy in capacity and commercialization Number of patients*1 Est. Market size*2 Indic
Slide 18: Vision for Achieving the REVOLUTION: Multi-Modality Innovator
Vision for Achieving the ”REVOLUTION” Partnerships through deployment of J-Brain CargoⓇ are expected to become a major business pillar Multi-Modality Innovator contributing to a wide range of disease areas Neuro-degeneration J-Brain CargoⓇ Platform Neuro-inflammation Focus on partnerships enabling (
Slide 19: JCR's Sustainability: Efforts during REVOLUTION
JCR’s Sustainability：Efforts during “REVOLUTION” Target Realization of sustainability through activities based on RD・E・S・G Corporate Rare Disease Environment Society Governance • Work environment that • Establish a governance • Meeting the Challenge of • Information Disclosure in fosters compatibili
Slide 20: New challenges in the final year and beyond
As the final year of the Midterm Business Plan New challenges in the final year and beyond Maximize product value by maximizing global development potential. Establish alliances with partners capable of maximizing the value of J-Brain CargoⓇ technology as a new business pillar. Achieve sustainable g
Slide 21: Business
Copyright © 2022 JCR Pharmaceuticals Co., Ltd. All rights reserved. Business 20
Slide 22: Appendix
Appendix Copyright © 2022 JCR Pharmaceuticals Co., Ltd. All rights reserved. Business 21
Slide 23: Lysosomal diseases: Expected Timeline
Lysosomal diseases：Expected Timeline Note: It is a plan at this stage and is subject to change FY2022 FY2023 FY2024 FY2025 • SAKIGAKE（PMDA） • Orphan Drug（PMDA） JR-141 Brazil： Filed • Orphan Drug（FDA） pabinafusp alfa • Fast Track（FDA） （MPS II） Global：Phase 3 trial (Ongoing) • Orphan Drug（EC） • PRIME（
Slide 24: Other pipeline (Growth Hormone and regenerative medicine area)
Other pipeline (Growth Hormone and regenerative medicine area) Note: It is a plan at this stage and is subject to change Expected timeline (Growth Hormone area) FY2022 FY2023 FY2024 FY2025 JR-142* Phase 2 （Pediatric short stature, GHD） Initiation of Phase 3 trial (Ongoing） *Long-acting growth hormon
Slide 25: JR-141 and JR-171 development pipeline (lysosomal diseases)
開発パイプライン（ライソゾーム病） JR-141 pabinafusp alfa: BBB-penetrating iduronate-2-sulfatase (rDNA origin) Indication : MPS type II (Hunter syndrome) Patient population*¹ : 250 (Japan) , 7,800 (WW) est. Est. Market size*² : 7.6 billion JPY (2019 Japan), 87.0 billion JPY (2019 WW) Disease overview : Hunter syndro
Slide 26: Progress of Other Compounds
Progress of Other Compounds JR-441 BBB-penetrating heparan N-sulfatase (rDNA origin) Indication : MPS type III A (Sanfilippo A syndrome) Patient population*¹ : 30 (Japan : Total of Type A&B）, 4,000 (WW) est. Est. Market size*² : >70.0 billion JPY (2019 WW: Total of Type A&B) Disease overview : An au
Slide 27: Progress of Other Compounds
Progress of Other Compounds JR-443 BBB-penetrating β-glucuronidase (rDNA origin) Indication : MPS type VII（Sly syndrome） Patient population*¹ : several (Japan) , 200 (WW) est. Est. Market size*² : 9.8 billion JPY est.（2019 WW） Disease overview : An autosomal recessive disease caused by deficiency of
Slide 28: Progress of Other Compounds
Progress of Other Compounds JR-142 Long-acting growth hormone (rDNA origin) Indication : Pediatric growth hormone deficiency Note : JCR’s proprietary half-life extension technology, based on a novel modified albumin, allows significant increase in the half-life of various biotherapeutics （Patent fil
Slide 29: Forward-Looking Statement
FORWARD-LOOKING STATEMENT This presentation contains forward-looking statements that are subject to a number of risks and uncertainties, many of which are outside our control. All forward-looking statements regarding our plans, outlook, strategy and future performance are based on judgments derived
Slide 30: Forward-Looking Statement
FORWARD-LOOKING STATEMENT The clinical development data mentioned in this document do not guarantee future results, nor do they guarantee the efficacy or effects of products under development. This document is not intended to guarantee or advertise the efficacy of the product under development. The