Kyowa Kirin Co., Ltd. · TSE:4151
Kyowa Kirin — Merger or Acquisition (April 2024)
25 slides from Kyowa Kirin's merger presentation, Apr 8, 2024
Slide 1: Kyowa Kirin - Orchard Therapeutics IR Meeting
Kyowa Kirin - Orchard Therapeutics IR Meeting April 8th, 2024
Slide 2: Today's Agenda
Today’s Agenda The Background of Acquiring Orchard Therapeutics ◆ – President and Chief Executive Officer Masashi Miyamoto, Ph.D. About Orchard Therapeutics and HSC-GT* ◆ – The CEO of Orchard Therapeutics plc, Bobby Gaspar, M.D., Ph.D. Future Plans Regarding HSC-GT ◆ – President and Chief Executive
Slide 3: Our Vision toward 2030
Our Vision toward 2030 Provide pharmaceuticals for unmet medical needs We are focused on developing medicines for diseases where there is a Our Vision toward 2030 clear patient need for new options. We make full use of multiple therapeutic modalities, including biotechnology Kyowa Kirin will realize
Slide 4: Story for Vision 2030
Story for Vision 2030 Strategies for creating and delivering life-changing value Disease Science Value Global deployment of delivery on products developed by Focus disease areas: bone & mineral, intractable taking full advantage of our own hematological diseases/hemato oncology, and rare diseases th
Slide 5: Why Orchard Therapeutics?
Why Orchard Therapeutics? Internal Changes External Changes • Focus on disease areas with strengths in bone & mineral, • Establishing treatments for diseases with no existing hematologic cancers & intractable blood diseases, and therapy rare diseases • Personalized medicine • Portfolio evolution thr
Slide 6: Bobby Gaspar – the CEO of Orchard Therapeutics plc
Bobby Gasper – the CEO of Orchard Therapeutics plc ◼ Bobby Gaspar, M.D., Ph.D. ⚫ Co-founder and chief executive officer of Orchard Therapeutics ⚫ Studied medicine and surgery at Kings College in London ⚫ Completed Ph.D. at the UCL Great Ormond Street Institute of Child Health © Kyowa Kirin Co., Ltd.
Slide 7: Imagine Limitless Possibilities
Imagine Limitless Possibilities 8 April 2024 Bobby Gaspar, M.D., Ph.D.
Slide 8: We aspire to end the devastation caused by genetic and other severe diseases
We aspire to end the devastation caused by genetic and other severe diseases through the curative potential of HSC gene therapy.
Slide 9: Orchard Therapeutics: A global gene therapy leader
Orchard Therapeutics: A global gene therapy leader Dedicated to ending the devastation caused by severe genetic diseases through the curative potential of Our Mission Our Mission hematopoietic stem cell (HSC) gene therapy Our Global Established footprint: presence in: Approach Our approach harnesses
Slide 10: Strategic long-term growth and value creation with expansion into larger indications
Strategic long-term growth and value creation with expansion into larger indications Mid-long VA L U E Execute and deliver term on rare disease portfolio Expand on HSC gene Continue to build out therapy approach for capabilities in HSC gene larger indications and therapy across regulatory, Near-mid
Slide 11: HSC gene therapy enables delivery of gene-corrected cells to multiple organ systems
HSC gene therapy enables delivery of gene-corrected cells to multiple organ systems Osteoclasts BONE HSC Viral Vector Microglia Granulocyte T cells MPS-IH B cells Integration BRAIN NK cells MLD MPS- IIIA Megakaryocyte LIVER Erythrocyte Intestinal macrophages Self-renewal Monocyte / Macrophage Kupf
Slide 12: Autologous ex vivo gene therapy approach
Autologous ex vivo gene therapy approach Administration: Blood cell collection: Genetically-modified cells Blood hematopoietic stem and are given back to the patient 6 1 progenitor cells (HSPCs) are taken intravenously from the patient via leukapheresis Patient can return home Conditioning: The pati
Slide 13: Advancing a pipeline to address serious genetic diseases
Advancing a pipeline to address serious genetic diseases 12
Slide 14: Strong operational execution already in 2024
Strong operational execution already in 2024 Built U.S. field Beneluxa team to set the MLD patients reimbursement BLA approval by Randomized first stage for identified for agreement FDA for patients in OTL- successful treatment driving expanded Lenmeldy in 203 pivotal study execution of the signific
Slide 15: Lenmeldy/Libmeldy (MLD): Potential significant clinical benefit
LenmeldyTM / Libmeldy® (MLD): Potential significant clinical benefit for a devastating genetic disease Severe Motor Impairment Free Survival (sMFS) Interval from birth to first occurrence GMFC-MLD ≥ 5 (no locomotion and unable to sit) or death Treatment with OTL-200 resulted in statistically signifi
Slide 16: MLD
All 7 surviving PSEJ patients maintained the ability to walk with normal performance for age (GMFC-MLD Level 0) Untreated NHx sibling Treated Child i We t GMFC-MLD Level 6 GMFC-MLD Level 0 3 years post-onset 8 years post-GT 8 years of age 12 years of age rchard therapeutics
Slide 17: Pioneering Commercial Operations Leading to Sustainability
Pioneering Commercial Operations Leading to Sustainability Access Reimbursement Treatment Secured for all eligible MLD children Early access program: AP2 granted and renewed (France) Treatment abroad: Named patient program in the Middle East established (Saudi Arabia) Cross border: European pathway
Slide 18: OTL-203 (MPS-IH): Disease background & NEJM interim proof-of-concept results
OTL-203 (MPS-IH): Disease background & NEJM interim proof-of-concept results Disease snapshot Before gene therapy Neuropsychological Tests over Time Cognitive Age-Equivalent Score (Overall) • Multisystemic neurometabolic condition affecting cognition, growth and skeletal function • Current standard
Slide 19: OTL-201 (MPS-IIIA): A progressive and devastating disease with no approved treatment options
OTL-201 (MPS-IIIA): A progressive and devastating disease with no approved treatment options Disease snapshot Early Neurocognitive Outcomes • Sanfilippo syndrome type A; • Change in cognitive function Pre-treatment Post-GT Treatment pathogenic variants in SGSH (age equivalent scores) against with GT
Slide 20: Success in MLD provides roadmap, common infrastructure for next programmes
Success in MLD provides roadmap, common infrastructure for next-in-line neurometabolic and CNS programmes MLD Other LSDs MPS-IH MPS-IIIA GRN-FTD Approved in Europe and U.S. Regulatory Supply Chain PLATFORM Manufacturing Treatment Sites SYNERGIES Distribution Referral Networks 19
Slide 21: Compelling fundamentals driving near-term value creation and long-term growth
Compelling fundamentals driving near-term value creation and long- term growth Commercial Diagnostics and Future Potential Manufacturing and Other Model Newborn Screening Regulatory Approvals Distribution Applications Establish scalable Leverage success in Implement a sustainable Advance scientific
Slide 22: The world's leading gene therapy company
The world’s leading gene therapy company 21
Slide 23: Story for Vision 2030
Story for Vision 2030 Strategies for creating and delivering life-changing value Disease Science Value Global deployment of delivery on products developed by Focus disease areas: bone & mineral, intractable taking full advantage of our own hematological diseases/hemato oncology, and rare diseases th
Slide 24: Future HSC-GT related business
Future HSC-GT related business ◼ Expansion of Libmeldy®/LenmeldyTM usage in EU and ◼ Steady progress in the current development pipeline US Target disease Status Code • US approval on March 18, 2024, followed by launch MPS-IH Registrational study OTL-203 • Ten prospective NBS* studies for MLD are ac
Slide 25: Kyowa Kirin Co., Ltd. Corporate Communications Dept., IR&PR Group
Kyowa Kirin Co., Ltd. Corporate Communications Dept., IR&PR Group +81-3-5205-7206 / ir@kyowakirin.com