Takeda Pharmaceutical Co. Ltd. · TSE:4502
Takeda Pharmaceutical — Investor Day (April 2021)
83 slides from Takeda Pharmaceutical's investor day presentation, Apr 6, 2021
Slide 1: Wave 1 Pipeline Market Opportunity Call (Part 2)
WAVE 1 PIPELINE MARKET OPPORTUNITY CALL (PART 2) April 6th, 2021 Takeda Pharmaceutical Company Limited
Slide 2: Important Notice
IMPORTANT NOTICE For the purposes of this notice, “presentation” means this document, any oral presentation, any question and answer session and any written or oral material discussed or distributed by Takeda Pharmaceutical Company Limited (“Takeda”) regarding this release. This presentation (includ
Slide 3: FY2021 Will Be an Inflection Year for the Pipeline
FY2021 WILL BE AN INFLECTION YEAR FOR THE PIPELINE 3 Up to 6 NME regulatory submissions anticipated by year-end FY21, with potential for 4 approvals Expect 7 NMEs in pivotal studies across 10 indications by the end of FY21 Strong R&D and Commercial partnership to ensure launch excellence and deliver
Slide 4: Agenda
AGENDA TIME (ET) TIME (JT) AGENDA 08:00 – 08:05 21:00 – 21:05 Introduction Christophe Weber, President & CEO Takeda 08:05 – 08:10 21:05 – 21:10 Delivering an Innovative Pipeline to Our Patients: Spotlight on Select Wave 1 Programs Andy Plump, President Research & Development 08:10 – 08:35 21:10 – 21
Slide 5: Delivering an Innovative Pipeline to Our Patients
Delivering an Innovative Pipeline to Our Patients Spotlight on Select Wave 1 Programs Andy Plump President, Research & Development Need New Image
Slide 6: R&D Focus: Innovative Biopharma, Innovative Pipeline
PLASMA-DERIVED THERAPIES VACCINES CELL THERAPY GENE THERAPY DATA SCIENCES ONCOLOGY GASTROENTEROLOGY (GI)NEUROSCIENCE RARE GENETIC & HEMATOLOGY R&D FOCUS INNOVATIVE BIOPHARMA INNOVATIVE PIPELINE • 11 Wave 1 NMEs 5 programs with BTD, 3 with FTD and 1 with Sakigake designation • ~30 Wave 2 NMEs A GLOBA
Slide 7: Takeda Labs in Key Innovative Centers
TAKEDA LABS IN KEY INNOVATIVE CENTERS SHONAN, JAPAN Neuroscience Research, T-CiRA, iPark CAMBRIDGE, MA R&D Center, Oncology, Cell therapy, GI Research SAN DIEGO, CA Specialized drug discovery technologies, GI and Neuroscience VIENNA, AUSTRIA Gene Therapy, Plasma Derived Therapy|7
Slide 8: Accessing Innovation Through Research Partnerships
RESEARCH PIPELINE1 ~80% NON SMALL MOLECULES IN-LICENSE RESEARCH COLLABORATIONS NEWCO FORMATION JOINT DEVELOPMENT ~ 165 ~ 45 ~ 20 ~ 25 WE ARE ACCESSING INNOVATION BY INTEGRATING TAKEDA’S WORLD CLASS LABORATORIES WITH A NETWORK OF PARTNERS Non-Viral Gene Therapies TAK-007 & CAR-NK Platform Psychiatry
Slide 9: Our Pipeline Is Poised to Deliver Now and in the Future
OUR PIPELINE IS POISED TO DELIVER NOW AND IN THE FUTURE 1. Projected approval dates depend on data read-outs; some Wave 1 target approval dates assume accelerated approval 2. Certain Wave 2 programs may be accelerated into Wave 1 depending on future data read outs 3. Approval date assumes filing on
Slide 10: Takeda's R&D Engine With Potential to Deliver Life-Transforming Medicines
TAKEDA’S R&D ENGINE WITH POTENTIAL TO DELIVER A SERIES OF LIFE-TRANSFORMING MEDICINES 10 1. Potential to accelerate into Wave 1 dependent on future data readouts. 2. Takeda is supporting global access to three different COVID-19 vaccines: Novavax to develop, manufacture and commercialize 250 million
Slide 11: Wave 1 Pipeline to Deliver Life-Transforming Treatments
WAVE 1 PIPELINE TO DELIVER LIFE-TRANSFORMING TREATMENTS TO GROWTH EMERGING MARKETS Therapeutic Areas TAK-003 Dengue Vaccine Mobocertinib (TAK-788) Pevonedistat (TAK-924) TAK-007 Maribavir (TAK-620) TAK-609 TAK-755 TAK-611 Soticlestat (TAK-935) Orexin Eohilia (TAK-721) 2021 2026+ Exon 20 NSCLC 2L Hig
Slide 12: Maribavir (TAK-620) in Post-Transplant CMV Infection
Maribavir (TAK-620) Potential Game Changer in the Treatment for Post- Transplant Cytomegalovirus (CMV) Infection Obi Umeh Global Program Lead, Rare Genetic and Hematology Claus Jepsen Head of Global Product and Launch Strategy, Rare Genetic & Hematology
Slide 13: Transplants Are Highly Limited, Precious, Life-Saving Treatments
TRANSPLANTS ARE HIGHLY LIMITED, PRECIOUS, LIFE-SAVING TREATMENTS Transplants • Are lifesaving • Save over 190k lives annually1,2 • Loss is devastating for patients & costly to society Takeda plans global filings in 2021 with the goal of bringing Maribavir to patients 1: https://www.who.int/transplan
Slide 14: Immunosuppression Is Both Necessary and Challenging
IMMUNOSUPPRESSION IS BOTH NECESSARY AND CHALLENGING Prevents Rejection thus Protects Transplant Disables Immune System ► Increased Risk of Deadly Infections NECESSARY CHALLENGING • Common virus, infects most people by adulthood • Infection is dormant (like chickenpox virus) until immune system is co
Slide 15: Post-Transplant CMV Infection More Than Doubles Risk of Transplant Loss
POST-TRANSPLANT CMV INFECTION MORE THAN DOUBLES THE RISK OF TRANSPLANT LOSS, MORTALITY AND TOTAL COST OF TRANSPLANTATION 1,2,3 Leading to: • Graft rejection (SOT) • Graft-versus-Host Disease (GvHD) • Immunosuppression • Fungal/bacterial co-infections Encephalitis, ventriculitis Retinitis Myocarditis
Slide 16: A Clear Unmet Need Exists for an Anti-CMV Agent
A CLEAR UNMET NEED EXISTS FOR AN ANTI-CMV AGENT WITH STRONG EFFICACY WITHOUT COMPROMISE Causes Toxicity - bone marrow and/or kidneys Leads to Resistance development & Treatment failure Tissue invasive disease, Transplant (graft) loss and mortality Untreated/Poorly Controlled Treatment with available
Slide 17: Maribavir's Novel Multi-Modal Mechanism of Action
MARIBAVIR HAS THE POTENTIAL TO REDEFINE SUCCESS IN POST-TRANSPLANT CMV DUE TO ITS NOVEL MULTI-MODAL MECHANISM OF ACTION Works at 3 different points (4, 5 & 6) in the viral lifecycle: viral DNA replication, maturation & encapsidation Only agent that targets pUL97 all other agents inhibit only viral r
Slide 18: Maribavir Extensively Studied in More Than 1500 Patients
MARIBAVIR, AN ORAL SAFE ANTIVIRAL EXTENSIVELY STUDIED IN MORE THAN 1500 PATIENTS TO DATE 2011 ODD US Granted for Treatment of Post-Transplant CMV December 2014 Two positive Ph 2 Studies in 1L & 2L (R/R) CMV infection using 400mg bid dose completed December 2016 Ph 3 Studies in 1L (Study 302) & 2L (S
Slide 19: Maribavir Met Primary and Secondary Endpoints in Phase 3 Study
MARIBAVIR MET ITS PRIMARY & SECONDARY ENDPOINTS IN THE PHASE 3 RESISTANT/REFRACTORY CMV INFECTION STUDY (Solstice Trial) Population Treatment Duration (8 Weeks) Post-Rx Follow-Up (12 Weeks) Primary Endpoint (End of Therapy) Confirmed clearance of plasma CMV DNA (CMV viremia clearance) at the end of
Slide 20: Global Trial Representative of Resistant/Refractory CMV Population
GLOBAL TRIAL, REPRESENTATIVE OF RESISTANT/REFRACTORY POST-TRANSPLANT CMV PATIENT POPULATION Broad Transplant Population Included adequate numbers of both solid organ and hematopoetic stem cell transplant recipients Large Global Trial • >140 sites, 12 countries, 3 continents • N = 352 transplant reci
Slide 21: Primary Endpoint: Superior Viremia Clearance
PRIMARY ENDPOINT: MARIBAVIR SHOWED CLINICALLY MEANINGFUL, SUPERIOR VIREMIA CLEARANCE VS. CONVENTIONAL THERAPIES >2x more efficacy vs comparator Treatment Difference = 32.8% MBV CM V c le ar an ce a t W ee k 8 (% ) 23.9 28 117 IAT 55.7 131 235 0 20 40 60 80 100 P-value <0.001 Strong Efficacy Across S
Slide 22: Secondary Endpoint: Superior Clearance and Symptom Control Through Week 16
SECONDARY ENDPOINT: MARIBAVIR MAINTAINED SUPERIOR VIREMIA CLEARANCE & SYMPTOM CONTROL THROUGH WEEK 16 (8 WEEKS OFF TREATMENT) 22 | IAT = investigator assigned treatment; MBV = maribavir Treatment Difference = 9.5% MBV CM V c le ar an ce & s ym pt om c on tr ol a t W ee k 16 (% ) 10.3 IAT 18.7 44 235
Slide 23: Safety - Tolerability
SAFETY - TOLERABILITY Key Treatment-related Adverse Events, % Category IAT (N=116) MBV (N=234) Neutropenia (V)GCV, n=56 25.0 1.7 Acute kidney injury FOS, n=47 19.1 1.7 Increased immunosuppressant drug levels 0 6.0 Taste disturbance 1.7 44.0 KEY SAFETY FINDINGS Maribavir was safe & well tolerated wit
Slide 24: Growing Body of Evidence in First-Line CMV Infection
MARIBAVIR HAS A GROWING BODY OF EVIDENCE IN TREATMENT OF FIRST-LINE POST-TRANSPLANT CMV INFECTION 24 | BID: twice daily; CMV: cytomegalovirus; HCT: hematopoietic cell transplant; MBV: maribavir; SOT: solid organ transplant; VGCV: valganciclovir MBV compared favorably to VGCV in CMV viremia clearance
Slide 25: Ongoing Phase 3 Trial in First-Line Post-Transplant CMV
Population Treatment Duration (8 Weeks) Post-Rx Follow-Up (12 Weeks) • 1st Line CMV Infection • Stem cell transplants only • Primary endpoint: CMV viremia clearance @ 8 weeks 1:1 Randomization Double-blind 900mg BID VGV (N=275) Double-blind 400mg BID MBV (N=275) Weekly/Bi-Weekly Follow-up Visits ONG
Slide 26: Summary
SUMMARY CMV infections threatens survival of transplant with devastating consequences for the patient and high cost for society Transplants are extremely precious life-saving treatments Currently available antivirals for treatment of CMV are toxic, develop resistance leading to treatment failure and
Slide 27: Maribavir – Market Opportunity
Maribavir – Market Opportunity
Slide 28: Organ Transplant Recipients Celebrate a Second Chance at Life
Jane married & mother of two “This is me on my one year lungiversary. Happy Breath Day. Here I am in the wilderness, enjoying life. I think a lot about how to honor my donor. It’s just about giving back, be happy in my career, caring for my friends and family. Simply being a good person” ORGAN TRANS
Slide 29: CMV Is the Most Challenging Infection Post-Transplant
CMV IS THE MOST CHALLENGING INFECTION POST-TRANSPLANT - AND AFFECTS TENS OF THOUSANDS OF PATIENTS WORLDWIDE Direct transplant cost increase6 2.0- 6.2x Higher Mortality5 2.6x 20- 30% Higher risk of transplant/graft failure4 ~190K Globally1 ~60K USA2 (HSCT & SOT transplants) leaves patients vulnerable
Slide 30: CMV Infections Place High Value Procedures and Organs at Risk
NOT ONLY DOES CMV INFECTIONS PLACE A HIGH VALUE PROCEDURE AT RISK CMV INFECTIONS ALSO RISK WASTING ORGANS THAT CANNOT BE “RE-ORDERED” 30 Costly Procedure Short Supply Cost of Liver Transplant Cost of Kidney Transplant Cost of Allogenic HSCT Est. annual cost of a transplant patient with CMV infection
Slide 31: Transplant Procedures Take Place in Few Specialized Centers
BOTH ORGAN AND HCST TRANSPLANT PROCEDURES ARE HIGHLY SOPHISTICATED AND TAKES PLACE IN FEW HIGHLY SPECIALIZED CENTERS • 90% of allogenic HSCT (adult only) covered at ~80 centers1 • 80% of specific organ transplants covered at a combined ~125 centers1 • Kidney 101 centers • Heart 72 centers • Lung 32
Slide 32: Current World of Treating CMV Is Full of Compromises
THE CURRENT WORLD OF TREATING CMV INFECTIONS IS FULL OF COMPROMISES Optimal immunosuppression to manage graft function and prevent rejection Increased risk of CMV infection and disease Treatment of CMV with current SoC at optimal efficacious dose and sufficient duration Risk of treatment AEs toxicit
Slide 33: Maribavir – A Potential CMV Game Changer
CMV is the most common infection post-transplant 1 Current options are sub optimal & require compromises 2 Maribavir has the potential to be a game changer in post-transplant CMV 3 Takeda has the ability to capture the full potential 4 MARIBAVIR – A POTENTIAL CMV GAME CHANGER |33 • 190K transplants/
Slide 34: Agenda
AGENDA |34 TIME (ET) TIME (JT) AGENDA 08:00 – 08:05 21:00 – 21:05 Introduction Christophe Weber, President & CEO Takeda 08:05 – 08:10 21:05 – 21:10 Delivering an Innovative Pipeline to Our Patients: Spotlight on Select Wave 1 Programs Andy Plump, President Research & Development 08:10 – 08:35 21:10
Slide 35: Soticlestat (TAK-935) Deep Dive
Soticlestat (TAK-935) Deep Dive: Novel MoA for Treatment of Dravet Syndrome and Lennox- Gastaut Syndrome Sarah Sheikh Head of Neuroscience Therapeutic Area Unit Erika Gill Head of Global Product and Launch Strategy, Neuroscience
Slide 36: The 2020s as the Decade of Neurology
THE 2020s AS THE DECADE OF NEUROLOGY I n c r e a s i n g a b i l i t y t o a d d r e s s d e v a s t a t i n g n e u r o l o g i c a l d i s e a s e s Innovat ion landscape Patient with SMA type 1 | 36
Slide 37: Takeda Neuroscience Roadmap
TAKEDA NEUROSCIENCE ROADMAP First launches of potentially transformative therapies in rare Neurology Wave 1 (thru FY2024) Wave 2 (FY2025+) Orexin (Narcolepsy Type 1) Potential approval in FY24 Soticlestat (DS and LGS) Potential approval in FY23 Huntington’s Disease / Ataxia Neuromuscular Diseases Ot
Slide 38: Key Inflections Set Our Future in Neuroscience
KEY INFLECTIONS SET OUR FUTURE IN NEUROSCIENCE Science & Innovation Focus on Rare Neurology Execution of Wave 1 programs Rare neurology NeuroD Soticlestat Portfolio: Programs per Disease Cluster |38
Slide 39: Key Takeaways for Soticlestat in DS and LGS
KEY TAKEAWAYS FOR SOTICLESTAT IN DRAVET SYNDROME AND LENNOX-GASTAUT SYNDROME Promising option for patients and caregivers Potential first-in-class therapy • Global capabilities and local footprint will enable worldwide development program • Regulatory approval in US, Europe, Japan, China, and other
Slide 40: Dravet Syndrome
• ~10K patients diagnosed in the US1,2 • Homogeneous population with SCN1A mutation found in ~85% of patients1 Rare Genetic Epilepsy Associated with Developmental Delay DRAVET SYNDROME 1. Wu Y. Pediatrics 2015;136:1310–5., 2. DS Takeda Internal estimates/data on file Feb 2021., 3. Anwar. A. Cureus 2
Slide 41: Lennox-Gastaut Syndrome
Patient population Seizure type Disease burden • ~30-50K patients diagnosed in the US1,2 • Heterogeneous patient population3 • Associated with multiple seizure types including drop seizures3 • ~60% of patients unable to perform activities of daily living independently3 • Mortality 14-fold higher tha
Slide 42: Current Treatments Leave Substantial Unmet Need
DS and LGS Treatment Needs Efficacy on top of current standard of care Treatments with fewer side effects Less complicated to prescribe, given high poly-pharmacy rates Low-burden therapies for physicians, caregivers, and patients CURRENT TREATMENTS LEAVE SUBSTANTIAL UNMET NEED DS and LGS Treatment C
Slide 43: Soticlestat With Potential First-in-Class MOA
1Salamone A, et al. ECE 2018., 2Nishi T, et al. AAN 2018. Annu. Rev. Biochem. 2009. 78:1017–40 SOTICLESTAT WITH POTENTIAL FIRST-IN-CLASS MOA Soticlestat inhibits cholesterol 24-hydroxylase (CH24H) enzyme1,2 Dose-dependent reduction in 24HC levels1,2 Reduced glutamatergic signaling & reduced inflamma
Slide 44: ELEKTRA Phase 2 Study Design
Key Inclusion Criteria • Aged ≥2 and ≤17 years • Currently taking 1–4 AEDs • ≥3 convulsive (DS); ≥ 4 Drop (LGS) seizures during 28-day Baseline Optional OLE study (ENDYMION) Endpoints: % change from baseline in • Primary: – Seizure frequency for combined DS & LGS patients (maintenance period) • Key
Slide 45: Soticlestat Met Primary Endpoint in ELEKTRA
1Hahn et al. AES 2020; 2Seizure frequency per 28 days; 3Asymptotic 95% confidence interval and Hodges-Lehmann estimation of the median of differences in % change between the two arms from un-adjusted rank statistics. Modified intent-to-treat (mITT) = All randomized subjects who received at least ≥ 1
Slide 46: Statistically Significant Seizure Reduction in Dravet Cohort
ELEKTRA1 - STATISTICALLY SIGNIFICANT SEIZURE REDUCTION IN DRAVET SYNDROME COHORT Dravet Syndrome (Convulsive Seizures) Soticlestat Placebo N=25N=26 Placebo-adjusted3: -46.0% (-68.5,-20.0) -40 -35 -30 -25 -20 -15 -10 -5 0 5 10 -33.8% 7.0%P=0.0007 1Hahn et al. AES 2020; 2Seizure frequency per 28 days;
Slide 47: Numerical Seizure Reduction in LGS Cohort
• • Placebo-adjusted median seizure reduction of 14.8% did not reach statistical significance • LGS cohort was not powered for efficacy • Broad range of drop seizure frequency at baseline of 4 to >5,000 drop seizures/28 days • Sensitivity analysis supportive of more stringent, countable drop seizure
Slide 48: Promising Emerging Safety and Tolerability Profile
TEAE = treatment emergent adverse events; SAE = serious adverse events PROMISING EMERGING SAFETY AND TOLERABILITY PROFILE SUPPORTIVE OF MOVING INTO PHASE 3 DEVELOPMENT ELEKTRA TEAEs 0% 25% 50% 75% 100% TEAE rates SAE rates Overall AE Rates Soticlestat Placebo TEAEs >5% in soticlestat & >3% differenc
Slide 49: Two Global Phase 3 Studies in DS and LGS
Trial Design • Trial design based on feedback from FDA, EMA & PMDA • Ages ≥2 years • Adjunctive to AEDs • Active seizures at baseline2 Entry into OLE Outcome Measures • Primary: – Frequency change in convulsive seizures (DS study) during full treatment period – Frequency change in MMD seizures (LGS
Slide 50: Soticlestat – Market Opportunity
Soticlestat – Market Opportunity
Slide 51: Soticlestat Potential to Extend Treatment Goals Beyond Seizure Reduction
Potential Soticlestat Benefits Based on Data to Date Novel MoA with demonstrated seizure reduction4 Low rates of adverse events4 Do not anticipate clinically relevant drug-drug interactions Less potential for safety or monitoring requirements SOTICLESTAT HAS THE POTENTIAL TO EXTEND TREATMENT GOALS B
Slide 52: Soticlestat Potential to Help the Majority of DS and LGS Patients
SOTICLESTAT HAS THE POTENTIAL TO HELP THE MAJORITY OF DS AND LGS PATIENTS 1Wu Y. Pediatrics 2015;136:1310–5., 2DS Takeda Internal estimates/data on file Feb 2021. 3Trevathan E. Epilepsia. 1997 Dec;38(12):1283-8., 4LGS Takeda Internal estimates/data on file Feb 2021. 5Samanta D. Neuropediatrics. 2020
Slide 53: Takeda Aspires to Raise DS and LGS Treatment Expectations
TAKEDA ASPIRES TO RAISE DS AND LGS TREATMENT EXPECTATIONS FOR PATIENTS, CAREGIVERS, AND PHYSICIANS Soticlestat First approval anticipated FY2023 Re-define treatment goals Establish soticlestat as therapy of choice Prepare for global launch 53 | Potential First-In-Class Seizure Reduction Treatment
Slide 54: Key Takeaways for Soticlestat in DS and LGS
KEY TAKEAWAYS FOR SOTICLESTAT IN DRAVET SYNDROME AND LENNOX-GASTAUT SYNDROME Promising option for patients and caregivers Potential first-in-class therapy • Global capabilities and local footprint will enable worldwide development program • Regulatory approval in US, Europe, Japan, China, and other
Slide 55: Orexin Franchise Strategy Update
Orexin Franchise Strategy Update First potential medicine to treat the underlying disease in patients with Narcolepsy Type 1 Elena Koundourakis Head of Orexin Franchise Development, Neuroscience TA Erika Gill Head of Global Product and Launch Strategy, Neuroscience
Slide 56: Key Takeaways for Orexin Franchise
KEY TAKEAWAYS FOR OREXIN FRANCHISE Narcolepsy Type 2 (NT2) & Idiopathic Hypersomnia (IH) to follow On track for First Approval of an Oral Orexin Agonist in Narcolepsy Type 1 (NT1) • TAK-925 IV: 5 ePOC established across multiple disease settings • TAK-861: Longer Oral Agonist enters clinic in FY2021
Slide 57: NT1, NT2 and IH Are Central Disorders of Hypersomnolence
NARCOLEPSY TYPE 1 (NT1), NARCOLEPSY TYPE 2 (NT2) AND IDIOPATHIC HYPERSOMNIA (IH) ARE ALL CENTRAL DISORDERS OF HYPERSOMNOLENCE WITH SIGNIFICANT UNMET NEED • Different disorders with overlapping clinical features especially Excessive Daytime Sleepiness (EDS) • Common challenge: misdiagnosis and undert
Slide 58: What Is It Like to Live With NT1?
WHAT IS IT LIKE FOR PEOPLE TO LIVE WITH NT1? Extreme SLEEPINESS FEAR of cataplectic attacks DISRUPTION of daily life MISUNDERSTOOD by HCPs and family “We take current meds to survive. We want new medications to help us live” Narcolepsy Patient Advisor (Takeda Sponsored Patient Advisory Board)58 |
Slide 59: Narcolepsy Type 1 Is Caused by Severe Loss of Orexin Neurons
1. Mahoney CE, et al. Nat Rev Neurosci. 2019;20(2):83-93. | 2. Chastain EM, et al. Biochim Biophys Acta. 2011;1812:265–274. | 3. Nishino S. Sleep Med. 2007;8(4):373-399. | 4. Saper CB, et al. Trends Neurosci. 2001;24(12):726-731. | 5. Yoshida Y, et al. Eur J Neurosci. 2001;14(7):1075-1081. Reduced a
Slide 60: Orexin Agonists Penetrate Blood Brain Barrier and Activate Receptor
* G-Protein coupled receptors (GPCR). Nature Cell Biology, 2000, 2(10), 703-708., Nature, 2001, 411(6837), 613-617., Nature, 2003, 422(6928), 173-176., Nature, 2014, 513(7516), 124-127., etc. Orexin 2 receptor ON Orexin peptides cannot penetrate blood brain barrier, but are synthesized in brain Our
Slide 61: TAK-925 IV Improved Wakefulness and Reduced Cataplexy in NT1
TAK-925 OREXIN IV FORMULATION IMPROVED MAINTENANCE OF WAKEFULNESS AND REDUCED CATAPLEXY IN NT1 2.3 5.8 3.2 1.8 0.0 0.00 2 4 6 8 10 12 14 16 Placebo (n=4) 11mg (n=4) 44mg (n=5) 2 23*** 40*** 0 5 10 15 20 25 30 35 40 45 Placebo (n=4) 11mg (n=4) 44mg (n=5) TAK-925 IV Day 7 average sleep latency in Main
Slide 62: TAK-925 IV Supports Broader Role of an Orexin Agonist
1.8 24.2 31.7 0 5 10 15 20 25 30 35 40 45 Placebo (N=5) TAK-925 44mg (N=3) TAK-925 112 mg (N=5) Narcolepsy Type 21 *** *** TAK-925 OREXIN IV FORMULATION SUPPORTS POTENTIAL FOR BROADER ROLE OF AN OREXIN AGONIST 8.6 25.4 38.8 0 5 10 15 20 25 30 35 40 45 Placebo (N=20) TAK-925 44mg (N=18) TAK-925 112 m
Slide 63: Preclinical Data Shows TAK-994 Similar Efficacy Potential as TAK-925
PRECLINICAL DATA SHOWS TAK-994 HAS THE POTENTIAL FOR SIMILAR EFFICACY AS TAK-925 1. Suzuki M.,, Presented at SLEEP 2018, Baltimore, USA, June 2-6, 2018 2. Kimura H., Presented at WORLD SLEEP, Vancouver, Canada, September 20-25, 2019 * * ** n=4, Mean±SEM *P ≤ 0.05, **P ≤ 0.01, compared with control (
Slide 64: TAK-994 Progressing in Clinical Trials in NT1 and NT2
FIRST ORAL OREXIN AGONIST TAK-994 IS PROGRESSING IN CLINICAL TRIALS IN NT1 AND NT2 A double-blind, ph2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of TAK-994 in patients with narcolepsy type 1 or narcolepsy type 2 Part A (NT1 Multiple Rising Dose) Part B (NT1 P
Slide 65: TAK-994 Met ePOC Criteria
0 5 10 15 20 25 30 35 40 Currently approved NT1 therapies TAK-994 <8 Comparison of sleep latency in the Maintenance of Wakefulness Test (MWT) Placebo adjusted (minutes) Ph2A Hurdle TAK-994 ORAL AGONIST MET ePOC CRITERIA AND HAS THE POTENTIAL TO TRANSFORM THE TREATMENT FOR PATIENTS WITH NT1 Exceeded
Slide 66: TAK-994 Achieved POC in Sleep Deprived Healthy Volunteers
FIRST ORAL OREXIN AGONIST TAK-994 ACHIEVED POC IN SLEEP DEPRIVED HEALTHY VOLUNTEERS WITH NORMAL OREXIN LEVELS (TAK-994-1503)* 12.6 31.8 37.4 0 5 10 15 20 25 30 35 40 45 Placebo (N=16) Low Dose (N=16) High Dose (N=17) *** Two doses of TAK-994 demonstrated statistically significant improvements in the
Slide 67: Orexin Franchise Next Steps and Key Milestones
OREXIN FRANCHISE NEXT STEPS AND KEY MILESTONES Narcolepsy Type 1 Narcolepsy Type 2 Pivotal Studies Ongoing Global Ph2 study in NT1 and NT2 with TAK-994 Data will inform Pivotal Studies Design NT1 Approval Potential Additional Indications Global launches Multiple assets FY2021 FY2021-FY2023 FY2024 FY
Slide 68: Takeda Is Pioneering the Field of Orexin Therapeutics
• Evaluate additional indications for TAK-994 • Assess potential indications for TAK-861 • Evaluate TAK-925 (IV) in hospital settings • Bring TAK-994 quickly to patients with highest unmet linked to Orexin deficiency • Launch with EDS and cataplexy data globally • Distinct biological effect of orexi
Slide 69: Oral Orexin Agonist TAK-994 – Market Opportunity
Oral Orexin Agonist TAK-994 – Market Opportunity
Slide 70: Key Takeaways for Oral Orexin Agonist TAK-994
KEY TAKEAWAYS FOR ORAL OREXIN AGONIST TAK-994 NARCOLEPSY TYPE 1 (NT1) Current NT1 treatments do not address underlying orexin deficiency NT1 is caused by an orexin deficiency, which disrupts sleep awake cycles • Anticipated first approval FY2024 • Label expansions planned, and data dependent, as par
Slide 71: NT1 Is Chronic and Severe
NT1 IS CHRONIC AND SEVERE CHARACTERIZED BY A PENTAD OF SYMPTOMS Excessive Daytime Sleepiness (EDS) Cataplexy Hallucinations Sleep Paralysis Disrupted Nighttime Sleep1 1 Sateia MJ. Chest 2014;146:1387–94 71 |
Slide 72: Patient's Journey to Diagnosis
A PATIENT’S JOURNEY GENERALLY BEGINS IN ADOLESCENCE BUT CAN TAKE DECADES TO GET TO A SLEEP SPECIALIST AND DIAGNOSIS SYMPTOM ONSET PRE-DIAGNOSIS DIAGNOSIS TREATMENT MEAN OF 15 YEARS TO DIAGNOSIS 72 |
Slide 73: Current NT1 Treatments Do Not Address Underlying Orexin Deficiency
Treatment escalation and polypharmacy are common Despite treatment, NT1 is not controlled Experience daily EDS despite treatment3 Experience 1-2 episodes of Cataplexy per day despite treatment3 Newly diagnosed patients progress to second line within 1 year1 Of second line patients receive more than
Slide 74: Patients and Physicians Make Significant Trade-Offs With Current Therapies
TAKEDA BELIEVES PATIENTS AND PHYSICIANS MAKE SIGNIFICANT TRADE-OFFS WITH CURRENT THERAPIES 1.Thorpy, Hopper, Patroneva, Burden of Narcolepsy: A Survey of Patients and Physicians, Neurology Apr 2019, 92 (15 Supplement) P3.6-036 2. Narcolepsy Network, 2018 Know Narcolepsy Survey. Available at: https:/
Slide 75: NT1 Rare, Underdiagnosed and Undertreated
NT1 RARE, UNDERDIAGNOSED AND UNDERTREATED Estimated diagnostic rate in developed countries (only 6% in China with largest prevalence)6 Mean diagnostic delay7 Adult NT1 Prevalence US 135K1 EU 66K2,3 JAPAN 64K4 CHINA 395K5 Treatment Rate8 1. Silber MH et al. Sleep 2002;25:197–202; Longstreth WT Jr. et
Slide 76: Potential to Transform Treatment With TAK-994
TAKEDA HAS THE POTENTIAL TO TRANSFORM TREATMENT WITH ORAL OREXIN AGONIST TAK-994 Increase Recognition and Diagnosis Rates Establish TAK-994 as a breakthrough treatment Prepare for NT1 launch and label expansions TAK-994 Oral Orexin Agonist First to target underlying cause of NT1 First approval expec
Slide 77: Key Takeaways for Oral Orexin Agonist TAK-994
KEY TAKEAWAYS FOR ORAL OREXIN AGONIST TAK-994 NARCOLEPSY TYPE 1 (NT1) Current NT1 treatments do not address underlying orexin deficiency NT1 is caused by an orexin deficiency, which disrupts sleep awake cycles • Anticipated first approval FY2024 • Label expansions planned, and data dependent, as par
Slide 78: Delivering an Innovative Pipeline to Our Patients
DELIVERING AN INNOVATIVE PIPELINE TO OUR PATIENTS SPOTLIGHT ON SELECT WAVE 1 PROGRAMS Uthra Sundaram EVP, Global Product and Launch Strategy
Slide 79: Strong R&D and Commercial Partnership Drives Overall Success
STRONG R&D AND COMMERCIAL PARTNERSHIP DRIVES OVERALL SUCCESS Transforming Partnership Transforming Lives Transforming Science 79 |
Slide 80: Bringing Our Pipeline to Life
BRINGING OUR PIPELINE TO LIFE Global Capabilities to Deliver Life Transforming Treatments LAUNCH EXCELLENCE Patient Journey & Diagnosis Data, Insights & Analytics Patient Services Value Based Partnerships Digital Evidence Generation 80 |
Slide 81: Market Opportunity and Peak Revenue Potential by Product
PRODUCT INDICATION FULL MARKET OPPORTUNITY2 TAKEDA’S PEAK REVENUE POTENTIAL5 mobocertinib (TAK-788) Exon 20 non-small cell lung cancer 1L Exon 20 non-small cell lung cancer 2L $300 – 600MN pevonedistat (TAK-924) Higher risk-Myelodysplastic syndromes Unfit Acute myeloid leukemia $400 – 800MN TAK-007
Slide 82: Upcoming Investor Events
UPCOMING INVESTOR EVENTS 4Q20 EARNINGS CALL ONCOLOGY STRATEGIC UPDATE CALL PEVONEDISTAT DEEP DIVE CALL JUNE 2021 (DATE TO BE CONFIRMED) MAY 11, 2021 2021 – DATA DRIVEN (DATE TO BE CONFIRMED) 82 |
Slide 83: Q&A Session
© 2021 Takeda Pharmaceutical Company Limited. All rights reserved QA Session