Alnylam Pharmaceuticals, Inc. · NASDAQ:ALNY
Alnylam Pharmaceuticals — Investor Presentation (September 2018)
32 slides from Alnylam Pharmaceuticals's investor presentation, Sep 11, 2018
Slide 1: ONPATTRO (patisiran) and Investigational ALN-TTRsc02 for ATTR Amyloidosis
ONPATTRO™ (patisiran) and Investigational ALN-TTRsc02 for the Treatment of ATTR Amyloidosis* September 11, 2018 *ONPATTRO is approved in the U.S. for the polyneuropathy of hereditary ATTR (hATTR) amyloidosis in adults, © 2018 Alnylam Pharmaceuticals, Inc. and in the EU for the treatment of hATTR amy
Slide 2: Agenda
Agenda Welcome • Christine Lindenboom, Vice President, Investor Relations & Corporate Communications Introduction and ONPATTRO (patisiran) • Eric Green, Vice President, General Manager, TTR Program A Payer’s Perspective • Michael Sherman, M.D., M.B.A., Chief Medical Officer and Senior Vice President
Slide 3: Reminders
Reminders Event will run for approximately 60-75 minutes Q&A session at end of presentation • Questions may be submitted at any time via the ‘Ask a Question’ field on the webcast interface. Replay, slides and transcript available at www.alnylam.com/capella 3
Slide 4: Alnylam Forward Looking Statements
Alnylam Forward Looking Statements This presentation contains forward-looking statements, within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. There are a number of important factors that could cause actual results to differ material
Slide 5: Agenda
Agenda Welcome • Christine Lindenboom, Vice President, Investor Relations & Corporate Communications Introduction and ONPATTRO (patisiran) • Eric Green, Vice President, General Manager, TTR Program A Payer’s Perspective • Michael Sherman, M.D., M.B.A., Chief Medical Officer and Senior Vice President
Slide 6: RNAi Therapeutics: New Class of Innovative Medicines
RNAi Therapeutics: New Class of Innovative Medicines Clinically Proven Approach with Transformational Potential Nobel Prize-winning science Silence any gene in genome Potent and durable mechanism of action Product engine for sustainable pipeline Now commercial 6
Slide 7: Alnylam Clinical Development Pipeline
Alnylam Clinical Development Pipeline Focused in 4 Strategic Therapeutic Areas (STArs): Genetic Medicines Cardio-Metabolic Diseases Hepatic Infectious Diseases HUMAN BREAKTHROUGH EARLY STAGE LATE STAGE COMMERCIAL CNS Diseases POC1 DESIGNATION (IND or CTA Filed-Phase 2) (Phase 2-Phase 3) REGISTRATION
Slide 8: Alnylam Clinical Development Pipeline
Alnylam Clinical Development Pipeline Focused in 4 Strategic Therapeutic Areas (STArs): Genetic Medicines Cardio-Metabolic Diseases Hepatic Infectious Diseases HUMAN BREAKTHROUGH EARLY STAGE LATE STAGE COMMERCIAL CNS Diseases POC1 DESIGNATION (IND or CTA Filed-Phase 2) (Phase 2-Phase 3) REGISTRATION
Slide 9: Hereditary ATTR (hATTR) Amyloidosis
Hereditary ATTR (hATTR) Amyloidosis Description Mutations in TTR gene lead to deposition of misfolded protein as GI: amyloid, causing polyneuropathy Diarrhea and other multisystem disease Nausea Vomiting manifestations1 CARDIAC: GU: Heart failure Proteinuria Arrhythmia Kidney failure Median survival
Slide 10: The first RNAi therapeutic is now approved
The first RNAi therapeutic is NOW APPROVED onpattro?%. (patisiran) ifscton WL Alnylam.
Slide 11: First and Only Therapy Approved for Patients with hATTR Amyloidosis in U.S.
First and Only Therapy Approved for Patients with hATTR Amyloidosis in U.S. Approved U.S. For the treatment of the polyneuropathy of Indication hereditary transthyretin-mediated amyloidosis in adults. Data in Label Includes data from APOLLO primary and select secondary endpoints. August 10, 2018 “Th
Slide 12: ONPATTRO U.S. Label Highlights
ONPATTRO U.S. Label Highlights Dosing & Dosing: Administration • 0.3 mg/kg (patients <100 kg) • 30 mg (patients ≥100 kg) Premedication (day of infusion): • IV dexamethasone, 10 mg • IV H1 and H2 blockers • Oral acetaminophen Administration: • Should be performed by healthcare professional. Safety No
Slide 13: Change From Baseline in mNIS+7
Change From Baseline in mNIS+71 Primary Endpoint: Change from baseline in mNIS+7, a specialized assessment of polyneuropathy in hATTR amyloidosisa-d • The mean change from baseline in mNIS+7 at 18 months was -6.0 points (improvement) for ONPATTRO-treated patients versus 28.0 points (worsening) for p
Slide 14: Change From Baseline in Norfolk QoL-DN Score
Change From Baseline in Norfolk QoL-DN Score1 Significant improvement in quality of life vs placebo, as measured by Norfolk QoL-DNa-d • For ONPATTRO-treated patients, the mean change from baseline in Norfolk QoL-DN at 18 months was -6.7 points (improvement) versus 14.4 points (worsening) for patient
Slide 15: Full Results of APOLLO Study Published July 2018
Full Results of APOLLO Study Published July 2018 15
Slide 16: U.S. Commercialization Underway
U.S. Commercialization Underway 1 2 3 Diagnosis Brand Choice Treatment Experience and Access TM TM Unified Commercial, Market Access, & Medical Affairs teams driving disease education, diagnosis, optimized patient experience, and access to care 16
Slide 17: No-charge, Third-party Genetic Testing and Counseling Program
: No-charge, Third-party Genetic Testing and Counseling Program Developed to reduce barriers to genetic testing and counseling to help people make more informed decisions about their health Alnylam provides financial support for the program, but the tests and services are performed by independent th
Slide 18: Ongoing Support from Alnylam Assist
Ongoing Support from Alnylam Assist™ Alnylam Assist is a comprehensive program dedicated to helping guide patients through treatment with ONPATTRO A dedicated Case Manager Alnylam Assist will connect patients with a dedicated Alnylam Case Manager who can provide personalized support throughout the t
Slide 19: First-Ever RNAi Therapeutic Approved in the EU
First-Ever RNAi Therapeutic Approved in the EU Approved EU For the treatment of hereditary transthyretin-mediated Indication amyloidosis (hATTR amyloidosis) in adults with stage 1 or stage 2 polyneuropathy Data in Label Includes data from APOLLO primary and secondary endpoints, as well as explorator
Slide 20: Global Disease Education Resources
Global Disease Education Resources Health care professionals Patients and their families www.hattramyloidosis.de www.hATTRbridge.de www.hattramyloidosis.fr www.hATTRbridge.fr www.hattramyloidosis.it www.hATTRbridge.nl www.hattramyloidosis.es www.hATTRbridge.es www.hattramyloidosis.pt www.hATTRbridge
Slide 21: Global Progress of ONPATTRO
Global Progress of ONPATTRO UK flag UK Japan • Provide patisiran to eligible adults in UK • Increasing presence in Japan with hATTR amyloidosis through MHRA – Head of Asia and seasoned leadership team Early Access to Medicines Scheme (EAMS) on board • On track for JNDA filing with Japanese Pharmaceu
Slide 22: Agenda
Agenda Welcome • Christine Lindenboom, Vice President, Investor Relations & Corporate Communications Introduction and ONPATTRO (patisiran) • Eric Green, Vice President, General Manager, TTR Program A Payer’s Perspective • Michael Sherman, M.D., M.B.A., Chief Medical Officer and Senior Vice President
Slide 23: A Payer's Perspective
A Payer’s Perspective In his role as Senior Vice President & Chief Medical Officer at Harvard Pilgrim Health Care, Dr. Sherman is responsible for Harvard Pilgrim’s medical trend management, network medical management, medical informatics, wellness and health promotion initiatives, care and disease m
Slide 24: Agenda
Agenda Welcome • Christine Lindenboom, Vice President, Investor Relations & Corporate Communications Introduction and ONPATTRO (patisiran) • Eric Green, Vice President, General Manager, TTR Program A Payer’s Perspective • Michael Sherman, M.D., M.B.A., Chief Medical Officer and Senior Vice President
Slide 25: Alnylam's Commitment to ATTR Amyloidosis Patients
Alnylam’s Commitment to ATTR Amyloidosis Patients Continued Innovation in ATTR with Investigational ALN-TTRsc02 Development Status for ALN-TTRsc02 Potential Product Attributes for ALN-TTRsc02 • Completed Phase 1 study in healthy volunteers POTENT AND SUSTAINED TTR KNOCKDOWN • Granted Orphan Drug Des
Slide 26: ALN-TTRsc02 Phase 1 Study: TTR Knockdown
ALN-TTRsc02 Phase 1 Study* Phase 1 Study Design ALN-TTRsc02 Achieves Robust and Durable Serum TTR Knockdown (KD) Mean max TTR KD of 83% after single 25 mg dose TTR Reduction Following Single Dose ALN-TTRsc02 Administration Safety (N=80): Mean [+/- SEM] TTR Relative to Baseline 1.4 Placebo (N=20) • N
Slide 27: ALN-TTRsc02 Phase 3 Study
ALN-TTRsc02 Phase 3 Study Planned Initiation Late 2018 Alignment reached on pivotal trial design Endpoints Details mNIS+7 & Norfolk-QOL (co-primary) open label certain cardiac parameters nine months ~120 patients with APOLLO placebo arm vs. hATTR amyloidosis results Reference arm of ~30 patients on
Slide 28: ALN-TTRsc02 Phase 3 Study Design
ALN-TTRsc02 Phase 3 Study Randomized, Open-label Study in Hereditary ATTR Amyloidosis Patients Patient Population N=~150 ALN-TTRsc02 Treatment Extension RANDOMIZATION† 25 mg SC q3 months • hATTR amyloidosis; any TTR mutation ALN-TTRsc02 Efficacy 25 mg SC • Neuropathy or Assessments q3 months Impairm
Slide 29: ALN-TTRsc02 Phase 3 Key Study Design Elements
ALN-TTRsc02 Phase 3 Study Key Study Design Elements Global Similar inclusion and Comprehensive Footprint exclusion criteria to APOLLO Evaluations Enrollment with various hATTR Patients with multi-systemic disease Assessments leveraging well- amyloidosis mutation types manifestations and a range of e
Slide 30: A Transformative and Historic Year for Alnylam and the TTR Programs
A Transformative and Historic Year for Alnylam and the TTR Programs • First-ever approval of a RNAi therapeutic in the U.S. and EU in August • Commercialization ongoing in U.S. and gearing up in EU • Continued global expansion with regulatory filings in Japan, Canada and Switzerland planned this yea
Slide 31: Agenda
Agenda Welcome • Christine Lindenboom, Vice President, Investor Relations & Corporate Communications Introduction and ONPATTRO (patisiran) • Eric Green, Vice President, General Manager, TTR Program A Payer’s Perspective • Michael Sherman, M.D., M.B.A., Chief Medical Officer and Senior Vice President
Slide 32: Thank You
32 Thank You © 2018 Alnylam Pharmaceuticals, Inc.
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