Corporate Overview August 2018
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1 Corporate Overview August Alnylam Pharmaceuticals, Inc.
2 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 There are a number of important factors that could cause actual results to differ materially from the results anticipated by these forward-looking statements. These important factors include our ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of our product candidates; pre-clinical and clinical results for our product candidates; actions or advice of regulatory agencies; delays, interruptions or failures in the manufacture and supply of our product candidates; our ability to obtain, maintain and protect intellectual property, enforce our intellectual property rights and defend our patent portfolio; our ability to obtain and maintain regulatory approval, pricing and reimbursement for products; our progress in establishing a commercial and ex-united States infrastructure; our ability to successfully launch, market and sell our approved products globally; our ability to successfully expand the indication for ONPATTRO (patisiran) in the future; competition from others using similar technology and developing products for similar uses; our ability to manage our growth and operating expenses, obtain additional funding to support our business activities and establish and maintain business alliances; the outcome of litigation; and the risk of government investigations; as well as those risks more fully discussed in our most recent report on Form 10-Q under the caption Risk Factors. If one or more of these factors materialize, or if any underlying assumptions prove incorrect, our actual results, performance or achievements may vary materially from any future results, performance or achievements expressed or implied by these forward-looking statements. All forward-looking statements speak only as of the date of this presentation and, except as required by law, we undertake no obligation to update such statements. 2
3 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 3
4 Alnylam Clinical Development Pipeline Focused in 4 Strategic Therapeutic Areas (STArs): Genetic Medicines Cardio-Metabolic Diseases Hepatic Infectious Diseases CNS Diseases HUMAN POC 1 BREAKTHROUGH DESIGNATION EARLY STAGE (IND or CTA Filed-Phase 2) LATE STAGE (Phase 2-Phase 3) REGISTRATION COMMERCIAL COMMERCIAL RIGHTS ONPATTRO (patisiran) 2 Polyneuropathy of Hereditary ATTR Amyloidosis Global Givosiran Acute Hepatic Porphyrias Global Fitusiran Hemophilia and Rare Bleeding Disorders Inclisiran Hypercholesterolemia 15-30% Royalties Milestones & up to 20% Royalties ALN-TTRsc02 ATTR Amyloidosis Global Lumasiran Primary Hyperoxaluria Type 1 Global Cemdisiran Complement-Mediated Diseases Global 4 1 POC, proof of concept defined as having demonstrated target gene knockdown and/or additional evidence of activity in clinical studies 2 Approved in the U.S. As of August 2018
5 Hereditary ATTR (hattr) Amyloidosis Description Mutations in TTR gene lead to deposition of misfolded protein as amyloid, causing polyneuropathy and other multisystem disease manifestations 1 GI: Diarrhea Nausea Vomiting Median survival 4.7 years from diagnosis CARDIAC: Heart failure Arrhythmia GU: Proteinuria Kidney failure UTI Incontinence Impotence Patient Population* ~50,000 worldwide AUTONOMIC: Falls Lightheadedness Weight loss PERIPHERAL: Numbness/tingling Pain Weakness Impaired walking 5 Kipper Living with hattr Amyloidosis 1 Coelho T, et al. N Engl J Med. 2013;369(9): * Ando et al., Orphanet J Rare Dis, 2013; Ruberg et al., Circulation, 2012
6 Patisiran Clinical Development Background Patisiran Therapeutic Hypothesis Production of mutant and wild type TTR Unstable circulating TTR tetramers reduced Randomized, double-blind, placebo-controlled study of patisiran in 225 hattr amyloidosis patients with polyneuropathy Organ deposition of monomers, amyloid (β-pleated) fibril prevented, clearance promoted Results published July 2018 Neuropathy stabilization or improvement 6
7 LS mean (SEM) mnis+7 from baseline LS mean (SEM) Norfolk-QOL from baseline Phase 3 Study Results Primary Endpoint Met Placebo Patisiran Worsening Improvement mnis p=9.26 x Worsening Baseline 9 Months 18 Months Improvement Norfolk-QOL p=1.10 x Baseline 9 Months 18 Months At 18 months -6.0 point change relative to baseline 34.0 point difference relative to placebo 56.1% of patients improved* At 18 months -6.7 point change relative to baseline 21.1 point difference relative to placebo 51.4% of patients improved* All secondary endpoints encompassing QOL, walk speed, activities of daily living, mbmi and autonomic dysfunction met 7 Adams D, et al. N Engl J Med. 2018;379:11-21 * Improvement defined as patients with <0 point increase from baseline to 18 months
8 Phase 3 Study Results Safety & Tolerability Profile Event Placebo Patisiran (N=77) (N=148) no. of patients (%) Any adverse event (AE) 75 (97) 143 (97) Any severe AE 28 (36) 42 (28) Any serious adverse event (SAE) 31 (40) 54 (36) AE leading to discontinuation of the trial regimen 11 (14) 7 (5) AE leading to withdrawal from the trial 9 (12) 7 (5) Death* 6 (8) 7 (5) Majority of AEs mild or moderate in severity No safety signals related to steroid pre-medication regimen or TTR reduction No hepatic, renal or hematologic (including platelet) safety signals 8 * No deaths were considered related to study drug by the investigator Adams D, et al. N Engl J Med. 2018;379:11-21
9 9
10 ONPATTRO (patisiran) Regulatory Status Approved U.S. Indication Data in Label For the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. Includes data from APOLLO primary and select secondary endpoints. This approval is part of a broader wave of advances that allow us to treat disease by actually targeting the root cause, enabling us to arrest or reverse a condition, rather than only being able to slow its progression or treat its symptoms New technologies like RNA inhibitors, that alter the genetic drivers of a disease, have the potential to transform medicine, so we can better confront and even cure debilitating illnesses. ~ Scott Gottlieb, MD, FDA Commissioner, Press Release, 8/10/2018 Recommended Indication in EU Recommended SmPC* For the treatment of hereditary transthyretin-mediated amyloidosis (hattr amyloidosis) in adults with stage 1 or stage 2 polyneuropathy. Includes data from APOLLO primary and secondary endpoints, as well as exploratory cardiac endpoints. The safety and efficacy of Onpattro was evaluated in a pivotal trial involving 225 patients with hattr amyloidosis and symptomatic polyneuropathy. The study showed clinically-relevant improvements in the neurological manifestations of the disease and on patients quality of life, as well as a positive impact on cardiac parameters. ~ CHMP Opinion Press Release, 7/27/2018 EC decision for approval expected in September JNDA Filing On track for submission in mid PMDA decision for approval anticipated June * SmPC, Summary of Product Characteristics
11 ONPATTRO on Market 1 st Business Day Post Approval ONPATTRO Launch Vision Alnylam is primed to flawlessly educate and serve all external stakeholders at launch, including patients, caregivers, healthcare professionals, and payers. Readiness, both internal and external, built through clear insights into stakeholder needs, full integration of launch plans, and alignment and deployment of a motivated, trained team. 48-Hour Launch Readiness Within 48-hours Alnylam was in position to: process a Patient Start Form, ship drug, collect revenue, and promote/engage with all stakeholders with excellence and integrity. 11
12 hattr Amyloidosis Market Opportunity Estimated Disease Prevalence* ~ 50,000 patients worldwide NEUROLOGIC PHENOTYPE > 50% have cardiomyopathy CARDIAC PHENOTYPE > 30% have neuropathy WITHIN ONPATTRO LABEL (based on approved U.S.P.I) 20K to 30K worldwide ~ 10K diagnosed 10K to 15K in U.S. < 3K diagnosed 12 * Based on Alnylam estimates from interviews with key opinion leaders, THAOS registry, recent clinical trials and literature ONPATTRO is approved in the U.S. for the polyneuropathy of hattr amyloidosis in adults; Not approved in EU on July 27, 2018, CHMP recommended for approval to treat hattr amyloidosis in adults with stage 1 or stage 2 polyneuropathy Current diagnosis rates difficult to confirm and may be lower in initial launch years
13 U.S. Commercialization Underway! Diagnosis Brand Choice Treatment Experience and Access TM TM Unified Commercial, Market Access, & Medical Affairs teams driving disease education, diagnosis & treatment, optimized patient experience, and access to care 13
14 Global Commercialization Efforts Underway EU and Asia in place EU Launch Ready Japan team on-board Preparing Canada and Switzerland Initiating Latin America, starting with Brazil UNITED STATES CEMEA* LATIN AMERICA ASIA Regional Support Market Access Supply Chain Country organizations built Country Manager Medical Affairs Sales and Marketing Local Market Access 14 * Canada, Europe, Middle East, North Africa
15 Mean [+/-SEM] TTR Knockdown Relative to Baseline (%) ALN-TTRsc02 Opportunity Advancing Continued Innovation for Patients with ATTR Amyloidosis Phase 1 Study Healthy Volunteers Placebo (N=20) 25mg (N=6) 100mg (N=6) 5mg (N=6) 50mg (N=6) 300mg (N=6) Mean max TTR KD of 97.1%; ~80% TTR KD at nearly 1 year after single 50 mg dose* Days Since First Dose Safety (N=80): No SAEs and no discontinuations due to AEs All AEs mild or moderate in severity 52 Inotersen DOSES PER YEAR 4 ALN-TTRsc02 DOSES PER YEAR ANTICIPATED Expect to initiate Phase 3 study in Late * As of data cutoff on 31May2017
16 ALN-TTRsc02 Market Opportunity* Potential for Significant Expansion in ATTR Amyloidosis hattr amyloidosis Asymptomatic hattr carriers Wild-type ATTR amyloidosis patients 16 * Intended to be illustrative and not intended to represent specific estimates of patient numbers
17 ALN-TTRsc02 Phase 3 Study Planned Initiation Late 2018 Alignment reached on pivotal trial design Endpoints mnis+7 & Norfolk-QOL (co-primary) certain cardiac parameters Details open label nine months ~120 patients with hattr amyloidosis vs. APOLLO placebo arm results Reference arm of ~30 patients on patisiran to be included. No formal comparisons between patisiran and ALN-TTRsc02 planned. Additional Phase 3 studies, including wild-type ATTR amyloidosis, planned for
18 Acute Hepatic Porphyrias Givosiran Description Family of ultra-rare orphan diseases causing incapacitating and potentially fatal attacks, leading to frequent hospitalizations and chronic pain Predominantly Severe, burning pain in abdomen, chest, back Weakness, numbness, respiratory failure female, commonly misdiagnosed Patient Population* 18 Rose Living with Porphyria ~5,000 Patients with sporadic attacks in U.S./EU ~1,000 Patients with recurrent attacks in U.S./EU * ORPHANET; The Porphyria Consortium Confusion, anxiety, seizures, hallucinations Lesions on sunexposed skin; chronic/blistering
19 Mean (SEM) AAR Mean (SEM) AAR Givosiran Interim Phase 1 and OLE Study Results Decreased Annualized Attack Rates (AAR)* Observed with up to 22 Months of Total Treatment in Phase 1 and OLE 25 83% Mean Decrease in AAR Givosiran Compared to Placebo 25 93% Decrease in AAR Givosiran Compared to Phase 1 Run-In % -75% 10-93% Placebo (N=4) 2.5 mg/kg (N=3) 5.0 mg/kg (N=4) mg/kg (N=3) mg/kg (N=3) 0 Run-in Treatment (givosiran**) 1.2 Quarterly Monthly Phase 1 (N=12) OLE (N=12) Phase 1 and OLE Safety: In OLE study (N=16): Two patients with SAEs, including one with anaphylactic reaction, assessed as definitely related to study drug. Patient had past history of asthma, oral allergy syndrome, and prior allergic reactions to acne cream and possibly latex gloves; patient discontinued from study Most common AEs: abdominal pain, nausea, injection site erythema, headache, injection site pruritis, fatigue, nasopharyngitis In Phase 1 (N=40): Six patients with SAEs, including one who developed acute pancreatitis complicated by pulmonary embolism resulting in death, considered unlikely related to study drug Majority of AEs mild-moderate in severity DURABILITY Monthly SC dose regimen 19 Phase 1 and interim OLE study results as of Feb 26, 2018; Sardh et al., EASL, April 2018 * Includes attacks treated in healthcare facility or with hemin ** Aggregated across all dose groups Mean time in Phase 1 run-in and treatment of 103 days and 165 days, respectively; mean time in OLE of 322 days
20 1:1 RANDOMIZATION Phase 3 Study Design Randomized, Double-Blind, Placebo-Controlled Study in Acute Hepatic Porphyria Patients N ~ 75 Patient Population Age 12 years Diagnosis of AHP 2 attacks within prior 6 months Willing to discontinue and/or not initiate hemin prophylaxis Givosiran SC qm 2.5 mg/kg or Placebo SC qm Interim analysis planned in mid-2018 Primary Endpoint Attacks requiring hospitalization, urgent care visit, home IV hemin at 6 months Key Secondary Endpoints ALA and PBG Hemin doses Symptoms QOL Open-Label Extension FDA Breakthrough and EMA PRIME Designations Enrollment completed well ahead of schedule 94 AHP patients, 36 sites, 18 countries Topline results on primary endpoint of annualized attack rate after 6 months of treatment expected in early 2019 Statistical Considerations: 70 patients will have at least 90% power to detect 45% reduction in annualized attack rate at 2-sided alpha of 0.05 Unblinded interim analysis of urinary ALA levels in 30 patients at 3 months 20
21 Annualized Attack Rate Urinary ALA (mmol/mol creatinine) Phase 3 Study Interim Analysis for Potential Accelerated Approval Alignment with FDA that reduction of urinary ALA is reasonably likely to predict clinical benefit Interim analysis with ~30 patients after 3 mo dosing; expect topline data in mid-2018 Expect NDA submission in late 2018 and potential FDA approval in mid Relationship of ALA Lowering with Annualized Attack Rate in Recurrent Attack Patients* 60 ALA Lowering in Recurrent Attack Patients at 2.5 mg/kg qm** Run-In Period Treatment Period 2.5 mg/kg/mo (N=3) placebo (N=4) % >0-25% >25-50% >50-75% >75% ULN ALA increased from baseline More ALA lowering from patient s baseline * Sardh et al., EASL, April 2018; Includes attacks treated in healthcare facility or with hemin ** Sardh et al., ICPP, June 2017 Time, Days
22 Givosiran Market Opportunity Givosiran has potential to address significant unmet needs Current treatment options inadequate 65% of patients have chronic symptoms during and between attacks Significant economic burden Average annual expenditure ranging from approximately $400,000 to $650,000, not reflecting indirect costs Disease significantly under-diagnosed Long diagnostic journey that can exceed 10 years Frequency of gene mutation (2-5:100,000) suggests much larger opportunity Education efforts underway to drive improved diagnosis and disease awareness Primary focus on neurologists, hematologists, gastroenterologists Partnerships with patient advocacy groups Initial opportunity in recurrent population Potential for further expansion Variegate porphyria Hereditary coproporphyria Sporadic attacks 123 tests submitted* 116 positive samples* 22 * As of July 13, 2018
23 Primary Hyperoxaluria Type 1 Lumasiran Description Rare autosomal recessive disorder of increased oxalate synthesis resulting in kidney stones and renal failure, with subsequent oxalate accumulation in extra-renal tissues Retinal Oxalosis Cardiomyopathy Onset generally pediatric, very limited treatment options Nephrocalcinosis Renal stones ESRD Patient Population ~3-5,000 U.S./EU Skeletal Involvement 23 Claire Living with PH1
24 Mean [+/- SEM] Urine Oxalate Content (mmol/24hr/1.73m 2 ) Lumasiran Phase 1/2 Study Initial Results* >60% Mean Reduction in Urinary Oxalate Mean maximal reduction in urinary oxalate of 64% relative to baseline after lumasiran dosing in PH1 patients in Cohorts 1-3 (N=12) Lumasiran 1.0 mg/kg monthly (N=4) Lumasiran 3.0 mg/kg monthly (N=4) Lumasiran 3.0 mg/kg quarterly (N=4) 0.50 ULN: Days since first dose qmonth q3m Part B Safety (N=20): No discontinuations due to AEs Most AEs mild or moderate, unrelated to study drug Two treatment-related AEs (mild and transient injection site reactions) No drug-related SAEs (most common: kidney stones (N=2)) 24 * Data as of March 29, 2018; only data points with at least 3 contributing patients are represented Placebo patient in quarterly cohort had not yet reached Day 85 post lumasiran dosing
25 Lumasiran Phase 3 Study Significant Acceleration in Advancement to Patients Expected Alignment reached on pivotal trial design Primary Endpoint reduction in urinary oxalate at 6 months Sample Size ~25 patients 2020 Initiate Phase 3 study (mid) Report topline results Submit NDA* (early) FDA Breakthrough and EMA PRIME Designations 25 * Assuming positive results
26 26 Other Programs to Watch
27 2:1 2:1 Median ABR 27 Hemophilia and Rare Bleeding Disorders (RBD) Fitusiran Description Genetic deficiency results in inability to generate thrombin and stop bleeding, leading to recurrent bleeds into joints, muscles, and major internal organs PATIENT POPULATION 200,000 itusiran Phase 3 Program* Early 2017 Adults and adolescents with hemophilia A or B with inhibitors On-demand N~50 Adults and adolescents with hemophilia A or B without inhibitors On-demand N~100 worldwide 4,000 with inhibitors Fitusiran OR Endpoints: ABR Bypassing agent (BPA) consumption Quality of life OD BPA Safety Re-initiated Phase 2 OLE and ATLAS Phase 3 studies in December 2017; Endpoints: Expect topline Fitusiran ATLAS results ABR in 2019 Factor VIII or IX OR consumption 1 Clinical results as of Jun 15, 2017; Pasi Quality of et life al., ISTH, July 2017; updated to reflect cerebral OD Factor Safety venous sinus thrombosis case noted in safety box Sanofi Genzyme is leading and fully funding development (post-transition) of fitusiran DURABILITY Monthly SC fixed dose regimen Fitusiran Phase 2 OLE Study in Inhibitor Patients N=14 Pre-Study Median ABR=0 with 6 months [1-11] median duration of dosing in observation period* 0 N=14 Fitusiran Treatment Updated Safety in Phase 2 OLE (N=33): 3 SAEs considered possibly related to study drug Includes one fatal cerebral venous sinus thrombosis enhanced bleed management guidelines and risk mitigation measures added to study protocols following event Majority of AEs mild or moderate in severity, unrelated to study drug Reversible ALT increases >3x ULN in 11 (33%) patients, all with prior history of HCV infection
28 Hypercholesterolemia Inclisiran Description Highly prevalent disease caused by elevated levels of LDL-C that increase risk of atherosclerotic cardiovascular disease (ASCVD) Inclisiran ORION-1 Phase 2 Study DURABILITY Biannual SC dose regimen PATIENT POPULATION ~31 million in U.S. have LDL-C levels >240 mg/dl Completed enrollment in ORION 9, 10, 11 Phase 3 trials with ~3,500 patients; Expect topline results in 2019 >50% mean LDL-C lowering at Day 180 after two quarterly doses* Safety (N=501)*: No drug-related SAEs, no discontinuations due to AEs Two patient deaths due to MI and stroke, both unrelated to study drug No LFT elevations related to study drug Majority of AEs mild or moderate in severity 28 * ORION-1 Phase 2 Study; ESC, Aug 2017 The Medicines Company is leading and funding development of inclisiran from Phase 2 onward and will commercialize program, if successful
29 Mean (+/- SEM) C5 knockdown relative to baseline (%) Complement-Mediated Diseases Cemdisiran (ALN-CC5) Description Numerous debilitating diseases caused by abnormal complement activity Paroxysmal nocturnal hemoglobinura (PNH), atypical hemolytic-uremic syndrome (ahus), myasthenia gravis, neuromyelitis optica, membranous nephropathy Cemdisiran Phase 1/2 Study in Healthy Volunteers mg (N=3) 600 mg (N=3) 200 mg (N=3) 900 mg (N=3) mg (N=3) Placebo (N=5) Days since first visit PATIENT POPULATION ~5,000 ahus >100,000 total complementmediated diseases Mean max C5 knockdown of 98%; Durability supports qm to q3m SC dose regimen 1 Part A Safety (N=20): No SAEs No discontinuations due to AEs All reported AEs mild or moderate Expect Phase 2 ahus initial data in Data as of 03/02/2016
30 RNAi Therapeutics in Development for CNS Diseases Limited Therapies to Prevent or Reverse Neurodegenerative Disease Sequence specific target knockdown across brain and spinal cord for two targets with single intrathecal dose* Confirmed sirna uptake in several different cell types Widespread distribution and knockdown in all key anatomical regions of brain and spinal cord tissue Examples of dominantly inherited genes within neurodegenerative diseases: Alzheimer s disease Parkinson s disease Frontotemporal dementia Huntington s disease Amyotrophic lateral sclerosis (ALS) Spinocerebellar ataxia Planned Next Steps 1 st DC in late st IND in late 2019/early INDs/year starting in * TIDES 2018 Annual Meeting
31 31 Guidance and Goals
32 32
33 Goal to Bring Innovation to Patients and Markets Around World 2018 ONPATTRO is indicated in the U.S. for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults^ Givosiran Lumasiran TTRsc02 Fitusiran Inclisiran Acute hepatic porphyrias Primary hyperoxaluria ATTR amyloidosis Hemophilia Hypercholesterolemia Partnered programs*: * Sanofi Genzyme is leading and funding development (post-transition) of fitusiran and will commercialize program, if successful; The Medicines Company is leading and funding development of inclisiran and will commercialize program, if successful ^ ONPATTRO is not yet approved in the EU; On July 27, 2018, CHMP recommended for approval to treat hattr amyloidosis in adults with stage 1 or stage 2 polyneuropathy Anticipated dates of launch based on current development timelines and assuming regulatory approval
34 34 Alnylam 2018 Goals 2018* *Early is Q1-Q2, Mid is Q2-Q3, and Late is Q3-Q4 PATISIRAN (hattr Amyloidosis) GIVOSIRAN (Acute Hepatic Porphyrias) FITUSIRAN (Hemophilia and RBD) ALN-TTRsc02 (ATTR Amyloidosis) INCLISIRAN (Hypercholesterolemia) LUMASIRAN (Primary Hyperoxaluria Type 1) ADDITIONAL CLINICAL PROGRAMS Additional APOLLO Phase 3 data FDA approval U.S. launch J-NDA submission EMA approval EU launch Additional ROW submissions Additional Phase 1/Phase 2 OLE data ENVISION Phase 3 interim analysis topline NDA filing Complete ENVISION Phase 3 enrollment Continue ATLAS Phase 3 enrollment Start Phase 3 Complete ORION 9/10/11 (LDL-C) enrollment Start ORION 4 (CVOT) Phase 3 Start Phase 3 Continue to advance early/mid-stage pipeline; File new INDs; Present clinical data Early Mid Late
35 Financial Summary and Guidance 2018 Q2 Financial Results Cash $1.48B Includes $44.8M in restricted investments GAAP Revenues $29.9M Total GAAP Operating Expenses $222.3M R&D Expenses $137.6M G&A Expenses $84.7M Total Non-GAAP Operating Expenses* $200.1M Non-GAAP R&D Expenses* $126.0M Non-GAAP G&A Expenses* $74.1M GAAP Net Loss $163.6M Non-GAAP Net Loss** $161.9M Shares Outstanding 100.6M Affirmed 2018 Financial Guidance Cash, including restricted cash and restricted investments, of ~$1.0B Annual Non-GAAP Operating Expenses Non-GAAP R&D Expenses* in the range of $420M to $460M Non-GAAP SG&A Expenses* in the range of $280M to $320M 35 * Non-GAAP operating expenses exclude stock-based compensation expenses. ** Non-GAAP net loss excludes stock-based compensation expenses and the gain on litigation settlement.
36 To those who say impossible, impractical, unrealistic, we say: CHALLENGE ACCEPTED Alnylam Pharmaceuticals, Inc.
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