Paradigm for Migraine Patients

Transcription

1 June Transforming 14, 2018 the Prevention Treatment Paradigm for Migraine Patients January 2019

2 Forward-Looking Statements This presentation and the accompanying commentary contains certain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Forward-looking statements include all statements that are not historical facts and typically contain words such as believe, will, may, estimate, continue, anticipate, intend, should, plan, approximately, expect, predict, could, support, potential, opportunity, positive, significant, unique, strong, unmet, need, design, strategy, advance, options, robust, unique, path, milestones, upcoming, enable, ensure, maintain, achieve, sufficient, projected, forecasted, new, sets, establishes, on track, freedom or the negative of these terms or other similar expressions. You should consider forward-looking statements carefully because they discuss future expectations, contain projections of future results of operations or financial condition, or state other forward-looking information. These statements relate to our possible and future results of operations, financial condition, business strategies, development plans, regulatory activities, competitive position, commercial plans, potential growth opportunities and effects of competition and the assumptions that underlie these statements. These forwardlooking statements are subject to certain risks and uncertainties that could cause actual results to differ materially from those anticipated in the forward-looking statements. Factors that might cause such a difference include, but are not limited to, the risks outlined under the caption Risk Factors set forth in Alder s Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2018, which was filed with the Securities and Exchange Commission (SEC) November 5, 2018 and is available on the SEC s website at and in other reports and filings we will make with the SEC from time to time. Forward-looking statements are based on our management s beliefs and assumptions and on information currently available to our management. These statements, like all statements in this presentation, speak only as of the date of this presentation (or an earlier date, where specifically noted), and except as required by law, we undertake no obligation to update or revise these statements in light of future developments. For investor audiences only. 2

3 Alder BioPharmaceuticals Compelling Value Proposition Poised to Transform the Treatment Paradigm for Migraine Prevention LARGE UNDERSERVED MARKET OPPORTUNITY EPTINEZUMAB LEAD CANDIDATE DIFFERENTIATED CLINICAL PROFILE EPTINEZUMAB WELL POSITIONED TO GAIN SIGNIFICANT MARKET SHARE ALD1910 PIPELINE PRODUCT POTENTIAL ALTERNATIVE PATHWAY FOR MIGRAINE PREVENTION 3

4 Migraine is a Serious Debilitating Neurological Disease Most disabling disease of people under the age of 501 Top cause of years lived with a disability during the most productive years, Attacks usually last 4-72 hours2 Depression, anxiety, and sleep disturbances2 are common for those with chronic migraine Symptoms include extreme pain, nausea, vomiting, extreme sensitivities to light and sound, gastrointestinal issues 1Steiner, Per year, $13B lost productivity in U.S million lost work days2 TJ, Stovner, LJ, & Vos, T. The Journal of Headache and Pain (2018) 19:17. Research Foundation. Migraine Facts. Available at: Accessed January 2, Migraine 4

5 U.S. Anti-CGRP Migraine Prevention Forecasted To Be A > $5 Billion Market1 13 Million Patient Candidates Eligible for Migraine Prevention2 Oral preventives fail to meet needs of many patients: Adherence, Safety, Tolerability Issues4 ONLY % 27 of Patients on Migraine Prevention2,3 BOTOX Chronic Migraine Est. 250K-275K patients treated in Newly Launched Anti-CGRP SubQ Products Est k NRx Patients in < 8 months6 1DRG 2018 Migraine Market report. of patients based on Alder estimates using third party publicly available data (US Census Bureau; Migraine Research Foundation; Buse DC, Manack AN, Fanning KM, et al. Chronic migraine prevalence, disability, and sociodemographic factors: Results from the American Migraine Prevalence and Prevention Study. Headache 2012;52: ). 3Data on file Marketscan, September Parsekyan D. Migraine prophylaxis in adult patients. West J Med. 2000;173(5): Estimates based on AGN Q3 public earnings statements. 6IQVIA NPA Weekly through 12/17/18. 2Number 5

6 Patients are Suffering & Need Preventive Medications that Work, and work More Quickly 1 2 % 87 rate effectiveness as important in determining whether they accept treatment (highest-rated) 1 rate fast acting as an important treatment feature when considering migraine prevention 1 79 % 64 % would always choose the most effective option regardless of administration method 1 90 % Say I will never give up fighting to find a solution 1 1 Alder proprietary patient market research, 2017 (N=250). 2 Migraine Research Foundation. Migraine Facts. Available at: Accessed November 19,

7 Alder BioPharmaceuticals Compelling Value Proposition Poised to Transform the Treatment Paradigm for Migraine Prevention LARGE UNDERSERVED MARKET OPPORTUNITY EPTINEZUMAB LEAD CANDIDATE DIFFERENTIATED CLINICAL PROFILE EPTINEZUMAB WELL POSITIONED TO GAIN SIGNIFICANT MARKET SHARE ALD1910 PIPELINE PRODUCT POTENTIAL ALTERNATIVE PATHWAY FOR MIGRAINE PREVENTION 7

8 Eptinezumab Differentiation Designed Into the Molecule Enabling Delivery To Meet Patient Needs Eptinezumab mab Quarterly Infusion High specificity with potent binding affinity to the ligand for rapid suppression of CGRP biology 1 Total administered dose is immediately active to inhibit CGRP with 100% bioavailability 1,2 Eptinezumab s highly competitive clinical profile 1. Baker B, Schaeffler B, Cady R, et al; Rational design of a monoclonal antibody (mab) inhibiting calcitonin gene-related peptide, ALD403 (eptinezumab), intended for the prevention of migraine. Poster presented at the American Academy of Neurology (AAN) 2017 Annual Meeting. 2. As compared to 50% -70% for subcutaneous anti-cgrps; Vu et.al., Pharm Res Sep; 34(9): ; Vermeersch, et al., J Pharmacol Exp Ther 354: , September

9 Completed Pivotal Studies Demonstrated Eptinezumab s Competitive Profile PROMISE 1 in Episodic Migraine Patients (N=888; baseline ~9 migraine days per month) Met primary and key secondary endpoints Safety and tolerability similar to placebo PROMISE 2 in Chronic Migraine Patients (N=1,072; baseline ~16 migraine days per month) Met primary and all key secondary endpoints Safety and tolerability similar to placebo RAPID Preventive benefit onset Day One post-infusion EFFECTIVE 50%, 75% and 100% reductions in migraine days SUSTAINED Efficacy sustained and further improved through repeated quarterly infusions NOTE: Comparisons are not based on data resulting from head-to-head trials and are not direct comparisons of safety or efficacy. Different protocol designs, trial designs, patient selection and populations, number of patients, trial endpoints, trial objectives and other parameters that are not the same between the relevant trials may cause any comparisons of results from different trials to be unreliable. 1. PROMISE 1 efficacy as compared with the best reported Phase 3 clinical data for anti-cgrp mabs as reported in press releases, published literature and product labels, where applicable. 2. PROMISE 2 efficacy as compared with the best reported Phase 3 clinical data for anti-cgrp mabs and onabotulinumtoxina as reported in press releases, published literature and product labels, where applicable. 9

10 Rapid: Statistically Significant Day 1 Onset of Prevention Demonstrated PROMISE 2: Chronic Migraine % of Patients with Migraine, Days % Infusion 1 Patients with Migraine, % 50% 40% 30% Average Daily Baseline Prevalence of Migraine: ~58% Day 1 Reduction in Migraine Prevalence Eptinezumab, 300mg, 52% reduction + Eptinezumab, 100mg, 50% reduction + Placebo 27% reduction 20% Day 0 Day 1 Day 7 Day 14 Day 21 Day 28 + statistically significant; Day 1 prevalence rate comparison between eptinezumab vs. placebo Lipton R et al. A Phase 3 Study to Evaluate Eptinezumab for Preventive Treatment of Chronic Migraine: Results of PROMISE-2 (PRevention Of Migraine via Intravenous eptinezumab Safety and Efficacy-2) Trial. Poster Presentation at the American Headache Society (AHS) 2018 Annual Meeting. June 28 July 1, 2018; San Francisco, CA. 10

11 Effective and Sustained: 50% Responder Rate by Month 1 and Further Improved with Subsequent Infusions PROMISE 2: Chronic Migraine 50% Migraine Responder Rates Eptinezumab 100 mg Eptinezumab 300 mg Placebo 64.0 Patients, %* INFUSION 1 Month 1 INFUSION 1 Months 1-3 INFUSION 2 Months statistically significant Lipton R et al. Eptinezumab for Prevention of Chronic Migraine: Results of 2 Quarterly Intravenous Infusions in the Phase 3 PROMISE-2 (PRevention Of Migraine via Intravenous eptinezumab Safety and Efficacy-2) Trial. Scientific Presentation at the American Headache Society (AHS) 2018 Annual Meeting. June 28 July 1, 2018; San Francisco, CA. 11

12 Effective and Sustained: 75% Responder Rate by Month 1 and Further Improved with Subsequent Infusions PROMISE 2: Chronic Migraine 75% Migraine Responder Rates 50 Eptinezumab 100 mg Eptinezumab 300 mg Placebo Patients, %* INFUSION 1 Month 1 INFUSION 1 Months 1-3 INFUSION 2 Months statistically significant Lipton R et al. Eptinezumab for Prevention of Chronic Migraine: Results of 2 Quarterly Intravenous Infusions in the Phase 3 PROMISE-2 (PRevention Of Migraine via Intravenous eptinezumab Safety and Efficacy-2) Trial. Scientific Presentation at the American Headache Society (AHS) 2018 Annual Meeting. June 28 July 1, 2018; San Francisco, CA. 12

13 Effective and Sustained: Average 100% Responder Rate Month 1 to 3 Further Improved with Subsequent Infusions PROMISE 2: Chronic Migraine 100% Migraine Responder Rates 30 Eptinezumab 100 mg Eptinezumab 300 mg Placebo Patients, %* INFUSION 1 Month 1 INFUSION 1 Months 1-3 INFUSION 2 Months statistically significant (nominal) Lipton R et al. Eptinezumab for Prevention of Chronic Migraine: Results of 2 Quarterly Intravenous Infusions in the Phase 3 PROMISE-2 (PRevention Of Migraine via Intravenous eptinezumab Safety and Efficacy-2) Trial. Scientific Presentation at the American Headache Society (AHS) 2018 Annual Meeting. June 28 July 1, 2018; San Francisco, CA. 13

14 PROMISE 2: Safety Data Consistent with Other Eptinezumab Studies TEAE = Treatment Emergent Adverse Event; * All Serious TEAEs judged unrelated to study drug PROMISE 2: 12-Week Safety Data Eptinezumab 300mg, N=350 n (%) Eptinezumab 100mg, N=356 n (%) Placebo N=366 n (%) Subjects with Any TEAE 155 (44) 136 (38) 144 (39) Subjects with Any Serious TEAE* 3 (<1) 3 (<1) 3 (<1) TEAEs >2% in eptinezumab dose groups Nasopharyngitis 22 (6) 13 (4) 15 (4) Nausea 12 (3) 6 (2) 6 (2) Upper Respiratory Infection 14 (4) 11 (3) 16 (4) Urinary Tract Infection 11 (3) 7 (2) 6 (2) Arthralgia 8 (2) 3 (<1) 3 (<1) Dizziness 9 (3) 5 (1) 4 (1) Anxiety 7 (2) 4 (1) 0 Fatigue 6 (2) 7 (2) 4 (1) Kudrow D et al. Eptinezumab Achieved Reductions in Migraine Activity as Early as Day 1 That Were Sustained Through Week 12: Results From PROMISE-2 (PRevention of Migraine via Intravenous eptinezumab Safety and Efficacy-2) Phase 3 Trial in Chronic Migraine. Poster Presentation at the American Academy of Neurology (AAN) 2018 Annual Meeting. April 21-27, 2018; Los Angeles, CA. 14

15 Eptinezumab Uniquely Competitive Profile (Chronic Migraine Absolute Data 1 ) Efficacy Endpoints Eptinezumab Promise-2 2 Absolute Data Range of Results Chronic Preventive Treatments 1 Absolute data Primary Endpoint: Mean Change from Baseline Monthly Migraine Days Day 1 Reduction in Migraine Risk Months 1-3 >50% Responder Rates Month 1 >75% Responder Rates Months 1-3 >75% Responder Rates -7.7 (100mg) -8.2 (300mg) (Months 1-3) 50% (100mg) 52% (300mg) 57.6% (100mg) 61.4% (300mg) 30.9% (100mg) 36.9% (300mg) 26.7% (100mg) 33.1% (300mg) Not Reported 28% % 4 Not Reported 17% 6-21% 6 NOTE: Comparisons are not based on data resulting from head-to-head trials and are not direct comparisons of safety or efficacy. Different protocol designs, trial designs, patient selection and populations, number of patients, trial endpoints, trial objectives and other parameters that are not the same between the relevant trials may cause any comparisons of results from different trials to be unreliable. 1 Sourced from Aimovig, AJOVY, Emgality and BOTOX product inserts and HCP promotional websites. 2 Data on File, Alder BioPharmaceuticals PROMISE 2 Study 011; Absolute data reported for 300mg and 100mg eptinezumab dose groups. 3 AJOVY (fremanezumab-vfrm) injection Current Prescribing Information. North Wales, PA: Teva Pharmaceuticals USA, Inc. Primary endpoint was reduction in monthly headache days of at least moderate severity (months 1-3). 4 Data at Month 3, Aimovig Prescribing Information. Thousand Oaks, CA: Amgen Inc. and East Hanover, NJ: Novartis Pharmaceuticals Corporation. 5 Emgality [Prescribing Information]. Indianapolis, IN: Lilly USA, LLC. 6 Data at Month 3, Aimovg HCP promotional website referencing Brandes JL, Dolezil D, Freeman MC, et al. Chronic migraine treatment with erenumab: responder rates. Poster presented at: American Headache Society, 59th Annual Scientific Meeting; June 8 11, 2017; Boston, MA. 15

16 Alder BioPharmaceuticals Compelling Value Proposition Poised to Transform the Treatment Paradigm for Migraine Prevention LARGE UNDERSERVED MARKET OPPORTUNITY EPTINEZUMAB LEAD CANDIDATE DIFFERENTIATED CLINICAL PROFILE EPTINEZUMAB WELL POSITIONED TO GAIN SIGNIFICANT MARKET SHARE ALD1910 PIPELINE PRODUCT POTENTIAL ALTERNATIVE PATHWAY FOR MIGRAINE PREVENTION 16

17 HCP Market Research Suggests Eptinezumab Well-Positioned for Adoption HCPs highly rated Eptinezumab on efficacy with 50% / 75% responder rates, reduction in monthly migraine days and rapid onset of effect 2 Procedure-Oriented Headache Specialists, Neurologists, Pain Specialists and PCPs 1 3 in 5 physicians believe less frequent administration and rapid onset of effect are important to anti-cgrp prescribing 2 ~20-30% 45 HCP feedback on % potential eptinezumab share of anti-cgrp market based on clinical profile 2 HCPs are likely to add or expand their IV capabilities 2 1 Alder proprietary physician market research, Alder proprietary ATU physician market research,

18 Specialty Sized Market Poised to Adopt New IV Procedures For Migraine Prevention Distribution of CGRP Rx to date by HCP 120% 100% 50% of scripts written by 820 HCPs 80% of scripts written by 2400 HCPs 20 % % of Business 80% 60% 40% 20% Top 2,432 HCPs (1,490 with infusion procedures) cover 80% CGRP, 43% Oral, 58% Botox 2 Of HCPs have prescribed 80% of anti-cgrp SubQ scripts 2 ~3,000 Procedure-Oriented Headache Specialists Neurologists, Pain Specialists and PCPs 1 See the largest number of highly impacted migraine patients Value patient adherence and product control Value practice economics 0% % Have IV access 1 63% have in-office # of HCPs 77% Have prescribed an IV to their patients access 1 for migraine 1 Specialty Size Sales Force of Alder proprietary physician market research, IQVIA Alder Migraine Target List Study

19 Market Research Suggests Highly Impacted Migraine Patients Prefer Eptinezumab Infusion ~5-7M Highly Impacted Chronic and More Severe Episodic Migraine Patients 1 74 % believe IV medications work the fastest when compared to other injection forms of administration 2 52 % of patients in market research would choose an infusion product with Eptinezumab s profile over a subcutaneous preventive therapy 2 1. Alder estimate of potential U.S. patient population for eptinezumab based on Alder proprietary market research. 2. Alder proprietary patient market research, 2017 (N=250) 19

20 Alder is Uniquely Positioned to Capture High Value Market Opportunity Migraine is a Serious Debilitating Disease Eptinezumab s Differentiated Characteristics2 ~ 3,000 Proceduralist HCP Migraine Specialists RAPID Preventive benefit achieved Day One post-infusion3 US Anti-CGRP Migraine Prevention Forecasted to be a >$5 Billion Market1 EFFECTIVE >50%, >75% and 100% reductions in migraine days SUSTAINED $ DRG 2018 Migraine Market report PROMISE 1 and PROMISE 2 studies 3 Benefit observed within the first infusion period 4 Alder proprietary market research ATU Alder proprietary patient market research, 2017 (N=250) Efficacy sustained for 3 months following a single administration ~20-30% potential Eptinezumab share of anticgrp prevention market4 ~ 5-7M Highly Impacted Chronic and More Severe Episodic Migraine Patients1 52% Of patients would choose Eptinezumab over a subcutaneous therapy5 1 2 Eptinezumab 20

21 Eptinezumab Key Milestones Event Completion PROMISE 1 Episodic Migraine PROMISE 2 Chronic Migraine Open Label Safety Study PK Comparability Study BLA Submission Eptinezumab Anticipated Launch Q Q

22 Alder BioPharmaceuticals Compelling Value Proposition Poised to Transform the Treatment Paradigm for Migraine Prevention LARGE UNDERSERVED MARKET OPPORTUNITY EPTINEZUMAB LEAD CANDIDATE DIFFERENTIATED CLINICAL PROFILE EPTINEZUMAB WELL POSITIONED TO GAIN SIGNIFICANT MARKET SHARE ALD1910 PIPELINE PRODUCT POTENTIAL ALTERNATIVE PATHWAY FOR MIGRAINE PREVENTION 22

23 ALD1910 Aims to Expand Migraine Prevention with Anti-PACAP Antibody PACAP inhibition offers a differentiated and complementary approach to migraine prevention ALD1910 Like CGRP, infusion of PACAP has been shown to induce migraine attacks in migraine patients ~75% 1 Estimated to respond well to eptinezumab (anti-cgrp) 2 Preclinical data indicate that PACAP and CGRP may have non-overlapping pharmacology with 5M respect to migraineassociated symptoms 2,3 ALD1910 is a first-in-class anti-pacap antibody Highly potent and selective humanized antibody Ligand approach allows blockage of the three peripheral PACAP receptors GMP manufacturing and IND-enabling studies underway to support potential 2H2019 IND 1 Schytz et al., Neurotherapeutics 2010; 7(2): Kaiser & Russo, Neuropeptides 2013;47(6): Alder unpublished research demonstrating that PACAP-driven migraine symptoms in rodents are not inhibited by anti-cgrp antibodies ALD1910 is an investigational product and has not been approved by the FDA or any other regulatory body. 23

24 Q Financial Results and Financial Outlook* Q Financial Results Cash position of $484.7M 1 as of Sept 30, cash investment 2 in the range of $250M and $270M Sufficient cash to meet projected operating requirements into 2020 with key activities including: BLA submission and filing Establishment of eptinezumab commercial drug supply chain Continued build out of Alder s commercial organization (e.g., marketing, sales, medical affairs, payor access, IT) Pre-launch market readiness Jan ATM facility established to provide financing optionality 1 Includes cash, cash equivalents, short and long-term investments and restricted cash 2 Net cash used in operating activities plus purchases of property and equipment as defined under U.S. Generally Accepted Accounting Principles. *Outlook as of November 5,

25 June Transforming 14, 2018 the Prevention Treatment Paradigm for Migraine Patients January 2019

26 Appendix 26

27 Rapid: Statistically Significant Day 1 Onset of Prevention Demonstrated PROMISE 1: Episodic Migraine % of Patients with Migraine, Days 1-28 Percent of Patients with Migraine 35% 30% 25% 20% Average (28 day) Baseline Prevalence of Migraine: ~ 30% Infusion 1 Day 1 Reduction in Migraine Prevalence Eptinezumab, 300mg, 55% reduction + Eptinezumab, 100mg, 52% reduction + Placebo 24% reduction 15% 10% Day 0 Day 1 Day 7 Day 14 Day 21 Day 28 + Statistically significant (unadjusted) Silberstein, S et al. Eptinezumab Results for the Prevention of Episodic Migraine Over 1 Year in the PROMISE-1 (PRevention Of Migraine via Intravenous eptinezumab Safety and Efficacy-1) Trial. Poster Presentation at the American Headache Society (AHS) 2018 Annual Scientific Meeting. June 28 July 1, 2018; San Francisco, CA. 27

28 50% Responder Rates by Month 1 Sustained and Further Improved with Subsequent Infusions Baseline: ~8.6 Migraine Days PROMISE 1: Episodic Migraine 50% Migraine Responder Rates 80 Eptinezumab 100 mg Eptinezumab 300 mg Placebo Patients, %* INFUSION 1 Month 1 INFUSION 1 Months 1-3 INFUSION 4 Months statistically significant; + statistically significant (unadjusted); statistically significant (post-hoc) Saper J et al. Neurology. 2018;90(suppl 15):S20. Scientific Platform Presentation by Silberstein S at the American Academy of Neurology (AAN) 2018 Annual Meeting, April 21-27, 2018; Los Angeles, CA Silberstein, S et al. Eptinezumab Results for the Prevention of Episodic Migraine Over 1 Year in the PROMISE-1 (PRevention Of Migraine via Intravenous eptinezumab Safety and Efficacy-1) Trial. Poster Presentation at the American Headache Society (AHS) 2018 Annual Scientific Meeting. June 28 July 1, 2018; San Francisco, CA. 28

29 75% Responder Rates by Month 1 Sustained and Further Improved with Subsequent Infusions Baseline: ~8.6 Migraine Days PROMISE 1: Episodic Migraine 75% Migraine Responder Rates 60 Eptinezumab 100 mg Eptinezumab 300 mg Placebo Patients, %* NS INFUSION 1 Month 1 INFUSION 1 Months 1-3 INFUSION 4 Months statistically significant; statistically significant (post-hoc); NS, not significant Saper J et al. Neurology. 2018;90(suppl 15):S20. Scientific Platform Presentation by Silberstein S at the American Academy of Neurology (AAN) 2018 Annual Meeting, April 21-27, 2018; Los Angeles, CA Silberstein, S et al. Eptinezumab Results for the Prevention of Episodic Migraine Over 1 Year in the PROMISE-1 (PRevention Of Migraine via Intravenous eptinezumab Safety and Efficacy-1) Trial. Poster Presentation at the American Headache Society (AHS) 2018 Annual Scientific Meeting. June 28 July 1, 2018; San Francisco, CA. 29

30 100% Responder Rates by Month 1 Sustained and Further Improved with Subsequent Infusions Baseline: ~8.6 Migraine Days PROMISE 1: Episodic Migraine 100% Migraine Responder Rates 40 Eptinezumab 100 mg Eptinezumab 300 mg Placebo Patients, %* NS INFUSION 1 Month 1 INFUSION 1 Months 1-3 INFUSION 4 Months statistically significant (unadjusted); NS, not significant Average percentage of patients with 100% response at any given month Saper J et al. Neurology. 2018;90(suppl 15):S20. Scientific Platform Presentation by Silberstein S at the American Academy of Neurology (AAN) 2018 Annual Meeting, April 21-27, 2018; Los Angeles, CA Silberstein, S et al. Eptinezumab Results for the Prevention of Episodic Migraine Over 1 Year in the PROMISE-1 (PRevention Of Migraine via Intravenous eptinezumab Safety and Efficacy-1) Trial. Poster Presentation at the American Headache Society (AHS) 2018 Annual Scientific Meeting. June 28 July 1, 2018; San Francisco, CA. 30