EX-99.2 3 exhibit992.htm EXHIBIT 99.2
FY18 Financial Results Conference Call & Webcast February 28, 2019

2 Forward-Looking Statements This presentation contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of our product candidates, the timing and reporting of results from preclinical studies and clinical trials, the prospects and timing of the potential regulatory approval of our product candidates, commercialization plans, manufacturing and supply plans, financing plans, and the projected revenues and cash position for the Company. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding the goals, progress, timing, and outcomes of discussions with regulatory authorities, and in particular the potential goals, progress, timing, and results of preclinical studies and clinical trials, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that results of clinical or preclinical studies indicate that the product candidates are unsafe or ineffective; the potential that it may be difficult to enroll patients in our clinical trials; the potential that regulatory authorities, including the FDA, EMA, and PMDA, may not grant or may delay approval for our product candidates; the potential that we may not be successful in commercializing Galafold in Europe, Japan, the US and other geographies or our other product candidates if and when approved; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues; the potential that we may not be able to manufacture or supply sufficient clinical or commercial products; and the potential that we will need additional funding to complete all of our studies and manufacturing. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results. With respect to statements regarding projections of the Company's revenue and cash position, actual results may differ based on market factors and the Company's ability to execute its operational and budget plans. In addition, all forward-looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2018 filed today. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof.

Introduction 3 Rare Disease Day – February 28, 2019 Rare Disease Day 2019 is “Bridging Health and Social Care”

Introduction 4 Amicus Highlights GALAFOLD’S EXTRAORDINARY LAUNCH AT-GAA IN POMPE: POTENTIAL TO LEADING GENE THERAPY PORTFOLIO IN RARE SUCCESS BECOME STANDARD OF CARE METABOLIC DISEASES • 650+ Patients and $91.2M Global Sales in FY18 • Continued Strength of Clinical Data • Pipeline of 14 Gene Therapies • FY19 Guidance of $160M-$180M • Granted Breakthrough Therapy Designation • 2 Clinical Stage Programs • $500M Potential Sales by 2023 • Multiple Data Expected Throughout 2019 • Established Global Research and Gene Therapy • $1B+ Addressable Market Opportunity by 2028 • 100+ Pompe Patients on AT-GAA by YE19 Center of Excellence in Philadelphia • $1B-$2B+ Market Opportunity • $1B+ Peak Recurring Market Opportunity FINANCIAL STRENGTH 2023 VISION • $500M+ Cash at 12/31/18 (runway into mid-2021) • 5,000+ Lives Transformed • Growing Contribution from Galafold Revenues • $1B+ in Revenue • Leading Global Rare Disease Biotech

Introduction 5 2019 Key Strategic Priorities 1 Nearly double annual revenue for Galafold (guidance $160M-$180M) Complete enrollment in AT-GAA Pivotal Study (PROPEL) and 2 report additional Phase 1/2 data Report additional 2-year clinical results in CLN6-Batten disease and 3 complete enrollment in ongoing CLN3-Batten disease Phase 1/2 study Establish preclinical proof of concept for Fabry and Pompe gene 4 therapies 5 Maintain strong financial position

Galafold® (migalastat) Global Launch… …taking a leadership role in the treatment of Fabry disease “We push ideas as far and as fast as possible” - Amicus Belief Statement

Galafold: Precision Medicine for Fabry Disease 7 Galafold Snapshot (as of December 31, 2018) $91.2M $160-180M One of the Most FY18 Galafold FY19 Global Revenue Galafold Successful Rare Rev. Guidance Disease Launches Geographic 24 Countries with Expansion in Pricing & 2019 Reimbursement 8 Regulatory 348 Approvals: Amenable Australia, Canada, EU, Variants in U.S. Israel, Japan, S. Korea, Switzerland , U.S. Label Galafold is indicated for adults with a confirmed diagnosis of Fabry Disease and an amenable mutation/variant. The most common adverse reactions reported with Galafold (≥10%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia. For additional information about Galafold, including the full U.S. Prescribing Information, please visit https://www.amicusrx.com/pi/Galafold.pdf. For further important safety information for Galafold, including posology and method of administration, special warnings, drug interactions and adverse drug reactions, please see the European SmPC for Galafold available from the EMA website at www.ema.europa.eu.

Galafold: Precision Medicine for Fabry Disease 8 Galafold Success and FY18 Galafold Revenue Guidance On Track to Nearly DOUBLE Revenue Again and Serve 1,000+ Patients in 2019 $160M-180M Guidance $91.2M $32.6M $36.9M $20.6M $80$21.3M-90M $4.9M $16.7M FY16 FY17 FY18 FY19

Galafold: Precision Medicine for Fabry Disease 9 Upside Potential Total Amenable Patient Population (“TAPP”) WORLDWIDE Estimate based on 35% - 50% amenability Diagnosis grows due to newborn screening in U.S. & Japan $1B+ Addressable Market TAPP: 4,700-6,750 Peak Potential Opportunity by 2028 WORLDWIDE Diagnosis continues at current rate TAPP: 4,200-6,000 Today WORLDWIDE* (U.S. & Japan Added) 2028 TAPP: 3,800-5,500 EU & ROW Only TAPP: 2,000-3,000 2018 2017 *WORLDWIDE includes total amenable patient population in all Fabry ERT commercial markets today Estimated effect of newborn screening on adult diagnostic rate.

AT-GAA Novel ERT for Pompe Disease “We encourage and embrace constant innovation” - Amicus Belief Statement

AT-GAA for Pompe Disease 11 AT-GAA: Breakthrough Therapy Designation U.S. FDA Granted BTD to AT-GAA in Late-Onset Pompe Disease (LOPD) BTD Criteria • Intended to treat a serious or life threatening disease or condition • Preliminary clinical evidence indicates drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints AT-GAA BTD Based on Ph 1/2 Clinical Efficacy • Improvements in 6-minute walk distance • Comparison to natural history of treated patients BTD Features • Intensive guidance on an efficient drug development program • Potential Rolling BLA • Organizational commitment involving senior managers • Potential for Priority Review • All Fast Track program features

AT-GAA for Pompe Disease 12 AT-GAA: 2019 Objectives Advance AT-GAA for as Many Patients Worldwide as Quickly as Possible  Additional Phase 1/2 Data (up to 24 Months)  Breakthrough Therapy Designation • Enroll PROPEL study (n=100) • Present additional Phase 1/2 data (Cohort 4) • Report natural history study data • Initiate supportive studies • Advance agreed upon CMC requirements to support BLA

Gene Therapy Pipeline “We have a duty to obsolete our own technologies” - Amicus Belief Statement

Amicus Pipeline 14 DISCOVERY PRECLINICAL PHASE 1/2 PHASE 3 REGULATORY COMMERCIAL Fabry Franchise Galafold®(migalastat) monotherapy Fabry Gene Therapy PENN Pompe Franchise AT-GAA (Novel ERT + Chaperone) Pompe Gene Therapy PENN Batten Franchise – Gene Therapies NCH CLN6 Batten Disease Advancing one NCH CLN3 Batten Disease of the most CLN8 Batten Disease NCH robust rare CLN1 Batten Disease NCH disease Rare CNS and Other Gene Therapies portfolios in CDKL5 Deficiency Disorder GTx / ERT PENN biotechnology Niemann-Pick Type C (NPC) NCH Tay-Sachs Disease NCH Wolman Disease NCH NCH / Other PENN

Amicus Gene Therapy Programs 15 Leading LSD Gene Therapy Portfolio Multiple Platforms Provide 14 Gene Therapy Programs and R&D Engine for Future Growth Gene Therapy Anticipated Milestones 2-Year CLN6 Batten Disease Phase 1/2 Data (Mid- 2019) CLN3 Batten Disease Phase 1/2 Study Enrolling CLN8 and CLN1 Batten Disease Preclinical Studies Underway Fabry and Pompe Preclinical Studies in Progress Engines for Future Growth

Amicus Gene Therapy Programs 16 Amicus Global Research and Gene Therapy Center of Excellence in Philadelphia Strengthens Amicus Capabilities as a Leading Global Rare Disease Biotechnology Company in Burgeoning Hub for Medical Breakthroughs » New 75,000 sqft state-of-the-art facility » Located in uCity Square Adjacent to Penn Campus » Global Amicus science organization and gene therapy leadership team headquarters » Co-led by Jeff Castelli, PhD, Chief Portfolio Officer and Head of Gene Therapy and Hung Do, PhD, CSO » Initial group occupying temporary space » Permanent space to eventually hold ~200 employees Artist rendering

Milestones “We have a duty to obsolete our own technologies” - Amicus Belief Statement

Upcoming Milestones 18 Anticipated Milestones: 2019 Well-Positioned to Create Significant Value for Shareholders and Patients in 2019 Galafold: Fabry Disease AT-GAA: Pompe Disease Gene Therapy Programs • FY19 revenue guidance $160M-  Additional Phase 1/2 data (21 • Ongoing CLN3 Batten disease $180M and 24 months) Phase 1/2 study enrollment • Growth in existing markets  Breakthrough therapy • Additional 2-year data from CLN6 designation (BTD) in LOPD Batten disease Phase 1/2 study • Expansion into new markets • PROPEL pivotal study enrollment • Preclinical proof of concept for • Diagnostic initiatives (n=100) Fabry, Pompe and CDD • Additional Phase 1/2 data • Preclinical work across additional (Cohort 4) neurologic LSDs • Natural history study data • Additional supportive studies • Advance CMC requirements to support BLA

A Rare Company Commercial Organizational Strength “We are business led and science driven” - Amicus Belief Statement

Commercial Organizational Strength 20 Global Footprint Global operations in North America EU place across the largest rare disease markets » employees supporting our global 150+ Galafold business » ~100 in Commercial » ~50 in Medical Affairs » countries with offices, including US, 11 EU5, Japan ROW APAC countries with direct presence (Amicus » 20 personnel) » 24 markets with reimbursement » markets with Amicus presence (direct or 27 distributor)

Commercial Organizational Strength 21 Experienced Passionate Commercial Team Proven global capabilities driving Galafold’s extraordinary launch success Diverse Experience in Background Passion for Rare Disease and Patients Drug Launch Experiences Galafold is indicated for adults with a confirmed diagnosis of Fabry Disease and an amenable mutation/variant. The most common adverse reactions reported with Galafold (≥10%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia. For additional information about Galafold, including the full U.S. Prescribing Information, please visit https://www.amicusrx.com/pi/Galafold.pdf. For further important safety information for Galafold, including posology and method of administration, special warnings, drug interactions and adverse drug reactions, please see the European SmPC for Galafold available from the EMA website at www.ema.europa.eu.

Commercial Organizational Strength 22 An Innovative Product Novel Mechanism, Precision Medicine, Commercial Execution Product Execution . Differentiated product profile – . Clear launch focus on priority first oral treatment option patient segments . Rapid determination of . Efficient outreach to key Fabry amenability (website) disease centers . Driving science through . Strong education efforts on Publications, FollowMe Registry importance of genotype and Post Marketing Studies Galafold is indicated for adults with a confirmed diagnosis of Fabry Disease and an amenable mutation/variant. The most common adverse reactions reported with Galafold (≥10%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia. For additional information about Galafold, including the full U.S. Prescribing Information, please visit https://www.amicusrx.com/pi/Galafold.pdf. For further important safety information for Galafold, including posology and method of administration, special warnings, drug interactions and adverse drug reactions, please see the European SmPC for Galafold available from the EMA website at www.ema.europa.eu.

Commercial Organizational Strength 23 Market Access Our medicines must be fairly priced and broadly accessible.” - Amicus Founding Belief . Proven patient-centric strategy of ensuring fair price and broad accessibility . Rapid, successful reimbursement in global markets . Amicus assist™ patient support program . Amicus PROMISE to limit price increases to consumer inflation . Amicus “pledge” to reinvest until there’s a cure Galafold is indicated for adults with a confirmed diagnosis of Fabry Disease and an amenable mutation/variant. The most common adverse reactions reported with Galafold (≥10%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia. For additional information about Galafold, including the full U.S. Prescribing Information, please visit https://www.amicusrx.com/pi/Galafold.pdf. For further important safety information for Galafold, including posology and method of administration, special warnings, drug interactions and adverse drug reactions, please see the European SmPC for Galafold available from the EMA website at www.ema.europa.eu.

Financial Summary Full Year Ended December 31, 2019 “Maximizing Value for Shareholders is the Foundation for Our Future Successes” - Amicus Belief Statement

Financial Summary 25 FY18 Select Financial Results FY18 Revenue of $91.2M Primarily from International Galafold Sales Dec. 31, 2018 (in thousands, except per share data) Dec. 31, 2017 Product revenue 91,245 36,930 Cost of goods sold 14,404 6,236 R&D expense 270,902* 149,310 SG&A expense 127,200 88,671 Changes in fair value of contingent consideration 3,300 (234,322) Loss on impairment of assets - 465,427 Loss from operations (328,777) (441,985) Income tax benefit 94 165,119 Net loss (348,995) (284,002) Net loss per share (1.88) (1.85) *Inclusive of upfront payment of $100 million for the Celenex asset acquisition

Financial Summary 26 Financial Summary and Guidance Strong Balance Sheet with $500M+ Cash at 12/31/18 - Cash Runway into 2021 FINANCIAL POSITION December 31, 2018 Cash ~$504M Cash Runway Into at least mid-2021 CAPITALIZATION Shares Outstanding 189,383,924 FINANCIAL GUIDANCE Projected YE 2019 Cash Balance ~$300M Galafold Revenue Guidance $160M-$180M

Closing 27 Impacting Lives >350 Patients* | ~$36M Global Sales >700 Patients* | ~$91M1 Global Sales 5,000 Patients* | $1B Global Sales YE17 YE18 2023 *Clinical & commercial, all figures approximate 1Preliminary unaudited

Appendix

AT-GAA for Pompe Disease 29 Pompe Patient Experience in Phase 1/2 Clinical Study (ATB200-02) Consistent and Durable Responses Across Key Measures of Safety, Functional Outcomes and Biomarkers in both ERT-Switch and ERT-Naïve Pompe Patients out to Month 18 6-Minute Walk Test (m) Change at Month 24a,b Cohort Baseline (n=10) (n=8) Mean (SD) Cohort 1 397.2 +53.6 ERT-Switch Ambulatory (96.8) (36.4) Change at Month 21 Cohort Baseline (n=5) (n=5) Mean (SD) Cohort 3 399.5 +54.8 ERT-Naïve (83.5) (34.7) FVC (% Predicted) Change at Month 24a,b,c Cohort Baseline (n=9*) (n=7) Mean (SD) Cohort 1 52.6 -0.6 ERT-Switch Ambulatory* (14.7) (2.8) Change at Month 21 Cohort Baseline (n=5) (n=5) Mean (SD) Cohort 3 53.4 +6.1 ERT-Naïve (20.3) (9.7) aOne patient in Cohort 1 discontinued from study (withdrew consent) before Month 24. bAt the time of this interim analysis, 1 patient in Cohort 1 had not reached Month 24. cBaseline FVC missing for 1 patient in Cohort 1

AT-GAA for Pompe Disease 30 PROPEL (ATB200-03) Study Design 52-Week Primary Treatment Period Long-Term Extension (Double-Blind) (Open-Label) Participants with Late-Onset AT-GAA Pompe Disease Bi-Weekly ~100 Patients AT-GAA 90 Clinical Sites Worldwide Bi-weekly Standard of Care ERT-Switch ERT-Naïve Bi-Weekly Primary Endpoint: 6-Minute Walk Test at Week 52 Multiple Secondary Endpoints

Thank You “Our passion for making a difference unites us” -Amicus Belief Statement