爱美医疗 2024年度报告

2 0 2 4A N N U A L R E P O R T AboutAmicus O U R M I S S I O N To develop and deliver transformativemedicines for people living with rare diseases O U R V I S I O N To become one of the leading raredisease-focused biotech companies To ourshareholders Bradley L. CampbellPresident and ChiefExecutive Officer 2024 represented a truly pivotal yearin the 20-plus history of Amicus. Onbehalf of our patient-centered team,I am delighted to share some of thehighlights of this past year with you,andour excitement about what’sahead for this special company. Wehave the foundation that we need to deliversustainable long-term growth: two approved medicineswithcompelling value propositions in growing rarediseasecategories;strong intellectual property forboth of these medicines; a leverageable commercialorganization; and an increasingly strong financial position. With the momentum we now have across our globalbusiness, we anticipate that we will deliver $1 billionin total revenue in 2028. Our growth trajectory, ourrare-diseasefocus,and our financial discipline willenable us to deliver double-digit growth and patientimpact in 2025 and for years to come, and to invest incontinued innovation in Fabry and Pompe disease andbusiness development. As we closed 2024, we reached several importantmilestones for Amicus. First and most importantly, wesurpassed 3,000 people living with a rare diseasetaking an Amicus medicine. We also delivered morethan $500 million in annual revenue and achieved non-GAAP profitability in 2024. We accomplished all of thisbecause of our laser focus on our mission to developand deliver transformative medicines for people livingwith rare diseases. A VISION FOR THE FUTURE Amicus is well positioned to become one of the leadingglobal rare disease companies and represents a rareblendof patient focus,demonstrated commercialperformance,sustainable long-term growth,andfinancial strength. Within our two disease areas, thereare more than 10,000 diagnosed patients – and morethan 100,000 undiagnosed patients. We’re resolute inour commitment to reach these individuals, and ouraim is to exceed expectations in the number of liveswe reach. Amicus stands out as a unique story in the biotechspace – both in terms of what we have accomplished,and what we are on track to do. OUR STRATEGIC PRIORITIES FOR 2025 This year we are focused on several key priorities,including: Thiswill be accomplished by executing on ourcommerciallaunches,fully leveraging our globalinfrastructureandcapabilities,anddevelopinginnovative new medicines– all while staying true towho we are, a patient-focused rare disease company.With a clear path forward, we are confident in our abilityto create lasting value for patients and shareholdersalike. Thank you for your continued support as we enterour next phase of success. 1.Driving continued double-digit revenue growththrough the uptake of both Galafold andPombiliti + Opfolda2.Advancing our ongoing studies to broadenindications and reinforce our leadership inFabry and Pompe disease3.Maintaining our financial discipline as we continueon our path to GAAP profitability Sincerely,Bradley Campbell SUSTAINABLE LONG-TERM GROWTH We’re proud of the progress that we’ve made, andexcited about what’s ahead in our journey to reachmore and more people around the world with ourtransformative medicines. A RareCompany Amicus Therapeutics is a global, patient-dedicatedbiotechnology company focused on discovering,developing and delivering novel, high-qualitymedicines for people living with rare diseases. (As of December 31, 2024) $528MNet Product Sales 2 ApprovedTherapies Galafold® and Pombiliti® + Opfolda® ~500Dedicated Employees Globally Global Footprint in20+ Countries LeverageableGlobal CommercialOrganization Non-GAAPProfitability Achieved in FY 20241 Amicus Therapeutics2024 Annual Report Reaching PatientsAcross the GlobeheeGGlobeeGGl bbPPatieents Amicus is a global organization, united bya passion for making a differenceby Our global footprint spans over 20 countries, including our globalheadquarters in Princeton, NJ and international headquarters in Marlow, UK.Additional international office locations include Australia, Canada, France,Japan, Germany, Italy, the Netherlands, and Spain. Core ValueDriversiDD Galafold: Building a LeadershipPosition in the Treatment ofFabry DiseaseLdBuld:afoGaentmrehe Tn thonitiPo 2,700+ patients and $458M global salesin FY24024n Fastest growing treatment for Fabrydisease globallyStandard of care in amenable populationallygloaseise pomecar Pombiliti + Opfolda: ResettingTherapeutic Expectations forPeople Living with Late-onsetPompe Disease ~220 patients treated or scheduled fortreatment and $70M global sales in FY24 Positive feedback increasing among patientsand physicians across all approved markets Securing broad patient access and initiatingmultiple successful launches throughout 2025 Corporate Outlook: A UniqueStory in Biotech with SignificantReven