PTC Therapeutics Inc 2024年度报告

Introduction PTC is a global biopharmaceutical company that discovers, develops andcommercializes clinically differentiated medicines that provide benefitsto children and adults living with rare disorders. Our ability to innovateto identify new therapies and to globally commercialize products is thefoundation that drives investment in a robust and diversified pipelineof transformative medicines. The company’s strategy is to leverage itsstrong scientific expertise and global commercial infrastructure to delivertransformative therapies for patients who have little to no treatment options. Our People Our Science Our Commitment Our focus on excellencebegins with developing andretaining a global workforcethat is equipped to lead intheir fields. By adapting newways of working, drivingexecution excellence,and staying true to ourpatient-focused missionwe further strengthen ourpatient-centric, compliantand innovative culture. Ourcommitment to patientsdrives us to think differentlyabout solutions and to workcollaboratively as One PTC. Our research efforts arefocused on two scientificplatforms - Splicingand Inflammation andFerroptosis - where PTChas unique expertise todiscover and advanceinnovative therapies to theclinic. We have a robustdevelopment portfolio witha number of potentiallypromising therapies for rareneurologic and metabolicdiseases includingphenylketonuria (PKU),Friedreich’s ataxia (FA) andHuntington’s Disease (HD). We are committed tochildren and adults livingwith serious diseases ofhigh unmet need. We workhard to provide resourcesand support to patients andtheir families throughouttheir rare disorder journeythrough compassionatecollaboration, throughoutthe drug developmentprocess. We strive to ensurewe deeply understand apatient’s disease journeyand involve them everystep of the way – fromearly research anddevelopment to clinicaltrials, to commercializationand support programs. A Message to Our Shareholders we received an additional $1.0billion. This strong cash positionprovides us with the resources tosupport our planned commerciallaunches, continue to invest inour innovative R&D platforms andengage in business developmentactivities to complement our existingcommercial and R&D portfolios.We now have the potential to becashflow breakeven without theneed to raise additional capital. revenue guidance despite significantheadwinds for our global Duchennemuscular dystrophy business.This commercial performance is atestament to our commercial teams’ability to effectively execute aroundthe globe, even in genericizedand competitive markets. As we began 2024, I shared ourambitious plans to position PTCfor future success. I am proudto say that 2024 was a year ofoutstanding execution acrossevery part of the company. We achieved all planned clinicaland regulatory milestones on time,including the submission of fourapproval applications to the U.S.Food and Drug Administration (FDA),all of which were accepted for review,including: Kebilidi™ (eladocageneexuparvovec-tneq) for AADCdeficiency which was approvedin November 2024 and is the first-ever direct-to-brain administeredgene therapy approved by FDA;sepiapterin for phenylketonuria (PKU)which has a regulatory action date ofJuly 29, 2025; Translarna™ (ataluren)for nonsense mutation Duchennemuscular dystrophy (nmDMD); andvatiquinone for Friedreich’s ataxia(FA) which was accepted with priorityreview and has a regulatory actiondate of Aug. 19, 2025. In additionto the U.S. filings, we submittedmarketing applications for sepiapterinin key markets globally including theEuropean Union (EU), Brazil, and Japan. In 2025, we are planning for anotheryear of execution and success. Thereare a number of potential importantvalue-creating milestones ahead,including the potential global launchof sepiapterin, expected data readoutfrom the PIVOT-HD Phase 2 study ofPTC518 in Huntington’s Disease (HD)patients, and a number of additionalscheduled regulatory decisionsin the U.S. and outside the U.S. The global launch of sepiapterin willbe PTC’s first-ever global launch. Thereremains a significant unmet needfor PKU patients as the vast majorityof patients are not well controlledby available therapies. The datacollected to date demonstrate thatsepiapterin can provide meaningfulbenefit to the full range of PKUpatients. Data from ongoing studies Through our revenue performance,effective management of operatingexpenses and the rapid and robustmonetization of the priority reviewvoucher received with the FDAapproval of Kebilidi, we ended 2024with over $1.1 billion in cash. In addition,following the closing of the NovartisPTC518 transaction in January 2025, We also had an outstanding year ofcommercial performance, exceeding first approximately 30 patientswho completed 12 months oftreatment. All key objectives weremet: dose-dependent and durablelowering of mutant Huntingtinprotein in blood and demonstrateddose-dependent lowering ofcerebrospinal fluid mutant Huntingtinprotein levels in