PTC Therapeutics Inc 2025年季度报告

FORM10-Q (Mark One) ☑QUARTERLY REPORT PURSUANT TO SECTION13 OR 15(d)OF THE SECURITIESEXCHANGE ACT OF 1934 For the quarterly period endedMarch31,2025 or ☐TRANSITION REPORT PURSUANT TO SECTION13 OR 15(d)OF THE SECURITIESEXCHANGE ACT OF 1934 For the transition period fromtoCommission file number:001-35969 PTC Therapeutics,Inc.(Exact name of registrant as specified in its charter) (State or other jurisdiction of incorporation ororganization) Securities registered pursuant to Section12(b)of the Act: Name of each exchange on whichregistered Common Stock, $0.001 par value pershare Indicate by check mark whether the registrant (1)has filed all reports required to be filed by Section13 or 15(d)ofthe Securities Exchange Act of 1934 during the preceding 12months (or for such shorter period that the registrantwas required to file such reports), and (2)has been subject to such filing requirements for the past 90days.YesþNo☐ Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to besubmitted pursuant to Rule405 of Regulation S-T (§232.405 of this chapter) during the preceding 12months (or forsuch shorter period that the registrant was required to submit such files).YesþNo☐ Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer,a smaller reporting company, or an emerging growth company. See the definitions of “large accelerated filer”,“accelerated filer,” “smaller reporting company,” and "emerging growth company" in Rule12b-2 of the ExchangeAct. If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transitionperiod for complying with any new or revised financial accounting standards provided pursuant to Section13(a)ofthe Exchange Act.☐ Indicate by check mark whether the registrant is a shell company (as defined in Rule12b-2 of the Exchange Act).Yes☐Noþ As of May 4, 2025, there were79,257,019shares of Common Stock, $0.001 par value per share, outstanding. TABLE OF CONTENTSPTC Therapeutics,Inc. PART II—OTHER INFORMATION Item6. Exhibits55 FORWARD-LOOKING STATEMENTS This Quarterly Report on Form10-Q contains forward-looking statements that involve substantialrisks and uncertainties. All statements, other than statements of historical facts, contained in thisQuarterly Report on Form10-Q, including statements regarding our strategy, future operations, futurefinancial position, future revenues, projected costs, prospects, plans and objectives of management,are forward-looking statements. The words “anticipate,” “believe,” “estimate,” “expect,” “intend,”“may,”“might,”“plan,”“predict,”“project,”“target,”“potential,”“will,”“would,”“could,”“should,” “continue,” and similar expressions are intended to identify forward-looking statements,although not all forward-looking statements contain these identifying words. The forward-looking statements in this Quarterly Report on Form10-Q include, among other things,statements about: ●the outcome of pricing, coverage and reimbursement negotiations with third-party payors forour products or product candidates that we commercialize or may commercialize in thefuture;●expectations with respect to sepiapterin for the treatment of phenylketonuria, including anyregulatorysubmissions and potential approvals,commercialization,and the potentialachievement of regulatory and sales milestones and contingent payments that we may beobligated to make●our ability to maintain our marketing authorization of Translarna for the treatment ofnonsense mutation Duchenne muscular dystrophy, or nmDMD, in Brazil, Russia and otherregions in which Translarna has been approved;●the effect of the European Commission’s adoption of the Committee for Medicinal Productsfor Human Use’s negative opinion not to renew the conditional marketing authorization forTranslarna in the European Economic Area on other regulatory bodies;●our ability to use the results of Study 041, a randomized, 18-month, placebo-controlledclinical trial of Translarna for the treatment of nmDMD followed by an 18-month open-labelextension, and from our international drug registry study to support a marketing approval forTranslarna for the treatment of nmDMD inthe United States;●whether investigators agree with our interpretation of the results of clinical trials and thetotality of clinical data from our trials of Translarna;●expectationswith respect to our license and collaboration agreement with NovartisPharmaceuticals Corporation, or Novartis, including our right to receive any development,regulatory and sales milestones, and profit sharing and royalty payments from Novartis;●expectations with respect to vatiquinone for the treatment of Friedreich’s ataxia, includingany regulatory submissions and potential approvals, commercialization, and the potentialachievement of regulatory and sales milestones and contingent payments that we may beobligated to make;●exp