| Forward - qLooqking Statements This prqesqeqntatioqn coqntaiqns forward - lookiqng statqemqeqnts withiqn thqe mqeaqniqng of Thqe Privatqe Sqecuritiqes Litigatioqn Rqeform Act of 1995 .. All statqemqeqnts coqntaiqnqed iqn this prqesqeqntatioqn, othqer thaqn statqemqeqnts of historic fact, arqe forward - lookiqng statqemqeqnts, iqncludiqng statqemqeqnts with rqespqect prqelimiqnary uqnauditqed 2022 fiqnaqncial iqnformatioqn with rqespqect to 2022 total qnqet rqevqeqnuqe aqnd 2022 DMD fraqnchisqe qnqet product rqevqeqnuqe, statqemqeqnts with rqespqect to guidaqncqe rqelatiqng to 2023 total qnqet product rqevqeqnuqe, 2023 DMD fraqnchisqe qnqet product rqevqeqnuqe aqnd 2023 opqeratiqng qexpqeqnditurqe guidaqncqe aqnd statqemqeqnts rqegardiqng: thqe futurqe qexpqectatioqns, plaqns aqnd prospqects for PTC, iqncludiqng with rqespqect to thqe qexpqectqed timiqng of cliqnical trials aqnd st udi qes, availability of data, rqegulatory submissioqns aqnd rqespoqnsqes aqnd othqer mattqers; qexpqectatioqns with rqespqect to Upstaza aqnd othqer programs withiqn PTC's gqeqnqe thqerapy platform, iqncludiqng aqny rqegulatory submissioqns, commqercializatioqn aqnd maqnufacturiqng capabilitiqes; advaqncqemqeqnt of PTC's joiqnt collaboratioqn program iqn SMA, iqncludiqng aqny rqegulatory submissioqns, commqercializatioqn or royalty o r m ilqestoqnqe paymqeqnts; PTC's qexpqectatioqns with rqespqect to thqe licqeqnsiqng, rqegulatory submissioqns aqnd commqercializatioqn of its products aqnd product caqndidatqes; PTC's stratqegy, futurqe opqeratioqns, futur qe f iqnaqncial positioqn, futurqe rqevqeqnuqes, projqectqed costs; aqnd thqe objqectivqes of maqnagqemqeqnt. Othqer forward - lookiqng statqemqeqnts may bqe idqeqntifiqed by thqe words, "guidaqncqe", "plaqn," "aqnticipatqe," "bqeliqevqe," "qestim atqe," "qexpqect," "iqntqeqnd," "may," "targqet," "potqeqntial," "will," "would," "could," "should," "coqntiqnuqe," aqnd similar qexprqessioqns. PTC's actual rqesults, pqerformaqncqe or achiqevqemqeqnts could diffqer matqerially from thosqe qexprqessqed or impliqed by forward - lookiqng sta tqemqeqnts it makqes as a rqesult of a variqety of risks aqnd uqncqertaiqntiqes, iqncludiqng thosqe rqelatqed to: qexpqectatioqns with rqespqect to thqe COVID - 19 paqndqemic aqnd rqelatqed rqespoqnsqe mqeasurqes aqnd thqeir qeffqects o qn PTC's busiqnqess, opqeratioqns, cliqnical trials, rqegulatory submissioqns aqnd approvals, aqnd PTC's collaborators, coqntract rqesqearch orgaqnizatioqns, suppliqers aqnd maqnufacturqers; thqe outcomqe of priciqng, cov qera gqe aqnd rqeimbursqemqeqnt qnqegotiatioqns with third party payors for PTC's products or product caqndidatqes that PTC commqercializqes or may commqercializqe iqn thqe futurqe; qexpqectatioqns with rqespqect to Upstaza aqnd othqer programs withiqn PTC's gqeqnqe thqerapy platform, iqncludiqng aqny rqegulatory submissioqns aqnd potqeqntial approvals, commqercializatioqn, maqnufacturiqng capabilitiqes aqnd thqe potqeqntial fiqnaqncial imp act aqnd bqeqnqefits of its lqeasqed biologics maqnufacturiqng facility aqnd thqe potqeqntial achiqevqemqeqnt of dqevqelopmqeqnt, rqegulatory aqnd salqes milqestoqnqes aqnd coqntiqngqeqnt paymqeqnts that PTC may bqe obligatqed to makqe; qexpqect ati oqns with rqespqect to thqe commqercializatioqn of Evrysdi uqndqer our SMA collaboratioqn; PTC's ability to maiqntaiqn its markqetiqng authorizatioqn of Traqnslarqna for thqe trqeatmqeqnt of qnmDMD iqn Brazil, Russia, thqe Europqeaqn Ecoqnomic Arqea (EEA) aqnd othqer rqegioqns, iqncludiqng whqethqer thqe Europqeaqn Mqediciqnqes Agqeqncy (EMA) dqetqermiqnqes iqn futurqe aqnqnual rqeqnqewal cyclqes that thqe bqeqnqefit - risk balaqncqe of Traqnslarqna authorizatioqn supports rqeqnqewal of such authorizatioqn; PTC's ability to complqetqe Study 041, which is a spqecific obligatioqn to coqntiqnuqed markqetiqng authorizatioqn iqn thqe EEA; PTC's ability to utilizqe rqes ults from Study 041, a raqndomizqed, 18 - moqnth, placqebo - coqntrollqed cliqnical trial of Traqnslarqna for thqe trqeatmqeqnt of qnmDMD followqed by aqn 18 - moqnth opqeqn - labqel qextqeqnsioqn, to support a markqetiqng approval for Traqnslarqna for thqe trqeatmqeqnt of qnmDMD iqn thqe Uqnitqed Statqes aqnd a coqnvqersioqn to a staqndard markqetiqng authorizatioqn iqn thqe EEA; qexpqectatioqns with rqespqect to thqe commqercializatioqn of Tqegsqedi aqnd Waylivra ; thqe rqesults of PTC's cliqnical trial for qemvododstat for COVID - 19; sigqnificaqnt busiqnqess qeffqects, iqncludiqng thqe qeffqects of iqndustry, markqet, qecoqnomic, political or rqegulatory coqnditioqns; chaqngqe s i qn tax aqnd othqer laws, rqegulatioqns, ratqes aqnd policiqes; thqe qeligiblqe patiqeqnt basqe aqnd commqercial potqeqntial of PTC's products aqnd product caqndidatqes; PTC's sciqeqntific approach aqnd gqeqnqeral dqevqelopmqeqnt progrqess ; P TC's ability to satisfy its obligatioqns uqndqer thqe tqerms of its lqeasqe agrqeqemqeqnts, iqncludiqng for its lqeasqed biologics maqnufacturiqng facility; PTC's ability to satisfy its obligatioqns uqndqer thqe tqer ms of thqe sqecurqed crqedit facility with Blackstoqnqe; thqe sufficiqeqncy of PTC's cash rqesourcqes aqnd its ability to obtaiqn adqeqqnPeuatqe fiqnaqnciqng iqn thqe futurqe for its forqesqeqeablqe aqnd uqnforqesqeqeablqe opqeratiqng qexpqeqnsqes aqn d capital qexpqeqnditurqes; aqnd thqe factors discussqed iqn thqe "Risk Factors" sqectioqn of PTC's most rqecqeqnt Aqnqnual Rqeport oqn Form 10 - K, as wqell as aqny updatqes to thqesqe risk factors filqed from timqe to timqe iqn PTC's othqer filiqngs with thqe SEC. You arqe urgqed to carqefully coqnsidqer all such factors. As with aqny pharmacqeutical uqndqer dqevqelopmqeqnt, thqerqe arqe sigqnificaqnt risks iqn thqe dqevqelopmqeqnt, rqegulatory approval aqnd commqerc ial izatioqn of qnqew products. Thqerqe arqe qno guaraqntqeqes that aqny product will rqecqeivqe or maiqntaiqn rqegulatory approval iqn aqny tqerritory, or provqe to bqe commqercially succqessful, iqncludiqng Traqnslarqna , Emflaza , Upstaza , Evrysdi, Tqegsqedi or Waylivra .. Thqe forward - lookiqng statqemqeqnts coqntaiqnqed hqerqeiqn rqeprqesqeqnt PTC's viqews oqnly as of thqe datqe of this prqesqeqntatioqn aqnd PTC doqes qnot uqndqertakqe or plaqn to updatqe or rqevisqe aqny such forward - lookiqng statqemqeqnts to rqeflqect actual rqesults or chaqngqes iqn plaqns, prospqects, assumptioqns, qestimatqes or projqectioqns, or othqer circumst aqnc qes occurriqng aftqer thqe datqe of this prqesqeqntatioqn qexcqept as rqeqqnPeuirqed by law. JP Morgan Presentation 2023 2 |
