UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM
CURRENT REPORT
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Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).
Emerging growth company
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
Item 2.02 Results of Operations and Financial Condition.
On January 9, 2023, PTC Therapeutics, Inc. (the “Company”) issued a press release (the “press release”) announcing certain preliminary (unaudited) financial information for its fiscal year ending December 31, 2022, including that the Company expects to report (i) total unaudited net revenue of approximately $710 million, (ii) total unaudited net product revenue of approximately $535 million, (iii) net product revenue for Translarna™ (ataluren) of approximately $289 million and net product revenue for Emflaza® (deflazacort) of approximately $218 million and (iv) collaboration and royalty revenue associated with Evrysdi of approximately $175 million. Final results are subject to completion of the Company’s year-end audit.
Item 7.01. Regulation FD Disclosure.
On January 9, 2023, the Company also announced financial guidance for its fiscal year ending December 31, 2023 in the press release, including that the Company anticipates (i) full year total revenues to be between $940 million and $1.0 billion (ii) full year net product revenues for the Duchenne muscular dystrophy franchise to be between $545 and $565 million (iii) GAAP R&D and SG&A expense for the full year 2023 to be between $1.01 and $1.06 billion with non-GAAP R&D and SG&A expense for the full year 2023 to be between $890 and $940 million, excluding estimated non-cash, stock-based compensation expense of approximately $120 million and (iv) up to $80 million of one-time payments upon achievement of potential clinical and regulatory success-based milestones from previous acquisition.
The Company announced that on Monday, January 9, 2023 at 10:30 am EST at the 41st Annual J.P. Morgan Healthcare Conference, the Company will provide an update on 2022 accomplishments and highlight upcoming 2023 potential value-creating milestones. The Company will also present its preliminary 2022 financial results and 2023 financial guidance. The presentation will be webcast live on the Events and Presentations page under the Investors section of the Company’s website.
A copy of the press release is attached to this Current Report on Form 8-K as Exhibit 99.1 and is incorporated by reference into this Item 7.01.
This Current Report on Form 8-K and Exhibits 99.1 and 99.2 include a forward-looking financial measure that was not prepared in accordance with accounting principles generally accepted in the United States (GAAP), non-GAAP R&D and SG&A expenses (which excludes non-cash stock-based compensation expense). Management uses this measure when assessing and identifying operational trends and, in management’s opinion, this non-GAAP measure is useful to investors and other users of its financial statements by providing greater transparency into the historical and projected operating performance of the Company and the Company’s future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP.
The information set forth in or incorporated by reference into Item 2.02 or this Item 7.01, including Exhibits 99.1 and 99.2, shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such a filing. All website addresses given in this Current Report on Form 8-K or incorporated herein by reference are for information only and are not intended to be an active link or to incorporate any website information into this Current Report on Form 8-K.
Forward Looking Statements: All statements, other than those of historical fact, contained in this Current Report on Form 8-K, are forward-looking statements, including reporting expectations with respect to financial information for fiscal year 2022 and financial guidance for fiscal year 2023. The Company's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to the preliminary nature of the Company's 2022 financial information, which is subject to completion of the Company's year-end audit; the assumptions underlying the Company's financial guidance for 2023; and the factors discussed in the “Risk Factors” section of the Company's Annual Report on Form 10-K for the year ended December 31, 2021 as well as any updates to these risk factors filed from time to time in the Company's other filings with the Securities and Exchange Commission. You are urged to carefully consider all such factors. The forward-looking statements contained herein and the exhibits hereto represent the Company's views only as of the date of this Current Report on Form 8-K and the Company does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this Current Report on Form 8-K except as required by law.
Item 9.01. Financial Statements and Exhibits.
(d) Exhibits
Exhibit No. |
| Description |
99.1 | Press Release, dated January 9, 2023 issued by PTC Therapeutics, Inc. | |
99.2 | Corporate Presentation – 41st Annual J.P. Morgan Healthcare Conference | |
104 | The cover page from this Current Report on Form 8-K, formatted in Inline XBRL |
Signature
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this Report to be signed on its behalf by the undersigned hereunto duly authorized.
PTC Therapeutics, Inc. | ||
Date: January 9, 2023 | By: | /s/ Emily Hill |
Name: | Emily Hill | |
Title: | Chief Financial Officer | |
EXHIBIT 99.1
PTC Therapeutics Provides an Update on Commercial Progress and R&D Pipeline
at 41st Annual J.P. Morgan Healthcare Conference
- ~$710 million unaudited 2022 total revenue, representing an impressive over 30% year-over-year growth –
- $940 million - $1.0 billion 2023 total revenue guidance –
- Results from three registration-directed clinical trials expected in 2023 –
- One additional registration-directed clinical trial expected to initiate in 2023 –
SOUTH PLAINFIELD, N.J., Jan. 9, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) will present an update on its commercial progress and R&D pipeline at the 41st Annual J.P. Morgan Healthcare Conference today, Monday January 9, at 10:30am EST/7:30am PST. Matthew Klein, M.D., Chief Operating Officer of PTC Therapeutics, will provide an update on 2022 accomplishments and highlight upcoming 2023 potential value-creating milestones. Preliminary 2022 unaudited financial results and 2023 financial guidance will also be provided. The presentation will be webcast live on the Events and Presentations page of the Investors section of PTC Therapeutics website at www.ptcbio.com.
Key 2022 Corporate Highlights:
| ● | Upstaza™, the first direct-administered gene therapy into the brain, was approved in the EU and UK for AADC deficiency patients. |
| ● | Unaudited net product revenue of $535 million in 2022 representing 25% year-over-year growth. |
| o | Strong year-over-year growth for the Duchenne muscular dystrophy (DMD) franchise, with unaudited net product revenue of $289 million for Translarna™ (ataluren) and $218 million for Emflaza® (deflazacort) in 2022. |
| ◾ | Translarna growth was driven by new patients in existing geographies and continued geographic expansion. |
| ◾ | Emflaza growth was due to continued new prescriptions, high compliance, fewer patient discontinuations and more favorable access. |
| o | Upstaza™ unaudited net product revenue was $13 million driven by patients being treated through early access programs and commercial access. |
| ● | Evrysdi® (risdiplam) is now approved in more than 90 countries. It has established market leadership in all major markets and is on track to become the global market leader in treatment of spinal muscular atrophy (SMA). Evrysdi is a product of the SMA collaboration between PTC, the SMA Foundation and Roche. |
| ● | PTC successfully advanced its clinical pipeline in 2022: |
| o | CardinALS, a registration-directed Phase 2 clinical trial of PTC857 in amyotrophic lateral sclerosis, was initiated. |
| o | PIVOT-HD, a Phase 2 clinical trial of PTC518 in Huntington’s disease, was initiated. |
| o | SunriseLMS, a registration-directed Phase 2 trial of unesbulin in leiomyosarcoma, was initiated. |
| o | The placebo-controlled portion of Study 041, a Phase 3 clinical trial of Translarna for nmDMD, was completed. Study 041 results supported submission of a Type II variation for conversion to standard marketing authorization in the EU. |
| o | FITE-19, a Phase 2/3 clinical trial of emvododstat for COVID-19, was completed. |
2023 Potential Key Value-Creating Milestones:
| ● | Results from the placebo-controlled portion of APHENITY, the Phase 3 registration-directed clinical trial of sepiapterin in patients with PKU, are expected in the first quarter of 2023. |
| ● | Results from MIT-E, the Phase 2/3 registration-directed clinical trial of vatiquinone in mitochondrial disease associated seizures, are expected in the first quarter of 2023. |
| ● | Results from MOVE-FA, the Phase 3 registration-directed clinical trial of vatiquinone in Friedreich ataxia, are expected in the second quarter of 2023. |
| ● | Results from the 12-week portion of PIVOT-HD, the Phase 2 study of PTC518 in Huntington's disease, are expected in the second quarter of 2023. |
| ● | Submission of a Biologics License Application (BLA) to the FDA for Upstaza is expected in the first half of 2023. |
| ● | A Phase 2/3 clinical trial of unesbulin in diffuse intrinsic pontine glioblastoma is expected to initiate in the fourth quarter of 2023. |
Preliminary Unaudited 2022 Financial Results:
| ● | Total unaudited net revenue for full year 2022 was approximately $710 million. |
| ● | Total unaudited net product revenue for full year 2022 was approximately $535 million. |
| ● | DMD franchise unaudited revenue for full year 2022 was approximately $507 million, including net product revenue for Translarna of approximately $289 million and for Emflaza of approximately $218 million. |
| ● | PTC expects to report approximately $175 million in 2022 collaboration and royalty revenue associated with Evrysdi. |
PTC is currently in the process of finalizing its financial results for the 2022 fiscal year. The above information is based on preliminary unaudited information and management estimates for the full year 2022, subject to the completion of PTC's financial closing procedures. Evrysdi royalty revenue estimates are based on sell side analyst consensus estimates.
2023 Financial Guidance:
| ● | PTC anticipates total revenues for the full year 2023 to be between $940 million and $1.0 billion. |
| ● | PTC anticipates net product revenues for the DMD franchise for the full year 2023 to be between $545 and $565 million. |
| ● | PTC anticipates GAAP R&D and SG&A expense for the full year 2023 to be between $1.01 and $1.06 billion. |
| ● | PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2023 to be between $890 and $940 million, excluding estimated non-cash, stock-based compensation expense of $120 million. PTC anticipates up to $80 million of one-time payments upon achievement of potential clinical and regulatory success-based milestones from previous acquisitions. |
Non-GAAP Financial Measures:
In this press release, the financial results and financial guidance of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in GAAP because
management uses these non-GAAP financial measures when assessing and identifying operational trends. In management's opinion, these non-GAAP financial measures are useful to investors and other users of PTC's financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company's future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the table below.
PTC Therapeutics, Inc.
Reconciliation of GAAP to Non-GAAP Projected Full Year 2023 R&D and SG&A Expense
(In thousands)
| Low End of Range | High End of Range |
Projected GAAP R&D and SG&A Expense | $ 1,010,000 | $ 1,060,000 |
Less: projected non-cash, stock-based compensation expense | 120,000 | 120,000 |
Projected non-GAAP R&D and SG&A expense | $ 890,000 | $ 940,000 |
About PTC Therapeutics, Inc.
PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. PTC's mission is to provide access to best-in-class treatments for patients who have little to no treatment options. PTC's strategy is to leverage its strong scientific and clinical expertise and global commercial infrastructure to bring therapies to patients. PTC believes this allows it to maximize value for all its stakeholders. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Instagram, Facebook, Twitter, and LinkedIn.
For More Information:
Investors
Kylie O'Keefe
+1 (908) 300-0691
kokeefe@ptcbio.com
Media
Jeanine Clemente
+1 (908) 912-9406
jclemente@ptcbio.com
Forward-Looking Statements:
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including the information provided under the heading
"2023 Financial Guidance", including with respect to (i) 2023 total revenue guidance, (ii) 2023 net product revenue guidance for the DMD franchise,(iii) 2023 GAAP and non-GAAP R&D and SG&A expense guidance and (iv) 2023 acquisition related one-time expense guidance, and statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses and other matters; expectations with respect to Upstaza and other programs within PTC's gene therapy platform, including any regulatory submissions, commercialization and manufacturing capabilities; advancement of PTC's joint collaboration program in SMA, including any regulatory submissions, commercialization or royalty or milestone payments; PTC's expectations with respect to the licensing, regulatory submissions and commercialization of its products and product candidates; PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words, "guidance", "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions.
PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: expectations with respect to the COVID-19 pandemic and related response measures and their effects on PTC's business, operations, clinical trials, regulatory submissions and approvals, and PTC's collaborators, contract research organizations, suppliers and manufacturers; the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; expectations with respect to Upstaza and other programs within PTC's gene therapy platform, including any regulatory submissions and potential approvals, commercialization, manufacturing capabilities and the potential financial impact and benefits of its leased biologics manufacturing facility and the potential achievement of development, regulatory and sales milestones and contingent payments that PTC may be obligated to make; expectations with respect to the commercialization of Evrysdi under our SMA collaboration; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in Brazil, Russia, the European Economic Area (EEA) and other regions, including whether the European Medicines Agency (EMA) determines in future annual renewal cycles that the benefit-risk balance of Translarna authorization supports renewal of such authorization; PTC's ability to complete Study 041, which is a specific obligation to continued marketing authorization in the EEA; PTC's ability to utilize results from Study 041, a randomized, 18-month, placebo-controlled clinical trial of Translarna for the treatment of nmDMD followed by an 18-month open-label extension, to support a marketing approval for Translarna for the treatment of nmDMD in the United States and a conversion to a standard marketing authorization in the EEA; expectations with respect to the commercialization of Tegsedi and Waylivra; the results of PTC's clinical trial for emvododstat for COVID-19; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; PTC's ability to satisfy its obligations under the terms of its lease agreements, including for its leased biologics manufacturing facility; PTC's ability to satisfy its obligations under the terms of the secured credit facility with Blackstone; the sufficiency of PTC's cash resources and its ability to obtain adequate financing in the future for its foreseeable and unforeseeable operating expenses and capital expenditures; and the factors discussed in the "Risk Factors" section of PTC's most recent Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors.
As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Translarna, Emflaza, Upstaza, Evrysdi, Tegsedi or Waylivra.
The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law.
| PKU Patient Matthew Klein, M.D., COO JP Morgan Healthcare Conference January 9, 2023 PTC 2023 |
| 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 |
| Treating Rare Diseases by Modulating Gene and Protein Expression 3 3 DNA Protein RNA JP Morgan Presentation 2023 |
| 4 Treating Rare Diseases by Modulating Gene and Protein Expression JP Morgan Presentation 2023 Bio - e Splicing Nonsense Suppression Gene Therapy |
| Building a Pipeline to Produce a Therapy Every Two to Three Years JP Morgan Presentation 2023 5 2 to 3 years New product every Commercialize Discover Develop |
| Development Research Oncology Metabolism Neurology A Diverse and Robust Portfolio To Continually Create Value JP Morgan Presentation 2023 6 6 SCA - 3 MAP - Tau 3 Undisclosed GT - AS GT - FA PTC518 HD Utreloxastat ALS Unesbulin DIPG Unesbulin LMS Sepiapterin PKU V atiquinone MDAS V atiquinone FA Commercial Small Molecule Gene Therapy DIPG, d:i:f:fuse :intr:ins:ic pont:ine gl:ioma; FA, Fr:iedre:ich atax:ia; ALS, amyotroph:ic lateral scleros:is; HD, Hunt:ington‘s d:isease; LMS , le:iomyosarcoma; MDAS, m:itochondr:ial d:isease assoc:iated se:izures; PKU, phenylketonur:ia; SCA - 3, sp:inocerebellar atax:ia type 3. 2 Undisclosed 13 Undisclosed |
| Continuing Strong Financial Performance Expected in 2023 JP Morgan Presentation 2023 7 *Non - GAAP measure wh:ich excludes est:imated non - cash, stock - based compensat:ion expense o:f approx:imately $120 m:ill:ion. GAAP R&D and SG&A expense :for the :full year 2023 :is ant:ic:ipated to be between $1,010 and $1,060 m:ill:ion. The Company ant:ic:ipates up to $80 m:ill:ion o:f one - t:ime payments upon ach:ievement o:f potent:ial cl:in:ical and regulatory success - based m:ilestones :from prev:ious acqu:is:it:ions. 2022 2023 Total Revenue Guidance $545 - 565 M $940 M - 1 B DMD Fran(c(hise Net Produ(ct Revenue Guidan(ce OPEX Guidan(ce* $890 - 940 M Unaudited Total Revenue ~$710 M Unaudited DMD Fran(c(hise Net Produ(ct Revenue $507 M |
| JP Morgan Presentation 2023 Continued Success Across Our Commercial Portfolio Distributed in 50+ countries with continued growth from new patients and geographic expansion First and only corticosteroid for all US DMD patients with growth from new patient starts and favorable access E stablished market leadership in all major markets with continued growth expected For treatment of hATTR with LATAM patients benefiting through early - access programs For treatment of FCS and FPL with LATAM patients benefiting through early - access programs First EMA approved disease - modifying treatment for AADC deficiency for patients 18 months and older 8 |
| JP Morgan Presentation 2023 9 Early Access Commercial Access Cross Border Building Foundation for Upstaza Success 2022 |
| JP Morgan Presentation 2023 10 Early Access Commercial Access Cross Border 2022 2023 and Beyond Substantial Growth in 2023 and Beyond with Additional Global Registrations |
| Substantial Revenue Growth from 2021 to 2023 JP Morgan Presentation 2023 11 Commercixaxl Sepiapterin | Vatiquinone | Unesbulin | PTC518 | Utreloxastat | Devexlopment $539 M 2021 Total revenue $116m Other $423m DMD Unaudited total revenue 2022 ~$710 M $ 203 m Other $507m DMD Potential future revenues 2023 $940 M - $1bn $395 - $435m Other $545 - $565m DMD |
| Strong R&D Execution and Value Creation in 2022 Clinical Achievements JP Morgan Presentation 2023 12 Regulatory Achievements Initiated SunriseLMS trial for unesbulin Initiated CardinALS study for utreloxastat Completed FITE - 19 study for emvododstat Waylivra approved in Brazil for treatment of FPL Completed enrollment for MIT - E Initiated PIVOT - HD study for PTC518 Completed Study 041 for Translarna Study 041 Filed type II variation for MAA for Translarna in EU and additional global approvals Upstaza EU and UK approval |
| JP Morgan Presentation 2023 13 Q1 2023 Q2 2023 Q3 2023 Q4 2023 Substantial Pipeline Progress Planned in 2023 |
| JP Morgan Presentation 2023 14 Q1 2023 Q2 2023 Q3 2023 Q4 2023 Substantial Pipeline Progress Planned in 2023 |
| ~ 58,000 Global Prevalence Sepiapterin Can Potentially Treat Broad PKU Population Including Classical PKU JP Morgan Presentation 2023 15 Disease Phenyxlketonurixa (PKU) is a metaboli(c (condition (caused by mutations to p(henylalanine (hydroxylase t(hat (can lead to (cognitive disabilities and seizures Current Treatments Majority of patients do not initially respond or are not well (controlled by standard of (care Mechanism of Action Sepixapterin is a more bioavailable pre(cursor t(han exogenously administered synt(heti(c BH4 and (has t(he potential to treat a broader range of PKU patients |
| qLarge Unmet Need Remains in PKU JP Morgan Presentation 2023 16 of pxatients xare wexlxl - controxlxled on Kuvxan Less thxan 10% 58,000 patients globally 30% therxapy nxaïve incxluding cxlxassicxaxl PKU 70% tried Kuvan 60% poorly controlled 40% well controlled Up to 70% fail ~30% respond 1. B:ioMar:in :f:irst quarter 2021 presentat:ion 1Q 2021. 2. Th:ird party reports prepared :for PTC Sepiapterin Potential Opportunity |
| Newborn screening with ~58,000 patients worldwide 1,2,3 Well - known metabolic centers of excellence across the world Connected and coordinated patient advocacy community Disease pathology well understood and documented Established Pillars for Commercial Success JP Morgan Presentation 2023 17 1. NPKUA; https::fn;t;i// www.npkua.org /, 2. Shoraka et. Al, Cl:in Exp Ped:iatr .. 2020; 63(2):: 34 – 43., 3. Borrajo G., Acta Ped Mex. 2012; 33(6)::fn;t;i 279 - 287. |
| Mechanistic Advantages of Sepiapterin Over Sapropterin : Increased Bioavailability GI Trxact Pxlxasmxa Cell Membrane Intrxacexlxluxlxar (xliver, brxain, kidney) Synthetic Sepiapterin (PTC923) Rapid Cross - Membrane Active Transport Sepiapterin Sepiapterin reductase Oxidation Synthetic BH 4 (Kuvan ® or sapropterin ) BH 4 BH 2 Kidney Clearance Inefficient Cross - Membrane Active Transport 7.8 - Dihydrobiopterin (BH 2 ) Dihydrofolate reductase Tetrahydrobiopterin (BH 4 ) JP Morgan Presentation 2023 18 Tyrosine Phenylalanine PAH BH 4 |
| APHENITY is a Global Registration - Directed Trial of Sepiapterin for PKU Primary Endpoint Reduction in blood phenylalanine levels Primxary Endpoint R sepiapterin Placebo 42 Days Open - Label Extension Placebo - Controlled s epixapterin 12 Months s epixapterin 14 Days Run - in 19 JP Morgan Presentation 2023 Part 1 Part 2 |
| JP Morgan Presentation 2023 20 APHENITY Part 1 Preliminary Data (n=104) Part 1 is an open - label run - in phase to identify subjects to be randomized The primary analysis population is those who have a ≥30% Phe reduction 0 10 20 30 40 50 60 70 - 65 % Sepiapterin - 29 % Kuvan 1 +122% 0 5 10 15 20 25 30 35 40 45 50 55 60 Sepiapterin - 57 % Kuvan Cxlxassicxaxl PKU ≥30% Responders Axlxl ≥30% Responders Mexan Bxlood Phe Levexls (%) Mexan Bxlood Phe Levexls (%) 1. KUVAN label ( sapropter:in d:ihydrochlor:ide) - Accessdata.:fda.gov Classical PKU patients minimally responsive to Kuvan |
| JP Morgan Presentation 2023 21 Q1 2023 Q2 2023 Q3 2023 Q4 2023 Substantial Pipeline Progress Planned in 2023 |
| JP Morgan Presentation 2023 22 Vatiqqk DTLuinone Has the Potential to Show Clinically Differentiated Improvement for MDAS Patients ~ 20,000 Global Prevalence Disease Mitochondrixaxl disexase xassocixated seizures (MDAS) is t(he (hig(hly morbid (condition of refra(ctory seizures in patients wit(h in(herited mito(c(hondrial disease Current Treatments No approved disease modifying treatments Mechanism of Action Vxatiquinone targets 15 - lipoxygenase, a regulator of t(he key energeti(c and oxidative stress pat(hways t(hat underpin seizures in t(hese patients |
| MIT - E is a Global Registration - Directed Trial of Vatiqqk DTLuinone for MDAS Primary Endpoint Cha nge from baseline in frequency of observable motor seizures PTC743 ( vatiquinone ) Placebo N = 30 24 weeks R N = 30 48 weeks PTC743 ( vxatiquinone ) N = 60 Open - Label Extension Placebo - Controlled Primary Endpoint Trial Status • Data expected 1Q 2023 JP Morgan Presentation 2023 23 Enrollment completed |
| JP Morgan Presentation 2023 24 Q1 2023 Q2 2023 Q3 2023 Q4 2023 Substantial Pipeline Progress Planned in 2023 |
| JP Morgan Presentation 2023 25 Vatiqqk DTLuinone Has the Potential to Provide Improvement in Neurological Function ~ 25,000 Global Prevalence Disease Friedreich xatxaxxlnkaixa (FA) is a rare, in(herited, progressive disease resulting from mito(c(hondrial dysfun(ction Current Treatments No approved disease modifying treatments Mechanism of Action Vxatiquinone targets 15 - lipoxygenase, a regulator of key energeti(c and oxidative stress pat(hways t(hat are disrupted in FA |
| MOVE - FA is a Global Registration - Directed Trial of Vatiqqk DTLuinone for FA Primary Endpoint Change in mFARS Key Secondary Endpoint Change in FA - ADL Open - Lxabexl Exxlnkatension Placebo - Controlled PTC743 ( vatiquinone ) Placebo N = ~ 60 72 weeks R N = ~ 60 24 weeks PTC743 ( vxatiquinone ) N = ~120 Primxary Endpoint Trial Status • Data expected in 2Q 2023 JP Morgan Presentation 2023 26 Enrollment completed |
| JP Morgan Presentation 2023 27 Q1 2023 Q2 2023 Q3 2023 Q4 2023 Substantial Pipeline Progress Planned in 2023 |
| PTC518 Reduces HTT mRNA and Protein to Target the Underlying Cause of HD JP Morgan Presentation 2023 28 ~ 135,000 Global Prevalence Disease Huntington’s disexase (HD) is a progressive brain disorder t(hat (causes un(controlled movements and (cognitive loss Current Treatments No approved disease modifying treatments Mechanism of Action PTC518 modulates spli(cing to indu(ce degradation of HTT mRNA, redu(cing expression of t(he toxi(c HTT protein |
| JP Morgan Presentation 2023 29 PIVOT - HD is a Global Phase 2 Trial of PTC518 for HD 3 mont(hs 9 mont(hs PTC518 5mg PTC518 10mg PTC518 20mg R Part 1 Part 2 Placebo PTC518 5mg PTC518 10mg PTC518 20mg Primary Endpoints • Safety and tolerability of PTC518 • Per(cent redu(ction in HTT mRNA and protein in blood Secondary Endpoints • Per(cent redu(ction in mHTT protein in CSF • C(hanges in neurofilament lig(ht (c(hain (( NfL ) in plasma and CSF • C(hange in brain volume on volumetri(c MRI imaging |
| PIVOT - HD Expanding Trial Target Population JP Morgan Presentation 2023 30 • Ambulatory Huntington’s patients ages 25 and older • CAG repeats 40 - 50 inclusive • Motor and Cognitive Function: - UHDRS - IS score of 100 - UHDRS TFC score of 13 • PIN HD score 0.18 - 4.93 - Multivariate calculation including SDMT, TMS, age, CAG Inclusion Criteria Stage 2 Inclusion Criteria Early Stage 3 • Ambulatory Huntington’s patients ages 25 and older • CAG repeats 40 - 50 inclusive • Motor and Cognitive Function: - UHDRS - IS score of less than 100 - UHDRS TFC score of 11 or 12 • PIN HD score 0.18 - 4.93 - Multivariate calculation including SDMT, TMS, age, CAG |
| Transformational Commercial Revenue in 2023 JP Morgan Presentation 2023 31 Commercixaxl $539 M 2021 Total revenue $116m Other $423m DMD Unaudited total revenue 2022 ~$710 M $ 203 m Other $ 507 m DMD Potential future revenues 2023 $940 M - $1bn $395 - $435m Other $545 - $565m DMD |
| JP Morgan Presentation 2023 32 Q1 2023 Q2 2023 Q3 2023 Q4 2023 Transformational Development Milestones in 2023 |
| JP Morgan Presentation 2023 33 Q1 2023 Q2 2023 Q3 2023 Q4 2023 DIPG BLA Subm:iss:ion Transformational Development Milestones in 2023 |
| PKU Patient Matthew Klein, M.D., COO JP Morgan Healthcare Conference January 9, 2023 PTC 2023 |
