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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): January 12, 2026
PTC THERAPEUTICS, INC.
(Exact Name of Company as Specified in Charter)
Delaware |
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001-35969 |
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04-3416587 |
(State or Other Jurisdiction |
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(Commission |
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(IRS Employer |
of Incorporation) |
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File Number) |
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Identification No.) |
500 Warren Corporate Center Drive |
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Warren, NJ |
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07059 |
(Address of Principal Executive Offices) |
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(Zip Code) |
Registrant’s telephone number, including area code: (908) 222-7000
Not applicable
(Former Name or Former Address, if Changed Since Last Report)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):
☐Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
☐Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
☐Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
☐Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))
Securities registered pursuant to Section 12(b) of the Act:
Title of each class |
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Trading Symbol(s) |
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Name of each exchange on which registered |
Common Stock, $0.001 par value per share |
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PTCT |
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Nasdaq Global Select Market |
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 12, 2026, 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, 2025, including that the Company expects to report (i) total unaudited net product and royalty revenue of approximately $823.4 million, (ii) total unaudited net product revenue of approximately $587.8 million, and (iii) unaudited net product revenue for Sephience™ (sepiapterin) of approximately $112.1 million. Final results are subject to completion of the Company’s year-end audit.
Item 7.01. Regulation FD Disclosure.
On January 12, 2026, the Company also announced financial guidance for its fiscal year ending December 31, 2026 in the press release, including that the Company anticipates (i) full year total product revenues of $700 million to $800 million, excluding Evrysdi® (risdiplam) royalty revenue and collaboration revenue and (ii) GAAP R&D and SG&A expense for the full year 2026 of $775 million to $815 million with non-GAAP R&D and SG&A expense for the full year 2026 of $680 million to $720 million, excluding estimated non-cash, stock-based compensation expense of $95 million.
The Company announced that on Monday, January 12, 2026 at 12:00 p.m. EST at the 44th Annual J.P. Morgan Healthcare Conference (the “Conference”), the Company will provide an update on 2025 accomplishments and highlight upcoming 2026 potential value-creating milestones. The Company will also present its preliminary 2025 unaudited financial results and 2026 financial guidance. The presentation will be webcast live on the Events and Presentations page under the Investors section of the Company’s website. Following the completion of the Conference, the Company will enter an investor quiet period until it provides its next corporate update.
A copy of the press release and the slide presentation are attached to this Current Report on Form 8-K as Exhibits 99.1 and 99.2 and are 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 2025 and financial guidance for fiscal year 2026. 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 2025 financial information, which is subject to completion of the Company's year-end audit; the assumptions underlying the Company's financial guidance for 2026; 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, 2024, 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. |
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Description |
99.1 |
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Press Release, dated January 12, 2026 issued by PTC Therapeutics, Inc. |
99.2 |
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Corporate Presentation – 44th Annual J.P. Morgan Healthcare Conference |
104 |
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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.
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PTC Therapeutics, Inc. |
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Date: January 12, 2026 |
By: |
/s/ Pierre Gravier |
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Name: |
Pierre Gravier |
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Title: |
Chief Financial Officer |
Exhibit 99.1
PTC Therapeutics Provides Update at J.P. Morgan Annual Healthcare Conference
– Strong Sephience™ (sepiapterin) launch continues with unaudited Q4 global revenue of $92.5M –
– Unaudited 2025 total product and royalty revenue of approximately $823M, exceeding guidance –
– 2026 product revenue guidance of $700–800M, representing 19–36% year-over-year growth –
– Strong cash position of approximately $1.94B as of December 31 –
WARREN, N.J., Jan. 12, 2026 - PTC Therapeutics, Inc. (NASDAQ: PTCT) today provided an update on the Company’s progress and its outlook for 2026. Matthew B. Klein, M.D., Chief Executive Officer of PTC, will discuss these updates at the Company’s presentation at the J.P. Morgan 2026 Healthcare Conference on Monday, January 12, 2026, at 9:00 a.m. PST / 12:00 p.m. EST.
“2025 was a highly successful year for PTC, highlighted by the initial regulatory approvals of Sephience and strong start to the global launch,” Dr. Klein said. “In 2026, we look forward to continuing the Sephience launch momentum, advancing our innovative earlier-stage programs and moving the company toward becoming cash flow breakeven.”
Key Corporate Highlights
| ● | Unaudited 2025 product and royalty revenue of approximately $823.4 million, exceeding guidance, and unaudited 2025 product revenue of approximately $587.8 million |
| ● | Unaudited Sephience Q4 2025 net revenue of approximately $92.5 million, including $81.6 million in the US and $10.9 million ex-US |
| o | Unaudited Sephience total net revenue of approximately $112.1 million in 2025 since launch |
| o | 1,134 patient start forms received in the US as of December 31, 2025 |
| o | 946 total patients on commercial therapy worldwide as of December 31, 2025 |
| o | Additional Sephience launches expected in 2026, including in Japan, Brazil and other geographies |
| ● | In December 2025, PTC sold the remainder of its Evrysdi® (risdiplam) royalty to Royalty Pharma for $240 million upfront and up to $60 million in sales-based milestones; PTC maintains the right to receive a $150 million milestone based on single-year Evrysdi sales of $2.5 billion by Roche |
| ● | Cash, cash equivalents, and marketable securities of approximately $1.94 billion as of December 31, 2025 |
| ● | End-of-Phase 2 meeting with FDA held in Q4 2025 to discuss the votoplam Huntington’s disease (HD) program: |
| o | Alignment reached on design of global Phase 3 trial, planned to initiate in H1 2026 |
| o | FDA confirmed openness to potential Accelerated Approval pathway given significant unmet need |
| ● | Type C meeting with FDA held in December 2025 to discuss vatiquinone Friedreich’s ataxia program; FDA requested additional information from MOVE-FA Phase 3 trial prior to providing guidance on next steps |
| ● | Translarna™ (ataluren) NDA remains under FDA review |
Unaudited 2025 Financial Results
| ● | Total unaudited product and royalty revenue for full-year 2025 was approximately $823.4 million |
| ● | Total unaudited product revenue for full-year 2025 was approximately $587.8 million |
| ● | Sephience unaudited total net revenue for 2025 was approximately $112.1 million |
| ● | DMD franchise unaudited revenue for full-year 2025 was approximately $381.8 million, including unaudited product revenue for Translarna of approximately $235.5 million and for Emflaza® (deflazacort) of approximately $146.3 million |
| ● | PTC expects to report approximately $235.7 million of full-year 2025 royalty revenue associated with Evrysdi |
PTC is finalizing its financial results for the 2025 fiscal year. The above information is based on preliminary unaudited information and management estimates for the full year 2025, subject to the completion of PTC's financial closing procedures. Evrysdi royalty revenue estimates are based on internal estimates and pending accounting treatment of Evrysdi royalty following the December 2025 transaction.
2026 Financial Guidance
For the full year 2026, PTC anticipates:
| ● | Total product revenue of $700 to $800 million, excluding Evrysdi royalty revenue and collaboration revenue |
| ● | GAAP R&D and SG&A expense of $775 to $815 million |
| ● | Non-GAAP R&D and SG&A expense of $680 to $720 million, excluding estimated non-cash, stock-based compensation expense of $95 million |
Non-GAAP Financial Measures
In this press release, the financial results and financial guidance of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management's opinion, these non-GAAP financial measures are useful to investors and other users of PTC's financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company's future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the table below.
PTC Therapeutics, Inc.
Reconciliation of GAAP to Non-GAAP Projected Full-Year 2026 R&D and SG&A expense
(In millions)
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Low End of Range |
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High End of Range |
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Projected GAAP R&D and SG&A expense |
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$ |
775 |
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$ |
815 |
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Less: projected non-cash, stock-based compensation expense |
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95 |
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95 |
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Projected non-GAAP R&D and SG&A expense |
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$ |
680 |
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$ |
720 |
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Acronyms:
DMD: Duchenne Muscular Dystrophy
FA: Friedreich’s ataxia
FDA: Food and Drug Administration GAAP: Generally Accepted Accounting Principles HD: Huntington’s Disease NDA: New Drug Application nmDMD: nonsense mutation Duchenne Muscular Dystrophy
PKU: Phenylketonuria
R&D: Research and Development
SG&A: Selling, General and Administrative
About PTC Therapeutics, Inc.
PTC is a global biopharmaceutical company that discovers, develops and commercializes clinically differentiated medicines that provide benefits to children and adults living 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. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Facebook, Instagram, LinkedIn and X.
For More Information:
Investors:
Ellen Cavaleri
+1 (615) 618-6228
ecavaleri@ptcbio.com
Media:
Jeanine Clemente
+1 (908) 912-9406
jclemente@ptcbio.com
Forward-Looking Statement
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 "2026 Financial Guidance", including with respect to (i) 2026 total product revenue guidance and (ii) 2026 GAAP and non-GAAP R&D and SG&A 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, meetings with regulatory agencies, commercialization and other matters with respect to 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," "aim," 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: 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 Sephience, including any regulatory submissions and potential approvals, commercialization, and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in Brazil, Russia and other regions; the effect of the European Commission's adoption of the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) on Translarna on other regulatory bodies; PTC's ability to use the results of 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, and from its international drug registry study to support a marketing approval for Translarna for the treatment of nmDMD in the United States; whether investigators agree with PTC's interpretation of the results of clinical trials and the totality of clinical data from its trials in Translarna; expectations with respect to PTC's license and collaboration agreement with Novartis Pharmaceuticals Corporation for votoplam for the treatment of Huntington's disease including its right to receive development, regulatory and sales milestones, profit sharing and royalty payments from Novartis, the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses, including potential accelerated approval; expectations with respect to Upstaza/Kebilidi, including commercialization, manufacturing capabilities, and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; expectations with respect to vatiquinone, including with respect to the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses and potential approvals and other matters; expectations with respect to the commercialization of Evrysdi under PTC's SMA collaboration; expectations with respect to the commercialization of Tegsedi and Waylivra; 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; 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 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 Sephience, Translarna, Emflaza, Upstaza, Kebilidi, 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.
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J.P. Morgan 2026 Healthcare Conference PTC Therapeutics 2026 Matthew B. Klein, MD CEO |
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J.P. Morgan 2026 Healthcare Conference FDA and EMA approvals of Sephience Advancement of early-stage R&D programs Strong start to Sephience global launch Drive revenue and effectively manage OpEx 2025: A Year of Focus and Execution 2 |
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J.P. Morgan 2026 Healthcare Conference This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this presentation, other than statements of historic fact, are forward-looking statements, including statements with respect to 2026 total product revenue guidance and 2026 operating 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, meetings with regulatory agencies, commercialization and other matters with respect to 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," "aim," 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: 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 Sephience, including any regulatory submissions and potential approvals, commercialization, and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in Brazil, Russia and other regions; the effect of the European Commission's adoption of the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) on Translarna on other regulatory bodies; PTC's ability to use the results of 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, and from its international drug registry study to support a marketing approval for Translarna for the treatment of nmDMD in the United States; whether investigators agree with PTC's interpretation of the results of clinical trials and the totality of clinical data from its trials in Translarna; expectations with respect to PTC's license and collaboration agreement with Novartis Pharmaceuticals Corporation for votoplam for the treatment of Huntington's disease including its right to receive development, regulatory and sales milestones, profit sharing and royalty payments from Novartis the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses, including potential accelerated approval; expectations with respect to Upstaza/Kebilidi, including commercialization, manufacturing capabilities, and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; expectations with respect to vatiquinone, including with respect to the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses and potential approvals and other matters; expectations with respect to the commercialization of Evrysdi under PTC's SMA collaboration; expectations with respect to the commercialization of Tegsedi and Waylivra; 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; 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 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 Sephience, Translarna, Emflaza, Upstaza, Kebilidi, Evrysdi, Tegsedi or Waylivra. The forward-looking statements contained herein represent PTC's views only as of the date of this presentation 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 presentation except as required by law. Forward Looking Statements 3 |
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J.P. Morgan 2026 Healthcare Conference 2025 Revenue Performance Exceeded Guidance 4 ~$823M* Product Revenue ~$588M * All revenue numbers are unaudited; revenue performance includes product and royalty revenue; royalty revenue is pending accounting treatment of Evrysdi |
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J.P. Morgan 2026 Healthcare Conference Sephience Launch Continues Strong Momentum 5 $92.5M Q4 Global Revenue* $81.6M US $10.9M ex-US 946 patients on commercial therapy worldwide** 1,134 Patient Start Forms in US** ** As of December 31, 2025 * Unaudited net revenue |
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J.P. Morgan 2026 Healthcare Conference 2026 Revenue and OpEx Guidance 6 YoY Product Revenue Growth $700 19-36% * -800M * Product revenue guidance excludes Evrysdi royalty and collaboration revenues; YoY based on $700-$800M guidance |
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J.P. Morgan 2026 Healthcare Conference 2026 Revenue and OpEx Guidance 7 * Product revenue guidance excludes Evrysdi royalty and collaboration revenues ** OpEx is a Non-GAAP measure that includes only R&D and SG&A expenses for full year 2026, and excludes estimated non-cash, stock-based compensation expense of approximately $95M. GAAP R&D and SG&A expense for full year 2026 is anticipated to be between $775 and $815M. Product Revenue* $700-800M OpEx $680-720M ** |
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J.P. Morgan 2026 Healthcare Conference 2026: Planning for Continued Success 8 Continue Sephience global launch momentum Move toward reaching cash flow breakeven Initiate votoplam HD Phase 3 trial Advance early-stage R&D programs |
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J.P. Morgan 2026 Healthcare Conference 9 Patient living w ith PKU Sephience PKU Program |
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J.P. Morgan 2026 Healthcare Conference Sephience Dual Mechanism of Action Supports Potential for Broad Uptake 10 * SR, sepiapterin reductase (enzyme that converts Sephience to BH2) ** DHFR, dihydrofolate reductase (enzyme that converts BH2 to BH4) 1. Precursor to BH4 SP Sephience 7.8-Dihydrobiopterin (BH2) Tetrahydrobiopterin (BH4) SR* DHFR** BH BH4 4 BH4 Sephience significantly increases intracellular BH4 levels 2. Independent Chaperone Function Misfolded PAH Enzyme Chaperone Effect Decreased Enzyme Activity Increased Enzyme Activity SP Chaperone effect stabilizes enzyme, increases function, and enables treatment of BH4-non-responsive mutations |
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J.P. Morgan 2026 Healthcare Conference Sephience Provides Significantly Greater Phe Reduction Compared With BH4 11 -437.0 -256.6 -600 -500 -400 -300 -200 -100 0 100 Absolute least squares mean change in blood Phe concentration (µmol/L) Sephience BH4 70.3% Greater reduction with Sephience p < 0.0001 Mean Absolute Change in Blood Phe from Baseline AMPLIPHY was a crossover study allowing for intra-subject comparison of Sephience and BH4 treatment effect |
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J.P. Morgan 2026 Healthcare Conference Sephience Provides Diet Liberalization, Cognitive and Quality of Life Benefits 12 Improved cognitive function and mood Improved concentration & slow thinking, and decreased irritability Meaningful diet liberalization 69% of subjects reached age-adjusted protein RDA Improved quality of life Reduced disease impact on emotional, social, and familial wellbeing Data presented at the 2025 International Congress of Inborn Errors of Metabolism (ICIEM) |
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J.P. Morgan 2026 Healthcare Conference Sephience Differentiated Profile Enables Penetration Into All Key Market Segments 13 On Treatment Treatment Failures Treatment Naive 17K Patients in US TOTAL ADDRESSABLE MARKET INCLUDING ALL AGES AND SEVERITIES |
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J.P. Morgan 2026 Healthcare Conference 2025: Launch Initiated in US, EU and Canada 14 UNIT ED STATES CANADA EUROPE |
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J.P. Morgan 2026 Healthcare Conference 15 2026: Expanding the Sephience Global Launch NORTH AMERICA EUROPE ASIA LATAM Anticipated Sephience geographic footprint for 2026 |
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J.P. Morgan 2026 Healthcare Conference 16 Votoplam Huntington’s Disease Program Patient living w ith HD |
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J.P. Morgan 2026 Healthcare Conference Phase 2 Placebo-Controlled Trial Achieved Key Efficacy and Safety Objectives 17 Long-term positive clinical trends at Month 24 relative to natural history and dose-dependent NfL lowering Continued favorable safety and tolerability profile with no treatment-related NfL spikes Primary endpoint met with durable, dose-dependent blood HTT protein lowering Favorable and dose-dependent clinical trends at Month 12 in Stage 2 subjects May 2025 data readout |
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J.P. Morgan 2026 Healthcare Conference Votoplam Phase 3 Trial Expected to be Initiated by Novartis in H1 2026 18 INVEST-HD Global Phase 3 Study • 3:2 randomization of votoplam: placebo • Target enrollment: ~770 participants in >30 countries • Primary endpoint: Change in cUHDRS from Baseline up to 36 months • Interim analysis planned for efficacy and futility |
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J.P. Morgan 2026 Healthcare Conference 19 Vatiquinone Friedreich’s Ataxia Program Patient living w ith FA |
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J.P. Morgan 2026 Healthcare Conference Vatiquinone Demonstrated Meaningful Slowing of Short- and Long-term Disease Progression 20 Vatiquinone Placebo Long-term Extension Studies 4.8-point benefit (p<0.0001) on mFARS** over 2 years in ambulatory and non-ambulatory adults 50% slowing (p<0.0001) of disease progression over 3 years** in MOVE-FA long-term extension study * MOVE-FA did not meet its primary endpoint of statistically significant change in total mFARS at 72 weeks ** Relative to matched FACOMS (Friedreich’s Ataxia Clinical Outcome Measures Study) natural history cohort 72-week Placebo-Controlled MOVE-FA Trial* Weeks Mean change from Baseline in mFARS Upright Stability score (+/- SE) 42% Slowing p=0.021 |
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J.P. Morgan 2026 Healthcare Conference 21 Innovative Research Platforms |
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J.P. Morgan 2026 Healthcare Conference PTSeek Platform Provides Rapid and Reliable Identification of Splicing Therapy Candidates 22 2005 2020 2023 Splicing Foundation Splicing Platform 2025 and beyond Targeted high-throughput screening Transcriptome-wide direct splicing interrogation Novel molecules for targets in multiple therapeutic areas SMA Huntington’s Disease Enhanced “splicing-centric” chemical library Significant expansion of potential targets Neurodegenerative Hematological Additional Targets Metabolic Oncology Neuromuscular |
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J.P. Morgan 2026 Healthcare Conference R&D Pipeline Includes Innovative Programs from Differentiated Scientific Platforms 23 MSH3: Huntington’s Disease, Myotonic Dystrophy I Undisclosed: Neurodegenerative Diseases Undisclosed: Brain Tumors & Metastases Undisclosed: Sickle Cell Disease, β-Thalassemia ATXN3: Spinocerebellar Ataxia (SCA) 3 Splicing Platform Inflammation & Ferroptosis Platform Ferroptosis: Parkinson’s Disease NLRP3 Inflammasome: Inflammatory Lung Diseases DHODH Inhibition: T-Cell Mediated Autoimmune Diseases NRF2 Activation: CNS, Non-CNS Indications |
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J.P. Morgan 2026 Healthcare Conference Well Positioned for Success in 2026 & Beyond 24 Strong Financial Position Robust Global Commercial Engine Innovative R&D Platforms |
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J.P. Morgan 2026 Healthcare Conference PTC Therapeutics 2026 Matthew B. Klein, MD CEO |
