PTC Therapeutics Provides Update at J.P. Morgan Annual Healthcare Conference

Rhea-AI Summary

PTC Therapeutics (NASDAQ: PTCT) reported unaudited 2025 product and royalty revenue of approximately $823.4M, with unaudited product revenue of about $587.8M. Sephience generated unaudited Q4 2025 net revenue of approximately $92.5M and total 2025 net revenue since launch of $112.1M. PTC ended 2025 with approximately $1.94B in cash, cash equivalents and marketable securities. In December 2025 PTC sold remaining Evrysdi royalty rights for $240M upfront plus up to $60M in milestones while retaining a separate $150M milestone right. For 2026 PTC guides total product revenue of $700–$800M (ex-Evrysdi royalties), GAAP R&D and SG&A of $775–$815M, and non-GAAP R&D and SG&A of $680–$720M. Clinical updates include Phase 3 alignment and Accelerated Approval openness for votoplam HD and FDA follow-up requests for vatiquinone MOVE-FA.

Positive

• Unaudited 2025 product and royalty revenue of approximately $823.4M
• Sephience total 2025 net revenue of approximately $112.1M since launch
• Sephience Q4 2025 net revenue of approximately $92.5M
• Cash and marketable securities of approximately $1.94B as of Dec 31, 2025
• Evrysdi royalty sale with $240M upfront improves near-term liquidity
• 2026 product revenue guidance of $700–$800M (19–36% YoY growth)

Negative

• Projected GAAP R&D and SG&A expense of $775–$815M for 2026
• Evrysdi royalty sale reduces future royalty income upside
• FDA requested additional MOVE-FA data for vatiquinone, risking delays
• Translarna NDA remains under FDA review, outcome uncertain

News Market Reaction – PTCT

+0.08%

3 alerts

+0.08% News Effect

+18.6% Peak Tracked

+$5M Valuation Impact

$6.22B Market Cap

2K Volume

On the day this news was published, PTCT gained 0.08%, reflecting a mild positive market reaction. Argus tracked a peak move of +18.6% during that session. Our momentum scanner triggered 3 alerts that day, indicating moderate trading interest and price volatility. This price movement added approximately $5M to the company's valuation, bringing the market cap to $6.22B at that time.

Data tracked by StockTitan Argus on the day of publication.

Key Figures

Q4 2025 Sephience revenue: $92.5M 2025 total product & royalty revenue: $823.4M 2025 product revenue: $587.8M +5 more

8 metrics

Q4 2025 Sephience revenue $92.5M Unaudited global net revenue in Q4 2025

2025 total product & royalty revenue $823.4M Unaudited 2025, exceeding guidance

2025 product revenue $587.8M Unaudited full-year 2025 product revenue

2025 Sephience revenue $112.1M Unaudited total net revenue in 2025 since launch

2026 product revenue guidance $700–800M Guided 2026 total product revenue, excluding Evrysdi royalty and collaboration

Cash & investments $1.94B Cash, cash equivalents, and marketable securities as of Dec 31, 2025

Evrysdi royalty sale upfront $240M Upfront payment from Royalty Pharma in Dec 2025

2026 non-GAAP R&D & SG&A $680–720M Projected 2026 non-GAAP R&D and SG&A expense excluding $95M stock comp

Market Reality Check

Price: $70.66 Vol: Volume 1,556,282 is 24% a...

normal vol

$70.66 Last Close

Volume Volume 1,556,282 is 24% above the 20-day average of 1,253,330 ahead of the update. normal

Technical Price $77.53 is trading above the 200-day MA of $58.03, reflecting a pre-existing uptrend.

Peers on Argus

PTCT gained 0.88% while close peers were mixed: KRYS up 2.71%, RNA flat to sligh...

PTCT gained 0.88% while close peers were mixed: KRYS up 2.71%, RNA flat to slightly positive, and CRSP, MRUS, TGTX down between -5.48% and -7.26%, pointing to stock-specific strength rather than a broad biotech move.

Historical Context

5 past events · Latest: Dec 22 (Positive)

Pattern 5 events

Date	Event	Sentiment	Move	Catalyst
Dec 22	Japan approval	Positive	+0.4%	Sephience approval in Japan for PKU across all ages and severities.
Dec 19	Inducement grants	Neutral	+0.4%	Small inducement stock option and RSU grants for five new employees.
Dec 18	Conference presentation	Neutral	-0.5%	Announcement of CEO presentation at J.P. Morgan Healthcare Conference.
Dec 01	Inducement grants	Neutral	-7.9%	Inducement equity awards to 15 new employees under Nasdaq rules.
Nov 20	R&D day announcement	Positive	+1.1%	Planned R&D Day detailing small molecule splicing and inflammation platforms.

Pattern Detected

Recent news events, including regulatory approvals and corporate updates, have generally seen modest, directionally consistent price reactions, with one notable selloff on otherwise routine inducement grant news.

Recent Company History

Over the past few months, PTC has reported a series of operational and corporate milestones. These include approval of Sephience for PKU in Japan on Dec 22, 2025, routine inducement equity grants on Dec 19 and Dec 1, 2025, and investor-focused events such as an upcoming J.P. Morgan conference presentation and an R&D Day. Price reactions to these items were mostly modest and aligned with the news tone, except for a sharper -7.9% move after the Dec. 1 inducement grants, suggesting occasional volatility even on neutral disclosures.

Market Pulse Summary

This announcement outlines a strong 2025 performance, including unaudited product and royalty revenu...

Analysis

This announcement outlines a strong 2025 performance, including unaudited product and royalty revenue of $823.4M, Sephience 2025 net revenue of $112.1M, and a robust cash and investment balance of about $1.94B. For 2026, PTC guided total product revenue to $700–800M and projected non-GAAP R&D and SG&A of $680–720M. Investors may track Sephience launch metrics, regulatory milestones for Translarna and other programs, and the company’s progress toward the stated goal of moving toward cash flow breakeven.

Key Terms

gaap, non-gaap, nda, fda, +4 more

8 terms

gaap financial

"In this press release, the financial results and financial guidance of PTC are provided in accordance with GAAP and using certain non-GAAP"

GAAP, or Generally Accepted Accounting Principles, are a set of standardized rules and guidelines that companies follow when preparing their financial statements. They ensure consistency, transparency, and comparability across different companies, making it easier for investors to understand and compare financial information accurately. This helps investors make informed decisions based on trustworthy and uniform financial reports.

non-gaap financial

"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."

Non-GAAP refers to financial measures that companies use to show their earnings or performance without including certain expenses or income that are often added back to give a different picture. It matters because it can make a company's results look better or more favorable, but it may also hide important costs, so investors need to look at both GAAP (official rules) and non-GAAP numbers to get a full understanding.

nda regulatory

"Translarna™ (ataluren) NDA remains under FDA review"

An NDA, or nondisclosure agreement, is a legal contract that keeps certain information private between parties. It’s like a promise not to share sensitive details, helping protect business ideas, strategies, or data from being leaked or used without permission. For investors, NDAs help ensure that confidential information remains secure, enabling trust and open communication during business discussions.

fda regulatory

"End-of-Phase 2 meeting with FDA held in Q4 2025 to discuss the votoplam"

The FDA is the U.S. federal agency that evaluates and approves medical drugs, devices, biological therapies and certain foods; think of it as the gatekeeper that decides whether a medical product is safe and effective for patients. For investors, FDA decisions determine whether a company can sell a product, affect expected revenue and introduce regulatory risk, so approvals, rejections or safety warnings can quickly move a company's valuation and stock price.

r&d financial

"GAAP R&D and SG&A expense of $775 to $815 million"

Research and development (R&D) is the work a company does to discover new products, improve existing ones, or develop better ways of making things — like a kitchen testing recipes to create a hit dish. For investors it matters because R&D is where future sales and competitive advantages are born, but it also uses cash and carries risk, so R&D spending and outcomes signal a company’s growth potential and uncertainty.

sg&a financial

"GAAP R&D and SG&A expense of $775 to $815 million"

SG&A stands for Selling, General, and Administrative expenses. It includes the costs a company spends on selling products, running the business day-to-day, and managing staff, like advertising, rent, and salaries. These expenses matter because they affect how much profit a company can make from its sales.

pku medical

"for the Treatment of Children and Adults Living with Phenylketonuria (PKU) in Japan"

An inherited metabolic condition in which the body cannot properly break down phenylalanine, an amino acid found in food, causing it to build up and potentially harm brain development if untreated; think of it as a factory line that can’t process one raw material, so the excess piles up. It matters to investors because routine newborn screening, lifelong specialized diets, medical foods and evolving drug or gene therapies create ongoing healthcare demand, regulatory pathways, and potential market opportunities tied to approvals, pricing and reimbursement.

accelerated approval regulatory

"FDA confirmed openness to potential Accelerated Approval pathway given significant unmet need"

Accelerated approval is a process that allows new medical treatments to be approved more quickly than usual if they address serious or life-threatening conditions and show promising early results. For investors, it signals that a treatment may reach the market sooner, potentially boosting a company's prospects, but it also involves some uncertainty since full evidence of effectiveness is still being gathered.

AI-generated analysis. Not financial advice.

– 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 /PRNewswire/ -- 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 towards 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
  • Unaudited Sephience total net revenue of approximately $112.1 million in 2025 since launch
  • 1,134 patient start forms received in the US as of December 31, 2025
  • 946 total patients on commercial therapy worldwide as of December 31, 2025
  • 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:
  • Alignment reached on design of global Phase 3 trial, planned to initiate in H1 2026
  • 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)

	Low End of Range			High End of Range
Projected GAAP R&D and SG&A expense	$	775		$	815
Less: projected non-cash, stock-based compensation expense	95			95
Projected non-GAAP R&D and SG&A expense	$	680		$	720

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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SOURCE PTC Therapeutics, Inc.

FAQ

What were PTC Therapeutics' unaudited 2025 total product and royalty revenues (PTCT)?

PTC reported unaudited 2025 total product and royalty revenue of approximately $823.4M.

How much revenue did Sephience generate in Q4 2025 and since launch (PTCT)?

Sephience reported unaudited Q4 2025 net revenue of approximately $92.5M and total 2025 net revenue since launch of $112.1M.

What is PTC's 2026 product revenue guidance (PTCT) and implied growth?

PTC guides 2026 product revenue of $700–$800M (excluding Evrysdi royalties), representing about 19–36% year-over-year growth.

How much cash did PTC have at year-end 2025 (PTCT)?

PTC had approximately $1.94 billion in cash, cash equivalents and marketable securities as of December 31, 2025.

What were the terms of PTC's December 2025 Evrysdi royalty transaction (PTCT)?

PTC sold remaining Evrysdi royalty rights for $240M upfront plus up to $60M in sales-based milestones and retains a separate $150M milestone right.

What clinical developments did PTC announce for votoplam and vatiquinone (PTCT)?

PTC reached Phase 3 design alignment and FDA openness to potential Accelerated Approval for votoplam HD; FDA requested additional MOVE-FA data for vatiquinone before next steps.