POverview
PTC Therapeutics develops and commercializes treatments for rare genetic disorders worldwide, leveraging RNA biology and post-transcriptional control. Its portfolio includes Translarna (ataluren) for nonsense-mutant Duchenne muscular dystrophy, Evrysdi (risdiplam) for spinal muscular atrophy, Emflaza (deflazacort) for DMD, and Upstaza for AADC deficiency, among others. The company uses an integrated model spanning discovery, clinical development, manufacturing, and global commercialization, with operations in 50+ countries and a European base in Dublin. Its goal is to provide safe, effective therapies for patients with limited treatment options and to grow a robust pipeline in rare diseases.
Work here?Claim your company
About PTC Therapeutics
Industries
Company Size
1,001-5,000
Company Stage
IPO
Headquarters
Warren Township, New Jersey
Founded
1998
Get referred to PTC Therapeutics
See people who can refer or advise you
Simplify's take
What believers are saying
- Q3 2026 revenue hit $358 million; management raised guidance to $1.29 billion-$1.32 billion.
- Sephience grew over 20% sequentially, with $145 million U.S. revenue in Q3.
- ST-920 BLA completes in Q4 2026, targeting 2027 approval and launch.
What critics are saying
- Emflaza lost orphan exclusivity in June 2026; generics already cut pricing.
- Translarna benefited from a one-off Russia order; Q4 lacks that support.
- Sephience funds the breakeven story; a reimbursement stall would crush 2026 cash flow.
What makes PTC Therapeutics unique
- PTC runs a rare-disease commercial machine; Sephience, Evrysdi royalties, and gene therapy span one platform.
- October 29, 2026, Sephience reached $182 million and over 2,000 active patients worldwide.
- ST-920 fits PTC's existing salesforce, avoiding a new commercial build.
Help us improve and share your feedback! Did you find this helpful?
Funding
Total Funding
$2B
Above
Industry Average
Funded Over
8 Rounds
Benefits
- Health Insurance
- Dental Insurance
- Vision Insurance
- 401(k) Retirement Plan
- Flexible Work Hours
- Performance Bonus
Stock Price
Growth & Insights and Company News
6 month growth
-1%1 year growth
-1%2 year growth
-1%PTC Therapeutics reported record third-quarter 2026 revenue of $358 million, with net product revenue of $276 million, up from $131 million a year earlier. Sephience, its phenylketonuria therapy, brought in $182 million, over 20% more than in the second quarter. The company raised full-year product revenue guidance to $960 million–$990 million and total revenue guidance to $1.29 billion–$1.32 billion. It expects to reach cash flow breakeven in 2026 and held $2.13 billion in cash and securities on September 30. PTC also acquired ST920, a Fabry disease gene therapy, for $111 million upfront plus up to $100 million in milestone payments. Approval and launch are possible in 2027. Sales of its Duchenne muscular dystrophy drugs face pressure from a new generic rival to Emflaza.
PTC sees $960M-$990M 2026 product revenue as Sephience expands and ST-920 filing targets Q4 2026. Earnings Call Insights: PTC Therapeutics (PTCT) Q3 2026 Management view. * "I'm pleased to share the results of another quarter of record product revenue and solid execution across the company." (CEO & Director Matthew Klein) * "Based on this performance, we are raising our full year 2026 product revenue guidance to $960 million to $990 million and now expect total revenue of $1.29 billion to $1.32 billion." (CEO & Director Klein) * "Beginning with Sephience, we are now 1 year into the global launch... all signs point to Sephience being a blockbuster rare disease product." (CEO & Director Klein) * "In the third quarter, worldwide Sephience revenue was $182 million... And as of September 30, we have over 2,000 active patients on Sephience worldwide." (CEO & Director Klein) * "Overall, the vast majority of the 17,000 patients in the U.S. have yet to try Sephience." (CEO & Director Klein) * "In the third quarter, we acquired the BLA-stage Fabry disease gene therapy asset, ST-920." (CEO & Director Klein) * "We expect to complete the rolling BLA submission for accelerated approval in Q4 2026 with the potential for approval and commercial launch in 2027." (CEO & Director Klein) * "Due to FDA scheduling, the Type C meeting to discuss the votoplam data package and a potential path to accelerated approval is now planned for the first quarter of 2027." (CEO & Director Klein) * "Total net product revenue across the commercial portfolio was $276 million... Third quarter 2026 product revenue includes Sephience net product revenue of $182 million and DMD franchise revenue of $74 million." (Chief Financial Officer Pierre Gravier) Outlook. * "This updated guidance represents 64% to 69% year-over-year product revenue growth." (CEO & Director Klein) * "With our continued effective expense management, we expect to achieve our goal of reaching cash flow breakeven in 2026." (CEO & Director Klein) * Compared with Q2, management increased 2026 product revenue guidance from "$850 million to $950 million" to "$960 million to $990 million" and increased 2026 total revenue guidance from "$1.18 billion to $1.28 billion" to "$1.29 billion to $1.32 billion." (CEO & Director Matthew Klein, Q2 vs. Q3 calls) * On pipeline and regulatory timing, management highlighted: "We expect to complete the rolling BLA submission... in Q4 2026" for ST-920, and said the votoplam Type C meeting is "now planned for the first quarter of 2027." (CEO & Director Klein) Financial results. * "Total revenue of $358 million, including $276 million of product revenue" was driven by Sephience and supported by other brands, while royalty revenue was tied to Evrysdi performance described as "approximately USD 607 million, resulting in royalty revenue of $82 million." (CEO & Director Matthew Klein; CFO Pierre Gravier) * Management detailed mature-brand contributions and variability drivers, including: "Translarna net product revenue was $49 million, including a government purchase order from Russia" and "Emflaza net product revenue was $25 million." (CFO Gravier) * Expense and liquidity metrics were framed as supporting the breakeven objective: "Non-GAAP R&D expense was $95 million" and "Non-GAAP SG&A expense was $67 million," while "Cash, cash equivalents and marketable securities totaled $2.13 billion as of September 30, 2026." (CFO Gravier) * On business development cash use and contingencies, management said: "In the third quarter of 2026, we acquired ST-920 for an upfront payment of $111 million and up to $100 million in contingent milestone payments tied to U.S. regulatory approval." (CFO Gravier) Q&A. * Kristen Kluska, Cantor Fitzgerald & Co.: asked where Sephience is in launch maturity and how to think about U.S. vs. ex-U.S. growth; CEO Klein answered, "we very much believe we're still in the very early innings here" and added, "we probably have at least 85% of PKU patients not yet tried Sephience." * Kristen Kluska, Cantor Fitzgerald & Co.: asked about FDA's rare-disease trial flexibility; CEO Klein said, "it's a really exciting time with what's going on for rare disease at FDA" and linked it to "upcoming discussions on votoplam with the Fabry program." * Tazeen Ahmad, BofA Securities: asked about U.S. vs. ex-U.S. long-term sales split and whether the U.S. launch is slowing; CEO Klein responded, "we really see ourselves now in a steady, durable growth phase" and said, "the majority of the revenue will be U.S." while ex-U.S. contributes with "periods of acceleration." * Benjamin Burnett, Wells Fargo: asked what raised guidance assumes across Sephience vs. DMD; CEO Klein said guidance is mainly Sephience-driven and noted DMD headwinds, including, "we're now seeing a new generic entrant... that has brought down the price" for Emflaza and "we do not expect in Q4 to have any additional large government purchase orders from Brazil or Russia" for Translarna. * Benjamin Burnett, Wells Fargo: asked about additional BD appetite; CFO Gravier said, "we will apply the same philosophy... We want to be very disciplined" and "make sure that we create value for shareholders." * Tejas (on for Eliana Merle), Barclays: asked about seasonality and persistence; CEO Klein said, "we did see some summer seasonality" and added, "we've been around low 20% cumulative discontinuations to date." * Lut Ming Cheng, JPMorgan: asked on Germany/Europe pricing timing; CBO Pauwels said Germany is "still in active negotiations" and for France/Italy/Spain, "we anticipate somewhere in the first half of 2027 for those to be finalized." * Eric Joseph, Citi: asked how ST-920 could launch (access, reimbursement, early adopters); CBO Pauwels said, "This market is incredibly large in the U.S. alone" and emphasized segmentation and durability messaging from "the STAAR study." * Faisal Khurshid, Jefferies: asked levers for continued U.S. growth and the votoplam meeting delay; CEO Klein pointed to "more and more of the switch dynamic" and said the meeting shift is "simply a matter of FDA scheduling." * Joseph Thome, TD Cowen: asked why Kuvan switchers haven't moved yet and where lapsed patients sit; CEO Klein cited clinic throughput and timing, adding, "access questions and payer questions become a when, not an if," and described patient-network pull from "social media." * Maxwell Skor, Morgan Stanley: asked whether accelerated approval could disrupt INVEST-HD enrollment and about ST-920 priority review; CEO Klein said INVEST-HD was designed to "serve as a confirmatory study" and added, "the potential for priority review comes along with that RMAT designation." * Asim Rana (on for Joon Lee), UBS: asked about Sephience dosing trends and ST-920 launch OpEx/accretion; CEO Klein said average weight assumptions have "been pretty stable" and that ST-920 spend would be "an incremental spend," adding, "we believe this could be accretive in 2027." Sentiment analysis. * Analysts' tone was slightly skeptical to neutral, repeatedly probing durability and slope of Sephience demand (e.g., BofA asking, "is the U.S. launch slowing?") and pushing for quantifiable launch indicators and pricing visibility in Europe. * Management's tone was positive and confident on Sephience and guidance, using reinforcing language such as "steady, durable growth phase" and "remain confident in the $2 billion-plus peak sales opportunity for Sephience," while sounding matter-of-fact on votoplam timing: "simply a matter of FDA scheduling." (CEO & Director Klein) * Versus Q2, management language became more definitive on cash flow and the 2026 target (Q3: "expect to achieve our goal" vs. Q2: "remain in a position to potentially reach"), while analysts' questions in Q3 leaned more into sustainability (U.S. slowing, seasonality, switch conversion) and near-term BD implications after the ST-920 deal. Quarter-over-Quarter comparison. * Q3 raised 2026 guidance ranges compared with Q2, and management shifted wording from "potentially reach" cash flow breakeven to "expect to achieve" it in 2026. (CEO & Director Klein, Q2 vs. Q3 calls) * Sephience scale markers moved from "1,647 commercial patients" in Q2 to "over 2,000 active patients" in Q3, while management's launch narrative emphasized being "at the 1-year launch mark" and "still in the very early innings." (CEO & Director Matthew Klein, Q2 vs. Q3 calls) * Q3 introduced a major strategic addition - "we acquired the BLA-stage... ST-920" - and tied it to a near-term regulatory plan, while Q2 framed BD more generally as flexibility supported by cash and infrastructure. (CEO & Director Klein; CFO Gravier) * Analysts in Q2 focused heavily on early access revenue recognition and HTA timelines; Q3 questions expanded to include U.S. growth durability, European pricing cadence, and launch mechanics for ST-920. (Q2 vs. Q3 Q&A) Risks and concerns. * Management flagged near-term pressure in the DMD franchise: "we're now seeing a new generic entrant... that has brought down the price" for Emflaza, and noted purchase-order variability for Translarna: "we do not expect in Q4 to have any additional large government purchase orders from Brazil or Russia." (CEO & Director Klein) * On European access timing risk, management said Germany pricing has "nothing specific yet" and Southern Europe negotiations "take a little bit longer" with expectations for finalization "somewhere in the first half of 2027." (CBO Eric Pauwels) * On regulatory timing uncertainty for votoplam, management attributed the delay to logistics: "this is simply a matter of FDA scheduling. There's nothing to read in there." (CEO & Director Klein) Final takeaway. PTC management emphasized record product revenue and raised 2026 revenue guidance while reiterating that Sephience remains in "the very early innings" with "over 2,000 active patients" and most eligible patients still not having tried therapy. The company highlighted a material near-term pipeline and commercial catalyst with the ST-920 Fabry gene therapy acquisition, targeting a rolling BLA completion in Q4 2026 and positioning for a potential 2027 launch, alongside continued preparations for regulatory engagement on votoplam in early 2027 and ongoing efforts to secure European Sephience pricing and reimbursement over 2027. Fresh Stock Ideas, Every Day Explore diverse investing perspectives with daily analysis from experts across the market. More on PTC Therapeutics. Seeking Alpha's Disclaimer: This article was automatically generated by an AI tool based on content available on the Seeking Alpha website, and has not been curated or reviewed by humans. Due to inherent limitations in using AI-based tools, the accuracy, completeness, or timeliness of such articles cannot be guaranteed. This article is intended for informational purposes only. Seeking Alpha does not take account of your objectives or your financial situation and does not offer any personalized investment advice. Seeking Alpha is not a licensed securities dealer, broker or US investment adviser or investment bank.
PTC Therapeutics reported a third-quarter loss of $51.7 million, or 62 cents per share. Adjusted for non-recurring costs and debt extinguishment, the company posted earnings of 61 cents per share. The New Jersey-based biopharmaceutical company beat Wall Street expectations. Four analysts surveyed by Zacks Investment Research had expected earnings of 17 cents per share. Revenue reached $357.8 million, above the $311.1 million that three analysts forecast. PTC expects full-year revenue of $1.29 billion to $1.32 billion.
PTC Therapeutics (NASDAQ:PTCT) releases quarterly earnings results, misses expectations by $0.76 EPS. October 29, 2026 Key points. * PTC Therapeutics missed earnings expectations: The company reported a quarterly loss of $0.62 per share versus the $0.14 consensus estimate, although revenue rose 69.6% year over year to $357.81 million, exceeding forecasts. * Management raised its outlook after a record quarter: 2026 product-revenue guidance increased to $960 million-$990 million, with cash-flow breakeven still expected this year. SEPHIENCE revenue reached $182 million, supported by more than 2,000 active patients worldwide. * The pipeline offers growth potential but faces execution risks: PTC expects to complete its ST-920 gene-therapy filing in the fourth quarter, while mature DMD products face generic and one-time-order pressure; the Votoplam FDA meeting was also delayed to early 2027. * MarketBeat previews top five stocks to own in November. PTC Therapeutics (NASDAQ:PTCT - Get Free Report) announced its quarterly earnings data on Thursday. The biopharmaceutical company reported ($0.62) earnings per share (EPS) for the quarter, missing the consensus estimate of $0.14 by ($0.76), FiscalAI reports. The firm had revenue of $357.81 million for the quarter, compared to analyst estimates of $306.43 million. The company's revenue was up 69.6% compared to the same quarter last year. During the same quarter last year, the firm posted $0.20 EPS. Here are the key takeaways from PTC Therapeutics' conference call: * Guidance was raised after a record quarter: Third-quarter total revenue reached $358 million, including $276 million in product revenue, and 2026 product revenue guidance increased to $960 million-$990 million. Management also reiterated its expectation to reach cash-flow breakeven this year. * SEPHIENCE launch momentum remains strong: Global SEPHIENCE revenue was $182 million, up more than 20% sequentially, with over 2,000 active patients worldwide and strong adherence. PTC said the U.S. launch is entering a durable growth phase and maintained its confidence in a $2 billion-plus peak-sales opportunity. * ST-920 adds a potentially near-term growth driver: PTC expects to complete its rolling BLA submission for the Fabry gene therapy in the fourth quarter, with potential approval and launch in 2027. The company said the $111 million acquisition could be accretive in 2027 and noted ST-920's RMAT designation may support priority review. * Mature DMD products face increasing pressure: A new Emflaza generic entrant has significantly reduced pricing, while Translarna's third-quarter revenue benefited from an unexpected Russian government order that PTC does not expect to repeat in the fourth quarter. * Votoplam's regulatory discussion was delayed: The FDA Type C meeting on the Huntington's disease program is now expected in the first quarter of 2027 rather than late 2026, although global enrollment in the 770-patient INVEST-HD Phase III study continues. PTC Therapeutics trading up 2.3%. PTCT stock traded up $1.44 during midday trading on Thursday, hitting $64.46. The stock had a trading volume of 1,524,172 shares, compared to its average volume of 996,871. The firm has a market capitalization of $5.38 billion, a price-to-earnings ratio of -111.14, a P/E/G ratio of 1.90 and a beta of 0.49. PTC Therapeutics has a 52 week low of $58.80 and a 52 week high of $90.87. The stock has a 50-day simple moving average of $65.57 and a 200 day simple moving average of $70.87. Insiders place their bets. In other news, VP Mark Boulding sold 435 shares of the firm's stock in a transaction dated Tuesday, October 6th. The shares were sold at an average price of $64.16, for a total transaction of $27,909.60. Following the completion of the transaction, the vice president directly owned 105,272 shares of the company's stock, valued at approximately $6,754,251.52. This trade represents a 0.41% decrease in their position. The transaction was disclosed in a legal filing with the Securities & Exchange Commission, which is accessible through the SEC website. The transaction was executed under a pre-arranged Rule 10b5-1 trading plan. Also, Director Michael Schmertzler sold 2,500 shares of the company's stock in a transaction dated Wednesday, October 14th. The shares were sold at an average price of $59.61, for a total transaction of $149,025.00. Following the completion of the sale, the director directly owned 1,291,594 shares in the company, valued at approximately $76,991,918.34. The trade was a 0.19% decrease in their position. The SEC filing for this sale provides additional information. The transaction was executed under a pre-arranged Rule 10b5-1 trading plan. Insiders have sold 77,370 shares of company stock worth $5,322,261 over the last 90 days. Corporate insiders own 5.30% of the company's stock. Analysts set new price targets. PTCT has been the subject of a number of research reports. Morgan Stanley increased their price target on shares of PTC Therapeutics from $93.00 to $104.00 and gave the company an "overweight" rating in a report on Friday, July 31st. Zacks Research downgraded shares of PTC Therapeutics from a "strong-buy" rating to a "hold" rating in a report on Thursday, August 6th. Barclays upped their price objective on shares of PTC Therapeutics from $120.00 to $122.00 and gave the company an "overweight" rating in a research report on Friday, August 14th. UBS Group assumed coverage on shares of PTC Therapeutics in a research note on Thursday, October 15th. They issued a "buy" rating and a $90.00 target price for the company. Finally, Raymond James Financial set a $106.00 target price on PTC Therapeutics in a research report on Tuesday, October 20th. One research analyst has rated the stock with a Strong Buy rating, ten have given a Buy rating, three have given a Hold rating and two have issued a Sell rating to the stock. Based on data from MarketBeat, the stock presently has a consensus rating of "Moderate Buy" and an average price target of $94.93. Discover more MarketBeat All Access MarketBeat Research Tools PTC Therapeutics company profile. PTC Therapeutics, Inc is a biopharmaceutical company focused on discovering, developing and commercializing medicines for rare diseases. Its research programs include small-molecule treatments, gene therapies and other approaches designed to address conditions with limited or no available treatment options. The company's commercial portfolio has included Emflaza (deflazacort), a corticosteroid approved in the United States to treat Duchenne muscular dystrophy, and Upstaza (eladocagene exuparvovec), a gene therapy authorized in the European Union for aromatic L-amino acid decarboxylase deficiency. This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to [email protected]. Before you consider PTC Therapeutics, you'll want to hear this. MarketBeat keeps track of Wall Street's top-rated and best performing research analysts and the stocks they recommend to their clients on a daily basis. MarketBeat has identified the five stocks that top analysts are quietly whispering to their clients to buy now before the broader market catches on... and PTC Therapeutics wasn't on the list. While PTC Therapeutics currently has a Moderate Buy rating among analysts, top-rated analysts believe these five stocks are better buys. Looking for higher dividend income in 2027? Discover seven high-yield dividend stocks with attractive payouts, proven dividend histories and growth potential. Continue following MarketBeat
PTC Therapeutics schedules October 29 results as stock trades below its high. Published on 10/20/2026 at 18:54 | Editorial responsibility: Rafael Müller, Editor-in-Chief AD HOC NEWS PTC Therapeutics reports third-quarter results on October 29, 2026. PTC Therapeutics stock costs EUR 55.60 on October 20, 2026. Reporting date October 29, 2026. * PTC Therapeutics will report third-quarter 2026 results on October 29, 2026. * Second-quarter 2026 revenue reached USD 360.52 million, up 101.50 percent year over year. * PTC Therapeutics stock was trading at EUR 55.60 at Lang & Schwarz on October 20, 2026 at 6:44 p.m. CEST. * Truist Financial assigned a USD 62.00 target and a Hold rating on October 16, 2026. PTC Therapeutics, Inc. (ISIN US69366J2006) is scheduled to report its third-quarter 2026 financial results on October 29, 2026. The company will host a conference call at 4:30 p.m. ET, according to PTC Therapeutics. The stock was trading at EUR 55.60 at Lang & Schwarz on October 20, 2026 at 6:44 p.m. CEST. Discover more Read Economics Trade Derivatives Order Calendars Revenue doubled in the second quarter. PTC Therapeutics reported USD 360.52 million in revenue for the second quarter of 2026, an increase of 101.50 percent from the same quarter of 2025. Net income reached USD 83.50 million, compared with a net loss in the prior-year quarter, while profit margin was 23.16 percent, according to Simply Wall St. The second-quarter performance gives the October 29 report a clear comparison point. The company had reported adjusted earnings per share of USD 0.92 for that period against a consensus estimate of USD 0.24, while revenue exceeded the USD 298.63 million estimate. What does the analyst view imply? Truist Financial resumed coverage of PTC Therapeutics with a Hold rating and a USD 62.00 price target on October 16, 2026, according to GuruFocus. The report cited concerns about long-term growth and the addressable market for Sephience in phenylketonuria. For comparison, the latest primary-market quote was USD 62.47 on Nasdaq at 12:53 p.m. ET on October 20, 2026. The same quote showed a 52-week range of USD 58.80 to USD 90.87 and a market capitalization of USD 5.2 billion. PTC Therapeutics therefore remains 31.25 percent below its 52-week high on that basis. October 29 is the next date. The next scheduled corporate event is the third-quarter report on October 29, 2026. PTC Therapeutics also completed the acquisition of the ST-920 Fabry disease gene therapy, a transaction listed on its investor-relations page alongside the second-quarter materials. Stock price and reporting date. Discover more Track Stock Market The October 29 reporting date places the next material company update within nine days. The second-quarter revenue increase of 101.50 percent and the USD 62.00 Truist target provide two quantified benchmarks for the upcoming release. The stock was trading at EUR 55.60 at Lang & Schwarz on October 20, 2026 at 6:44 p.m. CEST. The further course of trading is shown by the continuously updated real-time quote of PTC Therapeutics stock. PTC Therapeutics market facts. * Company: PTC Therapeutics, Inc. * ISIN: US69366J2006 * Ticker: PTCT * Primary exchange: Nasdaq * Price Lang & Schwarz (as of October 20, 2026, 6:44 p.m. CEST): EUR 55.60 * Sector / Industry: Healthcare / Biotechnology * Next earnings date: October 29, 2026 Sponsored Ad PTC Therapeutics stock: new analysis - 22 October. Fresh PTC Therapeutics information released. What's the impact for investors? Our latest independent report examines recent figures and market trends. Discover more Track Biotech Read Health Guides Invest in Stocks Disclaimer regarding our articles: This is not investment advice, nor is it a recommendation to buy or sell. Information regarding prices, companies, and markets is provided without guarantee; changes may occur at any time. Stock market transactions can result in significant losses. Our articles are created and reviewed, in whole or in part, automatically with the assistance of AI. en | US69366J2006 | PTCT | boerse | 70376274 | bgmi
PTC Therapeutics is Hiring for 42 Jobs on Simplify!
Find jobs on Simplify and start your career today
Don't see your dream role? Check out thousands of other roles on Simplify.Browse all jobs
About PTC Therapeutics
Industries
Company Size
1,001-5,000
Company Stage
IPO
Headquarters
Warren Township, New Jersey
Founded
1998
PTC Therapeutics is Hiring for 42 Jobs on Simplify!
Find jobs on Simplify and start your career today
Don't see your dream role? Check out thousands of other roles on Simplify.Browse all jobs