PTC Therapeutics

PTC Therapeutics

Biopharmaceuticals for rare genetic disorders

Overview

PTC Therapeutics is a global biopharmaceutical company that discovers, develops, and markets medicines for rare genetic diseases. It uses RNA biology and post-transcriptional control to create therapies, including small-molecule drugs and gene therapies. Its products include Translarna for nmDMD, Evrysdi for SMA, Emflaza, Upstaza for AADC deficiency, and Kebilidi for CNS indications, all supported by an integrated model from research to commercialization and operations in more than 50 countries with a European base in Dublin. The company aims to provide safe, effective treatments to patients with few or no options by building a strong pipeline and broad global access.

About PTC Therapeutics

Simplify's Rating
Why PTC Therapeutics is rated
B-
Rated B on Competitive Edge
Rated B on Growth Potential
Rated C on Differentiation

Industries

Biotechnology

Healthcare

Company Size

1,001-5,000

Company Stage

IPO

Headquarters

South Plainfield, New Jersey

Founded

1998

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Simplify's Take

What believers are saying

  • PTC raised 2026 guidance to $1.18 billion-$1.28 billion after Q2 beat.
  • Q2 2026 net income reached $83.5 million, proving operating leverage.
  • July 2026 Novartis milestone and ST-920 auction win extend pipeline value into 2027.

What critics are saying

  • FDA rejected Translarna U.S. approval on February 12, 2026, ending a 20-year pursuit.
  • European regulators refused Translarna renewal in 2024, shrinking PTC's DMD franchise further.
  • If Sephience adoption slows after 2026, PTC loses its only real growth engine.

What makes PTC Therapeutics unique

  • Sephience drove Q2 2026 revenue to $151 million across 1,647 patients worldwide.
  • PTC monetizes rare-disease commercialization, combining orphan launches, royalties, and regulatory execution.
  • ST-920 acquisition brings Fabry gene therapy without building a new commercial infrastructure.

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Funding

Total Funding

$2B

Above

Industry Average

Funded Over

8 Rounds

Post IPO Convertible funding comparison data is currently unavailable. We're working to provide this information soon!
Post IPO Convertible Funding Comparison
Coming Soon

Benefits

Health Insurance

Dental Insurance

Vision Insurance

401(k) Retirement Plan

Flexible Work Hours

Performance Bonus

Stock Price

Growth & Insights and Company News

Headcount

6 month growth

0%

1 year growth

0%

2 year growth

0%
The Pharma Letter
Aug 8th, 2026
PTC to expand rare disease portfolio with ST-920 buy.

PTC to expand rare disease portfolio with ST-920 buy. 13 August 2026 USA-based rare disease drug developer PTC Therapeutics (Nasdaq: PTCT) announced that it was selected as the winning bidder to acquire ST-920 - a biologics license application (BLA)-stage one-time administered AAV gene therapy for Fabry disease - from Sangamo Therapeutics in a competitive bankruptcy auction. "This transaction advances our strategy of leveraging our accomplished existing rare disease global commercial infrastructure to accelerate short- and intermediate-term revenue growth," said Dr Matthew Klein, chief executive, adding: "The ST-920 gene therapy program puts another innovative and valuable product in the demonstrated capable hands of our customer-facing teams. This was a unique opportunity with the potential for significant return on investment without the need for any development or commercial build and without impacting our objective of reaching cashflow break even in 2026. We look forward to working to bring ST-920 to all individuals who may benefit from this therapy as quickly as possible." This article is accessible to registered users, to continue reading please register for free. A free trial will give you access to exclusive features, interviews, round-ups and commentary from the sharpest minds in the pharmaceutical and biotechnology space for a week. If you are already a registered user please login. If your trial has come to an end, you can subscribe here. Try before you buy Free. 7 day trial access * All the news that moves the needle in pharma and biotech * Exclusive features, podcasts, interviews, data analyses and commentary from its global network of life sciences reporters. * Receive The Pharma Letter daily news bulletin, free forever. Become a subscriber £820. Or £77 per month * Unfettered access to industry-leading news, commentary and analysis in pharma and biotech. * Updates from clinical trials, conferences, M&A, licensing, financing, regulation, patents & legal, executive appointments, commercial strategy and financial results. * Daily roundup of key events in pharma and biotech. * Monthly in-depth briefings on Boardroom appointments and M&A news. * Choose from a cost-effective annual package or a flexible monthly subscription The Pharma Letter is an extremely useful and valuable Life Sciences service that brings together a daily update on performance people and products. It's part of the key information for keeping me informed Chairman, Sanofi Aventis UK More on this story... 24 June 2026 13 February 2026 20 August 2025 Companies featured in this story. Sign up to receive email updates Join industry leaders for a daily roundup of biotech & pharma news Today's issue. 13 August 2026 12 August 2026 Company spotlight. An American biotech company developing gene-modulating therapies, particularly for neuromuscular diseases. More features in biotechnology. 13 August 2026 12 August 2026

Yahoo Finance
Jul 31st, 2026
PTC Therapeutics Q2 revenue jumps 102% to $361M, beats estimates by 22%

PTC Therapeutics reported revenue of $360.52 million for the quarter ended June 2026, a 101.5% year-over-year increase. The biotech company posted earnings per share of $0.92, compared to a loss of $0.83 in the same period last year. The revenue figure exceeded the consensus estimate of $294.92 million by 22.24%. EPS also beat expectations, with analysts projecting a loss of $0.17 per share. Net product revenue reached $238.82 million, up 101.8% year over year. Royalty revenue came in at $71.11 million, a 23.4% increase. However, individual product revenues for Emflaza and Translarna declined 32.3% and 29.1% respectively. Shares have fallen 9.4% over the past month.

Yahoo Finance
Jul 31st, 2026
PTC Therapeutics hits record $361M revenue in Q2 2026, raises guidance to $1.18B-$1.28B

PTC Therapeutics reported record quarterly revenue of $361 million in Q2 2026, driven by strong product sales of $239 million—more than double the $118 million recorded in Q2 2025. The company raised its full-year 2026 product revenue guidance to $850 million to $950 million, with total revenue expected between $1.18 billion and $1.28 billion. Sephience led growth with $151 million in global net product revenue, up 21% quarter-over-quarter, serving 1,647 commercial patients globally. The company reported $71 million in Evrysdi royalty revenue based on Roche's global sales of approximately $628 million. PTC Therapeutics ended June 2026 with $2.23 billion in cash equivalents and marketable securities. The company refinanced convertible notes at 0% coupon with a 40% conversion premium, positioning itself for potential cash flow breakeven in 2026.

PR Newswire
Jul 30th, 2026
PTC Therapeutics raises 2026 revenue guidance to $1.18B–$1.28B on strong Sephience uptake

PTC Therapeutics reported second quarter 2026 revenue of $361 million, including $239 million in product revenue. The company raised its full-year 2026 total revenue guidance to $1.18 to $1.28 billion and increased product revenue guidance to $850 to $950 million from $750 to $850 million. Sephience generated $151 million in Q2 revenue, with 1,647 patients on commercial therapy worldwide as of 30 June 2026. The company held cash, cash equivalents, and marketable securities totalling $2.2 billion. In June 2026, PTC issued $550 million in senior convertible notes due in 2031 at 0% interest with a 40% premium conversion price. The company received a $50 million milestone payment from Novartis after dosing the first patient in the INVEST-HD Phase 3 study. PTC named PTC303 as its development candidate targeting Huntington's disease and myotonic dystrophy, planning clinical trials in 2027.

MarketBeat
Jul 30th, 2026
PTC Therapeutics (NASDAQ:PTCT) posts quarterly earnings results, beats expectations by $0.68 EPS.

PTC Therapeutics (NASDAQ:PTCT) posts quarterly earnings results, beats expectations by $0.68 EPS. July 30, 2026 Key points. * PTC Therapeutics beat quarterly expectations: The biopharmaceutical company reported $0.92 in EPS versus the $0.24 consensus estimate, while revenue of $360.52 million exceeded forecasts of $298.63 million. * Shares rose $1.03 to $75.52 in Thursday trading, giving PTC Therapeutics a market capitalization of approximately $6.26 billion; the stock remains below its 52-week high of $90.87. * Analysts maintain a "Moderate Buy" consensus rating with an average price target of $93.83, while insiders sold roughly $16.35 million of stock over the past three months under pre-arranged trading plans. * Five stocks to consider instead of PTC Therapeutics. PTC Therapeutics (NASDAQ:PTCT - Get Free Report) issued its quarterly earnings data on Thursday. The biopharmaceutical company reported $0.92 EPS for the quarter, beating analysts' consensus estimates of $0.24 by $0.68, Zacks reports. PTC Therapeutics had a negative return on equity of 362.45% and a negative net margin of 22.58%.The business had revenue of $360.52 million for the quarter, compared to analyst estimates of $298.63 million. PTC Therapeutics price performance. Shares of NASDAQ:PTCT traded up $1.03 during trading on Thursday, reaching $75.52. The stock had a trading volume of 1,373,818 shares, compared to its average volume of 1,459,631. The stock has a market capitalization of $6.26 billion, a PE ratio of -32.41, a P/E/G ratio of 6.56 and a beta of 0.53. PTC Therapeutics has a 52 week low of $43.17 and a 52 week high of $90.87. The business's 50-day moving average price is $77.47 and its 200 day moving average price is $72.79. Insider activity at PTC Therapeutics. In other news, Director Mary L. Smith sold 15,834 shares of the stock in a transaction on Thursday, July 9th. The stock was sold at an average price of $90.00, for a total transaction of $1,425,060.00. Following the sale, the director owned 21,813 shares in the company, valued at approximately $1,963,170. This represents a 42.06% decrease in their ownership of the stock. The sale was disclosed in a document filed with the Securities & Exchange Commission, which is accessible through this link. The transaction was executed under a pre-arranged Rule 10b5-1 trading plan. Also, VP Mark Elliott Boulding sold 5,079 shares of the stock in a transaction on Monday, July 6th. The stock was sold at an average price of $85.08, for a total value of $432,121.32. Following the sale, the vice president owned 105,272 shares in the company, valued at $8,956,541.76. The trade was a 4.60% decrease in their position. The disclosure for this sale is available in the SEC filing. The transaction was executed under a pre-arranged Rule 10b5-1 trading plan. Insiders sold 198,662 shares of company stock valued at $16,347,994 in the last three months. Corporate insiders own 5.50% of the company's stock. Institutional inflows and outflows. Several institutional investors and hedge funds have recently added to or reduced their stakes in PTCT. IFP Advisors Inc increased its position in PTC Therapeutics by 68.1% in the 3rd quarter. IFP Advisors Inc now owns 765 shares of the biopharmaceutical company's stock valued at $47,000 after acquiring an additional 310 shares in the last quarter. Virtus Investment Advisers LLC raised its stake in shares of PTC Therapeutics by 3.6% in the fourth quarter. Virtus Investment Advisers LLC now owns 9,033 shares of the biopharmaceutical company's stock worth $686,000 after acquiring an additional 316 shares during the last quarter. UMB Bank n.a. acquired a new position in shares of PTC Therapeutics during the fourth quarter worth approximately $26,000. Mariner LLC lifted its position in shares of PTC Therapeutics by 5.1% during the third quarter. Mariner LLC now owns 8,745 shares of the biopharmaceutical company's stock worth $537,000 after purchasing an additional 426 shares in the last quarter. Finally, Smartleaf Asset Management LLC lifted its position in shares of PTC Therapeutics by 96.4% during the fourth quarter. Smartleaf Asset Management LLC now owns 921 shares of the biopharmaceutical company's stock worth $70,000 after purchasing an additional 452 shares in the last quarter. Analyst upgrades and downgrades. PTCT has been the topic of several research analyst reports. Jefferies Financial Group raised PTC Therapeutics from a "hold" rating to a "buy" rating and raised their price target for the company from $76.00 to $85.00 in a research report on Wednesday, May 27th. Morgan Stanley reaffirmed an "overweight" rating and issued a $94.00 target price on shares of PTC Therapeutics in a report on Friday, May 8th. Citigroup raised PTC Therapeutics from a "neutral" rating to a "buy" rating and increased their price objective for the company from $75.00 to $108.00 in a research report on Tuesday, June 9th. Raymond James Financial initiated coverage on shares of PTC Therapeutics in a report on Friday, April 10th. They set an "outperform" rating and a $108.00 price objective on the stock. Finally, Wells Fargo & Company reduced their target price on shares of PTC Therapeutics from $95.00 to $93.00 and set an "overweight" rating for the company in a research note on Monday, July 20th. Two equities research analysts have rated the stock with a Strong Buy rating, nine have issued a Buy rating, one has assigned a Hold rating and two have issued a Sell rating to the stock. Based on data from MarketBeat.com, PTC Therapeutics currently has an average rating of "Moderate Buy" and an average target price of $93.83. Discover more Stock Market News Financial News Subscription Stock Split Calculator PTC Therapeutics company profile. PTC Therapeutics, Inc is a biopharmaceutical company focused on the discovery, development and commercialization of small molecule and biologic therapies for the treatment of rare genetic disorders. Since its founding in 1998, PTC has dedicated its efforts to addressing high unmet medical needs by targeting underlying genetic causes of disease. The company's research platform emphasizes mechanisms such as nonsense suppression and RNA modulation, enabling the development of novel treatments for conditions with limited therapeutic options. Among PTC's approved products is Translarna (ataluren), a first-in-class therapy designed to treat nonsense mutation Duchenne muscular dystrophy in select markets. Further reading. 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. With the proliferation of data centers and electric vehicles, the electric grid will only get more strained. 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