Actualités en direct • 20h
PTC Therapeutics to Acquire Late-Stage Fabry Gene Therapy Program for $111 Million PTC Therapeutics has been selected as the winning bidder to acquire Sangamo Therapeutics’ BLA-stage Fabry disease gene therapy, ST-920 (isaralgagene civaparvovec), for $111 million in cash upfront plus up to $100 million in potential milestone payments, with a rolling FDA BLA submission for accelerated approval expected by Q4 2026.
The deal brings PTC Therapeutics a one-time AAV gene therapy that is described as BLA-ready. It adds a late-stage rare disease asset that aligns closely with the company’s existing focus on genetic disorders and could broaden its potential treatment portfolio if regulatory milestones are met.
PTC Therapeutics’ shares trade at $71.97, with the stock down 8.7% over the past 30 days, which frames the acquisition against a period of recent share price pressure.
The key read-through is that PTC Therapeutics is committing meaningful capital to expand its rare disease pipeline with a late-stage gene therapy, concentrating both opportunity and development risk around future regulatory decisions. Recent Insider Transactions • Aug 07
CEO & Director recently sold US$706k worth of stock On the 4th of August, Matthew Klein sold around 10k shares on-market at roughly US$68.57 per share. This transaction amounted to 6.2% of their direct individual holding at the time of the trade. In the last 3 months, they made an even bigger sale worth US$917k. Matthew has been a net seller over the last 12 months, reducing personal holdings by US$4.2m. Recent Insider Transactions Derivative • Aug 05
CEO & Director notifies of intention to sell stock Matthew Klein intends to sell 10k shares in the next 90 days after lodging an Intent To Sell Form on the 4th of August. If the sale is conducted around the recent share price of US$68.57, it would amount to US$706k. For the year to December 2022, Matthew's total compensation was 4% salary and 96% other compensation. This indicates that these sales could comprise a meaningful part of their income for the year. Matthew currently holds less than 1% of total shares outstanding. Company insiders have collectively sold US$32m more than they bought, via options and on-market transactions in the last 12 months. Annonce • Aug 01
PTC Therapeutics, Inc. Announces Appointment of Hege Sollie-Zetlmayer to Board of Directors, Effective July 28, 2026 PTC Therapeutics, Inc. announced that Hege Sollie-Zetlmayer had been appointed to its board of directors, effective July 28, 2026, filling a vacancy on the Board. Ms. Sollie-Zetlmayer will serve as a Class III director with a term expiring at the annual meeting of stockholders to be held in 2028. Ms. Sollie-Zetlmayer has 30 years of experience in the biopharmaceutical and medical device industry, including the last eight with PTC Therapeutics, Inc. Ms. Sollie-Zetlmayer joined the Company in June 2018 as VP Human Resources Europe, Asia Pacific, and became VP Global Talent Management & Global HR Business Partners in November 2018, SVP, Global Human Resources in November 2020 and Chief Human Resources Officer from November 2023, a role she held until her retirement in June 2026. She serves as a director of PTC Therapeutics International Limited, its international headquarters and indirect wholly owned subsidiary, and previously served as a director of PTC Therapeutics Switzerland GmbH, its indirect wholly owned subsidiary. Ms. Sollie-Zetlmayer founded and manages HSZ Consulting GmbH, an executive advisory company, and previously served as the first female chairman of the Norwegian Medical Device Association. Ms. Sollie-Zetlmayer studied Human Resources at the Norwegian School of Business BI in Oslo and holds a certificate from Wharton Executive Education in Corporate Governance. Reported Earnings • Jul 31
Second quarter 2026 earnings: EPS and revenues exceed analyst expectations Second quarter 2026 results: EPS: US$1.01 (up from US$0.83 loss in 2Q 2025). Revenue: US$360.5m (up 101% from 2Q 2025). Net income: US$83.5m (up US$148.3m from 2Q 2025). Profit margin: 23% (up from net loss in 2Q 2025). Revenue exceeded analyst estimates by 21%. Earnings per share (EPS) also surpassed analyst estimates significantly. Revenue is forecast to grow 13% p.a. on average during the next 3 years, compared to a 22% growth forecast for the Biotechs industry in the US. Over the last 3 years on average, earnings per share has increased by 79% per year but the company’s share price has only increased by 21% per year, which means it is significantly lagging earnings growth. Actualités en direct • Jul 31
PTC Therapeutics Lifts 2026 Revenue Guidance After Clinical Progress and Board Appointment PTC Therapeutics raised its full-year 2026 product revenue guidance alongside reporting second quarter 2026 results, citing progress in clinical trials for Huntington’s disease and Friedreich’s ataxia and appointing Hege Sollie-Zetlmayer to its Board of Directors.
Management also reported a strong cash position as of 30 June 2026, which could give PTC Therapeutics more room to fund its R&D programs and support the clinical work highlighted in the update.
The stock trades at $75.52, with the share price up 15.9% over the past 90 days, while year to date it is slightly down 1.6%.
The combination of higher product revenue guidance, active late-stage trial work and a strengthened Board points to a company leaning on both its commercial base and pipeline. The cash position is important because it helps reduce immediate funding pressure as PTC Therapeutics advances its neurology programs. Annonce • Jul 16
PTC Therapeutics, Inc. to Report Q2, 2026 Results on Jul 30, 2026 PTC Therapeutics, Inc. announced that they will report Q2, 2026 results on Jul 30, 2026 Recent Insider Transactions Derivative • Jul 07
Chief Technical Operations Officer notifies of intention to sell stock Neil Almstead intends to sell 22k shares in the next 90 days after lodging an Intent To Sell Form on the 6th of July. If the sale is conducted around the recent share price of US$86.00, it would amount to US$1.9m. Neil currently holds less than 1% of total shares outstanding. Company insiders have collectively sold US$29m more than they bought, via options and on-market transactions in the last 12 months. Actualités en direct • Jun 25
Sephience Launch Drives 75% Rally as PTC Therapeutics Eyes $2 Billion Peak Sales PTC Therapeutics’ shares have rallied 75% since phenylketonuria drug Sephience was approved in July 2025, with the treatment generating US$124.6 million in sales in 1Q26 and underpinning management’s US$2 billion peak sales target.
Management reports a cash balance of US$1.89 billion and a collaboration with Novartis, which together provide funding capacity and a partner framework for future business development.
PTC Therapeutics’ stock last traded at US$84.41, with a 26.0% return over the past 90 days, reflecting strong recent trading momentum.
Sephience’s early sales performance and PTC Therapeutics’ cash position give the company room to fund commercialization and pursue pipeline or partnership opportunities, although execution on the Sephience ramp and outcomes from the Novartis collaboration remain key operational risks. Actualités en direct • Jun 15
Citi Upgrades PTC Therapeutics to Buy as Strong Sephience Sales Boost Outlook and Debt Plan Unveiled Citi upgraded PTC Therapeutics to Buy and set a US$108 price target, citing strong Sephience sales in phenylketonuria and an expected push into international markets.
The bank also highlighted pipeline drugs votoplam for Huntington's disease and vatiquinone for Friedreich's ataxia as underappreciated assets that could support future operations.
PTC Therapeutics announced a proposed US$500 million offering of Convertible Senior Notes due 2031, with an additional US$50 million overallotment option, aiming to refinance its 2026 convertible notes and repurchase about US$50 million of common stock.
Citi’s upgrade focuses attention on both Sephience’s current commercial traction and the broader pipeline. This comes at the same time that PTC is moving to reshape its balance sheet through a longer-dated convertible notes deal and a modest share buyback.
You should watch how the terms of the new notes, the scale of 2026 debt retired, and any updates on Sephience guidance or pipeline milestones affect the company’s future financial flexibility and dilution risk. Recent Insider Transactions Derivative • Jun 11
Chief Business Officer notifies of intention to sell stock Eric Pauwels intends to sell 21k shares in the next 90 days after lodging an Intent To Sell Form on the 10th of June. If the sale is conducted around the recent share price of US$75.00, it would amount to US$1.6m. Since September 2025, Eric's direct individual holding has increased from 20.06k shares to 25.12k. Company insiders have collectively sold US$24m more than they bought, via options and on-market transactions in the last 12 months. Annonce • Jun 10
PTC Therapeutics, Inc. Announces Resignation of Alethia Young from Director, Effective June 8, 2026 PTC Therapeutics, Inc. reported that on June 8, 2026, Alethia Young, a member of the board of directors of the company, notified the company of her resignation from the board effective June 8, 2026. Ms. Young has informed the company that her decision is based on her need to focus on commitments to her other work activities and was not due to any disagreement with the company. The company and Ms. Young entered into a consulting agreement effective as of June 8, 2026, pursuant to which Ms. Young will remain as an advisor to the company, with a term continuing through June 8, 2027. Ms. Young previously served as a member of the board of directors of PTC Therapeutics, Inc. and participated in the oversight of PTC and its activities until her resignation from the board effective as of June 8, 2026. Recent Insider Transactions Derivative • May 17
CEO & Director notifies of intention to sell stock Matthew Klein intends to sell 10k shares in the next 90 days after lodging an Intent To Sell Form on the 14th of May. If the sale is conducted around the recent share price of US$73.08, it would amount to US$731k. For the year to December 2022, Matthew's total compensation was 4% salary and 96% other compensation. This indicates that these sales could comprise a meaningful part of their income for the year. Since September 2025, Matthew's direct individual holding has increased from 77.77k shares to 125.11k. Company insiders have collectively sold US$23m more than they bought, via options and on-market transactions in the last 12 months. Reported Earnings • May 11
First quarter 2026 earnings: EPS and revenues exceed analyst expectations First quarter 2026 results: US$0.034 loss per share (down from US$11.09 profit in 1Q 2025). Revenue: US$272.6m (down 77% from 1Q 2025). Net loss: US$2.81m (down 100% from profit in 1Q 2025). Revenue exceeded analyst estimates by 21%. Earnings per share (EPS) also surpassed analyst estimates by 94%. Revenue is forecast to grow 16% p.a. on average during the next 3 years, compared to a 22% growth forecast for the Biotechs industry in the US. Over the last 3 years on average, earnings per share has increased by 83% per year but the company’s share price has only increased by 10% per year, which means it is significantly lagging earnings growth. Actualités en direct • May 05
PTC Therapeutics Surges Ahead With Positive Votoplam Data and US$50m Novartis Milestone PTC Therapeutics reported positive 24‑month interim data from the PIVOT-HD long-term extension study of votoplam in Stage 2 Huntington’s disease.
Patients on the 10 mg dose of votoplam showed a 52% reduction in disease progression vs natural history controls, while the 5 mg dose showed a 28% reduction, with a favorable safety and biomarker profile.
The data triggered a US$50m milestone payment as Novartis advanced votoplam into the global Phase 3 INVEST-HD trial, and Raymond James initiated coverage with an Outperform rating and a US$108 price target.
For investors, the key takeaway is that votoplam has moved further into late-stage development, with a large Phase 3 program in early-stage Huntington’s disease now underway. The reported dose-dependent effect on disease progression and the absence of new safety concerns, including no treatment-related neurofilament light chain spikes, highlight both the potential clinical relevance and the current risk profile seen in this dataset.
The US$50m milestone from Novartis and new analyst coverage following the data underscore how central votoplam appears to PTC Therapeutics’ pipeline, alongside the company’s launch of Sephience for phenylketonuria management. The planned April 28, 2026 conference call is expected to offer more detail on the PIVOT-HD results, the trial design for INVEST-HD, and management’s views on future development and commercialization paths. Major Estimate Revision • May 04
Consensus EPS estimates upgraded to US$0.76 loss The consensus outlook for fiscal year 2026 has been updated. 2026 losses forecast to reduce from -US$0.843 to -US$0.756 per share. Revenue forecast steady at US$990.8m. Biotechs industry in the US expected to see average net income decline 13% next year. Consensus price target broadly unchanged at US$88.07. Share price fell 5.9% to US$65.15 over the past week. Annonce • Apr 29
PTC Therapeutics Reports Positive Topline Results from Month 24 Interim Analysis of PIVOT-HD Extension Study of Votoplam PTC Therapeutics, Inc. reported positive topline results from the 24-month interim analysis of the PIVOT-HD long-term extension study, with favorable dose-dependent effects on disease progression for Stage 2 Huntington's disease (HD) patients following 24 months of votoplam treatment compared to an external natural history cohort. The PIVOT-HD study was a 12-month placebo-controlled study of two dose levels of votoplam in participants with Stage 2 and Stage 3 HD. The study met the primary endpoint of blood Huntingtin (HTT) protein lowering at 12 weeks, with persistent dose-dependent lowering at Month 12. PIVOT-HD participants then enrolled in the PIVOT-HD extension study in which those originally randomized to receive 5 mg or 10 mg of votoplam remained on those dose levels. Participants initially randomized to receive placebo were randomized to receive 5 mg or 10 mg. All participants and investigators remain blinded to initial PIVOT-HD treatment assignment. The objectives of the long-term extension study are to assess the safety and efficacy of long-term votoplam treatment. In the interim analysis following 24 months of votoplam treatment, there was evidence of dose-dependent benefit in slowing progression on the Composite Unified Huntington's Disease Rating Scale (cUHDRS) relative to a propensity weighted natural history cohort in Stage 2 participants, with 52% and 28% slowing for 10 mg and 5 mg participants, respectively. Signals of favorable treatment effects relative to natural history were recorded across the cUHDRS subscales for the 10 mg cohort. In addition, there were no treatment-related neurofilament light chain protein (NfL) increases and mean NfL levels remained below baseline at 24 months for both high and low dose cohorts – in contrast to the reported natural history that NfL levels increase over time in individuals with HD. In Stage 3 participants, potential signals of slowing of progression were observed at 24 months. The safety data at Month 24 for both dose levels and both stages were consistent with the previously established evidence of favorable safety. Novartis announced their initiation of the global Phase 3 INVEST-HD study on their First Quarter Earnings Call. This placebo-controlled study will enroll approximately 770 individuals with early-stage HD who will be randomized 3:2 to receive votoplam 10 mg or placebo. The primary endpoint will be the change from baseline up to month 36 in the cUHDRS. The INVEST-HD study is sponsored and funded by Novartis. Novartis and PTC will continue to review the data and discuss potential next steps including regulatory interactions. PIVOT-HD was designed as a 12-month placebo-controlled trial to assess pharmacodynamic effect and safety of votoplam at two dose levels, 5 mg and 10 mg, relative to placebo. Initially, the study included only Stage 2 patients. A Stage 3 cohort of similar size was subsequently added to help identify the best study population for future studies. The primary endpoints of PIVOT-HD were total blood Huntingtin (HTT) lowering at 12 weeks and safety events. Secondary endpoints included 12-month blood HTT levels, and other blood-and central nervous system (CNS) biomarkers as well as changes in Composite Unified Huntington's Disease Rating Scale (cUHDRS). Following 12 months, patients were eligible to enroll in a long-term extension study in which all subjects would receive votoplam. Those originally randomized to 5 mg and 10 mg would continue at that dose level; those initially randomized to placebo would be randomized 1:1 to 5 mg or 10 mg. All subjects and investigators remain blinded to initial treatment assignment. Votoplam (formerly PTC518) is a small molecule splicing modifier that acts via a unique mechanism to promote the inclusion of a novel pseudoexon containing a premature termination codon, thus triggering Huntingtin (HTT) mRNA degradation and subsequent reduction in HTT protein levels. Votoplam was discovered from PTC's validated splicing platform, following the successful discovery and development of Evrysdi (risdiplam) for spinal muscular atrophy (SMA). Votoplam was partnered with Novartis in December 2024. Following the completion of the PIVOT-HD clinical trial, Novartis assumed responsibility for votoplam's development, manufacturing and commercialization. Huntington's disease (HD) is a fatal, hereditary, genetic disorder of the central nervous system. It is caused by a defective gene. This gene produces a protein, called Huntingtin (HTT), which is involved in the functioning of the nerve cells in the brain (neurons). When the gene is defective, it produces an abnormal (or mutated) HTT protein that is toxic and causes neuron damage and neuron death. HD usually presents in people who are in their 30s or 40s. Symptoms can present earlier in life, and this is called Juvenile HD. There are also cases of infantile HD, when symptoms develop in children who are younger than 10 years old. While symptoms vary from person to person, the disease primarily affects the brain and results in abnormal movements, difficulties with speech, swallowing and walking, as well as a number of other symptoms including behavioral, cognitive and motor symptoms. While there are therapies approved for specific disease symptoms, currently, there is no cure for HD and there are no approved drugs that delay the onset or slow disease progression. Major Estimate Revision • Apr 24
Consensus EPS estimates fall by 15% The consensus outlook for fiscal year 2026 has been updated. 2026 expected loss increased from -US$0.692 to -US$0.798 per share. Revenue forecast unchanged at US$988.7m. Biotechs industry in the US expected to see average net income decline 12% next year. Consensus price target broadly unchanged at US$87.93. Share price fell 5.8% to US$68.23 over the past week. Annonce • Apr 22
PTC Therapeutics, Inc. to Report Q1, 2026 Results on May 07, 2026 PTC Therapeutics, Inc. announced that they will report Q1, 2026 results at 4:00 PM, US Eastern Standard Time on May 07, 2026 Annonce • Apr 21
PTC Therapeutics, Inc., Annual General Meeting, Jun 02, 2026 PTC Therapeutics, Inc., Annual General Meeting, Jun 02, 2026. Major Estimate Revision • Apr 15
Consensus estimates of losses per share improve by 15% The consensus outlook for earnings per share (EPS) in fiscal year 2026 has improved. 2026 revenue forecast increased from US$947.5m to US$984.3m. EPS estimate increased from -US$0.813 per share to -US$0.692 per share. Biotechs industry in the US expected to see average net income decline 12% next year. Consensus price target of US$87.87 unchanged from last update. Share price rose 3.2% to US$73.57 over the past week. Recent Insider Transactions Derivative • Apr 07
Chief Technical Operations Officer notifies of intention to sell stock Neil Almstead intends to sell 52k shares in the next 90 days after lodging an Intent To Sell Form on the 1st of April. If the sale is conducted around the recent share price of US$69.04, it would amount to US$3.6m. Since June 2025, Neil's direct individual holding has increased from 57.91k shares to 67.71k. Company insiders have collectively sold US$23m more than they bought, via options and on-market transactions in the last 12 months. Board Change • Apr 01
Insufficient new directors There is 1 new director who has joined the board in the last 3 years. The company's board is composed of: 1 new director. 5 experienced directors. 5 highly experienced directors. Director Jessica Chutter was the last director to join the board, commencing their role in 2026. The company’s insufficient board refreshment is considered a risk according to the Simply Wall St Risk Model. Annonce • Mar 25
PTC Therapeutics, Inc. Appoints Jessica Chutter to Board of Directors PTC Therapeutics, Inc. announced the appointment of Jessica Chutter to its Board of Directors. Ms. Chutter is a highly accomplished biotechnology investment banker with an extensive list of achievements over her long tenure at Morgan Stanley, including building and leading the bank's biotechnology franchise. Ms. Chutter recently retired as Managing Director and Chair of Biotechnology Investment Banking of Morgan Stanley after a distinguished career spanning over 40 years. She helped build the biotechnology banking practice at Morgan Stanley and is widely considered a pioneer in establishing the field of biotechnology investment banking. During her tenure at Morgan Stanley, Ms. Chutter was involved in approximately $80 billion of capital raising and $85 billion of strategic transactions. Ms. Chutter earned a BA in Commerce/Economics from McGill University and an MBA from Harvard University. Recent Insider Transactions Derivative • Mar 08
Executive VP & Chief Medical Officer notifies of intention to sell stock Lee Golden intends to sell 10k shares in the next 90 days after lodging an Intent To Sell Form on the 5th of March. If the sale is conducted around the recent share price of US$63.38, it would amount to US$634k. Since June 2025, Lee's direct individual holding has increased from 26.64k shares to 52.38k. Company insiders have collectively sold US$25m more than they bought, via options and on-market transactions in the last 12 months. Major Estimate Revision • Feb 26
Consensus EPS estimates upgraded to US$0.60 loss The consensus outlook for fiscal year 2026 has been updated. 2026 losses forecast to reduce from -US$1.00 to -US$0.596 per share. Revenue forecast steady at US$986.0m. Biotechs industry in the US expected to see average net income decline 7.9% next year. Consensus price target broadly unchanged at US$87.57. Share price was steady at US$67.76 over the past week. Recent Insider Transactions • Feb 24
CEO & Director recently sold US$511k worth of stock On the 18th of February, Matthew Klein sold around 7k shares on-market at roughly US$69.36 per share. This transaction amounted to 5.7% of their direct individual holding at the time of the trade. In the last 3 months, they made an even bigger sale worth US$630k. Matthew has been a net seller over the last 12 months, reducing personal holdings by US$2.9m. New Risk • Feb 22
New major risk - Revenue and earnings growth Earnings are forecast to decline by an average of 9.4% per year for the foreseeable future. This is considered a major risk. Ultimately, shareholders want to see a good return on their investment and that generally comes from sharing in the company's profits. If profits are expected to decline, then in most cases the share price will decline over time as well. In addition, if the company pays dividends it will also likely need to reduce or cut them, striking a dual blow to total shareholder returns. Currently, the following risks have been identified for the company: Major Risks Negative equity (-US$205m). Earnings are forecast to decline by an average of 9.4% per year for the foreseeable future. High level of non-cash earnings (97% accrual ratio). Minor Risks Significant insider selling over the past 3 months (US$3.3m sold). Recent Insider Transactions Derivative • Feb 20
CEO & Director notifies of intention to sell stock Matthew Klein intends to sell 7k shares in the next 90 days after lodging an Intent To Sell Form on the 18th of February. If the sale is conducted around the recent share price of US$69.36, it would amount to US$511k. For the year to December 2022, Matthew's total compensation was 12% salary and 88% other compensation. This indicates that these sales could comprise a meaningful part of their income for the year. Since March 2025, Matthew's direct individual holding has increased from 59.54k shares to 106.39k. Company insiders have collectively sold US$23m more than they bought, via options and on-market transactions in the last 12 months. Reported Earnings • Feb 20
Full year 2025 earnings: EPS and revenues miss analyst expectations Full year 2025 results: EPS: US$8.58 (up from US$4.73 loss in FY 2024). Revenue: US$1.73b (up 115% from FY 2024). Net income: US$682.6m (up US$1.05b from FY 2024). Profit margin: 39% (up from net loss in FY 2024). The move to profitability was primarily driven by higher revenue. Products in clinical trials Phase II: 2 Phase III: 1 Post-clinical trial products Pre-registration: 1 Approved (during full year): 1 Revenue missed analyst estimates by 6.2%. Earnings per share (EPS) also missed analyst estimates by 19%. Revenue is forecast to grow 1.3% p.a. on average during the next 3 years, compared to a 19% growth forecast for the Biotechs industry in the US. Over the last 3 years on average, earnings per share has increased by 89% per year but the company’s share price has only increased by 18% per year, which means it is significantly lagging earnings growth. Annonce • Feb 13
PTC Therapeutics Provides Regulatory Update on Translarna PTC Therapeutics, Inc. announced that it has withdrawn the New Drug Application (NDA) resubmission for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (DMD) following U.S. Food and Drug Administration (FDA) feedback on the application review. Annonce • Feb 05
PTC Therapeutics, Inc. to Report Q4, 2025 Results on Feb 19, 2026 PTC Therapeutics, Inc. announced that they will report Q4, 2025 results on Feb 19, 2026 Annonce • Jan 13
PTC Therapeutics, Inc. Provides Revenue Guidance for the Full Year 2026 PTC Therapeutics, Inc. provided revenue guidance for the full year 2026. For the full year 2026, PTC anticipates total product revenue of $700 million to $800 million, excluding Evrysdi royalty revenue and collaboration revenue. Recent Insider Transactions • Jan 12
CEO & Director recently sold US$630k worth of stock On the 7th of January, Matthew Klein sold around 8k shares on-market at roughly US$77.15 per share. This transaction amounted to 7.0% of their direct individual holding at the time of the trade. This was the largest sale by an insider in the last 3 months. Matthew has been a net seller over the last 12 months, reducing personal holdings by US$2.6m. Recent Insider Transactions • Jan 04
CEO & Director recently sold US$620k worth of stock On the 30th of December, Matthew Klein sold around 8k shares on-market at roughly US$76.64 per share. This transaction amounted to 8.0% of their direct individual holding at the time of the trade. This was the largest sale by an insider in the last 3 months. Matthew has been a net seller over the last 12 months, reducing personal holdings by US$2.2m. Recent Insider Transactions Derivative • Dec 31
CEO & Director notifies of intention to sell stock Matthew Klein intends to sell 8k shares in the next 90 days after lodging an Intent To Sell Form on the 30th of December. If the sale is conducted around the recent share price of US$76.64, it would amount to US$620k. For the year to December 2022, Matthew's total compensation was 12% salary and 88% other compensation. This indicates that these sales could comprise a meaningful part of their income for the year. Since December 2024, Matthew's direct individual holding has increased from 58.28k shares to 82.15k. Company insiders have collectively sold US$21m more than they bought, via options and on-market transactions in the last 12 months. Annonce • Dec 24
PTC Therapeutics Announces Approval of Sephience™? (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria (PKU) in Japan PTC Therapeutics, Inc. announced that Sephience™ (sepiapterin) has been approved by the Japanese Ministry of Health, Labor and Welfare (MHLW) for the treatment of children and adults living with phenylketonuria (PKU). The label includes individuals of all ages and the full spectrum of disease severity. PTC will now engage in pricing discussions which are expected to conclude in First Quarter 2026, with launch occurring shortly thereafter. Sephience™ is indicated for the treatment of adult and pediatric patients with phenylketonuria (PKU). Sephience is a natural precursor of the enzymatic co-factor BH4, a critical co-factor for phenylalanine hydroxylase (PAH). Through its mechanism of action, Sephience is able to effectively reduce blood phenylalanine (Phe) levels and has the potential to treat a broad range of PKU patients. Sephience is approved in the European Union /European Economic Area region, the United States and Japan. Recent Insider Transactions Derivative • Dec 19
Chief Business Officer notifies of intention to sell stock Eric Pauwels intends to sell 40k shares in the next 90 days after lodging an Intent To Sell Form on the 17th of December. If the sale is conducted around the recent share price of US$75.42, it would amount to US$3.0m. Since March 2025, Eric's direct individual holding has increased from 19.79k shares to 20.06k. Company insiders have collectively sold US$21m more than they bought, via options and on-market transactions in the last 12 months. Annonce • Dec 11
PTC Therapeutics Announces Health Canada Approval of Sephience™ (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria (PKU) PTC Therapeutics Canada ULC announced that Health Canada has approved Sephience™? (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The approval includes broad labeling for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month old and older with sepiapterin-responsive PKU. The Health Canada approval is based on the evidence of significant efficacy and safety from the Phase 3 APHENITY trial as well as durability of treatment effect in the APHENITY long-term extension study. Sephience is now commercially available in Canada. Sephience was recently approved by the U.S. Food and Drug Administration and granted marketing authorization by the European Commission. Recent Insider Transactions Derivative • Nov 13
Independent Director notifies of intention to sell stock Emma Reeve intends to sell 7k shares in the next 90 days after lodging an Intent To Sell Form on the 12th of November. If the sale is conducted around the recent share price of US$74.50, it would amount to US$546k. Since March 2025, Emma's direct individual holding has decreased from 8.53k shares to 4.33k. Company insiders have collectively sold US$14m more than they bought, via options and on-market transactions in the last 12 months. Reported Earnings • Nov 06
Third quarter 2025 earnings: EPS and revenues exceed analyst expectations Third quarter 2025 results: EPS: US$0.20 (up from US$1.39 loss in 3Q 2024). Revenue: US$211.0m (up 7.2% from 3Q 2024). Net income: US$15.9m (up US$122.6m from 3Q 2024). Profit margin: 7.5% (up from net loss in 3Q 2024). The move to profitability was primarily driven by lower expenses. Revenue exceeded analyst estimates by 20%. Earnings per share (EPS) also surpassed analyst estimates. Revenue is expected to decline by 6.8% p.a. on average during the next 3 years, while revenues in the Biotechs industry in the US are expected to grow by 21%. Over the last 3 years on average, earnings per share has increased by 76% per year but the company’s share price has only increased by 24% per year, which means it is significantly lagging earnings growth. Price Target Changed • Nov 05
Price target increased by 14% to US$76.27 Up from US$67.00, the current price target is an average from 15 analysts. New target price is 6.3% above last closing price of US$71.77. Stock is up 66% over the past year. The company is forecast to post earnings per share of US$9.23 next year compared to a net loss per share of US$4.73 last year. Annonce • Nov 05
PTC Therapeutics, Inc. Narrows Revenue Guidance for the Year 2025 PTC Therapeutics, Inc. narrowed revenue guidance for the year 2025. Total revenue of $750 to $800 million, which includes in-line products and royalty revenue from Evrysdi. Recent Insider Transactions Derivative • Oct 31
Independent Director notifies of intention to sell stock Emma Reeve intends to sell 16k shares in the next 90 days after lodging an Intent To Sell Form on the 30th of October. If the sale is conducted around the recent share price of US$69.50, it would amount to US$1.1m. Since March 2025, Emma's direct individual holding has decreased from 8.53k shares to 8.00k. Company insiders have collectively sold US$13m more than they bought, via options and on-market transactions in the last 12 months. Recent Insider Transactions Derivative • Oct 05
Chief Technical Operations Officer notifies of intention to sell stock Neil Almstead intends to sell 55k shares in the next 90 days after lodging an Intent To Sell Form on the 3rd of October. If the sale is conducted around the recent share price of US$66.05, it would amount to US$3.6m. Since December 2024, Neil's direct individual holding has increased from 41.34k shares to 57.91k. Company insiders have collectively sold US$9.5m more than they bought, via options and on-market transactions in the last 12 months. Recent Insider Transactions Derivative • Sep 10
Chief Business Officer notifies of intention to sell stock Eric Pauwels intends to sell 40k shares in the next 90 days after lodging an Intent To Sell Form on the 8th of September. If the sale is conducted around the recent share price of US$56.92, it would amount to US$2.3m. Since March 2025, Eric has owned 19.79k shares directly. Company insiders have collectively sold US$6.4m more than they bought, via options and on-market transactions in the last 12 months. Valuation Update With 7 Day Price Move • Sep 04
Investor sentiment improves as stock rises 17% After last week's 17% share price gain to US$58.01, the stock trades at a forward P/E ratio of 24x. Average trailing P/E is 16x in the Biotechs industry in the US. Total returns to shareholders of 7.7% over the past three years. Annonce • Aug 20
PTC Therapeutics, Inc. Receives Complete Response Letter for Vatiquinone NDA PTC Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) related to the New Drug Application (NDA) for vatiquinone for the treatment of children and adults living with Friedreich's ataxia. The FDA stated in the CRL that substantial evidence of efficacy was not demonstrated for vatiquinone and that an additional adequate and well-controlled study would be needed to support NDA resubmission. Vatiquinone has been evaluated in a number of clinical studies, many focused on pediatric patients, and has demonstrated an impact on mortality risk, and a number of neurological and neuromuscular disease symptoms. Reported Earnings • Aug 08
Second quarter 2025 earnings: EPS and revenues exceed analyst expectations Second quarter 2025 results: US$0.83 loss per share (improved from US$1.29 loss in 2Q 2024). Revenue: US$178.9m (down 4.2% from 2Q 2024). Net loss: US$64.8m (loss narrowed 35% from 2Q 2024). Revenue exceeded analyst estimates by 2.8%. Earnings per share (EPS) also surpassed analyst estimates by 19%. Revenue is expected to decline by 4.9% p.a. on average during the next 3 years, while revenues in the Biotechs industry in the US are expected to grow by 19%. Over the last 3 years on average, earnings per share has increased by 57% per year but the company’s share price has fallen by 5% per year, which means it is significantly lagging earnings. Recent Insider Transactions • Aug 07
CEO & Director recently sold US$556k worth of stock On the 5th of August, Matthew Klein sold around 11k shares on-market at roughly US$51.74 per share. This transaction amounted to 12% of their direct individual holding at the time of the trade. This was the largest sale by an insider in the last 3 months. Matthew has been a net seller over the last 12 months, reducing personal holdings by US$1.7m. Recent Insider Transactions Derivative • Aug 06
CEO & Director notifies of intention to sell stock Matthew Klein intends to sell 11k shares in the next 90 days after lodging an Intent To Sell Form on the 5th of August. If the sale is conducted around the recent share price of US$51.74, it would amount to US$556k. For the year to December 2022, Matthew's total compensation was 12% salary and 88% other compensation. This indicates that these sales could comprise a meaningful part of their income for the year. Since December 2024, Matthew's direct individual holding has increased from 58.28k shares to 63.23k. Company insiders have collectively sold US$5.7m more than they bought, via options and on-market transactions in the last 12 months. Valuation Update With 7 Day Price Move • Aug 01
Investor sentiment improves as stock rises 17% After last week's 17% share price gain to US$52.13, the stock trades at a forward P/E ratio of 10x. Average trailing P/E is 16x in the Biotechs industry in the US. Total loss to shareholders of 2.6% over the past three years. Major Estimate Revision • Jul 29
Consensus EPS estimates increase by 24% The consensus outlook for earnings per share (EPS) in fiscal year 2025 has improved. 2025 revenue forecast increased from US$1.63b to US$1.75b. EPS estimate increased from US$6.10 to US$7.55 per share. Net income forecast to shrink 28% next year vs 12% decline forecast for Biotechs industry in the US. Consensus price target up from US$62.81 to US$67.38. Share price rose 6.3% to US$49.29 over the past week. Annonce • Jul 29
PTC Therapeutics, Inc. Announces FDA Approval of Sephience™? (Sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria (PKU) PTC Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved SEPHIENCE™? (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The approval includes broad labeling for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive PKU. The FDA approval is based on the evidence of significant efficacy and safety from the Phase 3 APHENITY trial as well as durability of treatment effect in the APHENITY long-term extension study. SEPHIENCE and PDE-5 inhibitors (e.g., sildenafil, vardenafil, or tadalafil) induce vasorelaxation and may reduce blood pressure. Monitor for signs and symptoms of hypotension. Board Change • Jul 01
Insufficient new directors There is 1 new director who has joined the board in the last 3 years. The company's board is composed of: 1 new director. 4 experienced directors. 5 highly experienced directors. CEO & Director Matt Klein was the last director to join the board, commencing their role in 2023. The company’s insufficient board refreshment is considered a risk according to the Simply Wall St Risk Model. New Risk • Jun 25
New major risk - Revenue and earnings growth Earnings are forecast to decline by an average of 44% per year for the foreseeable future. This is considered a major risk. Ultimately, shareholders want to see a good return on their investment and that generally comes from sharing in the company's profits. If profits are expected to decline, then in most cases the share price will decline over time as well. In addition, if the company pays dividends it will also likely need to reduce or cut them, striking a dual blow to total shareholder returns. Currently, the following risks have been identified for the company: Major Risks Negative equity (-US$186m). Earnings are forecast to decline by an average of 44% per year for the foreseeable future. Minor Risk Annonce • Jun 24
PTC Therapeutics, Inc. Receives Marketing Authorization by the European Commission for the Treatment of Children and Adults Living with Phenylketonuria PTC Therapeutics, Inc. announced that Sephience (sepiapterin) was granted marketing authorization by the European Commission for the treatment of children and adults living with phenylketonuria (PKU). The authorization includes a broad label inclusive of all ages and disease severities. The European approval is based on the highly statistically significant results from the Phase 3 APHENITY trial as well as evidence of durable treatment effect and the ability of study participants to optimize their diet in the APHENITY long-term extension study. The marketing authorization is applicable to all 27 European Union member states as well as Iceland, Norway and Liechtenstein. The Sephience European launch will be initiated in Germany in the first half of July. A New Drug Application (NDA) for sepiapterin remains on schedule for its FDA target action date of July 29, 2025. Review of approval applications is ongoing in several other countries including Japan and Brazil. Valuation Update With 7 Day Price Move • May 13
Investor sentiment improves as stock rises 28% After last week's 28% share price gain to US$46.39, the stock trades at a forward P/E ratio of 14x. Average trailing P/E is 17x in the Biotechs industry in the US. Total returns to shareholders of 46% over the past three years. Major Estimate Revision • May 13
Consensus revenue estimates increase by 69% The consensus outlook for revenues in fiscal year 2025 has improved. 2025 revenue forecast increased from US$884.9m to US$1.50b. Now expected to report a profit of US$5.36 instead of losses of -US$1.17 per share. Biotechs industry in the US expected to see average net income decline 13% next year. Consensus price target down from US$64.79 to US$61.13. Share price rose 13% to US$46.01 over the past week. New Risk • May 07
New major risk - Financial position The company's debt is not well covered by operating cash flow. Currently running at an operating cash loss. This is considered a major risk. If the company's operating cash flows are too small relative to the size of their debt, it increases their balance sheet risk. The company has less cash from operations to cover its expenses from servicing large debt and it increases the risk of liquidity issues. It also extends the time it would take for the company to pay back the debt in full, meaning it may not be able to easily pay it all off in a distress scenario. Currently, the following risks have been identified for the company: Major Risks Debt is not well covered by operating cash flow (currently running at an operating cash loss). Negative equity (-US$186m). Earnings are forecast to decline by an average of 54% per year for the foreseeable future. Minor Risk Significant insider selling over the past 3 months (US$1.6m sold). Reported Earnings • May 07
First quarter 2025 earnings: EPS and revenues exceed analyst expectations First quarter 2025 results: EPS: US$11.09 (up from US$1.20 loss in 1Q 2024). Revenue: US$1.18b (up 460% from 1Q 2024). Net income: US$866.6m (up US$958.1m from 1Q 2024). Profit margin: 74% (up from net loss in 1Q 2024). The move to profitability was driven by higher revenue. Revenue exceeded analyst estimates significantly. Earnings per share (EPS) also surpassed analyst estimates significantly. Revenue is expected to decline by 6.1% p.a. on average during the next 3 years, while revenues in the Biotechs industry in the US are expected to grow by 18%. Over the last 3 years on average, earnings per share has increased by 35% per year but the company’s share price has only increased by 10% per year, which means it is significantly lagging earnings growth. Annonce • May 07
PTC Therapeutics, Inc. Provides Revenue Guidance for the Year 2025 PTC Therapeutics, Inc. provided revenue guidance for the year 2025. For the year, the company expects revenues for the full-year 2025 to be between $650 million and $800 million, which includes in-line products, potential new product launches, and royalty revenue from Evrysdi. Annonce • May 06
PTC Therapeutics, Inc. Announces Results from the Phase 2 PIVOT-HD Study of PTC518 (Votoplam) in Stage 2 and Stage 3 Huntington's Disease Patients PTC Therapeutics, Inc. announced results from the Phase 2 PIVOT-HD study of PTC518 (votoplam) in Stage 2 and Stage 3 Huntington's disease (HD) patients. The study met its primary endpoint of reduction in blood Huntingtin (HTT) protein levels (pIVOT-HD was designed as a 12-month placebo-controlled trial to assess pharmacodynamic effect and safety of PTC518 at two dose levels--5mg and 10mg, relative to placebo. Those originally randomized to 5mg and 10mg would continue at that dose level; those initially randomized to 5mg or 10mg. All subjects and investigators remain blinded to initial treatment assignment. Presentation of study results is expected at a scientific meeting later in the year. PTC518 is a small molecule splicing modifier that acts via a unique mechanism to promote the inclusion of a novel pseudoexon containing a premature termination codon, thus triggering Huntingtin (HTT) mRNA degradation and subsequent reduction in HTT protein levels. PTC518 was discovered from PTC's validated splicing platform, following the successful discovery and development of Evrysdi (risdiplam) for spinal muscular atrophy (SMA). Annonce • May 01
PTC Therapeutics, Inc., Annual General Meeting, Jun 17, 2025 PTC Therapeutics, Inc., Annual General Meeting, Jun 17, 2025. Annonce • Apr 25
PTC Therapeutics Receives Positive CHMP Opinion for Sephience™ (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria (PKU) PTC Therapeutics, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion on the marketing authorization application for Sephience™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The opinion includes a broad label inclusive of all ages and disease severities. Launch planning in Europe is well underway with priorities in Germany and other key European markets where named patient access will be immediately available. PTC expects the European Commission to ratify the marketing authorization for Sephience in approximately two months. The decision will be applicable to all 27 European Union member states, as well as Iceland, Norway and Liechtenstein. The New Drug Application (NDA) for sepiapterin is under review by the U.S. Food and Drug Administration (FDA) with a Prescription Drug User Fee Act (PDUFA) target action date of July 29, 2025. Review of approval applications is ongoing in several other countries including Japan and Brazil. Major Estimate Revision • Mar 30
Consensus EPS estimates upgraded to US$0.77 loss, revenue downgraded The consensus outlook for fiscal year 2025 has been updated. 2025 revenue forecast fell from US$955.4m to US$942.3m. 2025 losses expected to reduce from -US$1.07 to -US$0.77 per share. Biotechs industry in the US expected to see average net income decline 12% next year. Consensus price target of US$64.71 unchanged from last update. Share price fell 5.1% to US$54.24 over the past week. Annonce • Mar 29
PTC Therapeutics, Inc. Provides Regulatory Update on Translarna (Aaluren) in Europe PTC Therapeutics, Inc. announced that the European Commission (EC) has adopted the opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) to not renew the authorization of Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy. While this action effectively removes the drug's conditional marketing authorization in the European Economic Area, the EC indicated that individual countries within the European Union can leverage Articles 117(3) and 5(1) of the EU Directive 2001/83 to allow continued use of Translarna. Translarna (ataluren), discovered and developed by PTC Therapeutics, is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne. Translarna, the tradename of ataluren, is licensed in multiple countries for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged 2 years and older. Ataluren is an investigational new drug in the United States. Annonce • Mar 20
PTC Therapeutics Presents New Sepiapterin Data from Ongoing Studies PTC Therapeutics, Inc. shared new data being presented from the Phase 3 APHENITY trial and subsequent open-label extension study at the 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting. These data provide further evidence of the potential meaningful benefits of sepiapterin treatment for the full spectrum of phenylketonuria (PKU) patients, including significant diet liberalization. Key data presented include: Over 97% of subjects participating in the Phe tolerance protocol of the APHENITY open-label extension study demonstrate the ability to liberalize their diet while on sepiapterin treatment, with a mean increase in protein intake of 126%. 66% of subjects participating in the Phe tolerance sub study reached or exceeded the age-adjusted recommended daily allowance of protein intake for an individual without PKU, while maintaining control of blood Phe levels. Genetic variant analysis of subjects participating in the APHENITY study demonstrates that over 70% had a Genotype-Phenotype Value (GPV) consistent with classical PKU. Major Estimate Revision • Mar 16
Consensus EPS estimates fall by 39% The consensus outlook for fiscal year 2025 has been updated. 2025 expected loss increased from -US$0.766 to -US$1.07 per share. Revenue forecast unchanged at US$955.3m. Biotechs industry in the US expected to see average net income decline 13% next year. Consensus price target up from US$63.21 to US$64.93. Share price rose 3.6% to US$54.50 over the past week. 
