Here are 1-2 brief analogies for PTC Therapeutics (PTCT):

• Like Vertex Pharmaceuticals, but focused on developing treatments for rare genetic diseases such as Duchenne muscular dystrophy.
• A BioMarin Pharmaceutical specializing in medicines for a range of rare genetic and neurological disorders.

• Translarna (ataluren): A therapy for nonsense mutation Duchenne muscular dystrophy (DMD) patients aged two years and older.
• Emflaza (deflazacort): A corticosteroid indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients aged two years and older.
• Upstaza (eladocagene exuparvovec): A gene therapy for the treatment of Aromatic L-amino acid decarboxylase (AADC) deficiency.
• Tegsedi (inotersen): An antisense oligonucleotide indicated for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients with stage 1 or stage 2 polyneuropathy.
• Waylivra (volanesorsen): An antisense oligonucleotide indicated for the treatment of familial chylomicronemia syndrome (FCS) in adult patients with genetically confirmed FCS and high risk for pancreatitis, in addition to diet.

PTC Therapeutics (symbol: PTCT) primarily sells its pharmaceutical products to other companies, specifically major wholesale pharmaceutical distributors. These distributors then supply pharmacies, hospitals, and other healthcare providers.

The major customer companies for PTC Therapeutics, based on their financial disclosures, are:

• Cencora (formerly AmerisourceBergen Corporation) (Symbol: COR)
• Cardinal Health, Inc. (Symbol: CAH)
• McKesson Corporation (Symbol: MCK)

• Thermo Fisher Scientific Inc. (TMO)
• Lonza Group AG (LONN)
• Rentschler Biopharma SE

Matthew B. Klein, M.D. Chief Executive Officer

Dr. Klein has served as Chief Executive Officer and Director of PTC Therapeutics since March 2023. Prior to this role, he held positions at PTC Therapeutics as Chief Operating Officer, Chief Development Officer, and Global Head, Gene and Mitochondrial Therapies. Before joining PTC in 2019, Dr. Klein was the Chief Executive Officer and Chief Medical Officer of BioElectron Technology Corporation, a biotechnology company whose assets were acquired by PTC in 2019. At BioElectron, he led the development of two small molecules for central nervous system diseases and oversaw the formation of an oncology platform. He also served as Chief Medical Officer and Senior Vice President of Edison Pharmaceuticals. Earlier in his career, he was the Auth-Washington Research Chair of Restorative Burn Surgery at the University of Washington.

Pierre Gravier Chief Financial Officer

Mr. Gravier was appointed Chief Financial Officer of PTC Therapeutics in July 2023. He brings over 17 years of experience spanning investment banking, venture capital, and scientific roles. Before joining PTC, Mr. Gravier was a Managing Director in the healthcare group of Perella Weinberg Partners, where he advised biopharmaceutical and pharmaceutical companies on finance strategy and corporate development. He previously worked as a healthcare investment banker at Barclays Capital and as a Venture Capital Analyst at Société Générale Asset Management, focusing on early-stage biotechnology investments. He began his career as a Scientist at Ferring Pharmaceuticals.

Eric Pauwels Chief Business Officer

Mr. Pauwels became Chief Business Officer of PTC Therapeutics in 2020, having joined the company in March 2015. He possesses over 40 years of experience in healthcare, including launching more than 30 biopharmaceutical products globally, with a particular focus on rare diseases over the last 18 years. Prior to PTC, he served as Chief Commercial Officer and President of International for NPS Pharmaceuticals from 2011 to 2015, where he established global commercial operations for the launches of Gattex and Natpara. From 2005 to 2010, Mr. Pauwels was the first Chief Commercial Officer for Shire Human Genetic Therapies Rare Disease Business, leading the global expansion of its operations following the acquisition of TKT. His background also includes management roles at Bayer Healthcare Pharmaceuticals, Fournier Pharma, and Johnson & Johnson.

Christine Utter Senior Vice President, Chief Accounting Officer and Head of People Services

Ms. Utter has held the role of Senior Vice President, Chief Accounting Officer, and Head of People Services at PTC Therapeutics since 2019. Her tenure at PTC also includes serving as the company's Assistant Controller and Principal Financial Officer. Before joining PTC, she was the Assistant Corporate Controller at Barrier Therapeutics and a Financial Analyst at Engelhard Corp.

Neil Almstead, Ph.D. Chief Technical Operations Officer

Dr. Almstead serves as the Chief Technical Operations Officer at PTC Therapeutics.

1. Regulatory Challenges and Financial Dependence on Key Products: PTC Therapeutics faces significant business risk due to regulatory challenges, particularly concerning the marketing authorization of its key products. The company's financial health is heavily dependent on the commercial success and continued regulatory approvals for products like Translarna (ataluren) and Sephience. There is a risk of non-renewal of Translarna's marketing authorization in the European Economic Area (EEA), which could jeopardize revenue streams. Negative opinions from regulatory bodies, such as the Committee for Medicinal Products for Human Use (CHMP) regarding Translarna, highlight these ongoing regulatory hurdles.
2. Limited Product Diversification and Pipeline Launch Risk: The company is heavily reliant on revenue from a small number of products, primarily Translarna and Sephience. This concentration creates vulnerability to setbacks in a single product line, including issues with international reimbursements, regulatory approval, and market acceptance. While PTC Therapeutics has a pipeline of potential new therapies, its future growth is significantly dependent on the successful and often risky launches of these pipeline products.
3. Market Volatility and Intense Competition: PTC Therapeutics operates within the highly competitive and rapidly evolving biopharmaceutical industry. The company must constantly navigate market volatility and intense competition, requiring continuous strategic investments in research and development (R&D) and marketing to maintain its market position and stay ahead of competitors.

The emergence of gene therapies for Duchenne muscular dystrophy (DMD) represents a clear emerging threat. Specifically, Sarepta Therapeutics' Elevidys (delandistrogene moxeparvovec), which received accelerated approval from the FDA, offers a novel gene therapy approach to address the underlying genetic cause of DMD. This potentially disease-modifying treatment competes directly with PTC Therapeutics' existing DMD portfolio, including Translarna (ataluren) for nonsense mutation DMD and Emflaza (deflazacort) for all DMD patients. Gene therapy offers a fundamentally different and potentially more impactful treatment modality compared to PTC's small molecule and corticosteroid therapies, potentially eroding market share and long-term viability for these products by offering a superior and more comprehensive therapeutic option.

Below are the addressable markets for PTC Therapeutics' main products:

• Translarna (ataluren) and Emflaza (deflazacort) for Duchenne Muscular Dystrophy (DMD):
  • The global Duchenne muscular dystrophy (DMD) treatment market across the seven major markets (7MM: US, France, Germany, Italy, Spain, UK, and Japan) was approximately $2.3 billion in 2023 and is projected to reach $5.2 billion by 2033.
  • The United States is expected to account for 84.8% of the total DMD market share. In 2023, there were approximately 17,200 prevalent cases of DMD in the United States.
  • For Emflaza, PTC estimates an addressable market of approximately 10,000 DMD patients aged five years or older in the United States.
• Upstaza (eladocagene exuparvovec) for Aromatic L-amino acid decarboxylase (AADC) deficiency:
  • AADC deficiency is classified as a "very rare disease." A specific addressable market size in USD for this product is not publicly available.
• Tegsedi (inotersen) for hereditary transthyretin-mediated amyloidosis (hATTR):
  • The global patient population for hATTR ranges from 10,000 to 50,000 people.
  • North America holds the largest share in the Tegsedi market as of 2024. A specific numerical market size in USD is not publicly available in the provided search results.
• Waylivra (volanesorsen) for Familial Chylomicronemia Syndrome (FCS) and hypertriglyceridemia:
  • The Familial Chylomicronemia Syndrome (FCS) market size in the seven major markets (US, EU4, UK, Japan) was approximately $15 million in 2022.
  • Europe was the largest region in the Waylivra market in 2024. A specific global Waylivra market size in USD is not publicly available in the provided search results.
• Evrysdi (risdiplam) for Spinal Muscular Atrophy (SMA):
  • Global sales for Evrysdi were over $1.7 billion in 2024. (PTC Therapeutics receives royalties from these sales, as Roche markets the product).
• Sephience (sepiapterin) for Phenylketonuria (PKU):
  • Sephience generated $19.6 million in revenue in the third quarter of 2025. A specific overall addressable market size in USD for PKU is not publicly available.

PTC Therapeutics (PTCT) is expected to drive future revenue growth over the next 2-3 years through several key areas:

1. Global Launch and Expansion of Sephience: The successful launch of Sephience (sepiapterin), an FDA-approved therapy for phenylketonuria (PKU), is a primary revenue driver. Sephience received a positive CHMP opinion in Europe, anticipating a broad label for all ages and PKU patients. The global launch, initiated in the US and Europe, has shown strong initial uptake, generating $19.6 million in revenue in Q3 2025. Analysts project Sephience sales could reach $500 million by 2026. Further market expansion is anticipated with ongoing marketing authorization reviews, including in Japan, expected in Q4 2025.
2. Continued Contribution from the Duchenne Muscular Dystrophy (DMD) Franchise: PTC's DMD franchise, comprising Translarna and Emflaza, continues to be a significant revenue contributor. Despite the non-renewal of Translarna's conditional marketing authorization in the EU, PTC has secured agreements with several European countries to continue product supply, retaining approximately 25% of prior European revenue for the remainder of 2025. The DMD franchise generated $85.9 million in revenue in Q3 2025.
3. Roche Evrysdi Royalty Revenue: Royalty revenue from Evrysdi (risdiplam), a spinal muscular atrophy (SMA) treatment developed in collaboration with Roche, remains a steady source of income. Roche reported global sales of approximately $559 million in Q2 2025, contributing $58 million in royalty revenue to PTC. Q3 2025 royalty revenue from Evrysdi further increased to $70.8 million.
4. Advancement of PTC518 for Huntington's Disease: The progression of PTC518, an oral therapy for Huntington's disease (HD), through clinical development represents a future growth catalyst. The drug has demonstrated dose-dependent HTT reduction in Phase 2 trials. A significant licensing deal with Novartis, involving a $1 billion upfront payment and potential additional payments up to $1.9 billion tied to milestones, underscores its commercial potential. An FDA meeting for the Huntington's disease program is planned for Q4 2025, with potential approval by 2026 or 2027.
5. Global Commercialization of Upstaza: Upstaza (formerly GT-AADC), the first FDA-approved gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency, is expected to contribute to revenue as PTC continues its global commercialization efforts. Upstaza is approved in both the United States (as Kebilidi) and the European Union.

Share Issuance

• PTC Therapeutics' shares outstanding increased by 1.86% year-over-year to 0.078 billion for the quarter ending June 30, 2025.
• As of November 4, 2025, shares outstanding stood at 80.29 million, representing a 10.56% increase in one year.
• The company raised $35.9 million in a Post IPO funding round on May 16, 2023, and $141 million in another Post IPO round on May 14, 2020.

Inbound Investments

• PTC Therapeutics completed a Post IPO funding round on May 16, 2023, securing $35.9 million.
• An earlier Post IPO funding round on May 14, 2020, raised $141 million.

Outbound Investments

• PTC Therapeutics completed a Later Stage VC deal with ONODERA GT Pharma on October 31, 2022.
• In 2020, the company acquired Censa Pharmaceuticals, Inc., which focuses on developing CNSA-001 (sepiapterin) for orphan metabolic diseases.

Capital Expenditures

• For the last 12 months ending November 4, 2025, PTC Therapeutics reported capital expenditures of -$10.25 million, indicating a net sale of property, plant, and equipment or net divestitures.