PTC Therapeutics, Inc.
- Open
- 69.98
- Day high
- 70.06
- Day low
- 68.75
- Prev close
- 70.09
- Volume
- 841K
- Mkt cap
- $5.8B
- P/E (TTM)
- —
- EPS (TTM)
- —
- P/B
- -35.1
- P/S
- 5.8
- Yield
- —
- Per share
- —
- ▼Insiders net selling -$18.5M over the last 3 months (0 open-market buys, 47 sales)
- 🏛Institutions accumulating (13F)
PTC Therapeutics, Inc. (PTCT) is a Healthcare company listed on NASDAQ. The stock is up 32% over the past year. Over the trailing 3 months, insiders filed 0 open-market buys and 47 sales (SEC Form 4). Drillr has 2 published research articles covering PTCT.
PTC Therapeutics, Inc. (PTCT) financials & analyst ratings
Fundamentals (TTM)
Analyst consensus · 6 analysts
Source: exchange market data + company filings. Figures are trailing-twelve-month or as most recently reported. For informational purposes only — not investment advice.
PTCT earnings date, history & EPS estimates
| Report date | EPS est | EPS actual | Surprise | Revenue | Rev. surprise |
|---|---|---|---|---|---|
| Jul 30, 2026 | $0.24 | $0.92 | +289.3% | $361M | +20.7% |
| May 7, 2026 | $-0.45 | $-0.03 | +93.3% | $273M | +25.4% |
| Feb 19, 2026 | $-0.21 | $-1.67 | -695.2% | $165M | -33.3% |
| Nov 4, 2025 | $-1.19 | $0.20 | +116.8% | $211M | -25.0% |
| Aug 7, 2025 | $-1.07 | $-0.83 | +22.4% | $179M | -6.6% |
| Feb 27, 2025 | $-1.12 | $-0.24 | +78.6% | $213M | -16.0% |
| Nov 7, 2024 | $-1.54 | $-1.39 | +9.7% | $197M | +2.6% |
| Aug 8, 2024 | $-1.00 | $-1.16 | -16.0% | $187M | -1.2% |
| Apr 25, 2024 | $-1.21 | $-1.20 | +0.8% | $210M | +18.9% |
| Feb 29, 2024 | $0.29 | $-0.24 | -182.8% | $307M | -2.5% |
| Oct 26, 2023 | $-0.85 | $-1.76 | -107.1% | $197M | -8.2% |
| Aug 3, 2023 | $-1.58 | $-2.66 | -68.4% | $214M | +2.9% |
PTCT insider trading activity (SEC Form 4)
| Date | Insider | Type | Shares | Price |
|---|---|---|---|---|
| Aug 28, 2026 | SCHMERTZLER MICHAELdirector | Sell | 11,890 | $71.55 |
| Aug 28, 2026 | SCHMERTZLER MICHAELdirector | Sell | 5,626 | $73.18 |
| Aug 28, 2026 | SCHMERTZLER MICHAELdirector | Sell | 610 | $72.22 |
| Aug 28, 2026 | SCHMERTZLER MICHAELdirector | Sell | 6,874 | $72.74 |
| Aug 21, 2026 | SCHMERTZLER MICHAELdirector | Sell | 7,165 | $73.67 |
| Aug 21, 2026 | SCHMERTZLER MICHAELdirector | Sell | 9,419 | $70.78 |
| Aug 21, 2026 | SCHMERTZLER MICHAELdirector | Sell | 5,335 | $72.86 |
| Aug 21, 2026 | SCHMERTZLER MICHAELdirector | Sell | 2,390 | $71.88 |
| Aug 21, 2026 | SCHMERTZLER MICHAELdirector | Sell | 691 | $72.92 |
| Aug 19, 2026 | Boulding Mark Elliottofficer: EXEC. VP AND CLO | Sell | 969 | $72.90 |
| Aug 19, 2026 | Boulding Mark Elliottofficer: EXEC. VP AND CLO | Sell | 1,844 | $72.39 |
| Aug 19, 2026 | Boulding Mark Elliottofficer: EXEC. VP AND CLO | Option | 2,813 | $25.69 |
| Aug 6, 2026 | Klein Matthew B.director, officer: CHIEF EXECUTIVE OFFICER | Sell | 10,292 | $68.57 |
| Jul 30, 2026 | Sollie-Zetlmayer Hege Elisabethdirector | Grant | 4,000 | — |
| Jul 30, 2026 | Sollie-Zetlmayer Hege Elisabethdirector | Grant | 1,738 | $75.39 |
Source: PTCT SEC Form 4 filings, latest Aug 28, 2026. For informational purposes only — not investment advice.
See the full PTCT insider & 13F page →PTCT research & analysis
FDA Drops Sham Control for Rare-Disease Gene Therapy (CLPT, QURE)
The FDA told uniQure a sham-surgery control is no longer required, moving the cost of rare-disease gene therapy onto delivery hardware and endpoint instruments.
BCDABIIBCLPTuniQure (QURE), ClearPoint (CLPT): FDA Drops Sham-Surgery Control
uniQure, ClearPoint and BioCardia disclosed the FDA no longer requires a sham-surgery control in rare CNS gene therapy, moving the gate to delivery hardware.
BCDACLPTCYRX
PTC Therapeutics, Inc. company profile
Overview
PTC Therapeutics, Inc. (NASDAQ:PTCT) is a biopharmaceutical company founded in 1998 and headquartered in South Plainfield, New Jersey. The company went public in June 2013 and has evolved from a research-focused organization into a commercial-stage biopharmaceutical company with multiple marketed products. PTC specializes in discovering, developing, and commercializing treatments for rare diseases, with a particular focus on genetic disorders that affect small patient populations with high unmet medical needs.
Business
PTC Therapeutics operates in the rare disease biotechnology sector, developing and commercializing specialized medicines for patients with genetic disorders. The company's business spans multiple therapeutic areas within rare diseases, with products addressing conditions that typically affect thousands rather than millions of patients worldwide. The company's commercial portfolio includes several key franchises. The Duchenne Muscular Dystrophy (DMD) franchise represents the largest revenue segment, generating approximately 65-70% of total revenues through two main products: Translarna (ataluren), which treats nonsense mutation DMD by enabling cells to read through premature stop codons, and Emflaza (deflazacort), a corticosteroid that helps slow muscle degeneration. DMD is a progressive muscle-wasting disease primarily affecting boys, caused by mutations in the dystrophin gene. The royalty revenue segment contributes roughly 25-30% of revenues, primarily from Evrysdi (risdiplam), a treatment for spinal muscular atrophy developed by Roche. PTC receives royalties on global Evrysdi sales based on their contribution to the drug's discovery through their splicing platform technology. Spinal muscular atrophy is a genetic disorder that affects motor neurons, leading to muscle weakness and atrophy. The company also markets several other specialized therapies including Upstaza for AADC deficiency (a rare genetic disorder affecting dopamine production), and distributes Tegsedi and Waylivra for rare metabolic diseases in Latin America and the Caribbean. Additionally, PTC has recently received approval for KEBILIDI, another treatment for AADC deficiency. PTC's pipeline focuses on leveraging their proprietary platforms, particularly their splicing technology, to develop treatments for additional rare diseases including Huntington's disease, phenylketonuria (PKU), Friedreich's ataxia, and amyotrophic lateral sclerosis (ALS).
Revenue model
PTC Therapeutics generates revenue through multiple streams centered around rare disease treatments. The primary revenue model is direct product sales of their marketed therapies, where they sell directly to specialty pharmacies, hospitals, and treatment centers that serve rare disease patients. These products typically command premium pricing due to their specialized nature and the lack of alternative treatments. The company's customers are primarily specialty healthcare providers, rare disease treatment centers, and specialty pharmacies that have expertise in managing complex rare disease patients. Given the nature of rare diseases, PTC often works directly with a limited number of specialized treatment centers globally rather than broad distribution networks. Royalty revenues represent the second major income stream, where PTC receives ongoing payments based on sales of products they helped develop but are commercialized by partners. The most significant example is their royalty arrangement with Roche for Evrysdi, where PTC receives payments based on global sales performance. The company also generates revenue through licensing and collaboration agreements, as demonstrated by their recent $1 billion upfront payment from Novartis for rights to PTC518 (Huntington's disease program), along with potential milestone payments and future royalties. Several factors influence PTC's margins and profitability. Positive margin drivers include the premium pricing power inherent in rare disease treatments due to limited competition and high unmet medical need, the relatively low manufacturing costs for small-molecule drugs compared to biologics, and the potential for long-term patient relationships given the chronic nature of these conditions. Margin pressures come from high research and development costs required for clinical trials in rare diseases, significant regulatory and compliance expenses, the need for specialized commercial infrastructure to reach rare disease treatment centers, and potential pricing pressure from payers and health technology assessment bodies, particularly in international markets. Currency fluctuations also impact margins given the company's global commercial presence.
Competitive moat
PTC Therapeutics possesses a moderate competitive moat built primarily around their specialized expertise in rare disease drug development and their proprietary technology platforms. The company's strongest moat elements include their deep knowledge of rare disease regulatory pathways, established relationships with specialized treatment centers and key opinion leaders in rare disease communities, and their proprietary splicing platform technology that has demonstrated ability to generate multiple drug candidates. The company benefits from the inherent characteristics of rare disease markets, where high barriers to entry exist due to the specialized knowledge required, small patient populations that make it economically challenging for multiple competitors to enter, and the significant time and capital investment needed to develop treatments for these conditions. Once established in a rare disease indication, companies often enjoy sustained market positions due to physician and patient reluctance to switch treatments that are working. However, PTC's moat faces several vulnerabilities. The rare disease space is increasingly attracting larger pharmaceutical companies with greater resources, as demonstrated by the competitive dynamics in DMD where multiple companies are developing treatments. Regulatory risks pose ongoing challenges, as evidenced by the European regulatory setbacks with Translarna, where changing regulatory standards can significantly impact established products. The company's dependence on a limited number of products creates concentration risk, and their royalty revenues are subject to the commercial success and strategic decisions of partners like Roche. Competitive threats are emerging from several directions: gene therapies that could provide curative treatments for conditions PTC currently treats symptomatically, larger pharmaceutical companies entering rare disease markets with greater resources for development and commercialization, and potential biosimilar or generic competition as some of their products age. The company's future competitive position will largely depend on their ability to successfully launch new products from their pipeline and continue leveraging their platform technologies to generate additional drug candidates.
Risks & safety
PTC Therapeutics presents a moderate margin of safety with improving financial metrics but some underlying risks. • Liquidity position: Strong with $1.48 billion in cash and short-term investments as of Q1 2025, significantly improved from $780 million at end of 2024 due to the $1 billion Novartis upfront payment. Current ratio of 3.89 indicates solid short-term liquidity. • Cash flow dynamics: Dramatically improved from negative $108 million operating cash flow in 2024 to positive $870 million in Q1 2025, primarily driven by the Novartis collaboration payment. Free cash flow similarly improved to $868 million in Q1 2025. • Debt and solvency: The company shows negative shareholders' equity of approximately -$186 million, indicating total liabilities exceed total assets. However, the strong cash position provides adequate runway for operations and the negative equity primarily reflects accumulated losses from R&D investments rather than unsustainable debt levels. • Valuation metrics: Trading at relatively low multiples with P/E ratio of 1.15 and EV/EBITDA of 0.74 based on Q1 2025 results, though these metrics are distorted by the one-time Novartis payment. More normalized metrics would show higher valuations. • Revenue sustainability: Recurring revenue base from established products provides some stability, though regulatory risks (particularly in Europe) and competitive pressures create ongoing uncertainty.
Recent development
Over the past few years, PTC Therapeutics has undergone a significant strategic transformation from a primarily development-stage company to a diversified commercial organization with multiple revenue-generating products and a robust pipeline. The most significant recent development was the $1 billion collaboration agreement with Novartis in January 2025 for PTC518, their Huntington's disease program, which provides substantial financial resources and validates their splicing platform technology. The company has systematically built a pipeline of late-stage programs targeting large rare disease opportunities. Sepiapterin for PKU represents potentially their largest commercial opportunity, with management estimating over $1 billion in revenue potential globally and targeting approximately 58,000 PKU patients worldwide. The therapy received FDA approval and is preparing for global launch, with particular focus on the ability to allow patients to liberalize their restrictive diets. Regulatory strategy has become increasingly sophisticated, with PTC submitting four FDA applications in 2024 alone and successfully navigating complex rare disease regulatory pathways. The approval of KEBILIDI for AADC deficiency and the pending approvals for vatiquinone (Friedreich's ataxia) and potential Translarna approval in the US demonstrate their execution capabilities. The company has also made strategic portfolio decisions, including divesting non-core assets like their gene therapy manufacturing business while focusing resources on their highest-potential programs. Their approach to business development has evolved to include both in-licensing opportunities and out-licensing of programs like PTC518 where partnering provides better commercial prospects. Platform technology development continues to be a key focus, with their splicing platform generating multiple drug candidates and their inflammation platform showing early promise. This platform approach allows PTC to potentially generate multiple products from their core technological capabilities rather than relying on single-asset development programs.
PTCT company profile · for informational purposes only — not investment advice.
Track PTCT with Drillr
SEC filings, earnings calls, insider activity, alt-data signals — all queryable through Drillr's AI terminal and MCP API.
Try Drillr for free