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PTC Therapeutics Inc (PTCT)



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Upturn Advisory Summary
07/10/2025: PTCT (1-star) is currently NOT-A-BUY. Pass it for now.
1 Year Target Price $62.81
1 Year Target Price $62.81
7 | Strong Buy |
3 | Buy |
5 | Hold |
1 | Sell |
0 | Strong Sell |
Analysis of Past Performance
Type Stock | Historic Profit -7.42% | Avg. Invested days 33 | Today’s Advisory PASS |
Upturn Star Rating ![]() ![]() | Upturn Advisory Performance ![]() | Stock Returns Performance ![]() |
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Key Highlights
Company Size Mid-Cap Stock | Market Capitalization 3.92B USD | Price to earnings Ratio 7.61 | 1Y Target Price 62.81 |
Price to earnings Ratio 7.61 | 1Y Target Price 62.81 | ||
Volume (30-day avg) 16 | Beta 0.51 | 52 Weeks Range 29.02 - 58.38 | Updated Date 07/10/2025 |
52 Weeks Range 29.02 - 58.38 | Updated Date 07/10/2025 | ||
Dividends yield (FY) - | Basic EPS (TTM) 6.51 |
Analyzing Revenue: Products, Geography and Growth
Revenue by Products
Product revenue - Year on Year
Revenue by Geography
Geography revenue - Year on Year
Earnings Date
Report Date - | When - | Estimate - | Actual - |
Profitability
Profit Margin 33.56% | Operating Margin (TTM) 82.43% |
Management Effectiveness
Return on Assets (TTM) 24.42% | Return on Equity (TTM) -7740.12% |
Valuation
Trailing PE 7.61 | Forward PE 6.05 | Enterprise Value 2225421106 | Price to Sales(TTM) 2.21 |
Enterprise Value 2225421106 | Price to Sales(TTM) 2.21 | ||
Enterprise Value to Revenue 1.26 | Enterprise Value to EBITDA 2.65 | Shares Outstanding 79257000 | Shares Floating 64407424 |
Shares Outstanding 79257000 | Shares Floating 64407424 | ||
Percent Insiders 2.4 | Percent Institutions 101.06 |
Upturn AI SWOT
PTC Therapeutics Inc

Company Overview
History and Background
PTC Therapeutics, Inc. was founded in 1998. It is a biopharmaceutical company focused on the discovery, development, and commercialization of orally administered drugs targeting post-transcriptional control processes. They have grown from a research-focused entity to a commercial-stage company with multiple marketed products.
Core Business Areas
- Rare Diseases: Development and commercialization of therapies for rare diseases, with a focus on genetic disorders. Examples include Duchenne muscular dystrophy and spinal muscular atrophy.
- Oncology: Research and development of treatments for various types of cancer.
Leadership and Structure
The CEO of PTC Therapeutics is Matthew B. Klein. The organizational structure includes research, development, commercial, and administrative departments. The board of directors provides oversight and strategic guidance.
Top Products and Market Share
Key Offerings
- Translarna (ataluren): Translarna is an orally available small-molecule drug marketed for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged 2 years and older. Market share data is difficult to obtain precisely, but PTC is a key player. Competitors include Sarepta Therapeutics (gene therapy based treatments) and steroids (generic, but provides some relief from the symptoms of DMD).
- Emflaza (deflazacort): Emflaza is a corticosteroid indicated for the treatment of Duchenne muscular dystrophy (DMD). Acquired from Marathon Pharmaceuticals. Market share data is difficult to obtain precisely. Competitors include other steroids (generic). Revenue is significant, but being challenged with more targeted approaches.
- Evrysdi (risdiplam): PTC Therapeutics receives royalties from Roche on global sales of Evrysdi for spinal muscular atrophy (SMA). Evrysdi is the leading oral treatment for SMA. Other competitors include Novartis' Zolgensma and Biogen's Spinraza. This product does not directly add to PTC's revenue beyond royalty payments.
Market Dynamics
Industry Overview
The biopharmaceutical industry is characterized by high research and development costs, long development timelines, and regulatory hurdles. The rare disease segment is growing due to increased awareness and orphan drug designation incentives.
Positioning
PTC Therapeutics is positioned as a company focused on developing innovative therapies for rare diseases. Their competitive advantages include their expertise in RNA biology and their established commercial infrastructure.
Total Addressable Market (TAM)
The total addressable market (TAM) for rare disease therapeutics is estimated to be hundreds of billions of dollars globally and is expanding rapidly. PTC Therapeutics is positioned to capture a significant portion of this TAM through their existing product portfolio and pipeline.
Upturn SWOT Analysis
Strengths
- Established commercial infrastructure
- Expertise in RNA biology
- Diverse pipeline of product candidates
- Royalties from Evrysdi sales
Weaknesses
- High operating expenses
- Reliance on key products
- Regulatory challenges
- Competition from larger pharmaceutical companies
Opportunities
- Expansion into new therapeutic areas
- Acquisition of complementary assets
- Partnerships with other pharmaceutical companies
- Increased awareness and diagnosis of rare diseases
Threats
- Clinical trial failures
- Regulatory setbacks
- Generic competition
- Pricing pressures
Competitors and Market Share
Key Competitors
- SRPT
- VRTX
- BIIB
Competitive Landscape
PTC Therapeutics faces competition from larger pharmaceutical companies with greater resources. However, PTC's focus on RNA biology and rare diseases gives it a competitive advantage in certain areas. The landscape is constantly evolving due to scientific and clinical advances.
Major Acquisitions
BioElectron Technology Corporation
- Year: 2023
- Acquisition Price (USD millions): 240
- Strategic Rationale: Expand pipeline to include novel preclinical assets for mitochondrial diseases and other disorders.
Growth Trajectory and Initiatives
Historical Growth: Historical growth has been driven by the commercialization of Translarna and Emflaza, as well as royalties from Evrysdi. Growth is tied to regulatory approval and reimbursement in different countries. The success of new drug candidates is critical to future growth.
Future Projections: Future growth projections depend on the success of clinical trials, regulatory approvals, and market penetration. Analyst estimates vary but generally project continued revenue growth. Specific projections should be obtained from financial news sources.
Recent Initiatives: Recent initiatives include advancing pipeline programs, expanding into new geographic markets, and strategic collaborations.
Summary
PTC Therapeutics is a biopharmaceutical company focused on rare diseases and oncology with established products and royalty streams. While their expertise in RNA biology and focus on rare diseases provide a competitive edge, high operating expenses and reliance on key products pose challenges. The company needs to expand its pipeline and navigate regulatory hurdles successfully to achieve sustainable growth. Success also depends on the competition from pharmaceutical companies with greater resources.
Peer Comparison
Sources and Disclaimers
Data Sources:
- PTC Therapeutics Investor Relations
- SEC Filings
- Industry Reports
- Analyst Reports
Disclaimers:
This analysis is for informational purposes only and should not be considered financial advice. Market conditions and company performance can change rapidly. Consult with a financial professional before making any investment decisions.
AI Summarization is directionally correct and might not be accurate.
Summarized information shown could be a few years old and not current.
Fundamental Rating based on AI could be based on old data.
AI-generated summaries may have inaccuracies (hallucinations). Please verify the information before taking action.
About PTC Therapeutics Inc
Exchange NASDAQ | Headquaters Warren, NJ, United States | ||
IPO Launch date 2013-06-20 | CEO & Director Dr. Matthew B. Klein F.A.C.S., M.D., M.S. | ||
Sector Healthcare | Industry Biotechnology | Full time employees 939 | Website https://www.ptcbio.com |
Full time employees 939 | Website https://www.ptcbio.com |
PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines to children and adults living with rare disorders in the United States and internationally. The company offers Translarna and Emflaza for the treatment of Duchenne muscular dystrophy; Upstaza to treat aromatic l-amino acid decarboxylas (AADC) deficiency, a central nervous system disorder; Tegsedi and Waylivra for the treatment of rare diseases; and Evrysdi to treat spinal muscular atrophy (SMA) in adults and children. Its development pipeline products include Sepiapterin for the treatment of phenylketonuria; PTC518 splicing platform, which is being developed for the treatment of Huntington's disease; and inflammation and ferroptosis platforms, including vatiquinone to treat Friedreich's ataxia. The company distributes its products through third-party distributors. It has collaborations with F. Hoffman-La Roche Ltd., Hoffman-La Roche Inc., the SMA Foundation, National Taiwan University, Akcea Therapeutics, Inc., and Shiratori Pharmaceutical Co., Ltd. The company has license and collaboration agreement with Novartis Pharmaceuticals Corporation to develop PTC518 Huntington's disease program. It markets Upstaza, a gene therapy with the brand name Kebilidi in the United States. PTC Therapeutics, Inc. was incorporated in 1998 and is headquartered in Warren, New Jersey.

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