Home 
Today's Top Picks 
Top Performers 
Watchlist 
My Robo Portfolios 
ETF Zone 
Help & Tutorial 
Cancel anytime
- Chart
- Upturn Summary
- Highlights
- Revenue
- Valuation
Upturn AI SWOT - About
PTCT 
PTC Therapeutics Inc (PTCT)
- BUY Advisory
- SELL Advisory (Profit)
- SELL Advisory (Loss)
- Profit
- Loss
- Pass (Skip investing)
Stock price based on last close (see disclosures)
- ALL
- YEAR
- MONTH
- WEEK
Upturn Advisory Summary
08/28/2025: PTCT (1-star) is currently NOT-A-BUY. Pass it for now.
Upturn Star Rating
Not Recommended Performance
These Stocks/ETFs, based on Upturn Advisory, consistently fall short of market performance, signaling caution before investing.
Number of Analysts
16 Analysts rated it
Moderately tracked stock, growing coverage, gaining market and investor attention.
1 Year Target Price $66.69
1 Year Target Price $66.69
| 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  2.0 | Stock Returns Performance  1.0 |
| Profits based on simulation | Last Close 08/28/2025 |
Key Highlights
Company Size Mid-Cap Stock | Market Capitalization 3.96B USD | Price to earnings Ratio 7.06 | 1Y Target Price 66.69 |
Price to earnings Ratio 7.06 | 1Y Target Price 66.69 | ||
Volume (30-day avg) 16 | Beta 0.54 | 52 Weeks Range 30.41 - 58.38 | Updated Date 08/29/2025 |
52 Weeks Range 30.41 - 58.38 | Updated Date 08/29/2025 | ||
Dividends yield (FY) - | Basic EPS (TTM) 6.99 |
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 2025-08-07 | When - | Estimate -1.06 | Actual -0.83 |
Profitability
Profit Margin 35.65% | Operating Margin (TTM) -19.49% |
Management Effectiveness
Return on Assets (TTM) 23.81% | Return on Equity (TTM) -7740.12% |
Valuation
Trailing PE 7.06 | Forward PE 6.06 | Enterprise Value 2354238700 | Price to Sales(TTM) 2.24 |
Enterprise Value 2354238700 | Price to Sales(TTM) 2.24 | ||
Enterprise Value to Revenue 1.33 | Enterprise Value to EBITDA 2.8 | Shares Outstanding 79438096 | Shares Floating 65010547 |
Shares Outstanding 79438096 | Shares Floating 65010547 | ||
Percent Insiders 2.47 | Percent Institutions 101.83 |
Upturn AI SWOT
PTC Therapeutics Inc
Company Overview
History and Background
PTC Therapeutics, Inc. was founded in 1998 and is headquartered in South Plainfield, New Jersey. It focuses on the discovery, development, and commercialization of orally administered, small-molecule drugs targeting post-transcriptional control processes.
Core Business Areas
- Rare Disorders: Focuses on developing and commercializing therapies for rare diseases, particularly those with limited or no treatment options. This includes genetic disorders.
- Oncology: Engages in the development of targeted therapies for cancer, with a focus on addressing unmet needs in specific cancer subtypes.
Leadership and Structure
PTC Therapeutics is led by CEO Matthew B. Klein. The organizational structure is typical of a biotechnology company, with departments focused on research and development, clinical trials, commercial operations, and corporate functions.
Top Products and Market Share
Key Offerings
- Translarna (ataluren): Translarna is approved for the treatment of Duchenne muscular dystrophy (DMD) caused by a nonsense mutation in the dystrophin gene. Market share is difficult to obtain precisely but represents a significant portion of the nonsense mutation DMD market. Competitors include Sarepta Therapeutics with Exondys 51 and other exon-skipping therapies, as well as gene therapies in development.
- Emflaza (deflazacort): Emflaza is a corticosteroid approved for the treatment of Duchenne muscular dystrophy (DMD), regardless of the underlying mutation. Competitors include other corticosteroids like prednisone, as well as other DMD-specific therapies. Market share information is variable.
- Evrysdi (risdiplam) (Partnered with Roche and Genentech): Evrysdi is approved for the treatment of spinal muscular atrophy (SMA). While PTC shares in revenue, Roche and Genentech primarily commercialize the product. Competitors include Spinraza from Biogen and Zolgensma from Novartis.
Market Dynamics
Industry Overview
The pharmaceutical industry, particularly the rare disease segment, is characterized by high research and development costs, lengthy regulatory approval processes, and significant market exclusivity periods upon successful drug launch. The industry is driven by innovation, patient advocacy, and unmet medical needs.
Positioning
PTC Therapeutics is a player in the rare disease market, focusing on developing therapies for genetic disorders. Its competitive advantages include its expertise in RNA biology and its established commercial infrastructure for rare disease products.
Total Addressable Market (TAM)
The global rare disease market is estimated to be hundreds of billions of dollars. PTC is positioned to capture a share of this TAM through its approved therapies and pipeline products.
Upturn SWOT Analysis
Strengths
- Established commercial infrastructure for rare disease products
- Expertise in RNA biology and drug development
- Approved therapies addressing unmet medical needs
- Strong intellectual property portfolio
Weaknesses
- High research and development costs
- Reliance on a limited number of products for revenue
- Regulatory and clinical trial risks
- Competition from larger pharmaceutical companies
Opportunities
- Expansion into new rare disease indications
- Development of novel drug candidates
- Strategic collaborations and acquisitions
- Geographic expansion into new markets
Threats
- Competition from existing and emerging therapies
- Regulatory setbacks or delays
- Patent expirations
- Pricing pressures from payers
Competitors and Market Share
Key Competitors
- SRPT
- BIIB
- NVS
Competitive Landscape
PTC Therapeutics faces competition from established pharmaceutical companies with greater resources and broader product portfolios. PTC's competitive advantage lies in its specific focus on RNA biology and rare diseases.
Major Acquisitions
BioElectron Technology Corporation
- Year: 2023
- Acquisition Price (USD millions): 35
- Strategic Rationale: Expanded PTC's pipeline with a novel preclinical therapeutic for mitochondrial diseases.
Growth Trajectory and Initiatives
Historical Growth: Growth has been driven by the sales of Translarna and Emflaza, as well as revenue sharing from Evrysdi. Growth has also been volatile, impacted by regulatory approvals and market dynamics.
Future Projections: Future growth is dependent on the success of pipeline products and continued sales of existing therapies. Analyst estimates vary depending on the perceived potential of the pipeline.
Recent Initiatives: Recent initiatives include expanding clinical trials for pipeline candidates and pursuing regulatory approvals in new territories.
Summary
PTC Therapeutics is a pharmaceutical company focused on rare diseases, with established products and a pipeline of novel therapies. The company's strengths include its expertise in RNA biology and commercial infrastructure. PTC faces challenges, including high research costs, competition, and regulatory risks. The future of PTC depends on the success of its pipeline and continued sales of existing products.
Peer Comparison
Sources and Disclaimers
Data Sources:
- Company Website
- SEC Filings
- Analyst Reports
- Industry Publications
Disclaimers:
This analysis is based on publicly available information and should not be considered financial advice. Market share data is estimated and may not be precise. Financial data requires manual updating to the latest reports.
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.
Note: This website is maintained by Upturn Corporation, which is an investment adviser registered with the U.S. Securities and Exchange Commission. Such registration does not imply a certain level of skill or training. Investing in securities has risks. Past performance is no guarantee of future returns. No assurance is provided as to any particular investment return, and you may lose money using our services. You are strongly advised to consult appropriate counsel before making any investments in companies you learn about through our services. You should obtain appropriate legal, tax, investment, accounting, and other advice that takes into account your investment portfolio and overall financial situation. You are solely responsible for conducting due diligence on a potential investment. We do not affect trades for you. You will select your own broker through which to transact. Investments are not FDIC insured, they are not guaranteed, and they may lose value. Please see the Privacy Policy, Terms of Use, and Disclosure for more information.