PTC Therapeutics (PTCT) AI Stock Analysis

• Market Cap: $4.07B
• Dividend Yield: N/A
• Average Volume (3M): 1.05M
• Price to Earnings (P/E): 7.7
• Beta (1Y): 1.37
• Revenue Growth: 91.12%
• EPS Growth: N/A
• Country: US
• Employees: 939
• Sector: Healthcare
• Sector Strength: 37
• Industry: Biotechnology
• EPS (TTM): -0.83
• Shares Outstanding: 79,257,020
• 10 Day Avg. Volume: 1,406,571
• 30 Day Avg. Volume: 1,050,896
• PEG Ratio: N/A
• Price to Book (P/B): 0.00
• Price to Sales (P/S): 0.00
• P/FCF Ratio: 0.00
• Enterprise Value/Market Cap: 0.68
• Enterprise Value/Revenue: 1.56
• Enterprise Value/Gross Profit: 1.62
• Enterprise Value/Ebitda: 3.32
• 1Y Price Target: $68.85
• Price Target Upside: 36.72% Upside
• Rating Consensus: Moderate Buy
• Number of Analyst Covering: 15

• Rating: 77 Outperform
• Price Target: $58.00 ▲(15.17% Upside)

PTC Therapeutics' overall score is driven by strong financial performance improvements and strategic product launches. The company's robust cash position and recent regulatory approvals support its growth prospects. However, challenges such as negative equity and competition from generics remain.

Positive Factors

Financial Stability

PTC Therapeutics has strong financial stability with over $2 billion in cash reserves and partnerships in SMA and HD, providing a solid foundation for valuation.

Market Demand

Sephience is expected to have strong demand in the PKU market due to its efficacy and safety advantages, which may lead to it becoming the preferred adjunct therapy.

Regulatory Approval

Sephience has been approved by the FDA with a broad label for the treatment of hyperphenylalaninemia in phenylketonuria patients aged 1 month and older.

Negative Factors

Competitive Landscape

The presence of generic sapropterin continues to be a headwind.

Dietary Requirements

The availability of generic sapropterin and Sephience’s label requirement for a Phe-restricted diet are both headwinds to rapid and broad uptake.

Pricing Strategy

PTCT's wholesale acquisition cost for Sephience is considerably higher at $41,000 per month or ~$492,000 per year for an average patient.

PTC Therapeutics Business Overview & Revenue Model

Company Description

PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines to patients with rare disorders. Its portfolio pipeline includes commercial products and product candidates in various stages of development, including clinical, pre-clinical and research and discovery stages, focuses on the development of treatments for multiple therapeutic areas, such as rare diseases. The company offers Translarna and Emflaza for the treatment of Duchenne muscular dystrophy in the European Economic Area and the United States, as well as to treat nonsense mutation Duchenne muscular dystrophy in Brazil and Russia; commercializes Tegsedi and Waylivra for the treatment of rare diseases in Latin America and the Caribbean; and markets Evrysdi for the treatment of spinal muscular atrophy in adults and children two months and older in Brazil. The company's splicing platform includes PTC518, which is being developed for the treatment of Huntington's disease. PTC Therapeutics, Inc. has collaborations with F. Hoffman-La Roche Ltd and Hoffman-La Roche Inc., as well as the Spinal Muscular Atrophy Foundation to advance drug discovery and development research in regenerative medicine; and Akcea Therapeutics, Inc. to commercialize Tegsedi and Waylivra for the treatment of rare diseases in Latin America and the Caribbean. PTC Therapeutics, Inc. was incorporated in 1998 and is headquartered in South Plainfield, New Jersey.

How the Company Makes Money

PTC Therapeutics primarily generates revenue through the sale of its pharmaceutical products that are developed to treat rare and orphan diseases. The company's revenue model is based on the commercialization of its FDA-approved drugs, which are sold both directly to healthcare providers and through distribution partnerships. Key revenue streams include sales of its flagship products such as Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy and Emflaza (deflazacort), a corticosteroid for Duchenne muscular dystrophy. Additionally, PTC Therapeutics engages in strategic collaborations and licensing agreements with other biotechnology and pharmaceutical companies, which provide additional revenue through milestone payments and royalties. The company's focus on niche markets with high unmet medical needs often allows for premium pricing of its treatments, contributing significantly to its earnings.

PTC Therapeutics Financial Statement Overview

Summary

PTC Therapeutics has shown significant improvement in financial performance, with strong revenue growth and improved profitability in the TTM period. While cash flow management is strong, the negative equity and high liabilities remain concerns for financial stability.

Income Statement

PTC Therapeutics has shown a remarkable turnaround in its financial performance. The TTM (Trailing-Twelve-Months) gross profit margin is strong at 96.52%, indicating efficient cost management. The net profit margin has improved significantly to 33.55% in the TTM, reflecting a positive shift from previous losses. Revenue growth has been robust, with a 119.73% increase from 2024 to the TTM period. The EBIT and EBITDA margins have also turned positive, demonstrating improved operational efficiency. However, the company has experienced volatility in past years, which could pose a risk if not managed carefully.

Balance Sheet

The balance sheet of PTC Therapeutics presents a mixed picture. The debt-to-equity ratio is not meaningful due to negative stockholders' equity, indicating potential financial instability. However, the company has a strong cash position, with cash and equivalents exceeding total debt, resulting in negative net debt. The equity ratio is negative, which is a concern, but the company has managed to improve its asset base over time. The negative equity and high liabilities suggest a need for careful financial management to ensure long-term sustainability.

Cash Flow

PTC Therapeutics has shown a significant improvement in cash flow metrics. The TTM free cash flow is positive at $744.24 million, a substantial increase from previous negative figures, indicating better cash generation. The operating cash flow to net income ratio is 1.16, suggesting that the company is generating cash efficiently relative to its net income. The free cash flow to net income ratio is 1.25, further highlighting strong cash flow management. Despite these improvements, the company must continue to focus on maintaining positive cash flows to support its operations and growth.

Breakdown	Dec 2024	Dec 2023	Dec 2022	Dec 2021	Dec 2020
Income Statement
Total Revenue	806.78M	937.82M	698.80M	538.59M	380.77M
Gross Profit	749.38M	872.34M	654.12M	506.26M	361.82M
EBITDA	-120.46M	-330.34M	-367.78M	-374.44M	-297.00M
Net Income	-363.30M	-626.60M	-559.02M	-523.90M	-438.16M
Balance Sheet
Total Assets	1.71B	1.90B	1.71B	1.94B	2.21B
Cash, Cash Equivalents and Short-Term Investments	1.14B	876.74M	410.70M	773.38M	1.10B
Total Debt	389.30M	2.23B	703.63M	431.43M	420.44M
Total Liabilities	2.80B	2.71B	2.05B	1.94B	1.73B
Stockholders Equity	-1.10B	-818.55M	-347.09M	1.44M	481.98M
Cash Flow
Free Cash Flow	-114.19M	-279.04M	-509.11M	-336.66M	-250.05M
Operating Cash Flow	-107.69M	-158.42M	-356.65M	-251.33M	-194.07M
Investing Cash Flow	44.18M	-176.74M	290.18M	219.18M	-561.55M
Financing Cash Flow	255.87M	646.40M	167.95M	20.88M	668.72M

PTC Therapeutics Technical Analysis

• Technical Analysis Sentiment: Positive
• Last Price: 50.36
• 50DMA: Positive
• 100DMA: Positive
• 200DMA: Positive
• MACD: Negative
• RSI: Neutral
• STOCH: Neutral

Evaluating momentum and price trends is crucial in stock analysis to make informed investment decisions. For PTCT, the sentiment is Positive. The current price of 50.36 is above the 20-day moving average (MA) of 48.82, above the 50-day MA of 49.45, and above the 200-day MA of 47.53, indicating a bullish trend. The MACD of 0.69 indicates Negative momentum. The RSI at 54.18 is Neutral, neither overbought nor oversold. The STOCH value of 77.99 is Neutral, not indicating any strong overbought or oversold conditions. Overall, these indicators collectively point to a Positive sentiment for PTCT.

PTC Therapeutics Peers Comparison

Name	Overall Rating	Market Cap	P/E Ratio	ROE	Dividend Yield	Revenue Growth	EPS Growth
PTCT	77 Outperform	$4.07B	7.73	33.08%	―	91.12%	―
ACLX	64 Neutral	$3.97B	―	-35.56%	―	-41.66%	-180.15%
SWTX	61 Neutral	$3.54B	―	-51.57%	―	730.42%	31.63%
RNA	60 Neutral	$5.82B	―	-34.19%	―	-17.87%	-1.89%
RYTM	56 Neutral	$5.82B	―	-1334.54%	―	53.55%	30.87%
Sector *	51 Neutral	$7.39B	0.36	-62.86%	2.37%	15.48%	-2.68%
RARE	49 Neutral	$2.73B	―	-182.60%	―	26.77%	24.18%

PTC Therapeutics Corporate Events

PTC Therapeutics Enters Rights Satisfaction Agreement

On August 5, 2025, PTC Therapeutics entered into a Rights Satisfaction Agreement with former securityholders of Censa Pharmaceuticals, resulting in the cancellation of their rights to receive certain payments based on net sales of products containing sepiapterin. In exchange, PTC agreed to pay up to $250 million in cash and potential milestone payments based on sales thresholds. This agreement impacts the company’s financial obligations under the original merger agreement but maintains other rights and obligations intact.

The most recent analyst rating on (PTCT) stock is a Buy with a $63.00 price target. To see the full list of analyst forecasts on PTC Therapeutics stock, see the PTCT Stock Forecast page.

PTC Therapeutics Announces Q2 2025 Financial Results

On August 7, 2025, PTC Therapeutics announced its second quarter financial results and corporate updates, highlighting the European and FDA approval of Sephience™ for PKU, marking a significant milestone for the company. The global launch of Sephience is underway, expected to drive future growth and profitability. The company reported total revenues of $179 million for Q2 2025, with notable contributions from its DMD franchise and royalty revenues. Despite a decrease in net product revenue compared to the previous year, PTC’s strategic moves, including an agreement to purchase obligations related to Sephience, position it for continued expansion in the rare disease market.

The most recent analyst rating on (PTCT) stock is a Buy with a $63.00 price target. To see the full list of analyst forecasts on PTC Therapeutics stock, see the PTCT Stock Forecast page.

PTC Therapeutics Gains FDA Approval for Sephience

On July 28, 2025, PTC Therapeutics announced that the FDA approved Sephience™ (sepiapterin) for treating phenylketonuria (PKU) in patients one month and older. This approval, based on the Phase 3 APHENITY trial, marks a significant milestone for the PKU community, offering a new treatment option that could improve the quality of life for individuals with sepiapterin-responsive PKU. The approval also strengthens PTC’s position in the rare disease market, with Sephience already approved in the European Economic Area and under review in other countries.

The most recent analyst rating on (PTCT) stock is a Buy with a $63.00 price target. To see the full list of analyst forecasts on PTC Therapeutics stock, see the PTCT Stock Forecast page.

PTC Therapeutics Elects Directors at Annual Meeting

PTC Therapeutics held its Annual Meeting on June 17, 2025, where stockholders elected three Class III directors to serve until the 2028 annual meeting. Additionally, the appointment of Ernst & Young LLP as the independent registered public accounting firm for the fiscal year ending December 31, 2025, was ratified. The stockholders also approved a non-binding advisory proposal on named executive officer compensation.

The most recent analyst rating on (PTCT) stock is a Buy with a $63.00 price target. To see the full list of analyst forecasts on PTC Therapeutics stock, see the PTCT Stock Forecast page.