PTC Therapeutics (PTCT) AI Stock Analysis

• Market Cap: $5.41B
• Dividend Yield: N/A
• Average Volume (3M): 1.08M
• Price to Earnings (P/E): 9.8
• Beta (1Y): 1.19
• Revenue Growth: 96.00%
• EPS Growth: N/A
• Country: US
• Employees: 939
• Sector: Healthcare
• Sector Strength: 45
• Industry: Biotechnology
• EPS (TTM): -0.83
• Shares Outstanding: 79,438,095
• 10 Day Avg. Volume: 848,663
• 30 Day Avg. Volume: 1,076,701
• PEG Ratio: N/A
• Price to Book (P/B): N/A
• Price to Sales (P/S): N/A
• P/FCF Ratio: N/A
• Enterprise Value/Market Cap: 0.41
• Enterprise Value/Revenue: 1.26
• Enterprise Value/Gross Profit: 1.26
• Enterprise Value/Ebitda: 2.64
• 1Y Price Target: $68.43
• Price Target Upside: 0.47% Upside
• Rating Consensus: Moderate Buy
• Number of Analyst Covering: 16

• Rating: 61 Neutral
• Price Target: $71.00 ▲(4.24% Upside)

PTC Therapeutics shows strong technical momentum and a favorable valuation, which are the most significant factors driving the score. Financial performance is a concern due to negative equity and high leverage, but improvements in cash flow and operational efficiency provide some optimism. The positive sentiment from the earnings call further supports the stock's potential, although it was not included in the weighted score.

Positive Factors

Strong Cash Position

A robust cash position supports PTC's commercial and R&D initiatives, allowing for strategic investments and reducing the need for external financing.

Sephience Approval

Regulatory approval of Sephience in major markets enhances PTC's product portfolio and provides a new revenue stream with significant growth potential.

Operational Efficiency

Improved operational efficiency, as shown by better EBIT and EBITDA margins, indicates effective cost management and enhances long-term profitability.

Negative Factors

Negative Equity

Negative equity indicates financial instability, which can limit PTC's ability to raise capital and invest in growth opportunities, posing long-term risks.

Translarna Revenue Decline

The decline in Translarna revenue due to nonrenewal of EU marketing authorization impacts PTC's revenue base and could affect future financial performance.

Emflaza Market Erosion

Increased competition from generics erodes Emflaza's market share, potentially reducing revenue and impacting PTC's competitive position in the long term.

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 generates revenue primarily through the sale of its pharmaceutical products, which include Translarna™ and Emflaza™. The company operates a direct-to-pharmacy distribution model, allowing it to maintain control over the sales process and pricing strategies. Additionally, PTC may receive revenue from licensing agreements and collaborations with other pharmaceutical companies, which can include milestone payments and royalties based on product sales. The company also invests in research and development to expand its product pipeline, which can lead to new revenue opportunities as new therapies are developed and brought to market. Collaborations with academic institutions and other biotech firms may also contribute to revenue through shared research endeavors and joint ventures.

PTC Therapeutics Financial Statement Overview

Summary

PTC Therapeutics has shown a significant turnaround with strong revenue growth and improved profitability. The company has effectively managed its cash flows, leading to positive free cash flow. However, challenges remain with the negative equity position and high liabilities.

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	TTM	Dec 2024	Dec 2023	Dec 2022	Dec 2021	Dec 2020
Income Statement
Total Revenue	1.76B	806.78M	937.82M	698.80M	538.59M	380.77M
Gross Profit	1.76B	749.38M	649.70M	654.12M	506.26M	361.82M
EBITDA	838.50M	-120.46M	-330.34M	-367.78M	-368.18M	-297.00M
Net Income	629.17M	-363.30M	-626.60M	-559.02M	-523.90M	-438.16M
Balance Sheet
Total Assets	2.63B	1.71B	1.90B	1.71B	1.94B	2.21B
Cash, Cash Equivalents and Short-Term Investments	1.99B	1.14B	876.74M	410.70M	773.38M	1.10B
Total Debt	388.17M	2.47B	2.23B	1.46B	1.27B	420.44M
Total Liabilities	2.84B	2.80B	2.71B	2.05B	1.94B	1.73B
Stockholders Equity	-206.55M	-1.10B	-818.55M	-347.09M	1.44M	481.98M
Cash Flow
Free Cash Flow	749.48M	-114.19M	-279.04M	-509.11M	-336.66M	-250.05M
Operating Cash Flow	704.77M	-107.69M	-158.42M	-356.65M	-251.33M	-194.07M
Investing Cash Flow	-381.33M	44.18M	-176.74M	290.18M	219.18M	-561.55M
Financing Cash Flow	22.21M	255.87M	646.40M	167.95M	20.88M	668.72M

PTC Therapeutics Technical Analysis

• Technical Analysis Sentiment: Positive
• Last Price: 68.11
• 50DMA: Positive
• 100DMA: Positive
• 200DMA: Positive
• MACD: Positive
• 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 68.11 is above the 20-day moving average (MA) of 66.47, above the 50-day MA of 60.86, and above the 200-day MA of 51.84, indicating a bullish trend. The MACD of 1.89 indicates Positive momentum. The RSI at 64.04 is Neutral, neither overbought nor oversold. The STOCH value of 53.90 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	―	$5.41B	9.77	―	―	96.00%	―
TGTX	―	$5.40B	90.91	26.63%	―	30.96%	-38.30%
ACLX	―	$4.77B	―	-42.75%	―	-60.63%	-216.87%
MRUS	―	$7.20B	―	-48.54%	―	59.77%	-92.92%
CRSP	―	$5.56B	-11.27	-25.34%	―	-81.18%	-69.19%
Sector *	―	$7.86B	-0.30	-43.30%	2.27%	22.53%	-2.21%
PCVX	―	$5.65B	―	-21.65%	―	―	11.84%

PTC Therapeutics Corporate Events

PTC Therapeutics Completes Study on Vatiquinone for Friedreich Ataxia in Young Children

PTC Therapeutics has completed a clinical study titled ‘An Open-Label Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Vatiquinone in Children With Friedreich Ataxia Younger Than 7 Years of Age.’ The study aimed to assess the pharmacokinetics and safety of vatiquinone in young participants with Friedreich ataxia, a rare genetic disorder. This research is significant as it targets a vulnerable age group, potentially offering new treatment avenues.

PTC Therapeutics Receives FDA Complete Response Letter

On August 19, 2025, PTC Therapeutics announced that the FDA issued a Complete Response Letter regarding their New Drug Application for vatiquinone, a treatment for Friedreich’s ataxia. The FDA indicated that substantial evidence of efficacy was not demonstrated and requested an additional study. PTC plans to meet with the FDA to address these issues, reflecting the potential impact on their operations and market positioning.

The most recent analyst rating on (PTCT) stock is a Hold with a $51.00 price target. To see the full list of analyst forecasts on PTC Therapeutics stock, see the PTCT Stock Forecast page.

PTC Therapeutics’ Earnings Call Highlights Sephience Success

PTC Therapeutics’ recent earnings call painted a picture of a company on the upswing, buoyed by the successful approval and launch of Sephience. This development is expected to significantly bolster future revenues. Despite facing challenges such as market erosion for Emflaza and uncertainties surrounding the vatiquinone NDA, the overall sentiment was positive, with the Sephience launch being a major highlight.

PTC Therapeutics Reports Q2 2025 Financial Results

PTC Therapeutics, Inc. is a global biopharmaceutical company focused on the discovery, development, and commercialization of medicines for rare disorders, leveraging its scientific expertise and global infrastructure to provide treatments for patients with unmet medical needs.

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’ Future at Risk: Navigating Operational and Financial Challenges

PTC Therapeutics faces a significant business risk as outlined in their Annual Report on Form 10-K for the year ended December 31, 202. This risk could potentially impact their operational capabilities and financial performance. Stakeholders should closely monitor how the company addresses these challenges to mitigate potential negative outcomes. The company’s ability to navigate this risk will be crucial for its future stability and growth.

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.