PTC Therapeutics (PTCT) AI Stock Analysis

• Market Cap: $5.53B
• Dividend Yield: N/A
• Average Volume (3M): 1.13M
• Price to Earnings (P/E): 8.2
• Beta (1Y): 1.25
• Revenue Growth: 97.54%
• EPS Growth: N/A
• Country: US
• Employees: 939
• Sector: Healthcare
• Sector Strength: 45
• Industry: Biotechnology
• EPS (TTM): -1.67
• Shares Outstanding: 80,288,310
• 10 Day Avg. Volume: 1,036,636
• 30 Day Avg. Volume: 1,129,708
• PEG Ratio: -0.03
• Price to Book (P/B): -29.43
• Price to Sales (P/S): 3.49
• P/FCF Ratio: 8.60
• Enterprise Value/Market Cap: 0.91
• Enterprise Value/Revenue: 2.92
• Enterprise Value/Gross Profit: 3.21
• Enterprise Value/Ebitda: 5.67
• 1Y Price Target: $88.20
• Price Target Upside: 25.11% Upside
• Rating Consensus: Moderate Buy
• Number of Analyst Covering: 12
• EPS Forecast (FY): -1.18
• Revenue Forecast (FY): $963.97M

• Rating: 65 Neutral
• Price Target: $76.00 ▲(7.80% Upside)
• Action: Reiterated Date: 02/23/26
• Date: Action: Reiterated 02/23/26

The score is supported by a sharp TTM profitability/FCF turnaround and constructive earnings-call guidance (Sephience-driven growth outlook and stronger liquidity), plus a low earnings multiple. These positives are tempered by balance-sheet weakness (negative equity) and soft technicals (below key moving averages with negative MACD), which increase near-term risk despite improving fundamentals.

Positive Factors

Cash generation & liquidity

PTC's TTM operating and free cash flow turnaround and near parity of FCF to net income (~0.98) show strong cash conversion. This durable cash generation, plus a much larger cash balance, underpins runway for launches, R&D and optionality to reach cash‑flow breakeven in 2026.

High profitability margins

Exceptionally high gross and operating margins reflect a scalable product cost structure typical of specialty pharmaceuticals. Sustained margin levels support durable free cash flow generation and funding of pipeline programs, assuming the product mix and pricing dynamics remain intact.

Sephience commercial momentum

Rapid global approvals, early uptake (946 on therapy) and strong initial sales demonstrate repeatable commercial execution and payer acceptance. Durable launch traction reduces commercialization risk for Sephience and supports the company's 2026 product revenue growth trajectory.

Negative Factors

Negative shareholder equity

Persistent negative equity constrains conventional leverage metrics and reduces balance sheet resilience. Even with lower absolute debt versus prior years, negative equity magnifies funding risk for expensive trials or commercial scale‑ups and increases reliance on monetizations or external financing.

Evrysdi royalty monetization reduces recurring cash

Monetizing Evrysdi provided near‑term liquidity but materially reduces future predictable royalties, shifting revenue dependence to product sales and pipeline milestones. This lowers recurring cash visibility and increases execution risk if Sephience or other launches underperform.

DMD franchise pressure (generics)

Growing generic penetration in the DMD franchise erodes a historically durable revenue base, forcing PTC to replace income with newer launches and pipeline successes. That structural decline increases revenue volatility until newer products scale globally.

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 both commercially available drugs and those in various stages of clinical trials. The company's key revenue streams come from product sales, licensing agreements, and collaborations with other pharmaceutical companies. PTC has established significant partnerships with organizations such as the SMA Foundation and other biotech firms to enhance its research capabilities and expand its product pipeline. Additionally, the company benefits from grants and funding aimed at supporting its research in rare diseases, which further contributes to its overall earnings.

PTC Therapeutics Financial Statement Overview

Summary

Strong TTM profitability and cash generation (very high gross margin, solid EBIT/net margins, and strong operating/free cash flow with good conversion). The key offsets are elevated balance-sheet risk from negative equity and some softness in top-line momentum (recent revenue and FCF slightly down), raising sustainability questions if one-offs fade.

Income Statement

TTM (Trailing-Twelve-Months) results show a sharp profitability inflection: revenue of ~$1.73B with very strong gross profitability (~91% gross margin) and robust operating profitability (~49% EBIT margin), driving a solid net margin (~42%). This is a major improvement versus 2020–2024, which were consistently loss-making with negative operating and net margins. The key offset is growth: TTM revenue declined (~-2.7%) and 2024 revenue also dipped, suggesting the earnings surge may be mix/event-driven rather than a clean, steady demand expansion.

Balance Sheet

Balance sheet risk remains elevated. Stockholders’ equity is negative in TTM and across recent years, which weakens financial flexibility and makes leverage harder to interpret in a conventional way. While total debt in TTM (~$492M) is far lower than 2024 (~$2.47B), the company still carries meaningful leverage against a negative equity base, leaving it more exposed if profitability normalizes lower or if funding needs re-emerge.

Cash Flow

Cash generation improved materially in TTM (Trailing-Twelve-Months), with strong operating cash flow (~$711M) and free cash flow (~$699M), a dramatic turnaround from negative operating and free cash flow in 2020–2024. Free cash flow broadly tracks reported earnings (free cash flow to net income ~0.98), supporting earnings quality. The main weakness is momentum: free cash flow was slightly down (~-2.1% growth), and the multi-year history shows prior cash burn, so durability through cycles remains a key watch item.

Breakdown	Dec 2025	Dec 2024	Dec 2023	Dec 2022	Dec 2021
Income Statement
Total Revenue	1.73B	806.78M	937.82M	698.80M	538.59M
Gross Profit	1.66B	749.38M	872.34M	654.12M	506.26M
EBITDA	895.32M	-120.46M	-330.34M	-367.78M	-368.18M
Net Income	682.64M	-363.30M	-626.60M	-559.02M	-523.90M
Balance Sheet
Total Assets	2.90B	1.71B	1.90B	1.71B	1.94B
Cash, Cash Equivalents and Short-Term Investments	1.95B	1.14B	876.74M	410.70M	773.38M
Total Debt	398.70M	2.47B	2.23B	1.46B	1.27B
Total Liabilities	3.10B	2.80B	2.71B	2.05B	1.94B
Stockholders Equity	-205.31M	-1.10B	-818.55M	-347.09M	1.44M
Cash Flow
Free Cash Flow	702.34M	-181.94M	-279.04M	-509.11M	-336.66M
Operating Cash Flow	711.20M	-107.69M	-158.42M	-356.65M	-251.33M
Investing Cash Flow	-861.98M	44.18M	-176.74M	290.18M	219.18M
Financing Cash Flow	331.09M	255.87M	646.40M	167.95M	20.88M

PTC Therapeutics Technical Analysis

• Technical Analysis Sentiment: Negative
• Last Price: 70.50
• 50DMA: Negative
• 100DMA: Negative
• 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 Negative. The current price of 70.5 is below the 20-day moving average (MA) of 72.82, below the 50-day MA of 74.90, and above the 200-day MA of 61.87, indicating a neutral trend. The MACD of -1.56 indicates Positive momentum. The RSI at 39.99 is Neutral, neither overbought nor oversold. The STOCH value of 48.57 is Neutral, not indicating any strong overbought or oversold conditions. Overall, these indicators collectively point to a Negative sentiment for PTCT.

PTC Therapeutics Peers Comparison

Name	Overall Rating	Market Cap	P/E Ratio	ROE	Dividend Yield	Revenue Growth	EPS Growth
PTCT	65 Neutral	$5.53B	8.17	―	―	97.54%	―
TGTX	62 Neutral	$4.64B	10.60	111.96%	―	100.88%	―
PCVX	56 Neutral	$7.97B	-11.65	-20.83%	―	―	-5.21%
Sector *	51 Neutral	$7.86B	-0.30	-43.30%	2.27%	22.53%	-2.21%
CRSP	47 Neutral	$5.14B	-8.39	-30.18%	―	-81.10%	-99.21%
ACLX	40 Underperform	$3.99B	-16.98	-47.18%	―	-76.96%	-425.41%

PTC Therapeutics Corporate Events

PTC Therapeutics Tops 2025 Revenue Guidance, Boosts Cash

On Feb. 19, 2026, PTC Therapeutics reported that full-year 2025 product and royalty revenue reached $831 million, exceeding guidance, driven by the successful global launch of Sephience, which generated $111 million since its second-half 2025 debut and contributed to total 2025 revenues of $1.73 billion. The company strengthened its balance sheet to $1.95 billion in cash by year-end, boosted by a $998.4 million Novartis collaboration on votoplam and the sale of remaining Evrysdi royalties, while advancing its pipeline with FDA alignment on a Phase 3 Huntington’s trial and receiving feedback that an additional study is needed to resubmit vatiquinone in Friedreich’s ataxia, as it guides investors with 2026 product revenue expectations of $700 million to $800 million and higher R&D and SG&A spending.

The most recent analyst rating on (PTCT) stock is a Buy with a $124.00 price target. To see the full list of analyst forecasts on PTC Therapeutics stock, see the PTCT Stock Forecast page.

PTC Therapeutics Withdraws Translarna NDA After FDA Feedback

On February 12, 2026, PTC Therapeutics withdrew its New Drug Application resubmission for Translarna (ataluren), a protein restoration therapy for nonsense mutation Duchenne muscular dystrophy, after the U.S. Food and Drug Administration indicated the data were unlikely to meet its threshold for substantial evidence of effectiveness. The decision halts PTC’s two-decade effort to secure U.S. approval for Translarna in this rare and fatal neuromuscular disease, underscoring regulatory hurdles in rare disease drug development and potentially affecting patient access and the company’s positioning in the DMD treatment landscape.

The most recent analyst rating on (PTCT) stock is a Hold with a $75.00 price target. To see the full list of analyst forecasts on PTC Therapeutics stock, see the PTCT Stock Forecast page.

PTC Therapeutics Posts Strong 2025 Results, Updates 2026 Outlook

On January 12, 2026, PTC Therapeutics reported preliminary unaudited 2025 results showing approximately $823.4 million in total product and royalty revenue, above prior guidance, including $587.8 million in product revenue and $112.1 million from its newly launched Sephience, which generated $92.5 million in fourth-quarter sales alone and reached 946 patients worldwide by year-end. The company ended 2025 with about $1.94 billion in cash and securities, completed the sale of its remaining Evrysdi royalty stream for up to $300 million in consideration while retaining a potential $150 million milestone, advanced its pipeline with FDA alignment on a Phase 3 Huntington’s disease trial for votoplam and continued regulatory engagement on vatiquinone and Translarna, and issued 2026 guidance calling for $700 million to $800 million in product revenue and GAAP R&D and SG&A expenses of $775 million to $815 million, underscoring both strong launch momentum and sustained investment in late-stage programs as it moves toward cash-flow breakeven.

The most recent analyst rating on (PTCT) stock is a Buy with a $90.00 price target. To see the full list of analyst forecasts on PTC Therapeutics stock, see the PTCT Stock Forecast page.

PTC Therapeutics Fully Monetizes Evrysdi Royalty Interest

On December 29, 2025, PTC Therapeutics, Inc. entered into a second amendment to its Amended and Restated Royalty Purchase Agreement with Royalty Pharma Investments 2019 ICAV, under which PTC sold its remaining economic interest in royalties on worldwide net sales of Roche’s Evrysdi (risdiplam) and related products. In exchange for its retained royalty stake—representing 9.5111% of the royalty before a specified 2020 royalty cap is met and 16.6666% thereafter—PTC received $240 million in upfront cash and became eligible for up to three additional $20 million payments tied to future annual royalty payment thresholds for 2027, 2028 and 2029, resulting in Royalty Pharma now owning 100% of the Evrysdi royalty stream and PTC fully monetizing this asset to bolster its cash resources but relinquishing future royalty income from the product.

The most recent analyst rating on (PTCT) stock is a Buy with a $93.00 price target. To see the full list of analyst forecasts on PTC Therapeutics stock, see the PTCT Stock Forecast page.