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PTCT

PTC Therapeutics, Inc.

PTCT

PTC Therapeutics, Inc. • NASDAQ
$85.99 ▼ -0.30% (-0.26)
Market Cap $6.90 B
52w High $87.50
52w Low $35.95
Dividend Yield 0%
P/E 10
Volume 1.11M
Outstanding Shares 80.29M

Income Statement

Period Revenue Operating Expense Net Income Net Profit Margin Earnings Per Share EBITDA
Q3-2025 $211.007M ▲ $92.052M ▼ $15.896M ▲ 7.533% ▲ $0.2 ▲ $20.627M ▲
Q2-2025 $178.875M ▼ $202.412M ▲ $-64.849M ▼ -36.254% ▼ $-0.83 ▼ $-33.111M ▼
Q1-2025 $1.176B ▲ $193.009M ▼ $866.562M ▲ 73.681% ▲ $11.09 ▲ $971.141M ▲
Q4-2024 $213.172M ▲ $359.278M ▲ $-65.886M ▲ -30.907% ▲ $-0.85 ▲ $-46.875M ▲
Q3-2024 $196.786M $240.448M $-106.654M -54.198% $-1.39 $-49.896M

Balance Statement

Period Cash & Short-term Total Assets Total Liabilities Total Equity
Q3-2025 $1.707B ▼ $2.644B ▲ $2.799B ▼ $-155.758M ▲
Q2-2025 $1.989B ▼ $2.634B ▼ $2.841B ▼ $-206.546M ▼
Q1-2025 $2.027B ▲ $2.655B ▲ $2.841B ▲ $-185.76M ▲
Q4-2024 $1.14B ▲ $1.705B ▼ $2.803B ▼ $-1.098B ▼
Q3-2024 $1.013B $1.842B $2.897B $-1.054B

Cash Flow Statement

Period Net Income Cash From Operations Cash From Investing Cash From Financing Net Change Free Cash Flow
Q3-2025 $15.896M ▲ $-66.301M ▼ $-289.623M ▲ $15.219M ▲ $-344.769M ▲ $-69.746M ▼
Q2-2025 $-64.849M ▼ $-58.333M ▼ $-422.009M ▼ $3.961M ▼ $-462.793M ▼ $-62.327M ▼
Q1-2025 $866.562M ▲ $870.103M ▲ $-184.36M ▼ $9.41M ▼ $700.546M ▲ $867.571M ▲
Q4-2024 $-65.886M ▲ $-30.004M ▲ $267.712M ▲ $26.471M ▲ $255.526M ▲ $-36.501M ▲
Q3-2024 $-106.654M $-76.992M $-42.675M $-17.627M $-128.752M $-87.768M

Revenue by Products

Product Q4-2024Q1-2025Q2-2025Q3-2025
Collaboration and License Revenue
Collaboration and License Revenue
 $0 ▲$990.00M ▲$0 ▼$10.00M ▲
Product
Product
 $470.00M ▲$150.00M ▼$120.00M ▼$130.00M ▲
Royalty
Royalty
 $140.00M ▲$40.00M ▼$60.00M ▲$70.00M ▲
Manufacturing
Manufacturing
 $0 ▲$0 ▲$0 ▲$0 ▲

Five-Year Company Overview

Income Statement

Income Statement PTC’s revenue has grown steadily over the last five years, showing that its rare-disease products are gaining commercial traction. Gross margins appear strong, which means each dollar of sales is relatively profitable before overhead. However, research, development, and selling costs are still much larger than gross profit, so the company has reported sizable operating and net losses every year. Losses peaked recently and then narrowed somewhat in the latest year, but the business is still far from break-even. The path to profitability will likely depend on how quickly sales from existing and new drugs can scale relative to spending on R&D and commercialization.

Balance Sheet

Balance Sheet The balance sheet shows a company that has invested heavily but now carries a more stretched financial profile. Total assets have stayed fairly stable, and the cash balance has improved compared with a few years ago, offering some near-term breathing room. At the same time, debt has climbed sharply over the period, and shareholder equity has turned clearly negative, reflecting accumulated losses and a high liability burden. This combination means the company is more financially leveraged and has less cushion if results disappoint. Future flexibility will hinge on access to capital markets, partnership funding, and the ability to manage or refinance its debt load.

Cash Flow

Cash Flow PTC’s operations have consistently used, rather than generated, cash, but the cash burn from the core business has eased somewhat in the most recent year. Free cash flow has been negative throughout, with modest capital spending and the main outflow driven by ongoing operating losses. This indicates the company is not yet self-sustaining and has relied on external financing or upfront payments to fund its pipeline and commercial efforts. If new products ramp successfully, operating cash flow could improve; if not, the company may need additional funding sooner. Overall, cash dynamics remain a central risk factor to monitor alongside the clinical and regulatory news flow.

Competitive Edge

Competitive Edge PTC occupies a distinctive niche in biotechnology by focusing on rare, often severe genetic diseases where treatment options are limited. Its expertise in RNA splicing and gene therapy, plus an established global commercial footprint, gives it a differentiated position relative to many smaller biotechs. Orphan drug designations and specialized manufacturing capabilities further strengthen its competitive moat in selected indications. At the same time, it competes with large, well-funded pharmaceutical companies in areas like spinal muscular atrophy and Duchenne muscular dystrophy, and faces the usual orphan-drug challenges around pricing and reimbursement. The company’s reliance on a relatively concentrated portfolio of rare-disease products means that regulatory decisions or competitive shifts in a few key markets can have an outsized impact.

Innovation and R&D

Innovation and R&D Innovation is clearly PTC’s core strength. The company has built multiple technology platforms—RNA splicing, gene therapy, and Bio-e—that feed a broad pipeline across neurological and metabolic rare diseases. Its track record includes helping bring an oral SMA therapy to market and securing approval for a first-of-its-kind direct-to-brain gene therapy, underscoring real scientific depth. Late-stage programs in conditions such as phenylketonuria and Huntington’s disease could be transformational for patients and for the company if they succeed, but they carry the usual clinical and regulatory uncertainties. High R&D spending is a major reason for ongoing losses, yet it is also the main driver of potential long-term value, making disciplined project selection and trial execution especially important.

Summary

PTC Therapeutics is a science-driven rare-disease company that has successfully moved from pure R&D into a multi-product commercial stage, with solid revenue growth and strong underlying margins on its drugs. However, substantial and continuing spending on research and commercialization keeps the company firmly in loss-making territory, and its balance sheet now shows high debt and negative equity, signaling financial strain. The investment story revolves around whether its innovative platforms and late-stage pipeline can mature quickly enough to offset cash burn and support the capital structure. Key uncertainties include clinical trial outcomes, regulatory decisions, competitive dynamics in small but important markets, and access to funding if progress is slower than hoped. Overall, this is a high-science business with meaningful medical potential, but one that currently operates with significant financial and execution risk.

NEWS

November 24, 2025 · 6:01 PM UTC

Sandisk Set to Join S&P 500; Upwork, First Interstate BancSystem, PTC Therapeutics to Join S&P SmallCap 600

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November 20, 2025 · 8:00 AM UTC

PTC Therapeutics to Host R&D Day

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November 4, 2025 · 4:01 PM UTC

PTC Therapeutics Provides Corporate Update and Reports Third Quarter 2025 Financial Results

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October 31, 2025 · 8:00 AM UTC

PTC Therapeutics to Participate at Upcoming Investor Conferences

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October 27, 2025 · 4:30 PM UTC

PTC Therapeutics Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

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