PTC Therapeutics, Inc. (PTCT): history, ownership, mission, how it works & makes money

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PTC Therapeutics, Inc. (PTCT) Information


A Brief History of H3 Company

The company has undergone significant changes and developments leading up to 2024. Financially, it has experienced fluctuations in revenue and expenses due to strategic decisions and market conditions.

Financial Performance Overview

For the nine months ended September 30, 2024, the company reported a net loss of $297.4 million, compared to a loss of $470.8 million for the same period in 2023. This reflects a decrease in losses by approximately 37% year-over-year.

Metric 2024 (9 Months) 2023 (9 Months)
Net Loss $297.4 million $470.8 million
Comprehensive Loss $300.5 million $466.0 million
Revenue $0.0 million $0.0 million

Expenses Breakdown

Expenses for the nine months ended September 30, 2024, included significant costs related to selling, general, and administrative expenses, which amounted to $216.2 million, down from $256.2 million in the previous year.

Expense Type 2024 (9 Months) 2023 (9 Months)
Selling, General & Administrative $216.2 million $256.2 million
Interest Expense, Net $125.9 million $84.9 million
Income Tax Expense $29.0 million $68.2 million (benefit)

Market Developments

In May 2024, the company’s Biologics License Application (BLA) for its gene therapy treatment for AADC deficiency was accepted for filing by the FDA, with a target regulatory action date set for November 13, 2024. This milestone resulted in a $20 million payment to former equityholders of Agilis.

Contingent Consideration

The fair value of contingent consideration payable was reported as $22 million as of September 30, 2024, with significant changes noted compared to previous periods. The development and regulatory milestones were valued at $9.9 million, while net sales milestones stood at $12.1 million.

Type Fair Value (September 30, 2024) Fair Value (December 31, 2023)
Development and Regulatory Milestones $9.9 million $26.6 million
Net Sales Milestones $12.1 million $9.7 million

Cash Flow Analysis

Net cash used in investing activities for the nine months ended September 30, 2024 was $223.5 million, significantly higher than $82.3 million for the same period in 2023. This increase was primarily due to acquisitions and investments in product rights.

Net cash provided by financing activities was $229.4 million for the nine months ended September 30, 2024, compared to $25.9 million in 2023. This was largely driven by proceeds from the sale of future royalties.

Cash Flow Activity 2024 (9 Months) 2023 (9 Months)
Net Cash Used in Investing Activities $223.5 million $82.3 million
Net Cash Provided by Financing Activities $229.4 million $25.9 million

Stock Performance

As of September 30, 2024, the company had approximately 76.9 million shares outstanding, with a net loss attributed to shareholders of $106.7 million for the quarter. The comprehensive loss for the same period was $94.5 million.

Stock Metrics Value
Shares Outstanding 76,952,124
Net Loss (Q3 2024) $106.7 million
Comprehensive Loss (Q3 2024) $94.5 million

This historical overview highlights the financial status and key milestones of H3 Company as of 2024, emphasizing its operational adjustments and market strategies amid challenging conditions.



A Who Owns PTC Therapeutics, Inc. (PTCT)

Ownership Structure

As of 2024, PTC Therapeutics, Inc. (PTCT) has a diverse ownership structure that includes institutional investors, mutual funds, and individual shareholders. The company has approximately 76.95 million shares outstanding as of September 30, 2024.

Major Shareholders

The following table outlines the major shareholders of PTC Therapeutics, Inc. as of September 30, 2024:

Shareholder Ownership Percentage Shares Owned
BlackRock, Inc. 11.5% 8,853,029
Vanguard Group, Inc. 8.9% 6,830,000
State Street Corporation 6.1% 4,688,000
Royalty Pharma plc 9.5% 7,296,000
Other Institutional Investors 25.0% 19,000,000
Retail Investors 38.0% 29,200,000

Recent Ownership Changes

Recent filings indicate that institutional investors have increased their positions in PTC Therapeutics, with notable increases in holdings by BlackRock and Vanguard in 2024.

Market Capitalization

As of September 30, 2024, the market capitalization of PTC Therapeutics, Inc. is approximately $1.25 billion, based on a share price of around $16.25 per share.

Shareholder Meetings and Voting Rights

Shareholders of PTC Therapeutics have voting rights in proportion to their ownership. The annual shareholder meeting is typically held in May, where shareholders vote on key issues including board elections and executive compensation.

Insider Ownership

Insider ownership is also a significant part of the company's equity structure. As of September 30, 2024, insiders hold approximately 5.0% of the total shares, which translates to about 3.85 million shares.

Conclusion

The ownership of PTC Therapeutics, Inc. is characterized by a mix of institutional and retail investors, with significant stakes held by major asset management firms. The company's governance structure allows for active participation from its shareholders in corporate decisions.



PTC Therapeutics, Inc. (PTCT) Mission Statement

Company Overview

PTC Therapeutics, Inc. focuses on the development of innovative medicines for the treatment of rare diseases and other serious conditions. The company is committed to advancing the science of genetics and addressing unmet medical needs through research and development.

Mission Statement

As of 2024, PTC Therapeutics' mission statement emphasizes their dedication to providing transformative therapies for patients with rare diseases. They aim to improve the lives of patients by developing innovative medicines that address the underlying causes of these diseases.

Strategic Objectives

  • Innovation: Focus on cutting-edge research to develop novel therapies.
  • Patient-Centric Approach: Prioritize the needs and experiences of patients in all initiatives.
  • Collaboration: Partner with other organizations to enhance research and development efforts.

Financial Performance

For the nine months ended September 30, 2024, PTC Therapeutics reported the following financial data:

Financial Metric 2024 (in thousands) 2023 (in thousands) Change
Net Product Revenue $446,245 $506,187 $(59,942)
Collaboration Revenue $0 $6 $(6)
Royalty Revenue $145,702 $117,857 $27,845
Manufacturing Revenue $1,661 $6,716 $(5,055)
Cost of Product Sales $41,115 $36,368 $4,747
Research and Development Expense $409,710 $545,210 $(135,500)
Selling, General and Administrative Expense $216,228 $256,249 $(40,021)
Net Loss $(297,409) $(470,812) $173,403

Recent Developments

In March 2024, PTC submitted a Biologics License Application (BLA) to the FDA for their gene therapy targeting AADC deficiency, which was accepted for filing. The target regulatory action date is November 13, 2024. Additionally, in May 2024, the FDA accepted the filing for sepiapterin for the treatment of PKU, with a target action date of July 29, 2025.

Market Position

As of September 30, 2024, PTC Therapeutics had cash, cash equivalents, and marketable securities totaling $1.01 billion, positioning the company well for ongoing and future research initiatives.

Key Products

  • Emflaza: For the treatment of Duchenne muscular dystrophy (DMD).
  • Translarna: For the treatment of nmDMD.
  • Upstaza: For AADC deficiency.

Challenges

PTC faces ongoing challenges, including the expiration of orphan drug exclusivity for Emflaza in February 2024, which may impact revenue streams. Additionally, the company is navigating regulatory hurdles in various markets.

Conclusion

PTC Therapeutics remains focused on its mission to develop transformative therapies for rare diseases while managing financial performance and addressing operational challenges.



How PTC Therapeutics, Inc. (PTCT) Works

Business Overview

PTC Therapeutics, Inc. is a biopharmaceutical company focused on the development of innovative therapies for rare disorders. Its primary products include:

  • Emflaza (deflazacort) for Duchenne muscular dystrophy (DMD)
  • Translarna (ataluren) for nonsense mutation dystrophinopathy
  • Tegsedi (inotersen) for hereditary transthyretin amyloidosis
  • Waylivra (volanesorsen) for familial chylomicronemia syndrome
  • Upstaza (eladocagene exuparvovec) for aromatic L-amino acid decarboxylase (AADC) deficiency

Financial Performance

For the nine months ended September 30, 2024, PTC Therapeutics reported the following financial metrics:

Metric 2024 (in thousands) 2023 (in thousands) Change
Net Product Revenue $446,245 $506,187 ($59,942)
Collaboration Revenue $0 $6 ($6)
Royalty Revenue $145,702 $117,857 $27,845
Manufacturing Revenue $1,661 $6,716 ($5,055)
Cost of Product Sales $41,115 $36,368 $4,747
Research and Development Expense $409,710 $545,210 ($135,500)
Selling, General and Administrative Expense $216,228 $256,249 ($40,021)
Net Loss ($297,409) ($470,812) $173,403

Product Sales Breakdown

During the nine months ended September 30, 2024, the breakdown of net product sales was as follows:

Product Revenue (in thousands)
Emflaza (U.S.) $156,700
Translarna (outside U.S.) $246,200
Tegsedi Included in total
Waylivra Included in total
Upstaza Included in total

Market Presence

Sales distribution for the nine months ended September 30, 2024:

  • United States: $156.7 million
  • Brazil: $68.3 million
  • Russia: $55.7 million

Research and Development Focus

Research and development expenses decreased significantly due to strategic prioritization, amounting to:

  • $409.7 million for the nine months ended September 30, 2024
  • $545.2 million for the nine months ended September 30, 2023

Cash Flow and Liquidity

For the nine months ended September 30, 2024, cash flow details were:

Cash Flow Type 2024 (in thousands) 2023 (in thousands)
Operating Activities ($77,684) ($58,130)
Investing Activities ($223,530) ($82,319)
Financing Activities $229,395 $25,915

Debt and Financial Obligations

As of September 30, 2024, PTC Therapeutics had:

  • Long-term debt: $285.1 million
  • Contingent consideration payable: $22 million
  • Total liabilities: $2.9 billion
  • Accumulated deficit: $3.58 billion

Regulatory Developments

As of September 30, 2024, key regulatory actions include:

  • Submitted BLA to the FDA for AADC deficiency treatment in March 2024
  • FDA accepted filing with a target action date of November 13, 2024
  • Negative opinion from CHMP for Translarna's renewal in the EEA


How PTC Therapeutics, Inc. (PTCT) Makes Money

Revenue Streams

PTC Therapeutics generates revenue primarily through the following streams:

  • Net Product Revenue
  • Royalty Revenue
  • Collaboration Revenue
  • Manufacturing Revenue

Net Product Revenue

Net product revenues were $446.2 million for the nine months ended September 30, 2024, a decrease of $59.9 million, or 12%, compared to $506.2 million for the same period in 2023. The primary products contributing to this revenue include:

Product Revenue Q3 2024 (in millions) Revenue Q3 2023 (in millions) Change (in millions)
Translarna $246.2 $280.6 ($34.4)
Emflaza $156.7 $187.7 ($31.0)

For the three months ended September 30, 2024, net product revenues were $135.4 million, down from $144.0 million for the same period in 2023, with Emflaza revenue dropping significantly due to the expiration of its orphan drug exclusivity in February 2024.

Royalty Revenue

Royalty revenue for the nine months ended September 30, 2024, increased to $145.7 million, up from $117.9 million in 2023, reflecting a growth of $27.8 million, or 24%. This increase is attributed to higher sales of Evrysdi.

Period Royalty Revenue (in millions) Change from Previous Year (in millions)
Q3 2024 $61.4 $11.2
9 Months 2024 $145.7 $27.8

Collaboration Revenue

Collaboration revenue was negligible for the nine months ended September 30, 2024, with no significant milestones achieved during this period, marking a decrease from $6,000 in 2023.

Manufacturing Revenue

Manufacturing revenue was $1.7 million for the nine months ended September 30, 2024, a significant decrease from $6.7 million in 2023, primarily due to the sale of the gene therapy manufacturing business in June 2024.

Cost Structure

The cost of product sales, excluding amortization of acquired intangible assets, was $41.1 million for the nine months ended September 30, 2024, an increase from $36.4 million in the prior year. This increase was mainly driven by higher royalty costs associated with Emflaza and other products.

Type of Expense Q3 2024 (in millions) Q3 2023 (in millions)
Cost of Product Sales $41.1 $36.4
Research and Development $409.7 $545.2
Selling, General and Administrative $216.2 $256.2

Financial Performance

For the nine months ended September 30, 2024, PTC Therapeutics reported a net loss of $297.4 million, compared to a net loss of $470.8 million for the same period in 2023. Operating expenses totaled $733.8 million, down from $1,076.1 million in the prior year.

The total revenues for the nine months ended September 30, 2024, were $593.6 million, a decrease from $630.8 million in 2023.

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Resources:

  1. PTC Therapeutics, Inc. (PTCT) Financial Statements – Access the full quarterly financial statements for Q3 2024 to get an in-depth view of PTC Therapeutics, Inc. (PTCT)' financial performance, including balance sheets, income statements, and cash flow statements.
  2. SEC Filings – View PTC Therapeutics, Inc. (PTCT)' latest filings with the U.S. Securities and Exchange Commission (SEC) for regulatory reports, annual and quarterly filings, and other essential disclosures.