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PTCT on Nasdaq

PTC Therapeutics

Logo: PTC Therapeutics, Public domain, via Wikimedia Commons5

Pharmaceutical company

PTC Therapeutics, Inc. is a US pharmaceutical company focused on the development of orally administered small molecule drugs and gene therapy which regulate gene expression by targeting post-transcriptional control (PTC) mechanisms in orphan diseases. In September 2009, PTC entered into an agreement with Roche for the development of orally bioavailable small molecules for central nervous system diseases. PTC acquired the Bio-e platform in 2019.3 Its shares trade on Nasdaq under the symbol PTCT.1 Wikipedia text CC BY-SA 4.0

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Last updated Oct 21, 2026.

Overview

Jan 1, 1998Founded1

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  • TickerPTCT on Nasdaq1
  • Industrypharmaceutical industry1
  • HeadquartersSouth Plainfield1
  • Websitewww.ptcbio.com
  • Legal formDelaware corporation1
  • Incorporated inDE6
  • Business address500 Warren Corporate Center Drive, Warren, Nj 070596
  • Filer categoryLarge accelerated filer6
  • LEI549300UJLWOIWFDGB3184
  • StatusActive4
  • LEI statusIssued and current4
  • Registered addressc/o CORPORATION SERVICE COMPANY, 251 LITTLE FALLS DRIVE, 19808 Wilmington, United States4
  • Registered withDivision of Corporations, Department of State, 28782754

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PTC Therapeutics, Inc. is a US pharmaceutical company focused on the development of orally administered small molecule drugs and gene therapy which regulate gene expression by targeting post-transcriptional control (PTC) mechanisms in orphan diseases.3

Financials

From the company’s reports to the SEC. Every number links to the filing it came from.

Key numbers, fiscal 2025

50.1%Operating margin9
39.4%Net margin9
114.5%Revenue growth, 20259
$702.3MFree cash flow9

What sells

Revenue by product, business segment and region, as the company reports it to the SEC.

Products and services

Collaboration and License Revenue brought in 55% of the revenue PTC Therapeutics broke down this way in fiscal 2025.

  1. Collaboration and License Revenue$998.4M955%up 328307% from 2024
  2. Royalty$244.2M913%up 20% from 2024
  3. Translarna1(Member)$235.3M913%down 27% from 2024
  4. Emflaza$146.4M98%down 29% from 2024
  5. Sephience$111.2M96%
  6. Upstaza and Kebilidi$56.6M93%up 235% from 2024
  7. Other Products$37.3M92%up 1% from 2024
Products and services by year
Products and services202320242025
Collaboration and License Revenue$100.0M$304.0K$998.4M
Royalty$168.9M$203.9M$244.2M
Translarna1(Member)$332.8M$321.1M$235.3M
Emflaza$255.1M$207.2M$146.4M
Sephience$111.2M
Upstaza and Kebilidi$18.8M$16.9M$56.6M
Other Products$31.5M$36.9M$37.3M

Regions

United States brought in 31% of the revenue PTC Therapeutics broke down this way in fiscal 2025.

  1. United States$252.2M931%up 22% from 2024
  2. Rest of the world$235.9M929%down 40% from 2024
  3. All Other countries Other Than Russia, Brazil And united States$168.4M920%down 15% from 2024
  4. RU$100.4M912%down 5% from 2024
  5. Brazil$65.7M98%down 9% from 2024
Regions by year
Regions202320242025
United States$255.1M$207.2M$252.2M
Rest of the world$393.7M$235.9M
All Other countries Other Than Russia, Brazil And united States$238.5M$197.5M$168.4M
RU$86.0M$105.4M$100.4M
Brazil$58.6M$72.1M$65.7M

Where the money goes

Where each dollar of revenue went, fiscal 2025

  • Research and development26¢$455.2M
  • Selling, general and admin20¢$347.1M
  • Other costs13¢$231.6M
  • Income tax1¢$14.0M
  • Profit39¢$682.6M

Out of $1.7B in revenue.9

Where each dollar of operating cash went, fiscal 2025

  • Capital spending1¢$8.9M
  • Kept or used elsewhere99¢$702.3M

Out of $711.2M of cash from operations.9

Financial statements

Revenue101112139$1.7B in 2025+464% since 2019
0$500.0M$1.0B$1.5B$2.0B2019: $307.0M$307.0M20192020: $380.8M$380.8M2020+24%2021: $538.6M$538.6M2021+41%2022: $698.8M$698.8M2022+30%2023: $937.8M$937.8M2023+34%2024: $806.8M$806.8M2024−14%2025: $1.7B$1.7B2025+115%
Operating income101112139$866.9M in 2025
-$500.0M0$500.0M$1.0B2019: -$241.2M-$241.2M20192020: -$431.8M-$431.8M2020+79%2021: -$374.4M-$374.4M2021−13%2022: -$447.4M-$447.4M2022+19%2023: -$439.5M-$439.5M2023−2%2024: -$302.6M-$302.6M2024−31%2025: $866.9M$866.9M2025
Net income101112139$682.6M in 2025
-$1.0B-$500.0M0$500.0M$1.0B2019: -$251.6M-$251.6M20192020: -$438.2M-$438.2M2020+74%2021: -$523.9M-$523.9M2021+20%2022: -$559.0M-$559.0M2022+7%2023: -$626.6M-$626.6M2023+12%2024: -$363.3M-$363.3M2024−42%2025: $682.6M$682.6M2025

Every line of the income statement as filed: the last 3 fiscal years, latest first.9

Period ending2025-12-312024-12-312023-12-31
Total revenues$1.7B$806.8M$937.8M
Cost of product, collaboration and license sales, excluding amortization of acquired intangible assets$47.0M$57.4M$65.5M
Amortization of acquired intangible assets$24.7M$60.7M$222.6M
Research and development$455.2M$534.5M$666.6M
Selling, general and administrative$347.1M$300.9M$332.5M
Change in the fair value of contingent consideration-$800.0K-$4.5M-$127.7M
Intangible asset impairment$159.5M$217.8M
Tangible asset impairment and (gains) losses on transactions, net-$9.6M$750.0K
Total operating expenses$863.7M$1.1B$1.4B
Income (loss) from operations$866.9M-$302.6M-$439.5M
Interest expense, net-$152.2M-$167.0M-$129.2M
Other (expense) income, net-$18.1M$6.5M$10.1M
Gain on sale of priority review voucher$99.9M
Loss on extinguishment of debt-$137.6M
Income (loss) before income tax (expense) benefit$696.6M-$363.1M-$696.1M
Income tax (expense) benefit-$14.0M-$176.0K$69.5M
Net income (loss) attributable to common stockholders$682.6M-$363.3M-$626.6M
Basic (in shares)79.5M shares76.8M shares74.8M shares
Diluted (in shares)88.3M shares76.8M shares74.8M shares
Basic$8.58-$4.73-$8.37
Diluted$7.78-$4.73-$8.37

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Corporate structure

PTC Therapeutics sits at the top of its group. 7 entities, 2 countries on record. Switch between the flow chart and the network, and select any entity to see its details.

Major holders of PTC Therapeutics

  • PTC Therapeutics South Plainfield, New Jersey, United States
    • ClearPoint Neuro, Inc. Stake · Solana Beach, California
    • PTC Therapeutics GT, LLC Subsidiary · Delaware, United States
    • PTC Therapeutics HD, Inc. Subsidiary · Delaware, United States
    • PTC THERAPEUTICS INTERNATIONAL LIMITED Subsidiary · Dublin, Ireland
    • PTC Therapeutics MP, Inc. Subsidiary · Delaware, United States
    • PTC Therapeutics US, Inc. Subsidiary · Delaware, United States

Built from parent and subsidiary links from GLEIF, the Global Legal Entity Identifier Foundation (CC0); subsidiaries listed in Exhibit 21 of annual reports filed with the SEC; major holders from Schedules 13D and 13G; stakes of 5% or more it reported on Schedules 13D and 13G (1 companies). Each entity in the chart links to its source.

What it owns

What PTC Therapeutics reports owning in its annual reports to the SEC: buildings, land and equipment, real estate and locations, investments, intangible assets and inventory, at the end of its latest fiscal year and a year before.

Property and equipment

ItemLatestA year beforeChange
Property and equipment, before depreciation$102.4M9 Dec 31, 2025$94.3M9 Dec 31, 2024+8.5%
Accumulated depreciation$47.0M9 Dec 31, 2025$33.4M9 Dec 31, 2024+40.9%
Property and equipment, net$55.4M9 Dec 31, 2025$61.0M9 Dec 31, 2024−9.2%
Operating-lease assets (leased space)$80.1M9 Dec 31, 2025$56.7M9 Dec 31, 2024+41.2%

Investments

ItemLatestA year beforeChange
Cash and cash equivalents$984.6M9 Dec 31, 2025$779.7M9 Dec 31, 2024+26.3%
Marketable securities, current$960.7M9 Dec 31, 2025$360.0M9 Dec 31, 2024+166.9%
Debt securities available for sale$929.1M9 Dec 31, 2025$331.0M9 Dec 31, 2024+180.7%

Intangibles and goodwill

ItemLatestA year beforeChange
Goodwill$82.3M9 Dec 31, 2025$82.3M9 Dec 31, 20240%
Intangible assets, net (excluding goodwill)$388.8M9 Dec 31, 2025$118.8M9 Dec 31, 2024+227.2%
Indefinite-lived intangibles (excluding goodwill)$09 Dec 31, 2025$09 Dec 31, 2024
Finite-lived intangibles, net$388.8M9 Dec 31, 2025$118.8M13 Dec 31, 2024+227.2%

Inventory

ItemLatestA year beforeChange
Inventory, total$79.6M9 Dec 31, 2025$23.2M9 Dec 31, 2024+243.4%
Raw materials$2.3M9 Dec 31, 2025$2.5M9 Dec 31, 2024−9.7%
Work in process$55.7M9 Dec 31, 2025$12.2M9 Dec 31, 2024+355.8%
Finished goods$21.7M9 Dec 31, 2025$8.4M9 Dec 31, 2024+156.8%

Properties, in its own words

Our principal facility consists of approximately 180,000 square feet of office space and shell condition, modifiable space located at 500 Warren Corporate Center Drive in Warren, New Jersey, that we occupy under a lease that expires in 2039. The rental term for such facility commenced on June 1, 2022, with an initial term of seventeen years followed by three consecutive five-year renewal periods at our option. Additionally, we lease 103,000 square feet of laboratory and office space in Bridgewater, New Jersey. The rental term for such facility commenced on May 1, 2020 with an initial term of seven years and two consecutive five year renewal periods at our option. We lease approximately 11,500 square feet of office space in Dublin, Ireland, that we occupy under a lease that expires in 2034. Additionally, we lease approximately 5,000 square feet of office space in Sao Paulo, Brazil, that we occupy under a lease that expires in 2028. We also lease additional laboratory and office space in the U.S. and other countries to support our operations as a global organization, but these leases are not material to us.

Item 2 of the 10-K filed Feb 19, 2026.17

Leadership

Who has led PTC Therapeutics, and when. Pick a name to see everywhere that person has worked.

Chief executive

  1. Stuart Walter Peltz2013–2023
  2. Eric Pauwels2024–2025

President

  1. Claudia De Oliveira Ribeiro Hirawat2013–2015

Chief financial officer

  1. Shane William Charles Kovacs2013–2017
  2. Emily Luisa Hill2019–2023
  3. Pierre Gravier2023–now

Chief operating officer

  1. Marcio Souza2017–2020
200020052010201520202025
Board members and other officers (29)

Named in SEC Form D filings

People the company listed in its private-offering notices.

  • Dr. Klein is the Chief Executive Officer and the Director of the Development Committee of the Issuer.Matthew B. Klein, M.D. named May 8, 202352
  • Dr. Almstead is the Chief Technical Operations Officer of the Issuer.Neil B. Almstead, Ph.D named May 8, 202352
  • DirectorAllan D. Jacobson, Ph.D. named May 8, 202352
  • DirectorGlenn D. Steele Jr., M.D., Ph.D. named May 8, 202352
  • DirectorJerome B. Zeldis, M.D., Ph.D. named May 8, 202352
  • DirectorStepanie S. Okey named May 8, 202352

Products and brands

In 2017, PTC acquired Emflaza (deflazacort) from Marathon Pharmaceuticals. PTC also owns Translarna, (Ataluren) marketed for nonsense mutation Duchenne muscular dystrophy. Together, the two products generated revenues of 174 million dollars and 260 million dollars in 2017 and 2018 respectively.

PTC has the commercialization rights for WAYLIVRA (volanesorsen) in Latin America.

In 2018, PTC acquired Agilis Biotherapeutics and a gene therapy candidate, GT-AADC, with its compelling clinical data in treating aromatic L-amino acid decarboxylase (AADC) deficiency. AADC deficiency is a rare CNS disorder arising from reductions in the enzyme AADC that result from mutations in the dopa decarboxylase (DDC) gene. As of 2024, the AAV based treatment for AADC deficiency has been approved by the FDA and commercialized in the United States as Kebilidi. It is the first FDA-approved gene therapy treatment for AADC deficiency. This same product was granted EMA approval for use in the European Union under the name Upstaza.

From Wikipedia’s article on PTC Therapeutics, shared under CC BY-SA 4.0.7

Subsidiaries and ownership

Subsidiaries (Exhibit 21)

  • PTC Therapeutics GT, LLC Delaware8
  • PTC Therapeutics HD, Inc. Delaware8
  • PTC Therapeutics International Limited Ireland8
  • PTC Therapeutics MP, Inc. Delaware8
  • PTC Therapeutics US, Inc. Delaware8

The subsidiaries the company lists in Exhibit 21 of its annual report, as filed.

Former names

  • PTC THERAPEUTICS INCJan 2, 2004 to Oct 12, 20056

Timeline

  1. Sep 21, 20268-K Regulation FD disclosure; Other events53
  2. Aug 28, 20268-K Entered a material agreement54
  3. Aug 13, 20268-K Regulation FD disclosure; Other events55
  4. Jul 30, 20268-K Results of operations; Executive or director change; Regulation FD disclosure56
  5. Jun 18, 20268-K Entered a material agreement; New debt or obligation; Unregistered sale of shares57
  6. Jun 16, 20268-K Other events58
  7. Jun 9, 20268-K Executive or director change59
  8. Jun 5, 20268-K Shareholder vote60
  9. May 7, 20268-K Results of operations; Regulation FD disclosure61
  10. Apr 28, 20268-K Regulation FD disclosure62
  11. Mar 25, 20268-K Executive or director change; Regulation FD disclosure63
  12. Feb 19, 20268-K Results of operations; Regulation FD disclosure64
  13. Apr 19, 2024New chief executive Eric Pauwels becomes CEO18
  14. Jun 20, 2013Went public Listed on NASDAQ1
  15. Jun 19, 2013New chief executive Stuart Walter Peltz becomes CEO19
  16. Jan 1, 1998Founded Founded1

Calendar

Coming up

  1. Oct 29, 2026 · After the closeReports results for the quarter ending Sep/2026 A year earlier it reported $0.20 a share, on Nov 4, 2025.65

When it reported results

  1. Jul 30, 2026Reported quarterly or annual results56
  2. May 7, 2026Reported quarterly or annual results61
  3. Feb 19, 2026Reported quarterly or annual results64
  4. Jan 12, 2026Reported quarterly or annual results66
  5. Nov 4, 2025Reported quarterly or annual results67
  6. Aug 7, 2025Reported quarterly or annual results68
  7. May 6, 2025Reported quarterly or annual results69
  8. Feb 27, 2025Reported quarterly or annual results70

Upcoming dates from Nasdaq's calendar; past results from the company's 8-K filings (Item 2.02). The full markets calendar

Filings

662 filings to the SEC since October 2021: 6 annual reports, 15 quarterly reports, 72 major events, 290 insider forms, 46 large holders, 1 private raises, 232 other. The latest 20:

  1. Oct 13, 20264 EDGAR
  2. Oct 9, 2026144 EDGAR
  3. Oct 9, 20264 EDGAR
  4. Oct 7, 2026144 EDGAR
  5. Oct 7, 20264 EDGAR
  6. Oct 6, 2026144 EDGAR
  7. Oct 5, 2026144 EDGAR
  8. Oct 2, 20264 EDGAR
  9. Sep 30, 2026144 EDGAR
  10. Sep 21, 20268-K EDGAR
  11. Sep 18, 20264 EDGAR
  12. Sep 11, 20264 EDGAR
  13. Sep 9, 2026144 EDGAR
  14. Aug 28, 20264 EDGAR
  15. Aug 28, 20268-K EDGAR
  16. Aug 25, 2026144/A EDGAR
  17. Aug 21, 20264 EDGAR
  18. Aug 19, 2026144 EDGAR
  19. Aug 19, 20264 EDGAR
  20. Aug 17, 2026144 EDGAR

The 662 filings since October 2021 can be filtered by form type and year here when JavaScript is on.

Every filing from the last five years, summarized. The full record is on SEC EDGAR.

Insider trades

256 open-market trades by officers, directors and large owners since October 2021: 1 buys and 255 sales. Grants, option exercises and gifts are left out.

DateWhoTradeSharesPriceValueFiling
Sep 17, 2026SCHMERTZLER MICHAELDirectorSale10,000$67.85$678.5KForm 4
Sep 16, 2026SCHMERTZLER MICHAELDirectorSale10,000$67.12$671.2KForm 4
Sep 10, 2026SCHMERTZLER MICHAELDirectorSale10,000$67.34$673.4KForm 4
Sep 9, 2026SCHMERTZLER MICHAELDirectorSale10,000$67.84$678.4KForm 4
Aug 27, 2026SCHMERTZLER MICHAELDirectorSale12,500$71.58$894.8KForm 4
Aug 26, 2026SCHMERTZLER MICHAELDirectorSale12,500$72.94$911.7KForm 4
Aug 20, 2026SCHMERTZLER MICHAELDirectorSale12,500$71.11$888.9KForm 4
Aug 19, 2026SCHMERTZLER MICHAELDirectorSale12,500$73.32$916.6KForm 4
Aug 17, 2026Boulding Mark ElliottEXEC. VP AND CLOSale2,813$72.57$204.1KForm 4
Aug 4, 2026Klein Matthew B.CHIEF EXECUTIVE OFFICERSale10,292$68.57$705.7KForm 4
Jul 14, 2026Gravier PierreCHIEF FINANCIAL OFFICERSale3,494$80.35$280.7KForm 4
Jul 9, 2026Smith Mary L.DirectorSale15,834$90.00$1.4MForm 4
Jul 9, 2026Almstead Neil GregoryCHIEF TECHNICAL OPS OFFICERSale2,464$90.25$222.4KForm 4
Jul 7, 2026Almstead Neil GregoryCHIEF TECHNICAL OPS OFFICERSale24,613$88.89$2.2MForm 4
Jul 6, 2026Boulding Mark ElliottEXEC. VP AND CLOSale5,079$85.08$432.1KForm 4
Jul 6, 2026Almstead Neil GregoryCHIEF TECHNICAL OPS OFFICERSale22,161$86.00$1.9MForm 4
Jun 26, 2026Almstead Neil GregoryCHIEF TECHNICAL OPS OFFICERSale8,130$85.26$693.2KForm 4
Jun 25, 2026Almstead Neil GregoryCHIEF TECHNICAL OPS OFFICERSale20,960$85.97$1.8MForm 4
Jun 24, 2026Almstead Neil GregoryCHIEF TECHNICAL OPS OFFICERSale5,323$85.03$452.6KForm 4
Jun 22, 2026Almstead Neil GregoryCHIEF TECHNICAL OPS OFFICERSale23,749$80.90$1.9MForm 4

All 256 trades can be filtered by person here when JavaScript is on.

From Forms 4 filed with the SEC.

Major holders

Investors who own more than 5% of the shares must tell the SEC: a 13D if they may seek to influence the company, a 13G if they hold passively.

Stakes it holds

PTC Therapeutics has told the SEC it owns more than 5% of 1 company, latest first (Schedules 13D and 13G).

Money raised

Private offerings the company reported to the SEC on Form D.

  1. Apr 10, 2023Raised $35.9 million in a private offering of equity Form D: 40 investors so far Form D

Held by these funds

The largest holders among investment managers' 13F reports for the quarter ending Jun 30, 2026, the top 25 of 100. Change is in shares since the quarter before.

FundSharesValueShare of the fundChange
BlackRock, Inc.13,964,554$1.1B0.0%+1,015,837
Rtw Investments, LP7,746,415$631.9M5.4%No change
Jupiter Topco LLC7,236,402$590.2M0.2%New
Vanguard Portfolio Management LLC4,928,111$402.0M0.0%+123,517
State Street Corp4,598,952$375.1M0.0%+252,433
Wellington Management Group Llp4,128,273$336.7M0.1%+185,663
Vanguard Capital Management LLC3,739,127$305.0M0.0%+62,871
Geode Capital Management, LLC2,207,376$180.1M0.0%−2,578
Toronto Dominion Bank2,165,846$176.7M0.3%−860,117
Westfield Capital Management Co LP1,956,156$159.6M0.5%New
Hood River Capital Management LLC1,738,613$141.8M1.0%+74,054
William Blair Investment Management, LLC1,722,688$140.5M0.4%New
D. E. Shaw & Co., Inc.1,548,919$126.3M0.1%−91,462
Tang Capital Management LLC1,500,000$122.4M5.6%No change
Bank Of America Corp /de/1,452,277$118.5M0.0%−434,564
Driehaus Capital Management LLC1,440,107$117.5M0.7%+424,335
Fmr LLC1,398,261$114.1M0.0%−130,101
Jefferies Financial Group Inc.1,357,042$110.7M1.0%+2,583
Fuller & Thaler Asset Management, Inc.1,156,819$94.4M0.3%New
Pictet Asset Management Holding SA1,147,844$93.6M0.1%−35,876
Morgan Stanley1,103,675$90.0M0.0%+429,541
Armistice Capital, LLC1,071,395$87.4M2.4%−1,003,054
Blue Sparrow, LLC /DE1,050,700$85.7M0.8%+860,100
Franklin Resources Inc1,027,327$83.8M0.0%−94,061
Invesco Ltd.941,581$76.8M0.0%+76,130

13F reports cover disclosed long positions in U.S. stocks only.

SEC filer profile

How PTC Therapeutics is registered with the Securities and Exchange Commission, from its EDGAR record and the cover page of its latest annual report.

  • Name on EDGARPTC THERAPEUTICS, INC.2
  • CIK0001070081
  • Employer ID (EIN)04-341658717
  • Filer statusLarge accelerated filer not an emerging growth company; not a smaller reporting company; not a shell company17
  • Incorporated inDelaware17
  • Fiscal year endsDecember 312
  • Industry code (SIC)2834 Pharmaceutical Preparations2
  • SEC reviewing officeOffice of Life Sciences, Division of Corporation Finance2
  • Business address500 Warren Corporate Center Drive, Warren, NJ 070592
  • Phone90822270002
  • Shares outstanding83,460,802 as of Jul 29, 202615
  • Public float$3.4M as of Jun 30, 202517
  • AuditorErnst & Young LLP, Iselin, New Jersey PCAOB ID 42; it also attests to the company’s internal control over financial reporting17
  • On EDGAR sinceJan 2, 20042
  • Filings1,331 in all: 4 (573), 144 (204), 8-K (168), SC 13G/A (74), 10-Q (40), 3 (39), SC 13G (30), DEFA14A (14)2
    All 46 form types

    10-K (13), DEF 14A (13), S-8 (13), SCHEDULE 13G/A (13), UPLOAD (13), 424B5 (11), CORRESP (10), CT ORDER (10), D (10), SC 13D/A (10), S-3ASR (8), S-1/A (7), ARS (5), POSASR (5), SCHEDULE 13G (5), FWP (4), REGDEX (4), 8-K/A (3), S-1 (3), SC 13D (3), 3/A (2), 424B4 (2), EFFECT (2), PRE 14A (2), REGDEX/A (2), RW (2), S-1MEF (2), SEC STAFF LETTER (2), 10-K/A (1), 144/A (1), 4/A (1), 5 (1), 8-A12B (1), CERTNAS (1), DRS (1), DRS/A (1), RW WD (1), S-8 POS (1)

  • Latest filingsAnnual report (10-K) Feb 19, 2026
    Quarterly report (10-Q) Jul 30, 2026
    Proxy statement (DEF 14A) Apr 20, 2026
    Latest 8-K (8-K) Sep 21, 2026

Securities registered for trading

ClassSymbolExchange
Common Stock, $0.001 par value per sharePTCTNasdaq Global Select Market

From the cover page of the 10-K filed Feb 19, 2026 (Section 12(b) of the Exchange Act).17

Risk factors and legal proceedings

The risks PTC Therapeutics names in Item 1A of its 10-K filed Feb 19, 2026: 54 risks under these headings, as written.17

  • Risks Related to the Development and Commercialization of our Products and our Product Candidates
  • If we are unable to continue to execute our commercial strategy for our products, fail to obtain renewal of, or satisfy the conditions of our marketing authorization for our products, or if we experience significant delays in accomplishing such goals, our business will be materially harmed.
  • Delays or failures in obtaining regulatory approval would prevent us from commercializing our product candidates in the applicable territory and our ability to generate revenue will be materially impaired. Moreover, should we need to conduct additional development work, other than those we have planned, we expect to incur significant costs, which may have a material adverse effect on our business and results of operations.
  • We may be unable to continue to commercialize Translarna for nmDMD in the EEA if individual countries in the EU do not leverage Articles 117(3) and 5(1) of the EU Directive 2001/83 to allow continued commercial use of Translarna. The loss of the Translarna marketing authorization in the EEA may affect future reauthorizations in other jurisdictions.
  • We may use certain specialized pathways to develop our product candidates or to seek approval. We may not qualify for these pathways or such pathways may not ultimately speed the time to approval or result in product candidate approval.
  • If we or our collaborators experience any of a number of possible unforeseen events in connection with clinical trials related to our products or our product candidates, maintenance of our existing marketing authorization for our products and any additional potential marketing authorization or commercialization of our products or our product candidates could be delayed or prevented.
  • Subgroup, retrospective, post-hoc, and certain statistical analyses may not be reliable and typically will not form the basis for regulatory approval.
  • If we experience delays or difficulties in the enrollment of patients in our clinical trials, our receipt of necessary regulatory approvals could be delayed or prevented.
  • If serious adverse side effects are identified during the development of any product candidate or for any product for which we have or may obtain marketing approval, we may need to abandon or limit our development and/or marketing of that product or product candidate.
  • Certain of our products and product candidates may be difficult to produce, presenting manufacturing challenges that may delay product development and regulatory approval.
  • Any of our products or any other product candidate that receives marketing authorization may fail to achieve the degree of market acceptance by physicians, patients, third-party payors and others in the medical community necessary for commercial success.
  • If we are unable to establish or maintain sales, marketing and distribution capabilities or enter into agreements with third parties to market, sell and distribute our products or product candidates, we may not be successful in our continuing efforts to commercialize our products or any other product candidate if and when they are approved.
The other 42 risks
  • A significant portion of our commercial sales currently occurs in territories outside of the United States which subjects us to additional business risks that could adversely affect our revenue and results of operations.
  • Laws and regulations governing export restrictions and economic sanctions may preclude us from developing and selling certain products, generating revenue from such products, and manufacturing certain materials outside of the United States.
  • We face substantial competition, which may result in others discovering, developing or commercializing products before or more successfully than we do.
  • Our products or product candidates may become subject to unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives, which would harm our business.
  • There may be future and have been changes in legal and regulatory requirements that may materially impact our results of operation.
  • Risks Related to Our Financial Position and Need for Additional Capital
  • We have incurred significant losses since our inception and based on our current commercial, research and development plans, we expect to continue to incur significant operating expenses for the foreseeable future. We may never generate profits from operations or maintain profitability.
  • We may need additional funding. If we are unable to raise capital when needed, we could be forced to delay, reduce or eliminate our product development programs or commercialization efforts.
  • We may engage in strategic transactions to acquire assets, businesses, or rights to products, product candidates or technologies or form collaborations or make investments in other companies or technologies that could harm our operating results, dilute our stockholders’ ownership, increase our debt, or cause us to incur significant expense.
  • Raising additional capital may cause dilution to our stockholders, restrict our operations or require us to relinquish rights to our technologies or product candidates.
  • Our ability to use our net operating losses and certain other tax attributes to offset potential taxable income and related income taxes that would otherwise be due is subject to limitation under the provisions of Sections 382 and 383 of the Internal Revenue Code as a result of ownership changes of the Company and could be subject to further annual limitations under such provisions. In addition, we may not generate sufficient future taxable income to use our net operating losses and certain other tax attributes.
  • Changes in our effective income tax rates and future changes to U.S. and non-U.S. tax laws could adversely affect our results of operations.
  • Risks Related to Regulatory Approval of our Products and our Product Candidates
  • We may not be able to obtain orphan drug exclusivity for our products or product candidates in either the United States or the EU.
  • All pharmaceutical products for which marketing authorization has been granted are subject to extensive and rigorous governmental regulation and could be subject to restrictions or withdrawal from the market. We may also be subject to penalties or other enforcement if we fail to comply with regulatory requirements or if we experience unanticipated problems with our products, when and if any of them are approved, as well as our product candidates during development.
  • We may face competition from biosimilar, generic, and similar products approved through abbreviated pathways, as well as products approved pursuant to full applications.
  • Our relationships with customers, healthcare providers and professionals, patients, patient organizations, and third-party payors are or will be subject to applicable anti-kickback, fraud and abuse, transparency and other healthcare laws and regulations, which could expose us to criminal sanctions, civil penalties, contractual damages, reputational harm and diminished profits and future earnings.
  • Legislative and regulatory changes affecting the pharmaceutical industry or the healthcare system more broadly may increase the difficulty and cost for us to obtain or maintain marketing authorization of and commercialize our products and product candidates and affect the coverage and reimbursement we may obtain.
  • Risks Related to Our Business
  • We may expend our resources to pursue a particular product, product candidate or indication and fail to capitalize on product candidates or indications that may be more profitable or for which there is a greater likelihood of success.
  • We contract with third parties for the manufacture and distribution of our products and certain of our product candidates, which may increase the risk that we will not have sufficient quantities of our products or product candidates, such quantities may not meet the applicable regulatory quality standards, or such quantities at an acceptable cost, which could delay, prevent or impair our commercialization or development efforts.
  • We rely on third parties to conduct our preclinical and clinical trials, and those third parties may not perform satisfactorily, including failing to meet deadlines for the completion of such trials.
  • We currently depend, and expect to continue to depend, on collaborations with third parties for the development and commercialization of some of our products and product candidates. If those collaborations are not successful, we may not be able to capitalize on the market potential of these products and product candidates.
  • We may rely on third parties to perform many essential services for any products that we commercialize, including services related to warehousing and inventory control, distribution, government price reporting, customer service,
  • accounts receivable management, cash collection, and pharmacovigilance and adverse event reporting. If these third parties fail to perform as expected or to comply with legal and regulatory requirements, our ability to commercialize our product candidates will be significantly impacted and we may be subject to regulatory sanctions.
  • Our business and operations would suffer in the event of computer system failures, cyber-attacks, lack of operational resilience, or a deficiency in our, or our collaborators’ or third-party vendors’, cyber-security.
  • Product liability and other civil lawsuits against us could cause us to incur substantial liabilities and to limit clinical trials or commercialization of any current or future products. Our insurance program may not be extensive enough to adequately protect us against these risks.
  • If we fail to comply with environmental, health and safety laws and regulations, we could become subject to fines or penalties or incur costs that could have a material adverse effect on the success of our business.
  • Our future success depends on our ability to retain our chief executive officer and other key executives and to attract, retain and motivate qualified personnel.
  • Risks Related to our Intellectual Property
  • If we are unable to obtain and maintain patent protection for our technology and products, or if the scope of the patent protection is not sufficiently broad, and at such times as our patent protection expires, our competitors could develop and commercialize technology and products similar or identical to ours, and our ability to successfully commercialize our technology and products may be adversely affected.
  • We may become involved in lawsuits to protect or enforce our intellectual property, which could be expensive, time consuming and unsuccessful.
  • Third parties may initiate legal proceedings alleging that our patents are invalid and unenforceable or that we are infringing their intellectual property rights, the outcome of which would be uncertain and could have a material adverse effect on the success of our business.
  • We may be subject to claims by third parties asserting that we or our employees have misappropriated their intellectual property, or claiming ownership of what we regard as our own intellectual property.
  • Intellectual property litigation could cause us to spend substantial resources and could distract our personnel from their normal responsibilities.
  • Without patent protection, our marketed products may face generic competition.
  • If we are unable to protect the confidentiality of our trade secrets, our business and competitive position would be harmed.
  • We have not yet registered our trademarks in all of our potential markets, and failure to secure those registrations could adversely affect our business.
  • If we are not able to obtain adequate trademark protection or regulatory approval for our brand names, we may be required to re-brand affected products, which could cause delays in getting such products to market and substantially increase our costs.
  • Our rights to develop and commercialize Upstaza/Kebilidi are subject, in part, to the terms and conditions of licenses granted to us by others.
  • If we fail to comply with our obligations in our intellectual property licenses and funding arrangements with third parties, we could lose rights that are important to our business.
  • Risks Related to our Common Stock
  • Servicing the 2026 Convertible Notes requires a significant amount of cash. We may not have sufficient cash flow from our business to make payments on our debt, and we may not have the ability to raise the funds necessary for conversions of the 2026 Convertible Notes or to settle or pay principal and interest on the 2026 Convertible Notes at maturity, which could adversely affect our business, financial condition and results of operations.
  • Provisions in our corporate charter documents and under Delaware law could make an acquisition of us, which may be beneficial to our stockholders, more difficult and may prevent attempts by our stockholders to replace or remove our current management.
  • The price of our common stock may be volatile and fluctuate substantially, which could result in substantial losses for purchasers of our common stock and lawsuits against us and our officers and directors.
  • Because we do not anticipate paying any cash dividends on our capital in the foreseeable future, capital appreciation, if any, will be our stockholders’ sole source of gain.
  • The issuance of additional shares of our common stock or the sale of shares of our common stock by our stockholders could dilute our stockholders’ ownership interest in the Company and could significantly reduce the market price of our common stock.

Legal proceedings

From time to time in the ordinary course of our business, we are subject to claims, legal proceedings and disputes. We are not currently aware of any material legal proceedings which we are a party to or of which any of our property is the subject.

The opening of Item 3, Legal Proceedings.17

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