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IRON on Nasdaq

Disc Medicine, Inc.

In its 2026 annual report the company says: “We are a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases. We have assembled a portfolio of clinical and preclinical product candidates that aim to modify fundamental biological pathways associated with the formation and function of red blood cells, specifically heme biosynthesis and iron homeostasis.”4 It is headquartered in Watertown, Massachusetts.1

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Last updated Oct 21, 2026.

Overview

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  • TickerIRON on Nasdaq1
  • IndustryPharmaceutical Preparations1
  • HeadquartersWatertown, Massachusetts1
  • Business address321 Arsenal Street, Suite 101, Watertown, Ma 024722
  • Filer categoryLarge accelerated filer2

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We are a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases. We have assembled a portfolio of clinical and preclinical product candidates that aim to modify fundamental biological pathways associated with the formation and function of red blood cells, specifically heme biosynthesis and iron homeostasis. Our current pipeline includes bitopertin for the treatment of erythropoietic porphyrias, or EPs, including erythropoietic protoporphyria, or EPP, and X-linked protoporphyria, or XLP; DISC-0974 for the treatment of anemia of myelofibrosis, or MF, and anemia of inflammatory bowel disease, or IBD; and DISC-3405 for the treatment of polycythemia vera, or PV, sickle cell disease, or SCD, and other hematologic disorders. In addition, our preclinical programs include DI…4 From its latest annual report

Financials

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

Financial statements

Operating income5678-$236.0M in 2025+593% since 2020
-$300.0M-$200.0M-$100.0M02020: -$34.0M-$34.0M20202021: -$30.9M-$30.9M2021−9%2022: -$47.5M-$47.5M2022+53%2023: -$91.1M-$91.1M2023+92%2024: -$129.7M-$129.7M2024+42%2025: -$236.0M-$236.0M2025+82%
Net income5678-$212.2M in 2025+420% since 2020
-$300.0M-$200.0M-$100.0M02020: -$40.8M-$40.8M20202021: -$36.0M-$36.0M2021−12%2022: -$46.8M-$46.8M2022+30%2023: -$76.4M-$76.4M2023+63%2024: -$109.4M-$109.4M2024+43%2025: -$212.2M-$212.2M2025+94%

Every line of the income statement as filed: the last 3 fiscal years, latest first.8

Period ending2025-12-312024-12-312023-12-31
Research and development$170.6M$96.7M$69.3M
Selling, general and administrative$65.4M$33.0M$21.9M
Total operating expenses$236.0M$129.7M$91.1M
Loss from operations-$236.0M-$129.7M-$91.1M
Interest income$27.8M$21.3M$14.8M
Interest expense-$3.6M-$572.0K$0
Other income (expense)$45.0K-$2.0K-$2.0K
Total other income (expense), net$24.2M$20.7M$14.8M
Loss before income taxes-$211.8M-$109.0M-$76.3M
Income tax expense-$361.0K-$355.0K-$99.0K
Net loss per share, basic-$6.01-$3.96-$3.42
Net loss per share, diluted-$6.01-$3.96-$3.42
Weighted-average common shares outstanding-basic35.3M shares27.6M shares22.3M shares
Weighted-average common shares outstanding-diluted35.3M shares27.6M shares22.3M shares
Net loss-$212.2M-$109.4M-$76.4M
Foreign currency translation adjustments-$1.0K-$27.0K$0
Unrealized gain on marketable securities$677.0K$316.0K$0
Total other comprehensive income$676.0K$289.0K$0
Comprehensive loss-$211.5M-$109.1M-$76.4M

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Corporate structure

Disc Medicine, Inc. sits at the top of its group. 3 entities, 1 country on record. Switch between the flow chart and the network, and select any entity to see its details.

Major holders of Disc Medicine, Inc.

  • Disc Medicine, Inc. Watertown, Massachusetts
    • Disc Medicine Opco, Inc. Subsidiary · Delaware, United States
    • Disc Medicine Securities Corp. Subsidiary · Massachusetts, United States

Built from subsidiaries listed in Exhibit 21 of annual reports filed with the SEC; major holders from Schedules 13D and 13G. Each entity in the chart links to its source.

What it owns

What Disc Medicine, Inc. 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
Accumulated depreciation$401.0K11 Dec 31, 2024$245.0K11 Dec 31, 2023+63.7%
Property and equipment, net$1.2M8 Dec 31, 2025$751.0K8 Dec 31, 2024+54.3%
Operating-lease assets (leased space)$1.1M8 Dec 31, 2025$1.6M8 Dec 31, 2024−30.1%

Investments

ItemLatestA year beforeChange
Cash and cash equivalents$91.1M8 Dec 31, 2025$192.4M8 Dec 31, 2024−52.6%
Marketable securities, current$700.0M8 Dec 31, 2025$297.4M8 Dec 31, 2024+135.3%
Debt securities available for sale$700.0M8 Dec 31, 2025$297.4M8 Dec 31, 2024+135.3%

Properties, in its own words

Our principal office is located at 321 Arsenal Street, Suite 101, Watertown, MA 02472, where we lease and sublease a total of approximately 16,847 square feet of office space. Our sublease expires in November 2026 and our lease expires in December 2029. Upon expiration of the sublease, we will lease the same space through December 2029 pursuant to an existing agreement. We believe that these facilities will be adequate for our near-term needs. If required, we believe that suitable additional or substitute space will be available in the future on commercially reasonable terms to accommodate any such expansion of our operations.

Item 2 of the 10-K filed Feb 26, 2026.4

Leadership

Who has led Disc Medicine, Inc., and when. Pick a name to see everywhere that person has worked.

Chief executive

  1. Jason Patrick Meyenburg2021–2022
  2. John D. Quisel2023–now

Chair

  1. Georges Gemayel2022

President

  1. Jason Patrick Meyenburg2021

Chief financial officer

  1. Dennis D. Ryan2020
  2. Gregg Beloff2021
  3. Brian Piekos2021–2022
  4. Joanne Bryce2023–2024
  5. Jean M. Franchi2024–now

Chief operating officer

  1. Jonathan Yen-Wen Yu2025–now
2025
Board members and other officers (29)

Subsidiaries and ownership

Subsidiaries (Exhibit 21)

  • Disc Medicine Opco, Inc. Delaware3
  • Disc Medicine Securities Corp. Massachusetts3

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

Former names

  • Gemini Therapeutics, Inc. /DENov 27, 2020 to Dec 28, 20222
  • FS Development Corp.Jul 2, 2020 to Feb 8, 20212

Timeline

  1. Sep 10, 20268-K Regulation FD disclosure; Other events48
  2. Jul 30, 20268-K Results of operations49
  3. Jun 30, 20268-K Entered a material agreement; New debt or obligation50
  4. Jun 24, 20268-K Shareholder vote51
  5. May 5, 20268-K Results of operations52
  6. Feb 27, 20268-K Exit or restructuring costs53
  7. Feb 26, 20268-K Results of operations54
  8. Feb 17, 20268-K Regulation FD disclosure55
  9. Feb 13, 20268-K Regulation FD disclosure; Other events56
  10. Jan 12, 20268-K Results of operations; Regulation FD disclosure57
  11. Dec 8, 20258-K Other events58
  12. Nov 6, 20258-K Results of operations59
  13. Jan 3, 2023New chief executive John D. Quisel becomes CEO12
  14. Feb 10, 2021New chief executive Jason Patrick Meyenburg becomes CEO13

Calendar

Coming up

  1. Nov 5, 2026Reports results for the quarter ending Sep/2026 A year earlier it reported ($1.77) a share, on Nov 6, 2025.60

When it reported results

  1. Jul 30, 2026Reported quarterly or annual results49
  2. May 5, 2026Reported quarterly or annual results52
  3. Feb 26, 2026Reported quarterly or annual results54
  4. Jan 12, 2026Reported quarterly or annual results57
  5. Nov 6, 2025Reported quarterly or annual results59
  6. Oct 20, 2025Reported quarterly or annual results61
  7. Aug 7, 2025Reported quarterly or annual results62
  8. May 7, 2025Reported quarterly or annual results63

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

Filings

630 filings to the SEC since October 2021: 5 annual reports, 15 quarterly reports, 75 major events, 232 insider forms, 65 large holders, 238 other. The latest 20:

  1. Sep 16, 20264 EDGAR
  2. Sep 15, 20264 EDGAR
  3. Sep 11, 2026144 EDGAR
  4. Sep 10, 20268-K EDGAR
  5. Sep 9, 20264 EDGAR
  6. Sep 4, 2026144 EDGAR
  7. Sep 4, 2026144 EDGAR
  8. Sep 4, 2026144 EDGAR
  9. Sep 4, 2026144 EDGAR
  10. Sep 3, 2026144 EDGAR
  11. Sep 3, 2026144 EDGAR
  12. Sep 3, 2026144 EDGAR
  13. Sep 3, 2026144 EDGAR
  14. Sep 3, 20264 EDGAR
  15. Sep 2, 2026144 EDGAR
  16. Sep 2, 2026144 EDGAR
  17. Sep 2, 2026144 EDGAR
  18. Sep 2, 2026144 EDGAR
  19. Aug 26, 20264 EDGAR
  20. Aug 18, 20264 EDGAR

The 630 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

154 open-market trades by officers, directors and large owners since October 2021: 9 buys and 145 sales. Grants, option exercises and gifts are left out.

DateWhoTradeSharesPriceValueFiling
Sep 11, 2026Bitterman KevinDirectorSale27,136$70.11$1.9MForm 4
Sep 4, 2026Bitterman KevinDirectorSale1,689$79.96$135.1KForm 4
Sep 3, 2026Bitterman KevinDirectorSale5,203$79.98$416.1KForm 4
Sep 2, 2026Bitterman KevinDirectorSale49,164$80.17$3.9MForm 4
Sep 1, 2026Bitterman KevinDirectorSale129$79.95$10.3KForm 4
Aug 25, 2026Yu Jonathan Yen-WenChief Operating OfficerSale8,796$83.12$731.1KForm 4
Aug 24, 2026Yu Jonathan Yen-WenChief Operating OfficerSale300$82.53$24.8KForm 4
Aug 17, 2026Quisel John DChief Executive OfficerSale33,000$79.15$2.6MForm 4
Aug 12, 2026Yu Jonathan Yen-WenChief Operating OfficerSale804$82.64$66.4KForm 4
Aug 6, 2026Khara RahulChief Legal OfficerSale5,000$80.00$400.0KForm 4
Jul 13, 2026Quisel John DChief Executive OfficerSale33,000$74.96$2.5MForm 4
Jul 7, 2026Yu Jonathan Yen-WenChief Operating OfficerSale100$82.50$8.2KForm 4
Jun 29, 2026Khara RahulChief Legal OfficerSale1,649$75.00$123.7KForm 4
Jun 26, 2026Khara RahulChief Legal OfficerSale851$75.00$63.8KForm 4
Jun 26, 2026Yu Jonathan Yen-WenChief Operating OfficerSale300$72.50$21.8KForm 4
Jun 22, 2026Yu Jonathan Yen-WenChief Operating OfficerSale9,700$72.66$704.8KForm 4
Jun 18, 2026Khara RahulChief Legal OfficerSale7,500$70.22$526.7KForm 4
Jun 18, 2026Quisel John DChief Executive OfficerSale34,000$70.22$2.4MForm 4
Jun 16, 2026Yu Jonathan Yen-WenChief Operating OfficerSale18,612$69.19$1.3MForm 4
Apr 14, 2026Franchi Jean M.Chief Financial OfficerSale6,257$69.24$433.2KForm 4

All 154 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.

Executive pay

What the company paid its top executives, from the summary compensation table in its proxy statement. Total pay includes salary, bonus, stock and option awards and other pay, as the company valued them.

YearExecutiveAgeSalaryTotal pay
2025John QuiselJ.D., Ph.D.$695.0K$10.0M85
2025Jean Franchi59$531.0K$4.2M85
2025Pamela StephensonM.P.H.58$520.0K$4.2M85
2025William SavageM.D., Ph.D.$523.0K$3.5M85
2025Jonathan YuM.B.A.45$507.0K$3.5M85
2024President and Chief Executive Officer$645.0K$9.1M85
2024Chief Financial Officer$464.5K$5.3M85
2024Chief Commercial Officer$428.6K$5.2M85
2024Chief Medical Officer$505.0K$3.1M85
2024Chief Operating Officer$490.0K$2.9M85
2023President and Chief Executive Officer$562.0K$909.1K85
2023Chief Medical Officer$458.0K$687.7K85
2023Chief Operating Officer$419.0K$628.0K85

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
Fmr LLC4,894,473$358.0M0.0%−692,421
BlackRock, Inc.2,467,899$180.5M0.0%+194,068
Ra Capital Management, L.P.2,450,221$179.2M1.6%No change
Jpmorgan Chase & Co1,875,795$139.9M0.0%+504,315
Wellington Management Group Llp1,741,351$127.4M0.0%+5,975
State Street Corp1,648,836$120.6M0.0%−49,408
Vestal Point Capital, LP1,550,000$113.4M2.6%+850,000
Atlas Venture Life Science Advisors, LLC1,513,875$110.7M12.6%No change
Vanguard Capital Management LLC1,369,027$100.1M0.0%−6,403
Frazier Life Sciences Management, L.P.1,354,852$99.1M2.2%No change
TCG Crossover Management, LLC1,341,862$98.1M2.4%No change
Bvf Inc/il1,153,612$84.4M2.2%−118,700
Woodline Partners LP958,281$70.1M0.2%No change
Siren, L.L.C.888,401$65.0M1.3%−100,000
Logos Global Management LP875,000$64.0M2.8%+315,000
Opaleye Management Inc.855,000$62.5M5.7%+434,000
Geode Capital Management, LLC789,724$57.8M0.0%+45,502
Assenagon Asset Management S.A.768,316$56.2M0.0%+222,069
Deerfield Management Company, L.P.657,000$48.1M0.6%No change
Dimensional Fund Advisors LP569,018$41.6M0.0%+174,571
Bellevue Group AG560,000$41.0M0.9%+100,686
Saturn V Capital Management LP524,730$38.4M5.5%+74,804
Armistice Capital, LLC460,000$33.6M0.9%−76,000
Jennison Associates LLC439,520$32.1M0.0%−170,862
Aberdeen Group plc438,174$32.0M0.0%+62,560

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

SEC filer profile

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

  • Name on EDGARDisc Medicine, Inc.1
  • CIK0001816736
  • Employer ID (EIN)85-16128454
  • Filer statusLarge accelerated filer not an emerging growth company; not a smaller reporting company; not a shell company4
  • Incorporated inDelaware4
  • Fiscal year endsDecember 311
  • Industry code (SIC)2834 Pharmaceutical Preparations1
  • SEC reviewing officeOffice of Life Sciences, Division of Corporation Finance1
  • Business address321 Arsenal Street, Suite 101, Watertown, MA 024721
  • Phone(617) 674-92741
  • Shares outstanding38,392,844 as of Jul 23, 202610
  • Public float$1.4B as of Jun 30, 20254
  • AuditorErnst & Young LLP, Boston, Massachusetts PCAOB ID 42; it also attests to the company’s internal control over financial reporting4
  • On EDGAR sinceJul 2, 20201
  • Filings757 in all: 4 (228), 144 (154), 8-K (92), 3 (47), SC 13G (20), 10-Q (18), SC 13G/A (17), SC 13D/A (16)1
    All 42 form types

    424B3 (15), CORRESP (13), 424B5 (12), UPLOAD (11), 425 (10), EFFECT (10), S-4/A (9), SCHEDULE 13G/A (9), S-8 (7), 10-K (6), SC 13D (6), DEF 14A (5), DEFA14A (5), SCHEDULE 13D/A (5), ARS (4), POS AM (4), 144/A (3), 3/A (3), 4/A (3), S-3 (3), SCHEDULE 13G (3), 8-K/A (2), CERT (2), S-1 (2), S-1/A (2), S-4 (2), SEC STAFF LETTER (2), 25-NSE (1), 424B4 (1), 8-A12B (1), 8-A12B/A (1), DRS (1), S-1MEF (1), S-3ASR (1)

  • Latest filingsAnnual report (10-K) Feb 26, 2026
    Quarterly report (10-Q) Jul 30, 2026
    Proxy statement (DEF 14A) Apr 28, 2026
    Latest 8-K (8-K) Sep 10, 2026

Securities registered for trading

ClassSymbolExchange
Common Stock, $0.0001 Par ValueIRONThe Nasdaq Global Market

From the cover page of the 10-K filed Feb 26, 2026 (Section 12(b) of the Exchange Act).4

Risk factors and legal proceedings

The risks Disc Medicine, Inc. names in Item 1A of its 10-K filed Feb 26, 2026: 73 risks under these headings, as written.4

  • Risks Related to Our Limited Operating History, Financial Position and Capital Requirements
  • Our limited operating history may make it difficult for you to evaluate the success of our business to date and to assess our future viability.
  • We have incurred significant net losses since our inception and anticipate that we will continue to incur losses for the foreseeable future.
  • We have no products approved for commercial sale and have not generated any revenue from product sales.
  • Our existing and any future indebtedness could adversely affect our ability to operate our business.
  • We may need to raise substantial additional funding. If we are unable to raise capital when needed or on terms acceptable to us, we may be forced to delay, reduce or eliminate some of our product development programs or commercialization efforts.
  • Raising additional capital may cause dilution to our stockholders, restrict our operations or require us to relinquish rights to our technologies or product candidates.
  • Risks Related to the Discovery and Development of Our Product Candidates
  • We have not yet progressed any product candidates through a Phase 3 clinical trial and may be unable to successfully complete any additional clinical trials for any product candidates we develop. Certain of our programs are still in preclinical development and may never advance to clinical development.
  • Our programs are focused on the development of therapeutics for patients with hematologic diseases, which is a rapidly evolving area of science, and the approach we are taking to discover and develop product candidates is novel and may never lead to approved or marketable products.
  • Clinical development involves a lengthy and expensive process, with an uncertain outcome.
  • Because we are developing some of our product candidates for the treatment of diseases in which there is little clinical experience and, in some cases, using new endpoints or methodologies, the FDA or other regulatory authorities may not consider the endpoints of our clinical trials to predict or provide clinically meaningful results.
The other 62 risks
  • Interim, top-line, initial and preliminary data from our clinical trials that we announce or publish from time to time may change as more patient data become available and are subject to confirmation, audit and verification procedures that could result in material changes in the final data.
  • We may incur additional costs or experience delays in initiating or completing, or ultimately be unable to complete, the development and commercialization of our product candidates.
  • If we experience delays or difficulties in the enrollment of patients in clinical trials, our receipt of necessary regulatory approvals could be delayed or prevented.
  • Results from early preclinical studies and clinical trials of our programs and product candidates are not necessarily predictive of the results of later preclinical studies and clinical trials of our programs and product candidates. If we cannot replicate the results from earlier preclinical studies and clinical trials of our programs and product candidates in our later preclinical studies and clinical trials, we may be unable to successfully develop, obtain regulatory approval for and commercialize our product candidates.
  • Our clinical trials or those of our future collaborators may reveal significant adverse events not seen in prior preclinical studies or clinical trials and may result in a safety profile that could inhibit regulatory approval or market acceptance of any of our product candidates.
  • Some of our product candidates modulate pathways for which there are currently no approved or effective therapies, which may result in greater research and development expenses, regulatory issues that could delay or prevent approval or discovery of unknown or unanticipated adverse effects on safety or efficacy.
  • We have conducted and are currently conducting clinical trials for bitopertin, and may in the future conduct additional clinical trials of our product candidates, outside the United States, and the FDA and comparable foreign regulatory authorities may not accept data from such trials.
  • Although we intend to explore other therapeutic opportunities in addition to the programs and product candidates that we are currently developing, we may fail to identify viable new product candidates for clinical development for a number of reasons. If we fail to identify additional product candidates, our business could be materially harmed.
  • If we are not able to obtain, or if there are delays in obtaining, required regulatory approvals for any of our product candidates, we will not be able to commercialize, or will be delayed in commercializing, such product candidates, and our ability to generate revenue will be materially impaired.
  • A public health crisis, pandemic, epidemic, or outbreak of an infectious or highly contagious disease, may materially and adversely affect our business and financial results and could cause a disruption to the development of our product candidates.
  • Risks Related to Commercialization
  • We face substantial competition, which may result in others discovering, developing or commercializing products before or more successfully than we do.
  • If the market opportunities for our programs and product candidates are smaller than we estimate or if any regulatory approval that we obtain is based on a narrower definition of the patient population, our revenue and ability to achieve profitability could be materially adversely affected.
  • If our current product candidates or any future product candidates do not achieve broad market acceptance, the revenue that we generate from our sales may be limited, and we may never become profitable.
  • Even if we receive regulatory approval for any of our product candidates, we will be subject to ongoing regulatory obligations and continued regulatory review, which may result in significant additional expense. Additionally, our product candidates, if approved, could be subject to post-market study requirements, marketing and labeling restrictions and even recall or market withdrawal if unanticipated safety issues are discovered following approval. In addition, we may be subject to penalties or other enforcement action if we fail to comply with regulatory requirements.
  • Product liability lawsuits brought against us could cause us to incur substantial liabilities and could limit commercialization of any product candidates that we may develop.
  • Risks Related to Our Reliance on Third Parties
  • We rely on third parties to conduct our clinical trials for our product candidates, as well as potential investigator-sponsored clinical trials of our product candidates. If these third parties do not successfully carry out their contractual duties, comply with regulatory requirements, or meet expected deadlines, we may not be able to obtain regulatory approval for or commercialize our product candidates and our business could be substantially harmed.
  • We might not realize the anticipated benefits of our current collaborations with Mabwell or NIH, or any other collaborations we enter into in the future.
  • We have established collaborations with Mabwell and NIH and may seek to establish additional collaborations, and, if we are not able to establish them on commercially reasonable terms, or at all, we may have to alter our development and commercialization plans.
  • We contract with third parties for the manufacture of our product candidates for preclinical development and clinical testing and expect to continue to do so for commercialization. This reliance on third parties increases the risk that we will not have sufficient quantities of our product candidates or products or such quantities at an acceptable cost, which could delay, prevent, or impair our development or commercialization efforts.
  • The third parties upon whom we rely for the supply of the active pharmaceutical ingredients used in our product candidates are our sole sources of supply, and the loss of any of these suppliers could significantly harm our business.
  • The manufacture of biologics is complex and our third-party manufacturers may encounter difficulties in production. If any of our third-party manufacturers encounter such difficulties, our ability to provide supply of product candidates for clinical trials or products for patients, if approved, could be delayed or prevented.
  • If any third-party manufacturer of any of our product candidates is unable to increase the scale of its production of such product candidates or increase the product yield of its manufacturing, then our costs to manufacture such product or product candidate may increase and any commercialization may be delayed.
  • Risks Related to Our Intellectual Property
  • If we are unable to obtain and maintain patent and other intellectual property protection for our technology and product candidates, or if the scope of the intellectual property protection obtained is not sufficiently broad, our competitors could develop and commercialize technology and drugs similar or identical to ours, and our ability to successfully commercialize our technology and drugs may be impaired, and we may not be able to compete effectively in our market.
  • Obtaining and maintaining our patent protection depends on compliance with various procedural, document submission, deadlines, fee payment and other requirements imposed by governmental patent agencies, and our patent protection could be reduced or eliminated if we fail to comply with these requirements. We may miss a filing deadline for patent protection on these inventions.
  • If our trademarks and trade names for our products or company name are not adequately protected in one or more countries where we intend to market our products, we may delay the launch of product brand names, use different trademarks or tradenames in different countries, or face other potentially adverse consequences to building our product brand recognition.
  • If we are unable to adequately protect and enforce our trade secrets, our business and competitive position would be harmed.
  • We may initiate, become a defendant in or otherwise become party to lawsuits to protect or enforce our intellectual property rights, which could be expensive, time-consuming, and unsuccessful.
  • Intellectual property litigation and administrative patent office patent validity challenges in one or more countries could cause us to spend substantial resources and distract our personnel from their normal responsibilities.
  • We may be unable to obtain patent or other intellectual property protection for our current or future product candidates or our future products, if any, in all jurisdictions throughout the world, and we may not be able to adequately enforce our intellectual property rights even in the jurisdictions where we seek protection.
  • We may not obtain licenses or sublicenses to intellectual property rights in all markets on equally or sufficiently favorable terms with third parties.
  • If we fail to comply with our obligations in any agreements under which we may license intellectual property rights from third parties or otherwise experience disruptions to our business relationships with our licensors, we could lose license rights that are important to our business.
  • Any granted patents we may own or in-license covering our current or future product candidates or other valuable technology could be narrowed or found invalid or unenforceable if challenged in court or before administrative bodies in the U.S. or abroad, including the USPTO and the EPO. A patent asserted in a judicial court could be found invalid or unenforceable during the enforcement proceeding. Administrative or judicial proceedings challenging the validity of our patents or individual patent claims could take months or years to resolve.
  • Changes in patent law could diminish the value of patents in general, thereby impairing our ability to protect our current or future product candidates.
  • We may not identify relevant third-party patents or may incorrectly interpret the relevance, scope or expiration of a third-party patent, which might subject us to infringement claims or adversely affect our ability to develop and market our current or future product candidates.
  • Intellectual property rights do not guarantee commercial success of current or future product candidates or other business activities. Numerous factors may limit any potential competitive advantage provided by our intellectual property rights.
  • Risks Related to Government Regulation
  • Obtaining and maintaining regulatory approval of our product candidates in one jurisdiction does not mean that we will be successful in obtaining regulatory approval of our product candidates in other jurisdictions.
  • We may seek priority review designation for one or more of our product candidates, but we might not receive such designation, and even if we do, such designation may not lead to a faster regulatory review or approval process.
  • We may seek orphan drug designation for certain of our product candidates, and we may be unsuccessful or may be unable to maintain the benefits associated with orphan drug designation, including the potential for market exclusivity.
  • We have received rare pediatric disease designation for bitopertin. However, a marketing application for bitopertin, if approved, may not meet the eligibility criteria for a rare pediatric disease priority review voucher.
  • A breakthrough therapy designation and fast track designation by the FDA, even if granted for any of our product candidates, may not lead to a faster development, regulatory review or approval process, and each designation does not increase the likelihood that any of our product candidates will receive regulatory approval in the United States.
  • We sought approval from the FDA for bitopertin for EPP and XLP, and we may seek approval for any of our other product candidates, under the Accelerated Approval Program. If we are not able to use such program, we may be required to conduct additional clinical trials beyond those that are contemplated, which would increase the expense of obtaining, and delay the receipt of, necessary marketing approvals, if we receive them at all. In addition, even if the Accelerated Approval Program is available to us, it may not lead to expedited approval of our product candidates, or approval at all.
  • We received a Commissioner’s National Priority Voucher for bitopertin but the accelerated timeline for review of our NDA submission for bitopertin in EPP and XLP by the FDA did not result in an approval and it is unclear what, if any, benefits this voucher may provide in the future.
  • If our drug product candidates or any of our future drug product candidates obtain regulatory approval, additional competitors could enter the market with generic versions of such products, which may result in a material decline in sales of our competing products.
  • If approved, our investigational products regulated as biologics may face competition from biosimilars or interchangeable products approved through an abbreviated regulatory pathway.
  • The FDA, the EMA and other regulatory authorities may implement additional regulations or restrictions on the development and commercialization of our product candidates, and such changes can be difficult to predict.
  • The FDA and other regulatory agencies actively enforce the laws and regulations prohibiting the promotion of off-label uses.
  • Inadequate funding for the NIH, CMS, FDA, the SEC and other government agencies, including from government shut downs, or other disruptions to these agencies’ operations, including significant leadership, personnel, or policy changes, could prevent new products and services from being developed or commercialized in a timely manner or otherwise prevent those agencies from performing normal business functions on which the operation of our business may rely, which could negatively impact our business.
  • Current and future healthcare reform measures may have a material adverse effect on our business and results of operations.
  • Our relationships with customers, healthcare providers, physicians and third-party payors will be subject to applicable anti-kickback, fraud and abuse and other healthcare laws and regulations, which could expose us to criminal sanctions, civil penalties, exclusion from government healthcare programs, contractual damages, reputational harm and diminished future profits and earnings.
  • 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.
  • Compliance with U.S. and global privacy and security requirements could result in additional costs and liabilities to us or inhibit our ability to collect and process data globally, and the failure to comply with such requirements could expose us to criminal sanctions, civil penalties, contractual damages, reputational harm, administrative burdens and diminished profits and future earnings.
  • The use of new and evolving technologies, such as artificial intelligence, in our business may result in spending material resources and presents risks and challenges that can impact our business including by posing security and other risks to our confidential and/or proprietary information, including personal information, and as a result we may be exposed to reputational harm and liability.
  • Laws and regulations governing any international operations we may have in the future may preclude us from developing, manufacturing and selling certain products outside of the United States and require us to develop and implement costly compliance programs.
  • Risks Related to Employee Matters and Managing Growth
  • Our future success depends on our ability to retain key executives and experienced scientists and to attract, retain, and motivate qualified personnel.
  • We may be unable to adequately protect our information systems and infrastructure from cyberattacks or security compromises, cybersecurity incidents or data breaches, which could result in the disclosure of confidential or proprietary information, including personal data, damage our reputation, and subject us to significant financial and legal exposure.
  • We expect to expand our development and regulatory capabilities and potentially implement sales, marketing and distribution capabilities, and as a result, we may encounter difficulties in managing our growth, which could disrupt our operations.
  • General Risk Factors
  • Adverse developments affecting the financial services industry, such as actual events or concerns involving liquidity, defaults, or non-performance by financial institutions or transactional counterparties, could adversely affect the Company’s current and projected business operations and its financial condition and results of operations.
  • Changes in tax law may adversely affect us or our investors.
  • Our future taxable income may be subject to certain limitations.
  • We are subject to certain U.S. and foreign anti-corruption, anti-money laundering, export control, sanctions and other trade laws and regulations. We can face serious consequences for violations.
  • Significant political, trade or regulatory developments, unfavorable global economic conditions, and other circumstances beyond our control, could have a material adverse effect on our business, financial condition or results of operations.
  • Our employees, principal investigators, CROs and consultants may engage in misconduct or other improper activities, including non-compliance with regulatory standards and requirements or insider trading.

Legal proceedings

From time to time, we may be subject to legal proceedings and claims in the ordinary course of business. We are not currently aware of any such proceedings or claims that we believe will have, individually or in the aggregate, a material adverse effect on our business, financial condition or results of operations.

The opening of Item 3, Legal Proceedings.4

Sources

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