RYTM on Nasdaq

Rhythm (United states)

American pharmaceutical company based in Boston, founded in 20081

In its 2026 annual report the company says: “We are a global, commercial-stage biopharmaceutical company dedicated to transforming the lives of patients living with rare neuroendocrine diseases. We are focused on advancing melanocortin-4 receptor (MC4R) agonists, including our lead asset, IMCIVREE ® (setmelanotide), as precision medicines designed to treat hyperphagia and severe obesity caused by rare MC4R pathway diseases.”6 Its shares trade on Nasdaq under the symbol RYTM.1

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Last updated Oct 21, 2026.

Overview

2008Founded1

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  • TickerRYTM on Nasdaq1
  • Industrypharmaceutical industry1
  • HeadquartersBoston1
  • Websitewww.rhythmtx.com
  • Incorporated inDE4
  • Business address222 Berkeley Street, 12Th Floor, Boston, Ma 021164
  • Filer categoryLarge accelerated filer4
  • Legal formCorporation3
  • LEI529900JPNWXQ8DPLA5513
  • StatusActive3
  • LEI statusIssued and current3
  • Registered addressC/O CORPORATION SERVICE COMPANY, 19808 Wilmington, United States3
  • Registered withDivision of Corporations, Department of State, 52874583

We are a global, commercial-stage biopharmaceutical company dedicated to transforming the lives of patients living with rare neuroendocrine diseases. We are focused on advancing melanocortin-4 receptor (MC4R) agonists, including our lead asset, IMCIVREE ® (setmelanotide), as precision medicines designed to treat hyperphagia and severe obesity caused by rare MC4R pathway diseases. While obesity affects hundreds of millions of people worldwide, we are developing therapies for a subset of individuals who have hyperphagia, a pathological, insatiable hunger and impaired satiety accompanied by persistent and abnormal food-seeking behaviors, decreased energy expenditure and severe obesity due to diseases such as acquired or congenital hypothalamic obesity, Bardet-Biedl syndrome (BBS), Prader-Willi syndrome (PWS) and other diseases caused by impaired MC4R pathway signaling. The MC4R pathway is a…6 From its latest annual report

Financials

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

Key numbers, fiscal 2025

-101.2%Operating margin7
-103.6%Net margin7
45.8%Revenue growth, 20257
-141.3%Return on equity7
0.73×Debt to equity7
-$116.6MFree cash flow7

Where the money goes

Where each dollar of revenue went, fiscal 2025

  • Cost of revenue5¢$19.5M
  • Research and development44¢$167.3M
  • Selling, general and admin51¢$194.9M
  • Income tax0¢$497.0K

Out of $189.8M in revenue.7 The company lost money this year.

Financial statements

Revenue89107$189.8M in 2025
0$50.0M$100.0M$150.0M$200.0M2019: $0$020192020: $0$020202021: $3.2M$3.2M20212022: $23.6M$23.6M2022+649%2023: $77.4M$77.4M2023+228%2024: $130.1M$130.1M2024+68%2025: $189.8M$189.8M2025+46%
Operating income11129107-$192.0M in 2025+32% since 2019
-$300.0M-$200.0M-$100.0M02019: -$146.0M-$146.0M20192020: -$136.6M-$136.6M2020−6%2021: -$170.1M-$170.1M2021+25%2022: -$179.2M-$179.2M2022+5%2023: -$184.4M-$184.4M2023+3%2024: -$265.5M-$265.5M2024+44%2025: -$192.0M-$192.0M2025−28%
Net income11129107-$196.5M in 2025+40% since 2019
-$300.0M-$200.0M-$100.0M02019: -$140.7M-$140.7M20192020: -$134.0M-$134.0M2020−5%2021: -$69.6M-$69.6M2021−48%2022: -$181.1M-$181.1M2022+160%2023: -$184.7M-$184.7M2023+2%2024: -$260.6M-$260.6M2024+41%2025: -$196.5M-$196.5M2025−25%

Every line of the income statement as filed: the last 3 fiscal years, latest first.7

Period ending2025-12-312024-12-312023-12-31
Total revenues$189.8M$130.1M$77.4M
Cost of sales$19.5M$13.4M$9.3M
Research and development$167.3M$238.0M$135.0M
Selling, general, and administrative$194.9M$144.3M$117.5M
Total costs and expenses$381.8M$395.6M$261.8M
Loss from operations-$192.0M-$265.5M-$184.4M
Other income, net$1.3M$2.2M$190.0K
Gain on settlement of forward contract$0$8.9M$0
Interest expense-$20.6M-$20.6M-$13.9M
Interest income$15.3M$14.7M$13.9M
Total other income (expense), net-$4.0M$5.2M$243.0K
Loss before income taxes-$196.0M-$260.3M-$184.1M
Provision for income taxes$497.0K$346.0K$564.0K
Net loss-$196.5M-$260.6M-$184.7M
Accrued dividends on convertible preferred stock-$5.4M-$4.0M$0
Net loss attributable to common stockholders-$201.9M-$264.6M-$184.7M
Net loss per share attributable to common stockholders, basic (in dollars per share)-$3.11-$4.34-$3.20
Net loss per share attributable to common stockholders, diluted (in dollars per share)-$3.11-$4.34-$3.20
Weighted-average common shares outstanding, basic (in shares)65.0M shares61.0M shares57.7M shares
Weighted-average common shares outstanding, diluted (in shares)65.0M shares61.0M shares57.7M shares
Foreign currency translation adjustment-$1.3M$2.0K-$140.0K
Unrealized (loss) gain, net on marketable securities, net of tax$504.0K-$175.0K$366.0K
Comprehensive loss-$202.7M-$264.7M-$184.5M

Compare Rhythm (United states) with another company

Corporate structure

Rhythm (United states) sits at the top of its group. 12 entities, 10 countries on record. Switch between the flow chart and the network, and select any entity to see its details.

Major holders of Rhythm (United states)

  • JPMorgan Chase Filed SCHEDULE 13G on Rhythm (United states), latest Oct 13, 2026
  • PERCEPTIVE ADVISORS LLC Filed SCHEDULE 13G/A on Rhythm (United states), latest May 15, 2026 (7 filings since Feb 12, 2024)
  • VANGUARD CAPITAL MANAGEMENT LLC Filed SCHEDULE 13G on Rhythm (United states), latest Apr 30, 2026
  • VANGUARD GROUP INC Filed SCHEDULE 13G/A on Rhythm (United states), latest Mar 27, 2026 (4 filings since Feb 13, 2024)
  • RA CAPITAL MANAGEMENT, L.P. Filed SCHEDULE 13G/A on Rhythm (United states), latest Feb 17, 2026 (6 filings since Feb 14, 2022)
  • PRIMECAP MANAGEMENT CO/CA/ Filed SCHEDULE 13G/A on Rhythm (United states), latest Feb 12, 2026 (9 filings since Feb 10, 2022)
  • BAKER BROS. ADVISORS LP Filed SCHEDULE 13G/A on Rhythm (United states), latest May 15, 2025 (5 filings since Feb 14, 2022)
  • BlackRock Filed SCHEDULE 13G/A on Rhythm (United states), latest Apr 24, 2025 (6 filings since Feb 3, 2022)
  • Goldman Sachs Filed SCHEDULE 13G/A on Rhythm (United states), latest Feb 10, 2025 (5 filings since Jan 24, 2022)
  • NEW ENTERPRISE ASSOCIATES 13 LP Filed SC 13D/A on Rhythm (United states), latest Apr 30, 2024 (2 filings since Mar 29, 2023)
  • State Street Corporation Filed SC 13G/A on Rhythm (United states), latest Jan 23, 2024 (2 filings since Feb 3, 2023)
  • Federated Hermes Filed SC 13G/A on Rhythm (United states), latest Sep 7, 2023 (5 filings since Dec 10, 2021)
  • Rhythm (United states) Boston, Massachusetts, United States
    • Rhythm Pharma K.K. Subsidiary · Japan
    • Rhythm Pharmaceuticals Canada Inc. Subsidiary · Canada
    • Rhythm Pharmaceuticals France SAS Subsidiary · France
    • Rhythm Pharmaceuticals Germany GmbH Subsidiary · Germany
    • Rhythm Pharmaceuticals Italy S.r.l. Subsidiary · Italy
    • Rhythm Pharmaceuticals Netherlands B.V. Subsidiary · Netherlands
    • Rhythm Pharmaceuticals Spain, SL Subsidiary · Spain
    • Rhythm Pharmaceuticals Switzerland GmbH Subsidiary · Switzerland
    • Rhythm Pharmaceuticals UK Limited Subsidiary · United Kingdom
    • Rhythm Securities Corp. Subsidiary · Massachusetts, United States
    • Xinvento B.V. Subsidiary · Netherlands

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. Each entity in the chart links to its source.

What it owns

What Rhythm (United states) 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$6.4M7 Dec 31, 2025$5.4M7 Dec 31, 2024+17.7%
Accumulated depreciation$5.2M7 Dec 31, 2025$4.8M7 Dec 31, 2024+10.1%
Property and equipment, net$1.1M7 Dec 31, 2025$632.0K7 Dec 31, 2024+74.7%
Operating-lease assets (leased space)$3.0M7 Dec 31, 2025$3.5M7 Dec 31, 2024−12.3%

Investments

ItemLatestA year beforeChange
Cash and cash equivalents$54.3M7 Dec 31, 2025$89.1M7 Dec 31, 2024−39.1%
Marketable securities, current$334.6M7 Dec 31, 2025$231.4M7 Dec 31, 2024+44.6%
Debt securities available for sale$334.6M7 Dec 31, 2025$231.4M7 Dec 31, 2024+44.6%

Intangibles and goodwill

ItemLatestA year beforeChange
Finite-lived intangibles, net$5.3M7 Dec 31, 2025$6.2M7 Dec 31, 2024−13.8%

Inventory

ItemLatestA year beforeChange
Inventory, total$25.8M7 Dec 31, 2025$18.7M7 Dec 31, 2024+37.4%
Work in process$1.6M7 Dec 31, 2025$1.2M7 Dec 31, 2024+30.5%
Finished goods$17.4M7 Dec 31, 2025$10.7M7 Dec 31, 2024+62.2%

Properties, in its own words

Our corporate headquarters are located in Boston, Massachusetts, where we lease approximately 13,600 square feet of office space pursuant to lease agreements expiring in July 2030. This facility houses our research, clinical, regulatory, commercial and administrative personnel.

We believe that our existing facilities are adequate for our near-term needs, but we may need additional space as we grow and expand our operations. We believe that suitable additional or alternative office space would be available as required in the future on commercially reasonable terms.

Item 2 of the 10-K filed Feb 26, 2026.6

Leadership

Who has led Rhythm (United states), and when. Pick a name to see everywhere that person has worked.

Board members and other officers (25)

Subsidiaries and ownership

Subsidiaries (Exhibit 21)

  • Rhythm Pharma K.K. Japan5
  • Rhythm Pharmaceuticals Canada Inc. Canada5
  • Rhythm Pharmaceuticals France SAS France5
  • Rhythm Pharmaceuticals Germany GmbH Germany5
  • Rhythm Pharmaceuticals Italy S.r.l. Italy5
  • Rhythm Pharmaceuticals Netherlands B.V. The Netherlands5
  • Rhythm Pharmaceuticals Spain, SL Spain5
  • Rhythm Pharmaceuticals Switzerland GmbH Switzerland5
  • Rhythm Pharmaceuticals UK Limited United Kingdom5
  • Rhythm Securities Corp. Massachusetts5
  • Xinvento B.V. The Netherlands5

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

Former names

  • RHYTHM METABOLIC, INC.Aug 12, 2015 to Aug 12, 20154

Timeline

  1. Aug 4, 20268-K Results of operations; Regulation FD disclosure; Other events43
  2. Jun 25, 20268-K Shareholder vote44
  3. Jun 15, 20268-K Regulation FD disclosure; Other events45
  4. May 5, 20268-K Results of operations46
  5. Apr 3, 20268-K Executive or director change; Regulation FD disclosure47
  6. Mar 26, 20268-K Regulation FD disclosure; Other events48
  7. Mar 20, 20268-K Regulation FD disclosure; Other events49
  8. Mar 16, 20268-K Regulation FD disclosure; Other events50
  9. Mar 2, 20268-K Regulation FD disclosure; Other events51
  10. Feb 26, 20268-K Results of operations52
  11. Jan 9, 20268-K Results of operations; Regulation FD disclosure; Other events53
  12. Dec 19, 20258-K Executive or director change54
  13. Jul 21, 2020New chief executive David P. Meeker becomes CEO16
  14. Mar 31, 2020New chief executive Hunter C. Smith becomes CEO15
  15. Oct 4, 2017New chief executive Keith Michael Gottesdiener becomes CEO17
  16. Oct 4, 2017Went public Listed on NASDAQ1
  17. 2008Founded Founded1

Calendar

Coming up

  1. Nov 3, 2026Reports results for the quarter ending Sep/2026 A year earlier it reported ($0.82) a share, on Nov 4, 2025.55

When it reported results

  1. Aug 4, 2026Reported quarterly or annual results43
  2. May 5, 2026Reported quarterly or annual results46
  3. Feb 26, 2026Reported quarterly or annual results52
  4. Jan 9, 2026Reported quarterly or annual results53
  5. Nov 4, 2025Reported quarterly or annual results56
  6. Aug 5, 2025Reported quarterly or annual results57
  7. May 7, 2025Reported quarterly or annual results58
  8. Feb 26, 2025Reported quarterly or annual results59

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

Filings

492 filings to the SEC since October 2021: 5 annual reports, 16 quarterly reports, 60 major events, 243 insider forms, 56 large holders, 112 other. The latest 20:

  1. Oct 13, 2026SCHEDULE 13G EDGAR
  2. Sep 14, 2026144 EDGAR
  3. Sep 14, 20264 PRIMARY DOCUMENT EDGAR
  4. Sep 1, 20264 PRIMARY DOCUMENT EDGAR
  5. Aug 31, 2026144 EDGAR
  6. Aug 18, 20264 PRIMARY DOCUMENT EDGAR
  7. Aug 18, 20263 PRIMARY DOCUMENT EDGAR
  8. Aug 17, 20264 PRIMARY DOCUMENT EDGAR
  9. Aug 17, 20264 PRIMARY DOCUMENT EDGAR
  10. Aug 14, 20264 PRIMARY DOCUMENT EDGAR
  11. Aug 14, 20264 PRIMARY DOCUMENT EDGAR
  12. Aug 13, 2026144 EDGAR
  13. Aug 13, 2026144 EDGAR
  14. Aug 13, 2026144 EDGAR
  15. Aug 12, 2026144 EDGAR
  16. Aug 11, 2026144 EDGAR
  17. Aug 10, 2026144 EDGAR
  18. Aug 4, 202610-Q EDGAR
  19. Aug 4, 20268-K EDGAR
  20. Jul 2, 20264 OWNERSHIP DOCUMENT EDGAR

The 492 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

151 open-market trades by officers, directors and large owners since October 2021: 0 buys and 151 sales. Grants, option exercises and gifts are left out.

DateWhoTradeSharesPriceValueFiling
Sep 14, 2026Meeker David PPresident and CEOSale50,000$102.67$5.1MForm 4
Aug 31, 2026Smith Hunter CChief Financial OfficerSale62,774$105.46$6.6MForm 4
Aug 13, 2026Shulman JosephChief Technical OfficerSale5,079$119.75$608.2KForm 4
Aug 13, 2026Garfield AlastairChief Scientific OfficerSale7,763$117.57$912.7KForm 4
Aug 13, 2026Lee Jennifer KaydenEVP, Head of North AmericaSale75,834$117.38$8.9MForm 4
Aug 12, 2026Mazabraud YannEVP, Head of InternationalSale57,000$116.15$6.6MForm 4
Jun 22, 2026TETRAULT LYNN A.DirectorSale37,000$100.32$3.7MForm 4
May 15, 2026German Christopher PaulCorporate Controller & CAOSale4,643$92.21$428.1KForm 4
Mar 4, 2026German Christopher PaulCorporate Controller & CAOSale5,614$89.56$502.8KForm 4
Feb 19, 2026Smith Hunter CChief Financial OfficerSale4,385$101.26$444.0KForm 4
Feb 17, 2026Smith Hunter CChief Financial OfficerSale3,569$101.38$361.8KForm 4
Feb 12, 2026Smith Hunter CChief Financial OfficerSale1,592$98.23$156.4KForm 4
Feb 11, 2026Smith Hunter CChief Financial OfficerSale2,766$98.61$272.8KForm 4
Feb 10, 2026Smith Hunter CChief Financial OfficerSale2,713$103.00$279.4KForm 4
Feb 5, 2026Smith Hunter CChief Financial OfficerSale3,381$106.06$358.6KForm 4
Feb 4, 2026Smith Hunter CChief Financial OfficerSale3,247$105.99$344.1KForm 4
Dec 11, 2025Cramer Pamela J.Chief Human Resources OfficerSale3,350$111.00$371.9KForm 4
Nov 20, 2025Cramer Pamela J.Chief Human Resources OfficerSale3,350$105.00$351.8KForm 4
Nov 10, 2025Cramer Pamela J.Chief Human Resources OfficerSale3,515$100.00$351.5KForm 4
Nov 7, 2025Cramer Pamela J.Chief Human Resources OfficerSale19,351$98.82$1.9MForm 4

All 151 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
2025David P. Meeker71$807.7K$17.5M74
2025Hunter C. Smith58$540.5K$6.2M74
2025Yann Mazabraud53$557.2K$6.1M74
2025Jennifer Lee51$531.6K$5.6M74
2025Alastair GarfieldChief Scientific Officer46$505.4K$4.4M74
2024Chief Executive Officer$740.5K$14.7M74
2024Chief Financial Officer$499.4K$5.1M74
2024Executive VicePresident,$488.3K$4.3M74
2023Chief Executive Officer$704.7K$7.2M74
2023Chief Financial Officer$476.2K$2.3M74
2023Head of North America$464.7K$2.1M74
2023Head of International$448.8K$1.5M74

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
Ra Capital Management, L.P.6,666,837$740.2M6.4%No change
BlackRock, Inc.6,156,902$683.6M0.0%−80,249
Baker Bros. Advisors LP5,604,483$622.3M3.1%No change
Primecap Management Co/ca/3,168,853$351.8M0.2%−248,656
Vanguard Portfolio Management LLC3,136,895$348.3M0.0%+96,177
Vanguard Capital Management LLC3,005,977$333.8M0.0%+38,152
Jpmorgan Chase & Co2,652,746$292.2M0.0%+1,099,102
Perceptive Advisors LLC2,586,295$287.2M4.5%+212,500
State Street Corp2,573,529$285.7M0.0%+301,678
Westfield Capital Management Co LP2,208,595$245.2M0.8%+279,472
Goldman Sachs Group Inc2,126,502$236.1M0.0%+161,070
Federated Hermes, Inc.1,785,716$198.3M0.3%+499,413
Polar Capital Holdings Plc1,663,632$184.7M0.5%+48,046
Geode Capital Management, LLC1,533,698$170.3M0.0%+117,429
Capital Research Global Investors1,532,303$170.1M0.0%+416,843
Citadel Advisors LLC1,263,652$140.3M0.1%−154,967
Checkpoint Capital L.P.1,186,000$131.7M33.8%−41,200
Siren, L.L.C.1,136,275$126.2M2.6%+410,764
Morgan Stanley1,112,240$123.5M0.0%+408,262
Fmr LLC1,025,840$113.9M0.0%−663,831
Pictet Asset Management Holding SA957,785$106.3M0.1%+290,739
Jupiter Topco LLC925,490$102.8M0.0%New
Woodline Partners LP758,891$84.3M0.3%No change
Alliancebernstein L.P.894,723$77.8M0.0%−15,690
Clearbridge Investments, LLC696,473$77.3M0.1%+87,036

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

SEC filer profile

How Rhythm (United states) is registered with the Securities and Exchange Commission, from its EDGAR record and the cover page of its latest annual report.

  • Name on EDGARRHYTHM PHARMACEUTICALS, INC.2
  • CIK0001649904
  • Employer ID (EIN)46-21592716
  • Filer statusLarge accelerated filer not an emerging growth company; not a smaller reporting company; not a shell company6
  • Incorporated inDelaware6
  • Fiscal year endsDecember 312
  • Industry code (SIC)2834 Pharmaceutical Preparations2
  • SEC reviewing officeOffice of Life Sciences, Division of Corporation Finance2
  • Business address222 Berkeley Street, 12Th Floor, Boston, MA 021162
  • Phone857-264-42802
  • Shares outstanding68,852,082 as of Aug 1, 202614
  • Public float$3.8B as of Jun 30, 20256
  • AuditorErnst & Young LLP, Boston, Massachusetts PCAOB ID 42; it also attests to the company’s internal control over financial reporting6
  • On EDGAR sinceAug 12, 20152
  • Filings835 in all: 4 (376), 8-K (106), 144 (73), 3 (45), SC 13G/A (44), 10-Q (27), SC 13G (17), SCHEDULE 13G/A (15)2
    All 41 form types

    S-8 (13), SC 13D/A (11), DEFA14A (10), 10-K (9), 424B5 (9), DEF 14A (9), 4/A (8), CORRESP (8), UPLOAD (6), ARS (4), DRS/A (4), DRSLTR (4), SC 13D (4), CT ORDER (3), EFFECT (3), S-3ASR (3), 424B4 (2), D (2), DRS (2), S-1 (2), S-1/A (2), S-1MEF (2), SCHEDULE 13G (2), 10-Q/A (1), 424B7 (1), 5 (1), 8-A12B (1), 8-K/A (1), CERTNAS (1), D/A (1), DEFR14A (1), PRE 14A (1), S-3 (1)

  • Latest filingsAnnual report (10-K) Feb 26, 2026
    Quarterly report (10-Q) Aug 4, 2026
    Proxy statement (DEF 14A) Apr 29, 2026
    Latest 8-K (8-K) Aug 4, 2026

Securities registered for trading

ClassSymbolExchange
Common Stock, $0.001 par value per shareRYTMNasdaq

From the cover page of the 10-K filed Feb 26, 2026 (Section 12(b) of the Exchange Act).6

Risk factors and legal proceedings

The risks Rhythm (United states) names in Item 1A of its 10-K filed Feb 26, 2026: 72 risks under these headings, as written.6

  • Risks Related to Our Financial Position and Need for Capital
  • We are a commercial stage biopharmaceutical company with a limited operating history and have not generated significant revenue from product sales. We have incurred significant operating losses since our inception, anticipate that we will incur continued losses for the foreseeable future and may never achieve profitability.
  • We expect we may need to raise additional funding, which may not be available on acceptable terms, or at all. Failure to obtain this necessary capital when needed may force us to delay, limit or terminate our product development efforts or other operations.
  • Our Revenue Interest Financing Agreement with Healthcare Royalty Partners could restrict our ability to commercialize IMCIVREE, limit cash flow available for our operations and expose us to risks that could adversely affect our business, financial condition and results of operations.
  • Risks Related to the Development of Setmelanotide, Bivamelagon, RM-718 and our CHI Program
  • Positive results from earlier clinical trials of setmelanotide may not be predictive of the results of later clinical trials of setmelanotide. If we cannot generate positive results in our later clinical trials of setmelanotide, we may be unable to successfully develop, obtain regulatory approval for, and commercialize additional indications for setmelanotide.
  • Interim, “topline” and preliminary data from our preclinical and clinical trials that we announce or publish from time to time may change as more data become available and are subject to audit and verification procedures that could result in material changes in the final data.
  • The exclusive license agreement with LGC is important to our business. If we or LGC fail to adequately perform under the agreement, the development of bivamelagon could be delayed, or if we or LGC terminate the agreement, we would lose our rights to develop and commercialize bivamelagon.
  • The number of patients with each of the MC4R pathway variants we are targeting is small and has not been established with precision. If the actual number of patients is smaller than we estimate, our revenue and ability to achieve profitability may be materially adversely affected.
  • If we experience delays or difficulties in the enrollment and/or retention of patients in clinical trials, our regulatory submissions or receipt of additional marketing approvals could be delayed or prevented.
  • Failures or delays in the commencement or completion of our planned clinical trials could result in increased costs to us and could delay, prevent or limit our ability to generate revenue, achieve profitability and continue our business.
  • Research and development in the pharmaceutical industry is costly, risky, time-intensive and complicated. In particular, our CHI program is a pre-clinical discovery-stage program and we may not succeed in identifying a CHI program candidate to translate to development and even if we do we may not succeed in developing a CHI program product candidate.
The other 62 risks
  • Setmelanotide, RM-718 or bivamelagon may cause undesirable side effects that could delay or prevent additional regulatory approvals, limit the commercial profile of approved labeling, or result in significant negative consequences following marketing approval.
  • We may not be able to obtain or maintain orphan drug designations for setmelanotide, RM-718 or bivamelagon or to obtain or maintain orphan exclusivity. Even with exclusivity, competitors may obtain approval for different drugs that treat the same indications as setmelanotide, RM-718 and bivamelagon.
  • We may not be able to translate the once-daily, subcutaneous injection formulations of setmelanotide into alternate formulations, including alternate methods of delivery for setmelanotide or alternative methods of delivery for or other product candidates that would be acceptable to the FDA or other equivalent competent authorities in foreign jurisdictions or commercially successful.
  • We rely, and expect that we will continue to rely, on third parties to conduct research and discovery activities and in our clinical trials. If these third parties do not successfully carry out their contractual obligations or meet expected timelines, we may not be able to advance our pre-clinical and clinical programs or obtain, on a timely basis or at all, additional regulatory approvals for or commercialize our product candidates, and our business could be substantially harmed.
  • Risks Related to the Commercialization of IMCIVREE and, if Approved, our Products Candidates
  • The successful commercialization of IMCIVREE and any other product candidates for which we obtain approval will depend in part on the extent to which governmental authorities, private health insurers, and other third-party payors provide coverage and adequate reimbursement. Failure to obtain or maintain coverage and adequate reimbursement for IMCIVREE or our other product candidates, if any and if approved, could limit our ability to market those products and decrease our ability to generate revenue.
  • If we are unable to establish, maintain or expand our sales, marketing and distribution capabilities or enter into agreements with third parties to market, sell, and distribute IMCIVREE, we may not be able to generate revenue.
  • We may never receive regulatory approval to market setmelanotide outside of the United States, Canada, the European Union and the United Kingdom.
  • We may not achieve or maintain market acceptance for IMCIVREE, which would limit the revenue that we generate from the sale of IMCIVREE.
  • Our industry is intensely competitive. If we are not able to compete effectively against current and future competitors, we may not be able to generate revenue from the sale of IMCIVREE, our business will not grow and our financial condition and operations will suffer.
  • We face potential product liability exposure, and, if claims are brought against us, we may incur substantial liability.
  • We rely completely on third-party suppliers to manufacture our clinical and commercial drug supplies of setmelanotide, RM-718, and bivamelagon, and we intend to rely on third parties to produce preclinical, clinical and commercial supplies of any future product candidate.
  • Risks Related to Our Intellectual Property Rights
  • If we are unable to adequately protect our proprietary technology or maintain issued patents directed to setmelanotide, RM-718, and bivamelagon, others could compete against us sooner, which could have a material adverse impact on our business, results of operations, financial condition and prospects.
  • We may be involved in lawsuits to protect or enforce our patents or the patents of our licensors, which could be expensive, time consuming and unsuccessful.
  • We may infringe the intellectual property rights of others, which may prevent or delay our product development efforts and stop us from commercializing or increase the costs of commercializing IMCIVREE or our other product candidates.
  • We may be subject to claims challenging the inventorship or ownership of our patents and other intellectual property.
  • Issued patents covering setmelanotide or our other product candidates could be found invalid or unenforceable if challenged in court.
  • We do not seek to protect our intellectual property rights 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 are dependent on licensed intellectual property. If we were to lose our rights to licensed intellectual property, we may not be able to continue developing or commercializing setmelanotide or bivamelagon.
  • While we have registered trademarks for the commercial trade name IMCIVREE (setmelanotide) in the United States, the European Union, and other jurisdictions, we have not yet obtained trademark protection for IMCIVREE in certain foreign jurisdictions and failure to secure such registrations could adversely affect our business.
  • If we do not obtain additional protection under the Hatch-Waxman Amendments and similar foreign legislation by extending the patent terms and obtaining product exclusivity for setmelanotide and our other product candidates, our business may be materially harmed.
  • Changes in U.S. patent law could diminish the value of patents in general, thereby impairing our ability to protect our products.
  • We may be subject to damages resulting from claims that we or our employees have wrongfully used or disclosed alleged trade secrets of their former employers.
  • Risks Related to Regulatory Approval and Marketing of IMCIVREE and Other Legal and Compliance Matters
  • Even if we complete the necessary clinical trials, the regulatory and marketing approval process is expensive, time consuming and uncertain and may prevent us from obtaining additional approvals for the commercialization of our product candidates. If we are not able to obtain, or if there are delays in obtaining, required regulatory approvals for our product candidates, we will not be able to commercialize such candidates and our ability to generate revenue will be materially impaired.
  • Future regulatory legislation or regulation may increase the difficulty and cost for us to obtain marketing approval of and commercialize our product candidates.
  • Disruptions at the FDA, including those caused by changing policy priorities, funding shortages staffing or other resource limitations could hinder their ability to hire, retain or deploy key leadership and other personnel, or otherwise prevent new or modified products from being developed, approved or commercialized in a timely manner or at all, which could negatively impact our business.
  • Our failure to obtain marketing approval in foreign jurisdictions would prevent setmelanotide or our other product candidates from being marketed abroad, and any current or future approvals we have been or may be granted for setmelanotide or other products in the United States would not assure approval of setmelanotide or other products in foreign jurisdictions.
  • The terms of our current and future potential marketing approvals for setmelanotide and other product candidates and ongoing regulation may limit how we manufacture and market setmelanotide and other products, and compliance with such requirements may involve substantial resources, which could materially impair our ability to generate revenue.
  • Current and future healthcare reform legislation or regulation may increase the difficulty and cost for us and any future collaborators to commercialize setmelanotide and may adversely affect the prices we, or they, may obtain and may have a negative impact on our business and results of operations.
  • If we fail to comply with our reporting and payment obligations under the Medicaid Drug Rebate Program or other governmental pricing programs in which we participate, we could be subject to additional reimbursement requirements, penalties, sanctions and fines, which could have a material adverse effect on our business, financial condition, results of operations and growth prospects.
  • The FDA and other regulatory agencies actively enforce the laws and regulations prohibiting the promotion of off-label uses.
  • We may be subject to federal, state and foreign healthcare laws and regulations, including fraud and abuse laws, health information privacy and security laws, and antitrust laws. If we are unable to comply or have not fully complied with such laws and regulations, we could face criminal sanctions, damages, substantial civil penalties, reputational harm and diminished profits and future earnings.
  • Our employees may engage in misconduct or other improper activities, whether knowingly or unknowingly, including violating applicable regulatory standards and requirements or engaging in insider trading, which could significantly harm our business.
  • Actual or perceived failure to comply with data protection, privacy and security laws and regulations could lead to government enforcement actions and significant penalties against us, and adversely impact our operating results.
  • Our future growth depends, in part, on our ability to continue to penetrate foreign markets, where we will be subject to additional regulatory burdens and other risks and uncertainties.
  • Laws and regulations governing any international operations we may have in the future may preclude us from developing, manufacturing and selling setmelanotide or our other product candidates outside of the United States and require us to develop and implement costly compliance programs.
  • The results of the United Kingdom’s departure from the EU, and the resulting impact of differing regulations, may have a negative effect on our business.
  • Risks Related to Employee Matters and Managing Growth
  • Our future success depends on our ability to retain our key employees and consultants, and to attract, retain and motivate qualified personnel.
  • We will need to develop and expand our Company, and we may encounter difficulties in managing this development and expansion, which could disrupt our operations.
  • Our information technology systems, or those of our third-party CROs, CMOs or other contractors or consultants, may fail or suffer security breaches, which could result in a material disruption of setmelanotide and other product candidate development programs, regulatory investigations, enforcement actions and lawsuits.
  • Risks Related to Our Common Stock
  • Our directors and executive officers and their affiliated entities own a significant percentage of our stock and, if they choose to act together, will be able to exert significant influence over matters subject to stockholder approval.
  • Market volatility may affect our stock price and the value of your investment.
  • Our quarterly operating results may fluctuate significantly.
  • Our ability to use certain net operating loss carryovers and other tax attributes may be limited.
  • Substantial future sales or perceived potential sales of our common stock in the public market could cause the price of our common stock to decline significantly.
  • We may be at an increased risk of securities litigation, including class action litigation.
  • We do not intend to pay dividends on our common stock and, consequently, your ability to achieve a return on your investment will depend on appreciation in the price of our common stock.
  • Our common stock is subordinated to our Convertible Preferred Stock.
  • Provisions in our certificate of incorporation and bylaws and Delaware law might discourage, delay or prevent a change in control of our Company or changes in our management and, therefore, depress the market price of our common stock.
  • Our certificate of incorporation provides that the Court of Chancery of the State of Delaware is the exclusive forum for substantially all disputes between us and our stockholders and our bylaws designate the federal district courts of the United States as the exclusive forum for actions arising under the Securities Act, which could limit our stockholders’ ability to obtain an alternate preferred judicial forum for disputes with us or our directors, officers or employees.
  • General Risk Factors
  • We have in the past and may in the future acquire businesses or products, form strategic alliances or create joint ventures in the future, and we may not realize their benefits.
  • An active market for our common stock may not be maintained.
  • If securities or industry analysts do not continue to publish research or reports or publish unfavorable research or reports about our business, our stock price and trading volume could decline.
  • Raising additional capital may cause dilution to our existing stockholders, restrict our operations or require us to relinquish rights.
  • Unfavorable global political or economic conditions could adversely affect our business, financial condition or results of operations.
  • Business interruptions could adversely affect our operations.
  • We have incurred and will continue to incur substantial costs as a result of operating as a public company, our management will continue to devote substantial time to existing and new compliance initiatives and corporate governance policies, and we will need to hire additional qualified accounting, financial, legal and compliance personnel with appropriate public company experience.
  • We may in the future fail to maintain an effective system of internal control over financial reporting. This may prevent us from accurately reporting our financial results or preventing fraud. As a result, stockholders could lose confidence in our financial and other public reporting, which would harm our business and the trading price of our common stock.
  • We have previously identified a material weakness in our internal controls over financial reporting and may identify additional material weaknesses in the future or otherwise fail to maintain an effective system of internal controls, which may result in material misstatements of our consolidated financial statements or cause us to fail to meet our periodic reporting obligations.
  • The increasing focus on environmental sustainability and social initiatives could increase our costs, harm our reputation and adversely impact our financial results.
  • Short sellers of our stock may be manipulative and may drive down the market price of our common stock.

Legal proceedings

We are not currently a party to any material legal proceedings.

The opening of Item 3, Legal Proceedings.6

Sources

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