Private company
Avidity Biosciences, Inc.
American pharmaceutical preparations company based in San Diego1
In its 2026 annual report the company says: “We are a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates, or AOCs. Our proprietary AOC platform is designed to combine the specificity of monoclonal antibodies, or mAbs, with the precision of RNA therapeutics to target the root cause of diseases previously untreatable with such therapeutics. Our pipeline currently has three programs in clinical development.”11
Last updated Oct 20, 2026.
Overview
Financials
From the company’s reports to the SEC. Every number links to the filing it came from.
Key numbers, fiscal 2025
Where the money goes
Where each dollar of revenue went, fiscal 2025
- Research and development100¢$559.2M
Out of $18.8M in revenue.3 The company lost money this year.
Financial statements
| Fiscal year | 2019 | 2020 | 2021 | 2022 | 2023 | 2024 | 2025 |
|---|---|---|---|---|---|---|---|
| Revenue | $2.3M4 | $6.8M5 | $9.3M6 | $9.2M7 | $9.6M3 | $10.9M3 | $18.8M3 |
| Research and development | $14.5M4 | $37.6M5 | $101.2M6 | $150.4M7 | $191.0M3 | $303.6M3 | $559.2M3 |
| Operating expenses | $127.4M6 | $188.1M7 | $245.2M3 | $389.8M3 | $764.7M3 | ||
| Operating income | -$17.3M4 | -$44.3M5 | -$118.1M6 | -$178.9M7 | -$235.6M3 | -$378.9M3 | -$745.9M3 |
| Interest | $7.4M4 | $209.0K5 | $05 | $05 | |||
| Income before tax | -$212.2M3 | -$322.3M3 | -$684.6M3 | ||||
| Income tax | $04 | $05 | $06 | $07 | $03 | $03 | $03 |
| Net income | -$24.7M4 | -$44.4M5 | -$118.0M6 | -$174.0M7 | -$212.2M3 | -$322.3M3 | -$684.6M3 |
| Earnings per share | -$2.055 | -$2.856 | -$3.347 | -$2.913 | -$2.893 | -$4.973 | |
| Earnings per share (diluted) | -$2.055 | -$2.856 | -$3.347 | -$2.913 | -$2.893 | -$4.973 | |
| Shares (weighted, basic) | 41.4M shares6 | 52.2M shares7 | 73.0M shares3 | 111.6M shares3 | 137.7M shares3 | ||
| Shares (weighted, diluted) | 41.4M shares6 | 52.2M shares7 | 73.0M shares3 | 111.6M shares3 | 137.7M shares3 |
| Fiscal year | 2019 | 2020 | 2021 | 2022 | 2023 | 2024 | 2025 |
|---|---|---|---|---|---|---|---|
| Cash | $94.6M10 | $321.5M4 | $320.4M5 | $340.4M6 | $185.1M7 | $219.9M3 | $382.5M3 |
| Receivables | $2.4M6 | $1.1M7 | $07 | ||||
| Current assets | $95.7M10 | $331.7M4 | $411.1M5 | $622.9M6 | $611.3M7 | $1.5B3 | $1.8B3 |
| Property, plant and equipment | $4.8M5 | $6.3M6 | $8.4M7 | $12.7M3 | $23.3M3 | ||
| Total assets | $96.9M10 | $333.9M4 | $427.6M5 | $638.8M6 | $628.6M7 | $1.6B3 | $2.0B3 |
| Accounts payable | $2.8M5 | $4.6M6 | $8.8M7 | $8.5M3 | $12.4M3 | ||
| Current liabilities | $10.2M10 | $14.6M4 | $29.7M5 | $51.9M6 | $80.7M7 | $98.0M3 | $195.4M3 |
| Long-term debt | $1.8M10 | ||||||
| Total liabilities | $27.5M10 | $27.7M4 | $46.1M5 | $60.7M6 | $127.8M7 | $138.9M3 | $269.4M3 |
| Retained earnings | -$184.5M5 | -$358.5M6 | -$570.8M7 | -$893.1M3 | -$1.6B3 | ||
| Shareholders’ equity | -$65.4M5 | $306.2M6 | $381.4M7 | $578.1M3 | $500.8M3 | $1.4B3 | $1.7B3 |
| Shares outstanding | 3.0M shares10 | 37.6M shares4 | 47.8M shares5 | 69.8M shares6 | 79.3M shares7 | 119.9M shares3 | 151.2M shares3 |
Cash flow is reported year to date, not quarter by quarter, so there are no separate quarterly figures.
| Fiscal year | 2019 | 2020 | 2021 | 2022 | 2023 | 2024 | 2025 |
|---|---|---|---|---|---|---|---|
| Cash from operations | $2.5M4 | -$37.1M5 | -$94.8M6 | -$136.3M7 | -$119.1M3 | -$300.9M3 | -$650.4M3 |
| Stock-based compensation | $17.1M6 | $27.1M7 | $38.2M3 | $51.4M3 | $97.8M3 | ||
| Capital spending | $235.0K4 | $1.1M5 | $3.7M6 | $2.8M7 | $4.2M3 | $7.1M3 | $13.3M3 |
| Free cash flow | $2.3M | -$38.2M | -$98.6M | -$139.1M | -$123.3M | -$307.9M | -$663.8M |
| Cash from investing | -$82.5M6 | -$190.0M7 | -$130.1M3 | -$854.2M3 | -$38.0M3 | ||
| Cash from financing | $176.3M6 | $346.2M7 | $93.9M3 | $1.2B3 | $851.0M3 |
Corporate structure
Avidity Biosciences, Inc. sits at the top of its group. 3 entities, 2 countries on record. Switch between the flow chart and the network, and select any entity to see its details.
Major holders of Avidity Biosciences, Inc.
- RA CAPITAL MANAGEMENT, L.P. Filed SCHEDULE 13G/A on Avidity Biosciences, Inc., latest May 15, 2026 (6 filings since Dec 30, 2022)
- Avoro Capital Advisors LLC Filed SCHEDULE 13G/A on Avidity Biosciences, Inc., latest Feb 17, 2026 (2 filings since Nov 14, 2024)
- Janus Henderson Group Ltd. Filed SCHEDULE 13G/A on Avidity Biosciences, Inc., latest Feb 17, 2026 (6 filings since Feb 14, 2025)
- PRICE T ROWE ASSOCIATES INC /MD/ Filed SCHEDULE 13G/A on Avidity Biosciences, Inc., latest Feb 17, 2026 (8 filings since Feb 14, 2022)
- Wellington Management Company Filed SCHEDULE 13G/A on Avidity Biosciences, Inc., latest Feb 10, 2026 (3 filings since Nov 8, 2024)
- FMR LLC Filed SCHEDULE 13G/A on Avidity Biosciences, Inc., latest Nov 7, 2025 (7 filings since Feb 9, 2022)
- VANGUARD GROUP INC Filed SCHEDULE 13G/A on Avidity Biosciences, Inc., latest Oct 30, 2025 (5 filings since Feb 9, 2023)
- RTW INVESTMENTS, LP Filed SCHEDULE 13D/A on Avidity Biosciences, Inc., latest Oct 29, 2025 (5 filings since Feb 2, 2023)
- BlackRock Filed SCHEDULE 13G/A on Avidity Biosciences, Inc., latest Oct 17, 2025
- Cowen Financial Products LLC Filed SC 13G/A on Avidity Biosciences, Inc., latest Nov 13, 2024 (4 filings since Aug 18, 2022)
- State Street Corporation Filed SC 13G/A on Avidity Biosciences, Inc., latest Jan 23, 2024 (2 filings since Feb 3, 2023)
- EcoR1 Capital, LLC Filed SC 13G/A on Avidity Biosciences, Inc., latest Feb 14, 2023 (2 filings since Feb 14, 2022)
- Avidity Biosciences, Inc. DE
- Atrium Therapeutics, Inc. Subsidiary · Delaware, United States
- Avidity Biosciences Ireland Limited Subsidiary · Ireland
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 Avidity Biosciences, 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
| Item | Latest | A year before | Change |
|---|---|---|---|
| Property and equipment, before depreciation | $35.1M3 Dec 31, 2025 | $20.7M3 Dec 31, 2024 | +69.9% |
| Accumulated depreciation | $11.8M3 Dec 31, 2025 | $8.0M3 Dec 31, 2024 | +47.3% |
| Property and equipment, net | $23.3M3 Dec 31, 2025 | $12.7M3 Dec 31, 2024 | +84.2% |
| Operating-lease assets (leased space) | $51.3M3 Dec 31, 2025 | $5.6M3 Dec 31, 2024 | +812.6% |
Investments
| Item | Latest | A year before | Change |
|---|---|---|---|
| Cash and cash equivalents | $382.5M3 Dec 31, 2025 | $219.9M3 Dec 31, 2024 | +74.0% |
Properties, in its own words
We currently lease approximately 54,597 square feet of office and laboratory space in San Diego, California, under a lease that expires in 2026, with the option to extend the term of the lease for an additional five years. In April 2024, we entered into a sublease agreement, or the Sublease, to rent 105,000 square feet for office and laboratory space for the Company’s corporate headquarters. The Sublease commenced in August 2025, with a term of 9 years, 9 months. In March 2025, we also exercised the option to rent an additional 80,000 square feet in an adjacent available building under an amended sublease agreement, or the Amended Sublease, with Turning Point Therapeutics, Inc. The term of the Amended Sublease is approximately 9 years, 1 month with payments expected to begin in April 2026. We believe that our current and expected future facilities are adequate to meet our needs and that suitable additional alternative spaces will be available in the future on commercially reasonable terms, if required.
Item 2 of the 10-K filed Feb 23, 2026.11
Leadership
Who has led Avidity Biosciences, Inc., and when. Pick a name to see everywhere that person has worked.
Chief executive
President
Chief financial officer
Chief operating officer
- Chief executive officerSarah Boyce Jun 11, 2020 to now12
- PresidentSarah Boyce Jun 11, 2020 to now12
- Chief financial officerMichael F. Maclean Jun 11, 2020 to now13
- Chief operating officerJoseph Baroldi Jun 11, 2020 to Feb 4, 202114
Board members and other officers (22)
- Chief Technical OfficerCharles Calderaro Iii. Jan 8, 2025 to now15
- Chief Commercial OfficerEric Mosbrooker Jan 8, 2025 to now16
- Chief Program OfficerKathleen P. Gallagher Jan 8, 2025 to now17
- Chief Medical OfficerSteven George Hughes Jan 8, 2025 to now18
- Chief Human Resources OfficerTeresa McCarthy May 20, 2021 to now19
- MP and CIORoderick Wong Aug 6, 2021 to Aug 6, 202120
- Chief Scientific OfficerArthur A. Levin Jun 11, 2020 to Feb 15, 202321
- Board memberSimona Skerjanec May 16, 2024 to now22
- Board memberArthur A. Levin Mar 17, 2023 to now23
- Board memberJean Jinsun Kim Jan 11, 2021 to now24
- Board memberTamar Thompson Jan 11, 2021 to now25
- Board memberEric Mosbrooker Aug 27, 2021 to Jun 20, 202326
- Board memberBihua Chen Jun 11, 2020 to Jun 18, 202027
- Board memberCarsten Boess Jun 11, 2020 to now28
- Board memberEdward M. Kaye Md. Jun 11, 2020 to now29
- Board memberMichael B. Martin Jun 11, 2020 to Jun 11, 202030
- Board memberNoreen Henig Jun 11, 2020 to now31
- Board memberRoderick Wong Jun 11, 2020 to Jun 17, 202132
- Board memberSarah Boyce Jun 11, 2020 to now12
- Board memberTodd C. Brady Jun 11, 2020 to Jun 11, 202033
- Board memberTroy Edward Wilson Jun 11, 2020 to now34
- General counselJohn B. Moriarty Aug 5, 2024 to now35
Named in SEC Form D filings
People the company listed in its private-offering notices.
- Executive OfficerW. Michael Flanagan named Mar 19, 202436
Subsidiaries and ownership
Subsidiaries (Exhibit 21)
The subsidiaries the company lists in Exhibit 21 of its annual report, as filed.
Timeline
- Feb 27, 20268-K Ended a material agreement; Completed an acquisition or sale; Listing notice or transfer37
- Feb 26, 20268-K Shareholder vote38
- Feb 23, 20268-K Other events39
- Feb 3, 20268-K Regulation FD disclosure; Other events40
- Dec 18, 20258-K Executive or director change; Other events41
- Nov 10, 20258-K Results of operations42
- Oct 27, 20258-K Entered a material agreement; Regulation FD disclosure43
- Oct 14, 20258-K Other events44
- Sep 12, 20258-K Other events45
- Sep 10, 20258-K Executive or director change; Regulation FD disclosure; Other events46
- Aug 7, 20258-K Entered a material agreement; Results of operations47
- Jul 23, 20258-K Other events48
- Jun 11, 2020New chief executive Sarah Boyce becomes CEO12
Calendar
When it reported results
- Nov 10, 2025Reported quarterly or annual results42
- Aug 7, 2025Reported quarterly or annual results47
- May 8, 2025Reported quarterly or annual results49
- Feb 27, 2025Reported quarterly or annual results50
- Nov 7, 2024Reported quarterly or annual results51
- Aug 9, 2024Reported quarterly or annual results52
- May 9, 2024Reported quarterly or annual results53
- Feb 28, 2024Reported quarterly or annual results54
Upcoming dates from Nasdaq's calendar; past results from the company's 8-K filings (Item 2.02). The full markets calendar
Filings
435 filings to the SEC since October 2021: 5 annual reports, 13 quarterly reports, 64 major events, 198 insider forms, 53 large holders, 1 private raises, 101 other. The latest 20:
- May 15, 2026SCHEDULE 13G/A EDGAR
- Mar 9, 202615-12G EDGAR
- Feb 27, 20264 EDGAR
- Feb 27, 20264 EDGAR
- Feb 27, 20264 EDGAR
- Feb 27, 20264 EDGAR
- Feb 27, 20264 EDGAR
- Feb 27, 20264 EDGAR
- Feb 27, 20264 EDGAR
- Feb 27, 20264 EDGAR
- Feb 27, 20264 EDGAR
- Feb 27, 20264 EDGAR
- Feb 27, 20264 EDGAR
- Feb 27, 20264 EDGAR
- Feb 27, 20264 EDGAR
- Feb 27, 20264 EDGAR
- Feb 27, 20264 EDGAR
- Feb 27, 20264 EDGAR
- Feb 27, 20264 EDGAR
- Feb 27, 2026POSASR EDGAR
The 435 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
122 open-market trades by officers, directors and large owners since October 2021: 0 buys and 122 sales. Grants, option exercises and gifts are left out.
| Date | Who | Trade | Shares | Price | Value | Filing |
|---|---|---|---|---|---|---|
| Jan 21, 2026 | Hughes Steven GeorgeChief Medical Officer | Sale | 4,895 | $72.45 | $354.6K | Form 4 |
| Jan 21, 2026 | Boyce SarahPresident and CEO | Sale | 14,387 | $72.45 | $1.0M | Form 4 |
| Jan 21, 2026 | Flanagan W. MichaelChief Scientific Officer | Sale | 6,534 | $72.45 | $473.4K | Form 4 |
| Jan 21, 2026 | MacLean Michael FChief Financial Officer | Sale | 4,542 | $72.45 | $329.1K | Form 4 |
| Jan 21, 2026 | LEVIN ARTHUR ADirector | Sale | 1,758 | $72.45 | $127.4K | Form 4 |
| Jan 21, 2026 | McCarthy TeresaChief Human Resources Officer | Sale | 2,929 | $72.45 | $212.2K | Form 4 |
| Jan 7, 2026 | Boyce SarahPresident and CEO | Sale | 8,576 | $72.23 | $619.4K | Form 4 |
| Jan 7, 2026 | Calderaro Charles IIIChief Technical Officer | Sale | 3,727 | $72.23 | $269.2K | Form 4 |
| Jan 7, 2026 | McCarthy TeresaChief Human Resources Officer | Sale | 1,965 | $72.23 | $141.9K | Form 4 |
| Jan 7, 2026 | Moriarty John BChief Legal Officer | Sale | 2,374 | $72.23 | $171.5K | Form 4 |
| Jan 7, 2026 | MacLean Michael FChief Financial Officer | Sale | 1,974 | $72.23 | $142.6K | Form 4 |
| Jan 7, 2026 | Hughes Steven GeorgeChief Medical Officer | Sale | 2,373 | $72.23 | $171.4K | Form 4 |
| Jan 7, 2026 | Flanagan W. MichaelChief Scientific Officer | Sale | 2,373 | $72.23 | $171.4K | Form 4 |
| Oct 22, 2025 | Hughes Steven GeorgeChief Medical Officer | Sale | 2,209 | $47.54 | $105.0K | Form 4 |
| Oct 15, 2025 | McCarthy TeresaChief Human Resources Officer | Sale | 20,000 | $50.15 | $1.0M | Form 4 |
| Oct 3, 2025 | Mosbrooker EricChief Commercial Officer | Sale | 6,562 | $45.38 | $297.8K | Form 4 |
| Sep 22, 2025 | Hughes Steven GeorgeChief Medical Officer | Sale | 2,208 | $40.58 | $89.6K | Form 4 |
| Sep 15, 2025 | McCarthy TeresaChief Human Resources Officer | Sale | 15,000 | $41.41 | $621.1K | Form 4 |
| Sep 15, 2025 | Hughes Steven GeorgeChief Medical Officer | Sale | 1,542 | $45.41 | $70.0K | Form 4 |
| Sep 11, 2025 | Flanagan W. MichaelChief Scientific Officer | Sale | 20,000 | $38.94 | $778.8K | Form 4 |
All 122 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.
- RA CAPITAL MANAGEMENT, L.P.SCHEDULE 13G/A, 6 filings Dec 30, 2022 to May 15, 202655
- Avoro Capital Advisors LLCSCHEDULE 13G/A, 2 filings Nov 14, 2024 to Feb 17, 202656
- Janus Henderson Group Ltd.SCHEDULE 13G/A, 6 filings Feb 14, 2025 to Feb 17, 202657
- PRICE T ROWE ASSOCIATES INC /MD/SCHEDULE 13G/A, 8 filings Feb 14, 2022 to Feb 17, 202658
- Wellington Management CompanySCHEDULE 13G/A, 3 filings Nov 8, 2024 to Feb 10, 202659
- FMR LLCSCHEDULE 13G/A, 7 filings Feb 9, 2022 to Nov 7, 202560
- VANGUARD GROUP INCSCHEDULE 13G/A, 5 filings Feb 9, 2023 to Oct 30, 202561
- RTW INVESTMENTS, LPSCHEDULE 13D/A, 5 filings Feb 2, 2023 to Oct 29, 202562
- BlackRockSCHEDULE 13G/A, 1 filing Oct 17, 202563
- Cowen Financial Products LLCSC 13G/A, 4 filings Aug 18, 2022 to Nov 13, 202464
- State Street CorporationSC 13G/A, 2 filings Feb 3, 2023 to Jan 23, 202465
- EcoR1 Capital, LLCSC 13G/A, 2 filings Feb 14, 2022 to Feb 14, 202366
- Cormorant Asset Management, LPSC 13G, 2 filings Feb 14, 2022 to Dec 27, 202267
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.
| Year | Executive | Age | Salary | Total pay |
|---|---|---|---|---|
| 2024 | Sarah Boyce | 53 | $659.9K | $14.2M68 |
| 2024 | John B. MoriartyJr, J.D. | 57 | $208.3K | $9.7M68 |
| 2024 | Michael F. MacLean | 59 | $490.8K | $4.9M68 |
| 2024 | W. Michael FlanaganPh.D. | $500.3K | $4.8M68 | |
| 2024 | Teresa McCarthy | 61 | $456.9K | $4.6M68 |
| 2023 | President and Chief Executive Officer | $634.5K | $11.1M68 | |
| 2023 | Chief Scientific Officer | $470.6K | $4.9M68 | |
| 2023 | Chief Financial Officer | $471.9K | $4.5M68 | |
| 2023 | Chief Human Resources Officer | $439.3K | $3.7M68 | |
| 2022 | President and Chief Executive Officer | $601.4K | $5.4M68 | |
| 2022 | Chief Human Resources Officer | $420.3K | $2.3M68 | |
| 2022 | Chief Scientific Officer | $422.0K | $2.1M68 | |
| 2022 | Chief Financial Officer | $451.5K | $2.0M68 |
Money raised
Private offerings the company reported to the SEC on Form D.
- Mar 4, 2024Raised $400.2 million in a private offering of equity Form D: 45 investors so far Form D
SEC filer profile
How Avidity Biosciences, Inc. is registered with the Securities and Exchange Commission, from its EDGAR record and the cover page of its latest annual report.
- Name on EDGARAvidity Biosciences, Inc.1
- CIK0001599901
- Employer ID (EIN)46-133696011
- Filer statusLarge accelerated filer not an emerging growth company; not a smaller reporting company; not a shell company11
- Incorporated inDelaware11
- Fiscal year endsDecember 311
- Industry code (SIC)2834 Pharmaceutical Preparations1
- SEC reviewing officeOffice of Life Sciences, Division of Corporation Finance1
- Business address3020 Callan Road, San Diego, CA 921211
- Phone858-401-79001
- Shares outstanding155,149,646 shares as of Feb 13, 202611
- Public float$3.4B as of Jun 30, 202511
- AuditorDeloitte & Touche LLP, San Diego, California PCAOB ID 34; it also attests to the company’s internal control over financial reporting11
- On EDGAR sinceFeb 18, 20141
- Filings558 in all: 4 (219), 8-K (83), 144 (43), SC 13G/A (30), 3 (26), DEFA14A (20), SCHEDULE 13G/A (18), 10-Q (17)1
All 41 form types
SC 13G (16), 424B5 (12), DFAN14A (7), 10-K (6), S-8 (6), S-8 POS (6), DEF 14A (5), D (4), S-3ASR (4), SC 13D/A (4), ARS (3), CORRESP (3), POSASR (3), S-1/A (3), PRER14A (2), 15-12G (1), 25-NSE (1), 424B4 (1), 8-A12B (1), CERT (1), D/A (1), DEFM14A (1), DRS (1), DRS/A (1), DRSLTR (1), EFFECT (1), PREM14A (1), S-1 (1), S-1MEF (1), SC 13D (1), SCHEDULE 13D/A (1), SCHEDULE 13G (1), UPLOAD (1)
- Latest filingsAnnual report (10-K) Feb 23, 2026
Quarterly report (10-Q) Nov 10, 2025
Proxy statement (DEF 14A) Apr 29, 2025
Latest 8-K (8-K) Feb 27, 2026
Securities registered for trading
| Class | Symbol | Exchange |
|---|---|---|
| Common Stock, par value $0.0001 per share | RNA | The Nasdaq Global Market |
From the cover page of the 10-K filed Feb 23, 2026 (Section 12(b) of the Exchange Act).11
Risk factors and legal proceedings
The risks Avidity Biosciences, Inc. names in Item 1A of its 10-K filed Feb 23, 2026: 75 risks under these headings, as written.11
- Risks Related to Our Pending Acquisition by Novartis
- We may not complete the pending transaction with Novartis within the timeframe we anticipate, or at all, which could have an adverse effect on our business, financial results, and/or operations.
- The pendency of the Merger and Distribution could adversely affect our business, financial results and/or operations.
- While the Merger Agreement is in effect, we are subject to restrictions on our business activities.
- In certain instances, the Merger Agreement requires us to pay a termination fee to Novartis, which could require us to use available cash that would have otherwise been available for general corporate purposes.
- We have incurred, and will continue to incur, direct and indirect costs as a result of the pending transaction with Novartis.
- Litigation has arisen in connection with the Merger or the Distribution, which could be costly and divert management’s attention and otherwise materially harm our business.
- The Merger Agreement contains provisions that could discourage a potential competing acquirer of our company or could result in any competing proposal being at a lower price than it might otherwise be.
- The value of SpinCo Common Stock may not be as anticipated.
- If the Merger is consummated, our stockholders will not be able to participate in any further upside to the portion of our business acquired by Novartis.
- Risks Related to Our Limited Operating History, Financial Position and Capital Requirements
- We have a limited operating history, have incurred significant operating losses since our inception and expect to incur significant losses for the foreseeable future. We may never generate any product revenue or become profitable or, if we achieve profitability, we may not be able to sustain it.
The other 64 risks
- We will require substantial additional financing to achieve our goals, and a failure to obtain this necessary capital when needed on acceptable terms, or at all, could force us to delay, limit, reduce or terminate our development programs, commercialization efforts or other operations.
- 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, Development and Regulatory Approval of Our Product Candidates
- We have three product candidates in clinical development. All of our other development programs are in the preclinical or discovery stage. If we are unable to successfully develop, obtain regulatory approval for and ultimately commercialize product candidates, or experience significant delays in doing so, our business will be materially harmed.
- Interim, topline and preliminary data from our clinical trials and preclinical studies that we announce or publish from time to time may change as more patient data become available and are subject to audit and verification procedures that could result in material changes in the final data.
- Our approach to the discovery and development of product candidates based on our AOC platform is unproven, and we do not know whether we will be able to develop any products of commercial value, or if competing technological approaches will limit the commercial value of our product candidates or render our AOC platform obsolete.
- Preclinical and clinical development involves a lengthy and expensive process with an uncertain outcome, and the results of preclinical studies and early clinical trials are not necessarily predictive of future results. Our product candidates may not have favorable results in clinical trials or receive regulatory approval on a timely basis, if at all.
- Any difficulties or delays in the commencement or completion, or the termination or suspension, of our current or planned clinical trials could result in increased costs to us, or delay or limit our ability to generate revenue and adversely affect our commercial prospects.
- We may find it difficult to enroll patients in our clinical trials. If we encounter difficulties enrolling subjects in our clinical trials, our clinical development activities could be delayed or otherwise adversely affected.
- Use of our product candidates could be associated with side effects, adverse events or other properties or safety risks, which could delay or preclude approval, cause us to suspend or discontinue clinical trials, abandon a product candidate, limit the commercial profile of an approved label or result in other significant negative consequences that could severely harm our business, prospects, operating results and financial condition.
- As an organization, we have never completed any pivotal clinical trials or submitted a BLA for regulatory approval and may be unable to do so for any of our product candidates.
- Our product candidates are subject to extensive regulation and compliance, which is costly and time consuming, and such regulation may cause unanticipated delays or prevent the receipt of the approvals required to commercialize our product candidates.
- We may expend our limited resources to pursue a particular product candidate 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 may not be able to obtain or maintain Orphan drug designations for any of our product candidates, and we may be unable to maintain the benefits associated with Orphan drug designation, including the potential for market exclusivity.
- Receipt of Breakthrough Therapy designation or Fast Track designation by the FDA for one or more of our product candidates may not lead to a faster development or regulatory review or approval process and it does not increase the likelihood that our product candidates will receive marketing approval.
- We have obtained Rare Pediatric Disease designation for del-zota for the treatment of DMD44. However, there is no guarantee that FDA approval of will result in issuance of a priority review voucher.
- We are conducting certain of our clinical trials for our product candidates outside of the United States. However, the FDA and other foreign equivalents may not accept data from such trials, in which case our development plans will be delayed, which could materially harm our business.
- Disruptions at the FDA and other government agencies caused by funding shortages, staffing limitations or policy changes 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.
- Risks Related to Our Reliance on Third Parties
- We rely on third parties to conduct our preclinical studies and clinical trials. If these third parties do not successfully carry out their contractual duties, comply with applicable regulatory requirements or meet expected deadlines, our development programs and our ability to seek or obtain regulatory approval for or commercialize our product candidates may be delayed.
- We rely on third parties for the manufacture of our product candidates for preclinical and clinical development. 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.
- Our reliance on third parties requires us to share our trade secrets, which increases the possibility that a competitor will discover them or that our trade secrets will be misappropriated or disclosed.
- We are dependent on Lilly and BMS, as collaboration partners, for the development of certain targets. Under certain circumstances, Lilly or BMS may each unilaterally terminate its respective agreement with us for convenience, which could materially and adversely affect our business.
- We may seek to enter into additional collaborations, licenses and other similar arrangements and may not be successful in doing so, and even if we are, we may relinquish valuable rights and may not realize the benefits of such relationships.
- Risks Related to Commercialization of Our Product Candidates
- Even if we receive regulatory approval for any product candidate, 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 labeling and other restrictions on marketing or withdrawal from the market, and we may be subject to penalties if we fail to comply with regulatory requirements or if we experience unanticipated problems with our product candidates, when and if any of them are approved.
- Our product candidates for which we intend to seek approval as biologic products may face competition sooner than anticipated.
- The commercial success of our product candidates will depend upon the degree of market acceptance of such product candidates by physicians, patients, healthcare payors and others in the medical community.
- The FDA and other regulatory agencies actively enforce the laws and regulations prohibiting the promotion of off-label uses. If we are found or alleged to have improperly promoted off-label uses, we may become subject to significant liability.
- The successful commercialization of our product candidates, if approved, will depend in part on the extent to which governmental authorities and health insurers establish coverage, adequate reimbursement levels and favorable pricing policies. Failure to obtain or maintain coverage and adequate reimbursement for our products could limit our ability to market those products and decrease our ability to generate revenue.
- We face significant competition, and if our competitors develop technologies or product candidates more rapidly than we do or their technologies are more effective, our business and our ability to develop and successfully commercialize products may be adversely affected.
- If the market opportunities for our products are smaller than we believe they are, our revenue may be adversely affected, and our business may suffer.
- We currently have no marketing and sales organization and have no experience as a company in commercializing products, and we may have to invest significant resources to develop these capabilities. If we are unable to establish marketing and sales capabilities or enter into agreements with third parties to market and sell our products, we may not be able to generate product revenue.
- Our future growth may depend, in part, on our ability to operate in foreign markets, where we would be subject to additional regulatory burdens and other risks and uncertainties.
- Risks Related to Our Business Operations and Industry
- Our operating results may fluctuate significantly, which makes our future operating results difficult to predict and could cause our operating results to fall below expectations or any guidance we may provide.
- We are dependent on the services of our management and other clinical and scientific personnel, and if we are not able to retain these individuals or recruit additional management or clinical and scientific personnel, our business will suffer.
- We may encounter difficulties in managing our growth and expanding our operations successfully.
- We are subject to various federal, state and foreign healthcare laws and regulations, which could increase compliance costs, and our failure to comply with these laws and regulations could harm our results of operations and financial condition.
- Recently enacted legislation, future legislation and healthcare reform measures may increase the difficulty and cost for us to commercialize our product candidates and may affect the prices we may set.
- We intend to participate in the Medicaid Drug Rebate Program and other governmental pricing programs. If we fail to comply with our reporting and payment obligations under any programs we participate in, 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.
- If product liability lawsuits are brought against us, we may incur substantial liabilities and may be required to limit commercialization of our products.
- Our insurance policies are expensive and only protect us from some business risks, which will leave us exposed to significant uninsured liabilities.
- We and any of our current and potential future collaborators will be required to report to regulatory authorities if any of our approved products cause or contribute to adverse medical events, and any failure to do so would result in sanctions that would materially harm our business.
- Our business, including ongoing and planned clinical trials and preclinical studies, and financial condition, is subject to risks arising from pandemic and epidemic diseases.
- Our business could be affected by litigation, government investigations and enforcement actions.
- Our employees and independent contractors, including principal investigators, CROs, consultants and vendors, may engage in misconduct or other improper activities, including noncompliance with regulatory standards and requirements.
- We may engage in strategic transactions that could impact our liquidity, increase our expenses and present significant distractions to our management.
- Our ability to use net operating loss carryforwards and other tax attributes may be limited.
- Inflation could adversely affect our business and results of operations.
- Risks Related to Our Intellectual Property
- If we are unable to obtain and maintain patent protection for our therapeutic programs and other proprietary technologies we develop, or if the scope of the patent protection obtained is not sufficiently broad, our competitors could develop and commercialize products and technology similar or identical to ours, and our ability to successfully commercialize our therapeutic programs and other proprietary technologies we may develop may be adversely affected.
- We may not be able to protect our intellectual property and proprietary rights throughout the world.
- Obtaining and maintaining our patent protection depends on compliance with various procedural, document submission, fee payment, and other requirements imposed by government patent agencies, and our patent protection could be reduced or eliminated for non-compliance with these requirements.
- Changes in U.S. patent law could diminish the value of patents in general, thereby impairing our ability to protect our products.
- Issued patents covering our therapeutic programs and other proprietary technologies we may develop could be found invalid or unenforceable if challenged in court or before administrative bodies in the United States or abroad.
- If we do not obtain patent term extension for our product candidate, our business may be materially harmed.
- We may be subject to claims challenging the inventorship of our patents and other intellectual property.
- If we are unable to protect the confidentiality of our trade secrets, our business and competitive position would be harmed.
- We may not be successful in obtaining necessary rights to any product candidate we may develop through acquisitions and in-licenses.
- We may be subject to claims that our employees, consultants or advisors have wrongfully used or disclosed alleged trade secrets of their current or former employers or claims asserting ownership of what we regard as our own intellectual property.
- Third-party claims of intellectual property infringement, misappropriation or other violations against us or our collaborators may prevent or delay the development and commercialization of our therapeutic programs and other proprietary technologies we may develop.
- We may become involved in lawsuits to protect or enforce our patents and other intellectual property rights, which could be expensive, time consuming and unsuccessful.
- If our trademarks and trade names are not adequately protected, then we may not be able to build name recognition in our markets of interest and our business may be adversely affected.
- Intellectual property rights do not necessarily address all potential threats.
- Intellectual property discovered through government funded programs may be subject to federal regulations such as “march-in” rights, certain reporting requirements and a preference for United States-based companies. Compliance with such regulations may limit our exclusive rights and limit our ability to contract with non-United States manufacturers.
- We partially depend on intellectual property licensed from third parties, and our licensors may not always act in our best interest. If we fail to comply with our obligations under our intellectual property licenses, if the licenses are terminated or if disputes regarding these licenses arise, we could lose significant rights that are important to our business.
- We may not be successful in obtaining or maintaining necessary rights to product components and processes for our development pipeline through acquisitions and in-licenses.
Legal proceedings
We are not currently subject to any material legal proceedings. From time to time, we may be involved in legal proceedings or subject to claims incident to the ordinary course of business. Regardless of the outcome, such proceedings or claims can have an adverse impact on us because of defense and settlement costs, diversion of resources and other factors, and there can be no assurances that favorable outcomes will be obtained.
The opening of Item 3, Legal Proceedings.11
Sources
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- 12SEC EDGARSarah Boyce, Chief executive officer
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- 20SEC EDGARRoderick Wong, MP and CIO
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- 22SEC EDGARSimona Skerjanec, Board member
- 23SEC EDGARArthur A. Levin, Board member
- 24SEC EDGARJean Jinsun Kim, Board member
- 25SEC EDGARTamar Thompson, Board member
- 26SEC EDGAREric Mosbrooker, Board member
- 27SEC EDGARBihua Chen, Board member
- 28SEC EDGARCarsten Boess, Board member
- 29SEC EDGAREdward M. Kaye Md., Board member
- 30SEC EDGARMichael B. Martin, Board member
- 31SEC EDGARNoreen Henig, Board member
- 32SEC EDGARRoderick Wong, Board member
- 33SEC EDGARTodd C. Brady, Board member
- 34SEC EDGARTroy Edward Wilson, Board member
- 35SEC EDGARJohn B. Moriarty, General counsel
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- 38SEC EDGARShareholder vote
- 39SEC EDGAROther events
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- 42SEC EDGARResults of operations
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- 44SEC EDGAROther events
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- 48SEC EDGAROther events
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