CAMP on Nasdaq
Camp4 Therapeutics Corp
American pharmaceutical preparations company based in Cambridge1
In its 2026 annual report the company says: “We are a clinical-stage biopharmaceutical company pioneering the discovery and development of a new class of RNA-targeting therapeutics with the goal of upregulating gene expression and restoring healthy protein levels to treat a broad range of genetic diseases.”3 Its shares trade on Nasdaq under the symbol CAMP.1
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Last updated Oct 20, 2026.
Overview
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- TickerCAMP on Nasdaq1
- IndustryPharmaceutical Preparations1
- HeadquartersWatertown, Massachusetts1
- Incorporated inDE2
- Business address100 Talcott Avenue, Suite 201, Watertown, Ma 024722
- Filer category, Emerging growth company2
- Legal formCorporation
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We are a clinical-stage biopharmaceutical company pioneering the discovery and development of a new class of RNA-targeting therapeutics with the goal of upregulating gene expression and restoring healthy protein levels to treat a broad range of genetic diseases. Our lead product candidate, CMP-002, has the potential to be the first disease-modifying therapy for the treatment of synaptic Ras GTPase activating protein 1 (“SYNGAP1”)-related disorder, or SYNGAP1, a severe developmental and epileptic encephalopathy (“DEE”) characterized by seizures, developmental delays, and cognitive impairments. SYNGAP1 is caused by haploinsufficiency of the SYNGAP1 gene, where mutation of one gene copy results in a reduction in functional SYNGAP protein levels of up to 50%. While we believe that it remains underdiagnosed, we estimate that there are approximately 21,000 individuals living with SYNGAP1 in th…3 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
Where the money goes
Where each dollar of revenue went, fiscal 2025
- Research and development100¢$38.2M
Out of $3.5M in revenue.4 The company lost money this year.
Financial statements
| Fiscal year | 2022 | 2023 | 2024 | 2025 |
|---|---|---|---|---|
| Revenue | $350.0K5 | $652.0K4 | $3.5M4 | |
| Research and development | $40.6M5 | $38.8M4 | $38.2M4 | |
| Operating expenses | $52.2M5 | $53.7M4 | $56.1M4 | |
| Operating income | -$51.9M5 | -$53.1M4 | -$52.6M4 | |
| Income tax | $05 | $04 | $04 | |
| Net income | -$49.3M5 | -$51.8M4 | -$80.4M4 | |
| Earnings per share | -$124.805 | -$11.044 | -$2.654 | |
| Earnings per share (diluted) | -$124.805 | -$11.044 | -$2.654 | |
| Shares (weighted, basic) | 395.0K shares5 | 4.7M shares4 | 30.4M shares4 | |
| Shares (weighted, diluted) | 395.0K shares5 | 4.7M shares4 | 30.4M shares4 |
| Fiscal year | 2022 | 2023 | 2024 | 2025 |
|---|---|---|---|---|
| Cash | $84.5M5 | $38.4M5 | $64.0M4 | $109.5M4 |
| Current assets | $40.0M5 | $66.4M4 | $114.1M4 | |
| Property, plant and equipment | $4.8M5 | $3.9M5 | ||
| Total assets | $54.9M5 | $78.3M4 | $117.8M4 | |
| Accounts payable | $1.0M5 | $1.2M4 | $182.0K4 | |
| Current liabilities | $7.8M5 | $9.6M4 | $15.5M4 | |
| Total liabilities | $16.5M5 | $15.2M4 | $70.1M4 | |
| Retained earnings | -$160.0M5 | -$211.8M4 | -$292.2M4 | |
| Shareholders’ equity | -$77.5M5 | -$123.7M4 | $63.1M4 | $47.7M4 |
| Shares outstanding | 460.7K shares5 | 20.1M shares4 | 51.9M shares4 |
Cash flow is reported year to date, not quarter by quarter, so there are no separate quarterly figures.
| Fiscal year | 2022 | 2023 | 2024 | 2025 |
|---|---|---|---|---|
| Cash from operations | -$44.2M5 | -$45.6M4 | -$29.6M4 | |
| Depreciation and amortization | $1.7M5 | $1.7M4 | $1.6M4 | |
| Stock-based compensation | $2.9M5 | $4.0M4 | $4.0M4 | |
| Capital spending | $678.0K5 | $441.0K4 | $279.0K4 | |
| Free cash flow | -$44.8M | -$46.0M | -$29.8M | |
| Cash from investing | -$678.0K5 | -$441.0K4 | -$243.0K4 | |
| Cash from financing | $301.0K5 | $71.7M4 | $75.9M4 | |
| Dividend per share | $0.005 | $0.004 |
Corporate structure
Camp4 Therapeutics Corp sits at the top of its group. 1 entity, 1 country on record. Switch between the flow chart and the network, and select any entity to see its details.
Major holders of Camp4 Therapeutics Corp
- 5AM Ventures VI, L.P. Filed SCHEDULE 13D/A on Camp4 Therapeutics Corp, latest Aug 31, 2026 (4 filings since Oct 22, 2024)
- Polaris Management Co. VII, L.L.C. Filed SCHEDULE 13D/A on Camp4 Therapeutics Corp, latest Aug 19, 2026 (3 filings since Oct 22, 2024)
- BALYASNY ASSET MANAGEMENT L.P. Filed SCHEDULE 13G/A on Camp4 Therapeutics Corp, latest Aug 14, 2026 (2 filings since May 15, 2026)
- Trails Edge Capital Partners, LP Filed SCHEDULE 13G on Camp4 Therapeutics Corp, latest Aug 10, 2026
- Enavate Sciences GP, LLC Filed SCHEDULE 13D/A on Camp4 Therapeutics Corp, latest Mar 11, 2026 (4 filings since Oct 22, 2024)
- Vivo Opportunity Fund Holdings, L.P. Filed SCHEDULE 13G/A on Camp4 Therapeutics Corp, latest Feb 13, 2026 (2 filings since Sep 17, 2025)
- FMR LLC Filed SCHEDULE 13G/A on Camp4 Therapeutics Corp, latest Feb 5, 2026 (3 filings since Oct 7, 2025)
- Janus Henderson Group Ltd. Filed SCHEDULE 13G on Camp4 Therapeutics Corp, latest Jan 8, 2026
- Coastlands Capital LP Filed SCHEDULE 13G/A on Camp4 Therapeutics Corp, latest Dec 11, 2025 (2 filings since Sep 16, 2025)
- AH Bio Fund I, L.P. Filed SCHEDULE 13G/A on Camp4 Therapeutics Corp, latest Nov 14, 2025 (2 filings since Oct 22, 2024)
- Northpond Ventures II, LP Filed SCHEDULE 13G/A on Camp4 Therapeutics Corp, latest Nov 14, 2025 (2 filings since Oct 22, 2024)
- HARBOURVEST PARTNERS LLC Filed SCHEDULE 13G/A on Camp4 Therapeutics Corp, latest Oct 1, 2025 (2 filings since May 14, 2025)
- Camp4 Therapeutics Corp Watertown, Massachusetts
Built from major holders from Schedules 13D and 13G. Each entity in the chart links to its source.
What it owns
What Camp4 Therapeutics Corp 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 | $10.1M5 Dec 31, 2024 | $9.3M5 Dec 31, 2023 | +8.5% |
| Accumulated depreciation | $6.2M5 Dec 31, 2024 | $4.5M5 Dec 31, 2023 | +38.3% |
| Property and equipment, net | $3.9M5 Dec 31, 2024 | $4.8M5 Dec 31, 2023 | −19.6% |
| Finance-lease assets | $427.0K5 Dec 31, 2024 | $748.0K5 Dec 31, 2023 | −42.9% |
| Operating-lease assets (leased space) | $397.0K4 Dec 31, 2025 | $6.0M4 Dec 31, 2024 | −93.4% |
Investments
| Item | Latest | A year before | Change |
|---|---|---|---|
| Cash and cash equivalents | $109.5M4 Dec 31, 2025 | $64.0M4 Dec 31, 2024 | +71.0% |
Properties, in its own words
We currently lease approximately 30,000 square feet of office and laboratory space in Cambridge, Massachusetts, and approximately 5,300 square feet of office and laboratory space in Boulder, Colorado. In December 2025, we amended our Cambridge lease and expect to vacate the Cambridge facility during 2026. We have entered into a new lease for approximately 44,000 square feet of office and laboratory space in Watertown, Massachusetts, which is expected to commence in 2026. We believe our facilities will be adequate for the foreseeable future and that suitable additional or substitute space will be available as and when needed.
Item 2 of the 10-K filed Mar 5, 2026.3
Leadership
Who has led Camp4 Therapeutics Corp, and when. Pick a name to see everywhere that person has worked.
- Chief executive officerJosh Mandel-Brehm Oct 10, 2024 to now8
- Chief financial officerKelly Gold Oct 10, 2024 to now9
Board members and other officers (14)
- Chief Scientific OfficerDaniel Tardiff Oct 10, 2025 to now10
- Chief Scientific OfficerDavid Bumcrot Oct 10, 2024 to now11
- Chief Medical OfficerYuri Maricich Oct 10, 2024 to now12
- Board memberMichael F. Maclean Apr 3, 2026 to now13
- Board memberDouglas E. Williams Mar 19, 2025 to now14
- Board memberMurray Stewart Mar 19, 2025 to now15
- Board memberAmir Nashat Oct 10, 2024 to now16
- Board memberAndrew J. Schwab Oct 10, 2024 to now17
- Board memberIngo Chakravarty Oct 10, 2024 to Oct 10, 202418
- Board memberJames P. Boylan Oct 10, 2024 to now19
- Board memberJosh Mandel-Brehm Oct 10, 2024 to now8
- Board memberPaula Ragan Oct 10, 2024 to now20
- Board memberRavi I. Thadhani Oct 10, 2024 to now21
- Board memberSteven H. Holtzman Oct 10, 2024 to now22
Named in SEC Form D filings
People the company listed in its private-offering notices.
- Executive OfficerALFICA SEHGAL named Oct 29, 2021 to Jun 21, 202223
- Executive OfficerANN BARBIER named Jun 21, 202223
- Executive OfficerCALEB MOORE named Oct 29, 2021 to Jun 21, 202223
- Executive OfficerMICHELLE GATES named Oct 29, 2021 to Jun 21, 202223
- DirectorJORGE CONDE named Oct 29, 2021 to Jun 21, 202223
- DirectorMICHAEL HIGGINS named Oct 29, 2021 to Jun 21, 202223
- DirectorRICHARD YOUNG named Oct 29, 2021 to Jun 21, 202223
- DirectorSHAAN I. GANDHI named Oct 29, 2021 to Jun 21, 202223
Timeline
- Sep 28, 20268-K Regulation FD disclosure; Other events24
- Aug 13, 20268-K Results of operations25
- Aug 4, 20268-K Entered a material agreement; Unregistered sale of shares; Regulation FD disclosure26
- Jul 27, 20268-K Regulation FD disclosure; Other events27
- Jun 12, 20268-K Executive or director change; Shareholder vote28
- May 14, 20268-K Regulation FD disclosure29
- May 7, 20268-K Results of operations30
- Mar 24, 20268-K Executive or director change31
- Mar 5, 20268-K Completed an acquisition or sale; Regulation FD disclosure32
- Jan 9, 20268-K Regulation FD disclosure33
- Dec 23, 20258-K Entered a material agreement; Ended a material agreement34
- Dec 18, 20258-K Entered a material agreement; Regulation FD disclosure35
- Oct 10, 2024New chief executive Josh Mandel-Brehm becomes CEO8
Calendar
Coming up
- Nov 5, 2026Reports results for the quarter ending Sep/2026 A year earlier it reported ($0.55) a share, on Nov 6, 2025.36
When it reported results
- Aug 13, 2026Reported quarterly or annual results25
- May 7, 2026Reported quarterly or annual results30
- Nov 6, 2025Reported quarterly or annual results37
- Aug 14, 2025Reported quarterly or annual results38
- May 13, 2025Reported quarterly or annual results39
- Mar 27, 2025Reported quarterly or annual results40
- Nov 21, 2024Reported quarterly or annual results41
Upcoming dates from Nasdaq's calendar; past results from the company's 8-K filings (Item 2.02). The full markets calendar
Filings
187 filings to the SEC since October 2021: 2 annual reports, 6 quarterly reports, 25 major events, 84 insider forms, 28 large holders, 2 private raises, 40 other. The latest 20:
- Sep 28, 20268-K EDGAR
- Sep 2, 20264 EDGAR
- Aug 31, 2026SCHEDULE 13D/A EDGAR
- Aug 31, 20264 EDGAR
- Aug 26, 20264 EDGAR
- Aug 21, 20264 EDGAR
- Aug 19, 2026144 EDGAR
- Aug 19, 20264 EDGAR
- Aug 19, 2026SCHEDULE 13D/A EDGAR
- Aug 14, 2026SCHEDULE 13G/A EDGAR
- Aug 13, 202610-Q EDGAR
- Aug 13, 20268-K EDGAR
- Aug 12, 2026EFFECT EDGAR
- Aug 12, 2026424B3 EDGAR
- Aug 10, 2026SCHEDULE 13G EDGAR
- Aug 6, 2026S-3 EDGAR
- Aug 5, 2026SCHEDULE 13D/A EDGAR
- Aug 5, 20264 EDGAR
- Aug 5, 20264 EDGAR
- Aug 5, 20264 EDGAR
The 187 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
26 open-market trades by officers, directors and large owners since October 2021: 20 buys and 6 sales. Grants, option exercises and gifts are left out.
| Date | Who | Trade | Shares | Price | Value | Filing |
|---|---|---|---|---|---|---|
| Aug 31, 2026 | Gold KellyChief Financial Officer | Buy | 10,000 | $3.96 | $39.6K | Form 4 |
| Aug 27, 2026 | Schwab Andrew J.Director | Sale | 400,000 | $4.04 | $1.6M | Form 4 |
| Aug 24, 2026 | Schwab Andrew J.Director | Sale | 10 | $4.50 | $45 | Form 4 |
| Aug 21, 2026 | Schwab Andrew J.Director | Sale | 32,018 | $4.50 | $144.1K | Form 4 |
| Aug 20, 2026 | Schwab Andrew J.Director | Sale | 114,700 | $4.54 | $520.7K | Form 4 |
| Aug 19, 2026 | Tardiff DanielChief Scientific Officer | Sale | 1,738 | $4.76 | $8.3K | Form 4 |
| Aug 19, 2026 | Schwab Andrew J.Director | Sale | 45,147 | $4.76 | $214.9K | Form 4 |
| Aug 3, 2026 | Young Richard ADirector | Buy | 16,378 | $1.65 | $27.0K | Form 4 |
| Aug 3, 2026 | Maricich YuriChief Medical Officer | Buy | 6,551 | $1.65 | $10.8K | Form 4 |
| Aug 3, 2026 | Gold KellyChief Financial Officer | Buy | 6,551 | $1.65 | $10.8K | Form 4 |
| Aug 3, 2026 | Mandel-Brehm JoshChief Executive Officer | Buy | 6,551 | $1.65 | $10.8K | Form 4 |
| Sep 11, 2025 | Gold KellyChief Financial Officer | Buy | 6,060 | $1.65 | $10.0K | Form 4 |
| Sep 11, 2025 | Mandel-Brehm JoshChief Executive Officer | Buy | 6,060 | $1.65 | $10.0K | Form 4 |
| Sep 11, 2025 | Maricich YuriChief Medical Officer | Buy | 6,060 | $1.65 | $10.0K | Form 4 |
| Sep 11, 2025 | Young Richard ADirector | Buy | 15,151 | $1.65 | $25.0K | Form 4 |
| Sep 11, 2025 | 5AM Ventures VII, L.P.10% owner | Buy | 2,941,176 | $1.53 | $4.5M | Form 4 |
| Sep 11, 2025 | Polaris Management Co. VII, L.L.C.10% owner | Buy | 1,307,189 | $1.53 | $2.0M | Form 4 |
| Sep 11, 2025 | Nashat AmirDirector | Buy | 1,307,189 | $1.53 | $2.0M | Form 4 |
| Oct 15, 2024 | Northpond Ventures II, LP10% owner | Buy | 763,636 | $11.00 | $8.4M | Form 4 |
| Oct 15, 2024 | AH Equity Partners Bio I, L.L.C.10% owner | Buy | 727,272 | $11.00 | $8.0M | Form 4 |
All 26 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.
- 5AM Ventures VI, L.P.SCHEDULE 13D/A, 4 filings Oct 22, 2024 to Aug 31, 202642
- Polaris Management Co. VII, L.L.C.SCHEDULE 13D/A, 3 filings Oct 22, 2024 to Aug 19, 202643
- BALYASNY ASSET MANAGEMENT L.P.SCHEDULE 13G/A, 2 filings May 15, 2026 to Aug 14, 202644
- Trails Edge Capital Partners, LPSCHEDULE 13G, 1 filing Aug 10, 202645
- Enavate Sciences GP, LLCSCHEDULE 13D/A, 4 filings Oct 22, 2024 to Mar 11, 202646
- Vivo Opportunity Fund Holdings, L.P.SCHEDULE 13G/A, 2 filings Sep 17, 2025 to Feb 13, 202647
- FMR LLCSCHEDULE 13G/A, 3 filings Oct 7, 2025 to Feb 5, 202648
- Janus Henderson Group Ltd.SCHEDULE 13G, 1 filing Jan 8, 202649
- Coastlands Capital LPSCHEDULE 13G/A, 2 filings Sep 16, 2025 to Dec 11, 202550
- AH Bio Fund I, L.P.SCHEDULE 13G/A, 2 filings Oct 22, 2024 to Nov 14, 202551
- Northpond Ventures II, LPSCHEDULE 13G/A, 2 filings Oct 22, 2024 to Nov 14, 202552
- HARBOURVEST PARTNERS LLCSCHEDULE 13G/A, 2 filings May 14, 2025 to Oct 1, 202553
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 |
|---|---|---|---|---|
| 2025 | Josh Mandel-Brehm | 43 | $600.0K | $3.3M54 |
| 2025 | Kelly Gold | 48 | $465.0K | $1.8M54 |
| 2025 | Yuri MaricichMD | 45 | $500.0K | $1.8M54 |
| 2024 | Chief Medical Officer | $180.8K | $2.5M54 | |
| 2024 | Chief Executive Officer | $557.2K | $2.3M54 | |
| 2024 | Chief Financial Officer | $433.3K | $1.3M54 |
Money raised
Private offerings the company reported to the SEC on Form D.
SEC filer profile
How Camp4 Therapeutics Corp is registered with the Securities and Exchange Commission, from its EDGAR record and the cover page of its latest annual report.
- Name on EDGARCamp4 Therapeutics Corp1
- CIK0001736730
- Employer ID (EIN)81-11524763
- Filer statusNon-accelerated filer an emerging growth company; a smaller reporting company; not a shell company3
- Incorporated inDelaware3
- Fiscal year endsDecember 311
- Industry code (SIC)2834 Pharmaceutical Preparations1
- SEC reviewing officeOffice of Life Sciences, Division of Corporation Finance1
- Business address100 Talcott Avenue, Suite 201, Watertown, MA 024721
- Phone617-651-88671
- Shares outstanding62,753,200 as of Aug 12, 20267
- Public float$14.6M as of Jun 30, 20253
- AuditorErnst & Young LLP, Boston, Massachusetts PCAOB ID 42; no auditor attestation of internal control over financial reporting3
- On EDGAR sinceSep 25, 20181
- Filings189 in all: 4 (58), 8-K (25), 3 (23), SCHEDULE 13D/A (8), SCHEDULE 13G/A (8), SCHEDULE 13G (7), 10-Q (6), CORRESP (3)1
All 35 form types
D (3), DRS/A (3), S-3 (3), S-8 (3), SC 13D (3), UPLOAD (3), 10-K (2), 3/A (2), 424B3 (2), 424B5 (2), ARS (2), DEF 14A (2), DEFA14A (2), DRSLTR (2), EFFECT (2), S-1/A (2), SC 13G (2), SEC STAFF LETTER (2), 144 (1), 4/A (1), 424B4 (1), 8-A12B (1), CERT (1), D/A (1), DRS (1), FWP (1), S-1 (1)
- Latest filingsAnnual report (10-K) Mar 5, 2026
Quarterly report (10-Q) Aug 13, 2026
Proxy statement (DEF 14A) Apr 23, 2026
Latest 8-K (8-K) Sep 28, 2026
Securities registered for trading
| Class | Symbol | Exchange |
|---|---|---|
| Common stock, par value $0.0001 per share | CAMP | Nasdaq Global Market |
From the cover page of the 10-K filed Mar 5, 2026 (Section 12(b) of the Exchange Act).3
Risk factors and legal proceedings
The risks Camp4 Therapeutics Corp names in Item 1A of its 10-K filed Mar 5, 2026: 73 risks under these headings, as written.3
- Risks Related to Our Financial Position and Need for Additional Capital
- We have incurred significant losses since our inception, have no products approved for sale and we expect to incur losses for the foreseeable future.
- We have never generated revenue from product sales and may never achieve or maintain profitability.
- We will require substantial additional capital to finance our operations, 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 Research and Development of Our Product Candidates
- We are early in our development efforts. Our product candidates are in varying stages of development. As a result, it will be many years before we commercialize a product candidate, if ever. If we are unable to identify and advance product candidates through preclinical studies and clinical trials, obtain marketing approval and ultimately commercialize them, or experience significant delays in doing so, our business will be materially harmed.
- Our business is highly dependent on our lead product candidate, CMP-002, and we must complete clinical testing before we can seek regulatory approval and begin commercialization of any of our product candidates. If we are unable to obtain regulatory approval for, and successfully commercialize, our current product candidates, our business may be materially harmed, and such failure may affect the viability of any future product candidates.
- Our approach to the discovery and development of product candidates based on our RAP Platform is unproven, and we may not be successful in our efforts to develop and commercialize our product candidates and to identify and discover additional potential product candidates.
- Drug development is a lengthy and expensive process, and preclinical and clinical testing is uncertain as to the outcome. We may encounter substantial delays in the commencement, enrollment or completion of our clinical trials, or we may fail to demonstrate safety and effectiveness to the satisfaction of applicable regulatory authorities, which could prevent us from advancing or commercializing our product candidates on a timely basis, if at all.
- 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.
- The outcome of preclinical studies and earlier-stage clinical trials may not be predictive of future results or the success of later preclinical studies and clinical trials.
The other 63 risks
- Certain of the disorders we seek to treat, including SYNGAP1, have low prevalence and it may be difficult to identify and enroll patients with these disorders. If we experience delays or difficulties in the enrollment and/or maintenance of patients in clinical trials, our receipt of necessary regulatory approvals could be delayed or prevented.
- If any of our current or any future product candidates cause undesirable side effects or have other unexpected adverse properties, such side effects or properties could delay or prevent regulatory approval, limit the commercial potential or result in significant negative consequences following any potential marketing approval.
- We may expend our limited resources to pursue a particular program, product candidate or indication and fail to capitalize on programs, product candidates or indications that may be more profitable or for which there is a greater likelihood of success.
- We intend to conduct certain of our clinical trials globally. 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.
- Changes in the methods of manufacturing or formulation of our product candidates may result in additional costs or delay.
- If the market opportunities for any product candidates we develop are smaller than we believe they are, our revenue may be adversely affected, and our business may suffer. Because the target patient populations of certain of our programs are small, and the addressable patient population even smaller, we must be able to successfully identify patients and capture a significant market share to achieve profitability and growth.
- Changes in U.S. federal policy priorities and agency operations under the current U.S. presidential administration may adversely affect our business.
- Risks Related to Regulatory Approval and Commercialization
- Even if we complete the necessary preclinical studies and clinical trials, the marketing approval process is expensive, time-consuming and uncertain and may prevent us from obtaining approvals for the commercialization of any product candidates we may develop. If we are not able to obtain, or if there are delays in obtaining, required regulatory approvals, we will not be able to commercialize, or will be delayed in commercializing, product candidates we may develop, and our ability to generate revenue will be materially impaired.
- We may attempt to seek approval from the FDA or comparable foreign regulatory authorities, where applicable, under the accelerated approval pathways. We may fail to obtain approval under such accelerated approval pathways. Moreover, these pathways may not lead to a faster development, regulatory review or approval process and do not increase the likelihood that our product candidates will receive marketing approval.
- We may seek one or more designations or expedited programs for one or more of our product candidates, but we might not receive such designations or be allowed to proceed on expedited program pathways, and even if we do and proceed on such expedited program pathways in the future, such designations or expedited programs may not lead to a faster development or regulatory review or approval process, and each designation does not increase the likelihood that any of our product candidates will receive marketing approval in the United States.
- We have received orphan drug designation for CMP-001 for the treatment of UCDs, and we may pursue orphan drug designation for certain of our other product candidates. We may not be able to obtain or maintain the
- benefits of orphan drug designation, including potential orphan drug exclusivity, and even if we do, that exclusivity may not prevent regulatory authorities from approving other competing products.
- We have received rare pediatric disease designation from the FDA for CMP-001 for the treatment of UCDs, and we may pursue rare pediatric disease designation for certain of our other product candidates; however, there is no guarantee that a marketing application for such product candidates, if approved, will qualify for a rare pediatric disease priority review voucher.
- Obtaining and maintaining marketing approval or commercialization of our product candidates in the United States does not mean that we will be successful in obtaining marketing approval of our product candidates in other jurisdictions. Failure to obtain marketing approval in foreign jurisdictions would prevent any product candidates we may develop from being marketed in such jurisdictions, which, in turn, would materially impair our ability to generate revenue.
- Even if we obtain regulatory approval for any of our product candidates, we will still face extensive and ongoing regulatory requirements and obligations, which may result in significant additional expense.
- Even if any product candidate that we may develop receives marketing approval, it may fail to achieve the degree of market acceptance by physicians, patients, third-party payors and others in the medical community necessary for commercial success.
- The pricing, insurance coverage and reimbursement status of newly approved products is uncertain. Failure to obtain or maintain adequate coverage and reimbursement for our product candidates, if approved, could limit our ability to market those products and decrease our ability to generate product revenue.
- If we are unable to establish sales, marketing and distribution capabilities or enter into sales, marketing and distribution agreements with third parties, we may not be successful in commercializing any product candidates we may develop if and when they are approved.
- Our relationships with healthcare providers, physicians, patients and third-party payors may be subject to various anti-kickback, fraud and abuse, other healthcare laws and regulations, which could increase compliance costs, and our failure to comply with these laws and regulations could harm our reputation, subject us to significant fines and liability, or otherwise adversely affect our business.
- Current and future healthcare reform legislation or regulation may increase the difficulty and cost for us to obtain coverage for and commercialize our product candidates and may adversely affect the prices we may set.
- We are subject to privacy laws, regulations, and contractual obligations related to data privacy and security and changes in such laws, regulations, and contractual obligations and failure to comply with such requirements could subject us to significant fines and penalties, which may have a material adverse effect on our business, financial condition, results of operations or prospects.
- Laws and regulations governing any international operations we may have in the future may preclude us from developing, manufacturing and selling certain product candidates outside of the United States and require us to develop and implement costly compliance programs.
- Inadequate funding for the FDA, the Securities and Exchange Commission (the “SEC”), and other government agencies, including from government shut downs, or other disruptions to these agencies’ operations, could hinder their ability to hire and retain key leadership and other personnel, 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.
- Risks Related to Reliance on Third Parties
- We rely, and intend to continue to rely, on third parties to perform some of our preclinical studies and conduct our clinical trials. If these third parties do not successfully carry out their contractual duties, fail to comply with applicable regulatory requirements, or do not meet expected deadlines, our development programs may be delayed or subject to increased costs or we may be unable to obtain regulatory approval for or commercialize our product candidates.
- We currently depend on third-party suppliers for the manufacture of our product candidates. The loss of these or future third-party suppliers, or their inability to provide us with sufficient supply, could harm our business.
- Our reliance on third parties requires us to share our trade secrets, which increases the possibility that a competitor or other third party will discover our trade secrets or that our trade secrets will be misappropriated or disclosed.
- We may enter into collaborations with third parties for the research, development and commercialization of certain of the product candidates we may develop. If any such collaborations are not successful, we may not be able to capitalize on the market potential of those product candidates.
- If we are not able to establish collaborations on commercially reasonable terms, we may have to alter our development and commercialization plans.
- We are dependent on third-party vendors to provide certain licenses, products and services and our business and operations, including clinical trials, could be disrupted by any problems with our significant third-party vendors.
- Our research and development activities may involve the controlled use of potentially hazardous substances, including chemical and biological materials, by us or third parties such as our CROs and contract manufacturers.
- Risks Related to Our Intellectual Property
- Our rights to develop and commercialize our product candidates are subject, in part, to the terms and conditions of licenses granted to us by third parties. If we fail to comply with our obligations under these arrangements or otherwise experience disruptions to our business relationships with our current or any future licensors, we could lose such intellectual property rights that are important to our business.
- If we or our licensors are unable to obtain, maintain, enforce and adequately protect our intellectual property rights with respect to our product candidates and technology, or if the scope of any patent or other intellectual property 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 develop and commercialize our product candidates and technology may be adversely affected.
- Third parties may initiate legal proceedings alleging that we are infringing, misappropriating or otherwise violating their intellectual property rights, the outcome of which would be uncertain and could harm our business.
- We may be involved in lawsuits to protect or enforce our patents or other intellectual property or the intellectual property of our licensors, which could be expensive, time-consuming, and unsuccessful.
- We may not be able to protect our intellectual property 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 governmental patent agencies, and our patent protection could be reduced or eliminated for non-compliance with these requirements.
- Changes in patent law in the United States and in foreign jurisdictions could diminish the value of patents in general, thereby impairing our ability to protect our product candidates and our technology.
- Any patents covering our product candidates or our technology 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 and data exclusivity for our product candidates, our business may be materially harmed.
- We may be subject to claims challenging the inventorship or ownership of our patent and other intellectual property rights.
- 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 product candidates we may develop through acquisitions and in-licenses.
- We may be subject to claims asserting that our employees, consultants, independent contractors or advisors have wrongfully used or disclosed alleged trade secrets of their former employers or other third parties or claims asserting ownership of what we regard as our own intellectual property.
- 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 U.S.-based companies. Compliance with such regulations may limit our exclusive rights and limit our ability to contract with non-U.S. manufacturers.
- 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 face substantial competition, which may result in others discovering, developing or commercializing products before us or more successfully than we do.
- Our international activities subject us to various risks, and our failure to manage these risks could adversely affect our results of operations.
- We conduct certain research and development operations through our wholly-owned Australian subsidiary. If we lose our ability to operate in Australia, or if our subsidiary is unable to receive the research and development incentive payment allowed by Australian regulations, our business and results of operations could suffer.
- Our future success depends on our ability to retain key executives and to attract, retain and motivate qualified personnel.
- We may encounter difficulties in managing our growth and expanding our operations successfully.
- We may engage in strategic transactions that could impact our liquidity, increase our expenses, and present significant distractions to our management.
- Clinical trial and product liability lawsuits against us could divert our resources, could cause us to incur substantial liabilities and could limit commercialization of our product candidates.
- Our insurance policies are expensive and protect us from only some business risks, which will leave us exposed to significant uninsured liabilities.
- Our operations or those of the third parties upon whom we depend might be affected by the occurrence of a natural disaster, pandemic or other catastrophic event.
- Our business could be affected by litigation, government investigations, and enforcement actions.
- Our employees, consultants, collaborators, vendors, service providers and other contractors may engage in misconduct or other improper activities, including non-compliance with regulatory standards and requirements and insider trading.
- Our ability to utilize our net operating loss carryforwards and certain other tax attributes may be subject to limitations.
- Exchange rate fluctuations may affect our results of operations and financial conditions.
- International trade policies, including tariffs, sanctions and trade barriers, may adversely affect our business, financial condition, results of operations and growth prospects.
- Risks Related to Ownership of Our Common Stock
- An active, liquid, and orderly market for our common stock may not be sustained, or we may in the future fail to satisfy the continued listing requirements of Nasdaq.
- The trading price of the shares of our common stock could be highly volatile, and purchasers of our common stock could incur substantial losses.
Legal proceedings
From time to time, we may be subject to legal proceedings. We are not currently a party to or aware of any proceedings that we believe will have, individually or in the aggregate, a material adverse effect on our business, financial condition or results of operations. Regardless of outcome, litigation can have an adverse impact on us because of defense and settlement costs, diversion of management resources, and other factors.
The opening of Item 3, Legal Proceedings.3
Sources
The numbers on this page point here. Each one links to the record it came from.
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- 9SEC EDGARKelly Gold, Chief financial officer
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- 12SEC EDGARYuri Maricich, Chief Medical Officer
- 13SEC EDGARMichael F. Maclean, Board member
- 14SEC EDGARDouglas E. Williams, Board member
- 15SEC EDGARMurray Stewart, Board member
- 16SEC EDGARAmir Nashat, Board member
- 17SEC EDGARAndrew J. Schwab, Board member
- 18SEC EDGARIngo Chakravarty, Board member
- 19SEC EDGARJames P. Boylan, Board member
- 20SEC EDGARPaula Ragan, Board member
- 21SEC EDGARRavi I. Thadhani, Board member
- 22SEC EDGARSteven H. Holtzman, Board member
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- 25SEC EDGARResults of operations
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- 29SEC EDGARRegulation FD disclosure
- 30SEC EDGARResults of operations
- 31SEC EDGARExecutive or director change
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- 33SEC EDGARRegulation FD disclosure
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- 36NasdaqNasdaq calendar
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