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| Part I | | Financial Information | | |
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| Item 1. | | Financial Statements (Unaudited) | | 6 |
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| | | Condensed Balance Sheets | | 6 |
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| | | Condensed Statements of Operations and Comprehensive Loss | | 7 |
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| | | Condensed Statements of Stockholders’ Equity | | 8 |
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| | | Condensed Statements of Cash Flows | | 9 |
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| | | Notes to Condensed Financial Statements | | 10 |
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| Item 2. | | Management’s Discussion and Analysis of Financial Condition and Results of Operations | | 17 |
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| Item 3. | | Quantitative and Qualitative Disclosures About Market Risk | | 28 |
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| Item 4. | | Controls and Procedures | | 28 |
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| Part II | | Other Information | | 29 |
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| Item 1. | | Legal Proceedings | | 29 |
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| Item 1A. | | Risk Factors | | 29 |
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| Item 2. | | Unregistered Sales of Equity Securities and Use of Proceeds | | 101 |
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| Item 3. | | Defaults Upon Senior Securities | | 102 |
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| Item 4. | | Mine Safety Disclosures | | 102 |
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| Item 5. | | Other Information | | 102 |
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| Item 6. | | Exhibits | | 102 |
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| Signatures | | | | 104 |
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SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTS
This Quarterly Report on Form 10-Q (Quarterly Report) contains forward-looking statements that involve risks and uncertainties. All statements other than statements of historical facts contained in this Quarterly Report, including statements regarding our future results of operations and financial position, business strategy, development plans, planned preclinical studies and clinical trials, future results of clinical trials, expected research and development costs, regulatory strategy, timing and likelihood of success, as well as plans and objectives of management for future operations, are forward-looking statements. In some cases, you can identify forward-looking statements by terminology such as “may,” “will,” “could,” “would,” “should,” “likely,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “projects,” “potential,” “continue” or the negative of these terms or other comparable terminology. These forward-looking statements include, but are not limited to, statements about:
| ● | the safety and efficacy, and the ability of our preclinical studies and clinical trials to demonstrate the safety and efficacy, of our product candidates, and other positive results; |
| ● | our ability to utilize our proprietary drug discovery platform to develop a pipeline of product candidates to address muscle diseases; |
| ● | the timing, progress and results of preclinical studies and clinical trials for sevasemten, EDG-7500, EDG-15400, product candidates from our EDG-003 cardiometabolic discovery program and other product candidates we may develop, including statements regarding the timing of initiation and completion of studies or trials and related preparatory work, the period during which the results of the studies or trials will become available, potential registrational studies or cohorts and our research and development programs; |
| ● | the timing, scope and likelihood of domestic and foreign regulatory filings and approvals, including timing of final U.S. Food & Drug Administration (FDA) approval of or Investigational New Drugs of sevasemten, EDG-7500, EDG-15400, product candidates from our EDG-003 cardiometabolic discovery program and any other future product candidates; |
| ● | our ability to develop and advance our current product candidates and programs into, and successfully complete, clinical studies; |
| ● | our manufacturing, commercialization, operations and marketing capabilities, relationships with other businesses and other business strategies, systems and relationships; |
| ● | our plans relating to commercializing our product candidates, if approved, including the geographic areas of focus and sales strategy; |
| ● | the need to hire additional personnel and our ability to attract and retain such personnel; |
| ● | the size of the market opportunity for our product candidates, including our estimates of the number of patients who suffer from the diseases we are targeting and our expectations regarding the implementation of newborn screening for muscular dystrophy; |
| ● | our expectations regarding the approval and use of our product candidates in combination with other drugs; |
| ● | our competitive position and the success of competing product candidates and therapies that are or may become available; |
| ● | the ability of our programs to offer opportunities for us to expand into other severe muscle diseases; |
| ● | our estimates of the number of patients that we will enroll in our clinical trials; |
| ● | our estimates relating to the timing of completing enrollment for our clinical trials and natural history study; |
| ● | the beneficial characteristics, and the potential safety, efficacy and therapeutic effects, of our product candidates; |
| ● | our ability to obtain and maintain regulatory approval of our product candidates, and the timing or likelihood of regulatory filings and approvals, including our expectations to maintain the Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) for sevasemten and our expectation to seek special designations for our other product candidates; |
| ● | our plans relating to the further development of our product candidates, including additional indications we may pursue; |
| ● | existing regulations and regulatory developments in the United States, Europe and other jurisdictions; |
| ● | our expectations regarding the impact of public health pandemics on our business; |
| ● | our expectations regarding the impact of changes in the U.S. government administration and policy positions; |
| ● | our expectations regarding the impact of Russia’s war with Ukraine, and war and instability in the Middle East on our business; |
| ● | our expectations regarding the impact of instability in the U.S. banking and financial services sector and other macroeconomic trends; |
| ● | our intellectual property position, including the scope of protection we are able to establish and maintain for intellectual property rights covering sevasemten, EDG-7500, EDG-15400, product candidates from our EDG-003 cardiometabolic discovery program and other product candidates we may develop, including the extensions of existing patent terms where available, the validity of intellectual property rights held by third parties, and our ability not to infringe, misappropriate or otherwise violate any third-party intellectual property rights; |
| ● | our continued reliance on third parties to conduct additional preclinical studies and planned clinical trials of our product candidates, and for the manufacture of our product candidates for preclinical studies and clinical trials; |
| ● | our relationships with patient advocacy groups, key opinion leaders, regulators, the research community and payors; |
| ● | our ability to obtain, and negotiate favorable terms of, any collaboration, licensing or other arrangements that may be necessary or desirable to develop, manufacture or commercialize our product candidates; |
| ● | the pricing and reimbursement of sevasemten, EDG-7500, EDG-15400, product candidates from our EDG-003 cardiometabolic discovery program and other product candidates we may develop, if approved; |
| ● | the rate and degree of market acceptance and clinical utility of sevasemten, EDG-7500, EDG-15400, product candidates from our EDG-003 cardiometabolic discovery program and other product candidates we may develop; |
| ● | our estimates regarding expenses, future revenue, capital requirements and needs for additional financing which may be impacted by many factors including inflation; |
| ● | our financial performance; |
| ● | the period over which we estimate our existing cash, cash equivalents and marketable securities will be sufficient to fund our future operating expenses and capital expenditure requirements; and |
| ● | the impact of laws and regulations. |
We have based these forward-looking statements largely on our current expectations and projections about our business, the industry in which we operate and financial trends that we believe may affect our business, financial condition, results of operations and prospects, and these forward-looking statements are not guarantees of future performance or development. These forward-looking statements speak only as of the date of this Quarterly Report and are subject to a number of risks, uncertainties and assumptions described in the section titled “Risk Factors” and elsewhere in this Quarterly Report. Because forward-looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified, you should not rely on these forward-looking statements as predictions of future events. The events and circumstances reflected in our forward-looking statements may not be achieved or occur and actual results could differ materially from those projected in the forward-looking statements. Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events or otherwise.
In addition, statements that “we believe” and similar statements reflect our beliefs and opinions on the relevant subject. These statements are based upon information available to us as of the date of this Quarterly Report and while we believe such information forms a reasonable basis for such statements, such information may be limited or incomplete, and our statements should not be read to indicate that we have conducted an exhaustive inquiry into, or review of, all potentially available relevant information. These statements are inherently uncertain and you are cautioned not to unduly rely upon these statements.
