What changed in this filing

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Item 2. Management’s Discussion and Analysis

2024-11-07 compared with 2024-08-08 · 50 added, 50 removed, 196 unchanged (34% of the section changed)

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We are advancing two clinical-stage programs in muscular dystrophies and severe cardiac diseases.

Sevasemten (EDG-5506) is an orally administered skeletal myosin inhibitor in a pivotal stage trial for Becker muscular dystrophy as well as ongoing Phase 2 programs in Duchenne muscular dystrophy.

EDG-7500, currently in a Phase 2 trial, is a novel cardiac sarcomere modulator for the treatment of hypertrophic cardiomyopathy (HCM) and other disorders of diastolic dysfunction.

Sevasemten (EDG-5506) is an orally administered skeletal myosin inhibitor in a pivotal stage trial for Becker muscular dystrophy (Becker) as well as ongoing Phase 2 programs in Duchenne muscular dystrophy.

EDG-7500, currently in a multipart Phase 2 trial, is a novel cardiac sarcomere modulator for the treatment of hypertrophic cardiomyopathy (HCM) and other disorders of diastolic dysfunction.

We also continue to advance our preclinical pipeline, including characterization of novel cardiometabolic targets.

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We have incurred significant losses since the commencement of our operations.

Our net losses were $31.5 million and $60.0 million for the three and six months ended June 30, 2024, respectively and $21.5 million and $44.3 million for the three and six months ended June 30, 2023, respectively, and we expect to continue to incur significant losses for the foreseeable future as we advance our product candidates through preclinical development and clinical trials and seek regulatory approval of our product candidates.

Our net losses were $34.1 million and $94.2 million for the three and nine months ended September 30, 2024, respectively and $25.7 million and $70.0 million for the three and nine months ended September 30, 2023, respectively, and we expect to continue to incur significant losses for the foreseeable future as we advance our product candidates through preclinical development and

clinical trials and seek regulatory approval of our product candidates.

Our net losses may fluctuate significantly from period to period, depending on the timing of and expenditures on our planned research and development activities.

As of June 30, 2024, we had an accumulated deficit of $304.8 million.

As of September 30, 2024, we had an accumulated deficit of $338.9 million.

To date, we have financed our operations primarily through private placements of convertible preferred stock and public offerings of our common stock.

From inception through June 30, 2024, these private placements have provided gross proceeds of $160.7 million, and the initial public offering, follow-on public offering, issuance of our common stock under an “at the market offering” program (the ATM Program), and underwritten registered direct offering have generated net proceeds of $606.6 million.

From inception through September 30, 2024, these private placements have provided gross proceeds of $160.7 million, and the initial public offering, follow-on public offering, issuance of our common stock under an “at the market offering” program (the ATM Program), and underwritten registered direct offering have generated net proceeds of $606.6 million.

We believe that our existing cash and cash equivalents and marketable securities of $492.5 million will enable us to fund our planned operating expenses and capital expenditure requirements through at least the next 12 months.

Macroeconomic and Geopolitical Developments

We are monitoring macroeconomic and geopolitical developments, such as inflation, instability in the banking and financial services sector, tightening of the credit markets, the upcoming U.S.

presidential election, international conflicts, public health pandemics, cybersecurity and sanctions so that the Company can be prepared to react to new developments as they arise.

We are monitoring macroeconomic and geopolitical developments, such as inflation, instability in the banking and financial services sector, tightening of the credit markets, the upcoming change in the U.S.

presidential administration, international conflicts, public health pandemics, cybersecurity and sanctions so that the Company can be prepared to react to new developments as they arise.

Components of Our Results of Operations

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Product candidates in later stages of clinical development generally have higher development costs than those in earlier stages, primarily due to the increased size and duration of later-stage clinical trials.

We are currently conducting four Phase 2 clinical trials for sevasemten (CANYON, LYNX, FOX and DUNE), a potentially registrational, or pivotal cohort in individuals with Becker (GRAND CANYON), and a Phase 2 study for EDG-7500 (CIRRUS-HCM).

We are currently conducting four Phase 2 clinical trials with sevasemten for people with muscular dystrophy (CANYON, LYNX, FOX and DUNE), a potentially registrational, or pivotal cohort, in individuals with Becker (GRAND CANYON), and a multipart Phase 2 study with EDG-7500 for people with HCM (CIRRUS-HCM).

As a result, we expect that our research and development expenses will increase substantially over the next several years as we advance sevasemten, EDG-7500, a product from our EDG-003 cardiometabolic discovery program through clinical trials and additional product candidates;

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Results of Operations

Comparison of the three months ended June 30, 2024 and 2023

The following table summarizes our results of operations for the three months ended June 30, 2024 and 2023:

Three months ended June 30,

Comparison of the three months ended September 30, 2024 and 2023

The following table summarizes our results of operations for the three months ended September 30, 2024 and 2023:

Three months ended September 30,

(in thousands)

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The following table summarizes our research and development expenses:

Three months ended June 30,

Three months ended September 30,

(in thousands)

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Total research and development expenses

Research and development expenses were $30.7 million and $19.6 million for the three months ended June 30, 2024 and 2023, respectively.

The increase of $11.1 million was driven by an increase of $5.1 million in sevasemten clinical program expenses, which was primarily due to increased clinical trial activity in our LYNX trial from site activations and patient enrollment and costs incurred in connection with our GRAND CANYON, MESA, and FOX trials which were not active during the comparable period in 2023.

EDG-7500 clinical program expenses of $5.1 million are now shown separately in the above table due to advancing into a clinical trial in the third quarter of 2023.

These expenses include the trial costs as well as manufacturing costs to support current and future trials and nonclinical costs during the three months ended June 30, 2024.

Research and development expenses were $32.2 million and $23.8 million for the three months ended September 30, 2024 and 2023, respectively.

The increase of $8.4 million was driven by an increase of $4.6 million in sevasemten clinical program expenses, which was primarily due to increased clinical trial activity from site activations, patient enrollment, and manufacturing costs in our GRAND CANYON and FOX trials that were in start-up phases during the comparable period in 2023 and general clinical and outreach costs incurred in connection with all sevasemten trials.

Internal costs increased $3.1 million, primarily related to personnel costs and stock-based compensation resulting from increased employee headcount to support the growth of our research and development programs.

Discovery and preclinical expenses decreased $1.4 million as a result of EDG-7500 costs, now shown separately, which were a significant portion of the 2023 period’s discovery and preclinical expenses along with other costs from our EDG-003 program.

Discovery and preclinical expenses increased $0.6 million as a result of additional manufacturing and nonclinical costs related to our EDG-003 program and other cardio-metabolic research.

EDG-7500 clinical program expenses increased $0.1 million primarily related to the start-up, site activation, and patient enrollment for our multipart Phase 2 CIRRUS-HCM trial;

our Phase 1 trial was in a similar start-up phase in the comparable period in 2023.

General and administrative expenses

General and administrative expenses were $7.4 million and $5.8 million for the three months ended June 30, 2024 and 2023, respectively.

General and administrative expenses were $8.2 million and $5.7 million for the three months ended September 30, 2024 and 2023, respectively.

The increase of $2.5 million was primarily due to $1.6 million in increased personnel-related costs from increased headcount and stock-based compensation and $0.9 million in increased professional and consulting costs and other administrative costs.

Interest income

Interest income was $6.6 million and $3.9 million for the three months ended June 30, 2024 and 2023, respectively.

The increase of $2.7 million was primarily due to higher balances and interest rates on our marketable securities during the three months ended June 30, 2024 as compared to the three months ended June 30, 2023.

Comparison of the six months ended June 30, 2024 and 2023

The following table summarizes our results of operations for the six months ended June 30, 2024 and 2023:

Six months ended June 30,

Interest income was $6.3 million and $3.7 million for the three months ended September 30, 2024 and 2023, respectively.

The increase of $2.6 million was primarily due to higher balances of marketable securities during the three months ended September 30, 2024 as compared to the three months ended September 30, 2023.

Comparison of the nine months ended September 30, 2024 and 2023

The following table summarizes our results of operations for the nine months ended September 30, 2024 and 2023:

Nine months ended September 30,

(in thousands)

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The following table summarizes our research and development expenses:

Six months ended June 30,

Nine months ended September 30,

(in thousands)

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Total research and development expenses

Research and development expenses were $58.4 million and $39.4 million for the six months ended June 30, 2024 and 2023, respectively.

The increase of $18.9 million was primarily driven by an increase of $10.1 million in sevasemten clinical program expenses which was primarily due to the activation of our GRAND CANYON, LYNX, MESA, and FOX trials which were not active during the comparable period in 2023.

EDG-7500 clinical program expenses of $9.1 million are now shown separately in the above table due to advancing into a clinical trial.

These expenses include the trial costs as well as manufacturing costs to support current and future trials and nonclinical costs during the six months ended June 30, 2024.

Internal costs increased $5.1 million, primarily related to a $3.3 million increase in personnel costs related to salaries, benefits, and employee expenses resulting from increased employee headcount to support the growth of our research and development programs, a $1.2 million increase in stock-based compensation related to increased headcount and overall higher stock prices driving increased option fair values, and $0.6 million related to depreciation and other allocated costs.

Discovery and preclinical expenses decreased $5.4 million as a result of EDG-7500 costs, now shown separately, which were a significant portion of the 2023 period’s discovery and preclinical expenses along with other costs from our EDG-003 program.

Research and development expenses were $90.6 million and $63.2 million for the nine months ended September 30, 2024 and 2023, respectively.

The increase of $27.4 million was primarily driven by an increase of $14.7 million in sevasemten clinical program expenses which was primarily due an $8.6 million increase in our GRAND CANYON trial, $3.5 million increase in our FOX trial, $2.4 million increase in our LYNX trial, and $2.0 million increase in our MESA trial related to site activation and patient enrollment as the trials were in startup phases in the third quarter of 2023;

offset by a $1.3 million decrease in our CANYON trial, which is nearing completion.

EDG-7500 clinical program expenses increased $9.2 million related to the completion of our Phase 1 trial that was activated in late 2023 and manufacturing, site activation, and patient enrollment costs related to our CIRRUS-HCM trial that began in 2024.

Internal costs increased $8.3 million, primarily related to a $5.0 million increase in personnel costs related to salaries, benefits, and employee expenses resulting from increased employee headcount to support the growth of our research and development programs, a $2.5 million increase in stock-based compensation related to increased headcount and overall higher stock prices driving increased option fair values, and $0.8 million related to depreciation and other facilities costs.

Discovery and preclinical expenses decreased $4.8 million as a result of EDG-7500 costs, now shown separately, which were a significant portion of the first nine months of 2023’s discovery and preclinical expenses along with other costs from our EDG-003 program.

General and administrative expenses

General and administrative expenses were $14.5 million and $11.6 million for the six months ended June 30, 2024 and 2023, respectively.

General and administrative expenses were $22.7 million and $17.3 million for the nine months ended September 30, 2024 and 2023, respectively.

The increase of $5.4 million was primarily due to $3.8 million in increased personnel-related costs from increased headcount and stock-based compensation and $1.6 million in increased professional and consulting costs and other administrative costs.

Interest income

Interest income was $12.8 million and $6.7 million for the six months ended June 30, 2024 and 2023, respectively.

The increase of $6.1 million was primarily due to higher balances and interest rates on our marketable securities during the six months ended June 30, 2024, as compared to the six months ended June 30, 2023.

Interest income was $19.1 million and $10.5 million for the nine months ended September 30, 2024 and 2023, respectively.

The increase of $8.7 million was primarily due to higher balances of marketable securities during the nine months ended September 30, 2024, as compared to the nine months ended September 30, 2023.

Liquidity and Capital Resources

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To date, we have financed our operations primarily through private placements of convertible preferred stock and public offerings of our common stock.

As of June 30, 2024, we had cash, cash equivalents and marketable securities in the amount of $511.8 million.

From inception through September 30, 2024, these private placements have provided gross proceeds of $160.7 million, and the initial public offering, follow-on public offering, issuance of our common stock under the ATM Program, and underwritten registered direct offering have generated net proceeds of $606.6 million.

As of September 30, 2024, we had cash, cash equivalents and marketable securities in the amount of $492.5 million.

The following table summarizes our sources and uses of cash for each of the periods presented:

Six months ended June 30,

Nine months ended September 30,

(in thousands)

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Operating activities

Cash used in operating activities during the six months ended June 30, 2024 was primarily driven by our net loss for the period of $60.0 million, and was also impacted by changes in operating assets and liabilities which decreased net working capital by $0.9 million.

Cash used in operating activities during the nine months ended September 30, 2024 was primarily driven by our net loss for the period of $94.2 million, and was also impacted by changes in operating assets and liabilities which decreased net working capital by $3.4 million.

Cash used in operating activities was reduced by non-cash charges of $8.7 million relating to stock-based compensation expense of $17.0 million, amortization of premium and accretion of discounts, net on marketable securities of $9.9 million, depreciation of $1.5 million and amortization of right-of-use asset of $0.2 million.

Cash used in operating activities during the six months ended June 30, 2023 was primarily driven by our net loss for the period of $44.3 million, and was also impacted by changes in operating assets and liabilities which increased net working capital by $2.3 million.

Cash used in operating activities during the nine months ended September 30, 2023 was primarily driven by our net loss for the period of $70.0 million, and was also impacted by changes in operating assets and liabilities which increased net working capital by $1.4 million.

Cash used in operating activities was reduced by non-cash charges of $6.3 million relating to stock-based compensation expense of $12.4 million, amortization of premium and accretion of discounts, net on marketable securities of $7.3 million, depreciation of $1.1 million and amortization of right-of-use asset of $0.1 million.

Investing activities

Cash used in investing activities during the six months ended June 30, 2024 amounted to $230.9 million, which was due to $368.6 million in purchases of marketable securities and $0.5 million for purchases of equipment, which was partially offset by $136.4 million in maturities of marketable securities and $1.8 million in sales of marketable securities.

Cash provided by investing activities during the six months ended June 30, 2023 amounted to $52.5 million, which was due to $183.9 million in maturities of marketable securities and $5.1 million in sales of marketable securities, which was partially offset by $131.4 million in purchases of marketable securities and $5.1 million for purchases of equipment.

Cash used in investing activities during the nine months ended September 30, 2024 amounted to $205.6 million, which was due to $413.9 million in purchases of marketable securities and $1.0 million for purchases of equipment, which was partially offset by $196.6 million in maturities of marketable securities and $12.7 million in sales of marketable securities.

Cash provided by investing activities during the nine months ended September 30, 2023 amounted to $75.9 million, which was due to $278.4 million in maturities of marketable securities and $19.2 million in sales of marketable securities, which was partially offset by $216.1 million in purchases of marketable securities and $5.6 million for purchases of equipment.

Financing activities

Cash provided by financing activities during the six months ended June 30, 2024 was $243.0 million, which was due to cash proceeds of $7.0 million from the ATM Program, $240.0 million from the underwritten registered direct offering, $3.5 million in proceeds from the issuance of common stock upon exercise of stock options, and $0.4 million in proceeds from the employee stock purchase plan, which was partially offset by $7.9 million for the payment of underwriting commissions and offering costs.

Cash provided by financing activities during the six months ended June 30, 2023 was $0.4 million, which was due to cash proceeds of $52,000 from the issuance of common stock upon the exercise of stock options and $0.3 million in proceeds from the employee stock purchase plan.

Cash provided by financing activities during the nine months ended September 30, 2024 was $246.4 million, which was due to cash proceeds of $7.0 million from the ATM Program, $240.0 million from the underwritten registered direct offering, $7.1 million in proceeds from the issuance of common stock upon exercise of stock options, and $0.4 million in proceeds from the employee stock purchase plan, which was partially offset by $7.9 million for the payment of underwriting commissions and offering costs and $0.3 million for the payment of deferred offering costs.

Cash provided by financing activities during the nine months ended September 30, 2023 was $0.2 million, which was due to cash proceeds of $0.3 million in proceeds from the employee stock purchase plan and cash proceeds of 0.1 million from the issuance of common stock upon the exercise of stock options, which was partially offset by $0.2 million for the payment of deferred offering costs.

Funding requirements

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We do not currently have any long-term material capital requirements other than what will be required to fund operations for the foreseeable future and the amounts disclosed on the contractual obligations and commitments section below.

In order to complete the process of obtaining regulatory approval for our product candidates and to build the

sales, marketing and distribution infrastructure that we believe will be necessary to commercialize our product candidates, if approved, we will require substantial additional funding.

In order to complete the process of obtaining regulatory approval for our product candidates and to build the sales, marketing and distribution infrastructure that we believe will be necessary to commercialize our product candidates, if approved, we will require substantial additional funding.

We have based our projections of operating capital requirements on assumptions that may prove to be incorrect and we may use all of our available capital resources sooner than we expect.

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We expect that our existing cash and cash equivalents and marketable securities, will be sufficient to enable us to fund our planned operating expenses and capital expenditure requirements through at least the next 12 months.

Our short-term material cash requirements as of June 30, 2024 are to fund our operations, which consist primarily of research and development expenses related to our programs, and to a lesser extent, general and administrative expenses.

Our short-term material cash requirements as of September 30, 2024 are to fund our operations, which consist primarily of research and development expenses related to our programs, and to a lesser extent, general and

administrative expenses.

We have entered into contracts in the normal course of business with CROs, CDMOs and other third parties for preclinical research studies and testing, clinical trials and manufacturing services.

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Payments due upon cancellation consist only of payments for services provided and expenses incurred, including non-cancelable obligations of our service providers, up to the date of cancellation.

Our long-term cash requirements as of June 30, 2024 includes our lease obligations.

Our long-term cash requirements as of September 30, 2024 includes our lease obligations.

In January 2022, we entered into a lease agreement for approximately 18,614 square feet of office and laboratory space in Boulder, Colorado which includes escalating rent payments and an 8.2 year term, plus our share of operating expenses.

In February 2023, the lease was modified to occupy an additional 9,624 square feet of office space, with aggregate payments of approximately $1.5 million over the initial 7.3 year term, plus our share of operating expenses.

As of June 30, 2024 our total operating lease liability balance is $5.0 million, of which $1.0 million is a current liability.

As of September 30, 2024 our total operating lease liability balance is $4.4 million, of which $1.0 million is a current liability.

Critical Accounting Estimates

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Our critical accounting policies are described in the section titled “Management’s Discussion and Analysis of Financial Condition and Results of Operations—Critical Accounting Estimates” in our Annual Report on Form 10-K filed with the SEC on February 22, 2024 and the notes to the financial statements appearing elsewhere in this Quarterly Report on Form 10-Q.

During the three and six months ended June 30, 2024, there were no material changes to our critical accounting estimates from those discussed in our Annual Report on Form 10-K filed on February 22, 2024.

During the three and nine months ended September 30, 2024, there were no material changes to our critical accounting estimates from those discussed in our Annual Report on Form 10-K filed on February 22, 2024.

Recently Issued Accounting Pronouncements

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Emerging Growth Company and Smaller Reporting Company Status

Section 107 of The Jumpstart Our Business Startups Act of 2012 (JOBS Act) permits an “emerging growth company” such as us to take advantage of an extended transition time to comply with new or revised accounting

standards as applicable to public companies.

Thus, an emerging growth company can delay the adoption of certain accounting standards until those standards would otherwise apply to private companies.

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Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-Q

Item 2. Management’s Discussion and Analysis