Skip to main content

Scholar Rock Holding Corp

For the period ended Mar 31, 2025. Filed May 14, 2025.

View on SEC.gov

About this filing

A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.

Mar 31, 2025 · Filed May 14, 2025

SRRK$46.94+3.2%Stored close · as of Nov 6, 2026

Extracted filing text, with the paragraph anchors used by the comparison.

See the whole filing, with its images and exhibits

Notes to Consolidated Financial Statements

  1. Nature of the Business

Scholar Rock Holding Corporation (the “Company”) is a late-stage biopharmaceutical company focused on advancing innovative treatments for neuromuscular diseases, cardiometabolic disorders, and other serious diseases. As a global leader in transforming growth factor beta (“TGFβ”) superfamily biology, the Company’s novel understanding of the molecular mechanisms of growth factor activation enabled the development of a proprietary platform for the discovery and development of monoclonal antibodies that locally and selectively target the precursor, or latent, forms of growth factors. By targeting the signaling proteins at the cellular level and acting in the disease microenvironment, the Company believes that it may avoid the historical dose-limiting safety challenges associated with inhibiting growth factors for therapeutic effect. We believe our focus on biologically validated growth factors may facilitate a more efficient development path.

The Company’s first product candidate, apitegromab, is a highly selective, fully human, monoclonal antibody, with a unique mechanism of action that results in inhibition of the activation of the growth factor, myostatin, in skeletal muscle. Apitegromab is being developed as a potential first muscle-targeted therapy for the treatment of spinal muscular atrophy (“SMA”). In October 2024, the Company announced positive top-line results from SAPPHIRE, a pivotal Phase 3 clinical trial to evaluate the efficacy and safety of apitegromab in patients with nonambulatory Type 2 and Type 3 SMA. The study achieved its primary endpoint. The Company submitted a U.S. Biologics License Application (“BLA”) to the FDA in January 2025, which is now under priority review with a Prescription Drug User Fee Act (“PDUFA”) target action date of September 22, 2025. In March 2025 the Company submitted to the European Medicines Agency (“EMA”) and received validation of its marketing authorisation application (“MAA”) for apitegromab for the treatment of SMA. The FDA granted Fast Track designation, Rare Pediatric Disease designation and Orphan Drug designation to apitegromab for the treatment of SMA in May 2021, August 2020 and March 2018, respectively. The EMA granted Priority Medicines (“PRIME”) designation in March 2021 and the EC granted orphan medicinal product designation in December 2018 to apitegromab for the treatment of SMA. If apitegromab is approved on or around its PDUFA action date, the Company expects to initiate a commercial product launch in the fourth quarter of 2025 in the United States, with a commercial launch of apitegromab in Europe anticipated to follow in 2026.

In October 2023, the Company announced an expansion of its therapeutic focus into cardiometabolic disorders by advancing its anti-myostatin program with SRK-439, a novel, fully human anti-myostatin monoclonal antibody, for evaluation in cardiometabolic disorders, including obesity. The Company is developing SRK-439 towards a potential investigational new drug application submission in the third quarter of 2025. In 2024, the Company presented preclinical data at scientific conferences which support the potential of SRK-439 to increase lean mass and contribute to a favorable body composition in conjunction with GLP-1 receptor agonist (“GLP-1 RA”) treatment. To inform the development of SRK-439, in May 2024 the Company initiated the Phase 2 EMBRAZE proof-of-concept trial, designed to assess the safety and efficacy of apitegromab to preserve muscle mass in individuals living with obesity and on background therapy of a GLP-1 RA. In September 2024, the Company announced that it had completed enrollment in the Phase 2 EMRAZE proof-of-concept trial. Top-line results from this trial are expected in the second quarter of 2025.

The Company’s second product candidate, SRK-181, a highly selective inhibitor of the activation of latent TGFβ1, is being developed for the treatment of cancers that are resistant to checkpoint inhibitor therapies, such as anti-PD-1 or anti-PD-L1 antibody therapies (referred to together as anti-PD-(L)1 antibody therapies). SRK-181 is being evaluated in the Company’s Phase 1 DRAGON proof-of-concept clinical trial in patients with locally advanced or metastatic solid tumors that exhibit resistance to anti-PD-(L)1 antibody therapies. The Company completed enrollment of the Phase 1 DRAGON trial in December 2023 and continues to treat patients who remain on study. This two-part clinical trial consists of a dose escalation portion (Part A) and a dose expansion portion evaluating SRK-181 in combination with an approved anti-PD-(L)1 antibody therapy (Part B). Part B commenced in 2021 and includes the following active cohorts: urothelial carcinoma, cutaneous melanoma, non-small cell lung cancer, clear cell renal cell carcinoma and head and neck squamous cell carcinoma. Safety, efficacy and biomarker data were presentedin June 2024 at the American Society of Clinical Oncology annual meeting and in November 2024 at the Society for Immunotherapy of Cancer 39 th Annual Meeting.

Additionally, the Company continues to develop a pipeline of product candidates to deliver novel therapies to underserved patients suffering from a wide range of serious diseases, including neuromuscular disorders, cardiometabolic disorders, cancer, fibrosis, and iron-restricted anemia. The Company was originally formed in May 2012. Its principal offices are in Cambridge, Massachusetts.

Since its inception, the Company’s operations have focused on research and development of monoclonal antibodies that selectively inhibit activation of growth factors for therapeutic effect, as well as establishing the Company’s intellectual property portfolio and performing research and development activities. The Company has primarily financed its operations through various equity financings, as well as research and development collaboration agreements and the Company’s debt facility (see Note 10).

Revenue generation activities have been limited to two collaborations, both containing research services and the issuance of a license. No revenues have been recorded from the sale of any commercial product.

The Company is subject to a number of risks similar to other life science companies, including, but not limited to, successful discovery and development of its drug candidates, raising additional capital, development by its competitors of new technological innovations, protection of proprietary technology and regulatory approval and market acceptance of the Company’s product candidates. The Company anticipates that it will continue to incur significant operating losses for the next several years as it continues to develop its product candidates.

The Company believes that its existing cash, cash equivalents, and marketable securities at March 31, 2025 will be sufficient to allow the Company to fund its current operations through at least a period of one year after the date these financial statements are issued.

  1. Summary of Significant Accounting Policies

Summary of Significant Accounting Policies

The significant accounting policies used in preparation of the unaudited consolidated financial statements are described in the Company’s audited consolidated financial statements as of and for the year ended December 31, 2024, and the notes thereto, which are included in the Company’s Annual Report on Form 10-K. There have been no material changes to the significant accounting policies previously disclosed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2024.

Cash, Cash Equivalents and Restricted Cash

The following table reconciles cash, cash equivalents and restricted cash per the balance sheet to the statement of cash flows (in thousands):

​ · ​ · ​ · ​ · ​ · ​ · ​
​As of March 31,
​ · 2025 · 2024
Cash and cash equivalents​$137,926​$91,979
Restricted cash​2,407​2,407
​​$140,333​$94,386

Unaudited Interim Financial Information

The consolidated financial statements of the Company included herein have been prepared pursuant to the rules and regulations of the Securities and Exchange Commission (the “SEC”). The unaudited consolidated financial statements include the accounts of Scholar Rock Holding Corporation and its wholly owned subsidiaries. All intercompany transactions and balances have been eliminated in consolidation. In the opinion of management, the information furnished reflects all adjustments, all of which are of a normal and recurring nature, necessary for a fair presentation of the results for the reported interim periods. The Company considers events or transactions that occur after the balance sheet date but before the financial statements are issued to provide additional evidence relative to certain estimates or to

identify matters that require additional disclosure. The results of operations for interim periods are not necessarily indicative of results to be expected for the full year or any other interim period.

Use of Estimates

The preparation of financial statements in accordance with accounting principles generally accepted in the U.S. (“GAAP”) requires management to make estimates and judgments that may affect the reported amounts of assets and liabilities and related disclosures of contingent assets and liabilities at the date of the financial statements and the related reporting of revenues and expenses during the reporting period. Management bases its estimates on historical experience and on various assumptions that are believed to be reasonable under the circumstances. Actual results could differ from those estimates.

Recently Adopted Accounting Pronouncements

In November 2023, the Financial Accounting Standards Board (“FASB”) issued Accounting Standards Update (“ASU”) 2023-07,Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures (“ASU 2023-07”). The standard requires disclosure of incremental segment information on an annual and interim basis and allows for multiple measures of a segment’s profit or loss provided that one of those measures is consistent with GAAP. The amendments in this update do not change how a public company identifies its operating segments, aggregates those operating segments, or applies the quantitative thresholds to determine its reportable segments, but rather requires public entities to disclose significant segment expenses and other segment items on an annual and interim basis and provide in interim periods all disclosures about a reporting segment’s profit or loss and assets that are currently required annually. ASU 2023-07 becomes effective for the annual period starting on January 1, 2024, and for interim periods starting on January 1, 2025**.**The adoption of this standard did not have a material impact on the Company’s consolidated financial position and results of operations.

Recently Issued Accounting Pronouncements

In December 2023, the FASB issued ASU 2023-09,Income Taxes (Topic 740): Improvements to Income Tax Disclosures (“ASU 2023-09”), which enhances the transparency of income tax disclosures to provide information to investors to better assess how a company’s operations and related tax risks, tax planning and operational opportunities affect its tax rate and prospects for future cash flows. This requires public entities to disclose additional categories in the rate reconciliation regarding federal and state income taxes, and provide more details surrounding reconciling items if a quantitative threshold is met. The effective date for public companies is for annual periods starting on January 1, 2025. Early adoption is permitted for annual financial statements that have not yet been issued or made available for issuance, however, the Company has decided not to early adopt, does not anticipate a material impact to its net financial position, and is still evaluating the impact on its disclosures will be as a result of the adoption of ASU 2023-09.

In November 2024, the FASB issued ASU No. 2024-03,Income Statement - Reporting Comprehensive Income - Expense Disaggregation Disclosures (Subtopic 220-40):Disaggregation of Income Statement Expenses(“ASU 2024-03”), and in January 2025, the FASB issued ASU No. 2025-01,Income Statement - Reporting Comprehensive Income - Expense Disaggregation Disclosures (Subtopic 220-40): Clarifying the Effective Date. ASU 2024-03 requires additional disclosure of the nature of expenses included in the income statement as well as disclosures about specific types of expenses included in the expense captions presented in the income statement. ASU 2024-03, as clarified by ASU 2025-01, is effective for public companies for annual periods beginning after December 15, 2026, and interim reporting periods beginning after December 15, 2027. Early adoption is permitted. The Company is still evaluating the impact on its disclosures in future years as a result of the adoption of ASU 2024-03.

  1. Fair Value of Financial Assets and Liabilities

The following tables summarize the assets and liabilities measured at fair value on a recurring basis at March 31, 2025 and December 31, 2024 (in thousands):

​​​​​​​​​​​​​
​ · ​ · Fair Value Measurements at March 31, 2025
​ · Total · Level 1 · Level 2 · Level 3
Assets: · ​ · ​ · ​ · ​ · ​ · ​ · ​ · ​
Money market funds, included in cash and cash equivalents​$87,071​$87,071​$—​$—
U.S. treasury obligations, included in cash and cash equivalents​​31,907​​31,907​​—​​—
Marketable securities:​​​​
U.S. treasury obligations and government agency securities​​226,449​​226,449​​—​​—
Total assets​$345,427​$345,427​$—​$—
​​​​​​​​​​​​​
​ · ​ · Fair Value Measurements at December 31, 2024
​ · Total · Level 1 · Level 2 · Level 3
Assets: · ​ · ​ · ​ · ​ · ​ · ​ · ​ · ​
Money market funds, included in cash and cash equivalents​$97,290​$97,290​$—​$—
U.S. treasury obligations, included in cash and cash equivalents​​63,171​​63,171​​—​​—
Marketable securities:​​​​
U.S. treasury obligations and government agency securities​259,400​​259,400​​—​​—
Total assets​$419,861​$419,861​$—​$—

Cash, cash equivalents and marketable securities are Level 1 assets and include investments in money market funds, U.S. treasury obligations and government agency securities that are valued using quoted market prices. Accordingly, money market funds and government funds are categorized as Level 1 as of March 31, 2025 and December 31, 2024. There were no transfers of assets between fair value measurement levels during the three months ended March 31, 2025 or 2024.

The carrying amounts reflected in the balance sheets for prepaid expenses and other current assets, accounts payable, and accrued expenses approximate their fair values at March 31, 2025 and December 31, 2024, due to their short-term nature.

The Company believes the terms of its debt reflect current market conditions for an instrument with similar terms and maturity, therefore the carrying value of the Company's debt approximates its fair value based on Level 3 of the fair value hierarchy.

  1. Marketable Securities

The following table summarizes the Company’s investments as of March 31, 2025 (in thousands):

​ · ​ · ​ · ​ · ​ · ​ · ​ · ​ · ​ · ​ · ​ · ​ · ​
​​​​​Gross​​​
​ · ​ · Amortized · ​ · Unrealized · ​ · Estimated
​ · Cost · Gains · Losses · Fair Value
Marketable securities available-for-sale: · ​ · ​ · ​ · ​ · ​ · ​ · ​ · ​ · ​
U.S. treasury obligations and government agency securities​$226,320​$131​$(2)​$226,449
Total available-for-sale securities​$226,320​$131​$(2)​$226,449

The following table summarizes the Company’s investments as of December 31, 2024 (in thousands):

​ · ​ · ​ · ​ · ​ · ​ · ​ · ​ · ​ · ​ · ​ · ​ · ​
​​​​​Gross​​​
​ · ​ · Amortized · ​ · Unrealized · ​ · Estimated
​ · Cost · Gains · Losses · Fair Value
Marketable securities available-for-sale: · ​ · ​ · ​ · ​ · ​ · ​ · ​ · ​ · ​ · ​ · ​ · ​
U.S. treasury obligations and government agency securities​$259,240​$180​$(20)​$259,400
Total available-for-sale securities​$259,240​$180​$(20)​$259,400

Amortized cost approximated fair value for money market funds and U.S. treasury obligations included in cash and cash equivalents. The aggregate fair value of marketable securities with unrealized losses was $51.0 and $23.9 million at March 31, 2025 and December 31, 2024, respectively.** **At March 31, 2025 and December 31, 2024,11 investments and 12 investments, respectively, were in an unrealized loss position. All such investments have been in an unrealized loss position for less than a year and these losses are considered temporary. The Company has the ability and intent to hold these investments until a recovery of their amortized cost, which may not occur until maturity.

The Company believes that U.S. treasury obligations and government agency securities are subject to minimal credit risk. As a result, the Company did not record any charges for credit-related impairments for its available-for-sale securities for the three months ended March 31, 2025.

  1. Prepaid Expenses and Other Assets

At March 31, 2025 and December 31, 2024, prepaid expenses and other current assets consist of the following (in thousands):

​​​​​​​
​ · ​ · As of
​ · ​ · March 31, · December 31,
​ · 2025 · ​ · 2024
Prepaid external research and development expenses​$13,739​$7,716
Prepaid other​​4,239​​2,961
Receivables​​1,944​​2,151
Prepaid professional services expense​​639​​419
Prepaid insurance​​311​​640
​​$20,872​$13,887

At March 31, 2025 and December 31, 2024, other long-term assets consist of the following (in thousands):

​​​​​​​
​ · ​ · As of
​ · ​ · March 31, · December 31,
​ · 2025 · ​ · 2024
Prepaid external research and development expenses​$2,294​$2,395
Prepaid other​​553​​536
Prepaid insurance​​10​​14
​​$2,857​$2,945
  1. Accrued Expenses

At March 31, 2025 and December 31, 2024, accrued expenses consist of the following (in thousands):

​​​​​​​
​ · ​ · As of
​ · ​ · March 31, · December 31,
​ · 2025 · ​ · 2024
Accrued external research and development expense​$12,853​$12,116
Accrued payroll and related expenses​​8,386​​14,776
Accrued professional services expense​​5,652​​3,296
Accrued other​​749​​879
​​$27,640​$31,067

7. Common Stock

In June 2024, the stockholders approved an amendment to the Company’s amended and restated certificate of incorporation to increase the number of authorized shares of common stock from 150,000,000 to 300,000,000.

In October 2024, the Company entered into an underwriting agreement (the “Underwriting Agreement”) with J.P. Morgan Securities LLC, Jefferies LLC and Piper Sandler & Co., as representatives of the several underwriters named therein (the “Underwriters”), relating to the issuance and sale of an aggregate of 10,265,488 shares of the Company’s common stock at$28.25 per share and pre-funded warrants to purchase 353,983 shares of its common stock. The offering price per pre-funded warrant was$28.2499, which equals the per share public offering price for the common shares less the$0.0001 exercise price for each such pre-funded warrant. The pre-funded warrants are exercisable at any time and only expire when exercised in full. The offering closed on October 10, 2024. Pursuant to the Underwriting Agreement, the Underwriters were granted a 30-day option to purchase up to 1,592,920 additional shares (the “Option Shares”) of common stock, which was exercised in full on October 16, 2024. Total proceeds of the transaction, including the Option Shares were approximately$324.4 million, net of underwriting discounts and estimated offering expenses.

The Company has had a sales agreement in place during various time periods with Jefferies LLC (“Jefferies”) with respect to an at-the-market (“ATM”) offering program. Under this program, the Company is able to offer and sell, from time to time at its sole discretion, shares of its common stock through Jefferies as its sales agent. In an ATM offering, exchange-listed companies incrementally sell newly issued shares into the secondary trading market through a designated broker-dealer at prevailing market prices. The current ATM agreement, established in November 2022, allows for the sale of shares of common stock having an aggregate offering price of up to$100 million. As of March 31, 2025, the Company has sold 619,290 shares, generating net proceeds of$5.2 million, under the ATM program. No sales were made under the ATM program during the three months ended March 31, 2025.

The Company has issued pre-funded warrants to purchase common stock, as well as warrants to purchase common stock as part of its financing activities. Both the pre-funded warrants and warrants meet the conditions for equity classification and are recorded as a component of stockholders’ equity within additional paid-in capital. In October 2024, June 2022 and November 2020, the Company issued 353,983,25,510,205 and 2,179,487 pre-funded warrants, respectively. During the three months ended March 31, 2025 and 2024,0 and 2,526,833, respectively, of the Company’s pre-funded warrants were exercised. As of March 31, 2025, the Company has 17,362,147 pre-funded warrants outstanding. In June 2022, the Company also issued 10,459,181 warrants with an exercise price of$7.35. During the three months ended March 31, 2025 and 2024,0 and 830,660, respectively, of the Company’s warrants were exercised. As of March 31, 2025, the Company has 8,678,664 warrants outstanding.

  1. Equity-Based Compensation

The Company recorded equity-based compensation expense related to all equity-based awards, which was allocated as follows in the consolidated statements of operations and comprehensive loss for the three months ended March 31, 2025 and 2024 (in thousands):

​ · ​ · ​ · ​ · ​ · ​ · ​
​ · ​ · Three Months Ended
​ · ​ · March 31,
​ · 2025 · 2024
Research and development expense​$4,035​$3,531
General and administrative expense​9,378​4,633
​​$13,413​$8,164

Equity-based compensation during the three months ended March 31, 2025 includes $4.0 million related to the modification of certain equity awards.

The following table summarizes the Company’s unrecognized equity-based compensation expense as of March 31, 2025:

​​​​​
​​
​As of March 31, 2025
​ · ​ · Unrecognized Expense (in thousands) · Weighted Average Remaining Period of Recognition (years)
RSUs$63,427​2.7
Stock options​71,527​2.6
​$134,954​​

Restricted Stock Units

The following table summarizes the Company’s RSU activity for the current year:

​ · ​ · ​ · ​ · ​ · ​
​ · ​ · ​ · ​ · Weighted
​ · ​ · ​ · ​ · Average Grant
​ · Number of Units · Date Fair Value
RSUs as of December 31, 20243,486,668​$13.86
Granted942,116​$35.82
Vested(673,790)​$16.30
Forfeited(53,307)​$18.25
RSUs as of March 31, 20253,701,687​$18.94

The total fair value of RSUs vested during the three months ended March 31, 2025 was $28.3 million.

Stock Options

The following table summarizes the Company’s stock option activity for the current year:

​ · ​ · ​ · ​ · ​ · ​ · ​ · ​ · ​ · ​ · ​
​ · ​ · ​ · ​ · ​ · ​ · ​ · Weighted · ​ · ​ · ​
​​​​Weighted​Average​​​
​ · ​ · Number of · ​ · Average · ​ · Remaining · ​ · Aggregate
​ · Shares · Exercise Price · Contractual Term · Intrinsic Value
​​​​​​​(in years)​(in thousands)
Outstanding as of December 31, 20248,469,926​$15.19​7.83​$244,677
Granted1,256,169​$35.82​​​​​
Exercised​(375,939)​$13.40​​​​​
Cancelled(62,579)​$19.68​​​​​
Outstanding as of March 31, 20259,287,577​$18.02​7.86​$149,539
Options exercisable as of March 31, 20254,023,096​$17.43​6.61​$71,394

Using the Black-Scholes option pricing model, the weighted average fair value of options granted during the three months ended March 31, 2025 was $30.02.

The following weighted average assumptions were used in determining the fair value of options granted in the three months ended March 31, 2025 and 2024:

​​​​​
​ · Three Months Ended · ​
​ · March 31, · ​
​ · 2025 · 2024 · ​
Risk-free interest rate4.11%4.14%
Expected dividend yield0.0%0.0%
Expected term (years to liquidity)6.00​6.02​
Expected volatility108.68%91.39%
  1. Commitments and Contingencies

Operating Lease

In November 2019, the Company entered into a lease of office and laboratory space at 301 Binney Street in Cambridge, Massachusetts to be used as its new corporate headquarters (the “Lease”). The expiration date of the Lease was originally in August 2025 and included anoption to extend the term bytwo years. The base rent under the original Lease was $6.9 million per year, subject to an annual increase of 3.5%. Variable lease payments include the Company’s allocated share of costs incurred and expenditures made by the landlord in the operation and management of the building. The Lease included incentives of $14.1 million in the form of an allowance for tenant improvements related to the design and build out of the space. In connection with the Lease, the Company has secured a letter of credit for $2.3 million which renews automatically each year.

In May 2024, the Company entered into the First Amendment (the “Lease Amendment”) to the Lease to extend the term for approximatelytwo years, commencing on August 19, 2025 (the “First Extension Term”) with the base rent to start at approximately $6.2 million per year, followed by a 3% annual increase. Pursuant to the Lease Amendment, the Company also has anoption to extend the term of the Lease byfive years, upon the expiration of the First Extension Term.

Other information related to the Lease is as follows (in thousands, except lease term and discount rate):

​​​​​
​ · ​ · For Three Months Ended · ​
​ · March 31, · ​
​ · ​ · 2025 · ​
Lease cost:​​​​
Operating lease cost​$1,714​
Variable lease cost​​392​
Total lease cost​$2,106​
​​​​
​ · For Three Months Ended · ​
​ · March 31, · ​
​ · 2025 · ​
Other information:​​​
Operating cash flows used for operating leases$2,051​
Weighted average remaining lease term​2.4​
Weighted average incremental borrowing rate​13.1%

Legal Proceedings

The Company, from time to time, may be party to litigation arising in the ordinary course of its business. The Company was not subject to any material legal proceedings during the three months ended March 31, 2025 and 2024.

  1. Debt

On October 16, 2020 (the “Closing Date”) the Company entered into a Loan and Security Agreement with Oxford Finance LLC (“Oxford”) and Silicon Valley Bank (“SVB”) for $50.0 million (the “Loan and Security Agreement”). Tranche 1 of $25.0 million was funded on the Closing Date. The Company had an additional $25.0 million in loan proceeds available under Tranche 2 which was funded in December 2021, in conjunction with the Company entering into the First Amendment to Loan and Security Agreement with Oxford and SVB. The Loan and Security Agreement was to mature on May 1, 2025 and required interest-only payments through November 2022, with principal payments to commence in December 2022. Pursuant to the Loan and Security Agreement, the Company was required to maintain cash in an SVB account equal to the lesser of 100% of the Company’s consolidated cash or 105% of the dollar amount of the outstanding debt.

On November 10, 2022, the Company entered into the Second Amendment to the Loan and Security Agreement (“Amendment 2”) to increase the Company’s borrowing capacity under the Loan and Security Agreement to an amount up to $100.0 million, comprised of the original $50.0 million loan which remains outstanding and two additional $25.0 million tranches. The first $25.0 million tranche available under Amendment 2, was available at the Company’s discretion through December 2023 upon achievement of certain development and business performance milestones. The Company did not exercise this tranche. The second $25.0 million tranche available under Amendment 2, may be available upon the Company’s request, at Oxford and SVB’s discretion. Amendment 2 also extended the interest-only payment period for an additional 24 months through November 2024, with principal payments to commence in December 2024. The maturity of the loan was extended to November 2027.

Effective upon Amendment 2, the interest rate on the unpaid principal is the greater of the Wall Street Journal prime rate plus 4.60% or 9.35% per annum. Prepayment is permitted and may include a pre-payment fee ranging from 0% -3% (of the principal amount being prepaid), depending on when the prepayment is made. The Company is also required to make a final payment equal to 2% of the original principal amount.

In conjunction with Amendment 2, the Company was required to pay $0.9 million for the accrued portion of the final payment on the previous outstanding balance.

On March 10, 2023, SVB was closed by the California Department of Financial Protection and Innovation, which appointed the Federal Deposit Insurance Corporation (“FDIC”) as receiver. Afterward, the FDIC transferred all deposits

of the former Silicon Valley Bank to Silicon Valley Bridge Bank, N.A., as operated by the FDIC. On March 27, 2023, Silicon Valley Bridge Bank was closed by the Office of the Comptroller of the Currency, and the FDIC was appointed as receiver. First Citizens Bank then entered into an agreement with the FDIC to purchase out of FDIC receivership substantially all loans and certain other assets and assume all customer deposits and certain other liabilities of Silicon Valley Bridge Bank. On March 27, 2023, Silicon Valley Bridge Bank and its U.S. branches began operating as Silicon Valley Bank, a division of First Citizens Bank.

On April 18, 2023, the Company entered into Amendment 3 to the Loan and Security Agreement to amend certain provisions relating to the Company’s operating accounts.

On May 17, 2024, the Company entered into the Fourth Amendment to the Loan and Security Agreement (“Amendment 4”). The Company currently has $50.0 million outstanding under the Loan and Security Agreement, which was drawn down in two equal tranches. The third $25.0 million tranche is available at the Company’s discretion through December 2024, upon achievement of certain clinical and business milestones, which were amended under Amendment 4. The fourth $25.0 million tranche may be available through June 1, 2025 (or through December 1, 2025, upon achievement of certain business milestones) upon the Company’s request, at Oxford and SVB’s discretion. Amendment 4 also extends the interest-only payment period for an additional six months through May 2025, with principal payments to commence in June 2025. Additionally, upon achievement of certain business performance milestones, which were achieved in October 2024, the interest-only payment period was extended through November 2025, with principal payments to commence in December 2025.

On February 10, 2025, the Company entered into an Amended and Restated Loan and Security Agreement (the “Amended and Restated Loan and Security Agreement”) with Oxford. The Amended and Restated Loan and Security Agreement amends and restates in its entirety that certain Loan and Security Agreement, as amended.

The Amended and Restated Loan and Security Agreement provides for term loans in an aggregate principal amount of up to $200.0 million (each, a “Term Loan” and together, the “Term Loans”) subject to funding in four tranches. To date, the Company has received $50.0 million of Term Loans. The remaining three tranches, each in an amount of $50.0 million, are available to be borrowed (a) in the case of the second tranche, until December 31, 2025, (b) in the case of the third tranche, after the achievement of certain development and business performance milestones until September 30, 2026, and (c) in the case of the fourth tranche, after the achievement of certain development and business performance milestones until December 31, 2027. The Amended and Restated Loan and Security Agreement consolidates the existing outstanding loan tranches solely with Oxford and also extends the interest-only payment period through March 2029, with principal payments to commence in April 2029. In conjunction with the Amended and Restated Loan and Security Agreement, the Company was required to pay$0.9 million, which includes$0.5 million to SVB for the final payment on the outstanding tranche.

The outstanding principal of each Term Loan has an annual interest rate of (a) the greater of (i) the 1-Month CME Term SOFR on the last business day of the month that immediately precedes the month in which the interest will accrue and (ii)3%, plus (b)5.5%. Interest is payable on a monthly basis based on the principal amount outstanding during the preceding month. In addition, the Company is required to pay Oxford a final payment fee equal to 2.00% of the original principal amount of each Term Loan advanced to the Company.

  1. Net Loss per Share

The Company calculates basic net loss per share by dividing net loss by the weighted average number of common shares outstanding, excluding restricted common stock. The weighted average number of common shares used in the basic and diluted net loss per share calculation includes the pre-funded warrants issued in connection with the Company’s November 2020, June 2022 and October 2024 follow-on offerings as the pre-funded warrants are exercisable at any time for nominal cash consideration. As of March 31, 2025,10,681,528 pre-funded warrants have been exercised and 17,362,147 pre-funded warrants remain outstanding. The Company has generated a net loss in all periods presented, so the basic and diluted net loss per share are the same, as the inclusion of the potentially dilutive securities would be anti-dilutive.

The following table sets forth the outstanding common stock equivalents, presented based on amounts outstanding at each period end, that have been excluded from the calculation of diluted net loss per share for the periods indicated because their inclusion would have been anti-dilutive:

​​​​​
​ · ​ · Three Months Ended March 31,
​ · 2025 · 2024
RSUs​3,701,687​3,089,689
Stock options​9,287,577​9,048,138
Warrants​8,678,664​9,157,496
​​21,667,928​21,295,323

12. Segment Reporting

The Company operates and manages its business as asingle segment for the purposes of assessing performance and making operating decisions. The Company’s president and chief executive officer, who is the chief operating decision maker (“CODM”), reviews the Company’s financial information on a consolidated basis for purposes of evaluating financial performance and allocating resources and therefore we have presented segment information on the same basis. When evaluating the Company’s financial performance, the CODM regularly reviews net loss, non-operating expenses and operating expenses with non-cash expenses such as depreciation and equity-based compensation expense removed. The CODM considers net loss in making decisions on how to allocate resources. The measure of segment assets is reported on the balance sheet as total consolidated assets. All of the Company's long-lived assets are held in the United States.

The following table presents significant expense information about the Company’s operating segment:

​​​​​​​
​ · ​ · ​ · Three Months Ended March 31,
​ · ​ · 2025 · ​ · 2024
Operating expenses:​​​​​​
Employee related expense​$22,938​$16,080
External R&D expense - SMA​​17,074​​16,201
External R&D expense - oncology​​1,110​​2,747
External R&D expense - cardiometabolic/obesity​​6,348​​4,032
External R&D expense - Early research and other​​1,614​​741
External expense - G&A​​8,942​​5,001
Other segment related expense*​​5,258​​4,857
​​​​​​​
Employee related equity-based compensation expense​​13,413​​8,164
Depreciation and amortization expense​​393​​596
​​​​​​​
Interest income​​(4,229)​​(3,319)
Interest expense​​1,818​​1,732
Other non-operating expense/(income), net​​44​​21
​​​​​​​
Net loss​$74,723​$56,853
​​​​​​​

*Consists of other segment expenses related to supplies, corporate and facilities expenses.

13. Subsequent Events

Effective April 27, 2025, the Board appointed David Hallal as Chief Executive Officer, Akshay Vaishnaw as President of Research and Development, R. Keith Woods as Chief Operating Officer and Vikas Sinha as Chief Financial Officer, following Jay T. Backstrom’s transition from his role as the Company’s Chief Executive Officer and President to Senior Advisor. In connection with his appointment, Mr. Hallal was granted a stock option to purchase 275,167 shares of the Company’s common stock,250,000 RSUs and a target number of 500,000 performance stock units (“PSUs”) pursuant to the Company’s 2018 Stock Option and Incentive Plan (“2018 Plan”). Dr. Vaishnaw and Messrs. Woods and Sinha were each granted (i) a stock option to purchase 110,067 shares of the Company's common stock,100,000 RSUs and a target number of 200,000 PSUs, pursuant to the Company’s 2018 Plan in the case of Dr. Vaishnaw and pursuant to the Company’s 2022 Inducement Equity Plan in the case of each of Messrs. Woods and Sinha.

ContentsView on SEC.gov

The text of this filing, as the company filed it with the SEC, without its images or exhibits. This is not investment advice.