What changed in this filing

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Item 2. Management’s Discussion and Analysis

2025-05-02 compared with 2024-11-06 · 73 added, 124 removed, 201 unchanged (49% of the section changed)

Management’s Discussion and Analysis of Financial Condition and Results of Operations.

You should read the following discussion and analysis of our financial condition and results of operations together with our condensed consolidated financial statements and related notes appearing elsewhere in this Quarterly Report on Form 10-Q and the audited financial information and the notes thereto included in our Annual Report on Form 10-K, which was filed with the Securities and Exchange Commission (the "SEC") on March 5, 2024.

Some of the information contained in this discussion and analysis or set forth elsewhere in this Quarterly Report on Form 10-Q, including information with respect to our plans and strategy for our business and related financing, includes forward-looking statements that involve risks and uncertainties.

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Our platforms utilize a deliberate, pragmatic and patient-guided approach, leveraging a suite of translational tools, including novel transgenic and predictive translational animal models and electrophysiology markers, to enable an efficient path to proof-of-concept in patients.

Through this approach, we have established a diversified, multimodal CNS portfolio with four clinical-stage product candidates across movement disorders and epilepsy.

For our most advanced product candidate under the Cerebrum™ platform, ulixacaltamide, our Phase 3 Essential3 clinical trials in essential tremor, or ET, are ongoing.

We are planning to conduct an interim analysis in the first quarter of 2025.

Within our vormatrigine program (formerly known as PRAX-628), we announced positive results from our Photo-Paroxysmal Response ("PPR") study in the first quarter of 2024, and have initiated or plan to initiate four studies to generate patient eligibility, efficacy, safety and pharmacokinetics (PK) data for the program.

We initiated an observational study of vormatrigine in patients with epilepsy in the third quarter of 2024, and iniatiated or plan to initiate three efficacy studies.

The first efficacy study will be an open label eight-week study in patients with focal onset seizures or generalized epilepsy to be initiated in the fourth quarter of 2024, with topline results expected in the first half of 2025.

We have also initiated a double-blind, placebo-controlled, 12-week study in focal onset seizures in the fourth quarter of 2024, with topline results expected in the second half of 2025, and plan to initiate a third efficacy study in the first half of 2025.

Within our relutrigine program (formerly known as PRAX-562), we announced positive topline results from the first cohort of our EMBOLD study in the third quarter of 2024, and have initiated enrollment of the second cohort, with topline results expected in the first half of 2026.

For our most advanced product candidate under the Solidus™ platform, elsunersen (formerly known as PRAX-222), we shared results from Part 1 of the EMBRAVE study in the fourth quarter of 2023, and have initiated the first arm of our global confirmatory study in Brazil.

We are currently completing multiple global regulatory interactions to further expand the pivotal phase of the program later in 2024.

Through this approach, we have established a diversified, multimodal CNS portfolio with four clinical-stage product candidates across epilepsy and movement disorders.

For our vormatrigine program (formerly known as PRAX-628) within the Cerebrum™ platform, we announced positive results from our Photo-Paroxysmal Response, or PPR, study in the first quarter of 2024, and have initiated or plan to initiate four studies, called the ENERGY program, to generate patient eligibility, efficacy, safety and pharmacokinetics (PK) data for the program.

We initiated the EMPOWER study, an observational study of vormatrigine in patients with epilepsy, in the third quarter of 2024, and initiated or plan to initiate three efficacy studies.

The first efficacy study, RADIANT, is an open label eight-week study in patients with focal onset seizures or generalized epilepsy that is currently enrolling, with topline results expected by mid-year 2025.

We have also initiated the POWER1 study, a double-blind, placebo-controlled, 12-week study in focal onset seizures, with topline results expected in the second half of 2025, and plan to begin enrollment in the POWER2 study, a third efficacy study, in the second half of 2025.

Within our relutrigine program (formerly known as PRAX-562), we announced positive topline results from the first cohort of our EMBOLD study in the third quarter of 2024 and have initiated enrollment of the second cohort, with topline results expected no later than the first half of 2026.

We also plan to initiate the EMERALD study in a broader developmental and epileptic encephalopathies, or DEE, patient population in mid-2025.

For our ulixacaltamide program, we shared the results of a pre-planned statistic interim analysis of Study 1 of our Phase 3 Essential 3 clinical program for essential tremor in February 2025.

Based on the predefined decision framework for Study 1, the Independent Data Monitoring Committee, or IDMC, recommended that the study be stopped for futility, due to the results being unlikely to meet the primary efficacy endpoint under the parameters set by the statistical model.

The committee also indicated that some underlying assumptions of the statistical model might have influenced this outcome and encouraged us to explore alternative analysis methods.

Given the advanced state of enrollment for both Study 1 and Study 2 in the Essential3 program, and in the context of the advice received by the IDMC, we have decided to continue both studies to completion, with topline results expected in the third quarter of 2025.

The decision about whether the data supports the submission of an NDA will be made after analyzing the final results for Study 1 and Study 2.

For our most advanced product candidate under the Solidus™ platform, elsunersen (formerly known as PRAX-222), we shared results from Part 1 of the EMBRAVE study in the fourth quarter of 2023.

We are currently enrolling the second cohort of the EMBRAVE study in Brazil, with topline results expected in the first half of 2026, and plan to initiate EMBRAVE3, a Phase 3 registrational study, in mid-2025.

We also have three earlier stage novel ASOs with preclinical proof of mechanism.

PRAX-080 is focused on PCDH19 mosaic expression, PRAX-090 is targeting SYNGAP1 loss-of-function, or LoF, mutations which is a leading cause of severe intellectual disability and epilepsy in DEEs, and PRAX-100 is targeting SCN2A LoF mutations, the predominant generic link to de novo autism spectrum disorders, or ASD.

We anticipate nominating a development candidate for PRAX-100 by mid- 2025, and development candidates for PRAX-080 and PRAX-090 by the end of 2025.

We were incorporated in 2015 and commenced operations in 2016.

Since inception, we have devoted substantially all of our resources to developing our preclinical and clinical product candidates, building our intellectual property, or IP, portfolio, business planning, raising capital and providing general and administrative support for these operations.

We employ a “virtual” research and development model, relying heavily upon external consultants, collaborators, contract development and manufacturing organizations and contract research

organizations, or CROs, to conduct our preclinical and clinical activities.

We employ a “virtual” research and development model, relying heavily upon external consultants, collaborators, contract development and manufacturing organizations and contract research organizations, or CROs, to conduct our preclinical and clinical activities.

Since inception, we have financed our operations primarily with proceeds from the sale and issuance of equity securities.

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Our ability to generate product revenue sufficient to achieve profitability will depend heavily on the successful development and eventual commercialization of one or more of our product candidates, if approved.

We have incurred recurring operating losses since inception, including a net loss of $124.1 million for the nine months ended September 30, 2024.

As of September 30, 2024, we had an accumulated deficit of $778.1 million.

We have incurred recurring operating losses since inception, including a net loss of $69.3 million for the three months ended March 31, 2025.

As of March 31, 2025, we had an accumulated deficit of $906.0 million.

We expect to incur significant expenses and operating losses for the foreseeable future as we expand our research and development activities.

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We anticipate that our expenses will be maintained or increased in connection with our ongoing activities, as we:

• advance our lead product candidate, ulixacaltamide, through the Phase 3 Essential3 clinical trial program for ET;

• advance relutrigine (formally PRAX-562) in the EMBOLD and EMERALD clinical trials;

• advance elsunersen (formerly PRAX-222) into the pivotal stage of the program;

• advance vormatrigine (formerly PRAX-628) into efficacy clinical trials for focal onset seizures or generalized epilepsy;

• advance relutrigine (formally PRAX-562) in the EMBOLD clinical trial;

• advance elsunersen (formerly PRAX-222) into the registrational stage of the program;

• advance vormatrigine (formerly PRAX-628) into efficacy clinical trials for focal onset seizures;

• advance ulixacaltamide through the Phase 3 Essential3 clinical trial program for ET;

• advance our preclinical candidates to clinical trials;

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We may be unable to raise additional funds or enter into such other agreements or arrangements when needed on favorable terms, or at all.

If we fail to raise capital or enter into such agreements as and when needed, we may have to significantly delay, scale back or discontinue the development and commercialization of one or more of our product candidates or delay our pursuit of potential in-licenses or acquisitions.

If we fail to raise capital or enter into such agreements as and when needed, we may have to significantly delay, scale back or

discontinue the development and commercialization of one or more of our product candidates or delay our pursuit of potential in-licenses or acquisitions.

Because of the numerous risks and uncertainties associated with product development, we are unable to predict the timing or amount of increased expenses or when or if we will be able to achieve or maintain profitability.

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If we fail to become profitable or are unable to sustain profitability on a continuing basis, then we may be unable to continue our operations at planned levels and be forced to reduce or terminate our operations.

As of September 30, 2024, we had cash, cash equivalents and marketable securities of $411.2 million.

We expect that our cash, cash equivalents and marketable securities as of September 30, 2024 will be sufficient to fund our operating expenditures and capital expenditure requirements necessary to advance our research efforts and clinical trials into 2027.

The analysis included consideration of our current financial needs and ongoing research and

development plans.

As of March 31, 2025, we had cash, cash equivalents and marketable securities of $472.0 million.

We expect that our cash, cash equivalents, and marketable securities as of March 31, 2025 will be sufficient to fund our operating expenditures and capital expenditure requirements necessary to advance our research efforts and clinical trials into 2028.

The analysis included consideration of our current financial needs and ongoing research and development plans.

We have based this estimate on assumptions that may provide to be wrong, and we could exhaust our available capital resources sooner than we expect.

See “—Liquidity and Capital Resources.”

Reverse Stock Split

On November 28, 2023, we filed a Certificate of Amendment to our Amended and Restated Certificate of Incorporation with the Secretary of State of the State of Delaware to effect a 1-for-15 reverse stock split of our common stock, or the Reverse Stock Split.

The Reverse Stock Split became effective at 5:00 p.m., Eastern Time, on November 28, 2023, or the Effective Time.

As a result of the Reverse Stock Split, every 15 shares of our issued and outstanding common stock were automatically reclassified into one validly issued, fully-paid and non-assessable share of common stock, subject to the treatment of fractional shares as described below, without any action on the part of the holders thereof.

The Reverse Stock Split did not affect the number of authorized shares of common stock or the par value of the common stock.

No fractional shares were issued in connection with the Reverse Stock Split.

Stockholders who would otherwise be entitled to receive fractional shares as a result of the Reverse Stock Split were entitled to a cash payment in lieu thereof at a price equal to the fraction to which the stockholder would otherwise be entitled, multiplied by the closing price per share of the common stock (as adjusted for the Reverse Stock Split) on the Nasdaq Global Select Market on November 28, 2023, the last trading day immediately preceding the Effective Time.

Financial Operations Overview

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As discussed in Note 7 to our condensed consolidated financial statements, we entered into an Option and License Agreement, or the Collaboration Agreement, with UCB Biopharma SRL, or UCB, in December 2022.

We recognized $0.3 million and $1.1 million, respectively, of collaboration revenue from the Collaboration Agreement during the three and nine months ended September 30, 2024, compared to $0.5 million and $1.9 million, respectively, during the three and nine months ended September 30, 2023.

During the three months ended March 31, 2024, we recognized $0.4 million of collaboration revenue.

In December 2024, UCB exercised its option to in-license global development and commercialization rights under the terms of the Collaboration Agreement.

Accordingly, we did not recognize any collaboration revenue from the Collaboration Agreement during the three months ended March 31, 2025.

We have no further research service obligations under the terms of the Collaboration Agreement.

Operating Expenses

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Research and development expenses represent costs incurred by us for the following:

• costs to develop our portfolio;

• discovery efforts leading to development candidates;

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We expense research and development costs as incurred.

We recognize external development costs based on an evaluation of the progress to completion of specific tasks using information provided to us by our vendors and

our clinical investigative sites.

Payments for these activities are based on the terms of the individual agreements, which may differ from the pattern of costs incurred, and are reflected in our condensed consolidated balance sheets as prepaid expenses or accrued expenses.

We recognize external development costs based on an evaluation of the progress to completion of specific tasks using information provided to us by our vendors and our clinical investigative sites.

Payments for these activities are based on the terms of the individual agreements,

which may differ from the pattern of costs incurred, and are reflected in our condensed consolidated balance sheets as prepaid expenses or accrued expenses.

Non-refundable advance payments for goods or services to be received in the future for use in research and development activities are deferred and capitalized, even when there is no alternative future use for the research and development.

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Three Months Ended

September 30, Nine Months Ended

September 30,

$ 42,832 $ 12,911

$ 27,474 $ 5,832 $ 55,226 $ 23,857

1,327 2,806 4,197 16,330

Personnel-related (including stock-based compensation) 12,851 10,925

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Our income tax provision may be significantly affected by changes to our estimates.

There was no income tax provision recognized for the three and nine months ended September 30, 2024 and 2023.

There was no income tax provision recognized for the three months ended March 31, 2025 and 2024.

Results of Operations

Comparison of the Three Months Ended September 30, 2024 and 2023

Comparison of the Three Months Ended March 31, 2025 and 2024

The following table summarizes our condensed consolidated statements of operations for each period presented (in thousands):

Three Months Ended

September 30, Change

March 31, Change

Collaboration revenue $ — $ 431 $ (431)

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The $0.4 million decrease in collaboration revenue is associated with a decrease in the revenue recorded under the Collaboration Agreement with UCB that was executed in December 2022.

Changes in revenue recognized each period reflect timing of research services provided and costs incurred.

In December 2024, UCB exercised its option to in-license global development and commercialization rights for a development candidate as part of the Collaboration Agreement.

As such, we have no further research service obligations under the terms of the Collaboration Agreement and did not record any associated revenue during the three months ended March 31, 2025.

Research and Development Expense

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Three Months Ended

September 30, Change

$ 27,474 $ 5,832 $ 21,642

1,327 2,806 (1,479)

Personnel-related (including stock-based compensation) 10,863 6,840 4,023

Other indirect research and development expenses 2,217 1,782 435

Total research and development expenses $ 41,881 $ 17,260 $ 24,621

The $24.6 million increase in research and development expenses was primarily attributable to the following:

• $21.6 million increase in expense related to our Cerebrum™ platform, driven primarily by:

◦ a $15.7 million increase in spend for our ulixacaltamide program, primarily due to Essential3 study spend, as well as Phase 1 trial spend, partially offset by completion of our Essential1 study in the prior year;

◦ a $5.4 million increase in spend for our vormatrigine program, primarily related to current quarter POWER1, RADIANT and EMPOWER study startup activities, Phase 1 trial spend and manufacturing spend;

◦ a $0.5 million increase in spend for our relutrigine program, primarily related to the EMBOLD Phase 2 clinical trial.

• $4.0 million increase in personnel-related costs, including stock-based compensation expense, due to increased headcount;

• $0.4 million increase in indirect expenses driven primarily by consulting spend;

• $1.5 million decrease in expense related to our Solidus™ platform, driven by prior year spend associated with our elsunersen program.

General and Administrative Expense

The $6.5 million increase in general and administrative expenses was primarily attributable to the following:

• $4.6 million increase in personnel-related costs, mainly due to increased stock-based compensation expense;

• $1.4 million increase in professional expenses mainly due to increased legal, audit and other consulting expenses;

• $0.5 million increase in other expenses, none of which were individually significant.

Other income for the three months ended September 30, 2024 was comprised of interest income on our cash, cash equivalents, and marketable securities and investment premium and discount amortization.

Other income for the three months ended September 30, 2023 was compromised of interest income on our cash and cash equivalents.

Results of Operations

Comparison of the Nine Months Ended September 30, 2024 and 2023

The following table summarizes our condensed consolidated statements of operations for each period presented (in thousands):

Nine Months Ended

September 30, Change

Collaboration revenue $ 1,090 $ 1,932 $ (842)

Operating expenses:

Research and development 96,125 68,378 27,747

General and administrative 41,174 32,121 9,053

Total operating expenses 137,299 100,499 36,800

Loss from operations (136,209) (98,567) (37,642)

Other income:

Other income, net 12,069 2,168 9,901

Total other income 12,069 2,168 9,901

Net loss $ (124,140) $ (96,399) $ (27,741)

Collaboration Revenue

The $0.8 million decrease in collaboration revenue is associated with a decrease in the revenue recorded under the Collaboration Agreement with UCB that was executed in December 2022.

Changes in revenue recognized each period reflect timing of research services provided and costs incurred.

Research and Development Expense

The following table summarizes our research and development expenses for each period presented, along with the changes in those items (in thousands):

Nine Months Ended

September 30, Change

March 31, Change

$ 42,832 $ 12,911 $ 29,921

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• $29.9 million increase in expense related to our Cerebrum™ platform, driven primarily by:

◦ a $29.5 million increase in spend for our ulixacaltamide program, primarily due to clinical-related activities for our Phase 3 program in ulixacaltamide and Phase 1 trial spend, partially offset by prior year Essential1 study activity;

◦ a $3.2 million increase in clinical-related spend for our vormatrigine program, primarily driven by current quarter POWER1, RADIANT, and EMPOWER study startup activities and manufacturing spend;

◦ a $0.3 million increase in spend for our relutrigine program related to our EMBOLD study;

◦ a $1.6 million decrease in activities for our earlier stage assets.

• $12.1 million decrease in expense related to our Solidus™ platform, driven by a prior year $6.9 million milestone payment due to Ionis Pharmaceuticals Inc., or Ionis, upon initiation of our elsunersen EMBRAVE study, as well as prior year study activity;

• $8.5 million increase in personnel-related costs, primarily stock-based compensation expense.

◦ a $13.0 million increase in spend for our vormatrigine program, primarily driven by spend related to the ENERGY program;

◦ a $12.3 million increase in spend for our ulixacaltamide program, primarily due to increased Essential3 activity compared to prior year;

◦ a $4.3 million increase in spend for our relutragine program, primarily related to EMBOLD clinical trial costs and current quarter manufacturing spend.

• $1.9 million increase in personnel-related costs, primarily due to increased headcount;

• $1.9 million increase in indirect expenses driven primarily by software and consulting spend to support operations.

General and Administrative Expense

The $9.1 million increase in general and administrative expenses was primarily attributable to the following:

• $8.3 million increase in personnel-related costs mainly due to increased stock-based compensation expense;

• $0.3 million increase in professional fees, primarily audit fees;

• $0.5 million increase in other expenses, none of which were individually significant.

Other income for the nine months ended September 30, 2024 and 2023 was comprised of interest income on our cash, cash equivalents, and marketable securities and investment premium and discount amortization.

The $1.4 million decrease in general and administrative expenses was primarily attributable to the following:

• $2.8 million decrease in personnel-related costs mainly due to decreased stock-based compensation expense;

• $1.4 million increase in indirect expenses, primarily consulting expenses to support operations.

Other income for the three months ended March 31, 2025 and 2024 was comprised of interest income on our cash, cash equivalents, and marketable securities and investment premium and discount amortization.

Liquidity and Capital Resources

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To date, we have financed our operations primarily with proceeds from the issuance of redeemable convertible preferred stock and from the sale of common stock through an initial public offering, common stock and pre-funded warrants through follow-on public offerings and common stock from at-the-market offerings under our shelf registration statement.

From inception through September 30, 2024, we have raised $1.0 billion in aggregate cash proceeds from such transactions, net of issuance costs.

As of September 30, 2024, we had cash, cash equivalents and marketable securities of $411.2 million.

In November 2021, we entered into an Open Market Sale Agreement, or the 2021 Sales Agreement, with Jefferies LLC, or Jefferies, to provide for the offering, issuance and sale of up to an aggregate amount of $125.0 million of common stock from time to time in at-the-market offerings for which Jefferies acted as sales agent.

We terminated the 2021 Sales Agreement in June 2023.

On June 21, 2023, we completed a public offering of:

(i) an aggregate of 4,296,646 shares of common stock at a public offering price of $14.25 per share, including the underwriters' full exercise of their option to purchase 619,979 additional shares of common stock, and (ii) pre-funded warrants to purchase 470,000 shares of common stock at a public offering price of $14.2485 per share.

The purchase prices per share for each pre-funded warrant represents the per share offering price for the common stock, less the $0.0015 per share exercise price for each underlying share.

Total net proceeds generated from the offering were approximately $63.4 million, after deducting

underwriting discounts, commissions and other offering expenses payable by us.

As of June 30, 2024, all warrants associated with this offering were exercised on a cashless basis with no proceeds received by the Company.

In December 2023, we entered into an Open Market Sale Agreement, or the 2023 Sales Agreement, with Jefferies, to provide for the offering, issuance and sale of up to an aggregate amount of $75.0 million of common stock in at-the-market offerings.

From inception through March 31, 2025, we have raised $1.2 billion in aggregate cash proceeds from such transactions, net of issuance costs.

As of March 31, 2025, we had cash, cash equivalents, and marketable securities of $472.0 million.

The 2023 Sales Agreement was terminated in January 2024.

During the three months ended March 31, 2024, we issued and sold an aggregate of 192,190 shares under the 2023 Sales Agreement for aggregate net proceeds of $5.3 million, after deducting commissions and offering expenses payable by us.

On January 16, 2024, we completed a public offering of:

In January 2024, we completed a public offering of:

(i) an aggregate of 3,802,025 shares of our common stock at a public offering price of $35.50 per share, including the underwriters' full exercise of their option to purchase 633,750 additional shares of common stock, and (ii) pre-funded warrants to purchase 1,056,725 shares of common stock at a public offering price of $35.4999 per share of common stock underlying the warrants.

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Total net proceeds generated from the offering were approximately $161.6 million, after deducting underwriting discounts, commissions and other offering expenses payable by us.

As of September 30, 2024, 152,145 warrants associated with this offering were exercised on a cashless basis with no cash proceeds received by the Company.

As of March 31, 2025, 325,988 warrants associated with this offering were exercised on a cashless basis with no cash proceeds received by us.

In March 2024, we entered into an Open Market Sale Agreement, or the 2024 Sales Agreement, with Jefferies, to provide for the offering, issuance, and sale of up to an aggregate amount of $150.0 million of common stock in at-the-market offerings.

During the nine months ended September 30, 2024, we issued and sold an aggregate of 42,851 shares under the 2024 Sales Agreement for aggregate net proceeds of $2.3 million, after deducting commissions and offering expenses payable by us.

On April 2, 2024, we completed a public offering of:

During the three months ended March 31, 2024, we issued and sold an aggregate of 17,662 shares under the 2024 Sales Agreement for aggregate net proceeds of $0.9 million, after deducting commissions and offering expenses payable by us.

In April 2024, we completed a public offering of:

(i) an aggregate of 3,849,558 shares of our common stock at a public offering price of $56.50 per share, including the underwriters' full exercise of their option to purchase 530,973 additional shares of common stock, and (ii) pre-funded warrants to purchase 221,238 shares of common stock at a public offering price of $56.4999 per share of common stock underlying the warrants.

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Total net proceeds generated from the offering were approximately $216.0 million, after deducting underwriting discounts, commissions and other offering expenses payable by us.

In December 2024, we entered into an amendment to the 2024 Sales Agreement with Jefferies to provide for the offering, issuance and sale of up to an aggregate of $250.0 million of common stock from time to time in at-the-market offerings.

During the three months ended March 31, 2025, we issued and sold an aggregate of 694,212

shares under the amended 2024 Sales Agreement for aggregate net proceeds of $54.9 million, after deducting commissions and offering expenses payable by us.

The following table provides information regarding our cash flows for each period presented (in thousands):

Nine Months Ended

September 30,

Three Months Ended

Net cash (used in) provided by:

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Financing activities 54,269 182,757

Net increase in cash, cash equivalents and restricted cash $ 87,345 $ 39,470

Net increase (decrease) in cash, cash equivalents and restricted cash $ (49,805) $ 70,680

Operating Activities

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The cash used in operating activities resulted primarily from our net losses adjusted for non-cash charges and changes in operating assets and liabilities, which are primarily the result of increased expenses and timing of vendor payments.

During the nine months ended September 30, 2024, net cash used in operating activities of $75.6 million was primarily due to our $124.1 million net loss, partially offset by $15.8 million in changes in operating assets and

liabilities primarily related to increases in accounts payable and accrued expenses, and $32.7 million of non-cash charges primarily related to stock-based compensation.

During the nine months ended September 30, 2023, net cash used in operating activities of $87.3 million was primarily due to our $96.4 million net loss and $11.0 million in changes in operating assets and liabilities primarily related to decreases in accrued expenses and accounts payable, partially offset by $20.1 million of non-cash charges primarily related to stock-based compensation.

During the three months ended March 31, 2025, net cash used in operating activities of $53.0 million was primarily due to our $69.3 million net loss, partially offset by $8.4 million in changes in operating assets and liabilities primarily related to an increase in accounts payable and $7.9 million of non-cash charges primarily related to stock-based compensation.

During the three months ended March 31, 2024, net cash used in operating activities of $20.9 million was primarily due to our $39.6 million net loss, partially offset by $1.5 million in changes in operating assets and liabilities primarily related to a decrease in accounts payable and $17.2 million of non-cash charges primarily related to stock-based compensation.

Investing Activities

During the nine months ended September 30, 2024, net cash used in investing activities of $237.6 million was primarily related to purchases of marketable securities partially offset by maturities of marketable securities.

During the nine months ended September 30, 2023, net cash provided by investing activities of $39.0 million was related to maturities of marketable securities.

During the three months ended March 31, 2025, net cash used in investing activities of $51.1 million was primarily related to purchases of marketable securities offset by maturities of marketable securities.

During the three months ended March 31, 2024, net cash used in investing activities of $91.2 million was related to maturities of marketable securities.

Financing Activities

During the nine months ended September 30, 2024, net cash provided by financing activities of $400.6 million consisted primarily of net proceeds from our January 2024 and April 2024 follow-on public offerings, our at-the-market offerings and our collaboration and license agreement with Tenacia.

During the nine months ended September 30, 2023, net cash provided by financing activities of $87.7 million consisted primarily of net proceeds from at-the-market offerings and our June 2023 follow-on public offering.

During the three months ended March 31, 2025, net cash provided by financing activities of $54.3 million consisted primarily of net proceeds from our at-the-market offerings.

During the three months ended March 31, 2024, net cash provided by financing activities of $182.8 million consisted of net proceeds from our January 2024 public offering, our at-the-market offerings and our collaboration and license agreement with Tenacia Biotechnology (Shanghai) Co., Ltd.

Plan of Operation and Future Funding Requirements

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• experience any delays or encounter any issues with any of the above, including but not limited to failed studies, complex results, safety issues or other regulatory challenges.

We are unable to estimate the exact amount of our working capital requirements, but based on our current operating plan, we believe that our cash, cash equivalents, and marketable securities as of September 30, 2024 will be sufficient to fund our operating expenses and capital expenditure requirements into 2027.

We are unable to estimate the exact amount of our working capital requirements, but based on our current operating plan, we believe that our cash, cash equivalents, and marketable securities as of March 31, 2025 will be sufficient to fund our operating expenses and capital expenditure requirements into 2028.

However, we have based this estimate on assumptions that may prove to be wrong and we could exhaust our capital resources sooner than we expect.

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Actual results may differ from these estimates under different assumptions or conditions.

There have been no changes to our critical accounting policies from those described under the heading “Management’s Discussion and Analysis of Financial Condition and Results of Operations – Critical Accounting Policies and Significant Judgments and Estimates” included in our Annual Report on Form 10-K filed with the SEC on March 5, 2024, other than as disclosed in Note 2 to our condensed consolidated financial statements appearing elsewhere in this Quarterly Report on Form 10-Q.

There have been no changes to our critical accounting policies from those described under the heading “Management’s Discussion and Analysis of Financial Condition and Results of Operations – Critical Accounting Policies and Significant Judgments and Estimates” included in our Annual Report on Form 10-K filed with the SEC on February 28, 2025, other than as disclosed in Note 2 to our condensed consolidated financial statements appearing elsewhere in this Quarterly Report on Form 10-Q.

Recently Issued Accounting Pronouncements

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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