What changed in this filing

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Item 2. Management’s Discussion and Analysis

2026-08-06 compared with 2026-05-07 · 84 added, 37 removed, 226 unchanged (35% of the section changed)

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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 ulixacaltamide program within the Cerebrum™ platform, we announced positive topline results for the two studies in the Phase 3 Essential3 program in essential tremor, or ET, in October 2025.

We submitted a New Drug Application, or NDA, for ulixacaltamide HCl for the treatment of ET to the FDA, and it was accepted for review with a target action date under the Prescription Drug User Fee Act, or PDUFA, of January 29, 2027.

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

For our ulixacaltamide program within the Cerebrum™ platform, we submitted a New Drug Application, or NDA, for ulixacaltamide HCl for the treatment of essential tremor, or ET, to the FDA based on the positive results of our two studies in the Phase 3 Essential3 program in ET, and it was accepted for review with a target action date under the Prescription Drug User Fee Act, or PDUFA, of January 29, 2027.

The mid-cycle meeting was successfully completed and the FDA has identified no major safety or efficacy concerns to date.

The agency confirmed that no advisory committee meeting is planned.

In anticipation of potential approval, commercial preparations and pre-launch activities are ongoing.

For our relutrigine program within the Cerebrum™ platform, in December 2025, we announced positive results from the registrational cohort of the EMBOLD study in SCN2A and SCN8A developmental and epileptic encephalopathies, or DEEs, after receiving a recommendation from the Data Monitoring Committee to stop the study early for efficacy.

We submitted an NDA for relutrigine for the treatment of SCN2A-DEE and SCN8A-DEE to the FDA, and it was accepted for priority review with a target PDUFA action date of September 27, 2026.

In July 2026, we entered into a research collaboration and license agreement with Remagine Labs, Inc., a developer of electronically controlled transdermal drug delivery systems, to develop an iontophoretic transdermal patch for the delivery of ulixacaltamide.

For our relutrigine program within the Cerebrum™ platform, we submitted an NDA for relutrigine for the treatment of SCN2A and SCN8A developmental and epileptic encephalopathies, or DEEs, to the FDA based on the positive results from the registrational cohort of the EMBOLD study in SCN2A-DEE and SCN8A-DEE, and it was accepted for priority review with a target PDUFA action date of September 27, 2026.

In June 2026, the FDA notified us that it had extended by three months the review period for our NDA for relutrigine, setting an updated PDUFA action date of December 27, 2026.

The mid-cycle meeting was successfully completed and the FDA has identified no major safety or efficacy concerns to date.

The agency confirmed that no advisory committee meeting is planned.

In anticipation of potential approval, commercial preparations and pre-launch activities are ongoing.

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Assuming approval of the NDA for relutrigine, we believe the EMERALD study, if positive, could serve as the basis for a supplemental NDA, or sNDA, submission in 2027.

The FDA has completed its Bioresearch Monitoring, or BIMO, inspection as part of its ongoing review of the ulixacaltamide and relutrigine NDAs.

The BIMO inspection was conducted jointly for both applications, and the

scope of the inspection was comprehensive, spanning overall quality and clinical operations, data integrity, including statistical and interim analyses, and safety, amongst other standard areas in the BIMO program.

The inspection was completed successfully, and no form 483 was issued.

For our vormatrigine program within the Cerebrum™ platform, our ENERGY program is ongoing, which consists of five studies to generate patient eligibility, efficacy, safety and pharmacokinetics, or PK, data in patients with focal onset seizures, or FOS, or generalized epilepsy.

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We initiated the EMPOWER study, an observational study of vormatrigine in patients with epilepsy, in the third quarter of 2024.

For our POWER1 study, a double-blind, placebo-controlled, 12-week study in FOS, we expect to announce topline results in the second quarter of 2026.

our third efficacy study, is enrolling patients, with completion expected in the second half of 2026 and topline results expected in 2027.

We intend to initiate POWER3, a clinical trial evaluating vormatrigine as a single-agent treatment for FOS, in the first half of 2026.

For our most advanced product candidate under the Solidus™ platform, elsunersen, we announced positive topline results from the EMBRAVE Part A study evaluating the safety and efficacy of elsunersen versus sham procedure in April 2026 for treating SCN2A gain-of-function DEE.

We are currently enrolling EMBRAVE3, a Phase 3 registrational study for SCN2A gain-of-function DEE, and expect to announce topline results in 2027.

In June 2026, we announced that the primary endpoint was not met in our POWER1 study, a double-blind, placebo-controlled, 12-week study in FOS.

We paused enrollment in POWER2, our third efficacy study, to reassess the vormatrigine program and determine potential modifications.

We intend to restart enrollment of the POWER2 study and initiate the POWER3 study, a clinical trial evaluating vormatrigine as a single-agent treatment for FOS, in the fourth quarter of 2026.

For our most advanced product candidate under the Solidus™ platform, elsunersen, we announced positive topline results in April 2026 from the EMBRAVE Part A study evaluating the safety and efficacy of elsunersen versus sham procedure for treating SCN2A gain-of-function DEE.

In June 2026, we received FDA breakthrough therapy designation for elsunersen for the treatment of seizures associated with SCN2A DEEs caused by gain-of-function variants.

We are currently enrolling EMBRAVE3, a Phase 3 registrational study for early-seizure-onset SCN2A DEE, and expect to announce topline results in 2027.

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 genetic link to de novo autism spectrum disorders, or ASD.

We anticipate nominating a development candidate for PRAX-080, PRAX-090 and PRAX-100 in the first half of 2026.

We anticipate nominating several development candidates in 2026.

We were incorporated in 2015 and commenced operations in 2016.

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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 $92.6 million for the three months ended March 31, 2026.

As of March 31, 2026, we had an accumulated deficit of $1.2 billion.

We have incurred recurring operating losses since inception, including a net loss of $176.3 million for the six months ended June 30, 2026.

As of June 30, 2026, we had an accumulated deficit of $1.3 billion.

We expect to incur significant expenses for the foreseeable future as we expand our research and development activities and prepare for commercialization of our product candidates.

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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 March 31, 2026, we had cash, cash equivalents and marketable securities of $1.4 billion.

We expect that our cash, cash equivalents, and marketable securities as of March 31, 2026 will be sufficient to fund our operating expenditures and capital expenditure requirements necessary to advance our operating activities into 2028.

As of June 30, 2026, we had cash, cash equivalents and marketable securities of $1.4 billion.

We expect that our cash, cash equivalents, and marketable securities as of June 30, 2026 will be sufficient to fund our operating expenditures and capital expenditure requirements necessary to advance our operating activities into 2028.

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

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Three Months Ended

June 30, Six Months Ended June 30,

2026 2025 2026 2025

$ 40,444 $ 46,977 $ 92,433 $ 89,809

8,655 1,979 12,989 3,327

Personnel-related (including stock-based compensation) 15,877 11,459 32,528 24,310

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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 months ended March 31, 2026 and 2025.

There was no income tax provision recognized for the three and six months ended June 30, 2026 and 2025.

Results of Operations

Comparison of the Three Months Ended March 31, 2026 and 2025

Comparison of the Three Months Ended June 30, 2026 and 2025

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

Three Months Ended

March 31, Change

June 30, Change

Operating expenses:

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Three Months Ended

March 31, Change

June 30, Change

Cerebrum™ $ 40,444 $ 46,977 $ (6,533)

Solidus™ 8,655 1,979 6,676

Personnel-related (including stock-based compensation) 15,877 11,459 4,418

Other indirect research and development expenses 4,440 2,591 1,849

Total research and development expenses $ 69,416 $ 63,006 $ 6,410

The $6.4 million increase in research and development expenses was primarily attributable to the following:

• $6.7 million increase in expense related to our Solidus™ platform, driven primarily by a $5.0 million milestone payment due to Ionis Pharmaceuticals Inc., or Ionis, upon completion of our EMBRAVE Part A study, as well as spend for our EMBRAVE3 pivotal clinical trial;

• $6.5 million decrease in expense related to our Cerebrum™ platform, driven primarily by:

◦ a $6.6 million decrease in spend for our ulixacaltamide program, driven primarily by decreased activity in the Essential3 study, partially offset by increased manufacturing costs to support commercialization;

◦ a $4.8 million decrease in spend for our vormatrigine program, primarily driven by lower spend for our ENERGY program, partially offset by,

◦ a $4.2 million increase in activities for our earlier stage assets as we advance our portfolio.

• $4.4 million increase in personnel-related costs mainly due to increased headcount;

• $1.8 million increase in indirect expenses driven primarily by consulting spend to support operations.

General and Administrative Expense

The $14.4 million increase in general and administrative expenses was primarily attributable to the following:

• $7.2 million increase in professional expenses, primarily attributable to increased commercial spend as we prepare for the potential launches of ulixacaltamide and relutrigine;

• $6.3 million increase in personnel-related costs mainly due to increased headcount.

Results of Operations

Comparison of the Six Months Ended June 30, 2026 and 2025

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

Six Months Ended

June 30, Change

Operating expenses:

Research and development $ 147,403 $ 123,812 $ 23,591

General and administrative 55,361 26,983 28,378

Total operating expenses 202,764 150,795 51,969

Loss from operations (202,764) (150,795) (51,969)

Other income:

Other income, net 26,483 10,372 16,111

Total other income 26,483 10,372 16,111

Net loss $ (176,281) $ (140,423) $ (35,858)

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):

Six Months Ended

June 30, Change

$ 92,433 $ 89,809 $ 2,624

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◦ a $7.6 million increase in spend for our relutrigine program, primarily related to our EMERALD trial activities and manufacturing spend to support commercialization;

◦ a $6.9 million increase in spend for our vormatrigine program, primarily driven by spend for our ENERGY program;

◦ a $5.9 million increase in activities for our earlier stage assets as we advance our portfolio;

partially offset by,

◦ a $2.1 million increase in spend for our vormatrigine program, primarily driven by spend for our ENERGY program, partially offset by,

◦ a $13.0 million decrease in spend for our ulixacaltamide program, driven primarily by decreased activity in the Essential3 study, partially offset by increased manufacturing costs to support commercialization and the PDUFA fee paid in the first quarter of 2026.

• $3.8 million increase in personnel-related costs mainly due to increased headcount;

• $3.0 million increase in expense related to our Solidus™ platform, driven by elsunersen program spend for EMBRAVE Part A clinical trial and our EMBRAVE3 pivotal clinical trial.

• $9.7 million increase in expense related to our Solidus™ platform, driven primarily by a $5.0 million milestone payment due to Ionis Pharmaceuticals Inc., or Ionis, upon completion of our EMBRAVE Part A study, as well as spend for our EMBRAVE3 pivotal clinical trial;

• $8.2 million increase in personnel-related costs due to increased headcount;

• $3.1 million increase in indirect expenses driven primarily by consulting spend to support operations.

General and Administrative Expense

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• $15.5 million increase in personnel-related costs mainly due to increased stock-based compensation expense and increased headcount;

• $3.5 million increase in professional expenses, primarily attributable to increased commercial spend as we prepare for the potential launch of ulixacaltamide and relutrigine.

• $11.3 million increase in professional expenses, primarily attributable to increased commercial spend as we prepare for the potential launches of ulixacaltamide and relutrigine.

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 March 31, 2026, we have raised $2.4 billion in aggregate cash proceeds from such transactions, net of issuance costs.

As of March 31, 2026, we had cash, cash equivalents, and marketable securities of $1.4 billion.

From inception through June 30, 2026, we have raised $2.4 billion in aggregate cash proceeds from such transactions, net of issuance costs.

As of June 30, 2026, we had cash, cash equivalents, and marketable securities of $1.4 billion.

In March 2024, we entered into an Open Market Sale Agreement, or the 2024 Sales Agreement, with Jefferies LLC, or 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.

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 amount 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.

During the six months ended June 30, 2025, we issued and sold an aggregate of 1,351,689 shares under the amended 2024 Sales Agreement for aggregate net proceeds of $83.3 million, after deducting commissions and offering expenses payable by us.

The amended 2024 Sales Agreement was terminated in September 2025.

In September 2025, we entered into a Sales Agreement, or the 2025 Sales Agreement, with TD Securities (USA) LLC, or TD Cowen, 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, 2026, we did not issue or sell any shares of common stock under the 2025 Sales Agreement.

During the six months ended June 30, 2026, we did not issue or sell any shares of common stock under the 2025 Sales Agreement.

In October 2025, we completed a public offering of:

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Total net proceeds generated from the offering were $567.1 million, after deducting underwriting discounts, commissions, and other offering expenses payable by us.

As of March 31, 2026, none of the pre-funded warrants had been exercised and all remained outstanding.

As of June 30, 2026, none of the pre-funded warrants had been exercised and all remained outstanding.

In January 2026, we completed a public offering of 2,543,800 shares of our common stock at a public offering price of $260.00 per share, including the underwriters' full exercise of their option to purchase 331,800 additional shares of common stock.

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

Three Months Ended

Six Months Ended

Net cash (used in) provided by:

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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.

During the three months ended March 31, 2026, net cash used in operating activities of $86.2 million was primarily due to our $92.6 million net loss and $10.3 million in changes in operating assets and liabilities primarily related to a decrease in accrued expenses, partially offset by $16.7 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 six months ended June 30, 2026, net cash used in operating activities of $163.7 million was primarily due to our $176.3 million net loss and $15.1 million in changes in operating assets and liabilities primarily related to a decrease in accrued expenses, partially offset by $27.7 million of non-cash charges primarily related to stock-based compensation.

During the six months ended June 30, 2025, net cash used in operating activities of $107.7 million was primarily due to our $140.4 million net loss, partially offset by $17.8 million in changes in operating assets and liabilities primarily related to an increase in accounts payable and $14.9 million of non-cash charges primarily related to stock-based compensation.

Investing Activities

During the three months ended March 31, 2026, net cash used in investing activities of $344.3 million was primarily related to purchases of marketable securities offset by 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 six months ended June 30, 2026, net cash used in investing activities of $333.3 million was primarily related to purchases of marketable securities offset by maturities of marketable securities.

During the six months ended June 30, 2025, net cash used in investing activities of $33.2 million was primarily related to purchases of marketable securities offset by maturities of marketable securities.

Financing Activities

During the three months ended March 31, 2026, net cash provided by financing activities of $609.4 million consisted primarily of net proceeds from our January 2026 follow-on 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 six months ended June 30, 2026, net cash provided by financing activities of $614.0 million consisted primarily of net proceeds from our January 2026 follow-on offering.

During the six months ended June 30, 2025, net cash provided by financing activities of $82.9 million consisted primarily of net proceeds from our at-the-market offerings.

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 March 31, 2026 will be sufficient to fund our operating expenditures and capital expenditure requirements into 2028.

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 June 30, 2026 will be sufficient to fund our operating expenditures 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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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