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Item 2. Management’s Discussion and Analysis

2025-08-07 compared with 2025-05-08 · 65 added, 38 removed, 165 unchanged (38% of the section changed)

MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS

The following discussion and analysis of our condensed consolidated financial condition and results of operations should be read in conjunction with our unaudited condensed consolidated financial statements and related notes included in this Quarterly Report on Form 10-Q (this Quarterly Report) and the audited financial statements and notes thereto as of and for the year ended December 31, 2024 included with our Annual Report on Form 10-K, filed with the SEC on February 27, 2025 (the Annual Report).

The following discussion and analysis of our financial condition and results of operations should be read in conjunction with our unaudited condensed consolidated financial statements and related notes thereto included in this Quarterly Report on Form 10-Q (this Quarterly Report), the audited financial statements and notes thereto as of and for the year ended December 31, 2024 included with our Annual Report on Form 10-K, filed with the SEC on February 27, 2025 (our Annual Report), and our unaudited condensed consolidated financial statements and related notes thereto as of and for the quarter ended March 31, 2025 included with our Quarterly Report on Form 10-Q, filed with the SEC on May 8, 2025.

Past operating results are not necessarily indicative of results that may occur in future periods.

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Readers of this Quarterly Report are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date on which they are made.

We undertake no obligation to update or revise publicly any forward-looking statements.

We undertake no obligation to update or revise publicly any forward-looking statements except as required by law.

Actual events or results may differ materially from our expectations.

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Our neuro-rare disease franchise is anchored by the commercial product DAYBUE, which is the first and only drug approved for the treatment of Rett syndrome.

Net product sales from these two commercial products totaled $244.3 million for the three months ended March 31, 2025, compared with $205.8 million for the three months ended March 31, 2024.

Net product sales from these two commercial products totaled $508.9 million for the six months ended June 30, 2025, compared with $447.8 million for the six months ended June 30, 2024.

In addition to these commercial products, we have a portfolio of product candidates and research programs that are designed to address significant unmet medical needs in CNS disorders and rare diseases.

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Hyperphagia is an intense persistent sensation of hunger accompanied by food preoccupations, an extreme drive to consume food, food-related behavior problems, and a lack of normal satiety.

In November 2023, we initiated the Phase 3 COMPASS PWS study evaluating the efficacy and safety of ACP-101 for the treatment of hyperphagia in PWS.

In November 2023, we initiated the Phase 3 COMPASS PWS study evaluating the efficacy and safety of ACP-101 for the treatment of hyperphagia in PWS for which we completed enrollment in the second quarter of 2025.

We expect to report top-line results from this study early in the fourth quarter of 2025.

In the event of positive study results, we plan to submit a New Drug Application (NDA) to the FDA in the first quarter of 2026, with the potential for FDA approval in the third quarter of 2026 as a resubmission.

Our next most advanced product candidate is ACP-204 for the treatment of Alzheimer’s disease psychosis (ADP).

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We plan to initiate an additional Phase 2 study of ACP-204 in Lewy Body Dementia with Psychosis (LBDP) in the third quarter of 2025.

We have several product candidates in earlier stages of development for the treatment of CNS disorders and rare diseases, including ACP-711 for the treatment of essential tremor, for which we expect to initiate a Phase 2 study in 2026.

We have several product candidates in earlier stages of development for the treatment of CNS disorders and rare diseases.

These include ACP-711 for the treatment of essential tremor, with a Phase 2 study expected to begin in 2026;

ACP-211 for the treatment of major depressive disorder, for which a Phase 2 study is anticipated to start in the fourth quarter of 2025;

and ACP-271, a GPR88 agonist, with a first-in-human study in healthy volunteers planned for the fourth quarter of 2025.

We have incurred substantial operating losses since our inception due in large part to expenditures for our research and development activities.

As of March 31, 2025, we had an accumulated deficit of $2.2 billion.

As of June 30, 2025, we had an accumulated deficit of $2.2 billion.

Contingent on the level of business development activities we may complete as well as pipeline programs we may advance, we may incur operating losses as we incur significant research and development costs and costs for continued commercialization of our products.

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Cost of Product Sales

Cost of product sales consists of third-party manufacturing costs, freight, and indirect overhead costs associated with sales of our products.

Cost of product sales consists of third-party manufacturing costs, freight, duties, and indirect overhead costs associated with sales of our products.

Cost of product sales may also include period costs related to certain inventory manufacturing services, excess or obsolete inventory adjustment charges, unabsorbed manufacturing and overhead costs, and manufacturing variances.

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Cost of sales for a newly launched product does not include the full cost of manufacturing until the initial pre-launch inventory is depleted, and additional inventory is manufactured and sold.

Thus, the cost of sales as a percentage of net sales of DAYBUE for the three months ended March 31, 2025 was affected by use of the initial pre-launch inventory, which was previously expensed as research and development expense, and is referred to as zero cost inventories.

Thus, the cost of sales as a percentage of net sales of DAYBUE for the three and six months ended June 30, 2025 was affected by use of the initial pre-launch inventory, which was previously expensed as research and development expense, and is referred to as zero cost inventories.

However, we do not expect that the cost of sales as a percentage of net sales of DAYBUE will increase significantly once we commence the sales of full cost inventories.

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The FDA has released us from one of the five post-marketing requirements (PMRs).

In addition, we have fulfilled one of the five PMRs.

Of the remaining three PMRs, we have also completed one and we are awaiting the FDA’s acknowledgement and acceptance of that completed PMR.

In addition, we have fulfilled two of the five PMRs.

Of the remaining two PMRs, we have also completed one and we are awaiting the FDA’s acknowledgement and acceptance of that completed PMR.

We will be responsible for all costs incurred for these PMRs.

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To the extent that external expenses are not attributable to a specific project, they are allocated proportionally to each of the projects.

The following table summarizes our research and development expenses for the three months ended March 31, 2025 and 2024 (in thousands):

Three Months Ended March 31,

The following table summarizes our research and development expenses for the three and six months ended June 30, 2025 and 2024 (in thousands):

Three Months Ended June 30,

Six Months Ended June 30,

Costs of external service providers:

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Early-stage programs

Upfront and milestone payments

Internal costs

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Likewise, we are unable to determine with certainty the anticipated completion dates for our current research and development programs.

Clinical development and regulatory approval timelines, probability of success, and development costs vary widely.

Clinical development and regulatory approval timelines, probability of success, and development costs vary widely across our development programs.

While our current development efforts are primarily focused on advancing the development of ACP-101, ACP-204 and other early-stage product candidates, we anticipate that we will make determinations as to which programs to pursue and how much funding to direct to each program on an ongoing basis in response to the scientific and clinical success of each product candidate, as well as an ongoing assessment of the commercial potential of each candidate and our financial position.

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Income tax expense may fluctuate from quarter to quarter due to adjustments related to non-recurring transactions, timing of revenue and expense across different tax jurisdictions and changes in certain tax assessments.

On July 4, 2025, the OBBBA was signed into law, which enacts substantial changes to U.S.

tax and related laws.

Some of the provisions of the new tax law affecting corporations include but are not limited to expensing of domestic research expenses, increasing the limit of the deduction of interest expense deduction to thirty percent of earnings before interest, taxes, depreciation, and amortization (EBITDA), and one hundred percent bonus depreciation on eligible property acquired after January 19, 2025.

We are currently evaluating the impact the new tax law will have on our financial condition and results of operations.

The impact of the tax law changes from the OBBBA will be included in our financial statements beginning in the three months ended September 30, 2025.

Critical Accounting Policies and Estimates

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Due to these fluctuations, we believe that the period-to-period comparisons of our operating results are not a good indication of our future performance.

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

Comparison of the Three Months Ended June 30, 2025 and 2024

Product Sales, Net

Net product sales, comprised of NUPLAZID and DAYBUE, were $244.3 million and $205.8 million for the three months ended March 31, 2025 and 2024, respectively.

Net product sales of NUPLAZID were $159.7 million and $129.9 million for the three months ended March 31, 2025 and 2024, respectively.

Net product sales, comprised of NUPLAZID and DAYBUE, were $264.6 million and $242.0 million for the three months ended June 30, 2025 and 2024, respectively.

Net product sales of NUPLAZID were $168.5 million and $157.4 million for the three months ended June 30, 2025 and 2024, respectively.

The increase in net product sales of NUPLAZID of $11.1 million was due to the growth in NUPLAZID unit sales as well as a higher average net selling price of NUPLAZID in 2025 compared to 2024.

Net product sales of DAYBUE were $84.6 million and $75.9 million for the three months ended March 31, 2025 and 2024, respectively.

Net product sales of DAYBUE were $96.1 million and $84.6 million for the three months ended June 30, 2025 and 2024, respectively.

The increase in net product sales of DAYBUE of $11.5 million was due to the growth in DAYBUE unit sales as well as a higher average net selling price of NUPLAZID in 2025 compared to 2024.

Cost of Product Sales

Cost of product sales was $20.7 million and $18.2 million for the three months ended June 30, 2025 and 2024, respectively, or approximately 8% and 8% of net product sales, respectively.

Cost of product sales as a percentage of net product sales remained relatively flat during the three months ended June 30, 2025 as compared to the same period of 2024.

Certain manufacturing related expenses incurred prior to DAYBUE receiving FDA approval were classified as research and development expenses, resulting in zero cost inventory.

If cost of product sales included previously expensed inventories, the total cost of sales with these manufacturing costs included for the three months ended June 30, 2025 would have increased by approximately $1.5 million.

Research and Development Expenses

Research and development expenses increased to $78.0 million for the three months ended June 30, 2025 from $76.2 million for the three months ended June 30, 2024.

The increase in research and development expenses was primarily related to increased expenditures for ACP-204 and ACP-101 as well as increased personnel expenses, offset by reduced expenditures for ending studies that took place in 2024.

Selling, General and Administrative Expenses

Selling, general and administrative expenses increased to $133.5 million for the three months ended June 30, 2025 from $117.1 million for the three months ended June 30, 2024.

The increase in selling, general and administrative expenses was primarily driven by increased expenditures for both DAYBUE and NUPLAZID in the U.S., including the planned expansion of the DAYBUE team.

Comparison of the Six Months Ended June 30, 2025 and 2024

Product Sales, Net

Net product sales, comprised of NUPLAZID and DAYBUE, were $508.9 million and $447.8 million for the six months ended June 30, 2025 and 2024, respectively.

Net product sales of NUPLAZID were $328.2 million and $287.3 million for the six months ended June 30, 2025 and 2024, respectively.

The increase in net product sales of NUPLAZID of $40.9 million was due to the growth in NUPLAZID unit sales as well as a higher average net selling price of NUPLAZID in 2025 compared to 2024.

Net product sales of DAYBUE were $180.7 million and $160.5 million for the six months ended June 30, 2025 and 2024, respectively.

The increase in net product sales of DAYBUE of $20.2 million was primarily due to the growth in DAYBUE unit sales.

The following table provides a summary of activity with respect to our sales allowances and accruals for the three months ended March 31, 2025 (in thousands):

The following table provides a summary of activity with respect to our sales allowances and accruals for the six months ended June 30, 2025 (in thousands):

Distribution Fees,

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Credits/payments for prior period sales

Balance as of March 31, 2025

Balance as of June 30, 2025

Cost of Product Sales

Cost of product sales was $20.4 million and $23.0 million for the three months ended March 31, 2025 and 2024, respectively, or approximately 8% and 11% of net product sales, respectively.

Cost of product sales as a percentage of net product sales for NUPLAZID remained relatively flat during the three months ended March 31, 2025 as compared to the same period of 2024.

The decrease in cost of product sales was primarily due to the decreased license fees and royalties expensed for DAYBUE, including royalties due to Neuren based on net sales of DAYBUE and the amortization of the milestone payments capitalized under our 2018 license agreement with Neuren.

The license fees and royalties expensed during the three months ended March 31, 2025 were $11.2 million as compared to $12.9 million in the same period of 2024.

During the three months ended March 31, 2024, we had a one-time cumulative catch-up of $3.9 million of expense for the capitalization and amortization of the $50.0 million sales milestone to Neuren.

Cost of product sales was $41.1 million and $41.2 million for the six months ended June 30, 2025 and 2024, respectively, or approximately 8% and 9% of net product sales, respectively.

Cost of product sales as a percentage of net product sales remained relatively flat during the six months ended June 30, 2025 as compared to the same period of 2024.

Certain manufacturing related expenses incurred prior to DAYBUE receiving FDA approval were classified as research and development expenses, resulting in zero cost inventory.

Prior to receiving FDA approval for DAYBUE in March 2023, we manufactured inventory and recorded approximately $29.9 million related to the zero cost inventory as research and development expense.

Utilizing the actual direct costs to manufacture DAYBUE prior to receiving FDA approval, had the previously expensed inventory been capitalized and recognized when sold, the total cost of sales with these manufacturing costs included for the three months ended March 31, 2025 would have increased by approximately $2.8 million.

Utilizing the actual direct costs to manufacture DAYBUE prior to receiving FDA approval, had the previously expensed inventory been capitalized and recognized when sold, the total cost of sales with these manufacturing costs included for the six months ended June 30, 2025 would have increased by approximately $4.4 million.

We do not expect our cost of product sales for DAYBUE to increase significantly as a percentage of net product sales in future periods as we continue to produce inventory for future sales.

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Research and Development Expenses

Research and development expenses increased to $78.3 million for the three months ended March 31, 2025, including $3.4 million in stock-based compensation expense, from $59.7 million for the three months ended March 31, 2024, including $4.1 million in stock-based compensation expense.

The increase in research and development expenses was mainly due to increased costs from clinical-stage programs.

Research and development expenses increased to $156.2 million for the six months ended June 30, 2025 from $135.9 million for the six months ended June 30, 2024.

The increase in research and development expenses was mainly related to increased expenditures for ACP-204 and ACP-101 as well as increased personnel expenses, offset by reduced expenditures for ending studies that took place in 2024.

Selling, General and Administrative Expenses

Selling, general and administrative expenses increased to $126.4 million for the three months ended March 31, 2025, including $7.6 million in stock-based compensation expense, from $108.0 million for the three months ended March 31, 2024, including $10.5 million in stock-based compensation expense.

The increase in selling, general and administrative expenses was primarily driven by the costs related to the consumer activation program to support the NUPLAZID franchise and higher commercial operation expenses for the planned expansion of the DAYBUE team.

Selling, general and administrative expenses increased to $259.9 million for the six months ended June 30, 2025 from $225.1 million for the six months ended June 30, 2024.

The increase in selling, general and administrative expenses was primarily driven by costs related to our consumer activation program to support NUPLAZID and the planned expansion of the DAYBUE team.

Liquidity and Capital Resources

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the payment will be set off against the allowance for such rebate that we have accrued up to the date of payment.

At March 31, 2025, we had $681.6 million in cash, cash equivalents, and investment securities, compared to $756.0 million at December 31, 2024.

This $74.4 million decrease was due to cash used in operating activities and material cash payments made to Neuren, including for one third of the net proceeds ($48.8 million) from our sale of the PRV and the annual net sales milestone ($50 million) as our aggregate net revenue of trofinetide in North America for the treatment of Rett syndrome exceeded $250.0 million in 2024.

Net cash provided by operating activities totaled $20.3 million for the three months ended March 31, 2025 compared to $29.1 million for the three months ended March 31, 2024.

This decrease in cash provided by operations primarily resulted from an increase in our research and development cost and selling, general and administrative cost partially offset by the increase in product revenue.

Net cash used in investing activities increased to $124.0 million for the three months ended March 31, 2025 compared to $14.0 million for the three months ended March 31, 2024.

The increase in net cash used in investing activities for the three months ended March 31, 2025 compared to the three months ended March 31, 2024 was primarily due to increased net purchases of investment securities.

Net cash provided by financing activities increased to $1.8 million for the three months ended March 31, 2025 compared to $1.0 million for the three months ended March 31, 2024.

This increase in net cash provided by financing activities for the three months ended March 31, 2025 was attributable primarily to an increase in proceeds resulting from the exercise of employee stock options.

At June 30, 2025, we had $762.0 million in cash, cash equivalents, and investment securities, compared to $756.0 million at December 31, 2024.

This $6.0 million increase was due to cash provided by operating activities offset by material cash payments made to Neuren, including for one third of the net proceeds ($48.8 million) from our sale of the PRV and the annual net sales milestone ($50 million) as our aggregate net revenue of trofinetide in North America for the treatment of Rett syndrome exceeded $250.0 million in 2024.

Net cash provided by operating activities totaled $84.3 million for the six months ended June 30, 2025 compared to $54.1 million for the six months ended June 30, 2024.

This increase in cash provided by operations primarily resulted from the increase in product revenue partially offset by an increase in our research and development cost and selling, general and administrative cost.

Net cash used in investing activities increased to $167.3 million for the six months ended June 30, 2025 compared to $70.2 million for the six months ended June 30, 2024.

The increase in net cash used in investing activities for the six months ended June 30, 2025 compared to the six months ended June 30, 2024 was primarily due to increased net purchases of investment securities.

Net cash provided by financing activities increased to $17.8 million for the six months ended June 30, 2025 compared to $4.6 million for the six months ended June 30, 2024.

This increase in net cash provided by financing activities for the six months ended June 30, 2025 was attributable primarily to an increase in proceeds resulting from the exercise of employee stock options and awards.

Off-Balance Sheet Arrangements

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We do not have any direct investments in auction-rate securities or securities that are collateralized by assets that include mortgages or subprime debt.

If a 10 percent change in interest rates were to have occurred on March 31, 2025, this change would not have had a material effect on the fair value of our investment portfolio as of that date.

If a 10 percent change in interest rates were to have occurred on June 30, 2025, this change would not have had a material effect on the fair value of our investment portfolio as of that date.

Due to our investment in investment-grade, interest-bearing securities, as of the date of this Quarterly Report on Form 10-Q, we do not expect anticipated changes in interest rates to have a material effect on our interest rate risk in future reporting periods.

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