Carbetocin

SAN DIEGO--( BUSINESS WIRE )--Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced its financial results for the second quarter ended June 30, 2024. “ In the second quarter of 2024, Acadia delivered $242.0 million in net product sales, highlighted by 11% year-over-year growth in NUPLAZID net sales and 11% sequential growth in DAYBUE net sales,” said Steve Davis, Chief Executive Officer. “ Additionally, we are advancing enrollment in our Phase 3 trial in Prader-Willi syndrome and our Phase 2 / Phase 3 program in Alzheimer’s disease psychosis. With two successful commercial products, a strong pipeline of late and early-stage assets and a growing cash balance, Acadia is in a strong position to support our long-term growth.” Company Updates The journal Med published clinical data from two open-label extension studies, LILAC-1 and LILAC-2, which showed that patients treated with DAYBUE™ (trofinetide) who completed these studies experienced improvement in Rett syndrome symptoms as measured by the Rett Syndrome Behaviour Questionnaire (RSBQ). Presented interim data from the open-label real-world LOTUS™ study at the 2024 International Rett Syndrome Foundation (IRSF) Annual Scientific Meeting. LOTUS is an ongoing, open-label caregiver-reported study evaluating the efficacy and tolerability outcomes in patients with Rett syndrome treated with DAYBUE. Financial Results Revenues Total revenues, comprised of net product sales from NUPLAZID ® and DAYBUE, were $242.0 million for the three months ended June 30, 2024, up 46% year-over-year. Total revenues were $447.8 million for the six months ended June 30, 2024. Net product sales of NUPLAZID were $157.4 million and $142.0 million for the three months ended June 30, 2024 and 2023, respectively. The 11% year-over-year increase in net product sales of NUPLAZID included 6% growth in unit sales in 2024 compared to 2023. Net product sales of NUPLAZID were $287.3 million and $260.5 million for the six months ended June 30, 2024 and 2023, respectively. Net product sales of DAYBUE were $84.6 million and $23.2 million for the three months ended June 30, 2024 and 2023, respectively. Net product sales of DAYBUE were $160.5 million and $23.2 million for the six months ended June 30, 2024 and 2023, respectively. The increase in net product sales of DAYBUE was due to the growth in DAYBUE unit sales. Research and Development Research and development expenses were $76.2 million, compared to $58.8 million for the three months ended June 30, 2024 and 2023, respectively. The increase was mainly due to increased costs from ACP-101, ACP-204 and early-stage programs, partially offset by a reduction in costs associated with our pimavanserin negative symptoms of schizophrenia program. For the six months ended June 30, 2024 and 2023, research and development expenses were $135.9 million and $127.9 million, respectively. The increase was mainly due to increased costs from ACP-101, ACP-204 and early stage programs, partially offset by the elimination of pre-FDA approval trofinetide commercial supply expenses that were included in the previous year, as well as a reduction in costs associated with our pimavanserin negative symptoms of schizophrenia program. Selling, General and Administrative Selling, general and administrative expenses were $117.1 million and $96.0 million for the three months ended June 30, 2024 and 2023, respectively. For the six months ended June 30, 2024 and 2023, selling, general and administrative expenses were $225.1 million and $197.2 million, respectively. The increase in selling, general and administrative expenses in both periods was primarily driven by upfront costs related to a new consumer activation program to support the NUPLAZID franchise, increased marketing costs in the U.S. to support DAYBUE, and investments to support commercialization of trofinetide outside the U.S. Net Income (Loss) For the three months ended June 30, 2024, Acadia reported net income of $33.4 million, or $0.20 per common share, compared to net income of $1.1 million, or $0.01 per common share, for the same period in 2023. Net income for the three months ended June 30, 2024 and 2023 included $15.7 million and $15.2, respectively, of non-cash stock-based compensation expense. For the six months ended June 30, 2024, Acadia reported net income of $49.9 million, or $0.30 per common share, compared to a net loss of $41.9 million, or $0.26 per common share. Net income for the six months ended June 30, 2024 included $30.4 million of non-cash stock-based compensation expense. Net loss for the six months ended June 30, 2023 included $29.9 million of non-cash stock-based compensation expense. Cash and Investments At June 30, 2024, Acadia’s cash, cash equivalents and investment securities totaled $500.9 million, compared to $438.9 million at December 31, 2023. Full Year 2024 Financial Guidance Acadia is updating its 2024 guidance: NUPLAZID net product sales guidance is increased to a range of $590 to $610 million from the previous range of $560 to $590 million, reflecting stronger underlying demand driving recent higher unit volume. DAYBUE net product sales guidance is decreased to a range of $340 to $370 million from the previous range of $370 to $420 million. Total revenue guidance is revised to a range of $930 to $980 million from the previous range of $930 million to $1.01 billion. R&D expense guidance is narrowed to the lower end of the previous range and is now expected to be between $305 to $315 million. SG&A expense guidance is narrowed to the higher end of the previous range and is now expected to be between $465 to $480 million. Conference Call and Webcast Information Acadia will host a conference call to discuss the second quarter 2024 results today, Tuesday, August 6, 2024 at 1:30 p.m. PT/4:30 p.m. ET. The conference call may be accessed by registering for the call here . Once registered, participants will receive an email with the dial-in number and unique PIN number to use for accessing the call. About NUPLAZID ® (pimavanserin) Pimavanserin is a selective serotonin inverse agonist and antagonist preferentially targeting 5-HT2A receptors. These receptors are thought to play an important role in neuropsychiatric disorders. In vitro, pimavanserin demonstrated no appreciable binding affinity for dopamine (including D2), histamine, muscarinic, or adrenergic receptors. Pimavanserin was approved for the treatment of hallucinations and delusions associated with Parkinson’s disease psychosis by the U.S. Food and Drug Administration in April 2016 under the trade name NUPLAZID. About DAYBUE ™ (trofinetide) Trofinetide is a synthetic version of a naturally occurring molecule known as the tripeptide glycine-proline-glutamate (GPE). The mechanism by which trofinetide exerts therapeutic effects in patients with Rett syndrome is unknown. Trofinetide was approved for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older by the U.S. Food and Drug Administration in March 2023 under the trade name DAYBUE. About Acadia Pharmaceuticals Acadia is advancing breakthroughs in neuroscience to elevate life. For 30 years we have been working at the forefront of healthcare to bring vital solutions to people who need them most. We developed and commercialized the first and only FDA-approved drug to treat hallucinations and delusions associated with Parkinson’s disease psychosis and the first and only FDA-approved drug for the treatment of Rett syndrome. Our clinical-stage development efforts are focused on Prader-Willi syndrome, Alzheimer’s disease psychosis and multiple other programs targeting neuropsychiatric symptoms in central nervous system disorders. For more information, visit us at acadia.com and follow us on LinkedIn and Twitter . Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements other than statements of historical fact and can be identified by terms such as “may,” “will,” “should,” “could,” “would,” “expects,” “plans,” “anticipates,” “believes,” “estimates,” “projects,” “predicts,” “potential” and similar expressions (including the negative thereof) intended to identify forward-looking statements. Forward-looking statements contained in this press release, include, but are not limited to, statements about: (i) our business strategy, objectives and opportunities, including support for our early-stage pipeline and business development opportunities; (ii) plans for, including timing, development and progress of commercialization or regulatory timelines for, NUPLAZID, DAYBUE (both within and outside the U.S.) and our product candidates; (iii) benefits to be derived from and efficacy of our products, including the potential advantages of NUPLAZID and DAYBUE; (iv) the timing and conduct of our clinical trials, including continued enrollment of our clinical trials in Prader-Willi syndrome and Alzheimer’s disease psychosis, and the timing and content of our presentations regarding our clinical trials; and (v) our estimates regarding our future financial performance, profitability or capital requirements, including our full year 2024 financial guidance. Forward-looking statements are subject to known and unknown risks, uncertainties, assumptions and other factors that may cause our actual results, performance or achievements to differ materially and adversely from those anticipated or implied by our forward-looking statements. Such risks, uncertainties and other factors include, but are not limited to: our dependency on the continued successful commercialization of NUPLAZID and DAYBUE and our ability to maintain or increase sales of NUPLAZID or DAYBUE; our plans to commercialize DAYBUE outside the U.S.; the costs of our commercialization plans and development programs, and the financial impact or revenues from any commercialization we undertake; our ability to obtain necessary regulatory approvals for our product candidates and, if and when approved, market acceptance of our products; the risks associated with clinical trials and their outcomes, including risks of unsuccessful enrollment and negative or inconsistent results; our dependence on third-party collaborators, clinical research organizations, manufacturers, suppliers and distributors; the impact of competitive products and therapies; our ability to generate or obtain the necessary capital to fund our operations; our ability to grow, equip and train our specialized sales forces; our ability to manage the growth and complexity of our organization; our ability to maintain, protect and enhance our intellectual property; and our ability to continue to stay in compliance with applicable laws and regulations. Given the risks and uncertainties, you should not place undue reliance on these forward-looking statements. For a discussion of these and other risks, uncertainties and other factors that may cause our actual results, performance or achievements to differ, please refer to our quarterly report on Form 10-Q for the quarter ended March 31, 2024 as well as our subsequent filings with the Securities and Exchange Commission (SEC) from time to time, including our quarterly report on Form 10-Q for the quarter ended June 30, 2024 being filed with the SEC today, which will be available at www.sec.gov . The forward-looking statements contained herein are made as of the date hereof, and we undertake no obligation to update them after this date, except as required by law. ACADIA PHARMACEUTICALS INC. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (in thousands, except per share amounts) (Unaudited) Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Revenues Product sales, net $ 241,963 $ 165,235 $ 447,794 $ 283,697 Total revenues 241,963 165,235 447,794 283,697 Operating expenses Cost of product sales (1)(2) 18,230 7,459 41,181 9,126 Research and development (2) 76,233 58,771 135,912 127,915 Selling, general and administrative (2) 117,063 95,968 225,054 197,203 Total operating expenses 211,526 162,198 402,147 334,244 Income (loss) from operations 30,437 3,037 45,647 (50,547 ) Interest income, net 6,359 4,550 11,865 8,350 Other income (loss) 386 (1,244 ) 672 3,601 Income (loss) before income taxes 37,182 6,343 58,184 (38,596 ) Income tax expense 3,793 5,229 8,240 3,311 Net income (loss) $ 33,389 $ 1,114 $ 49,944 $ (41,907 ) Earnings (net loss) per share: Basic $ 0.20 $ 0.01 $ 0.30 $ (0.26 ) Diluted $ 0.20 $ 0.01 $ 0.30 $ (0.26 ) Weighted average common shares outstanding: Basic 165,551 163,458 165,174 163,109 Diluted 166,174 165,046 166,391 163,109 (1) Includes license fees and royalties (2) Includes the following stock-based compensation expense Cost of product sales, license fees and royalties $ 362 $ 200 $ 515 $ 368 Research and development $ 3,749 $ 3,666 $ 7,842 $ 7,638 Selling, general and administrative $ 11,574 $ 11,288 $ 22,078 $ 21,853 ACADIA PHARMACEUTICALS INC. CONDENSED CONSOLIDATED BALANCE SHEETS (in thousands) June 30, 2024 December 31, 2023 (unaudited) Assets Cash, cash equivalents and investment securities $ 500,942 $ 438,865 Accounts receivable, net 103,698 98,267 Interest and other receivables 6,695 4,083 Inventory 71,525 35,819 Prepaid expenses 46,056 39,091 Total current assets 728,916 616,125 Property and equipment, net 4,144 4,612 Operating lease right-of-use assets 52,390 51,855 Intangible assets, net 107,859 65,490 Restricted cash 5,770 5,770 Long-term inventory 14,544 4,628 Other assets 476 476 Total assets $ 914,099 $ 748,956 Liabilities and stockholders’ equity Accounts payable $ 19,882 $ 17,543 Accrued liabilities 317,969 236,711 Total current liabilities 337,851 254,254 Operating lease liabilities 47,186 47,800 Other long-term liabilities 12,362 15,147 Total liabilities 397,399 317,201 Total stockholders’ equity 516,700 431,755 Total liabilities and stockholders’ equity $ 914,099 $ 748,956

An Acadia Pharmaceuticals drug that’s already approved for treating psychosis in Parkinson’s disease patients could not beat a placebo in a pivotal schizophrenia study, spelling the end of efforts to develop a medication the company once envisioned as having potential applications across a range of neurological disorders. The clinical trial failure announced Tuesday evaluated the drug, pimavanserin, as a treatment for negative symptoms of schizophrenia. The main goal was to show a change in score according to an assessment used to measure the disease’s symptoms. According to the preliminary results, the pimavanserin arm achieved a numerical change in score that was similar to the change reported in a prior Phase 2 study. But San Diego-based Acadia also said the placebo effect in Phase 3 was higher. Consequently, the score change in the Phase 3 trial was not enough to be statistically significant. Pimavanserin was initially approved in 2016, a regulatory decision that made the drug the first treatment for the hallucinations and delusions experienced by some Parkinson’s patients. In this indication, the once-daily capsule is marketed under the brand name Nuplazid. The Acadia drug is a small molecule designed to bind to 5-HT2A, a serotonin receptor that plays a role in psychosis. Parkinson’s psychosis was just the lead indication for the drug. The company conducted additional clinical trials to support potential label expansion. In 2021, the FDA rejected Acadia’s application for pimavanserin in dementia-related psychosis, pointing to a lack of statistical significance in some patient subgroups. The following year, FDA turned an application for the drug as a treatment for psychosis in Alzheimer’s patients, asking the company to conduct another clinical trial. Acadia instead discussed a resubmission based on additional analyses of existing clinical data. That resubmission was also rejected, leading the company to cease efforts to develop the drug for Alzheimer’s psychosis. The latest failure in schizophrenia marks the last time the company will try to expand pimavanserin’s label beyond Parkinson’s psychosis. “We are disappointed the trial did not meet its primary endpoint given the significant unmet need in patients with negative symptoms of schizophrenia,” Acadia CEO Steve Davis said in a prepared statement. “We will continue to analyze these data with our scientific advisors, but we do not intend to conduct any further clinical trials with pimavanserin.” Nuplazid accounted for $549.2 million in revenue in 2023, a 6.1% increase over sales of the product in 2022, according to Acadia’s financial reports. The company also has a new contributor to revenue, Daybue. A year ago, this drug became the first FDA-approved treatment for Rett syndrome, a rare genetic neurological disorder. In 2023, Daybue accounted for $177.2 million in sales. Acadia reported a cash position of $438.9 million at the end of 2023. In addition to supporting commercialization of Nuplazid and Daybue, the capital will also support development of a pipeline of drug candidates in various stages of development for central nervous system disorders and rare diseases. After the wind down of pimavanserin, Acadia’s next most advanced program is ACP-101, a drug in Phase 3 clinical trials for treating the insatiable appetite caused by the rare disease Prader-Willi syndrome. The pipeline also includes ACP-204, a novel molecule that has reached Phase 2 testing in Alzheimer’s disease psychosis.