ACP-319

- 2Q22 net sales of $134.6 million, a 17% increase over 2Q21 - NDA submitted for trofinetide for the treatment of Rett syndrome SAN DIEGO--(BUSINESS WIRE)-- Acadia Pharmaceuticals Inc. (Nasdaq: ACAD), today announced its financial results for the second quarter ended June 30, 2022. “In the second quarter of 2022, NUPLAZID net sales increased 17% year-over-year,” said Steve Davis, Chief Executive Officer. “Last month, we submitted an NDA for our second potential commercial product, trofinetide which would be the first FDA-approved treatment for Rett syndrome. Together with a strong balance sheet, our late and early-stage portfolio, including our Phase 3 program for pimavanserin for the treatment of the negative symptoms of schizophrenia, and a continued focus on strategic business development, we are well-positioned for long-term growth.” Company Updates Submitted a New Drug Application (NDA) to the FDA for trofinetide as a potential treatment for Rett syndrome in adults and pediatric patients two years of age and older. Trofinetide could be Acadia’s second commercial product and the first and only approved treatment in the U.S. for Rett syndrome. Received a Complete Response Letter from the FDA for the resubmitted supplemental NDA for pimavanserin for the treatment of hallucinations and delusions associated with Alzheimer’s disease psychosis (ADP). Presented two late-breaker posters at the American Society of Clinical Psychopharmacology (ASCP) Annual Meeting in June 2022, adding to the growing body of evidence of safety of pimavanserin in the treatment of Parkinson’s disease psychosis. Acadia is developing an internally discovered new molecule, ACP-204, currently in Phase 1. ACP-204 builds upon the learnings of pimavanserin in the treatment of neuropsychiatric symptoms. Acadia is discontinuing the development of ACP-044 in acute and chronic pain, based on evaluation of the final data set from a previously completed Phase 2 bunionectomy study, and ACP-319 an M1 PAM modulator, based on a pro does not support advancement to Phase 2. Financial Results Revenue Net sales of NUPLAZID® (pimavanserin) were $134.6 million for the three months ended June 30, 2022, an increase of 17% as compared to $115.2 million reported for the three months ended June 30, 2021. For the six months ended June 30, 2022 and 2021, Acadia reported net product sales of $250.0 million and $221.8 million, respectively. Research and Development Research and development expenses for the three months ended June 30, 2022 were $75.6 million, compared to $56.9 million for the same period of 2021. The increase was primarily due to increased costs of our development activities for ACP-044, ACP-319 and other early stage programs. For the six months ended June 30, 2022 and 2021, research and development expenses were $204.5 million and $113.9 million. The increase was mainly due to the $60 million upfront payment made to Stoke Therapeutics for the license and collaboration agreement in 2022. Selling, General and Administrative Selling, general and administrative expenses for the three months ended June 30, 2022 were $89.9 million, compared to $96.8 million for the same period of 2021. For the six months ended June 30, 2022 and 2021, selling, general and administrative expenses were $186.6 million and $208.5 million, respectively. The decrease in both periods was primarily due to decreased advertising and promotional costs and personnel expenses. Net Loss For the three months ended June 30, 2022, Acadia reported a net loss of $34.0 million, or $0.21 per common share, compared to a net loss of $43.9 million, or $0.27 per common share, for the same period in 2021. The net losses for the three months ended June 30, 2022 and 2021 included $20.5 million and $22.0 million, respectively, of non-cash stock-based compensation expense. For the six months ended June 30, 2022, Acadia reported a net loss of $147.1 million, or $0.91 per common share, compared to a net loss of $110.3 million, or $0.69 per common share, for the same period in 2021. The increase was mainly due to the $60 million upfront payment made to Stoke Therapeutics for the license and collaboration agreement in 2022. The net losses for the six months ended June 30, 2022 and 2021 included $35.5 million and $35.2 million, respectively, of non-cash stock-based compensation expense. Cash and Investments At June 30, 2022, Acadia’s cash, cash equivalents, and investment securities totaled $436.4 million, compared to $520.7 million at December 31, 2021. 2022 Financial Guidance NUPLAZID net sales guidance is updated to $510 to $540 million from the previous range of $510 to $560 million. GAAP R&D guidance is updated to $340 to $360 million from $355 to $375 million, which includes approximately $25 million of stock-based compensation expense. GAAP SG&A guidance of $360 to $380 million is reiterated, which includes approximately $45 million of stock-based compensation expense. Conference Call and Webcast Information 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. The registration link will also be available on Acadia’s website, under the investors section and will be archived there until September 5, 2022. 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. In addition, Acadia is developing pimavanserin in other neuropsychiatric conditions. About Trofinetide Trofinetide is an investigational drug. It is a novel synthetic analog of the amino‐terminal tripeptide of IGF-1 designed to treat the core symptoms of Rett syndrome by potentially reducing neuroinflammation and supporting synaptic function. Trofinetide is thought to stimulate synaptic maturation and overcome the synaptic and neuronal immaturities that are characteristic of Rett syndrome pathophysiology. In the central nervous system, IGF-1 is produced by both of the major types of brain cells – neurons and glia. IGF-1 in the brain is critical for both normal development and for response to injury and disease. Trofinetide has been shown to inhibit the production of inflammatory cytokines, inhibit the overactivation of microglia and astrocytes, and increase the amount of available IGF-1 that can bind to IGF-1 receptors. Trofinetide has been granted Fast Track Status and Orphan Drug Designation for Rett syndrome and has also been granted Rare Pediatric Disease (RPD) designation by the FDA. About Acadia Pharmaceuticals Acadia is advancing breakthroughs in neuroscience to elevate life. For more than 25 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 approved therapy for hallucinations and delusions associated with Parkinson’s disease psychosis. Our clinical-stage development efforts are focused on treating the negative symptoms of schizophrenia, Rett syndrome and neuropsychiatric symptoms in central nervous system disorders. For more information, visit us at and follow us on LinkedIn and Twitter. Forward-Looking Statements Statements in this press release that are not strictly historical in nature are forward-looking statements. These statements include, but are not limited to, statements related to: the potential opportunity for future growth in sales of NUPLAZID; the timing of ongoing and future clinical studies for pimavanserin; the development and commercialization of trofinetide; and guidance for full-year 2022 NUPLAZID net sales for Parkinson’s disease psychosis only and certain expense line items. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties. Actual events or results may differ materially from those projected in any of such statements due to various factors, including the uncertainty of future commercial sales and related items that would impact net sales during 2022, the risks and uncertainties inherent in drug development, approval and commercialization, and the fact that past results of clinical trials may not be indicative of future trial results. For a discussion of these and other factors, please refer to Acadia’s annual report on Form 10-K for the year ended December 31, 2021 as well as Acadia’s subsequent filings with the Securities and Exchange Commission. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. All forward-looking statements are qualified in their entirety by this cautionary statement and Acadia undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof, 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, 2022 2021 2022 2021 Revenues Product sales, net $ 134,563 $ 115,221 $ 250,031 $ 221,775 Total revenues 134,563 115,221 250,031 221,775 Operating expenses Cost of product sales, license fees and royalties (1) 2,667 5,206 5,617 9,898 Research and development (1) 75,646 56,935 204,501 113,908 Selling, general and administrative (1) 89,901 96,789 186,580 208,450 Total operating expenses 168,214 158,930 396,698 332,256 Loss from operations (33,651 ) (43,709 ) (146,667 ) (110,481 ) Interest income, net 580 133 685 333 Other (loss) income (497 ) 178 (157 ) 323 Loss before income taxes (33,568 ) (43,398 ) (146,139 ) (109,825 ) Income tax expense 443 473 928 494 Net loss $ (34,011 ) $ (43,871 ) $ (147,067 ) $ (110,319 ) Net loss per common share, basic and diluted $ (0.21 ) $ (0.27 ) $ (0.91 ) $ (0.69 ) Weighted average common shares outstanding, basic and diluted 161,654 160,421 161,443 160,217 (1) Includes the following stock-based compensation expense Cost of product sales, license fees and royalties $ 346 $ 423 $ 669 $ 586 Research and development $ 7,232 $ 7,319 $ 12,696 $ 12,149 Selling, general and administrative $ 12,934 $ 14,263 $ 22,110 $ 22,454 ACADIA PHARMACEUTICALS INC. CONDENSED CONSOLIDATED BALANCE SHEETS (in thousands) June 30, 2022 December 31, 2021 (unaudited) Assets Cash, cash equivalents and investment securities $ 436,351 $ 520,706 Accounts receivable, net 67,953 64,366 Interest and other receivables 936 978 Inventory 6,327 7,881 Prepaid expenses 20,952 23,892 Total current assets 532,519 617,823 Property and equipment, net 7,016 8,047 Operating lease right-of-use assets 57,417 58,268 Restricted cash 5,770 5,770 Long-term inventory 6,205 6,217 Other assets 3,839 3,997 Total assets $ 612,766 $ 700,122 Liabilities and stockholders’ equity Accounts payable $ 11,854 $ 6,876 Accrued liabilities 105,827 89,192 Total current liabilities 117,681 96,068 Operating lease liabilities 54,693 56,126 Other long-term liabilities 5,544 7,034 Total liabilities 177,918 159,228 Total stockholders’ equity 434,848 540,894 Total liabilities and stockholders’ equity $ 612,766 $ 700,122