ACADIA Pharmaceuticals, Inc.

SAN DIEGO--(BUSINESS WIRE)--Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced the appointment of Jonathan M. Poole to its Board of Directors, effective March 3, 2026. Mr. Poole will serve on the Company’s Audit Committee and brings deep biopharmaceutical finance leadership experience in supporting global growth and innovation across multiple therapeutic areas and modalities. “Jonathan brings extensive biotechnology corporate leadership experience and a proven track record of supporting significant business growth across complex, global organizations,” said Stephen R. Biggar, M.D., Ph.D., Chairman of Acadia’s Board of Directors. “His appointment further strengthens the Board’s financial and operational expertise at a pivotal time as Acadia continues to execute its growth strategy, advance its commercial portfolio and pipeline, and deliver sustained, long‑term value for shareholders.” “I am honored to join Acadia’s Board of Directors,” said Mr. Poole. “I look forward to working with the Board and management team to support the Company’s mission of developing and delivering innovative therapies for patients with significant unmet needs.” Since March 2020, Mr. Poole has served as Senior Vice President, Finance at Vertex Pharmaceuticals, Inc., where he has led the global finance team during a period of significant R&D portfolio and global commercial growth and diversification as well as the execution and integration of multiple acquisitions. Previously, from March 2018 to March 2020, Mr. Poole served as Chief Financial Officer of Evelo Biosciences, Inc., a biotechnology company developing orally delivered product candidates for inflammation and cancer. Prior to that, from April 2014 to March 2018, he served as Chief Financial Officer of Genocea Biosciences, Inc., a biotechnology company focused on developing infectious disease and neoantigen cancer vaccines. From July 2018 through March 2020, Mr. Poole also served as a director of Codiak Biosciences, Inc., where he was Chair of the Audit Committee. Mr. Poole received a B.Sc. in Biological Sciences from Durham University in the United Kingdom and an M.B.A. from London Business School. About Acadia Pharmaceuticals Acadia is committed to turning scientific promise into meaningful innovation that makes the difference for underserved neurological and rare disease communities around the world. Our commercial portfolio includes the first and only FDA-approved treatments for Parkinson’s disease psychosis and Rett syndrome. We are developing the next wave of therapeutic advancements with a robust and diverse pipeline that includes mid- to late-stage programs in Alzheimer’s disease psychosis and Lewy body dementia psychosis, along with earlier-stage programs that address other underserved patient needs. At Acadia, we’re here to be their difference. For more information, visit us at acadia.com and follow us on LinkedIn and X . Contacts Investor Contact: Acadia Pharmaceuticals Inc. Al Kildani (858) 261-2872 ir@acadia-pharm.com Acadia Pharmaceuticals Inc. Jessica Tieszen (858) 261-2950 ir@acadia-pharm.com Media Contact: Acadia Pharmaceuticals Inc. Deb Kazenelson (818) 395-3043 media@acadia-pharm.com

Acadia is "committed to working constructively with EU regulators" following a recent European rejection for Rett syndrome drug Daybue, CEO Catherine Adam Owens said. Acadia Pharmaceuticals isn’t taking its recent rejection from the European Medicines Agency lying down, vowing to seek a re-examination of the decision from the regulator's Committee for Medicinal Products for Human Use (CHMP) that limits the reach of its Rett syndrome drug Daybue.The CHMP’s negative decision, issued Feb. 26, poked holes in Acadia's pivotal Lavender trial, which tested Daybue (trofinetide) against placebo in 187 female patients with Rett syndrome, a rare neurodevelopmental disorder. Now, after reviewing the CHMP’s rejection, Acadia has pledged to request a re-examination of the opinion, maintaining in a March 2 release that it now has a better understanding of the European regulator's concerns. “While we are disappointed by the CHMP’s recommendation to refuse approval, we continue to be encouraged by the meaningful benefits trofinetide has demonstrated for people living with Rett syndrome,” CEO Catherine Owen Adams said in a statement. “We remain committed to working constructively with EU regulators to explore next steps and to bring this therapy to patients.” In Acadia's study, patients were randomized to receive Daybue or placebo over 12 weeks and primarily evaluated through a caregiver assessment, the Rett Syndrome Behaviour Questionnaire (RSBQ), and a physician Clinical Global Impression–Improvement (CGI-I) scale. The FDA signed off on Daybue in 2023 based on this study, as did Canada’s health regulators in 2024 and Israel’s health ministry more recently. The CHMP, however, took the opinion that based on the trial, “the benefits of Daybu in the treatment of Rett syndrome have not been demonstrated,” the agency explained in its rejection, using the drug's would-be commercial moniker in the EU. The EMA found that the size of the drug’s effects after 12 weeks was “too small” and not expected to be clinically meaningful for patients, pointing out that the trial did not assess “several key symptoms of Rett syndrome.” Acadia’s long-term effectiveness data, meanwhile, were “complicated” by the “large” number of patients who withdrew from the study, the agency said.Ultimately, 154 out of the 187 patients in Lavender chose to roll over to the company’s open-label extension study, while the company's long-term study, Lilac-2, consisted of 77 patients who had previously completed both trials. European regulators also determined that the proposed Daybue use was “not representative of the patients included in the main study, as the latter did not involve patients across the different disease stages.”Acadia had been planning for a re-examination since Feb. 2, when the CHMP informed the company of a “negative trend vote” during a closed-door meeting on its application. “While the negative trend vote is disappointing and not what we hoped for, we believe the strong data that supported the approval of trofinetide for the treatment of Rett syndrome in the United States, Canada, and Israel speak to the meaningful benefits that trofinetide can deliver,” Owen Adams said at the time, pointing to more than 1,000 patients globally across a broad age range who are currently on treatment. She also cited a U.S.-based real-world experience study that shows outcomes that “closely mirror” the impact observed in Acadia's clinical trials.   EU legislation allows a drug applicant to request a re-examination within 15 calendar days of the official opinion, Acadia further explained at the time. The company must then submit its grounds for the request within 60 days, after which the CHMP has up to 60 days to examine its opinion again. Acadia’s Daybue is the world's first drug specifically approved to treat Rett syndrome, a complex genetic disorder that occurs in one of every 10,000 to 15,000 female births worldwide. In the U.S., Acadia estimates the disease affects around 6,000 to 9,000 patients. In Europe, where the company first filed Daybue for approval in January 2025, the drug could help about 9,000 to 12,000 patients with the disease, the company has said. In 2025, the company achieved Owen Adams’ goal of locking down over $1 billion in annual sales for the first time, based on $391 million in Daybue sales and $692 million from older Parkinson’s disease psychosis med Nuplazid. The company is expecting between $460 million and $490 million from Daybue this year as it grows its oral solution version of the drug, a powder called Daybue Stix, following the new product's December FDA approval.

Plus, news about Acadia Pharmaceuticals, Poplar Therapeutics and Antiverse: 🅰️ RTW’s Prolium gets $50M: The New York-based biotech Prolium Bioscience has $50 million in Series A funds from RTW Investments. The funding was announced 14 months after Prolium’s T cell engager deal with China-based Keymed Biosciences. That pact got Prolium a CD20xCD3 bispecific, rebranded as PRO-203, that will enter Phase 1/2 in systemic sclerosis next quarter. — Kyle LaHucik 🔬 COUR reports Phase 2a data: One year into the study, the biotech said its candidate CNP-104 demonstrated “durable clinical effects” in patients with a chronic liver autoimmune disease called primary biliary cholangitis. The company reported results for biomarkers indicative of disease progression, including liver stiffness and function. CEO Dannielle Appelhans said the readout will “inform our ongoing evaluation of the program’s development strategy, including potential collaboration with a strategic partner.” — Nicole DeFeudis 🏢 Acadia to ask EU regulators to re-examine Rett syndrome drug: The move follows the Committee for Medicinal Products for Human Use (CHMP) giving a negative opinion on trofinetide. CHMP said Acadia’s clinical data didn’t capture “all core symptoms” of Rett syndrome. Trofinetide is marketed in the US as Daybue. — Anna Brown 💰 Chip Baird’s latest bet lands another $45M: The former 2seventy bio and bluebird bio executive has collected a Series A extension for Poplar Therapeutics just two months after the startup debuted with $50 million . The new total of $95 million will help the Cambridge, MA-based biotech develop a food allergy candidate known as PHB-050. — Kyle LaHucik 🏴󠁧󠁢󠁷󠁬󠁳󠁿 Welsh biotech nabs $9.3M: Antiverse got the Series A love from Soulmates Ventures, DOMiNO Ventures and others. The startup has raised $20 million to date for its AI antibody design work, and it has a research agreement with the Cystic Fibrosis Foundation. — Kyle LaHucik