Acadia Pharmaceuticals to Present Data at the 2026 American Academy of Neurology Annual Meeting

Published: 2026-04-20 09:28:11 ET ACAD

Published on 04/20/2026 at 09:28 am EDT

SAN DIEGO - Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) announced that it will present multiple original data presentations spanning its portfolio at the 2026 American Academy of Neurology (AAN) Annual Meeting, taking place April 18-22, 2026 in Chicago, IL.

The Company will present real-world data from a sub-group analysis of the ongoing, Phase 4, prospective, observational, open-label LOTUS study evaluating the benefits and tolerability of DAYBUE (trofinetide) in adults with Rett syndrome in routine clinical practice. In support of NUPLAZID (pimavanserin) in Parkinson's disease psychosis (PDP), Acadia will present exploratory analyses evaluating heterogeneity in treatment response trajectories and the impact of baseline sleep disturbances among PDP patients treated with pimavanserin. The Company is also debuting translational and pharmacokinetic research supporting the continued development of ACP-711, an investigational drug, for essential tremor. Collectively, these data reflect Acadia's ongoing commitment to advancing scientific knowledge across a wide range of neurological conditions.

About DAYBUE (trofinetide) and DAYBUE STIX (trofinetide)

Trofinetide is a synthetic analog of the N-terminal tripeptide of insulin-like growth factor-1. The mechanism by which trofinetide exerts therapeutic effects in patients with Rett syndrome is unknown. In animal studies, trofinetide has been shown to increase branching of dendrites and synaptic plasticity signals.

Indication and Important Safety Information for DAYBUE (trofinetide) and DAYBUE STIX (trofinetide)

Indication

DAYBUE and DAYBUE STIX are indicated for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older.

Important Safety Information

Warnings and Precautions

Diarrhea: In a 12-week study and in long-term studies, 85% of patients treated with DAYBUE experienced diarrhea. In those treated with DAYBUE, 49% either had persistent diarrhea or recurrence after resolution despite dose interruptions, reductions, or concomitant antidiarrheal therapy. Diarrhea severity was mild or moderate in 96% of cases. In the 12-week study, antidiarrheal medication was used in 51% of patients treated with DAYBUE.

Advise patients to stop laxatives before starting DAYBUE or DAYBUE STIX. If diarrhea occurs, patients should notify their healthcare provider, consider starting antidiarrheal treatment, and monitor hydration status and increase oral fluids, if needed. Interrupt, reduce dose, or discontinue DAYBUE or DAYBUE STIX if severe diarrhea occurs or if dehydration is suspected.

Vomiting: In a 12-week study, vomiting occurred in 29% of patients treated with DAYBUE and in 12% of patients who received placebo.

Patients with Rett syndrome are at risk for aspiration and aspiration pneumonia. Aspiration and aspiration pneumonia have been reported following vomiting in patients being treated with DAYBUE. Interrupt, reduce dose, or discontinue DAYBUE or DAYBUE STIX if vomiting is severe or occurs despite medical management.

Weight Loss: In the 12-week study, 12% of patients treated with DAYBUE experienced weight loss of greater than 7% from baseline, compared to 4% of patients who received placebo. In long-term studies, 2.2% of patients discontinued treatment with DAYBUE due to weight loss. Monitor weight and interrupt, reduce dose, or discontinue DAYBUE or DAYBUE STIX if significant weight loss occurs.

Adverse Reactions: The common adverse reactions (=5% for DAYBUE-treated patients and at least 2% greater than in placebo) reported in the 12-week study were diarrhea (82% vs 20%), vomiting (29% vs 12%), fever (9% vs 4%), seizure (9% vs 6%), anxiety (8% vs 1%), decreased appetite (8% vs 2%), fatigue (8% vs 2%), and nasopharyngitis (5% vs 1%).

Drug Interactions: Effect of DAYBUE and DAYBUE STIX on other Drugs

Trofinetide, a weak inhibitor of CYP3A and an inhibitor of P-gp, can increase the plasma concentrations of CYP3A and/or P-gp substrates (e.g., loperamide), which may increase the risk of adverse reactions associated with these substrates.

Closely monitor patients when DAYBUE or DAYBUE STIX is administered concomitantly with sensitive CYP3A and/or P-gp substrates for which a minimal increase in substrate plasma concentration (i.e., drugs with a narrow therapeutic index) may lead to serious adverse reactions.

Use in Specific Population: Renal Impairment

DAYBUE and DAYBUE STIX are not recommended for patients with severe renal impairment.

DAYBUE is available as an oral solution (200 mg/mL).

DAYBUE STIX for oral solution powder is available in 5,000 mg, 6,000 mg, and 8,000 mg packets.

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 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.

Contact:

Al Kildani

Tel: (858) 261-2872

Email: [email protected]

Deb Kazenelson

Tel: (818) 395-3043

Email: [email protected]