Trofinetide

Acadia Pharmaceuticals is scrapping development of intranasal carbetocin after the experimental Prader-Willi syndrome (PWS) drug failed a Phase III trial, sending company shares down nearly 12% on Wednesday.The decision scuttles Acadia's effort to bring a second treatment option to patients struggling with the rare genetic disorder's hallmark symptom of insatiable hunger. Soleno Therapeutics' Vykat XR (diazoxide choline) became the first drug approved by the FDA specifically for hyperphagia in PWS this past March.Intranasal carbetocin, also known as ACP-101, is a synthetic analogue of oxytocin, a neuropeptide involved in regulating eating behaviour that is deficient in PWS patients.The 12-week COMPASS PWS trial tested a 3.2-mg three-times-daily dose in 175 children and adults ages 5 through 30 years old, measuring changes from baseline in the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) as the main outcome. Acadia reported no statistically significant improvement over placebo on the primary or any secondary measures.The outcome dashes hopes raised by an earlier Phase III study, CARE PWS, which had hinted at benefit with the same low dose, but not a higher one. Ahead of Wednesday's readout, analysts at Citizens JMP said investor expectations were "muted" for COMPASS PWS given the inverse dose response seen in the previous Phase III trial. Acadia has suggested that the lack of efficacy at the high dose may have been due to activity on vasopressin receptors, which offsets the beneficial activity driven through oxytocin receptors.Still, the analysts noted that learnings from that earlier study were used to optimise COMPASS PWS, including focusing solely on the lower dose and extending the duration of treatment from 8 to 12 weeks, in hopes of replicating the efficacy signal. They were also projecting a "highly conservative" $250 000/year price at launch, compared to the roughly $500,000 expected average annual cost of Vykat XR."Despite this disappointment, Acadia is well-positioned to deliver long-term, sustainable growth supported by two approved products projected to generate over $1 billion in net sales in 2025, and a robust pipeline," remarked CEO Catherine Owen Adams. "Looking ahead, we anticipate seven Phase II or III study starts through 2026 and four data readouts by the end of 2027."Acadia's two marketed products are Nuplazid (pimavanserin) for Parkinson's disease psychosis and the Rett syndrome therapy Daybue (trofinetide).

Biomed Industries, Inc. Presents Four Pivotal Studies at AAIC 2025 Highlighting Breakthrough Therapies for Alzheimer’s, Rett Syndrome, and Obesity NA-831 is the only drug to date that has halted Alzheimer’s disease progression. Combining it with existing drugs like Donanemab could optimize therapeutic efficacy and reduce serious side effects.” — Dr. Lloyd L. Tran, CEO of Biomed SAN JOSE, CA, UNITED STATES, August 4, 2025 / EINPresswire.com / -- Biomed Industries, Inc., a leading biopharmaceutical innovator developing transformative therapies for neurological and metabolic diseases, today announced the presentation of four major scientific papers at the Alzheimer’s Association International Conference (AAIC), held July 27–31, 2025, in Toronto, Canada. The presentations featured Biomed’s next-generation oral therapies for Alzheimer’s disease, Rett syndrome, and obesity, with a focus on novel combination strategies designed to enhance safety, efficacy, and accessibility. Featured AAIC 2025 Presentations: 1. The End of the Amyloid Era? Evidence for a Paradigm Shift in the Quest to Treat Alzheimer’s Disease 2. A Phase 3 Clinical Protocol of NA-831 Combined with Donanemab in Early Alzheimer’s Disease: A Placebo-Controlled, Double-Blind Study 3. Associations Between Alzheimer’s Disease and Rett Syndrome: Clinical Trials of NA-831 and NA-921 4. Neuro-Metabolic Link Between Alzheimer’s Disease and Obesity: Clinical Evaluation of NA-831 and NA-931 1. PRESENTATION: The End of the Amyloid Era? A Paradigm Shift in Alzheimer’s Research For over three decades, the “amyloid hypothesis” has dominated Alzheimer’s disease (AD) research, asserting that amyloid-β accumulation is a primary driver of neurodegeneration. Biomed’s comprehensive analysis of Phase 3 trial data from seven anti-amyloid drugs — including aducanumab, lecanemab, donanemab, gantenerumab, bapineuzumab, crenezumab, and solanezumab — challenges this paradigm. Across all trials, both treatment and placebo groups exhibited similar cognitive decline, as measured by CDR-SB and ADAS-Cog. The average differences between treatment and placebo arms were minor and not clinically significant (CDR-SB: -0.25; ADAS-Cog: -0.79). Slope comparisons further revealed near-identical rates of decline between groups. “Our analysis of Phase 3 clinical trial data for seven anti-amyloid drugs, including FDA-approved Aducanumab, Lecanemab, and Donanemab, indicates that none could halt disease progression in a clinically meaningful way — and all carried serious safety concerns,” said Dr. Zung Tran, VP of Biostatistics and AI at Biomed. 2. PRESENTATION: Phase 3 Clinical Protocol of NA-831 Combined with Donanemab NA-831, Biomed’s lead oral candidate, is a first-in-class therapy that promotes neuroprotection, neurogenesis, and memory enhancement. Phase 2 trials demonstrated its disease-modifying potential with a favorable safety profile compared to traditional anti-amyloid drugs. In this upcoming Phase 3 study, NA-831 will be evaluated in combination with Donanemab, a recently FDA-approved monoclonal antibody, to explore synergistic effects. The goal is to lower Donanemab dosing, thereby reducing risks such as cerebral edema and microbleeds, while enhancing cognitive outcomes. “NA-831 is the only drug to date that has halted disease progression in Phase 2 trials,” said Dr. Lloyd Tran, CEO of Biomed Industries. “Combining it with existing drugs like Donanemab could optimize therapeutic efficacy and reduce serious side effects.” 3. PRESENTATION: Alzheimer’s and Rett Syndrome: Shared Mechanisms and Dual Therapeutic Potential Biomed also presented findings on NA-921, a structural analog of NA-831 developed for Rett syndrome, a rare X-linked neurodevelopmental disorder. NA-921 modulates MeCP2 expression, targeting the disorder’s core epigenetic dysfunction. A double-blind, placebo-controlled Phase 2/3 trial (ClinicalTrials.gov ID: NCT06849973) demonstrated promising results: Clinical Global Impression–Improvement (CGI-I) at week 12: NA-921: 3.60 | Placebo: 3.83 | P = 0.0020 | Effect size = 0.42 NA-921 was well tolerated, with a significantly improved safety profile compared to trofinetide: - Diarrhea: Trofinetide 82%, NA-921 24%, Placebo 19% - Vomiting: Trofinetide 29%, NA-921 9%, Placebo 11% - Fever: Trofinetide 9%, NA-921 5%, Placebo 4% These findings underscore a possible biological link between Alzheimer’s disease and Rett syndrome, opening new cross-indication opportunities for NA-831 and NA-921. 4. PRESENTATION: Neuro-Metabolic Connections: NA-831 and NA-931 in Alzheimer’s and Obesity Biomed’s data also revealed compelling evidence of a neuro-metabolic bridge between Alzheimer’s disease, diabetes, and obesity. In addition to NA-831’s CNS benefits, Biomed is advancing NA-931, an oral quadruple receptor agonist (IGF-1, GLP-1, GIP, and glucagon) designed to treat obesity. In a 13-week Multiple Ascending Dose (MAD) study: - Mean body weight reduction: Up to 13.8% at 150 mg daily, 12.4% greater than placebo - ≥12% weight loss achieved by 72% of NA-931-treated patients vs. 2% in placebo group NA-931 demonstrated a strong safety profile, with mild and transient GI-related adverse events, and no observed muscle loss. “Our pipeline shows how targeting interconnected pathways across the CNS and metabolic systems can unlock significant clinical potential,” said Michael Willis, VP of Business Development. “With six active programs across Alzheimer’s, ALS, Rett syndrome, stroke, obesity, and MASH, we are building a diversified platform to accelerate innovation and value creation.” ABOUT BIOMED INDUSTRIES, INC. Biomed Industries, Inc. is a pioneering biopharmaceutical company committed to developing novel therapeutics that address unmet medical needs. Its innovative research platform has produced treatments for conditions including Alzheimer’s disease, ALS, Traumatic Brain Injury, Major Depressive Disorder, Diabetes, Obesity, MASH, Stroke, and rare diseases such as Huntington Disease and Rett Syndrome. (Website: https://www.biomedind.com ) Michael Willis Biomed Industries, Inc. email us here Visit us on social media: LinkedIn X Biomed introduction video Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.