ACADIA Pharmaceuticals, Inc.

Neuren Pharmaceuticals to receive a third of the $150 million proceeds from Acadia Pharmaceuticals' sale of a Rare Pediatric Disease priority review voucher (PRV) granted by the FDA, following the approval of Daybue in April 2023.

Stoke Therapeutics released its Q3 2023 Form 10-Q, reporting $4.9M in revenue (+$1.6M QoQ) and a net loss of $26.4M. The company is advancing clinical programs for zorevunersen (Dravet syndrome) and STK-002 (ADOA), with regulatory updates planned for H2 2024. Stoke faces challenges including clinical trial uncertainty and regulatory approval risks.

Acadia Pharmaceuticals sells its Rare Pediatric Disease Priority Review Voucher for $150 million, with one-third of the proceeds to be paid to Neuren Pharmaceuticals. The funds will support commercial operations, R&D programs in central nervous system and rare diseases, and future business development.

Chapters cover methodology, executive summary, market variables, segmentation by disease, molecule, biological target, mechanism, and region, and competitive landscape.

Health Canada approves Daybue (trofinetide) for Rett syndrome in adults and children 2+ years old, making it the first approved treatment in Canada. Acadia Pharmaceuticals aims to provide the treatment quickly, with plans to seek approvals in Europe and Japan. Daybue targets inflammation and nerve cell connections, showing improvements in behavior and communication in clinical trials.

Health Canada approves DAYBUE™ (trofinetide) as the first treatment for Rett syndrome in Canada, following positive Phase 3 LAVENDER study results.

Axsome Therapeutics' Auvelity, launched in 2022 for MDD, generated $118.4M in H1 2024, up 172.8% YoY. Auvelity is being studied for AD agitation and smoking cessation. Sunosi, acquired from Jazz in 2022, contributed $42.2M in H1 2024, up 35.2% YoY. AXSM's pipeline includes AXS-07 for migraine, AXS-14 for fibromyalgia, and AXS-12 for narcolepsy. Competition from Acadia and Jazz's other drugs poses challenges.

Clinical trials for Rett syndrome need better outcome measures to objectively confirm symptom improvement. Existing measures lack specificity for motor and autonomic functions. Acadia Pharmaceuticals' Daybue became the first FDA-approved treatment, while Anavex Life Sciences' ANAVEX2-73 failed in Phase III. Experts advocate for a holistic approach including behavioural and biomarker assessments. Video motor assessments and wearable biosensors are suggested for more objective evaluations.

Pre-ordering for epinephrine nasal spray (neffy) has launched for treating allergic reactions, including anaphylaxis. The FDA approved trofinetide (Daybue) for Rett syndrome, marking a milestone in treatment. A study in JAMA Pediatrics found not all screen time before bed negatively impacts sleep in children. Another study in The American Journal of Clinical Nutrition linked fish intake during pregnancy to reduced autism risk in children.

Acadia Pharmaceuticals' COMPASS-PWS Clinical trial, enrolling PWS patients aged 5-30 with hyperphagia, aims to evaluate carbetocin's safety and efficacy via nasal spray. The phase 3 study involves 170 participants in a double-blind setup, with key eligibility criteria including no recent food interventions or treatment studies. The trial includes 5 clinic visits and an optional 3-year open-label extension. For details, visit compasspws.com.