ACADIA Pharmaceuticals, Inc.

Neurocrine Biosciences' new FDA-approved non-steroidal drug, Crenessity, treats classic congenital adrenal hyperplasia (CAH), offering an alternative to steroid treatments with fewer side effects. Approved for both adults and children aged 4+, it reduces ACTH and adrenal androgens, with potential for significant revenue. Neurocrine plans to disclose pricing upon launch, with projections of $150 million in 2025 and peak sales of $1.47 billion.

A survey at the AES 2024 Annual Meeting found that 86% of prescribers reported titrating trofinetide improved tolerability in Rett syndrome patients, reducing diarrhea severity and treatment discontinuations, and enhancing quality of life for patients and caregivers.

Global Human Growth Hormones Market to reach USD 10 billion by 2030, driven by increasing prevalence of growth hormone deficiency, product approvals, and adoption of recombinant HGH. North America dominates the market, with key players including Novo Nordisk, Pfizer, and Eli Lilly. Market growth is fueled by advancements in biotechnology and rising demand for targeted therapies, despite challenges like stringent regulations and high treatment costs.

Kronos Bio to lay off 83% of workforce, CEO steps down; Idorsia in talks to sell blood pressure drug rights for $35M; PTC Therapeutics shelves failed ALS drug, plans to sell review voucher for $150M; Acadia Pharmaceuticals acquires global rights to Saniona's experimental medicine; Soleno Therapeutics' FDA decision on Prader-Wili syndrome drug delayed to March 2025; Alnylam Pharmaceuticals awaits FDA decision on ATTR-CM drug by March 23.

Stoke Therapeutics is advancing RNA-based drugs, focusing on up-regulating proteins to treat rare neurological diseases like Dravet syndrome. After overcoming safety issues, they are preparing for a phase 3 trial of zorevunersen, aiming to improve cognition and behavior in patients. Stoke emphasizes innovation in RNA science, addressing manufacturing and patient access challenges, and is collaborating with Acadia Pharmaceuticals for further development.

The neurodegenerative disorder therapeutics market is projected to grow from $18.53 billion in 2023 to $28.33 billion in 2028 at a CAGR of 9.0%, driven by factors like aging population, rising prevalence of neurodegenerative disorders, and strategic collaborations among pharmaceutical companies.

FDA approves first gene therapy, Kebilidi, for aromatic L-amino acid decarboxylase (AADC) deficiency, administered directly into the brain. Tested in 13 pediatric patients, Kebilidi showed gross motor function improvement in 8 of 12 patients at 48 weeks, with better outcomes for younger patients. The therapy, already approved in Europe, aims to replace the mutated DDC gene with a functional one, increasing dopamine production. The FDA's accelerated approval requires additional data, with long-term follow-up planned. PTC projects peak revenue of $266.3 million in 2026, and the approval includes a priority review voucher.

Acadia Pharmaceuticals sold its rare pediatric disease priority review voucher (PRV) for $150 million, with Neuren Pharmaceuticals set to receive $50 million. PRVs, awarded by the FDA for developing drugs for rare pediatric diseases, can be sold to accelerate drug reviews, highlighting their value in licensing deals.

Marvel Biosciences Corp. reported promising interim results from its MB204 study for Rett syndrome, showing near-normal social interaction restoration in a preclinical model. MB204 outperformed Trofinetide in most endpoints, with a distinct carry-over effect. Marvel aims to complete the study and publish the data.

Acadia Pharmaceuticals sold its priority review voucher for $150 million, with $50 million going to Neuren Pharmaceuticals. The voucher, awarded for FDA approval of Daybue for Rett syndrome, shortens drug review time from 10 to 6 months. The sale reflects high demand as the FDA plans to wind down the voucher program. Daybue sales reached $251.7 million in the first nine months of 2024, with 2024 sales forecasted at $340-350 million.