Pharma Focus Asia

ACADIA Pharmaceuticals and Neuren Pharmaceuticals Announce Exclusive License Agreement

Tuesday, August 07, 2018

ACADIA Pharmaceuticals Inc. and Neuren Pharmaceuticals Limited announced today that they have entered into an exclusive North American License Agreement for the development and commercialization of trofinetide for Rett syndrome and other indications. Neuren retains rights to develop and commercialize trofinetide for all indications outside of North America.

ACADIA plans to initiate a Phase 3 randomized, double-blind placebo-controlled study evaluating trofinetide in the second half of 2019 following completion of additional manufacturing activities. This study will evaluate trofinetide and placebo in approximately 180 girls with Rett syndrome and will measure the Rett Syndrome Behavior Questionnaire (RSBQ), a caregiver assessment, and the Clinical Global Impression of Improvement (CGI-I), a physician assessment, as co-primary efficacy endpoints.

“A potential treatment for Rett syndrome is a perfect fit with ACADIA’s mission to develop novel therapies to improve the lives of patients with central nervous system disorders,” said Serge Stankovic, M.D., M.S.P.H, Executive Vice President, Head of Research and Development at ACADIA. “Today there are no approved treatments for the girls and women suffering from Rett syndrome. We look forward to initiating a Phase 3 study in the second half of 2019 to further explore the potential benefits of trofinetide for patients and their caregivers.”

Neuren Executive Chairman Dr. Richard Treagus commented, “We are delighted to be partnering with ACADIA in North America. ACADIA’s team has a proven record in developing and commercializing medicines in central nervous system disorders with no approved therapies and high unmet needs. ACADIA’s additional capabilities and resources will immediately make a very significant difference, enabling us to advance our shared goal of developing this novel treatment option for Rett syndrome patients.”

Steve Kaminsky, Ph.D., Chief Science Officer of, commented, “ is grateful to Neuren for their dedication to the development of trofinetide. ACADIA’s commitment to advance trofinetide to Phase 3 brings us closer to the first potential treatment for Rett syndrome with a drug designed to address the underlying biology and improve the lives of those suffering from the condition.”

Trofinetide is a novel synthetic analog of the amino?terminal tripeptide of IGF-1 designed to treat the core symptoms of Rett syndrome by reducing neuroinflammation and supporting synaptic function. In the central nervous system, IGF-1 is produced by both of the major types of brain cells – neurons and glia. IGF-1 in the brain is critical for both normal development and for response to injury and disease1,2. Trofinetide has been granted U.S. Food and Drug Administration (FDA) Fast Track Status and Orphan Drug Designation in the U.S. and Europe.

Neuren conducted a Phase 2 double-blind placebo-controlled dose ranging study in girls aged 5 to 15 years with Rett syndrome, in which statistically significant and clinically meaningful improvement was demonstrated on the RSBQ and the CGI-I. This followed positive trends observed in an earlier Phase 2 trial in adolescents and adults aged 16 to 45 years with Rett syndrome3. In addition, Neuren has completed an exploratory study in Fragile X syndrome.

Dr. Daniel Glaze, Medical Director at the Blue Bird Circle Rett Center, Texas Children’s Hospital commented, “The trofinetide Phase 2 results in Rett syndrome are very promising in terms of both safety and clinical benefit. For many families, these improvements would provide meaningful improvement in their child’s quality of life.”

Under the terms of the License Agreement, Neuren is eligible to receive US $10 million upfront plus potential milestone payments of up to US $455 million. In addition, Neuren is eligible to receive tiered, escalating, double-digit percentage royalties on net sales of trofinetide in North America and one third of the market value of any Rare Pediatric Disease Priority Review Voucher, if awarded by the U.S. FDA upon approval of a New Drug Application for trofinetide. The potential milestone payments to Neuren consist of US $105 million subject to achievement of development milestones in Rett syndrome and Fragile X syndrome and up to US $350 million subject to achievement of thresholds of annual net sales of trofinetide in North America. ACADIA will fund and execute the remaining development for trofinetide in Rett syndrome in North America, except for the completion by Neuren of certain in-progress preparatory activities. ACADIA and Neuren will form a Joint Steering Committee to direct the development of trofinetide in all indications, including the next clinical trial for Fragile X syndrome. Any data and regulatory filings generated by ACADIA or Neuren may be used by either party for the development and commercialization of trofinetide in their respective territories. ACADIA has a right of first negotiation to acquire a license to develop and commercialize trofinetide outside North America. Neuren has an obligation not to develop a competing product in indications for which ACADIA develops and commercializes trofinetide.

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