Acadia making plans to expand patient access to Daybue in Rett
New deal grants company rights to therapy outside North America
Acadia Pharmaceuticals has expanded its licensing agreement with Neuren Pharmaceuticals to commercialize Daybue (trofinetide) — the first and only therapy approved for Rett syndrome — outside of North America.
Daybue was originally developed by Neuren, which licensed its rights in North America to Acadia in 2018.
“This expanded worldwide agreement solidifies Acadia’s position as the global leader in addressing the unmet needs of people with Rett syndrome,” Steve Davis, Acadia’s president and CEO, said in a press release.
“We have successfully delivered Daybue, the first FDA-approved therapy that treats the core symptoms of Rett syndrome, and are deeply committed to broadening access to this important therapy for patients worldwide,” Davis said.
Acadia expects to submit an application seeking the approval of Daybue in Canada in the next 18 months. Plans for similar requests in Europe, Asia, and other regions are expected at a later date.
Acadia also acquires rights to Neuren’s NNZ-2591
Daybue, a liquid medication taken by mouth or a feeding tube, was approved in the U.S. in March. A support program called Acadia Connect is available for patients starting or continuing on the therapy.
The main ingredient in Daybue is an analogue of glypromate, a protein fragment occurring naturally in the brain that helps promote development of connections between nerve cells, which is impaired in Rett syndrome.
Under the terms of the new licensing agreement, Acadia also has secured the worldwide rights to Neuren’s investigational therapy NNZ-2591 for Rett and fragile X syndrome. Also a rare genetic disorder, fragile X is characterized by cognitive impairment, learning issues, and behavioral challenges.
NNZ-2591 also is being tested for neurological disorders that manifest in early childhood, including Prader-Willi syndrome (PWS) and Angelman syndrome. A Phase 2 trial of NNZ-2591 in PWS patients is underway.
The therapy is a lab-made version of cyclo-glycyl-proline (cGP) protein, a naturally occurring peptide — a chain of amino acids, the building blocks of proteins — that regulates the signaling of insulin-like growth factor 1, known as IGF-1. The treatment candidate is expected to normalize IGF-1, which is needed for proper brain development, and restore normal communication between brain cells.
Per the agreement’s terms, Neuren will receive an upfront payment of $100 million, and is eligible for potential milestone and royalty payments for Daybue and NNZ-259.
Neuren also is eligible to receive additional payments upon the achievement of certain commercial milestones outside of North America, and to a percentage of sales. No changes have been made regarding the previous license agreement for Daybue in North America.
The potential future payments to Neuren related to NNZ-2591 in Rett syndrome and fragile X syndrome are in conformity with those of Daybue in and outside North America, the companies noted.
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