Cypress Bioscience, Inc., a pharmaceutical company engaged in the development of innovative drugs to treat central nervous system (CNS) disorders, announced that it has acquired patent rights and technology related to a novel, intranasal formulation of carbetocin, a potential breakthrough treatment for the core symptoms of autism. The therapy, which was developed by Marina Biotech, Inc., is expected to further strengthen Cypress’ CNS pipeline.
Under the terms of the agreement, Cypress will make an upfront payment to Marina Biotech of $750,000. Subject to certain late-stage clinical and regulatory milestones, including approval in the United States, Cypress may make up to $27 million in additional payments to Marina Biotech. Cypress will also fund all continuing development activities and pay single-digit royalties to Marina Biotech based on commercial sales, if any.
“We are enthusiastic about this acquisition, which exemplifies our strategy to build one of the highest quality CNS pipelines in the industry,” said Jay D. Kranzler, MD, PhD, chairman and chief executive officer of Cypress Bioscience. “Given the number of autism cases in children, we believe that the addition of this novel therapy will enable us to address an important unmet medical need. The intranasal formulation of carbetocin is based on well-established pharmacology, and we believe that this therapy could be a breakthrough in the treatment of the core symptoms of autistic spectrum disorder. We plan to work expeditiously and expect to announce details regarding the clinical program in the coming months.”
“Over the past decade, the rates of diagnosis of autistic spectrum disorders have increased by more than 50 per cent,” said Christopher McDougle, MD, Albert E. Sterne professor and chairman of the Department of Psychiatry, Indiana University School of Medicine, Indianapolis. “Carbetocin represents a potential treatment that, for the first time, targets the core symptoms of autism, particularly the social impairments and repetitive behaviours associated with this devastating disease. If successful, carbetocin could revolutionize the treatment of autism, thereby improving the lives of those individuals and families affected by this condition.”
Carbetocin is a synthetic version of the endogenous hormone, oxytocin, modified for greater serum half-life. This compound was originally developed by Ferring Pharmaceuticals and is sold in multiple countries outside of the US solely in a parenteral form for the treatment of postpartum haemorrhage. Marina Biotech, with its expertise in intranasal peptide delivery originating from its predecessor company, Nastech Pharmaceuticals, developed an intranasal formulation of this agent with the goal of assessing its efficacy for the treatment of autism. Phase 1 studies have been completed with this novel formulation.
Autistic spectrum disorders are developmental disorders that affect one in 110 children in the US, per recent surveys completed by the Centers for Disease Control2. These disorders are characterized by social impairments, repetitive behaviours, and, in more severe subgroups, by language and communication deficits as well as intellectual impairments2,3. The social impairments can take the form of abnormal eye contact and body language, a lack of age-appropriate peer relationships, and a lack of social reciprocity. The spectrum of repetitive behaviours can include restricted interests, a need for rigid routines or rituals, stereotypes, and odd preoccupations. Both of these core symptoms have profound effects upon the lives of affected individuals and their caregivers. The cost to society on the whole in the US alone is estimated at over $35 billion per yea.
Marina Biotech (formerly known as MDRNA, Inc.) is a biotechnology company focused on the development and commercialization of therapeutic products based on RNA interference (RNAi).
Cypress Bioscience is a pharmaceutical company dedicated to the development of innovative drugs targeting large unmet medical needs for patients suffering from a variety of disorders of the central nervous system.