UCB announced an agreement to acquire the rights to Proximagen's Midazolam Nasal Spray (USL261), an anti-epilepsy drug (AED) developed as an intended rescue treatment for acute repetitive seizures (ARS, also known as serial, recurrent or cluster seizures) in patients with epilepsy.
USL261 is a novel investigational midazolam formulation, which has been specifically designed for intranasal delivery without active inhalation. It has been granted orphan drug designation and fast track designation by the United States Food and Drug Administration (FDA), reflecting the significant unmet need which currently exists for ARS rescue treatment.
Rectally administered benzodiazepines, such as diazepam, are commonly prescribed for the treatment of ARS. However, whilst this has traditionally provided patients and caregivers with a much-needed treatment option, this route of administration may be cumbersome and problematic in social settings. A treatment administered nasally would provide important additional treatment options.
USL261 has demonstrated strong results in a significant Phase 3 clinical trial program and the intention is to file USL261 as a New Drug Application (NDA) in the course of 2018.
"There is a real and pressing need for effective and convenient rescue treatments in ARS that rapidly end ongoing seizures as well as those that prevent seizure reoccurrence," explained Jean-Christophe Tellier, CEO of UCB. "Midazolam Nasal Spray has delivered strong Phase 3 results; our acquisition of this program, when approved, will expand and diversify the treatment choices we are able to provide to the epilepsy community, complementing our strong internal portfolio and building on our extensive knowledge, passion and expertise in the field of epilepsy."
UCB estimates that more than 150,000 people with refractory epilepsy also experience ARS.
These types of seizures pose multiple risks to patients, which include repeated emergency room related hospitalizations each year and possible evolution into status epilepticus, a potentially life-threatening seizure state.
"Rescue treatment options for acute repetitive seizures have historically been very limited. As a global leader in epilepsy, with a pioneering commitment to improving patient value, UCB was the natural choice to progress the development journey of midazolam nasal spray," said Bill Pullman, Chief Scientific Officer and President, Proximagen. "In making this important new medicine available, following approval, UCB will be delivering an effective rescue treatment option for patients and caregivers living with ARS. I'd like to take this opportunity to thank our investigators, along with the teams at Upsher-Smith and Proximagen who have brought us to this major milestone," said Mark Evenstad, Executive Chairman of ACOVA, parent company of Proximagen.
UCB believes USL261 has the potential to complement its already successful portfolio of epilepsy medicines, significantly improving its ability to provide additional treatment choice and value to millions of people living with poorly controlled seizures.