Rare Daily Staff
Marinus Pharmaceuticals, a company focused on rare genetic epilepsies and seizure disorders, entered into collaboration and exclusive license agreement with Finnish pharma Orion whereby Orion received exclusive rights to commercialize the oral and intravenous (IV) dose formulations of ganaxolone in the European Economic Area, United Kingdom and Switzerland for CDKL5 deficiency disorder, tuberous sclerosis complex, and refractory status epilepticus.
“Orion has a strong presence across Europe in rare neurological disorders, brings an extensive commercial infrastructure and is an ideal partner to introduce ganaxolone in Europe,” said Scott Braunstein, CEO of Marinus. “This collaboration not only serves as an important step in our global development strategy for ganaxolone, it also represents the confidence Orion has in its potential. I believe that the collaboration allows both parties to share in the long-term success of ganaxolone.”
Ganaxolone, a positive allosteric modulator of GABAA receptors, is being developed in intravenous and oral formulations intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings. Ganaxolone exhibits anti-seizure and anti-anxiety activity via its effects on synaptic and extrasynaptic GABAA receptors. Marinus completed the first ever phase 3 pivotal study in children with CDKL5 deficiency disorder in 2020 and just submitted a New Drug Application to the U.S. Food and Drug Administration in that indication. It is also conducting a phase 2 trial in tuberous sclerosis complex and a phase 3 trial in refractory status epilepticus.
Under the agreement, Orion will pay Marinus $30 million (€25 million) in an upfront cash payment. Marinus is eligible to receive up to an additional $115 million (€97 million) in R&D reimbursement and cash milestone payments based on specific clinical and commercial achievements, as well as tiered royalty payments based on net sales ranging from the low double-digits to high teens for the oral programs and the low double-digits to low 20s for the IV program. The upfront payment is subject to certain provisions associated with additional preclinical testing by Marinus, which is expected to be completed in the first quarter of 2022.
“We are pleased to be the partner of choice for Marinus in Europe. “What we have seen so far with ganaxolone is encouraging and signifies an ongoing commitment to the rare epilepsy communities,” said Satu Ahomäki, senior vice president, Commercial Operations, Orion. “Ganaxolone could be a promising treatment option for patients suffering from rare epilepsies.”
As part of the agreement, Marinus will continue to be responsible for clinical development of ganaxolone, as well as regulatory approvals and submissions, including interactions with the European Medicines Agency (EMA). Marinus expects to file a European marketing authorization application (MAA) for ganaxolone for the treatment of seizures associated with CDD by the end of the third quarter of 2021. Marinus will continue to manufacture and supply product to Orion pursuant to a supply agreement between the parties. Ganaxolone is expected to have at least 10 years of regulatory data protection in Europe.
Orion will be responsible for all pricing and reimbursement approvals as well as commercialization of ganaxolone in the European Economic Area (comprised of all 27 members of the European Union, Iceland, Liechtenstein and Norway). Additional territories include the United Kingdom and Switzerland. A Joint Steering Committee, comprised of two representatives from each party, will be set up to monitor and coordinate the development and commercialization activities.
“We believe that Orion’s commercial leadership has exceptional expertise in rare orphan diseases as well as a significant hospital and ICU presence to support development of IV ganaxolone alongside our oral franchise,” said Christy Shafer, Chief Commercial Officer of Marinus. “We remain committed to identifying opportunities throughout the rest of the world to help improve the lives of patients suffering from rare genetic epilepsies and seizure disorders.”
Photo: Scott Braunstein, CEO of Marinus Pharmaceuticals