Chiesi acquires ex-US Raxone marketing rights

The deal – worth up to CHF105m (€93m) including sales milestones – gives Santhera financial backing to advance its the development of its clinical-stage neuromuscular and pulmonary programmes. It provides Chiesi with marketing rights of the benzoquinone idebenone in Leber’s hereditary optic neuropathy (LHON) and all other ophthalmological indications worldwide except the US and Canada, where Santhera retains rights. Following the completion of certain reimbursement and post-regulatory commitments on the part of Santhera, orphan drug specialist Chiesi Group will have the option to fully acquire Santhera’s Raxone business.

“In the past eighteen months we strategically diversified our pipeline," said said Dr Thomas Meier, CEO of Santhera. “We have built a strong portfolio in neuromuscular and pulmonary rare disease areas with last year’s acquisition of option-rights to vamorolone for Duchenne muscular dystrophy (DMD) and other inflammatory diseases, and the acquisition of POL6014 for cystic fibrosis and other pulmonary disorders. Major upcoming inflection points will be the decision on our marketing authorization application for Puldysa in DMD in Europe, which we plan to submit shortly. Another milestone expected for next year are results of the pivotal trial with vamorolone in DMD. With positive results and upon exercising the option to acquire this asset, we could seek regulatory approvals in the US and Europe,” he added.    

About 95% of patients with LHON harbour one of three pathogenic mutations of the mitochondrial DNA, which cause a defect in the complex I subunit of the mitochondrial respiratory chain. This defect leads to decreased cellular energy (ATP) production, increased reactive oxygen species (ROS) production and retinal ganglion cell dysfunction, which cause progressive loss of visual acuity and blindness. 

Raxone (idebenone), a synthetic short-chain benzoquinone  circumvents the complex I defect, reduces and scavenges ROS, restores cellular energy levels in retinal ganglion cells and promotes recovery of visual acuity in up to 50% of patients.