• Axovant will license two products from Oxford BioMedica
• The gene therapy based products have demonstrated long term improvement in motor function
• Axovant shares are up 102% in premarket trading

Axovant Sciences (NASDAQ:AXON) today announced that it has licensed the exclusive worldwide rights to develop and commercialize OXB-102, now AXO-Lenti-PD, from Oxford BioMedica. AXO-Lenti-PD is an investigational gene therapy for Parkinson’s disease that delivers three genes encoding a critical set of enzymes required for dopamine synthesis in the brain. Oxford BioMedica is a world leader in lentiviral vector product development and manufacturing, and will be the clinical and commercial supplier of AXO-Lenti-PD. Axovant expects to initiate a Phase 1/2 dose escalation study of AXO-Lenti-PD in patients with advanced Parkinson’s disease by the end of 2018.

Under the terms of the license agreement with Oxford BioMedica, Axovant obtained rights to AXO-Lenti-PD, as well as its predecessor product ProSavin, for an initial payment of $30 million in cash, $5 million of which will be applied as a credit against the process development work and clinical supply that Oxford BioMedica will provide to Axovant. Oxford BioMedica is also eligible to receive additional development, regulatory, and commercial milestone payments potentially in excess of $812 million, and tiered royalties on net sales of AXO-Lenti-PD, if approved. Roivant has agreed to purchase $25 million of Axovant common shares, which will support the clinical development of AXO-Lenti-PD and additional business development activities

Fraser Wright, PhD, will join Axovant as Chief Technology Officer overseeing the company’s gene therapy initiatives. Dr. Wright is the Co-Founder and former Chief Technology Officer of Spark Therapeutics and has over 20 years of leadership experience in the development of novel vector-based biologic products. At Spark he oversaw process development and clinical-stage manufacturing for Luxturna.

“This is an exciting time to join Axovant, and I look forward to the opportunity to work closely with  and help build gene therapy capabilities at Axovant,” said Dr. Wright. “AXO-Lenti-PD is a strong foundation for Axovant’s new pipeline, and I am excited to begin preparing the Phase 1/2 clinical study in advanced Parkinson’s disease later this year.”

AXO-Lenti-PD (formerly OXB-102) is a second-generation gene therapy, which utilises Oxford BioMedica’s LentiVector platform technology to deliver three genes that encode key dopamine synthesis enzymes. When injected into the brain’s striatum, OXB-102 genetically modifies cells to produce dopamine, replacing that which is lost during the course of the disease. Unlike current drug treatment, which loses efficacy with long-term use, OXB-102 is designed to provide patient benefit for a number of years following a single administration. In a phase I/II study, AXO-Lenti-PD met its primary endpoint and demonstrated a statistically significant improvement in motor function at six and twelve months post treatment.

ProSavin is the first generation of the product. In a small study conducted in France, ProSavin was tested in 15 patients. Motor improvement was noted in all patients and the effect was dose dependent. Though not a cure (the disease continued to progress), the long term increase in dopamine production and improvement in motor function appeared to last up to four years.

As of this writing Axovant shares are up 102% in premarket trading.