Axovant enters gene therapy field, gaining rights to Oxford BioMedica's experimental Parkinson's disease treatment

Axovant Sciences announced Wednesday an exclusive licensing deal potentially worth over $800 million to gain global rights to Oxford BioMedica's experimental gene therapy OXB-102 for Parkinson's disease. Axovant, whose shares jumped more than 120 percent on the news, said that it expects to initiate a Phase I/II dose-escalation study of the therapy, renamed AXO-Lenti-PD, in patients with advanced Parkinson's disease by the end of 2018.

Under the agreement, Axovant will obtain rights to AXO-Lenti-PD, as well as its predecessor product ProSavin, for an initial cash payment of $30 million. As part of the deal, Oxford BioMedica is also eligible to receive development milestones of up to $55 million, regulatory and commercial milestone payments of potentially $757.5 million, as well as tiered royalties in the range of 7 percent to 10 percent on net sales of AXO-Lenti-PD, if approved.

Axovant noted that in relation to the transaction, parent company Roivant Sciences agreed to purchase $25 million of common shares, which will support the clinical development of AXO-Lenti-PD and additional business development activities. AXO-Lenti-PD, which uses Oxford BioMedica's LentiVector platform, is designed to deliver three genes encoding a critical set of enzymes required for dopamine synthesis in the brain.

In addition, Axovant noted that Fraser Wright will join the company as chief technology officer (CTO) to oversee the gene therapy initiatives. Axovant CEO Pavan Cheruvu, who assumed the role in February, said Wright "brings over two decades of experience in gene therapy manufacturing, and will be committed to building world-class gene therapy capabilities at Axovant."

The appointment of Wright, who is the co-founder and former CTO of Spark Therapeutics, follows Axovant's recent hires of Gavin Corcoran, the current chief medical officer of Allergan, as executive vice president of R&D, and Michael Hayden, the former chief scientific officer at Teva, as chairman of the scientific advisory board.

The overhaul of Axovant's drug pipeline and executive team comes after the departure earlier this year of CEO David Hung and chief operating officer Marion McCourt. Their exit came shortly after the company announced that it would end development of intepirdine after the experimental 5-HT6 receptor antagonist failed to meet the main efficacy endpoint of a mid-stage study in patients with dementia with Lewy bodies. The drugmaker previously disclosed that a late-stage study of intepirdine in patients with mild-to-moderate Alzheimer's disease who were receiving background donepezil therapy also failed to meet its co-primary efficacy endpoints.

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