Axovant Obtains Worldwide License to Develop AXO-Lenti-PD, Potential Parkinson’s Gene Therapy

José Lopes, PhD avatar

by José Lopes, PhD |

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Axovant Sciences licensed the exclusive worldwide rights from Oxford BioMedica to develop and market AXO-Lenti-PD, a gene therapy candidate for Parkinson’s disease.

Formerly known as OXB-102, AXO-Lenti-PD uses a modified, harmless type of virus called lentivirus to deliver three genes that provide instructions to make a key enzyme in the synthesis of dopamine, the neurotransmitter produced at low levels in Parkinson’s patients.

The investigational medication was designed to provide durable restoration of dopamine levels after a single administration.

Oxford recently completed a Phase 1/2 trial of ProSavin (NCT00627588), OXB-102’s predecessor, which demonstrated favorable safety and tolerability and a significant improvement of motor function at six and 12 months in Parkinson’s patients. This benefit was sustained for up to four years in most patients.

Compared with ProSavin, preclinical studies of OXB-102 showed increased production of the key enzymes, as well as at least a fivefold greater potency in improving behavior and movement in an animal model of the disease.

Axovant plans to start a Phase 1/2 dose escalation trial of AXO-Lenti-PD in patients with advanced Parkinson’s by the end of 2018. Oxford will be the clinical and commercial supplier of AXO-Lenti-PD.

Under the terms of the agreement, Axovant obtained rights to both AXO-Lenti-PD and ProSavin for an initial $30 million. Roivant Sciences, Axovant’s parent company, agreed to purchase $25 million of Axovant’s shares, which will support the development of AXO-Lenti-PD.

“Axovant remains committed to developing innovative treatments for serious neurodegenerative conditions such as Parkinson’s disease, and we are excited to partner with Oxford BioMedica,” Pavan Cheruvu, MD, CEO of Axovant, said in a press release. “We will continue to pursue promising new therapeutic approaches based on transformative science, and will further expand our pipeline with high-quality assets like AXO-Lenti-PD.”

Axovant also announced that Fraser Wright, PhD, co-founder and former chief technology officer of Spark Therapeutics, will join the company as the new chief technology officer, overseeing the gene therapy program.

“I look forward to the opportunity to work closely with Oxford BioMedica and help build gene therapy capabilities at Axovant,” Wright said. “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.”

At Spark, Wright oversaw the development and manufacturing of Luxturna (voretigene neparvovec-rzyl). He had previously been involved in the development and human testing of both Luxturna and Kymriah (tisagenlecleucel), developed by Novartis, at the Children’s Hospital of Philadelphia.

Wright’s more than 20 years of experience in gene therapy will help build world-class capabilities at Axovant, Cheruvu said.

John Dawson, CEO of Oxford, believes Axovant’s expertise in neurological disorders, including Parkinson’s, makes it an ideal partner “to advance the development of AXO-Lenti-PD for the treatment of patients with Parkinson’s.”

“We are delighted to sign this significant agreement which not only underlines our LentiVector-enabled platform and product development strategy but further demonstrates Oxford BioMedica’s ability to build multiple partnerships with leaders in their respective therapeutics fields,” he said.

Axovant held a conference call on June 6 to discuss the agreement. A replay of the webcast is now available here.