First patient successfully dosed with Oxford BioMedica's OXB-102
Oxford Biomedica
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16:40 27/12/24
Gene and cell therapy group Oxford BioMedica noted on Thursday that its partner, Axovant Sciences - a company developing innovative gene therapies for neurologic and neuromuscular diseases - has announced the successful dosing of the first patient in a clinical study of AXO-Lenti-PD, also known as OXB-102.
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The London-listed firm described that as an investigational gene therapy for patients with Parkinson's disease.
It said AXO-Lenti-PD is a novel gene therapy that enabled the expression of a set of three critical enzymes required for end-to-end dopamine synthesis in the brain.
The therapy was originally developed by Oxford BioMedica, and subsequently out-licensed to Axovant in June.
AXO-Lenti-PD was administered to the first patient on the study at the Clinical Research Facility affiliated with the National Institute for Health Research (NIHR) and University College London Hospitals (UCLH).
The patient experienced no complications related to the surgery or to administration of the vector and was discharged home as planned, Oxford BioMedica said.
“The ongoing clinical study of AXO-Lenti-PD will evaluate safety and tolerability, as well as collect efficacy data including standard measures of motor function in patients with Parkinson's disease,” the board reported.
“Initial data from the first cohort of patients in the clinical trial is expected in the first half of 2019.
“This study will build upon the long-term safety and efficacy results reported in the phase I/II trial of ‘ProSavin’, which showed up to six-year durability of reductions in UPDRS Part III ‘OFF’ scores in data shared last week at the Annual Congress of the European Society of Gene and Cell Therapy and recently published in Human Gene Therapy, Clinical Development.”
Under the terms of the agreement with Axovant, Oxford BioMedica received a $30m (£22m) upfront payment, including $5m as pre-payment for manufacturing activities related to OXB-102, now renamed AXO-Lenti-PD.
Oxford BioMedica was also eligible to receive $55m upon the achievement of specified development milestones, and $757.5m upon the achievement of specified regulatory and sales milestones, with 7% to 10% tiered royalties on net sales of AXO-Lenti-PD.