The first dose level of a novel gene-based treatment (RetinoStat, Oxford BioMedica) for neovascular age-related macular degeneration (AMD) is safe and well-tolerated at 1 month following treatment, the company has announced.
Oxford, England-The first dose level of a novel gene-based treatment (RetinoStat, Oxford BioMedica) for neovascular age-related macular degeneration (AMD) is safe and well-tolerated at 1 month following treatment, the company has announced.
The treatment was designed and developed using the company’s proprietary gene-delivery technology (LentiVector). It is the lead program of the ocular agreement the company signed with Sanofi in 2009.
Researchers of the ongoing phase I study expect to enroll 18 patients with wet AMD at the Wilmer Eye Institute, Johns Hopkins University School of Medicine, Baltimore. They will evaluate three dose levels and assess safety, aspects of visual acuity, and ocular physiology. Three patients received the first dose level of the treatment, and 1-month results were assessed by the study’s independent data safety monitoring board (DSMB). Complete first results from the study are expected to be announced in the first half of 2012.
Highlights of the first patient cohort at 1 month (dose level 1), according to the company, include a favorable safety profile with no serious adverse events related to the treatment or its method of administration, no signs of inflammation in the eye, and DSMB support to proceed to dose level 2 in the next patient cohort.
“This clinical study is breaking new ground as the first to administer directly a lentiviral, vector-based treatment to patients in the United States, and the favorable safety profile of [the treatment] is encouraging,” said John Dawson, chief executive officer of Oxford BioMedica. “We continue to work closely with the regulatory bodies . . . to advance our novel ocular programs as rapidly as possible and, together with Sanofi, we look forward to further progress during 2011.”
The company also has two vision-related orphan drug products entering clinical development. The first patient in the phase I/IIa study of a novel gene-based treatment (StarGen) for Stargardt’s disease was treated in June at the Oregon Health & Science University’s Casey Eye Institute. Another product (UshStat), for the treatment of Usher syndrome type 1B, is expected to enter phase I/II clinical development before the end of this year.
“We are delighted to see Oxford BioMedica’s lentiviral gene therapy products successfully moving into human studies,” said Stephen Rose, PhD, chief research officer of the Foundation Fighting Blindness, a non-profit organization that provided early funding for the company’s pre-clinical ocular program. “The company’s innovative treatments hold great promise for saving vision in people affected by a broad range of devastating eye diseases for which current treatments are limited or non-existent.”