Press Releases
Lineage Cell Therapeutics Reinitiates Patient Enrollment in Clinical Study of OpRegen® for the Treatment of Dry AMD With Geographic Atrophy
First OpRegen Patient Dosed at
“OpRegen is a promising candidate for the future treatment of dry AMD and I am excited to have dosed the first patient at our institute,” stated
“We are pleased to safely resume patient enrollment in our OpRegen study under the direction of
Recently, Lineage reported the first known finding of retinal tissue regeneration in a patient receiving OpRegen for the treatment of atrophic dry AMD. This unprecedented finding supports the view that dry AMD is not an irreversible, degenerative condition and that some portion of diseased retinal tissue may be recoverable in atrophic end-stage disease patients. The loss of RPE cells over time creates progressively larger areas of geographic atrophy (GA) in the adult retina, leading to impaired vision or complete blindness. Humans lack the innate ability to regenerate retinal tissue and replace lost retina cells, which has led to a presumption that progression of GA may someday be slowed or halted but cannot be reversed. The unique finding from the ongoing OpRegen clinical trial supports a different view, in which an RPE cell transplant can potentially replace or rescue retinal cells in patients who suffer from retinal lesions or degeneration. Lineage has reported evidence from a patient with atrophic end-stage disease who received a transplant of allogeneic RPE cells and showed substantial restoration of retinal tissue within the area of GA. Specifically, the area of GA assessed at 9 months was approximately 25% smaller than the patient’s pre-treatment baseline. These findings were initially observed by an independent external advisor using multiple imaging technologies and were subsequently confirmed by the reading center and additional experts in the field of retinal imaging.
The Company has also observed evidence of benefit in some patients, including increases in Best Corrected Visual Acuity (BCVA), reduction in the growth of GA and increases in reading speed. The addition of signs of retinal tissue regeneration provides further support that OpRegen may be a viable treatment for the millions of individuals living with dry AMD, one of the leading causes of vision loss in the world.
About the OpRegen Phase 1/2a Clinical Study
This is a Phase 1/2a open-label, dose escalation safety and efficacy study of a single injection of human retinal pigment epithelium cells derived from an established pluripotent cell line and transplanted subretinally in patients with advanced dry AMD with GA. The study will enroll patients into 4 cohorts, with 18 of 21 expected patients enrolled to date. The first 3 cohorts enrolled solely legally blind patients, with best corrected visual acuity (BCVA) of 20/200 or worse. The fourth cohort, which is currently enrolling, will include patients with vision ranging from 20/250 to as high as 20/64. Cohort 4 also includes patients treated with one of two formulations of OpRegen, (i) a formulation which required preparation of cells one day prior to use in a dose preparation laboratory; or (ii) a new “off-the-shelf” or “thaw-and-inject” formulation of OpRegen which can be shipped directly to sites and used immediately upon thawing, removing the complications and logistics of having to use a dose preparation facility. The primary objective of the study is to evaluate the safety and tolerability of OpRegen as assessed by the incidence and frequency of treatment emergent adverse events. Secondary objectives are to evaluate the preliminary efficacy of OpRegen treatment by assessing the changes in ophthalmological parameters measured by various methods of primary clinical relevance. Additionally, for the patients in Cohort 4 that receive subretinal delivery of OpRegen utilizing Gyroscope Therapeutics’ Orbit Subretinal Delivery System (Orbit SDS), objectives will include the evaluation of the safety of delivery of OpRegen using the Orbit SDS.
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Forward-Looking Statements
Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements, in some cases, can be identified by terms such as “believe,” “may,” “will,” “estimate,” “continue,” “anticipate,” “design,” “intend,” “expect,” “could,” “plan,” “potential,” “predict,” “seek,” “should,” “would,” “contemplate,” project,” “target,” “tend to,” or the negative version of these words and similar expressions. Such statements include, but are not limited to, statements relating to the anticipated completion of patient enrollment in Lineage’s Phase 1/2a clinical study of OpRegen. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineage’s actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including risks and uncertainties inherent in Lineage’s business and other risks in Lineage’s filings with the
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