Press Releases
Lineage Cell Therapeutics Presents New OpRegen® Data for Dry AMD With GA at 2020 American Academy of Ophthalmology Annual Meeting
- Improved Visual Acuity Continues to be Observed in Cohort 4 Patients
- First Known Clinical Report of Retinal Tissue Regeneration Persisted to 23 Months with Further Improvement in Visual Acuity
- Patient Enrollment Recently Completed
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Therapeutic Expert Call with Principal Investigator
Christopher D. Riemann , M.D. Scheduled forNovember 17, 2020 at4:00 pm Eastern Time
“These new data increasingly suggest to us that treatment with OpRegen can provide clinically meaningful outcomes in dry AMD patients with GA, particularly for those with earlier-stage disease,” stated
OpRegen Data Update & Highlights from the AAO Presentation (data presented on 20 patients, dosed through
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Continued progressive functional improvement.
- In Cohort 4, 6 out of 7 (86%) of patients’ treated eyes measured above their baseline vision (Best Corrected Visual Acuity, or BCVA) at 12 months, a clinically relevant timeframe, or as of the longest available timepoint less than 12 months (data collection continues for more recently-treated patients).
- Data to date demonstrate a localized slowing of GA progression in the treated areas with a trend towards slower GA growth in treated versus fellow eyes in pooled analyses.
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Long-term engraftment is supported with imaging observations up to more than 4 years, even with a short immunosuppression regimen.
- In all Cohort 4 patients receiving OpRegen TAI formulation, per protocol, immunosuppressants have been discontinued as scheduled, typically within 90 days post-operatively, and no cases of acute or delayed rejection or inflammation have been reported.
- One Cohort 4 patient was treated only with mycophenolate mofetil and received no tacrolimus for immunosuppression.
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Anatomical restoration of retinal tissue.
- A Cohort 4 patient with evidence of retinal restoration and confirmed history of GA growth, which was first reported at 9 months, continues at month 23 to have an area of GA smaller than at baseline.
- This patient also experienced additional improvement in BCVA from 9 to 23 months post-treatment, while the untreated eye has experienced further reduction in visual acuity.
- Long-term monitoring on this patient is expected to continue.
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Treatment overview.
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As of
October 5, 2020 , 16 patients were treated via pars planar vitrectomy (PPV), while 4 were treated with the Gyroscope SDS. -
As of
November 10, 2020 , 17 patients were treated via PPV, while 7 were treated with the Gyroscope SDS. - Enrollment in the phase 1/2a study is complete; follow-up continues for safety and efficacy.
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As of
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Safety and tolerability.
- The primary objective of the study is to evaluate the safety and tolerability of OpRegen at 12 months, and in patients which have reached this time point OpRegen appears well tolerated.
- There have been no unexpected adverse events (AEs) or treatment-related systemic serious AEs reported in enrolled patients.
- The most common and expected ocular AEs were the formation or exacerbation of mild to moderate epiretinal membranes (ERMs) and a single report of a retinal detachment, with cause unknown (all occurring in patients receiving OpRegen via the PPV route of administration).
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The Gyroscope SDS is an alternative to the PPV route and is designed to avoid ERM formation.
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Through
October 2020 , 16 patients were treated via PPV while 4 were treated with the Gyroscope SDS. ERMs were observed in 13 PPV patients. - One patient treated with the Gyroscope SDS developed a mild choroidal neovascularization (CNV) at the site of needle penetration 6 months post-treatment which was successfully treated with a single dose of an approved anti-VEGF agent. The cause was unknown.
- One patient treated via PPV developed a mild CNV at > 24 months post-treatment.
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Through
- Other changes observed following OpRegen treatment persisted through the last time point examined (> 4 years in some patients), including subretinal pigmentation and hyper-reflective areas seen on optical coherence tomography (OCT).
The results were presented at the 2020 American Academy of Ophthalmology Annual Meeting (AAO 2020). The presentation, “Phase 1/2a Study of Subretinally Transplanted hESC-Derived RPE Cells in Advanced Dry-Form AMD Patients” was featured as part of the Original Paper Session, OP02V Retina, Vitreous Original Papers on
KOL Call and Webcast
Lineage will host a therapeutic area expert call with
About OpRegen
OpRegen is currently being evaluated in 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 enrolled 24 patients into 4 cohorts. The first 3 cohorts enrolled only legally blind patients with best corrected visual acuity (BCVA) of 20/200 or worse. The fourth cohort enrolled 12 better vision patients (vision from 20/65 to 20/250 with smaller areas of GA). Cohort 4 also included patients treated with a new “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. In total, 17 patients were treated via PPV, while 7 were treated with the Gyroscope SDS. 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 the Gyroscope SDS, objectives will include the evaluation of the safety of delivery of OpRegen using the Gyroscope SDS.
OpRegen is a registered trademark of
About Dry AMD
Dry age-related macular degeneration (AMD) is a leading cause of adult blindness in the developed world. There are two forms of AMD: wet AMD and dry AMD. Dry AMD is the more common of the two types, accounting for approximately 85-90% of cases. Wet AMD is the less common of the two types, accounting for approximately 10-15% of cases. Global sales of the two leading wet AMD therapies were in excess of
About
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 development plans for OpRegen and post-enrollment timing expectations. 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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(ir@lineagecell.com)
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Solebury Trout IR
(Gogawa@troutgroup.com)
(646) 378-2949
Nic.johnson@russopartnersllc.com
David.schull@russopartnersllc.com
(212) 845-4242
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