OpRegen® Data Update to Be Featured at 2021 Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting in Presentation by Christopher D. Riemann, MD

Rhea-AI Summary

Lineage Cell Therapeutics (NYSE: LCTX) announced updated interim results from a Phase 1/2a study of OpRegen®, a cell therapy aimed at treating dry age-related macular degeneration (AMD). The study enrolled 24 patients in four cohorts, assessing safety, tolerability, and preliminary efficacy. Notably, Cohort 4 utilized a new 'thaw-and-inject' formulation, enhancing logistical efficiency. Results will be presented at the 2021 ARVO Annual Meeting on May 6, 2021. Dry AMD affects 85-90% of AMD cases, and no FDA-approved treatments currently exist for this condition.

Positive

• Interim results from Phase 1/2a study of OpRegen show promising safety and efficacy.
• Introduction of a 'thaw-and-inject' formulation simplifies treatment logistics.
• Presentation of results at a prestigious international conference boosts visibility.

Negative

• Limited visibility on specific efficacy outcomes may raise concerns among investors.
• The absence of FDA-approved treatments for dry AMD enhances competitive pressure.

Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs, announced today that updated interim results from a Phase 1/2a study of its lead product candidate, OpRegen^®, a retinal pigment epithelium cell transplant therapy currently in development for the treatment of dry age-related macular degeneration (AMD), will be presented at the 2021 Association for Research in Vision and Ophthalmology Annual Meeting (ARVO 2021), to be held virtually (May 1 – 7, 2021). The presentation, “Phase I/IIa Clinical Trial of Transplanted Allogeneic Retinal Pigmented Epithelium (RPE, OpRegen) Cells in Advanced Dry Age-Related Macular Degeneration (AMD): Interim Results” will be featured as part of the Stem cells/Gene Therapy/Transplantation Session, on May 6, 2021 between 5:15 pm and 6:45 pm EDT by Christopher D. Riemann, M.D., Vitreoretinal Surgeon and Fellowship Director, Cincinnati Eye Institute (CEI) and University of Cincinnati School of Medicine. (abstract number 3538173).

The Association for Research in Vision and Ophthalmology, Inc. (ARVO) was founded in 1928 in Washington, DC by a group of 73 ophthalmologists. ARVO is the largest and one of the most respected eye and vision research organizations in the world and its members include nearly 11,000 researchers from over 75 countries. The membership is multidisciplinary and consists of both clinical and basic researchers. ARVO advances research worldwide into understanding the visual system and preventing, treating and curing its disorders. For more information, please visit https://www.arvo.org/ or follow the association on Twitter @ARVOInfo.

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. 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. Additional objectives include the evaluation of the safety of delivery of OpRegen using the Orbit Subretinal Delivery System (Orbit SDS). OpRegen is a registered trademark of Cell Cure Neurosciences Ltd., a majority-owned subsidiary of Lineage Cell Therapeutics, Inc.

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 $10 billion in 2019. Nearly all cases of wet AMD begin as dry AMD. Dry AMD typically affects both eyes. There are currently no U.S. Food and Drug Administration (FDA) or European Medicines Agency (EMA) approved treatment options available for patients with dry AMD.

About Lineage Cell Therapeutics, Inc.
Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineage’s programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed to either replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineage’s clinical programs are in markets with billion dollar opportunities and include three allogeneic (“off-the-shelf”) product candidates: (i) OpRegen^®, a retinal pigment epithelium transplant therapy in Phase 1/2a development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of acute spinal cord injuries; and (iii) VAC, an allogeneic dendritic cell therapy platform for immuno-oncology and infectious disease, currently in clinical development for the treatment of non-small cell lung cancer. For more information, please visit www.lineagecell.com or follow the Company on Twitter @LineageCell.

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FAQ

What are the interim results of the Phase 1/2a study for OpRegen by Lineage Cell Therapeutics?

The interim results indicate promising safety and efficacy for OpRegen in treating dry AMD.

When will the results of the OpRegen study be presented?

Results will be presented on May 6, 2021, at the 2021 ARVO Annual Meeting.

What is the purpose of the OpRegen study?

The study aims to evaluate the safety, tolerability, and preliminary efficacy of OpRegen for advanced dry AMD.

How many patients were involved in the OpRegen trial?

The trial enrolled 24 patients across four cohorts.

What is dry age-related macular degeneration (AMD)?

Dry AMD is a leading cause of adult blindness, accounting for 85-90% of AMD cases, with no approved treatments available.