Lineage Cell Therapeutics, Inc. (NYSE and TASE: LCTX) and its subsidiary Cell Cure Neurosciences, Ltd, recently entered into an exclusive worldwide collaboration and license agreement with Roche and Genentech, a member of the Roche Group, for the development and commercialization of OpRegen, a retinal pigment epithelium (RPE) cell therapy for the treatment of ocular disorders, including advanced dry age-related macular degeneration (dry AMD) with geographic atrophy (GA).
Under the terms of the collaboration agreement, Genentech will assume responsibility for further clinical development and commercialization of the OpRegen program which is currently being evaluated in a Phase 1/2a open-label, dose-escalation clinical safety, and efficacy study in patients with advanced dry AMD with GA. Lineage will complete activities related to the ongoing OpRegen clinical study, for which enrollment is complete, and perform certain manufacturing activities. Genentech will pay Lineage a $50 million upfront payment and Lineage is eligible to receive up to $620 million in additional development, approval, and sales milestone payments, in addition to tiered double-digit royalties.
Tamar Raz, CEO, Hadasit stated, “Cell Cure was established in 2002 based on the technology of two leading scientists at Hadassah Medical Center, Prof. Benjamin Reubinoff, Director of the Hadassah Center for Embryonic Stem Cell Research and an expert in obstetrics and gynecology and Prof Eyal Banin, Director of the Center for Retinal Degeneration Diseases at Hadassah‘s Department of Ophthalmology, an expert of AMD. Rami Skaliter, M.Sc., Ph.D., is the CEO of Cell Cure Neurosciences, where the manufacturing and related research and development activities for Lineage’s regenerative medicine platform take place. Dr. Skaliter obtained his B.Sc. in Biology at the Ben-Gurion University and his M.Sc. and Ph.D. in Biochemistry at the Weizmann Institute in Israel. He specialized in the biochemistry of replication and mutagenesis mechanisms. Dr. Skaliter also completed a post-doctoral fellowship at Stanford University.”
“Genentech is a clear global leader in ophthalmology and has demonstrated a longstanding commitment to patients, innovative research, and successful product development,” said Brian M. Culley, Lineage’s CEO. “Their desire to combine our cell therapy technology with their ophthalmology expertise and capabilities will help advance the OpRegen program more rapidly and we believe successfully to patients with serious ocular disorders, such as dry age-related macular degeneration. Lineage’s objective is to pioneer a new branch of regenerative medicine, based on transplanting whole cells into the body to restore activity lost to aging, injury, or disease. We believe the results we have demonstrated to date with OpRegen represent a paradigm change many did not believe possible with cell therapy, by restoring retinal tissue and potentially halting or reversing the expansion of geographic atrophy. I am incredibly proud of what the Lineage team has accomplished with the OpRegen program and look forward to joining forces with the Genentech team as they work to take this program to the next level and potentially to patients in need of treatment.”
“Genentech has a longstanding commitment to discovering and developing novel drugs for the treatment of serious eye disorders such as with advanced dry AMD with GA, which is one of our focus areas within ophthalmology,” said James Sabry, M.D., Ph.D., global head of Pharma Partnering, Roche. “We are excited to partner with Lineage Cell Therapeutics, Inc. to advance potential new therapies in an area of high unmet medical need.”
OpRegen has been developed in part through contributions and financial grants made by Hadasit Medical Research Services and Development Ltd. (“Hadasit“) and the Israeli Innovation Authority (the “IIA”).
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 a best-corrected visual acuity (BCVA) of 20/200 or worse. The fourth cohort enrolled 12 better vision patients (BCVA from 20/65 to 20/250 with smaller mean 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 was 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.
OpRegen has been well-tolerated to date and there have been no new, unexpected ocular or systemic adverse events or serious adverse events related to OpRegen or study procedures that have not been previously reported.
About Lineage Cell Therapeutics
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, which is now being developed under a worldwide collaboration with Roche and Genentech, a member of the Roche Group;
(ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of acute spinal cord injuries;
(iii) VAC2, an allogeneic dendritic cell therapy produced from Lineage’s VAC technology platform for immuno-oncology and infectious disease, is currently in Phase 1 clinical development for the treatment of non-small cell lung cancer.
Cell Cure Neurosciences, a subsidiary of Lineage, is a biotechnology company developing cell therapies for retinal and neural degenerative diseases and is located at the Jerusalem Bio Park, which is sited at the Hadassah University Hospital campus.
For more information, please visit www.lineagecell.com or follow the Company on Twitter @LineageCell.
About Cell Cure Neurosciences, Ltd
The OpRegen program was established in part through intellectual property licensed from Hadasit Medical Research Services and Development Ltd., the technology transfer company of Hadassah Medical Center, and with financial grants from the Israeli Innovation Authority, an independent agency created to address the needs of global innovation ecosystems. Lineage is obligated to pay a portion of the upfront, milestone, and royalty payments it receives to Hadasit and the Israeli Innovation Authority. Each of these parties’ contributions began when the OpRegen program was in its earliest and most speculative stage of development. A significant portion of early development on the OpRegen program was conducted by Cell Cure Neurosciences, Ltd., which over time became a 99%-owned subsidiary of Lineage. Cell Cure was originally established by the Hadassah Medical Center, where the intellectual property underlying the differentiation and manufacture of RPE cells originated. Monetary support provided by the Israeli Innovation Authority through a series of research grants began in 2007. Development of OpRegen in subsequent years was led by Lineage, a California company, and will continue hereafter under the leadership of Genentech, a global pharmaceutical company.
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