Lineage Establishes Exclusive Global Collaboration with Genentech for the Development and Commercialization of OpRegen® RPE Cell Therapy for the Treatment of Eye Disorders

CARLSBAD, Calif.–(BUSINESS WIRE)–Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), today announced that Lineage and its subsidiary, Cell Cure Neurosciences Ltd., have entered into an exclusive worldwide collaboration and licensing agreement with rock and Genentechmember of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), for the development and commercialization of a retinal pigment epithelial (RPE) cell therapy for the treatment of ocular disorders, including advanced dry macular degeneration age-related (dry AMD) with geographic atrophy (GA).

Genentech will assume responsibility for the continued clinical development and commercialization of Lineage’s OpRegen program, which is currently being evaluated in an open-label, dose-escalating Phase 1/2a clinical study of safety and efficacy in patients with advanced dry AMD with AG. Under the terms of the collaboration agreement, Lineage will complete activities related to the ongoing clinical study, for which recruitment has been completed, and perform certain manufacturing activities. Genentech will pay Lineage an upfront payment of $50 million and Lineage is eligible to receive up to $620 million in additional development, approval and sales milestone payments, in addition to double-digit tiered royalties.

“Genentech is an undisputed global leader in ophthalmology and has demonstrated a long-standing commitment to patients, innovative research and successful product development,” said Brian M. Culley, CEO of Lineage. “Their desire to combine our cell therapy technology with their expertise and capabilities in ophthalmology will help move the OpRegen program forward more quickly and we believe in successfully helping patients with serious eye conditions, such as dry macular degeneration related to age. Lineage’s goal is to pioneer a new branch of regenerative medicine, based on the transplantation of whole cells into the body to restore activity lost due to aging, injury or disease. sickness. We believe the results we have demonstrated to date with OpRegen represent a paradigm shift that many did not believe possible with cell therapy, 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 partnering with the Genentech team as they work to take this program to the next level and potentially to patients. needing treatment.

Mr. Culley continued, “Looking forward, Lineage will continue to focus on advancing our spinal cord injury and oncology programs, as well as announcing new disease settings in which we plan to work. deploy our technology, either on our own or through strategic alliances. At Lineage, we are all immensely proud to have the opportunity and responsibility to advance a new and exciting branch of medicine, and our goal is to have a profound impact on the patients who inspire us.

“Genentech has a long-standing commitment to the discovery and development of new drugs for the treatment of serious ocular disorders such as advanced dry AMD with GA, which is one of our focus areas in ophthalmology,” said said James Sabry, MD, Ph.D., Global Head of Pharmaceutical Partnerships, Roche. “We are excited to partner with Lineage Cell Therapeutics to advance potential new therapies in an area of ​​significant unmet medical need.”

Conference call information

Lineage will host a live conference call and webcast today starting at 8 a.m. ET to discuss the collaboration with Roche Group and Genentech. Interested parties may access the conference call by dialing (866) 888-8633 from within the United States and Canada and (636) 812-6629 from anywhere outside the United States and Canada and should ask for the “Lineage Cell Therapeutics Appeal”. A live webcast of the conference call will be available online at Investors section of the Lineage website. A replay of the webcast will be available on the Lineage website for 30 days and a telephone replay will be available until December 27, 2021, by dialing (855) 859-2056 from the United States and Canada and (404 ) 537-3406 by the way outside. in the United States and Canada and entering the conference ID number 5174206.

About OpRegen

OpRegen was developed in part through financial contributions and grants from Hadasit Medical Research Services and Development Ltd. (“Hadasit”) and the Israeli Innovation Authority (“IIA”). Lineage is obligated to pay a portion of the initial, milestone, and royalty payments it receives to Hadasit and The IIA. OpRegen is currently being evaluated in an open-label, dose-escalating Phase 1/2a study of the safety and efficacy of a single injection of human retinal pigment epithelium cells derived from a pluripotent cell line established and transplanted via the subretinal route in patients with advanced dry AMD with GA. The study recruited 24 patients in 4 cohorts. The first 3 cohorts recruited only legally blind patients with best corrected visual acuity (BCVA) of 20/200 or less. The fourth cohort recruited 12 patients with better vision (BCVA from 20/65 to 20/250 with smaller average AG surfaces). Cohort 4 also included patients treated with a new “thaw and squirt” formulation of OpRegen, which can be shipped directly to sites and used immediately after thawing, removing the complications and logistics of using a facility of dose preparation. The primary objective of the study was to assess the safety and tolerability of OpRegen based on the incidence and frequency of treatment-related adverse events. The secondary objectives are to assess the preliminary efficacy of OpRegen treatment by evaluating 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 the study procedures not previously reported.

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 proprietary and robust cell therapy platform and associated in-house development and manufacturing capabilities. With this platform, Lineage develops and manufactures specialized and differentiated end-stage human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed to replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a way to help the body mount an effective immune response against cancer. Lineage’s clinical programs are in billion-dollar opportunity markets 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, one of the leading causes 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 injury; and (iii) VAC2, an allogeneic dendritic cell therapy produced from Lineage’s VAC technology platform for immuno-oncology and infectious diseases, currently in phase 1 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.

Forward-looking statements

Lineage cautions you that all statements, other than statements of historical fact, contained in this press release are forward-looking statements. Forward-looking statements, in some instances, may be identified by words such as “believe”, “aim”, “may”, “will”, “estimate”, “continue”, “anticipate”, “conceive”, “have the intention of, “”expect”, “could”, “may”, “plan”, “potential”, “predict”, “seek”, “should”, “would”, “contemplate”, ” project”, “target”, “tend to”, or the negative version of these similar words and expressions. Such statements include, but are not limited to, statements relating to the collaboration and license agreement with Roche and Genentech and planned activities under the collaboration and licensing agreement, initial, milestone and royalty consideration payable to Lineage, potential benefits of processing with OpRegen, and Lineage’s plans to advance its programs spinal cord injury and oncology and announce new disease settings where it plans to deploy its technology. 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 any future results, performance or achievements expressed or implied by the forward-looking statements of this press release, including the risk that competing alternative therapies may adversely impact the commercial potential of OpRegen, which could materially adversely affect the milestone and royalty payments payable to Lineage under of the Collaboration and Licensing Agreement, the risk that Roche and Genentech may not be successful in completing further clinical trials for OpRegen and/or obtaining regulatory approval for OpRegen in a particular jurisdiction, and the risks and uncertainties inherent in Lineage’s business and other risks in documents filed by Lineage a from the Securities and Exchange Commission (SEC). Lineage’s forward-looking statements are based on its current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Further information regarding these and other risks is included under “Risk Factors” in Lineage’s periodic filings with the SEC, including Lineage’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the SEC and its other reports, which are available on the SEC’s website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date they were made. Lineage undertakes no obligation to update these statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.