Lineage Initiates Clinical Study of OPC1 for Spinal Cord Injury

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About the DOSED Study

The Delivery of Oligodendrocyte Progenitor Cells for Spinal Cord Injury: Evaluation of a Novel Device (DOSED) clinical study is an open label, multi-center, device safety study, in 3-5 subacute and 3-5 stable chronic subjects with complete (ASIA Impairment Scale A) or incomplete (ASIA Impairment Scale B), traumatic, focal SCI affecting either cervical (C4-C7) or thoracic (T1-T10) vertebrae. The primary objective of this study is to evaluate the safety of a novel Manual Inject Parenchymal Spinal Delivery System (MI PSD System) to administer OPC1 to the spinal parenchyma. The primary endpoint is frequency and severity of the MI PSD System- or injection procedure-related adverse events (AEs) through 30 days (1 month). Secondary endpoints are frequency and severity of AEs through 90 days (3 months) following injection of OPC1 and/or the concomitant immunosuppression administered. Exploratory endpoints include measurements of neurological impairment and function, as well as pain, evaluated by changes from baseline on the following endpoints: changes in neurological function as measured by sensory and motor scores and motor level on International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI) examinations; changes in post-injection pain, defined as a worsening of pain or neuropathic pain of greater than 7 days duration from baseline levels, as assessed by the International Spinal Cord Injury Pain Basic Data Set or occurrence of allodynia; changes from baseline at 30, 90 and 365 days post-injection of OPC1 in: ISNCSCI, SCIM, International Spinal Cord Injury Pain Questionnaire; patient and clinical impressions of changes in quality of life as reported by changes from baseline at 30, 90, and 365 days post-injection of OPC1 as measured by: Patient Global Impression of Severity (PGI-S), Patient Global Impression of Change (PGI-C), Clinician Global Impression of Severity (CGI-S) and Clinician Global Impression of Change (CGI-C).

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel, “off-the-shelf,” cell therapies to address unmet medical needs. Lineage’s programs are based on its proprietary cell-based technology platform and associated development and manufacturing capabilities. From this platform, Lineage designs, develops, manufactures, and tests specialized human cells with anatomical and physiological functions similar or identical to cells found naturally in the human body. These cells are created by applying directed differentiation protocols to established, well-characterized, and self-renewing pluripotent cell lines. These protocols generate cells with characteristics associated with specific and desired developmental lineages. Cells derived from such lineages are transplanted into patients in an effort to replace or support cells that are absent or dysfunctional due to degenerative disease, aging, or traumatic injury, and to restore or augment the patient’s functional activity. Lineage’s neuroscience focused pipeline currently includes: (i) OpRegen^®, a retinal pigment epithelial cell therapy in Phase 2a development under a worldwide collaboration with Roche and Genentech, a member of the Roche Group, for the treatment of geographic atrophy secondary to age-related macular degeneration; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of spinal cord injuries; (iii) ReSonance™ (ANP1), an auditory neuronal progenitor cell therapy for the potential treatment of auditory neuropathy; (iv) PNC1, a photoreceptor neural cell therapy for the potential treatment of vision loss due to photoreceptor dysfunction or damage; and (v) RND1, a novel hypoimmune induced pluripotent stem cell line being developed under a gene editing partnership. For more information, please visit www.lineagecell.com or follow the company on X/Twitter @LineageCell.

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,” “aim,” “may,” “will,” “estimate,” “continue,” “anticipate,” “design,” “intend,” “expect,” “could,” “can,” “plan,” “potential,” “predict,” “seek,” “should,” “would,” “contemplate,” “project,” “target,” “tend to,” or the negative version of these words and similar expressions. Lineage’s forward-looking statements are based upon its current expectations and beliefs and involve assumptions that may never materialize or may prove to be incorrect. Such statements include, but are not limited to, statements relating to: the plans and expectations with respect to OPC1 and its potential benefits, and the anticipated conduct of the DOSED study for OPC1, including the ability to improve recovery, or allow a patient to regain more functional activity and/or mobility than what could otherwise be expected, or improve a patient’s quality of life; and the potential approval by FDA of the introduction of an immediate-use formulation of OPC1 into clinical testing. 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, but not limited to, the following risks: that Lineage may not receive additional funding from CIRM to support the DOSED study; that the Institutional Review Board does not approve the DOSED study or requires material modifications to the study in order to approve it; that the FDA provides feedback or additional information requests related to the Investigational New Drug amendment for OPC1 or the DOSED study that adversely impacts the study’s progress; Lineage’s ability to design and conduct successful clinical trials, to enroll a sufficient number of patients, to meet established clinical endpoints, to avoid undesirable side effects and other safety concerns, and to demonstrate sufficient safety and efficacy of its product candidates; Lineage’s dependence on third parties to conduct clinical trials, including the UC San Diego Sanford Stem Cell Clinical Center; the risk that positive findings in early clinical and/or nonclinical studies of a product candidate may not be predictive of success in subsequent clinical and/or nonclinical studies of that candidate; Lineage’s ability to develop, obtain FDA approval for, and commercialize its product candidates and to do so on communicated timelines, as well as OPC1’s applicability to a potential chronic SCI patient population; and those risks and uncertainties inherent in Lineage’s business and other risks discussed in Lineage’s filings with the Securities and Exchange Commission (SEC). Further information regarding these and other risks is included under the heading “Risk Factors” in Lineage’s periodic reports with the SEC, including Lineage’s most recent Annual Report on Form 10-K filed with the SEC and its other subsequent reports, which are available from the SEC’s website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Lineage undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. All forward-looking statements are expressly qualified in their entirety by these cautionary statements.

View source version on businesswire.com: https://www.businesswire.com/news/home/20250211889206/en/

Lineage Cell Therapeutics, Inc. IR

Ioana C. Hone

([email protected])

(442) 287-8963

Russo Partners – Media Relations

Nic Johnson or David Schull

([email protected])

([email protected])

(212) 845-4242

KEYWORDS: California United States North America

INDUSTRY KEYWORDS: Science Neurology Stem Cells Biotechnology Research Pharmaceutical Health Clinical Trials

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