Lineage Announces FDA Clearance of IND Amendment for OPC1 Cell Transplant for the Treatment of Spinal Cord Injury
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Insights
The FDA clearance of the Investigational New Drug amendment for OPC1 represents a pivotal step for Lineage Cell Therapeutics in advancing its cell therapy program. Oligodendrocyte progenitor cells (OPCs) are crucial in the production of myelin, which insulates nerve fibers and is often damaged in spinal cord injuries (SCIs). The potential therapeutic effects of OPC1 could be ground-breaking, given that there are currently limited treatments for SCI that can significantly restore function.
Previous clinical trials have demonstrated safety and some efficacy, but the sample sizes were small. The DOSED study's inclusion of chronic SCI patients is particularly noteworthy as this population has fewer treatment options and typically exhibits less natural recovery over time. If OPC1 can demonstrate efficacy in this group, it could substantially shift the treatment paradigm for chronic SCI. However, efficacy assessments in chronic conditions are complex and may require extended follow-up to ascertain tangible benefits.
From a financial perspective, the progress of the OPC1 program into further clinical trials is a critical inflection point for Lineage Cell Therapeutics. The biotech sector is highly sensitive to regulatory milestones and FDA clearance is often a catalyst for investor interest. The potential market for SCI treatments is significant, with few competitors offering restorative therapies, positioning OPC1 as a potentially disruptive technology in this space.
Investors will be closely monitoring the enrollment and execution of the DOSED study, as delays or issues could impact the company's valuation and burn rate. The pursuit of external financial support from the California Institute for Regenerative Medicine (CIRM) is a strategic move to mitigate financial risk and extend the company's cash runway. The outcome of the CIRM grant application will be an important financial event to watch.
The healthcare system bears a substantial economic burden from spinal cord injuries, including long-term care costs and lost productivity. An effective treatment like OPC1 could reduce this burden by improving outcomes for SCI patients. The economic implications extend beyond direct healthcare savings; successful treatments can enhance patients' quality of life and societal participation, contributing to overall economic productivity.
However, the cost-effectiveness of such treatments must be evaluated. Cell therapies are typically expensive and reimbursement by insurance companies will hinge on the therapy's ability to demonstrate clear benefits over existing treatments. The long-term economic impact will depend on the balance between the treatment's cost and the potential to reduce the economic burden of SCIs.
The DOSED Clinical Study Will Evaluate a Novel Delivery Device in Subacute and Chronic Spinal Cord Injury Patients
“Lineage’s oligodendrocyte cell transplants are designed to replace or support cells that are absent or dysfunctional due to traumatic injury, with the goal of helping to improve the quality of life and restore or augment functional activity to persons suffering from traumatic cervical or thoracic spinal cord injuries. The clearance of our INDa and the initiation of OPC1 clinical testing under our sponsorship represents a significant milestone for this program, and reflects our commitment to developing modern cell therapy product candidates,” stated Brian M. Culley, Lineage CEO. “We are excited by the opportunity to build upon the promising results achieved with OPC1 in previous trials, and to continue to seek improvements in how our therapy is prepared and administered. The DOSED clinical study in particular is intended to evaluate the safety and performance of a new delivery device, which is compatible with our forthcoming immediate-use formulation, and which does not require cessation of patient ventilation during administration. We believe these improvements can lead to a safer surgical procedure for surgeons and patients. This study also will mark the first time that OPC1 is administered to chronic SCI patients, and we will be collecting efficacy assessments in addition to the primary outcome measures of safety. Completing this regulatory step also enables us to proceed with our planned CLIN-2 grant application to CIRM to request external financial support for the OPC1 program.”
OPC1 has been tested in two clinical trials to date: a five patient Phase 1 clinical safety trial in acute thoracic SCI, where all subjects have been followed for at least 10 years; as well as a 25 patient Phase 1/2a multicenter dose-escalation clinical trial in subacute cervical SCI, where all subjects were evaluated for at least two years, one of the first cell therapy clinical trials supported by the California Institute for Regenerative Medicine under Proposition 71. Results from both studies have been published in the Journal of Neurosurgery: Spine. The data from the Phase 1/2a clinical study of OPC1 in subacute cervical SCI is available here and the data from the Phase 1 clinical study of OPC1 in acute thoracic SCI is available here.
About the DOSED Clinical 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 approximately 3-5 subacute and 3-5 stable chronic subjects with complete (
About OPC1
OPC1 is an oligodendrocyte progenitor cell (OPC) transplant therapy designed to provide clinically meaningful improvements to motor recovery in individuals with spinal cord injuries (SCI). OPCs are naturally occurring precursors to the cells which provide electrical insulation for nerve axons in the form of a myelin sheath. SCI most often occurs when the spinal cord is subjected to a severe crush or contusion injury and typically results in severe functional impairment, including limb paralysis, aberrant pain signaling, and loss of bladder control and other body functions. There are approximately 18,000 new spinal cord injuries annually in the
About the California Institute for Regenerative Medicine (CIRM)
CIRM,
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 and preclinical programs are in markets with billion dollar opportunities and include five allogeneic (“off-the-shelf”) product candidates: (i) OpRegen®, a retinal pigment epithelial cell therapy in Phase 2a development for the treatment of geographic atrophy secondary to age-related macular degeneration, is 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, a dendritic cell therapy produced from Lineage’s VAC technology platform for immuno-oncology and infectious disease, currently in Phase 1 clinical development for the treatment of non-small cell lung cancer; (iv) ANP1, an auditory neuronal progenitor cell therapy for the potential treatment of auditory neuropathy; and (v) PNC1, a photoreceptor neural cell therapy for the potential treatment of vision loss due to photoreceptor dysfunction or damage. 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 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. Such statements include, but are not limited to, statements relating to: the ability of cell transplant therapies, including OPC1, to improve recovery, allow a patient to regain more function than what could otherwise be expected, or improve a patient’s quality of life; the potential future achievements of our clinical, preclinical and development programs, the planned initiation of clinical trials, including the expected initial site opening for the DOSED clinical study in the second quarter of 2024; that the improvements in the delivery device for OPC1 can lead to a safer surgical procedure; and the timing and availability of clinical data updates related to our programs. 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 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; 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). Lineage’s forward-looking statements are based upon 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 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 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.
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Lineage Cell Therapeutics, Inc. IR
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Source: Lineage Cell Therapeutics, Inc.
FAQ
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