CARLSBAD, Calif.--(BUSINESS WIRE)--Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, announced today that the Company has submitted an Investigational New Drug amendment (INDa) to the U.S.

加利福尼亚州卡尔斯巴德（商业新闻短讯）--Lineage Cell Therapeutics，Inc.（纽约证券交易所和塔斯社：LCTX），一家临床阶段的生物技术公司，为未满足的医疗需求开发同种异体细胞疗法，今天宣布该公司已向美国提交了一份研究性新药修正案（INDa）。

Food and Drug Administration (FDA), for OPC1, its investigational allogeneic oligodendrocyte progenitor cell transplant, for the treatment of spinal cord injury (SCI). Receipt of FDA clearance of the INDa would enable the Company to initiate its DOSED (Delivery of Oligodendrocyte Progenitor Cells for Spinal Cord Injury: Evaluation of a Novel Device) clinical study, to evaluate the safety and utility of a novel spinal cord delivery device in both subacute and chronic SCI patients.

美国食品和药物管理局（FDA）针对OPC1，其研究性异基因少突胶质细胞祖细胞移植，用于治疗脊髓损伤（SCI）。获得FDA对INDa的批准将使该公司能够启动其DOSED（脊髓损伤少突胶质细胞祖细胞的递送：新型装置的评估）临床研究，以评估新型脊髓递送装置在亚急性和慢性SCI患者中的安全性和实用性。

The OPC1 cell transplant is designed to replace or support cells that are absent or dysfunctional due to traumatic injury, with a goal to help improve the quality of life and restore or augment functional activity in persons suffering from traumatic cervical or thoracic SCIs..

OPC1细胞移植旨在替代或支持因创伤性损伤而缺失或功能失调的细胞，目的是帮助改善生活质量，恢复或增强患有创伤性宫颈或胸部SCI的人的功能活动。。

“The submission of our IND amendment for OPC1 and its return to clinical testing represents a significant milestone for this program and a diligent effort by our team,” stated Brian M. Culley, Lineage CEO. “With so few opportunities for SCI patients to participate in clinical trials, it is a privilege to be able to re-engage with the community as part of our efforts to improve outcomes for individuals with a debilitating condition for which there currently are no FDA-approved treatments.

Lineage首席执行官布莱恩·卡利（BrianM.Culley）表示：“我们提交的OPC1 IND修正案及其恢复临床测试代表了该计划的一个重要里程碑，也是我们团队的辛勤努力。”。“脊髓损伤患者参加临床试验的机会如此之少，因此能够重新参与社区是我们努力改善目前尚无FDA批准治疗的虚弱患者预后的一部分。

Our team has made a significant effort to introduce improvements to the OPC1 program, including enhancements to the production process and quality of our product candidate and an improved delivery system compatible with our immediate use, thaw-and-inject formulation. We are encouraged by the meaningful quality of life and safety results observed with OPC1 in previous trials, and we look forward to building on that promising work.

我们的团队已经做出了巨大的努力来改进OPC1计划，包括改进生产过程和候选产品的质量，以及改进与我们的即时使用，解冻和注射配方兼容的输送系统。我们对OPC1在以前的试验中观察到的有意义的生活质量和安全性结果感到鼓舞，我们期待着在这项有希望的工作的基础上再接再厉。

We are excited by the opportunity to initiate the DOSED study and the opening of the first clinical sites in early 2024, assuming no further comments are received from the FDA in the 30 days following our INDa submission.”.

假设在我们提交INDa后的30天内没有收到FDA的进一步评论，我们很高兴有机会在2024年初启动DOSED研究并开放第一个临床站点。”。

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.

迄今为止，OPC1已在两项临床试验中进行了测试；一项针对急性胸部SCI的五名患者的1期临床安全性试验，所有受试者均已随访至少10年；以及亚急性宫颈SCI的25名患者1/2a期多中心剂量递增临床试验，所有受试者均接受了至少两年的评估，这是加州再生医学研究所根据第71号提案支持的首批细胞治疗临床试验之一。

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..

这两项研究的结果均已发表在《神经外科杂志：脊柱》上：此处提供了亚急性颈椎SCI中OPC1的1/2a期临床研究数据，此处提供了急性胸部SCI中OPC1的1期临床研究数据。。

About the DOSED Study

关于剂量研究

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 (ASIA Impairment Scale A) or incomplete (ASIA Impairment Scale B), traumatic, focal SCI affecting either cervical (C4-C7) or thoracic (T1-T10) vertebrae.

少突胶质祖细胞在脊髓损伤中的应用：新型装置（DOSED）临床研究的评估是一项开放标签，多中心，装置安全性研究，在大约3-5名亚急性和3-5名稳定的慢性受试者中进行，这些受试者具有完全（ASIA损伤量表a）或不完全（ASIA损伤量表B），创伤性，局灶性SCI影响颈椎（C4-C7）或胸椎（T1-T10）椎骨。

The primary objective of this study is to evaluate the safety of a novel spinal cord delivery device to administer OPC1 to the spinal parenchyma. The primary endpoint is safety, as measured by the frequency and severity of adverse events (AEs) through 30 days following OPC1 injection that are related to the injection procedure.

这项研究的主要目的是评估一种新型脊髓输送装置将OPC1施用于脊髓实质的安全性。主要终点是安全性，通过与注射程序相关的OPC1注射后30天内不良事件（AE）的频率和严重程度来衡量。

Secondary endpoints are safety and tolerability, as measured by the frequency and severity of AEs, including AEs of special interest, through 90 days following OPC1 injection, that are related to OPC1 and/or the concomitant short-term immunosuppression. Safety parameters will be evaluated by magnetic resonance imaging (MRI) data evaluating evidence of deterioration or changes in the following: intramedullary hemorrhage, cerebral spinal fluid (CSF) leak, epidural abscess, infection; evidence of an expanding cyst or mass at the injection site or elsewhere in the central nervous system (CNS); evidence of inflammatory lesion(s) at injection site or elsewhere in CNS and evidence of CSF flow obstruction.

次要终点是安全性和耐受性，通过注射OPC1后90天内与OPC1和/或伴随的短期免疫抑制有关的AE（包括特别感兴趣的AE）的频率和严重程度来衡量。安全参数将通过磁共振成像（MRI）数据评估以下情况恶化或变化的证据：髓内出血，脑脊液（CSF）渗漏，硬膜外脓肿，感染；注射部位或中枢神经系统（CNS）其他部位囊肿或肿块扩张的证据；注射部位或中枢神经系统其他部位炎性病变的证据以及脑脊液流动阻塞的证据。

Exploratory endpoints include: (1) potential improvements in neurological impairment, function, and 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) exami.

探索性终点包括：（1）神经功能障碍，功能和疼痛的潜在改善，通过以下终点的基线变化进行评估：根据国际脊髓损伤神经分类标准（ISNCSCI）exami的感觉和运动评分以及运动水平测量的神经功能变化。

About OPC1

关于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.

OPC1是一种少突胶质祖细胞（OPC）移植疗法，旨在为脊髓损伤（SCI）患者的运动恢复提供临床上有意义的改善。OPCs是细胞的天然前体，以髓鞘的形式为神经轴突提供电绝缘。

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 U.S.

脊髓损伤最常发生在脊髓受到严重挤压或挫伤损伤时，通常会导致严重的功能障碍，包括肢体瘫痪，异常疼痛信号传导以及膀胱控制和其他身体功能丧失。美国每年约有18000例新的脊髓损伤。

and there currently are no FDA-approved drugs or interventions specifically for the treatment of SCI. The OPC1 program has been partially funded by a $14.3 million grant from the California Institute for Regenerative Medicine. OPC1 has received Regenerative Medicine Advanced Therapy (RMAT) designation and Orphan Drug designation from the U.S.

目前还没有FDA批准的专门用于治疗SCI的药物或干预措施。OPC1计划的部分资金来自加利福尼亚再生医学研究所1430万美元的赠款。OPC1已获得美国再生医学高级治疗（RMAT）和孤儿药的称号。

Food and Drug Administration (FDA)..

美国食品和药物管理局（FDA）。。

About the California Institute for Regenerative Medicine (CIRM)

关于加利福尼亚再生医学研究所（CIRM）

CIRM, California’s Stem Cell Agency, was created by the voters of California in 2004 with the passing of Proposition 71, which authorized $3 billion in funding for stem cell research in California. The agency funds stem cell research at institutions and companies throughout California (as well as institutions and companies outside of the state that conduct a portion of their research in California) with the goal of accelerating treatments to patients with unmet medical needs.

加州干细胞管理局（CIRM）是加州选民于2004年通过71号提案创建的，该提案授权30亿美元用于加州干细胞研究。该机构为整个加利福尼亚州的机构和公司（以及在加利福尼亚州进行部分研究的州外机构和公司）的干细胞研究提供资金，目的是加速对未满足医疗需求的患者的治疗。

In 2020, California voters approved to continue funding California’s Stem Cell Agency through the passage of Proposition 14. CIRM’s mission is to accelerate world class science to deliver transformative regenerative medicine treatments in an equitable manner to a diverse California and world as well as to accelerate stem cell treatments to patients with unmet medical needs.

2020年，加州选民通过第14号提案，批准继续资助加州干细胞局。CIRM的使命是加速世界一流的科学，以公平的方式向加利福尼亚州和世界各地提供变革性再生医学治疗，并加速对未满足医疗需求的患者进行干细胞治疗。

By promoting and encouraging the growth of the stem cell biotechnology sector, the agency is also helping attract the best scientists to the state and establishing California as a global leader in stem cell research. For more information, please visit https://www.cirm.ca.gov/ and follow the agency on Twitter: @CIRMnews..

通过促进和鼓励干细胞生物技术部门的发展，该机构还帮助吸引最优秀的科学家来到加州，并将加州打造为干细胞研究的全球领导者。有关更多信息，请访问https://www.cirm.ca.gov/并在推特上关注该机构：@CIRMnews。。

About Lineage Cell Therapeutics, Inc.

关于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.

Lineage Cell Therapeutics是一家临床阶段的生物技术公司，为未满足的医疗需求开发新型细胞疗法。Lineage的计划基于其强大的专有细胞治疗平台以及相关的内部开发和制造能力。通过这个平台，谱系从其多能和祖细胞起始材料中开发和制造专门的，终末分化的人类细胞。

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.

这些分化的细胞被开发用于替代或支持由于退行性疾病或创伤性损伤而功能失调或不存在的细胞，或者作为帮助身体对癌症产生有效免疫应答的手段。Lineage的临床和临床前项目在市场上拥有数十亿美元的机会，其中包括五种同种异体（“现成”）候选产品：（i）OpRegen®，一种在2a期开发中的视网膜色素上皮细胞疗法，用于治疗继发于年龄相关性黄斑变性的地理萎缩，正在与罗氏集团成员罗氏和基因泰克的全球合作下开发；（ii）OPC1，一种在1/2a期发育中用于治疗急性脊髓损伤的少突胶质细胞祖细胞疗法；（iii）VAC2，一种由Lineage的免疫肿瘤学和传染病VAC技术平台产生的树突状细胞疗法，目前正在进行治疗非小细胞肺癌的第一阶段临床开发；（iv）ANP1，一种用于潜在治疗听神经病的听觉神经元祖细胞疗法；（v）PNC1，一种光感受器神经细胞疗法，可用于治疗由于光感受器功能障碍或损伤引起的视力丧失。

For more information, please visit www.lineagecell.com or follow the company on Twi.

有关更多信息，请访问www.lineagecell.com或在Twi上关注该公司。

Forward-Looking Statements

前瞻性声明

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提醒您，本新闻稿中包含的所有声明（历史事实声明除外）均为前瞻性声明。在某些情况下，前瞻性陈述，可以通过诸如“相信”、“目标”、“可能”、“会”、“估计”、“继续”、“预期”、“设计”、“打算”、“期望”、“可能”、“能”、“计划”、“潜力”、“预测”、“寻求”、“应该”、“会”、“沉思”、“项目”、“目标”、“倾向于”等术语来识别，或者这些单词的负面版本和类似版本表达式。

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; and the potential future achievements of our clinical, preclinical and development programs, the planned initiation of clinical trials, including the DOSED study in early 2024, and the timing and availability of clinical data updates related to our programs.

这些陈述包括但不限于以下陈述：细胞移植疗法（包括OPC1）改善恢复的能力，使患者恢复比预期更多的功能，或改善患者的生活质量；以及我们的临床，临床前和开发计划的潜在未来成就，计划开始的临床试验，包括2024年初的DOSED研究，以及与我们的计划相关的临床数据更新的时间和可用性。

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; that the feedback received from the FDA for OPC1 may not enable further clinical development; and those risks and uncertainties inherent in Lineage’s business and other risks discussed in Lineage.

前瞻性声明涉及已知和未知的风险、不确定性和其他因素，这些因素可能导致Lineage的实际结果、业绩或成就与本新闻稿中前瞻性声明所表达或暗示的未来结果、业绩或成就存在重大差异，包括但不限于，以下风险：候选产品早期临床和/或非临床研究的阳性结果可能无法预测该候选产品随后的临床和/或非临床研究的成功；FDA对OPC1的反馈可能无法进一步进行临床开发；以及Lineage业务中固有的风险和不确定性以及Lineage中讨论的其他风险。