NervGen 脊髓损伤药 NVG-291 获 FDA 突破性疗法认定：3期试验招募约150人
NervGen 11月12日在 Florida 州 Miami 宣布，美国食品药品监管局 FDA 已授予其药物 NVG-291 突破性疗法认定，用于治疗慢性创伤性颈段运动不完全脊髓损伤。NVG-291 被开发为有望成为首个促进慢性脊髓损伤患者神经再生的疗法，此前已获 FDA 快速通道认定与欧洲药品管理局孤儿药认定。
认定依据是 1b/2a 期 CONNECT SCI 研究慢性队列的安全性与疗效数据。该研究中，NVG-291 在评估手部功能这一临床重要终点上显示出实质改善，患者自报的总体变化印象与盲法质性访谈也记录到，相较安慰剂，患者日常功能与独立性有所提升。药物总体耐受良好，没有严重不良事件，也没有患者因此停药。
NervGen 首席监管事务官 Shamim Ruff 表示，这一认定说明 FDA 认可 NVG-291 的临床数据前景，该药旨在帮助身体发挥自身的神经再生与修复能力，目标是让受伤多年后的功能恢复成为可能，公司将与 FDA 继续协作推进正在进行的 3 期 RESTORE 研究。脊髓损伤公益组织 Wings for Life USA 首席执行官 Andrew Wagner 表示，美国有超过 30 万人带着创伤性脊髓损伤生活，功能上哪怕一点小的进步，也能给生活质量与日常独立带来深远差别。
3 期 RESTORE 研究是一项多中心、随机、双盲、安慰剂对照的注册性研究，计划在美国与加拿大招募约 150 名慢性创伤性颈段运动不完全脊髓损伤成人患者。主要终点以 GRASSP 定量抓握量表评估手功能改善，这是颈段脊髓损伤患者最看重的恢复目标之一。入组者为受伤 1 至 10 年、双侧运动平面在 C7 或以上、美国脊髓损伤协会残损分级为 C 或 D 级的患者，按 1 比 1 随机接受 NVG-291 或安慰剂、每日一次皮下注射 12 周，其后观察 4 周，研究结束后还计划 12 周开放标签延长期。试验登记号为 NCT07799532。
脊髓损伤会中断大脑与身体之间的信号传导，造成运动、感觉与自主功能的长期损害。美国约有 31.2 万人带着创伤性脊髓损伤生活，每年新增约 1.85 万例。损伤后，硫酸软骨素蛋白聚糖水平升高，会抑制神经纤维再生与发芽，限制恢复。目前尚无 FDA 批准的药物疗法能恢复慢性脊髓损伤患者已丧失的神经功能。
需要说明的是，突破性疗法认定旨在加快针对严重疾病、且初步证据显示可能较现有疗法有实质改善的药物的开发与审评，可带来优先审评资格与滚动提交等便利，但不保证药物最终获批，早期研究结果也未必能预测后续结果。（来源：NervGen）
NervGen Spinal Cord Injury Drug NVG-291 Wins FDA Breakthrough Therapy Designation: Phase 3 Trial Enrolling About 150
NervGen announced in Miami, Florida, on November 12 that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to NVG-291 for the treatment of chronic traumatic cervical motor-incomplete spinal cord injury. NVG-291 is being developed as potentially the first therapy to promote nerve regeneration in people living with chronic spinal cord injury, and it has also received FDA Fast Track designation and European Medicines Agency Orphan Drug designation for spinal cord injury.
The designation was based on safety and efficacy data from the chronic cohort of the Phase 1b/2a CONNECT SCI study. Preliminary evidence showed substantial improvements on a clinically significant endpoint assessing functional hand use, supported by Patient Global Impression of Change results and blinded qualitative interviews documenting participant-reported gains in everyday function and independence compared with placebo. NVG-291 was generally well tolerated, with no serious adverse events or treatment discontinuations.
Shamim Ruff, NervGen's Chief Regulatory Affairs Officer, said the designation underscores the FDA's recognition of the promising clinical data and that NVG-291 is designed to facilitate the body's natural ability to regenerate and repair its nervous system, with the goal of achieving functional recovery even years after injury, adding that the company looks forward to building on the collaboration as it advances its ongoing Phase 3 RESTORE study. Andrew Wagner, CEO of Wings for Life USA, said the designation brings renewed hope to the more than 300,000 individuals living with traumatic spinal cord injury in the United States, and that even small gains in function can make a profound difference to quality of life and everyday independence.
The Phase 3 RESTORE study is a multicenter, randomized, double-blind, placebo-controlled registrational study expected to enroll approximately 150 adults living with chronic traumatic cervical motor-incomplete spinal cord injury across the United States and Canada. Its primary endpoint assesses improvement in hand function, a leading recovery priority for people with cervical injury, as measured by GRASSP Quantitative Prehension. Eligible participants are one to ten years post-injury, have a motor level at C7 or higher on both sides and an American Spinal Injury Association Impairment Scale classification of C or D. Participants are randomized 1:1 to receive NVG-291 or placebo by once-daily subcutaneous injection for 12 weeks, followed by a four-week observation period, with an optional 12-week open-label extension planned. The study is registered as NCT07799532.
Spinal cord injury disrupts communication between the brain and body and can result in lasting impairments in voluntary movement, sensation and autonomic function. Approximately 312,000 people in the United States are living with traumatic spinal cord injury, and about 18,500 new injuries occur each year. After injury, increased levels of chondroitin sulfate proteoglycans contribute to an environment that inhibits nervous system repair. There are currently no FDA-approved pharmacologic therapies to restore lost neurologic function in individuals living with chronic spinal cord injury.
Breakthrough Therapy designation is designed to expedite the development and review of drugs for serious conditions where preliminary evidence indicates the drug may demonstrate substantial improvement over available therapy, and it can bring eligibility for Priority Review and rolling submission. It does not guarantee regulatory approval, and results from earlier studies may not be predictive of subsequent ones. (Source: NervGen)
来源：NervGen
发布：GSM·美国（发布链接）
图片来源：图：正常与损伤脊髓示意图，右为损伤后胶质瘢痕与神经修复方向。Wikimedia Commons，CC BY 4.0。 / Image: Diagram of normal and injured spinal cord, showing glial scar and repair pathways after injury. Via Wikimedia Commons, CC BY 4.0.
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