CNM-Au8 对多发性硬化症表现出长期改善

在 2 期临床试验 (VISIONARY-MS) 中，使用 Clene Nanomedicine 的 CNM-Au8 进行长期治疗表明，复发缓解型多发性硬化症 (RRMS) 患者的神经和髓磷脂健康状况在三年内持续改善。 CNM-Au8 是一种含有金纳米颗粒的口服疗法，以神经细胞能量产生为目标，这是一种不同于其他多发性硬化症疗法的新机制。这些发现支持 Clene 的第三阶段试验计划和潜在的药物批准申请。

Clene Nanomedicine's CNM-Au8 Demonstrates Long-Term Improvements in Multiple Sclerosis

Clene Nanomedicine 的 CNM-Au8 显示对多发性硬化症的长期改善

Denver, Colorado, April 18, 2024 - Data from the three-year VISIONARY-MS clinical trial presented at the American Academy of Neurology (AAN) 2024 Annual Meeting provide further evidence of the potential therapeutic benefits of Clene Nanomedicine's experimental oral therapy CNM-Au8 in relapsing-remitting multiple sclerosis (RRMS).

科罗拉多州丹佛，2024 年 4 月 18 日 - 在美国神经病学学会 (AAN) 2024 年年会上公布的为期三年的 VISIONARY-MS 临床试验的数据进一步证明了 Clene Nanomedicine 的实验性口服疗法 CNM-Au8 的潜在治疗益处复发缓解型多发性硬化症（RRMS）。

Sustained Nerve and Myelin Improvements

神经和髓磷脂持续改善

Long-term treatment with CNM-Au8 led to sustained improvements in the health of nerves and myelin over three years, according to the findings. This was evident in improvements in nerve function, as measured by visual evoked potentials, and in markers of myelin repair, or remyelination, as measured by magnetic resonance imaging (MRI).

研究结果显示，三年来长期使用 CNM-Au8 治疗可以持续改善神经和髓磷脂的健康状况。这在神经功能的改善（通过视觉诱发电位测量）以及髓磷脂修复或髓鞘再生标志物（通过磁共振成像（MRI）测量）方面表现得很明显。

Mechanism of Action

作用机制

CNM-Au8 is a liquid suspension of gold nanoparticles that is designed to boost nerve cells' energy production. This mechanism is distinct from approved disease-modifying therapies (DMTs) for MS, which work to reduce inflammation in the brain.

CNM-Au8 是一种金纳米粒子的液体悬浮液，旨在促进神经细胞的能量产生。这种机制与已批准的多发性硬化症疾病缓解疗法 (DMT) 不同，后者旨在减少大脑炎症。

Phase 3 Trial Planned

计划进行第三阶段试验

The findings support Clene's plans to launch a Phase 3 trial of CNM-Au8 in MS. Such a trial would provide further evidence of the therapy's efficacy and safety and may support applications toward its approval.

这些发现支持 Clene 计划在 MS 中启动 CNM-Au8 的 3 期试验。这样的试验将为该疗法的有效性和安全性提供进一步的证据，并可能支持其批准申请。

VISIONARY-MS Extension Study

VISIONARY-MS 推广研究

The VISIONARY-MS clinical trial (NCT03536559) initially tested CNM-Au8 against a placebo in 73 people with RRMS who had not had a relapse for at least six months before entering the study. After a year, those given CNM-Au8 had significant improvements in vision, suggesting that the therapy improved the health of nerve fibers in the brain.

VISIONARY-MS 临床试验 (NCT03536559) 最初在 73 名 RRMS 患者中测试了 CNM-Au8 与安慰剂的对比，这些患者在进入研究之前至少六个月没有复发。一年后，接受 CNM-Au8 治疗的患者视力有了显着改善，这表明该疗法改善了大脑神经纤维的健康。

Fifty-five participants from the original trial then entered into an extension study (VISIONMS-LTE; NCT04626921), where they were treated with CNM-Au8 for an additional two years, for a total of three years of treatment.

来自原始试验的 55 名参与者随后进入了一项扩展研究（VISIONMS-LTE；NCT04626921），他们接受 CNM-Au8 治疗额外两年，总共治疗三年。

Long-Term Efficacy Data

长期疗效数据

Data from the extension study showed that the improvements in vision and cognitive function observed after one year of CNM-Au8 treatment were maintained for three years. Additionally, those who switched from a placebo to CNM-Au8 in the extension study saw improvements in these measures.

扩展研究的数据显示，CNM-Au8 治疗一年后观察到的视力和认知功能改善可维持三年。此外，那些在扩展研究中从安慰剂转向 CNM-Au8 的人看到了这些指标的改善。

Improvements in Nerve Damage

改善神经损伤

The new data presented at AAN included further evidence that the therapy may be repairing damaged optic nerves. Visual evoked potential latency and amplitude measures improved in patients who started on CNM-Au8 in the placebo-controlled part of the trial, suggesting that the damaged optic nerves were repairing.

AAN 上公布的新数据进一步证明该疗法可能正在修复受损的视神经。在试验的安慰剂对照部分中，开始使用 CNM-Au8 的患者的视觉诱发电位潜伏期和振幅测量有所改善，表明受损的视神经正在修复。

Remyelination Markers

髓鞘再生标记物

MRI data also indicated that CNM-Au8 led to long-term increases in markers of myelin repair. Myelin is a fatty substance that wraps around nerve fibers to help them send electrical signals, but is damaged by the misguided inflammatory attacks in MS.

MRI 数据还表明 CNM-Au8 导致髓磷脂修复标记物的长期增加。髓磷脂是一种脂肪物质，包裹在神经纤维周围，帮助神经纤维发送电信号，但会因多发性硬化症中误导性炎症攻击而受损。

Expert Commentary

专家评论

"These results are particularly exciting and provide a clear impetus for a Phase 3 study," said Michael Barnett, PhD, a clinical advisor for the trial, during his presentation at AAN. "The development of adjunctive therapies that not only prevent neurodegeneration, but also improve neuronal function with measurable clinical benefit, will fill a major unmet need for people living with MS."

该试验的临床顾问 Michael Barnett 博士在 AAN 的演讲中表示：“这些结果特别令人兴奋，为 3 期研究提供了明显的推动力。” “辅助疗法的开发不仅可以预防神经退行性变，而且可以改善神经元功能并具有可测量的临床益处，这将满足多发性硬化症患者未满足的主要需求。”

"Observing such a profound clinical benefit with corresponding improvements in physiologic measures utilizing a mechanism that does not target immune system modulation has never been demonstrated in prior multiple sclerosis trials," added Benjamin Greenberg, MD, head of medicine at Clene.

Clene 医学主任本杰明·格林伯格医学博士补充道：“在之前的多发性硬化症试验中，从未证明过利用不针对免疫系统调节的机制观察到如此深远的临床益处以及相应的生理措施改善。”

Conclusion

结论

The long-term data from the VISIONARY-MS clinical trial provide further evidence of the potential therapeutic benefits of Clene Nanomedicine's CNM-Au8 in RRMS. The therapy demonstrates sustained improvements in nerve and myelin health and may represent a promising new treatment option for people with this debilitating condition.

VISIONARY-MS 临床试验的长期数据进一步证明了 Clene Nanomedicine 的 CNM-Au8 在 RRMS 中的潜在治疗效果。该疗法证明了神经和髓磷脂健康的持续改善，对于患有这种衰弱疾病的人来说可能是一种有前途的新治疗选择。