FDA 授予 LioCyx-M004 用于 HBV 相关肝细胞癌的快速通道指定 2021

StephenW

FDA 授予 LioCyx-M004 用于 HBV 相关肝细胞癌的快速通道指定
2021 年 12 月 24 日
马修福勒

FDA 快速通道指定是基于一项研究 LioCyx-M004 输注治疗原发性 HBV 相关肝细胞癌的 1 期试验的总体生存改善。

根据负责治疗乙型肝炎病毒 (HBV) 相关肝细胞癌 (HCC) 患者的潜在一流疗法 LioCyx-M004，FDA 授予其快速通道指定资格。药物，Lion TCR Pte Ltd.1

该指定是疗效数据的结果，特别关注总生存期 (OS)，与 LioCyx-M004 在乙型肝炎表面抗原 (HBsAg) 阳性 HCC 患者中使用有关，这些患者复发或对先前的全身治疗无效。

“全球每年有超过 420,000 人发生与 HBV 相关的 HCC，大多数晚期 HCC 患者在初始治疗后迅速复发。然而，现有的治疗方法非常有限，尤其是在提高总体生存率方面，” Lion TCR 的首席运营官兼首席营销官 Tina Tingting Wang 在一份新闻稿中说。 “我们相信，我们创新的 TCR-T 疗法可以满足这一紧迫且重要的未满足医疗需求。有了这个快速通道指定，我们期待与 FDA 进行更频繁的沟通，希望我们的产品能够更快地获得药物批准，以供患者使用。”

在之前的研究中，一项 1 期研究 (NCT03634683) 调查了 LioCyx-M004 在原发性 HBV 相关 HCC 中的输注。接受治疗的患者中没有发生细胞因子释放综合征 (CRS)，也没有神经毒性迹象。疾病控制率为60%，缓解持续时间为27.7个月。该人群的中位 OS 为 33.1 个月。2

评估 LioCyx-M004 作为单一疗法以及与乐伐替尼 (Lenvima) 联合治疗 HBV 相关 HCC 患者的安全性和有效性的未来 1b/2 期多中心研究获得批准。

“T细胞疗法领域充满活力和竞争。通过快速通道等加速程序，创新疗法的发布速度比以往任何时候都快。连同我们因在 HCC 中使用 HBV 特异性 TCR T 细胞疗法而获得的孤儿药物指定，我们相信这一快速通道批准可以推动我们专有的一流 TCR T 细胞疗法加速监管批准。” Lion TCR 首席执行官彭晓明在一份新闻稿中表示。

快速通道指定旨在为可以解决可能危及生命的情况的代理加速获得批准。该指定侧重于可以解决未满足的医疗需求的代理，通过与 FDA 的早期和频繁会议来加快批准过程。

StephenW

FDA Grants Fast Track Designation to LioCyx-M004 for HBV-related Hepatocellular Carcinoma
December 24, 2021
Matthew Fowler

FDA fast track designation comes based on overall survival improvements from a phase 1 trial investigating LioCyx-M004 infusions in primary HBV-related hepatocellular carcinoma.

The FDA granted Fast Track Designation to a potential first-in-class therapy, LioCyx-M004, for the treatment of patients with Hepatitis B virus (HBV)–related hepatocellular carcinoma (HCC), according to a press release from the company responsible for the drug, Lion TCR Pte Ltd.1

The designation is a result of efficacy data, specifically focusing on overall survival (OS), related to LioCyx-M004 use in patients with Hepatitis B surface antigen (HBsAg)–positive HCC who relapsed or were refractory to prior systemic treatment.

“HBV-related HCC occurs in over 420,000 people every year worldwide and majority of advanced HCC patients relapse quickly after initial treatment. However, existing treatments are very limited especially on improving overall survival,” Tina Tingting Wang, COO and CMO of Lion TCR, said in a press release. “We believe that our innovative TCR-T therapy can fill this urgent and important unmet medical needs. With this Fast Track designation, we look forward to having more frequent communication with the Agency in the hope to attain a more expedited drug approval for our product for patient access.”

In previous research, a phase 1 study (NCT03634683) investigated LioCyx-M004 infusions in primary HBV-related HCC. There were no occurrences of cytokine release syndrome (CRS) among patients who received the therapy, as well as no signs of neurotoxicity. The disease control rate was 60% and duration of response was 27.7 months. Median OS in that population was 33.1 months.2

Clearance was obtained for a future phase 1b/2 multicenter study evaluating the safety and efficacy of LioCyx-M004 as a monotherapy and in combination with lenvatinib (Lenvima) for patients with HBV-related HCC.

“The field of T-cell therapy is highly dynamic and competitive. Innovative therapies are released faster than ever through expediated programs like Fast Track. Together with our Orphan Drug Designation obtained for the use of HBV-specific TCR T-cell therapy in HCC, we believe this Fast Track approval can drive forward the accelerated regulatory approval our proprietary first-in-class TCR T cell therapy.” Peng Xiaoming, CEO of Lion TCR, said in a press release.

Fast Track Designation is designed to grant accelerated access to approval for agents that can address potentially life-threatening conditions. The designation focuses on agents that can address unmet medical needs, expediting the approval process through early and frequent meetings with the FDA.