/PRNewswire/ -- Amphix Bio, a pre-clinical stage company developing a new therapeutic modality for regenerative medicine, received an Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for its lead candidate AMFX-200 for treatment of acute spinal cord injury (SCI). SCI is a devastating condition often leading to permanent paralysis, with approximately 18,000 new cases each year in .

/PRNewswire/ -- Amphix Bio是一家处于临床前阶段的公司，致力于开发一种用于再生医学的全新治疗方式，其主导候选药物AMFX-200已获得美国食品药品监督管理局（FDA）授予的孤儿药资格，用于治疗急性脊髓损伤（SCI）。脊髓损伤是一种破坏性极强的疾病，常导致永久性瘫痪，每年约有18,000例新发病例。

the United States

美国

AMFX-200 is based on a technology platform called Supramolecular Therapeutic Peptides (STPs), where peptides function both as a drug to activate cell receptors and as a physical nanofiber scaffold to support tissue regeneration. This technology was pioneered in the research group of Professor

AMFX-200基于一个名为超分子治疗肽（STPs）的技术平台，其中肽既作为激活细胞受体的药物，又作为支持组织再生的物理纳米纤维支架。这项技术是由教授的研究团队开创的。

Northwestern team made a key breakthrough in 2021 by discovering the ability to control the collective motion of molecules within the nanofibers, a strategy that enhanced biological potency and stimulated unprecedented neural regeneration.

西北大学团队在 2021 年取得了关键性突破，发现了控制纳米纤维内部分子集体运动的能力，这一策略增强了生物效力，并刺激了前所未有的神经再生。

Leveraging the STP platform, Amphix Bio is advancing regenerative therapeutics for neurological and musculoskeletal conditions. Starting with established biology and known high-impact targets for cellular regeneration, the company bridges the gap between scientific discovery and clinically viable therapeutics.

利用STP平台，Amphix Bio正在推进针对神经和肌肉骨骼疾病的再生疗法。公司以已确立的生物学基础和细胞再生领域中已知的高影响力靶点为起点，弥合了科学发现与临床可行疗法之间的差距。

In preclinical models of acute spinal cord injury, a single injection of AMFX-200 into the spinal cord enables motor neurons from the brain to regrow past the injury site, re-establish severed connections, and restore motor function — effectively reversing paralysis..

在急性脊髓损伤的临床前模型中，向脊髓注射一次AMFX-200能够使大脑的运动神经元在损伤部位重新生长，重建断裂的连接，并恢复运动功能——有效逆转瘫痪。

'Since we first published these results in 2021, we have further validated the ability of these molecules to regenerate functional neural tissue in additional preclinical models, different types of spinal cord injury, and in other neural injuries and neurodegenerative diseases,' said Stupp, who also serves as Chief Scientific Officer of Amphix Bio.

“自从我们在2021年首次发布这些结果以来，我们进一步验证了这些分子在其他临床前模型、不同类型脊髓损伤以及其他神经损伤和神经退行性疾病中再生功能性神经组织的能力，”同时担任Amphix Bio首席科学官的斯图普表示。

'This represents a paradigm shift in the development of neural regeneration therapeutics.'.

“这代表了神经再生治疗发展中的一个范式转变。”

The company received preliminary feedback from the FDA on the drug development program for acute SCI late last year, and is now focused on completing safety studies required for regulatory approval to begin a first-in-human clinical trial in SCI patients.

该公司去年年底收到了 FDA 关于急性 SCI 药物开发计划的初步反馈，目前正专注于完成监管机构要求的安全性研究，以启动首个针对 SCI 患者的人体临床试验。

'Experiencing a spinal cord injury is devastating for patients and their families, as it leads to a lifetime of lost mobility, high healthcare costs, and a heavy reliance on caregivers,' said Dr.

“脊髓损伤对患者及其家人来说是毁灭性的，因为它会导致终身行动能力丧失、高昂的医疗费用以及严重依赖护理人员，”博士说道。

James Guest

詹姆斯·格斯特

, a neurosurgeon at the

，一位神经外科医生在

University of Miami

迈阿密大学

and the Miami Project to Cure Paralysis. 'Previous attempts at treatments have faced many limitations but Amphix Bio is taking a completely new approach to neural regeneration, with the potential for a breakthrough in the field.'

以及迈阿密瘫痪治愈计划。‘以往的治疗尝试面临许多限制，但Amphix Bio正在采取一种全新的神经再生方法，有望在该领域取得突破。’

About Orphan Drugs

关于孤儿药

The FDA's Orphan Drug program is designed to encourage and support the development of treatments for rare diseases or conditions. Benefits of Orphan Drug Designation include financial incentives such as tax credits for clinical trials, exemption from user fees, and up to seven years of market exclusivity after approval.

FDA的孤儿药计划旨在鼓励和支持针对罕见疾病或病症的治疗开发。获得孤儿药资格的好处包括财务激励措施，例如临床试验的税收抵免、免除用户费用，以及在获批后可享有长达七年的市场独占期。

Companies are also eligible to apply for clinical trial grants through the Office of Orphan Product Development to offset the cost of efficacy and/or safety studies..

公司也有资格通过孤儿产品开发办公室申请临床试验资助，以抵消疗效和/或安全性研究的成本。

About Amphix Bio

关于Amphix Bio

in 2021. The company is developing a new platform technology, Supramolecular Therapeutic Peptides (STPs), that instruct cells to initiate regenerative processes and restore function lost from injury, disease, or aging. These therapies trigger targeted signaling pathways while forming scaffolds to support tissue growth.

2021年，该公司正在开发一种新的平台技术——超分子治疗肽（STPs），这种技术能够指导细胞启动再生过程，恢复因受伤、疾病或衰老而丧失的功能。这些疗法在触发靶向信号通路的同时形成支架以支持组织生长。

Amphix Bio's goal is to use regenerative medicine to increase human healthspan — the length of time that people can live productive, fulfilling lives..

Amphix Bio 的目标是利用再生医学延长人类的健康寿命——即人们能够过上富有成效、充实生活的时长。