PHILADELPHIA--(BUSINESS WIRE)--Aro Biotherapeutics, a clinical-stage biotechnology company working to develop potent and tissue-targeted medicines, today announced the initiation of the Phase 1b portion of its clinical trial of ABX1100, a novel potential treatment for late-onset Pompe disease (LOPD).

费城--（商业新闻短讯）--致力于开发强效和组织靶向药物的临床阶段生物技术公司Aro Biotherapeutics今天宣布开始其ABX1100临床试验的1b期部分，这是一种治疗迟发性庞贝病（LOPD）的新型潜在疗法。

By targeting a known receptor called CD71, ABX1100 delivers siRNA therapy to muscle tissue, where it inhibits the production of the GYS1 enzyme, which is responsible for synthesizing glycogen. In the planned 1b study, investigators seek to enroll adults with LOPD to evaluate the safety and bioactivity of ABX1100.

通过靶向一种名为CD71的已知受体，ABX1100将siRNA疗法传递到肌肉组织，从而抑制负责合成糖原的GYS1酶的产生。在计划的1b研究中，研究人员试图招募患有LOPD的成年人，以评估ABX1100的安全性和生物活性。

More information about the trial is available at ClinicalTrials.gov using the identifier NCT06109948..

有关该试验的更多信息，请访问ClinicalTrials.gov，使用标识符NCT06109948。。

“The limitations of enzyme replacement therapy for Pompe disease underscore the need for new therapies with novel mechanisms of action,” said Aneal Khan, MD, principal investigator of the trial and a pediatrician and medical geneticist at the Metabolic and Genetic in Calgary (MAGIC) Clinic in Alberta, Canada.

该试验的首席研究员、加拿大阿尔伯塔省卡尔加里代谢与遗传（MAGIC）诊所的儿科医生和医学遗传学家阿内尔·汗（Aneal Khan）医学博士说：“庞贝病的酶替代疗法的局限性突显了对具有新作用机制的新疗法的需求。”。

“ABX1100 is being evaluated as a new therapeutic approach for individuals with late-onset Pompe disease, and we are eager to begin this trial in the underserved Pompe disease population.”.

“ABX1100正在被评估为晚发性庞贝病患者的一种新治疗方法，我们渴望在服务不足的庞贝病人群中开始这项试验。”。

Pompe disease is a rare neuromuscular disorder driven by the toxic buildup of glycogen, a stored form of sugar used for energy, in the muscle. This over-accumulation of glycogen leads to progressive loss of muscle function, weakness, and disability that can eventually progress to death from respiratory failure.1 Currently available enzyme replacement therapies (ERTs) for Pompe disease have limited long-term efficacy.

庞贝氏症是一种罕见的神经肌肉疾病，由肌肉中糖原（一种储存能量的糖）的毒性累积引起。糖原的过度积累导致肌肉功能逐渐丧失，无力和残疾，最终可能因呼吸衰竭而死亡。目前可用的庞贝病酶替代疗法（ERT）的长期疗效有限。

Preclinical studies suggest that inhibiting the production of GYS1 may be a valuable addition or alternative to ERT..

临床前研究表明，抑制GYS1的产生可能是ERT的有价值的补充或替代品。。

“The encouraging preclinical and clinical evidence generated to date provides a solid foundation for ongoing clinical studies of ABX1100, which has the potential to be the first new treatment for Pompe disease beyond ERT. We anticipate dosing the first patient with Pompe disease in the Phase 1b study before the end of 2024,” noted Susan Dillon, Ph.D., co-founder, president and chief executive officer of Aro..

“迄今为止产生的令人鼓舞的临床前和临床证据为正在进行的ABX1100临床研究提供了坚实的基础，该研究有可能成为ERT以外的首例庞贝病新疗法。我们预计在2024年底之前，在1b期研究中为第一例庞贝病患者服用药物，”Aro联合创始人、总裁兼首席执行官苏珊·狄龙博士指出。。

“ABX1100 was well tolerated in a recently completed Phase 1a trial conducted in normal healthy volunteers, and it showed durable GYS1 mRNA knockdown in muscle biopsies, with effects lasting through at least 10 weeks following a single dose,” added Dr. Dillon. “The study achieved the goal of identifying a safe and effective starting dose for studies in patients with LOPD.

Dillon博士补充道：“在最近完成的一项针对正常健康志愿者的1a期试验中，ABX1100耐受性良好，在肌肉活检中显示出持久的GYS1 mRNA敲低，单次给药后至少持续10周。”。“该研究实现了为LOPD患者的研究确定安全有效的起始剂量的目标。

We plan to present the Phase 1a results at a future medical conference.”.

我们计划在未来的医学会议上介绍1a期的结果。”。

About ABX1100

关于ABX1100

ABX1100, an investigational treatment for Pompe disease, is comprised of a CD71 receptor-binding Centyrin conjugated to a small interfering RNA (siRNA) that specifically interferes with expression of GYS1 messenger RNA (mRNA), thereby reducing levels and overall activity of the GYS1 enzyme in muscle tissues.

ABX1100是庞贝氏病的一种研究性治疗方法，由CD71受体结合的Centyrin组成，该Centyrin与小干扰RNA（siRNA）结合，特异性干扰GYS1信使RNA（mRNA）的表达，从而降低肌肉组织中GYS1酶的水平和总体活性。

ABX1100 demonstrated a favorable safety profile in toxicology studies, with durable reductions in GYS1 mRNA in muscles, supporting the potential for quarterly dosing. ABX1100 has received Orphan Drug Designation and Rare Pediatric Disease status from the United States Food and Drug Administration (FDA)..

ABX1100在毒理学研究中表现出良好的安全性，肌肉中GYS1 mRNA的持久减少，支持了每季度给药的潜力。ABX1100已获得美国食品和药物管理局（FDA）的孤儿药指定和罕见儿科疾病状态。。

About Aro Biotherapeutics

关于Aro生物治疗学

Aro Biotherapeutics is a biotechnology company working to develop potent and versatile tissue-targeted genetic medicines with a platform based on a proprietary protein technology called Centyrins. The company is developing a wholly owned pipeline of Centyrin-based therapeutic candidates for tissue-specific targeting of therapeutics for a diverse set of diseases.

。该公司正在开发一条全资拥有的基于Centyrin的候选治疗药物管道，用于针对多种疾病的组织特异性靶向治疗。

For more information, visit www.arobiotx.com..

有关更多信息，请访问www.arobiotx.com。。

Reference

参考

Pompe disease. U.S. Department of Health and Human Services, National Institutes of Health, National Institute of Neurological Disorders and Stroke (NINDS); 2024. https://www.ninds.nih.gov/health-information/disorders/pompe-disease#:~:text=What%20is%20Pompe%20disease%3F,the%20heart%20and%20skeletal%20muscles.

庞贝病。U、 美国卫生与公众服务部，国立卫生研究院，国家神经疾病和中风研究所（NINDS）；2024https://www.ninds.nih.gov/health-information/disorders/pompe-disease#:~：text=什么是庞贝病，心脏病和骨骼肌。

Accessed October 15, 2024..

2024年10月15日访问。。