WATERTOWN, Mass.--(BUSINESS WIRE)--Nido Biosciences (Nido Bio), a clinical stage company developing precision medicines for debilitating neurological diseases, today announced positive results from its Phase 1 study of NIDO-361 in healthy male subjects and interim results from its observational study at the 2023 International Kennedy’s Disease Patient Conference in London, November 4 – 5.

马萨诸塞州沃特敦-（商业线）-Nido Biosciences（Nido Bio）是一家开发用于使人衰弱的神经系统疾病的精确药物的临床阶段公司，今天宣布其在健康男性受试者中对Nido-361进行的1期研究取得了积极成果，其于11月4日至5日在伦敦举行的2023年国际肯尼迪病患者会议上的观察性研究的中期结果。

The company believes data from both studies support the advancement of the clinical evaluation of NIDO-361 in patients..

该公司认为这两项研究的数据都支持NIDO-361在患者中的临床评估进展。。

NIDO-361 is in development for the treatment of patients suffering from Spinal and Bulbar Muscular Atrophy (SBMA), also known as Kennedy’s disease. SBMA is caused by an X-linked genetic mutation on the androgen receptor that results in the progressive loss of skeletal muscle and motor neuron function.

NIDO-361正在开发中，用于治疗患有脊髓和延髓性肌萎缩症（SBMA）的患者，也称为肯尼迪病。SBMA是由雄激素受体上的X连锁遗传突变引起的，该突变导致骨骼肌和运动神经元功能的逐步丧失。

The novel small molecule binds to a distinct site on the androgen receptor and corrects transcriptional dysregulation to restore healthy cell function..

新型小分子与雄激素受体上的不同位点结合，并纠正转录失调以恢复健康的细胞功能。。

The Phase 1 study of NIDO-361 was a randomized double-blind placebo-controlled trial in healthy male subjects. The study was designed to evaluate the safety, tolerability, and pharmacokinetics (PK) of NIDO-361 delivered orally as a single dose in dose-escalated cohorts followed by multiple doses in dose-escalated cohorts.

NIDO-361的1期研究是健康男性受试者的随机双盲安慰剂对照试验。该研究旨在评估NIDO-361在剂量递增队列中作为单剂量口服递送的安全性，耐受性和药代动力学（PK），随后在剂量递增队列中以多剂量递送。

Sixty-four subjects were enrolled and received study drug, with eight enrolled in each cohort at a ratio of 3:1, active: placebo. The positive results presented at the conference demonstrate that NIDO-361 was generally safe and well-tolerated when administered as a single or as multiple doses, with no serious Treatment Emergent Adverse Events (TEAE)..

64名受试者参加并接受研究药物，每个队列中有8名以3:1的比例参加，活性：安慰剂。会议上提出的积极结果表明，NIDO-361单剂量或多剂量给药时通常安全且耐受性良好，没有严重的治疗引起的不良事件（TEAE）。。

Additionally, Nido Bio has announced the completion of interim analyses in patients with SBMA enrolled in an ongoing observational study. The two-year observational study being conducted at the National Institute of Neurological Disorders and Stroke (NINDS)/National Institute of Health (NIH), the University College London (UCL), and the Department of Neurology, Nagoya University Graduate School of Medicine (Nagoya), was designed to assess the relationship between muscle MRI and clinical outcomes.

此外，Nido Bio宣布完成正在进行的观察性研究中SBMA患者的中期分析。这项为期两年的观察性研究正在国立神经疾病和中风研究所（NINDS）/国立卫生研究院（NIH），伦敦大学学院（UCL）和名古屋大学医学研究生院神经内科进行（名古屋），旨在评估肌肉MRI与临床结果之间的关系。

Interim analyses conducted to date with available data at 6 months and 12 months post-baseline generated results in support of the hypothesis that muscle MRI measures are associated with disease progression measures..

迄今为止进行的中期分析以及基线后6个月和12个月的可用数据产生的结果支持了肌肉MRI测量与疾病进展测量相关的假设。。

“The Phase 1 study results and interim analyses of our ongoing observational study support the further advancement of our clinical program by enabling the selection of active and safe doses and confirming muscle MRI as a reliable primary endpoint for our upcoming Phase 2 trial which we anticipate will begin enrollment in Q1 2024,” said Vissia Viglietta, M.D., Ph.D., CMO of Nido Bio.

“我们正在进行的观察性研究的第一阶段研究结果和中期分析支持了我们临床计划的进一步发展，因为我们能够选择有效和安全的剂量，并确认肌肉MRI是我们即将进行的第二阶段试验的可靠主要终点，我们预计这将在2024年第一季度开始报名，”Nido Bio博士，博士Vissia Viglietta说。

“We are excited by the potential of NIDO-361 to be a medicine that matters for SBMA patients as we continue our efforts to bring transformative therapies to patients suffering from debilitating neurological diseases.”.

“我们对NIDO-361作为一种对SBMA患者很重要的药物的潜力感到兴奋，因为我们继续努力为患有使人衰弱的神经系统疾病的患者带来变革性治疗。”。

Presentation Details

演示细节

Title: Nido-361 for the Treatment of SBMA: Updates on clinical development program

题目：Nido-361用于治疗SBMA：临床开发计划的更新

Presented by: Vissia Viglietta, M.D., Ph.D., Chief Medical Officer, Nido Biosciences

提交人：Vissia Viglietta，M.D.，Ph.D.，Nido Biosciences首席医疗官

Date: November 5, 2023

日期：2023年11月5日

Time: 2:00 p.m. GMT

时间：GMT下午2:00

About Spinal and Bulbar Muscular Atrophy (SBMA)

关于嵴髓性和延髓性肌肉萎缩症

SBMA, also known as Kennedy’s disease, is a rare inherited X-linked neuromuscular disorder caused by a genetic mutation of the androgen receptor (AR) that results in the loss of skeletal muscle and motor neuron function. Manifesting in men, SBMA causes progressive weakness and wasting of limb, facial and swallowing muscles, which results in impaired mobility, speech, and swallowing..

SBMA，也称为肯尼迪氏病，是一种罕见的遗传性X连锁神经肌肉疾病，由雄激素受体（AR）的基因突变引起，导致骨骼肌和运动神经元功能丧失。SBMA表现在男性中，会导致进行性无力和肢体，面部和吞咽肌肉的消瘦，从而导致活动能力，言语和吞咽能力受损。。

About Nido Biosciences

关于Nido生物科学

Nido Bio is translating today’s neuroscience breakthroughs into tomorrow’s treatments for severe neurological diseases. Leveraging human genetics, we develop precision medicines that address the fundamental biology of disease and restore healthy cell function. NIDO-361, our clinical-stage candidate, is a treatment for Spinal and Bulbar Muscular Atrophy which is a rare and debilitating neuromuscular disease.

Nido Bio正在将今天的神经科学突破转化为明天严重神经疾病的治疗方法。利用人类遗传学，我们开发精准药物，解决疾病的基本生物学和恢复健康的细胞功能。NIDO-361是我们的临床分期候选者，是治疗脊髓和延髓性肌萎缩的一种罕见且使人衰弱的神经肌肉疾病。

Additional pipeline programs center around a novel target with the potential to address multiple disease mechanisms and that has broad clinical application across neurodegenerative and peripheral inflammatory diseases. We are creating a sustainable pipeline for the company by utilizing a functional genomics discovery platform based on human cell lines to identify novel targets.

额外的管道计划围绕着一个新的目标，有可能解决多种疾病机制，并在神经退行性疾病和外周炎症性疾病中具有广泛的临床应用。我们正在利用基于人类细胞系的功能基因组学发现平台为公司创建可持续的管道，以确定新的目标。

Through our approach we seek to transform patient lives in meaningful ways. www.nidobio.com..

通过我们的方法，我们寻求以有意义的方式改变患者的生活。www.nidobio.com。。