TOKYO--(BUSINESS WIRE)--RIBOMIC Inc. (TOKYO:4591), a clinical-stage pharmaceutical company specializing in aptamer therapeutics, today announced that 13 patients have been enrolled in an observational study 1 of umedaptanib pegol (anti-FGF2 aptamer) in pediatric patients (5-14 years old) with Achondroplasia in an early phase II study..

东京--（商业新闻短讯）--RIBOMIC Inc.（东京：4591），一家专门从事适体治疗的临床阶段制药公司，今天宣布，在一项早期II期研究中，有13名患者参加了umedaptanib pegol（抗FGF2适体）对患有软骨发育不全的儿科患者（5-14岁）的观察性研究1。。

A low-dose subcutaneous study (Cohort1 2) to evaluate the efficacy and safety of umedaptanib pegol in six patients who have completed the observational study is ongoing and is expected to be completed in November of this year.

一项低剂量皮下研究（Cohort1 2）正在进行中，以评估umedatanib pegol对6名完成观察性研究的患者的疗效和安全性，预计将于今年11月完成。

These patients will continue in the extension study and three have already completed Cohort 1 and entered the extension study.

这些患者将继续进行扩展研究，其中三名已经完成队列1并进入扩展研究。

RIBOMIC also announced that the first patient in a high-dose subcutaneous study (Cohort 2 3) of umedaptanib pegol has been dosed in patients who have completed the observational study has been administered. No safety concerns have been identified in the ongoing early phase II clinical study and extension study, including this case..

RIBOMIC还宣布，umedaptanib pegol高剂量皮下研究（队列2-3）的第一名患者已经在完成观察性研究的患者中服用。正在进行的早期II期临床研究和扩展研究（包括本例）未发现安全问题。。

2

2

After confirming the safety and tolerability of subcutaneous administration at a dose of 0.3 mg/kg once every 2 weeks for 8 weeks (total of 4 doses), the dosing interval is changed to once weekly for 26 weeks (total dosing period: 34 weeks).

在确认0.3 mg/kg剂量皮下给药的安全性和耐受性后，每2周一次，持续8周（共4次），给药间隔改为每周一次，持续26周（总给药期：34周）。

ABOUT umedaptanib pegol

关于umedaptanib pegol

umedaptanib pegol is a novel oligonucleotide-based aptamer formerly designated RBM-007, with potent anti-FGF2 (fibroblast growth factor 2) activity and is expected to be a fundamental treatment that directly targets the pathogenic mechanism of Achondroplasia.

umedaptanib pegol是一种新型的基于寡核苷酸的适体，以前称为RBM-007，具有强大的抗FGF2（成纤维细胞生长因子2）活性，有望成为直接针对软骨发育不全致病机制的基本治疗方法。

The drug has demonstrated clinical POC in exudative age-related macular degeneration.

该药物已证明渗出性年龄相关性黄斑变性的临床POC。

ABOUT Achondroplasia

关于软骨发育不全

Achondroplasia is disease in which a genetic mutation of the fibroblast growth factor receptor type 3 (FGFR3) causes FGFR3 to be activated, resulting in an excessive influx of FGF signals that inhibit the normal growth of cartilage and other tissues, causing short stature with limb shortening and other symptoms.

软骨发育不全是一种疾病，其中成纤维细胞生长因子受体3型（FGFR3）的基因突变导致FGFR3被激活，导致FGF信号过度流入，抑制软骨和其他组织的正常生长，导致身材矮小，肢体缩短和其他症状。

It is a rare disease with an incidence of 1 in 25,000 newborns and is considered intractable. The development of effective new drugs is required..

这是一种罕见疾病，每25000名新生儿中就有1名发病，被认为是难治性疾病。需要开发有效的新药。。

ABOUT RIBOMIC

关于RIBOMIC

RIBOMIC is a clinical-stage biopharmaceutical company specializing in the discovery and development of aptamer therapeutics, a type of nucleic acid medicine with great potential for the development of next-generation drugs. The RiboART system, the company’s core drug discovery platform, can be used to discovery many types of aptamer drugs.

。RiboART系统是该公司的核心药物发现平台，可用于发现多种适体药物。