RIBOMIC Announces Completion of IND Submission for an Observational Study for Continuous Phase 2 Trial of RBM-007 for Treatment of Achondroplasia

TOKYO–(BUSINESS WIRE)–RIBOMIC, Inc., a clinical stage pharmaceutical company specializing in aptamer therapeutics (TOKYO:4591), today announced that it submitted an Investigational New Drug Application (IND) for an observational study to the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan. The purpose of the observational study is to obtain clinical basic data, including height growth, and to select subjects for the early phase II study.

If there is no comment from PMDA for 14 days after submission, the observational study will be initiated.

Overview of the study

Subjects	Patients with achondroplasia
Target number of subjects	14
Purpose of the study	The purpose of this study is to obtain clinical basic data including height growth in children (aged 5-14 years) with achondroplasia, which will enable the evaluation of efficacy and safety in the continuous early Phase II study using RBM-007 as a comparative data, and to select subjects for the early phase II study appropriately.
Study design	Observational study (no therapeutic intervention)
Observation period	26 weeks or more, up to 2 years

About RBM-007

RBM-007 is a novel nucleic acid medicine (oligonucleotide-based aptamer) developed in-house at RIBOMIC�s research facilities in Tokyo. RBM-007 has been shown to have potent effects in limiting excessive interactions between fibroblast growth factors, which are known to cause achondroplasia.

About Achondroplasia

Achondroplasia is a rare disease characterized by short stature (adult height of approximately 130 cm for males and approximately 125 cm for females) with short limbs. Achondroplasia has no known cure, and is designated as an intractable disease by the Ministry of Health, Labour and Welfare in Japan. This disease results mainly from a genetic defect in FGFR3 (fibroblast growth factor type 3 receptor). This genetic change causes the receptor to be overly active to growth factors such as FGF2, which leads to reduced growth of chondrocytes, resulting in a short stature. Achondroplasia occurs in a frequency of 1 in approximately 25,000 normal live births and is estimated to affect approximately 250,000 people worldwide.

By inhibiting the binding of FGF2 to FGFR3, RBM-007 has demonstrated therapeutic effects in studies using animal models of achondroplasia and patient-derived iPS (induced pluripotent stem) cells.

ABOUT RIBOMIC

RIBOMIC is a clinical stage bio-pharmaceutical company specializing in the discovery and development of aptamer therapeutics, which is one type of nucleic acid medicine, a field with much potential for the development of next-generation drugs. The RiboART system, the company’s core drug discovery platform, can be used for the discovery of many types of aptamer drugs. RIBOMIC is dedicated to the discovery and development of drugs that target the broad field of unmet medical needs, which encompasses eye disorders, rare disease of short stature in children and many other diseases.

See RIBOMIC website for more information.

https://www.ribomic.com/eng/

Forward-Looking Statements

This announcement contains forward-looking statements relating to current plans, estimates, strategies, belief and the future performance of Company. These statements are based on Company’s current expectations in light of the information and assumptions currently available so that Company does not promise the realization and these expectations may differ materially from those discussed in the forward-looking statements. These factors include, but not limited to, i) changes in general economic conditions and in laws and regulations, relating to pharmaceutical markets, ii) currency exchange rate fluctuations, iii) claims and concerns on the product safety and efficacy, iv) completion and discontinuation of clinical trials, v) infringement of Company’s intellectual property rights by third parties.?

Information on pharmaceutical products (including products currently in development), which is included in this press release is not intended to constitute an advertisement or medical advice.

“RIBOMIC,” “RiboART system” and the RIBOMIC logo are registered trademarks or trademarks of RIBOMIC Inc. in various jurisdictions.

Contacts

Contacts for inquiries or additional information:

Shoji Yonebayashi

RIBOMIC Inc.

Finance and Accounting

ir.inquiry@ribomic.com