RIBOMIC Announces First Injection in the Phase 2 Clinical Trial of RBM-007 (TOFU Study) in Subjects with Wet Age-Related Macular Degeneration

RIBOMIC, Inc., a clinical stage pharmaceutical company specializing in aptamer therapeutics and traded on the Mothers Market of the Tokyo Stock Exchange (Code Number: 4591), today announced that first patient has received injection in the phase 2 trial of RBM-007 for the treatment of exudative age-related macular degeneration (AMD) in the United States. The first site started enrollment at the end of December 2019 and five sites are now active across the United States.

About RBM-007 and development background

RBM-007 is a novel oligonucleotide-based aptamer with potent anti-FGF2 (fibroblast growth factor 2) activity. Currently approved therapies for wet AMD, intravitreal injections of anti-VEGF drugs, have shown dramatic visual benefits for wet AMD patients. However, a significant portion of wet AMD patients exhibit incomplete response to therapy, and over the extended management course can lose vision, with the formation of submacular fibrosis as one risk factor. RIBOMIC investigated a novel therapy for wet AMD targeting fibroblast growth factor 2 (FGF2), which is implicated in not only angiogenesis but also fibrosis in several diseases, and created RBM-007, a novel oligonucleotide-based aptamer with potent anti-FGF2 activity. RBM-007 is chemically synthesized, and pharmacokinetic studies of RBM-007 in the rabbit vitreous revealed high and relatively long-lasting profiles, which are superior to the other approved anti-VEGF drugs. The dual action of RBM-007 (anti-angiogenic and anti-scarring) holds promise as an additive or alternative therapy to anti-VEGF treatments for wet AMD.

About TOFU study

A Multi-Center, Randomized, Double Masked and Active Controlled Phase II Study Assessing the Efficacy and Safety of Intravitreal Injections of RBM-007 monotherapy and RBM-007 in Combination with Eylea^® Compared to Eylea^® Monotherapy in Subjects with Wet Age-related Macular Degeneration (TOFU Study) is Phase 2 Study assessing the safety, efficacy and durability of RBM-007.

Study Design	Multicenter, active-controlled, double masked study
Patient Population	Patients with wet AMD who are non or low responders to existing anti-VEGF drugs
Administration	Four monthly intravitreal injections of RBM-007. Eylea® dosed every other month as per label.
Primary Endpoints	Mean change in Best Corrected Visual Acuity from Baseline and safety
Study Arms	RBM-007 (monotherapy) RBM-007 and Eylea® (Anti-VEGF drug) (combination) Eylea® (Anti-VEGF drug) (monotherapy)
Number of Subject	81 (27 per arm)
Duration	5 months (primary endpoint at month 1 after last injection)
Location	10 or more sites across the United States

See ClinicalTrials.gov for more information.
https://clinicaltrials.gov/ct2/show/NCT04200248

About wet Age-related Macular Degeneration

Wet (exudative) age-related macular degeneration, is the leading cause of blindness in the United States and Europe. It is caused by the formation of abnormal and leaky new blood vessels under the retina, termed choroidal neovascularization. The leakage of fluid from the vessels causes retinal thickening and retinal degeneration including fibrotic scar formation, and leads to severe and rapid loss of vision.

ABOUT RIBOMIC

RIBOMIC is a bio-venture company centered on drug discovery. The company is engaged in the field 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, pain and many other problems.
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.

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Contact information

Contacts for inquiries or additional information:
RIBOMIC Inc.
Corporate Planning Department
Shoji Yonebayashi
ir.inquiry@ribomic.com