RIBOMIC Announces Preliminary Topline Data From Phase 2 Trials of RBM-007 for Wet Age-Related Macular Degeneration

TOKYO–(BUSINESS WIRE)–RIBOMIC, Inc., a clinical stage pharmaceutical company specializing in aptamer therapeutics (TOKYO:4591), today announced the topline data from the Phase 2 TOFU study of RBM-007 in patients with Wet Age-Related Macular Degeneration (wAMD).

TOFU study is a double-masked, randomized, active-controlled Phase 2 trial (n=86) evaluating the efficacy and safety of RBM-007 monotherapy and RBM-007 in combination with Eylea^� compared to Eylea^® monotherapy in patients with wAMD who are previously treated with Standard of Care (anti-VEGF drugs).

As a result of the analysis, RBM-007 monotherapy or RBM-007 in combination with Eylea^® did not demonstrate vision improvement over Eylea^® monotherapy in this patient population.

On the other hand, in treatment naïve wAMD patients, preliminary interim data from the ongoing phase 2 TEMPURA investigator sponsored trial evaluating the safety and efficacy of RBM-007 monotherapy are showing improvement in vision and retinal anatomy.

Additional analyses of secondary endpoints of TOFU are ongoing. The company is planning to share the detailed results together with those from its RAMEN extension study and TEMPURA study as full dataset.

About RBM-007

RBM-007 is a novel oligonucleotide-based aptamer with potent anti-FGF2 (fibroblast growth factor 2) activity. FGF2 is implicated in not only angiogenesis but also fibrosis in several diseases including wAMD. The dual action of RBM-007 (anti-angiogenic and anti-scarring) holds promise as an additive or alternative therapy to anti-VEGF treatments for wAMD. Three studies are currently ongoing for wAMD: 1. Active-controlled, double masked trial, TOFU study (NCT04200248); 2. Single-arm, open-label extension trial, RAMEN (NCT04640272); and 3. Investigator sponsored trial with treatment naïve wAMD patients, the TEMPURA study (NCT04895293).

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 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:

RIBOMIC Inc.

Administration Division

Shoji Yonebayashi

ir.inquiry@ribomic.com