RIBOMIC Phase I and II Data Published in the Eye: Full TOFU/RAMEN/TEMPURA Trial Results Demonstrate Clinical Proof of Concept of Umedaptanib Pegol in Exudative Age-Related Macular Degeneration (nAMD)

Phase 1Clinical ResultPhase 2
TOKYO--(BUSINESS WIRE)--RIBOMIC Inc., a clinical stage pharmaceutical company specializing in aptamer therapeutics (TOKYO:4591), today announced that the Eye has published full results from the phase 1 (SUSHI) and phase 2 (TOFU/RAMEN/TEMPURA) trials evaluating the efficacy and safety of intravitreal umedaptanib pegol in nAMD1,2. TOFU is a randomized, double-masked study assessing the efficacy of intravitreal umedaptanib pegol monotherapy or in combination with Eylea®, compared to Eylea® monotherapy in 86 subjects with anti-VEGF pretreated nAMD. RAMEN is an extension study of TOFU, in which 22 subjects who had exited the TOFU study received 4 monthly intravitreal injections of umedaptanib pegol. TEMPURA is an investigator-sponsored, single-center, open-label, 4-month study of umedaptanib pegol in five treatment-naïve nAMD patients.
Throughout these studies umedaptanib pegol was safe, well tolerated, and effective to treat nAMD patients with no or short history of anti-VEGF treatment. Striking improvements in visual acuity and anatomy were observed in some of the treatment naïve patients. In nAMD patients with a long history of anti-VEGF treatment, umedaptanib pegol showed no additional benefit of monotherapy or the combination over Eylea®. Nevertheless, the pre-existing fibrosis remained stable without worsening in these studies. Notably, visual acuity decreased slightly in patients who switched from anti-VEGF (Eylea®) to umedaptanib pegol in the RAMEN study. Therefore, we believe that umedaptanib pegol is an excellent alternative as a first-line medication before patients are exposed to anti-VEGF treatments to mitigate risk of damage caused by fibrosis. Further studies are needed on the efficacy of umedaptanib pegol early in the onset of nAMD as well as in coordination or combination with anti-VEGF drugs.
“We are very pleased that The Royal College of Ophthalmologists journal (Eye) recognized the trial results as a viable Proof-of-Concept for umedaptanib pegol in nAMD therapy. This first clinical evidence is an exciting breakthrough which raises hope that a non-VEGF target can lead to successful monotherapy in nAMD and provide a first-line alternative to treatment naïve nAMD patients.” commented Yoshi Nakamura, Ph.D., the CEO of RIBOMIC Inc.
Pereira, D.S., Akita, K., Bhisitkul, R.B., Nishihata, T., Ali, Y., Nakamura, E., Nakamura, Y.: Safety and tolerability of intravitreal umedaptanib pegol (anti-FGF2) for neovascular age-related macular degeneration (nAMD): a phase 1, open label study. Eye, published online: 01 December 2023. URL: https://www.nature.com/articles/s41433-023-02849-6.pdf
Pereira, D.S., Maturi, R.K., Akita, K., Bhisitkul, R.B., Nishihata, T., Sakota, E., Ali, Y., Nakamura, E., Bezwada, P., Nakamura, Y.: Clinical proof of concept for anti-FGF2 therapy in exudative age-related macular degeneration (nAMD): phase 2 trials in treatment-naïve and anti-VEGF pretreated patients. Eye, published online: 30 November 2023. URL: https://www.nature.com/articles/s41433-023-02848-7.pdf
Umedaptanib pegol is a novel oligonucleotide-based aptamer formerly called RBM-007 with potent anti-FGF2 (fibroblast growth factor 2) activity. FGF2 is implicated in not only angiogenesis but also fibrosis in several diseases including nAMD. The dual action of umedaptanib pegol (anti-angiogenic and anti-scarring) holds promise as an additive or alternative therapy to anti-VEGF treatments for nAMD. The three completed Phase 2 studies in nAMD are: 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 nAMD patients, the TEMPURA study (NCT04895293).
Exudative age-related macular degeneration (nAMD), 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.
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.
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