TOKYO--(BUSINESS WIRE)--RIBOMIC, Inc., a clinical stage pharmaceutical company specializing in aptamer therapeutics (TOKYO:4591), today announced the presentation of preliminary interim results from the ongoing two Phase 2 studies, TOFU and the RAMEN extension study in patients with Wet Age-Related Macular Degeneration (wAMD) by Dr. Raj Maturi at the 54th Annual Scientific Meeting of the Retina Society, Chicago, USA.
TOFU study is a double-masked, randomized, active-controlled Phase 2 trial evaluating the efficacy and safety of RBM-007 in patients with wAMD who are previously treated and sub-optimal responders to Standard of Care (anti-VEGF drugs). RAMEN study is a single-arm, open-label extension trial. The primary endpoint for both studies is mean change in Best Corrected Visual Acuity (BCVA) from Baseline.
The TOFU study remains masked and all of the interim data from the trial are on a masked basis (RBM-007 arm, RBM-007+Eylea® arm, and Eylea® arm combined). In the TOFU study, a total of 86 subjects were enrolled and as of cut-off date of August 31, approximately 50 subjects completed Week16 (Primary Endpoint) and 44 completed the entire study. The remainder of the subjects are at different time-points in the trial depending on their start date. The masked preliminary interim safety data from the three arms combined, showed that the AEs reported were consistent with the Standard of Care, with no retinal vasculitis or occlusive events, and as for the preliminary BCVA outcome in subjects who completed week 16, approximately 70% had improved or stable vision.
In the on-going open-label RAMEN study, monthly injections of RBM-007 showed good safety and tolerability with no unexpected AE as of the cut-off date.
The Last Patient Last Visit in both studies is expected in December 2021 and topline results are planned to be reported in Q1 2022.
“We are pleased that the abstract was accepted for a podium presentation and excited to see Dr. Maturi present the masked interim data at the prestigious Retina Society,” commented Padma Bezwada, PhD., CEO of RIBOMIC USA Inc. “We are encouraged by the preliminary data, especially considering the study population as incomplete response to anti-VEGF therapy represents a significant clinical unmet need in the management of wAMD. While the data increases our confidence in RBM-007, we must caution that the TOFU data are masked and we do not know the breakdown between the three treatment arms. The study and data will continue to remain masked until we complete the study and reach database lock following which we will unmask, analyze the full dataset and share the topline in Q1 2022”
The title and date/time of the presentation:
Title: Intravitreal RBM-007, a novel anti-angiogenic and anti-scarring agent, in recalcitrant exudative AMD patients: interim safety update on the Phase 2 TOFU and the RAMEN extension study
Presenter: Raj Maturi, MD
Date/Time: Thursday, September 30, 2021 from 9:46 am CT
The slide presentation will become available on RIBOMIC’s website https://www.ribomic.com/eng/ir.php on October 1, 2021, JST.
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). The TOFU and RAMEN studies are active but closed to recruitment.
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.
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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