Safety and Efficacy of Intravitreal Risuteganib for Non-Exudative AMD: A Multicenter, Phase 2a, Randomized, Clinical Trial
- Creators
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Boyer, David S.
- Gonzalez, Victor H.
- Kunimoto, Derek Y.
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Maturi, Raj K.
- Roe, Richard H.
- Singer, Michael A.
- Xavier, Samantha
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Kornfield, Julie A.
- Kuppermann, Baruch D.
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Quiroz-Mercado, Hugo
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Aubel, Janine
- Karageozian, Hampar L.
- Park, John Y.
- Karageozian, Vicken H.
- Karageozian, Lisa
- Sarayba, Melvin A.
- Kaiser, Peter K.
Abstract
Background and Objective: To evaluate the safety and efficacy of 1.0 mg risuteganib in subjects with nonexudative age-related macular degeneration (AMD). Patients and Methods: This was a phase 2a, prospective, double-masked, sham-controlled study. Eyes with nonexudative (dry) AMD and Early Treatment Diabetic Retinopathy Study (ETDRS) best-corrected visual acuity (BCVA) between 20/40 and 20/200 were included. Subjects were randomized to intravitreal 1.0 mg risuteganib or sham injection. At Week 16, subjects in the risuteganib group received a second 1.0-mg dose and the sham group crossed over to receive a dose of 1.0 mg risuteganib and were evaluated at Week 28. The primary endpoint was proportion of subjects with 8 letters ETDRS or more BCVA gain from baseline to Week 28 in the risuteganib group versus baseline to Week 12 for the sham group. BCVA was tested and subjects were observed for adverse events (AEs) every 4 weeks until completion of the study at 32 weeks. Results: Forty-five subjects (risuteganib, n = 29; sham, n = 16) were enrolled in the study, of whom 39 (risuteganib, n = 25; sham, n = 14) completed the study and were included in the per protocol efficacy analysis. At baseline, mean age was 78.8 and 75.9 years and mean BCVA was 67.1 and 64.4 letters in the sham and risuteganib groups, respectively. The primary endpoint was met by 48% of the risuteganib group at Week 28 and 7% of the sham group at Week 12 (P = .013). Of the risuteganib subjects, 20% gained 15 letters or more at Week 28, whereas no patients in the sham group at Week 12 achieved this visual acuity gain. The only ocular treatment-related treatment-emergent AE was vitreous floaters, which spontaneously recovered without sequelae. No drug-related serious AE was reported. Conclusions: Risuteganib demonstrated significant BCVA improvement in patients with non-exudative AMD. No drug-related AEs were seen during a 32-week observation period.
Additional Information
© 2021 Boyer, Gonzalez, Kunimoto et al. This is an Open Access article distributed under the terms of the Creative Commons Attribution-NonCommercial 4.0 International ( https://creativecommons.org/licenses/by-nc/4.0). This license allows users to copy and distribute, to remix, transform, and build upon the article non-commercially, provided the author is attributed and the new work is non-commercial. Originally submitted December 17, 2020. Revision received February 18, 2021. Accepted for publication March 23, 2021. Dr. Boyer reports grants and personal fees from Allergan, Aerpio, Allegro, Allergan, Boehringer Ingelheim, Clearside, Ionis, Genentech, Graybug, Novartis, Iveric, Regeneron, and Stealth; personal fees from Alcon, Acucela, Bausch + Lomb, BioMotiv, Glaukos, Kala, and Thea; and grants from Bayer and Santen outside the submitted work. Dr. Gonzalez is a consultant for and has received research support from Genentech, Regeneron, Oxurion (a Thrombogenics Inc. subsidiary), Alcon/Novartis, Allergan, Valeant, Santen Pharmaceutical, and Astellas Institute for Regenerative Medicine; is a consultant for, has received research support from, and has personal financial interests in Alimera; is a consultant for Bausch + Lomb, Topcon, Beaver-Visitec International Inc., and Abbvie; has personal financial interest in Panoptica; and has received financial support from Iconic Therapeutics, Allegro Ophthalmics, DRCRNet, Boehringer Ingelheim, Insite Vision Inc., Graybug Vision, Inc., Chengdu Kanghong Biotechnology Co, Ltd, Mallinckrodt ARD Inc., Opthea Ltd., 60° Pharmaceuticals, Apellis Pharmaceuticals, and Ribomic USA Inc. outside the submitted work. Dr. Kunimoto is a consultant for and has received research grants from Allegro, Allergan, and Genentech; is a consultant for Bausch + Lomb, D.O.R.C., Novartis, and Regeneron; and has received research grants from Roche outside the submitted work. Dr. Maturi has received research grants from Aerpio, Allegro, Allergan, Boehringer Ingelheim, Genentech, Graybug, Inc., Gyroscope, Kalvista, Samsung Bioepis, Santen, and Oxurion outside the submitted work. Dr. Singer is a consultant for and has received research grants from Aerie, Allegro, Kodiak, and Santen; is a consultant/speaker for and has received research grants from Allergan, Genentech, Novartis, and Regeneron; is a consultant for Eyepoint; is a speaker for Mallinckrodt and Spark; and has received research funding from DRCRNet, Icon, Ionis, Kalvista, Ophthea, Optos, Senju, and Sydnexis outside the submitted work. Dr. Xavier has received grants from Allegro Ophthalmics, LLC outside the submitted work. Dr. Kornfield received grants and non-financial support from Allegro Ophthalmics, LLC, during the conduct of this study, as well as profits interest units (PIU) in Allegro Ophthalmics outside the submited work. Dr. Kupperman has received clinical research funding from Alcon, Apellis, Clearside, GSK, and Ionis; is a consultant for and has received clinical research funding from Allergan, Genentech, IVERIC Bio, jCYTE, Novartis, and Regeneron; and is a consultant for Allegro, Aprea, Cell Care, Dose, Eyedaptic, Galimedix, Glaucos, Interface Biologics, Oculis, Revana, Ripple Therapeutics, and Theravance Biopharma outside the submitted work. Dr. Quiroz-Mercado has stock in Allegro Ophthalmics, LLC. Ms. Aubel, Dr. H Karageozian, Dr. Park, Dr. V Karageozian, Ms. Karageozian, Dr. Sarayba, and Dr. Kaiser are employees of and have stock in Allegro Ophthalmics, LLC. Dr. Roe reports no relevant financial disclosures.Attached Files
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Additional details
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- Resolver ID
- CaltechAUTHORS:20210823-142252660
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2021-08-24Created from EPrint's datestamp field
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2021-08-24Created from EPrint's last_modified field