September 2016
Volume 57, Issue 12
Open Access
ARVO Annual Meeting Abstract  |   September 2016
A combined phase 2a/b study of the efficacy, safety, and tolerability of repeated topical doses of regorafenib eye drops in treatment-naïve patients with neovascular age-related macular degeneration (nAMD)
Author Affiliations & Notes
  • Antonia M Joussen
    Charite, University Medicine Berlin, Berlin, Germany
  • Sebastian Wolf
    Inselspital, Bern, Switzerland
  • Peter K Kaiser
    Cole Eye Institute, Cleveland, Ohio, United States
  • David S Boyer
    Retina-Vitreous Associates Medical Group, Beverly Hills, California, United States
  • Thomas Schmelter
    Bayer Pharma AG, Berlin, Germany
  • Rupert Sandbrink
    Bayer Pharma AG, Berlin, Germany
    Heinrich-Heine-Universität, Dusseldorf, Germany
  • Oliver Zeitz
    Bayer Pharma AG, Berlin, Germany
  • Michael K Boettger
    Bayer Pharma AG, Berlin, Germany
  • Brigitte Stemper
    Bayer Pharma AG, Berlin, Germany
  • Footnotes
    Commercial Relationships   Antonia Joussen, Allergan (C), Bayer (C), Novartis (C); Sebastian Wolf, Alcon (C), Allergan (C), Bayer (C), Heidelberg Engineering (S), Novartis (C), Optos (S), Zeiss (S); Peter Kaiser, Alcon (C), Bayer (C), Genentech (C), Kanghong (C), Neurotech (C), Novartis (C), Ohr (C), Ophthotech (C), Regeneron (C); David Boyer, Alcon (C), Allergan (C), Bayer (C), Genentech (C), Neurotech (C), Novartis (C), Ohr (C), Regeneron (C), Roche (C); Thomas Schmelter, Bayer (E); Rupert Sandbrink, Bayer (E); Oliver Zeitz, Bayer (E); Michael Boettger, Bayer (E); Brigitte Stemper, Bayer (E)
  • Footnotes
    Support  None
Investigative Ophthalmology & Visual Science September 2016, Vol.57, No Pagination Specified. doi:
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      Antonia M Joussen, Sebastian Wolf, Peter K Kaiser, David S Boyer, Thomas Schmelter, Rupert Sandbrink, Oliver Zeitz, Michael K Boettger, Brigitte Stemper; A combined phase 2a/b study of the efficacy, safety, and tolerability of repeated topical doses of regorafenib eye drops in treatment-naïve patients with neovascular age-related macular degeneration (nAMD). Invest. Ophthalmol. Vis. Sci. 201657(12):.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

Purpose : To study the efficacy, safety, and tolerability of regorafenib eye drops (REG) in treatment-naïve patients with nAMD. REG is a multi-kinase inhibitor that acts mainly on the intracellular signalling of vascular endothelial growth factor receptors, which are known to play a primary role in the development and progression of nAMD.

Methods : DREAM was a multicenter, single-arm, open-label, Phase 2a/b study in which patients with choroidal neovascularization (CNV) secondary to nAMD received 1 drop (0.75 mg, 25 µL) of topical REG 3 times daily for 12 weeks in the study eye. The primary endpoint was the mean change in best-corrected visual acuity ETDRS letter score (BCVA) from baseline to Weeks 4 and 12. Anatomic and safety outcomes were also measured at Weeks 4 and 12.

Results : A total of 51 nAMD patients received treatment (mean age: 75 years; mean baseline BCVA: 59 letters; mean baseline central retinal thickness [CRT]: 463.7 µm; mean CNV lesion size: 7.1 mm2; 84.3% had occult CNV and 15.7% had predominantly classic CNV). The mean changes in BCVA were +1.2 letters and –2.4 letters at Weeks 4 and 12, respectively. Patients with occult CNV lost fewer letters than those with classic CNV. The mean changes in CRT were +26 µm and +47 µm at Weeks 4 and 12. Ocular treatment-emergent adverse events (TEAEs) were reported in the study eye of 21 patients (41.2%) by Week 12; the majority were mild to moderate in severity (95.2%). There were 1 serious ocular TEAE (visual acuity reduced) and 2 serious nonocular TEAEs (ankle fracture and acute myocardial infarction resulting in 1 death), but none were drug related. A total of 21 patients required rescue medication (intravitreal ranibizumab).

Conclusions : DREAM was a Phase 2a/b study investigating regorafenib eye drops given 3 times daily for 12 weeks in patients with treatment-naïve nAMD. The study was terminated after completion of Phase 2a because efficacy was not sufficient and did not approach historic levels seen with other nAMD treatments. There were no drug-related safety concerns.

This is an abstract that was submitted for the 2016 ARVO Annual Meeting, held in Seattle, Wash., May 1-5, 2016.

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