Interim results from a phase 2 trial presented at the Euretina congress show Sanofi's SAR402663 intravitreal gene therapy reduced treatment burden by 97% at a medium dose in wet age-related macular degeneration patients. The single in-office AAV2 injection secretes sFLT01 to inhibit vessel growth, enabling over 90% of medium-dose patients to eliminate supplemental anti-VEGF injections. Supported by a 19-week corticosteroid prophylaxis, both evaluated doses maintained a favorable safety profile.
Efficacy and treatment burden reduction
- ▪Interim phase 2 trial results presented at the Euretina congress in Vienna showed Sanofi's SAR402663 gene therapy demonstrated sustained visual function and retinal anatomic control in wet age-related macular degeneration patients.
- ▪In the phase 2 trial, more than 90% of wet age-related macular degeneration patients receiving the medium dose of Sanofi's SAR402663 required zero supplemental anti-VEGF injections.
- ▪At a medium dose level, Sanofi's SAR402663 gene therapy achieved a 97% reduction in treatment burden for wet age-related macular degeneration patients in phase 2 interim trial results.
Phase 2 study design and patient profile
- ▪In the phase 2 trial for Sanofi's SAR402663 gene therapy, enrolled patients had visual acuity ranging from 20/25 to 20/200 and a documented prior anti-VEGF treatment response.
- ▪The phase 2 study of Sanofi's SAR402663 evaluated the safety and efficacy of low and medium doses in 21 and 22 previously treated neovascular age-related macular degeneration patients, respectively.
Safety profile and inflammation management
- ▪Phase 2 interim results for Sanofi's SAR402663 gene therapy showed both evaluated doses were well tolerated with no reported cases of endophthalmitis, vasculitis, hypotony, or choroidal effusions.
- ▪To mitigate immune-mediated safety risks associated with gene therapies, the SAR402663 trial protocol incorporated a 19-week prophylactic regimen of corticosteroids.
- ▪Pooled phase 1 and phase 2 data for Sanofi's SAR402663 showed few cases of intraocular inflammation, all of which were manageable with corticosteroids, including one event that resolved spontaneously.
Phase 1 trial burden reductions and safety
- ▪During Sanofi's early DFI17940 phase 1 program, a high dose level 3 of SAR402663 was evaluated but excluded from phase 2 testing due to high rates of intraocular inflammation.
- ▪Phase 1 trial data for Sanofi's SAR402663 showed dose-dependent treatment burden reductions of 63% for dose level 1, 83% for dose level 2, and 92% for dose level 3.
Mechanism of action
- ▪Sanofi's investigational SAR402663 is an adeno-associated virus 2 (AAV2) gene therapy administered via a single in-office intravitreal injection to provide sustained anti-VEGF activity.
- ▪Sanofi's SAR402663 gene therapy secretes sFLT01, a VEGF-neutralizing protein engineered to inhibit abnormal blood vessel growth in wet age-related macular degeneration.
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