One-year trial outcomes demonstrate efficacy of ABBV-RGX-314 gene therapy for DR
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ROME — One-year results of the phase 2 ALTITUDE trial showed efficacy and safety of a one-time in-office injection of ABBV-RGX-314 for the treatment of diabetic retinopathy.
“This investigational drug is an AAV8 vector with anti-VEGF Fab similar to ranibizumab,” Lejla Vajzovic, MD, said at the FLORetina-ICOOR meeting.
Injected into the suprachoroidal space, ABBV-RGX-314 reduces leaky blood vessel formation by giving ocular cells the ability to produce an anti-VEGF Fab.
ALTITUDE is a dose-escalating trial including patients with moderately severe and severe nonproliferative diabetic retinopathy (NPDR) as well as mild proliferative diabetic retinopathy receiving ABBV-RGX-314 (Regenxbio, AbbVie) at two dose levels in three cohorts.
At 1 year, ABBV-RGX-314 at dose level 1 and 2 was well tolerated, with no cases of chorioretinitis, vasculitis, occlusion and hypotony. There were a few mild and transient cases of episcleritis and intraocular inflammation that responded to topical therapy.
“I will point out that no prophylactic steroids were given to these patients,” Vajzovic said. Best corrected visual acuity was stable through 1 year.
The primary endpoint of the trial was a two-step improvement in ETDRS severity score, and this was achieved with dose level 1 and 2 by 33% and 20% of NPDR patients, respectively, as compared with only 12% of controls.
Dose level 2 had the best outcomes, with 100% of NPDR patients showing stable or improved disease severity and 70.8% showing improvement as compared with 25% in the control group. None of the patients at dose level 2 experienced a two-step worsening as compared with 37.5% in the control group. There was also a significant reduction in vision-threatening events up to almost 90%.
“I would like to thank all the investigators, especially the patients, who are helping us build more novel therapies, potentially a single injection in the clinic, that will help long-term disease patients,” Vajzovic said.
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Vajzovic L. Suprachoroidal delivery of ABBV-RGX-314 gene therapy for diabetic retinopathy (DR) without center-involved diabetic macular edema (CI-DME): The phase II ALTITUDE study. Presented at: FLORetina-ICOOR; Nov. 30-Dec. 3, 2023; Rome.
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