RegenxBio Inc. released Q1 2023 updates regarding ongoing development and commercialization plans for ABBV-RGX-314, its newly-renamed investigational gene therapy for the treatment of chronic retinal diseases.
Let’s talk about this gene therapy first.
Formerly known as RGX-314, ABBV-RGX-314 is in development as a novel, one-time subretinal injection.
Leveraging a novel adenosine-associated virus (N + AAV= NAV for short) vector, this gene therapy could treat patients with retinal diseases such as wet age-related macular degeneration (AMD) who might have been administered anti-vascular endothelial growth factor (anti-VEGF) in the past.
How does the NAV vector work?
The NAV vector is administered via injection or infusion, reaching the target cells and delivering the gene—thus allowing the cells to make the necessary body’s needed protein for various rare diseases.
And in ABBV-RGX-314?
ABBV-RGX-314 includes the NAV adeno-associated virus 8 (AAV8) vector containing a gene encoding for a monoclonal antibody fragment.
Through protein expression, this modified AAV vector is designed to neutralize VEGF activity, blocking the pathway where new, leaky blood vessels may grow and cause fluid to accumulate in the retina.
RegenxBio has a number of potential pipeline therapies as part of its NAV Technology Platform.
OK .. now back up. Why the name change?
RegenxBio renamed ABB-RGX-314 as part of transferring its investigational new drug (IND) applications to AbbVie—which it partnered with in 2021 to develop and commercialize ABB-RGX-314—for all nine current and ongoing clinical trials in the United States and Canada, including two pivotal trials.
Tell me more about these pivotal trials.
The ongoing phase 2b/3 ATMOSPHERE (NCT04704921) and phase 3 ASCENT (NCT05407636) pivotal trials are assessing the safety and efficacy of ABBV-RGX-314 for wet AMD.
Both studies are evaluating two doses of ABBV-RGX-314 (compared to an active comparator), with the primary endpoints being mean change in best-corrected visual acuity (BCVA) relative to ranibizumab (ATMOSPHERE) or aflibercept (ASCENT).
Patient enrollment is ongoing for both trials.
When can we expect results?
According to Clinical Trials, the studies are estimated to be completed on the following dates:
ATMOSPHERE → March 2024
ASCENT → December 2024
What else?
RegenxBio also announced that the IND applications transfer will support a global expansion of the ATMOSPHERE and ASCENT trials, with AbbVie taking over as the lead regulatory party.
Further, AbbVie will now be responsible for the global development and commercialization of ABBV-RGX-314; however, both companies will continue collaboration on additional clinical trials of the gene therapy.
What about these other trials?
Funny you should ask … the company reported that a non-randomized, long-term, follow-up Fellow Eye Treatment study (NCT03999801) has been initiated
The trial is assessing the safety, efficacy, and immunogenicity of ABBV-RGC-314 via subretinal delivery into the eyes of patients (from the ATMOSPHERE and ASCENT studies) with bilateral disease who already received a subretinal injection.
Per Clinical Trials, the study is slated to complete in December 2028.