Ocular Gene Therapy: Regenxbio Data Shows Consistent Preservation Of Visual Acuity, Retinal Thickness

Regenxbio Inc RGNX released Tuesday, Jan. 16, interim data from the Phase 2 AAVIATE trial of ABBV-RGX-314 for wet age-related macular degeneration (wet AMD) using suprachoroidal delivery. 

Investigational ABBV-RGX-314 using suprachoroidal delivery is designed as a one-time, in-office treatment.

The new data presented includes six-month results from two additional dose level 3 cohorts (Cohorts 5 and 6). 

ABBV-RGX-314, being developed in collaboration with AbbVie Inc ABBV, is also being investigated as a potential one-time gene therapy for diabetic retinopathy and other chronic retinal conditions.

ABBV-RGX-314 continues to be well tolerated in over 100 patients from three dose levels with no drug-related serious adverse events

ABBV-RGX-314 in over 50 patients at the third dose level demonstrated the highest reduction in treatment burden:

• 80% reduction in annualized injection rate.
• 50% injection-free.

Zero cases of intraocular inflammation were observed in patients who received short-course prophylactic topical steroid eye drops.

Baird writes that while pivotal data for RGX-314 is anticipated in about two years, the forthcoming results from the suprachoroidal administration in age-related macular degeneration (AMD) and diabetic retinopathy (DR) during the second half of 2023 are expected to provide valuable insights. 

These results could potentially support the program’s transition from a subretinal operative procedure to an in-office administration, presenting a significant improvement in commercial potential. 

Baird analyst notes that the decrease in the number of patients receiving injections is a common occurrence in Phase 2 studies. While this and competitive developments in the wet AMD field may pose challenges for REGENXBIO’s commercial prospects, the impact on reducing treatment burden is still considered significant. It keeps the Outperform rating with a price target of $34.

JMP Securities notes that the six-month data update from Cohorts 5 and 6 provides additional evidence that ocular gene therapy can be effectively and safely delivered to the suprachoroidal space via Clearside Biomedical Inc’s CLSD Microinjector, which would be paradigm-shifting for the field. 

Price Action: RGNX shares are down 7.98% at $14.05 on the last check Wednesday.