RegenxBio has presented new interim data from several ongoing clinical trials in ophthalmology and genetic disorder indications investigating its adeno-associated vector (AAV) gene therapies.
Alongside the clinical trial development, Regenxbio is working to bring a new drug candidate into the clinic in a couple of years.
Go deeper with GlobalData
At the Virtual Investor Day, Regenxbio announced updates on its retinal programme investigating ABBV-RGX-314, which acts by neutralising the activity of vascular endothelial growth factor A (VEGF-A).
The gene therapy is developed in collaboration with AbbVie.
The interim data were from two Phase II ophthalmology trials called AAVIATE (NCT04514653) in wet age-related macular degeneration (wet AMD) and ALTITUDE (NCT04567550) in diabetic retinopathy (DR).
The data revealed that the third dose level cohorts did not demonstrate intraocular inflammation after receiving ABBV-RGX-314 suprachoroidal delivery in combination with a short course of prophylactic topical steroid eye drops. The third dose level consists of 1.0×10¹² genome copies per eye.
How well do you really know your competitors?
Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.
Thank you!
Your download email will arrive shortly
Not ready to buy yet? Download a free sample
We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form
By GlobalDataAs of 12 June, ABBV-RGX-314 was well tolerated in patients with wet AMD from the sixth cohort of the AAVIATE trial, and no serious drug-related adverse events were reported. The seven-week course of ocular steroid prophylaxis reduced mild to moderate intraocular inflammation seen in previous cohorts.
Regenxbio expects to report additional interim data and full six-month results from cohorts five and six from the AAVIATE trial in January 2024.
The drug candidate was also well tolerated in patients with DR from cohorts four and five in the ALTITUDE trial. Both cohorts received the third dose level of the gene therapy and topical steroid eye drops, with no reported cases of intraocular inflammation. Regenxbio expects to report additional interim data from the ALTITUDE trial in November 2023.
Duchenne programme
With clinical developments in ophthalmology, Regenxbio provided updates on its clinical programme in Duchenne muscular dystrophy (DMD) investigating RGX-202, a one-time AAV therapeutic that potentially restores partial muscle function.
As of 6 July, RGX-202 was well-tolerated in two patients who have been dosed to date with no drug-related serious adverse events.
The Phase I/II AFFINITY DUCHENNE trial is recruiting the paediatric population aged four to 11 years. The US Food and Drug Administration (FDA) granted a fast-track designation for the drug in April 2023.
Regenxbio plans to share additional interim data and longer-term safety and microdystrophin protein expression levels in muscle at three months in October 2023.
The company also announced its plans to bring a new potential therapy for DMD into the clinic. The new candidate is based on a novel exon-skipping construct. Regenxbio estimated that nearly half of DMD patients have mutations of the DMD gene that are amenable to exon skipping.
Regenxbio is initiating investigational new drug (IND) application-enabling studies and expects to submit an IND to the FDA in H1 2025.
Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.
