RegenxBio Inc. is kicking off another late-stage clinical trial for its eye disease treatment after striking a significant development and commercialization deal with Chicago’s AbbVie Inc.
The Rockville company said Monday it has initiated its second phase 3 study of its experimental drug for patients with wet age-related macular degeneration (wet AMD), part of its collaboration with AbbVie — a deal worth potentially more than $1 billion for the Maryland biotech.
Under that deal, RegenxBio (NASDAQ: RGNX) could receive up to $1.38 billion in development, regulatory and commercial milestones from AbbVie (NYSE: ABBV), following an initial $370 million upfront.
This trial, called Ascent, is now live and screening patients. It's the second of two trials to assess this gene therapy candidate, called RGX-314, as a one-time treatment for wet AMD. The other, called Atmosphere, launched in January 2021.
Successful results from both trials would help pave the way for the company to submit its application to the Food and Drug Administration in 2024, according to RegenxBio. Regulatory approval would open the door for the business to take that product to market and to patients.
Within the agreement with AbbVie, RegenxBio handles the candidate’s clinical studies, which in addition to these trials include a phase 2 study in diabetic retinopathy.
Should the product reach the commercialization stage, both companies would equally share profits from U.S. sales and AbbVie would pay RegenxBio tiered royalties on international sales.
In addition, RegenxBio will lead manufacturing for the candidate’s clinical development and domestic commercial supply, AbbVie will spearhead manufacturing for its commercial supply outside of the U.S., the companies will tag-team a commercialization plan within the U.S. and AbbVie will lead clinical development and commercialization globally.
Patients with wet AMD experience vision loss because new blood vessels form, and leak, in the retina. So these trials set out to give these patients a one-time treatment alternative to the current standard of care for wet AMD and diabetic retinopathy, which involves monthly or bimonthly injections into the eye.
RegenxBio is also advancing several other gene therapy programs including for rare diseases such as Hunter syndrome, a debilitating disease in young children, which kicked off a phase 1/2 study in 2020. The company is also developing gene therapy treatments for hereditary angioedema, a rare genetic condition involving painful swelling episodes, and Duchenne muscular dystrophy, a syndrome marked by frequent falls, learning disabilities and other symptoms that affects people with muscular dystrophy.
It all comes as the business settles into a 132,000-square-foot headquarters where its more than 300 employees moved in 2021. That site, still in Rockville at 9800 Medical Center Drive, grows and centralizes the biotech’s previous 80,000-square-foot footprint that was distributed across five locations. RegenxBio's new manufacturing facility, which is part of the new headquarters project, is slated to open in late spring, the company confirmed Monday.
