Dive Brief:
- A gene therapy from Beacon Therapeutics succeeded in a late-stage study involving people with a rare, often blinding eye condition, setting the company up to seek regulatory approval.
- Called laruparetigene zovaparvovec, or laru-zova, the treatment helped a higher proportion of trial enrollees with X-linked retinitis pigmentosa read at least 15 more letters on a visual acuity test in low-light conditions than untreated participants after a year of follow-up. Beacon said the adverse events associated with treatment were “predominantly mild to moderate.”
- Further details from the study will be revealed at a medical meeting in October. The biotechnology firm plans to begin submitting a “rolling” approval application, which can be submitted in piecemeal fashion, by the end of the year.
Dive Insight:
In retinitis pigmentosa, certain light-detecting cells in the retina decay over time, causing progressive vision loss and possibly total blindness. The “X-linked” form, or XLRP, primarily affects boys, accounts for about 14% of those with the disease and is particularly severe.
Laru-zova is one of a few gene therapies young companies have been developing for the condition. These treatments deliver functioning copies of a gene, RPGR, that’s mutated in XLRP. Drugmakers have hoped this approach might preserve and restore the function of those light-sensing cells, though study results so far have been mixed. One program Biogen purchased in an acquisition failed in testing in 2021. Another, from MeiraGTx, missed its main objective in a pivotal study but is still headed toward a regulatory review.
Laru-zova could be different. The treatment was first developed by Applied Genetic Technologies Corp., which healthcare investor Syncona took private in 2022 and folded into what has since become Beacon. The late-stage study Beacon subsequently ran has become the “first and only pivotal trial” in XLRP to achieve its main goal, the company said.
In the trial, Beacon tested two different doses of the gene therapy in 85 male patients who have XLRP and were between the ages of 12 and 48 at the study’s start. Beacon’s goal was for treatment to help a higher proportion of patients achieve a 15-letter-or-more improvement on a low-lighting vision test.
According to Beacon, 24% of participants getting a low-dose of laru-zova and 31% of those on a high-dose achieved that goal, compared to none in the control group. The company also pointed to “supportive positive trends” on certain secondary measures, such as how well a part of the retina detects faint spots of light during an eye test. Some of those findings weren’t statistically significant, however.
“Beacon selected endpoints that would best capture improvements that matter to patients, particularly their ability to see in low-light conditions, which is one of the most challenging aspects of living with XLRP,” Robert Sisk, a professor of ophthalmology at the University of Cincinnati, said in a statement provided by Beacon.
Two serious adverse events were reported, but Beacon attributed both to the surgical procedure used to administer treatment.
The results “are both statistically significant and clinically meaningful, representing an important milestone for ocular gene therapy and demonstrating the potential for a one-time treatment to change the course of an inherited retinal disease,” Lance Baldo, Beacon’s CEO, said in a statement.
