Beacon Therapeutics has reported a pivotal clinical success for its gene therapy in X-linked retinitis pigmentosa (XLRP), a rare inherited eye disorder that causes progressive vision loss. The positive visual-acuity results put the biotech in position to begin seeking regulatory approval for the treatment.
The Phase 2/3 Vista study evaluated two doses of laruparetigene zovaparvovec, or laru-zova, in 85 boys and men with XLRP, a disease that predominantly affects males. A year after receiving treatment, none of the participants in the control group met the study’s criteria for a visual-acuity response. The threshold required patients to correctly identify at least 15 additional letters on a low-light eye chart.
Laru-zova produced responses in both treatment groups. The responder rate reached over 24% among participants receiving the lower dose and 31% among those given the higher dose, allowing the study to achieve its primary goal.
Beacon CEO Lance Baldo said the pivotal findings demonstrate the extent of the benefit that the therapy could potentially provide for people living with a debilitating, vision-robbing disease that affects hundreds of thousands of individuals worldwide. He described the coming years as an important period for the company as it moves the program toward potential approval.
The biotech intends to move quickly toward regulatory review. Baldo said Beacon plans to begin a rolling biologics license application with the FDA before the end of the year. The company is also preparing to seek authorization in the European Union and the U.K., although Baldo acknowledged that partnerships could become important if Beacon is to fully commercialize laru-zova across multiple markets.
He said the company, which has a workforce of more than 80 people, is considering whether to commercialize the therapy independently or work with partners that have established infrastructure and market-specific expertise. For now, Beacon is keeping both approaches under consideration.
Beacon’s achievement comes after larger pharmaceutical companies encountered difficulties developing gene therapies for XLRP. Baldo said the company benefited from lessons generated by earlier programs from Biogen and Johnson & Johnson. Biogen’s XLRP gene therapy failed to meet its primary endpoint in a Phase 2/3 study in 2021, while J&J experienced a Phase 3 failure the following year. J&J’s partner, MeiraGTx, subsequently regained the therapy and is still pursuing a potential approval despite the unsuccessful trial.
According to Baldo, one important distinction was the way Beacon designed its clinical trial. Biogen selected retinal sensitivity as its primary measure, whereas J&J’s study evaluated participants’ ability to get through a virtual maze. Both approaches were influenced by trial methodologies previously used for other diseases, which was understandable given the limited knowledge surrounding XLRP when those studies were designed.
Baldo said being able to learn from earlier programs can give later developers an advantage. He credited not only the companies that conducted the earlier research but also the patients and clinicians who participated in those studies, generating knowledge that helped subsequent researchers refine their approach.
The other major factor, according to Baldo, is the design of laru-zova itself. The therapy delivers a complete version of the retinitis pigmentosa GTPase regulator (RPGR) gene using a viral capsid, with the treatment injected directly into the back of the eye.
Beacon Therapeutics has reported an important clinical development for its investigational gene therapy laru-zova, which is being developed for X-linked retinitis pigmentosa (XLRP), a rare inherited retinal disease that can cause progressive vision loss and blindness. The latest results from the pivotal Phase 2/3 VISTA trial could support the company’s next steps toward regulatory review.
Beacon Reports Positive VISTA Trial Results
The VISTA trial evaluated laru-zova in 85 male patients with XLRP. According to Beacon, the study met its FDA-endorsed primary endpoint, showing a statistically significant improvement in low-luminance visual acuity compared with the untreated control group.
At 12 months, 24.1% of participants receiving the low dose and 31% receiving the high dose met the trial’s responder definition, while no participants in the untreated control group met that definition. The responder measure was based on achieving at least a 15-letter improvement on a visual acuity test under low-light conditions.
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