Beacon's gene therapy shows vision improvements in early phase 2 data

Beacon Therapeutics is making strides in the field of gene therapy with its promising treatment for X-linked retinitis pigmentosa (XLRP), a genetic disorder leading to progressive vision loss. Their investigational therapy, laruparetigene zovaparvovec (laru-zova), has shown encouraging preliminary results in improving visual function.

XLRP is a hereditary retinal disease that affects approximately 1 in 25,000 males in regions like the U.S., Europe, and Australia. It typically leads to significant vision decline starting in childhood, often resulting in blindness by middle age. Currently, there are no approved treatments targeting this debilitating condition.

Beacon Therapeutics' laru-zova aims to restore vision by delivering a functional copy of the retinitis pigmentosa GTPase regulator (RPGR) gene, crucial for normal visual function. This gene therapy is intended to enhance the functionality of both rods and cones in patients' eyes. In July of last year, Beacon secured $170 million in series B funding to further this promising treatment and to support regulatory applications in both the U.S. and Europe by integrating results from their ongoing phase 2 study with data from an earlier phase 1/2 trial.

The company recently disclosed interim six-month safety and efficacy data from their phase 2 trial, called Dawn, which examines the untreated eye of patients who have already received gene therapy in one eye. This open-label, non-randomized trial provided positive early signals, showcasing improvements in several critical visual function measures, such as low luminance visual acuity and eye sensitivity. While the study is primarily focused on safety, the data suggest potential benefits of laru-zova in enhancing vision for XLRP patients.

Beacon Therapeutics CEO Lance Baldo, M.D., emphasized the significance of the latest findings, asserting that the company is encouraged by the data and is eager to continue developing this innovative treatment option. Over the past five years, Beacon has compiled a solid foundation of safety and efficacy data for laru-zova through multiple clinical studies.

In addition to the Dawn study, the company is conducting the phase 2/3 Vista trial, which is a randomized, controlled study. This will provide more comprehensive insights into the therapy's efficacy, safety, and tolerability by comparing two study groups receiving laru-zova with an untreated control group.

The positive interim results from Dawn offer renewed hope for addressing a challenging disease area where other pharmaceutical companies have faced setbacks. Notably, Johnson & Johnson recently reported that their investigational gene therapy, botaretigene sparoparvovec, did not achieve its primary goal in a phase 3 trial. Even though it showed some improvements in secondary measures, the company did not find these results to be statistically significant and is continuing to analyze the data.

Meanwhile, another company, 4D Molecular Therapeutics, decided to discontinue its XLRP program earlier this year. Their gene therapy was under evaluation in a phase 1/2 trial, which was anticipated to yield results in the following year.

Beacon Therapeutics' ongoing research efforts and promising findings offer a potential breakthrough for XLRP patients, providing them with a glimmer of hope in a field where effective treatment options have been elusive.