Beacon Therapeutics launches with focus on retinal disease gene therapies
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Key takeaways:
- Beacon Therapeutics launches with an X-linked retinitis pigmentosa therapy in phase 2 trials.
- Preclinical assets include therapies for dry age-related macular degeneration and cone-rod dystrophy.
Beacon Therapeutics has launched with the goal of developing a new generation of gene therapies for a range of retinal diseases, according to a press release.
Syncona Limited and additional investors including Oxford Science Enterprises provided $120 million to fund the acquisition of AGTC and capital for Beacon’s other development candidates.
Beacon’s lead clinical asset is AGTC-501, a gene therapy program in phase 2 clinical trials for the treatment of X-linked retinitis pigmentosa (XLRP). The therapy, which was part of Syncona’s acquisition of AGTC in 2022, addresses the photoreceptor damage caused by XLRP by expressing the full-length RPGR protein.
“Beacon Therapeutics combines a broad development pipeline, a deep scientific foundation, a strong clinical network, and a highly experienced management team to drive forward a unique late-stage clinical and preclinical pipeline,” David Fellows, CEO of Beacon, said in the release. “With the 12-month data from our phase 2 SKYLINE trial for AGTC-501 expected shortly and two highly innovative and differentiated pipeline assets for prevalent and rare blinding diseases, we are excited to be building a new leader in the ophthalmic gene therapy space.”
Beacon’s preclinical assets include an intravitreally delivered AAV-based program for dry age-related macular degeneration and a program that targets cone-rod dystrophy.
Perspective
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It is exciting to see such a confident investment in the field of ocular gene therapy with the recent launch of Beacon Therapeutics having just been announced on June 12, 2023. With $120 million in funding, the new company was able to acquire the well-known gene therapy company AGTC’s lead clinical asset: AGTC-501 (laruparetigene zovaparvovec) for patients with X-linked retinitis pigmentosa (XLRP). Their pre-clinical therapeutic pipeline is now also augmented with novel investigational therapies including an intravitreal gene therapy for non-exudative or dry age-related macular degeneration and cone-rod dystrophy (CRD).
Beacon Therapeutics' lead therapeutic under clinical investigation is AGTC-501. This is currently in Phase 2/3 clinical trials evaluating its safety and efficacy in patients with XLRP, a disease that leads to widespread photoreceptor – both rods and cones – degeneration causing impaired vision, constriction of the field of vision, and blindness. AGTC-501 has the potential to help patients with XLRP by targeting mutations in the retinitis pigmentosa GTPase (RPGR) gene. By correctly expressing the full-length gene product (ie, the RPGR protein), AGTC-501 has the potential to mitigate damage to and loss of both rods and cones. Interim results of the randomized, controlled, masked, multi-center Phase 2/3 clinical trial with 63 participants between the ages of 8 and 50 are anticipated in the second half of 2023, with study completion targeted for January 2024. Similar to Spark Therapeutics (Genentech/Roche) revolutionary drug Luxturna (voretigene neparvovec-rzyl) for Leber’s congenital amaurosis, AGTC-501 is delivered via subretinal injection. 4D Molecular Therapeutics also has a candidate drug in Phase 1/2 clinical trials for XLRP with an intravitreal approach using the R100 vector. Therefore, the addition of Beacon Therapeutics into this increasingly competitive space represents a welcome investment.
Additional therapeutics targets from Beacon Therapeutics in dry AMD and CRD are also attractive for clinicians and patients alike due to the chronic nature of these conditions which don’t lend themselves nicely to such frequent interventions as monthly or bimonthly intraocular injections. We will have to patiently wait to see how developments in these other indications fare as they enter clinical studies in the future.
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