Beacon Therapeutics Granted FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for laru-zova (AGTC-501)

• RMAT designation provides benefits including enhanced communications with FDA throughout development
• Recognizes preliminary clinical evidence from the Phase 2 DAWN and SKYLINE trials of laru-zova in X-linked retinitis pigmentosa (XLRP)
• Laru-zova also holds Fast Track designation in US, Priority Medicines (PRIME) designation in EU and Innovative Licensing and Access Pathway (ILAP) designation in UK

LONDON and CAMBRIDGE, Mass., Jan. 28, 2025 /PRNewswire/ -- Beacon Therapeutics Holdings Limited ('Beacon Therapeutics' or 'the Company'), a leading ophthalmic gene therapy company with a mission to save and restore the vision of patients with blinding retinal diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to laru-zova (laruparetigene zovaparvovec) for the treatment of X-linked retinitis pigmentosa (XLRP).

RMAT designation was created to expedite the development and review of regenerative medicine therapies that have the potential to address unmet need in serious or life-threatening diseases, based on preliminary clinical evidence. RMAT designation provides benefits including interactive communications with the FDA throughout development, the opportunity to discuss appropriate endpoints to support accelerated approval, and eligibility for expedited programs such as priority review.

The FDA's RMAT designation for laru-zova recognizes preliminary clinical evidence from the Phase 2 DAWN and SKYLINE trials evaluating the efficacy, safety and tolerability of laru-zova in patients with XLRP caused by mutations in the RPGR gene. This evidence includes emerging data from DAWN showing improvements in low luminance visual acuity (LLVA), which provides support for the measure as a clinically meaningful endpoint in XLRP.

"The FDA's decision to grant RMAT designation to laru-zova is a significant milestone for the XLRP patient community, and underscores our promising data and the potential for laru-zova to significantly improve the lives of patients who suffer from XLRP," said Lance Baldo, MD, chief executive officer of Beacon Therapeutics. "We look forward to working closely with the FDA on continued development activities to support an expedited pathway for laru-zova."

Laru-zova also holds several additional regulatory designations, including Fast Track designation from the FDA, PRIME designation from the European Medicines Agency, and ILAP designation from the Medicines and Healthcare products Regulatory Agency in the UK.

Beacon Therapeutics recently presented updates on several studies of laru-zova, including positive interim 3-month data in the Phase 2 DAWN trial, positive interim 24-month data from the Phase 2 SKYLINE trial, and ongoing enrollment in the Phase 2/3 VISTA trial.

About laru-zova 
Laru-zova is a gene therapy currently being investigated for the treatment of patients with XLRP. Laru-zova expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss, representing a potential best-in-class treatment for progressive vision loss in patients with XLRP.

About the SKYLINE, DAWN and VISTA Studies
SKYLINE is an ongoing, fully enrolled, Phase 2, randomized, controlled study evaluating the safety, efficacy and tolerability of laru-zova in 14 male patients with XLRP caused by mutations in the RPGR gene. The study's primary endpoint is the proportion of response by microperimetry between the study and fellow eye at Month 12. NCT06333249

DAWN is an ongoing Phase 2, non-randomized, open-label study of laru-zova in the fellow eye of male participants with XLRP who have previously been treated with an AAV vector-based gene therapy delivering the full-length RPGR protein. The objective of DAWN is to assess two different dose levels of laru-zova for efficacy, safety and tolerability in the target population. DAWN is also evaluating the changes in visual function and functional vision, and is the first trial in the laru-zova clinical development program that is collecting and evaluating LLVA data. NCT06275620

VISTA is a currently enrolling, Phase 2/3, randomized, controlled, masked, multi-center pivotal study evaluating the efficacy, safety and tolerability of laru-zova in two study groups compared to an untreated control group. VISTA is currently enrolling. NCT04850118.

About XLRP
XLRP is a severe, aggressive, inherited retinal disease that often leads to blindness by middle age, with no treatment options available. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. XLRP primarily affects young males, with an estimated prevalence of 1 in 25,000 males in the US, Europe and Australia having XLRP with RPGR mutations.

About Beacon Therapeutics
Beacon Therapeutics is an ophthalmic gene therapy company founded to save and restore the vision of patients with a range of prevalent and rare retinal diseases that result in blindness. The Company has an established scientific foundation that includes a late-stage development candidate to treat XLRP and two preclinical programs, one targeting dry age-related macular degeneration (AMD) and another targeting an inherited cone-rod dystrophy (CRD).

Beacon is supported by funds from Syncona Limited, Forbion, Oxford Science Enterprises, TCGX, Advent Life Sciences and additional investors. Find out more about Beacon Therapeutics at beacontx.com.

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SOURCE Beacon Therapeutics