Beacon Therapeutics Reports Positive Phase 2 Data for Potential Best-in-Class Gene Therapy Laru-zova (AGTC-501) in X-Linked Retinitis Pigmentosa

Dec 09, 2024 07:30 CST Updated 07:30

Beacon Therapeutics

Ophthalmology Gene Therapy Technology Researcher

▎WuXiEdited by the Kant Content Team

Beacon Therapeutics recently announced the 3-month interim safety and efficacy data from the Phase 2 clinical trial, DAWN, evaluating the investigational gene therapy laru-zova (AGTC-501) for the treatment of patients with X-linked retinitis pigmentosa (XLRP).

Data from three months indicates:

Laru-zova was well tolerated in all participants, with no treatment-emergent adverse events related to the study drug reported, including no ocular inflammatory adverse events.
The data also showed good signs of early improvement in low-luminance visual acuity (LLVA), a key visual function indicator.
The benefit-risk assessment results support continuing the study for Retinitis Pigmentosa GTPase Regulator (RPGR) Clinical development for patients with XLRP caused by gene mutations.

▲Laru-zova Improves Patients' Low-Light Vision in Phase 2 Clinical Trial DAWN (Source: Beacon Therapeutics Official Website)

XLRP is a severe aggressive hereditary retinal disease that primarily affects young males, often leading to blindness by middle age, with no available treatments currently.Laru-zova is a gene therapy based on an adeno-associated virus vector that expresses the full-length RPGR protein. Therefore, it has the potential to address all photoreceptor damage caused by XLRP, including the loss of rod and cone cells, representing a potential "best-in-class" treatment option for progressive vision loss in XLRP patients.

Currently, Beacon Therapeutics is conducting the pivotal Phase 2/3 clinical trial VISTA, developing laru-zova as a potential therapy for patients with XLRP, and patient enrollment is ongoing.

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References:

[1] Beacon Therapeutics Announces Positive 3-Month Data from Phase 2 DAWN Trial of laru-zova (AGTC-501) in Patients with X-Linked Retinitis Pigmentosa (XLRP). Retrieved December 6, 2024, from https://www.prnewswire.com/news-releases/beacon-therapeutics-announces-positive-3-month-data-from-phase-2-dawn-trial-of-laru-zova-agtc-501-in-patients-with-x-linked-retinitis-pigmentosa-xlrp-302324627.html

[2] Subretinal Gene Therapy laru-zova (AGTC-501) for X-Linked Retinitis Pigmentosa (XLRP) Phase 2 Multicenter Study (DAWN): Preliminary Results. Retrieved December 6, 2024, from https://www.beacontx.com/wp-content/uploads/2024/12/2024-Dec-3-FLORetina-2024-DAWN-3M-data-presentation-FINALw.pdf

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