Beacon Therapeutics, based in London and Oxford, UK, have recently announced an investment of £96 million (€112 million) to fund the acquisition of AGTC (Applied Genetic Technologies Corporation, Nasdaq: AGTC), previously a clinical-stage company focused on the commercialization of adeno-associated virus (AAV)-based gene therapies. Beacon is now to be a leading ophthalmic gene therapy company to restore and improve the vision of patients with retinal diseases, developing a new generation of gene therapies to treat rare and prevalent retinal diseases. Syncona Limited, a UK-based investment funder focused on the life-sciences and Oxford Science Enterprises (OSE), will be the lead investors.
Beacon’s lead clinical asset is AGTC-501 (previously named as rAAV2tYF-GRK1-RPGR), a proposed treatment of X-linked RP (XLRP) that demonstrated meaningful efficacy and a good safety profile in the Phase I/II HORIZON trial, and expects to present 12-month data the second half of 2023. The clinical asset was acquired as part of Syncona’s acquisition of AGTC in November 2022. XLRP, one of the most common forms of retinitis pigmentosa with mutations in one gene, the RP GTPase regulator gene (RPGR gene), is thought to account for approximately 75% of XLRP recorded cases. The gene encodes a ciliary protein that regulates trafficking of proteins to the outer segment of photoreceptors. Males are more severely affected by the X-linked pathology with night blindness generally occurring within the first decade of life, followed by restriction of the visual field and loss of visual acuity leading to legal blindness in most patients by the fourth to fifth decade of life. Unlike other approaches in the gene therapy space, AGTC-501 correctly expresses the full length RPGR protein, thereby addressing the entirety of photoreceptor damage caused by XLRP, including both rod and cone loss. Beacon Therapeutics is awaiting feedback from the US Food and Drug Administration (FDA) regarding the study design of its upcoming VISTA clinical trial, a Phase II/III study to assess the effect of AGTC-501 on XLRP.
Other pre-clinical opportunities include an intravitreally (IVT) delivered novel AAV based program for dry AMD) and a further pre-clinical asset targeting cone-rod dystrophy (CRD) which is caused by a null mutation in the Cadherin Related Family Member 1 (CDHR1) gene. Commenting on the investment, David Fellows, Chief Executive Officer of Beacon stated that the company “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 pre-clinical pipeline. With the 12-month data from our Phase II 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.”