Beacon raises $170m for eye gene therapies

Beacon Therapeutics has secured $170 million in a Series B funding round, aimed at accelerating the development of its leading candidate, AGTC-501, for treating X-linked retinitis pigmentosa (XLRP). This financing was spearheaded by Forbion, a life sciences venture capital firm, and included contributions from existing investors such as Syncona Limited, Oxford Science Enterprises, and the University of Oxford, with additional investments from TCGX and Advent Life Sciences.

In October 2023, Syncona acquired Applied Genetic Technologies Corporation (AGTC) for $23.5 million. This acquisition transferred a portfolio of ophthalmic pipeline candidates to Beacon, including AGTC-501, an adeno-associated virus (AAV) gene therapy. XLRP is a genetic disorder leading to progressive vision loss, primarily affecting males due to retinal degeneration. AGTC-501 has undergone various clinical trials focused on this condition.

Promising interim data was revealed in February 2024 from the Phase II SKYLINE trial (NCT03316560), indicating that 63% of eyes treated with a high dose of AGTC-501 showed an improvement in retinal sensitivity of at least seven decibels (dB) in at least five loci after 12 months. Following these results, the first patient was enrolled in June 2024 for the pivotal Phase II/III VISTA registrational trial. Beacon intends to use data from this and previous trials to support a biologics license application (BLA) in the United States and a marketing authorization application (MAA) in Europe.

XLRP is mainly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. AGTC-501 works by expressing the full-length RPGR protein, which is expected to restore the function of both rod and cone photoreceptors. This approach is unique compared to other treatments as it aims to address the full spectrum of photoreceptor damage.

Beacon Therapeutics, based in London, was launched in 2023 with an initial $120 million to develop gene therapies for eye-related conditions. The latest funding round brings the company's total financing to $290 million. This additional capital will also support a Phase I/II trial for its programme targeting dry age-related macular degeneration.

David Fellows, CEO of Beacon, emphasized their focus on advancing their pipeline of ophthalmic gene therapies to preserve and restore vision for patients suffering from a variety of common and rare retinal diseases that lead to blindness.