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Beacon Therapeutic raises $170M for XLRP gene therapy development

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Beacon Therapeutics Holdings Limited (Beacon Therapeutics) announced it has raised a total of $170 million in Series B funding to support its lead asset in clinical development for x-linked retinitis pigmentosa (XLRP).

First, a refresh of the company.

The ophthalmic gene therapy company was established in June 2023 by London-based Syncona Limited, a life sciences investment company.

  • This followed Syncona’s November 2022 acquisition of AGTC, a publicly-treated biotechnology company developing genetic therapies for rare inherited conditions.

Its purpose: Restoring and improving vision in patients with prevalent and rare retinal diseases that lead to blindness through the following:

  • An XLRP candidate (we’ll get to that shortly)
  • Two preclinical programs:
    • Dry age-related macular degeneration (AMD)
    • Cone-rod dystrophy (CRD)

Now this financing.

The funding round was led by Syncona Limited and other existing investors—Oxford Science Enterprises and the University of Oxford—as well as TCGX and Advent Life Sciences, with initial investments.

  • To date: The company has now raised an estimated $290 million in funding
    • Beacon previously secured $120 million in financing when it first launched, with that funding also being used toward its XLRP asset’s clinical development

And what is it being used for?

Two purposes:

  • To continue clinical development of this XLRP lead asset
  • To generate phase 1/2 clinical trial data for the dry AMD program

Now give me the rundown on this asset.

As a late-stage and lead development candidate for XLRP, AGTC-501 expresses “the full length RPGR (retinitis pigmentosa GTPase regulator) protein” via subretinal injection.

  • As a result: It’s intended to address all photoreceptor damage caused by XLRP, including both rod and cone loss
    • Note: XLRP is primarily a result of mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene
  • Why this is important: The blinding orphan disease currently has no commercially available treatments

Has it undergone clinical studies yet?

Yes! In February 2024, the company released positive 12-month data from the phase 2 SKYLINE trial  (NCT06333249), which analyzed AGTC-501 as a follow-up to its ongoing phase 1/2 HORIZON trial.

The study details: XLRP patients were randomized to receive two doses of AGTC-501:

  • High: 6.8 E+11 vg/eye
  • Low: 7.5 E+10 vg/eye

And those findings?

Beacon reported that the high dose of AGTC-501 demonstrated a 63% response rate in patients’ study, with those patients also demonstrating a “robust improvement in visual function—including mean retinal sensitivity.”

  • Overall: The asset was well tolerated, demonstrating a favorable safety profile with no clinically significant safety events associated with treatment

And more recently?

Just last month, Beacon reported it had dosed the first patient in its randomized, multicenter phase 2/3 VISTA study (NCT04850118).

The trial’s focus: Assessing safety, efficacy, and tolerability of two AGTC-501 doses (high/low) versus control when randomized (1:1:1) in male XLRP patients.

  • Special note: After 12 months (and if eligible), participants will have the option to switch to a high dose of AGTC-501 in their fellow eye.

Study duration: While participants will be followed for 24 months, the total study is 5 years

Click here for more details on this study design.

And what’s the end goal for this AGTC-501?

Ultimately, Beacon plans to use its long-term results on AGTC-501 from all three clinical trials—HORIZON, SYLINE, and VISTA—as supporting clinical data for a Biologics License Application (BLA) submission to the FDA.

In the meantime, keep your eyes peeled for interim data from the VISTA trial!

Will do! Lastly, any other Beacon updates to know about?

Yes, actually … CEO David Fellows noted that the company’s Board of Directors welcomed three new members:

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