Beacon Succeeded in XLRP Where Biogen and J&J Failed, on an Endpoint Neither of Them Used

Beacon Therapeutics said on 21 September 2026 that laru-zova met the FDA-endorsed primary endpoint of VISTA, a 12-month randomised trial in 85 males aged 12 to 48 with X-linked retinitis pigmentosa. A gain of at least 15 letters in low-light visual acuity was reached by 31.0% on the high dose (p=0.0019) and 24.1% on the low dose (p=0.0106), against none of the untreated controls.
At least a 10-letter gain was reached by 48.3% on the high dose, 58.6% on the low dose and 3.7% of controls. Macular sensitivity on microperimetry improved by 1.201 dB on the high dose (p=0.0614) and 1.312 dB on the low dose (p=0.0405).
Ocular adverse events were mostly mild to moderate, balanced across groups and largely attributed to surgery. Drug-related events occurred in 25% of the high dose group and 38% of the low dose group, and both ocular serious adverse events, in the low dose group, were attributed to surgery.
Beacon plans to begin a rolling BLA later this year. Chief executive Lance Baldo told Fierce Biotech it will also seek approval in the EU and UK and may look for partners there. Full data go to the AAO’s Retina Subspecialty Day on 10 October.

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The result says as much about how XLRP trials are designed as about the gene therapy itself.

The endpoint made the difference, and the microperimetry data show it. Biogen’s programme used retinal sensitivity as its primary endpoint and missed in 2021; J&J’s used a virtual maze and missed last year, according to Fierce Biotech. On microperimetry, the measure closest to Biogen’s, Beacon’s high dose narrowly missed significance while the low dose reached it. On that primary endpoint, VISTA would have read as a partial miss.
There is no clean dose response. The high dose led on the primary endpoint, 31.0% against 24.1%, while the low dose led on the 10-letter response, 58.6% against 48.3%, and on microperimetry, and carried more drug-related adverse events, 38% against 25%. Regulators will ask which dose belongs on the label, and the topline does not settle it.
The first XLRP filings will rest on very different evidence. MeiraGTx bought its gene therapy back from J&J and plans to seek approval this year despite the failed phase 3, per Fierce Biotech. Beacon, a company of just over 80 people that says it may partner outside the US, now holds the only pivotal XLRP trial to meet its primary endpoint.