Interim results positive for gene therapy to treat X-linked retinitis pigmentosa
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Key takeaways:
- AGTC-501 gene therapy is under investigation for the treatment of X-linked retinitis pigmentosa.
- Beacon Therapeutics is planning to begin a phase 2/3 trial of AGTC-501 later this year.
Beacon Therapeutics announced the presentation of interim safety and efficacy results from the phase 2 SKYLINE trial evaluating AGTC-501 gene therapy for X-linked retinitis pigmentosa.
At 12 months, 63% of study eyes treated with high-dose AGTC-501 experienced a response rate, defined as an improvement in retinal sensitivity of at least 7 dB in at least five loci, according to a company press release. Untreated eyes in the high-dose cohort and eyes that received low-dose AGTC-501 experienced a 0% response rate. Patients in the high-dose cohort also showed improvement in visual function.
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The therapy was well tolerated, and there were no reports of clinically significant treatment-associated safety events.
“These data, which demonstrate a favorable safety profile and notable improvement in visual function, are another positive step in the development of AGTC-501 for [X-linked retinitis pigmentosa], a blinding, orphan disease for which there is currently no approved treatment,” Nadia Waheed, MD, chief medical officer of Beacon, said in the release. “We look forward to announcing the initiation of our phase 2/3 VISTA trial in the first half of 2024.”
The data were presented at the Macula Society meeting.
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