Nanoscope’s eye disease gene therapy shows mixed results in PhII

30 Mar 2023
endpts.com
Phase 2Phase 3Gene TherapyClinical ResultDrug Approval
Dallas-based biotech Nanoscope Therapeutics unveiled Phase II results on its gene therapy for a rare eye disease Thursday morning. In the RESTORE trial, 18 patients with retinitis pigmentosa got a gene therapy called MCO-010 while nine got placebo. On a vision test called the MLYMT, the treatment group had a one-point greater change over one year in their score compared to the placebo group, the primary endpoint of the study. However, the 95% confidence interval was 0.0 to 3.0, meaning the result was not statistically significant. The p-value was not provided. All in all, 12 of 18 people with retinitis pigmentosa who got the therapy saw a two level or greater improvement on the MLYMT at one year, Nanoscope said. In the placebo group, 3 of 9 people saw that level of improvement. Nanoscope declined to comment beyond the press release. In retinitis pigmentosa, the retina breaks down over time and can lead to blindness. Nanoscope’s therapy is injected directly into the eye with the goal of restoring the eye’s ability to detect low light levels. According to the press release, the therapy came with “no serious or severe adverse events,” though Nanoscope provided few details on safety outside of that. Roche’s Luxturna is already approved for certain subtypes of the eye disease caused by genetic mutations. A number of others are also working on rare eye disease gene therapies — Janssen and MeiraGTxMeiraGTx are testing a therapy for X-linked retinitis pigmentosa in a Phase III trial set to complete in March of next year, according to a federal clinical trials database . Nanoscope is also testing its gene therapy candidate in another rare eye condition known as Stargardt disease.
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