Gene therapy improves vision in patients with retinitis pigmentosa in phase 2b trial
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Key takeaways:
- MCO-010, a mutation-agnostic gene therapy, demonstrated vision improvement in retinitis pigmentosa.
- Nanoscope plans to submit a biologics license application to the FDA for MCO-010 later this year.
MCO-010 demonstrated positive topline results in vision improvement in patients with permanent and severe vision loss from advanced retinitis pigmentosa, according to a press release from Nanoscope Therapeutics.
The mutation-agnostic gene therapy also demonstrated sustained durability after a single intravitreal injection, with improvements in visual function persisting or increasing after week 52 of the 2-year phase 2b RESTORE trial.
“MCO-010 would, for the first time, provide ophthalmologists a treatment option that actually gives vision back to the patients suffering permanent vision loss from advanced retinitis pigmentosa (RP), no matter which of the 100-plus mutations caused the retinal degeneration,” Samuel Barone, MD, chief medical officer of Nanoscope, told Healio. “Since MCO-010 is delivered via simple in-office intravitreal injection, we anticipate rapid adoption globally, once approved.”
The randomized, controlled clinical trial involved 28 subjects with severe vision loss and a diagnosis of RP. One subject withdrew consent before receiving intervention, leaving 18 subjects to receive MCO-010 and nine subjects to receive sham control. In the MCO-010 group, nine subjects received a high dose of 1.2E11 gc per eye, and nine subjects received a low dose of 0.9E11 gc per eye. The high dose will be the commercial dose.
The trial met its primary endpoint of a statistically significant improvement of best corrected visual acuity at week 52 in both the high-dose group (0.337 logMAR; P = .021) and the low-dose group (0.382 logMAR; P = .029) compared with the sham control group (0.05 logMAR). The high-dose group also had a statistically significant BCVA improvement at week 76 (0.539 logMAR; P = .001).
“The visual acuity gains occurred regardless of underlying genetic mutation and were supported by improvements on other functional vision assessments,” Barone said.
MCO-010 was well tolerated, with the most common adverse events being mild or moderate anterior chamber cell and ocular hypertension.
Nanoscope plans to submit a biologics license application to the FDA for MCO-010 before the end of the year, Barone said.
“We are motivated to get this to patients as soon as possible, and because this well-tolerated treatment is showing significant benefit in a high unmet need population, we are confident in the pathway to potential approval,” he said.
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