Nanoscope Therapeutics, Inc.

Pictured: Illustration of a gene therapy showing a syringe injecting a treatment into a DNA strand/iStock, Ilya Lukichev Nanoscope Therapeutics claimed a hit on the changed primary endpoint of its Phase IIb vision loss gene therapy trial Tuesday, as the biotech looks to FDA approval of the candidate this year. The study compared two doses of MCO-010, a mutation-agnostic gene therapy, to a sham control in 27 people with retinitis pigmentosa. The chronic genetic eye disease is characterized by degeneration of the retina. Spark Therapeutics, part of Roche, won FDA approval for Luxturna (voretigene neparvovec) in the condition in 2017 but the authorization only covers patients with mutations in the RPE65 gene. Nanoscope designed MCO-010 to improve the vision of all patients with retinitis pigmentosa, regardless of the genetic basis of their condition. The gene therapy consists of an adeno-associated virus vector that delivers multi-characteristic opsins to re-enable retinal bipolar cells to detect light. Last year, an investigator in the clinical trial said the study did not meet its primary endpoint, which, at that time, was assessing the efficacy of MCO-010 using the multi luminance y- mobility test at 52 weeks. Spark tracked the change in multi-luminance mobility test scores over 52 weeks to show the efficacy of Luxturna. The test measures changes in functional vision, as assessed by the ability of a subject to navigate a course accurately and at a reasonable pace at different levels of light. Nanoscope changed the primary endpoint of its Phase IIb trial in January 2024 and released a statement about its talks with regulatory agencies the next week. According to Nanoscope, the FDA said the change from baseline in a measure of visual acuity in low vision patients could be an appropriate primary efficacy endpoint to support a filing for approval. Tuesday, Nanoscope presented data on the change in best-corrected visual acuity (BCVA) at week 52. The Phase IIb study linked both doses of the gene therapy to statistically significant improvements in BCVA, a vision test, causing the trial to hit its new primary endpoint. Nanoscope changed the primary endpoint after publishing 52-week results on the original measure of efficacy, although the biotech said it finalized and submitted a revised statistical analysis plan before the completion of week 76. The low dose failed to beat control on BCVA at week 76 but the high dose was significantly better than the sham treatment. The biotech tracked reductions in logMAR from baseline of 0.539 and 0.374, respectively, in the high and low dose arms after 76 weeks. Nanoscope said in January that the Icelandic Medicines Agency endorsed a 0.3 logMAR change as being clinically meaningful for patients with severe vision loss. The biotech is now preparing to FDA approval in the second half of 2024. Nick Paul Taylor is a freelance pharmaceutical and biotech writer based in London. He can be reached on LinkedIn.

Retinitis pigmentosa is a group of rare genetic eye disorders. Nanoscope Therapeutics is back with more data assessing its gene therapy that uses light-sensitive molecules to treat retinal disease. With data demonstrating vision improvement in hand, the biotech is now planning to submit the therapy for FDA approval later this year. The two-year phase 2b study tested MCO-010 among patients with permanent and severe vision loss from advanced retinitis pigmentosa (RP), a term that encapsulates a group of rare genetic eye disorders. The new data build off early findings presented a year ago that found that 16 of the 18  (89%) patients injected with MCO-010 demonstrated an improvement of two or more luminance levels across two visual tests at 12 months, compared to four of the nine (44%) patients who received placebo.     Now, the Texas-based biotech has shared that the trial, dubbed RESTORE, also demonstrated a statistically significant improvement in best-corrected visual acuity (BCVA) after 52 weeks for 18 patients split among a high-dose and low-dose group, compared to nine participants in the control arm. The measure was the trial’s primary endpoint. “We observed significant vision restoration in many patients with severe vision loss, including those who were completely blind,” David Boyer, M.D., an investigator in the trial and ophthalmology professor at the University of Southern California Keck School of Medicine, said in a March 26 release. “Many patients treated with MCO-010 derived a clinically meaningful benefit measurable on the primary visual function test, and this effect was confirmed by a parallel improvement in functional vision assessments.” A single intravitreal injection of MCO-010 also showed statistically significant BCVA improvement at Week 76 among the high-dose group. However, improvement in the low-dose arm was not statistically significant at the same extended time stamp. The gene therapy was generally well tolerated, and no treatment-related serious or severe adverse events were reported, according to Nanoscope.    With new data in hand, Nanoscope intends to apply for FDA approval in the second half of 2024. The biotech plans to use the higher dose of MCO-010 (1.2E11gc/eye) as the commercial dose. Currently, there is no available cure for RP. MCO-010, which has already snagged fast-track and orphan tags from the FDA, is an ambient-light activatable multicharacteristic opsin optogenetic therapy. Unlike other eye-targeted gene therapies, which take aim at a specific gene mutation in the outer retinal cells, Nanoscope’s therapy is designed to target inner retinal neurons to restore vision lost as RP progresses. Nanoscope is also testing out the mutation-agnostic gene therapy in Stargardt disease, another rare eye disease.

Plus, news about bluebird bio, Q32 Bio and Aquestive Therapeutics: Edgewood Oncology’s $20M Series A: The Brookline, MA-based biotech plans to use the money to support Phase 2a studies in acute myeloid leukemia and targeted breast cancer. It’s financed by Alta Partners. — Kyle LaHucik Nanoscope to seek FDA approval for eye gene therapy: The Dallas-based biotech said its mutation-agnostic gene therapy MCO-010 passed a Phase 2b study in retinitis pigmentosa, significantly improving vision measured on an eye chart at one year at both the high and low doses compared to a sham control. It expects to submit an application for MCO-010 to the FDA in the second half of this year. — Lei Lei Wu