Optogenetic Monotherapy for Retinitis Pigmentosa IND Approved, Phase 2b Study Enrolling


The announcement follows positive results from the phase 1/2a trial of MCO-010 for RP.

The FDA has approved the investigational new drug (IND) application for Nanoscope Therapeutics’ MCO-010 for the treatment of retinitis pigmentosa (RP).1

MCO-010, an ambient-light activatable optogenetic monotherapy, is now set to be studied in a phase 2b clinical trial. 

"We hope our therapy will restore vision in ambient light without any external device, eliminating risk of photo-toxicity," said Sulagna Bhattacharya, chief executive officer, Nanoscope, in a statement, while board chairman Al Guillem, PhD, added, “these are major milestones for Nanoscope with potential benefit for patients suffering from retinal degeneration."

The randomized, double-blind, sham-controlled, multi-center trial has begun enrolling patients with advanced RP to receive a single intravenous injection of MCO-010. The treatment’s efficacy and safety will be evaluated. 

MCO-010 consists of an AAV2 vector to deliver the MCO gene payload to bipolar retinal cells, which then express polychromatic opsins to enable vision in different color environments. The therapy is applicable for patients regardless of underlying gene mutations and can be administered in an office setting. The FDA previously granted the treatment orphan drug designation for both RP and Stargardt disease.

READ MORE: Targeting Human Retinal Progenitor Cell Injections for Retinitis Pigmentosa

"Our gene therapies reprogram retinal cells and make them photosensitive to restore vision. MCO delivery with proprietary viral vector has allowed MCO-transduction in patients' retina, confirmed by sustained fluorescence reporter expression," Samarendra Mohanty, PhD, Nanoscope's president and chief scientific officer, said in a statement.

"We are excited by the guidance received from FDA regarding improvising the characterization of our product, the primary endpoint and potency assays that will hopefully accelerate our clinical program to make the restorative drug available to RP patients," Mohanty added.

A recent phase 1/2a open-label study demonstrated that MCO-010 was safe and was associated with consistent vision improvements through 1 year, with expected improvements in quality of life in patients with advanced RP. The study enrolled 11 patients, 3 of which received a low dose of 1.75 × 1011 VG per eye and 8 of which received a high dose of 3.5 × 1011 VG per eye. Successful gene transduction was confirmed with florescence imaging.

At 1 year, 6 (86%) high-dose patients gained >0.3 logMAR (15 letters) while the seventh patient could not be evaluated due to COVID-19 after 31 weeks. All participants evaluated showed both objective and subjective improvements in functional vision and shape discrimination accuracy to at least 90% compared with baseline. Participants’ performance in 2 different mobility tests also improved, with a 50% reduction in time to touch lighted panel in 1 test. Patient-reported outcomes were also positive and correlated with these mobility test outcomes.

“After MCO-treatment, the patients reported long-lasting improvements in outdoor light sensitivity and daily activities. We were pleasantly surprised that after eight weeks of treatment, some subjects could attend their follow-up visits during the study without the assistance of a chaperone. Some of the patients even gained the ability to read letters on a wall or even the large text in a newspaper, use a cell phone, watch television, and could even thread a needle,” principal investigator Santosh Mahapatra, MBBS, MS, ophthalmologist and eye surgeon, said in a statement at the time.

1. Nanoscope Therapeutics announces FDA approval of IND for phase 2b clinical trial of optogenetic gene monotherapy to restore vision in patients with retinitis pigmentosa. News release. Nanoscope Therapeutics. June 23, 2021. https://www.biospace.com/article/releases/nanoscope-therapeutics-announces-fda-approval-of-ind-for-phase-2b-clinical-trial-of-optogenetic-gene-monotherapy-to-restore-vision-in-patients-with-retinitis-pigmentosa/
2. Nanoscope’s optogenetic gene therapy restores clinically meaningful vision in 11 patients blinded by retinitis pigmentosa. News release. Nanoscope Therapeutics. June 3, 2021. https://nanostherapeutics.com/2021/06/03/nanoscopes-optogenetic-gene-therapy-restores-clinically-meaningful-vision/
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