Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for treatment of retinal degenerative diseases, on June 23 announced that its Investigational New Drug application (IND) is cleared to open for Phase 2b clinical trial of MCO-010, an ambient-light activatable optogenetic monotherapy to restore vision in patients with advanced retinitis pigmentosa (RP).
An IND is a request from a clinical study sponsor to obtain authorization from the Food and Drug Administration (FDA) to administer an investigational drug or biological product to humans.
“Congratulations to the Nanoscope team and I am excited to be part of it. We hope our therapy will restore vision in ambient light without any external device, eliminating risk of photo-toxicity,” said Sulagna Bhattacharya, CEO of Nanoscope, which has been a client of the TechFW incubator/accelerator since February 2016.
The impressive clinical data presented at theAmerican Academy of Ophthalmology 2020 annual meeting has persisted till the end of Phase1/2a study. “These are major milestones for Nanoscope with potential benefit for patients suffering from retinal degeneration,” said Board Chairman Al Guillem, PhD.
Guillem was one of the co-founders of ZS Pharma, a TechFW client that received an investment from the Texas Emerging Technology Fund. In 2015, Anglo-Swedish drug firm AstraZeneca acquired ZS Pharma in a deal worth $2.7 billion.
For Nanoscope Therapeutics, the Phase 2b trial, which starts in June, will be a randomized, double-blind, sham-controlled, multi-center trial in the U.S. for patients with advanced RP. The treatment will involve a single intravitreal injection of MCO-010 to confirm its ability to improve the patients’ vision. MCO has received orphan drug designations for RP and Stargardt disease from the FDA.
Nanoscope’s gene therapy uses an AAV2 vector to deliver the MCO genes into bipolar retinal cells, where they express polychromatic opsins enabling vision in different color environments. The therapy can be administered in an office setting. Additionally, the therapy is applicable for RP regardless of underlying gene mutations.
A Phase 1/2a open label study of MCO-010 demonstrated the optogenetic monotherapy was safe with improved quality of life consistent with functional vision and visual function in advanced RP patients.
“Our gene therapies re-program retinal cells and make them photosensitive to restore vision. MCO delivery with proprietary viral vector has allowed MCO-transduction in patients’ retinas, confirmed by sustained fluorescence reporter expression,” said Samarendra Mohanty, PhD, Nanoscope’s President and Chief Scientific Officer.
“We are excited by the guidance received from the 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.”
About RP: from Nanoscope
In normal eyes, opsins are expressed by photoreceptor cells and intrinsically photosensitive Retinal Ganglion Cells in the retina. When activated by light, they trigger the physiological process of vision. RP, linked to more than 60 different gene mutations, encompasses a group of rare genetic disorders where photoreceptors degrade gradually, leading to impaired vision and eventual blindness.