Nanoscope's Gene Therapy Secures Phase II Victory in Treating Retinitis Pigmentosa.
The primary objective of a Phase IIb trial aiming to treat retinitis pigmentosa was achieved by MCO-010, which was compared to a placebo in a small study.
The gene therapy arena has earned another victory as Nanoscope Therapeutics based in Dallas, Texas, declared upbeat results in a Phase IIb study of MCO-010 for innovative retinitis pigmentosa treatments.
The Phase IIb RESTORE trial, which consisted of 27 patients, has successfully achieved its primary efficacy goal based on the Multi-Luminance Y-Mobility Test (MLYMT). The MLYMT is a test that measures a patient's capacity to move through an area filled with LEDs lights of varying brightness levels. In the trial, 18 of the participants received MCO-010 while the remaining 9 received an intravitreal injection that did not contain the active ingredient, also known as a sham injection.
) The authorities believe that a difference of no less than two degrees of brightness on the MLYMT is significant enough to show improvement in a patient's ability to move around in a darker room after treatment. In the RESTORE study, 16 out of 18 patients who were given MCO-010 met this standard of effectiveness, while only 4 out of 9 patients who were given a placebo achieved it. (p value not provided).
The MCO-010 had a favorable impact on the other measures apart from the primary endpoint, which included Multi-Luminance Shape Discrimination Test (MLSDT) and Best-Corrected Visual Acuity (BCVA). These measures assess the patient's capability to identify shapes or numbers from a distance.
Regarding safety, there were no significant negative incidents reported for patients who received MCO-010 treatment. The most frequently reported side effects that arose during treatment included the presence of anterior chamber cells, elevated pressure in the eyes, and bleeding in the conjunctiva.
Nanoscope has developed a gene therapy that can provide white opsin to the cells in the eye. This can help to restore vision based on seeing white light and reduce ongoing damage to the retinal cells. The World Health Organization recently designated the official name, "sonpiretigene isteparvovec”, for this therapy also known as MCO-010.
A set of uncommon genetic disorders referred to as retinitis pigmentosa involves the gradual deterioration of the retina, leading to visual impairment and eventual blindness. MCO-010 has been granted Fast Track recognition by the FDA, as well as orphan drug designations for both Stargardt disease and retinitis pigmentosa.
Advancements in Gene Therapy Field
Nanoscope's MCO-010 is currently undergoing the RESTORE trial, which is one of five significant gene therapy trials expected to have significant results in the first six months of 2022.
Applied Genetic Technologies is conducting the AGTC-501 (NCT04850118) Phase II/III investigation in the field of vision loss alongside MCO-010, with interim findings scheduled for H1 2023. The trial focuses on X-linked retinitis pigmentosa prompted by mutations in the RPGR gene and assesses the effects of AGTC-501, also called laruparetigene zovaparvovec.
With more and more investments being made in late-stage gene therapies, there is also a large influx of funds being directed towards developing delivery systems for gene therapy, such as adeno-associated viruses (AAVs). Additionally, by 2023, we may witness the very first CRISPR gene therapy receiving approval from the FDA.
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