Nanoscope Therapeutics Eyes FDA Approval After Gene Therapy Trial Success

In a significant advancement for retinitis pigmentosa treatment, Nanoscope Therapeutics announced positive outcomes from its Phase IIb trial of MCO-010, a pioneering gene therapy, on Tuesday. The company is now gearing up to seek FDA approval later this year, marking a potential leap forward in the fight against vision loss.

Retinitis pigmentosa, a chronic genetic eye disease leading to retinal degeneration, affects thousands worldwide. Until now, treatments have been limited, with Spark Therapeutics’ Luxturna being the only FDA-approved gene therapy for the condition, catering exclusively to patients with specific RPE65 gene mutations. Nanoscope’s MCO-010 aims to broaden this horizon by offering a mutation-agnostic solution that could benefit all patients with the disease, irrespective of their genetic mutation.

The therapy uses an adeno-associated virus vector to deliver multi-characteristic opsins into the retina, re-enabling retinal bipolar cells’ ability to detect light. This innovative approach showed promise in the recent trial, which compared two doses of MCO-010 against a sham control in 27 participants.

Initially, the trial’s primary endpoint aimed to measure the efficacy of MCO-010 through the multi luminance mobility test at 52 weeks. However, in a strategic pivot, Nanoscope revised this endpoint in January 2024 after consultations with regulatory bodies. The new metric focused on changes in best-corrected visual acuity (BCVA), a critical measure of vision clarity.

This alteration paid off when the 52-week data revealed statistically significant improvements in BCVA for both doses of MCO-010, signifying the trial’s success under its new primary endpoint. Despite the low dose not outperforming the control at week 76, the high dose demonstrated a significant advantage over the sham treatment, with notable reductions in logMAR scores, indicating improved vision clarity.

Nanoscope’s confidence is further bolstered by the Icelandic Medicines Agency’s endorsement of a 0.3 logMAR change as clinically significant for patients with severe vision loss. This external validation adds weight to the therapy’s potential impact, positioning Nanoscope for a promising FDA submission in the latter half of 2024.

The journey of MCO-010 from conception to this pivotal moment reflects the dynamic nature of clinical research, where adaptability and patient-centric endpoints can drive breakthroughs in treatment. For patients with retinitis pigmentosa, Nanoscope’s developments offer a beacon of hope, heralding a future where vision loss could be substantially mitigated or even reversed, regardless of genetic background. As the biotech industry watches closely, Nanoscope’s strides may not only revolutionize retinitis pigmentosa treatment but also set new precedents for gene therapy applications across a spectrum of genetic diseases.

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