Samuel Barone, MD, is the chief medical officer (CMO) of Nanoscope Therapeutics, a late-stage clinical biotechnology company developing gene therapies for inherited retinal diseases and geographic atrophy (GA) secondary to AMD. Dr. Barone is a practicing board-certified ophthalmologist and vitreoretinal surgeon with clinical development and regulatory experience across a range of indications in ophthalmology, dermatology, rare diseases, and cell and gene therapy.
Ophthalmology Management: Can you discuss your role as CMO at Nanoscope and how you got started in this role?
Samuel Barone, MD: As CMO, I am responsible for the execution of our clinical pipeline in retinitis pigmentosa (RP), Stargardt disease and soon central GA from advanced dry AMD. I have been CMO since September 2023 but have served as an advisor to the company since 2018 and helped design our first-in-human Phase 1/2a study.
OM: Why did you move in this direction with your career and how?
SB: Relatively early in my ophthalmology and retina career, an opportunity presented itself at the FDA. I pursued it to gain a better understanding of the regulatory process with the intention of leveraging the experience with a full-time clinical practice that could be broadened by clinical investigations and research. During my time at the FDA as a senior medical officer, I discovered I really enjoyed the public health component of drug development and recognized how many more lives I could potentially impact if I could bring a drug to market.
OM: Can you discuss the significance of having a practicing ophthalmologist and vitreoretinal surgeon as CMO?
SB: I find it to be a very helpful skill set, particularly with developing products at Nanoscope that target retinal diseases. Continued retinal practice provides me with first-hand understanding that allows me to relate and speak to the patient experience and practice dynamics, which are directly applied to considerations like high unmet needs, safety, administration procedures and messaging.
Staying clinically active serves as a reminder of why we do what we do in clinical development, which is ultimately for patients. It is very meaningful and something that I truly value. I would not take a role that would not allow me to continue to practice. I thank my CEO, Sulagna Bhattacharya, for recognizing the value of this and supporting it.
OM: As a former senior medical officer at the FDA, how will that experience help in advancing Nanoscope’s lead program MCO-010 for retinitis pigmentosa?
SB: My FDA experience in the direct office and center where our MCO-010 product resides has been quite helpful as we move forward. We are fortunate that the current posture of FDA leadership is one of support for innovative therapies for serious conditions with high unmet needs. But it is not straightforward and requires ongoing discussion and collaboration. My understanding of the FDA’s position, processes and procedures goes a long way toward helping these interactions be fruitful for both sides so that we can together bring transformative therapy to the clinic.
OM: Nanoscope announced positive results from the Phase 2b RESTORE trial of MCO-010. Can you give us an overview?
SB: The randomized, controlled Phase 2b RESTORE trial showed meaningful vision gains in patients with severe vision loss from permanent photoreceptor loss from RP. In this study, we randomized 27 individuals to one of two MCO-010 dose groups administered by a single intravitreal injection, or to sham control. Inclusion criteria included BCVA no better than 1.9 LogMAR (20/1600 Snellen equivalent) in the study eye and 1.6 LogMAR (20/800 Snellen equivalent) in the better-seeing eye. The primary endpoint was mean change in BCVA at week 52 with the only key secondary endpoint at week 76.
In both dose groups and at both time points, we observed mean BCVA gain >0.3 LogMAR, the threshold that has been used to support approval in other ophthalmology indications like macular degeneration and diabetic retinopathy. This was statistically significant compared to control. There were no serious or severe ocular adverse events and no inflammatory adverse events. The results in RESTORE are consistent with the safety and efficacy findings in our previous Phase 1/2a trial, and we anticipate submitting a Biologic License Application to the FDA by the end of the year.
OM: Can you discuss the other programs in your pipeline?
SB: Last year, we presented 1-year results from our open label Phase 2a STARLIGHT study evaluating MCO-010 in six patients with severe vision loss from Stargardt disease. MCO-010 continued to be safe, and gains in vision were observed across multiple parameters. We look forward to starting a Phase 3 registrational study before the end of the year.
In addition, MCO-020 is our next-generation, non-viral vector program that uses lower power OCT-guided laser to induce transfection. With MCO-020, we will be targeting vision loss from central GA in advanced dry AMD with the potential to restore vision. MCO-020 is currently in investigational new drug (IND)-enabling studies, and we anticipate opening an IND before the end of the year. OM
