Week in review: Tocilizumab, fast track, retinas in a dish

A weekly roundup of ophthalmic news from around the web.

Tocilizumab shows promise for patients suffering from COVID-19–associated pneumonia. New phase 3 results reveal that patients receiving the drug—an interleukin-6 inhibitor approved for giant cell arteritis—along with standard care were 44% less likely to progress to mechanical ventilation or death by day 28 compared with those receiving placebo. Moreover, this global phase 3 trial is the first to primarily enroll underserved and minority patient populations. “We have been striving to improve inclusion and diversity in our trials,” said Jamie Freedman, MD, PhD, head of Genentech’s U.S. Medical Affairs. “During the COVID-19 pandemic, we saw how high the stakes were for many communities of color and made diversity the centerpiece of this trial.” The company plans to share the data with the FDA and other health authorities. Genentech

A gene therapy for dry AMD has been fast-tracked by the FDA. Gyroscope Therapeutics’ investigational AAV-based gene therapy is delivered under the retina of people with geographic atrophy who have a specific mutation in complement factor I (CFI). “Research suggests people with dry AMD who have certain CFI mutations that correlate with low CFI levels in the blood have a higher risk of developing AMD.” said chief medical officer Nadia Waheed, MD, MPH. A phase 2 trial is already underway, and another larger trial is slated to begin later this year. Gyroscope Therapeutics

Santen has acquired Eyevance Pharmaceuticals, paying $225 million in cash for the young ophthalmic company. Since coming onto the scene in 2017, Eyevance has focused on developing and commercializing topical treatments targeting the ocular surface and anterior segment. Their products include the first and only FDA approved ophthalmic antifungal (Natacyn) and the sole available acetate derivative of fluorometholone (Flarex). Two drugs that were being developed—a vancomycin ophthalmic ointment (Visovanq) and an ophthalmic gel for persistent corneal epithelial defects (Nexagon)—were excluded from the deal. Santen, Eyevance

A novel gene therapy approach for retinal diseases may bypass the need for invasive surgery. The key? Microneedles. Unlike current gene therapies that require more invasive, complex surgeries, the new method uses needles less than 1 mm in length to inject viral particles into the suprachoroidal space and is simple enough to perform in an office setting. Experiments in rhesus monkeys demonstrated that these suprachoroidal injections can produce diffuse, peripheral expression in retinal pigment epithelial cells but does induce localized inflammation—a side effect that will need further investigation. The authors say phase 3 clinical trials have shown these microneedles can be used to deliver steroids to successfully treat inflammatory eye diseases. UC Davis Health, Molecular Therapy Methods & Clinical Development

Production of personalized, functional retinas in the lab may soon be a reality, thanks to a team of Swiss scientists. The group has developed a model in which stem cells can self-organize into light-sensitive, multilayered retinal organoids with functional synapses and can give rise to most retinal cell types within 38 weeks. Even more impressive is that the approach enables researchers to generate thousands of highly uniform retinal organoids. "The research addresses a fundamental unmet need, which is to develop model retinas that closely resemble the real organ," said study investigator Cameron Cowan, PhD. “It opens up the possibility of developing treatments in a dish tailored to individual patients." Institute of Molecular and Clinical Ophthalmology Basel, Cell
(Featured image courtesy of IOB.ch)