Weekly Ophthalmic Newsletter

1. Novartis’ Eye Drug Triggers Rare Retinal Side Effects in AMD Patients

Two recent studies have found that a drug called brolucizumab, which was approved in 2019 and developed by Swiss pharmaceutical company Novartis AG to treat wet?age-related macular degeneration?(AMD), can cause rare retinal side effects due to its interaction with the human immune system.

Although the medication has been approved in over 70 countries and was considered safe during pre-approval testing, reports emerged a few months after its launch indicating rare retinal disorders in a small percentage of patients who had been treated with the drug.

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2. AstraZeneca's Ultomiris Nears Approval for NMOSD Treatment in EU

AstraZeneca's Ultomiris (ravulizumab) has received a recommendation for marketing authorization in the European Union (EU) for the treatment of adult patients who are anti-aquaporin-4 (AQP4) antibody positive (Ab+) and have neuromyelitis optica spectrum disorder (NMOSD).

If authorized, Ultomiris would become the first and only long-acting C5 complement inhibitor to be approved for the treatment of AQP4 Ab+ NMOSD in the EU.

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3. Gene Therapy Restores Vision in Teen with Leber Congenital Amaurosis

In a groundbreaking clinical trial sponsored by Atsena Therapeutics, 16-year-old Aiden Van De Rostyne-Moore was given the gift of sight after years of blindness caused by?Leber congenital amaurosis. Thanks to an experimental gene therapy treatment, Aiden was able to see snowflakes for the first time in her life.

Atsena's ATSN-101 gene therapy is designed to deliver working copies of the GUCY2D gene, which is critical for vision, to the retina. The treatment sends molecular machinery to the back of the eye, where it can provide the necessary gene to replace the mutated one causing the blindness.

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4. Extremely Rare Gene Variants Identified as Possible Cause of AMD

The National Eye Institute (NEI) conducted a study that found rare genetic variants associated with?age-related macular degeneration?(AMD).

The genetic variants generate malformed proteins that affect the stability of the membrane attack complex (MAC) and could trigger a chronic inflammatory response in the retina. Published in the journal iScience, the study suggests that MAC could be a target for therapy to slow down or prevent the progression of AMD.

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5. Bloomberg: Big Pharma Companies Eyeing Apellis for Acquisition

Apellis Pharmaceuticals has become the subject of takeover interest amid a recent wave of M&A deals in the biopharma industry, according to a Bloomberg report.

The company is reportedly in talks with advisors to assess its options and may consider partnerships or licensing agreements. However, despite the interest, a buyout is not guaranteed, as two sources told Bloomberg that the company would prefer to remain independent.

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