Chiesi Group’s idebenone faced a regulatory setback last month after the U.S. FDA issued a complete response letter (CRL) to the company’s NDA for Leber hereditary optic neuropathy (LHON), a rare inherited disorder that causes sudden vision loss.But a chance missed for Chiesi may be an opportunity for gene therapies, including Gensight Biologics SA’s lenadogene nolparvovec (Lumevoq; GS-010).

Biogen Inc. hardly blinked at the competition faced by Apellis Pharmaceuticals Inc. as the two companies sealed a deal whereby the former has agreed to acquire all outstanding shares of the latter for $41 each, or about $5.6 billion.

Analysts were sounding pleased and the company intends to go ahead with a regulatory filing, but investors seem to have wanted more from Viridian Therapeutics Inc.’s top-line data from the elegrobart (formerly VRDN-003) Reveal-1 phase III trial in active thyroid eye disease (TED). Viridian shares (NASDAQ:VRDN) closed March 30 at $18.53, down $8.86, or 32%.

After previous setbacks with the program, investors largely brushed aside ocular-focused Kodiak Sciences Inc.’s anticipated phase III Glow2 data of tarcocimab tedromer in diabetic retinopathy (DR), so the positive top-line superiority results revealed March 26 caught many by surprise as it sets the company up for an accelerated multi-indication BLA submission.

Innovent Biologics Inc.’s efdamrofusp alfa (IBI-302) met the primary endpoint in the phase III Star trial in neovascular age-related macular degeneration (nAMD), and the Suzhou, China-based company will submit an NDA to China’s National Medical Products Administration.

It’s déjà vu all over again for Aldeyra Therapeutics Inc., which disclosed its third complete response letter (CRL) for dry eye disease candidate reproxalap, with the U.S. FDA citing a lack of substantial and consistent evidence of efficacy.

Scientists at Duke University have uncovered how macrophages help maintain intraocular pressure and have found that a specific type, resident macrophages, is essential for proper drainage of intraocular fluid. When these cells are removed, drainage becomes impaired and intraocular pressure rises, contributing to the development of glaucoma.

Similarities among three pediatric brain tumors that arise in different structures of the CNS – pineoblastoma, retinoblastoma and Group 3 medulloblastoma – have been linked to their shared origin during pineal gland development. Scientists at St. Jude Children’s Research Hospital have identified the molecular signatures that drive these tumors from pinealocyte progenitor cells that conserve a common differentiation program, providing a shared therapeutic target for these three cancer types.

Ocular Therapeutix Inc.’s wet age-macular degeneration candidate, Axpaxli, beat anti-VEGF therapy Eylea (aflibercept) from Regeneron Pharmaceuticals Inc. in the phase III head-to-head trial called Sol-1, but not by enough of a margin for Wall Street. Shares of the firm (NASDAQ:OCUL) closed Feb. 17 at $6.99, down $1.89, or 21%, as investors mulled the top-line findings.