Ocuphire Pharma Inc. is marching ahead despite falling short of the primary endpoint in top-line data from the Zeta-1 phase II study testing oral APX-3330 in diabetic retinopathy (DR). The trial did not achieve its goal with regard to the percentage of patients with a ≥2-step improvement in Diabetic Retinopathy Severity Score at week 24 in the study eye. Ocuphire plans to schedule an end-of-phase II meeting with the U.S. FDA to review the results.
The eyes certainly have it. There are plenty of ocular gene therapies being studied in a crowded field and now there is a new one as Perceive Biotherapeutics Inc. has closed on a $78 million series B financing.
Word that big pharma firms are sniffing around Horizon Therapeutics plc as a takeover candidate caused not only that firm’s shares to jump but also provided a boost for Viridian Therapeutics Inc., which has a thyroid eye disease (TED) candidate set to challenge Horizon’s Tepezza (teprotumumab-trbw), cleared by the U.S. FDA in January 2020.
A lackluster efficacy signal has prompted Editas Medicine Inc. to pause enrollment in a phase I/II trial of its CRISPR/Cas9-based gene editing therapy, EDIT-101, which is in development for patients with a particular form of Leber congenital amaurosis type 10.
Newco Breye Therapeutics ApS is poised to repurpose an intravenously administered drug that failed in ischemia reperfusion injury as an orally available treatment for diabetic retinopathy, after closing a €4 million (US$4.1 million) seed round.
Shares of Oyster Point Pharma Inc. (NASDAQ:OYST) soared to $11.57, or 38.6% on word of the buyout by Viatris Inc., which is also taking over privately held, development-stage Famy Life Sciences Ltd. to create an ophthalmology franchise that Viatris estimates could mean at least $1 billion in sales by 2028 as well as at least $500 million more in adjusted EBITDA.
“Big genomics” specialist Replay Holdings LLC has unveiled the first of four satellite genomic medicine companies it is forming to apply its high capacity herpes simplex viral (HSV) vector to next generation gene therapies. Eudora Therapeutics will specialize in inherited retinal eye diseases. It arrives on the scene with programs targeted at retinitis pigmentosa, Stargardt disease and Usher syndrome type 1B.
With roughly a decade of work behind it, Valitor Inc. is ready to put its Multivalent Polymer (MVP) platform to the test, having recently announced its presence with a $28 million series B round and appointment of biopharma veteran Steven Lo as CEO. The next step is IND-enabling studies for its lead ophthalmology candidate, with clinical testing slated to start in 2024.
Eye diseases specialist Oculis SA is set to add $200 million to its balance sheet by merging with the European Biotech Acquisition Corp., a special purpose acquisition company (SPAC) formed to invest in the European life sciences industry.
Over the next two weeks, the team at NGM Biopharmaceuticals Inc. will be digging through the data from its phase II trial of complement 3 inhibitor NGM-621 in patients with geographic atrophy (GA) secondary to age-related macular degeneration, following reports of a top-line miss Oct. 17 and a severe reaction from investors, who sent shares (NASDAQ:NGM) plummeting 70.5% to close at $3.41.