It took resolving issues from two complete response letters for Fennec Pharmaceuticals Inc. to finally land U.S. FDA approval of Pedmark. The injectable formulation of sodium thiosulfate is for reducing the risk of platinum-induced ototoxicity associated with cisplatin in pediatric patients 1 month and older with localized, non-metastatic solid tumors. Approval of the NDA arrived Sept. 20, three days before its Sept. 23 PDUFA date.
Ocular gene therapy firm Sparingvision SA raised €75 million (US$75 million) in a series B round to fund its transition to clinical development. The company is about to move its lead program, the mutation-agnostic gene therapy SPVN-06, into a phase I trial in retinitis pigmentosa (RP). “We are in the middle of the regulatory submission process,” CEO Stéphane Boissel told BioWorld.
Success in two pivotal studies of high dose Eylea (aflibercept) has significantly strengthened Regeneron Pharmaceuticals Inc.’s market position. But the company will face stiff competition from biosimilars and Roche Holding Group’s recently approved Vabysmo. After struggling for much of the year, the Tarrytown, N.Y.-based company’s stock (NASDAQ: REGN) rose about 20% this week. It closed 2% upward on Sept. 9 at $724.32 per share.
Questioners probed the fine points during Iveric Bio Inc.’s conference call, but news proved unmistakably good with Zimura (avacincaptad pegol) in geographic atrophy (GA), and shares of the firm (NASDAQ:ISEE) closed Sept. 6 at $15.70, up $6.26, or 66%, on positive top-line data from Gather 2, the firm’s second phase III study.
Taiwan’s Brim Biotechnology Inc. announced a capital raise of $18 million to advance its lead candidate, regenerative peptide therapy BRM-421, to phase III trials for dry eye syndrome. It’s designed to offer relief from dry eye symptoms by stimulating proliferation and differentiation of stem cells to repair damage to the cornea. “Our goal is restoration and repair of the damage caused by DES,” said Brim founder and CEO Haishan Jang.
Ophthalmic therapy and device developer Alcon SA, of Geneva, further strengthened its portfolio by buying Aerie Pharmaceuticals Inc. for about $770 million in equity. Aerie’s financial guidance for its glaucoma franchise puts net product revenue at $130 million to $140 million for all of 2022.
Opthea Ltd. has secured nondilutive funding to complete phase III development and commence commercialization of its wet age-related macular degeneration (wet AMD) treatment OPT-302, in a $170 million agreement with Launch Therapeutics, an operating company set up earlier this year by the private equity group Carlyle to manage biotech investments. Under the terms of the deal, Launch will now commit $120 million in three instalments at fixed time points, with an option to invest a further $50 million.
Positive data from Unity Biotechnology Inc.’s phase II study of UBX-1325, a senolytic BCL-XL inhibitor for treating diabetic macular edema, boosted the stock out of the penny range. Shares of the South San Francisco-based company’s stock (NASDAQ:UBX) rose sharply, 54% on Aug. 12, to $1.31 each.
The phase III miss disclosed Aug. 11 by Kubota Pharmaceutical Holdings Co. Ltd. subsidiary Kubota Vision Inc. in Stargardt disease put more eyes on the rare, inherited, juvenile-onset form of macular degeneration, for which nothing is approved.
Researchers are closer to better diagnosing and treating age-related macular degeneration (AMD) after discovering new genetic signatures of the disease by reprogramming stem cells to generate high-resolution disease models.
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