Lianbio Co. Ltd. announced mixed phase III top-line results on Oct. 30 for its in-licensed Demodex blepharitis treatment called TP-03, with the U.S. FDA-approved eyedrop hitting just one co-primary endpoint in the Libra trial on Chinese patients.
Investors awaiting the announcement of a partnering deal for RASP modulator reproxalap ahead of the drug’s Nov. 23 PDUFA date were in for a rude awakening, as Aldeyra Therapeutics Inc. disclosed that the U.S. FDA had raised issues with the NDA, putting its imminent approval in dry eye disease in doubt. The update, disclosed in an SEC filing, sent shares (NASDAQ:ALDX) sinking 66%, or $3.60, to close Oct. 16 at $1.83.
The NDA for the formerly named Nyxol is now an open and shut case for Ocuphire Pharma Inc. and Viatris Inc. The U.S. FDA has approved Ryzumvi (phentolamine ophthalmic solution) for treating pharmacologically induced mydriasis, better known as dilating the pupil. Ryzumvi, a small-molecule eye drop, reduces the pupil’s diameter after it is dilated with adrenergic agonists such as phenylephrine or parasympatholytic agents such as tropicamide. The treatment reduces pupil size by acting on the iris dilator muscle.
Iveric Bio Inc.’s regulatory win Aug. 4 drew attention to the already hot eye-disease space, where intriguing new developments include the possibility of an oral therapy for Stargardt disease. Belite Bio Inc. in late July finished enrollment of a phase III study with once daily tinlarebant, a retinol binding protein 4 antagonist for Stargardt’s. Data from 90 adolescent subjects in the study called Dragon are due in mid-2024.
Galimedix Therapeutics Inc. plans to move its lead compound, GAL-101, into a phase IIa proof-of-concept trial next year, in order to test an intriguing hypothesis associated with certain retinal degeneration conditions. GAL-101 selectively binds misfolded amyloid beta species, and the company believes that this mechanism may benefit patients with the dry form of age-related macular degeneration or with glaucoma.
In its second big pharma deal to date, Shape Therapeutics Inc. drew Otsuka Pharmaceutical Co. Ltd. to the table in a potential $1.5 billion-plus collaboration initially aimed at developing gene therapies for ocular diseases. The multitarget agreement, which includes options for additional targets and tissue types, will combine Shape’s AI-driven adeno-associated virus (AAV) platform and Otsuka’s expertise in ophthalmology to develop intravitreally delivered AAV therapies.
After hitting the safety and efficacy endpoints in a pivotal study of wet age-related macular degeneration (AMD), Outlook Therapeutics Inc.’s CEO said he had not expected to receive a complete response letter (CRL) from the U.S. FDA. The BLA for ONS-5010 (bevacizumab-vikg) now is on hold, the company said, because the agency said chemistry, manufacturing, and controls management problems were getting in the way, along with “open observations from pre-approval manufacturing inspections, and a lack of substantial evidence.”
A nonsteroidal eye drop formulation of dazdotuftide, TRS-01 missed its late-stage primary endpoint for controlling inflammation in uveitis, but developer Tarsier Pharma Ltd. said post-hoc analysis showing a boosted benefit-risk profile over steroids may be a ray of hope.
Opthea Ltd. plans to raise AU$80 million (US$51.2 million) via a AU$10 million private placement and a AU$70 million entitlement offer to continue its pivotal phase III trials in wet age-related macular degeneration (wet AMD) for lead candidate OPT-302.
On the heels of the U.S. FDA clearance for Veopoz (pozelimab-bbfg) from Regeneron Pharmaceuticals Inc. to treat Chaple disease, the regulatory rollercoaster ride for the firm’s bid with higher-dose Eylea (aflibercept) ended with a green light for that compound as well. Eylea HD is indicated for wet age-related macular degeneration, diabetic macular edema, and diabetic retinopathy.
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