What does it mean for a confirmatory trial to be “underway”? That’s a question that’s been plaguing some drug sponsors, especially those in the ultra-rare disease space, since the U.S. Congress, in 2023, gave the FDA the authority to require that a confirmatory trial be underway at the time accelerated approval is granted.
Proper survival and regeneration of retinal ganglion cells (RGCs), which constitute the optic nerve, is crucial for managing traumatic or ischemic optic nerve injury, optic neuropathy, or optic pathway gliomas. Researchers from Wenzhou Medical University described the synthesis of novel spirocyclic chalcone derivatives designed to combat optic nerve damage.
Ophthalmic diseases specialist Oculis Holding AG announced positive phase II data showing its serum-glucose corticoid kinase-2 activator OCS-05 protected nerve cells and improved vision in patients with acute optic neuritis. The placebo-controlled, randomized trial assessed the neuroprotective effect of OCS-05 in combination with standard-of-care steroid injections, against steroids alone, with a benefit seen in preserving both ganglions and retinal cell nerve fibers.
Scientists at Admare Bioinnovations Inc., Kingston General Hospital (KGH) and Queen’s University have described cytochrome P450 26B1 (CYP26B1) inhibitors reported to be useful for the treatment of acne, dry eye, meibomian glands dysfunction, fibrodysplasia ossificans progressive, heterotopic ossification (HO) and multiple osteochondroma.
Sigma-2 receptor modulation using exogenous ligands reduces cellular damage associated with central nervous system degeneration in age-related pathologies such as Alzheimer’s disease, Parkinson’s disease or dry age-related macular degeneration. Researchers from Cognition Therapeutics Inc. described the neuroprotective effect of CT-2074, a sigma-2 receptor in preclinical models of open-angle glaucoma.
Certa Therapeutics Pty Ltd. has acquired Occurx Pty Ltd. in a move to strengthen its pipeline to target multiple fibrotic diseases as both companies share a focus on targeting GPR68, a defined G protein-coupled receptor (GPCR) receptor that mediates signaling pathways associated with inflammation and fibrosis and is thought to be a master switch of fibrosis.
Certa Therapeutics Pty Ltd. has acquired Occurx Pty Ltd. in a move to strengthen its pipeline to target multiple fibrotic diseases as both companies share a focus on targeting GPR68, a defined G protein-coupled receptor (GPCR) receptor that mediates signaling pathways associated with inflammation and fibrosis and is thought to be a master switch of fibrosis.
Vigeneron GmbH has received IND approval from the FDA for VG-801, a novel mRNA trans-splicing gene therapy to treat Stargardt disease and other retinal dystrophies associated with mutations in the ABCA4 gene. IND clearance enables initiation of a phase I/II trial, with a clinical trial application (CTA) submission to the EMA also planned in the coming months.
Lento Bio Inc. has synthesized cysteine derivatives reported to be useful for the treatment of presbyopia. An exemplified compound (Cpd in row 4 in tbl1 pg 20, claim 18) decreased in lens stiffness in C57BL/6 mice (at 20 mM; as eye drops).
Splicebio S.L. has gained IND clearance from the FDA for its lead program SB-007, a protein splicing gene therapy to address the root genetic cause of Stargardt disease.
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