Regulatory Roundup FINAL

May 9, 2025 - Regulatory Roundup Updated every Friday

The Food and Drug Administration granted Regenerative Medicine Advanced Therapy (RMAT) designation to the following two companies and candidates:

  • 4D Molecular Therapeutics’ 4D-150 for the treatment of diabetic macular edema. 4D-150 is a potential backbone therapy that is designed to provide multiyear sustained delivery of the anti–vascular endothelial growth factor (VEGF) aflibercept and anti-VEGF-C from the retina with a single intravitreal injection. 4D-150 is also being developed for neovascular age-related macular degeneration.
  • Opus Genetics’ OPGx-LCA5, its investigational gene therapy for Leber congenital amaurosis (LCA) due to genetic variations in the LCA5 gene. Opus has also been invited to participate in the FDA’s Initial Comprehensive Multidisciplinary RMAT Meeting to support Opus’ development and manufacturing strategy. Lastly, Opus has been invited to join the FDA’s Chemistry, Manufacturing, and Controls Development and Readiness Pilot program, which provides additional guidance for accelerating product development under an investigational new drug application.

RMAT designation was created in the 21st Century Cures Act to expedite review of regenerative medicine therapies.

The Food and Drug Administration granted Fast Track designation to urcosimod, formerly called OK-101, OKYO Pharma’s drug candidate for the treatment of neuropathic corneal pain.

Clinical Observations - Square - Edited

May 9, 2025 - Clinical Observations Updated every Friday

Galimedix Therapeutics reports completion of the single ascending-dose part of the Phase I study of oral GAL-101, a small-molecule specifically designed to target misfolded amyloid beta monomers in Alzheimer’s disease, dry age-related macular degeneration (AMD), and glaucoma. The results showed that GAL-101 was well tolerated with a favorable safety profile, Galimedix reports. Galimedix also is conducting a Phase II trial with GAL-101 eyedrops for dry AMD.

The following trial readouts were reported at Association for Research in Vision and Ophthalmology (ARVO 2025) Annual Meeting.

  • Biopharmaceutical company Perfuse Therapeutics has reported 24-week results from its completed Phase I/IIa trial of PER-001, an endothelin antagonist intravitreal implant for glaucoma. The results demonstrated clinically meaningful increases in optic nerve head blood flow, and improved vision.
  • Isarna Therapeutics presented final results from its Phase II BETTER trial of ISTH0036, a selective TGF-β2-blocking antisense oligonucleotide, in patients with neovascular age-related macular degeneration (nAMD) and diabetic macular edema (DME). Patients who received intravitreal injections of ISTH0036 every eight weeks had stable or improved best-corrected visual acuity, along with anatomical improvements in the retina.
  • Opus Genetics announced one-year results from adults treated in the Phase I/II trial of OPGx-LCA5, its lead gene therapy candidate for treatment of Leber congenital amaurosis 5, a potentially blinding inherited retinal degeneration. Preliminary data showed gains in cone-mediated vision at one year. The results were from three adult patients (aged 19 to 34 years old), all of whom received subretinal injections in a single eye of up to 300 µl of low-dose (1x1010 vector genome per eye) OPGx-LCA5. Among outcomes, visual acuity improvements were equivalent to a 3.5-line gain across the three participants. Two additional adolescent patients have now also been treated.
  • Beacon Therapeutics reported six-month interim safety and efficacy results from the Phase II DAWN trial of its lead program, laru-zova (laruparetigene zovaparvovec), in patients with X-linked retinitis pigmentosa, an inherited retinal disease often caused by mutations to the RPGR gene. The data showed nonserious ocular treatment-emergent adverse events, early improvements in low-luminance visual acuity, and early and sustained improvements in mean sensitivity in study eyes, as observed by microperimetry.

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Rich Kirkner

Rich Kirkner

Contributing Editor
rkirkner@ois.net

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