The double blind, randomized, placebo-controlled, two-center study published in Investigative Ophthalmology & Visual Science assessed the safety and efficacy of daily subcutaneous 1, 4, 8 mg cibinetide or placebo for 28 days in 64 patients with painful sarcoid neuropathy. Cibinetide met its pre-specified primary efficacy endpoint: to assess small nerve fiber loss and repair as quantified by corneal confocal microscopy (CCM). A significant increase in corneal nerve fiber area versus placebo was noted for cibinetide 4 mg dose, a treatment effect corresponding to ≈ 23% increase from baseline. Cibinetide nerve regenerative effects were also demonstrated by a significant increase in skin intraepidermal growth associated protein-43 nerve fiber length. Improved patient functional activity was correlated with the increase in nerve fibers, and clinically meaningful placebo-corrected decreases in pain intensity were noted in patients with moderate to severe pain at baseline. No significant safety issues were observed for any treatment group in the trial.
