Report on Telbermin (DB12639): A Comprehensive Review of its Development for Diabetic Foot Ulcers and Amyotrophic Lateral Sclerosis

Abstract

Telbermin (DrugBank ID: DB12639), a recombinant human vascular endothelial growth factor A165 (rhVEGF-A165), is a biotech therapeutic agent investigated for its potential in promoting angiogenesis and neuroprotection. This report provides a comprehensive review of Telbermin's development, focusing on its clinical trials for diabetic foot ulcers (DFU) and amyotrophic lateral sclerosis (ALS). As a VEGF mimetic, Telbermin was hypothesized to stimulate new blood vessel formation, crucial for wound healing in DFU, and to exert neurotrophic effects, potentially slowing motor neuron degeneration in ALS. Clinical development for DFU, primarily led by Genentech, showed promising trends in early Phase I trials regarding wound healing and a good local safety profile with topical administration. However, subsequent, presumably Phase II, trials failed to meet the U.S. Food and Drug Administration (FDA)-accepted primary efficacy endpoints for complete wound closure, leading to the discontinuation of its development for this indication.[1] For ALS, Telbermin, also known as sNN0029 and developed by Newron Pharmaceuticals, was administered via intracerebroventricular (ICV) infusion. Early Phase I studies demonstrated that this delivery route achieved detectable drug levels in the cerebrospinal fluid and showed preliminary signals of potential benefit, alongside an FDA Orphan Drug Designation.[2] However, the ALS program was prematurely terminated in October 2015 due to critical issues with the third-party supplier of the investigational drug delivery catheter, rather than concerns about the drug's intrinsic efficacy or safety at that stage of development.[5] This report synthesizes the available data on Telbermin's identifiers, mechanism of action, clinical trial designs, efficacy and safety outcomes in DFU and ALS, and the dist