Policy & Regulation
Neuropore Receives Orphan Drug Designation for NPT520-34 for the Treatment of Amyotrophic Lateral Sclerosis
16 August 2019 - - US-based biopharmaceutical company Neuropore Therapies, Inc.  has received orphan drug designation for NPT520-34 for the treatment of amyotrophic lateral sclerosis or ALS, the company said.

The orphan drug designation will facilitate the development of NPT520-34 in the treatment of this debilitating medical condition for which limited treatments are available.

In addition, Neuropore reports the successful completion of the initial, single dose, safety and the food effect studies of NPT520-34 in healthy volunteers.

NPT520-34 is now being assessed in a multiple dose safety study designed to evaluate the safety, tolerability and pharmacokinetics of repeated doses of NPT520-34 in normal healthy volunteers at potentially therapeutically relevant exposures.

NPT520-34 is an orally bioavailable, blood-brain barrier penetrating small molecule. Its mode of action is to attenuate microglial and astroglial mediated neuroinflammation, resulting in reduced burden of neuropathic proteins such as superoxide dismutase-1, alpha-synuclein and beta-amyloid in animal models of neurodegenerative disorders such as ALS, Parkinson's disease and Alzheimer's disease, respectively.

Neuropore is currently pursuing NPT520-34 for the treatment of both ALS and Parkinson's disease.

NPT520-34 is a clinical stage orally bioavailable small molecule that reduces astrocytic and microglial markers of neuroinflammation with robust beneficial effects on neuropathology and motor function in animal models of Parkinson's disease.

NPT520-34 also has been shown to reduce the expression of markers of neuroinflammation and neuropathology in animal models of amyotrophic lateral sclerosis and Alzheimer's disease.

NPT520-34 is currently being evaluated for safety, pharmacokinetics and target engagement in Phase 1 clinical studies in healthy volunteers.

Biomarker-based proof-of-mechanism studies in patients with neurodegenerative disorders are scheduled to start in 2020.

Neuropore Therapies Inc. is a San Diego, California-based biopharmaceutical company developing novel disease modifying small molecule therapeutics for the treatment of neurodegenerative disorders.