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ALS-205 (formerly known as PMX205) is an orally bioavailable small-molecule antagonist of the complement C5a receptor 1 (C5aR1), currently under development by Alsonex Pharmaceuticals. The drug is designed to inhibit complement C5a-mediated neuroinflammation, which is implicated in the progression of various neurodegenerative diseases. ALS-205 is a potent and selective antagonist of the human C5a1 receptor with no reported activity at the C5a2 receptor. It is lipophilic and capable of crossing the blood-brain barrier to access the central nervous system. ALS-205 has been granted Orphan Drug Designation by both the U.S. FDA and the European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND).
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