AV-101

AV-101 is an investigational drug code name used by two different companies for two distinct drug formulations. One formulation, developed by **Vistagen Therapeutics**, is an oral prodrug known chemically as **4-Cl-KYN**. It targets the N-methyl-D-aspartate receptor (NMDAR), an ionotropic glutamate receptor in the brain, and is being investigated for neurological disorders including neuropathic pain, major depressive disorder (MDD), and other CNS disorders. Preclinical studies have shown antinociceptive effects similar to gabapentin with a better side effect profile. The U.S. FDA has granted Fast Track designation for this oral formulation as a potential non-opioid treatment for neuropathic pain and as an adjunctive treatment for MDD. The second formulation, developed by **Aerovate Therapeutics**, is an **inhaled dry powder formulation of imatinib** specifically for pulmonary arterial hypertension (PAH). This formulation is designed to deliver imatinib directly to the lungs to maximize potential clinical benefit while limiting systemic adverse effects. This inhaled formulation was evaluated in the IMPAHCT Phase 2b/3 trial for PAH. While the drug was well tolerated, the study did not meet its primary endpoint for improvement in pulmonary vascular resistance (PVR) compared to placebo.

Other names

inhaled imatinibdry powder inhaled imatinib