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PharmatrophiX

Frank Longo, MD, PhD | California, United States

PharmatrophiX

Frank Longo, MD, PhD | California, United States

Phase 1 Study for P75 Small Molecule Ligand

PharmatrophiX (PTX) is a virtual biotech company founded by Dr. Longo in 2005 and is focused on the development of small molecule ligands directed to neurotrophin receptors for the treatment of Alzheimer's disease and other neurological disorders. The test drug is a small molecule ligand of the p75 receptor that when administered orally once per day inhibited deleterious signaling, excess tau phosphorylation and neuronal and synaptic degeneration and cognitive deficits in three AD mouse models. In October 2012, the test drug received FDA approval for phase 1 testing in normal young and elderly subjects. This drug has never been tested in humans thus as per FDA guidance, Phase 1 testing will consist of young and elderly normal subjects in a PK and safety study. Phase 1 will consist of 6 single ascending dose (SAD) cohorts and 2 or 3 (depending on funding availability) multiple ascending dose (MAD) cohorts. The 5th and 6th SAD and each MAD cohort will consist of elderly subjects. The trial is designed to assess safety over various dose ranges and make possible an anticipated phase 2a study in which the drug will be tested early AD subjects over a dose range for its ability to affect various imaging biomarkers which will be indicative of potential effectiveness in AD therapy. The majority of phase 1 costs will be supported by private funding already raised by PTX and the goal of this application is to obtain ADDF support for the final key support that will make this trial possible.