In silico screening with medicinal chemistry
to create new small molecule drugs

PTX technology

Neurotrophin receptors on the surface of neurons are powerful regulators of signaling networks within
neurons that determine whether a synapse or neuron survives or degenerates. These same signaling networks
are affected in AD and other neurological disorders. The Longo and Massa Laboratories at UCSF and UNC,
with peer-reviewed funding from the Alzheimer's Drug Discovery Foundation (ADDF), Alzheimer's Association
and the National Institutes of Aging (NIH) pioneered the development of compounds that bind to these
receptors to prevent degenerative signaling and promote regenerative signaling. Compounds have been
developed that target various combinations receptor targets. Examples include drugs that target the p75 or
TrkB receptor individually and drugs that target the TrkB and TrkC receptors combined. PTX has the exclusive
license to these compounds and has developed them in the commercial context including the establishment
of extensive intellectual property. Through a combination of licensed patents and novel chemistry executed
by PTX, the company has two development programs: p75 receptor program and Trk ligand program. Each
program is targeting a spectrum of neurological and non-neurological disorders.

Modulation of p75 has shown efficacy in mouse models of AD, traumatic brain injury, spinal cord injury and
chemotherapy-induced neuropathy. Compounds directed to TrkB have shown efficacy in mouse models of
Rett syndrome, Huntington's disease, post-traumatic epilepsy, multiple sclerosis, stroke rehabilitation,
anterior-ischemic-optic neuropathy and traumatic brain injury. Targeting of the TrkB-TrkC receptors has
shown improved outcomes in mouse models of Alzheimer's and Parkinson's disease and in
Charcot-Marie-Tooth disease, the most common form of inherited peripheral neuropathy.