Addressing
neurodegenerative diseases

Alzheimer’s, other tauopathies, Huntington’s, and related disorders share a single fingerprint at the cellular level: synaptic failure.

Neurodegenerative diseases like Alzheimer’s arise from a complex “perfect storm” within the brain.

Multifactorial biology.

Most neurodegenerative conditions are not caused by a single glitch. Instead, they exhibit multifactorial biology — the result of many intersecting pathological pathways.

  • Protein misfolding — aggregates like amyloid-beta, tau or alpha-synuclein promote degeneration.
  • Inflammation — the brain’s immune cells (microglia) can become hyper-active, attacking normal structures including synapses.
  • Mitochondrial failure — the “power plants” of the cells stop producing energy, leading to oxidative stress.

Drugs that target only one of these factors often fail to stop the overall progression.

Progressive synapse loss.

There is often a focus on the death of the neuron “cell body”, but disease usually begins at the connections. Progressive synapse loss is frequently the earliest sign of trouble.

  • Synapses are the chemical bridges between neurons where communication happens.
  • In diseases like Alzheimer’s, synapses start disappearing long before the actual neuron dies.

By the time a patient shows noticeable memory loss or motor symptoms, synaptic connections have already begun to be “pruned” away.

The blood‑brain barrier.

Even when we develop a promising drug, getting it to the target is a logistical challenge. The blood-brain barrier is a highly selective “security fence” that protects the brain from toxins in the blood — but it also keeps out about 99% of potential small-molecule drugs, limiting delivery to the brain.

LM11A-31 effectively crosses the blood-brain barrier.

Our Role

Targeting the receptors that decide survival or promote degeneration.

PharmatrophiX’s disease-modifying small molecule technology targets receptors that regulate signaling networks determining whether neurons survive or degenerate. Neurotrophin receptors p75 and Trk participate in development, synaptic function, and survival of neurons and their synapses.

p75 receptor compounds that decrease degeneration of synapses and neurons. Ligand program develops compounds targeting TrkB — or TrkB and TrkC receptors together — to promote survival signaling.

Explore our Research & Development →