ENDECE for Niemann-Pick Disease Type C (NPC1)

NDC-1308 (MC2) has a Dual Mechanism of Action (MOA) that may both slow the progression of Niemann-Pick disease type C (NPC1) and potentially reverse the functional losses associated with NPC1. Using a nebulizer, NDC-1308 (MC2) will be directly delivered to the central nervous system to reduce inflammation, and remyelinate patients with the two key pathologies that drive NPC1.
 

  1. Chronic Progressive Neuroinflammation
  2. Demyelination / Hypomyelination
NDC-1308

NPC1: A RARE PROGRESSIVE DISEASE

Niemann-Pick disease type C (NPC1) is a rare genetic disorder in which gene mutations are passed from parents to children in a pattern called autosomal recessive inheritance. Patients with NPC1 are unable to transport cholesterol and fatty substances (lipids) out of lysosomes causing an accumulation of these compounds leading to progressive and lethal neurodegeneration.

Niemann-Pick disease type C (NPC1) primarily affects children, but can occur at any age. Symptoms Include:

Clumsiness and difficulty walking

Excessive muscle contractions (dystonia) or eye movements

Sleep disturbances

Difficulty swallowing and eating

Recurrent Pneumonia

ENDECE SARS-CoV-2 COVID-19

HOW NDC-1308 (MC2) WORKS FOR NPC1

TARGET ONE:
Directly Polarizes Macrophages to the M2 Anti-inflammatory State

TARGET TWO:
Directly Induces Remyelination