Michael Hayden, PhD, CEO of Prilenia, discusses recent clinical trials testing pridopidine for the treatment of amyotrophic lateral sclerosis (ALS) and Huntington’s disease.

 

 

ALS, also referred to as Lou Gehrig’s disease, is a motor neuron disease which leads to problems with muscle control and movement. There are various types of ALS that are distinguished by symptoms and, in some cases, genetic cause. Early symptoms may include:

  • Muscle twitching
  • Cramping
  • Stiffness
  • Weakness
  • Slurred speech
  • Difficulty chewing or swallowing

 

As the disease progresses, people may become weaker and are eventually wheelchair-dependent. Most people with ALS have a sporadic, as opposed to inherited, form of ALS. It is believed that these cases are caused by an interaction between genetic and environmental factors. 

Huntington’s disease is an inherited condition that causes progressive degeneration of neurons in the brain. It is caused by changes in the HTT gene and is inherited in an autosomal dominant manner.

Pridopidine is an oral investigational sigma-1 receptor (S1R) agonist. The S1R protein is responsible for the regulation of brain and spinal cord processes, which are often affected by neurodegenerative diseases such as ALS and Huntington’s disease. Pridopidine stimulates pathways that enhance autophagy, axonal transport, mitochondrial energy production and respiration, and restores calcium homeostasis.

Clinical Trials

The phase 2 HEALEY trial for ALS results presented with a tolerable safety profile. While the primary endpoint of change from baseline through 24 weeks in Full Analysis Set was not met, significant improvements were observed in participants’ speech through their speaking and articulation rate. Positive results were also observed in multiple disease progression measures and survival time.

The phase 3 PROOF-HD trial for Huntington’s disease results were similar in that a tolerable safety profile was observed. The primary endpoint of change from baseline to 65 weeks in Total Functional Capacity was not met. However, improvements were observed in composite Unified Huntington’s Disease Rating Scale, motor function, cognition, functional capacity, and quality of life. 

A phase 3 study testing pridopidine in ALS is set for later this year.

 

To learn more about rare neurological diseases, visit https://checkrare.com/diseases/neurology-nervous-system-diseases/