ABOUT
ALS
ALS, also known as Lou Gehrig’s Disease or Motor Neuron Disease, is a chronic progressive neurodegenerative disease that affects people worldwide, with approximately 20,000 in the U.S. alone. The majority of ALS cases (~90%) are sporadic. However, ~10% of ALS cases are caused by genetic mutations.
Several genes have been discovered that are causative for ALS, including the gene encoding the sigma-1 receptor (S1R).
In patients with ALS, motor neurons in the brain and spinal cord that convey messages to the muscles degenerate, affecting the brain’s ability to communicate with muscles.
This leads to muscle wasting and progressive paralysis. Patients rapidly lose their ability to walk, speak, eat and breathe and become fully dependent on their caretakers.
ALS is a devastating disease, for patients, their families and caretakers.
Pridopidine for ALS
Pridopidine was assessed in the HEALEY ALS platform trial for the treatment of ALS.
In February 2023, Prilenia announced initial results from the pridopidine arm of this Phase 2 study. These results provide valuable insights, which will inform future clinical development for pridopidine in ALS. Additional analyses are underway, including from the open-label extension study, and complete study results will be presented at upcoming scientific meetings.
Pridopidine is a promising drug candidate for the treatment of ALS for several reasons:
Pridopidine activates sigma 1 receptor and improves motor neurons survival and muscle function in animal models of ALS.
Recent data show that some individuals with decreased or loss of function in the sigma 1 receptor develop ALS.
Pridopidine is an oral drug with an established safety profile.
Pridopidine demonstrated potential clinical efficacy in maintaining total functional capacity in HD patients. Many aspects of functional capacity are shared between HD and ALS.
These findings highlight the therapeutic potential of pridopidine for treating ALS. In addition, Prilenia holds Orphan Drug Designation for pridopidine in ALS in both the U.S. and Europe.
Learn more about pridopidine for ALS:
