Recently, clinical stage biotechnology company Prilenia Therapeutics B.V. (“Prilenia”) announced that the first patient has been enrolled in a Phase 2/3 clinical trial evaluating pridopidine for patients with amyotrophic lateral sclerosis (ALS). The HEALEY ALS Platform Trial is the first ever platform trial specifically centered around ALS.

Pridopidine

Pridopidine is an orally administered, selective small-molecule Sigma-1 Receptor (S1R) agonist. Normally, S1R plays a part in several bodily pathways that contribute to neurodegenerative disorders. Through activating and expressing S1R, Pridopidine may inhibit neurodegeneration and prompt protective measures. Beyond ALS, this therapy has also been evaluated for patients with Huntington’s disease.

The safety, efficacy, and tolerability of pridopidine is being tested within the HEALEY ALS Platform trial. In an article published in Clinical Trials, a platform trial is described as:

a clinical trial with a single master protocol in which multiple treatments are evaluated simultaneously. Adaptive platform designs offer flexible features such as dropping treatments for futility, declaring one or more treatments superior, or adding new treatments to be tested during the course of a trial.

In this case, the HEALEY ALS Platform trial is exploring somewhere around 30+ investigational treatments for ALS. Since there are currently only three approved treatments for ALS (Riluzole, edaravone, Nuedexta), this trial offers the potential to find new, effective, and more accessible therapies.

In particular, pridopidine is being evaluated through the Regimen D Pridopidine arm of the trial. During the trial, patients will receive a 45mg dose of the treatment.

Amyotrophic Lateral Sclerosis (ALS)

Also known as Lou Gehrig’s disease, amyotrophic lateral sclerosis (ALS) is a progressive neurological disease causing nerve cell death in the brain, brain stem, and spinal cord. The exact cause of the condition is unknown, though family history may play a role in a small number of cases. As nerve cells die, muscles begin to weaken. Many patients lose voluntary movement and control. Typically, ALS is fatal as the chest muscles weaken, causing difficulty breathing. ALS often affects males more than females, particularly white males between the ages of 60 and 69. Symptoms vary but may include:

• Depression and anxiety
• Frequent tripping and falling
• Muscle weakness and cramps
• Difficulty with small movements or everyday activities
• Poor posture
• Difficulty speaking and swallowing
• Changes in speech
• Inability to move the entire body