Enrollment Fills Early for Phase 3 Trial of ALS Drug Candidate Arimoclomol

According to a press release from the Danish biopharmaceutical company Orphazyme, the Company has filled target enrollment for its phase 3 clinical study of arimoclomol in patients with amyotrophic lateral sclerosis.

About Amyotrophic Lateral Sclerosis

You’ve probably heard of amyotrophic lateral sclerosis (ALS), but you might not know much about it. Despite what many people think, ALS is technically a group of conditions that chiefly involve the progressive damaging and wasting of nerve cells in the brain (called neurons) that are responsible for controlling voluntary muscle movement.

ALS is characterized by the progressive degeneration of the upper and lower motor neurons. Voluntary motion is triggered when upper motor neurons in the brain communicate with lower motor neurons — similar nerve cells found in the spinal cord. These lower motor neurons then stimulate muscle groups directly, acting out the original signal sent by the brain. As these nerve cells degenerate, the brain struggles to effectively communicate with lower motor neurons and, subsequently, the rest of the body. What may begin with muscle cramping and general weakness eventually progresses to full-body paralysis — with patients no longer able to swallow or even breathe without assistance.

ALS is exceptionally rare — only affecting an estimated 14,000-15,000 Americans every year. However, the disease is universally devastating — there is no cure, and the few treatments that are available for ALS patients are used only to alleviate the worst of the symptoms.

Enrollment in Phase 3 Trial Complete

The phase 3 trial of arimoclomol filled to capacity ahead of schedule. 213 participants will randomly receive either arimoclomol or a placebo (the groups sized 2:1, respectively) for a 76-week period. Additional space has been allotted for up to 18 individuals who are treated with edaravone — one of the few drugs currently used to treat the symptoms of ALS. Participants who remain with the study through the end of the 76th week will be invited to participate in an open-label extension trial.

Because enrollment was completed early, headline results from the study may be published as soon as early 2021. Calaneet Balas, President and CEO of the ALS Association, highlighted the desperate need for more ALS-targeted clinical trials. “We urgently need new treatments for ALS and we look forward to the results,” she said.


ALS is extremely challenging to formulate treatments for. A phase 3 trial can make or break a drug candidate — are you excited or nervous about the results of the upcoming clinical trial? Share your thoughts with Patient Worthy!

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