ALS, which is also known as Lou Gehrig’s Disease, is a progressive neurological disease that affects the nerve cells in the brain and spinal cord, which ultimately causes the complete loss of voluntary muscle control over time. The loss of muscle control typically begins in the hands and feet before progressing to the rest of the body.
According to the ALS Association, more than 5,000 people in the US are diagnosed with ALS every year. The average life expectancy is at least three years.
The pharmaceutical company’s CENTAUR study, which was done in conjunction with the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital in Boston, determined that its drug, AMX0035, showed a “statistically significant slowing of ALS disease progression.”
The 24-week clinical trial included the participation of some 137 subjects who had been diagnosed with ALS within 18 months of symptoms first appearing. Aged between 18 and 80, many of the patients had already developed problems such as difficulty with walking and speaking, as well as arm weakness.
In a news release, Amylyx noted that the trial saw a portion of subjects given the AMX0035 drug, while others were given a placebo in addition to ALS drugs that they had already been prescribed. The duration of the trial was six months.
“With these results, Amylyx now has a responsibility to move ahead as efficiently as possible, as people living with ALS don’t have time to wait,” Justin Klee, president and co-founder of Amylyx, said in a statement.
“We will work closely with the [US Food and Drug Administration (FDA)] and the ALS community, including the wonderful Northeast ALS Consortium leadership and member sites that conducted the CENTAUR trial, to decide next steps and will continue to keep everyone informed.”
Although the company has largely held off on releasing the details of the drug and the full analysis of its trial, Klee did explain to the Wall Street Journal in a Tuesday article that AMX0035 is a combination of two different ALS medications. Additionally, the Amylyx co-founder noted that the results of the experiment showed that the concoction had a larger impact on the neurodegenerative disease’s progression, rather than on specific ALS-caused complications.
Kristina Golji, one of the trial’s participants who received her ALS diagnosis in March 2017, told the Journal that prior to starting the study she had been noticing a gradual decline in her ability to speak; however, after starting on AMX0035, the progression of her symptoms began to decelerate.
“I believe my ALS slowed down somewhat during the trial,” Golji said, before noting that her symptoms quickly worsened after the trial wrapped and she was off the new medication. As part of an open label extension under Amylyx’s study, Golji was later able to continue using AMX0035.
At present, according to the ALS Association, there are four drug treatments approved by the FDA for the condition: Radicava, Riluteck, Tiglutik and Nuedexta. Although there is no cure for the disease, the drugs help prolong life expectancy by three to six months and slow the decline of muscle functions.
Amylyx is expected to publish the trial’s results in a medical journal and present them at a medical meeting in early 2020.