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Trial Shows Some ALS Drug Benefit for Patients

Man in wheelchair

(PXHere)

3 Sept. 2020. Results from a clinical trial show a drug to treat amyotrophic lateral sclerosis slows the functional decline in ALS patients compared to a placebo. Findings from the trial testing the drug code-named AMX0035 developed by Amylyx Pharmaceuticals Inc. in Cambridge, Massachusetts appear in today’s issue of New England Journal of Medicine (subscription required).

ALS, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disorder where neurons or nerve cells controlling muscles in the body begin to waste away, and can no longer send or receive signals from the brain or spinal cord. As the nerve cells stop functioning, muscles in the limbs, and later speech and breathing muscles, begin weakening and eventually stop functioning. Most people with the disease die of respiratory failure. According to Johns Hopkins University, ALS affects some 30,000 people in the U.S., with 6,000 new cases reported each year.

Amylyx Pharmaceuticals is developing AMX0035 that aims to slow the disease’s progression by targeting two sources of stress on nerve cells believed to contribute to cell death and inflammation associated with ALS. One stress source is the mitochondria or energy center of the cell, while the second source is the endoplasmic reticulum, a network of sacs and tubes that takes up much of the internal space of the cell, and transfers molecules between the nucleus and the rest of the cell. The company says both of these stress pathways need to be addressed to treat neurodegenerative diseases such as ALS.

AMX0035, says Amylyx, is made of two approved compounds that together work on both of the stress sources: phenylbutyrate for mitochondrial stress and tauroursodeoxycholic acid for endoplasmic reticulum.  Both compounds were tested individually with ALS patients earlier, but AMX0035 is designed to combine and optimize their effects.

The mid- and late-stage clinical trial enrolled 137 participants diagnosed with ALS symptoms in the previous 18 months, from an initial screening of 177 patients. Participants at the 25 sites in the U.S. were randomly assigned to receive AMX0035 or a placebo on a two-to-one basis, administered once a day for three weeks, then twice a day for 21 weeks. The study team looked primarily for changes in participants’ scores on a standard 48-point rating scale of muscle activity and day-to-day functions among ALS patients. Participants are also rated on isometric limb strength, lung capacity, and chemical and imaging biomarkers of nerve cell death and integrity.

The results show functional ratings for the 89 participants receiving AMX0035 declined at slower pace, 1.24 points per month, than the 1.66 points per month for the 48 participants receiving the placebo, a difference large enough for statistical reliability. While AMX0035 recipients scored higher on many of the trial’s secondary measures, differences with placebo recipients were not large enough to be considered reliable.

The company says AMX0035 was well tolerated, but nearly all participants in the trial — 97 percent of AMX0035 and 96 percent of placebo recipients — experienced some adverse affects. Among AMX0035 recipients, adverse effects were gastrointestinal, including diarrhea, nausea, salivary secretions, and abdominal discomfort. However, serious adverse effects occurred more often among placebo recipients. About one in five AMX0035 recipients (19%) discontinued the trial early compared to 8 percent of placebo recipients.

“The data published today,” says Calaneet Balas, president of The ALS Association in an Amylyx statement, “makes a clear and compelling case that AMX0035 should be made available to people with ALS as soon as possible. We look forward to working with Amylyx, the FDA, and the entire community to help make that happen.” ALS Association is a co-sponsor of the clinical trial.

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