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ALS Drug Maker Gains $135M in New Funds

Man in wheelchair


20 July 2021. A developer of treatments for people with amyotrophic lateral sclerosis, or ALS, is raising $135 million in its third venture financing round. Amylyx Pharmaceuticals Inc. in Cambridge, Massachusetts says it plans to apply those funds to a late-stage clinical trial and prepare for regulatory approval of its lead product.

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 5,000 new cases reported each year.

Amylyx Pharmaceuticals’ lead therapy candidate is 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.

Slower decline and longer life extension

As reported in Science & Enterprise in September and October 2020, findings from a clinical trial show AMX0035 is shown to slow functional decline and extend survival in ALS patients compared to a placebo. The mid- and late-stage trial enrolled 137 patients diagnosed with ALS symptoms in the previous 18 months at 25 sites in the U.S. Participants were randomly assigned to receive AMX0035 or a placebo on a two-to-one basis for six months, 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.

In the September 2020 data published in New England Journal of Medicine, results show 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. The October results, published in the journal Muscle & Nerve, report on patients taking part in an extension of the original six-month study. Those findings show participants originally receiving AMX0035 lived for a median of 25 months, compared to 18.5 months for participants receiving the placebo, a difference of 6.5 months.

The new venture round, the company’s third, is raising $135 million for Amylyx, led by Viking Global Investors in Greenwich, Connecticut. Taking part in the round are new investors Bain Capital Life Sciences, Perceptive Advisors, Rock Springs Capital, Woodline Partners, Marshall Wace, Tybourne Capital Management, Verition Fund Management, aMoon Fund, and Falcon Edge. Existing investors, including Morningside Ventures, 683 Capital Management, Belinda Termeer and Polaris Founders Capital, also participated.

According to Crunchbase, Amylyx Pharmaceuticals raised $67.2 in earlier venture funding. The company says it plans to use the new funds for a late-stage clinical trial of AMX0035 to prepare for final regulatory approval. Amylyx says it already filed for AMX0035’s approval in Canada and plans to apply for European Medicines Agency approval before the end of the year.

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