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Amylyx Pharmaceuticals' New Drug for ALS Makes Progress With the FDA

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Jan. 5 2022, Published 1:00 p.m. ET

Amylyx is a Cambridge-based pharmaceutical company that seeks to develop new therapies for ALS (amyotrophic lateral sclerosis), Alzheimer’s disease, and other neurodegenerative diseases. The company's latest endeavor involved submitting an NDA (New Drug Application) to the FDA while announcing its IPO.

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On September 15, 2021, Amylyx announced that it would be submitting an NDA to the FDA for its AMX0035 drug. The drug will be used to treat ALS. Where does AMX0035 stand in its review currently?

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Doctor reaches for pill bottle

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Amylyx is focusing on AMX0035 trials and its IPO.

On December 29, 2021, Amylyx announced that the FDA accepted the NDA. The FDA reportedly assigned a target date for completion of the review on June 29, 2022. AMX0035 was submitted to the FDA based on findings from the phase 2 CENTAUR trial, which evaluated 137 people with ALS. People who received AMX0035 exhibited a reduction in clinical decline by the end of the trial.

AMX0035 is composed of two agents, taurursodiol (TURSO or ursodoxicoltaurine) and sodium phenylbutyrate (PB). Both agents were combined in an effort to reduce the death of neurons and reduce neuronal dysfunction. The drug will target the neuronal pathways and endoplasmic reticulum for ALS and other diseases that are neurodegenerative.

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AMX0035 shows promise in trials.

The decline, or lack thereof, is measured by the Revised ALS Functioning Rating Scale, which is used in clinics globally to measure ALS function. By the end of the trial, Amylyx reportedly has marketing applications under review for AMX0035 in the U.S. and Canada for the phase 3 PHOENIX trial. Phase 3 will aim to focus on efficacy and additional safety for regulatory purposes. Phase 3 will enroll 600 patients with “definite or clinically probably ALS and within 24 months from symptom onset.”

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On Jan. 3, Amylyx announced its IPO of 8,750,000 shares between $18.00 and $20.00 per share. It will be listed on the Nasdaq under the ticker symbol “AMLX.” The company anticipates a 30-day option for underwriters to purchase 1,312,500 additional shares of stock at the IPO price. Amylyx expects to raise $175 million. The company was valued at $580 million in 2020 by Pitchbook data. The company has also received backing from investors like Viking Global and Morningside.

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ALS Association tweets support of AMX0035

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AMX0035 is being tested for ALS and Alzheimer's disease.

A separate clinical study was also done to test the effectiveness of AMX0035 as an Alzheimer’s treatment. The trial enrolled 95 people and went from August 27, 2018, to August 15, 2021. The study investigated the effects of the drug for “late mild cognitive impairment or early dementia due to Alzheimer’s disease.” The results from the trial show that the drug had a significant impact on biomarkers for Alzheimer's disease.

Dr. Steven E. Arnold, a Harvard professor and the Translational Neurology Head of the Interdisciplinary Brain Center at Massachusetts General Hospital commented on the trial results. Dr. Arnold said that he was encouraged by the critical biomarker changes and he anticipates using the data for further investigation of the drugs potential and possible effects on long-term cognition.

Joshua Cohen, the Co-CEO, Chairman, and Co-Founder of Amylyx Pharmaceuticals commented on the submission. He said, “We are thrilled to move toward the U.S. submission of an NDA for AMX0035 and look forward to continuing to work with the FDA.” Justin Klee, who's also a Co-CEO weighed in on the ALS treatment. He said that since ALS is a time-sensitive illness, Amylyx’s priority is getting the drug through clinical development as efficiently as possible.

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