Late yesterday evening, Amylyx Pharmaceuticals Inc. (AMLX:NASDAQ), which is focused on the development of therapeutics for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, announced that "the U.S. Food and Drug Administration's (FDA) Peripheral and Central Nervous System Drugs Advisory Committee (PCNSDAC) voted (seven yes votes and two no votes) that the available evidence of effectiveness is sufficient to support approval of AMX0035 (sodium phenylbutyrate and taurursodiol [also known as ursodoxicoltaurine]) for the treatment of amyotrophic lateral sclerosis (ALS)."
The company has completed U.S. enrollment and is now actively recruiting participants in Europe for its 48-week global Phase 3 PHOENIX clinical trial. The primary objective of the randomized study is to further investigate the safety and efficacy of AMX0035 in treating ALS. The study will focus on ALSFRS-R total score progression and overall survival over 48 weeks. The company advised that initial results from the trial are expected in 2024.
The company noted that the decision by the PCNSDAC committee took into account all of the available data submitted to the review panel through September 7, 2022, including the information that was presented previously at the PCNS meeting held on March 30, 2022.
Amylyx Pharmaceuticals' Head of Scientific Communications, Jamie Timmons, M.D., stated, "The Committee's thoughtful review of the data and support of the benefit that AMX0035 may bring to the ALS community if approved is promising … The CENTAUR trial data has consistently demonstrated potential benefits of AMX0035 on function and overall survival."
The company explained that ALS is a neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. The disease causes deteriorating muscle function, which progresses to the inability to move and speak, respiratory paralysis, and, ultimately, death.
Amylyx mentioned that even though the PCNSDAC recommendations are not binding, they are expected to serve as an integral part of the FDA's overall review and approval process for the company's pending New Drug Application (NDA) for AMX0035. The firm advised that the FDA granted Priority Review for AX0035 in December 2021 and noted that the current target action date for the FDA's ruling on the NDA is scheduled for September 29, 2022.
The firm indicated that AX0035 is an oral medication with marketing applications pending in the U.S. and the EU. The company added that the drug had been approved by Health Canada under the name ALBRIOZA™ for use in treating ALS.
Amylyx highlighted that if the FDA grants final approval for AMX0035, "it will be the first treatment in ALS that has demonstrated a significant slowing of disease progression and functional decline, as well as extended survival, in a randomized, placebo-controlled clinical trial, as a standalone therapy or when added to existing approved treatments."
Leading up to today's announcement, AMX0035 has been evaluated in several clinical studies, including the multicenter Phase 2 CENTAUR clinical trial, which enrolled 137 patient subjects diagnosed with ALS. The study met its primary efficacy endpoint and demonstrated that AMX0035, together with standard of care, significantly slowed the rate of functional decline as measured by the ALS rating scale (ALSFRS-R) total score versus placebo and standard of care after 24 weeks.
The company pointed out that "the CENTAUR trial was funded, in part, by the ALS ACT grant and the ALS Ice Bucket Challenge, and was supported by The ALS Association, ALS Finding a Cure (a program of The Leandro P. Rizzuto Foundation), the Northeast ALS Consortium, and the Sean M. Healey & AMG Center for ALS at Mass General."
The company has completed U.S. enrollment and is now actively recruiting European participants for its 48-week global Phase 3 PHOENIX clinical trial. The primary objective of the randomized study is to further investigate the safety and efficacy of AMX0035 in treating ALS. The study will focus on ALSFRS-R total score progression and overall survival over 48 weeks. The company advised that initial results from the trial are expected in 2024.
Amylyx is a pharmaceutical firm headquartered in Cambridge, Mass., that concentrates its work on developing new potential therapies for treating ALS and other neurodegenerative diseases.
The firm endeavors to work closely with patients, their families, and the healthcare professionals who support them. The company's primary product pipeline asset is AMX0035, a dual UPR-Bax apoptosis inhibitor composed of sodium phenylbutyrate and taurursodiol currently being developed as an ALS treatment.
Amylyx Pharma started off the day with a market cap of around $1.05 billion with approximately 58.53 million shares outstanding and a short interest of about 5.8%. AMLX shares opened 75% higher today at $31.30 (+$13.40, +74.86%) over yesterday's $17.90 closing price. The stock traded today between $27.00 and $32.80 per share and closed for trading at $27.03 (+$9.13, +51.01%).
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