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Amylyx Pharmaceuticals Announces Outcome of FDA Advisory Committee Meeting on AMX0035 for the Treatment of ALS

Amylyx Pharmaceuticals Announces Outcome of FDA Advisory Committee Meeting on AMX0035 for the Treatment of ALS

CAMBRIDGE, Mass., March 30, 2022–(Company WIRE)–Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) introduced these days the outcome of the U.S. Food stuff and Drug Administration’s (Fda) Peripheral and Central Anxious Technique Medications Advisory Committee (PCNSDAC) conference to evaluation the Company’s New Drug Application (NDA) for AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO; also known as ursodoxicoltaurine]) for the procedure of amyotrophic lateral sclerosis (ALS).

On the dilemma, “Do the data from the solitary randomized, controlled demo and the open-label extension study [Phase 2 CENTAUR trial] create a conclusion that sodium phenylbutyrate/taurursodiol [AMX0035] is powerful in the treatment method of people with amyotrophic lateral sclerosis (ALS)?,” the PCNSDAC voted 4 (certainly) and 6 (no).

“We continue being assured in the facts from the Section 2 CENTAUR trial and the probable rewards of AMX0035 as a treatment method selection for folks residing with ALS,” reported Jamie Timmons, M.D., Head of Scientific Communications of Amylyx. “We are enthusiastic by the hundreds of men and women impacted by this devastating disorder who shared their own testimonies, both penned and spoken, in conjunction with today’s assembly. We are also encouraged by the pro ALS doctors who shared their clinical perspectives. Our software is less than overview by the Fda, and we remain dedicated to pursuing its approval offered the urgent need to have for new treatment plans for ALS.”

While the Food and drug administration considers the recommendations of its advisory committees, the recommendations by the panel are non-binding. The final choice with regards to acceptance of a pending NDA is produced by the Fda. As beforehand noted, the Fda has granted Precedence Assessment to the NDA for AMX0035 and is expected to make a decision on the approval of AMX0035 by June 29, 2022 under the Prescription Drug Person Cost Act.

ALS is a relentlessly progressive and deadly neurodegenerative condition brought on by motor neuron dying in the brain and spinal cord. Motor neuron reduction in ALS leads to deteriorating muscle mass function, the lack of ability to move and converse, respiratory paralysis, and finally death.

About AMX0035

AMX0035 is a proprietary oral mounted-dose blend of two smaller molecules: sodium phenylbutyrate (PB), which is a compact molecular chaperone developed to cut down the unfolded protein response (UPR), stopping cell loss of life ensuing from the UPR, and taurursodiol (TURSO also recognised as ursodoxicoltaurine), which is a Bax inhibitor intended to lower cell death by apoptosis. PB and TURSO were being mixed in a fixed-dose formulation in an hard work to cut down neuronal loss of life and dysfunction. AMX0035 is built to goal the endoplasmic reticulum and mitochondrial-dependent neuronal degeneration pathways in ALS and other neurodegenerative diseases.

About the CENTAUR Demo

CENTAUR was a multicenter Phase 2 clinical demo in 137 members with ALS encompassing a 6-thirty day period randomized placebo-controlled period and an open up-label lengthy-phrase observe-up section. The trial fulfilled its main efficacy endpoint of minimizing functional decrease as calculated by the ALS Purposeful Rating Scale-Revised (ALSFRS-R).

In general, reported rates of adverse activities and discontinuations ended up very similar concerning AMX0035 and placebo teams through the 24-week randomized period nevertheless, gastrointestinal gatherings occurred with higher frequency (≥2%) in the AMX0035 team. Detailed data from CENTAUR is published in the New England Journal of Drugs (NEJM) and Muscle mass and Nerve.

The CENTAUR demo was funded, in aspect, by the ALS ACT grant and the ALS Ice Bucket Challenge, and was supported by The ALS Association, ALS Getting a Treatment (a method of The Leandro P. Rizzuto Basis), the Northeast ALS Consortium, and the Sean M. Healey & AMG Centre for ALS at Mass Normal.

About Amylyx Pharmaceuticals

Amylyx Prescription drugs, Inc. is a medical-phase biopharmaceutical company functioning on producing a novel therapeutic for ALS and other neurodegenerative ailments. For far more info, stop by www.amylyx.com and abide by us on LinkedIn and Twitter. For traders please pay a visit to www.traders.amylyx.com

Forward-Wanting Statements

Statements contained in this press release about issues that are not historical facts are “ahead-searching statements” within the that means the of Private Securities Litigation Reform Act of 1995, as amended. For the reason that this sort of statements are topic to pitfalls and uncertainties, real effects might differ materially from those expressed or implied by these forward-on the lookout statements. These types of statements include, but are not confined to, statements with regards to the opportunity regulatory approval of AMX0035 and the likely of AMX0035 as a therapy for ALS and anticipations with regards to our for a longer time-time period strategy. Any ahead-on the lookout statements in this assertion are primarily based on management’s present-day expectations of foreseeable future gatherings and are matter to a amount of pitfalls and uncertainties that could bring about genuine final results to differ materially and adversely from those set forth in or implied by these kinds of forward-hunting statements. Dangers that add to the unsure character of the ahead-searching statements include: the good results, price tag, and timing of Amylyx’ method progress actions, Amylyx’ means to execute on its tactic, regulatory developments, anticipations concerning the timing of regulatory review of AMX0035, Amylyx’ capacity to fund functions, and the impact that the ongoing COVID-19 pandemic will have on Amylyx’ functions, as well as those people challenges and uncertainties established forth in Amylyx’ United States Securities and Exchange Commission filings. All ahead-seeking statements contained in this press release converse only as of the date on which they were being manufactured. Amylyx undertakes no obligation to update this kind of statements to replicate situations that occur or instances that exist immediately after the day on which they were being built.

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Contacts

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Becky Gohsler
Finn Associates
(646) 307-6307
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Traders
Lindsey Allen
Amylyx Prescribed drugs, Inc.
(857) 320-6244
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