Amylyx Pharmaceuticals Announces Posting of Briefing Documents for Second FDA Advisory Committee Meeting on AMX0035

Rhea-AI Summary

Amylyx Pharmaceuticals announced the reconvened meeting of the Peripheral and Central Nervous System Drugs Advisory Committee (PCNSDAC) by the FDA to review the New Drug Application (NDA) for AMX0035, a treatment for amyotrophic lateral sclerosis (ALS). Scheduled for September 7, 2022, this follows a previous meeting on March 30, 2022. The NDA's target action date is September 29, 2022, which was extended for further analysis of clinical data. AMX0035 is under regulatory review in the U.S. and EU, and is already approved in Canada as ALBRIOZA™.

Positive

• FDA reconvenes PCNSDAC meeting for AMX0035 review, indicating continued regulatory engagement.
• Target action date extended to September 29, 2022, allowing further review of clinical data.

Negative

• FDA's extension of the target action date could imply potential delays in approval.

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx or the “Company”) today announced that the U.S. Food and Drug Administration (FDA) posted briefing documents for the reconvened Peripheral and Central Nervous System Drugs Advisory Committee (PCNSDAC) meeting to review the New Drug Application (NDA) for AMX0035 (sodium phenylbutyrate and taurursodiol [also known as ursodoxicoltaurine]) for the treatment of amyotrophic lateral sclerosis (ALS).

The second meeting of the PCNSDAC for the AMX0035 NDA is scheduled for 12:00 p.m. ET on Wednesday, September 7, 2022. The briefing materials and webcast information can be accessed here. The Company is not responsible for the content of, nor the statements made in, the briefing materials that were prepared by the FDA.

The PCNSDAC previously met on March 30, 2022 to discuss the NDA for AMX0035 for the treatment of ALS. The Prescription Drug User Fee Act (PDUFA) target action date for the NDA is September 29, 2022, which was extended by the FDA to allow more time to review additional analyses of data from the Company’s clinical studies.

About AMX0035
AMX0035 (sodium phenylbutyrate and taurursodiol) is an oral fixed-dose medication with marketing applications pending in the United States and European Union and approved with conditions as ALBRIOZA™ to treat amyotrophic lateral sclerosis (ALS) in Canada. The combination of sodium phenylbutyrate and taurursodiol may reduce neuronal cell death, hypothesized to occur by simultaneously mitigating endoplasmic reticulum stress and mitochondrial dysfunction. AMX0035 is also being explored for the potential treatment of other neurodegenerative diseases.

About Amylyx Pharmaceuticals
Amylyx Pharmaceuticals, Inc. is committed to supporting and creating more moments for the neurodegenerative community through the discovery and development of innovative new treatments. Amylyx is headquartered in Cambridge, Massachusetts and has operations in Canada and EMEA. For more information, visit amylyx.com and follow us on LinkedIn and Twitter. For investors, please visit investors.amylyx.com.

Forward-Looking Statements
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding beliefs about the clinical study analyses of AMX0035, the potential regulatory approval of AMX0035 and the potential of AMX0035 or other future therapeutic candidates as a treatment for ALS and other neurodegenerative diseases. Any forward-looking statements in this statement are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include: the success, cost, and timing of Amylyx’ program development activities, Amylyx’ ability to execute on its strategy, regulatory developments, expectations regarding the timing of regulatory review of AMX0035 and the inherent unpredictability of the regulatory review process, Amylyx’ ability to fund operations, and the impact that the ongoing COVID-19 pandemic will have on Amylyx’ operations, as well as those risks and uncertainties set forth in Amylyx’ United States Securities and Exchange Commission (SEC) filings, including Amylyx’ Annual Report on Form 10-K for the year ended December 31, 2021, and subsequent filings with the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Amylyx undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220831005780/en/

Media

Becky Gohsler

Finn Partners

(646) 307-6307

amylyxmediateam@amylyx.com



Investors

Lindsey Allen

Amylyx Pharmaceuticals, Inc.

(857) 320-6244

Investors@amylyx.com

Source: Amylyx Pharmaceuticals, Inc.

FAQ

What is the purpose of the FDA meeting on September 7, 2022, for AMLX?

The meeting is to review the New Drug Application for AMX0035, a treatment for ALS.

When is the target action date for the AMX0035 NDA?

The target action date is September 29, 2022.

What was discussed in the previous PCNSDAC meeting on March 30, 2022?

The previous meeting discussed the NDA for AMX0035 for ALS treatment.

What does AMX0035 aim to treat?

AMX0035 aims to treat amyotrophic lateral sclerosis (ALS).

Is AMX0035 approved in any countries?

Yes, AMX0035 is conditionally approved in Canada as ALBRIOZA™.