Amylyx Pharmaceuticals Inc.

Amylyx Pharmaceuticals Inc. Ushering in a new era for treating diseases with high unmet needs

12/23/2025

As 2025 comes to a close, our team took time to reflect on the milestones that shaped this year and the opportunities ahead in 2026.

To the incredible communities we work alongside: thank you for your trust and collaboration. Wishing you peace, joy, and a wonderful holiday season!

12/19/2025

Wolfram syndrome is a rare genetic disorder that affects multiple organs and systems over time.

Most people with Wolfram syndrome have mutations in the WFS1 gene, which encodes wolframin, a protein essential for maintaining endoplasmic reticulum (ER) homeostasis and normal cellular function.

When wolframin is impaired, cells experience chronic ER stress and mitochondrial dysfunction. Learn more about Wolfram syndrome on our website at https://www.amylyx.com/

Post-bariatric hypoglycemia (PBH) places a heavy burden on those it affects, with sudden and unpredictable low blood sug...
12/17/2025

Post-bariatric hypoglycemia (PBH) places a heavy burden on those it affects, with sudden and unpredictable low blood sugar events that can cause severe symptoms like confusion, fainting, and seizures. These events can make daily activities – like working, driving, or even being alone – difficult or unsafe.

As we work toward developing a much-needed PBH treatment, Amylyx is also committed to supporting the community with education and resources. That’s why we developed the “Understanding PBH” guide in close collaboration with the PBH community. View and download here: https://bit.ly/4pL23aZ

12/11/2025

Maria and her family had big plans: travel adventure, and a new chapter after bariatric surgery. But post-bariatric hypoglycemia ( ) brought these dreams to a halt. In this clip, Maria and her daughter talk about the emotional toll of living with PBH and the need for treatment options.

Watch her full story here: https://bit.ly/4oQuNxA

Amylyx cares deeply about supporting and investing in our people, and we’re thrilled that Tapan agrees. Hear what he sha...
12/10/2025

Amylyx cares deeply about supporting and investing in our people, and we’re thrilled that Tapan agrees. Hear what he shared about our company culture and find out more about our approach to learning and development: https://www.amylyx.com/careers

12/05/2025

Today, we announced we will proceed with cohort 2 enrollment in our LUMINA trial in following cohort 1 safety data demonstrating our investigational A*O was generally well-tolerated, with no treatment-related serious adverse events.

We look forward to presenting these data at the 36th International Symposium on ALS/MND. Learn more and see additional presentation details here: https://bit.ly/445x0Oo

Next week, our co-CEOs Justin Klee and Josh Cohen are participating in the 2025 Advancing Drug Development Forum, where ...
12/04/2025

Next week, our co-CEOs Justin Klee and Josh Cohen are participating in the 2025 Advancing Drug Development Forum, where they’ll discuss what’s driving transformation right now in the Boston biotech hub and how to scale with strength, stability, and focus.

See more session details below.

12/03/2025

Calpain-2 (CAPN2) is an important contributor to axonal degeneration, or nerve fiber damage, which plays a critical role in ALS progression.

Research indicates that calpain-2 activation can disrupt key cellular pathways and reduce the structural integrity of nerve fibers, contributing to neurodegeneration. Learn more about the connection between calpain-2 and ALS on our website at https://www.amylyx.com/.

11/24/2025

Sometimes, the first sign that something’s wrong isn’t a test but a nudge from a loved one. Maria’s dog Blue began alerting her to her hypoglycemic episodes before she even knew what was happening.

Her family’s intuition and persistence led to a diagnosis of post-bariatric hypoglycemia ( ).

Watch Maria’s full story here: https://bit.ly/43JDC4Q

Amylyx’ SVP of Medical Affairs, Jamie Timmons, MD, will be presenting this Saturday, November 15, at the The Snow Founda...
11/12/2025

Amylyx’ SVP of Medical Affairs, Jamie Timmons, MD, will be presenting this Saturday, November 15, at the The Snow Foundation and Wolfram Syndrome UK (DIDMOAD) Foundation Virtual Global Research and Trial Update. Dr. Timmons will share Week 48 results and updates from our Phase 2 open-label HELIOS clinical trial in adults living with . Learn more and register for the virtual event here: https://wolframsyndrome.co.uk/ws-conference/ #:~:text=Wolfram%20Syndrome%20UK,15th%20November%20Agenda

10/31/2025

What we’re doing, why, and how we’re doing it is deeply interwoven. Learn more about each of the core values guiding below.

There are no approved therapies for the approximately 160,000 people in the U.S. who have post-bariatric   ( ). That’s r...
10/22/2025

There are no approved therapies for the approximately 160,000 people in the U.S. who have post-bariatric ( ). That’s roughly 8% of people who have undergone the two most common types of bariatric surgery, Roux-en-Y gastric bypass (RYGB) or sleeve gastrectomy, over the last decade. Learn more about this rare condition on our website at https://amylyx.com

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