Amylyx Pharmaceuticals Has Submitted a New Drug Application to the FDA for AMX0035 to Treat ALS
Amylyx Pharmaceuticals today announced it has submitted a New Drug Application (NDA) to the FDA for AMX0035 for the treatment of ALS.
This New Drug Application is based on data from the CENTAUR trial, a placebo-controlled study evaluating 137 people with ALS. In this study, participants receiving AMX0035 had statistically significant slowing of functional decline at the end of the 6-month randomized phase as measured by the Revised ALS Functional Rating Scale (ALSFRS-R), the most commonly used scale in clinics worldwide to measure function in ALS.
In addition, participants who started on AMX0035 during the placebo-controlled phase of CENTAUR showed a 44% lower risk of death.
Amylyx also intends to initiate a global Phase 3 clinical trial in the fourth quarter of 2021. The Phase 3 PHOENIX trial of AMX0035 for the treatment of people with ALS will assess the safety and efficacy of AMX0035 in an international population of approximately 600 participants and build upon findings from the CENTAUR trial.
Our Les Turner ALS Center at Northwestern Medicine will start enrollment later this fall in the Phase 3 PHOENIX trial of AMX0035 and the Expanded Access Program, if implemented by Amylyx.
Saturday is Casino Night – You’re Invited!
Feeling lucky? Want to meet fun, kind-hearted folks? Then join us at All-In for ALS Casino Night hosted by the Les Turner’s Young ProfessionALS Group (YPG) this Saturday (November 6) from 7:00 – 11:00pm at the elegant East Bank Club in Chicago.
This in-person ALS fundraising event will feature favorite casino games like Blackjack, Roulette and Craps, as well as opportunities to win incredible prizes. Appetizers and refreshments will also be available. Proof of Covid vaccination is required at Casino Night, so remember to bring your vaccination card.
Tickets:
- General Admission: $85 online/$100 at the door (includes beer, wine, and hors d’oeuvres)
- VIP Admission: $125 online/$145 at the door (includes open bar and hors d’oeuvres)
ALS Learning Series Webinars – All Past Recordings are Now Available to View On-Demand
Our next webinar in the ALS Learning Series, Hospice and Palliative Care for People with ALS, will take place on November 11 from 12pm – 1pm CT.
Dr. Martha Twaddle, Waud Family Medical Director for Palliative Medicine & Supportive Care at Northwestern Medicine, will cover how to talk about end of life, what to say, and when to say it. She’ll discuss the differences between Palliative Care and Hospice Care, as well as their benefits, services offered, when to seek out this care, and what you need to know before signing-up for hospice services.
We look forward to seeing you at our November webinar.
Each month, the Les Turner ALS Foundation presents a new online webinar to help viewers make better informed decisions about ALS care.
Webinar attendees get their questions answered by leading subject-matter experts, often sourced from the clinicians at our very own Les Turner ALS Center at Northwestern Medicine.
In case you missed a topic which is now relevant to your stage in the ALS journey, all previous webinars are conveniently available to view on-demand.
On-Demand Webinars
Click on any of the following topics to watch the webinar:
- Family Planning and ALS
- Adaptive Technology for Continuous Communication
- Managing Speech and Swallowing Problems in ALS
- Nutrition Optimization in Patients with ALS
- Dissecting ALS Research Articles
- Respiratory Care in ALS: Take a Breath
- Grief and Loss in Children and Youth in Families Living with ALS
- Talking with Children about ALS
- Coping with the Loss of a Loved One (During COVID-19)
- My ALS Communication Passport to Quality Care
- Clinical Trials 101
- Genetics and ALS
Recap of the 11th Annual Les Turner Symposium on ALS
Thank you to all who attended and presented at the 11th Annual Les Turner Symposium on ALS yesterday (Monday, November 1).
Nearly 450 registrants from across the United States and 20 other countries participated in the Symposium.
Special thanks go out to our keynote speaker, Dr. Robert H. Brown of the University of Massachusetts Medical School, for his insightful and valuable presentation on “New Directions in ALS Therapies.”
Dr. Brown’s role in ALS research cannot be overstated. From his early work in the mid-1980s and the discovery of the SOD1 gene 30 years ago along with our own Dr. Teepu Siddique, he has been on the forefront of ALS translational research.
Dr. Brown described a range of promising therapies currently in various stages of development and sees druggable molecular pathways emerging in ALS and convergence points for both familial and sporadic ALS.
Dr. Brown states, “I am completely confident that the technology is out there that we will turn off these ALS genes.”
We’re grateful to be able to connect with all of you in our shared vision of creating a world free of ALS.
If you were unable to attend yesterday’s Symposium, all of the presentations will soon be available for viewing on our website. Please check back for the recordings in the coming days, and feel free to share the presentations with family, friends or others who may find the information valuable.
In the meantime, learn more about the Les Turner Symposium on ALS and view recordings from the previous 2020 Symposium here.
