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New Research Update for Proposed ALS Drug Treatment—AMX0035

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We’re excited to share another milestone in the search for better drug treatments for ALS. Amylyx announced today that the company will soon submit a New Drug Application (NDA) for AMX0035 to the FDA. The ALS Association has been closely following the development of this treatment, even providing serious funding to propel its development.

Supporting the Development of a New Treatment

Many of these donations were received during the record-breaking ALS Ice Bucket Challenge in 2014. The ALS Association provided a grant of $750,000 for a clinical trial pilot in June 2016. The next month, a $1.46 million grant was provided to the Northeast ALS Consortium (NEALS) to fund the phase 2 CENTAUR clinical trial of AMX0035.

In September 2020, the concluded trial reports were released. They showed promise for this new drug. Those who received this AMX0035 experienced a significant slower progression of ALS than those who received the placebo. In terms of side effects, the drug presented itself as well-tolerated with a better benefit to risk balance. Read more about the CENTAUR phase 2 results here.

The Crucial Role of ALS Advocates

As soon as the results of phase 2 were released for AMX0035, ALS advocates jumped into action. In a team effort with I AM ALS, the ALS Association issued a petition. It urged the FDA and the drug company to make the treatment widely available as soon as possible. When the average lifespan is just 2 – 5 years after an ALS diagnosis, we can’t wait!

That’s just what ALS advocates communicated to the FDA just earlier this year in May. Eight ALS representatives from across the United States hosted a We Can’t Wait Action Meeting with the FDA. The meeting urged the FDA to act swiftly when it comes to approving new drug therapies for those living with ALS.

Our advocates play just as much as a role in the development and approval of new drugs as does the funding provided by generous supporters. With both these functions working in tandem, we can only hope for speedy approvals of future drug treatments.

What Comes Next?

Amylyx is currently conducting a third clinical trial (PHOENIX) of AMX0035; however, they can concurrently submit the NDA. It’s estimated that the drug company will submit this application sometime in the Fall 2021.

Following the application submission, the FDA will determine if they want to grant the new treatment approval. Or, contrarily, the FDA could require an additional study. This review process will take several months from the time of submission. In addition to the United States, Amylyx has also filed for approval of AMX0035 in Canada and the European Union.

In the meantime, the ALS Association plans to put pressure on the FDA to approve this treatment as soon as possible.

Read the full press release

View the we can't wait action meeting

Read the FDA's response to the we can't wait action meeting

ALS Drug Shows Further Promise, Slowing Progression and Increasing Survivability

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Muscle and Nerve has published the results of the open label extension study of AMX0035, a promising new drug therapy for people with ALS developed by Amylyx. The study showed that people who received the drug lived about 6.5 months longer than people who received the placebo during the trial. This finding is in addition to the findings from the phase 2 trial, showing that people with ALS receiving this drug experienced a significantly slower decline in disease progression, compared to those on a placebo.

As quoted in the New York Times article, Neil Thakur, chief mission officer of the ALS Association, states, “That is a very meaningful benefit for people affected by this devastating, fatal disease.”

These findings further show that the treatment should be made available to people with ALS as soon as possible. We urge the FDA and Amylyx to work together to bring this treatment to market as soon as possible and we stand ready to help ensure that happens. You can help by signing our petition to the FDA and Amylyx at als.org/petition.

ALS Drug Slows Progression, Petition for FDA Approval

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The New England Journal of Medicine (NEJM) has published the results of a phase 2 trial (known as CENTAUR) of the compound AMX0035, produced by Amylyx, a Massachusetts-based pharmaceutical company. The results of the trial were very promising – people with ALS receiving this drug experienced a significantly slower decline in disease progression, compared to those on a placebo. Additionally, this drug shows to be safe and well-tolerated indicating a positive benefit and risk consideration for people with ALS.

The ALS Association and I AM ALS have now issued a petition calling on the drug company and the FDA to make the treatment widely available as soon as possible. The ALS Association and I AM ALS are asking that Amylyx and the FDA move with urgency to ensure this drug is a treatment option for all people living with ALS.

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The ALS Association committed $750,000 to Amylyx in funding for this research, and $1.4M to the NEALS clinical trial consortium to support the trial. These grants were made possible through the ALS Ice Bucket Challenge. The trial included 137 people with ALS and was conducted across 25 top medical centers through the Northeast ALS (NEALS) consortium. All of the participants enrolled in the trial were diagnosed with sporadic or familial ALS within the previous eight months and had a rapid progression of the disease.

With a typical survival time of between 2 to 5 years, people with ALS cannot wait for the full experimental process of this drug to continue. In light of the substantive data of AMX0035’s effectiveness and safety, it has been requested that the FDA and Amylyx to work together to bring this to people with ALS as soon as possible by approving the drug prior to a Phase 3 trial. To access and sign the petition, visit www.als.org/petition.

Sign the Petition Today!

ALS Association Awards $1 Million to Fund Potential ALS Treatment

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Today marks a monumental day for ALS research as the first in-human clinical trials of AT-1501 have begun. AT-1501 is an antibody therapeutic developed by the ALS Therapy Development Institute (ALS TDI), with support from The ALS Association, ALS ONE, and ALS Finding a Cure®. The drug blocks immune cell activation and protects nerves against the progression of ALS. As of today researchers at Anelixis Therapeutics, lead by Dr. Steve Perrin, have successfully administered a dose of AT-1501 to the study’s first participant.

This is a great progressive step on our journey for a cure, and we’re happy to announce that the ALS Association awarded an additional $1 million to Anelixis to help fund this groundbreaking research.

“We are proud to partner with Anelixis and other ALS organizations to help advance this promising compound into clinical trials,” said Calaneet Balas, President and CEO of The ALS Association.

The safety trial of this potentially new treatment is currently enrolling volunteers, both those with ALS and those without, to participate in the study. In total, eight people with ALS will receive AT-1501 and will be monitored for safety , tolerability, and how the drug moves through the body. If it’s proven safe the trial will then move on to Phase II.

We’re excited for the prospect of this new treatment and we remain hopeful that together with patients, caretakers, donors, and researchers we can create a world without ALS.

Help us create a world without ALS by donating today.

A Letter from the Board President: Multidisciplinary Clinic Access Expands in Texas

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When my father was diagnosed with ALS in November 2011, he and my mother began attending an ALS multidisciplinary clinic. He had been sick for three to four years and had seen doctor after doctor trying to get a diagnosis.  When he started attending clinic, the ease of a one-stop place for patients to see all the disciplines necessary to treat ALS gave my parents both relief and empowerment.

Aside from the relief of constant travel to numerous doctor’s appointments, multidisciplinary clinics help provide a better quality of life for a patient and caregiver.  From bonding with another patient or caregiver in the lobby over a shared experience to the ability to learn about new developments in treatments and research, the benefits are endless.

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Dodgeball Tournament Raises Over $55,000 in Three Years to Support ALS Research

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ALS dodgeball tournament hosts

Three days before Christmas 2009, Turner Corbett’s family got the devastating news that his mom, Jennifer, had been diagnosed with ALS. Turner was just 12 years old.

Wanting to make the most of a difficult situation, in 2015, Turner created a benefit event that would raise money for ALS research. After running through some ideas with his friend Alex Doswell, they decided a dodgeball tournament would be the most fun and family friendly.

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Frequently Asked Questions about Radicava™ (Edaravone)

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Based on information as of 5/5/2017

What is Radicava?

Radicava™ (edaravone) is a prescription medicine approved by the U.S. Food and Drug Administration (FDA) to treat people with amyotrophic lateral sclerosis (ALS).1

In clinical trials, some people given Radicava showed significantly less decline in physical function compared to placebo as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R), a validated rating instrument for monitoring the progression of disability in patients with ALS.1,2

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FDA Approves New ALS Drug

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Radicava is First Approved Treatment for ALS in Decades

The Food and Drug Administration’s (FDA) announced today that it has approved Radicava (edaravone), the first new treatment approved specifically for ALS in 22 years. The FDA approved Radicava less than a year after Mitsubishi Tanabe Pharma Corporation submitted a New Drug Application. The only other approved treatment specifically for ALS, riluzole, was approved in 1995.

“We thank the FDA and MT Pharma for working together to expedite the approval of the first new ALS-specific treatment in decades,” said Barbara Newhouse, president and CEO of The ALS Association. “We hope today’s announcement signals the beginning of a new chapter in the fight against this terrible disease. There are several drugs to treat ALS currently in clinical trials and we are hopeful that people living with ALS have even more therapies available to them sooner rather than later.”

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New Research Brings Help, Hope to People with ALS

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ALS is a devastating disease with no cure.

But researchers are working to change that. The increased awareness and donations provided by events like the ALS Ice Bucket Challenge—and by people like you—are making a real difference in the pace of discoveries, bringing us ever closer to the end of ALS.

Here are just a few examples of recent advancements in ALS research and technology:

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ALS Texas: 2016 By the Numbers

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At ALS Texas, we’re dedicated to serving people with ALS in any way we can. That includes running clinics, hosting support groups, providing grants, loaning equipment, funding research, educating people about ALS, and much more.

It’s only through the support of people like you that we’re able to do all this, and we want to thank you for helping make the past year a success. In 2016, ALS Texas provided more services to more people—about 25% more, compared to 2015. Here’s a look at the numbers that help show what we were able to accomplish:

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