Amylyx Pharmaceuticals launches phase 2 trial for AMX0035 Wolfram syndrome therapy

Cambridge, Massachusetts–based Amylyx has dosed the first participant in its phase 2 HELIOS clinical trial of AMX0035 focusing on Wolfram syndrome. The condition is an ultra-rare genetic disorder involving the endocrine system. Symptoms of Wolfram syndrome can include diabetes insipidus, diabetes mellitus, optic atrophy and hearing loss.

In September, Amylyx won FDA approval for AMX0035 (sodium phenylbutyrate and taurursodiol), marketed as Relyvrio, for adults with amyotrophic lateral sclerosis (ALS).

Both sodium phenylbutyrate and taurursodiol are smal molecules demonstrating neuroprotective effects in preclinical studies.

Trial design

The HELIOS is an exploratory open-label proof of biology study. Amylyx anticipates topline results from HELIOS in 2024.

The HELIOS trial is an exploratory open-label, single-group proof of biology study, with Amylyx anticipating topline results in 2024. The trial aims to assess the safety and efficacy of AMX0035 in…

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FDA approves Amylyx ALS drug Relyvrio 

Amylyx Pharmaceuticals (Nasdaq:AML) has won FDA approval for Relyvrio (AMX0035; sodium phenylbutyrate and ursodoxicoltaurine), notching the first win for amyotrophic lateral sclerosis (ALS) in five years.

The drug received Health Canada approval in June. The drug is known as Albrioza in that country.

In a summary, FDA reviewers note that the limited clinical data available for the drug have resulted in “in a degree of residual uncertainty about the evidence of effectiveness” of the drug. The agency notes that the lack of clarity is acceptable given the significant disease burden of ALS.

“This approval provides another important treatment option for ALS, a life-threatening disease that currently has no cure,” said Dr. Billy Dunn, director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research, in a news release. “The FDA remains committed to facilitating the development of additional ALS treatments.”

AML shar…

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Inside Canada’s approval of Amylyx’s ALS drug Albrioza

One of the things that differentiates amyotrophic lateral sclerosis (ALS) from other rare diseases is that “it is a catastrophic diagnosis,” said Chris Aiello, Head of Canada and General Manager, Amylyx Pharmaceuticals. “Patients do not really have much to go on in terms of pharmacological treatments.” 

Last week, Amylyx Pharmaceuticals received Health Canada approval for the ALS therapy Albrioza (AMX0035) with conditions. 

The regulatory nod represents the first approval for an ALS drug in recent memory. 

A 2017 article in Frontiers in Aging Neuroscience noted that “the overwhelming majority” of human clinical trials for ALS drug candidates failed to show efficacy. Since 1980, there have been more than 80 randomized controlled trials in ALS, and only two therapies, riluzole and edaravone, have found widespread use. 

Chris Aiello

“These patients …

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Understanding the promise of Albrioza: Q&A with the CEO of ALS Society of Canada

Amyotrophic lateral sclerosis (ALS) remains a bewildering disease. Although it is rare, it is not uncommon. The lifetime risk of developing the condition is roughly 1 in 300 by age 85. 

The heterogeneity of the disease is also unique, said Tammy Pighin Moore, CEO of the ALS Society of Canada. “About 5% to 10% of the people diagnosed with ALS have a familial or hereditary connection to it that we can easily understand because of the genes implicated,” Moore said.  

Symptoms can begin in the limb (known as ‘limb onset’) or first affect speech or swallowing (known as ‘bulbar onset’). Some patients with the disease die months after diagnosis, while others may live for decades. For example, the famed physicist Stephen Hawking lived with the condition for 55 years. The average life expectancy after diagnosis is two to five years. 

Health Canada recently became the first country to approve the ALS drug Albrioza (AMX0035) from…

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Amylyx notches first global approval for oral ALS therapy Albrioza

Amylyx Pharmaceuticals (Nasdaq:AML) has received Health Canada approval for Albrioza (AMX0035).

The FDA is anticipated to decide on Albrioza, which consists of sodium phenylbutyrate and ursodoxicoltaurine, by September 29, 2022.

Amylyx is the first new therapy for ALS to win approval in Canada since 2018.

The approval also is the first global regulatory approval for the drug.

Health Canada decided to greenlight the drug after reviewing clinical trial data showing that it slowed disease progression and loss of functional decline in ALS patients.

“We are excited with Health Canada’s decision to approve Albrioza with conditions. Albrioza is a therapy that demonstrated in our CENTAUR trial a statistically significant and clinically meaningful impact on function, alone or in addition to existing ALS therapies,” said Justin Klee and Joshua Cohen, co-CEOs and co-founders of Amylyx, in prepared remarks. “We are grateful to the people who participat…

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Amylyx Pharmaceuticals preps NDA filing for ALS drug AMX0035  

Cambridge, Massachusetts–based Amylyx Pharmaceuticals intends to submit a New Drug Application (NDA) to FDA for AMX0035 (sodium phenylbutyrate and taurursodiol) to treat amyotrophic lateral sclerosis (ALS), which is also known as Lou Gehrig’s disease. 

The company decided to file after a series of meetings with the FDA, including a pre-NDA meeting on July 15.  

AMX0035 was the subject of the Phase 2 CENTAUR trial, which involved 137 participants with ALS. In that trial, the drug met its primary efficacy endpoint of slowing ALS based on the ALS Functional Rating Scale-Revised (ALSFRS-R). 

A forthcoming Phase 3 trial will evaluate the safety and efficacy of the drug over 48 weeks. 

Before reversing course, FDA had asked Amylyx to submit positive Phase 3 trial results before filing a New Drug Application.

To date, there are limited treatment options available for ALS. A 2020 paper in Neuropharmacology. 

Concluded that the two drugs with…

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Amylyx hires new CFO 

Amylyx Pharmaceuticals, a company dedicated to developing new treatments for neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), will install James Frates as chief financial officer (CFO) on Jan. 25, 2021. Frates has currently worked as the CFO of the global biopharma company Alkermes (NSDQ: ALKS) since 1998.

“We are so excited for Jim to join us at this critical juncture for Amylyx,” said Joshua Cohen, co-CEO and co-founder of Cambridge, Mass.-based Amylyx, in a statement. “Jim’s depth and breadth of experience as a biopharmaceutical executive coupled with his financial acumen and personal mission for bringing promising treatments to patients with ALS make him a fantastic addition to our leadership team.”

 “Jim brings the unique combination of experience with growing biopharma companies focused on the central nervous system, both as a CFO and a board member, and passion for the people we serve,” said Justin Klee, co-CEO and co-founder of Am…

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