Back to top

Image: Bigstock

Amylyx (AMLX) Withdraws ALS Drug, Cuts Workforce by 70%

Read MoreHide Full Article

Amylyx Pharmaceuticals, Inc. (AMLX - Free Report) announced that it will remove its amyotrophic lateral sclerosis (ALS) drug Relyvrio (sodium phenylbutyrate and taurursodiol, also known as AMX0035) from the U.S. markets and it has already started a process with the FDA on the same.

The drug, approved under the brand name Albrioza in Canada, will also be removed from Canadian markets.

While the formal decision to remove the drug from these markets has come recently, the company was contemplating the same following the failure of the late-stage PHOENIX study last month, which led to a massive crash in the company’s shares.

In March 2024, Amylyx announced disappointing top-line results from PHOENIX, a global, 48-week, randomized, placebo-controlled phase III clinical trial of AMX0035 in ALS patients. PHOENIX did not meet its primary endpoint of reaching statistical significance as measured by a change from baseline in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) total score at week 48. The study also failed to achieve statistical significance in secondary endpoints.

Consequently, Relyvrio/Albrioza will no longer be available for new patients. Nevertheless, the patients currently on therapy in the United States and Canada who, in consultation with their physician, wish to continue can be transitioned to a free drug program.

Meanwhile, the PHOENIX Open Label Extension (OLE) is ongoing. Amylyx will now focus on advancing two key programs investigating its lead candidate, AMX0035 in Wolfram syndrome and progressive supranuclear palsy (PSP) and AMX0114, an antisense oligonucleotide targeting calpain-2, in ALS.

Concurrently, Amylyx announced a restructuring plan, whereby it will reduce its workforce by approximately 70% and decrease financial commitments outside its priority areas. Amylyx expects to have a cash runway into 2026 by undertaking this plan.

AMLX’s shares were trading up in pre-market on the news.

Amylyx’s shares have lost 81.2% year to date compared with the industry’s 5.4% decline.

Zacks Investment Research

 

Image Source: Zacks Investment Research

The company will present interim data from its phase II HELIOS study of AMX0035 in Wolfram syndrome, later this month. Wolfram syndrome is a rare, genetic, fatal neurodegenerative disease with no FDA-approved treatment options.

A phase III study, ORION, is ongoing to evaluate AMX0035 for the treatment of progressive supranuclear palsy, a rare neurodegenerative disorder characterized as a tauopathy. An interim analysis is planned for mid-2025.

Relyvrio is the only marketed drug in Amylyx’s portfolio and the decision to withdraw the drug is a huge blow for the company.

The FDA approval of Relyvrio/Albrioza in 2022 was based on data from the phase II CENTAUR study, wherein treatment with AMX0035 showed a significant slow-down of ALS progression and functional decline. Prior to granting this approval, the agency held two advisory committee meetings in 2022 – the first held in March, which voted 6:4 against approving the drug and another in September, which voted 7:2 favoring the drug’s approval after Amylyx submitted additional data on AMX0035.   

Zacks Rank & Key Picks

Amylyx currently carries a Zacks Rank #4 (Sell).

Some better-ranked stocks from the biotech sector are ADMA Biologics, Inc. (ADMA - Free Report) , Galapagos (GLPG - Free Report) and ANI Pharmaceuticals, Inc. (ANIP - Free Report) , each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, estimates for ADMA Biologics’ 2024 earnings per share (EPS) have improved from 22 to 30 cents. In the past year, shares of ADMA have surged 102.6%.

ADMA Biologics’ earnings beat estimates in three of the trailing four quarters and met once, delivering an average surprise of 85.00%.

In the past 60 days, estimates for GLPG’s 2024 loss have narrowed from $1.68 per share to 40 cents.

In the past 60 days, estimates for ANI Pharmaceuticals’ 2024 EPS have improved from $4.06 to $4.43. In the past year, shares of ANIP have surged 75.6%.

The company’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 109.06%.

 

Published in