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Amylyx considers scrapping ALS drug following Phase III flop

2024-03-08

临床3期临床2期临床结果上市批准疫苗

Amylyx will spend the next eight weeks reviewing the data with the FDA and ALS community. Image credit: Shutterstock / David Herraez Calzada.

Amylyx Pharmaceuticals’s Phase III trial for amyotrophic lateral scFDAosis (ALS) Relyvrio has failed to meet any of its endpoints.

Amylyx PharmaceuticalsI flop, Amylyx said it amyotrophic lateral sclerosis (ALS)hRelyvrioand the broader ALS community, to discuss the results from the Phoenix trial over the next eight weeks and decide how to move forward. The company added this could include withdrawing Relyvrio from the market.

The candidate was approved by Amylyx Food and Drug Administration (FDA) in September 2022 based on findinALSfrom the multicentre Phase II Centaur trial that enrolled 137 ALS patients. In October 2023, the drug, which was set to be marketed as Albrioza in Europe, was rejectRelyvriopproval by the European Medicines Agency (EMA).

Once the market opened, the companUS Food and Drug Administration (FDA)ALS Albrioza European Medicines Agency (EMA)

The global, 48-week, randomised, placebo-controlled Phase III clinical trial of Relyvrio (AMX0035) did not meet its primary endpoint of reaching statistical significance from baseline in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) total score. There was also no statistical significance seen in secondary endpoints.

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The trial saw 664 patients randomised into two arms, Relyvrio or placebo, with both treatment groups receiving standard of care (SoC).

In a statement, Amylyx co-CEOs Justin Klee and JoshParkinson’s vaccineare surprised and deeply disappointed by the Phoenix results following the positive data from the Centaur trial. We will be led in our decisions by two key principles: doing what is right for people living with ALS, informed by regulatory authorities and the ALS community, and by what the science tells us.”

Despite the disappointing efficacy results, Relyvrio was well-tolerated with no new safety signals.

European participants who completed the 48-week randoRelyvrioase had the option to enrol in an open-label extension of the trial of up to two years in duration, which remains ongoing.

Momna Ali, healtAmylyxanalyst at GlobalData, said: “When Relyvrio was initially approved, there was a swirl of controversy anyway. It’s a shame that the late-stage trial found no significant difference between ALS patients treated with Relyvrio and those on placebo after 48 weeks.ALS ALS

“There are approximately 4,000 people in theRelyvrioelyvrio and if Amylyx does pull its drug, sadly patients would only be left with three approved therapies. One, from Biogen, which targets a very small portion of the population with certain genetic mutations. The other two, called Radicava and riluzole, have shown modest therapeutic effects on both function and survival.”

GlobalData is the parent company of the Clinical Trials Arena.

Amylyx has two other ongoing studies with Relyvrio, a PhaRelyvriotudy in progressive supranuclear palsy (PSP) and a Phase II study in Wolfram syndrome.ALS Relyvrio

Relyvrio is a combination of sodium phenylbutyrate RelyvrioursodiolAmylyxh cause a reduction of damage to nerve cells.Biogen Radicava riluzole

ALS landscape

Amylyxnown as classical motor neuron diseaRelyvriois a progressive and faprogressive supranuclear palsy (PSP)d by motor neuron death iWolfram syndromespinal cord. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and eventually, death.

Relyvriota has predicted globsodium phenylbutyrateto hitaurursodiol 2029.

ALSeloping disease-modifying for ALS has proven challenging, with a high failure rate often attributed to the disease’s unclear aetiology and complex pathophysiology.

Earlier this year, Sanofmotor neuron diseaseutALS’s SAR443820 failed to meet neurodegenerative disorderPhase II trial and Ferrer Internacional’s FAB122 failed to meet its primary endpoALS in a Phase III study.respiratory paralysis

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