HQ Team
April 4, 2024: Amylyx Pharmaceuticals announced it has started a process with the USFDA and Health Canada to voluntarily discontinue the marketing authorisations for its amyotrophic lateral sclerosis (ALS) therapy after the drug failed in a last-stage trial.
The therapy, marketed as Relyvrio in the US and Albrioza in Canada and also known as AMX0035, did not meet its primary and secondary goals in the phase III trial called Phoenix, according to a March 8 statement from Amylyx.
“There was no significant difference observed between participants treated with AMX0035 and placebo in ALSFRS-R total score change ( a measurement of clinical benefit) from baseline at Week 48.
“No significant difference was observed in the subset of participants who met the (Phase-II) Centaur trial criteria. There were also no significant differences observed across secondary endpoints,” according to the March 8 statement.
Free drug program
Patients currently on therapy in the US and Canada who, in consultation with their physician, wish to stay on treatment can be transitioned to a free drug program, according to the April 4 statement.
As a result of the market withdrawal of the therapy, Amylyx plans to lay off 70% of its workforce “to focus the company’s financial resources on upcoming clinical milestones” and decrease external financial commitments outside of its priority areas.
Relyvrio pulled in a revenue of $381 million in 2023.
The milestones include clinical trials named HELIOS AMX0035 for Wolfram syndrome, ORION AMX0035 in progressive supranuclear palsy, and its planned trial of AMX0114 in amyotrophic lateral sclerosis.
ALS community
Amylyx expects to have a cash runway into 2026, which will allow the organization to deliver on key upcoming milestones, including data readouts.
“The decision to remove Relyvrio/Albrioza from the market and provide therapy free of charge for those who wish to continue was informed by the Phoenix trial results, engagement with regulatory authorities, and discussions with the ALS community,” said Joshua Cohen and Justin Klee, Co-CEOs of Amylyx.
The decision by Amylyx to pull out from the market will pose grave consequences to amyotrophic lateral sclerosis patients. There are few options to treat the fatal neurodegenerative disease that affects motor neurons.
Motor neuron death
Amyotrophic lateral sclerosis, also known as motor neuron disease, is a relentlessly progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord.
Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and eventually, death.
More than 90% of people with ALS have sporadic disease, showing no clear family history.
ALS affects around 30,000 people in the US, and more than 30,000 people are estimated to be living with ALS in Europe (European Union and United Kingdom). People living with ALS have a median survival of approximately two years from diagnosis.
