Successful trial of new drug brings ALS treatment closer to FDA approval

Amyotrophic lateral sclerosis (ALS) is a degenerative neurological condition, but there may soon be another treatment option for the disease as Amylyx Pharmaceuticals finishes successful phase 2 of human trials and moves into phase 3 of a new experimental drug. 

The new treatment, called AMX0035, is a co-formulation of two separate drugs: sodium phenylbutyrate and taurursodiol and hopes to slow, if not stop completely, neurodegeneration associated with motor neuron dysfunction.

Phase 2 of the trials involved human trials with 137 subjects and found that the 24-week treatment led to “clinically meaningful and statistically significant” benefits. When given the opportunity to extend treatment after the trial period, most participants chose to continue with the drug. 

The drug was associated with a 44 percent lower risk of death and the treated group lived, on average, six and half months longer than the placebo group.

Phase 3 of the trial is expected to begin shortly, but given the success of phase 2, the ALS Association, a co-funder of the experiment, is urging the FDA to make the drug publicly available before the completion of phase 3. Amylyx Pharmaceuticals says it plans to continue conversations with the FDA to pursue the potential for an early and safe release of the drug to individuals that critically need it.

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Successful trial of new drug brings ALS treatment closer to FDA approval

Amyotrophic lateral sclerosis (ALS) is a degenerative neurological condition, but there may soon be another treatment option for the disease as Amylyx Pharmaceuticals finishes successful phase 2 of human trials and moves into phase 3 of a new experimental drug. 

The new treatment, called AMX0035, is a co-formulation of two separate drugs: sodium phenylbutyrate and taurursodiol and hopes to slow, if not stop completely, neurodegeneration associated with motor neuron dysfunction.

Phase 2 of the trials involved human trials with 137 subjects and found that the 24-week treatment led to “clinically meaningful and statistically significant” benefits. When given the opportunity to extend treatment after the trial period, most participants chose to continue with the drug. 

The drug was associated with a 44 percent lower risk of death and the treated group lived, on average, six and half months longer than the placebo group.

Phase 3 of the trial is expected to begin shortly, but given the success of phase 2, the ALS Association, a co-funder of the experiment, is urging the FDA to make the drug publicly available before the completion of phase 3. Amylyx Pharmaceuticals says it plans to continue conversations with the FDA to pursue the potential for an early and safe release of the drug to individuals that critically need it.

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