Last Patient Dosed in Phase 2 Study of Experimental ALS Treatment

In a recent press release, Massachusetts-based biotech company Amylyx Pharmaceuticals announced the completion of dosing in the 24 week study of their experimental amyotrophic lateral sclerosis treatment AMX0035.

About Amyotrophic Lateral Sclerosis

Amyotrophic lateral sclerosis (ALS), is a rare neurological disease characterized by the progressive loss of certain sensory and motor functions, and sometimes by personality and cognitive changes.

The symptoms of ALS are caused by the deterioration and eventual death of motor neurons — the nerve cells responsible for muscle control by physically linking the central nervous system to various muscle groups throughout the body.

As these motor neurons deteriorate, the brain’s ability to effectively coordinate important motor functions like walking, chewing, or talking is increasingly reduced. What may start only as weakness in the hands will eventually progress, eventually making it impossible to swallow, speak, or even breathe without assistance. Ultimately, the disease is fatal.

In rare cases (5 to 10 per cent), ALS is genetically inherited. The vast majority of cases, however, are caused by some unknown combination of factors. It is thought that certain risk factors like age, sex, and possibly environmental factors may increase the likelihood of an individual developing ALS.

Although some treatments exist that can help temporarily control the symptoms of ALS, there is no cure for the disease. Physicians may additionally prescribe other treatments that manage specific symptoms of ALS without treating the underlying disease.

About AMX0035 and the Amylyx Study

AMX0035 is an experimental agent for the treatment of ALS. It is a proprietary formulation of the small molecules sodium phenylbutyrate and tauroursodeoxycholic acid. Researchers hope AMX0035 will safely slow the degeneration and death of motor neurons.

The last of the just under 300 ALS patients who participated in a 24-week, double-blind, placebo-controlled phase II study of AMX0035 recently received their final dosing of the drug.

“We’re thankful to everyone involved,” said Dr. Patrick Yeramian, Amylyx’s Chief Medical Officer. He and his colleagues will be working hard to deliver compiled and analyzed data “as quickly as possible over the coming months.”

An open-label extension to the phase II study is currently underway, allowing patients who participated to continue receiving the drug.


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