The past few days have been very pleasant for biotech company Amylyx Pharmaceuticals (AMLX -7.16%). As is often the case with companies in that sector, Amylyx's stock has recently seen a dramatic rise thanks to news from the laboratory. The company's stock was rising by a bit over 10% week to date as of Friday before market open, according to data compiled by S&P Global Market Intelligence.
Tackling a rare disorder
Amylyx's encouraging news concerned its pipeline drug AMX0035, which targets the rare genetic disorder Wolfram Syndrome. The company reported that a phase 2 clinical trial conducted on 12 adults with the affliction met its primary efficacy endpoint. This was to demonstrate improvement in the function of the pancreas; Wolfram typically degrades this over time, in addition to affecting other organs in the body.
The biotech added that longer-term data indicate "sustained improvement," while being administered the drug. Overall, the treatment was well tolerated by the patients during the study.
In the press release detailing the trial's results, Amylyx said that it now plans to meet with officials from the U.S. Food and Drug Administration (FDA) regarding advancing the drug to phase 3 testing.
Hope for future success
Since Wolfram Syndrome is an uncommon disorder (it affects roughly 3,000 people in the U.S., according to Amylyx), AMX0035 has been granted orphan drug status in both the U.S. and the European Union. Due to the very limited patient population, it won't come close to being a blockbuster. But still, its performance is encouraging and it makes the biotech company's stock well worth tracking.
