Orphazyme is taking the ax to most of its workforce after its beleaguered single asset has been hit by rejections and serial flops in the clinic.
The history of the drug has been a horror show: Back in March, the Copenhagen-based biotech’s experimental drug, known as arimoclomol, was being tested in 150 patients against placebo for inclusion body myositis, a progressively debilitating muscle-wasting disease, but failed to hit its primary or secondary endpoints.
The drug had also been trialed in amyotrophic lateral sclerosis (ALS), a neurodegenerative disease, with data out back in May showing it too had failed, and spectacularly, missing both its primary and secondary endpoints in helping ALS patients.
There was still hope, however, with the drug, seen by the company as a “pipeline in a product” given its (now diminishing) hits on goal, as it was also going after Niemann-Pick disease type C (NPC), a rare disorder in which cholesterol builds up in patients’ cells and leads to neurological problems that often prove fatal, which had been given a speedy FDA review.
A decision was expected by late June; it came two weeks ago and was roundly rejected by the regulator, which wanted far more data than the company has offered.
These setbacks, especially the latest, led its stock to free fall; now, it is inevitably cutting costs by cutting staffers, around two-thirds all told, as it looks to “advance its corporate strategy” and put its eggs in the NPC basket for arimoclomol.
It’s also axing Rémi Droller, Martijn Kleijwegt and Anders Hedegaard from its board, offering no replacements.
As part of the restructuring, Orphazyme will “significantly scale back its global organization, including teams based in the U.S. and Europe, with the purpose of reducing the number of employees to those who will support essential activities moving forward,” it said in a statement.
This includes going after regulatory approval in Europe as well as “assessing the path forward in partnership with the FDA in the U.S.” Its only other disease area left, according to its latest updates, is the rare inherited metabolic disorder Gaucher disease, for which arimoclomol is in midstage testing (though there is already a fairly large market for this disease).
“As a result of the restructuring of the company and our rigorous cost saving program, we will have to part ways with many of our most valued and talented colleagues,” said Orphazyme CEO Christophe Bourdon, who replaced former chief Kim Stratton when she abruptly quit six months ago.
“I thank each of them for their strong commitment to Orphazyme and dedication to showing up for patients in need. The immediate actions we are taking are necessary to protect and support the ongoing approval process in Europe and the evaluation of a path forward in the U.S.”