Orphazyme saw its stock sink more than 30% in Europe Monday morning after it failed to to hit any of the endpoints in a phase 2/3 trial.
The experimental drug, 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 primary was to see how well the med could prevent disease progression as measured by the inclusion body myositis functional rating scale.
The Copenhagen, Denmark-based biotech said “no important safety concerns were detected” but gave no more data as it combs over the trial. Full findings from the study will be “shared in a future scientific forum,” it added in a statement.
The drug is designed to boost production of so-called heat shock proteins, which can “rescue” defective misfolded proteins, clear protein aggregates and improve the function of lysosomes.
This also comes just a few days before new CEO and Amgen veteran Christophe Bourdon is set to take the top seat, replacing interment chief and Chief Financial Officer Anders Vadsholt on April 1.
“We recognize these data are disappointing for patients and families who continue to eagerly await a promising option for IBM. We believe the data collected will be useful to the community, since this trial represents one of the largest, long-term studies ever conducted in this disease and will help inform future research in the category,” said Thomas Blaettler, M.D., chief medical officer at Orphazyme.
This is certainly not the end of the road for arimoclomol, with a readout from a pivotal phase 3 trial of the drug in amyotrophic lateral sclerosis, a neurodegenerative disease, slated in the coming weeks.
More importantly for its commercial future, it is also under a speedy review at the FDA for Niemann-Pick disease type C, a rare disorder in which cholesterol builds up in patients' cells and leads to neurological problems that often prove fatal, with a decision expected by late June.