Benzinga | Mar 29, 2021 02:45PM EDT

Why Is Orphazyme (ORPH) Stock Trading Lower On Monday?

* Orphazyme A S (NASDAQ: ORPH) saw its stock plunge on Monday after failing to meet primary and secondary endpoints in a Phase 2/3 trial.

* The experimental drug, arimoclomol, was being tested in 150 inclusion body myositis patients against a placebo, a muscle-wasting disease.

* The primary objective was to see how well the treatment could prevent disease progression measured by the inclusion body myositis functional rating scale.

* No significant safety concerns were detected but gave no more data. Full findings from the study will be "shared in a future scientific forum," the company added in a statement.

* The drug is designed to boost the production of heat shock proteins, which can "rescue" defective misfolded proteins, clear protein aggregates, and improve lysosomes' function.

* This is certainly not the end of the road for arimoclomol. A Phase 3 data readout of the drug in amyotrophic lateral sclerosis, a neurodegenerative disease, is expected in the coming weeks.

* Arimoclomol is also under a 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, with a decision expected by late June.

* Price Action: ORPH shares are down 29.5% at $8.74 in market trading hours on the last check Monday.