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

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  • 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.
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