Shares of Orphazyme A/S (ORPH) are gaining over 12% on Tuesday morning. The late-stage biopharmaceutical company on Monday revealed plans to lay off most of its employees due to a series of failures for its lead investigational product. The company also presented 36-month data supporting durable response to arimoclomol, its lead candidate.
ORPH is currently trading at $8.16, up $0.92 or 12.71%, on the Nasdaq. In the 52 week period, the stock has traded between $4.75 and $77.7.
Orphazyme is developing Arimoclomol for rare diseases including Niemann-Pick disease type C (NPC) and Gaucher disease.
On Monday, Copenhagen, Denmark-headquartered Orphazyme announced 24-month interim results of an open-label extension trial, providing efficacy and safety data for its investigational treatment arimoclomol in Niemann-Pick disease type C for up to 36 months.
The study results show that arimoclomol provided a sustained benefit to study participants by reducing NPC progression as measured by the 5-domain NPC Clinical Severity Scale.
“Following on the outcomes from the 12-month double-blind phase, which indicated a clinically meaningful effect on disease progression, these longer-term data provide an encouraging picture that arimoclomol could deliver a sustained benefit and consistent safety profile over time,” said Marc Patterson, MD, Professor of Neurology, Pediatrics and Medical Genetics, Mayo Clinic Children’s Center in Rochester, MN.
The company on Monday also announced that it will lay off two-thirds of its workforce.
Orphazyme said it remains committed to pursuing regulatory approval in Europe and assessing a path forward for arimoclomol in the U.S. following receipt of a Complete Response Letter from the U.S. Food and Drug Administration (FDA) on June 17, 2021.
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