Orphazyme Inc. (NASDAQ: ORPH) stock gained by 4.16% at last close while the ORPH stock price surges by 15.17% in the pre-market trading. Orphazyme is a late-stage pharmaceutical startup working on arimoclomol for the treatment of Niemann-Pick disease. Orphazyme is based in Denmark, with offices in the United States and Switzerland.
ORPH stock’ Current Development
The results of a Phase 2/3 study of arimoclomol, an experimental heat-shock protein amplifier, in Niemann-Pick disease type C (NPC), were reported in the peer-reviewed Journal of Inherited Metabolic Disease, according to Orphazyme (JIMD).
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A prospective, randomized, double-blind, placebo-controlled Phase 2/3 trial was conducted. Fifty patients between the ages of 2 and 18 were randomly assigned 2:1 to arimoclomol: placebo, stratified by miglustat usage. Clinical treatment was maintained on a regular basis. Arimoclomol was taken three times a day orally. According to Mengel et al.1 and Patterson et al.2, the primary outcome was the modification in 5-domain NPC Clinical Severity Scale (NPCCSS) score from baseline to 12 months. The ambulation, cognitive, fine motor skills, speech, and swallowing areas of the 5-domain NPCCSS were judged to be the most therapeutically useful to patients, caretakers, and clinicians (Cortina-Borja et al.3). According to a recent verification of the 5-domain NPCCSS, a clinically significant change for caregivers/patients and physicians is a shift of 1 point or more on the overall score (Patterson et al2).
Arimoclomol was well accepted, with adverse reactions occurring in 88.2 percent of arimoclomol patients and 75.0 percent of placebo patients. When compared to placebo, arimoclomol had fewer severe adverse events.
Christophe Bourdon, Chief Executive Officer at ORPH stock stated,
They are dedicated to supporting the NPC community and are working hard to provide this potentially life-saving drug to patients as soon as possible. Arimoclomol is undergoing regulatory assessment in Europe, with a CHMP opinion expected in Q4 2021, and we are continuing to assess the route ahead in the United States in light of the recent FDA response.