Copenhagen, June 4, 2018 – Orphazyme A/S, a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, today announced that Orphazyme is presenting preclinical, clinical, and novel biomarker data on Niemann-Pick disease Type C (NPC) at the 2018 Parseghian Scientific Conference for Niemann-Pick Type C Research in Tucson, Arizona, USA on June 2-4, 2018.
The data presented will be the top-line results from the multi-center, non-interventional observational trial (NPC-001) on 36 pediatric patients with NPC conducted as a “lead-in” study before they entered the Phase II/III randomized, placebo-controlled interventional trial (NPC-002) with arimoclomol. In addition, preclinical data on heat-shock protein amplification with arimoclomol in NPC will be presented. The highlights of the data presented during the conference are as follows:
NPC-Clinical Severity Score (NPC-CSS) used to measure disease progression showed a mean increase of 1.47 per 6 months
An abbreviated 5-domain NPC-CSS was also used to measure disease progression and showed a mean increase in 0.75 per 6 months
The abbreviated 5-domain NPC-CSS was highly correlated to the full 17-domain NPC-CSS
HSP70 levels were shown to be markedly decreased in patients with NPC compared to normal patient controls
Glycosphingolipids and cholesterol-derivatives showed promise as biomarkers of disease, including a novel glycosphingolipid that was shown to be elevated in patients with NPC
New preclinical data supported the unique mechanism of action of arimoclomol in NPC and other sphingolipidoses
“The rate of clinical progression observed in the NPC-001 trial and measured by the NPC-CSS is in line with previously published data and importantly there is a strong correlation between the full NPC-CSS and the abbreviated 5-domain NPC-CSS.”, said Dr. Eugen Mengel, University Medical Center in Mainz, Germany and the Coordinating Principle Investigator for Orphazyme’s clinical NPC trials.
Results from the NPC-002 trial are expected in Q3 2018.