Orphazyme presents 36-month data supporting durable response to arimoclomol at meet

  Orphazyme A/S (ORPHA.CO; ORPH), a late-stage biopharmaceutical company, today announced 24-month interim results of an open-label extension (OLE) trial, providing efficacy and safety data for its investigational treatment arimoclomol in Niemann-Pick disease type C (NPC) for up to 36 months. The data are featured in a presentation as part of the Parseghian Scientific Conference for Niemann-Pick disease type C Research.

The results demonstrate that arimoclomol provided a sustained benefit to study participants by reducing NPC progression as measured by the 5-domain NPC Clinical Severity Scale (5D-NPCCSS). A slowing of progression from baseline was observed through 36 months in participants who received arimoclomol from the start of the double-blind phase (mean change, 3.5 points). By comparison, disease progression among NPC patients receiving routine clinical care was estimated to be a mean increase of 5.2 points after three years, based on a statistical model combining placebo data from the NPC-002 double-blind study and prospective data from the observational NPC-001 study. The effect was consistent across pre-specified subgroups, including among participants more than four years of age and those treated with miglustat. Also, slowing of progression through 24 months was observed in those participants who initiated arimoclomol treatment upon entering the open-label period (mean change, 0.9 points).

“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.

Arimoclomol demonstrated a consistent safety profile throughout the 36-month treatment period. Adverse events observed during the open label extension phase were similar to those observed in the double-blind phase. A total of 41 patients joined the OLE following the double-blind period; 33 have now completed up to 36 months of treatment.

Data from the 36-month period support the findings from the 12-month double-blind period, which showed a clinically meaningful difference on the 5-domain NPCCSS, with a significant p-value of 0.046 (previously calculated at p=0.0537).

Orphazyme continue to pursue regulatory approval in Europe and evaluate a path forward for arimoclomol in NPC in the US.

https://www.biospace.com/article/releases/orphazyme-presents-36-month-data-supporting-durable-response-to-arimoclomol-during-parseghian-scientific-conference-for-npc-research/