Orphazyme A/S, a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, today announced encouraging top-line results for its clinical Phase II/III trial with orally administered arimoclomol for the treatment of patients with Niemann-Pick disease Type C (NPC).
The trial was a multi-center, prospective, double-blinded, placebo-controlled interventional study with a 12-month duration. In total, 50 patients were enrolled in the EU and US. The purpose of the trial was to assess the efficacy and safety of arimoclomol, compared to placebo, in the treatment of NPC, administered in addition to the patient's standard-of-care. The primary endpoints, 5-domain NPC-CSS and Clinical Global Impression of Improvement (CGI-I), evaluated the treatment difference between the arimoclomol-treated and the placebo group after 12 months of treatment.
Overall, baseline characteristics were well-balanced across treatment arms. Arimoclomol was well-tolerated. The overall incidence of adverse events (AEs) was similar for arimoclomol (85.7%) and placebo (81.3%). Serious AEs occurred less frequently in the arimoclomol group (14.3%) compared to placebo (37.5%). The top-line data demonstrated a 74% reduction in progression on the primary endpoint, corroborated by consistent benefit across sub-populations. Placebo progression rates on the CGI-I were lower than expected impeding the ability to show a positive effect.
The full data set, including biomarker data, will become available and be analyzed in Q4 2018. Furthermore, 24-month data will become available from the on-going open-label extension trial in Q2 2019.
Anders Hinsby, Chief Executive Officer of Orphazyme, said: "We are highly encouraged that the top-line data show a strong positive trend on the 5-domain NPC-CSS. We now look forward to receiving the full analysis of data. In addition, the open-label extension trial will provide data on the clinical benefit of arimoclomol over a longer period of time. We are determined to make arimoclomol available to patients as quickly as possible. We are grateful for the strong support we have received from the patient community, the expert physicians, and especially the participants and their families".
Orphazyme will engage with the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) to determine the best path towards making arimoclomol available to those suffering from NPC.
Thomas Blaettler, Chief Medical Officer of Orphazyme, said: “We observed compelling, clinically-relevant trends in favor of arimoclomol in this first trial in NPC. I am encouraged by the 74% reduction in disease progression on the 5-domain NPC-CSS. This is further corroborated by a similar effect on the full scale. Assessing the effect in patients 4 years of age and older (44 out of 50 patients), the effect becomes even more pronounced, with a p-value of 0.027”.
About Orphazyme A/S
Orphazyme is a biopharmaceutical company focused on bringing novel treatments to patients living with life-threatening or debilitating rare diseases. Our research focuses on developing therapies for diseases caused by misfolding of proteins and lysosomal dysfunction. Arimoclomol, the company’s lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C, Gaucher disease, sporadic Inclusion Body Myositis, and Amyotrophic Lateral Sclerosis. The Denmark-based company is listed on Nasdaq Copenhagen (ORPHA.CO). For more information, please visit www.orphazyme.com.
Arimoclomol is an investigational drug candidate that amplifies the production of heat-shock proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally, crosses the blood brain barrier, and has been studied in seven Phase I and three Phase II trials. Arimoclomol is in clinical development for NPC, Gaucher disease, sIBM, and ALS.