Zevra announces arimoclomol featured in two poster presentations

Zevra Therapeutics announced that arimoclomol, the Company’s investigational product candidate being developed as a treatment for Niemann-Pick disease type C, is being featured in two poster presentations during the 19th Annual WORLDSymposiu 2023, the annual research conference dedicated to lysosomal diseases being held February 22-26, 2023 in Orlando, Florida, USA. Research presented at WORLDSymposium 2023 included an interim analysis from the ongoing four-year open-label extension of the Phase 2/3 clinical trial of arimoclomol. Results from this analysis, based on up to four years of continuous treatment, suggest that arimoclomol may reduce the long-term progression of NPC. Zevra plans to include these data as part of the updated New Drug Application for arimoclomol, which the Company expects to resubmit to the U.S. Food and Drug Administration as early as the third quarter of 2023. The first poster, titled, "Evaluation of the Long-Term Effect of Arimoclomol in NPC," reported 48-month data from the open-label extension of the Phase 2/3 clinical trial of arimoclomol in NPC. Upon fulfilling the randomized double-blinded portion of the phase 2/3 clinical trial, both placebo- and arimoclomol-treated patients were given the option to continue into the four-year OLE phase of the study with arimoclomol treatment provided in addition to their current standard of care. Progression of NPC disease through the DB and OLE phases was assessed utilizing the five-domain NPC Clinical Severity Scale and compared with an estimated progression calculated from the combination of untreated patients from the NPC-001 observational trial and placebo patients from the NPC-002 Phase 2/3 trial. The second poster titled, "Association Between NPC Severity Score Domains and Corresponding Items of the Performance-based Scale for the Assessment and Rating of Ataxia, SARA," reported research investigating correlations between relevant 5DNPCCSS domains and corresponding SARA test items to potentially provide further supportive evidence for 5DNPCCSS validity as a tool for evaluating NPC progression. Based on a comparative analysis of both measurements, it was determined that individual 5DNPCCSS domains and relevant performance-based SARA test items showed strong associations and alignment between the two instruments for all analysis methods used.