Zevra Therapeutics presents data for arimoclomol, OLPRUVA at SSIEM 2024

Zevra Therapeutics announced the presentation of five posters at the Society for the Study of Inborn Errors of Metabolism 2024 Annual Symposium. Four posters focused on data from multiple studies showing the efficacy and safety of arimoclomol as a treatment for people living with Niemann-Pick disease type C, and one poster highlighted data from pharmacokinetic modeling studies of OLPRUVA, a therapy for the long-term management of certain adult and pediatric patients with urea cycle disorders involving deficiencies of carbamylphosphate synthetase, ornithine transcarbamylase or argininosuccinic acid synthetase. The data for arimoclomol presented at SSIEM is summarized below: Title: Efficacy Results from a 12-month Double-blind Randomized Trial of Arimoclomol for Treatment of Niemann Pick Disease Type C – presenting an improved 4-Domain NPC Clinical Severity Scale: Summary: The treatment effect of arimoclomol was evaluated in a 12-month, double-blind, placebo-controlled clinical trial using the original 5-domain Niemann pick type C clinical severity scale and the modified 4-domain Niemann Pick type C clinical severity scale. A statistically significant treatment effect was shown using the modified 4DNPCCSS and the prespecified 5DNPCCSS primary endpoint in the 12-month clinical trial, representing a clinically meaningful reduction in disease progression with arimoclomol treatment compared to placebo. Title: Long-term Efficacy and Safety Evaluation of Arimoclomol Treatment in Patients with Niemann Pick Type C – Data from 48 Months Open Label Trial: Summary: The long-term safety and efficacy of arimoclomol in the 12-month double-blind, and 48-month open-label extension portion of the clinical trial were presented using the 4-Domain Niemann Pick Type C Clinical Severity scale which evaluates ambulation, speech, swallowing and fine motor skills. For those patients transitioning from placebo to arimoclomol at the start of the open-label extension period, the mean annual rate of disease progression reduced from an annual rate of change of 1.9 points during the double-blind phase, to a rate of 0.3 in the first 12 months of treatment, remained numerically smaller for the rest of the trial, and was comparable between the double-blind phase of the trial and the open-label extension phase of the trial. Additionally, arimoclomol was well tolerated with no new safety signals observed. Title: Arimoclomol for the Treatment of NPC in a Real-World Setting: Long-term Outcomes from an Expanded Access Program in the USA. Summary: The long-term safety and efficacy of arimoclomol in a real-world setting were evaluated in a total of 56 adult and pediatric patients in the U.S. arimoclomol expanded access program trial. Data presented included over 3 years of U.S. EAP clinical outcomes and demonstrated that adult and pediatric patients treated with arimoclomol, including those with and without miglustat as a component of their routine clinical care, experienced relatively stable disease as measured by the 5DNPCCSS and 4DNPCCSS and show that arimoclomol was well tolerated. Title: Arimoclomol safety profile in the treatment of NPC in a Real-World setting: Long-term data from an Expanded Access program in the USA: Summary:Safety data from 94 adult and pediatric participants in the arimoclomol US EAP were presented. Safety outcomes reported from exposure periods spanning as much as 46 months of treatment, demonstrated a safety profile consistent with the published clinical trial experience of arimoclomol in NPC. The data for OLPRUVA presented at SSIEM is summarized below: Summary:Pharmacokinetic modeling evaluating whether sodium phenylbutyrate could be safely and effectively administered while fasting showed greater absorption and bioavailability with increased drug exposure in fasted administration of NaPBA compared to fed administration of NaPBA and glycerol phenylbutyrate in both adult and child virtual patients, which is predicted to increase efficacy in proportion to increased drug exposure, theoretically allowing for a 30% dose decrease compared to fed conditions.