Zevra Therapeutics to Present New MIPLYFFA Data at International Congress, Poster Earns Best Poster Award

Zevra Therapeutics, Inc. (NASDAQGS: ZVRA), a commercial-stage company focused on therapies for rare diseases, announced that four posters on MIPLYFFA (arimoclomol) will be presented at the International Congress of Inborn Errors of Metabolism (ICIEM), held Sept. 2-6, 2025, in Kyoto, Japan. MIPLYFFA, approved in the U.S. for the treatment of Niemann-Pick disease type C (NPC), will be highlighted in a Best Poster award-winning presentation (#BP-19) detailing its unique mechanism of action targeting NPC pathophysiology.

Additional presentations will feature positive new data from a pediatric substudy in patients younger than two years old and a prespecified efficacy analysis in patients on miglustat who switched from placebo to MIPLYFFA. The presentation of these findings at a major international conference underscores the scientific community's recognition of MIPLYFFA's potential impact on rare disease treatment. The data being presented represents significant advancements in understanding how this therapy works and its effectiveness across different patient populations.

The pediatric substudy data is particularly important as it addresses the critical need for effective treatments in very young patients with NPC, a progressive and fatal genetic disorder. The prespecified efficacy analysis provides valuable insights into how patients already receiving miglustat therapy respond when switching to MIPLYFFA treatment. These presentations contribute to the growing body of evidence supporting MIPLYFFA's role in managing this rare disease. The full details of these presentations can be found in the official press release available at https://ibn.fm/XjkBY.

The recognition of the MIPLYFFA mechanism of action poster with a Best Poster award at ICIEM 2025 signifies the scientific importance and novelty of this research. International conferences like ICIEM serve as crucial platforms for sharing cutting-edge research that can accelerate the development of treatments for rare metabolic disorders. The data being presented may influence clinical practice guidelines and treatment approaches for NPC patients worldwide. For more information about the conference and its proceedings, visit the official website at https://www.iciem2025.org.

These presentations represent ongoing research efforts to better understand and optimize treatment strategies for Niemann-Pick disease type C. The accumulation of clinical data across different patient subgroups helps build a more comprehensive understanding of MIPLYFFA's therapeutic profile. The findings being shared at this international forum may also inform future research directions and potential regulatory considerations in other markets beyond the United States. The continued investigation into MIPLYFFA's effects supports the broader rare disease community's efforts to develop effective treatments for conditions with limited therapeutic options.