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

Zevra Therapeutics, Inc. (NASDAQGS: ZVRA) announced that four posters on MIPLYFFA (arimoclomol) will be presented at the International Congress of Inborn Errors of Metabolism (ICIEM), scheduled for September 2-6, 2025, in Kyoto, Japan. The presentations will include a Best Poster award-winning presentation (#BP-19) that details MIPLYFFA's unique mechanism of action targeting Niemann-Pick disease type C (NPC) pathophysiology. MIPLYFFA is approved in the U.S. for the treatment of NPC, a rare and fatal genetic disorder.

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. These findings are significant as they provide deeper insights into the drug's efficacy across different patient populations, particularly in very young children who are often the most severely affected by NPC. The data could influence treatment protocols and offer hope for improved outcomes in this vulnerable demographic.

The implications of this announcement extend to the rare disease community, healthcare providers, and investors. For patients and families affected by NPC, the new data may validate MIPLYFFA's role as a critical therapy and potentially support expanded use in younger populations. Clinicians may gain valuable information to optimize treatment strategies, while the recognition at a prestigious conference like ICIEM underscores Zevra Therapeutics' commitment to advancing rare disease therapies. The company focuses on combining science, data, and patient need to create transformational therapies for rare diseases with limited or no treatment options, as detailed on their website at https://www.zevra.com.

From an industry perspective, the presentation of robust data at an international congress highlights Zevra's progress in drug development and could enhance its reputation among peers and stakeholders. The Best Poster award signifies peer recognition of the scientific merit of their research, potentially attracting further investment and collaboration opportunities. For the broader rare disease sector, such advancements emphasize the importance of targeted therapies and may encourage similar innovations, ultimately benefiting patients worldwide by addressing unmet medical needs.