Core Insights - Zevra Therapeutics presented new positive data on MIPLYFFA for Niemann-Pick disease type C (NPC) at the International Congress of Inborn Errors of Metabolism (ICIEM) [1][2] - MIPLYFFA has shown efficacy in reducing annual disease progression in patients who switched from placebo while on concomitant miglustat treatment [4] - The company received a nomination for Best Poster Award for its presentation on MIPLYFFA's mechanism of action [2] Data Highlights - MIPLYFFA activates transcription factors that enhance NPC1 protein levels, addressing NPC etiology through multiple pathways [3] - In a pediatric substudy, MIPLYFFA was well tolerated in children aged 6 to under 24 months, with no new safety signals observed [5] - Long-term outcomes from an expanded access program confirmed the effectiveness and safety of MIPLYFFA, consistent with results from the pivotal Phase 3 study [6] Product Information - MIPLYFFA (arimoclomol) is approved for treating neurological manifestations of NPC in patients aged 2 years and older, in combination with miglustat [9] - The drug received FDA approval on September 20, 2024, and has Orphan Medicinal Product designation from the European Medicines Agency [7][8] - MIPLYFFA has been administered to over 270 NPC patients globally, making it part of the most extensive clinical development program for NPC to date [7][8] Company Overview - Zevra Therapeutics focuses on developing therapies for rare diseases with limited treatment options, aiming to provide life-changing therapeutics [19] - The company employs unique, data-driven strategies to overcome drug development challenges in the rare disease community [19]

Core Insights - Zevra Therapeutics announced the publication of long-term efficacy and safety results for MIPLYFFA (arimoclomol) in treating Niemann-Pick Disease Type C (NPC), demonstrating sustained disease progression halting over multiple years [1][2] Company Overview - Zevra Therapeutics is a commercial-stage company focused on therapies for rare diseases, aiming to provide transformational treatments for conditions with limited options [14] Product Information - MIPLYFFA is an approved therapy for NPC, indicated for use in combination with miglustat for patients aged 2 years and older [4] - The drug received FDA approval on September 20, 2024, and has shown the ability to activate transcription factors that regulate lysosomal function [3] Clinical Trial Results - The Phase 2/3 trial demonstrated that MIPLYFFA halted disease progression compared to placebo over a one-year duration, with long-term data showing sustained efficacy for at least five years in over 270 patients [2][3] - The open-label extension study confirmed no new safety concerns, aligning with previous trial results [2] Safety Information - Common adverse reactions reported include upper respiratory tract infection, diarrhea, and decreased weight, with hypersensitivity reactions noted in some patients [5][9][10] - MIPLYFFA may cause increases in serum creatinine without affecting glomerular function, primarily occurring in the first month of treatment [8]

Core Insights - Zevra Therapeutics, Inc. announced presentations on MIPLYFFA (arimoclomol) at the NNPDF Conference, highlighting its role as the first FDA-approved treatment for Niemann-Pick disease type C (NPC) [1][2] - MIPLYFFA is indicated for use in combination with miglustat for treating neurological manifestations of NPC in patients aged 2 years and older [7][6] Presentation and Research Findings - Dr. Barbara K. Burton presented an overview of MIPLYFFA, emphasizing its unique mechanism that improves lysosomal function and halts disease progression at 12 months in a pivotal study [2][3] - The pivotal trial demonstrated that arimoclomol combined with miglustat halted disease progression compared to placebo, confirmed by a 48-month open-label extension study [4][6] - Two posters were presented: one detailing long-term effectiveness and safety of arimoclomol, and another providing mechanistic evidence of arimoclomol's action through the CLEAR network [4][5] Drug Mechanism and Clinical Significance - MIPLYFFA enhances the activation of transcription factors TFEB and TFE3, leading to the upregulation of CLEAR genes and improved lysosomal function [6][17] - The drug has shown to reduce unesterified cholesterol in NPC fibroblasts, although the clinical significance of this finding remains to be fully understood [6] Company Overview - Zevra Therapeutics focuses on developing therapies for rare diseases with limited treatment options, aiming to create transformational therapies through data-driven strategies [18]

Core Insights - Zevra Therapeutics, Inc. has published significant findings regarding the mechanism of action of its therapy MIPLYFFA (arimoclomol) for Niemann-Pick disease type C (NPC), highlighting its potential to address the underlying pathology of the disease [1][2][3] Group 1: Mechanism of Action - The publication details how arimoclomol enhances the translocation of translation factors EB and E3 (TFEB & TFE3) from the cytosol to the nucleus, initiating a cascade that upregulates coordinated lysosomal expression and regulation (CLEAR) genes, including NPC1 [2] - Increased expression of CLEAR genes leads to higher NPC1 protein levels in lysosomes, improving cholesterol trafficking and correlating with better neurological behaviors in animal models [2] Group 2: Clinical Significance - MIPLYFFA, approved by the U.S. FDA on September 20, 2024, is indicated for use in combination with miglustat for treating neurological manifestations of NPC in patients aged 2 years and older [3][4] - In a pivotal phase 3 trial, MIPLYFFA demonstrated the ability to halt disease progression compared to placebo, as measured by the NPC Clinical Severity Scale [3] Group 3: Safety Information - Common adverse reactions reported in clinical trials include upper respiratory tract infection, diarrhea, and decreased weight, with hypersensitivity reactions such as urticaria and angioedema noted in some patients [5][8][9] - MIPLYFFA may cause increased serum creatinine levels without affecting glomerular function, primarily occurring within the first month of treatment [7] Group 4: Company Overview - Zevra Therapeutics focuses on developing therapies for rare diseases, aiming to create transformational treatments for conditions with limited or no options [13] - The company employs data-driven strategies to navigate complex drug development challenges, striving to make new therapies accessible to the rare disease community [13]