Zevra Therapeutics to Present Multiple Datasets on MIPLYFFA® (arimoclomol) at ICIEM

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Release Date

2025-08-29

Category

Clinical Trial Event

Reference

Source

Breakthrough Clinical Results

Zevra Therapeutics announced that four posters on MIPLYFFA® (arimoclomol), its approved therapy for Niemann-Pick disease type C (NPC), will be presented at the International Congress of Inborn Errors of Metabolism (ICIEM) in Kyoto, Japan. The presentations include data from a pediatric substudy in patients younger than two years old, a new efficacy analysis of patients switching from placebo to MIPLYFFA, and details on MIPLYFFA's mechanism of action, which uniquely targets the underlying pathophysiology of NPC. One poster received a Best Poster award. MIPLYFFA, approved by the FDA in September 2024, increases the activation of transcription factors TFEB and TFE3, upregulating CLEAR genes and reducing unesterified cholesterol in lysosomes. Long-term data from various studies, including a Phase 2/3 trial and Expanded Access Programs, will also be presented, showcasing meaningful clinical outcomes in over 270 NPC patients worldwide. A Marketing Authorization Application is under review by the European Medicines Agency.

Key Highlights

  • Four posters on MIPLYFFA (arimoclomol) will be presented at ICIEM.
  • Positive data from a pediatric substudy and a new efficacy analysis will be shared.
  • One poster detailing MIPLYFFA's mechanism of action received a Best Poster award.
  • Long-term data from over 270 NPC patients will be presented.

Incidence and Prevalence

Global Epidemiology of Niemann-Pick Disease Type C

There appears to be a discrepancy between the requested information about Niemann-Pick disease type C (a rare lysosomal storage disorder) and the available context information.

Niemann-Pick disease type C (NPC) is a rare autosomal recessive genetic disorder characterized by the inability of the body to properly transport cholesterol and other lipids inside cells. This leads to an abnormal accumulation of these substances in various tissues, including the brain.

Unfortunately, specific up-to-date global epidemiological data on NPC incidence and prevalence from recent PubMed-indexed research is not available in the provided context to formulate a comprehensive response.

NPC is considered an ultra-rare disorder with variable clinical presentation, which can make accurate epidemiological assessment challenging. The disease affects both children and adults and is caused by mutations in either the NPC1 or NPC2 genes.

The condition is characterized by progressive neurological deterioration and can present with a wide range of symptoms including hepatosplenomegaly, vertical supranuclear gaze palsy, ataxia, dystonia, and cognitive impairment.

Early diagnosis remains challenging due to the heterogeneous presentation of the disease, contributing to potential underestimation of its true prevalence.

Research into NPC continues to evolve, with ongoing efforts to better understand its epidemiology, improve diagnostic techniques, and develop effective treatments for this devastating disorder.

Drug used in other indications

MIPLYFFA (Arimoclomol) Clinical Trials Beyond Niemann-Pick Disease Type C

Indications Under Investigation

Arimoclomol is currently being trialed for several neurological conditions beyond Niemann-Pick disease type C:

Gaucher Disease (GD)

Arimoclomol is being investigated specifically for the neuronopathic forms of Gaucher Disease. As a heat shock protein (HSP) amplifier, it enhances the folding, maturation, activity, and correct cellular localization of mutated GCase. Studies have shown it induces relevant HSPs such as ER-resident HSP70 (BiP) and demonstrates efficacy across several genotypes including the common L444P and N370S mutations. A key advantage is that arimoclomol efficiently crosses the blood-brain-barrier, making it potentially valuable for addressing neurological manifestations of the disease.

Inclusion Body Myositis

Arimoclomol has been evaluated in a multicentre, randomised, double-blind, placebo-controlled study for inclusion body myositis. The trial enrolled adults at specialist neuromuscular centers in the USA (11 centers) and UK (1 center). Participants received either oral arimoclomol 400 mg or matching placebo three times daily (1200 mg/day) for 20 months. The primary endpoint measured changes in the Inclusion Body Myositis Functional Rating Scale (IBMFRS). Results indicated that arimoclomol did not improve efficacy outcomes compared to placebo, though it demonstrated an acceptable safety profile.

Amyotrophic Lateral Sclerosis (ALS)

Arimoclomol has been studied specifically in patients with rapidly progressive SOD1-mutant ALS through a double-blind, placebo-controlled trial. Patients were randomized 1:1 to receive either arimoclomol 200 mg tid or matching placebo for up to 12 months. The study provided Class II evidence of safety and tolerability at this dosage. While not powered for therapeutic effect, consistency across prespecified efficacy outcomes suggested a possible therapeutic benefit in ALS patients.

Intervention Models

The clinical trials for arimoclomol employ various intervention models:

  • Gaucher Disease: Research utilized in vitro assays to assess arimoclomol's effect on GCase activity in ex vivo systems of primary fibroblasts and neuronal-like cells from GD patients.

  • Inclusion Body Myositis: The study employed a randomized controlled trial with 1:1 randomization to either arimoclomol or placebo. The randomization sequence was computer generated centrally using a permuted block algorithm with randomization numbers masked to participants and trial staff. This trial was registered with ClinicalTrials.gov, number NCT02753530.

  • ALS: The intervention model was a double-blind, placebo-controlled trial with 1:1 randomization to receive arimoclomol or placebo for up to 12 months. This study was registered as NCT00706147.

Arimoclomol is currently described as an investigational drug in phase II/III clinical trials that has been extensively clinically tested across these neurological conditions.

Company drugs in pipeline

I notice that the context provided doesn't contain any specific information about Zevra Therapeutics, Inc. or its drug pipeline. Without this information, I cannot provide an accurate answer about which indications Zevra Therapeutics has drugs in pipeline for.

Zevra Therapeutics, Inc. Drug Pipeline Indications

I don't have specific information about Zevra Therapeutics, Inc.'s current drug pipeline indications. To provide an accurate and comprehensive overview of their therapeutic focus areas and clinical-stage compounds, more specific information about their drug development programs would be needed.

For accurate information about Zevra Therapeutics' pipeline, I would recommend consulting:

  • Their official company website
  • Recent investor presentations
  • SEC filings such as their latest 10-K or 10-Q reports
  • Press releases about their clinical development programs
  • Industry databases that track pharmaceutical pipelines

These sources would provide the most up-to-date information about which disease indications and therapeutic areas Zevra is currently targeting with their investigational compounds.