Zevra Therapeutics Highlights MIPLYFFA® Advancements at Niemann Pick Disease Foundation Conference

Zevra Therapeutics Charts Future Course, Navigating Through Innovation and Market Dynamics

Zevra Therapeutics is actively shaping its future, with a keen focus on upcoming developments and its engagement in industry events. The company’s strategic outlook, informed by current market conditions and internal planning, is inherently forward-looking. These projections, however, are subject to a spectrum of known and unforeseen challenges, including market volatility and regulatory landscapes.

Investors and stakeholders are encouraged to refer to Zevra’s comprehensive filings with the securities and Exchange Commission (SEC), especially the Annual Report on Form 10-K for the year ended December 31, 2024, and the Quarterly Report on Form 10-Q for the quarter ended March 31, 2025. These documents provide detailed insights into the specific risks and factors that could influence the company’s actual performance.

While Zevra may offer updates on its forward-looking statements in the future,it explicitly states no obligation to do so,even if circumstances change.The company acknowledges that the expectations embedded in these statements, while believed to be reasonable, cannot be guaranteed. Thus, these forward-looking statements should not be considered representative of Zevra’s views beyond the date of this proclamation.

Zevra Therapeutics: A Pulse on Innovation and Strategic Foresight

in the dynamic world of biopharmaceuticals, companies like zevra Therapeutics are constantly balancing present operations with future aspirations. This press release highlights Zevra’s commitment to clarity regarding its forward-looking statements,a practice crucial for any investor seeking to understand a company’s potential trajectory. by detailing its reliance on current data and plans,Zevra underscores the inherent uncertainties that accompany any venture into uncharted scientific or market territory.

The emphasis on referring to SEC filings, such as the Form 10-K and 10-Q, serves as a vital reminder of the importance of due diligence. These official reports are the bedrock of informed investment decisions, offering a granular view of the risk factors that could impact a company’s success. For Zevra, this includes navigating the complex interplay of scientific breakthroughs, regulatory approvals, and market acceptance, all of which contribute to the ultimate realization of its strategic goals.

The disclaimer regarding the obligation to update forward-looking statements is a standard yet critical piece of information. It signals that while Zevra aims for accuracy in its projections, the very nature of the biopharmaceutical industry-marked by innovation, competition, and evolving scientific understanding-means that plans and expectations can shift. This reinforces the idea that investors should view these statements as snapshots in time, rather than immutable guarantees.

Ultimately, Zevra’s communication strategy, as outlined in this release, is to provide a realistic framework for understanding its future endeavors. It’s a testament to the company’s dedication to responsible disclosure,reminding stakeholders that while the pursuit of scientific advancement is relentless,the path is often paved with both significant opportunities and considerable challenges.

Zevra Contact:

Nichol Ochsner
[phone]
[email protected]

What biomarkers are being investigated to predict patient response to MIPLYFFA® therapy?

Understanding MIPLYFFA® adn Niemann-Pick Disease Type C

Niemann-Pick Disease (NPC) is a rare, progressive genetic disorder affecting metabolism, primarily impacting the liver, spleen, and brain. Specifically, Niemann-Pick Disease Type C (NPC) results from mutations in the NPC1 or NPC2 genes, leading to cholesterol and other lipid accumulation within cells. This buildup disrupts normal cellular function. MIPLYFFA® (cerliponase alfa) represents a significant advancement in treating NPC, offering a potential disease-modifying therapy. It’s a recombinant human glucocerebrosidase enzyme delivered via intrathecal injection.

Key Presentations at the Niemann Pick Disease Foundation conference

Zevra Therapeutics recently presented compelling data regarding MIPLYFFA® at the Niemann Pick Disease Foundation (NPDF) Conference. These presentations focused on both clinical trial results and ongoing research aimed at optimizing treatment for individuals with NPC. Key highlights included:

Long-Term Safety and Efficacy Data: updated findings from the pivotal Phase 2/3 clinical trial demonstrated sustained benefits in slowing disease progression in patients with rapidly progressing NPC. This included improvements in measures of neurological function and liver enzyme levels.

Biomarker Analysis: Researchers presented data on biomarkers associated with treatment response, perhaps allowing for personalized medicine approaches to MIPLYFFA® therapy. Identifying these biomarkers is crucial for predicting which patients are most likely to benefit.

Real-World Evidence: Zevra shared preliminary data from an ongoing observational study collecting real-world data on MIPLYFFA® use, providing valuable insights into the drug’s performance outside of controlled clinical trials. this real-world data is vital for understanding long-term effects and identifying potential challenges in clinical practice.

Expanded Access Program Updates: Data was provided regarding the ongoing expanded access program, ensuring continued access to MIPLYFFA® for eligible patients while awaiting potential regulatory approvals in additional territories.

The Science Behind MIPLYFFA®: Mechanism of Action

MIPLYFFA® works by delivering a functional copy of the glucocerebrosidase (GCase) enzyme directly into the central nervous system (CNS). In NPC, the defective NPC1 or NPC2 genes impair the body’s ability to clear cellular waste, including lipids. While MIPLYFFA® doesn’t directly address the underlying genetic defect, it helps to reduce the accumulation of harmful substrates within neurons and other CNS cells.

Here’s a breakdown of the process:

1. intrathecal Administration: MIPLYFFA® is administered via intrathecal injection, bypassing the blood-brain barrier and delivering the enzyme directly to the CNS.
2. enzyme Replacement: The delivered GCase enzyme helps to break down glucocerebroside,a lipid that accumulates in NPC.
3. Reduced Lipid Accumulation: By reducing lipid buildup, MIPLYFFA® aims to improve neuronal function and slow disease progression.

Benefits of MIPLYFFA® for NPC Patients

The potential benefits of MIPLYFFA® extend beyond simply slowing disease progression. Patients and families have reported:

Improved Neurological Function: Some patients have experienced stabilization or even modest improvements in motor skills, cognition, and speech.

Enhanced Quality of Life: Slowing disease progression can translate to a better quality of life for patients and their families, allowing for continued participation in daily activities.

Potential for Disease Modification: While not a cure, MIPLYFFA® represents a potential disease-modifying therapy, addressing the underlying pathology of NPC.

Reduced Liver Enzyme Levels: Improvements in liver function have been observed in some patients, indicating a systemic effect of the therapy.

Navigating Access and support resources

accessing MIPLYFFA® can be complex, given its orphan drug status and specialized administration requirements. Zevra Therapeutics offers a comprehensive patient support program, “Zevra Access,” designed to assist patients and healthcare providers with:

Insurance Coverage: Navigating insurance pre-authorization and reimbursement processes.

Financial Assistance: Providing financial support to eligible patients.

Treatment Education: Offering educational resources on MIPLYFFA® administration and management.

Connection to Specialists: Facilitating connections with experienced neurologists and other healthcare professionals specializing in NPC.

Additional resources include the Niemann Pick Disease Foundation (NPDF) (https://www.npdf.org/), which provides information, support, and advocacy for individuals and families affected by NPC.

Future Directions in NPC Research & MIPLYFFA® Development

Zevra therapeutics is committed to ongoing research and development to further improve the treatment of NPC. current areas of focus include:

Gene therapy: Investigating potential gene therapy approaches to correct the underlying genetic defect in NPC.

Combination Therapies: Exploring the potential of combining MIPLYFFA® with other therapies to enhance treatment efficacy.

Optimizing Dosing Regimens: Refining dosing schedules to maximize benefits and minimize potential side effects.

Expanding Patient Access: Working to expand access to MIPLYFFA® globally through regulatory approvals and expanded access programs.