MIPLYFFA's Q1 2025 Earnings Call: Contradictions Unveiled in Patient Enrollment and Reimbursement Challenges

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Commercial Success of MIPLYFFA:
- Zevra Therapeutics has enrolled over one-third of diagnosed NPC patients in the U.S., reaching more than 120 prescription enrollment forms since launch.
- The success is driven by the unique clinical profile of MIPLYFFA, which has been shown to halt disease progression and is supported by robust clinical data.

Talent and Culture:
- Zevra's team is recognized for strong execution, leading to the company being acknowledged on Fast Company's top 10 most innovative companies list.
- The company's passionate and experienced team focused on its mission has been instrumental in achieving these milestones.

Financial Strength and Strategic Investments:
- The monetization of the pediatric rare disease priority review voucher added $148.3 million non-dilutive capital to Zevra's balance sheet.
- This bolstered financial strength enables Zevra to invest in its strategic priorities independently from capital markets.

Celiprolol Phase 3 Trial Enrollment:
- Enrollment in the Celiprolol trial has reached 32 patients, despite inheriting the trial with paused enrollment.
- Efforts to target COL3A1 patient centers and genetic testing have begun to accelerate enrollment in this high unmet need area.