Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) announced its financial results for the third quarter of 2024, reporting significant progress driven by the FDA approval and launch of MIPLYFFA for Niemann-Pick disease type C (NPC). The company is focused on addressing unmet needs in rare diseases.
MIPLYFFA Approval and Launch
On September 20, 2024, the FDA approved MIPLYFFA (arimoclomol) capsules, the first drug approved for NPC, indicated for neurological manifestations in adult and pediatric patients (2 years and older) in combination with miglustat. Zevra received a rare pediatric disease Priority Review Voucher (PRV) upon approval.
As of October 31, 90 prescription enrollment forms for MIPLYFFA have been received, with 30% approved for reimbursement and ready for fulfillment, aligning with the company's expectations of 8-12 weeks post-launch. Zevra has also launched AmplifyAssist™, a support program for NPC and UCD caregivers.
OLPRUVA and UCD Focus
OLPRUVA saw three new patient enrollments in Q3, with reimbursement coverage at approximately 76% of covered lives. Zevra is refining its commercial strategy for OLPRUVA, focusing on patient segments with fewer access barriers. In partnership with The National Urea Cycle Disorders Foundation, a patient education campaign has been launched to emphasize prompt and accurate testing of blood ammonia levels.
KP1077 and Celiprolol Development
Zevra completed an End-of-Phase 2 meeting with the FDA for KP1077, with the agency indicating that a single pivotal study with confirmatory evidence would suffice for NDA submission. Strategic alternatives are being evaluated to advance clinical development and commercialization.
The Phase 3 DiSCOVER trial of celiprolol for Vascular Ehlers-Danlos Syndrome (VEDS) is ongoing, with 19 patients now enrolled. Zevra is expanding partnerships with patient advocacy groups and treatment centers.
Financial Highlights
Key financial highlights from Q3 2024 include:
- Revenue, Net: $3.7 million, including $2.6 million from the French expanded access program (EAP) for arimoclomol and $1.1 million in royalties from the AZSTARYS® license agreement.
- R&D Expenses: $10.9 million, a decrease from Q3 2023 due to the completion of the KP1077 phase 2 trial.
- SG&A Expenses: $16.2 million, reflecting commercial launch activities and patient services initiatives.
- Net Loss: ($33.2) million, or ($0.69) per share.
- Cash Position: $95.5 million as of Sept. 30, 2024, expected to extend the cash runway into 2027.
Strategic Outlook
Zevra has completed a portfolio assessment and developed a strategic plan for 2025, focusing on Commercial Excellence, Pipeline and Innovation, Talent and Culture, and Corporate Foundation. The company has discontinued in-house drug discovery activities, opting for outsourced early research and development.
"The third quarter was one of the most exciting and transformational periods in Zevra’s journey," said Neil F. McFarlane, Zevra’s President and Chief Executive Officer. "After years of tireless effort, our team has achieved a major milestone with FDA approval of MIPLYFFA™ (arimoclomol), and we’re celebrating with the Niemann-Pick disease type C (NPC) community."
