Release Date: March 11, 2025

For the complete transcript of the earnings call, please refer to the full earnings call transcript.

Positive Points

• Zevra Therapeutics Inc (NASDAQ:ZVRA) successfully transitioned to a commercial stage company with the launch of MyPIFA for Niemann-Pick disease Type C (NPC), marking a significant milestone.
• The early launch of MyPIFA exceeded expectations with 109 patient enrollment forms received in Q4, indicating strong demand and unmet needs in the NPC community.
• Zevra Therapeutics Inc (NASDAQ:ZVRA) plans to expand MyPIFA's availability outside the US, with a focus on submitting a marketing authorization application in Europe in the second half of 2025.
• The company entered into an agreement to monetize its priority review voucher for $150 million, providing non-dilutive capital to support future growth.
• Zevra Therapeutics Inc (NASDAQ:ZVRA) reported a record level of quarterly product revenue in Q4 2024, driven by MyPIFA's launch and other commercial activities.

Negative Points

• Operating expenses for Q4 2024 were $24.5 million, contributing to a net loss of $35.7 million for the quarter.
• The launch of Alprova for urea cycle disorders (UCDs) has progressed slower than expected, with limited patient pull-through and reimbursement support.
• Zevra Therapeutics Inc (NASDAQ:ZVRA) faces challenges in accelerating patient enrollment for its phase 3 Discover trial for vascular Ehlers-Danlos syndrome (vEDS).
• Many commercial plans have not yet formalized reimbursement coverage for MyPIFA, posing potential hurdles for market access.
• The company experienced a decrease in cash equivalents and investments by $20 million compared to the end of Q3 2024, highlighting financial pressures.

Q & A Highlights

• Warning! GuruFocus has detected 5 Warning Signs with ZVRA.

Q: Can you provide more specific guidance on the European filing timelines for MyPIFA and how the European market dynamics might influence your strategy? A: Neil McFarlane, CEO, explained that they plan to file a marketing authorization application in Europe in the second half of 2025. The European market is more mature due to the long-standing presence of Miglustat, which has led to more diagnosed and treated patients. They believe MyPIFA, in combination with Miglustat, can halt disease progression, offering a significant opportunity in Europe. Adrian Quartel, Chief Medical Officer, added that they are consulting with experts to address previous regulatory feedback and are confident about the filing's acceptance.

Q: How conservative is your cash runway guidance, and does it include potential European approval? A: Leanne Clifton, CFO, stated that the cash runway guidance is conservative and does not include proceeds from the priority review voucher sale. The focus is on executing current plans, and the guidance does not account for potential European approval.

Q: Can you provide more details on the sales during the quarter and expectations for net patient additions? A: Leanne Clifton, CFO, mentioned that Q4 revenue included initial patient dispenses and some refills, with stock levels maintained at target levels. Josh Schaffer, Chief Commercial Officer, highlighted the rapid conversion of expanded access program patients and the focus on identifying and treating diagnosed but untreated patients, as well as those not yet diagnosed.

Q: What are the key variables influencing the trajectory of patient enrollment forms for MyPIFA? A: Josh Schaffer, Chief Commercial Officer, noted that many patients were ready to transition from the expanded access program. The focus is now on expanding beyond these sites to reach more diagnosed patients and those at centers of excellence.

Q: How are reimbursement conversations progressing, and what is the expected net revenue per patient? A: Josh Schaffer, Chief Commercial Officer, explained that they are emphasizing MyPIFA's clinical benefits in discussions with payers. Most patients are getting coverage through formulary or medical exceptions. Leanne Clifton, CFO, mentioned that it's too early to discuss gross-to-net details, but they expect a solid position without aggressive pricing strategies.

For the complete transcript of the earnings call, please refer to the full earnings call transcript.