Release Date: November 12, 2024

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

Positive Points

• Zevra Therapeutics Inc (NASDAQ:ZVRA) successfully launched Myle, the first FDA-approved therapy for Niemann-Pick disease type C, with 90 prescription enrollment forms submitted by October 31st.
• The company received a rare pediatric disease priority review voucher (PRV) with the approval of Myle, which they plan to monetize for non-dilutive capital.
• Zevra Therapeutics Inc (NASDAQ:ZVRA) completed an end of phase two meeting with the FDA for KP1077, receiving clear direction for a phase three path forward.
• The company has a strategic plan for 2025 focusing on commercial excellence, pipeline and innovation, talent and culture, and corporate foundation.
• Zevra Therapeutics Inc (NASDAQ:ZVRA) completed a public offering, raising approximately $64.5 million, extending their cash runway into 2027.

Negative Points

• The launch of Aprova, a treatment for urea cycle disorders, is underperforming with only three prescription enrollment forms received in the third quarter.
• Zevra Therapeutics Inc (NASDAQ:ZVRA) reported a net loss of $33.2 million for the third quarter, a significant increase from the previous year.
• The company decided to discontinue in-house pro-drug discovery activities and close facilities in Iowa and Virginia, indicating a shift in strategy.
• There are challenges in raising patient awareness and overcoming reimbursement hurdles for Aprova in the urea cycle disorder market.
• Zevra Therapeutics Inc (NASDAQ:ZVRA) has halted some development activities, including Osanit, as part of their pipeline review.

Q & A Highlights

• Warning! GuruFocus has detected 8 Warning Signs with ZVRA.

Q: Can you provide an update on the manufacturing and distribution timeline for Myle, and what are the next steps for patients who have received reimbursement approval? A: Neil McFarlane, CEO, stated that the manufacturing and distribution timeline for Myle is on track, with the drug expected to be in the channel within the 8 to 12-week timeframe post-approval. Josh Shaer, Chief Commercial Officer, added that there are no additional hurdles for reimbursement, and the company is working through the benefits investigation process for both EAP and new patients.

Q: How should we model Myle sales for Q4 2024, and what are the next steps for European expansion? A: Neil McFarlane, CEO, mentioned that the first shipment of Myle to the specialty pharmacy is expected in Q4, with revenue recognition following. For European expansion, the company is prioritizing regulatory submission but cannot provide specific timing yet. They plan to update on this in early 2025.

Q: Can you provide more details on the status of the remaining 60 patients who have started enrollment forms for Myle? A: Josh Shaer, Chief Commercial Officer, explained that as of October 31st, 30% of the 90 enrollment forms have been processed and approved for reimbursement. The company continues to work through the remaining forms, with a high level of confidence in achieving further approvals.

Q: What is the level of awareness and excitement for Myle outside of the EAP program, and will you provide sales guidance in early 2025? A: Neil McFarlane, CEO, stated that there is no intention to provide sales guidance in the early phases of the launch. However, the company is focused on raising awareness and identifying new patients, with a strong engagement from patient advocacy groups and prescribers.

Q: Regarding KP 1077, is there a preference for the types of strategic alternatives you might be contemplating? A: Neil McFarlane, CEO, indicated that the company is exploring strategic alternatives to maximize the value of KP 1077, considering the infrastructure needs for commercialization. They are open to partnerships to unlock value but have not specified a preference for any particular type of strategic alternative.

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