• Net Revenue: $4.4 million, including $3.1 million from French EAP for arimoclomol and $1.3 million from AZSTARYS royalties and reimbursements.
• OLPRUVA Revenue: De minimis revenue recognized due to transition to a new specialty pharmacy.
• Cost of Goods Sold: $3.2 million obsolescence reserve recognized for OLPRUVA inventory nearing expiration.
• R&D Expenses: $10.5 million, slightly decreased due to completion of KP1077 Phase II trial.
• SG&A Expenses: $12.6 million, reflecting full-quarter engagement in commercial activities for OLPRUVA.
• Net Loss: $19.9 million or $0.48 per share.
• Cash, Cash Equivalents, and Investments: $49.3 million as of June 30, 2024, decreased by $3.4 million from March 31.
• Long-term Debt: $58.3 million as of June 30, 2024.
• Public Offering Proceeds: $64.5 million net from issuing approximately 10.6 million shares at $6.50 per share.
• Pro Forma Cash, Cash Equivalents, and Investments: $113.8 million, extending cash runway into Q1 2027.

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

Release Date: August 13, 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 completed a public offering, raising $64.5 million, which extends their cash runway into Q1 2027.
• The FDA Advisory Committee voted favorably on the effectiveness of arimoclomol for Niemann Pick Disease Type C, indicating potential approval.
• Zevra's commercial infrastructure is strategically built to optimize the launch of both OLPRUVA and arimoclomol, targeting genetic metabolic diseases.
• KP1077 showed positive results in Phase II trials for idiopathic hypersomnia, demonstrating clinically meaningful benefits and good tolerance.
• The company has increased OLPRUVA coverage to 75% of covered lives, improving formulary status and patient services programs.

Negative Points

• OLPRUVA's patient enrollments are not yet at the desired level, indicating challenges in building awareness and engagement.
• The transition to a new specialty pharmacy impacted OLPRUVA's net sales revenue for the quarter, resulting in minimal revenue recognition.
• Cost of goods sold was inflated due to a $3.2 million obsolescence reserve against OLPRUVA inventory nearing expiration.
• Net loss for Q2 2024 was $19.9 million, reflecting increased investments in commercial infrastructure.
• There is uncertainty regarding the final pricing and market acceptance of arimoclomol, pending FDA approval and labeling discussions.

Q & A Highlights

Q: How do you view OLPRUVA sales in the second half of 2024, and what are your initial thoughts on pricing for arimoclomol if it gets approved? Also, what is the patent protection and marketing exclusivity for arimoclomol? A: We rely on orphan drug exclusivity for arimoclomol for up to seven years. It's too early to discuss pricing as we are in labeling discussions, but our goal is to make arimoclomol widely available. For OLPRUVA, the first half focused on building awareness, and we expect increased enrollments in the second half due to enhanced resources and tactics.

Q: After the AdCom, how do you see the combination use of arimoclomol with miglustat in the real world? What feedback have you received from patient advocacy groups post-AdCom? A: We studied arimoclomol versus placebo with routine care, which could include miglustat. We expect the label to reflect this study design. The advisory committee meeting showed overwhelming support from prescribers, caregivers, and patients, reflecting the benefits seen in our Expanded Access Program.

Q: Have you received any additional information requests from the FDA during the labeling discussions for arimoclomol? A: We have received additional information requests, which is typical in this process, but no requests for new data beyond what was shared with the FDA.

Q: Assuming arimoclomol is approved in late September, what are your expectations for product availability? Do you need a different reimbursement support infrastructure for this new indication? A: We are on track to have product available within 8 to 12 weeks post-launch. Our commercial team is already in place, optimized for both OLPRUVA and arimoclomol, and is engaging with prescribers and payers to ensure readiness for commercialization.

Q: Regarding the EAP population, is there anything you can do to expedite their transition to commercial products once arimoclomol is available? A: We aim to transition EAP patients to commercial supply within the first year post-launch. We are working with investigators to ensure a seamless transition while continuing to support patients in the EAP until commercial access is available.

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