Release Date: May 08, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Mirum Pharmaceuticals Inc (MIRM, Financial) reported a significant increase in total net product sales, reaching $68.9 million this quarter, marking a 137% increase from the first quarter of 2023.
- The company successfully launched Marley in new international markets, including Italy, contributing to sustained demand growth across all geographies.
- Mirum Pharmaceuticals Inc (MIRM) announced the U.S. approval of live Marley for Pacific, an important milestone for the company and the patient community it serves.
- The company is making progress on expanding its commercial medicines through new approvals and label expansions, enhancing its market presence and treatment impact.
- Mirum Pharmaceuticals Inc (MIRM) is advancing volixibat in PSC and PBC, with significant market opportunities due to the lack of approved therapies for these conditions.
Negative Points
- The company experienced a temporary disruption due to a healthcare cybersecurity incident affecting their specialty pharmacy, which impacted insurance claims processing and resulted in an estimated $3 million impact on Q1 sales.
- Mirum Pharmaceuticals Inc (MIRM) reported a net loss of $25.3 million for the quarter, indicating ongoing challenges in achieving profitability.
- Total operating expenses for the quarter were high at $95.7 million, which included substantial R&D and SG&A expenses.
- The company faces significant risks and uncertainties, as noted in their forward-looking statements, which could materially affect future results.
- Despite the approval and positive reception of new treatments, there are challenges in payer discussions and reimbursement processes that could affect the speed and extent of revenue realization from new launches.
Q & A Highlights
Q: Can you remind us what the background medications patients are allowed to be on in the volixibat studies that may also address pruritis, and how that factors into your expectations for the results?
A: (Joanne Quan, Chief Medical Officer) - In the volixibat studies, patients are either on or not on ursodeoxycholic acid, with varying levels of alkaline phosphatase. This broad inclusion criteria is designed to make the studies applicable to a real-world setting, potentially translating to first-line use for PBC.
Q: Regarding the $3 million impact from the Change Healthcare issue, is this a one-time loss or will it pop up in future quarters?
A: (Peter Radovich, President and Chief Operating Officer) - The $3 million impact is a one-time effect that concluded by the end of the quarter. There are no expected lingering effects in future quarters. The impact was generally proportional to the size of the products.
Q: What are your expectations for the placebo arm in the volixibat trial for PSC?
A: (Joanne Quan, Chief Medical Officer) - The study design for volixibat in PSC assumes a conservative treatment difference of 1.75 points in pruritis, active compared to placebo. This is based on historical data and is a starting point for the study.
Q: Can you provide any updates on the potential for orphan drug status for Maralixibat in the EU?
A: (Christopher Peetz, CEO) - Discussions with European regulators are ongoing, and the company hopes to have an update soon. There is strong conviction in the data supporting Maralixibat's benefits for patients.
Q: How are you managing the label disparity and reimbursement dialogue for Maralixibat, especially with the upcoming label expansion?
A: (Peter Radovich, President and Chief Operating Officer) - Feedback on Maralixibat's efficacy and broader genetic types included in the label has been very positive from clinicians and patients. Payer conversations are progressing well, and updates to policies after label expansion are expected to occur quickly.
Q: What are the main drivers for your acute care business and how do you see the growth prospects?
A: (Peter Radovich, President and Chief Operating Officer) - The acute care business, particularly the bile acid products, is expected to see mid-single digit year-on-year growth. The company is excited about potential FDA approval next year for new indications, which could help identify more patients.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
