Release Date: August 01, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- RUCONEST sales grew by 23% in Q2 2024 compared to the previous year, and by 16% in the first half of 2024.
- Joenja sales increased by 16% in Q2 2024 compared to Q1 2024, and by 44% in the first half of 2024 compared to the last half of 2023.
- Pharming Group (PHGUF, Financial) is embarking on a Phase 2 study for a new indication for leniolisib and exploring a third indication.
- The company has identified significant commercial potential for Joenja in both APDS and subsequent indications.
- Pharming Group (PHGUF) is focusing on in-licensing or acquiring clinical stage opportunities in rare diseases to broaden its portfolio.
Negative Points
- Operating expenses increased from $65.8 million to $70.1 million, reflecting continued investment in Joenja and other areas.
- Net profit turned into a loss of $1.2 million in Q2 2024 compared to a profit of $1.3 million in Q2 2023.
- Cash and marketable securities decreased by almost $42 million, primarily due to bond repurchases.
- The CHMP review for leniolisib has been extended to January 2026, delaying potential European market entry.
- There is significant competition in the hereditary angioedema market, which could impact RUCONEST sales despite its unique position.
Q & A Highlights
Q: Could you share more about the third indication for leniolisib and its potential impact on the addressable patient population?
A: (Anurag Relan, Chief Medical Officer) The third indication we are exploring is another primary immune deficiency with immune dysregulation, similar to APDS. This will expand the potential patient population significantly. We are currently obtaining regulatory feedback on the proposed clinical development plan.
Q: Can you elaborate on the VUS validation studies and the 1,200 patients identified in the US?
A: (Anurag Relan, Chief Medical Officer) The 1,200 patients identified have had genetic tests showing variants of uncertain significance (VUS) in the PIK3CD or PIK3R1 genes. We expect about 20% of these VUS results to be reclassified as disease-causing, significantly increasing the APDS patient population.
Q: How many patients have you identified in the UK, and what are your expectations for getting them on paid therapy?
A: (Sijmen de Vries, CEO) We have identified 61 patients in the UK, with 37 over 12 years of age. We expect these patients to go on paid therapy quickly once reimbursement is secured, likely in the first half of next year.
Q: Can you provide an update on your business development efforts?
A: (Sijmen de Vries, CEO) We have broadened our search and have a Chief Business Officer on board. We are actively assessing assets and have issued non-binding offers, although due diligence sometimes reveals unexpected issues.
Q: What impact do you expect from the anticipated launch of competing products for RUCONEST in 2025 and beyond?
A: (Sijmen de Vries, CEO) We believe RUCONEST will continue to serve its unique patient population effectively. While some patients may try new competitors, RUCONEST's consistent response rates and ability to address all three pathways of hereditary angioedema attacks make it a reliable choice.
Q: Can you provide more details on the regulatory work needed for leniolisib in the EU and the expected approval timeline?
A: (Sijmen de Vries, CEO) We plan to respond to the CHMP by January 2026. We expect a positive opinion by the end of Q1 2026, with potential market entry in Germany by the end of Q2 or beginning of Q3 2026.
Q: What is the process for getting diagnosed patients in the US enrolled in Joenja therapy?
A: (Stephen Toor, Chief Commercial Officer) The process involves genetic testing, working with our patient services, and securing payer approval. Our approval rate is 98%, although some patients face delays due to other medical conditions or managed care system complexities.
Q: Can you provide a timeline for the Phase 2 study of leniolisib's second indication and the subsequent Phase 3 trial?
A: (Sijmen de Vries, CEO) We expect insights from the Phase 2 study by the end of next year. The timeline for the Phase 3 trial will be formulated based on these insights.
Q: What is the expected timeline for regulatory approval in Canada and Australia for leniolisib?
A: (Sijmen de Vries, CEO) We anticipate regulatory action in 2025, although the exact timing within the year is not specified.
Q: How do you plan to manage the potential impact of new competitors on RUCONEST?
A: (Sijmen de Vries, CEO) We are confident in RUCONEST's unique position and consistent response rates. While some patients may try new competitors, RUCONEST's ability to address all three pathways of hereditary angioedema attacks makes it a reliable choice for severely affected patients.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
