Pharming Group (PHARM) Wells Fargo 2024 Healthcare Conference summary

Business overview and product performance

• Ruconest has been on the U.S. market for nearly 10 years, showing 16% growth in the first half of this year compared to last year, with FY23 revenue of $227.1M and 1H24 revenue of $87.6M, driven by increased patient enrollments and prescriber growth.

• Ruconest is a recombinant C1 esterase inhibitor for acute hereditary angioedema attacks, serving patients not responsive to bradykinin/kallikrein pathway therapies and maintaining a strong market position for a unique patient subset.

• No lifecycle management or market expansion plans for Ruconest; focus remains on the U.S. market.

• Regulatory exclusivity for Ruconest expires in 2026, but significant barriers to entry are expected to limit competition.

• RUCONEST® remains a key therapy for acute HAE attacks, with strong U.S. demand and a high rate of single-dose effectiveness.

Joenja (leniolisib) launch and growth strategy

• Joenja launched for APDS, an ultra-rare immune disorder, with about 91 of 150 eligible U.S. patients on therapy after five quarters, generating $20.7M in H1 sales (44% growth over H2 last year), and is the first and only FDA-approved treatment for APDS.

• Patient finding strategies, including genetic and family testing and VUS validation, are expanding the diagnosed APDS population, potentially increasing the U.S. patient pool to 500+.

• Pediatric label expansion is expected, with trials for ages 4–12 nearly complete and submission planned for early next year, potentially accelerating growth in 2025–2026.

• European and Japanese launches are targeted for 2026, with early access programs generating revenue in France and regulatory submissions underway in the U.K. and Japan; approvals anticipated in Israel (received), U.K. (Q4 2024), and Australia (2025).

• Joenja pricing is set at $566,000 per year (net ~$500,000), with U.S. pricing at $47,220 per 30-day supply, high adherence rates (~85%), a 15% GTN discount, and only cost-of-living price increases anticipated.

Pipeline expansion and future outlook

• No current competition for Joenja in APDS; leniolisib is being developed for PIDs with immune dysregulation, targeting a population of ~5 per million, with a Phase II trial underway and regulatory feedback sought for a third PID indication.

• Phase 3 for the new indication could start in 2026, with potential launch by 2027–2028.

• Joenja demonstrated significant clinical benefits in APDS, including reduced lymphadenopathy, increased naïve B cells, and reduced splenomegaly, with a favorable safety profile and long-term tolerability.

• Pharming is actively seeking additional rare disease assets with clinical proof of concept, focusing on immunology, hematology, respiratory, and gastroenterology, aiming for launches within three to four years.

• Financially strong position with expected 2024 revenues of $280–295 million (14–20% growth), gross profit for 1H24 of $113.3M, cash and marketable securities of $161.8M as of June 30, 2024, and a focus on profitability and growth through new product launches.