Pharming Group N.V. Q3 FY2025 earnings call

• Strong Q3 performance with total revenues up 30% year-on-year and operating profit jumping to $15.8 million. - RUCONEST grew 29% YoY, fueled by new prescribers and patient enrollments, with a unique value proposition for severe HAE patients. - Joenja had 35% YoY revenue growth, with 25% YoY increase in patients on treatment and progress in finding new APDS patients. - Upgraded full-year 2025 revenue guidance to $360 million - $375 million from previous $335 million - $350 million. - Reduced G&A headcount to optimize capital deployment. - Pipeline progress: KL1333 for primary mitochondrial disease in registrational study, APDS pipeline advancements including FDA priority review for pediatric sNDA, and implications from Cell publication on APDS prevalence suggesting higher prevalence and broader symptoms.

In the third quarter, RUCONEST contributed significantly with revenue growth of 29% year-on-year, reaching $82 million, which is approximately 84.3% of the Q3 total revenue of $97.3 million. Joenja saw a 35% year-on-year revenue increase, generating $15.1 million, accounting for about 15.5% of Q3 total revenue. RUCONEST's growth was driven by new prescribers and patient enrollments despite the launch of a new oral therapy, while Joenja's growth was due to a 25% year-on-year increase in patients on treatment and success in finding new APDS patients.

• Raised full-year 2025 revenue guidance to $365 million - $375 million, implying 23% - 26% growth. - Expect total operating expenses between $304 million - $308 million, including $10.2 million nonrecurring Abliva acquisition-related transaction expenses and excluding ~$7 million restructuring costs. - Available cash and future cash flows expected to cover current pipeline and prelaunch costs. - Focus on profitable growth, efficient capital allocation, and improved financial transparency.

• Uncertainties in VUS reclassification processes for APDS patients. - Challenges in regulatory compliance and market acceptance for international rollout of Joenja. - Uncertainties surrounding APDS prevalence estimates as per Cell publication findings, which may impact patient identification and market potential.

Q: Given your more disciplined approach, what are your priorities for capital allocation? Can we expect another M&A transaction similar in size to Abliva? And second, how is your basket PID trial progressing?

A: Priorities for capital allocation include profitable growth and efficient deployment. No rush for M&A, will pursue value accretive opportunities. Basket PID trial progressing nicely, expected readout in second half of 2026.

Q: I just wanted to ask a question about the new formulation. If you could give any more color on this new pediatric formulation for the 1- to 6-year-old group? And if there's any specific manufacturing hurdles that you may need to clear for this formulation?

A: New pediatric formulation for 1-6-year-olds is granules. Manufactured them, did PK work, and engaged with FDA on formulation and study design, remains on track.

Q: Firstly, I just wanted to ask around your revenue guidance uplift, just confirming how much of this comes from better-than-expected RUCONEST versus Joenja. And then in particular, for RUCONEST, how you're expecting that to develop into Q4 and 2026, given that you're not seeing much pressure from competition and also continue to see increases in prescribers and patients there? My second question is on Joenja and the international rollout. It seems that this is contributing around 11% this quarter. So curious to hear a bit more about how that's evolving and how you expect that mix to evolve over time? And then thirdly, just around the RUCONEST withdrawal from ex U.S. markets. Are you able to comment a bit on the savings you'll make from this and where you plan to redirect those resources?

A: Revenue guidance uplift driven by strong performance of both RUCONEST and Joenja. RUCONEST expected to continue growing with more severe patients. Joenja international rollout planned in 8 markets, focus on reimbursement. RUCONEST withdrawal has minimal financial impact, resources redirected to growth initiatives.

Q: Firstly, I just wanted to ask around your revenue guidance uplift, just confirming how much of this comes from better-than-expected RUCONEST versus Joenja. And then in particular, for RUCONEST, how you're expecting that to develop into Q4 and 2026, given that you're not seeing much pressure from competition and also continue to see increases in prescribers and patients there? My second question is on Joenja and the international rollout. It seems that this is contributing around 11% this quarter. So curious to hear a bit more about how that's evolving and how you expect that mix to evolve over time? And then thirdly, just around the RUCONEST withdrawal from ex U.S. markets. Are you able to comment a bit on the savings you'll make from this and where you plan to redirect those resources?

A: Revenue guidance uplift driven by strong performance of both RUCONEST and Joenja. RUCONEST expected to continue growing with more severe patients. Joenja international rollout planned in 8 markets, focus on reimbursement. RUCONEST withdrawal has minimal financial impact, resources redirected to growth initiatives.

Q: I've just got 2 questions. So you recorded a gross margin of 92.7% in the third quarter, which I think compares with 90% in H1 and 89% in '24. I was just wondering how we should think about the gross margin in your existing markets going forward? So do you think this 93% is sustainable going forward? And then you also say in the presentation, I mean, you said you've got -- you've seen an increase in more severe frequent attack patients. Does that mean that these more severe frequent attack patients are actually increasing as a proportion of the overall number of patients?

A: RUCONEST serves more severe patients, which is reinforcing. Gross margin sustainable with Joenja share growth, but specifics on forward-looking not given.