Pharming Group N.V. Q3 FY2024 Earnings Call

Good day, and thank you for standing by. Welcome to the Pharming Group NV 3Q 2024 Results Conference Call and Webcast. At this time, all participants are in a listen-only mode. After the speakers' presentation, there will be a question-and-answer session. Please be advised today's conference is being recorded. I'd now like to hand the conference over to your first speaker today, Sijmen de Vries. Please go ahead.

Thank you very much, and good morning, good afternoon, ladies and gentlemen. Welcome to our results call. I'm happy to introduce my colleagues here, Stephen Toor, Chief Commercial Officer; Anurag Relan, our Chief Medical Officer; and Jeroen Wakkerman, our Chief Financial Officer. We will take you through the results and, of course, answer all your questions. So before I do that, I'd like to have the next slide and point you to the forward-looking statements because during this presentation, we will be making the usual forward-looking statements, which can differ significantly from future results. So having said that, I'd like to start with the next slide. You see a slide of my face there. And you're seeing, no doubt, the announcement that after 16 years here at the helm, which is actually precisely next week, I have informed the Board of Directors that I will not be available for reelection. The company is in great shape today, and I'm reaching a beautiful age of 65 next week. So it would be a good moment to hand over the helm to a successor who can continue to build the company, as we have been over the last few years, into this rare disease company that we are building as we speak with a great pipeline and a global geographical presence. Having said that, I would like them to go to the next slide because they see how we are going to build that. RUCONEST, obviously, on slide number 5, please. RUCONEST is, of course, the pillar of the company and has been for a long time. You see that the results of RUCONEST continue to grow with more prescribers and more patients using RUCONEST in this increasingly competitive hereditary angioedema market, resulting in a three-quarter results increase of almost $64 million and almost $173 million for nine months in 2023. In other words, RUCONEST delivers significant cash flows from which we can actually build that company and build that portfolio and commercial presence with, of course, Joenja, leniolisib, the first and only FDA-approved treatment for APDS, a new ultra-rare immune disorder where Anurag Relan will speak later on. The revenues are marching quite nicely for this ultra-rare launch, making $32 million in the first nine months of this year, which is, of course, an incredible growth versus last year, which is no mean feat since it's a new disease. We'll talk later about the ways how we find these patients and what's going on regarding the further exploitation of additional markets beyond the currently approved UK, US, and Israel. Last but not least, we continue to focus on extending the portfolio further with an emphasis on in-licensing or acquiring clinical stage opportunities and other rare diseases to leverage our commercialization presence in the US, UK, Europe, and future markets like Japan and Australia. When you see the next slide, you see the pipeline depicted visually; we're beginning to build that pipeline and are working hard to extend it with additional assets. I would like to show you another thing, and that's the next slide, where you see an enormous number of products available for hereditary angioedema patients. It's really good news for patients in the US that there are so many products available. There are a lot of prophylactic therapies available and many acute products as well. However, there is, of course, one outstanding product, and that is Ruconest. RUCONEST is a high-dose protein replacement therapy. All the products on the market are primarily used for the treatment of acute and breakthrough attacks; they are often not IV, which RUCONEST is. Our patients are confident to perform IV self-injections, while those products are, for instance, subcutaneous. There are also oral opportunities reviewed by the FDA and currently in clinical stages for acute attacks, tested in patients who are responsive to Firazyr, which is a significant difference in terms of the patient base we serve with Ruconest. Every patient that uses Ruconest does so not by choice but because they must, meaning they are not responding to Firazyr therapy. Hence, there is clear market segmentation. RUCONEST serves its own segment on the severe end; it remains crucial, as it blocks all pathways and serves that unique patient segment effectively. This explains our confidence in the ongoing strength and future of Ruconest. RUCONEST has delivered substantial cash flows, enabling us to develop further growth potential. I would like to hand over to Steven to talk about the commercial results. Steven, over to you, please.

Thank you, Sijmen. Good morning, everybody. If you could move to the next slide, please. Thank you. Sijmen did a really nice job of describing our patient population and why RUCONEST is used. As you can see on this slide, RUCONEST is the second most prescribed acute product in the US market. That really speaks to the product's features; it's an IV, it gives a bolus of C1, it's highly effective and quick-acting. As Sijmen said, that's critical for the patient population we serve that has a more severe form of HAE. We continue to grow our prescriber base; we have gained 57 new prescribers this year, heading towards 800 in total. This success is illustrated through leading metrics, which experienced double-digit growth this year, including new enrollments gaining momentum; over 100 in Q3, a 25% increase year-on-year. All of this translates into around 12% revenue growth so far this year, outperforming last year when we achieved 10% revenue growth. Moving to the Joenja launch, we achieved a strong initial start to our US launch, and that growth has steadied, which was expected as we approach significant inflection points. Over the 18 months since launch, we have found approximately 50% of the known patients, of which 25% are pediatric. Among eligible adult patients, we currently have 93 on paid therapy with five pending insurance authorization this quarter, which has been going smoothly. Significant growth opportunities are upcoming: we anticipate the UK launch, an influx of US patients in 2025, and pediatric patients in 2026. This translates to $32 million in sales year-to-date. We continue to drive growth opportunities beyond APDS with our teams working on life cycle management opportunities for leniolisib with significant potential value to our customers.

Thanks, Steve. Let's start by reviewing some details about our activities in APDS. We have multiple strategies to help find and diagnose new APDS patients. First, on the education front, we continue to present new data on APDS, highlighting the seriousness of the condition and the use of leniolisib in these patients, including presentations at recent conferences, like the European Society for Immune Deficiency meeting in France. We also have programs to help diagnose new patients, including sponsored no-cost testing and family testing initiatives. Importantly, there are a significant number of patients with variants of uncertain significance (VUS) who may actually have APDS. We are conducting a functional testing study and a large-scale project that analyzes all possible variants in the relevant genes, with expectations that 20% of patients with VUS could turn out to have APDS, potentially doubling our diagnosed patient population. On the next slide, we see the sources of future growth for Joenja. We are addressing the CHMP's remaining CMC request and have positive clinical benefit and safety conclusions for leniolisib in APDS patients. We are making progress in Japan and expect to file there in mid-2025. We are also handling numerous requests for leniolisib access in other markets and providing this through expanded access programs. Additionally, our pediatric study enrollment has been completed, with results expected shortly, allowing us to file for younger patients down to age four. We're keen to accelerate treatment access for the younger population, especially for a disease that starts in early childhood. Furthermore, we're investigating leniolisib in primary immune deficiencies where there is immune dysregulation. Currently, we are conducting a Phase 2 proof-of-concept study to assess leniolisib’s potential for various primary immune deficiencies, with significant unmet needs. We're excited about the potential here, as this group of disorders has a clinical basis, and we believe leniolisib can address a significant patient population. We’re ready to launch this clinical trial, with expectations for the first patients to be dosed shortly.

Thank you very much, Anurag. I'll first take you through the financial highlights for this quarter and then the year-to-date figures. Revenues in Q3 increased by 12%. RUCONEST grew by 6%, and Joenja by 73%, primarily driven by volume growth. Gross profit increased by $9.7 million, in line with revenues, with a slight increase in gross margin. Operating expenses rose due to ongoing investments in Joenja and increased payroll expenses. The operating profit amounted to $4.1 million compared to $1.9 million last year, primarily due to increased gross profit, offset by heightened operational expenses. We had a net loss of $1 million in the quarter compared to a net profit of $3.4 million last year, driven by higher finance expenses. Cash and equivalents grew from $161.8 million to $173.3 million, primarily due to positive cash flows from operations. Now, moving on to the figures for the first nine months of the year—the total revenues rose by 25% to $204.5 million. RUCONEST revenues were up 12% at $172.6 million, while Joenja surged by 210%. The gross profit increased by $35.3 million, mainly due to revenue growth. Operating expenses rose due to continuing investments and the expansion of the leniolisib franchise. The operating loss reached $15.3 million, reflecting increased expenses. The net loss for the first nine months was $14.7 million compared to a loss of $7.4 million last year, impacted by other income from the PRV and milestone payment from last year. Regarding operating expenses, we expect them to be stable moving forward. In terms of financial guidance for this year, we maintain our overall revenue range of $82 million to $95 million, projecting to finish near the midpoint. Joenja's growth remains a significant revenue driver, and we expect continued growth in patients’ paid therapy.

Thanks, Jeroen. Yes, and the outlook, as you heard from Jeroen, we expect to land in the middle of the range regarding total revenues. We anticipate significant growth in patient numbers due to the clarification of the US situation and the expected pediatric label expansion, both of which will act as major growth engines moving into 2026. Furthermore, we are excited about the Phase 2 trial recently initiated for a larger population with PID and immune dysfunction. We are actively pursuing business development opportunities and hope to update you on securing a deal soon. We’ll now open the floor for any questions that you may have for me or my colleagues. Thank you.

We will now take our first question. This is from the line of Sushila Hernandez from Kempen. Please go ahead.

Yes, thank you for taking my questions. I have a few on RUCONEST and Joenja, if I may. I'll start with RUCONEST. So on RUCONEST, again, over 100 new patient enrollments this quarter. Do you see refills happening from previous quarters? And also, how do you see the ODP market developing in the coming years in terms of size? Thank you.

Would you like to answer that one, Stephen?

Yes. Thank you, Sijmen. Yes, we do see refills, and they generally reflect the patient's course of disease. Some patients need them more frequently than others based on how often they experience attacks. But yes, many patients return for refills.

How do you see the ODP market developing in the coming years in terms of size?

In the ODP market, we've seen continued demand even with prophylactic therapies. Over 50% of patients still experience regular acute attacks. We haven't seen a huge decline, but we should note that with the new therapies entering the market, there may be some revenue shifts; however, the demand remains.

Okay. That's clear. And then on Joenja in the UK, how are your discussions with NICE progressing? Any additional information that you can share?

With NICE, the discussions are productive and ongoing, but given their confidentiality, I wouldn't want to comment further, except to say we’re engaging in good dialogue.

And in the US, how many of the 30 identified patients do you expect to convert to Joenja and within what timeline?

The speed at which patients begin therapy depends on their discussions with insurance providers. Most eligible patients will eventually go on therapy that want to be on it, as we have had no refusals. Different processes take place depending on the insurer, but most have gone smoothly.

Thank you. We'll now move to our next question. This is from Joe Pantginis from H.C. Wainwright. Please go ahead.

Hey everybody. Thanks for taking the questions. First off, Sijmen, I want to congratulate you on everything that you've done for the company and wish you the best of luck in your future endeavors. For Jeroen, could you provide any insights on the current plan for your remaining convertible bonds?

The old convertible bond is completely redeemed, and there is nothing left in the market. For the current one, we plan to keep it on the market without a plan for redemption at this time.

Got it. Can you describe any hurdles or headwinds you are currently facing for Joenja?

Stephen, would you like to take that one?

Certainly. We've seen the initial big wave of patients, and followed by a more steady growth pace as we tap into fewer eligible patients. There may be some delays in identifying new patients, but we have upcoming inflection points with pediatric patients and market launches which should help accelerate our growth. However, we do face the typical challenges of educating physicians and patients about a new disease, which we are addressing.

Thank you for those details. Regarding pipeline expansion and business development, can we expect mature discussions about potential deals?

That's a forward-looking statement, but indeed, we have been in several mature discussions. However, until a deal is signed, it remains speculative.

Thank you. We will now take our next question. This is from Simon Scholes from First Berlin. Please go ahead.

I was just wondering if you could give us an update on what percentage of the market for RUCONEST is derived from those using it as a rescue product. Which segment is growing more rapidly?

In the evolution of both markets, I believe they are fairly stable. RUCONEST is primarily used for patients on rescue therapy, as they have effective prophylactic options. Most of our patients likely use it for acute attacks.

Got it. Regarding pathways and patient attacks, do individual patients experience attacks through multiple pathways?

It is complicated. Patients deficient in C1 inhibitor may interact with various pathways, and the production of bradykinin can occur regardless of a certain pathway being blocked. Known targeted therapies treat some elements but may not capture the entire disease process, explaining why patients may still experience breakthrough attacks.

Thank you for the insight. As we continue to engage with our stakeholders and update on our progress, we remain dedicated to patient care.

We will now take additional questions from written ones.

Thank you for attending this results conference. We expect to land in the middle of our revenue guidance. We've discussed important growth opportunities in the future, including the effort to clarify the US patient population, expansion into pediatric indications, and further regulatory activities in various countries. We also initiated a Phase 2 trial for a larger patient population, which we regard as a crucial inflection point for future growth. Thank you for participating; we look forward to updating you in March during our full year results call.

Thank you. This concludes today's conference call. Thank you for participating, and you may now disconnect.