Pharming (PHAR) Q3 2026 Earnings Call Transcript

Image source: The Motley Fool.

DATE

Tuesday, Oct. 27, 2026

CALL PARTICIPANTS

• Interim Co-Chief Executive Officer - Kenneth Lynard
• Interim Co-Chief Executive Officer and Chief Commercial Officer - Leverne Marsh
• Chief Medical Officer - Anurag Relan

TAKEAWAYS

• Total Revenue -- $117.6 million, representing 21% year-over-year growth driven by the return of RUCONEST to positive performance and continued expansion of Joenja.
• RUCONEST Global Revenue -- $93.8 million, growing 14% year over year as U.S. demand stabilized at prior-year levels.
• Joenja Global Revenue -- $23.8 million, increasing 58% year over year supported by patient growth in the U.S. and new international market launches.
• Operating Profit -- $29.9 million, up 89% year over year due to revenue growth and disciplined expense management.
• Adjusted Operating Profit -- $35 million, representing a significant increase from $16 million a year ago after excluding a $5.1 million one-time manufacturing-related inventory impairment.
• Full-Year 2026 Revenue Guidance -- $380 million to $390 million, reflecting an expected 14% annual growth rate.
• Full-Year 2026 Operating Expense Guidance -- $310 million to $315 million, adjusted downward to reflect approximately $9 million in structural G&A savings.
• Net Profit -- $22.4 million, compared to $7.5 million in the prior-year period, benefiting from a favorable foreign exchange result.
• Operating Cash Flow -- $32.4 million, contributing to a quarter-end cash position of $190.9 million.
• U.S. Joenja Patient Count -- 136 patients on paid therapy, an increase of 17% year over year and four patients since the end of the second quarter.
• International Joenja Patient Count -- 75 patients receiving paid therapy, including 30 in launch markets and 45 through paid access programs in other regions.
• APDS Patient Identification -- 304 identified patients in the United States and 1,060 patients globally as of Sept. 30.
• RUCONEST U.S. Revenue Guidance -- an expected decline of approximately 3% for the full year at the midpoint, factoring in early-year volume declines.
• FALCON Study Enrollment -- management expects 145 of the planned 180 participants to be enrolled by the end of 2026, with completion projected for the first half of 2027.
• Incremental R&D Investment -- approximately $35 million for the full year, focused on advancing the clinical pipeline for leniolisib and nepasimone.
• Gross Margin -- projected at 88% for the full year, including a total of $10 million in manufacturing inventory impairments recorded across the second and third quarters.
• U.S. Pediatric APDS Opportunity -- management identified over 60 children aged 4 to 11 in the U.S., with 38 immediately eligible for newly approved doses.

Need a quote from a Motley Fool analyst? Email pr@fool.com

RISKS

• Lynard noted that third-quarter RUCONEST revenue "included an early seasonal increase in channel inventory and fourth quarter revenue may reflect inventory timing," potentially impacting near-term reported growth.
• Marsh stated that Joenja international revenue benefited from ordering patterns that were "temporary," resulting in $2 million to $3 million of revenue pull-forward from the fourth quarter into the third quarter.
• Marsh warned that the company is anticipating the entry of a "new oral competitor that, to date, has a PDUFA date at the end of April," which may impact the RUCONEST patient base in 2027.

SUMMARY

Pharming Group N.V. (NASDAQ:PHAR) reported 21% year-over-year revenue growth for the third quarter, supported by a stabilization in RUCONEST demand and the continued commercial ramp of Joenja. Management tightened full-year revenue guidance while lowering operating expense projections, citing structural savings and operational discipline during a period of leadership transition. The company expanded Joenja's geographic footprint with launches in Germany and Japan and received U.S. Food and Drug Administration approval for a pediatric label expansion, which began shipping in October. Clinical efforts are focused on expanding leniolisib into larger patient populations with common variable immune deficiency (CVID), with top-line data expected to inform the design of a potential registrational study.

• Management stated that the search for a permanent Chief Executive Officer is ongoing, with the board conducting a formal search following the departure of Fabrice Chouraqui in September.
• The company reported that Japan is the first market where Joenja is commercially available for children aged 4 to 11, following a launch in August.
• Relan explained that the company enriched its CVID Phase 2 study for a specific endotype characterized by lymphoproliferation to better align with the mechanism of action of leniolisib.
• Marsh reported that RUCONEST demand was unaffected by new treatments launched in 2025, maintaining over 90% of its active patient base.
• Management indicated that the bottleneck for FALCON study enrollment was primarily investigator site activation, which has been resolved with over 50 sites now recruiting.

INDUSTRY GLOSSARY

• APDS: Activated PI3K delta syndrome, a rare primary immunodeficiency caused by mutations that overactivate the PI3K delta pathway.
• CVID: Common variable immune deficiency, a condition characterized by low levels of protective antibodies and an increased susceptibility to infections.
• HAE: Hereditary angioedema, a rare genetic disorder that causes episodes of severe swelling in various parts of the body.
• PDUFA: Prescription Drug User Fee Act, the date by which the U.S. Food and Drug Administration is expected to complete its review of a drug application.
• PID: Primary immunodeficiency, a group of disorders in which part of the body's immune system is missing or does not function properly.
• PI3K delta: Phosphoinositide 3-kinase delta, an enzyme that regulates immune cell development and activity.
• rhC1INH: Recombinant human C1 esterase inhibitor, a protein therapy used to treat acute HAE attacks.

Full Conference Call Transcript

Operator: Good day, and thank you for standing by. Welcome to the Farming Group NV Q3 26 Results Conference Call and Webcast. After the speakers' presentation, there will be a question and answer session. To ask a question during the session, you will need to press *11 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, please press 11 again. Please be advised that today's conference is being recorded. I would now like to hand the conference over to your speaker today, Kenneth Lynard Please go ahead.

Kenneth Lynard EMBA: Thank you, operator. Good morning and good afternoon, everyone. And thank you for joining PharmEx third quarter 26 call. I am joined today by Leverne Marsh, my fellow interim co-CEO, our chief commercial officer, and Dr. Anurag Relan, our chief medical officer. After my opening remarks, Leverne, will review our commercial performance, Anurag will cover R&D, and I will return to take you through the financials and outlook before we open the line for questions. As you know, on September 29, Fabrice Chouraqui stepped down as CEO and the board appointed Leverne, and me as interim co-CEOs while it conducts a formal search for a permanent successor. As of now, there are no additional updates to report.

And despite these changes, our strategy pipeline, and commitment to patients remain unchanged. Before we begin, please note that we will be making forward looking statements on this call. These are based on our current expectations and plans, and actual results may differ materially. Please refer to the full statement on this slide. We are pleased to report a strong third-quarter performance. Before I go further, I want to comment on why we decided to report results today. Rather than on November 5 as originally scheduled. Because our third quarter results came in above analyst consensus, we did so on October 22 in a pre-release with top-line revenues and operating expenses only.

We appreciated that once these numbers were public, investors would want to see the full details behind the numbers as soon as possible and have the opportunity to ask questions. As a result, we moved the date up for our full third quarter disclosure to today. The change in date is not related to any transaction or restructuring of the company. Getting back to this slide, as many of you know, as many of you who have followed farming know our strategy rests on 3 pillars. RUCONEST continues to provide a durable source of cash flow and I am pleased to report it has returned to growth this quarter.

Joenja, a high growth asset still early in its life cycle, is expanding geographically and with a new pediatric indication. And a high value clinical pipeline with 2 potential billion dollar opportunities. leniolusib and broader primary immunodeficiencies, including CVID, and nepasimone in mitochondrial DNA driven primary mitochondrial disease. Turning to the quarter in more detail. Total revenue grew 21% to $117.6 million ahead of market expectations. This quarter, RUCONEST returned to revenue growth Joenja sales grew 58%, and our operating profit increased by 89%.

We are also tightening our full year revenue guidance to $380 million to $390 million and lowered our operating expense guidance to $310 million to $315 million Both of our commercial products contributed to growth, RUCONEST returned to growth in the quarter with U. S. Demand back to prior year levels in volume terms. Joenja continued to grow strongly in The US and internationally. That performance carried through to a significant increase in operating profit and strong operating cash flow. Leverne, will take you through each product shortly and I will come back to the full financials and our guidance later in the call.

At the start of the second half, we laid out 3 goals for 2026, and I am happy to report that we made notable progress on each of these during the quarter. First, RUCONEST, which stabilized demand and returned revenue to growth in the quarter ahead of our own projections and, furthermore, saw stabilization in underlying demand. Second, Joenja and APDS geographic and pediatric expansion continued. We launched in Germany in July and Japan in August, with Japan becoming the first market where Joenga is commercially available for children aged 4 to 11.

In the U.S., the FDA approved our pediatric label expansion on September 11, ahead of the PDUFA date and shipments for this newest market segment began this month. Third, advancing leniolusib in conditions beyond APDS. On October 14, we presented positive phase 2 top line data and genetic PIDs with immune dysregulation and we expect to report top line data from our CVID study in November. These updates represent substantial growth for leniolusib in the years ahead. With that, I will hand the call over to Leverne, to discuss our commercial performance. Leverne?

Leverne, Marsh: Thank you, Kenneth. Good morning. Good afternoon, everyone. I will provide an update on our third quarter commercial performance, and the growth opportunities ahead for RUCONEST and Joenja. Starting with RUCONEST, the third quarter marked a return to revenue growth for RUCONEST. As you heard from Kenneth, RUCONEST revenue reached $93.8 million an increase of 14% year over year. Importantly, we have stabilized underlying US demand with volume returning to prior year levels. This compares favorably to declines we reported in Q1 and Q2, and we expect this positive trend in demand to continue into Q4. We are encouraged by the stabilization that came only 1 year after the introduction of the first oral on demand treatment.

And as we communicated previously, RUCONEST demand has not been impacted by any new treatments that were launched in 2025. We continue to maintain the overwhelming majority of our active patient base with particularly strong retention amongst patients experiencing more or frequent or severe HAE attacks. And we continue to attract new patients, and new prescribers. To put our revenue performance into context, though, there is 1 additional factor worth highlighting. RUCONEST's third quarter revenue benefited from a seasonal increase in channel inventory that came a quarter earlier than expected. This contributed approximately 9 percentage points of the quarter's 14% year over year growth.

While these inventory levels can fluctuate, the underlying patient demand remains our most meaningful indicator of the franchise performance. And so the stabilization that we have seen in RUCONEST demand reinforces our confidence in its resilient and differentiated position within the HAE treatment landscape. While treatment options are expanding choices for patients, we know that HAE is not a 1-size-fits-all disease. Patients who are experiencing more frequent or more severe attacks often have very distinct treatment needs, and RUCONEST continues to play an important role for this population. By replacing deficient or dysfunctional C1-esterase inhibitor, RUCONEST targets the root cause of HAE across all pathways, offering high efficacy and a fast complete resolution of attacks.

We believe that these attributes are particularly important for patients with a higher disease burden and helps explain the strong patient retention that we have seen. More than a year after the launch of the first oral on demand competitor, the overwhelming majority of RUCONEST patients have remained on therapy. And physicians continue to prescribe RUCONEST for new patients, with more severe disease. In addition, RUCONEST's highly specialized manufacturing process may present challenges for direct biosimilar competition, and these factors combined further support our confidence in its long term commercial position. Now turning to Joenja.

We continue to build on our commercial momentum in the third quarter, with global revenue increasing 58% year over year to $23.8 million This is driven by continued U.S. patient growth, and an increased contribution from international markets in the U.S., revenue increased 19% to $15.9 million supported by continued growth in patients on therapy, We ended the quarter with 136 US patients on paid therapy, up 17% year over year. also an increase of 4 patients from the end of the second quarter. Internationally, our revenue increased to $7.9 million compared with 1.7 million a year ago. And right now, the international business represents approximately 1-third of Joenja's global revenue.

This growth reflects our expanding international presence, including launches in Germany and Japan, alongside continued penetration in the U.K. At quarter-end, 75 patients were receiving paid therapy internationally, including 50 patients in launch markets, and 45 patients through paid access programs in other markets. it is worth noting that international revenue for Joenja also benefited from ordering patterns in certain markets, that we consider temporary. As a result, there is a $2 million to $3 million in earlier revenue recognition that benefited Q3 we originally expected in Q4. Importantly, the growing number of patients receiving treatment across markets demonstrates continued progress in expanding JosephAnja's global commercial reach.

And additionally, our third quarter results do not yet reflect any revenue contribution from the newly approved U.S. pediatric doses which began shipping in October. This brings me to our next slide. A notable next phase of Joenja's US growth is now underway. Following FDA approval of the new pediatric doses in September. This approval expands access to Joenja for eligible children aged 4 to 11, an important population that was previously outside of the approved age range. On September 11, as Kenneth mentioned, the FDA approved our new drug application for the 40 and 50 milligram twice daily dose for children. aged 4 to 11, weighing at least 27 kilograms.

This happened ahead of the anticipated October 24 PDUFA date. Our shipments began in October, marking a significant milestone in our efforts to expand treatment across younger patients with APDS. We are also pursuing further expansion of our pediatric label, and in July, we submitted a separate supplemental application for the lower doses for children aged 4 to 11 between 13 and less than 27 kilograms. The FDA has accepted the application with priority review and we have an assigned PDUFA date of January 30, 2027. To date, we have identified over 60 children aged 4 to 11 with APDS in the United States.

The newly approved doses address 38 of these patients, with remaining children potentially becoming eligible under the lower dose application if approved. We have seen a strong start to the pediatric launch in the U.S., and we are expecting an initial bolus of new patients on therapy throughout Q4. In the range of 15 to 20 patients on therapy, And we look forward to providing an update on patients receiving treatment when we report our full-year results early in 2027. Looking ahead, we see several growth drivers that can build on Joenja's established commercial foundation in APDS in the U.S., we expect continued patient finding, increased genetic testing, and expanded treatment access for younger pediatric patients.

As of September 30, we had identified 304 patients with APDS in the United States and 1.06 thousand globally, reflecting continued progress in identifying individuals living with this rare disease. Internationally, our focused geographic expansion can result in a doubling of the addressable APDS opportunity. Joenja is now approved in all our target launch markets with Germany launching in July, Japan in August, building on our established presence in the U.K. Beyond APDS, our clinical development programs are exploring leniolumab's potential in significantly larger populations of patients with immune dysregulation.

For common variable immune deficiency or CVID with immune dysregulation, where we plan to pursue a registrational trial there are more than 13 thousand patients in the United States, a 26-times prevalence compared to APDS. Anurag will review our clinical programs with leniolumab in just a few minutes. While this remains investigational, it certainly represents a potentially significant expansion of Joenga's addressable opportunity. And together, these growth drivers provide a clear path to build on Joenja's existing foundation with continued expansion in APDS, to the potential treatment of substantially larger patient populations in CVID. Ambition is to establish Joenja as a broader immunology franchise with blockbuster potential.

And with that, I will turn the call over to Anurag to discuss our clinical development programs.

Anurag Relan: Thank you, Leverne. Before turning to our new PID programs, let's quickly review some background. Specifically, that PI3K delta is a central regulator of immune cell development and activity. In APDS, where the pathway is overactive, Joenja has shown durable reductions in lymphoproliferation and has a well established safety profile with up to 7 years of treatment. Now let's turn to what is new. Our first clinical data for leniolusib beyond APDS and what to expect from our CVID read-out in November. The rationale for going beyond APDS is that there are other genetically defined PIDs and CVID patients with immune dysregulation who show similar PI3K delta driven biology.

As a result, these populations share the same clinical manifestations we see in APDS. Namely, lymphoproliferation, autoimmunity, gastrointestinal disease, and pulmonary disease. The 2 Phase 2 studies we are running cover patient populations that are substantially larger than APDS as Leverne, just mentioned. But they share a similar unmet need. And the prevalence, as you heard, is 26 times greater than the APDS prevalence and this includes the overlap with the genetic PID population. The 2 Phase 2 studies that I have described have similar endpoints and objectives that we have discussed previously, and you can see here on this slide.

They include spleen and lymph node size, blood cell counts, lung and liver involvement, and patient and clinician reported outcomes as well as biomarkers. Together, these studies will inform our plans for a potential registrational study. Because PI3K delta is a shared driver across these populations, we continue to anticipate a single Phase 3 study in this broader CVID indication incorporating patients from both studies. Here's an overview of the genetic PID study results we presented at a late breaking abstract at the European Society for Immunodeficiency Conference 2 weeks ago. As you can imagine, are excited by these results as they are the first clinical data establishing proof of concept for leniolusib in primary immune deficiency beyond APDS.

The study enrolled 13 participants, 12 of whom completed treatment. The mean age in the study was 32.5 years, And importantly, 9 of the 13 participants also had a CDID diagnosis, which is relevant as we look ahead to the CVID read-out. The data showed that treatment with leniolusib resulted in improvements across a number of objective measures consistent with the PI3K-delta mechanism, including spleen volume, lymph node size, lung gas exchange, and liver stiffness. leniolusib was well tolerated with no new safety signals. Infections were the most common adverse event, as would be expected in a PID population, and 2 patients had serious adverse events considered unrelated to leniolizumab.

Also important, all 12 participants who completed the study chose to continue to enter the 3-year long term extension study. Which we see as a meaningful signal from both patients and physicians. Now looking ahead to the CDID readout, which we expect to report top-line results from next month. This is a 20-patient single arm open label multicenter study with CVID and CVID with immune dysregulation with or without a genetically identified cause. We will report across the same measures as in the previous PID study, including lymphoproliferation, cytopenias, lung and liver disease, biomarkers, and safety. Now the CDID population is more heterogeneous and many patients have no identified genetic cause.

So we will be looking for a consistent direction of benefit across measures and a dose to take forward. Together with the genetic PID data, the CDID results will inform a potential registrational study. In fact, we have already planned several regulatory interactions to discuss the endpoints and design of a phase 3 study including an end-of-Phase 2 meeting with FDA. Now turning to nepasimone, or KL1.33 thousand which is being developed to treat mitochondrial DNA driven primary mitochondrial disease, which is in a registrational-enabling study. This is a rare disorder in which mutations in DNA cause debilitating fatigue and muscle weakness.

With more than 30 thousand diagnosed patients in the EU, in the U.S., EU4, and the U.K., there is no approved disease modifying treatment. Nepasimone targets the underlying pathology by normalizing the NAD plus to NADH ratio and mitochondrial function. The primary endpoints of our pivotal FALCON study are improvements in fatigue and a measure of muscle weakness called the sit-to-stand test. Both endpoints have cleared a futility analysis at the interim look at the data, which was conducted after 40 patients had gone through 24-week treatment. The study continues to enroll patients with more than 50 sites now recruiting and where there is been a significant expansion in the U.S.

Based on the patients already screened, we expect that 145 of the planned 180 participants to be enrolled by the end of this year, and we expect enrollment to be complete during the first half of 27. Top line results are therefore expected approximately 1 year after enrollment is complete. And with that, I will turn it back to Kenneth.

Kenneth Lynard EMBA: Thanks, Anurag. I would now like to take a moment to walk you through the financial highlights of Q3 26 versus Q3 25. Our third quarter revenue was $118 million, up 21%, with Joenja up 58%, and RUCONEST up 14% year over year. And 30% sequentially. Operating profit was $29.9 million up 89%. Excluding a $5.1 million 1-time manufacturing-related inventory impairment, adjusted operating profit increased significantly to $35 million compared to $16 million a year ago. Operating expenses were $74.6 million in line with a year ago, and the result also benefited from higher other income mainly from received R&D tax incentives. Net profit was $22.4 million compared to $7.5 million last year, including a favorable foreign exchange result.

Operating cash flow was $32.4 million and cash and marketable securities rose $31.4 million in the quarter to $190.9 million driven by profitability and partially offset by normal activity driven working capital movements, including an increase in trade and other receivables. For the first 9 months of the year, revenue was $280.2 million up 4%. Joenja grew 45% to $55.7 million, and RUCONEST was 3% lower at $224.5 million. Adjusted operating profit was $38 million compared with $29.7 million driven by higher revenue and total operating expenses, 2.8% below the prior year.

Operating cash flow was $24.7 million year to date, with cash and marketable securities increased $9.8 million since year end. to $190.9 million, net of the $12.3 million settlement for the early termination of the DSP facility lease in the first quarter. For the full year 2026, we have tightened our revenue guidance to $380 million to $390 million, representing 14% growth. And lowered our operating expenses guidance to $310 million to $315 million representing 0% to 1% growth. That includes approximately $35 million incremental R&D investment and about $9 million of structural G&A savings.

Our full year guidance continues to assume that RUCONEST revenue will stabilize and return to growth in the second half of 26 and that Joenja will deliver significant and accelerating growth for the year. At the midpoint of our guidance range, RUCONEST U. S. Revenue is expected to decline approximately 3% for the full year. For Joenja, we continue to expect annual revenue growth in the mid-30s%, as compared to 29% growth in 2025 with international launches and the U.S. pediatric revenues in the fourth quarter contributing.

For the full year, we expect cost of goods sold to be approximately 12% of revenue including the $10 million manufacturing inventory impairments recorded in Q2 and Q3, corresponding to a gross margin of 88%. With 9 months revenue of $280.2 million our guidance implies Q4 revenue of roughly $100 million to $110 million As Leverne noted, third quarter RUCONEST revenue included an early seasonal increase in channel inventory and fourth quarter revenue may reflect inventory timing. For Joenja, the $2 million to $3 million early international revenue recognition we saw in Q3 was pulled forward from Q4.

Finally, our cash position of $190.9 million, together with future operating cash flows, is expected to cover our current pipeline and prelaunch costs. In closing, this quarter showed what our business can deliver. RUCONEST returned to growth in the quarter with stable US demand and a resilient patient base and remains a durable source of cash flow. Joenja grew 58% and is still early in its life cycle. New pediatric doses began shipping in the U.S. earlier this month. With a strong start to the launch We have a PDUFA date of January 30, 2027 for lower pediatric doses, and our launches in Germany and Japan are well underway.

Moving to our pipeline, leniolusib delivered positive data in genetic PIDs, and we expect top line CVID data in November which will shape our path to a potential registrational study. Falcon enrollment continues to progress. Towards completion in the first half of 27. We are running the company with discipline, Operating expenses were flat in the quarter, Operating profit rose 89%. And we ended the quarter with $191 million in cash. Leverne, and I together with the leadership team, and the board, are focused on execution consistent with our strategy. With that, operator, please open the line for questions.

Operator: Thank you. As a reminder, to ask a question, you will need to press 11 on your telephone, and wait for your name to be announced. And our first question comes from the line of Benjamin Jackson from Jefferies. Please go ahead. Your line is open.

Benjamin Jackson: Great. Thank you for the question or thanks for the presentation. Very clear across the full spectrum, I guess. Just a couple from me. The first, I just wanted to touch on the RUCONEST patient initiation you saw during 3Q. I guess, we look historically, the third quarter is typically seasonally lower. But relative to historical, still probably a lower value than perhaps we anticipated, and we made a fairly big deal about it being a leading indicator back at 2Q. So perhaps you can provide a little bit of color around the I think it was the 43 patient additions you saw this quarter and how that is tracking in the first month of the fourth quarter.

And secondly, any color you can provide in and around the CEO search and how the board is thinking about a profile for this individual Are they looking both internally and externally? And kind of what well, I mean, just making that profile a little bit more clear to us in terms of what they are looking for. And then the third and final 1 for me, just if you could add any color behind the slower Falcon recruitment. Be interested if there is something specifically about patient identification or competing trials running at the same time that perhaps is impacting the timelines versus your prior expectation? Thank you.

Leverne, Marsh: Great. Thanks, Benjamin, for the question. I think your first remark is important, right, because we do see new patient enrollments naturally vary from quarter to quarter. And exactly as you said, we typically see Q3, if we look back, somewhat softer than Q2. So that pattern certainly continues. However, we do not necessarily view a single quarter as a reliable indicator of the long term demand trend.

And so what I am asking everyone to pay attention to is that RUCONEST is a combination of new enrollments the retention that we have seen, and we have maintained over 90% of our active patient base still 1 year after an oral competitor, We also review the retention amongst high burden patients, which tend to be our high vial users. And in this area, we disproportionately retain high burden patients, patients who suffer from severe attacks, that supports what we see in the volume growth. So when we talk about stabilizing demand for RUCONEST, and vial volumes returning to the same level it was in Q3, those are all the aggregate factors that are contributing to demand.

So new enrollments, a disproportionate retention of high burden patients, and patients returning back to RUCONEST. Based on what we have seen in Q3, on the back of the clients that we reported in Q1, Q2 in volume demand, Q3 then returned to volume stabilization prior year levels, and we expect that strong momentum in demand to continue into Q4.

Kenneth Lynard EMBA: Benjamin, let me. Thanks for the question regarding the CEO, the leadership transition. So let me take the opportunity to reiterate that you know, despite the leadership transition announced there are no changes to our strategy pipeline or the commitment to patients. That all remain unchanged. Leverne, and I are as interim co CEOs driving the organization to execute on all the priorities that we have. Talked about earlier and which we have given an update today on the--on-- and that we will continue to do Meanwhile, the board is conducting a formal search for a permanent successor. And we do not really have any further updates on that at this point of time.

But with the commitment that in due time, of course, the company will provide updates as to the progress and the conclusion of that of that process.

Anurag Relan: Hi, Benjamin. On the question of FALCON enrollment, so we. As I mentioned, we have more than 50 sites now recruiting. With many of those sites in the U.S., and the year end estimate is actually based on patients who have already gone through or entered the screening process. The main bottleneck really was site activation, and that is largely now Investigator engagement and patient engagement actually remains very high. So the sites that we have gotten up and running just in the past few months are act very active, and we have seen a very high number of, enrollments or screenings come just in the past couple of months.

So I think we really have this main bottleneck now largely behind us.

Leverne, Marsh: Great. Thank you for all of that.

Operator: Thank you. We will now move on to our next question. And our next question comes from the line of Sushila Hernandez from Van Lanschott Kempen. Please go ahead. Your line is open.

Sushila Hernandez: Yes. Thank you for taking my questions. I also have a few. At the moment, there are 30 patients on paid therapy in international launch markets. What is the Joenja opportunity for Japan, Germany, and The UK? And could there be a trigger for further acceleration? Then a second question on the CVID study. What should we expect from this study to read out? What will be a meaningful reduction in the spleen volume? And then the third, just to clarify, you mentioned available cash coverage prelaunch cost. Is this for KL1.33 thousand? Or would you need additional cash? Thank you.

Leverne, Marsh: I will take the first question. Thanks. You shall allow me-- Joenja As you say, we have 30 patients on therapy in our launch markets, and that is in addition to 45 patients in non launch markets. so they are in international markets on a named patient basis In terms of the breakdown by country, because the launches in Japan and Germany is so early, we will not be disclosing country level on therapy quite as yet.

What I can say is that we have seen a really, really healthy uptake both in Germany and in Japan on the back of established presence in The UK, and future catalysts for growth in these 3 launch in particular, are a for The UK, an expansion in the pediatric population, and the pediatric indication for the higher doses similar to what we have now in The US. So that is 1 growth catalyst. And then continued progress both in Germany and Japan as we engage physicians, as we educate physicians on the positioning of JosephAnja for patients with APDS. And so those 2 key elements for Germany and Japan is going to be really critical.

Anurag Relan: Hi, Sushila. So on the question of CVID and what we are looking for here is we are looking to see if there is a direction of benefit across lymphoproliferation cytopenias, and the end organ measures. So, you know, take a look back at what we have done in APDS and the what we have seen is that there have been consistent improvements in lymph proliferation. So we have seen spleen volumes decrease. We have seen lymph nodes decrease in size. Of course, lymph node reduction was the primary endpoint in the APDS study, so obviously important from a regulatory standpoint too.

We have also seen now in the genetic PID study that just read out, we have seen a consistent, improvement there also. In lymphoproliferation. So seeing an improvement in lymphoproliferation would be important. Of course, it is expected based on this mechanism, but we need to establish the that can also be, seen in patients with CVID. So I think that is probably 1 of the most important things that we will be looking for. Obviously, these patients, as I said earlier, are heterogeneous. So they have other manifestations of the disease. So the cytopenias and other end organ measures we will be looking at.

We will also be looking at clinician reported and patient reported measures because we know these are important obviously to patients, but also to regulators. And then lastly, we are looking at tolerability and safety. And trying to determine, what a what the dose should be to move forward in a larger study.

Kenneth Lynard EMBA: And, Sushila, maybe just on the on the cash aspect So, no, we do not need to raise additional financing. The cash position at the end of September, together with the future cash flows that we are projecting are covering, let's say, all our current pipeline costs. And also prelaunch costs related to those assets. Thanks for the questions.

Sushila Hernandez: Okay. that is clear. Thank you.

Operator: Thank you. We will now move on to our next question. And our next question comes from the line of Joe Pantginis from H. C. Wainwright. Please go ahead. Your line is open.

Joseph Pantginis: Everybody, good morning. Thanks for taking the questions and all the details today. So I have a question here, but how would you envision the integration of Joenja when PID and CVID indications potentially get approved. Namely, could this be a potentially easier integration because of the current physician base and also what kind of physician or specialties could be expanded into? Thank you.

Anurag Relan: Great question, Joe. And in fact, the doctors that diagnose and see APDS patients are largely the same physicians that see patients with CVID and these genetic PIDs. These are primarily immunologists and they have a lot of experience in immune deficiency. So it is really the same group of doctors that are seeing all 3 groups of patients. Thank you.

Leverne, Marsh: And then in terms of execution, Joe, obviously, with Joenja, we have established commercial medical affairs market access, patient services capability, in The US with a very successful Joenja launch in APDS. All of those capabilities will be transferable to CDID. So calling on the same physicians, engaging payers with the value proposition for CVID. Expanding our patient services program to accommodate these CVID and then continued commercial and medical affairs execution. So excited to be able to build on those capabilities that have served us well for Joenja in APDS.

Joseph Pantginis: Great. Thanks for the color.

Operator: Thank you. We will now move on to our next question. And our next question comes from the line of Simon Scholes from First Berlin. Please go ahead. Your line is open.

Simon Scholes: Yes. Good morning and good afternoon. Thanks for taking my questions. I have just got 1 actually. And so you mentioned that in Q3, your numbers benefited from inventory build, an early inventory build, by 1 specialty pharmacy. Might it not be the case that in Q4, your numbers benefit from inventory build by other specialty pharmacies?

Leverne, Marsh: Hi, Simon. Thanks for the question and astute observation. So, yes, Right? So what we are expecting to see typically in Q4 is specialty pharmacies increasing inventory, before year end, and we see that across the--and you are 100% right. We have seen 1 specialty pharmacy pull that forward into Q3. So while we are expecting, additional seasonal adjustments, as the rest of the channel adjusts in Q4. I would think about it in exactly as you say. RUCONEST components as you look forward to Q4 would be demand. So as we have stabilized our demand to prior year levels, recovering from the first and second quarter declines. We are expecting that momentum on demand to continue in Q4.

We are expecting some seasonal adjustment from the remainder of the channel, including this including the specialty pharmacy that just bought in. And then finally, as you think about revenue, accommodate for price increases, and some gross to net adjustments, and that will make up RUCONEST for Q4.

Simon Scholes: Okay. Thanks very much. that is very helpful.

Operator: Thank you. Thank you. Once again, to ask a question, you will need to press 1-1 on your telephone, and wait for your name to be announced. To withdraw your question, please press 11 again. Our next question comes from the line of Fanyi Zhong from OpCo. Please go ahead. Your line is open.

Fanyi Zhong: Thanks for taking my question. This is Fanyi on for Jeffrey Jones from Oppenheimer. For the question about RUCONEST, so given the guidance of 2026 revenues, it looks like you estimate a softer Q4 revenue for RUCONEST versus 3Q. So should we expect a similar revenue pattern next year with a softer first half followed by a stronger second half And for leniolumab, what would you define as a successful November with the CVID readout? And I have. I probably have 1 follow-up. Thank you.

Leverne, Marsh: All right. Thanks so much for the question, Peng. So, for RUCONEST, we are at midpoint of guidance, forecasting a 3% annual decline. Right? So you remember the Q1, Q2 declines. We have now seen a return to revenue growth in Q3 and a stabilization of demand in Q3. Now for Q4, the components will be as what I have discussed. We are expecting demand growth to continue. We are expecting that momentum on demand to continue. But we are expecting inventory adjustments in the fourth quarter that will impact revenue and that is the biggest factor now.

A seasonality perspective into next year, we are anticipating a new oral competitor that, to date, has a PDUFA date at the end of April. What we are planning for is a similar pattern of impact that we have seen with the first oral competitor, So, as we look at how the quarters have evolved since the berotralstat introduction, we are planning for a similar impact going forward. But the underlying business for us is really important. We have maintained 90% more than 90% of our active patient base since the introduction of that first oral competitor.

We have built back volumes to the previous year levels, and that is on the back of a disproportionate retention of high burden patients that tend to use more vials, and we continue to start new and engage new prescribers. So that is what--those are the different components that I would take into account for 2027 for RUCONEST.

Anurag Relan: Yeah. And, Fanyi, thanks for the question on CDID. I think it is important to remember that in CVID, we are enrolling patients with immune dysregulation. So not all CVID. So the population that we selected for the study is really enriched for a specific endotype. And immune dysregulation and CVID converges on this endotype, which includes this expanded population of certain B cells called CD21 low B cells, elevated levels of CXCL13, and most importantly, lymphoproliferation. Lymphoproliferation is manifest as enlarged lymph nodes and a big spleen. And what we have seen from the genetic PID data as well as the data, of course, originally in APDS is that leniolisib can positively impact lymphoproliferation, and we have seen that.

And so that is obviously going to be 1 of the important things that we are going to be looking for in the CVID data. But we know that these CVID patients have other manifestations too, so we are also going to be looking at what the response to those manifestations, including in the lung, as well as in the liver. So, lots of things to look at and try to see if we can positively impact and have a like I said earlier, sort of a consistent direction of benefit.

Fanyi Zhong: In these patients. Got it. Thank you so much.

Operator: There are no further questions at this time, so I will hand the call back to Kenneth for closing remarks. Thank you very much.

Kenneth Lynard EMBA: Yes, this indeed concludes our Q&A. Thank you all for joining us today and for your questions. We look forward to sharing our CVID results in November and updating you further with our full year results. Thank you everyone for joining, and have a good day.

Operator: This concludes today's conference call. Thank you for participating. You may now disconnect. Speakers, please stand by.

This article is a transcript of this conference call produced for The Motley Fool. While we strive for our Foolish Best, there may be errors, omissions, or inaccuracies in this transcript. Parts of this article were created using Large Language Models (LLMs) based on The Motley Fool's insights and investing approach. It has been reviewed by our AI quality control systems. Since LLMs cannot (currently) own stocks, it has no positions in any of the stocks mentioned. As with all our articles, The Motley Fool does not assume any responsibility for your use of this content, and we strongly encourage you to do your own research, including listening to the call yourself and reading the company's SEC filings. Please see our Terms and Conditions for additional details, including our Obligatory Capitalized Disclaimers of Liability.

The Motley Fool has no position in any of the stocks mentioned. The Motley Fool has a disclosure policy.