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Catalyst Pharmaceuticals delivered exceptional results for the full year 2025, surpassing revenue guidance with $589 million in total revenue, a 19.8% year-over-year increase. The company's flagship product, FIRDAPSE, reached $358.4 million, while its newest offering, AGAMREE, surged 154% to $117.1 million. This growth effectively offset the anticipated decline of FYCOMPA following the entry of generic competition. For 2026, Catalyst provided revenue guidance of $615 million to $645 million, reflecting continued confidence in its rare disease portfolio despite IRA-related gross-to-net headwinds. The company ended the year with a strong cash position of $709.2 million and a focus on expanding its portfolio through strategic business development. Key operational highlights included the implementation of AI-driven patient identification leads and pharmacy programs that improved treatment persistence by 12%. During the earnings call, management detailed a March 2026 trial date for pending IP litigation and expressed optimism regarding AGAMREE's adoption in younger DMD patients, noting a drop in the median age of new enrollees. Catalyst remains focused on maximizing the $1 billion addressable market for LEMS and advancing lifecycle management for AGAMREE while actively seeking value-accretive acquisitions in the rare disease space with peak sales potential of $500 million.

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Full Earnings Call Transcript — Q4 • 2026-02-26

Operator: Good morning, and thank you for standing by. My name is John, and I will be your conference operator today. At this time, I would like to welcome everyone to the Catalyst Pharmaceuticals Fourth Quarter and Full Year 2025 Financial Results Conference Call. [Operator Instructions] I would now like to turn the conference over to Mike Kalb, Chief Financial Officer.
Michael Kalb: Thank you. Good morning, everyone, and thank you for joining our conference call to discuss Catalyst's fourth quarter and full year 2025 financial results and business highlights. Rich Daly, President and CEO, will lead the call today; and Jeff Del Carmen, our Chief Commercial Officer and I will also present. Additionally, other members of our management team will be available for the Q&A. Before we begin, I would like to remind you that in our remarks this morning and in the Q&A session, we will make statements about expected future results, which may be forward-looking statements for purposes of federal securities laws. These statements reflect our current expectations, estimates and projections and do not guarantee future performance. They involve risks, uncertainties and assumptions that are difficult to predict and may not prove to be accurate. Actual results may vary from the expectations stated in our forward-looking statements. These forward-looking statements should be considered only in conjunction with the detailed information contained in our SEC filings, including the risk factors described in our 2025 annual report on Form 10-K filed yesterday, February 25, 2026, with the SEC. At this time, I'll turn the call over to Rich. Rich?
Richard John Daly: Thanks, Mike. Good morning, everyone, and thank you for joining us today. I'd like to begin with a review of 2025, which was another fantastic year for Catalyst before moving on to the plans we have set for 2026. 2025 was defined by notable growth as evidenced by another year of record revenues, execution of our strategy to maximize the value of our best-in-class commercial portfolio and at the center of all we do, personalized support for patients living with rare diseases. For the full year 2025, total revenues grew by 19.8% year-over-year to $589 million, exceeding our previous guidance, which was the upper end of our range of $565 million to $585 million and highlighting our ability to capitalize on market opportunities while maintaining operational excellence. Full year net product revenue for 2025 reached $588.8 million, an exceptional 20.3% increase over 2024. This is driven by a number of factors, most notably continued patient identification and market penetration. And as demonstrated by our 2026 guidance of total revenue between $615 million and $645 million, we are confident in the continued growth trajectory of our differentiated products. Let's begin with the 2026 forecast down by product, starting with our promoted products, FIRDAPSE and AGAMREE. FIRDAPSE guidance for 2026 is $435 million to $450 million, reflecting an increase of 21.4% to 25.6%. AGAMREE guidance of $140 million to $150 million, forecasting a 19.6% to 28.1% growth. And finally, FYCOMPA, $40 million to $45 million, which effective at the beginning of 2026 is no longer promoted as a result of generic competition that entered the market in 2025. Now let's take a closer look at our 2025 performance. Revenue for our flagship product, FIRDAPSE, was $358.4 million, an increase of 17% for the full year and 18% when comparing quarter 4 2025 to quarter 4 2024. FIRDAPSE remains the only evidence-based FDA-approved therapy for Lambert-Eaton myasthenic syndrome, or LEMS, a debilitating nerve muscle communication disorder that results in progressive weakness and fatigue. We are making significant headway in the 2 distinct markets for the product, idiopathic LEMS and cancer-associated LEMS, and we believe there is still significant opportunity for growth in both of these submarkets. Combined, we view the LEMS addressable market opportunity to be in excess of $1 billion. Jeff will cover the brand performance, driven by exciting initiatives that we believe will help our team deliver continued growth for FIRDAPSE. As you know, in 2025, we finalized settlement with 2 of the 3 first filers. One suit remains against Hetero USA, and a trial has been set to start on March 23, 2026, which is prior to the expiration of the automatic 30-month stay on May 26, 2026. We remain confident in our ability to protect our IP. Moving to AGAMREE. Our differentiated corticosteroid medication approved for use in the treatment of Duchenne Muscular Dystrophy, or DMD, a rare and life-threatening neuromuscular disorder. AGAMREE delivered 154.3% year-over-year growth with 2025 revenues of $117.1 million. Our launch strategy targeting centers of excellence penetration delivered outstanding results. With this success, we have now pivoted our efforts to going deeper in each of these core institutions. Our goal is to ensure the greatest possible use of AGAMREE an effective and differentiated steroid in what we believe has a greater than $1 billion addressable market. We plan to tap into the full potential of AGAMREE through our ongoing SUMMIT study, a 5-year follow-up study evaluating approximately 250 DMD patients once enrollment is completed. By increasing the full body of data that assesses the potential long-term benefit over current standard of care, we believe AGAMREE can be further differentiated, allowing us to build more awareness and drive further growth. With regard to maximizing the full value of AGAMREE, we are presently conducting a Phase I study to evaluate dose equivalence between AGAMREE and other steroids and potential immunosuppressive activity as well. We are also currently assessing potential indications beyond DMD, where AGAMREE may serve a broader array of patients with rare diseases. We look forward to updating you further on our expansion initiatives with AGAMREE as the year unfolds. Lastly, FYCOMPA. Despite its loss of exclusivity in May of 2025, the product delivered net revenue of $113.3 million in the year, outperforming our expectations. Due to generic competition, we are forecasting sales of FYCOMPA in 2026 of between $40 million and $45 million, which reflects our expectation that FYCOMPA will remain a solid revenue producer for us. Beyond our portfolio optimization initiatives, we are pursuing our evolved and focused business development strategy aimed at identifying the right opportunities to supplement our strong organic growth. Our business development engine conducted over 100 assessments in 2025. Notably, about 90% of those were inbound, underscoring our reputation in the industry as a proven leader that delivers value through launching, supporting and growing our promoted assets. With our industry-leading rare disease expertise, best-in-class commercial capabilities, established plug-and-play infrastructure and trusted status within the rare disease community, we are confident in our ability to drive continued long-term repeatable success through business development. As we assess the broader landscape and levers at our disposal to create value, we are focused on remaining nimble and acting opportunistically to ensure we are well-positioned to identify, assess and onboard rare disease products that will grow our portfolio and positively impact the rare disease community. To be clear, we will maintain the same guiding principles that have enabled our prior success, remaining disease and modality agnostic while prioritizing on-market and near-market differentiated rare disease products. In addition, as we reported during our JPMorgan presentation earlier this year, we have now expanded our search to include therapies in late-stage development with positive proof of concept, a differentiated profile and a well-characterized regulatory path. We will also continue to focus on assets with peak sales of up to $500 million, which is where we believe we can be most competitive and best suited to integrate with our existing infrastructure. With that, I'll turn the call over to Jeff, who will provide additional insights into our commercial performance. Jeff?
Jeffrey Del Carmen: Thanks, Rich. We are very pleased with our exceptional performance in 2025, marked by full year combined total revenues of $589 million, surpassing the upper end of our updated guidance of $565 million to $585 million. This outstanding achievement was driven by FIRDAPSE reaching a record high of $358.4 million, the continued successful commercialization of AGAMREE and the strong contribution from FYCOMPA despite the entry of generic competition. Let's start by reviewing our advancements with FIRDAPSE, the only evidence-based FDA-approved treatment for Lambert-Eaton in myasthenic syndrome. In the fourth quarter of 2025, net revenues amounted to $97.6 million, showcasing a remarkable Q4 2025 versus Q4 2024 growth of 18.3%. Furthermore, FIRDAPSE's full year 2025 net revenues showed strong 17.1% growth year-over-year. FIRDAPSE's performance this year reflects deliberate disciplined commercial execution across the patient journey. First, by expanding and optimizing our lead-generating channels, we increased our data leads of identified LEMS patients in active diagnostic stages by 40% in the fourth quarter. These patients consistently represent approximately 50% of new starts each quarter, reinforcing the importance of our sustained investment in early patient identification. Second, we significantly improved our conversion efficiency through tighter coordination across field teams, commercial analytics and marketing, we increased the rate at which qualified leads transition to treatment. As a result, new patient enrollments for FIRDAPSE exceeded our 2025 forecast, demonstrating the impact of more focused execution. In the second half of 2025, we also saw increased VGCC testing by more than 1/3 versus the first half of 2025, reflecting targeted initiatives to accelerate diagnosis among LEMS patients. By shortening time to diagnosis, our awareness activities are expanding the treated population and improving the overall patient journey. Finally, in June, we launched a pharmacy outreach program designed to support newly enrolled patients in achieving their optimal therapeutic dose. This initiative contributed to a significant reduction in new patient discontinuations, strengthening early persistence and reinforcing long-term value. Overall, FIRDAPSE's performance underscores the effectiveness of our end-to-end commercial strategy, expanding the top of the funnel, improving conversion, accelerating diagnosis and enhancing patient retention to drive sustainable growth. Our next phase of growth for FIRDAPSE will come from both idiopathic LEMS and cancer-associated LEMS. We will continue to leverage the increased data leads to accelerate new patient acquisition while continuing to deploy initiatives to accelerate the diagnostic journey for LEMS patients. As for cancer-associated LEMS, our primary focus in the first half of 2026 is and will continue to be pursuing relationships with leading oncology networks to integrate the updated NCCN guidelines into their care pathways, which we believe will lead to more addressable patients in the second half of 2026. It's important to note that we have already seen significant uptake in the number of new positive VGCC tests ordered by oncologists in the second half of 2025. Turning to AGAMREE. AGAMREE continues to build strong commercial momentum as a differentiated therapy for the treatment of patients living with DMD. For full year 2025, the first full year of AGAMREE being on the market in the U.S., we delivered net product revenue of $117.1 million, representing 154.3% year-over-year growth. In the fourth quarter alone, net product revenue reached $35.3 million, up 67.5% compared to the fourth quarter of 2024, clear evidence of accelerating demand and effective execution. Adoption across top DMD centers of excellence continues to expand. To date, 100% of the top DMD centers of excellence, which represent about 80% of all DMD patients have enrolled at least 1 patient on AGAMREE and 270 unique health care providers have submitted enrollment forms. This reflects broad and deepening engagement across the treatment community as well as growing confidence in AGAMRE's differentiated clinical profile. Importantly, we saw a meaningful reduction in discontinuations and cancellations in the second half of 2025. This improvement was driven primarily by increased provider familiarity and experience with AGAMREE as well as fewer disruptions from DMD market events. We believe that this trend reinforces the durability of demand as the market matures. Since launch, approximately 45% of patients have transitioned from prednisone and 42% from EMFLAZA, underscoring AGAMREE's relevance across established treatment paradigms and its ability to compete effectively within multiple segments of care. In addition, the median age of new enrollees has dropped 1 year in the most recent quarters compared to when we launched in 2024. Reimbursement performance remains strong with success rates above 85%, consistent with our expectations. Our commercial organization continues to execute with precision, prioritizing targeted provider education, optimizing field force impact and deepening payer engagement to support sustained uptake and long-term market expansion. FYCOMPA delivered full year 2025 net product revenue of $113.3 million, exceeding the upper end of our guidance. As previously noted, we expect continued revenue erosion from increased generic competition to impact FYCOMPA performance moving forward. As of December 31, we discontinued personal promotion and assistance programs for FYCOMPA. However, we forecast that 2026 net product revenues from this product will continue to be meaningful. In closing, our commercial organization continues to operate with rigor and accountability, translating strategy into consistent portfolio performance while laying the groundwork for our next wave of growth for FIRDAPSE and AGAMREE. Our diversified portfolio, combined with differentiated commercial capabilities and disciplined execution gives us confidence that we will be able to achieve our 2026 revenue guidance. Our focus is unwavering, elevating commercial excellence across markets, expanding and accelerating patient access and unlocking the full value of our portfolio to drive durable growth and sustained shareholder returns. I want to recognize and thank our entire commercial team for their commitment to execution, performance and most importantly, the patients we serve. Their dedication is fundamental to our continued success. At this time, I would like to turn the call back over to Mike.
Michael Kalb: Thank you, Jeff. Our fourth quarter and full year 2025 financial results demonstrated another strong year, driven by our solid financial performance, financial discipline and strong execution. Our total revenues for 2025 were $589.0 million, an approximate 19.8% increase when compared to total revenues of $491.7 million for 2024. 2024 included approximately $2.4 million of license and other revenue, which consisted principally of a milestone payment earned from our sublicensee in Japan, receiving regulatory approval to commercialize FIRDAPSE for the treatment of patients with LEMS in Japan compared to license and other revenue in 2025 of approximately $182,000. For the fourth quarter of 2025, total revenues were $152.6 million, representing a 7.6% year-over-year increase. FIRDAPSE's fourth quarter of 2025 net product revenue increased 18.3% over the fourth quarter of 2024 and 5.9% compared to Q3 2025. AGAMREE's fourth quarter 2025 product revenue net increased 67.5% over the fourth quarter of 2024 and approximately 9% over Q3 2025. For 2026, we are forecasting FIRDAPSE net product revenue to be between $435 million and $450 million, which takes into account an increase in gross to net driven by the IRA impact on our Medicare Part D net product revenue. We expect that the IRA impact will continue to increase annually. We are forecasting AGAMREE 2026 net product revenue to be between $140 million and $150 million. We are forecasting FYCOMPA 2026 net product revenue to still remain meaningful at between $40 million and $45 million. The expected decrease is a result of the entry of generic versions of FYCOMPA during 2025. Net income before income taxes for 2025 was $283.5 million, a 31.1% increase compared to $216.3 million for 2024. We reported GAAP net income for 2025 of $214.3 million or $1.68 per diluted share. GAAP net income increased by 30.8% compared to GAAP net income for 2024 of $163.9 million or $1.31 per diluted share. Non-GAAP net income for 2025 was $346.2 million or $2.72 per diluted share, which excludes from GAAP net income, amortization of intangible assets related to our acquisitions of FYCOMPA, AGAMRE and Ruzurgi of $37.5 million stock-based compensation expense of $24.8 million, income tax provision of $69.2 million and depreciation of $0.4 million. Non-GAAP net income for 2025 was $346.2 million or $2.72 per diluted share, which excludes from GAAP net income, amortization of intangible assets related to our acquisitions of FYCOMPA, AGAMRE and Ruzurgi of $37.4 million stock-based compensation expense of $22.8 million, income tax provision of $52.4 million and depreciation of $0.4 million. Our effective tax rate for 2025 was 24.4% compared to 24.2% for 2024. The effective tax rate is affected by many factors, including the number of stock options exercised in any given period which we expect will be relatively consistent for 2026, but will likely fluctuate from quarter-to-quarter. Cost of sales expense was approximately $87.3 million in 2025 compared to $68.8 million in 2024 and consisted principally of royalties. As a reminder, AGAMRE royalties paid to the ultimate product licensor equals 7% of net sales up to $250 million with additional increases as net sales increase. Additionally, through December 31, 2025, we were also required to pay 5% on net sales up to $100 million to our direct licensor. Further, we are also required to pay our direct licensor royalties of 7% of net sales in excess of $100 million and up to $200 million with additional increases as net sales increase. The company is also required to make a $12.5 million milestone payment when AGAMREE's net product revenue reached $100 million, which was achieved during the fourth quarter of 2025. This milestone payment obligation was capitalized during the fourth quarter of 2025 and is being amortized over the estimated remaining useful life of the asset. Further details of our royalty obligations for AGAMRE as well as FIRDAPSE and FYCOMPA are disclosed in our 2025 Form 10-K. Beginning in July 2026, as per our contractual arrangement with Eisai, we will be required to pay FYCOMPA royalties equal to 6% of net product revenue to the product licensor for FYCOMPA. Research and development expenses were $12.7 million in 2025 compared to $12.6 million in 2024. Our R&D spending during 2025 was comprised mainly of costs to support our ongoing AGAMRE studies. Relative to the normal course of business, absent another acquisition, we are forecasting research and development costs in 2026 to be between $17.5 million and $22.5 million. Selling, general and administrative or SG&A expenses for 2025 totaled $193.8 million as compared to $177.7 million for 2024, primarily attributable to an increase in employee compensation related to annual merit increases and an increase in headcount. Further, general and administrative expenses increased due to consulting fees related to multiple business initiatives, including business development activities, which were offset due to decreases in contributions to 501(c)(3) organizations supporting patient assistance programs. While we are no longer actively marketing FYCOMPA, we anticipate that 2026 SG&A expenses will increase slightly compared to 2025 due to the costs associated with our continued efforts focused on increasing the awareness of FIRDAPSE for the potential treatment of cancer-associated LEMS. As reported, we ended 2025 with cash and cash equivalents of $709.2 million compared to $517.6 million at December 31, 2024. The increase in cash of $191.6 million was largely driven by $208.7 million in cash generated from operating activities, partially offset by $17 million of cash used in financing activities, which includes the repurchase of $25.3 million of common stock during the fourth quarter of 2025. We believe that our life cycle management and our business development activities will not be adversely affected by our share repurchases under our share repurchase plan. We believe our current funds, along with our expected continued generation of cash from operations, continue to provide us the financial flexibility to fund our existing R&D programs, meet our potential contractual obligations and support our strategic initiatives, business development and portfolio expansion efforts, leading to long-term growth and value creation. More detailed information and analysis of our 2025 financial performance may be found in our annual report on Form 10-K, which was filed with the Securities and Exchange Commission yesterday, February 25, and can be found on the Investor Relations page of our website. At this time, I will turn the call back over to Rich. Rich?
Richard John Daly: Thanks, Mike. As you heard on today's call, Catalyst is entering 2026 from a position of strength and with significant momentum. Our strategic priorities remain clear, and we are primed to drive continued growth. On FIRDAPSE we'll focus on executing our dual market expansion strategies, prioritizing patient identification efforts for idiopathic LEMS and ongoing education to promote the updated NCCN guidelines for cancer-associated LEMS. For AGAMRE, we plan to progress the multiple initiatives underway to maximize the potential of this differentiated asset, including facilitating earlier disease detection and deepening our market penetration to drive commercial expansion as well as advance the ongoing SUMMIT study and other life cycle management activities. Lastly, we will continue to pursue strategic and thoughtful business development with an eye toward exciting and low-risk opportunities where we believe we can unlock meaningful value. We are proud of our progress in 2025 and plan to deliver continued success in the year ahead. I want to end by thanking our employees, partners and shareholders for their support and dedication. I'll now turn the call back over to the operator for questions.
Operator: [Operator Instructions] Our first question comes from the line of Samantha Semenkow with Citi.
Unknown Analyst: This is Ben on for Sam. To start, can you speak to the drivers that underpin the growth implied within your 2026 guidance for FIRDAPSE and AGAMREE?
Richard John Daly: Thanks for the question. I'll turn it over to Jeff to start the answer.
Jeffrey Del Carmen: Absolutely. So, Ben, when we look at FIRDAPSE first, we are extremely excited about the opportunity that still remains. You look at both sides of FIRDAPSE that we've specified and that's idiopathic LEMS, where we feel we're only about 30% penetrated at this point. And then you have the cancer-associated LEMS where we're well under 10% penetrated. So again, significant opportunity there. As I mentioned on the call, we have a pool of patients there that we've stated 500 in the past and that we've seen significant growth from specifically our data leads in the fourth quarter. And so now that number is greater than 600. So based on those identified patients that are somewhere in their LEMS diagnostic journey, we believe that there's plenty of opportunity here in the short term to help those patients convert on to FIRDAPSE treatment. We've also identified some pharmacy intervention programs that have helped us reduce the discontinuations of patients in the first 4 months of treatment where we saw some higher-than-expected discontinuations. So based on those intervention programs, we've seen these patients able to reach their optimal therapeutic dose within the first 4 months. And at that point, we've seen a reduction in the discontinuation of those patients by 12%. So that's very significant, too. So, from the cancer associated side, we've seen a 21% increase in all VGCC testing year-over-year in '25 versus '24. With that, patient identification also is critical. So, we know that number will, in the long term, turn into patients' potential leads for us to help. And then we also expect that screening to take place in the first half of the year. But then in the second half of the year, we expect more arrangements with group practices that will help us convert those leads into patients on therapy. So that's FIRDAPSE.
Richard John Daly: Jeff, just on the FIRDAPSE side, you talked about the number of leads that we have. Can you talk a little bit about the improvement in quality of those leads?
Jeffrey Del Carmen: Absolutely. So, we've become much more efficient in helping these patients go from a diagnosis of LEMS on to treatment when appropriate. We use AI, we use machine learning, and we have 5 different data sources that we utilize to qualify the leads and ensure they are unique and identify which of these patient leads are the highest priority and have the most urgency to get on treatment. So, we point our sales force into that direction where to go. So, we score these leads for them. And that's been extremely helpful in helping these patients convert on to treatment sooner. For AGAMREE, we're very pleased with the performance last year. And based on the low side of our guidance, the 20% growth this year. We expect in at least a 20% growth in 2026. I mentioned broad adoption and across the top 45 or the top COEs that make up 80% of all DMD patients. So, we expect to not only continue that, but to deepen that penetration within those sites. We've seen many physicians now. The -- with the experience that they have, we've seen discontinuations and cancellations that have also decreased over the last 2 to 3 quarters. So that's also a positive sign of greater experience and strong adoption and acceptance of AGAMREE.
Operator: Our next question comes from the line of Joon Lee with Truist Securities.
Asim Rana: Congrats on the quarter. This is Asim on for Joon. So just kind of on the last one, I mean, your FIRDAPSE guide for '26 is above the historical 15% to 20% that we've typically seen. So, I just want to understand of all the factors that you mentioned in the answer to the previous question, what's the biggest driver of that increase? And is that how we should be thinking of FIRDAPSE growth beyond '26 in 2027? And then just on BD, I mean, would it be fair to assume we'll see a deal this year?
Richard John Daly: Jeff, do you want to build on the previous answer to that?
Jeffrey Del Carmen: Sure. Asim, the thing that really gives us the most confidence is really in the short term, the greater than 600 pool of patients, the patients that are somewhere in their diagnostic journey to LEMS, but we know they're LEMS patients not yet on treatment. So over 50% of our new enrollments comes from that pool of patients. So that gives us significant confidence. The other big reason for confidence is the 21% growth in VGCC test year-on-year. We're actually seeing a 9% growth quarter-on-quarter of VGCC test. So that volume, the velocity of new tests that are being done also will help us identify and help these patients sooner and shorten their diagnostic journey. So that's one of the big things. And our ability to be more efficient, like I mentioned, helping these patients that are diagnosed get on to treatment faster. And then on the second half of the year, that's when we talked about incremental patients coming from the cancer-associated LEMS opportunity. So that's when we truly expect both idiopathic and CA-LEMS patients to increase here in the second half of this year.
Richard John Daly: Before I take the BD question, Asim, just to build on Jeff's answer, too, think about the timing of some of the things we put in place. So, this will be a full year of the dedicated sales forces. We changed that in April of last year. So we expect there to be some dedication provide some value. But also, Jeff, can you speak to the timing of the pharmacy program because that, again, was not a full year program either, and we're seeing the nice results there, too.
Jeffrey Del Carmen: Thanks, Rich. We initiated the pharmacy intervention program in June of last year. And we saw a significant decrease in patients that were not able to get to the -- their optimal therapeutic dose. We saw a reduction in that. And like I mentioned, we saw a 12% decrease in discontinuations of those patients after we initiated that program. So again, like Rich mentioned, we only have basically half a year of that program in place. And we feel that in the long-term, that will help patients get to their optimal therapeutic dose and when appropriate, stay on treatment.
Richard John Daly: I'll take question -- thanks for the questions here today. So we're going to continue along the line that we've pursued, which is a diligent and thoughtful approach to looking at opportunities and as I think we've talked about before, we really look at a couple of elements in this. We're obviously looking for a differentiated product profile rather and something that really can improve patient care. The second element for us is we have to be aligned on the vision with our potential partner or licensor on not only how the product will launch, but what might be life cycle management opportunities. So we think that's really important. And then, again, focusing on that near-term accretive opportunity is really important to us as well. Our BD team has done a phenomenal job of exercising these elements and looking for the ideal opportunity. The one other thing we have to take into account as we assess opportunity is especially as we go deeper into the pipeline, working our way back into post proof-of-concept opportunities with a relatively clear regulatory path is the regulatory environment today. And so we are, again, diligent and thoughtful in our approach. As to whether or not we can say we'll do a deal in the year, obviously, we want to improve the portfolio that we have to improve care for patients. And so we'll continue our diligent efforts here and bring in products that we believe we can add true value to and the products that will also add value to the lives of the patients we serve.
Operator: Our next question comes from the line of Pavan Patel with Bank of America.
Pavan Patel: Congrats on the commercial execution this quarter. I thought it was pretty strong. So I will focus on AGAMREE commercial. And my two questions. So the first is on the detail on the median age of new AGAMREE enrollees dropping by a year. I thought that was interesting. So maybe if you can speak to whether you're seeing a meaningful shift where physicians are using AGAMREE as a first-line steroid for newly diagnosed boys rather than just switching older patients who are already experiencing toxicities from either prednisone or EMFLAZA? And how does that capturing that younger demographic impact assumptions around longer-term patient durability? And then the second question is with regards to the reimbursement. I know you mentioned that it's tracking above 85%. So maybe if you can just speak to what's the pushback that you're seeing in the remaining 15%. Is that just largely a step edit requirement of try prednisone first? And do you expect that 85% rate to pick up as we move through the quarters in 2026?
Jeffrey Del Carmen: Sure. Great questions, and I'll take the latter first. And when we think about the AGAMREE reimbursement landscape, we're very pleased with that greater than 85% approval rates. And to your point, yes, absolutely, there are some step edits. But over time, those steps, those patients are able to get on to treatment. What we do is we provide bridge treatment or free drug for patients while they're going through those steps. And until they're able to get approved, then we convert those patients under reimbursed patients over time. So we believe that's very, very strong. And in fact, we're closer to 90% as far as reimbursement rates go. When we look at the average age, the decrease by 1 year, so the average age in the last 6 months has come down to closer to 11-ish versus the 12 that was before. So that's very significant. And you had mentioned it, why it's significant is because younger patients, they have greater adherence to therapy. And really, they're more likely to experience the positive tangible benefits for longer. So it's a great thing for patients but we're seeing that play out. And you asked what does that mean that patients are coming over without doing a step or going to prednisone or generic EMFLAZA or EMFLAZA. And about 10% of our patients actually come directly without ever having experienced a steroid before. So it is the first steroid for the DMD treatment. So we are seeing that.
Richard John Daly: Jeff, to build on Pavan's question, when we think about the change in age, obviously, the dosing is start at the top end of the dose and work down. So these patients are coming in, they're younger. One would anticipate maybe a different dose? Are we experiencing that?
Jeffrey Del Carmen: No, actually, we're seeing the same dose.
Richard John Daly: So I think it's really positive for longevity, persistency on the drug and then obviously, for the dosing for the patient to get the benefit.
Jeffrey Del Carmen: Absolutely.
Operator: Our next question comes from the line of Leland Gershell with Oppenheimer.
Unknown Analyst: Congrats on the quarter. And this is Jason on here for Leland Gershell. Question on AGAMREE. One and how does the SUMMIT open-label expansion affect the forecast for AGAMREE going forward? And to what degree are the assumptions built into the 2026 guidance? And maybe one more point. How should we think about additional indications to support AGAMREE growth going forward?
Richard John Daly: So before -- I have Will join in, I just want to touch on the fact that the focus here for SUMMIT is enrolling patients, getting the patients into the trial. And with that, I'll just turn it over to Will to talk about what some of the expectations are. Will?
William Andrews: Yes. Thank you, Rich. Thank you, Jason, for the question. We are -- continue to be excited about the SUMMIT trial. We continue to bring in new sites and to enroll additional patients towards our goal of getting this to a trial where we have a significant enough patients where we can really pull out some robust data analysis from this trial. We're also excited about it because of Santhera's announcement from last November as well as their upcoming MDA conference poster presentations and abstract presentations on their Guardian trial as well as evaluation of other open-label data that they have that look at really the same -- many of the same parameters that we're looking at in SUMMIT, where they actually show some important positive top line data, including comparisons to large natural history data sets that show normal growth in the AGAMREE-treated patients compared to stunted growth in the corticosteroid treated population, a decreased rate of vertebral fractures, decreased rate of cataracts, no cases of glaucoma, and these are all important endpoints in our SUMMIT trial. We are also additionally looking at progression of cardiac effect, et cetera. So this is a trial that we are purposely driving to evaluate long-term potential benefit in these really important glucocorticoid side effects. And for, I think, the latter part of your question, Jason, I'll pass it back to Rich in regard to how we look at it as a potential impact on performance of the medication.
Richard John Daly: Thanks, Will. This is a great question, Jason. So thank you. When we look at these data that we see for Santhera, again, it's very encouraging for SUMMIT. But we have to keep in mind that none of the endpoints that we see in the data from Santhera are in our label at this point in time. So presently, we don't have a great impact built in. I don't think we have any impact because we can't promote it. So -- but again, we're highly encouraged by these data. We're really excited to continue the SUMMIT trial to see if we can build on the data set. And obviously, physicians can use the product as they deem appropriate, but we need a robust data set, and the Santhera data is a great start for us.
Operator: Our next question comes from the line of Luke Herrmann with Baird.
Luke Herrmann: Looks like a nice FIRDAPSE guide. Can you just help provide any additional color on the extent to which you expect the growth for FIRDAPSE to be sort of backloaded? Or does the traction you're seeing with idiopathic sort of smooth things out in the first half? And I have a follow-up.
Jeffrey Del Carmen: So yes, we do expect strong enrollments from the idiopathic LEMS here in the first half to help as we build the screening for cancer-associated LEMS. Like I mentioned, we do expect incremental patients for cancer-associated LEMS in the second half of the year.
Richard John Daly: I think when you build on this, when you think about how this is -- the patients are diagnosed in the journey they go through, Jeff did a really nice job of explaining how we're accelerating that when it's appropriate. And we'll continue to see that. But there is a cadence to this that we've -- I think we've seen over the last 6 or 7 years. And on the cancer side, we're working to advance that, but we would expect there to be an incremental opportunity in the second half, especially when we see the VGCC tests increasing, I think that's a really good sign, but it does take time to get those patients into the treatment queue.
Luke Herrmann: Great. And then just a follow-up on business development. I guess, in light of the favorable trajectory for XBI recently, do you think this could make BD activities more challenging? Or have you not seen much of a change to the breadth or quality of inbounds at this point?
Richard John Daly: We're seeing no change to the quality of the inbounds. We like the opportunities we're currently evaluating. And it comes down to the 3 or 4 points I looked at before when we get into due diligence, making sure that we have good alignment opportunity to offer differentiated and improved care and then obviously return -- get a return as quickly as possible, as reasonably possible.
Operator: Our next question comes from the line of Sudan Loganathan from Stephens Inc.
Kesav Chandrasekhar: This is Kesav on for Sudan. Congrats on wrapping up 2025. So I understand that VGCC testing initiatives are primarily expected to materialize during the second half of this year. But could you all provide some color on the magnitude testing volume has possibly already had on FIRDAPSE sales? And then my second question is, could you talk about how the Phase 1a readout in this quarter on establishing translational dosing is expected to impact your plans for AGAMREE going forward?
Richard John Daly: Jeff, why don't you take the first one and then Will shall take the second.
Jeffrey Del Carmen: Like I had mentioned, there is significant growth year-on-year, 21% in the year-on-year for the VGCC test. So that's always a positive sign. We know it's tough to tease out what -- how much of that is the cancer-associated LEMS or test ordered by an oncologist versus a neurologist. So I would say primarily, a lot of that increase is still stemming from the idiopathic LEMS side. So there's plenty of opportunity still remaining to continue to increase the VGCC testing within oncologists. So that presents a significant opportunity still for us moving forward.
Richard John Daly: Jeff, as you look at the VGCC testing and that is an indicator of conversion to therapy, where would that rank as an indicator of conversion?
Jeffrey Del Carmen: It's of our patients coming in each month, it's about 50% to 60% of our new enrollments come from these new leads.
Richard John Daly: So that's increasing?
Jeffrey Del Carmen: Absolutely.
Richard John Daly: Will, do you want to take the next question? And I'm sorry, could you just repeat the question so we have clarity on it?
Sudan Loganathan: Yes, for sure. So like for the Phase 1a readout in this quarter, first quarter of 2026 on establishing that translational dose for AGRAMEE, -- could you kind of just provide some color on like how that readout might impact your plans going forward for AGRAMEE or if there was going to be any substantial impact?
William Andrews: Yes. And happy to answer that question. Thank you for the question. One quick point of clarification is that we've announced that we will have analyses of that data within the first half of 2026, so not this quarter, just for that clarification. And effectively, what you're referring to is our evaluation in this study of biomarkers of inflammation as well as immunosuppressive biomarkers on multiple doses of AGAMREE. As to how that might impact business development or I should say, life cycle management opportunities for AGAMREE is essentially if we see, say, strong immunosuppressive effects, it might direct us towards certain life cycle management opportunities. And if we see minimal immunosuppressive effects, again, that might direct us to other life cycle management opportunities. The life cycle management evaluations broadly for AGAMREE are important for us and active, and we're excited about a number of the potential additional target indications that we're evaluating currently.
Richard John Daly: And to build on that from a business impact, when Jeff mentioned on the call that we're seeing high retention rates, so there could be a potential business impact to getting the right patient on, but we are seeing a reduction in patients who are not staying on therapy, so a higher retention rate. And remember, about 45% of the patients switch from prednisone, 45% switch from an EMFLAZA, whether it's branded or generic. And so, the physicians are getting more and more comfortable over time. And this could just improve that with a little bit more direction should we be able to get it into the label, but that's yet to be seen. But just to build on that, we're happy with the progress we're making on retention as well.
Operator: And at this time, we have no further questions. That concludes our question-and-answer session and today's conference call. We would like to thank you for your participation. You may now disconnect your lines. Have a pleasant day.