Catalyst Pharmaceuticals Inc (CPRX) 2025 Q4 法說會逐字稿

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 in full year 2025 financial results conference call.

All lines have been placed on mute to prevent any background noise. After the speaker's remarks, there will be a question-and-answer session.

(Operator Instructions)

I would now like to turn the conference over to Mike Kalb, Chief Financial Officer. Please go ahead.

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.

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 was 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's 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 forecasts 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 two distinct markets for the product, idiopathic limbs and cancer‑associated limbs, and we believe there's 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.

In 2025, we finalized settlement with two of the first filler. 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 thirty‑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 excellent 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 five‑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 of our 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 one 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 $40.45 million dollars, 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 one hundred 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'll provide additional insights into our commercial performance. Jeff.

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 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 enrolments 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 a third 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 lens and cancer associated lens. 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 lens, 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 a full year 2025, the first full year of AGAMREE being on the market in the US, 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 one patient on AGAMREE, and 270 unique healthcare providers have submitted enrollment forms. This reflects broad and deepening engagement across the treatment community. As well as growing confidence in AGAMREE'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 one 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 a 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 guide.

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 sustain 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.

Thank you, Jeff.

Our fourth quarter and full year 2025 financial results demonstrate 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 sub-licensee 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 this 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's 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're 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 $40.45 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, AGAMREE, 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.

This compares to non-GAAP net income for 2024 of $276.3 million, or $2.21 per diluted share, which excludes from GAAP net income amortization of intangible assets related to our acquisitions of FYCOMPA AGAMREE and Ruzurgi of $37.4 million, stock-based compensation expense of $22.3 million, the 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, the AGAMREE 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 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 AGAMREE, as well as FIRDAPSE and FYCOMPA, are disclosed in our 2025 Form 10-K.

Beginning in July 2026, as per our contractual arrangement with FSSAI, 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 AGAMREE 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 head count.

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 cost 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'll turn the call back over to Rich. Rich.

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 limbs, and ongoing education to promote the updated NCCN guidelines for cancer associated limbs.

For AGAMREE, 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.

Thank you, ladies and gentlemen. We will now begin the question-and-answer session.

(Operator Instructions)

Thank you.

Samantha Semenkow, Citi Investment Research.

Hi, this is Ben on for Sam. Thanks so much for taking the questions. To start, can you speak to the drivers that underpin the growth implied within your 2026 guidance for FIRDAPSE and AGAMREE?

And thanks for the question. I'll turn it over to Jeff to start the answer.

Absolutely. So, Ben, when we look at FIRDAPSE first, we're extremely excited about the opportunity that still remains.

You look at both sides of FIRDAPSE that we, we've specified, and that's idiopathic limbs where we feel we're only about 30% penetrated at this point, and then you have the cancer associated lens 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 are plenty, there's plenty of opportunity here in the short-term to help those patients convert onto FIRDAPSE's treatment. 3

We've also identified some pharmacy intervention programs that have helped us reduce the discontinuations of patients in the first four 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 four months, and at that point, we've seen a reduction in the discontinuation of those patients by 12%. So that, 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 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.

Jeff, just on the 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?

Absolutely. So, we've become much more efficient in helping these patients go from a diagnosis of LEMS onto treatment, when appropriate. We use AI, we use machine learning, and we have five different data sources that we utilize to qualify the leads and ensure they are unique and identify which of these patients, 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 onto treatment sooner.

For AGAMREE, we, we're very pleased with the performance last year and based on the low side of our guidance, that's 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, with the experience that they have, we've seen discontinuations and cancellations that have also decreased over the last two to three quarters. So that's also a positive sign of greater experience and strong adoption and acceptance of AGAMREE.

Joon Lee, Truist Securities.

Congrats on the quarter and thanks for taking the questions. 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's growth, beyond '26, even in 2027, and then just on BD, I mean, would it be fair to assume we'll see a deal this year?

Thank you.

Jeff, do you want to build on the previous answer for that?

Sure, awesome, 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 tests year on year. We Actually seeing a 9% growth, quarter on quarter of VGCC tests.

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 on, get onto 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 LEMS patients to increase here in the second half of this year.

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 did, 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.

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 a half a year, of that program in place, and we feel that in the long-term, that'll help patients get to their optimal therapeutic dose and when appropriate, stay on treatment.

Thanks. And Asim, I'll take your 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 portfolio or 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 creative opportunity is a real, 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, 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 a 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 you know 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.

Thank you.

Thank you.

Pavan R. Patel, Bank of America Global Research.

Hey guys, congrats on the commercial execution, this quarter. I thought it was pretty strong. So, I will focus on again re commercial, and my two questions. So, the first is on the detail on the median age of new again re-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 capture the younger demographic-- 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?

Thank you.

Sure, great questions, and I'll take the latter first. And when we talk-- think about the gamma reimbursement landscape, we're very pleased with that greater than 85%, approval rates. And to your point, yeah, absolutely, there are some step edits, but over time, those steps, those patients are able to, get onto treatment. 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 on reimbursed patients over time. So, we believe it's-- that's 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 one year, it's, so the average age in the last six months has come down to closer to eleven-ish versus the twelve 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 ask, 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 their first steroid for their DMD, treatment. So, we are seeing that.

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, are, one would anticipate maybe a different dose. Are we experiencing that?

No, actually, well, we're seeing the same dose.

Right. 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.

Absolutely.

Thank you guys.

Leland Gershell, Oppenheimer and Co.

Hey, congrats on the quarter, and this is Jason on here for Leland Gershell. I have a question on AGAMREE. When 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? Thank you.

So, before I have Will join in, I just want to touch on the fact that the focus here is for a summit is enrolling patients, getting the patients into the clinic, into the trial, and, with that, I'll just turn it over to Will to talk about, we know what some of the expectations are. Will?

Yes, thank you.

Thank you, Richard.

Thank you, Jason, for the question.

We are continued 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, MBA 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 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, etc.

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.

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. We don't have, 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, 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 that they deem appropriate, but, we need a robust data set and this Centera data is a great start for us.

Luke Herrmann, Robert W. Baird & Co.

Hi team, thanks for the questions. 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 back loaded, or does the traction you're seeing with idiopathic sort of smooth things out in the first half, and I have a follow-up. Thanks.

So, yeah, we do expect strong, enrollments from the idiopathic lens here in the FIRDAPSE to help, as we build the screening, for cancer associated lens.

Like I mentioned, we do expect incremental patients or cancer associated limbs in the second half of the year.

I think when you build on this, when you think about how this is, the patients are diagnosed and 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 six or seven years, and on the cancer side, we're working to make, 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.

Great, and then just to 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 the inbounds at this point?

We're seeing no change to the quality of the inbounds. We like the opportunities we're currently evaluating, and you know it comes down to the three or four points I've looked at before we get into due diligence, making sure that we have good alignment opportunities to offer differentiated and improved care, and then obviously get a return as quickly as possible, as reasonably possible.

Great thanks team.

Thank you.

Sudan Loganathan, Stephens & Co.

Hey, Catalyst team, this is Keith Salvon for Sudan.

Thank you for taking my question and, 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 as possibly already had on FIRDAPSE's sales, and then my second question is, could you talk about how the Phase 1A readout in this quarter on establishing transitional dosing is expected to impact your plans for AGAMREE going forward?

Thank you.

Jeff, why don't you take the first one, I'm sure.

Like I had mentioned, there's significant growth year on year, 21% in 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 lens or tests ordered by an oncologist versus a neurologist.

So, I would say primarily a lot of that increase is still stemming from the idiopathic lens side. So, there's plenty of opportunities still remaining to continue to increase the VGCC testing within oncologists, so that presents a significant opportunity still for us moving forward.

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?

It's of our patients coming in each month, it's about 50% to 60% of our new enrollments, come from these new leads. So.

So we'll get it increasing.

Absolutely.

That's likely increases.

Absolutely.

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?

Yeah, for sure.

So, like for the Phase 1A readout, in this quarter, first quarter of 2026 on establishing that translational dose, for AGAMREE, it could you kind of just provide some color on like how that readout might impact your plans going forward for AGAMREE or if there was going to be any substantial impact?

Thank you.

Yeah, 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 analysis 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 the 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 lifecycle 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.

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 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 we're just to build on that, we're happy with the progress we're making on retention as well.

Okay, got it. Thank you.

Thank you.

And at this time, we have no further questions. That concludes our question-and-answer session in today's conference call. We would like to thank you for your participation. You may now disconnect your line. Have a blessed day.