Catalyst Pharmaceuticals - Q1 2023

Transcript

Operator (participant)

Greetings, welcome to the Catalyst Pharmaceuticals first quarter 2023 financial results conference call. At this time, all participants are in a listen-only mode. A brief question-and-answer session will follow the formal presentation. If anyone should require operator assistance during the conference, please press star 0 on your telephone keypad. As a reminder, this conference is being recorded. It is now my pleasure to introduce your host, Ali Grande, Chief Financial Officer. Thank you, Ali. You may begin.

Alicia Grande (CFO)

Good morning, everyone. Thank you for joining our conference call to discuss Catalyst's first quarter 2023 financial results and corporate highlights. Leading the call today is Patrick J. McEnany, Chairman and Chief Executive Officer. We're also joined by Dr. Steven R. Miller, our Chief Operating Officer and Chief Scientific Officer, and Jeffrey Del Carmen, our Chief Commercial Officer. For the Q&A session, we'll have Dr. Gary Ingenito, our Chief Medical and Regulatory Officer. 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 relate to our current expectations, estimates, and projections and are not guarantees of future performance. They involve risks, uncertainties, and assumptions that are difficult to predict and may prove not to be accurate.

Actual results may vary from the expectations contained 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 2022 annual report on Form 10-K. At this time, I'll turn the call over to Pat.

Patrick McEnany (Chairman and CEO)

Thanks, Ali, welcome everyone to our first quarter 2023 results conference call. We started 2023 strong as we made meaningful progress across all our functional areas. I'm very pleased to report on our record-breaking first quarter 2023 financial performance as shared in yesterday's press release, I will now highlight several key points. We achieved total revenues of $85.4 million during the quarter, representing 98% growth year-over-year. The exceptional performance reflects Firdapse product net revenues of $57.5 million, achieving a net revenue increase of 34% year-over-year. Total revenues were bolstered by Fycompa net product revenues of $27.8 million for the approximately 2 months that we owned the US rights to the product during the 2023 first quarter.

non-GAAP income for the first quarter was $46.8 million, or $0.44 per basic share and $0.41 per diluted share. This excludes from GAAP net income, non-cash stock-based compensation, depreciation, amortization of intangible assets in our income tax provision. GAAP net income for the first quarter was $29.6 million, or $0.28 per basic share and $0.26 per diluted share, which reflect, among other expenses, the expenses related to the amortization of the intangible assets associated with the acquisition of Fycompa and Ruzurgi, which were approximately $6.5 million. The expenses related to the transition services agreement with Eisai for Fycompa were approximately $7 million for the two-month period, and $2.9 million in non-cash stock-based compensation. We ended the quarter with a cash position of about $148 million.

The outstanding first quarter performance reinforces the belief in our strategy. We reaffirm 2023 total revenue guidance of between $375 million-$385 million, representing a 75%-80% increase in total revenue compared to 2022. We expect operating expenses of $160 million-$170 million for the full year 2023, which includes amortization expenses of about $32 million for this year. This also includes the operating expenses directly related to Fycompa, which at this time we estimate to be $40 million-$45 million per year. Keep in mind that we are still fine-tuning our pro forma expenses to account for the Fycompa acquisition. We are also forecasting that non-cash, non-GAAP net income for 2023 will be between $195 million-$205 million.

During the first quarter, we announced that we successfully completed the purchase of the US rights to Fycompa by January 24, 2023. This marks an important milestone for Catalyst as we broadened our commercial product portfolio with the first and only non-competitive AMPA receptor antagonist for epilepsy, further augmenting the already strong revenue growth of Firdapse. Effective May 1, we onboarded the full US commercial team for the Fycompa brand, including marketing, promotion, and medical support activities. I'm pleased to report that the Fycompa commercial medical affairs integration is for the most part completed, and our newly established franchise teams are actively field engaged as of this week. We're excited to welcome our new Fycompa team members who bring a wealth of passion, experience, and expertise in the epilepsy field to the Catalyst family.

Building upon our exceptional commercial capabilities, we are poised to fully benefit from our diversified portfolio to achieve targeted run rate synergies that position Catalyst to benefit from this long-term revenue and income opportunity. Moving on to our clinical business. We are making meaningful progress focused on expanding the use of Firdapse. The phase 3 registrational study in Japan is progressing on plan. We anticipate study completion by the end of 2023, and assuming success in that study, we anticipate an NDA submission to the Japanese regulatory agency by our partner, DyDo Pharma, in the second quarter of 2024. Based upon an acceptance of the NDA to market the product in Japan, we also plan to accelerate our global growth expansion strategy for Firdapse into other countries in Asia and South and Central America.

Initiatives are already underway to identify potential partnership opportunities in these territories as part of our longer-term strategic growth plans. Along with our global expansion plans, we are also planning to seek an increase from the FDA in the maximum daily dose of Firdapse from 80 milligrams per day to 100 milligrams per day. As we know, there are quite a number of Firdapse patients that could benefit from being treated with 100 milligrams per day. Just last week, we received the minutes from our Type C meeting with the FDA regarding the supplemental NDA submission requirements for the application, seeking to expand the maximum daily dosage for Firdapse from 80 to 100 milligram. Based on the positive response from the meeting, we anticipate filing the sNDA, seeking approval of the change early in the third quarter of this year.

During this quarter, we've gained significant ground in identifying acquisition opportunities towards building a diversified, rare disease portfolio. Our key priority on the strategy front remains to smartly continue to broaden and diversify our product portfolio through collaborative global partnerships, acquisition of commercial stage assets, and/or acquisitions of companies that own those type of assets. Our company is fully resourced, there is capacity from multiple perspectives to tuck in rare disease programs with a preference towards neurology, commercial or late-stage assets. Our mission, which has been driven our strategy since inception, is to improve the lives of those who suffer from rare and difficult to treat diseases. This commitment to providing these patients with safe and effective medicine while enabling affordability and accessibility remains core to our values.

With that in mind, we are proud to share that earlier this month, we released our inaugural ESG report highlighting our commitment and progress on environmental, social, and governance initiatives that benefit our patients, people, and communities. We look forward to advancing our ESG journey as we continue building our strong and sustainable foundation. Without question, we achieved meaningful results as we successfully executed across multiple initiatives while delivering an exceptional financial performance that exceeded expectations. Looking ahead, we are well-positioned for sustained growth with our two branded products and a sound strategy for our company's future, supported by our recent achievements. Coupled with our expanding capabilities and prudent approach to capital allocation, we will continue executing our long-term vision to drive sustainable growth and deliver value for all our various stakeholders.

I'll now turn the call over to Jeff Del Carmen, our Chief Commercial Officer, who will provide an update on our commercial activities.

Jeffrey Del Carmen (CCO)

Thanks, Pat. Good morning, everyone. As Pat mentioned, Catalyst had an excellent start to 2023, driven by the combination of strong Firdapse net revenues and compelling revenues from Fycompa. The record Q1 total net revenues of $85.4 million positions Catalyst well to achieve our combined 2023 revenue guidance of $375 million-$385 million. First, let me start with Firdapse performance. Q1 net sales of $57.5 million represents a 34% increase quarter over the same quarter last year, a direct result of a sustained cadence of new patient starts, high compliance, and consistent low discontinuation rates.

Prescription approval rates were greater than 90% across all payers, government or private commercial insurers. Patients enrolled in Catalyst Pathways, including those who are covered by Medicare in accessing foundation assistance, had an average copay of less than $2 per month. Our strategic growth initiatives will continue to drive organic growth moving forward. LEMS education programs targeting healthcare providers have already resulted in a significant increase in voltage-gated calcium channel antibody tests, which will shorten the diagnostic journey for LEMS patients and lead to more patients being eligible for treatment with Firdapse. Additionally, the recently hired oncology thought leader liaison will focus on the top 30 thoracic oncology centers to accelerate diagnosis of small cell lung cancer LEMS patients. Currently, greater than 80% of small cell lung cancer LEMS patients are undiagnosed.

We continue to maintain a pipeline of approximately 450 diagnosed LEMS patients that are not yet on Firdapse, which contribute around 50% of new patient enrollments each quarter. Let me provide an update on Fycompa. We have established a new commercial Fycompa franchise business that is highly synergistic with our Firdapse business. The integration has gone exceptionally well, a credit to the entire organization and our commercial capability to execute flawlessly without interruption to the Firdapse business. The Fycompa sales force and marketing team are fully staffed with 34 highly experienced personnel with a deep understanding of Fycompa and a passion to help patients living with epilepsy. During the first week of May, we successfully held our Fycompa launch meeting, in which our Fycompa commercial team was trained and certified.

On conclusion of our Fycompa franchise launch meeting, the entire Fycompa team and our corporate leadership were all energized about future growth opportunities. A Fycompa sales team of 27 regional account managers and 3 area business directors were deployed into the field effective May 5th. Q1 net revenues for Fycompa were $27.8 million for the approximately 2 months that we own the US right to the product, which adds further confidence towards achieving our Fycompa full year 2023 net product revenue forecast of $130 million. Adding Fycompa to our portfolio is an ideal strategic fit for our existing commercial infrastructure, with a 45% overlap in Fycompa and Firdapse physician call points and an increased share of voice that will benefit both franchises in driving growth.

In summary, we are making meaningful progress on sustained organic growth for Firdapse, as well as the successful execution of the launch of our Fycompa commercial business. We are highly focused on continued commercial execution excellence and remain confident in our 2023 total revenue guidance of $375 million-$385 million. I wanna thank the entire team at Catalyst for their unwavering commitment to patients and look forward to a successful 2023. I will now turn the call over to Dr. Steven Miller, our Chief Operating Officer and Chief Scientific Officer, for an update on R&D activities.

Steven Miller (COO and Chief Scientific Officer)

Thanks, Jeff. Our clinical development and regulatory strategy for Firdapse continues to focus on expanding access to all LEMS patients, enhancing the Firdapse patent estate to maximize its commercial potential, and integrating the newly acquired Fycompa product into Catalyst's organization. First, I would like to discuss our development efforts to increase the indicated maximum dose of Firdapse from 80 milligrams per day to 100 milligrams. As announced earlier this week, the agency has responded that they accept our approach for the filing of a supplemental NDA that we expect the sNDA submission to be submitted early in the third quarter of this year. Currently, there are a number of LEMS patients who are already being treated at a 100 milligram daily dosage of Firdapse after their physician worked with the pharmacy and insurance providers to justify the higher dose.

Other patients on the current indicated maximum dose of 80 milligrams per day, their physicians have expressed a need to increase the patient's daily dosage to 100 milligrams to optimize therapy. This planned supplement, if approved, will help those patients. We believe that our data set constitutes an acceptable basis for seeking a 100 milligram maximum indicated daily dosage for Firdapse. Regarding our global expansion, our sub-licensee partner, DyDo Pharma in Japan, has completed enrollment in their Firdapse phase 3 clinical trial, which is required to seek approval for the Japanese market. The safety follow-up phase of that study is ongoing. We continue to anticipate completion of that trial by the end of the year and filing of a Japanese NDA submission in the second quarter of 2024. It is estimated that there are about 1,200 to 1,300 LEMS patients in Japan.

Under our license agreement, the filing of the NDA in Japan is an important milestone for Firdapse and Catalyst because it expands our territory to further advance our efforts to expand the global footprint of Firdapse into other markets in Asia and in Central and South America. As previously re-reported, we recently completed the acquisition of the US rights to Fycompa or perampanel, which is the first and only approved AMPA receptor antagonist or inhibitor. Catalyst Fycompa integration efforts are progressing well, as Jeff reported, and we have completed the successful integration of the commercial teams. We are also well along with the process of integrating Fycompa's manufacturing quality and pharmacovigilance functions into our organization, and anticipate beginning to distribute Fycompa in Catalyst trade dress by the 4th quarter of this year. AMPA receptors enable fast excitatory synaptic transmission throughout the central nervous system.

Epileptologists believe that seizure generation and spreading can be dependent on overactivation of AMPA receptors. Hyperactivity induced by seizures might also alter AMPA receptor function and duration of excitation. Hyperactivation of AMPA receptors is highly toxic, and epileptologists believes it adds to secondary Fycompa, as an AMPA receptor antagonist, can act to reverse these effects and improve seizure control. Fycompa is approved as an anti-seizure medication to treat partial-onset seizures with or without secondary generalized seizures in people with epilepsy who are 4 years of age and older, and with other medications to treat primary generalized tonic-clonic seizures in people with epilepsy who are 12 years of age and older.

Moving on to our medical information function, Catalyst Neuromuscular Medical Science Liaisons or MSLs are actively reaching out to oncology healthcare providers to build relationships and provide education about the importance of testing their patients for LEMS in order to expand the use of Firdapse by those patients. Oncologists that already treat LEMS in their practices have found that patients treated with Firdapse maintain muscle strength, improving the patient's and physician's perception of well-being and the patient's ability to maintain functional mobility. All of these domains are critical for the patient's quality of life. Catalyst has also onboarded the new Fycompa MSL team and their new director, all with prior epilepsy experience to support Fycompa. Fycompa is a mature product for which extensive published information and real-world data is available.

The MSL team will bring this information to healthcare providers to treat epilepsy and also to address any questions that these physicians may have about using Fycompa. As previously reported, a recent publication identified a number of rare epilepsies that were shown to respond to Fycompa treatment. As a service to the physician community, Catalyst provides support for the development of continuing medical education, or CME programs, that are part of the formal ongoing education of healthcare providers. Catalyst has, over the past three years, provided support for three CME programs for various aspects of the diagnosis, treatment, and management of LEMS. Over this period of time, over 8,000 healthcare providers have utilized the CME program's learning modules, and about 2,500 have taken the CME test in order to be granted CME credit for the maintenance of their medical licenses.

Last quarter, we responded, we sponsored a new LEMS CME program that targeted oncologists that treat small cell lung cancer due to the correlation between this cancer and associated LEMS. In just a few months, this recent program has already had 1,200 learners and 280 CME test takers. In short, through these programs, we have now reached far more healthcare providers than there are LEMS patients, and these program successes may be one of the many contributing factors to the overall commercial success of Firdapse for the treatment of LEMS. Catalyst will continue to support the creation of new CME programs for both Firdapse and now for Fycompa in the future.

Moving on to business development, Pat described our significant portfolio expansion with the acquisition of Fycompa in the first quarter. We are continuing to identify additional assets for potential acquisition. It is also important to point out how the Fycompa acquisition fits into our broader business development strategy. The specific biochemical origins of epilepsy are increasingly being elucidated, the field of epilepsy treatment is evolving into a precision medicine approach, with increasing numbers of rare epilepsies being identified and targeted therapies being developed for each. A rare disease company, Catalyst acquisition of Fycompa is our entree into this expanding therapeutic area. Catalyst is actively pursuing additional therapies that are commercial stage or very late-stage products in development, including those that extend beyond epilepsy and neuromuscular rare disease. At this time, I would like to turn the call over to Alicia Grande, our CFO.

Alicia Grande (CFO)

Thanks, Steve. Results from Catalyst first quarter 2023 have set us on pace for another year of exceptional financial performance and strong execution. On the business development front, we were very busy as we closed the previously announced acquisition of the US rights to Fycompa for $160 million in cash, and integration efforts are underway. I remind you that Fycompa operations, including the results for the first quarter of 2023, include only a little over 2 months of activities since we acquired the product in late January. Our total revenues for the first quarter of 2023 were $85.4 million, a 98% increase when compared to total revenues of $43.1 million for the first quarter of 2022.

Product revenue net for the first quarter of 2023 for the Fycompa product acquired in late January, we expect to amortize on a straight line basis over the next approximately 5 years. Amortization for intangibles acquired in connection with both the Fycompa product acquired in late January 2023 and the Ruzurgi product acquired during July 2022 was approximately $6.5 million for the first quarter of 2023. We did not have any amortization in the comparable 2022 quarter, as we had not acquired either product during the first quarter of 2022. We expect intangible related amortization for our acquired products to be approximately $8.5 million per quarter for the rest of 2023.

Our effective tax rate for the first quarter of 2023 on an annualized basis was 20.8% compared to 24.2% for the first quarter of 2022. For 2023, the difference to the statutory federal income tax rate of 21% was primarily driven by state income taxes and anticipated annual permanent differences, offset by the Orphan Drug Tax Credit claim. The effective tax rate is affected by many factors, including the number of stock options exercised in any given period, and is likely to fluctuate in future periods. Cost of sale expenses were approximately $9.9 million in the first quarter of 2023, compared to $5.9 million in the first quarter of 2022, and consisted principally of royalties.

As a reminder, royalties for Firdapse have increased by 3% when net product sales exceed $100 million in any calendar year, we expect cost of sales to trend higher as the year progresses. Cost of sales for Fycompa in 2023 is exclusive of amortization of intangible assets. Research and development expenses were $3.6 million in the 1st quarter of 2023, slightly up from $3.4 million in the 1st quarter of 2022. SG&A expenses for the 1st quarter of 2023 totaled $29.7 million, compared to $16.4 million in Q1 2022. SG&A expenses decreased slightly as a percentage of total operating expenses to 60% for Q1 2023, compared to 64% for Q1 2022.

The overall difference in SG&A expenses in the first quarter of 2023 was principally due to commercial expenses under the transition services agreement related to Fycompa, filed during January 2023, and our contributions during the first quarter of 2023. The 501(c)(3) organizations, where the contributions will be used over the full year 2023, as previously noted. As reported, we ended the quarter with cash and cash equivalents of $148 million. As a reminder, during January 2023, we used approximately $162 million of our available cash for the acquisition of the US rights for Fycompa. We believe our remaining funds continue to allow us the financial flexibility to fund our existing R&D programs, as well as support our strategic initiative of acquiring opportunities leading to future growth and value creation.

More detailed information and analysis of our first quarter 2023 financial performance may be found in our quarterly report or form, on Form 10-Q, which was filed with the Securities and Exchange Commission yesterday, May 10th, and can be found on our investor relations page on our website at www.catalystpharma.com. With that, I'll turn the call over to Pat.

Patrick McEnany (Chairman and CEO)

Thanks, Ali. In closing our prepared remarks, we are very optimistic about our path forward as we continue to strengthen our position as a leader within the neuroscience space. We are well-positioned to drive sustainable growth with our sights set on further expansion that aligns with our core mission to deliver value to patients, healthcare providers, and shareholders. I'm proud of our team's accomplishments, and I thank all of our employees for their dedication and ongoing commitment to the patient communities we serve. At this time, I'd like to turn the call over to the operator to open the line for questions.

Operator (participant)

Thank you. We will now be conducting a question and answer session. If you would like to ask a question, please press star one on your telephone keypad. The confirmation tone will indicate your line is in the question queue. You may press star two if you'd like to remove your question from the queue. For participants using speakerphone, it may be necessary to pick up your handset before pressing the star key. One moment please while we poll for questions. Thank you. Our first question is from Joon Lee with Truist Securities. Please proceed with your question.

Les Sulewski (Analyst)

Good morning. This is Les on for Joon. Thank you for taking my questions. First I'll start on Firdapse. Can you talk about any seasonal trends that you might have seen and any other impacts that drove the quarter-over-quarter slide in the Firdapse sales?

Patrick McEnany (Chairman and CEO)

Did you say slide in the product sales?

Les Sulewski (Analyst)

Quarter-over-quarter, sequentially, yes.

Patrick McEnany (Chairman and CEO)

Yeah. Jeff, do you wanna take that question?

Jeffrey Del Carmen (CCO)

Sure. What we saw in Q1, we're very pleased with the net revenue results in Q1. The typical seasonal factors impacted the quarter. You know, such as what patients try to do in December, to ensure that they have medication over the holidays, they try to pull forward their prescriptions or their shipments into December. That impacts January a little bit, as well as some of these patients wanna get their drug before some of their benefits are reset, like co-pays and deductibles in January. That always impacts. It's not just for Firdapse, but it's for the industry in general. That's what happens in Q1. Also, for gross to net, the accrual % is always higher.

You know, you accrue the Medicare manufacturing rebates for all Medicare patients into Q1, and then gross to net will fall a little bit throughout the rest of the year. That's why you see inflated gross to net in the first quarter. I already also talked about the reauthorization process, but during that reauthorization process that for the most part happens in January, we provide bridge shipments for these patients so that they don't have an interruption in any of their medication. Those are the seasonal factors that we have, as well as, again, like I mentioned, all the other products in the industry get impacted with, for the most part, in the first quarter.

Patrick McEnany (Chairman and CEO)

Yeah.

Jeffrey Del Carmen (CCO)

That's very helpful.

Patrick McEnany (Chairman and CEO)

Jeff, Les, that's typical, especially in the specialty pharmaceutical area. Where you're dealing with one specialty distributor, and as Jeff pointed out, we gave away. Because on the bridge shipments, we can't charge for that. That's free of charge to patients that are waiting for reauthorizations. That's pretty typical in the business. That delay can be anywhere from one week to three weeks getting that reauthorization in which you're not only giving away free product, but you're giving away your revenue, if you will. I think if you look back at us historically, with a few exceptions, you'll see that Q4 versus Q1 is sort of flat. You know, there are exceptions, like in Q1 of last year, we onboarded a number of the Ruzurgi patients, so that was skewed. Otherwise, that's pretty typical.

Les Sulewski (Analyst)

I'll leave it with this, is once again, like we said throughout the call today, we confirm our forecast and guidance for Firdapse as well as Fycompa for 2023, given the strong performance with Firdapse revenues in Q. That's very helpful. I understand the impacts and the industry-wide, and I just wanted to get a sense if it's the same for you or if there was something else. Just thank you for the explanation and the details. Regarding just, I guess, you touched on the guidance. Has there been a shift in the mix of the guidance or is that still the same as you provided last quarter for the separate product line?

Patrick McEnany (Chairman and CEO)

Right now there's no change in the mix. The proportion is the same as we talked about last time, Les.

Les Sulewski (Analyst)

Got it. Okay. If we normalize Fycompa sales for the full 3-month run rate, that would imply a very strong quarter. I mean, probably, you know, more so than what everyone on the street expected, I'd say. Can you provide any color that you think drove that upside? Can we expect the remaining quarters to be similarly strong? I mean, if we do like a normalized run rate, something around $40 million per quarter, is that seem, you know, reasonable or is that a high number?

Patrick McEnany (Chairman and CEO)

Yeah. That's a great question, I'll let Jeff take that as well.

Jeffrey Del Carmen (CCO)

Sure. One quick thing is, with any integration, you have to allow for a transition period. You know, what we're doing is we feel like we're gonna be much more efficient with our sales force, where we are taking about 40% from a size perspective or a number of field personnel versus what was previously used for Fycompa and larger territories. With that, we wanna allow for that transition patient period to take hold. That's why we're very confident in the flat 2023 versus 2022.

Patrick McEnany (Chairman and CEO)

Let me add to that, Les. As Jeff pointed out in your earlier question, you know, this is more of a retail product, and as a result, your, you know, your rebates and your reauthorizations and things like that also are affected in the month of January. I don't think you can merely take those two months and extrapolate for the full year. I think that would be a mistake. I think that right now for the moment, and if that changes, we'll certainly provide an update on the guidance. I think that we're still very comfortable sitting with that 130 for this year.

Jeffrey Del Carmen (CCO)

Correct. We are very confident that we can demonstrate growth in 2024 with Fycompa.

Les Sulewski (Analyst)

Well, guys, thank you for the color. Good luck on the progress. Thank you.

Patrick McEnany (Chairman and CEO)

Great. Thank you.

Operator (participant)

Thank you. Our next question is from Joseph Catanzaro with Piper Sandler. Please proceed with your question.

Joseph Catanzaro (Analyst)

Hey, everybody. Thanks so much for taking my questions. Maybe first one for me. Just wondering if you could comment on the cadence of new patient enrollments in the quarter and the split you're seeing between non-tumor LEMS and tumor LEMS, and whether that dynamic is changing in any way with some of your efforts in oncology. With regards to your comments on, you know, further global expansion opportunities, wondering if we should think about that in the same way you sort of struck a collaboration with DyDo Pharma in Japan. Maybe related to DyDo Pharma, are there any milestones associated with the completion of their phase three and then subsequent NDA filing? Thanks.

Patrick McEnany (Chairman and CEO)

Sure. Jeff, do you want to take the first question?

Jeffrey Del Carmen (CCO)

Hi, Joe. Thanks for the question. The cadence of new patient enrollments in Q1 exceeded forecast. We had a strong new patient enrollment there in Q1, and we continue to see that strong cadence here thus far in Q2. As far as the mix goes, we estimate about a 25% of our new enrollments in Q1 are tumor LEMS patients. Keep in mind that, you know, some patients come in with that tumor LEMS ICD-10 code, there are some patients that come in that are LEMS patients and then over, we have to make an assumption. Over the next two years that they get their scans, they will be diagnosed with it. Based on all those factors, we're estimating that we're about 25% mix of our total enrollments are tumor LEMS.

Patrick McEnany (Chairman and CEO)

Joe, with regard to our global efforts, you know, the DyDo Pharma transaction for Japan serves as a really good model for other territories, in Asia and South and Central America. With regard to milestones, there is an undisclosed milestone based on a filing and acceptance by PMDA.

Joseph Catanzaro (Analyst)

Okay. Got it. That's all helpful. Thanks for taking my questions.

Patrick McEnany (Chairman and CEO)

Sure. Thanks, Joe.

Operator (participant)

Thank you. Our next question is from Charles Duncan with Cantor Fitzgerald. Please proceed with your question.

Pete Stavropoulos (Research Analyst)

This is Pete Stavropoulos on for Charles. Hi, patent team. Congratulations on all the progress for the quarter. You know, our first question is, you know, there's greater than, you know, 800 LEMS diagnosed patients, you know, that are being treated with Firdapse. You know, wondering what you can say, or what that number suggests, you know, in terms of penetration into the total LEMS opportunity. Are there any specifics you can give into penetration as a result of the oncology outreach efforts?

Patrick McEnany (Chairman and CEO)

I'll turn that over to Jeff Del Carmen.

Jeffrey Del Carmen (CCO)

Hey, Pete. What we're seeing right now is about a 25% penetration of current patients that are still living and diagnosed with LEMS. That's what we're seeing is about a 25% penetration. As far as your... What was your other question, Pete, about that in with the tumor LEMS patients, what we think we can get as far as penetration?

Pete Stavropoulos (Research Analyst)

Yeah. I was asking, like, what do you think the oncology efforts, outreach efforts has been, you know, impacting.

Jeffrey Del Carmen (CCO)

Excellent. What I can say there is the unfortunate news for small cell lung cancer LEMS patients that greater than 80% of these patients are undiagnosed. It's up to us to help these patients get diagnosed so that they can get benefit from the treatment with Firdapse. We've seen an uptick recently, just a slight uptick in 25% or so into the percent of our new enrollments that are small cell lung cancer LEMS patients. We're hopeful that we can get to about 30% of the mix will be the tumor LEMS patients. That's what our all our efforts are geared towards.

Pete Stavropoulos (Research Analyst)

Okay. You know, of the patients on Firdapse, you know, can you provide any, you know, the proportion of or any color about our patients, you know, who would like to go from 80 milligrams and be up dosed to 100 milligrams?

Jeffrey Del Carmen (CCO)

Yes. Right now, there are 40%. 40% of our current patients are on 80 milligrams or higher. We know that LEMS is a progressive disease and also from hearing from patients and physicians and from former Ruzurgi patients that were higher, that patients will benefit from a higher dose, some of these patients will. We feel like that's a huge benefit again to these LEMS patients. We do see some physicians that and patients that don't wanna go to the max dose. When you have a cap of 80, those patients that are at 60 or even 70 milligrams don't wanna go to 80 milligrams if that's the max dose. Now having 100, that allows for some of these patients to also benefit from a higher dose if that's what is necessary to help treat their disease.

Pete Stavropoulos (Research Analyst)

All right. Thank you. One question I guess on Fycompa. You know, so you know, it's, you know... What's the potential for developing Fycompa in rare epilepsy indications? You know, if I heard you correctly, you mentioned earlier in the call that epilepsy is evolving into a precision medicine approach. You know, are there any specific epilepsies you believe Fycompa can be utilized in?

Steven Miller (COO and Chief Scientific Officer)

There have already been 7 different rare epilepsies that were identified in a recently published paper that we did a press release on not too long ago. With regard to other rare epilepsies that may develop, the current label for Fycompa actually contains a fairly broad description of clinical characteristics of epilepsies that can be treated with the drug. If a specific rare epilepsy presents with those clinical characteristics, a physician would be able to prescribe Fycompa on label for that rare epilepsy and determine whether or not the patient responds well to that. As I said, there's already 7 that have been identified. Because of the relative maturity of this product, we do not anticipate doing any formal clinical trials to update the label to specifically call out specific rare epilepsies.

Our team of MSLs will continue to provide any information that is relevant for the treatment of rare epilepsies to the physician community with Fycompa.

Pete Stavropoulos (Research Analyst)

Okay. Thank you for taking our questions and congratulations on the quarter once again.

Patrick McEnany (Chairman and CEO)

Great. Thank you, Pete.

Operator (participant)

Thank you. There are no further questions at this time. I would like to hand the floor back over to Patrick McEnany for any closing comments.

Patrick McEnany (Chairman and CEO)

Yeah. I'd like to thank everybody for joining us today, and we look forward to our next earnings call. Have a great day.

Operator (participant)

This concludes today's conference. You may disconnect your lines at this time. Thank you for your participation.