Zevra Therapeutics (NASDAQ:ZVRA) reported third-quarter financial results on Tuesday. The transcript from the company’s third-quarter earnings call has been provided below.
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Summary
Zevra Therapeutics reported a 45% increase in total net revenue for Q3 2026, reaching $37.8 million, with $34 million from myplifa’s U.S. sales.
The company is focused on expanding myplifa’s label to include infants under 24 months and plans to file an sNDA in Q1 2027.
Zevra continues to expand its Global Expanded Access Program and is evaluating celiprolol for vascular Ehlers-Danlos syndrome in a Phase 3 trial.
The company has more than $270 million in cash and investments, providing flexibility to pursue strategic opportunities.
Management is optimistic about the potential expansion of the diagnosed patient population for NPC and continues to enhance disease awareness and diagnosis through strategic initiatives.
Full Transcript
OPERATOR (Moderator)
Please stand by. Your meeting is about to begin. Good afternoon and thank you for joining Zevra Therapeutics’ third quarter 2026 financial results and Corporate Update conference call. Today’s call is being recorded and will be available via the Investor Relations section of the company’s website later today. The host for today’s call is Nichol Ochsner, Zevra Therapeutics’ Vice President of Investor Relations and Corporate Communications.
Nichol Ochsner (Vice President, Investor Relations and Corporate Communications)
Thank you and welcome. To those who are joining us today, we will provide an overview of our recent accomplishments followed by a review of our third quarter 2026 financial results. I encourage you to read our financial results news release which was distributed this afternoon and is available in the Investors section of our website. Before we begin the call, please note that certain information shared today will include forward-looking statements. Actual results may differ materially from those stated or implied by any forward-looking statements due to risks and uncertainties associated with Zevra Therapeutics’ business.
Forward-looking statements are not promises or guarantees and are inherently subject to risks, uncertainties and other important factors that may lead to actual results differing materially from the projections made and should be evaluated together with the Risk Factors section in our most recent quarterly report on Form 10-Q, our annual report on Form 10-K and our other filings with the SEC. I’m pleased to welcome Zevra Therapeutics’ management team members participating in today’s call: Neil McFarlane, Zevra Therapeutics’ President and Chief Executive Officer, Josh Schaefer, our Chief Commercial Officer, and Justin Renz, our Chief Financial Officer. Now it’s my pleasure to hand the call over to Neil.
Neil McFarlane (Chief Executive Officer)
Thank you, Nichol, and welcome to everyone joining our quarterly call this afternoon. Zevra Therapeutics is a purpose-driven company redefining what is possible and bringing life-changing therapies to people living with rare diseases. In rare disease, the path is complex, the needs are urgent and the responsibility is significant. The people and families behind these needs are at the heart of what we do every day. Progress with the U.S. launch of myplifa, the first approved therapy for Niemann-Pick disease type C, or NPC, reflects the disciplined execution of our strategic plan and the determination, expertise and commitment of our people. Together we are building a trusted company with the capabilities and resolve to make a lasting difference. I’m pleased to share that total net revenue for the third quarter was $37.8 million, an increase of 45% compared to the third quarter of 2025, and net U.S. product revenue for myplifa was $34 million.
What we’re seeing in the market suggests that it may be just the beginning, particularly as we strengthen our understanding of the NPC community in the United States, where an estimated 900 people are living with NPC, of whom 300 to 350 have been diagnosed. These estimates are derived from a study published in 2021 prior to any approved treatment for NPC. In the third quarter we received 13 prescription enrollment forms, bringing the cumulative since launch to 197.
In rare disease, the number of identified patients often grows over time as awareness increases, more patients receive a diagnosis and new therapies are approved. Josh will share more details on how our initiatives are driving patient identification, diagnosis and treatment later in the call. Our commitment to realizing myplifa’s full potential and supporting the NPC community is driving our investment to expand patient access. In the second quarter, we published data from a multicenter open-label pediatric substudy evaluating treatment of NPC with arimoclomol in combination with miglustat in infants 6 months to less than 24 months of age. The substudy assessed safety and tolerability for up to 36 months of treatment and found that arimoclomol was well tolerated with no new observed safety signals.
On the heels of this, in the third quarter we engaged with the FDA in a Type B meeting to explore the potential of expanding the label to include infants less than 24 months of age. Based on the feedback, we expect to file an sNDA in the first quarter of 2027. Importantly, we believe myplifa is supported by a strong intellectual property portfolio. Myplifa is covered by patents listed in the Orange Book with expiration dates extending to 2041 and has U.S. orphan drug exclusivity for the treatment of NPC through 2031.
These provide distinct layers of protection: regulatory-based exclusivity and listed patent coverage. As mentioned on previous calls, we are also pursuing a potential patent term extension through the U.S. Patent and Trademark Office, and based on the current processing times, we now anticipate a decision from the USPTO in 2027. In the EU, the reexamination process for our Marketing Authorization Application, or MAA, for arimoclomol is underway by the Committee for Medicinal Products for Human Use, or CHMP. As part of the reexamination, a new Rapporteur and Co-Rapporteur have been appointed to lead the CHMP’s evaluation. Additionally, an ad hoc expert group, or AHEG, will meet to review the data and provide their specialized scientific and clinical expertise. This is an important forum for experts in the field to offer perspectives that will contribute to the CHMP’s procedural discussions.
We believe the totality of the clinical and real-world evidence supports the clinically meaningful benefits observed in patients treated with arimoclomol. The significant unmet need in Europe motivates us to keep working on behalf of people living with NPC, and we expect the CHMP’s opinion in the fourth quarter. Due to the proximity of the AHEG and CHMP meetings, we will be brief in addressing questions regarding the ongoing procedure. Turning to our Global Expanded Access Program, or EAP, we remain steadfast in our commitment to the NPC community and supporting access to arimoclomol for eligible patients. Our Global EAP has enrolled 134 patients and is comprised of compassionate use and named patient reimbursement programs. As previously discussed, quarterly reimbursement amounts fluctuate based on the timing and cadence of orders. Of note, this past quarter we expanded the program to include an additional territory and continue to evaluate more opportunities to expand access to patients globally.
Moving to our late-stage pipeline, celiprolol is being evaluated for the treatment of vascular Ehlers-Danlos syndrome, or vEDS. vEDS is a rare inherited connective tissue disorder caused by a change in the COL3A1 gene leading to fragile walls in blood vessels and hollow organs, which can cause arterial rupture or dissection, among other complications. With our Phase 3 DISCOVER trial, we are confirming the ability of celiprolol, our selective adrenoceptor modulator, to reduce the mechanical stress on these tissues, thereby lowering the risk of major events.
We enrolled 6 new patients in the third quarter for a total of 72. As a reminder, the trial was designed with an interim analysis triggered at 28 events and there have been four confirmed events through the end of Q3. We’ve discussed previously that we’re focused on advancing the ongoing DISCOVER trial and are exploring options to accelerate the program by engaging with the FDA. Following a Type C meeting in the first quarter, we recently held a second Type C meeting to discuss a potential submission strategy with the addition of real-world data sets.
While we await formal meeting minutes to confirm next steps, we’re developing a statistical analysis plan to include the retrospective data sets with the intent to have a follow-up discussion with the agency in the first half of 2027. Across our business, we remain focused on advancing key opportunities while maintaining a strong financial position with more than $270 million in cash, cash equivalents and investments as of the end of Q3. We believe we have the flexibility to pursue near-term opportunities with myplifa in the U.S. and broadening access for patients in global markets as well as advancing our development pipeline. I’ll now ask Josh to review our commercial momentum.
Josh Schaefer (Chief Commercial Officer)
Thank you, Neil. We continue to build the foundation for sustained MIPLYFA growth through patient identification, disease awareness and stakeholder engagement. Our progress is reflected not only in the 197 prescription enrollment forms submitted since launch, but also in our broader activity and growing awareness across the NPC community. In the third quarter, the 13 prescription enrollment forms we received included a balance of adult and pediatric patients, as well as a meaningful proportion of newly diagnosed patients.
As a reminder, NPC is a fatal lysosomal storage disorder leading to abnormal lipid accumulation, which over time causes progressive dysfunction of the nerves, brain and other organs. This results in neurodegenerative and visceral symptoms, including negative impacts on speech, cognitive function, fine motor skills and the ability to swallow. The symptoms are variable across patients, and many people with NPC experience a prolonged period before diagnosis due to the lack of disease awareness.
Disease progression is irreversible and varies based on the age of symptom onset. MIPLYFA, in combination with miglustat, has been established as a foundational treatment for NPC and has demonstrated a halting of disease progression at 12 months and rapid improvement by the first assessment at week 12 based on the NPC Clinical Severity Scale. This is further reinforced by the recently published clinical practice guidelines, as well as real-world evidence confirming MIPLYFA’s long-term efficacy and safety profile. We published new data in the third quarter and at the recent SSIEM Annual Symposium.
Findings were presented from our early access programs showing sustained disease stabilization across age groups, including adults. Since launch, we have observed high patient retention and refill rates consistent with what we observed in our EAP study. This data helps support our market access position and payer coverage, which is currently 69%. The majority of remaining patients receive coverage through medical exception pathways. This broad coverage is facilitated through our ongoing dialogues and engagement with payers, including regular presentations and conference participation.
Our integrated commercial strategy is focused on identifying new patients, accelerating their diagnosis and expanding access to treatment. Because NPC symptoms vary widely, the disease can be difficult to diagnose. We support overall disease awareness with our campaign called Learn NPC: Read Between the Signs. We also support early diagnosis through collaborations with genetic testing providers. Additionally, we identify healthcare professionals who may be treating NPC patients through our proprietary analytics model, and we engage these professionals in discussions focused on disease awareness and relevant treatment options.
Many of these clinicians may be treating their first and perhaps only NPC patient. To facilitate education and strengthen the understanding of treatment options, our Expert Connect program provides clinicians less experienced with NPC direct access to experts in the field. We are encouraged by the impact of these efforts. Our digital presence spans multiple platforms, including the disease awareness website, the MIPLYFA-branded website and our patient and caregiver channels. In the third quarter, we saw increased traffic across all digital channels with growth in display impressions, search clicks and click-through rates among HCP and patient audiences. Specifically, our disease awareness website delivered strong results with an increase of 123% in site visits quarter over quarter, from 25,000 in Q2 to nearly 56,000 in Q3.
This was in part driven by the launch of our new NPC Knowledge Hub, which provides educational resources for patients and caregivers. These results demonstrate some of the impact we’re making in expanding disease awareness and supporting Zevra’s focus on accelerating time to NPC diagnosis, activities which have directly led to enrollments. In addition, we expanded our engagement with the NPC patient advocacy community and strengthened our scientific leadership through participation in national and regional congresses. During the quarter, we also held scientific steering committee meetings with NPC experts to gather input on diagnosis, treatment decision-making and long-term disease management. Based on analysis of medical claims data, we believe that the number of individuals with NPC in the US may be higher than previous estimates.
With our flexible commercial model, we are able to scale quickly to respond to new information. As a result, we modestly expanded our customer-facing teams and broadened our reach in community practices outside of the Centers of Excellence where we continue to focus. Together, these efforts reflect a comprehensive approach to connecting patients with appropriate treatments and access. We’re grateful for our partnerships across the NPC community and proud to support those living with this devastating disease.
With that, I’ll turn the call over to Justin.
Justin Renz (Chief Financial Officer)
Thank you, Josh. Before I begin, I want to encourage everyone to refer to our quarterly report on Form 10-Q, which we intend to file later today, for more detailed financial information. Our revenue growth and the strength of our balance sheet driven by MIPLYFA, we believe we have a solid financial foundation to support ongoing operations to execute our corporate priorities. In the third quarter of 2026, we generated total net revenue of $37.8 million, which was an increase of $11.7 million, or 45%, compared to $26.1 million in Q3 2025. This is comprised of $34.0 million for MIPLYFA in net sales in the US, $0.2 million from OLPRUVA and $3.6 million in net reimbursements from the global EAP. Revenue from MIPLYFA in the US grew 12.6% compared to the second quarter this year and 52% compared to the third quarter of last year.
During the third quarter of 2026, our operating expenses were $24.1 million, which was an increase of $3.7 million compared to the same quarter a year ago. R&D expense was $4.9 million for Q3 2026, which was an increase of $1.5 million compared to Q3 2025 due primarily to our ongoing investment in arimoclomol and in the Swap World Development Program. SG&A expense was $19.1 million for Q3 2026, which was an increase of $2.2 million compared to Q3 2025, primarily due to commercial and infrastructure-related costs.
We recorded a $0.5 million income tax provision in Q3 2026. Net income for the third quarter of 2026 was $19.2 million, or $0.31 per basic and $0.26 per diluted share, compared to a net loss of $0.5 million, or $0.01 per basic and diluted share, in the same quarter a year ago. In the third quarter, we recorded a favorable mark-to-market fair value non-cash credit of $5.5 million related to our outstanding common stock warrants, which is included in the other income section in our consolidated statements of operations.
As of September 30, 2026, total cash, cash equivalents and investments were $270.9 million, which was an increase of approximately $10.7 million compared to June 30, 2026. In summary, we are in a strong financial position. We have the capacity and flexibility to advance our strategic priorities. Now I’ll turn the call back to Neil for his closing remarks.
Neil McFarlane (Chief Executive Officer)
Thanks, Justin. We believe we’re well positioned to build on our momentum with MIPLYFA in the US, expanding access to arimoclomol globally and the path to potentially accelerate the development of Soliprolol. With a solid corporate foundation underpinning our strategic plan and a shared commitment to putting patients first, we are dedicated to making a meaningful impact for all Zevra stakeholders.
OPERATOR (Moderator)
Please open the line for questions. At this time, if you wish to ask a question, please press star one on your telephone keypad. You may remove yourself from the queue by pressing star two. We’ll take our first question from Kristin Kluska with Cantor Fitzgerald. Your line is open. Please go ahead.
Kristin Kluska (Analyst at Cantor Fitzgerald)
Hi everyone. Congrats on a really strong quarter. Thanks for taking the questions. I had two of them. The first question is, during your prepared remarks you noted that you are looking at near-term opportunities for MIPLYFA in the US. Given your strong cash balance, was hoping you could further elaborate on that. Is that driven by your comments about the sNDA? Excuse me,
Neil McFarlane (Chief Executive Officer)
Kristin, I’m not sure if I understood the question, but let me see if I can answer it two ways. One, yes, we are looking at near-term opportunities for MIPLYFA, and the fact that we published our pediatric sub-study earlier this year, and after that we went to the agency to explore a Type B meeting and the opportunity to potentially expand the label to include patients less than 24 months of age. That feedback really informed our strategy to move forward with a planned sNDA in Q1 of ’27.
So we do think that that is a great opportunity, especially now that we’ve got this intellectual property Orange Book listing, as we mentioned in our last quarter, out to 2041.
Kristin Kluska (Analyst at Cantor Fitzgerald)
Thanks so much for that. And then just one question on the US commercial launch. It seems like with the new numbers today that collectively you have about two-thirds of the US patient population, which is really impressive in slightly over a year. Just looking forward, I’m curious how we should be setting expectations. Do you still expect there are more to unlock for the already diagnosed patients, or should we be thinking that more of the near-term new enrollment forms are really going to be driven by any new potential patient adds?
Thanks so much again.
Neil McFarlane (Chief Executive Officer)
Yeah, thanks, Kristin. So yes, you’re right. We have come out of the gate and, in the first seven full quarters post launch, been able to get to a large portion—you said two-thirds—of the diagnosed patient population. The good part about what we see today, and I think what’s providing us a lot of confidence—confidence that we’re somewhere between 350 and 900 patients in the United States—is because we’re learning a lot. We’re seeing newly diagnosed patients.
We’re seeing the efforts that we have, and I’ll ask Josh to talk a little bit more about some of these specifics. The learnings around adult patients as well as the pediatric patient split, around 50% each, that we really have now seen, that the market we expect is estimated to be bigger than what we had originally anticipated between that 350 and 900. So we’ve got a long way to go. But I’ll ask Josh to talk a little bit about some of these learnings and, quite frankly, why we did this modest expansion.
Josh Schaefer (Chief Commercial Officer)
Yeah. Thanks, Kristin, for the question. As we noted, we have 197 enrollments since launch and we’ve been really pleased to see that it’s this great balance between pediatric patients and adult patients. In addition, we’re seeing a pretty stable number of new patients coming in every quarter. All of that, supplemented then by this analysis that we’re doing of the claims data, which we’ve really been digging into, suggests that there are more patients out there than we had originally estimated.
We’ve consequently expanded our sales team to be able to find those patients. It’s early days, but we do have confidence that those patients are out there and that the market potential may be larger than we had originally estimated.
OPERATOR (Moderator)
Thank you. We’ll move on now to Sumant Kulkarni with Canaccord. Your line is open. Please go ahead.
Sumant Kulkarni (Analyst at Canaccord Genuity)
Thanks for taking your questions. I have three. You made some qualitative comments in your prepared remarks, but given the numbers in diagnosed and treated patients and prevalence data, which were as of 2021, what’s your latest understanding on how much higher those numbers could be now that MIPLYFA has been approved for a little more than two years in the US, and what fraction of the patient population is less than 24 months of age?
Neil McFarlane (Chief Executive Officer)
I’ll see if I can start that off and ask Josh to answer some of the specifics. You know, Sumant, we mentioned this a number of times throughout the last year and a half, and a lot of times what you see when you get a product launched is, first time in a rare disease, you start to see the tide rising and you see newly diagnosed patients come in, which we’ve now seen for about the last five quarters in a row, a meaningful amount of newly diagnosed patients in addition to children and adults.
And you start to see a little bit of the disease state awareness and the integrated strategy kind of lifting the awareness of the disease area, and you start to get more patients diagnosed. I think this is all, and a lot of these learnings are, what’s really driving our confidence that the market of what we thought were the 300 to 350 diagnosed patients, but a prevalent number around 900. We’re now starting to see that kind of tide rising that allows us to see between that 350 and 900.
So I’ll ask Josh to talk a little bit about what the 2021 publication, which is what we quote, versus what some of the learnings we’ve seen over the last seven quarters or so.
Josh Schaefer (Chief Commercial Officer)
So the early estimates were based off of a publication in 2021 that was looking retrospectively at claims data and looking at patients with the diagnosis of NPC subsequently. Now, fast forward five years, we’re seeing, well, first of all, the dataset is much more comprehensive than it was previously. But also as Neil has mentioned, with the launch of my plifa, with greater disease awareness, and as we noted, our disease awareness campaign has nearly doubled in terms of the number of visits to the site, both with clinicians and patients.
I think all of that is really helping to build a greater awareness of NPC. We’re also seeing diagnosis rates increasing, and so all of that gives us confidence that the number is greater than the original estimate.
Neil McFarlane (Chief Executive Officer)
I think you also asked a question. Sorry, Steven, I think you were also asking a question in regards to the pediatric population and what our thoughts were around the 900 patients, what that pediatric population looks like.
Josh Schaefer (Chief Commercial Officer)
Yeah, in terms of total addressable market and an increase with that expanded label potentially of 24 and under, it’s not a significant number of additional patients. It’s probably in the ballpark of 5%. But it’s very meaningful for us because it shows a continued increase in our expansion of the label, our ability to bring access to more and more patients. So we’re very excited about that. But in terms of absolute numbers, it does help grow the market, but it’s a relatively small patient size.
Sumant Kulkarni (Analyst at Canaccord Genuity)
Got it. And then how do you expect competitive dynamics to change in the NPC market now that Shionogi is acquiring IntraBio? And in your opinion, what might hold the key to the EMA potentially reversing its earlier decision on arimoclomol as part of this re-examination process?
Neil McFarlane (Chief Executive Officer)
Well, maybe I’ll start with the last question, then I’ll ask Josh to talk a little bit about the addition of additional companies coming into the marketplace, which we see as great. I’m going to be measured in my comments, Sumant, in regards to the activities ongoing with the AHEG that I discussed, as well as the CHMP opinion, much more to say that we continue to be confident in the totality of our data package, the fact that there’s a high level of unmet need, and the fact that we now have these experts that are coming to be able to actually opine on the clinical meaningfulness and statistical relevance of our data.
So with that I’ll ask you to stay tuned. And we do still expect our opinion in Q4. Josh, you want to talk a little bit about the additional companies coming into the NPC space?
Josh Schaefer (Chief Commercial Officer)
Yeah. So we view this as a really good thing for a number of reasons. One, I think it validates what we’re saying, that the market opportunity is larger, and really validates that there is room to continue to grow within the NPC marketplace. We have a great deal of respect for Shionogi, and we think that this is great for patients in general and this is going to help to increase awareness and diagnosis. And importantly, this helps reinforce my plifa’s strong foundational position, given that we are the only drug that has data showing that we work in combination with miglustat, and we know that combination therapies are the fastest-growing part of the market and are supported by the consensus guidelines. So overall, we see this as a really positive thing for the market.
Sumant Kulkarni (Analyst at Canaccord Genuity)
Thank you.
Neil McFarlane (Chief Executive Officer)
Maybe I’ll add a little bit to that, because we do think that part of the overall expansion of the market and the increase in the newly diagnosed patient population is because of the fact that we have a number of companies that are investing into disease state awareness. And we’ve seen that in the year-over-year numbers just out of the claims data alone and the number of patients in 2026 that have been newly diagnosed versus those patients in previous years.
So we’re really fortunate to be able to have the investment that is rising the NPC marketplace. And again, as Josh mentioned, the estimated numbers that we think the market of those diagnosed patients we believe now is higher than what we’ve seen from the previous numbers. We got some. We’re going to go test it and get it out there. But now, with the other folks that are coming in the marketplace, we think we can address it quicker with the COP with the strength of our data.
Sumant Kulkarni (Analyst at Canaccord Genuity)
Got it, thanks.
OPERATOR (Moderator)
Thank you. We’ll move on now to Lachlan Hanbury Brown with William Blair. Your line is open.
Truman (Analyst at William Blair)
Hi, this is Truman on for Lachlan, and congrats on the quarter. And you spoke on the sNDA for my plifa that you’ll be filing, but do you foresee any additional data will be needed for that, or will that be ready to go?
Josh Schaefer (Chief Commercial Officer)
So the feedback that we received post our pediatric sub-study is informing our submission strategy. We won’t get into the specific data, but we feel confident that we can file an sNDA in Q1 of 2027.
Truman (Analyst at William Blair)
Great, thank you.
OPERATOR (Moderator)
Thank you. We’ll move on now to Brandon Folks with H.C. Wainwright. Your line is open. Please go ahead.
Brandon Folks (Analyst at H.C. Wainwright)
All right, thanks for taking my questions, and congrats on another good quarter. A few from me, if I may. I know this one’s probably tricky to answer, but understanding you don’t contract, you know, and a lot goes through medical exception, can you just talk about the trend you’re seeing on net realized price per patient on therapy? You know, has it increased in the year? Has it been relatively stable? And does that change if a patient is on multimodal therapy?
Neil McFarlane (Chief Executive Officer)
I might start that one off and just let you know that we continue to have very strong reimbursement rates through both our covered lives as well as with the medical exception and the strength of our data driving a lot of those decisions. But Josh, talk about some of the specifics that you asked about.
Josh Schaefer (Chief Commercial Officer)
Yeah, so we continue to see really strong reimbursement rates. We continue to see really strong refill rates, very similar to what we saw in the Expanded Access programs. So, you know, I think that’s largely driven based on the strength of our clinical data and the work that our team is doing to get in front of payers. You know, we have data showing that we work in combination with miglustat. So, you know, certainly that helps to support that reimbursement, as well as the long-term efficacy data that we’ve been able to present publicly.
So all that really lends itself to, you know, continued high reimbursement and retention and refill rates. And yeah, we don’t see any change in that.
Neil McFarlane (Chief Executive Officer)
Yeah. And then just to be specific, you know, we see the patient enrollment forms that come in for my plifa and we see what enrollments are actually, and the high level of those enrollments that go on to commercial product. What we cannot answer for you is what happens with anybody else.
Brandon Folks (Analyst at H.C. Wainwright)
Thank you very much. Maybe just following on from an earlier question, just given the changing dynamic in the market with Shionogi and potential additional competitors, does that change how you think about sales and marketing spend in 2027? I know you talked about sort of the collective investment being a rising boat, but just how do you view your spend in 2027?
Neil McFarlane (Chief Executive Officer)
I’ll maybe kick that over to Justin in a moment, but you know, for us, we’ve been very disciplined in regards to our execution and I consider that we’ll continue to do that, but we may not provide you full guidance for 2027, Brandon.
Justin Renz (Chief Financial Officer)
Thank you. The modest expansion next year, we don’t foresee any meaningful increases from this year. So again, as Neil mentioned, we’re in a solid financial position, we have the capacity and flexibility, but I would not look to make major changes to our marketing spend going forward.
Brandon Folks (Analyst at H.C. Wainwright)
Great. Thanks for taking my questions, and congrats on a good quarter.
Neil McFarlane (Chief Executive Officer)
Thank you.
OPERATOR (Moderator)
Thank you. We’ll move on now to Jason Butler with Citizens. Your line is open.
Jason Butler (Analyst at Citizens)
Hi. Thanks for taking the question, and congrats on the quarter. Can you speak to the characteristics of the newly diagnosed patients that you’re seeing enrollments for in terms of age and disease severity? And is there any evidence that you’re actually seeing an acceleration of diagnosis in the population? And specifically, are any of these patients coming directly from your efforts to expand genetic testing? Thanks.
Neil McFarlane (Chief Executive Officer)
That’s great. These sound like a lot of really good specific questions. Exactly for Josh. But overall, I think we’re quite, you know, back to the learnings, we’re learning every day about what this market looks like. But really the newly diagnosed patients is what’s provided us a lot of confidence that this market is bigger than the 350 and somewhere between 350 and 900.
Josh Schaefer (Chief Commercial Officer)
Yeah. And I wish I could give you some sort of generalized characterization of these newly diagnosed patients, but they’re really across the entire marketplace. So we see some older patients, we see some younger patients. Many of them have come through either genetic testing, through our collaboration with some of those genetic testing providers. I think some of it is just coming from our efforts to really increase the disease awareness. So there is no real sort of overarching characteristic that I can provide to you.
Neil McFarlane (Chief Executive Officer)
I think a lot of this, Jason, like anything else, there’s no silver bullet. This is an integrated strategy that we have to be able to unlock earlier diagnosis through disease state awareness, being able to provide treatment options for physicians and make that education really readily available. And we see the opportunity to educate more people now because we’ve, hence the reason we did this modest expansion, because we see a larger number of potential patients out there in terms of what we’ve uncovered so far.
And then lastly, we want to make sure that we continue to provide access. Those three things we’ve invested in. And I think the team is doing an outstanding job in that. But it’s hard when you have such small numbers to be able to give you characteristics that might be meaningful. Except what I’ve said on previous calls, we always thought that this disease was a little bit more of a childhood-level disease. And the fact that we still see a fairly consistent 50% kids, 50% adults, and newly diagnosed in the same kind of ratios, those are learnings for us that we’re now piggybacking on to be able to go out and make sure we can do more disease state awareness, get more patients diagnosed, and then provide access to them.
Jason Butler (Analyst at Citizens)
Great. Thanks for taking the question.
OPERATOR (Moderator)
Thank you. And once again, if you would like to ask a question, that is star one on your telephone keypad, we’ll move next to Eddie Hickman with Guggenheim Securities. Your line is open.
Eddie Hickman (Analyst)
Hey, guys. Good afternoon. Congrats on all the progress and thanks for taking the question. You noted about half of the enrollments are adults who are milder. For the 13 forms this quarter, how did the adult share and the community practice share compare with the first half? And because the constraints seem to be, like, getting new clinicians rather than finding patients, do you think you would need a bigger field team or a medical fair spend to reach those milder adult patients?
And then on the sNDA, how many patients do you think are under 24 months that you could add to the TAM? Thanks.
Neil McFarlane (Chief Executive Officer)
All right, I’m going to take the first question, the last question. I’ll ask Josh to opine in the middle. Eddie, thank you for your question. And I want to make sure that I make something clear: just because you’re an adult patient doesn’t mean that you have milder disease. The trajectory and the natural history show that all patients will continue to progress. But it is not unusual for an adult patient who has progressed very slowly to then actually have a change that will allow them to start progressing at a much more rapid pace and be more impacted.
So I don’t want this to, I hope that this doesn’t come across that an adult patient means that they’re milder. That’s not always the case. And I think that we all need to be cognizant of the fact that these patients that are adults also, in order to be treated with bad in my Piplitha, you actually need neurological impact. And these patients are seeing neurological impact. So these adults that we’re seeing that are about 50% of our current prescription enrollment forms, we’re learning that some have been misdiagnosed with other diseases. We’ve had somebody recently who was misdiagnosed with multiple sclerosis but then finally ended up getting an NPC diagnosis many, many years later, genetically diagnosed.
And that patient was progressing. So we see a lot of these types. This is just one anecdote of many because, as you know, one patient, you know one patient. So that was one. Adults can have very aggressive disease as well as children. So to clarify that, the next thing I wanted to talk to you about was the sNDA. Our sNDA is an opportunity to be able to expand the label. But these patients we think are in the 900-patient prevalent numbers today. And as we continue to see newly diagnosed patients, we wanted to be able to provide both the physicians with the understanding of how to utilize the product as well as the payer community to be able to know how to be able to make it easier for them to gain access to the product.
So it’s not increasing the TAM per se or the prevalence per se. It’s part of this increased confidence that we’re seeing of the number of diagnosed patients that we believe is estimated to be larger than our original 300 to 350.
Josh Schaefer (Chief Commercial Officer)
Yeah. And then in terms of the size of the team to be able to reach these new patients, this is based on our analysis and increasing understanding of the claims data. And what I’m really pleased with was that we’ve been able to identify that there are more patients out there with NPC diagnosis. And very quickly we were able to expand the team modestly, but we were able to expand the team very quickly in order to respond to that new understanding of the marketplace.
I think this is a model that we’re going to adopt, one that allows us to scale up or down as we learn more about the market, as we identify these patients. So I think we’re right-sized for where we are today, but I’m really pleased with our ability to pivot quickly if need be based on the data. Thank you.
OPERATOR (Moderator)
Thank you. We’ll move on now to Kambiz Yazdi with U.S. Bancorp. Your line is open.
Kambiz Yazdi (Analyst at U.S. Bancorp)
Hi Dean, thank you for the question. Beyond intellectual property strengthening and potential label expansion to infants under 24 months, can you give us any color on what the next layer of life cycle management looks like for the Miplife franchise? Are there any formulation, combination, or next-generation compound strategies in the pipeline that we should be thinking about over the longer-term horizon? Thank you.
Neil McFarlane (Chief Executive Officer)
Yeah, thanks, Kambiz. And I appreciate the question. You know, as I mentioned, I believe on one of the earlier calls this year we looked at our Orange Book listing going out to 2041. Along with strengthening the moat of our patent portfolio with the patent term extension and the continued investments that we’re looking at, we also see the opportunity to be able to explore what additional indications or other areas we may want to look at. It’s really too early for us to come back to you and tell you anything at this point, but I do want to say that we are always exploring opportunities to maximize the franchise and even provide optionality for patients with NPC.
OPERATOR (Moderator)
Thank you and it appears that we have no further questions at this time. I’m happy to return the call back over to our hosts for any concluding remarks.
Neil McFarlane (Chief Executive Officer)
Thank you for joining our call today and we look forward to keeping you apprised of our future progress.
OPERATOR (Moderator)
Thank you. This brings us to the end of today’s meeting. We appreciate your time and participation. You may now disconnect.
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