Zevra Therapeutics - Earnings Call - Q2 2025

Executive Summary

• Revenue and commercial traction accelerated: net revenue grew to $25.9M in Q2 2025 from $20.4M in Q1 and $4.4M in Q2 2024, driven by MIPLYFFA net revenue of $21.5M and broader market access at 52% of covered lives.
• Reported GAAP diluted EPS was $1.21, buoyed by $148.3M net proceeds from the PRV sale; excluding the PRV and OLPRUVA impairment/obsolescence, adjusted net loss was $(3.2)M with adjusted EPS of $(0.06).
• S&P Global consensus for Q2 2025 revenue ($22.49M*) was beaten; however, consensus Primary EPS ($1.61*) contrasted with S&P’s actual Primary EPS of $(0.06)*, signaling a large headline miss if consensus assumed PRV-related gains. Clarify GAAP vs adjusted framing for investors [Values retrieved from S&P Global].
• Strategic catalysts: EU MAA submission for arimoclomol (MIPLYFFA) was filed, meeting 2H25 guidance, and European EAP expanded to 89 patients, setting up for potential country-by-country pull-through post approval.
• Risk/concern: OLPRUVA adoption remained slow (one enrollment in Q2), triggering a $58.7M non-cash intangible impairment and $11.7M inventory write-down; watch competitive dynamics and payer posture.

What Went Well and What Went Wrong

What Went Well

• Commercial momentum in MIPLYFFA: net revenue increased 26% Q/Q to $21.5M; covered lives rose to 52% with strong conversion/retention through patient services and medical exceptions.
• EU expansion: arimoclomol MAA submitted to EMA, meeting 2H25 timing; European EAP reached 89 patients, supporting future conversion upon country reimbursement.
• Balance sheet strength: $148.3M net PRV proceeds recognized as other income; cash, cash equivalents and securities at $217.7M; total debt ~$60.7M, providing flexibility independent of capital markets.

Quoted management:

• “Q2 net revenue reached $25,900,000… We completed the sale of our PRV for $150,000,000 and strengthened our balance sheet.”
• “We believe the remarkable performance in the second quarter sets a strong foundation for continued momentum.”
• “Our commercial organization has reached critical mass… prioritizing and executing on key strategies to deliver value [to] patients living with rare diseases.”

What Went Wrong

• OLPRUVA softness: only one Q2 enrollment, continued slow adoption; coverage at 79% but market dynamics (authorized generic) shifting against uptake.
• Non-cash charges: $58.7M impairment of OLPRUVA-related intangibles and $11.7M inventory write-down; cost of product revenue elevated to $14.0M including amortization and obsolescence.
• Operating losses before PRV: excluding the PRV gain and non-cash charges, the quarter reflects an adjusted net loss of $(3.2)M; SG&A elevated due to proxy-related professional fees and commercial launch activities.

Transcript

Speaker 0

Good afternoon, and thank you for joining Zevra Therapeutics' second quarter 2025 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. Please go ahead.

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 second quarter financial results. I encourage you to read our financial news release, which was distributed this afternoon and is available in the investor relations 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 actual results to differ materially from the projections made, and should be evaluated together with the risk factors section in our most recently filed quarterly report on Form 10-Q, annual report on Form 10-K, and other filings with the SEC. In addition, we will disclose certain non-GAAP information on today's call, specifically adjusted net loss and adjusted net loss per share. Reconciling information to GAAP can be found in our financial results news release. I'm pleased to welcome Zevra Therapeutics' management team members participating in today's call: Neil McFarlane, Zevra Therapeutics' President and Chief Executive Officer, LaDuane Clifton, our Chief Financial Officer, and Josh Schafer, our Chief Commercial Officer. Our Chief Medical Officer, Adrian Quartel, will be available for today's question and answer session.

Now, it's my pleasure to hand the call over to Neil.

Speaker 4

Thank you, Nichol, and thank you to everyone joining our update call this afternoon. At Zevra Therapeutics, our vision is to build a leading life sciences company whose core mission is to serve patients by bringing life-changing therapeutics to people living with rare diseases. We are demonstrating our ability to execute in a complex regulatory, clinical development, and commercial environment to advance promising therapies with an approach that balances science with patient need. As a reminder, this vision is driven by the four pillars of our corporate strategy: commercial excellence, pipeline and innovation, talent and culture, and corporate foundation. The execution of our strategy delivered remarkable performance in the second quarter and set the strong foundation for continued momentum in Q3. On today's call, we'll highlight several key achievements. Q2 net revenue reached $25.9 million, reflecting robust demand and effective operational execution.

We completed the sale of our Priority Review Voucher for $150 million to strengthen our balance sheet. We also submitted our Marketing Authorization Application, or MAA, for our Aramucamol in Europe, marking an important milestone in our geographic expansion efforts. Let's dive in with an update on the U.S. launch of MyQlifi, the first approved treatment for people diagnosed with Niemann-Pick disease type C, or NPC, and the only treatment that has been shown to halt disease progression by addressing its underlying pathology. As a quick reminder, NPC is an ultra-rare, relentlessly progressive neurodegenerative disease caused by inherited lysosomal storage disorder and leads to premature mortality. In the U.S., we estimate that roughly 300 to 350 patients have been diagnosed with NPC out of the estimated 900 people living with the disease.

The approval of MyQlifi marked a fundamental shift in the treatment paradigm, and we believe that its ability to address disease progression is an important differentiator that will lead to long-term success. We are incredibly proud of the work our team is doing to support the healthcare community and eligible patients with an intense sense of urgency. We've received positive feedback from both patients and physicians, which is reflected in a strong performance since launch, with a total of 129 prescription forms through the end of Q2, of which seven were enrolled in the quarter. Through rapid uptake in the early stages of launch, we have been able to reach over one-third of those diagnosed with NPC in the U.S.

This group primarily included patients in our Expanded Access Program, or EAP, who have been treated with MyQlifi for up to seven years, with the majority of patients remaining on therapy. Following the success of our early launch penetration, our commercialization efforts are focused on increasing both diagnosis and treatment access. The second segment of patients that we're focused on are those who have received an NPC diagnosis but may or may not be currently receiving treatment. These patients typically were diagnosed at centers of excellence and managed in coordination with a local provider for ongoing care. We've mapped these referral patterns beyond the centers of excellence and are engaging the broader group of prescribers. The last segment of our patients are those living with NPC but who have not been diagnosed.

Part of our contribution and responsibility to the patient community is to increase awareness and shorten the time to diagnosis for this devastating disease. We are demonstrating leadership through our Disease State Awareness Campaign to help drive early diagnosis. The treatment of NPC is multifaceted, and our program offers education and genetic testing options for individuals with suspected lysosomal storage disorders. With growing awareness of MyQlifi, we look forward to extending our reach to serve additional patients. Our work with advocacy organizations is very important, and our support has been well received. We often provide more details on our strong presence at recent conferences where Zevra Therapeutics and our specialty pharmacy engage with caregivers and people living with NPC. These are some of the ways that we're building our reputation as a reliable partner.

Another exciting opportunity for MyQlifi is in Europe, where we estimate approximately 1,100 people are living with NPC. One of our priorities is to secure European approval and determine the optimal go-to-market strategy to ensure access for a greater number of patients. Just a few weeks ago, we announced the submission of the Aramucamol MAA to the European Medicines Agency, delivering on the early side of our second half of 2025 guidance. This is another significant and timely milestone for Zevra Therapeutics.

To support the submission, we received guidance from EU regulators and assembled a robust data package, including new mechanistic and long-term clinical data generated since our FDA submission. We have demonstrated a synergistic effect with Nidostat, which is approved in Europe for NPC and is the standard of care in this more mature market, where we have an established Expanded Access Program, which increased to 89 patients enrolled at the end of the second quarter. To further our efforts in the scientific and medical community, our data from the Open Label Extension Study was recently published in the Journal of Molecular Genetics and Metabolism. The paper presents the impressive long-term efficacy and safety outcomes for NPC patients treated with MyQlifi in the 48-month Open Label Extension phase following the end of the 12-month pivotal trial.

Additionally, we published data showing that MyQlifi upregulates expression of genes belonging to the coordinated lysosomal expression and regulation network, or the CLIR network, targeting the underlying pathophysiology of NPC to improve pathology, reduce cholesterol accumulation, and prevent cell death. As the NPC marketplace evolves, shaping treatment guidelines will become more important for the NPC community. We believe our success in establishing MyQlifi's benefit to the largest data set of NPC clinical trial patients and having these data published in peer-reviewed journals will help inform treatment decisions. Turning now to Opruva, which is a treatment for certain urea cycle disorders, we previously discussed the limited pull-through we experienced in this launch, which continued in the second quarter with one prescription enrollment form.

Our refined marketing efforts over the last several months have focused on identifying the patients for whom Opruva is best suited, strengthening our patient support services, and addressing reimbursement challenges. However, adoption continues to be slow, and we remain cautious. Josh will provide more detail on the product performance, and LaDuane will provide financial details on the non-cash charge related to the prevailing trend later in the call. Moving on to our pipeline, we are advancing SoliPro as a potential treatment of vascular Ehlers-Danlos syndrome, or VEDS, in the phase 3 discovery trial. VEDS is a severe genetic connective tissue disorder characterized by a risk of dissection and rupture of the arteries, gastrointestinal tract, and uterus. SoliPro is an angiotensin receptor modulator believed to decrease mechanical stress on the vascular wall of large arteries and hollow organs.

In the second quarter, we enrolled seven patients in the discovery trial, bringing the total to 39 patients enrolled out of the 150 patients required for complete enrollment of the trial. The genetic testing initiative to identify patients who have the COL3A1 gene mutation, which causes the disease, is ramping up in collaboration with patient advocates, KOLs, treating physicians, and clinics, providing increased visibility and has the potential to accelerate future enrollment. We are making progress in advancing our vision of becoming a leading rare disease company through focusing on the patients we serve. Our strategy balances near-term operational excellence with the agility and financial flexibility to support initiatives that build sustainable value for patients and shareholders alike. Let me turn the call over to Josh, who will provide more details on our commercial products. Josh?

Speaker 1

Thank you, Neil, and good afternoon. Let's begin with MyQlifi for NPC. MyQlifi, in combination with Nidostat, is the only treatment shown to halt disease progression, 12 months, in a pivotal placebo-controlled study using the NPC Clinical Severity Scale, which is the only validated measurement of NPC progression, assessing clinically meaningful markers such as impairment of mobility, cognition, speech, and swallowing. In the second quarter, we received seven prescription enrollment forms, and we have increased our product net revenue by 26% quarter over quarter, a reflection of new patient demand as well as access and retention. A new enrollment is defined as a prescription submitted to our specialty pharmacy, which begins the benefits investigation process to determine reimbursement eligibility. In the second quarter, our coverage reached 52% of all covered lives. This is consistent with our expectations at this stage of the launch.

For those other patients who were not immediately covered, we've been able to achieve reimbursement through medical exception pathways. We remain actively engaged in discussions with payers to facilitate reimbursement, and our current focus is on demonstrating the clinical value of MyQlifi to payers, especially leveraging our long-term open label extension data, which showed durable clinical benefit for up to five years. Our field team, including case managers, continues to play a critical role on the front lines, working with patients and healthcare providers to navigate the reimbursement landscape. Their efforts have been instrumental in helping to overcome access barriers and allowing patients to receive timely support to secure coverage.

Additionally, our Amplify Assist Program, which is a centralized resource that assists clinicians, offices, and patients in navigating the reimbursement challenges and supporting coverage for all of our commercialized products, has been well received. To further bolster our penetration into the second segment of patients, those NPC patients who have been diagnosed and are either on other treatments or not being treated, we have a number of initiatives underway. We are emphasizing the robustness of our data with its strong presence at scientific, medical, and advocacy-related conferences where physicians and patients can learn more about MyQlifi's therapeutic impact. Last month, at the National Niemann-Pick Disease Foundation Conference, Dr. Barbara Burton, a key opinion leader and Professor at Northwestern University's Feinberg School of Medicine, presented an overview of MyQlifi and our unprecedented long-term data. We had a similarly strong presence at the Southeastern Regional Genetics Group Conference with four poster presentations.

As Neil mentioned, the recent publication of our Open Label Extension Study puts our long-term 48-month data to the forefront for physicians, highlighting the duration of clinical benefit. Specifically, the improvement in disease progression was seen at the first evaluated time point at 12 weeks and then continued for more than five years in a heterogeneous population of NPC patients with no new safety concerns. These results align with our pivotal trial data, which showed that MyQlifi, in combination with Nidostat, halted disease progression compared to placebo over a one-year study duration. The presentation of key data and insights like those provided in these recent publications are an important part of our strategy to raise awareness of the clinically meaningful impact that MyQlifi can have for patients, and we will continue to execute our publication strategy to support prescribing decisions.

The third segment of patients, those that are undiagnosed, are an important population for us. It is critical to shorten the time to diagnosis to halt the progression of disease sooner. Our Disease State Awareness Campaign, Learn NPC: Read Between the Signs, is designed to provide educational and genetic testing resources to support the diagnosis of new NPC patients. This program is helping to drive awareness of the disease, as well as identifying patients who were previously undiagnosed and may be candidates for MyQlifi. Turning to Opruva, UCDs are rare inherited metabolic disorders characterized by an excess accumulation of ammonia, which can be neurotoxic and lead to neurocognitive damage or even death. Although current treatments are effective, over 25% of hyperammonemia crises are caused by poor adherence to treatment.

Opruva was launched based on an established efficacy and safety profile, but with an innovative formulation that offers a palatable option in convenient premeasured single-dose envelopes for ease of use and ammonia control on the go. We believe in the benefits that Opruva offers people with UCDs. However, we have seen slower than expected uptake. The mature UCD market and patient satisfaction with existing treatments resulted in one prescription enrollment form in the second quarter. An authorized generic to the market leaders is anticipated, eventually creating a shift in their competitive dynamics. We believe Opruva's profile, as well as our patient support activities, will position us to compete in this changing landscape. We have been actively engaged in strategic negotiations with payers to facilitate reimbursement. In the second quarter, we saw our overall coverage increase to 79%.

In summary, our commercial organization has reached critical mass, and the strength of our capabilities is being leveraged across our portfolio, with the primary focus on MyQlifi. We are prioritizing and executing on key strategies to deliver value to patients living with rare diseases. I will now pass the call to LaDuane, who will present the financial results for the second quarter of 2025.

Speaker 3

Thank you, Josh, and good afternoon, everyone. In addition to the financial details included in today's call, we encourage you to refer to Zevra Therapeutics' quarterly report on Form 10-Q for more detailed information, which we intend to file later today. In the second quarter of 2025, we reported net revenue of $25.9 million, which includes $21.5 million from MyQlifi, $300,000 from Opruva, $2.6 million in net reimbursements from the French Expanded Access Program for Aramucamol, $1.2 million from royalties and other reimbursements under the Astaris license, and an upfront payment of $300,000 from the out-license of Dextrosin during the period. For our commercial products, MyQlifi and Opruva, we recognize revenue when shipments are received by the specialty pharmacy. Cost of product revenue for Q2 2025 was $14 million, which includes $1.6 million of non-cash intangible asset amortization.

We conduct quarterly assessments of the recoverability of certain intangible assets in inventory in accordance with U.S. GAAP. Based on the prevailing trends for Opruva, including enrollments, paid authorizations, as well as market and competitive landscape dynamics, we have recognized a non-cash impairment charge of $58.7 million for impairment of intangible assets and an inventory write-down of $11.7 million as of June 30, 2025. Operating expense for the second quarter was $24.2 million, which was an increase of $1.1 million compared to the same quarter a year ago. R&D expenses were $3.4 million for Q2 2025, which was a decrease of $7.1 million compared to Q2 2024 due to a decrease in third-party and personnel-related costs following the completion of the KP1077 Phase II trial.

SG&A expenses were $20.8 million for Q2 2025, which was an increase of $8.2 million due to professional fees incurred related to the proxy contest earlier this year, as well as other expenses associated with our commercial activities. During Q2 2025, we also recognized $147.9 million in other income, which includes $148.3 million in net proceeds from the sale of the Priority Review Voucher asset, which was completed on April 1, 2025. This one-time transaction has provided significant non-dilutive capital for the company and has further strengthened our financial position. Net income for the quarter was $74.7 million or $1.24 per basic share and $1.21 per diluted share. Excluding the one-time PRV sale, the one-time non-cash impairment charge, and the inventory obsolescence charge recognized during the quarter, adjusted net loss was $3.2 million or $0.06 adjusted net loss per basic and diluted share for Q2 2025.

For the same quarter in 2024, we reported a GAAP net loss of $19.9 million or $0.48 per basic and diluted share. We are pleased with the early stages of the MyQlifi launch and our continued progress in building our corporate foundation through solid execution. Our focus remains on executing the launch of our commercial products and on the development of our pipeline, which includes supporting both the Aramucamol MAA in Europe and the ongoing phase 3 trial for SoliPro. As of June 30, 2025, total cash, cash equivalents, and investments were $217.7 million compared to $68.7 million at the end of the prior quarter. Total debt was approximately $60.7 million. We believe that our existing capital resources continue to be sufficient to allow us to execute on our strategic priorities independent of the capital markets. Now, I will turn the call back to Neil for his closing remarks.

Speaker 4

Thank you, LaDuane. We are fortunate to be in a unique position with commercial therapies, a late-stage development program, and a solid balance sheet to execute on our priorities. We attribute our success and momentum to staying true to our mission and the dedication of our team committed to making an impact on the lives of people living with rare diseases. Our focus moving forward remains firmly on executing across our four strategic pillars to accelerate our trajectory for transformative growth and long-term value creation. Thank you. We'll now open the call for questions. Operator?

Speaker 0

Thank you. At this time, if you would like 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 Brandon Foulks with HC Wainwright.

Speaker 5

Hi, thanks for taking my question. Congratulations on all the progress. Maybe just starting in the U.S., in the most new patients start in Q2, or just any enrollments, and any trends you're starting to see in the U.S. in terms of enrollments, in terms of where they may be coming from?

Speaker 3

Brandon, you cut out a little bit in the first part of your comment. It sounded like you were asking about U.S. MyQlifi and any trends, but can I ask you to repeat the question?

Speaker 5

Yeah, absolutely. Just any specialties contributing most to new patient enrollments in Q2 and since launch as well? Just any trends you see in the U.S. in terms of patient enrollments who weren't in the Expanded Access Program?

Speaker 3

I lost Josh to comment on the specifics around specialties, but maybe I'll take the opportunity to be able to talk a little bit about the strong performance in Q1 to Q2 overall. You know, in Q1, we had MyQlifi revenue of about $17.1 million and a 26% growth from Q1 to Q2 at $21.5 million. That's really a lot to do with the pull-through of patients from the patient enrollment forms on our patient services group and reimbursement, walking through to help to drive patients through that funnel, through the reimbursement hurdles to get them onto commercial products. Tremendous job by the team in pulling that through. If you look at our total enrollments from Q1 to Q2, we're at 122 total enrollments, and then ending Q2 at 129.

That's coming from a fairly small number, but it's still a third of the patient population today that's been diagnosed, the 300 to 350. I'll pass it off to Josh a little bit to talk a little bit about the specialties and trends, but recall, these are small numbers in terms of the total number of patients in the U.S.

Speaker 1

Yeah, thanks for the question. We're seeing initially, you know, as we expected, that the early patients were coming from those clinicians who were part of our Expanded Access Program, and those are largely neurologists and pediatricians. Now, keep in mind that while many of these clinicians might have done training as a pediatrician, they are seeing both children and adult patients with Niemann-Pick disease type C. We're also seeing medical geneticists treating these patients, and we're starting to see an emerging small group of psychiatrists who are seeing these patients as well. This is very consistent with what we expected, and we're very pleased with what we're seeing so far.

Speaker 5

Great, thanks very much. If I may just shift gears to Europe, you've obviously done a tremendous job in the U.S. in terms of EAP conversion. Can you help us just think through the pushes and pulls of converting the EAP patients in Europe? Should you be approved there versus what we saw in that really quick and very good execution in the U.S.?

Speaker 3

Yeah, thanks, Brandon. You're absolutely right. If you're taking note, we've gone from this time last year, maybe 70 to 80 patients in our, what we coin as our global Expanded Access Program, which is in Europe primarily and a small number of markets. Each quarter, we continue to give you a number that's a little higher. We ended with 89 patients at the end of Q2, and that's from a small number of European markets that we have this Expanded Access Program. These patients, if we think about the durability and how long they've been on therapy, and then the ability for us at a market-by-market level after approval, reimbursement in Europe is going to be by country.

It's variable in regards to making that happen, but I do think our continued growth in the Expanded Access Program in Europe, along with the durability of patients that have been in the program for a long period of time, bodes well for us to be able to pull those patients through once we get reimbursement in those countries.

Speaker 5

Great, thank you very much, and congratulations on all the progress.

Speaker 3

Thanks.

Speaker 0

We'll go next to Kristin Kloska at Cantor.

Speaker 2

Hi, everyone. Congrats on another good quarter. First, I wanted to ask, you talked about the different, you talked about patient segments out there, one of them being patients that are diagnosed but not on any therapies yet. Can you walk us through what that means? Are these newly diagnosed and they're taking the steps? Are they interested in therapies, hoping to learn more? I have a follow-up.

Speaker 3

All right, Kristin, I'm going to try and quarterback this year, but that one I think goes directly to Josh to be able to talk a little bit about our diagnosed patient population and how we plan to get the rest of those patients exposed to MyQlifi, and then while we're continuing to unlock the newly diagnosed patients as well, which we're starting to see as well, Josh?

Speaker 1

Yeah, specifically your question around those patients who are diagnosed but not receiving treatment, those are typically patients who maybe were diagnosed a year ago prior to any therapies being on the market, and at the time, maybe their symptoms weren't severe enough for them to receive any treatment. We're finding that those patients are now coming back into the offices, either their local clinicians who are now being made aware of treatment options or going back to those COEs. We're finding those patients now that we've been on the market for nine months or so, and we're building that awareness and being able to bring those patients who had not previously received treatment to be eligible for MyQlifi.

Speaker 3

Yeah, and Kristin, one of the things I might just follow up on Josh, you know, and maybe from the prior question as well, when we think about these centers of excellence and we think about the local physicians who are working in concert with the Diagnosing Center of Excellence, as we've talked about in the prepared mark, we've mapped those physicians that are not in the actual centers of excellence, and our field organization's optimized to be able to go out and make sure that we can educate and offer MyQlifi to those physicians in the event that they had more than one, right? A lot of these physicians have only ever seen one patient and only have one patient and rely on that center of excellence.

Now that they've got experience with MyQlifi, we have the opportunity to be able to make sure that we're providing them with the Disease State Awareness Campaign and everything else to know that if they see it once, maybe they can see it twice.

Speaker 2

Okay, thanks. Can you provide us a sense of what % of patients are on paid MyQlifi? Our checks support that those patients that are on combination, that reimbursement has gone well, but curious what you're seeing from your end. Thanks so much again.

Speaker 3

I might just start that one off. Kristin, we give metrics that are around the prescription enrollment forms coming in the top that allow for us to show kind of the top of the funnel, if you will. We provide that covered lives, and we provide also the reimbursement, or sorry, the revenue number. When we talk about those patients that are on paid drug, you'll see that you go from $17.1 million in Q1 to $21.5 million in Q2. That's a 26% increase quarter over quarter in paid drug. Going from 122 enrollment forms to 129, it's obviously not 26%. We're hopeful that the guidance we're giving you around revenue, which is what comes out of the bottom, will be able to provide that takeaway on paid performance.

Speaker 1

Yeah, and I would just add to that part of the question, Kristin, that our patient services team has done a phenomenal job of being able to convert enrollments to paid patients and make sure that these patients are able to receive MyQlifi. Also, importantly, making sure that they continue to get their refills, and the retention rate of these patients is also really quite high. I think you had a second question, which was really around combination and what are we seeing in terms of patients receiving a combination of therapy.

Speaker 2

You mean specifically from reimbursement? Sorry.

Speaker 1

Reimbursement, yeah. I would remind you that by label, patients who are receiving MyQlifi are also receiving Nidostat. That by definition is a combination of those two therapies. We're not seeing any pushback or very limited pushback from a reimbursement standpoint. As we noted in the prepared remarks, we currently have 52% coverage, and for those who are not immediately covered, we're able to get reimbursement through medical exception pathways.

Speaker 2

Thanks again.

Speaker 3

Thanks, Kristin.

Speaker 0

Once again, ladies and gentlemen, it's star one if you had a question. We will go next to Jason Butler with Citizens.

Speaker 4

Thanks for taking the questions and congrats on the quarter. Two questions that I understand that you're still early in the launch and you may need to run time to get complete clarity on, but do you have a sense now that you're a couple quarters in of what the average time is taking from enrollment form to getting a patient on reimbursed drug? In terms of retention, do you have enough data yet to speak to whether the retention rate for patients that were not in the Expanded Access Program is consistent with the retention rate that you saw in the Expanded Access Program? Thanks.

Speaker 3

Yeah, Jason, I appreciate the call. I'll ask Josh to be able to talk a little bit about the time from enrollment form to getting those patients through the system and then into commercial drug. It's improving for sure. I'll have Josh talk about that. In regards to our retention rate of patients that are newly on product, we're only in the second full quarter of launch. It's really hard for us to be able to tell you about persistency rates and/or adherence rates to prescriptions at this point, only the second quarter. Some patients have only received one commercial fill, others have received seven. I think it's a little early for us to go there, and usually, we need about six quarters to be able to actually get a true persistency and adherence rate. It'll take a little bit more time for us to get to that.

One thing I can say is that of our Expanded Access Program patients who converted to commercial product, the majority of those patients have been consistent on product, and we've had limited, if any, folks come off product.

Speaker 1

I would just add to that point that we're really not seeing any differentiation from a payer perspective as to whether a patient was on the Expanded Access Program or not. We're looking at retention rates more holistically and haven't seen any differences really materially between a patient who was enrolled in the Expanded Access Program and those new patients who have come to MyQlifi since then who are not on the Expanded Access Program. Our retention remains quite high across all of those patients. In terms of the time from enrollment to paid, that varies widely based on the type of plan that a patient might be on, whether that's commercial or Medicaid, and then within commercial, there's a high degree of variability.

What I can tell you is that that timeframe across all payers is being reduced dramatically, so much that in some instances, we're seeing patients getting covered within 72 hours. Not all of them, of course, but we are seeing some patients with very rapid conversion from enrollment to paid, and we continue to see that improve quarter over quarter.

Speaker 4

Great, and then just one more for me on Opruva. Obviously, you're thinking carefully about the strategy here. Can you speak to what the magnitude of investment in commercializing the product that doesn't overlap directly with MyQlifi is? Just trying to get a sense of essentially how much you're investing in the product and how you're thinking about continuing that investment.

Speaker 3

Thanks, Jason. I think hopefully we made it really clear in our prepared remarks, and I'll reiterate it here. We have today got a commercial infrastructure that is actually running really well. When we talk about handling our enrollment forms as well as pulling those patients through our field reimbursement support, all the things that are working for us to be able to deliver this strong performance in Q2 for MyQlifi, it's synergistic with Opruva all the way. This question you're asking around if we took out, I think is what you're asking, if we took out MyQlifi out of this, what would it be costing you to be able to run Opruva?

The reality is that it would be fairly similar to what you see in our SG&A today because of the high level of overlap and our ability to be able to truly execute on what our business plan is. Having these centers of excellence or so across the country that we can provide value across the chain from commercial to medical to disease state awareness and really help this rare disease community. It's hard for me to break it out. It's very limited in regards to one or the other, but the synergies are huge for us. That provides us with a great opportunity to be able to do this really well, earn the right to go do it again someday.

Speaker 4

Great, thank you. Thanks for taking the questions.

Speaker 0

We'll go next to Sumant Kulkarni with Canaccord.

Speaker 5

Yeah, thanks for taking that question. You said these seven additional patient enrollment forms on MyQlifi takes you up to 129 in total. Is it in line with your expectations at the stage of the launch? How should we think about growth in patient enrollment forms going forward, and how do you think the availability of ACNURSA, which is also approved for Niemann-Pick type C, is impacting patient enrollment?

Speaker 3

Yeah, thanks, Jumon, and I appreciate the question, and we're looking forward to spending time with you at your conference tomorrow. You know, you kind of set up the question nicely. When we think about 300 to 350 patients diagnosed in the U.S., having 129 of those patients enrolled into our program is, quite frankly, just I'm incredibly proud of this organization in delivering that. When we think about moving forward in the variability of enrollment on a quarter-to-quarter basis or a month-to-month basis, unlocking this opportunity between the 300 and 350 to the 900 prevalent in the United States, we want to be more like Europe in this regard.

You know, a decade ago, when Nidostat was approved in Europe, there were a small number of patients who had actually been diagnosed, and with a prevalence of 1,100 in Europe, a majority of those patients are now being diagnosed and on some type of treatment. I'll ask Josh to talk a little bit about some of the areas and things that he's doing to be able to ensure that we can continue to drive the performance in the U.S., but we've got a great comp in Europe that shows us that as we continue to invest, we can be much more like this mature market having a product approved for a while in Europe.

Speaker 1

Yeah, we're really focusing on these two patient segments, those that are diagnosed and perhaps not yet receiving treatment, and that's really around building awareness and reemphasizing the clinical differentiation of MyQlifi and all the benefits that it brings in terms of, you know, it is the only drug that has demonstrated that it halts the progression of the disease at 12 months. We've been able to demonstrate at the first clinical time point, which was 12 weeks, that we have an impact on disease. We've recently published new data around the mechanism of action, which really draws a strong connection between MyQlifi and its ability to affect the underlying pathology of the disease. Importantly, the recent open label extension data shows that the durability of that effect lasts up to five years. We're really emphasizing that data and using every opportunity that we can to reinforce that.

For the undiagnosed patients, we have a number of different tactics to try and bring as many of those patients to diagnosis so that they can receive treatment as quickly as possible and ultimately halt the progression of disease. We have a disease awareness campaign, Read Between the Signs. Connected to that is a genetic testing capability to allow physicians to test those patients that they might have suspicion around whether or not they have NPC. We're using other really sort of sophisticated machine learning to be able to identify signs and symptoms of patients who have not yet been diagnosed based on a profile of an NPC patient, which will allow us to help continue to educate clinicians around how to identify these patients. We're really excited about those things.

As Neil mentioned, we see Europe as a model that can help us sort of determine or look forward to how we can see that diagnosis rate increase over time.

Speaker 3

Yeah, Sumant, maybe I'll just add one more thing. I'll add to that with another question.

Speaker 5

Oh, yeah, I did on the pipeline. You enrolled seven patients in the discovery trial for SoliPro for vascular Ehlers-Danlos syndrome. How happy are you with the pace of enrollment, and at what point will you be able to let us know when we might expect top-line data on it?

Speaker 3

Adrian's here with us, but I'll kind of kick it off and hand it off to him. When we think about the fact that it was at the end of Q3, early start of Q4, that we reestablished the enrollment of this trial and started to invest in the tactics to screen the outstanding patients that had been in the queue, but also some of the tactics we've been executing on that I'll ask Adrian to talk more about to actually get a higher quality CALL381, you know, genetically identified already patient that could then go into the trial and have the opportunity to be screened and then enrolled. I think we're starting to see some traction here, but Adrian, why don't you give an update here?

Speaker 5

Yeah, Sumant, we started a genetic testing initiative a couple of months ago. We've also connected with most of the centers where those patients are actually seen and treated, and I mean the CALL381 positive test patients. We've got a serious amount of physicians that are interested in referring the patients into the trial and significant patient interest. It takes a bit of time to see that come to fruition because it takes quite a bit of time to get those patients to the screening process. We're very hopeful to report positive outcomes of that in the next quarter.

Speaker 3

Thanks. Jumon, what I was going to say earlier on to your question, when we think about Niemann-Pick C in the U.S., it sometimes goes in the face of what we've been looking at, that we're launching this product in MyQlifi with the largest clinical data set of NPC patients that have ever been put together. In addition to that, we're not just launching a product with 12 months' worth of data. We're now launching a product that now has got five years' worth of clinical data, reinforcing the durability of treatment effect in the open label extension study that was published in this last quarter. We're now also being able to elucidate the mechanism of action work that we've talked about as well. I think this is just the beginning. We are in a very fortunate position.

Usually, you're not launching a product for the first time with five years of data. When, you know, Josh is talking about the pull-through, the work his team is doing on driving the awareness with payers with the strength of our data is what's delivering the strong performance or part of what's delivering the strong performance in what we're doing. The ability for us to be able to have the MAA file as a new MAA with this new, you know, very robust data sets, it puts us in a wonderful position to be able to bring MyQlifi to Europe and to the rest of the globe and take care of NPC patients outside of the United States.

We are very fortunate that we're able to be able to really jump on the back of a lot of folks that are out there and have been working in this disease area for a long time and now execute for patients with NPC.

Speaker 5

All right, thanks. I have a lot more questions for you tomorrow.

Speaker 3

Thank you. We'll forward it.

Speaker 0

We'll move next to Sammy Corwin with William Blair.

Speaker 2

Hey, team. Congrats on the progress this quarter, and thanks for taking our questions. I was curious how many unique MyQlifi prescribers there are now and how that's grown over the last quarter, and if you are approaching prescriber saturation. You expanded the MyQlifi covered lives significantly compared to last quarter. How much further do you think you can expand the extent of covered lives, and are you aware of any policies that are still being finalized? Thank you.

Speaker 3

Thanks, Sammy. I will actually pass both of those over to Josh, the unique prescribers, as well as if there's any prescriber fatigue, which definitely there is now.

Speaker 1

Yeah, so we have seen a pretty significant growth in the number of prescribers. When we first launched and we were dealing with the Expanded Access Program patients, those were patients who were under the care of just a handful of Expanded Access Program investigators, and you might have seen clinicians with anywhere from 12 to 8 patients. Now, as we move past that into this next cohort of patients, we're seeing clinicians who are treating three, four, maybe one or two patients. Naturally, the number of prescribers, the unique prescribers, has grown pretty significantly. We expect that to continue as this really is an ultra-rare disease where now we're getting into those clinicians who are seeing one or two patients over the course of a year, and that prescriber base will continue to grow. We expect that the number of lives and patients will grow as well.

I think you asked a question around reimbursement and covered lives. As noted, we're at 52% today, which is very consistent with what we'd expect for this stage of a launch. That's really more a reflection of plans who just haven't yet put MyQlifi in front of their PMT committee, and some plans wait anywhere from 9 to 12 months after a product has been launched before they make those decisions. We expect that number to increase over the course of our launch. A good analog is looking at Opruva, which has been out on the market for a little bit longer in a more mature market. As we noted, that's at 79%. You could see us absolutely growing from the 52% of covered lives to where we are today.

Speaker 2

That's very helpful. Thank you.

Speaker 0

We'll go next to Eddie Hickman with Guggenheim.

Speaker 4

Hi, good afternoon, everyone. Thank you for taking my questions. Congrats on all the progress. I wanted to follow up on a previous question just to sort of double-click on this penetration. If we think specifically about the sort of two-thirds of diagnosed patients that you know have NPC and that you have yet to penetrate, should we expect sort of a similar cadence of new patient enrollment forms throughout the next couple quarters, or do you reach a point where it starts to get sort of difficult to find those patients? This is sort of beyond, you know, finding new patients, but just within those sort of known diagnosed patients.

Also, just the logistical question, can patients start Nidostat and MyQlifi at the same time, or do payers ever ask for, or doctors ever ask for a delay in getting a patient sort of stably on Nidostat before trying to get coverage for MyQlifi? Appreciate it.

Speaker 3

Yeah, thanks, Eddie. I think those questions I'll hand over to Josh to handle.

Speaker 1

Yeah, patients can absolutely start Opruva and MyQlifi at the same time, and we've seen some new patients new to therapy starting both at the same time. That is absolutely possible. Your other question was really around the cadence of enrollments and what we expect in future quarters. I would just have to say it's really early in the launch to be able to give you any sense of that and be able to give you a sense of trends. I think as you look at where we are with our enrollments, that is a good reflection of the demand that we're seeing. More importantly, looking at the performance from a net sales perspective of a 26% increase really speaks to our ability to convert those patients who have come in and be able to convert them to paid and then retain those patients.

As we really begin to pull through the initiatives that we've been talking about with new patient identification, building awareness around clinical differentiation, we expect the enrollments to come in over the next couple of quarters, but again, probably too early to give you any sense of trends at this point.

Speaker 4

Got it. Thank you.

Speaker 0

Just a final reminder with star one if you had a question. It does not appear we have any other questions holding. This concludes the Q&A portion of today's call. I will now turn the program back to our presenters for any additional or closing remarks.

Speaker 3

Thank you, Operator, and thanks everybody for joining the call today. We'll look forward to keeping you appraised on future progress. Have a great week.

Speaker 0

Thank you. This does conclude today's program. Thank you for your participation. You may disconnect at any time.