ZVRA

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Transcript Summary

Zevra reported a strong first quarter of 2026, with total net revenue rising 78% year-over-year to $36.2 million. This growth was led by the successful U.S. launch of MIPLYFFA for Niemann-Pick disease type C (NPC), which contributed $24.6 million in sales, and $10.2 million from international Expanded Access Programs. Strategically, the company streamlined its operations by divesting the non-core SDX portfolio for $50 million, using the proceeds to retire all outstanding debt and strengthen its cash position to $236.8 million. Commercial highlights for MIPLYFFA include its inclusion in the Journal of Inherited Metabolic Disease clinical guidelines and reaching nearly half of the diagnosed U.S. patient population. Zevra is also advancing its Phase III DiSCOVER trial for Celiprolol in Vascular Ehlers-Danlos Syndrome and is in active discussions with the FDA to accelerate its development. With a clean balance sheet and a growing global footprint through EMA regulatory engagement, Zevra is positioned as a durable rare disease company with significant growth potential. Management remains focused on leveraging AI targeting to find undiagnosed patients and expanding MIPLYFFA's market reach both in the U.S. and Europe.

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Zevra Therapeutics (ZVRA): One-time asset liquidation masks core commercial failure and significant insider exits.

Full Earnings Call Transcript — Q1 • 2026-05-07

Operator: Good afternoon, and thank you for joining Zevra's First 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's Vice President of Investor Relations and Corporate Communications.
Nichol Ochsner: 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 first 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 in any forward-looking statements due to risks and uncertainties associated with Zevra's 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 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 filings with the SEC. I am pleased to welcome Zevra's management team members participating in today's call. Neil McFarlane, Zevra's President and Chief Executive Officer; Josh Schafer, our Chief Commercial Officer; and Justin Renz, our Chief Financial Officer. Our Chief Medical Officer, Adrian Quortel, will also be available for today's question-and-answer session. Now it's my pleasure to hand the call over to Neil.
Neil McFarlane: Thank you, Nichol, and welcome to everyone joining our quarterly call this afternoon. We are building a durable rare disease company grounded in disciplined execution, financial strength and a commitment to patients. We continue to advance our strategic plan, delivering strong performance and positioning ourselves for long-term growth. We made substantial progress in establishing MIPLYFFA as a foundational treatment for Niemann-Pick disease type C or NPC in the U.S. by delivering meaningful clinical impact to patients and pursuing multiple pathways to expand patient access globally. We are also advancing our late-stage asset, Celiprolol, through a Phase III study for the treatment of Vascular Ehlers-Danlos Syndrome, or VES, and executing several approaches to accelerate its development. To sharpen our focus on high-impact activities and remove operational distractions, we optimized the portfolio by divesting noncore assets with the sale of the SDX portfolio, to Commave Therapeutics for $50 million and concurrently resolved the legal dispute. Our balance sheet is strong with a cash position of $236.8 million and no outstanding debt, providing financial flexibility to drive growth. In the first quarter, total net revenue was $36.2 million, which is a 78% increase over Q1 2025. We've now reached a total of 170 prescription enrollment forms for MIPLYFFA from launch through March 31, nine of which were received in the first quarter. Recall, the estimated prevalence of NPC patients in the U.S. is approximately 900, of whom 300 to 350 are currently diagnosed. Thus, we have successfully reached roughly half of this patient population and continue to have traction in newly diagnosed patients. This is a significant early launch achievement and remains consistent with the meaningful opportunity ahead for continued growth. As a reminder, we've established a solid patent position for MIPLYFFA. It received orphan drug designation in the U.S., enabling marketing exclusivity through 2031. Consistent with our strategy to maximize the potential growth drivers for our business, we're pursuing a patent term extension through the U.S. Patent Office and await their decision, which could provide coverage beyond 2031. Through our global Expanded Access Program, or EAP, we are able to deliver a much-needed treatment to patients with NPC in certain European countries and select territories outside of Europe, including the U.K. As of the end of the quarter, we have a total of 122 patients enrolled in the EAP across geographies. Our global EAP is comprised of multiple access programs, including compassionate use and named patient reimbursement. Within each, we expect variability in enrollment and reimbursement over the first few years until the patient base has stabilized, consistent with our experience in the French EAP program. In Europe, there are an estimated 1,100 individuals with NPC. Diagnosis rates exceed those in the U.S., largely because the earlier approval of miglustat established physician awareness and enabled patient identification. To support the geographic expansion with a potential approval of Arimoclomol in Europe, we have a marketing authorization application under review by the European Medicines Agency, or EMA. We submitted our responses to the EMA's 120-day list of questions within the 90-day clock stop period and are progressing along the standard review process to make Arimoclomol available to the European NPC community. As a reminder, we have also received orphan medicinal product designation in Europe for the treatment of NPC. Turning to our late-stage pipeline. Our Phase III DiSCOVER Trial is evaluating Celiprolol for the treatment of beds, a rare inherited connective tissue disorder caused by COL3A1 gene mutations that weaken the walls of blood vessels and hollow organs and can cause arterial rupture or dissection among other complications. Approximately 90% of patients experience an event by the age of 40. There are roughly 7,500 individuals living with beds. Celiprolol is a selective adrenoceptor modulator that works by inducing vascular dilation and smooth muscle relaxation and thereby reducing mechanical stress on tissues of the arterial wall and hollow organs. Currently, there are no approved therapies for beds. Our commercialization strategy for Celiprolol is focused exclusively in the U.S., where there's a clear opportunity to fill an unmet need. While not approved in Europe for beds, Celiprolol is the primary off-label treatment in several European countries. This use is supported by the results of several studies, including long-term European cohorts. We've enrolled a total of 62 patients in the DiSCOVER Trial with 10 patients enrolled in the first quarter. This is an event-driven study, and we have two confirmed events out of the 28 events required to trigger the interim analysis. We continue to implement activities aimed at driving enrollment, including building a network of genetic testing centers to improve diagnosis as well as strengthening connections with key specialists who manage these patients. In parallel, following the FDA Type C meeting we had in the first quarter, we are preparing for a follow-up meeting in the second half of the year to explore pathways to accelerate its clinical development. In summary, we have a clear vision to become a leading rare disease therapeutics company, and we're motivated by the momentum and the opportunity that this phase of growth brings. I'll now turn the call over to Josh to review our commercial performance in more detail. Josh?
Joshua Schafer: Thank you, Neil, and good afternoon. Before reviewing the quarterly progress with MIPLYFFA, I'll provide a quick background on NPC. NPC is a rare lysosomal storage disorder caused by mutations in genes that impair intracellular cholesterol and lipid trafficking, leading to the abnormal lipid accumulation in the brain, liver, spleen and other organs. The onset and course of disease are heterogeneous, ranging from infancy to adulthood with progressive neurodegeneration that can vary in both speed of onset and clinical presentation. The extensive data generated for MIPLYFFA and NPC has shown long-term meaningful patient outcomes through the most expansive clinical development program in NPC to date. We have more than 5 years of data across more than 270 NPC patients worldwide through clinical studies, including our pivotal trial, open-label extension study, global EAP and pediatric substudy. All demonstrating MIPLYFFA's efficacy and safety. Notably, MIPLYFFA in combination with miglustat is the first and only disease-modifying therapy shown to halt disease progression at 12 months in a randomized controlled trial based on the validated NPC clinical severity scale. The onset of benefit is rapid with improvements noted at the first clinical evaluation time point of 12 weeks and has durable efficacy with treatment effects sustained for over 5 years. We are pleased to announce that MIPLYFFA was added to the NPC clinical practice guidelines, which were recently published in the Journal of Inherited Metabolic Disease, marking the first update to the guidelines since initial publication in 2018. These guidelines discuss the heterogeneity of the disease and reinforce that the NPC Clinical Severity Scale and genetic testing are the most reliable clinical endpoints and confirmation of diagnosis. The guidelines also point out, consistent with our messaging that early detection is critical to delay disease progression. MIPLYFFA's mechanistic and clinical differentiation is resonating with prescribers and patients and is driving adoption. As Neil shared, we have received a total of 170 prescription enrollment forms since launch with 9 enrollment forms coming in Q1. Our commercial strategy is focused on three key priorities: accelerating time to diagnosis and treatment, driving demand and facilitating access to MIPLYFFA. NPC remains significantly underdiagnosed and often diagnosed late due to heterogeneous symptoms. To enable earlier diagnosis, we have focused on education and engagement within the medical community through a strong presence at medical present data and through our ongoing disease awareness campaign called Learn NPC, Read Between the Signs. Additionally, we have built a custom AI-driven targeting model to find likely NPC patients and have collaborations with providers of genetic testing to accelerate their diagnosis. As a result, we continue to see new enrollments from previously diagnosed as well as newly diagnosed patients. We are also finding patients and seeing demand for MIPLYFFA increase outside of the centers of excellence. Our prescriber base is expanding to include community-based prescribers, which we believe reflects the success of our targeting and education efforts. Many of these new prescribers did not know that they had NPC patients in their practice and were previously unfamiliar with the disease and treatment options. We help facilitate medical education efforts through various initiatives such as the recently launched Expert Connect, which connects HCPs unfamiliar with NPC to experts who can address questions regarding disease state and available treatment options. Our patient mix has grown to include adults and children equally. These trends give us confidence in the estimated prevalence of 900 people in the U.S. living with NPC, and we remain focused on reaching as many patients as possible and expanding the total addressable market. From a market access standpoint, we have stable coverage of 69% and continue to achieve reimbursement through the medical exception pathway. Payer engagement continues to be focused on emphasizing our robust clinical safety and efficacy data and the extensive real-world evidence seen in clinical practice that supports MIPLYFFA's value. We believe clinical benefit support services and broad patient access and independent market research suggests MIPLYFFA is the preferred NPC therapy most trusted by clinicians and shown to improve balance, swallowing, cognition, speech and reduce falls. We received heartwarming letters from families noting how positively impacted they have been by MIPLYFFA and the support of our Amplify Assist patient services program. Together, this feedback reflects the impact of our commercial activities and sets the stage for continued growth. With that, I will turn the call over to Justin to review our financial results.
Justin Renz: Thank you, Josh. In addition to the financial details included in today's call, we encourage you to refer to our quarterly report on Form 10-Q for more detailed information, which we intend to file shortly. As Neil mentioned, in the first quarter of 2026, we generated net revenue of $36.2 million, which was an increase in total net revenue of $15.8 million compared to $20.4 million in Q1 of 2025. This is comprised of $24.6 million from MIPLYFFA net sales in the United States, $0.3 million from OLPRUVA, $10.2 million in net reimbursements from the global EAP for Arimoclomol and $1.1 million in royalty revenue. It is worth noting that we had 1 less shipment week of MIPLYFFA in the U.S. due to the first quarter delivery calendar. And as a result, channel inventory fell below the low end of our targeted range. Turning to recent business transactions. In March, we executed an agreement with Commave Therapeutics for the sale of the SDX portfolio for $50 million, monetizing assets that were not central to our core investment thesis. Under our contractual obligations, Aquestive Therapeutics received 10% or $5 million of gross proceeds. In the first quarter, we received $40.5 million of the $45 million in net proceeds, and we received a final payment of $4.5 million in April. In connection with this transaction, we reviewed our capital allocation strategy and retired our debt early, saving on average approximately $8 million a year in future interest expense. We are now debt-free and strategically positioned for growth, supported by a clean balance sheet. These onetime transactions impacted our first quarter financials. Accordingly, we recorded a onetime gain of approximately $43.3 million, partially offset by an approximately $10 million expense associated with the early extinguishment of debt, which is noted in the other income and expense section in our financial statements. Pivoting back to normal operations. During the first quarter of 2026, our operating expenses were $25.2 million, which was an increase of $2.4 million compared to the same quarter a year ago. R&D expense was $4.4 million for Q1 2026, which was an increase of $1.1 million compared to Q1 2025 due primarily to increases in third-party costs and professional fees. SG&A expense was $20.8 million for Q1 2026, which was an increase of $1.2 million compared to Q1 2025, primarily due to an increase in professional fees, partially offset by a decrease in third-party spending. We have utilized the vast majority of our usable net operating loss carryforwards. And as a result of the multiple onetime transactions that we recorded in the first quarter, we incurred an estimated tax provision of $6.9 million. Net income for the first quarter of 2026 was $37.9 million or $0.62 per basic and $0.60 per diluted share compared to a net loss of $3.1 million or $0.06 per basic and diluted share for the same quarter a year ago. Excluding the onetime transactions as well as the related tax provision for clear comparability across periods, the estimated quarterly net income reported would be $11.5 million or $0.18 per diluted share. As of March 31, 2026, Total cash, cash equivalents and investments were $236.8 million, which was a decrease of $2.1 million compared to December 31, 2025. As mentioned earlier, this decrease is attributable to deleveraging driven by our debt payoff, partially offset by the non-dilutive capital proceeds from the sale of the SDX portfolio and supported by our operating income. Collectively, these factors have further fortified our balance sheet, and we remain well positioned with the financial capacity to execute on our strategic priorities independent of the capital markets. And now I'll turn the call back to Neil for his closing remarks. Neil?
Neil McFarlane: Thanks, Justin. Our corporate profile has evolved significantly with the execution against the strategic pillars we introduced a little over a year ago. We are delivering strong commercial execution while thoughtfully monetizing assets that are not core to our business. We relocated our corporate headquarters to Boston, a hub of biotech innovation, and we strengthened our leadership by attracting seasoned professionals to our executive management team and Board of Directors, bringing valuable experience and perspective. These efforts, combined with prudent financial stewardship, are positioning us to deliver on our vision. As we advance through 2026, we are anchored by a clinically meaningful commercial product with multiple opportunities for global growth, a late-stage pipeline and a strong financial position. Our collective team at Zevra is energized by the numerous opportunities we have to expand our impact for people living with rare diseases. Operator, please open the line for questions.
Operator: [Operator Instructions] Our first question today comes from Kristen Kluska with Cantor Fitzgerald.
Unknown Analyst: This is [ Ian ] on Kristen's line. Congrats on the quarter update here. First, now that MIPLYFFA has been added to the NPC clinical practice guidelines, we're just wondering what this means for physician adoption. I mean are these doctors now formally advised to consider MIPLYFFA when treating these patients?
Neil McFarlane: We're really pleased. And I think as we've been previously communicating, these guidelines were in process and really came out quite fast after the introduction of new products that were approved in the U.S. It really reinforces the NPC severity scale as the tool that shows disease progression, the genetic testing as a key endpoint and diagnostic tool. The complexity of the disease. When we think about the heterogenicity of both infantile version and adult versions of the disease, it's really important to early detect in order to be able to delay progression. But really importantly, the combination therapy being considered for NPC was one of the major takeaways. I'll ask Josh to talk a little bit about some of the impact that we see and quite frankly, the hurdles that are lowered with these guidelines that have been published.
Joshua Schafer: Yes. Neil, just to add to what Neil said, we're also really pleased that the guidelines addressed the need for early detection and that early treatment helps delay progression. I also went on to talk about the importance of using combination therapy for patients who have been diagnosed with NPC. And this is really important because these are the opinions of a select group of key opinion leaders that we're now able to use and communicate and help build confidence and consistency in the way that some HCPs who might not be as familiar with NPC can now use this as a consistent guide for their treatment.
Unknown Analyst: And then second, do you have a sense of the proportion of the patients that are identified through the genetic testing that you're conducting and the AI-driven predictive model that ultimately convert onto the drug? I guess -- and just... go ahead.
Joshua Schafer: No, sorry, please finish your question.
Unknown Analyst: Yes. Just related to that, I was wondering what feedback you're hearing from the physicians that are managing these patients in terms of like how receptive they are being to the data-driven identification approaches that you're using?
Neil McFarlane: Wonderful question, and thanks for outlining our commercial strategy really clearly for us. Let me ask Josh to touch on some of the key priorities that we're executing against and some of the feedback that we're getting.
Joshua Schafer: Yes. As we mentioned in the prepared remarks, we have three key priorities from a commercial perspective. The first is to accelerate the time to diagnosis and treatment, and we're seeing that through our disease awareness campaign as well as some of the collaborations with the genetic testing companies. Our second priority is to really drive demand and you're seeing that in the enrollments that we've been able to get over the past quarter and more and then facilitate access with 69% of covered lives -- or 69% of covered lives currently able to access MIPLYFFA and the others we're able to do through medical exception pathways. So we feel really confident in our line of sight for more patients, given what we're seeing today, which is a nice mix of newly diagnosed as well as previously diagnosed patients.
Operator: And our next question comes from Kambiz Yazdi with BTIG.
Kambiz Yazdi: EAP revenue growth was quite robust. Is France driving that growth or other countries? And how should we think about geographic composition of the EAP as a leading indicator for where commercial demand may concentrate post EU approval?
Neil McFarlane: And welcome to the analyst coverage. As we reported, 122 patients enrolled in Q1 2026. This really encompasses multiple access programs. We have and multiple territories. In Europe, we've had outstanding for quite some time, our French EAP experience, which we've previously guided and consistently receive about $10 million net per year now that our patient base has stabilized in that territory, and that was per year. Today, we have new markets that are coming on. And that variability in ordering pattern and the rates of new enrollments and the reality is that we've got both compassionate use as well as named patient reimbursement. All of these new attributes lead to the variability that we continuously guide towards. It's early. We have new distributors. We're really pleased, though, with our new distributors and actually all of our distributors and how fast they're able to be able to get the named patient request for individual patients, then drive that back through to being able to get through our system and the product to patients in these territories. So we're really pleased with the continued inbound in these markets. And our goal is to really expand access to as many markets as we can while still focusing on our key territories in the U.S. and expanding the diagnosis and treatment, along with our global expansion, i.e., Europe and the MAA as well.
Kambiz Yazdi: And then maybe quickly as a follow-up, can you share with us any quantitative milestones or leading indicators you're tracking internally around your bespoke AI-driven patient identification and genetic testing efforts in identifying newly diagnosed NPC patients?
Neil McFarlane: Let me ask Josh to work on some of those strategies that are starting to really give us confidence in the TAM.
Joshua Schafer: Yes. And we monitor and measure a number of things, which will remain internal and metrics that we use just internally. But we are measuring how many patients are we finding that are newly diagnosed. We're looking at how many new prescribers we're able to bring into the mix as well. And then other interesting dynamics about the patient's journey, perhaps any other treatments that they may have been on, other diagnoses and other symptoms, all of that is tracked as we look to really understand as much as we can about these patients. This program is working extremely well, and it's allowing us to look at those patients who we know are diagnosed with NPC and find like patients who have not yet been diagnosed and be able to work with clinicians in accelerating their diagnosis and treatment.
Neil McFarlane: Yes. Let me add a little bit to that. I think it's important because we talk about the heterogeneity of the disease. The patient journey in NPC, unlike a lot -- so I should say, not unlike a lot of other rare diseases, it is you know one patient, you know one patient. We're talking about children and the primary data that has been developed over many, many years in NPC has been really child-based. In our experience and the expanded access program in the U.S., we built that out, and we saw that it was about 50% of the patients that were children and 50% of the patients that were adults. In our real-world experience moving forward, we still see that. Now that means that we have to continue to learn about the journey of the patient that is the adult patient and understand how we can then make the tools and continue to evolve our commercial strategy and educate physicians that patients in later ages also can present with NPC. So this is early in our launch. We're really pleased with the 170 total enrollments, the 50% children and adults, but we're still learning a lot about what it means for adult patients.
Operator: Our next question comes from Sumant Kulkarni with Canaccord.
Sumant Kulkarni: I have two, one on MIPLYFFA, one on Celiprolol. You mentioned that MIPLYFFA is now at almost 50% share of the diagnosed and treated patients in the U.S. What does your competitive intelligence tell you about your share of the competitor AQNEURSA in the remainder and your ability to gain further share in this 300 to 350 patients? And do you know of any cases where payers are allowing concurrent use of both MIPLYFFA and AQNEURSA in the same patient?
Neil McFarlane: We'll take one question at a time here in regards to the MIPLYFFA question. You are correct, and I'll ask Josh to double-click on this a little. You are correct that also based on the treatment guidelines that were just recently established that a lot of patients, and I can't give you the exact amount, but a lot of patients because of the complexity of the disease as well as the symptomatology of the disease are on multiple therapies, and we're actually seeing success in getting those patients on miglustat and MIPLYFFA and other therapies to be able to get them covered from a commercial perspective. These guidelines, I think, are going to continue to reinforce that combination therapy should be considered for NPC patients. We believe that, that bodes really well for us. Our label is in combination with miglustat. We see that's where our clinical differentiation is in terms of disease modification, halting the progression of the disease and the durable effects that we see. So we see this as a real positive. And I'll ask Josh maybe to talk a little bit about what he's seeing on the ground.
Joshua Schafer: Yes. Just to add a little bit more color there. We have been seeing in clinical practice that clinicians want to be able to treat patients with as many different modalities and to approach the treatment from as many different angles as possible. And so we've seen this combination therapy for some time. And now that opinion is really reinforced by the treatment guidelines. This -- what this means is that those 50% of diagnosed patients that we're treating now, some of them very likely may be on other therapies. And this is not an either/or marketplace. This is really an and marketplace. And so we're seeing that continuing to grow. We're also seeing that coverage is continuing to exist for patients. We have 69% of total covered lives today. Those who are not able to get that through direct formulary, we're able to get coverage even for combination therapy through medical exception pathways.
Sumant Kulkarni: Got it. On Celiprolol, we know you plan to meet with the FDA again in the second half of this year, but what are some of the specific options that you might have on the table in terms of your ability to move faster to bring this product to VEDS patients?
Neil McFarlane: Yes. We mentioned in our last call that we had a Type C meeting in Q1 and that we're exploring pathways to accelerate the clinical development of Celiprolol. I would categorize the conversations as constructive, also informative. We're now -- this is the early stages. We're now in the process of preparing for the follow-up meeting in the second half of 2026. And quite frankly, it's too early for us to tell you what strategies we're going at. But the reality is that we're going in two directions. One, to continue to boost enrollment activities and the other is to accelerate the clinical development through additional pathways with additional data.
Operator: Our next question comes from Eddie Hickman with Guggenheim Securities.
Eddie Hickman: Congratulations on all the progress. Apologies if this has already been asked, I'm jumping between a few calls. But really nice performance outside the U.S. as well. So I'm wondering if you can give an update on the type of questions that you got from the 100 day, day 120 day. And are they clinical CMC or more commercial? And then what's the sort of time line for that updated CHMP opinion?
Neil McFarlane: Yes. Eddie, and you have been the first to ask the question in regards to our marketing authorization application. As we mentioned in the prepared remarks, we did respond to the 120-day list of questions within the appropriate clock stop period. We provided the standard responses that are there. I won't get into the specifics of what it is the questions were or how we address them. But what I would continue to say is that since we've submitted a new application to the agency, we have not actually seen any new questions that we did not see out of the previous European submission and the FDA submission that we were able to then provide. So the substantial amount of data that we have now and the robustness of the package. And as a reminder, it's the totality of the data is over 270 treated patients. including data from our Phase II/III study, the open-label extension study that's got 5 years of data, our EAP that's got over 5 years of data, along with the pediatric substudy. So it's new data and the engagement is fairly standard. So now we're continuing to engage along the path, and we look forward to our next engagement with the European regulators.
Operator: Our next question comes from Jason Butler with Citizens.
Jason Butler: Two for me. Can you talk about the profile of the newly diagnosed patients that you're seeing? And again, speak to heterogeneity here. How early in the disease are they versus patients that may have had disease for a long time, but the diagnosis is delayed? And then second question, you mentioned that the inventory was below the lower end of the range for range at the end of the quarter. Has that reversed so far in 2Q?
Neil McFarlane: Yes. Jason, let me start with the first question, the profile of the newly diagnosed patients. And I know I say this a lot, but when you know one patient, you know one patient because of this heterogeneity of the disease, it's really important. One patient may present with a primary symptom that's an epilepsy. Another may present with gait instability. Another may present with cognitive challenges. And because of that, we're continuing to refine our -- as Josh talked about, the predictive modeling and claims data and all the things that we're doing, but we're learning along the way. So every time we get a new patient, it's further informing how we go out and try to continue to expand. And as I mentioned, this 300 patients to 350 patients, we have a really good line of sight to those diagnosed patients already. We've talked about the prevalence of the 900 in the United States. We now have a lot more confidence that, that 350 and the 900, we're somewhere in between those according to the TAM and what we see as the addressable patient population. So let me ask Josh to talk a little bit about some of the characteristics we see, but the confidence is getting -- is growing every time we see newly diagnosed patients.
Joshua Schafer: Yes. Just to bring this to life a little bit. We recently saw the diagnosis of a toddler who had an enlarged spleen and then went through genetic testing and was confirmed to have NPC. On the other end of the spectrum, there was an adult who had been misdiagnosed with MS for years and then changed physicians who happen to be aware of NPC and did genetic testing and confirmed that it was NPC. So it really runs the gamut, but there are some similarities and some characteristics both in terms of the symptoms as well as the journey that these patients go on that we're able to really learn from. And this is feeding our disease awareness campaign that we're continuing to educate physicians. We have a strong presence at medical conferences. We published a lot of data on this as well as the strong commercial efforts that we're putting in place.
Neil McFarlane: And Jason, let me hand off to Justin to keep him going here this evening on the inventory question.
Justin Renz: Yes. Jason, the way the Q1 delivery calendar fell, we just had one less shipment of MIPLYFFA in the U.S. than typical. And so as a result, our inventory in the channel was a little less than the low end of our targeted range. And so as a result, we do expect this to fall back within our targeted range by the end of the second quarter.
Operator: [Operator Instructions] Our next question will come from Brandon Folkes with H.C. Wainwright.
Brandon Folkes: Congrats on all the progress. Maybe just two for me. Any color on the time from submitting an enrollment form to getting on product and how that is trending at this stage of the launch? And then secondly, is there a difference in net revenue realized per patient if it goes through a medical exception versus a patient whose insurance falls into the 69% of covered lives currently?
Neil McFarlane: Brandon. let me ask Josh to talk a little bit about the enrollment numbers that we're seeing now.
Joshua Schafer: Yes. I think your question was really around the time from an enrollment comes in until once a patient receives therapy. And it's varied. It is largely dependent on the payer type, whether it's government or whether it's commercial. We are seeing -- in Q1, we had a number of patients who went through a reauthorization process, which is very typical at the beginning of the year as patients might either change plans or plans might change their policies. And so that had an impact on the first quarter, and we're working through those reauthorizations. So it's a little difficult to give you kind of what the average or the standard is just because of that reauthorizations that took place in the first quarter.
Neil McFarlane: Your second question, might have to -- this was about net revenue, and I'm not sure that I quite got that. Can you repeat that question for us?
Brandon Folkes: I guess what's the priority to grow the 69% of covered lives? Given your success under medical exceptions, is there a difference in net realized revenue per patient that goes through insurance versus the medical exception?
Neil McFarlane: Yes. No, that's a great question. I'll ask Josh to opine. But the 69% of covered lives allows for you to be on a formulary. It doesn't necessarily have a preferred position on the formulary. And it does speed up the process. A lot of our education that's gone to payers so far has really allowed us to be able to educate on the clinical benefits with the medical directors of this [ varying ] plans and achieve what we believe is a really good covered life plan. The important component of the question I think you just asked, which is maybe get into the potential for gross to net as well. We do not contract currently today. So it's standard government discounts along with distribution margins and the like. So the net price hasn't really changed except for the variability in gross to net on a quarter-over-quarter basis.
Joshua Schafer: Yes. And I think you're asking a very salient question around the 69% and our intent to grow that. We absolutely do, and we are making steady progress in that area, largely by talking about the clinical differentiation of MIPLYFFA. The guidelines certainly will help those discussions as well. But as you point out, it really doesn't impact the overall ability for a patient to receive MIPLYFFA because every plan has a medical exception pathway. What it does is it reduces a little bit of the time, it reduces some of the burden. We have a very robust patient services resources that we provide to help patients and offices navigate this. And so our goal is to make MIPLYFFA as accessible to patients as possible. And we do that both through increasing the covered lives, but also providing these patient services.
Operator: Thank you. This concludes the Q&A portion of today's call. I will now turn the call back to Neil for any additional or closing remarks.
Neil McFarlane: Thank you for joining our call today. We look forward to keeping you appraised of our future progress. Have a wonderful evening.
Operator: Thank you. This brings us to the end of today's meeting. We appreciate your time and participation. You may now disconnect.