8/5/2026

speaker
Neil McFarlane
President and Chief Executive Officer

Good afternoon and thank you for joining Zavra's second 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.

speaker
Justin Renz
Chief Financial Officer

The host for today's call is Nichol Ochsner, Zavra's Vice President of Investor Relations and Corporate Communications.

speaker
Nichol Ochsner
Vice President of 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 second quarter 2026 financial results. I encourage you to read our financial results news release, which was distributed this afternoon and is available in the investor 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 Debra's business. Follow-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 factor 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. This call will also reference Thank you for joining us. I am pleased to welcome Zebra's management team members participating in today's call. Neil McFarlane, Zebra's President and Chief Executive Officer, Josh Schafer, our Chief Commercial Officer, and Justin Renz, our Chief Financial Officer. Now, it's my pleasure to hand the call over to Neil.

speaker
Neil McFarlane
President and Chief Executive Officer

Thank you, Nichol, and welcome to everybody joining our quarterly call this afternoon. At VEVRA, our mission is to redefine what is possible in bringing life-changing therapies to people living with rare disease. Our strategy to unlock long-term value through focused execution has translated into measurable results. In the second quarter, our net revenue was $39.7 million, a 53% increase year over year. This was driven by $30.2 million in net U.S. revenue for MyPlypha. Our commercial progress reflects continued momentum in reaching patients and successfully establishing Miplypa in combination with Niglostat as a foundational treatment for Niemann-Pick disease type C, or NPC, in the U.S. As of June 30th, we had received a total of 184 prescription enrollment forms, 14 of which were received in the second quarter, bringing the total in the first six months of the year to 23. Additionally, we have strengthened our intellectual property position for MyPlypha with an orange book listing through 2041. We published long-term data program and the findings from our pediatric sub-study, further demonstrating the efficacy and safety of MyPlypha. We requested a re-examination of our Aromathlomol Marketing Authorization Application, or MAA, by the Committee for Medicinal Products for Human Use, or CHMP, in the European Union. And we continued to build a strong financial foundation with more than $260 million in cash, cash equivalents, and investments. Our strategic focus... This is reflected in the thoughtful portfolio decisions we are making, while maintaining an unwavering sense of urgency on behalf of patients. Most recently, we announced that following our oral explanation, the CHMP issued a negative opinion regarding the Arimaximal MAA. Subsequently, we are advancing the reexamination process. which will provide the opportunity to address the specific grounds cited in the CHMP opinion and have our comprehensive evidence package reviewed by a new rapporteur and co-rapporteur. This process typically takes 120 days from the submission of a reexamination to the opinion from the CHMP, which is expected in Q4. Importantly, as we advance through the European regulatory process, Our commitment to the patient and the NPC community remains steadfast. We will maintain access to Arimathamol for eligible patients through our Global Expanded Access Program, or EAP, which is comprised of compassionate use and reimbursed main patient programs that have enrolled 132 patients with 10 added in the second quarter. As previously guided, we expect reimbursements beyond the French EAP to be highly variable in the first few years until the patient base has stabilized. Ordering patterns for patients outside of Europe remain unpredictable, with some territories ordering a full year's supply and others ordering on a monthly or quarterly basis. Simultaneously, we are evaluating opportunities to expand access to MyPlypha through the global EAP in response to strong interest from additional countries. This reflects our commitment to helping eligible patients outside the U.S. access treatment while building meaningful partnerships across the rare disease community. Additionally, we strengthened our U.S. intellectual property position for MyPlyfa with an Orange Book listing that provides protection through 2041. We are still awaiting the determination for the separately requested patent term extension through the U.S. Patent Office, which, if granted, would fall within the Orange Book coverage period. Together, our IP portfolio provides a strong foundation for the long-term commercial opportunity for MyPlyfa. and we continue to invest in expanding and strengthening our protection beyond our current portfolio. At the same time, we are advancing Soliptolol, a selective adrenoceptor modulator that induces vascular dilation and smooth muscle relaxation for the treatment of vascular Ehlers-Danlos Syndrome, or VEDS. VEDS is a rare genetic connective tissue disorder caused by changes in the COL3A1 gene. Leading to fragile walls in blood vessels and hollow organs, which can cause arterial rupture or deception, among other complications. Silipilol has been shown to reduce the mechanical stress on these tissues. In our ongoing phase three DISCOVER trial, we've enrolled a total of 66 patients, including four who were enrolled in the second quarter. As an event-driven study, 28 events are required to trigger the interim analysis. and we have three confirmed events thus far. We are focused on accelerating this program by implementing strategies to drive enrollment, including expanding our network of genetic testing centers and exploring options to advance our clinical development by engaging with the FDA. Following a Type C meeting in Q1, we remain on track to reengage with the FDA in the second half of this year. In summary, our priorities remain clear. Delivering innovation for people living with rare disease, creating sustainable value for our stakeholders, and fulfilling our commitment to the patients, healthcare providers, and communities we serve. I'm now turning the call over to Josh to share more details regarding MyPlypus commercial performance. Josh?

speaker
Josh Schafer
Chief Commercial Officer

Thank you, Neil, and good afternoon. NPC is an ultra-rare genetic disease that causes significant neurological impairment and places a substantial burden on patients and families. Clinical manifestations can include dysfunction of ambulation, fine motor skills, swallowing, and speech. The disease and its symptoms vary significantly across patients, reflecting its complexity and severity. Earlier this year, the NTC clinical practice treatment guidelines were updated to further characterize the disease and reflect new advances in treatment since its last publication in 2018, including the addition of Miflifil. Importantly, the guidelines recommend considering combination therapy for all patients with confirmed NTC. As a reminder, MIPLISA is differentiated as the only FDA-approved treatment for NPC with established efficacy and safety indicated for use in combination with MIGLISA. The randomized controlled pivotal study demonstrated a combination halted NPC disease progression at 12 months as assessed by the rescored four-domain NPC clinical severity scale, a validated measurement of NPC progression. The onset of benefit was rapid, with clinical effects sustained over the long term. MyPlexus clinical data, combined with a growing awareness across the NPC community, continues to support its adoption. As Neil mentioned, we have received 184 prescription enrollment forms since launch through June 30th. The 14 we received in the second quarter included patients from Puerto Rico, reflecting our reach into U.S. territories. As we have noted previously, an enrollment form is a prescription submitted to our specialty pharmacy, which then begins the benefits investigation process to determine reimbursement eligibility. Notably, patients initiating treatment are being enrolled from centers of excellence and community practices with representation across both newly diagnosed and previously diagnosed patients, as well as adult and pediatric populations. This distribution supports our confidence in the estimated prevalence of 900 patients living with MTC in the U.S., of whom 300 to 350 are diagnosed. Our commercial strategy is centered on three key priorities, accelerating diagnosis and treatment, driving demand, and facilitating access to MIPLIFA. And the progress we are seeing reflects the growing impact of initiatives being implemented across each of these areas. Our disease awareness campaign, Learn NPC, Read Between the Signs, is driving the identification of patients based on symptoms and has led to new patient enrollments for MyPlaifa. Our genetic testing collaborations and our custom AI-driven targeting model are helping to support the identification and diagnosis of patients with NPC, allowing for earlier intervention. We also launched our Expert Connect program, which has enabled healthcare professionals who are less familiar with NPC to consult one-on-one with clinical experts to further strengthen knowledge and confidence. Additionally, we expanded and enhanced our digital engagement through the launch of NPC Knowledge Hub, providing resources to help patients and caregivers navigate the treatment journey. From a market access standpoint, care coverage remained at 69% of covered lives, consisting with our expectations. We continue to share presentations with payers on the recent update to the NTC clinical practice guidelines, as well as the growing body of newly published clinical data on myFlyflop. Additionally, we convened payer advisory boards with key decision makers to inform our strategy on how best to support prescribers and patients as they navigate the reimbursement landscape. We maintained strong relationships with the rare disease community and assembled steering committees to gather expert insights and help shape NPC disease awareness. In the second quarter, we participated in national and regional congresses, including the American Academy of Neurology, The Society of Inborn Metabolic Disorders, and Manual Pediatric Neurosymposium to enhance scientific visibility and educate clinicians and researchers. This strong engagement has continued into the third quarter with our participation at the National Neiman-Tick Disease Foundation Conference, where the importance and interest in a combination strategy for MTC treatment was highlighted prominently. Before turning the call over to Justin to review the financials, we want to reiterate our deep gratitude for the opportunity to make a meaningful impact in the lives of patients and their loved ones. We remain committed to being a trusted partner in the community by providing compassionate, patient-centered support. Justin?

speaker
Justin Renz
Chief Financial Officer

Thank you, Josh. Underpinning our achievements is this robust cash position, which provides the flexibility to invest in commercial execution, advance our pipeline, and pursue future growth opportunities while maintaining financial discipline. In addition to the financial details included in today's call, we encourage you to refer to our quarterly report on Form 10Q for more detailed information, which we intend to file later today. In the second quarter of 2026, we generated total net revenue of $39.7 million, which was an increase of $13.8 million, or 53%, compared to $25.9 million in Q2 2025. As a reminder, we recognize U.S. commercial revenue when my-ply-for-shipments are received by the specialty pharmacy. Of note, channel inventory has returned to target levels as compared to Q1 when it fell below the low end of our range. The second quarter of 2026 total net revenue is comprised of $30.2 million from my parts and net sales in the U.S., $0.2 million from Opruba, $9.0 million in debt reimbursements from the Global EAP for Aramaka Mall, and $0.3 million in past due royalty revenue from Comas. Revenue from MyQuaiza in the U.S. grew 23% compared to the first quarter of this year and 40% compared to the second quarter of last year. During the second quarter of 2026, our operating expenses were $21.0 million, which was a decrease of $3.2 million compared to the same quarter a year ago. R&D expense was $4.5 million for Q2 2026, which was an increase of $1.1 million compared to Q2 2025 due primarily to ongoing Aramaka Mall efforts. S, G, and A expense was $16.6 million for Q2 2026, which was a decrease of $4.2 million compared to Q2 2025. primarily due to a decrease in professional fees and third-party spending, partially offset by an increase in personnel-related costs. We allocated $4.0 million for our Q2 2026 income tax provision. Net income for the second quarter of 2026 was $8.8 million, or 14 cents per basic and diluted share, made a net income of $74.7 million, or $1.24 per basic and $1.21 per diluted share, for the same quarter of a year ago. This quarter, we incurred a mark-to-market fair value non-cash charge of $6.4 million related to our outstanding common stock warrants. Including this adjustment that impacts period comparison, That income for Q2 2026 would have been $15.2 million or $0.25 per share. In Q2 2025, we would have had an adjusted net loss of $2.4 million or $0.04 per share by excluding one-time transactions and other adjustments that contributed a net positive of $77.1 million to Q2 2025's results. As of June 30th, 2026, total cash, cash equivalents and investments were $260.2 million, which was an increase of approximately $23.4 million compared to March 31st, 2026. In summary, we remain well positioned with the financial capacity to execute on our strategic priorities independent of the capital markets. Now I'll turn the call back to Neil for his closing remarks. Neil?

speaker
Neil McFarlane
President and Chief Executive Officer

Thanks, Justin. Our strong second quarter performance demonstrates the strength of our strategy, disciplined execution, and continued momentum across the business. We remain steadfast in our commitment to putting patients first, a principle that is reflected in our approach to growing our U.S. commercial business, expanding our global presence, and fueling our portfolio to deliver long-term shareholder value. With that, operator, please open the line for questions.

speaker
Miplyfa

At this time, if you wish to ask a question, please press star 1 on your telephone keypad. You may remove yourself from the queue by pressing star 2. We will take our first question from Kristen Kluska with Cantor Fitzgerald. Your line is now open.

speaker
Kristen Kluska

Hi, everyone. Thanks so much for taking the questions here. I had a couple this afternoon. First, you're sitting on a very robust balance sheet and have a continued line of sight into more revenues as the MIT FIFO business grows. So, curious how you're leveraging this cash and ways you're looking to potentially explore using that. Second, I wanted to talk about the EMA. You mentioned there's a new repertoire here. Can you tell us a little bit about how that process works when there's really a new team here looking? Is there anything that you can do differently? And we have seen some recent reversals specifically in the rare disease space. We're curious about your thoughts there. Thank you so much.

speaker
Neil McFarlane
President and Chief Executive Officer

Thank you, Kristen. Maybe I'll start with your second question, and then I'll pivot over to Justin to help talk a little bit about the strong financial foundation we've got. So you are correct. In our re-examination process, we will have the opportunity to bring in a new rapporteur and co-rapporteur to further interpret the evidence that we have submitted so far. I can't actually comment on Thank you for joining us. and I think that as we continue to move forward with our reexamination process, we're looking forward to bringing new expert voices into the procedural discussions and allowing for the comprehensive evidence package to shine. On that note, I'll ask Justin to talk a little bit more about our Strong Financial Foundation and what we're doing moving forward.

speaker
Justin Renz
Chief Financial Officer

Thank you, Neil, and thank you, Kristen. As you noted, we ended Q2 with $260.2 million in cash for closing investments, and that really provides us meaningful flexibility. Our priorities are unchanged as we execute against our strategic plan. You know, mainly we're driving MyPlypho growth, pursuing global opportunities for MyPlypho, community advance to Wepperwall, and, you know, again, evaluate, of course, aligned external opportunities. So, we're going to maintain that discipline capital allocation strategy we've always talked of. We're going to be prudent in our investments and, you know, really focus on creating opportunities when we can find them.

speaker
Kristen Kluska

Thank you very much.

speaker
Miplyfa

and we will take our next question from Sumant Kulkarni with Canaccord Genuity. Your line is open.

speaker
Canaccord

Good afternoon. Thanks for taking that question. This one is about the PMA process. What exactly could you do to strengthen your, I guess, the request for the examination? Is there anything new you can file in terms of the real-world data you've mandated? I guess the best way I could ask that is what is going to be new in this request versus what was denied in the prior or documents that you sent in. Thank you.

speaker
Neil McFarlane
President and Chief Executive Officer

Thanks so much. So, I think it's a really important question. You know, the opinion that the CHMP provided reflects their assessment on the submitted evidence under this European regulatory framework that they have. Under the reexamination process, we'll have the opportunity to focus specifically to address these specific grounds that have been cited for the refusal. We believe strongly that the comprehensive evidence package, I mean, we've got Thank you for joining us. This robust and comprehensive package that we've resubmitted as part of this application, we're now going to go into the further interpretation of the evidence that are specific to the specific grounds that were cited for the refusal and bring it to light. And that comes with new expert voices into the procedural discussion that will allow us to continue to educate the CHMP on this, you know, Again, I've said it a few times, but robust and comprehensive evidence package.

speaker
Miplyfa

Thanks. And we'll take our next question from Eddie Hickman with Guggenheim Securities. Please go ahead.

speaker
Eddie Hickman

Hey, good afternoon. Appreciate you taking the questions, and congrats on all the progress so far. Just two from me. On the U.S. side, can you just give us maybe some directional color on which of the segments you're seeing and a number of others. The sort of variable non-French EAP numbers, sort of, you know, I know it's going to be sort of up and down maybe, but can you maybe sort of give us a min-max or some sort of bracketing for the next couple quarters on how we should model that? Appreciate it.

speaker
Neil McFarlane
President and Chief Executive Officer

Let me start with your second question in regards to the global EAP. And I think you're specifically asking about the reimburse section of that. We've said previously that our French program has been very consistent. It's approximately two and a half. Thank you for joining us. The variability in ordering patterns that come through other named patient reimbursement programs can come in one patient at a time. And it can be one patient that orders for an annual basis, one patient that orders for quarterly or a monthly supply. So it's really hard for me to tell you or guide you in regards to the brackets that you're asking for because it remains unpredictable. It's driven by the timing, the order volume, the program type. and also the geography. So the only thing that I can tell you is that we remain very committed in the French EAP program, which has been consistent at about $10 million net per year or approximately $2.5 million per quarter. Let me switch quickly to the U.S. business. Really great questions. You know, the U.S. business – We're learning more about every day. We are seeing an equal mix between children and adults. As you know, this has been primarily a child disease for many years and kind of characterizes that. Now we're seeing quite a bit of adult patients. So those learnings are coming through. You know, we don't have a single tactic that is working to uncover the number of patients we have. And remember, we're at 184 enrollment forms now with 14 in the quarter. This is really an integrated strategy, and I'll ask Josh to talk more about some of the things he's seen on the ground, but an integrated strategy versus a single tactic that is continuing to drive this and kind of unlocking this dam that we think is between the 350 and 900. Josh?

speaker
Josh Schafer
Chief Commercial Officer

Thanks. Eddie, thanks for the question. As Neil mentioned, I wouldn't attribute the enrollment to any one tactic. In fact, we have a really well integrated commercial strategy that's focused on and the rest of the team. And we're seeing patients who are young as well as adult patients. So, you know, we're really pleased with the diversity and the distribution of the enrollments that we're seeing, most notably because it really gives us confidence in the overall addressable market that we've been talking about, which is, we believe, somewhere between 350 and 900 patients.

speaker
Justin Renz
Chief Financial Officer

Appreciate it.

speaker
Miplyfa

Thank you. And we will take our next question from Jason Butler with Citizens. Please go ahead.

speaker
Neil McFarlane
President and Chief Executive Officer

Hi. Thanks for taking the question. just wondering if you could give us some kind of perspective on the age distribution of newly diagnosed patients and then secondly when you look at the tools that you're using like the AI targeting and the expert connect program are you seeing any growth in the number of prescribers over the last couple of quarters? Thank you.

speaker
Josh Schafer
Chief Commercial Officer

Yeah, sure. In terms of the age distribution of Newly diagnosed patients. You know, we are seeing patients who are coming in who are both under the age of 18 as well as some who are over the age of 18. And just within the past, this first half, we had some very specific instances. There was a newly diagnosed child who was about two years old, something that had just been presented and the parents had her tested and confirmed with MPC. On the other hand, we have a patient who is in his mid-20s and has been misdiagnosed with epilepsy for five or six years and was finally tested and confirmed to have MPC. So we're really seeing a diversity of instances and age groups reinforcing the heterogeneity of the disease. and this is largely driven by the tactics that we put in place. The AI targeting, our disease awareness campaign, all of these things are really helping to drive enrollments and again, giving us confidence that there are more patients out there yet to be diagnosed.

speaker
Neil McFarlane
President and Chief Executive Officer

Yeah, Jason, just to put a little bit of a finer point on this, you know, we've said that the age distribution between adults and kids is about 50-50 in what we see today. So of these 184 enrollment forms, you just think of that as half adults and half children. When it comes to the newly diagnosed patients, It's really difficult for us to be able to say, you know, and provide that level of guidance. So for us, you should consider to say this is not just a childhood disease as we've been thinking about this for many, many decades. These adult patients that are coming in today have got delayed diagnosis. They've got complex disease histories. And we're starting to unlock that. So the 50-50 ratio remains of our total population.

speaker
Jason

Great. Thank you.

speaker
Miplyfa

And once again, as a reminder, it is star one on your telephone keypad to ask a question. We will move next to Brandon Fultz with HC Wainwright. Your line is open.

speaker
Jason

Hi, thanks for taking my question. Congrats on another good quarter. Neil, maybe just how do you feel about enrollment forms today, halfway through 2026 when we think about the full year compared to The enrollment forms you generated in 2025, it does look like things are trending up on an enrollment basis, but do you have better visibility into enrollment trends today? And are you starting to get more comfortable with sort of a quarterly level of enrollment forms you expect going forward?

speaker
Neil McFarlane
President and Chief Executive Officer

Thanks, Brandon. So, yes, you asked a question in regards to 2025, we have 52 enrollments for the year. And halfway through this year, we're at about 23 enrollments. I think what gives me a lot of confidence is really around the opportunity to unlock newly diagnosed patients. We started seeing that around this time last year. And then in Q3, we saw more newly diagnosed patients, Q4, newly diagnosed patients. And these efforts that Josh just mentioned previously around our bespoke AI model that's getting the EMR data along with the claims data together and helping our reps get to places where they can educate physicians that we believe have NPC patients. It's really driving the newly diagnosed as well as the previously diagnosed patients. So I'm pleased with where we are through the year, but I'm even more confident about the TAM being somewhere between that 350 and 900 because we're seeing these newly diagnosed patients earlier in the launch process than we would have expected today. And I'm hopeful that... with continued investments on our side and others in the area and MPC will continue to drive that forward.

speaker
Jason

Thanks very much. And then maybe just one more for me. You know, I heard your earlier commentary on slipper law, but any additional color you can provide coming out of that meeting, that 1Q meeting and sort of the continued dialogue with the FDA? Did you come out of the meeting more confident in going ahead, or what gave you confidence in terms of sort of moving that program forward, coming out of that meeting? Any color would be helpful. Thank you.

speaker
Neil McFarlane
President and Chief Executive Officer

I would coin the meeting as informative. We went in to try and find ways to accelerate the clinical development program, and the questions we asked were responded to with opportunities for us to go out and do some homework. and we kind of have several approaches that we're working on here. It's a parallel track, right? One is that we want to try and find ways to accelerate the clinical development through regulatory pathways and the other is really around us and the ability to drive enrollment so we can get to the events and get to this interim analysis. As you saw last quarter, we had two confirmed events. We have three confirmed events now in Q2. We're up to 66 patients enrolled of 150 patients in the study, but we'd like to be able to find ways to put our foot on the gas and that's what our second half plans are going to be discussing with the FDA.

speaker
Jason

Great. Thanks very much.

speaker
Miplyfa

and we will take our next question from Lachlan Hanbury-Brown with William Blair. Your line is open.

speaker
William Blair

Hey guys, thanks for the question. Maybe the first, Neil, going back to some of the prior questions, but just wondering what gives you confidence that you can be successful in the re-examination? I understand your point about confidence in the overall package, but confidence in maybe being able to get the rapporteurs over the line or beyond the hurdles that they've They've seen in this initial review, because I typically associate EME with being overly flexible. And then maybe a second question, just wondering if the IP win for MyPlyfor in the US changes how you think about your ability or maybe appetite to invest in the MyPlyfor franchise in the US?

speaker
Neil McFarlane
President and Chief Executive Officer

So let me start with your first question. I'm going to be thoughtful in the level of detail I provide, but our confidence is fully grounded in the totality of the evidence and the unmet need. We mentioned, again, 132 patients in our global EAP. The majority of those patients are patients that are in the European Union, and I think that that's an important perspective. So, we continue to see the demand from patients. When it comes to the CHMP and their assessment, This process that we're working through and the stage that we're in around the reexamination process is really around now focusing on that further interpretation of the evidence that we've provided around the open label extension, the EAP, and the pediatric sub-studies, but it's also It starts with our pivotal study, right? It's primarily centered on the analysis of the pivotal study as the foundation of this application. So, we've got a positive, clinically meaningful, and statistically significant clinical study. That's what gives us the first part of confidence. The rest of it is around the comprehensive evidence package. So I can't give you a probability, but what I can tell you is that we're bolstering our clinical trial with long-term clinical experience supported by multi-years of safety database with no new safety concerns. And we believe strongly that the benefit-risk is in favor of MyPlyfor for patients in the EU. The second question that you had was in regards to, I'm going to take that as lifecycle management and IP. Yes, we're... Very happy with the longer duration of intellectual property protection today. And that does give us a lens towards looking at how we can further invest the capital Justin was talking about earlier today into the U.S. business, but also into the European and global expansion along with the CILIPA law program. I kind of leave it at that at this point in the game. I don't want to say that we have a specific action that we'll take with the current extension, but we are looking forward to maximize the value of my platform over the long haul.

speaker
Josh Schafer
Chief Commercial Officer

Thanks.

speaker
Miplyfa

And we will take our next question from Kambiz Yazdi with BTIG. Your line is open.

speaker
Justin Renz
Chief Financial Officer

I think congratulations on the quarter. It's just a goal for me. On the re-examination, are you thinking about the role of the SAG meeting and KOL testimonies potentially in the process? And also maybe on your strong chaos revision, can you speak about your philosophy on shared buybacks?

speaker
Neil McFarlane
President and Chief Executive Officer

I'll take the first one and I'll ask Justin to comment on our capital allocation plans. The answer to that question is we did resubmit for reexamination and we have requested a SAG meeting. That's part of bringing new expert voices into the procedural discussions and we will take advantage of that. Justin?

speaker
Justin Renz
Chief Financial Officer

Yeah, thank you. And again, reiterating what we said earlier, we're in growth mode. Our priorities remain unchanged. Thank you very much.

speaker
Miplyfa

And we will take a follow-up question from Sumant Kulkarni with Canaccord Genuity. Please go ahead.

speaker
Canaccord

Thanks for the follow-up. So is there anything you can share about what your latest comparative intelligence tells you about the nature of the diagnosed and treated patients in the U.S. that are not yet on myPlaifa, especially in terms of their dependence on the other approved products like Syntabios and Ursa?

speaker
Neil McFarlane
President and Chief Executive Officer

Yeah, I recall this question. You know, it's important. We believe there are complementary mechanisms of action, and I think the treatment guidelines reinforce what we've been saying for many years now, that when you have a heterogeneous disease that has multi-symptomatology and just devastating progression, It's important to have as many complementary mechanisms as possible that you can utilize. I'll ask Josh to talk a little bit more about what we see in the marketplace, but what we have been seeing is the guidelines along with our current real-world evidence that patients and physicians want options, and we're seeing combination therapy being not just a recognized approach, but more common than not.

speaker
Josh Schafer
Chief Commercial Officer

Yeah, as Neil mentioned and as we've talked about previously, Suma, the market is really moving towards a combination therapy because the treatments that are available are very complementary to one another. And this is reinforced in the treatment guidelines that came out earlier this year. Also important in those guidelines, it was recognized that Miplyfa was a disease-modifying treatment and is the only treatment that the FDA indicated in combination use with Miglasat. But as the market moves towards combination therapy, I think it really supports Miplyfa's ongoing use as foundational therapy. Your question about other products out there, we don't really see this as an either-or market. Many patients, certainly we know that many myelitis patients are receiving migla stats and perhaps other treatments. And so we look at this really as an opportunity to continue to grow patients. Our share of those diagnosed patients, and more importantly, the undiagnosed patients. The activities that we're doing now, we're seeing new enrollments coming in on a regular basis for newly diagnosed patients, all of which gives us real confidence that the overall market size is what we think it is of 350 to 900 patients. Thank you.

speaker
Miplyfa

and we will move next to Jonathan Ashok with Roth Capital Partners. Your line is open.

speaker
Jonathan Ashok

Jonathan Ashok Thank you. Solid quarter, guys. Just two things. Can you tell us anything about patient attrition, any commercial patients who are no longer taking the drug and maybe a percentage there? And how did the end of 2Q inventory compare to the end of 1Q?

speaker
Neil McFarlane
President and Chief Executive Officer

Jonathan, I'll ask Josh to talk a little bit about the refill rate persistency and what we see in the market today, and then a little bit about the Indian inventory. Yeah.

speaker
Josh Schafer
Chief Commercial Officer

With regards to attrition, first of all, as we noted, we've got about 69% of covered lives, so we are very confident in the access that we're able to provide for patients, either through direct formulary for those 69% Thank you for joining us. The patients that we do lose, unfortunately, are, you know, it's really a reflection more of the severity of the disease. It is an oftentimes fatal disease, and patients will progress to the point of dying. And the patients who we have lost, they're no longer taking my play for the vast majority of those are patients who have unfortunately passed away.

speaker
Neil McFarlane
President and Chief Executive Officer

Yeah, Jonathan, just to put a final edge on that, you know, we had discussed that patients that get on MyPlypha seem to stay on MyPlypha. Our EHP data was very... are supportive of that effort. The rates that we saw in terms of the patients who, as Josh mentioned, unfortunately passed away, those rates were really super small. We're continuing to see those similar kind of rates in our long-term data so far. Now, we're almost two years in. At the end of this year, we should be able to start seeing some more persistency, and maybe we'll be able to provide that metric later on. Justin, you want to talk a little bit about inventory?

speaker
Justin Renz
Chief Financial Officer

Okay. So, in general, you know, we have a channel inventory target that we work with our specialty pharmacy partner on. And our channel inventory fell below our targeted range in the Q1. It was truly because of the shift in timing and the dynamics around that. And for Q2, it was essentially back to the normal target level that we aspire to. So, I believe you'll see a normalization going forward of this level.

speaker
Jonathan Ashok

Okay. So, just a wee bit higher. Back to normal is what you're saying. Correct. Thank you very much, guys.

speaker
Miplyfa

And this does conclude the Q&A portion of today's call. I'd like to turn the call back over to Neil McFarlane for any closing remarks.

speaker
Neil McFarlane
President and Chief Executive Officer

Thank you for joining the call today and we look forward to keeping you apprised of our future progress. Have a great evening.

speaker
Miplyfa

Thank you. This brings us to the end of today's meeting. We appreciate your time and participation. You may now disconnect.

Disclaimer

This conference call transcript was computer generated and almost certianly contains errors. This transcript is provided for information purposes only.EarningsCall, LLC makes no representation about the accuracy of the aforementioned transcript, and you are cautioned not to place undue reliance on the information provided by the transcript.

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