Q1 2025 Zevra Therapeutics Inc Earnings Call

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Good afternoon and thank you for joining Zevra's first 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.

Speaker Change: The host for today's call is Nicole Ochsner, severed vice presidents.

Please go ahead.

Speaker Change: 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 first quarter 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 the R website.

Speaker Change: Before we begin the call, please note that certain information shared today will include forward-looking statements.

Speaker Change: Actual results may differ materially from those stated or implied by any forward-looking statements due to risks and uncertainties associated with Severus business.

Speaker Change: Power-looking statements are not promises or guarantees, and are inherently subject to risks, uncertainties and other important factors that may lead actual results differing materially from projections made.

Speaker Change: These four looking statements should be evaluated together with the cautionary statements contained in the risk factors section and our most recently quarterly report on Form 10Q.

Speaker Change: Annual Report on Form 10K and our other filings with the SEC.

Speaker Change: I am pleased to welcome Zevra's management team members participating in today's call.

Neal McFarlane: Neil McFarlane, Severus President, and Chief Executive Officer, Ludwain Clifton, or Chief Financial Officer, and Josh Schafer, or Chief Commercial Officer.

Neil McFarlane: Our Chief Medical Officer, Adrian Quartel, will also be available for today's question and answer session. Now, it's my pleasure to turn the call over to Neil.

Neil McFarlane: Thank you, Nicole, and thank you for joining us this afternoon to review our strong progress and the ongoing execution of our four strategic pillars.

Let me start with commercial excellence.

Neil McFarlane: We are delivering on our two commercial launches to bring innovative therapies to people living with rare diseases.

Neil McFarlane: We have exceeded our internal expectations for the my-pipe launch, and I've enrolled over one-third of those diagnoses with Neiman-Pick Disease Type-C or NPC in the United States.

Neil McFarlane: With all proof of this treatment of certain urea cycle disorders for UCDs, we will find our marketing efforts to further increase awareness and access for patients and providers.

Moving on to Pipeline and Innovation

Neil McFarlane: We are focused on where our capabilities have the greatest impact for patients as we progress our development programs.

Neil McFarlane: Our Phase III Discover Trial of Salippo Law, our late stage asset for the potential treatment of Vascular Eilers Danlos Syndrome for VEDS, which has received both orphan drug and breakthrough therapy designations, continues to progress with patient moments.

Neil McFarlane: Additionally, we continue assessing value-enhancing strategic alternatives for our FACE-3 Ready Asset, KP 1077, Ferrer Sleep Disorders

Neil McFarlane: These clinical stage assets provide optionality and diversification to our portfolio.

turn it to talent and culture.

Neil McFarlane: We have a passionate, highly motivated, experienced and mission-driven team focused on our vision to bring a life-changing therapies to people living with rare diseases.

Neil McFarlane: Our team's strong execution is being acknowledged. For example, we earned a spot on fast companies top 10 most innovative companies list in the medicines, therapeutics, and pharmaceuticals categories.

Finally, Corporate Foundation.

Neil McFarlane: This underpins all other pillars and allows us to responsibly invest in our long-term transformation through disciplined capital allocation while maintaining our position of financial strength.

Neil McFarlane: With the recent monetization of our pediatric rare disease priority review voucher, or PRV, we added $148.3 million of non-dilutive capital to our balance sheet.

Bolster, our financial strength.

Neil McFarlane: With this, we have ample resources and financial flexibility to execute on our strategic priorities independent from the capital markets.

Neil McFarlane: Driving a little deeper into our commercial programs, we are pleased with the early success in the My Plethora launch for the treatment of MPC, which is an ultra rare neurodegenerative progressive and fatal disease.

The launch is characterized by three unique patient cohorts [inaudible]

Neil McFarlane: Those who are participants of our expanded access program or DAP.

Neil McFarlane: Those who are diagnosed and may or may not be receiving treatment, and finally, those who are undiagnosed and livid with MPC for whom we seek to accelerate diagnosis and treatment.

Neil McFarlane: We've received a total of 122 prescription enrollment forms from a launch through March 31st with 13 in the first quarter of 2025.

Neil McFarlane: Even in these early days of the launch, we have received prescription enrollment forms from all three patient cohorts, affirming the early impact of our commercial strategy.

Notably,

Neil McFarlane: As we have previously reported, all active USEP participants have been enrolled to receive my plipa, and we have now closed the program, including locking the database and deactivating study centers ahead of our previously stated guidance.

Neil McFarlane: We are focused on providing my plifea to people living with NPCs and have been educating prescribers and payers on the clinical benefit which demonstrates in combination with megalostat, a halting of disease progression through 12 months of treatment.

Neil McFarlane: Beyond the Pivotal trial, my pipe of safety profile and the long-term treatment effect have been evaluated in open label extension studies and through our multi-center EAP.

Neil McFarlane: with patients having as many as five to seven years of experience on treatment, which significantly differentiates my plipa and positions it as the cornerstone therapy.

Neil McFarlane: Joseph will provide additional details related to our launch metrics later in today's call.

Neil McFarlane: Beyond the successful U.S. launch, our goal is to provide access to as many people living with NPCs as possible by expanding the availability of my plipa outside of the U.S.

Neil McFarlane: We are diligently working towards expanding our regulatory submissions with an immediate focus on a marketing authorization application or MAA in Europe in the second half of this year.

Neil McFarlane: Where we estimate approximately 1,100 people are living with NPC. As a reminder, the European market is already well established with access to a marketed treatment for more than a decade.

Neil McFarlane: Further, we're expanding our global EAP, which has recently increased to approximately 85 NPC patients, compared to the previously reported 70 to 80 patients.

Neil McFarlane: Our continued investment in my life as global EAP, in combination with a more established market, could accelerate commercialization upon approval in Europe .

Turning to our prover.

Neil McFarlane: from initial product availability in July of 2023, and including Zevra's promotion of All Prove Us in late January of 2024.

Neil McFarlane: Through the end of the first quarter, 2025, we've received a total of 28 prescription enrollment forms with five prescription enrollment forms during the first quarter of this year.

Neil McFarlane: In November , we outlined a plan to refine our commercial strategy towards the adult and OTC-deficient populations, for whom all previous portability and ease of administration may provide the greatest benefit.

Neil McFarlane: The enrolments received in the first quarter were all within this patient segment, suggesting early science of this strategy is gaining traction.

Neil McFarlane: Before turning the call over to Josh, I'll share a few more updates on our clinical stage assets.

Neil McFarlane: Through our Phase III Discover Trial, Salippo Law is being evaluated as a potential treatment of vets, a devastating connective tissue disorder caused by the Call 3A1 gene mutation, and characterized by spontaneous arterial aneurysms, hollow organ ruptures, and aortic dissections.

Neil McFarlane: In the U.S., approximately 7,500 people are living with vets, where the standard of care relies on reactive and invasive surgical interventions with no approved treatments.

Neil McFarlane: Celepelol is also currently used off-label as a standard of care for vets in certain European countries.

Neil McFarlane: Here in the first quarter, we enrolled five additional subjects in the Discover Trial, bringing the total to 32.

Neil McFarlane: This is an event-driven trial, and the rate of enrollment is important, and we're implementing various strategies, including increasing our efforts to focus on providers and clinics specializing in genetically confirmed call 3a1 positive patients.

Neil McFarlane: In the first quarter, we began receiving leads from a recently initiated genetic testing program, providing our team with a new group of confirmed VADS patients for outreach.

Neil McFarlane: Importantly, we're optimistic in this strategy to drive enrollment and expect to see an increase in the enrollment rate in the upcoming quarters.

Neil McFarlane: There are also several updates related to the review of our extensive IP portfolio as we seek to extract value by determining where we continue to invest as well as where we choose to stop investing based on our strategic plan.

Neil McFarlane: I'm pleased to report that earlier this month we've outlicensed intellectual property related to a pre-clinical pro-drug when undisclosed party for a nominal upfront payment, potential future milestones, and mid-single-digit royalties on net sales.

Neil McFarlane: We will continue to optimize and curate our IP portfolio through a variety of avenues to realize value for the company and our shareholders.

Neil McFarlane: Additionally, regarding Appadass, which was previously approved for the treatment of pain and has not been commercially available since 2023, we have made a strategic decision to formally withdraw the NDA.

Neil McFarlane: By limiting the regulatory activities associated with maintaining approval, we are further reducing costs.

Neil McFarlane: We are continuing Zevra's transformation into a patient centric commercial stage rare disease

Neil McFarlane: With our bolstered cash balance, we are well positioned to support our stated strategic priorities and execute on our long-term vision for the company.

Neil McFarlane: by establishing my PIPA as a cornerstone treatment for NPP globally, maximizing our commercial opportunity with OPPRUVA, and supporting our growth through our development pipeline, including the ongoing Phase 3 trial for Singapore.

Neil McFarlane: Continue to build on our track record of execution, positioning us to make future investments that are in alignment with our strategic plan. Let me turn the call over to Josh who will give an update on our commercial products.

Thank you, Neil, and good afternoon.

Josh: K, while discussing our commercial products, we will provide the following metrics.

Josh: Description Enrollment Forms for the Quarter, The Percent of Covered Lives, and Net Revenue.

Josh: Beginning with my plifea, as Neil stated, the launch is progressing well and exceeding our expectations.

Josh: With our EAP cohort fully enrolled, we're focused on reaching the diagnosed and undiagnosed cohorts.

Josh: We've seen steady growth in the first quarter, with 13 prescription enrollment forms, all representing individuals new to my apply for, which indicates a broadening of both the patient and prescriber base.

Josh: This is a result of our ongoing efforts to raise awareness of NPCs and to educate prescribers about my playlists, clinically differentiated profile.

Josh: We estimate that there are approximately 900 people living with NTC in the US, of which only 300 to 350 have been diagnosed.

Josh: Since launch, through March 31st, we have received a total of 122 prescription enrollment forms.

Josh: Meaning that roughly one-third of the estimated individuals diagnosed with NPC in the U.S. have been enrolled to receive my pli-fa.

Josh: As a reminder, a prescription enrollment form is a prescription submitted to our specialty pharmacy initiating the benefits investigation process to determine reimbursement and can lead to a 30-day paid dispense of my plipa.

Josh: Regarding market access, many payers have not yet formalized their formulary coverage or reimbursement policies and our team has been actively engaging with payers to secure reimbursement.

Josh: Through the end of the first quarter, we have achieved 38% of covered lives, which is in line with our expectations at this stage of launch.

Josh: We have been able to secure reimbursement authorization for many of our patients through direct formulary coverage or via the medical exception process.

Josh: While initial denials are commonplace among rare disease products, we are very pleased with our team's ability to swiftly address pair challenges by presenting my plife as robust and differentiated clinical data.

Josh: NPC is a lysosomal storage disorder that is caused by progressive lipid buildup leading to cell death and ultimately organ dysfunction in the spleen, liver, and brain.

Josh: The difficulty in diagnosing NPC patients centers around variability in age of disease onset and the heterogeneity of symptoms.

Josh: As a result, the disease progression is measured by the only clinically validated tool.

the four domain NPC, Clinical Severity Scale.

which evaluates four key domains.

Josh: Dean by NPC experts to be the most important, including amulation, fine motor skills, speech, and importantly, swallow abilities.

Josh: My Plypha is the only product approved by the FDA based on the NPC clinical severity scale. And the data in our label demonstrate that my Plypha, in combination with MIGLISTED, halts disease progression through 12 months of treatment.

Josh: As shown by a greater than two-point improvement in patients receiving my python, Miglistat compared to those receiving Miglistat alone.

Josh: is important to note that only a one-point improvement is needed to demonstrate a clinically meaningful difference with a well-tolerated safety profile.

Josh: Additionally, my plethora is the only FDA-approved product for NPC with more than five years of clinical data in its label and with more than 270 NPC patients.

Josh: who participated in our pivotal trial, our Open Label Extension Study, or our EAP.

Josh: In our view, these data, in addition to its well-tolerated safety profile, establishes my plan to apply for as the cornerstone of therapy for NPC.

Josh: Building on our strong body of evidence, we recently issued a publication discussing mechanistic insights into my life as mediated effects on light-as-home function and NPC in the Journal of Molecular Genetics and Metabolism.

Josh: The elucidation of my wife has differentiated mechanism of action marks a critical step in understanding its interactions with NPC at a cellular level.

Josh: These insights substantiate how my playfully addresses the underlying pathology of NPC and supports the long-term benefit observed in our clinical trials.

Josh: During our fourth quarter call, we unveiled numerous initiatives to reach the MPC patient cohorts, namely those who are diagnosed and may or may not be receiving treatment, as well as the undiagnosed population.

Continue to analyze claims data to identify existing patients.

Josh: and we are employing targeting techniques to identify new and undiagnosed patients based on related symptoms and conditions.

Josh: In addition, our targeted media campaign to build awareness and educate about early signs and symptoms of NPC is proving to be successful. We have expanded to a national scale reaching households across the U.S.

Josh: Our team is amplifying the reach of these news segments through branded geotargeting efforts in the corresponding regions.

Josh: and these new segments are increasing awareness of NPC and the availability of treatment options and are primarily designed to resonate with individuals who have been diagnosed but are not yet receiving treatment.

Josh: We also launched our disease state awareness campaign, LearnNPC, read between the signs on rare disease state this past February , and its driving disease recognition and early diagnosis.

Josh: treatment of NPC is multi-faceted and our program provides education and genetic testing options for individuals with suspected lysosomal storage disorders.

Josh: We have already seen the impact of this initiative with new patients who were not previously diagnosed with NPC, keen identified, enabling us to facilitate earlier diagnosis and offer my plipa as a treatment option.

Josh: In summary, we are encouraged by the early results of our efforts to engage the diverse patient cohorts, and we're looking forward to sharing our ongoing progress and future success with the program.

Josh: Now let's turn to Alprevo, our commercial product for the treatment of certain UCDs.

Josh: UCD's are a group of rare inherited metabolic disorders resulting from a defect in one of the six enzymes for two transporters in the urea cycle.

Josh: causing an accumulation of ammonia known as hyperanoninia, which can be toxic and lead to neurocognitive damage or even death.

Josh: As we have discussed in prior quarters, we have moderated our expectations for the pace of the launch, given the unique dynamics of the UCD commercial landscape.

Josh: From initial product availability in July of 2023 and including Zevra's promotion about Prova from January 2024 through the end of this first quarter.

Josh: 28 total prescription enrollment forms for approval have been received, including five in this first quarter of 2025.

Josh: In the case of Alpruva, our prescription enrollment forms may also include our 30-day free trial program.

As Neil highlighted, we have refined our marketing strategy.

to more specifically target the adult patient, seeking more independence.

as well as the OTC deficient and carrier population.

Josh: who may receive greater benefit from the portability and palatability offer bioprova while encountering fewer reimbursement obstacles.

Josh: All enrollment forms in the first quarter align with our targeted patient segment, suggesting that this positioning resonates with prescribers.

Josh: Given that patients with this disease typically only see their physician one to two times a year, we anticipate that the impact of our refined marketing strategy will require additional quarters to gain further traction.

Josh: For both our commercial products, we remain committed to patient access and focused on delivering comprehensive patient services through our in-house program Amplify Assist.

Josh: Our Field Case Managers, who support both my Plypha and Ophruva, are demonstrating early impact through their ability to assist with reimbursement hurdles.

Josh: Currently, our market access team continues to engage with payers to identify opportunities for Alpruva to improve its formulary position.

Josh: We maintain regular communication with pairs and have increased covered lives to 78%. I look forward to sharing additional updates in the coming quarters.

Speaker Change: I will now pass the call to Ludwig, who present the financial results for the first quarter of 2025.

Thank you, Josh, and good afternoon, everyone.

Speaker Change: In addition to the financial details included in today's call, we encourage you to refer to Zevra's quarterly report on Form 10Q for more detailed information which we intend to file shortly.

Speaker Change: In the first quarter of 2025, we reported net revenue of $20.4 million, comprised of 17.1 from my plight for revenue.

Speaker Change: 0.1 from O'Proof of Revenue, 2.3 Annette reimbursements from the French EAP for Aramaquemole, and 0.9 from Royaltees and other reimbursements under the Astaurus license.

Speaker Change: For our commercial products, my plypha and opruva, we recognize revenue when shipments are received by the specialty pharmacy.

Speaker Change: Our operating expense for the first quarter was $22.8 million, which was a decrease of 0.6 compared to the same quarter a year ago.

Combined with a decrease in personnel-related costs.

Speaker Change: SGNA expenses were 19.5 for Q1 2025, which was an increase of 9.6.

Speaker Change: Period-over-period increase in S-DNA expenses was primarily related to an increase in personnel-related costs.

Speaker Change: professional fees and other expenses associated with our commercial, medical, and launch activities.

Speaker Change: Net Loss for the first quarter of 2025 was $3.1 million, or $6.00 per basic and diluted chair compared to $16.6 million or $0.40 per basic and diluted chair for the same quarter a year ago.

Speaker Change: As of March 31, 2025, Total Cash, Cash Equivalence and Investments were $68.7 million, which was a decrease of 6.8 compared to December 31, 2024.

Speaker Change: Combined with the net proceeds of 148.3 from the sale of the PRV received just after the end of the quarter on April 1st.

Cash, Cash Equivalent and Investments would be 217 million.

Total debt was approximately sixty [inaudible]

Speaker Change: As mentioned earlier, adding the non-delutive capital from the PRV sale has further enhanced our financial flexibility to support our strategic priorities, which includes executing on the commercial launches of my Plypha and O'Proofa.

and supporting our ongoing Phase 2 trial for Slipper Law.

Speaker Change: We currently have ample resources to execute on our strategic priorities independent from the capital markets.

Speaker Change: Our financial results for Q1 2025 reflect the solid momentum in building a leading rare disease therapeutics company.

Speaker Change: and we are pleased with the opportunities we have in 2025 and beyond to drive value creation through disciplined investments where we can win.

Neil McFarlane: Now, I'll turn the call back to Neil for his closing remarks.

Neil McFarlane: 2025 is off to a terrific start for Zevra. The launch of my Plypha is exceeding expectations, our course corrections and marketing strategy for O'Proof are gaining traction, and we're advancing our development pipeline.

Neil McFarlane: Today, driven by our sustained execution, we believe we're well positioned for future growth and see Zevra as a promising commercial stage rare disease therapeutic company with a clear plan to drive shareholder value.

Neil McFarlane: Our vision for the future is bright, and we have many opportunities to serve the needs of patients and caregivers as we execute on our mission.

Neil McFarlane: Thank you for joining the call, and we'll now open the call for questions. Operator?

Speaker Change: 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 at any time by pressing star two.

Once again, let us star one to ask a question.

We will go first to Sumant Kulkarni at Canacord.

Simant Kulkarni: Good afternoon. It's nice to see the progress and thanks for taking our questions. I have a couple. So first, relative to the 122 patient enrollment forms, could you give us any details on the number of patients that are actually on my plipher right now and what the spread of reimbursed patients might be?

Simant Kulkarni: Thanks, Sumant. And just before I answer that question, I want to let everybody know that we're taking the call from multiple locations so that may necessitate a slight delay in responding as we get the folks who are going to be answering the question. I'll hand that question directly off the Josh in regards to the strong performance we've seen with my play for so far.

Josh: Yes, thanks, Sumant. So, as noted, we have seen 122 or received 122 enrollments.

since the launch through the end of the first quarter.

Josh: We do not report on how many of those are actively on drug, but I can't say that the majority of them...

Aron Drug,

Josh: and those who are not yet are going through a process of benefits investigation.

Josh: and are in some stage of pending. Some might be receiving what we call Quick Start, which is free drug while we investigate the benefits and then intend to convert those patients to paid. And then there may be others that are just in some various stage of benefits investigation.

Speaker Change: Got it. And one more before I hop into the queue. You mentioned denials of reimbursement at early stages in red disease launches are to be expected. That's understandable, but could you give us any sense of what the top reason might be for the denial of my plethora?

Speaker Change: Patience out there, excuse me, the total number of people out there with insurance.

Speaker Change: 38% of those total-covered lives who are receiving commercial insurance, federal insurance, VA are covered currently, and then any challenges that we're finding right now.

Speaker Change: Products that have to go through some sort of prior authorization before they can get approved. We've been able to get coverage for almost all of our patients, either through direct formulary physicians.

Speaker Change: either direct formulary status, or through some sort of medical exception process, which might require a physician writing a letter of medical necessity, but we've had great success in getting that covered. It's largely due to...

Speaker Change: The demonstrated differentiation that my life has with it being the only drug that's demonstrated the halting of disease progression through 12 months. It's got five to seven years of patient experience with more than 270 patients treated.

Speaker Change: and we have the only clinically validated end point for NPC, which is the Neiman Tick, so that a clinical severity skill scale.

Thank you.

Yes. So, Sumant, maybe I'll bring to the forefront.

Layed it out beautifully.

Our differentiation, the clinical profile, is what's-

Speaker Change: really driving the sprunk performance, the feedback from physicians and so far how we've been able to address the...

Speaker Change: with the strength of the data. That's really what's driving a lot of our performance today.

Thanks.

Speaker Change: Once again, if you would like to ask a question, please press star one on your telephone and keep that down.

We'll go next to Jason Butler with Citizens.

Jason Butler: Hi, thanks for taking the questions and congrats on the quarter. A couple for me on my play, so first of all,

Jason Butler: How much visibility do you have into whether patients are coming from that undiagnosed bucket versus diagnosed but not on a therapy right now? And just how should we think about bringing those patients on board, the cadence you can bring those patients on board throughout the year? And then second question, just qualitatively, can you speak to how inventory levels end of the quarter versus the end of 2024? Thank you.

Speaker Change: So Jason, thanks for the question. I'll try to quarterback this a little bit. I'll ask Josh to talk a little bit specifically in regards to the three cohorts that we talked about and the visibility. And then I'll ask him to hand it off to the Dwayne, which again may take a quick second to talk about inventory levels and our targets then and where we are today. Thank you.

Speaker Change: Jason, as we've mentioned, there are three distinct cohorts that we think about, the total prevalence

for NPC as roughly 900 patients.

you know, waiting and watching.

Speaker Change: Of that subset, we had 83 patients who were in our expanded access program, and those patients have all been enrolled to receive my plife up.

Speaker Change: We reported at the end of the fourth quarter 109 patients, and that included those EAP patients plus a combination of diagnosed and undiagnosed.

Speaker Change: We continue to see a number of the two cohorts, the diagnosed and undiagnosed patients coming in as a direct response to the initiatives that we've rolled out to bring more awareness to the disease and also to my play for the treatment option.

Del Dwayne, if you can ask.

Thank you. Thank you.

Jason Butler: Sure, thank you. And so, Jason, with regard to inventory, these are the early days of the launch.

and we seek to support patients and remain very nimble.

Jason Butler: So, we're managing that closely using a target days on hand. When we reported out in Q4, we explained that we had reached it and stocked at that target level, and we are maintaining that as we come into the end of Q1 as well.

Great. Thanks for taking the questions.

We'll go next to Eddie Hickman with Guggenheim.

Eddie Hickman: Hey, afternoon. Thanks for taking my questions and grouts on this progress so far. Any quality of metrics on refill rates or patient retention or average net price that you can give us that might be helpful for modeling the durability and treatment effects or trajectory going into the second half of the year? Should we expect this cadence of the NOVO patients of maybe around three or four per month to continue throughout 2025? And you know, what are the challenges in continuing to penetrate those non-EP patients? Thanks.

Yeah.

Eddie Hickman: So, I can't give you a little bit of color to say that, are those patients that have come in?

Eddie Hickman: and for whom we've received enrollments and who are receiving active drug.

Eddie Hickman: The vast majority of them have received refills and are continuing to stay on drug.

you know, certainly as we as we continue through the launch.

Eddie Hickman: We would expect to see that maybe diminish a little bit.

Eddie Hickman: But it really, if you reflect back to our EAT, we had patients who stayed on drug for as long as five to seven years, and so that really speaks to the clinical benefit of my plife as well as the durability of response, and we would expect to see that commercially as well.

Your other questions were around Net Price.

and, again, what we're seeing in ...

Eddie Hickman: in the EAP, with the vast majority of patients receiving one or two of the higher doses.

Eddie Hickman: So that can give you kind of a sense of what the whack price is and give you a sense of how that's trending as well.

Eddie Hickman: Yeah, Eddie, but before, maybe you have a follow-up question here but before we move forward, you know, when we actually launched the program back in September , we talked about the fact that we're utilizing the average dosing of our EAP.

Eddie Hickman: Patience, which, you know, the clinical trial leaned more to younger, lighter weight patients. And then our expanded access program really brought more adult patients than two. So what our real world experience has been so far and the average pricing that we had reported out based on the weight distribution of our 83 patients has really kind of continued the same. So that guidance that we provided is guidance that I would continue to have you move forward with.

Eddie Hickman: Thanks, and then I just, you know, it's curious sort of about the cadence of those new patients starts throughout the year. So we can, you know, continue to sort of think about 12 to 13 per quarter or with that to minister grow throughout the year.

Eddie Hickman: Thanks again for the question. I think it's difficult for us. We're not going to provide guidance. We're going to continue to be disciplined in what we do around

Eddie Hickman: The metrics that Josh talked about in new patient enrollment forms, as well as market access and then revenue to be able to pull through. What I would say to you, though, is that as we went through the three cohorts.

Eddie Hickman: and accelerating the expanded access programs and continuing to get them really rapidly in roll, which we would originally think would take us 12 months. We got it done.

You know, just a quarter and a little bit.

Eddie Hickman: We're now in the process of getting to these two other cohorts, the diagnosed patients who have not been on therapy as well as those patients who are not yet on been diagnosed. We've seen all three of those cohorts.

Eddie Hickman: MQ-1, and we expect that we'll continue to see all three of those cohorts come out of that, the EAP, those are done, but the other two cohorts, those patients are diagnosed and undiagnosed, continue as the strength of the product. [inaudible]

Eddie Hickman: and the benefits, you know, lead to getting patients on therapy. So I can't give you guidance in terms of where the cadence is going to be, but I can tell you that the strong performance today is based on our data.

Got to try. Thank you so much.

Speaker Change: Again, if you would like to ask a question, please press star one on your telephone keypad.

We will go next to Lachlan Hanbury Brown with William Blair Blair.

Lachlan Hanbury-Brown: Hey guys, thanks for taking the questions and congrats on the progress. I wanted to ask on the coverage. You said 38% of lives are covered. Can you just elaborate on what that coverage looks like? What the requirements are, did you contract for that? [inaudible]

Lachlan Hanbury-Brown: and maybe how much of an impact does having that coverage make in terms of the time to get a patient from sort of prescription enrollment form to actually on drug causative, it still requires a prior authorization.

Speaker Change: and then if I could just clarify as well, on the EAP patients he said that they'd all been enrolled just to clarify that are they all receiving drug through the commercial channel or are some of them still through sort of in the benefit investigation stage.

Speaker Change: Well, I'll take your first question around what the 38% of covered lives means.

Speaker Change: So again, most commercial plans and federal plans will evaluate a drug based on its clinical merits and some plans will not by policy.

Speaker Change: Evaluate a product until it's been on the market for six months, and some it might be as long as 12 months.

Speaker Change: and so the 38% of covered lives means that those plans, roughly 75 to 100 plans out there covering several hundreds of millions of lives.

Speaker Change: have reviewed my plightha and have made it available to their patients. And that represents about 38% of the total lives out there that have insurance. That does not mean that only 38% of our patients will receive.

Speaker Change: Grug, or be able to get drug reimbursed. That just means that there's a direct route through a formulary status or position.

Speaker Change: All plans have a medical exception pathway, and we've been very successful in getting our patients reimbursed through a medical exception pathway. We expect that 38% to increase as more plans evaluate.

Speaker Change: My play is going forward and we will continue to see that number increase and we will continue to have patients reimbursed either through direct formulary status or through the medical exception pathway.

Speaker Change: Just to follow up on an answer that are clouding your questions, you have to, if all of the patient enrollment forms.

have been put on to a commercial product.

Speaker Change: and that's the process that we work through through this medical exception pathway as well as others to get patients through and convert them from.

Speaker Change: That timeline is not immediate. Some of them are within 24 hours, they can get through the process, and others take time to work through this.

Speaker Change: Peter Pierre, Medical Exception, so on and so forth process of getting especially pharmacy products through what I'm very proud of as a team has actually worked diligently to be able to ensure that when a prescription form comes in we do everything that we can to make sure that patient and their family received the product as soon as possible while we're going through the benefits investigation process and they're actually rapidly working through this really large number again 122 enrollment forms [inaudible]

a dramatic number of forms and really strong performance.

Speaker Change: and getting through that process. Some are going to take 24 hours, some are going to take 30 days, some are going to take much longer than that. So I would say that right now, our team is doing a great job and you see that based on the revenue numbers that we've got in just our first full quarter post launch of converting those patients. Thank you very much.

No thanks.

We'll return now to Sumant Kulkarni at Canacord.

Thanks for the follow-up. This is on Celeprolol, which seems to be flying under the radar a little bit.

Speaker Change: You said you have 32 patients enrolled. It's an event driven trial where you're increasing efforts to drive enrollment. If I remember right, this is a 150 patient trial. Looks like the last update on clinical trials.gov came about a year ago.

Speaker Change: Given that the Phase 3 trial for a comparator and the starting has been suspended for some time now, what does that mean in terms of your ability to give us a timeline on when enrollment might be complete?

Thank you.

Speaker Change: I think it's important to just take a quick step back.

Speaker Change: We inherited this trial after it had actually paused enrollment for some time with a large number as we said publicly previously of screenings that were ready to go into the trial. We kicked that off in Q3 with our first patients being re-enrolled and re-dosed in the trial.

Speaker Change: and then we've been actually investing in getting through the screening of the previous patients but also now targeting how we're going to be able to drive.

Future Enrollment

Speaker Change: We talked about our ability to go to call 3-A1 patient centers and physicians to be able to find more readily diagnosed patients who are more keen to be able to go into the trial. And we also talked about some genetic testing that we're also doing as part of our screening. So we get more.

Speaker Change: Patients who are already diagnosed have the genetic defect and we can then offer them the opportunity to be in the trial.

Speaker Change: That, those activities we feel very confident will continue to drive and now accelerate enrollment of the trial, but it's really only been two quarters since we got the trial up and going. The learnings as we've walked through and now we've got these five additional patience. Thank you very much.

Speaker Change: 32 patients enrolled to date and there's a high on met need and no approved therapy as you know but we're working through with these clinics and testing centers now to really ramp it so.

Speaker Change: You know, in a nutshell, I can't tell you exactly when we will finalize it, but the strategies are starting to pay off in terms of our commitment to moving forward.

Got it. Thanks.

Speaker Change: and that will conclude the Q&A session. I want to turn the program back over to Neil McFarlane, for any additional or closing remarks.

Speaker Change: Thanks, operator, and thanks everybody for joining our call today. We look forward to keeping your praise of future progress and look to see you in Q2.

Speaker Change: Thank you, sir. This does conclude today's program. We thank you for your participation. You may disconnect at any time.

Thank you for your time, and I'll see you in the next video.