Zevra Therapeutics at Canaccord Conference: Strategic Growth Insights

On Wednesday, 13 August 2025, Zevra Therapeutics (NASDAQ:ZVRA) presented at Canaccord Genuity’s 45th Annual Growth Conference. The discussion centered on the promising launch of MyPlifa for Niemann-Pick Type C, with a strong 40% enrollment rate in the U.S. However, concerns were raised about the sustainability of this growth. Revenue saw a notable increase, yet challenges remain in other product areas like Alpruva.

Key Takeaways

• MyPlifa has enrolled 40% of diagnosed U.S. patients, with revenue growing 26% quarter-over-quarter.
• Zevra’s European expansion is underway, with an MAA filed six months early.
• Alpruva faces challenges with low enrollment and market competition, leading to an impairment charge.
• Zevra maintains a strong cash position of $217 million after selling its PRV asset.

Financial Results

Zevra Therapeutics reported $21.5 million in sales for MyPlifa, marking a 26% increase from $17 million in the previous quarter. Despite this growth, the company recognized an impairment charge on Alpruva due to lower-than-expected enrollment and the emergence of a generic competitor. Zevra’s cash balance stands at $217 million, bolstered by $148 million from the sale of its PRV asset, maintaining a neutral cash burn for the quarter.

Operational Updates

Patient enrollment for MyPlifa reached 129, representing 40% of the diagnosed Niemann-Pick Type C population in the U.S. The company added seven new enrollments in the recent quarter, down from thirteen previously. Zevra has filed its Marketing Authorization Application in Europe earlier than expected and is focusing on increasing enrollment in the Celeprolol phase 3 trial for vascular Ehlers-Danlos syndrome.

Future Outlook

Zevra aims to expand MyPlifa’s reach by increasing awareness and securing broader payer coverage. The company is leveraging its European Early Access Program experience to achieve rapid market penetration and favorable pricing. A timeline for the Celeprolol trial data will be announced post-enrollment completion.

Q&A Highlights

During the Q&A, Zevra refrained from providing guidance on peak sales for MyPlifa, instead drawing parallels with European market experiences. The company noted that 80-90% of diagnosed Niemann-Pick Type C patients in the U.S. have used miglustat, highlighting the synergistic effect of combining it with MyPlifa. Zevra is exploring commercialization partnerships in Europe.

Readers are encouraged to refer to the full transcript for a detailed understanding of Zevra Therapeutics’ strategic direction.

Full transcript - Canaccord Genuity’s 45th Annual Growth Conference:

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: afternoon, everyone. I’m Sumant Kulkarni, a senior biotechnology analyst with Canaccord Genuity, and it’s my pleasure to have Zebra Therapeutics here today. Zebra is focused on rare disease, and they really are changing what the company is all about going all into rare disease. They have a couple of products on the market already, both of which target high unmet need indications. I think most of the investor focus is on the product called MyPlifa or adimoclomol for Niemann Pick type c that’s been out in the market for a few quarters already.

And, with that, I’ll turn it over to we have, Neil, the CFO CEO, the Duane CFO, and Josh, the, chief commercial officer. So you guys, can feel free to give a little bit of intro about yourselves, and then we’ll head straight into q and a.

Neil McFarlane, CEO, Zebra Therapeutics: Sure. Quick intros, and then I’ll talk a little bit about the company.

Josh Schafer, Chief Commercial Officer, Zebra Therapeutics: Yeah. Josh Schafer, chief commercial officer. I’ve been with, Zebra for two and a half years.

Ladwyane Clifton, CFO, Zebra Therapeutics: And I’m Ladwyane Clifton, the CFO. I’ve been with the company since 02/2015, so a little more than ten years pre IPO, but we’ve come a long way since then.

Neil McFarlane, CEO, Zebra Therapeutics: Yeah. Thanks. Neil McFarlane, CEO. I joined in October 2023. Just to give you a quick little flavor around the company, we’re a commercial stage biotech company.

We have two ultra rare disease products that are in the market today, one that is for certain urea cycle disorders and another that’s for Niemann Pick disease type c, both ultra orphan indications. We also have a pipeline in a program that is being developed for another rare disease called vascular Ehlers Danlos syndrome, a rare connective tissue disorder that is in phase three and enrolling patients now, and an internally developed program called KP1077, which has been developed for idiopathic hypersomnia, and that program ran out its phase two, and we’re in the process of looking for strategic alternatives for that program. Underlying all of that, we actually have royalties and milestones from another program that was developed that’s in the ADHD space that we receive quarterly regulatory payments from. I think that’s that’s good for now. I’ll have ask LaDuena to talk about the balance sheet as we get through the the conversation.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: Cool. I thought you’re gonna read the whole forward looking statement thing. We also have Nicole Ocean in the audience. She heads up IR for the firm. So I’ll start with, with the topic of the day, I guess, which is my Plyfa.

You reported earnings last night. We saw your patient enrollment. You had 129 patient enrollment forms so far. You added seven in the quarter. To be fair to you, you’ve never set any expectations on exactly what we should expect on the outside for patient enrollment.

You said that this was in line with your expectations. How should we think about growth on patient enrollments going forward, which is somewhat like what I asked you last night, but I’d like to peel the layers of the onion a little bit more here.

Neil McFarlane, CEO, Zebra Therapeutics: Yeah. Great great question, and and thank you for that. Just to shape the question a little bit, let me give a little bit of a background for those who may not be aware. In The United States, Niemann Pick c has a prevalence of around nine hundred patients, and the most recent data we have says that three hundred to three fifty of those patients have been diagnosed. Through our, what I would call not meeting expectations, but exceeding expectations launch, we’ve actually enrolled in a which we call a patient enrollment form, one hundred and twenty nine patients.

That’s about forty percent of the diagnosed patient population. And at this stage of an ultra rare disease launch, that’s pretty impressive. When we think about seven patients going from thirteen patients in q one to seven patients in q two, we’re bit of a product of our own success. But the reality is is that’s still a 2% market share growth in seven patients in a market that is an ultra rare disease space. So the important components that we see in terms of our launch and how we’re gauging success is absolutely bringing patients in the top of the funnel.

Forty percent of the diagnosed patient populations is exceptional at two full quarters of post launch. The other component is how are we able to be able to pull those patients through? Josh can talk a little bit more about the outstanding effort of the team, but how do you get those patient enrollment forms into through the payer system, through the reimbursement system, and then into commercial revenue? And what you saw was a 26% quarter over quarter growth in revenue from, like, q one to q two. So with that, maybe I’ll ask Josh to talk a little bit about some of the outstanding work that the team’s doing to pull patients through

Josh Schafer, Chief Commercial Officer, Zebra Therapeutics: and bring

Neil McFarlane, CEO, Zebra Therapeutics: them on the top

Josh Schafer, Chief Commercial Officer, Zebra Therapeutics: of the funnel. Yeah. So as Neil mentioned, we had seven enrollments in the quarter, which is a reflection of our ability to find these patients who are either previously diagnosed, maybe not getting a treatment for NPC, or new patients who have never previously been diagnosed. And this is through our efforts to raise awareness around the disease as well as making available genetic testing so that more people can receive the benefit of as early as possible. Because MyPlifer is the only drug that’s been able to show that it can halt the progression of disease through twelve months, and more recently, we released some data from an open label extension study that shows that it halts the progression of the disease up to five years.

So we’re eager to find these patients, get them diagnosed as early as possible so that they can get on treatment and halt the progression of their disease. Also, you know, what Neil was talking about was trying to get patients into the top of the funnel is really sort of a measure of of immediate demand. But then getting those patients who come through, getting them paid, going helping them through their reimbursement challenges and overcoming any any hurdles that they might have there, and then making sure that they continue to stay on drug. And our patient services team has done an exceptional job in getting those patients who come in despite maybe an initial denial or some pushback from payers, we were able to get almost all of these patients covered and then importantly continue to receive drug month after month after month, which is reflected in the 26 quarter over quarter growth in net revenue.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: Right. We know the prevalence of Neiman big type c. We know the diagnosed patients. We know your patient enrollment forms, which is one twenty nine. Share is around 40%.

Could you kind of comment on any peak sales or share expectation that you might have and the timeline you might take to achieve that?

Neil McFarlane, CEO, Zebra Therapeutics: No. No. We don’t. But what we do have in in providing guidance and and peak, penetration at this point, what we do have is a comp, and that comp is actually based on Europe. In Europe, there was a product filed and has been approved for well over a decade.

Unlike in The US where we have nine hundred patient prevalence, in Europe there are eleven hundred patient prevalence, And throughout that decade plus of having an approved product with a label, which happens to be miglustat, which is the product that our US label has in combination with miglustat is where you see the greatest value on treatment effect for for Niemann Pick C patients. The majority of those patients have been identified over a decade plus time frame. So when we think about our ability to be able to get The US market, which has not ever had a product approved for NPC, which we’re the first product approved, The ability to be able to unlock that three hundred to three fifty patients and get closer to the prevalence patients by the workout that Josh talked about, all of our disease state awareness, the fact that these initial bolus of patients that were in our EAP were able to pull through in less than a year, which is, again, an impressive feat, but now those physicians and those centers of excellence, we’ve also mapped the local physicians who are taking the diagnosed patients out of the COEs, going back into the centers, to the local centers, and then driving those patients.

We’ve done that as well. So great opportunity for us, and I can’t give you a number cause we’re two quarters into the launch of the product, but what we see is an opportunity to unlock that diagnosed patient population and get closer to the prevalent patient population in The US over time. Hopefully, it won’t be ten years, but over time, if that allows us a good comp. Okay.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: So you probably know this, but I’m gonna ask you this question again in a different way. Of the three hundred to three fifty patients that are approved, we know the off label standard of care is Miglestat in The US. What’s your understanding of what the share of Miglestat is given Miglestat’s been approved in The US for Gaucher’s for some time now?

Neil McFarlane, CEO, Zebra Therapeutics: Yeah. Really tough question because you you hit it on the money. You said that it’s off label. It’s a very unique situation that we’re in where we have a product that’s in that we use in combination with MyPlifer that is actually off label, and utilized via multiple generics and also utilized in varying disease states. So it’s very hard for us to triangulate, but what we know from the physician community that’s taking care of patients as well as the patient community is that although miglostat can be challenging with the side effect profile that it has, the majority of patients are being tried, or at least experienced miglostat, after they get a diagnosis of Niemann Pick C.

So, our impression through our own experience with our clinical trial was about eighty percent. We’ve seen other clinical trials that are between eighty and ninety percent of patients on miglustat. And then we also have the European experience, which I think is the most mature experience we have, and that’s also fairly high.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: Yep. In So The US, eighty to ninety percent of diagnosed patients have been on miglestat at some point?

Neil McFarlane, CEO, Zebra Therapeutics: We believe that’s right. Right.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: Okay. Because it’s almost like you’ve

Josh Schafer, Chief Commercial Officer, Zebra Therapeutics: I would just also add that of those diagnosed patients, many of them are on some other treatment for another underlying symptom related to NPC, whether that’s epilepsy or seizures or any movement disorder treatment. And so we’re able to find those patients. They’ve got a diagnosis of NPC but might not be on a an NPC specifically indicated treatment at this point.

Neil McFarlane, CEO, Zebra Therapeutics: Right. Right.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: Yeah. I was trying to say it’s like miglustat is almost on label given it’s on your label now.

Neil McFarlane, CEO, Zebra Therapeutics: Unfortunately, that’s not how the FDA sees it, but we do promote the product to our label.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: Right. So, you reported 21,500,000.0 in sales of MyPlaza. We know that this is a rare disease product, ultra rare disease product. It’s sold through specialty pharmacies. So is it fair to assume it’s just in time kind of inventory management on that?

Or I know you made some comments on shipping versus pull through yesterday.

Ladwyane Clifton, CFO, Zebra Therapeutics: Yeah, that’s right. We have a target level of inventory that we maintain and we are careful to keep that pretty narrow. Our specialty pharmacy is doing a great job of getting it to patients when they present, and so but we maintain that every quarter so that it doesn’t really impact revenue.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: So on reimbursement trends, how satisfied are you with the trend as you see it now?

Neil McFarlane, CEO, Zebra Therapeutics: I’ll ask Josh to to comment, but maybe I’ll give him a big kudos here. What what we have seen is, from the initial part of our launch in terms of reimbursement to where we are today, a faster time to reimbursement, which has to do with a lot of what Josh and the team have been doing, but probably more importantly than that, the ability for our data and the strength of our data in working with payers for them to be able to actually say, okay, you’re halting the progression of the disease. That’s what we’ve been asking for, and that’s what we’re willing to be able to pay for. So, yeah, you wanna

Josh Schafer, Chief Commercial Officer, Zebra Therapeutics: go for it. I would just

Neil McFarlane, CEO, Zebra Therapeutics: Take the victory lap.

Josh Schafer, Chief Commercial Officer, Zebra Therapeutics: I won’t do that yet, but I would say that we’re at 52% of covered lives, meaning all the commercial and federally covered patients, we are on formulary of fifty two percent of those right now, and that’s exactly where we would expect to be at this point in launch. That number is gonna increase as more commercial plans review the data and and put MyPlayFit in front of its PNT, but we feel good about where we are in terms of covered lives. But more importantly, as Neil was alluding to, we are able to get coverage for those patients through other medical exception pathways, and we have had, an extraordinarily high success rate in terms of getting patients paid, or getting getting coverage for those patients and getting them paid and and staying on drug.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: Correct. What’s your denominator for the covered lives calculation? I’m sorry? What’s your denominator for the covered lives? 5052% of

Josh Schafer, Chief Commercial Officer, Zebra Therapeutics: 52 of, all covered lives, and I think that’s roughly about a 180,000,000 US lives. Okay. So that’s all the commercial plans, Medicaid, Medicare, and all the supplementals. Right. So

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: So then, how would that translate to the like, how do patient enrollment forms translate to the number of paid patients that you have on the product as of now?

Josh Schafer, Chief Commercial Officer, Zebra Therapeutics: So we don’t report on that, but I think the best you know, again, if you look at 129 enrollments that are coming in, the best way to measure the paid rates is to look at the the net sales and to be able to see that the quarter over quarter, we went from 17 last quarter to 21.5 this quarter. That’s sort of the calculus that needs to be done to be able to determine paid lives.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: Right. If you look at patient enrollment forms for the next quarter or two, not I mean, you can give numbers if you like, but qualitatively, what should we be thinking about the direction of those enrollment forms relative to the seven you had this quarter and the numbers you had in the prior quarters?

Josh Schafer, Chief Commercial Officer, Zebra Therapeutics: Well, again, we’re we’re only two quarters into the launch. So I think it’s really challenging for us to to give any sense of of trend or or guidance at this point. What I can tell you is having been out now for a little more than two quarters, we’re seeing awareness of MyPlifer raise, particularly outside of those centers of excellence. We’re seeing that more of the patients are, you know, they get maybe initially diagnosed in a center of excellence, then they go to a community neurologist or physician. And the awareness within that community doctor or within the community is really raising.

So again, too early to tell, but, you know, we are seeing some some positive trends and more patients coming in who were previously undiagnosed, which I think is also a good harbinger of Yeah. Of, you know, our our tactics and initiatives are really starting to take traction.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: Right. So this is because we’ve got a lot of investor focus on enrollment forms, and that’s the reason why I’m asking you so many questions on this. So the seven patients. Right? Is that somewhat of a steady state growth wise?

And, like, any kind of info you might give there would be really helpful growth rate wise or steady state wise.

Neil McFarlane, CEO, Zebra Therapeutics: I wanna try this

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: hit the three fifty.

Neil McFarlane, CEO, Zebra Therapeutics: I appreciate I appreciate that, and I think that it’s an important question because our goal here is to be able to impact as many patients’ lives as we can. There’s also a a expectation that we have that we’re gonna be able to maximize MyPlaypha for every patient with NPC. So the efforts that we’re putting forth and the tactics and the resources that we’re putting into this marketplace from patient advocacy to we’ve been at every one of the meetings. We brought our specialty pharmacy to the patient and caregiver meetings in North Carolina at the annual meeting to make sure that patients that are impacted have their pharmacy there to help them in the event that they need to. All the tactics and efforts that we’re doing about impacting and maximizing the value of NPC.

What I can tell you though is that this time last year, we didn’t have this number of patients and a 40% share of the market in in our numbers for the next for the last for the next couple of years. So you’re at you’re you’re you’re rightfully asking the question around, is it gonna be seven next quarter? And what I can tell you is that we’re making no lack of investment and effort, and and I and I talked about the sense of urgency that this team has in terms of its ability to go out and try to impact patients. Everything is being done to do that. So we have a great model in the ability to not just, as Josh mentioned, go out and actually get patients who’ve never been diagnosed.

We’re offering genetic testing today. That genetic testing is identifying patients who’ve never been diagnosed before, and every time we impact the patients’ lives, it’s a big deal. So if it’s seven, if it’s if it’s more than that, if it’s less than that, we’re gonna continue to drive to make sure that we can get closer to the nine hundred people living with NPC.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: Got it. So on that note, targeting the eventual nine hundred, is there an initiative to get the FDA to or to convince the FDA that your product could be used as a monotherapy?

Neil McFarlane, CEO, Zebra Therapeutics: No. No. There isn’t. And the reason and, actually, that’s a really great question. We actually just published mechanism of action data talking about some of the early work we’ve done to be able to put that that biologic plausibility from early stage mechanism of action all the way to our clinical data that we’ve seen.

Now we’ve been able to uncover long term data. Not uncover. We’ve been developing it for some time, and what you see is a synergistic effect when you give MyPlifa and Miglostat together where you don’t see it as monotherapy. So, now, I can’t say that you don’t see it, but we didn’t have enough patients who were not on it in our trial to be able to see it, but what you do see is a halting of progression of a devastating neurodegenerative disease. That is the hallmark of what patients and families and physicians have been asking for.

How do you stop or halt the progression of this disease? So with in mind, the label that we have allows us the great, and now this is five year data in our OLE, we will be publishing even longer data in our EAP data. The database was locked at the end of Q1, so this now gives us this ability to go out and really drive. The biggest value we can make for patients is when you give it the way that the label is.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: Right. So in The US, how are you thinking about exclusivity, patent life, and life cycle management around the intellectual property associated with Mitlaf?

Neil McFarlane, CEO, Zebra Therapeutics: Yeah. So today, we have orphan drug exclusivity. The intellectual property in terms of patent term extension, we have filed, and we’ll we’ll you know, working through that with the with the office. I can’t tell you what that is until we get that completed. And in addition to that, last year prior to our advisory committee, we did file additional intellectual property around combination therapy and and and other things that are that were important for us to to protect.

So I think the team’s done a wonderful job. That’ll take time through the through the the patent office and and the speed at which they operate for us to understand. But once we know more, we’ll be able to get back

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: to you on that. Okay.

Ladwyane Clifton, CFO, Zebra Therapeutics: Got it.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: So you mentioned the synergistic effect of using Myplafir with miglustat. What does that mean for the philosophy that the European Medicines Agency has around this product and its place as a treatment for Neenahpic?

Neil McFarlane, CEO, Zebra Therapeutics: Well, so thank you for that question. The I get back to the core. The core of the questions that we had from the first time with the CHMP, you know, conversations and the EMA conversations we had when the product was previously filed in Europe. Those questions were very similar to The US’s CRL. Not only we we’ve been able to refile that two weeks ago, we filed our MAA application early, I might add.

The guidance was in the 2025, and we filed it in the July, about six months ahead of what most people were expecting. And that came with this additional data that we I just discussed. So it came with our ability to have the OLE published in a peer reviewed journal. It came with the mechanism of action data that I discussed a moment ago, and it also came with the pediatric data that we had been enrolling since our original application in trying to put the PIP together. That study’s completed.

We have not shared that data publicly, but that data was put in. So the robustness of the package that we have in Europe now, we believe, will allow us to be able to overcome any previous hurdles, but actually also allow us to be able to have this combination and show to the European authorities, hey, if you’re on miglustat alone, we know what that looks like. It’s a two point progression. One point’s clinically meaningful. It’s a difference between somebody being in a wheelchair and being able to walk.

Two points is significant. So we see this delta will go into the health technology assessments and evaluations with Europe to be able to show the magnitude of of impact that will have on on patients.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: Okay. So you do have data in hand that shows that patients on miglustat alone would not would progress at a faster rate or Yes. Adding Mycliophile

Neil McFarlane, CEO, Zebra Therapeutics: would That’s in our label in The US today. Right. It’s about a two point progression. That’s exactly right.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: Yep. And that that’s gonna augment the application relative to what you’d filed or what was filed in the past.

Neil McFarlane, CEO, Zebra Therapeutics: Previously. And that’s also with this duration. We have now five years of published OLE data, and we submitted to the agency our EAP data in The US where we count we captured data, and that’s upwards of seven years of data Right. That that we’re we’re looking forward to being able to publish soon, but that was submitted to the EMA.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: Got it. Europe is typically more price sensitive than The US. Now now that we have a list price in The US, how are you thinking about pricing of this product when it gets approved?

Neil McFarlane, CEO, Zebra Therapeutics: Really too early for us to talk about pricing. We need to we need to talk about label and and getting through the data before we do that. What I will say though is that we have been actively, and Josh and some of the other folks in the team have been actively refreshing our pricing quarter work based on the value proposition that we’ve been talking about in this spread of clinical meaningfulness at a country by country level to understand how would we do sequencing and how is the best way for us to optimize our go to market strategy. Today, with the filing and now knowing the strength of the data that we’re bringing, it’s an opportunity for us to really look if we’re gonna do it ourselves. We’ve been having a lot of capabilities presentations from regional partners, multinationals, local distributors, and we have an eye with 89 patients in our European EAP now.

This time last year, we’re about 70. 89 patients in our European EAP, we believe that we can do the similar type of rapid penetration in those markets where we can get early access and pricing that we’d be able to launch those markets as well. So it’s a little early, but we feel pretty confident that the strength of the data, the fact that we’re continuing our EAP in Europe, and it is it is growing, in a small number of countries, it gives us a foot ahead when we think about how to commercialize in Europe.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: So on that note, commercialization in Europe, do you plan to do it yourselves, or are you looking for partners? How’s that

Neil McFarlane, CEO, Zebra Therapeutics: It’s open right now, Sumat. We are we are exploring all alternatives, and the goal here is for us to be able to, make the best choice, while we’re under review.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: Got it. So I’ll move then to Alprova for urea cycle disorders. You had you’ve had that product now for some time. You’ve you’ve launched it because this is a true new launch effort on Alprova. There are some dynamics there that are affecting

Could you go into that, and how are you thinking about the product in the longer term, especially relative to the, the accounting concept of taking an impairment charge?

Neil McFarlane, CEO, Zebra Therapeutics: I’ll I’ll ask, you can start, and then you can talk about the the impairment charge. Yeah.

Josh Schafer, Chief Commercial Officer, Zebra Therapeutics: Alpruva is a treatment for certain urea cycle disorders, and that is a very mature market. There are several other products in the marketplace now, and and just to be quite honest, the enrollments in this quarter, we had one enrollment not living up to the expectations that we had. We are nonetheless, we believe that Alpruva has a place in therapy, but the market is quite satisfied with the market leader right now, which is Revicti. There is an authorized generic to that product coming into the market any day now, and we believe that that is really an opportunity for there will be a little bit of chaos and disruption in in the marketplace, and it’s an opportunity for us to continue to emphasize the the clinical benefits that Elprova has, but also to really continue to differentiate ourselves based off of the patient services and the capabilities that we have there, which are, you know, used for MyPlayfa but also for Alpruva. And and we believe that, you know, the combination of our product profile and our patient services are really gonna help.

As patients are being forced off of the the market leader, we will be a viable alternative for them and we believe we would offer significant benefits to those patients.

Ladwyane Clifton, CFO, Zebra Therapeutics: Related to this quarter, we did recognize an impairment charge. Oprovo was one of the assets that came with our acquisition of Acer in 2023 and so it gave rise to these assets on the balance sheet that have been amortizing. The accounting guidance is a bit more strict and probably doesn’t take into account all the opportunities that we see as Josh just mentioned. So there were indications of impairment based on the prevailing enrollment trends and the entrance of generic that, an authorized generic that could impact that. So the auditors look at that with a bit more skepticism and through that analysis determined we needed to take this impairment charge this quarter.

So we did that, also recognized some inventory write off. But at the same time, I think the original premise not only was that we were going to get an asset with Oprova, but also that the infrastructure that came with that really allowed us to build out what we needed to support MyPlaypha at the same time. So, again, elements of value from that that aren’t really reflected in this accounting determination, but, we continue to believe in the product and believe that Josh’s team and this plan of execution will take advantage of those opportunities that might be presented in this next chapter. Got it.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: In the last minute or so, a couple of quick questions. Celeprolol for vascular Ehlers Danlos syndrome, a lot of unmet need in that. At what point will you be able to give us a timeline on when we can expect top line data on that phase three? Yep. And, Ladewena, a question for you.

What’s your latest cash runway?

Neil McFarlane, CEO, Zebra Therapeutics: Okay. I’ll take the first one. So that trial is a 150 patient trial. It’s an event driven trial. So we have to get all enrollments, and then I can give you a time frame as to when we expect those events because that’s gonna be more statistically driven that can pass that along to you.

Two good things about this trial, I know that we’re past time. One is is that there’s an interim analysis at 28 events that allows us to be able to and it’s being run under a SPA. I might just say that. It allows us to be able to take an interim look and with that data, fileable if it is if it hits what we need it to hit. If not, we go to four to six events, and, that’ll be the ultimate outcome of, of the trial.

So as soon as we get all enrolled patients, timeline coming your way. And our goal right now, as we mentioned on the call, all the activities, we’re starting to see that ramp in the enrollment of COL three a one positive patients. Our goal is to be able to see that, continue, in the next few quarters. Got it.

Ladwyane Clifton, CFO, Zebra Therapeutics: And during q two, we sold our PRV asset that we had also received with the approval of MiPLAFA, and so bringing that onto the balance sheet, that was a 148,000,000 in net proceeds, brought our cash balance to $2.17, and we just reported yesterday the June 30 balance was still at $2.17, meaning that our cash burn was neutral during the quarter. We can’t really predict or project how that’s going to go into the future, but we’ve made good progress and we have a solid balance sheet to execute against our priorities, supporting MyPlifa, supporting the MAA application, and then the saliprolol trial.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: Got it. Thanks for that. Eager to see your patient enrollment next quarter.

Neil McFarlane, CEO, Zebra Therapeutics: Appreciate it. Thanks so much.

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