Zebra Therapeutics at H.C. Wainwright Conference: Strategic Focus on Rare Diseases

On Tuesday, 20 May 2025, Zebra Therapeutics (NASDAQ:ZVRA) presented at the H.C. Wainwright 3rd Annual BioConnect Investor Conference 2025, offering insights into its strategic initiatives. The company, known for its focus on rare diseases, highlighted both the promising launch of MyPlifa and challenges in patient diagnosis. CEO Neil McFarlane emphasized Zebra’s strong financial footing and its ability to operate independently of capital markets.

Key Takeaways

• Zebra Therapeutics has $217 million in cash, enabling independent operations.
• The MyPlifa launch is exceeding expectations with 122 patient enrollment forms.
• Zebra is actively pursuing a marketing authorization application in Europe.
• The company is investing in genetic testing partnerships to enhance diagnosis rates.
• Ciliprolol is in phase three development, with 32 patients enrolled in a 150-patient trial.

Financial Results

• Zebra maintains a robust cash position of $217 million.
• This financial strength allows the company to focus on executing current programs without relying on external capital markets.

Operational Updates

• MyPlifa (Niemann Pick Disease Type C)

- 122 patient enrollment forms since launch.

- The launch is surpassing expectations, with a focus on European market expansion.

- Partnerships with genetic testing organizations aim to improve diagnosis rates.

- Successful conversion of 83 patients from an expanded access program to commercial scripts.

• Olpruva (Urea Cycle Disorders)

- Strategy pivoted to target active patients, resulting in 5 enrollments.

• Ciliprolol (Vascular Ehlers Danlos Syndrome)

- 32 patients enrolled in a 150-patient phase three trial.

Future Outlook

• Zebra is prioritizing the marketing authorization application for MyPlifa in Europe in the second half of the year.
• The company is exploring strategic approaches to maximize the potential of its pipeline, including ciliprolol.
• Zebra is assessing the European market from both macro and policy perspectives to refine its go-to-market strategy.

Q&A Highlights

• MyPlifa Patient Enrollment

- 122 patient enrollment forms have been completed since the product’s launch.

- A third of the diagnosed patient population has been enrolled.

• Diagnosis Challenges

- The heterogeneous nature of Niemann Pick Disease Type C complicates diagnosis.

- Zebra is investing in raising disease state awareness to address this issue.

• Reimbursement

- Approximately 38% of covered lives are secured, reflecting ongoing efforts to enhance reimbursement strategies.

In conclusion, Zebra Therapeutics’ strategic focus on rare diseases, supported by a solid financial foundation, positions the company well for future growth. For further details, readers are encouraged to refer to the full transcript below.

Full transcript - H.C. Wainwright 3rd Annual BioConnect Investor Conference 2025:

Brandon Folks, Biopharma Analyst, H. C. Wainwright: Alright. Good afternoon, everyone. My name is Brandon Folks. I’m one of the biopharma analysts here at H. C.

Wainwright. And joining me for a fireside chat from Zebra Therapeutics is CEO, Neil McFarlane. Neil, thank you very much.

Neil McFarlane, CEO, Zebra Therapeutics: Yeah. Thank you for having us.

Brandon Folks, Biopharma Analyst, H. C. Wainwright: So maybe just to start, do you wanna just spend a few minutes giving people a quick overview of Zebra and the products that you currently have?

Neil McFarlane, CEO, Zebra Therapeutics: Yeah. Absolutely. And, I’d I’d wanna make sure I make the disclosure that, we will, maybe making forward looking statements. Take a look at our most recent SEC filings, for the most up to date information. And, again, thanks for having me and, and the Zebra team here.

It’s been a great conference so far. Zebra is a commercial stage rare disease company. We’ve got two commercial programs in The U. S. Market today along with a pipeline and a program that is in phase three as well as an end of Phase two, Phase three ready program that we have as well.

Well capitalized, and we’re in the process today of executing on the launch of our most recently approved product. Let me go through just quickly in regards to the products and and and the disease areas that we’re in. We have a program that that is most recently launched, as I mentioned, for Niemann Pick disease type c, and that is MyPlifa, And that is in its launch phase right now. We have another program in your certain urea cycle disorders, which is called Olpruva. We can get into some more of this as we go through the meeting.

In phase three development, we have a program, which is called ciliprolol, and it’s being developed for vascular Ehlers Danlos syndrome, a rare connective tissue disorder. And then, as I mentioned, our end, we finished a phase two and and developed a package to the end of phase two meeting with the agency. We have a single pivotal, phase three with, confirmatory data and some safety information that we’ll need to bring forth for our ten seventy seven program and rare sleep disorders that we made the determination to go and, look for strategic alternatives at the, end of last year.

Brandon Folks, Biopharma Analyst, H. C. Wainwright: Great. Thanks very much. And maybe can you just talk a little bit more about MyPlifer and especially the patient population that it serves?

Neil McFarlane, CEO, Zebra Therapeutics: Sure. So as I mentioned, my Plypho is most recently approved first in class product for the treatment of Niemann Pick disease type c. It’s a rare lysosomal storage disorder that gets cholesterol and lipid buildup in the cell and causes cell death and heterogeneous disease presentations. And and in The United States, there are about nine hundred patients from a prevalence perspective. In Europe, about eleven hundred patients from a prevalence perspective.

And again, bringing it back to The US, in our most recent launch here, we are, of those nine hundred patients, about three hundred to three fifty of those patients are have been diagnosed in The U. S. Of which, in our most recent quarterly call, we spoke about 122 patient enrollment forms in, total since launch at the end of last year. So in our first full, quarter and, let’s call it, five weeks at the end of, q four, we’re about a third of the patient pop of the diagnosed patient population with enrollment form. So the the the launch is going very well in in the in The US and exceeding expectations for sure.

In in Europe, I wanna talk quickly about Europe because in our call that we had about few weeks ago here in our earnings call, we talked about reaffirming our, MAA filing in the second half of of this year, which we’re moving towards right now. In Europe, about eleven hundred patients from a prevalence perspective. However, it’s a much more mature market in Europe. There’s been an approved product in Europe for well over a decade, and then a large number of those eleven hundred patients are actually been diagnosed and are have been on some type of treatment. So in in The US, we’re a little bit behind in regards to the number of diagnosed patients, but, we’re hopeful that disease state education along with, a lot of the product awareness that we’re putting in will continue to, identify patients and and treat them earlier.

Brandon Folks, Biopharma Analyst, H. C. Wainwright: And you touched a little bit. Maybe, you know, given the ultra orphan nature of the disease, can you just talk more about the launch and how you approach a launch in this space?

Neil McFarlane, CEO, Zebra Therapeutics: Yeah. We see three cohorts of patients of the nine hundred patients in The United States. The first cohort was the patients who had early access or expanded access, and they were in our trials. We ended or in our our EAP program, we ended that with about eighty three patients. So that’s eighty three of the three hundred to three hundred fifty patients diagnosed.

Then we talk about that diagnosed cohort of that three hundred to three hundred fifty patients. You’ve been diagnosed, You’re on some type of therapy. It could be you’re on a treatment for a symptom, but you, at that point, were not on disease modifying therapy. With Myplifa in combination with miglustat, our label indicates that you halt the progression of this devastating disease versus on miglustat alone. And that’s with an acceptable safety profile.

So that that diagnosed patient population, we’re we are, we’re also, moving forward. And the third cohort is how are we unlocking the undiagnosed patients? So that’s building the three hundred to three fifty to the nine hundred. And I and I’m pleased to say that, our efforts around, disease state awareness and and product awareness and going after from from earned media to, what we do with, with the physicians to date and conferences and data and so on and so forth. We’re actually identifying patients in all three cohorts.

The first cohort, we’ve actually enrolled all of our EAP patients into enrollment forms. So that cohort is now finalized. And then we’re moving in the other two, the diagnosed the rest of the diagnosed patients as well as the undiagnosed patients.

Brandon Folks, Biopharma Analyst, H. C. Wainwright: Great. And you talked about sort of the undiagnosed patients and sort of the nine hundred prevalence with the three hundred to three fifty being diagnosed. Why haven’t those patients been diagnosed, and what are some of the challenges to diagnosis for these patients?

Neil McFarlane, CEO, Zebra Therapeutics: Yeah. That’s a great question. NPC is a is a heterogeneous disease. It can present with in in the early forms with more visceral type symptoms, I e, hepatosplenomegaly and failure to thrive, other types of, neurological symptoms as well, all the way to patients that are, adult patients that actually will present with a psychotic break, or they’ll present with, epilepsy as their primary, symptoms. So as you think about rare disease in general, not just Niemann Pick C, you know one patient, you know how they might progress one patient at a time.

So the the heterogeneous nature of the disease makes it somewhat complicated to to note you know, notice that a patient with epilepsy might have additional symptoms that then you would test them for for, Neiman Pick disease type c. So our efforts and what we’re what we’re investing in around disease state awareness where you may see epilepsy, but if you see something else that goes along with it, then it you know, we’d like you to make sure that you think about Niemann Picksey because there’s now a treatment available that, based on our label, halts the progression of this of this, you know, progressive disease.

Brandon Folks, Biopharma Analyst, H. C. Wainwright: Great. Thanks. And, you know, maybe just what are the next steps in the launch here? You sort of touched on sort of going into all the three pillars and going into those three pillars. How do you get further penetration?

Would you say those three hundred to three fifty patients that are diagnosed, are most of them, if not all of them, good candidate candidates for MyPliva?

Neil McFarlane, CEO, Zebra Therapeutics: Yeah. Well, I think there’s a couple of questions in there. Let me see if I can try to answer the first one. How do we get to both the undiagnosed patients and the diagnosed patients? We we fully expect that, our efforts and looking at the the the playbook, if you will, of, Europe, which, with 1,100 patients, there was an approved product and a lot of investment in disease state awareness as well as product awareness, and the rate of diagnosis in Europe is much higher than that of The US.

We’re taking that playbook to The US now that we have an approved product, and we’re investing in all of those types of, activities around raising the awareness, making sure from a physician perspective, a patient perspective, community perspective, we’re raising the awareness of NPC and the the fact that there’s a disease modifying treatment. So our hope is is that we’ll be able to utilize those same activities that have proven successful in Europe, also in The US. Along, though, with what we announced, some of the efforts that we announced on our earnings call around genetic testing. We’ve partnered with a leading genetic testing organization that’s been testing for Niemann Pick C for some time through lysosomal storage disease panels. And, and we’re working with them to make sure that as an NPC patient may come up in the panel, we have the ability to educate, those physicians and let them know that there’s product availability to to to to halt the progression of the disease.

But also, in addition to that, make treating sorry, diagnosing patients also available. So we’re making that testing available with this partner that we have. So, again, the the efforts that we’re putting into raising awareness are are not really that unique to us, but we’re targeting those efforts, primarily those physicians, patients, and and and and groups that can most benefit. Thank

Brandon Folks, Biopharma Analyst, H. C. Wainwright: you. And where are the majority of the Niemann Pick patients diagnosed? You know, what’s what is the physician? And then similarly, how should we think about those patients that have Niemann Pick but are undiagnosed? Where might they be being treated for something else currently?

Neil McFarlane, CEO, Zebra Therapeutics: It’s a question that we still are in the process of answering. I don’t think I can give you a definitive there. We’re in the We’re in the first quarter of launch. What we’ve heard from physicians to date that are part of these, let’s call them centers of excellence, that have a large number of patients in our EAP that we’re treating not one patient, but treating a dozen patients. Obviously, those patients were being diagnosed and taken care of at those sites.

We’re now moving outside of those sites to where, neurologists and geneticists and and, you know, pediatric folks are getting and and actually, even on the psychiatry side, patients are being diagnosed. So it’s a little early for me to tell you that we know everything about, where these patients are and and and what what, they’re presenting with. But I will tell you that we’re making a lot of effort and focusing that effort into where we can, hone in the NPC, the diagnosis, and and and then treat patients faster. I have to try.

Brandon Folks, Biopharma Analyst, H. C. Wainwright: All right. And then you talked about the EAP, right, and the very good enrollment that you’ve had to date. Can you just help us understand the journey that these patients have from the EAP to enrollment to Zevra transitioning them into paid patients?

Neil McFarlane, CEO, Zebra Therapeutics: Sure. So I think it’s important to note that prior to our advisory committee last summer, we had approximately 70 patients in the EAP. These patients were some patients who were in our original clinical trial that then enrolled over into the OLE program. And then from from the open label extension, they enrolled them into the EAP program. And, and that 70 patients was pretty pretty, level.

After our advisory committee, then we had an influx of patients who went to these sites and, and asked to be part of the EAP. We ended about six weeks later, a month or so later, with about 83 patients. I think one of the things that’s been, most most, impactful is the majority of these patients that were in the EAP program have been in the program for a very long period. We’re talking about some patients with data up to five years. And and that’s meaningful because as when you when you launch a program, and for those who have been in the commercial business for a while, you usually launch a program, and then you go and present the the the package insert to a physician.

You provide all the data, they say, this is great, but I need more data. I need another year’s worth of data. And then, you know, you go with another year’s worth, and they say, well, I need two years’ worth of data. The the good part about what we have and and continuing the EAP program for this amount of time is we have longitudinal data of patients who’ve been on therapy in an EAP and benefiting from it. So when we now go to physicians and talk and payers, by the way, as well, And we talk about the impact of our label and the opportunity to halt the progression of a devastating disease versus, you know, the the the the the non con the control arm, I think it’s meaningful that we have this longitudinal data that patients who’ve been through this process are now converted to a a commercial script.

Brandon Folks, Biopharma Analyst, H. C. Wainwright: Great. And and just two follow ons. You said two you touched on two very important points there. Right? So maybe just firstly, how do you feel about current reimbursement levels?

Neil McFarlane, CEO, Zebra Therapeutics: I’m very proud of the team. And actually, I would say I’m proud of the team, but I’m also very happy that physicians and patients are also willing to work through the hurdles that the payer community has around high value therapeutic and new products to market. We don’t talk about specific reimbursement rates, but we give enrollment forms that come into the top, which is a leading indicator. We talk about covered lives, and and on our most recent call, we talked about about 38% of covered lives. And as you know, covered lives doesn’t equal reimbursement.

Reimbursement is the heavy lifting that’s done through the medical exception process that allows for patients to then come and get on to to commercial therapy. We have been very successful, and I and I and I, again, I I applaud the families that have to do a lot of heavy lifting, the physicians who have to do a lot of heavy lifting. But I also will say that between our field reimbursement specialists that are out there helping the families and physicians to to navigate, our our our our medical folks that are in there every day, educating payers around the disease, our sales reps that are in the in the offices, making sure they’re educating on the label. This collective effort has pulled through a number of patients, and I and and when we talk about conversion of a patient enrollment form to a script on the bottom that is, that’s being, reimbursed. It’s a lot of effort, and, all the pieces of the pie have come together.

So really happy with that so far. We have to continue doing that. That’s not something that you stop, doing. So it is, from a payer perspective, I think the strength of the data, as well as the pull from the patients and the families and the physicians willing to do the work, is what you see in our performance.

Brandon Folks, Biopharma Analyst, H. C. Wainwright: All right. And the other part of that that you mentioned that in your EAP, you had patients on therapy for a very long time. The EAP obviously then suggests that patients do stay on therapy. Can you just talk about once a patient goes on therapy, do they generally stay on therapy? And then how much support does Zevran need to put behind those patients to keep them on therapy?

Well, we’re only a quarter in one full quarter into the launch, but I but I do think that the stability of patients that have remained into the expanded access program in The US, but also in Europe. We have an expanded expanded access program, which

Neil McFarlane, CEO, Zebra Therapeutics: we call our global EAP, that has, you know, historically had 70 to 80 patients enrolled in in in, in that program. After our approval and we announced this last quarter, that’s up to 85 patients now in a small number of European countries, and they’ve remained fairly consistent and stable there too. So I I it’s too early for me to tell you what the duration of therapy is gonna be in of the patients who have transitioned to commercial product. I will say, though, that we’re here for those patients, in terms of supporting them through the payer landscape, in terms of supporting them through our patient services group. And, and if history repeats itself, those patients will continue on therapy because they really have stayed in a long time.

And I I think it’s important to note an expanded access program is is is not an easy program for families either. That’s a commitment to have to travel to a center out, maybe sometimes outside of your state once a quarter to get evaluated and, you know, we collect data along with that in addition to then get your supply from the sites, then go back to your center, you know, go back to your home. So it’s a commitment, and and, I think that that has paid off. The the the patients and the support of the physicians obviously helped us with our with our data collection, and now we have the ability to get this data out to the community and help to support the long term use of MyPlifa.

Brandon Folks, Biopharma Analyst, H. C. Wainwright: Fantastic. And you you you’ve touched on Europe before, but, you know, it’s quite interesting the sort of prevalence and number of of diagnosed patients out there. I guess so what do they do differently to have a higher diagnosis rate in Europe versus The US? And, you know, do you feel whatever it is that they do do or the reasons for that higher diagnosis rate, is that directly transferable to a US model?

Neil McFarlane, CEO, Zebra Therapeutics: Well, I do think it is transferable. Let me start with that. It is. It’s a great comparable for us to think about what has worked. Now, the bit it it my my my, answer to that is is that when you have and this is not just for Niemann Pick Sibre.

In all rare diseases that I’ve that have been associated with. When you have no available therapy, it is challenging for a physician to think about what they would do for that disease state. As therapies become approved and, and the the the momentum starts to pick up, you start to see physicians thinking about that disease. I don’t think it’s any different, for for Europe. Europe had an approved product well over a decade ago.

They went from less diagnosed patients than we had in The US, and now have been able to really ramp up the diagnosed patient population. And a lot of times that has to do with, obviously, interest of the patients and the caregivers to to to to get diagnosed and and have access to earlier diagnosis. It’s about the physicians who are willing to, do the heavy lifting to help those patients to get through the diagnostic odyssey earlier, pulling them earlier, getting them tested, and then on to some type of a therapy. And, obviously, the companies that are out there that have been investing for well over or the company that’s been investing for well over a decade of, physician education, patient awareness, and and, and disease state awareness. So I I think it is absolutely transferable to The US.

We’re starting to see that now by having all three cohorts that I talked about, the EAP, the diagnosed patients, as well as the nondiagnosed patients. In our first full quarter, we’ve had patients from all three of those cohorts. So I think it bodes well for the future, but Europe is a very good baseline for us to build on.

Brandon Folks, Biopharma Analyst, H. C. Wainwright: Great. And you talked about the MAA or potential MAA filing. How are you thinking about entering Europe and helping those patients with MIPLYFA, especially in terms of commercial footprint versus partnering? What are you considering at this stage?

Neil McFarlane, CEO, Zebra Therapeutics: Yes. So our focus is squarely on MAA in the second half of this year. As as as we’ve mentioned before, a lot of the issues that the CHMP brought up were very similar to that of the CRL. We’ve made a lot of effort in regards to ensuring that we can answer those questions and then resulting in an FDA approval. We’ll build on that information and provide it to to the European authorities to to allow them to know that, you know, the the ultimate impact that Myplyfa in combination with miglustat makes on on the Niemann Pick C patient population is significant.

We we strongly believe that this is a product that is worthwhile approving in Europe, but we’re gonna provide that additional data. As I mentioned also, we are now continuing to invest in our EAP program and expanding that EAP program within Europe. We’ve gotten inbounds that we’re able to now, satisfied by this program. We’ve gone from 70 to 80 patients up to 85 now. We’ll continue to invest in that.

And then, you know, we’ll we’ll also explore our go to market strategy in Europe. There’s nothing off the table today. We we we will look at all of the, avenues that we can take. But most importantly, we’re we’re we’re investing today in understanding what that market looks like from a macro lens perspective, from a policy lens perspective, which, you know, in today’s market, it is a changing policy and pricing and how pharmaceuticals are dealt with, both The US and abroad. It’s a dynamic space for us right now.

So we’re putting all that into the equation, and we have enough time to be able to make a very good decision for both our patients to make sure that the patients are taken care of and our shareholders.

Brandon Folks, Biopharma Analyst, H. C. Wainwright: Great. Zivra is a lot more than just my plie for us. I do want to just shift gears with, I think, the three minutes we have left here. Can you just talk about your other products and programs that you have and what success looks like for those?

Neil McFarlane, CEO, Zebra Therapeutics: Yes. So the other commercial product, I’ll start there, is Olprova, as I mentioned, it’s approved for certain urea cycle disorders, where it’s an ammonia scavenger, a nitrogen scavenger. And that product came out of the chute a little slow. We moderated our expectations. We’ve actually pivoted our strategy at the end of Q3 last year to be able to go after really where the benefits of the product really resonate with the patients.

Active patients that are looking for both the palatability, the portability and the personalized dosing, which is the differentiation that Olproofer brings to the table. We announced, a few weeks ago in our first quarter results that, we had five enrollments in, exactly in the strategy in which we’ve been moving forward. So I think there’s some optimism there for us that that we’re gonna be able to continue to, see that over the next few quarters, around, the strategy that we put forth. So we need some time, but making progress. The other product that we have that I that we also announced is is that we reestablished enrollment at the end of q three last year around our salipolol program.

As a reminder, salipolol is being developed for vascular Ehlers Danlos syndrome, Devastating disease, that is a deficient collagen deficiency, and and genetic footprint is COL3A1 positive patients. You can have, aortic, or or or aneurysms and dissections, as well as hollow organ ruptures. We are now investing in driving the enrollment of that. It’s a 150 patient, two to one randomized trial that we now have 32 patients enrolled in the trial. And and we’re focusing our efforts into those centers who are taking care of already genetically diagnosed COL3A1 patients and allowing them to go to this decentralized trial.

Decentralized trial sounds easy. You don’t have physical sites, and and you’re not managing a lot of different sites. You’re managing one site. However, getting patients to actually drive to those sites and getting patients who are, appropriate patients to drive to those sites does take a lot of effort. So it’s not as easy as it sounds, and

Brandon Folks, Biopharma Analyst, H. C. Wainwright: I think we’ve got the right prescription as we’re now seeing some uptake in in the enrollment. Fantastic. I’m gonna sneak one more in. And what I’m not gonna ask you is your cash runway. But what I’m going to ask you is given how you shored up your balance sheet of late, how do you think about capital deployment across the board?

You’ve touched on the number of programs you have going on right now. But you know, given your strong balance sheet, how do we think about Zebra deploying that capital over the next couple years?

Neil McFarlane, CEO, Zebra Therapeutics: Yeah. Just so so for clarity purposes, we announced on our earnings call that, with our our pro form a $217,000,000 in cash in the bank, we’re we have the ability to operate independent of the capital markets at this point, which we believe strongly in. And then that will allow that capital will allow us to be able to do the things that that we’re really focused on. One is the commercial launch of our two products, making sure that we do everything to get patients taken care of and commercializing those products. The last one is to make sure that we’re investing into our, soliprolol program and maximizing the efforts there.

And and lastly, I think this is an area that we haven’t talked a lot about previously because we needed to be focused on what we’re doing. But the but our our European, filing is an important and significant future milestone for the company, and we’re executing on that. So today, it’s about executing what’s in front of us today before we think about what we do next.

Brandon Folks, Biopharma Analyst, H. C. Wainwright: Great. Well, it’s a good position to be in. I’m gonna leave it there. I think we are out of time, but, Neil, thanks so much for joining us.

Neil McFarlane, CEO, Zebra Therapeutics: Appreciate having

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