Good morning, and welcome to Ionis conference call to discuss the FDA approval of ZANVASTRO for treatment of Alexander disease. As a reminder, this call is being recorded. At this time, I would like to turn the call over to Wade Walke, Senior Vice President of Investor Relations, to lead the call. Please begin.
Thank you, Joelle, and t hank you to everyone joining us today as we discuss ZANVASTRO, which is now the first and only FDA-approved disease-modifying therapy for the treatment of Alexander disease. Please be sure to visit the investor section of the Ionis website to see the press release Ionis has issued earlier today, along with the slides accompanying today's webcast. Before we begin, I would like to remind you that our discussion today will contain forward-looking statements that are based on our current expectations and beliefs. Such statements are subject to certain risks and uncertainties, and our actual results may differ materially. I encourage you to consult the risk factors contained in our SEC filings for additional detail. With me on the call today are Brett Monia, Chief Executive Officer; Holly Kordasiewicz, Chief Development Officer; and Kyle Jenne, Chief Global Product Strategy Officer.
Our agenda today will be as follows: Brett will provide opening remarks; Holly will provide a brief review of the unmet need associated with Alexander disease, as well as the data that support the approval and label of ZANVASTRO; and Kyle will review our launch strategy for ZANVASTRO, which represents Ionis' first independent commercial launch in neurology. After Brett's brief conclusion, we will open the call for your questions. With that, I'll turn the call over to Brett.
Thanks, Wade. Good morning and thanks everybody for joining us on today's call. Today is a landmark day for people with Alexander disease and Ionis, as the FDA has approved ZANVASTRO, the first and only disease-modifying treatment for this ultra-rare, progressive, and often fatal neurological disorder. The FDA approves ZANVASTRO with a broad label enabling treatment of both children and adults with Alexander disease, and w ith today's approval coming nearly three weeks early, we are positioned to bring ZANVASTRO to patients even sooner than anticipated. ZANVASTRO was approved based on positive data from our pivotal study in children and adults with Alexander disease. The study achieved a statistically significant and clinically meaningful benefit on the primary endpoint measuring motor function. Additionally, trends favoring ZANVASTRO across key symptom domains were also observed, along with a favorable safety and tolerability profile.
For Ionis, this approval builds on our long history of discovering and developing first-in-class treatments for rare neurological diseases such as SPINRAZA, first therapy ever approved to treat SMA, and QALSODY, the only disease-modifying treatment approved for a genetic form of ALS. ZANVASTRO builds on our legacy of innovation and reinforces the transformational power of our technology, and a s the first independent launch from our industry-leading neurology pipeline that today includes eight wholly owned medicines addressing similarly challenging diseases like Dravet syndrome, prion disease, and Angelman syndrome, ZANVASTRO provides a strong foundation to support these potential future launches. Furthermore, as Ionis' third wholly owned commercial medicine, ZANVASTRO joins TEGSEDI and DAWNZERA in demonstrating our ability to successfully capitalize on the opportunities from our rich and growing pipeline. We were pleased to receive a Priority Review Voucher from the FDA in connection with ZANVASTRO's approval.
This voucher provides an opportunity to accelerate the FDA review of a future Ionis medicine and potentially accelerate our ability to bring it to patients sooner. In addition to launching in the U.S., we are also expanding access to ZANVASTRO to people with Alexander disease outside the U.S. through our commercial partner Recordati, which remains on track to file for approval in the EU and Japan next year, with additional submissions to follow. Before I turn the call over to Holly, I want to extend my sincere thanks to the Alexander disease community, including the patients, families, investigators, advocacy organizations, and research partners who helped make this approval possible. I also want to recognize the Ionis team for their innovation, dedication to patients, and commitment in bringing this important medicine forward. With that, I'll turn the call over to Holly.
Thank you, Brett. Before I begin, I would also like to thank the Alexander disease community for their partnership and support that made the approval of ZANVASTRO possible. It is a powerful, determined community, and it has been a privilege to work with them. Alexander disease is an ultra-rare, progressive, and severe neurological disease caused by pathogenic variants in the GFAP gene. Because it affects the brain's white matter, Alexander disease can involve multiple regions of the brain and present with complex symptomatology. While symptoms often present first in infancy or childhood, onset can occur at any age, with most patients experiencing progressive motor and cognitive dysfunction, loss of mobility and independence, and difficulty controlling the muscles needed for swallowing, airway protection, and purposeful movement, a nd sadly, it is almost always fatal. Alexander disease is estimated to affect approximately one in 1 million- 3 million people worldwide.
Until today, in the absence of any approved disease-modifying treatments, care for people with Alexander disease has largely focused on symptom management and support as the disease progresses. Given the small size of this population and the urgent need for treatment, and with the support of the FDA and Alexander disease community, we took an innovative approach in designing our ZANVASTRO registrational trial. We combined first-in-human dose finding and pivotal studies into one controlled integrated trial protocol, which provided the results that supported today's approval. The ZANVASTRO trial enrolled 54 people, ages two to 53, the majority of whom were under the age of 18 at enrollment, reflecting the real-world Alexander disease population. The primary objective of this study was to evaluate ZANVASTRO's impact on stabilization of gross motor function in children and adults with Alexander disease.
The primary endpoint in the study measured stabilization of gait speed in participants aged five and up using the 10-m walk test, a commonly used measure of gross motor function in neurological diseases. The study also measured gross motor function in younger participants, as well as a number of secondary and exploratory endpoints aimed at capturing the constellation of Alexander disease symptoms. The approved dosage of ZANVASTRO is 50 mg and is administered quarterly by intrathecal injection, the same well-established route of administration as our other approved neurology medicines. We initially tested a lower 25 mg quarterly intrathecal dose in a small cohort of patients, which enabled us to establish ZANVASTRO safety and pharmacokinetic profile. Once complete, we tested the approved ZANVASTRO 50 mg dose in a larger cohort of patients, which represented the pivotal portion of the trial.
The study met its primary endpoint, showing a statistically significant and clinically meaningful stabilization of gait on the 10-m walk test in participants treated with ZANVASTRO 50 mg. This was a very exciting outcome as it represented the first time a medicine demonstrated disease-modifying benefit in this patient population. Secondary and exploratory endpoints also consistently favored ZANVASTRO. In participants aged two to four , ZANVASTRO improved gross motor function compared with control on the Gross Motor Function Measure-88 or GMFM-88, a well-established measure appropriate for this age group. ZANVASTRO also substantially reduced plasma GFAP compared with control, providing evidence that ZANVASTRO engaged the intended target and is modulating the underlying mechanism of Alexander disease in ZANVASTRO-treated patients. ZANVASTRO also demonstrated a favorable safety and tolerability profile in the trial.
Most adverse reactions observed in the trial were mild to moderate in severity, and serious adverse reactions occurred less frequently with ZANVASTRO compared to control. These efficacy and safety results supported a broad label for children and adults reflecting the real-world Alexander disease population. Importantly, we believe ZANVASTRO is well-positioned to become an important new treatment option and the standard of care for people living with Alexander disease. With that, I'll turn the call over to Kyle.
Thank you, Holly. We are thrilled to bring ZANVASTRO, the first disease modifying treatment for Alexander disease, to people in the U.S. who are living with this ultra rare progressive neurological disorder. This represents an important new medicine for a community with significant unmet need. We are launching ZANVASTRO with a broad label enabling children and adults diagnosed with Alexander disease to access treatment, and w e are working quickly to get product into the channel so we can bring this important treatment to patients in need shortly. With our customer facing team already in the field, we are ready to launch ZANVASTRO. Although exact prevalence is not well understood, we estimate that approximately 300 people in the U.S. are living with Alexander disease, approximately half of whom have been identified through medical claims.
And among the patients who currently receive supportive treatment for their disease, many do so at one of the approximately 12 leukodystrophy centers of excellence in the U.S. At launch, our team will focus on these centers to ensure identified patients can gain access to ZANVASTRO if appropriate. In parallel, we will work to reach treaters in neurology centers outside this concentrated network in an effort to identify new patients who may benefit from ZANVASTRO. We will do this strategically leveraging omnichannel resources as needed to extend our team's reach. We have four initial launch priorities. First, the team will work to transition patients currently receiving ZANVASTRO in the clinical study and expanded access program to commercial drug while ensuring continuity of care for these patients throughout the process.
Second, we will work to get patients who have already been diagnosed with Alexander disease and identified through ICD- 10 codes and patient registries on treatment. Third, we will work to raise disease awareness among physicians who care for people with this rare neurological disease to drive new patient identification. Finally, we will work with payers to ensure coverage and access for ZANVASTRO. We have also tailored our Ionis Every Step patient and HCP support program to meet the unique needs of the Alexander community. For healthcare providers and site staff, we have adapted our access and reimbursement support services specifically for ZANVASTRO, which is intrathecally administered, dosed quarterly, and will be reimbursed mostly under the patient's medical benefit. For patients and families, they will have access to a comprehensive suite of education and reimbursement support services designed to help patients start and stay on treatment.
Reflecting the value of ZANVASTRO can bring to the ultra-rare Alexander disease community, we are launching with a price of $285,000 per dose. We have financial assistance and reimbursement support programs in place to help eligible patients access treatment. Out-of-pocket costs for commercially insured patients may be reduced to as little as $0 per prescription. With our focused commercial approach, an experienced customer-facing team, and a high-touch patient services model, we are positioned to support timely access to ZANVASTRO for people living with Alexander disease. More broadly, the ZANVASTRO launch represents an important step in Ionis' continued growth, expanding our commercial capabilities, establishing a foundation for future independent neurology launches, and advancing our goal of delivering a steady cadence of new transformational medicines for people in need. With that, I'll turn the call back over to Brett.
Thanks, Kyle. Today's approval is an important milestone for the Alexander disease community and for Ionis. Over the years, we've had the privilege to hear directly from many families impacted by this disease, and to day, we want to again extend our heartfelt gratitude for their insights, support, and partnership in making this monumental breakthrough possible. For Ionis, with ZANVASTRO now approved, we are expanding our commercial portfolio and laying a solid foundation for our future neurology launches. We are laser-focused on delivering launch success for ZANVASTRO and across our entire commercial portfolio, thereby driving accelerating value for patients, shareholders, and all Ionis stakeholders. With that, we'll open the call up for questions. Given the importance of today's news, we'll keep the Q&A session focused entirely on ZANVASTRO or, if you want, questions applying to our whole neurology pipeline as they relate to ZANVASTRO. Operator?
Thank you. Ladies and gentlemen, we will now begin the question- and- answer session. Should you have a question, please press star followed by the one on your touch-tone phone. You will hear a prompt that your hand has been raised. Should you wish to decline from the polling process, please press star followed by the two. If you are using a speakerphone, please lift the handset before pressing any keys. One moment please for your first question. Your first question comes from Yaron Werber with TD Cowen. Your line is now open.
Thank you very much. This is Steve Ionov on for Yaron. Congratulations again management on the approval. One question from us. Of the roughly 150 identified U.S. patients, of which 54 were enrolled in the study, how many more are in the OLE or expanded access program and potentially positioned for near-term conversion? And how quickly do you expect that conversion to happen? Thank you very much.
Let me start with the numbers, and I will let Kyle comment on the time it will take for the conversion. So, we are not providing specific numbers on the expanded access program or how many patients are continuing the OLE at this time. As Kyle mentioned, one of the key areas of focus to convert patients over to commercial and as quickly as possible, I can say that, and Holly please add anything you want to this, t he enthusiasm and the number of patients that rolled over into the open label extension from the phase III study was very robust. Do you want to jump in really quick?
Yep. No, that is exactly it. So, the majority of patients have rolled onto the OLE, and all have continued.
They all have continued, and t he EAP has gone quite well. We are really, really pleased with how enthusiastic and how efficient it has been to bring in a good number of patients in the expanded access program. Kyle, what do you think about converting these over to a commercial product?
Yeah. Just in terms of timing, the back half of this year, it is going to take us a little bit of time, right? These are quarterly dosed patients, number one. Number two, we do not expect a bolus here at the beginning. There are some logistical as well as reimbursement dynamics that the institutions will have to navigate through. That is the second piece to this. Then, the other part is some of these patients that have been diagnosed might end up being referred back to local centers where they reside. So, there are some dynamics there in terms of making sure that they can find and identify a treater within their local community that can get onto therapy. But we will work as quickly as we can. Product will be in channel as quickly as we can get that in.
And we, obviously, will be supporting all the patients that are on drug today to make sure that there is no disruption in treatment.
And I will just add one last thing, Steven. Our partner, Recordati, is working with us very closely and aggressively to open up an EAP in Europe as well. So, stay tuned for that. Thank you. Next question please.
Thank you so much. Congratulations again.
Thanks, Steve.
Your next question comes from Gary Nachman with Canaccord Genuity. Your line is now open.
Hey guys, good morning. This is Denis Reznik on for Gary. Congrats on the approval and thanks for taking our questions. While I recognize these are fully different therapeutic areas and different launch strategies but maybe talk about some of the learnings you've gotten from the successful TRYNGOLZA and DAWNZERA launches that could be applied to this launch, particularly maybe with the Ionis Every Step program. Then, maybe talk a little bit more about how conversations with the payers have progressed with regards to your WAC price and what the overall path to full reimbursement access looks like. Thanks.
Yeah, happy to touch on that. Let me, I'll just first start. The execution on the launches that you referenced with TRYNGOLZA and DAWNZERA have really been very, very strong. So, we've got all of our back-office capabilities and operational components that are in place, and the team has done a very, very good job of making sure that we can do things like get drug and channel quickly, make sure that drug can obviously get to patients when the prescriptions come in, et cetera. So, we've learned a lot in terms of the operational dynamics of commercialization, and that's gone extremely well. As it relates to the Ionis Every Step program, that is really one of the differentiators and one of the things that we really take to heart here at Ionis. We map what we describe as the patient journey for every single program that we have.
We think about from the very beginning of when a patient either needs to be diagnosed or is diagnosed all the way through to when that patient ends up going onto therapy and remaining on therapy. How can we support them along the way? And what's needed from a disease state standpoint? What's needed from a product education standpoint? Obviously, access and reimbursement is important and making sure that out-of-pocket expenses and things can be managed appropriately. But those are the types of ways that we design our patient services program, Ionis Every Step. We've done exactly that with ZANVASTRO. I think that's a really important piece here for us is, this is going to be the first time that we're moving into a buy and bill model.
We understand the distribution, we understand the needs from the payer standpoint, we understand the institutions, and also the patient community in terms of what they're going to need in order to have support from an access and reimbursement standpoint. So, that's a very strong focus for us. As it relates to working with the payers, we took a very similar approach that we've taken with our previous programs. We go out and we speak to the HCPs that are the treaters of this disease. How are they going to use the product and what do they need in terms of reimbursement and support? Then, we go to the payers, and we have conversations about prevalence of the disease, which as we've talked about is an ultra-rare condition here.
And we also look at other analogs within similar disease areas as well as outside of those disease areas to make sure that we can parallel that, and that we ultimately end up with a price that is in alignment with what the value recognizes for the therapy. And so, we're very pleased with the conversations we've had with payers early on. We believe this is going to be a medical exception process. The institutions are very familiar with the way that they go through that process and how they manage this. And obviously, we have the resources and capabilities to help support the institutions in that process moving forward.
That's great. Thanks so much, and congrats again.
Your next question comes from Salveen Richter with Goldman Sachs. Your line is now open.
Good morning. Thanks for taking my question. Could you speak to the overlap the centers of excellence that you're targeting have with Angelman treaters, and how you can use this launch to establish referral networks for pediatric neurology more broadly? Thank you.
Yeah. So, there is some overlap. The focus here are going to be in the leukodystrophy centers, which there will be a little distinction there. But beyond the leukodystrophy centers, we're also going to be focused on about 25 of the key neurological centers that are out there. In those neurology centers, that's where we see some of the overlap between what we're working on here and the program with Angelman. I'll also mention we have our medical affairs team that has been out working in the neurology space for several years now, a very strong team, well educated. They know the key opinion leaders in the Alexander disease space as well as in the Angelman space.
And they are appropriately going out and educating on the disease and supporting those HCPs in the science and the needs that they have in order to learn and understand how to diagnose and treat these patients.
Just to remind everybody, this disease also is a disease very similar like Angelman in that it affects both children and adults. About 2/3 are children, the other 1/3 are adults, similar to the Angelman population. So, those are seeing those neurologists and have a lot of those same challenges with converting from doctors, from the pediatric neurologists to the more standard neurologists, and t hose are all communities that are talking and working together because it is a unique challenge for some of these diseases.
Your next question comes from Jason Gerberry with Bank of America. Your line is now open.
Hey. Good morning. This is Chi on for Jason. Congrats on the approval. I wanted to ask the pipeline related to ZANVASTRO questions. If you may, can you remind us what ASO backbone technology is ZANVASTRO based on? What other key neural pipeline programs of yours use the same backing technology? And how today's approval change your confidence of the delivery mechanism for the broader neuro pipeline given perhaps renewed focus on this topic in light of recent competitive news? Thank you.
So, thank you for the question. This is a 2'-MOE mixed backbone gapmer oligonucleotide, so it's the same chemistry as diranersen, our tau program that is partnered with Biogen. Within our wholly owned pipeline, it's the same chemistry as our Angelman syndrome program, as well as our Pelizaeus-Merzbacher disease and MECP2 duplication syndrome program, all in our pediatric neurology pipeline. So, it's a chemistry that we're extremely familiar with. It's a similar chemistry that was used for QALSODY. Between QALSODY and now, and ZANVASTRO, we had improved our screening paradigms. So, ZANVASTRO is using the latest optimized screening techniques similarly to our Angelman syndrome program. Like our Alexander program, our Angelman program is also quarterly dosed and at a similar dose range. ZANVASTRO is dosed at 50 mg, and our Angelman syndrome program, which is in phase III right now is dosed at 80 mg.
So, just to put a fine point on that, t hanks, Holly, i t's a proven platform, Chi. Chemistry is absolutely critical, and i t goes beyond chemistry, however. We also have what I believe is by far the most rigorous approach to selection, screening, and identification of optimized molecules to maximize potency and avoid off target effects of oligonucleotides. That coupled with the chemistry that Holly highlighted is also very, very important to select safe and effective molecules. That's what we do with ZANVASTRO, that's what we do with Angelman's and the other programs that are in our wholly owned pipeline that Holly just touched on.
Thanks so much.
Your next question comes from Mike Ulz with Morgan Stanley. Your line is now open.
Hey guys, it's Avi Novick on the line for Mike. Thank you for taking our questions and congratulations on the approval. I guess one, can you talk about feedback you've heard from KOLs and how enthusiastic are they about prescribing ZANVASTRO to their patients? Then, second, can you also maybe speak to your efforts to identify the approximately 50% of patients who are not yet diagnosed? Thanks.
Yeah. So, I can touch on the first point. So, the community is overjoyed. The flooding of emails and notes that we have gotten is extremely strong. The KOLs, as soon as we saw the data, have been encouraged and looking forward to this day today. And you can see that also in our EAP program where we have had significant demand from the patients as well as the KOLs to participate in that and to get their patients on drugs. So, the enthusiasm is absolutely there from the community and the KOLs.
Yeah. In terms of patient identification, we've already launched programs to do disease awareness, disease education through our medical affairs group. We will have a very strong presence on different channels through our omnichannel capabilities and our digital non-personal channels to be able to do even broader education. But it will obviously take time. It's not easily done. The other thing I'll mention is there are a lot of things related to AI that are happening now to help triangulate and potentially help accelerate identifying some of these patients. So, between the claims data, our tactics and resources to educate broadly through our marketing channels, and then combined with some of the AI technologies, we're going to do everything we can to try to help this community and identify as many patients as we can.
All right. Great. Thank you for taking our questions and congrats.
Thanks, Avi.
Your next question comes from Jessica Fye with JP Morgan. Your line is now open.
Hi, it's Silvia on for Jess Fye. Thanks for taking our questions. Just one from me. How do you see the trajectory towards peak sales? Like, has that number changed and how would that launch might differ to other rare disease launch trajectories? Thanks.
Yeah, great.
Go ahead.
Yeah, great question. Peak sales is estimated right now to be greater than $100 million for this program. We've maintained that number for quite some time. It's going to take a while to get to that peak sales number purely because of the patient identification and doing what I was describing earlier in terms of figuring out where these patients are and making sure that we can get them treated as quickly as possible. But we're still confident in the greater than $100 million, and we'll do everything we can to help as many patients as we can with this disease.
Thank you.
Your next question comes from Eric Joseph with Citi. Your line is now open.
Hi, good morning. Let me add my congrats on the approval and thanks for taking the question. Just coming back to the patient ID effort, can you talk about the role that newborn screening might potentially have here and the extent to which that's well established for Alexander disease? Also, from a resourcing standpoint, would just be curious to know how you size the MSL and salesforce here detailing to COEs and sort of local care centers. Thank you.
Alexander disease currently is not on the newborn screening panel. Th at is not something that's actively being done. The way this is typically diagnosed is the symptoms begin to onset, then it's diagnosed by MRI and then genetic confirmation of testing.
And maybe, Holly, go into a little bit why, what the challenges are.
Yes.
About getting it onto newborn screening, which is formidable.
Yes. So, the main challenge with this is you have to have an assay that can be done easily and simply. Then, you have to go to each individual jurisdiction to show the evidence that this can be done reproducibly, and then to get approval to do this. It is a long, arduous process. There is also the challenge that not every mutation in Alexander disease is fully penetrant. Then, that adds an additional challenge with doing newborn screening.
Yeah. Great question on the field force sizing. There are a couple of different teams that we have. Medical affairs, I will mention first. That team has been in the field and deployed for quite some time. They are covering the major neurology centers across the U.S. because they do more than just Alexander disease, as we were talking about earlier. They also cover the Angelman program and other pipeline programs that we have for neurology. In addition to the 25 neurology centers, there are also obviously the 12 leukodystrophy centers specific to Alexander disease. That is a part of that. So, that is our medical affairs team. We are modestly sized for our sales efforts. We know based on the number of institutions that we want to get to, that we can right size for the number of people across the United States.
It's a small team at this point because we're able to support that team with our omnichannel and digital capabilities to go out and educate more broadly around this disease with some of our marketing tactics. We also have our patient education managers, which are going to be a critical component to this. This is part of our Ionis Every Step program. These are individuals that will be able to work directly with the patients and caregivers of them to make sure that they understand the process and have disease resources, product education, reimbursement support, et cetera, associated with that. We've got all of the right teams and the right size to be able to accommodate for the known patients today that we were talking about clinical trial EAP, and also the ability to help support those that are already identified and just not yet on treatment.
So, we are rightsized and it's a very efficient organization. Thanks, Eric.
Your next question comes from David Lebowitz with BMO Capital Markets. Your line is now open.
Thank you very much for taking my question. I'm curious, at this stage in the game, where do you expect patients will get their intrathecal injections and to what extent is the ability to provide such injections as we get further from the academic centers?
Yeah. The fortunate thing here is we've had SPINRAZA for a long time, right? Obviously, Biogen's running that program, but we've been very close partners with them, and we understand exactly how they've orchestrated that model where the local communities are educated and set up with the capability to be able to do intrathecal administration. So, we have mapped the United States. We've got a good sense of where those capabilities are today. I expect the majority of patients will fall into one of those centers that has a capability in existence currently. If they don't, we'll be able to work effectively with them to get them set up and help them with that process.
Yeah, and just to add to that, Kyle, David, it's a really good question because this, of course, is a chronic therapy. ZANVASTRO is. Over time, we do expect patients wanting to, families wanting to move towards their more local community settings away from those centers of excellence and leukodystrophy centers of excellence. We will support them. That will happen over time. We recognize that, and that will be one of the challenges that lies ahead for us is to make sure that they are not losing access to the drug due to administration issues and those sorts of things, w orking with those local communities to ensure that they're fully capabilized to administer IT. But it will evolve over time. Right now, it's primarily the centers of excellence and leukodystrophy centers which are well- equipped and well- experienced.
But like Kyle said, SPINRAZA and QALSODY, and other medicines that we've developed over the years, has really changed the landscape on the capabilities of IT administration across the country.
Thanks for taking my question and congratulations.
Thank you.
Your next question comes from Yale Jen with Laidlaw & Company. Your line is now open.
Thanks for taking the questions and congrats on the approval. You mentioned earlier that you will use buy and bill model. Should that be the case given the price of the drug, should they identifying the patient first would be the key things before the physicians will stock the drug to their centers for the subsequent treatments administrations?
Yeah, great question on the logistics here and how this works. So, this will be a medical benefit product, right, so it will go through the medical benefits side of Medicare Part B, for example, or medical benefit under the commercial plans. It will be a buy and bill scenario where the institution will purchase the product and then seek reimbursement on the back end, very consistent with what they do with a lot of other therapies that they're currently using. Medical exception will be the predominant payer coverage that we believe ZANVASTRO will be covered by, so i t will be a patient-by-patient request for the therapy. They will submit the request based on the diagnosis consistent with the clinical trial, and then they wouldn't purchase product until that patient has been approved through their payer so that they know that reimbursement is going to be accepted.
That's part of the process and the steps that we'll work through, but it 's not uncommon for the institutions to do this. We've done a lot of research and spoken with many of the institutions that will be treating these patients already. They know how to do it. It's more of a time component and the back and forth with payers in order to get all of those steps of the process completed.
Okay, great. Thanks a lot, appreciate it and congrats.
Thanks, Yale.
Your next question comes from Myles Minter with William Blair. Your line is now open.
Hey everyone, thanks for taking the questions. First one's just on the 25 neurology specialty centers you're targeting. Do every single one of them already have prescribers with experience in using SPINRAZA and/or QALSODY? That's the first one. The second one's just on the safety profile: the vomiting, the back pain, the post lumbar puncture syndrome. Is that different between pediatrics and adults considering you're approved in both populations? Thanks very much.
I can answer the safety question first. No, there aren't major differences between the profile in adults and kids. It's very similar.
On the 25 centers, I don't know that all of them do have experience with SPINRAZA and QALSODY. Many of them do because these are the key centers in where neurology is being practiced and where a lot of these patients are being identified. But regardless if they have the experience or they don't, we've actually mapped, as I mentioned, across the United States capabilities in all of the major cities to figure out where we are going to be able to help these patients and get them treated.
That being said, depending upon where those institutions are referring patients back to or where patients might reside today, there could be some additional work for us to set up the capability and help them make sure that they're able to administer intrathecal administrations within the institution or the group in which they work with. But we've got the resources to help them and make sure that that happens.
Yeah, and I'll just add one other thing, Myles. Obviously, pediatric neurologists are the central physician population that manages SMA and that'll be similar for ZANVASTRO. So, they're well- experienced with SPINRAZA and certainly well aware of SPINRAZA because they're treating heads with these rare genetic diseases. And maybe now, we have time for one last question before closing out.
Our last question comes from Moritz Reiterer with Guggenheim Securities. Your line is now open.
Hi, this is Moritz on for Debjit. Thanks so much for taking the question, and congrats on the approval again. Could you just reiterate your plans for the P riority Review Voucher? Are you planning on using that internally for an Ionis program, or are you considering selling it?
Thanks, Moritz. To be determined. But right now, our focus is on our wholly owned pipeline. We have eight medicines now in clinical development that are wholly owned in neurology that certainly could potentially benefit from the usage of our PRV. That's the priority now, and that pipeline is going to grow. We're expecting additional neurology medicines to enter the clinic in the near future. So, no commitment one way or the other right now, but certainly, we see a lot of potential to utilize our PRV for our rich and growing wholly owned neurology pipeline. So, stay tuned for that. Thanks for the question, and thanks everybody for joining us today, for participating in our call. We're really looking forward to an exciting second half of the year and looking forward to sharing all the progress we're making at Ionis along the way.
Thanks again for participating, and everybody, have a great Labor Day weekend.
Goodbye. Ladies and gentlemen, this concludes your conference call for today. We thank you for participating and ask that you please disconnect your lines.