All right. Good morning, everyone. Tyler Van Buren here, Senior Biotech Analyst at TD Cowan. Thank you very much for joining TD Cowan's 46th Annual Healthcare Conference for our next session. Very excited to be hosting a fireside chat with Soleno Therapeutics, and it's my pleasure to introduce Anish Bhatnagar, the CEO, and Meredith Manning, the Chief Commercial Officer of Soleno. Anish, Meredith, it's a privilege to have you both here with us. Thank you very much for joining me.
Thanks for having us, Tyler.
So, before we get into the questions, maybe we'll give you the opportunity to start with some high-level comments on Soleno and the Vicette XR launch, especially given the recent earnings announcement and updates on the franchise and in Q4 heading into this year.
Thanks, Tyler. So as most of you, I'm sure know, we're in the rare disease space. We have launched a drug, the first ever drug for hyperphagia in Prader-Willi syndrome, VicatXR. We've had a tremendous launch. We've had about 190 million in revenue in the last three quarters. We are in the process of expanding the Vicat franchise. We recently talked about the next indication, which is glycogen storage disease. We think it's a high probability of success. It's something that makes a lot of sense. No FDA-approved therapies, et cetera. So, yeah, looking forward to the conversation.
So just to follow up on that and staying at a high level, now that you guys are three-quarters or three-plus quarters into launch, What's been the biggest learn-as-you-go insights as you bring, you know, the first approved therapy for Prader-Willies to patients?
I think the most interesting thing has been that it's sort of verification that this is a big market. You know, we knew going in that the claims data were telling you that there's a lot of patients out there. And we've seen that pan out. What we've also seen pan out is interest from the community, interest from the physicians. if you look at our number of prescribers you know more than 600 prescribers is something that no one had really thought would be possible that quickly so that's been very rewarding and the other thing that's been very interesting is our reimbursement our our coverage we're seeing you know more than 180 million lives covered we're seeing payments from more than 45 medicades we're seeing medicare be one of our best pairs we're seeing all the large pbms remove new to market blocks so overall
it's been it's been great so maybe we'll move to start forms can you uh you know discuss uh how that's progressed over the course of the launch and what your expectation is for start forms moving forward in q1 and subsequent quarters yeah as we all know we had a huge bolus to begin
with not one we were expecting but very large bolus and we've seen meaningful numbers for the rest of the year and the fourth quarter was about 200 plus start forms which we think was we're getting towards steady state and we've said publicly that we expect to do about a thousand over nine to twelve months and it's really driven by two major factors one is you know we ask our sales team to do a bottoms-up assessment on what they can do and the other is we know that the larger practices are crowded, unable to insert people sort of at will, and that they'll be seeing patients every six to 12 months. And when that time comes, they'll offer drug to them. So that's what drives that number.
Okay. And 1,009 to 12 months, should we expect 250 plus a quarter, or could there be some lumpiness between quarters as we look out over the course of the year?
Yeah, we discourage people from looking at quarters. we prefer people look at the year. So there's definitely lumpiness to be expected.
Has what you've seen so far in January and February given you confidence in reaching that 1,000 over the next nine to 12 months?
Not really commenting on the quarter. I had to ask.
Sure. As we think about start forms translating to revenue, revenue what do you feel what does that time frame look like in terms of start form to actually getting drug to titrating up to the full dose and the full price and really adding to sales
marida do you want to take that yeah happy to take that we've stated that um the typical turnaround time that we're looking at is anywhere from 30 to 45 days but i would just share the pathway of the way that a start form comes in to our specialty pharmacy sorry there's a little bit of feedback thank you comes into our specialty pharmacy so first you know we work on the benefits verification and then making sure that the families are coached up and ready to to get started once we make sure that the payers have the ability to reimburse then we we move forward and shipping is pretty easy. We're very pleased with the way that Panther is able to get the product Vicadexar out to the patients very quickly. We've been, as we've mentioned, we've been very successful at the reimbursement. The overwhelming majority are on paid versus free, but we do have a comprehensive program that if there are people who need either a quick start or a bridge or a free drug, that we have that available as well.
And just to follow up on the PSFs from Q3 to Q4, was there holiday related seasonality in Q4 that impacted that start form number? And is that something that we could see reverse in the new year?
You know, I think seasonality for sure, especially, you know, food related holidays like Christmas, Thanksgiving, et cetera, are meaningful for this population. But I do think that there's peculiarities that are specific to the disease itself. And I'm not sure that they're so seasonal. So you have situations within families, high divorce rates. We had a recent example of a patient who had a script but did not start because the parents divorced. So we see situations like this, which we find peculiar to this disease state. So yes, while there is seasonality, I think there's other things that happen in this disease state that are very specific to it.
Maybe you could just elaborate what you're seeing in terms of persistence or discontinuations and what you expect that to look like over the long-term.
Yeah, the discontinuation numbers we said for adverse events as of the fourth quarter launch rate were about 12%, overall about 15%. We have said in the past that we expect long-term discontinuation rates to be in the 15 to 20% range. And the one thing that we're tracking is in the trials, we saw discontinuations to be earlier in the process. So if you stayed on drug for about six months or so, you were likely to stay on drug long-term. So that's a metric we're watching very carefully. Early indicators are that is indeed the case here, but we'll watch that carefully.
Okay, and so the discontinuations are primarily due to adverse events early on, you're not seeing discontinuations due to efficacy yet? How are you thinking about as patients get to kind of six to 12 months on treatment, especially for those that may not have as good of a response as others?
Overall, we feel that if you stay on drug, a vast majority will have a response. And we see that, we see responses coming on early, we see them deepening over time. So I think there's this narrative about the six to 12 months, I think it's more related to the fact that our data shows maximal effects in those six to nine or six to 12 months. We see efficacy coming on early. So I think it'll be unusual for someone to stay on drug for a year and not see efficacy.
And in terms of determining max benefit or response in a patient, do you think a doctor really needs six months or it's really that kind of first full year, 12 months that's required to determine that?
I mean, honestly, I don't think it's the doctor as much as the family, because these are really observed behaviors. And while some of them could be observed in clinic, generally they're observed by the caregivers. So the doctor might ask the relevant questions, but the answer really comes from the caregivers.
And maybe you could just give us the latest on coverage, where you're at in coverage, and when you expect to reach peak coverage?
Yeah, so as Anish had mentioned, we're at the end of Q4, we were at over 180 million lives covered. And so we'd look at the various sources that you all probably look at MMIT to track the covered lives. As Anish mentioned as well, we have over 45 state Medicaid programs that either have a policy or have covered. I think that we also mentioned in our earnings call that we're probably going to, after Q1, stop reporting out on the covered lives, because as you'll notice in rare disease programs, Medicare Part D doesn't usually list the product or the medicines on their formulary because they don't want populations going and searching for specific plans. And so if you look at MMIT, it's gonna be underreported and most rare disease stop at around 60% covered. But we're very pleased. We've had really strong coverage in Medicare. We mentioned about the 45 states and Medicaid. All of our top national PBMs last year removed the new to market block. So we're super pleased. And also early trends last year, looking at reauthorization were very strong and we haven't had any issues with that.
What needs to be accomplished to get some of these last payers on board over the next few months?
Yeah, the majority of them are really just state Medicaid, so the last couple of states, and they just take time, and they go through the P&T and the DUR drug utilization review, and so it's just timing on those.
you could discuss the latest on the average weight and price per patient that you all are
seeing? Yeah, so as we've said in the past, the clinical trial weight was about 61 kilograms on average, 30 and a half years of age. What we've seen over time, we've said a vast majority of patients are between 4 and 26 years of age, and therefore on average heavier than what we have seen we've talked about the whack being north of about 500 and overall we feel that we'll start we have started to see older patients come in so the patients will be heavier on average perhaps we don't expect large step ups though we see this more as sort of a gentle cadence moving upwards and on average
how many patients titrate all the way up to the maximal dose or their optimal
dose and how long does that take we're tracking that information i think it's fair to say that a vast majority of them will get to where they need to um when the clinical trials for example virtually everyone did so we're tracking it carefully but right now i'd say a good majority
is getting there and uh obviously uh the ex some of the more experienced doctors they'll pause titration if they see some safety events and then allow it to recover and then go back to titrating but assuming there's no pause remind us how long that should take for the average patient it's about six weeks six weeks all right um and uh gross to net is it should we still expect it to be somewhere in the range of 15 to 20 percent or should that could that continue
to grow over time or so as we've said we expect it in the long term to be that we've seen it to be somewhat lower until now but as we pointed out the first quarter you should expect it to be higher for the reasons that we have discussed one is new policies coming in and you know needing to provide free drug for a period of time and the other is copays resetting so as you know for commercial payers we do pay for the copays so we expect the gross trend to be higher in the first quarter
And what percentage of patients are on free drug, and how long, on average, does it take to move them from free drug to paid drug?
So we're not giving full percentages, but the vast majority are on paid. And as many people might know, if you start a Medicare patient on free drug, you have to carry them out through the full year. Medicaid, you can actually convert onto paid and then commercial, we work through that on a day-by-day, week-by-week basis, but we've been really quite successful at converting to paid.
And I would add that a lot of the free programs are actually short-term, so there's three programs that we have. One is Quick Start, one is Bridge, and the third is actual, like, true free drug. and that category is actually very small at this time.
And maybe just, we could touch on the prior auth process. What's that been like for most doctors?
I think if you've heard any of the interviews, they're all quite surprised at how smooth it's gone. So kudos to our market access and medical team for really laying the groundwork with the payers. As we've said before, a lot of the reauthorizations that are coming in with the policies are anywhere from six months to 12 months, with the majority sitting around 12 months. And as I mentioned, some of those patients who had six months reauthorization in November and December have gone through quite well.
And just following up on discontinuation, how many patients are you seeing discontinuing and then coming back onto drug or being reintroduced?
This is starting to happen. And I think it's a steady stream of people coming back. And interestingly, you know, one would expect that if you had a more severe adverse event that led to discontinuation, you might not want to, but we've actually had a meaningful number come back on drug or want to come back on drug in spite of serious reaction.
As you've noted, well over 600 prescribers, many with few patients. So maybe you could talk about your plan to kind of reach these prescribers that may be less familiar with VicatXR as well as the patients and just your educational efforts and events that you all are hosting.
Yeah, so we have really placed a large emphasis in the field team that we have gone to market with in the go-to-market launch, focusing on those who are high volume prescribers. at the same time we recognize that there were a lot in the community with regard to the long tail so we've put into place other teams like hybrid teams who are able to generate leads and pass that off to our sales team we did that early on at launch and we've seen that to be very effective that raises awareness across the community especially in those settings where they may not be as steeped into pws we have our msl team and the medical affairs team who is really putting a lot of resources towards educating across both academic settings as well as the community settings and when we look at the metrics of awareness and growing awareness it's very strong and we're also looking at the knowledge around vicatxr and that's really a proxy for comfort
and we're seeing that grow as well when you um host these events are you seeing uh like an uptick in prescriptions immediately following those events how do you assess kind of return on investment and return on time with respect to your promotional efforts yeah so i think you're
speaking about the patient events so we both have um the patient webinars where usually usually we have one of our top pws experts talking and dialoguing with a family caregiver we actually had a patient who came on and did a webinar and then the other events that we have are the live patient events we actually had one last year in october in philly we just did one last weekend in north carolina and we had two physicians join that as well and it's early but we are hearing of patients who reach out to us families who reach out to us and say thank you for hosting that because of that i went in and i asked um to be prescribed by cat xr and said i'm not leaving until i get it so it's it's been very successful and before we get to um ex-us and uh the newly
announced a pipeline indication just in the us what do you believe what's your latest thoughts on the ultimate market opportunity for viket xr how large of a drug do you think it will be over
the long term i think the numbers speak for themselves and the claims data itself at the time of launch we said we were seeing confirmed 12 500 cases which is a very meaningful number so even if you were to assume a very modest penetration of that this is a very large drug
so the math is not that difficult to do so um turning to europe so you responded to day 120 questions i think you said day 180 were expected maybe the day after you reported earnings so presumably you've got those questions are there any comments you could provide with respect to those questions and and maybe even how you if you've responded or how you plan to respond and next steps I can acknowledge that we received
the questions we're processing them right now it'll take us some time were
surprised by any of them no comments fair enough how do you think about the opportunity in Europe and your ability to launch by cat there with without a
partner let me talk about the opportunity I think it's it's very meaningful if you just look at the EU 4 plus UK you're seeing about nine nine thousand five hundred patients based on primary market research when you look at prevalence estimates we're seeing north of 15,000 in the EU 27 so it's a very meaningful market and i think it is interesting that in many ways it's a lot more structured than what we see in the u.s particularly in markets like france and germany center centers of excellence really drive it and we've started to work on a team and i'll let meredith deal with
yeah so i think um it's as anish had mentioned a very attractive opportunity right now we're We're working on looking at the workload and the number of sales force that we need. We already have some of our medical team and the MSLs who are already starting and talking with KOLs and understanding the market landscape and doing market development activities. I think that what we're very pleased about is by those who are watching us in Europe and seeing the success that we've had here in the US and getting to know the PWS community. That's very special. have a lot of individuals who are very expert in rare disease and want to join seleno so just to
follow up on the structured comment uh you mean the like the infrastructure like the treatment infrastructure the practices you're it's just it's easy to identify the practices and the institutions that you need to go after is that fair to say relative to the u.s i think the uk
might be an exception but other than that yes got it is that fair yeah a little more concentrated
you know if you look at germany they're just they have more of the established centers of excellence than the u.s although the u.s is growing we've commented a couple times where we're hearing like even here in boston boston children's um finally got approval to set up their pws clinic really recently and so um but that's a little bit already established in europe is the diagnosis rate in
europe similar to the u.s or could it be better given what you just mentioned uh it varies by
country so if you look at france there's published data that shows within the first couple of months diagnosis happens really early but we've already done market research where other countries there's some work that we need to do but really strong advocacy across the physicians and i think not this past december but december of 2024 we were able to bring together i think it was almost 60 thought leaders across europe and the great thing is that they're a tight community and they share and they share best practices and assuming approval in europe you guys are committed to
launching it yourselves as opposed to partnering or is partnering still an option you know it remains
an option there's a lot of interest but i think the ongoing challenges or uncertainty with mfn certainly makes us believe that it's best to have control not made the final decision yet but we
are certainly thinking more in that direction yep that makes sense certainly hearing that from other companies as well um okay uh so maybe uh moving to um uh the new indication gsd1 that you announced during q4 earnings can you just elaborate on that indication the mechanistic rationale to pursuing that indication with bicat xr and if there's been any thing observed historically
in terms of case studies or clinical experience yeah glycoin storage disease in particular type 1 is a terrible condition it's a situation where your body is not producing enough glucose or having enough glucose in the blood and leads to potentially fatal hypoglycemic episodes and the way these people survive is by taking multiple feeds of uncooked cornstarch during the day so that their glucose levels remain high enough it is not a great way to live life if you speak with one of these families people have been able to survive and they live fairly normal lives so they don't have the sort of comorbidities that we see in pws but it's a very tough way of living multiple efforts have been initiated to try and deal with this the only one that's bared fruit to some extent is the ultrogenics gene therapy the data from that phase three study and it's now in front of the fda shows that there is a very modest decrease in cornstarch use for about four from about five to about four times a day which is pretty modest in addition about 20 to 30 percent of those patients have antibodies to AAV so they will not be candidates in addition there are known problems with AAV therapies related to the liver and the liver is a primary target organ in GST so it leads to a situation where these three to four thousand people in the U.S. are really in dire need of a treatment option and interestingly there is very limited case reports that have been published from a very long time ago that show that the parent molecule can actually be used effectively and i've spoken with the kol who has actually used that molecule in the past successfully but could not continue to use it because of its side effect profile given it's multiple times a day use in very high doses so we think vicatxr has the opportunity to provide a very meaningful solution mechanistically it makes a lot of sense because as you know the drug decreases the secretion of insulin from the pancreas as a direct effect so what we need to do is to do exactly that raise blood glucose levels just enough so we can titrate the dose very carefully get these people to where they
need to be with a once daily dose and what's the status of initiating a study and what might that look like in terms of timeline to data something we need to
discuss with the FDA the process has started as we've said we expect to start the process as we speak and start a trial later this year we keep you posted
on study design as we get more clarity from the FDA and just in general what about lifecycle extension strategy for Vicat XR is that something that you all continue to think about and could we get an update on that front anytime soon we
continue to think about it there are some interesting options and yes you
should think of an update in the coming quarter great so maybe to close out I'll ask you what you believe is the most underappreciated aspect of this Leno
story by investors right now I think you have to look at the market you have to look at the fact that this is not when we talk about patient numbers these are not prevalent estimates these are real patients so there is a very meaningful market out there each patient is very valuable and we don't need a very high penetration of this town to be a very meaningful drug but it's anything you
want to add I would just say that you know there's a high unmet need similar to the numbers but I think we've been very pleased with the interaction with the advocacy organizations in the community and they're very receptive and and just very appreciative of the approach that we've taken. And I think that that tightness with the community will continue the momentum that we have.
Wonderful. Anish, Meredith, thank you very much for your time.
Thank you. Thank you, Tyler.