Good morning. My name is Ashley, and I will be your conference operator today. At this time, I would like to welcome everyone to Harmony Biosciences Phase III IN TUNE Study Top Line Results conference call. All participant lines have been placed on mute to prevent any background noise. After the speaker's remarks, there will be a question and answer session. If you would like to ask a question at that time, please press star one on your telephone keypad. Please be advised that today's conference call may be recorded. Lastly, if you should require operator assistance, please press star zero. I will now turn the call over to Luis Sanay, Head of Investor Relations. Please go ahead.
Thank you, operator. Good morning, everyone, and thank you for joining us today to discuss the top line results from the Phase III IN TUNE Study. Our speakers on today's call are Dr. Jeffrey Dayno, President and CEO, and Dr. Kumar Budur, Chief Medical Officer. Following our prepared remarks, Sandip Kapadia, Chief Financial Officer and Chief Administrative Officer, will also be available for the Q&A portion of the call. During today's call, we will be making forward-looking statements which are based on our current expectations and beliefs. These statements are subject to certain risks and uncertainties. Our actual results may differ materially, and we undertake no obligation to update these statements even if circumstances change. We encourage you to consult the risk factors referenced in our SEC filings for additional details. I would now like to turn the call over to Dr. Jeffrey Dayno. Jeff?
Thank you, Luis, and good morning, everyone. I also want to thank you for joining our call this morning and for your interest in Harmony and the top-line results from the IN TUNE Study. First, I would like to take a moment to thank all the patients and their families and the clinical investigators and their staff who participated in the IN TUNE Study, along with all my colleagues at Harmony, who oversaw the conduct of this trial and supported the patients and clinicians who were involved in the trial. Based on our initial read of the top-line data from the IN TUNE Study, we are encouraged by the magnitude of the response that patients experienced in the initial open label treatment period and by the totality of the data thus far, that clearly shows patients with IH experienced a positive clinical effect with pitolisant.
In fact, almost 90% of the patients who completed the trial elected to continue into the long-term extension trial. Although the separation between pitolisant and placebo did not reach statistical significance on the primary endpoint during the randomized withdrawal phase of the trial, there was a positive trend for pitolisant on this endpoint, as well as many of the pre-specified secondary endpoints, including the Idiopathic Hypersomnia Severity Scale and the Sleep Inertia Questionnaire. As you know, the IN TUNE Study completed enrollment nine months ahead of plan, reflecting the significant interest from both the patient and healthcare professional communities. We also received orphan drug designation for pitolisant for the treatment of idiopathic hypersomnia, reflecting the FDA's interest in the potential utility of pitolisant in patients with IH.
Based on our careful listening to the IH community, we know that they have a strong desire for a non-scheduled treatment option to treat their IH symptoms. We remain committed to the IH patient community and pursuing an indication for pitolisant in IH. At this point, we will continue our review of the full data set, which will inform the path forward. We believe that based on the totality of the evidence that we have seen thus far, along with pitolisant receiving orphan drug designation for IH, there is an opportunity for us to work with the FDA on a path forward for pitolisant in idiopathic hypersomnia. Once we have completed our review of all the data, we look forward to engaging with the agency. I will now turn the call over to Dr. Kumar Budur, our Chief Medical Officer, to provide additional details on the IN TUNE Study.
Kumar?
Thank you, Jeff. Good morning, everyone, and thank you for joining the call. I'd like to echo Jeff's comments in thanking those who participated in the IN TUNE Study, and I'm also encouraged with the totality of the data that clearly showed patients experienced a positive clinical benefit with pitolisant. I would like to start my comments with a brief overview of the study design. The IN TUNE Study was a placebo-controlled, double-blind, randomized, withdrawal trial conducted in adult patients with idiopathic hypersomnia at 52 clinical trial sites across the U.S. The primary objective was to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness in patients with idiopathic hypersomnia. The secondary objectives were to assess the impact of pitolisant on other important aspects of idiopathic hypersomnia, such as idiopathic hypersomnia severity, sleep inertia, daytime functioning, and cognitive performance.
Other outcome measures include patient impression of overall change in symptoms and investigator assessment of overall disease severity. The study itself consisted of two periods. An eight-week open-label period consisting of three weeks of dose titration, three weeks of dose optimization, and two weeks of stable dose period, followed by a four-week double-blind randomized withdrawal period to assess the primary, key secondary, and other endpoints. During the open-label period, all patients were treated with pitolisant for eight weeks, and 83% of the patients, that's 83% of the patients who completed the eight-week open-label period, were considered as treatment responders, as defined by three or higher point improvement in Epworth Sleepiness Scale. A total of 139 patients were randomized in the double-blind randomized withdrawal phase, where half of them continued to receive pitolisant, while the other half received placebo.
All efficacy assessments were measured from the end of stable dose period to end of randomized withdrawal period and analyzed to compare the differences between the pitolisant group and the placebo group. Now, moving on to the data. We are very encouraged by the magnitude of response seen in the initial open label treatment period, where 83% of the patients completing this period responded with an average of 9.4 points improvement in the Epworth Sleepiness Scale. A positive trend favoring pitolisant was observed during the 4-week double-blind randomized withdrawal period. However, no statistically significant difference was observed between pitolisant and placebo group on the primary endpoint of ESS. Positive trends favoring pitolisant were also observed across all pre-specified endpoints, including the Idiopathic Hypersomnia Severity Scale, which approached statistical significance, as well as other endpoints, including PROMIS-SRI, FOSQ-10, and Sleep Inertia questionnaires.
Equally encouraging is the number of patients, that's almost 90%, almost 90% who completed the randomized withdrawal period, elected to continue into the 12-month long-term extension study, which is currently ongoing. Importantly, the safety and tolerability profile of pitolisant was consistent with the established safety profile of pitolisant, and no new safety signals were observed in adult patients with idiopathic hypersomnia. As Dr. Jeffrey Dayno mentioned earlier, we remain committed to the idiopathic hypersomnia patient community, and we understand their strong desire for a non-scheduled treatment option for idiopathic hypersomnia. Following a thorough review of the full data set, we will work closely with the FDA to discuss next steps and a path forward for pitolisant in idiopathic hypersomnia. We look forward to sharing additional information in the future.
In closing, I would like to thank all the patients and their family members who participated in the study, as well as the clinical investigators and site personnel for their efforts and commitment in helping us advance this development program. I would also like to thank the patient advocacy group for their relentless efforts on behalf of patients with idiopathic hypersomnia. I now turn the call over to the operator to start the Q&A session. Operator?
Thank you. At this time, if you would like to ask a question, please press star one on your telephone keypad. If you wish to remove yourself from the queue, may you do so by pressing star two. We remind you to please pick up your handset and please limit yourself to one question and one follow-up question. We'll take our first question from Charles Duncan with Cantor Fitzgerald. Please go ahead.
Hey, good morning, gentlemen. Thanks for taking my question. Congrats on the responder rate, but the randomized withdrawal makes this a little bit more complex to interpret. So I guess I'm wondering, what rate of relapse had you assumed in the randomized withdrawal portion? And in terms of next steps, could you imagine conducting a new trial with a parallel group design, or give us a little bit of color on the timing of your discussions with the agency and path forward? Thanks.
Sure, Charles. Good morning, Charles. Thanks for the question. With regards to... Yeah, I think that, you know, the initial, we were very pleased and encouraged by, you know, the magnitude of the response, as you mentioned, you know, 83%, you know, as well as the, the range of response, almost a 10-point improvement on the Epworth Scale in the open label phase. As for what we, you know, have seen initially, and it's early in the randomized withdrawal phase as we, you know, keep sort of, going through the data, the placebo response group, and, you know, the sort of the worsening of about four points, was not, consistent with the expectation, of, you know, what we thought in that, you know, in that group.
So the, you know, the dynamic during the four-week randomized withdrawal phase is obviously what we are looking at much more closely as we go through, you know, the full data set. And I think that, you know, that—based on that, it's really, it's, it's too early to... We are just going through the full data set, and as for... you know, next steps, you know, with the agency, we'll provide an update on that, you know, after the full data analysis. I think we're encouraged, you know, with regards to what we've seen thus far in the data, you know, with regards to the, the robust effect in the, the open label phase, and, you know, up to 90% of patients electing to go into the long-term extension study.
So I think what it tells us is a positive clinical effect overall in this patient population that we've seen. And along with pitolisant receiving Orphan Drug Designation by the FDA, you know, we're encouraged that we'll have a step forward, and obviously, we'll provide an update on that at that time.
Okay, that's helpful. If I may, one follow-up, and that is regarding that open label extension study.
Phase of the study. I'm intrigued to, about what, not only the interest in rolling over, but also the persistence. First of all, with regard to the control patients in the randomized withdrawal, are they given an opportunity to restart pitolisant? Secondarily, what have you seen in terms of persistence within the open label extension for those patients currently on pitolisant? Thanks.
Yes, Charles, great question. I'll turn that over to Kumar to, you know, respond to that one.
Yeah. Hey, good morning, Charles. Thanks for the question. The long-term extension study was always in the plan, along with the main study. So all patients who completed the randomized withdrawal period were offered an opportunity to participate in the long-term extension study. And as I mentioned earlier, almost 90% of the patients elected to participate in the long-term extension study. The study is ongoing, and we have seen very little attrition in the long-term extension study. We are collecting safety and efficacy data from the long-term study as well, and those data will be available in due course of time.
I'm sorry to be dense, but the patients, I guess all patients were, had an opportunity to continue on the pitolisant in the long-term extension, even if they were on the, you know, call it control or placebo arm in the randomized withdrawal?
Correct, Charles. So whoever completed the randomized withdrawal portion of the study, whether they were on placebo or on pitolisant, because when they complete the study, we wouldn't know what they were taking because the study was still ongoing at that time. They were given an opportunity to participate in the long-term extension study, and they started on pitolisant at 8.9, titrated to 17.8, and 35.6, based on the tolerability and the efficacy response.
Okay, very good. Look forward to more data. Thanks for taking my questions.
Thanks, Charles.
Thank you. We will take our next question from François Brisebois with Oppenheimer. Please go ahead.
Hi. Thanks for taking the question. Just, so I was just wondering, in terms of the randomized withdrawal, can you just help us understand maybe the thought process behind using that study design? Thanks.
Yes, good morning, Frank. So I think that, you know, with regards to the randomized withdrawal design, with idiopathic hypersomnia being a sort of adjacency to narcolepsy, you know, the thinking is, once the efficacy is established, you know, has been established in, with pitolisant and narcolepsy, then a randomized withdrawal design is available with regards to an enriched design, you know, what is thought to be an enriched design, you know, looking at maintenance of effect. Obviously, I think as you're aware, there's, you know, there's precedent with IH in this space, you know, based on, on the trial that, you know, Xywav conducted and regulatory sort of precedence there.
The thinking was, you know, with a randomized withdrawal and enriched design, it would give us the opportunity to, you know, demonstrate in open label the effect there, and then, you know, the benefit with regards to going forward. You know, what... As I mentioned before, given the mechanism of action, you know, of pitolisant, which is, you know, different than some of the other agents, you know, in this class, the way that we saw in the randomized withdrawal phase in the placebo group did not have, you know, as much of the worsening over that time as, you know, as was expected.
And we're, obviously, we're looking into that more closely in terms of that dynamic, and, you know, why that was the case. But, you know, the overall thinking was an enriched design, you know, opportunity to assess, you know, the efficacy and safety of pitolisant in this patient population. Kumar, any additional thoughts?
No, I think you covered everything, Jeff. And also, the minimizing the number of patients who are on placebo, and to the extent they'll be on placebo, that also played a role into the randomized withdrawal study design. As we mentioned earlier, it's just that the response rate in the open label period, 83%, not only the response rate, but the magnitude of response of 9.4% from baseline was pretty impressive. But in the randomized withdrawal period, we are looking into the factors that could have potentially affected the rate at which placebo declines. So we are evaluating those data further.
Okay. If I could just sneak in a follow-up here. Are you guys ready to discuss hypotheticals of what could have happened to the placebo arm that maybe didn't happen? You know, we talked about other trials, maybe in Xywav or whatnot. Are you guys just going to look through the data more before you're ready to discuss maybe hypotheticals of the placebo response? Just on that back, you know, with the FDA open, how important in a disease like this with, you know, with the treatment options that are out there or the treatment option that's out there, is open label data, you think, to the FDA? Thank you.
Sure, Frank. I think that, with regards to your question, you know, it's early and I don't want to speculate, but some of the thinking in the design, in the randomized withdrawal period, we actually extended to a full four weeks in the randomized withdrawal period. The thinking of that was we knew that the mechanism of action, pitolisant, sort of being different, obviously the other study conducted with Xywav and Nitazoxanide, the mechanism of action is different with regards to histamine modulation.
And from a pharmacodynamic perspective, you know, the potential, you know, with regards to patients coming off of active drugs, you know, pitolisant in the open label phase, you know, how long, it would take with regards to, experiencing, you know, the placebo response and washing out. So that, that's some, you know, early thinking that we are digging into the data, looking at, you know, the trends over that four-week period.
With regards to open label data, you know, I think that as we talked about, you know, the totality of the data and, you know, the trends not only in the primary endpoint but the, the key secondary outcomes, you know, namely the idiopathic hypersomnia severity scale that approach significance, along with the, you know, the overall benefit-risk profile pitolisant and as a non-scheduled treatment option. So, after review of all the data and all these factors, you know, we will work with the agency. And, and I think, you know, we're, we're optimistic in terms of identifying a path forward, and we're committed to, you know, pitolisant in, in the IH patient population and pursuing the indication.
Thank you.
Thanks, Frank.
Thank you. We'll take our next question from David Amsellem with Piper Sandler. Please go ahead.
Thanks. So I just wanted to switch gears a little bit and get your thoughts on commercial implications here, given the overlap between NT2 and IH in terms of how patients present and symptoms. So do you think that there is the potential that this could that physicians could interpret the data in a way that perhaps has them putting the brakes on further usage or significant usage in the NT2 subgroup? And just how do you think about that from a commercial perspective to the extent that there is, you know, any ill effects? Thanks.
Yeah. Good morning, David. Thanks for the question. I mean, I think at this point, you know, IH is a distinct diagnosis with regards to, you know, diagnostic, you know, codes, first of all, differentiating IH from NT2, and then, you know, the diagnostic criteria, you know, with regards to IH, you know, versus NT2, based on, you know, the ICSD-3, you know, criteria. So I think that and with regards to, in a clinical trial, part of the inclusion, exclusion criteria. So, you know, that the initial thinking is the populations are, you know, distinct. And with regards to, you know, the overall response and the, you know, the meaning of the data, it's obviously still early as we explore the full data set.
So, you know, we're not at this point. This is an idiopathic hypersomnia patient population, and I think I can have Kumar comment with regards to, you know, the study population that we were investigating, aside from, you know, in terms of the commercial impact.
Thank you, Jeff. Thank you, David, for the question. Good morning. Yes, Jeff mentioned idiopathic hypersomnia is a distinct ICSD-3 diagnostic category with a clear characteristic feature, and those were our inclusion, exclusion criteria in the clinical trial. If you look at the baseline characteristics of our study, these are the patients who had a diagnosis of idiopathic hypersomnia, and any NT1 or NT2 patients were clearly excluded from the study. Even in this patient population, granted that the primary endpoint did not separate during the randomized withdrawal period, the totality of the data that I alluded to earlier in terms of the open label period, where 83% of the patients showed response, and the response was defined as greater than or equal to three points improvement in ESS score.
Which, by the way, is larger than what ASM guidelines suggest, which is a two-point change, is considered clinically meaningful. Also the magnitude of response, which is 9.4 points from baseline to the end of open-label period, is pretty impressive. Also in the randomized withdrawal period, as Jeff alluded to earlier, Idiopathic Hypersomnia Severity Scale, which actually measures all aspects of idiopathic hypersomnia. It approached almost statistical significance, and all the other endpoints, like PROMIS, FRI, FOSS, Sleep Inertia Questionnaire, they all trended in a favorable direction to pitolisant, and of course, the long-term extension study, where we have almost 90% of the patients electing to participate in that study.
The totality of the data in idiopathic hypersomnia patients is encouraging, and we are evaluating this data further, especially in the randomized withdrawal period, to see, to basically better understand the data.
Yeah,[crosstalk] David. Yeah, go ahead, David.
Yeah. Yeah, if I may sneak in a follow-up. I mean, and I might have missed this. Can you comment on background medications and how that could have been a confounding factor here?
Yes, sure, David. So I think that patients were allowed in the trial on background medications as long as their baseline level of sleepiness you know met the inclusion/exclusion criteria. So that is you know an important follow-on analysis that you know is being conducted along with others with regards to you know impact of other potential confounders.
You know, to your original question, I think that, you know, in terms of, you know, real-world treatment and real-world management, you know, we're encouraged with regards to in the open label phase. We've got, you know, the magnitude of response that Kumar shared, you know, 83% responders at close to a 10-point improvement in sort of open label, real-world dosing, you know, in this patient population, as well as the intrigue of patients up around 90% electing to go into the long-term extension. You know, with regards to a clinical effect outside of the four-week randomized withdrawal phase that we're obviously digging in real hard to the full data set. You know, we're encouraged by that clinical, you know, response.
Whether it's patients with IH or overlap with NT2, I think that, you know, that's our current, you know, sort of position on this.
Okay. Appreciate it. Thank you.
Thank you. We'll take our next question from Greg Suvannavejh with Mizuho. Please go ahead.
Hey, thanks for taking my questions. I was wondering, I didn't, I think I didn't see a p-value included in the press release. I was wondering if you could share what the p-value was, and also if you could share what the exact placebo response rate, so we can kind of assess what the delta was. And, lastly, a question around if you have any additional data cuts from this study before you have a chance to meet with FDA. Thanks.
Sure, Greg. Good morning. Kumar, would you like to-
Right.
Respond to that?
Hey, good morning, Greg, and thanks for the question. We saw worsening of about 4.4 points in the placebo group during the randomized withdrawal period, and pitolisant group changed by about 3.3 points, and the p-value was 0.22. As mentioned earlier during the call, we are evaluating this data further, as there could be some outliers that might have affected the data. Although the primary endpoint did not make statistical significance, as I mentioned earlier, Greg, the numerical difference was in favor of pitolisant, not just on the primary endpoint, but also on all the other endpoints that we evaluated in this study, including Idiopathic Hypersomnia Severity Scale, which almost approached statistical significance at 0.06 from SRI, Sleep Inertia Questionnaire, FOSS-10, and others.
So we continue to evaluate the data, and in terms of your other question, other data cuts, it's too early. We just received the top-line data. We'll do a deeper dive into all the data sets and chart our next course of action.
Yeah. And Greg, we will, you know, we will do that in terms of the further analyses, and, you know, further cuts of the data, if you will, before. That will inform our path forward with the agency. So that'll be conducted before we engage with the agency.
Thank you.
Thank you. We will take our next question from Danielle Brill with Raymond James. Please go ahead.
Hey, guys, this is Alex under Danielle. Thanks for taking our question. So we're just kind of curious, looking back, you know, given enrollment completed nine months ahead of schedule, is it possible that they were not enrolling patients properly diagnosed with IH? Any indication of study conduct issue?
Good morning, Alex. Thanks for the question. There is no indication at this point of any study conduct issues. And I think that I'll, you know, allow Kumar to expand on that, but no indication of any, you know, problems with the conduct of the trial. And again, in terms of inclusion, exclusion and ICSD-3 criteria, in terms of patient diagnosed with, you know, with IH, you know, that's how the trial was conducted. Kumar?
Sure. Thanks, Jeff. Good morning, and thanks for the question. Yes, there is no reason to believe that the patients enrolled in the study were inappropriate patients because the inclusion, exclusion criteria were very rigorous. We kept a very close eye on the study conduct. And, in fact, I mean, we still believe that the rapid enrollment is really the result of excitement from the patient community and from the clinicians to try a novel mechanism of action, like pitolisant, in patients with idiopathic hypersomnia. And, the data, to some extent, reflects, not to some extent, to a large extent, reflects that the response we saw in the open label phase, not just the response rate, but also the magnitude of response, and also the fact that almost 90% of the patients elected to continue in the long-term extension study.
There is very little attrition in our ongoing long-term extension study.
Great. Thanks so much.
Thanks, Alex.
Thank you. We will take our next question from Jason Gerberry with Bank of America. Please go ahead.
Hey, guys. Good morning. Just wanted to confirm, did I hear that the WAKIX arm and the randomized withdrawal worsened by 3.3 points? Just wanted to clarify that because I guess with the Xywav trial, patients remained stable during randomized withdrawal while on Xywav. So wondering if the pitolisant arm didn't perform as you were expecting during the randomized withdrawal phase. And have you looked at how the data cut at two weeks versus four weeks into the randomized withdrawal? I know that Jazz also had a shorter randomized withdrawal period, so wondering to what extent if you had kind of conducted the trial similar to Jazz, how that might have produced a different result.
Thanks for the question, Jason. Kumar, you want to, sure, expand on it?
Hey, good morning, Jason. Thanks for the question. Yes, as I mentioned earlier, the placebo worsened by about 4.4 points during the randomized withdrawal period, and we also saw a change in the pitolisant arm, where we saw a change of about 3.3 points, which is slightly larger than we anticipated. With the Xywav study, the patients either remained stable or they worsened by about 1-2 points in their randomized withdrawal phase. So we are evaluating this data further to see why we did not see a larger drop in the placebo group. And also, we are evaluating the pitolisant arm as well, to see if any of the outliers might have influenced some of these changes. Too early to comment on any of these things. It's just to see the top-line data.
We will be looking deeper into the data to understand this phenomenon and see if there is anything that could explain this phenomenon. But also important to note, again, as I mentioned earlier, is the favorable trend that we saw on all the other endpoints, including idiopathic hypersomnia severity scale, sleep inertia questionnaire, FOSS-10, PROMIS-SRI and other things that we measured in this study.
Yeah. And Jason, with regards to your second question on the two-week data point, as Kumar said, it's early, but let me just shed a little light on what we've seen so far, and it's early. We actually... The trend in that period of four-week randomized withdrawal, the separation between the pitolisant and placebo actually, you know, begins, you know, after the two-week period. Which is consistent with, you know, the understanding with regards to the mechanism of action, working through histamine modulation. So pharmacodynamically different than, you know, how an oxybate, you know, product, you know, works, where there is more a rapid decrement. And obviously, if you look at the publication on the Xywav trial, you see that pattern.
So we're looking much more closely at this aspect, where the separation is beginning after the two-week point, and continuing. And we took it out to four weeks, and, you know, we will look closely, you know, in terms of that part of the design, explaining. Because we see clinical effect upfront, in the open label, you know, a robust effect, an interest of patients to go into long-term extension, and we need to capture that four-week window and understand it better.
If I could just squeeze one quick follow-up then. So can you confirm that, you know, as you engage with FDA, under no scenarios, would you be doing another full-fledged phase III with WAKIX and IH? That if you were to kind of contemplate-
... further clinical evaluation in this area, it would be with one of your next generation pitolisants?
Too early to comment on that, Jason. Too early to comment with regards to what the opportunities may be, and you know, how quickly we'd be able to execute on them, so it's too early to comment on that.
Okay. Thanks.
Thank you.
Thank you. We will take our next question from Corinne Jenkins with Goldman Sachs. Please go ahead.
Good morning. I was hoping you could help us understand how you're thinking about, you know, what you saw in that drug arm during the randomized withdrawal, particularly as it relates to the durability of the drug's activity or some sort of, like, issue with tachyphylaxis. Perhaps you could draw on your experience in the narcolepsy population to help us understand this better.
Yeah, sure, Corinne. Good morning. So with regards to, that's what we are looking at more closely. So first of all, from all the narcolepsy data clinical experience, there's no evidence of tachyphylaxis with pitolisant. And I, I think that's been demonstrated pretty clearly from the full development program of pitolisant in narcolepsy. And, and, you know, and we didn't expect that in this study. With regards to movement, on the ESS score, you know, where at the end of the open label period, and you go into, you know, the randomized withdrawal phase. And as we've seen also in the Xywav trial, there, you know, there is some movement kind of on worsening on, on the ESS score scale initially, and then, you know, that either stabilizes or can continue, you know, to get worse.
The reason for that is when you have a robust response in the open label phase, and in this trial, close to 10-point improvement, then you're improving patients down to a level where many of them is, you know, quote, normalized in terms of level of sleepiness. There is the potential for movement up and down around that endpoint in both the active treatment group and the placebo. The dynamic that I just, you know, explained within how quickly the decrement or worsening in the placebo arm and what was happening in the pitolisant arm in that four-week window is what we're looking at very closely.
Yeah, and also-
Good morning, Corinne. Thank you for the question. Also we have the long-term extension study. As mentioned, almost 90% of the patients elected to participate, and there is very little attrition in that study, and we are looking at both safety and efficacy data that will provide us information on the maintenance of effect of pitolisant in patients with idiopathic hypersomnia.
As you think about potential conversations with regulators, are you waiting on additional data from that open label extension, or do you plan to go to them kind of as soon as you can get on the schedule?
I mean, it is too early to comment on that. We are still evaluating the data. We will also look at the data from the long-term extension study, but more importantly, we will look at exactly what happened during the randomized withdrawal phase and then chart our next course of action.
Yeah, but I think, yeah, I think that there will be, you know, potentially we'll have the opportunity for some additional data from the long-term extension to share with the agency and inform, you know, as Kumar said, you know, the maintenance of response for pitolisant.
Okay. Thank you.
Mm-hmm. Thank you.
Thank you. We will take our last question from Ami Fadia with Needham. Please go ahead.
Hi, good morning. Thanks for taking my question. I have two quick ones. Firstly, how much of a follow-up period have you had in the long-term extension portion so far? And can you give us any color on how the ESS scores have evolved as patients have been in that portion of the study?
Morning, Ami. Thank you for your questions. So I'll turn it over to Kumar with regards to the long-term extension that, you know, was part of the program plan from the beginning, patients continuing on into them, and with regards to when we anticipate further, you know, data cuts from that.
Hey, good morning, Ami. Thanks for the question. Off the top of my head, I cannot say exactly how many patients have completed what duration of the study, but majority of the patients are, in fact, 119 patients elected to participate in the long-term extension study. We are collecting the safety and efficacy data from the patient population, and we are going to have a data cut from that study relatively soon. Once we have a data cut, we will get to know the efficacy and the safety data from the long-term extension. At this moment, we don't have that information.
Okay, great. Thank you. And then just a second question. Perhaps, you know, I, I suppose you're going to need to look through the data more closely to understand why the randomized withdrawal portion did not hit and what might be the appropriate next steps. But just stepping back, could you comment on whether you're committed to pursuing IH as an indication, whether it is with pitolisant or a next generation version of it?
Sure, Ami. Yes, sure. Yeah, we're absolutely committed to pursuing IH as an indication. And again, I think that, you know, we're very encouraged by the signals that, you know, we have seen from this trial. And, you know, beginning with the overall clinical effect and magnitude of effect, you know, that we've been describing. There is more work to be done in understanding the dynamic around the, you know, the 4-week randomized withdrawal period. We look forward to more data coming out of the long-term extension trial. And we will, you know, we will put that all together, and, you know, kind of build our case with regards to engagement with the agency in, you know, in pursuing next steps and an indication for pitolisant in patients with IH.
So we remain committed to that. We, you know, we think that given the product profile, given the non-scheduled status, given the interest that we've seen in this trial and what we've heard from the IH patient community, you know, we will persevere and pursue that indication.
Thank you.
Thank you. I'm showing no further questions. I would now like to turn the call back over to Jeff Dayno for closing remarks.
Thank you, Ashley, and thanks, everyone, for joining our call today and for your interest in Harmony and the results from the IN TUNE study. As I said, we remain committed to the IH patient community and pursuing an indication for pitolisant in patients with IH. As we've shared, we'll complete our review of the full data set and provide an update at that time. Thanks, everyone, and have a great day.
Thank you, and this does conclude today's program. Thank you for your participation. You may now disconnect and have a wonderful day.