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spk03: Ladies and gentlemen, thank you for standing by. Our conference will begin momentarily. Once again, thank you for standing by. Our conference will begin momentarily.
spk00: Thank you. Thank you.
spk03: Greetings and welcome to the Avidel Pharmaceuticals first quarter 2021 earnings call. At this time, all participants are in a listen-only mode. A question and answer session will follow the formal presentation. If anyone should require operator assistance during the conference, please press star zero on your telephone keypad. As a reminder, this conference is being recorded. It is now my pleasure to introduce Tom McHugh, Chief Financial Officer Thank you, you may begin. Good morning, and thank you for joining us on our conference call.
spk05: This morning, we share a press release providing a corporate update and financial results for the quarter-ended March 31st, 2021. The release can be accessed on our website, www.avido.com. As a reminder, before we begin, the following presentation includes several matters that constitute forward-looking statements within the meaning of the Privacy Securities Litigation Reform Act of 1995. Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those contemplated in such forward-looking statements. These risks include risks that products in the development stage may not achieve scientific objectives or milestones or meet stringent regulatory requirements, uncertainties regarding market entry and acceptance of products, and the impact of competitive products and pricing. These and other risks are described more fully in Avidel's Public Filings and Exchange Act, including the Form 10-K for the year ended December 31, 2020, which was filed on March 9, 2021, and subsequent SEC filings. Except as required by law, Avidel undertakes no obligation to update or revise any forward-looking statements contained in this presentation to reflect new information, future events, or otherwise. On the call with me today are Greg Divis, our Chief Executive Officer, Richard Kim, our Chief Commercial Officer, and Dr. Jennifer Gudeman, our VP of Medical and Clinical Affairs. At this time, I'll turn the call over to Greg.
spk12: Thank you, Tom. Good morning, everyone, and thank you for joining us on our first quarter 2021 conference call. I will begin by providing an update on our business, highlighting the significant progress we continue to make towards achieving FDA approval of and preparation for the potential commercialization of once-nightly FT218. If approved, FT218 will be the first and only once-nightly Oxibate treatment available for people suffering from the debilitating orphan disease of narcolepsy. I will then turn the call over to Jennifer, who will offer an overview of the progress we've made with our medical affairs and scientific communication plans for FT218. Richard will then provide an update on our commercialization and launch planning as we move closer to the PDUFA date and a potential approval. And finally, Tom will provide a review of the financial results for the quarter, and we will conclude with a Q&A session. With that as an outline for the call, let's get started. As we are now nearly halfway into 2021, I can say that it has already been a significant year for Avidel with several key regulatory, clinical, and launch readiness milestones achieved, as we continue to rapidly advance our investigational once-nightly FT218 program, most notably being the acceptance of the NDA filing with an assigned PDUFA date of October 15th. Considering this progress and the momentum we are experiencing across the entire FT218 program, from NDA execution to data dissemination and launch readiness, the second half of 2021 is lining up to be both exciting and potentially historic for Avidel and for people suffering from narcolepsy. In this regard, the progress we are making on the regulatory front is arguably the most relevant and important near-term milestone for Avidel. As we have said in the past, as it relates to our public disclosures on the NDA process, and specifically our regulatory filing strategy, we've said no news is good news. And as such, I am pleased to report, as it relates to our regulatory filing strategy perspective, we have no news to share. As we are now approaching the midpoint of the review timeline for the NDA, and based on the actual review to date, we still have not been asked by the agency to certify paragraph four against any orange book listed patents, and we don't believe, based on the data and regulatory strategy of our FT218 NDA submission, there is any basis to request such a certification. Our team over the last two years has executed this program, and specifically this NDA, exceptionally well, and we remain highly confident in our regulatory filing strategy as we head toward our October 15th PDUFA date. Furthermore, as Jennifer will cover, the data from the Reston study, her key secondary endpoints, recently presented at the American Academy of Neurology Conference, confirms the clinical profile and promise of our investigational once-nightly FT218. We look forward to even more data being presented and disseminated throughout the scientific community during the balance of 2021. Lastly, this promise of FT218 can only be realized through both the successful execution of our NDA and the subsequent commercialization of FT218, if approved. For the past few months, we've accelerated our launch planning and execution with the appointment of Richard Kim as our Chief Commercial Officer, And Richard has quickly immersed himself into our plans, continues to actively engage with key stakeholders, and is building a team of exceptional and proven industry professionals that is quickly advancing our level of readiness across multiple work streams. The caliber of the people who are raising their hand, wanting to join us in this journey, is some of the best I've seen in my over 30 years of industry experience. which we believe is reflective of the opportunity and the value that Once Nightly FT218 can deliver. As many of you know, it was just a little more than 24 months ago when we decided to focus our business on the prospects and the potential of our investigational Once Nightly FT218. That is exactly what we have done, and our execution on that decision has brought us to where we are today, with a strong balance sheet, having over $200 million of cash on hand to support our launch readiness and a regulatory pathway to a potential approval and future launch. Our team has remained incredibly focused, has executed at the highest level, and is building great momentum across the company and in the narcolepsy community as we seek to establish our investigational once-nightly FT218 as the oxidative choice and disrupt this multibillion-dollar narcolepsy market. So to summarize, the overall key takeaway from today's call is that we remain on track across all aspects of the once-nightly FT218 plan. This includes NDA execution, which continues to advance according to plan, and we remain confident in our regulatory filing strategy as we head toward mid-cycle review. Furthermore, the opportunity for FT218 only improves as we continue to announce new, relevant data and actively engage the medical community while accelerating our overall launch readiness. We look forward to what will be an exciting second half of 2021 and beyond. And with that as the batch up, let's get into some of the details. And for now, I'll turn the call over to Jennifer.
spk09: Thanks, Greg. It's great to be here today to discuss the progress we've made with our scientific communications for FT218. While the Avidel team has known for some time how robust the broader Reston data set is, we are now fully engaged in externalizing these data so that the medical and payor community can also fully appreciate our positive findings with once nightly FT218. Additionally, we have expanded our medical affairs team to further our connectivity in scientific exchange with key opinion leaders. As Greg mentioned, we were excited to present new positive secondary endpoint data at the 2021 American Academy of Neurology Annual Meeting, which was held last month. There are two key takeaways from these secondary data, including, first, FT218 demonstrated significant consolidation of sleep, significant increase in time in deep sleep, and significant decrease in light sleep compared to placebo, for all doses evaluated, six gram, seven and a half gram, and nine gram, beginning by week three. Disturbed nocturnal sleep is a frequent and bothersome complaint of patients living with narcolepsy. While most therapies for narcolepsy address only daytime symptoms, this newly presented sleep data from Reston supports that once nightly FT218, if approved, could address nighttime symptoms of narcolepsy without having to wake up in the middle of the night. Second, FT218 demonstrated a significant improvement in the upward sleepiness scale, a patient-reported outcome, as well as significantly improving patient perceptions of both the quality and the refreshing nature of sleep, also for all doses evaluated. The Epworth Sleepiness Scale is commonly used in clinical practice as it evaluates eight domains to ask patients about their likelihood of dosing off during various activities, such as watching TV or when stopped at a traffic light. At all doses and beginning at week three, which was the first formal evaluation, FT218 significantly improved, that is reduced, the Epworth score. For context, a score of 16 out of 24 is characteristic of narcolepsy. A score of 10 or below is considered normal. At week 13, with the 9 gram dose, the endpoint mean was a score of 10.4, indicating not only market improvement from baseline, but also approaching a normal at-worth score for many of the participants. As a reminder, we have previously shared the top-line data from the pivotal Phase III Reston trial, which reported that FT218 met all three co-primary efficacy endpoints compared to placebo for all three doses evaluated. These results were highly statistically significant with all p-values less than 0.001 and clinically meaningful as assessed by the maintenance of wakefulness tests, clinical global impression improvement, and mean weekly cataplexy attacks. With the new secondary endpoint data presented at AAN, clinicians can now begin to more fully appreciate the totality of the positive results demonstrated with FT218. The upcoming annual sweep congress, which commences in exactly one month, is dedicated to clinicians focused on sleep disorders. As such, I am really looking forward to our expansive data dissemination strategy that we have scheduled. We will be presenting a total of six posters with one oral presentation. These educational assets, along with a newly created mechanism of action video, will be housed in our virtual medical affairs booth as we look to engage with these narcolepsy specialists. Additionally, we are supporting a symposium titled, How Narcolepsy Management is Evolving, which will be comprised of an expert panel discussion conveying three primary points. First, it is often necessary to manage both daytime and nighttime symptoms. Second, the importance of shared decision making and criticality of considering the patient's perspective for treatment of this chronic condition. And third, reviewing the nearly 20 years' worth of immediate release sodium oxidate data showing no signal of increased cardiovascular risk. Turning now to our publication plans. As you would expect, there are numerous publications planned and already in process. We very recently reached an important milestone with the submission of our primary manuscript from the Reston trial with an excellent group of key opinion leaders and investigators as co-authors. I look forward to providing updates on the content and timing of this primary manuscript as well as additional publications in the future. As a final remark, what really excites me is the perspective we hear from key opinion leaders for what FT218 could mean for their patients suffering from this chronic condition. Whether it is working on publications, holding advisory boards, or simply one-on-one meetings, the consistent takeaway from our interactions is that FT-218, if approved, will provide a significant clinical advancement in the treatment of narcolepsy as the only once-nightly Oxibate product. Let me now hand the call over to Richard for an update on the commercial planning activities. Richard, the floor is yours.
spk02: Thanks, Jennifer. And let me say that I am excited to be on the call today and provide an update on our launch preparations for once nightly FT218. During the March earnings call, I shared some of my initial insights and enthusiasm for joining the Avidel team and for the amazing opportunity we have to transform the narcolepsy market. Well, I can say that over the last few months, I've had a chance to validate my initial impressions that, if approved, once nightly, FD218 has the potential to gain market-leading share in the Oxibate class. Previously, I shared from our market research some key insights about unmet needs in the Oxibate market, including that, Almost half of patients report refusing twice-nightly sodium oxibate when offered by a physician, primarily due to the need to take a second dose in the middle of the night, two and a half to four hours later. And almost 60% of patients reported negative treatment experiences. What we see is that despite a time to diagnosis of about eight to 15 years, patients on average are usually initiated with pharmacotherapy around three months post-diagnosis. In addition, patients tend to have their narcofibrosis treatment switched or supplemented relatively quickly, with the average time to modify treatment from first to second line being about a month and a half, and the average time to modify from second to third line treatment being about only two weeks. From this research and other work we have done, the data sheds light on the challenges with twice-nightly oxibate treatment, and the propensity for patients to seek new options when available as they continue to search for new ways to get more control over the daily impact that narcolepsy has on their lives. In the last couple of months, I have also had the opportunity to speak with treating physicians as well as patients living with narcolepsy. Here are a few key insights that continue to stick with me from those conversations. It's hard for someone who does not suffer from narcolepsy to really understand the challenges that those with it go through every day. Like the choices some patients make to not participate in happy life events because of the fear that positive emotions will trigger a major cataplectic event. Like when I heard about a father not attending his daughter's wedding. Narcolepsy can have a profound impact on what most of us would consider regular day-to-day activities. like cooking, driving, or sleeping through the night. One of the amazing patients I spoke with said that since her diagnosis and treatment of stimulants, antidepressants, and twice-nightly oxibate, she has not slept through the night in 21 years. At the end of the day, like most of us, people with narcolepsy are seeking more normalcy in their lives and the opportunity to live more independently. I have also learned that OxyBait therapy can be a game changer for some patients, but also that the challenges of twice nightly therapy go well beyond having to wake up during the middle of the night. I'm so appreciative of the time people spent with me as the conversations have been both emotional and inspirational. And overall, I continue to appreciate just how relentless narcolepsy can be. That's why we will continue to base our decisions on what we learn from patients, physicians, and payers, and why we will also be relentless in how we bring once-nightly FD218 to the market if approved. Now let me transition to an update on some of our specific launch preparations. Overall, we continue to make significant progress in ensuring we are ready to launch once-nightly FD218, and that we develop and execute programs focused on bringing exceptional clinical value to all of our customers. On the REMS patient hub and distribution front, we've made significant progress choosing our partners to ensure our network is ready and will deliver customer-focused support when HCPs and patients are seeking to get one slightly FD218 if approved. We also continue to focus on gaining more patient insights under the guidance of pre-approval information exchange, which provides the opportunity to engage with payers about once-nightly FD218. In addition, our team keeps advancing on other key launch initiatives, like targeting, field force sizing, data integration, pricing, and messaging. And we will roll out our first corporate narcolepsy campaign to coincide with the Sleep Congress next month as we ramp up our customer engagement activities. To support these and other commercial planning activities, we are also actively growing our launch team, and we have momentum in the marketplace attracting top-tier talent to lead our launch. For example, we have recently made key leadership hires in marketing, patient services and distribution, and insight and data analytics, and are making great progress against our overall hiring plan. It's been really cool to see that our top candidates clearly understand our value proposition and embrace what we are doing to reshape the narcolepsy market for patients. Well, it's been a quick first three months for me, but I am really pleased with our progress in our launch preparations. I am more convinced today that we have a unique opportunity to transform a market, to build a world-class team, to create exceptional clinical value, deliver strong shareholder returns, and most importantly, potentially bring an essential once-nightly treatment option to patients suffering from narcolepsy. I look forward to providing further updates on our progress on future calls. I will now turn the call back over to Tom to provide an overview of our financial results for the quarter. Over to you, Tom.
spk05: Thank you, Richard. and I'm pleased to provide an overview of Avidel's financial results. From a balance sheet perspective, we ended the quarter in a strong cash position with $205 million of cash, cash equivalents, and marketable securities. And as a reminder, in addition to the cash on hand at March 31st, we expect to collect the remaining $8.3 million from the sale of sterile injectable drug portfolio that we sold last year on June 30th of 2020. Turning to our income statement, As a result of the sale of the sterile injectable products, we did not record any revenue in the quarter ended March 31, 2021, and we also did not record any expense for cost of products and tangible asset amortization and changes in fair value of contingent consideration. The total of our R&D and SG&A expenses in the first quarter of 2021 were $14.8 million compared to $13.4 million in the prior year. As our preparations for the launch of FT218 continue to accelerate, we expect that our operating expenses will increase quarter over quarter during the remainder of the year and be more heavily weighted in the second half of 2021 as we approach the PDUFA date in Q4. With regards to R&D, expenses decreased 1.6 million year over year to 3.9 million in the first quarter of 2021 versus 5.5 million in the prior year. The decrease is due primarily to the completion of the Phase III Reston clinical study for FT218, which concluded during the first quarter of 2020. SG&A expenses increased $3.1 million year-over-year to $11 million in the first quarter of 2021 versus $7.9 million in the prior year. The majority of the year-over-year increase is attributable to costs for planning and preparing for the launch of FT218, if approved. Income tax benefit was $2.6 million in the first quarter of 2021 compared to $9.5 million in the prior year. The year-over-year decrease is primarily due to benefits recognized in 2020 from the Coronavirus Aid, Relief, and Economic Security Act. Net loss for the first quarter of 2021 was $13.4 million, or $0.23 per diluted share, compared to a net loss of $0.9 million or two cents per diluted share for the same period in 2020. The increase in net loss and diluted loss per share is primarily the result of the year-over-year decrease in revenue due to the sale of the sterile injectable products. Diluted shares outstanding increased to 58.4 million shares this year versus 41.1 million shares last year. The increase in the number of shares is due primarily to the 190 million of gross proceeds raised from equity issuances during the first half of 2020. Finally, as Greg noted earlier, we are incredibly pleased with our progress to date and the momentum we are carrying into the rest of the year. We believe we're in a strong financial position with 205 million of cash on hand to fund the financial investments needed to complete the NDA review process, compile additional supporting scientific data, position FT218 in the market and continue to ramp up our launch preparations for FT218. Before turning the call back to Greg for closing remarks, we're going to open up the call for Q&A, and I'll now turn the call over to the operators.
spk03: Thank you. We will now be conducting a question and answer session. If you would like to ask a question, please press star 1 on your telephone keypad. A confirmation tone will indicate your line is in the question queue. You may press star two if you would like to remove your question from the queue. For participants using speaker equipment, it may be necessary to pick up your handset before pressing the star keys. We ask that you please limit yourself to one question and one follow-up question. One moment, please, while we poll for your questions. Our first questions come from the line of Paul Matias, but Steve, please proceed with your questions. Great.
spk13: Thanks so much. Appreciate the call and questions. So you guys have said again that no news is good news. I guess, how do you think about certain inflection points in the review? And if there's any kind of, like, I guess this mid-cycle review meeting or some other kind of scheduled interaction where the key issues that are related to certification and possible labeling are going to come up. Just kind of curious if there's anything that you would point to or how we should think about the timelines as it relates to Freedom to Operate. And then separately, I wanted to ask, are you expecting to get orphan drugs to exclusivity? How should we think about that? And if you do, what do you think that means for other once-nightly sodium oxidates that are in the pipeline, even those that might be low sodium? Thanks so much.
spk12: Yeah, thanks, Paul. Appreciate the questions. I think as it relates to the review, I think, you know, our commentary, you know, we've tried to provide a little bit of context here that we're very, you know, again, we haven't been asked to certify. We're very pleased with the progress we've made on this front and really the types of questions or the comments we've received during the review process to date, right? I think the one thing we'll say is that, you know, the FDA doesn't typically notify an applicant when, you know, some portion of the NDA is kind of complete and they've checked the box and moved on, they really just move on. So, you know, as we enter the second half of the review, we'll certainly move to label negotiations and things of that matter that we would expect. I would say that our position remains the same. We certainly are not aware and don't believe there's any basis for certification on our side whatsoever, given the data we've provided, how that is reflected in our label, and our overall regulatory filing strategies. That being said, even if we've been confirmed already or we learn at a later date during the review process that we've effectively navigated through any sort of certification risk, we certainly aren't going to speak on it publicly in advance of the FDA making the first comment publicly in the form of their decision on or around October 15th. That being said, I think we feel really good about where we are, how we've progressed this aspect of the NDA, and where the and where we sit today currently. As it relates to orphan drug, you're correct. We were granted orphan drug designation on the plausible hypothesis that once nightly FT-218 could be clinically superior to the reference product, right? We believe that we provided a robust and complete rationale for our exclusivity request that has been and is part of our NDA review. So we certainly look forward to the FDA's decision on it. It's not something we're relying on for our exclusivity protection, because I'll remind you that we've had a number of intellectual property patents granted already and many more in the queue, so to speak, that we believe will build the appropriate protections for the company. It was the first one to innovate and demonstrate a modified or extended release control GHB-related product. that can work for patients, and we certainly are going to protect that in every way, shape, or form. So as its impact on other potential products that could come in the marketplace, if we're granted Orpin drug, it would be something that they would have to demonstrate that they're clinically superior to our one slightly product is how we think about it. Thanks.
spk13: Thanks, Greg. Appreciate it.
spk03: Our next question has come from the line of David Amsell with Piper Sandler. Please proceed with your questions.
spk06: Hey, thanks. So just a couple. So on the review, I don't know if you can talk about this, but has the REMS portion of the discussion come up, or when in the review does does the REMS piece actually come up? I'll just understand that if you can. And then secondly, on the commercial landscape, I'm sure you're going to get this question an awful lot, but I have to ask it, which is with Jazz converting a significant number of patients over to low sodium, what are your thoughts on getting some of those patients, capturing some of those patients and I guess as part of that, with Jazz's success here, does that mean you have to contract more aggressively in order to try to capture some shares? So how philosophically do you think about that? Thank you.
spk12: Yeah, thanks, David. I'll take the first question, and then Richard, maybe I'll turn it over to you to have any perspective on the commercial question. As it relates to the REMS program, I would say yes, Again, I think for context and background, it is not a topic that the first time we would be engaging the FDA on it would be during the review. It's something we would have obviously, given the criticality of it, engaged with the agency in advance of even our initial submission. I just remind everyone that as a 505 application, it's not subject to any sort of single shared system. And we can, in essence, have our own REMS program from that perspective that will be obviously geared and specific to our own label, you know, should we get approved. In terms of the review process, I would say that, you know, there's little I think we can say specifics around it other than overall I would say we're quite confident that the FDA has been through, you know, all aspects of our NDA. And it wouldn't get accepted if it wasn't in there already and for sure because it's part of our submissions. So I would say generally speaking, whether it's REMS or anything else, we're quite confident the FDA has been through at least initially all aspects of our NDA.
spk02: Yeah, and thanks, Ray. I'll take over the rest of the question from you, David. So, you know, I guess first from our perspective, David, it's always great to see new treatment options come to the market no matter how large or small the benefit is that's added. I think the key thing that we think of the early uptake of mixed salts is that it really shows us, confirms our market research that there, in general, there's a high degree of patient willingness to try new treatments to gain more normalcy in their lives and a fair degree of dissatisfaction in the market. We actually think this bodes well for the value proposition that, you know, for one slightly FE218 is we believe that the majority of patients switching to mixed salt now, they'll continue to demonstrate a propensity to seek new ways to improve their treatment as they go forward. especially with the significant advancement like a once-nightly oxibate coming to the marketplace. And for us, we remind ourselves that sodium was really not much of an issue. It's clearly when there's no other options, it makes sense. But we also know that from the systematic literature review that's been done, that experts in the field have concluded that the sodium and sodium oxibate really doesn't create any additional cardiovascular risk for patients. So at the end of the day for us, the sodium oxibate has demonstrated a lot of great utility over the last couple of years and nearly two decades safety profile. And that's really why we believe that ultimately when given options, narcolepsy patients will also focus on getting more consolidated sleep without the need to take a second dose during the middle of the night. And as far as the payers are concerned, it's always been our position that we're going to fight this battle on our clinical value proposition. We don't want this to turn into convenience or salt play. Having said that, we're clearly going to be very active with the payers. We don't want to get into pricing wars or anything, but we also know that at some point in time in the United States, there usually is some sort of contracting that we will build in. But we are beginning that next level of discussions with payers, and we're looking forward to providing some more updates in the future.
spk06: Great, thanks.
spk03: Our next questions come from the line of Francois Brisbraugh with Oppenheimer. Please proceed with your questions.
spk11: Hey, thanks for taking the questions. In terms of the data at AN, is there anything, you know, some of the data for Zarum is extremely old, but any comparisons here to twice nightly for the extra secondary endpoints, or is it mostly placebo? Any kind of, I know it's difficult to do, but any cross comparisons that are out there?
spk12: Jennifer, want to answer that?
spk09: Yes. Hi. Good morning, Francois. Thank you for your question. You know, my general philosophy is that I think FT218's data should stand on its own. We know that there are inherent challenges whenever we're having any sort of cross-study comparisons, obviously potentially differences in trial design as well as inpatient demographics or clinical characteristics. That being said, I talked a little bit about the Epworth score in my opening remarks, and perhaps that's relevant to highlight. We're extremely pleased with the fact that our end-of-study score with the 9-gram dose was 10.4. As I mentioned, the score of 10 or below is considered normal. And with all of the caveats that I described in terms of limitations with cross-study comparisons, I think it is still relevant to recognize with the twice-nightly sodium oxibate, their end-of-study score with the 9-gram dose was 12. So, you know, as Richard had mentioned in his remarks, Our belief is that patients who have this chronic condition that once they're diagnosed, they're living with for the rest of their life, that their aspiration is returning to normal. And with our F-word scores coupled with the fact that one does not need to wake up in the middle of the night to take that second dose, we believe that that's going to be a very attractive option for patients suffering from this condition.
spk12: Male Speaker 1 Yeah, Frank, and I would just add to Jen's thanks, great comments. As she noted, it's hard to make those comparisons. And I think just to reiterate the point, I think everything we present and all the data we've looked at and the data we're going to share in sleep only continues to just reinforce and support the overall pivotal study results that really, like, you can't quite, our data is compelling, and with an adverse reaction profile that looks exceptionally well, you know, strong as well. So we're just excited about the prospects of how 218 looks and the data it presents, and I think the more we get an opportunity to share this data with the medical community, you can see the more interesting and excited they get about it as they're learning more about us from that perspective.
spk11: That's great. Thank you very much. And just in terms of the education, maybe this is more for Richard. I would assume that, you know, this is a fairly straightforward kind of new product in terms of, you know, comparing it to twice nightly. Is this something when you speak to physicians that you feel like the physician education will be difficult or, you know, at conferences from your interviews, is this something that's pretty straightforward and they get it?
spk02: Frank, I've learned to realize in my life not to take anything for granted. So I think the messaging is straightforward and simple. But, you know, I think the great news is with the work that Jennifer has done is now we have this great scientific foundation to go forward with. And if approved, of course, it'll really give us a great foundation to really have a very clear, simple message. So the good news is, as you know, physicians are very deeply familiar with using Oxibate treatment. We just hope to message and deliver something that's simpler for them and their patients to be able to use. So, yeah, I look forward to getting out there and having more of those conversations when we can, but I think they should be, our goal is to make this a simple communication to build off of their experience that physicians already have.
spk11: Okay, great. And then just all that data that you guys are working on, the extra secondary endpoints, now that it's all filed and accepted, there's nothing here on the NDA that can affect the label, or is this still something that the FDA can look at?
spk12: Yeah, I think when you think about the label, it's predominantly your primary endpoint data that will be most represented in our label. So, any post hoc analysis that we'll do that you'll see at the suite meeting will not be in our label at this stage, but it's additional analysis we think is relevant for the clinical community to understand. you know, in different patient populations as an example that we think is important for as they think about the use of one-sidedly FT218 in the future to have more data to look at and really just to round out the complete clinical profile of FT218. Okay.
spk11: Okay, great. And I guess my point was a little more, you know, from the sales rep's perspective, if it's not in the label, how much can they discuss it? And on that note, have you shared how many reps or when you would hire them?
spk02: Yeah. Absolutely. Great. Sorry. Uh, and Frank yet, you know, as far as the label, I mean, first Jennifer's team is doing a fantastic job in beginning this real, um, push for our clinical data that'll be out there. So, uh, we, we, we really, uh, believe and trust in the work that they continue to do. And as far as the Salesforce is concerned, we haven't made any final decisions yet. Uh, I think we're getting into those stages now where we're really starting to get, uh, zone in some, some of the numbers. We sort of traditionally guide it towards 50 or 60, but I think for us, we're still assessing sort of the face-to-face, the digital communications that will go on. And the other thing for me as well is ultimately we know that this is a relatively concentrated marketplace, as we've spoken about. Around 1,600 physicians make up 80% of the prescription volume and less than 500 make up 50%. So the good news for us is that will give us some flexibility in how we want to go to this marketplace. So those decisions will be coming up in the upcoming months. as we get ready to support the launch of Once Nightly FD218.
spk11: Great. Thank you very much. Thanks, Ryan.
spk03: Thank you. Our next questions come from the line of Mark Goodman with SVB Lyric. Please proceed with your questions.
spk07: Hi. Thanks. This is from Mark. We just had a question on sort of when you're thinking of disclosing other products that you are internally developing that leverage your controlled release technology.
spk12: Yeah, thank you. I think it's a great question because we certainly know how important it is as we move through a potential approval and launch that the what's next question will certainly come about. And I would describe it this way. We've done quite a bit of planning and have even initiated some early work on what we would characterize as what's next beyond FT218. But I don't think we're at a place where we'll discuss specifics at this stage. I mean, we're very focused on executing against the current regulatory and launch readiness strategy, which is very important for us. But I think as you think about it, right, in potential future areas for, you know, expansion, you can think about it in the form of lifecycle management. You can think about, you know, whether it's a pipeline and a product. You can think about our leveraging technology, our technology platform both in sleep or in relevant adjacencies from that standpoint as kind of a couple of legs of the stool, if you will, in terms of how we think about going what's next. But I would say at this stage we remain very focused on 218 while we plan and begin to do a little work on the other aspects of our portfolio, and at the right time we'll come forward and provide some more insights to our shareholders for sure. Thank you.
spk09: Thanks.
spk03: Our next questions come from the line of David Sherman with LifeSide Capital. Please proceed with your questions.
spk04: Hi, guys. I was wondering if you could talk a little bit more about how you're planning to engage with non-prescribers in an effort to expand the existing Oxabate prescriber base. And then I was curious to hear a little bit more about just how you're planning to kind of engage and advertise to consumers down the road if it's approved. Richard, you want to go first?
spk02: Yeah, sure. Thanks for the question, David. So as far as non-prescribers, we know that sort of within any sort of given year, there's around 4,000 to 4,500 physicians who prescribe OxyBates. So we believe that's relatively concentrated. So to your question around non-prescribers, to be very candid, I think the initial thrust of our focus will be on current prescribers of OxyBates who have the experience, but Because of the proposition of a once nightly FE218, where I think you're going is we do believe that there is an opportunity to expand the treater base going forward as well. So, you know, I think what we see is interest has grown in the narcolepsy class with other new novel therapies who have come to the marketplace. So we intend to sort of capture some of this momentum and just, you know, I think also we're not going to be too specific around who's going to be invited to some of our speaker programs going forward as well. And as far as sort of how to sort of connect with a lot of the patients that are out there, we believe that the patient voice is really critically important in the treatment of narcolepsy, as there really aren't, as you know, markers, blood markers or scans that really help assess impact of this disease. So, you know, our commitment is really to sort of focus on getting the appropriate education through the venues that patients seek information today. And we think there's really good opportunities for us to get out there because the narcolepsy community is, in general, pretty active online. So that really, digital communication provides a very focused opportunity for us to reach out and make sure that the proper education information is out there on what we're doing in narcolepsy and upon approval for once-nightly FE218 as well.
spk12: Yeah, David, just one additional comment to Richard's comments around market expansion or prescriber expansion. I want to differentiate those a little bit, only because we believe within the current prescriber audience of these 4,000-plus per year and those who are within that subset who prescribe a lot more, there is a fairly reasonable-sized cohort of patients you know, and every practice is a little bit different, but in aggregate, that are sodium oxibate eligible as defined by those current prescribers who aren't going on sodium oxibate, as Richard described a little earlier. Whether that's because the physician has decided not to treat them or the patient has decided not to go on it, the predominant reason is dosing related. So in that category, we believe there's expansion opportunities within the current prescriber base, which makes it even that much more efficient for us accordingly. Okay, thanks for taking my question.
spk03: Thank you. Our next question has come from the line of Robin Garner with Craig Holland. Please proceed with your question.
spk08: Hi, good morning, and thanks for taking my question. You shared some new patient insights earlier in the call. For context, can you share with us a ballpark of how many patients you've been able to engage during your market research?
spk02: Yeah, thanks, Ram. Patient market research is one of the interesting things that we do here as well. And I would say, in general, what we've seen is we've been able to engage with hundreds of physicians, patients, and payers. We don't always sort of break out the exact numbers of what we've done there, but it's fair to sort of say that it's dozens upon dozens of patients. And then what we've also done is also done individual conversations as well to get more qualitative perspectives. And going forward, we will continue to do this, not only through market research, but our engagement through patient advocacy and other sources as well. So I can say from my perspective, the feedback from the payer work that we've done, if it's in market research, speaking to advocacy groups or individuals, is generally pretty consistent. And I think even though that there are some individual needs of patients out there as well. So hopefully that gives you some of the perspective from what we do in patient research.
spk12: Male Speaker 1 Yeah. And, Robin, I'll just add one more comment to that, to Richard's, and that is, you know, in particular, as time continues to go and the market continues to evolve, we're doing robust research. And it is a combination of both qualitative insights, as Richard described, but statistically and market research-based quant studies that are robust and, you know, appropriately designed and with demographics that are meant to represent, you know, our target audience and geographically dispersed accordingly. So we try to get, you know, quant work that's large to give us the right, you know, right views and not make decisions based on very, very small sample sizes, but on large, robust work products.
spk08: Okay, thank you for that. And then just lastly, will you be presenting any new data points at the SEAP Congress?
spk12: So just to be clear, are we presenting any new data at the Sleep Congress is what you asked? Yes, that's right. Yeah, okay. Jen, you want to answer that?
spk09: Yes, I do. Hi, Robin. Thank you for your question. So the new data that is being presented are post hoc analyses, and this is data that we think is really relevant for clinicians to understand. So the abstracts have been published for the SLEEP Congress, and our three post hocs are looking at efficacy stratified by subgroups, so NT1 and NT2. looking at efficacy by the subgroups of stimulant use or no stimulant use, and also examining the weight loss that occurred with FT218. And as I mentioned in my opening remarks, the consistency of benefit that is seen with FT218 underscores just how robust these findings are and how consistent the efficacy is. And so we look forward to having that full presentation in just a month's time.
spk08: Okay, thank you.
spk03: Thanks, Robin. Thank you. Our next questions come from the line of Oren Livnat with HC Rainway. Please proceed with your questions. Hi, guys.
spk10: Can you hear me?
spk12: Yeah. Hi, Oren. Good morning.
spk10: Hi. Good morning. I just want to return back to the review process, you know, with the caveat, you know, we understand you can't comment on anything you can't comment on. But I'm getting a lot of questions as we get closer towards the PDUFA regarding, you know, any unknowns. Obviously, your tolerability profile looked really good in Phase 3, at least as good as Irem. But people are asking me, what do we know, or what have you already submitted with regards to the safety or risks around you? your drug delivery technology in general. Specifically, I'm getting asked about dose dumping work you've done or food effect work you've done and how that might factor into, you know, the benefit-risk calculation. And then also just in the review, on the manufacturing front, I think in the past you've mentioned bridging studies that you, you know, need or plan to do. Can you just remind us, is that just for your backup domestic supplier for post-approval? Or, you know, and how confidently the FDA can inspect your your original European manufacturer in time. Thanks.
spk12: Yeah, great questions. You know, as it relates to the technology itself, although I would say that this formulation is unique on its own to a certain extent, the technology has been approved in a prior product, CoreHCR, which is Carbidolol. It's a beta blocker commercialized by another product, another company in the past. So the technology has been through and if you will, an FDA process, so to speak, from that perspective. Although, you know, again, this is a, you know, an application of that technology and that process with, you know, with this, you know, tailored to, you know, sodium oxibate in that regard. But I would say that as it relates to all of the non-clinical work that we had to do in our submission, Again, we believe it's robust, it's complete, whether that's our food effect data or otherwise, and the work we've provided to, you know, validate, you know, this formulation and how this formulation performs in different settings that are required relative to our NDA. We feel really good about our submission, and obviously that includes, you know, the drug-drug interaction data that we provided as well as part of our NDA submission. As it relates to our CMC, yeah, you know, we have a primary supplier who's been making this product for well over five years. We filed with over three years of stability data. During the review process, we'll take over four years actually of stability data. So they've been making it at this scale for quite some time. So we feel we have a robust manufacturer who's been doing it for a long time, but we also recognize we wanted to have another source and a backup source. We've done all that work. We've completed those bridging studies, if you will, and that will be an action that will occur post-approval, as you referenced.
spk10: And with regards to access to your European facility given, you know, COVID dynamics, you know, October's still a ways off.
spk12: Yeah, sorry. Again, you know, that, you know, we've seen some of the recent guidance from the FDA that has recently come out about how they'll, you know, prioritize inspections. The limited number, you know, less than one-half of 1% or so that they claim of facilities that didn't get inspected tied to a review over the last 18 months that they needed to inspect. That being said, this facility was recently inspected just prior to the pandemic and with no observations, no 43s, if you will. We've done all of our work to prepare for any sort of PAI with our partner, our CMO. So at this stage, I would say there's nothing more to say on it at this point. And if anything comes up, we'll certainly update accordingly. But we see no reason that a PAI would be a deterrent at this point. But, you know, again, we have to work and see how the NDA process unfolds. All right. Appreciate it. Thanks.
spk03: Thank you. Our next questions come from the line of Matt Kaplan with Lavenberg Development. Please proceed with your questions.
spk01: Oh, hi. This is Raymond in for Matt. Thanks for taking our questions and congrats on all the progress so far. Maybe just the question on the market research. You mentioned that there were high levels of treatment refusals and high discontinuations after treatment starts. And you said the second dosing as a main issue, but can you provide any color on any other sources of refusals and discontinuation and how FT218 might be positioned to capitalize on that? And my other question is just any updates you can provide on the switch study at the moment? Thanks.
spk12: Richard, do you want to start with the first one and maybe Jen take the second?
spk02: Sure. No problem. Thanks, Raymond. As far as the research is concerned, we often focus on the inconvenience of getting up during the middle of the night for a second dose for the twice nightly oxibates. But beyond that, there's a lot of other concerns that go on around the fear of waking up a partner, the leaving out your medications out at the nightstand with kids running around the house. There's a lot of stress on these patients that we find from the research in regards to more than just getting up during the middle of the night. And I think what we generally sort of see there is it's not easy. Also, a lot of some of the patients have now gone to adjusting their dose. It's called asymmetric dosing to maybe take more of their dose earlier on and less later on during the middle of the night. So I think there's a lot of factors that continue to go on there with these patients that go beyond just the perceived inconvenience of the second dose. And I'll pass it over now to Jen on the second part of your question.
spk09: Thank you, Richard, and thank you for the question, Raymond. So we are now at just about 60 patients, or approximately a 20% increase from our last call. We've got a number who are in the screening process right now, so we're looking to add to that overall number. We're also continuing to activate sites. And, of course, all of this is happening still in the remainder of the backdrop of a worldwide pandemic. There's also the unique aspect with FT218, because it is not yet FDA approved, it's considered a Schedule I medication, which necessitates patients having to come in once a month to pick up the medication. What we're really pleased with in regard to this open label switch study are the insights that we're gathering. And the primary purpose, of course, is long-term safety and tolerability. And we're also seeing, in addition to good results there, sustained efficacy with FT218 as well. And then lastly, it's preliminary, but the feedback that we're getting in regard to the preferred dosing regimen once or twice nightly has been extremely positive for once nightly FT218.
spk01: Thanks. Thank you.
spk03: There are no further questions at this time. I would like to turn the call back over to Greg Divitz for any closing comments.
spk12: Thank you again, and thank you everyone for your questions. In closing, To say I'm proud of every team member's contributions to where we are today would be an understatement. It's been a very, very busy few months, but it's also exciting to see us closing in on the PDUFA date and ramping up our preparations for the next chapter in our company's history. And I just want to reiterate and rest assured that this entire team is working very hard every day to deliver the best possible results for all key stakeholders. That includes patients and providers, but also and importantly so includes our shareholders. And we'll continue to keep you apprised of our progress. And with that, we thank you for joining the start of the week and the start of your day with us and wish you a great rest of the day. Thank you.
spk03: Thank you for your participation. This does conclude today's teleconference. You may disconnect your lines at this time. Have a great day. Have a great day.
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