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spk13: Greetings and welcome to the Avidel Pharmaceuticals fourth quarter and full year 2021 earnings call. At this time, our participants are in a listen-only mode. A question-and-answer session will follow the formal remarks. As a reminder, this call is being recorded. It is now my pleasure to introduce Brandi Robinson. Thank you. You may begin.
spk09: Good morning, and thank you for joining us on our conference call. This morning, we've issued our press release providing a corporate update and financial results for the quarter and full year ended December 31st, 2021. The release can be found on our website, www.avidel.com. As a reminder, before we begin, the following presentation includes several matters that constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are subject to risk and uncertainties that could cause actual results to differ materially from those contemplated in such forward-looking statements. These risks include information regarding products in development stage that 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 under the Exchange Act included in the Form 10-K for the year ended December 31st, 2021, which was filed yesterday, as well as any 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 today are Greg Divis, Chief Executive Officer, Dr. Doug Williamson, Chief Medical Officer, and Tom McHugh, Chief Financial Officer. Richard Kim, Chief Commercial Officer, and Dr. Jennifer Guteman, our Vice President of Medical and Clinical Affairs, will additionally be available for Q&A following the call. At this time, I'll turn the call over to Greg.
spk07: Thank you, Brandi. Good morning, everyone, and thank you for joining us for our call. I'll start off by providing an update on our business and highlighting the progress made toward potential FDA approval and commercialization of FC-218, including a brief update on our commercial readiness activities. Dr. Williamson will then provide a summary of the new data recently presented at World Sleep 2022. Tom will then review the financial results for the quarter, including an update on our recent convertible maturity extension, We will conclude with a Q&A session when we will be joined by Richard and Jennifer. As we head toward what we believe will be a full approval and launch for FT218, there are four priorities for the company that are mission critical to ensure we realize the full potential and value that FT218 can create for patients, for shareholders, and for Avidel at large. Number one, and most importantly, is the FDA taking a formal action on our FT218 NDA and granting our full approval. Number two, continuing our deep understanding of the market opportunity and the clear, unmet need for OxyBait-eligible patients that goes well beyond the current OxyBait-treated patient population. Number three, the significant progress we are making in preparing the company and the narcolepsy community for the potential future launch of FT218, including launch readiness and data generation. And finally, number four, the strengthening of our balance sheet to ensure maximum flexibility to achieve our business objectives. So let's begin our update with priority number one, the one topic on everyone's mind, which is the status of our NDA review for FD218. Earlier this year, we sent in additional edits to the draft label, and we confirmed with the agency at that time there were no additional questions or information requests pending. We were given no specifics on the timing of the completion of our review, and subsequently requested a meeting with more senior officials from the FDA to clarify timing and any remaining review items. Based on these and subsequent exchanges, let me share with you what we have been told directly by the FDA. First, we have reconfirmed that there are currently no outstanding questions or information requests and that the last and final remaining matters are as communicated to us administrative, and internal to the FDA. Furthermore, within the past week, the agency confirmed to us that they are working on the FT218 label and expect to send it along with any potential follow-up comments back to us soon, which is the language used in writing to us by the FDA. We share this detail so our stakeholders know we are continuing to take action to move this forward with all means at our disposal. And we have always expected that the next step upon completion of the FDA's internal and administrative process is for us to receive an updated, proposed, and potentially final label. And now, based on this very recent and direct feedback, we have been informed that it is currently being worked on and is expected to happen soon per the FDA. Completing this next step on the label, we believe, is an important step forward toward the potential full approval of FT218, which takes us to priority number two. As it relates to the market opportunity and unmet need of OxyBate eligible patients, we have continued to conduct extensive patient and healthcare professional research, research which continues to inform and confirm that FT218, if approved, can command a meaningful share of the narcolepsy treatment market among OxyBate eligible patients including the potential to expand the treated oxibate patient pool. We believe this is important to understand because, despite the introduction of an additional twice-nightly oxibate formulation over the last 15-plus months, there has not been a meaningful increase in the number of narcolepsy patients being treated with any form of the twice-nightly oxibates. And through our extensive research, we have learned that the introduction of once-at-bedtime FT218 will be well-received by current high-volume oxibate prescribers and can expand the utilization of oxibates with physicians who currently prescribe at low volumes or even not at all. Physicians we have surveyed indicated their intent to switch patients as well as increase their oxibate utilization upon the potential approval and launch of FT218, including those who currently do not prescribe any twice-nightly oxibates. Furthermore, very recent patient research informs us that the majority of oxibate-eligible patients, whether naive to therapy, previously treated with oxibates, or currently on either of the twice-nightly formulations, have self-reported as being interested or significantly interested in learning more about once-at-bedtime FP218 and, if approved, interested or significantly interested in initiating therapy with FP218. and this includes those who have recently switched to the twice-nightly mixed salts formulation as well. All of this only confirms what we have seen in our past research, that there is a clear need for a once-at-bedtime option like FT218 for OxyBait-eligible patients, whether on therapy currently or not, due to the challenges associated with having to wake up in the middle of the night, thus providing Avidel a significant opportunity to positively expand the OxyBait-treated pool almost exclusively to our benefit. The potential full approval and launch of FT2 and 8, combined with a market opportunity that is meaningfully larger than the current approximately 16,000 patients and nearly $2 billion, requires significant market preparation and launch readiness actions, for which over the last year and specifically the last few months, we have made tremendous progress and leads us to priority number three, Under the leadership of Richard and his experienced team, they have significantly progressed our overall launch readiness across all commercial and launch-related functions. This includes but is not limited to the selection and setup of our specialty pharmacy distribution partners, the continued development of our patient services center and REMS, which are prepared to advance upon FDA approval, meaningful engagement with payers with a focus on the three large PBMs representing approximately 85% of commercially insured lives, the identification and selection of nearly 90% of our sales team with offers accepted pending FDA approval, and lastly, customer-facing activity generating significant interest from healthcare providers at live medical congresses, including most recently at the World Sleep Congress in Rome that just finished yesterday. Another key component of our launch readiness is to highlight the publication of key data to continue to scientifically demonstrate the potential clinical benefits of once-at-bedtime FT218. Before we hand it over to Tom to conclude with our fourth priority, which is our financial performance and balance sheet-related actions, I am pleased to introduce to you Dr. Doug Williamson, our newly appointed Chief Medical Officer, to review the highlights of the most recently published clinical data from WorldSleep 2022. Doug, the floor is yours.
spk11: Well, thank you, Greg, and good morning, everyone. I want to spend a few minutes to highlight some of the new data we recently presented at World Sleep 2022, which continued to support the proposition of FT218 as a potential once-at-bedtime option to treat the devastating symptoms of narcolepsy. As you saw from our press release issued earlier today, we had a strong presence at World Sleep, with a total of eight narcolepsy posters presented. To highlight some of our data, let's start with the discrete choice experiment, or DCE. DCEs are a widely accepted way to isolate the specific attributes that ultimately drive choice in medication selection. Building upon a prior study, we surveyed 100 OxyBait-experienced clinicians, along with 120 OxyBait-experienced patients. And this time included the attributes of the two times nightly mixed salt oxidate product as well. This second DCE confirmed that once nightly dosing, when compared to either of the twice nightly dosing options, and notably regardless of sodium content, was the most important attribute driving both patient and clinician preference, as well as patient quality of life and the reduction of patient anxiety and stress. In addition to this DCE publication, our pivotal Phase III Rest On clinical trial of FT218 continues to yield relevant post-hoc data. About two-thirds of people with narcolepsy experience disrupted nighttime sleep. This is further exacerbated by the vast majority of medications used to treat excessive daytime sleepiness in narcolepsy, which share the common side effect of insomnia. In the Rest On study, 63% of participants received stable doses of concomitant stimulants. Yet the FT218 group had improvements in disruptive nighttime sleep when compared to placebo, not only regardless of narcolepsy type, but also importantly regardless of whether or not they took a stimulant. In other post hoc analyses from Reston, FT218 demonstrated improvement compared to placebo in subjective measures of daytime sleepiness, sleep quality, and refreshing nature of sleep as early as week one with the 4.5-gram starting dose, with even greater improvement at week two soon after starting the 6-gram dose. Finally, World Sleep 2022 offered the opportunity for our first presentation of interim data from the ongoing RESTORE study, which was designed to evaluate the long-term safety and tolerability of FT218. Among the participants treated in this open-label study some for more than 18 months, FT218 has been generally well tolerated with a low rate of discontinuation due to adverse reactions and no unexpected safety signals. Upcoming congresses this spring will include additional analyses from Restore on patient preference data for those switching from either of the twice-nightly products, as well as data on dose initiation and titration. Based on a growing body of clinical evidence and the feedback we receive from patients, we continue to be motivated by the tremendous potential of FT218, if approved, for people with narcolepsy. With that, I'll now turn the call over to Tom to detail our balance sheet actions and financial results. Tom?
spk06: Thank you, Doug. I'll provide a few highlights for the quarter and also note that full financial results are available in the press release and the 10-K. Okay. We just announced an exchange of $117.4 million of $143.8 million of senior unsecured convertible notes that were originally issued in February 2018 that had a maturity date of February of 2023. Following this exchange, $26.4 million remain outstanding from that original issuance. But most importantly, the $117.4 million of notes that were exchanged have a maturity date of October 2023 or eight months longer with no change to the interest rate of 4.5 percent or the exchange price of $10.79. Our rationale for extended maturity date can be summarized as follows. With $157.2 million of cash on the balance sheet at December 31st, we believe we're in a strong liquidity position and don't need to raise capital at this time. We believe the cash on hand based on current plans and expectations, is sufficient to prepare for the launch of FT218, if approved. Extended maturity by eight months removes a significant near-term cash requirement and provides much greater flexibility and time to execute not only a potential launch of FT218, but also time to consider financing options that address Avidel's longer-term capital needs. We are appreciative of the note holders who participated in the exchange and look forward to their continued support of Avidel. I'll now turn to a brief commentary on operating expenses. Total operating expenses in the quarter ended December 31st, 2021, were approximately $23 million, which is a $9 million increase versus the prior year. The increase is due to a $12 million increase in SG&A, offset by a $3 million decrease in R&D. The year-over-year decrease in R&D resulted primarily from lower clinical expenses and purchases of active pharmaceutical ingredients used in the research and development of FT218. The year-over-year increase in SG&A is attributable to a number of factors, including commercial launch planning costs, higher legal and professional fees, and higher compensation costs associated with higher headcount. Net loss for the fourth quarter of 2021 was approximately $22 million, or 38 cents per diluted share, compared to net loss of approximately $11 million, or 19 cents per diluted share in the same period in 2020. The year-over-year increase in net loss and loss per share resulted from the increase in operating expenses. And as noted a moment ago, as review of our NDA for FT218 progresses and our preparations for launch continue, we believe we're in a strong financial position with 157 million of cash on hand, coupled with the eight-month maturity extension on 117 million of the convertible notes. to fund the financial investments needed for the potential launch of FT218. I will now turn the call back to Greg for closing comments before we open up the line for Q&A.
spk07: Thank you, Tom. And again, thank you all for taking the time to join us today and your continued support of AVIDO. You know, to wrap up briefly before Q&A, we are fully aware of everyone's interest in the status, the timing, and most importantly, the completion of the FT218 NDA review. Rest assured, we are directing all our energy, sparing no effort, and will continue to deploy all available resources, both internal and external, to successfully bring FT218 to an approval as soon as possible. We have not learned anything, including in our most recent exchanges with FDA, that compromises our belief in the potential approvability of FT218. We look forward to the FDA, as they have recently communicated to us, completing their work soon, bringing us closer to our mission, of delivering this important treatment to people living with narcolepsy. The progress we have made to both prepare ourselves and the market for the potential launch has been significant and clearly demonstrates a much larger Oxibate-eligible patient pool that we can potentially serve given the clearly defined unmet need for and the importance of once-at-bedtime dosing for patients, providers, and caregivers. And once approved, we will be ready to accelerate our FT218 launch activities and fully maximize the value of FP2 and 8 for all stakeholders, including patients, shareholders, and Avidel. So with that, let's open the lineup for Q&A.
spk13: As a reminder, to ask a question, you need to press star 1 on your telephone, and to withdraw your question, just press the panel key. One moment for questions. Our first question will come from Chris Howerton from Jefferies. You may begin.
spk16: Hi, good morning. I got to say, Greg, really exciting news. I can't wait to hear from the FDA really soon, so thanks for the update today and, of course, for everything else. You know, the questions for me, I guess, would be with respect to kind of the NDA review and the labeling, if there's any features of the label that you're currently negotiating that you can kind of give us more color on, that would be really interesting. The second question that I would have maybe is from just a logistical perspective. I know when we were closer to the original PDUFA date, there was some discussion around a hearing for preliminary injunction. Just curious if there was any updates on the status and timing of that as it appears we're getting closer to a full approval, as you said. And then if I may, if you want to address a third question, you can say no thanks, Chris. You've already asked too many. But the third one would be if you've considered strategically, you know, obviously narcolepsy is the most near-term opportunity, but have you considered strategically label expansion opportunities into things like idiopathic hypersomnia where other oxibate formulations have been successful? Thank you.
spk07: Yeah, Chris, thanks. Maybe I'll take those briefly. You know, from a labeling perspective, the pen is really in, I would describe it as, in FDA's hands. We provided the last turn of it earlier this year, as we referenced. I would describe those edits as minor. They're data-related, making sure all information is accurate per the sources in our NDA submission. And so that sits in their hand. We've always known that we would get a label back. It could be a final approved label. It could have edits to it. based on whatever the agency is proposing. But we don't believe that that's an important next step for us to head toward a full approval. So there's not much more I think we can provide relative to the specifics around it. Other than that, we've had a number of exchanges on the label previously. So as we've said to many of you, I believe, in the past, we felt the label was in really good shape. And if it ends up anything remotely where it was in our last turn, we'll be really, really pleased with the construct of our final approved label. Secondly, on the PI, there really is nothing to report from that front. You know, there isn't a PI filed, there isn't a hearing planned at this stage of the game. I think whether one gets filed or not is not a question we can answer, but I think our view based upon how the court was preparing to handle a potential PI should one have been filed back last year. We have no reason to believe that the court isn't going to take the same perspective and the same view of we'll have an approved asset that patients really need, and we'll want to act on that as efficiently as the court schedule will allow it to do that. So that's how I think where we sit from the PI. There's really no information to report on. In terms of other label expansions, we've done a lot of work in terms of really evaluating different opportunities. But let's be clear, what's most important for us and for patients we're serving and for our shareholders is to get the approval and launch FT218 as the next major catalyst for the company. But we have done a bit of work in looking at other opportunities, and certainly we'll talk more about that in the future. And I would say the most important priority for us from that regard is lifecycle management. Clearly, there's studies being done in idiopathic hypersomnia. We'll see how that data and those launches progress over the coming months. And it's obviously something we're very interested in and something that we hear from a lot of KOLs, including just this past week at the World Sleep Congress in Rome, a tremendous amount of interest in wanting to study FT218 and idiopathic hypersomnia. So we'll have more to talk about that in the future. In addition, what we've also done is we've begun some early work on some alternate formulation work for that subset of patient population that may need something, a different formulation with a different salt content, given that we have a technology platform that allows us to do that in a once-nightly. So that work is ongoing, and there will be more to talk about that in the future, but it's still early at this point in time. So thanks, Chris.
spk16: Awesome. Interesting little tidbit there at the end, Greg. So thank you for that, and I appreciate all the progress.
spk13: Our next question will come from David Anselm from Piper Sandler. You may begin.
spk15: Thanks. So maybe I'll turn the discussion over to the commercial landscape. And what I wanted to get your thoughts on is how you think the Xyrem generic market will Obviously, we know that there's an authorized generic coming, and there are significant royalties attached to that. So I guess the question here, Greg, is how do you think about, you know, 218's place in the market to the extent that we see significant uptake of an AG? So that's my first question, and then I have a follow-up. Sure. Thanks, David.
spk07: Maybe I'll ask Richard, our chief commercial officer, to answer this one.
spk05: Yeah. Hey, thanks, Greg, and thanks, David, for the question. So, yeah, you know, obviously we did all hear from Jazz sort of anticipation of the AG for the second half of this year. And, you know, I can sort of say that's pretty consistent with where we view this marketplace. If by chance the AG happens to grow this marketplace, I think we view that as positive for more potential oxibate utilization that goes on overall. But I also think at the end of the day, the AG and the existing two branded compounds in this marketplace, and even one day when you get the multi-source generics, they're all twice nightly oxibates. And that's a very consistent value proposition. We think the proposition of FD218 as only once at bedtime treatment options is a very differentiated treatment option that's going to be well needed, as you've heard from Greg saying earlier. And I think our value proposition is very well understood by not only patients and physicians, but also by the payers that we engage with as well. So our view on the AG and the multisource generics potentially in the future hasn't changed at all, and we believe we're well positioned in this marketplace with or without them coming to the market.
spk07: I just think I would only add that we have no expectation at this stage, given your commentary on royalties, that there's going to be a substantial reduction in the pricing in the marketplace of the AG. And if that is the case, if it's more typically priced or even higher than more typically priced, given what the AG providers have to give back to the reference listed drug holder, our view and our research with payers would tell us that it's going to be treated like an extension to the brand. So we don't believe it. We don't expect it to be a step at it. We don't expect it to be preferential. And candidly, as Richard noted, every twice nightly product patient is a candidate for us, right, whether they're on therapy or not, quite frankly. So, again, I think we don't see it being a material shakeup to the marketplace, nor mitigates the opportunity one bit for FT218.
spk15: Okay, that's helpful. Maybe I'll ask sort of the commercialization or commercial landscape question a little differently, which is that you've talked about whether it's an AG or the brand, it's still a twice-nightly product. But as the pie for the low-sodium product increases, do you look at that as a landscape where your pie could be shrinking, or do you have confidence that potentially you can get some low-sodium patients moved over to 218 and Obviously, once nightly, but it is going back to high sodium. So how do you think about that?
spk07: Yeah, thanks, David. Richard, do you want to go?
spk05: Yeah, sure. No, it's a great question again, David. So, you know, Greg commented earlier on the, you know, growing body of market research that we continue to do. And what we do know is when we show our product profile to patients who are either on, you know, the sodium oxibate or the mixed salt version right now, is there's a very high interest in learning about FE218. We also know that there is also interest in potentially switching to this agent. And in fact, David, in patients who are on the mixed salt version, they tend to be patients who have changed therapy quicker over their treatment duration than on the twice nightly sodium oxidate. So we actually view the opportunity if patients are switched on to the mixed salt version as still a very robust opportunity for us. They tend to be more early adopters of treatment change as well. So we view both the pool of currently stable sodium-oxidate twice-naughty patients and those who are on the mixed salt version as both being very viable candidates to be considered upon approval for FT2 and A. So we don't see the mixed salt version shrinking our ability to penetrate the marketplace or to grow it at all in the future either.
spk07: Yeah, I think in addition to that, I think where your commentary and from our perspective doesn't represent is that we believe the pie of OxyBait-treated patients can grow. And we don't believe that certainly hasn't been demonstrated with the introduction of the mixed salts product. But based on our research, we think there's more patients out there that don't require a switch, but yet we hear from patients on therapy today who have switched from both the twice-nightly original formulation and the more recent Mixol formulation. We have those patients in our restore study now. We hear how they're doing, and we think as, you know, that body of experience grows from being in an open-label extension study to being in the market, we think that will be a major catalyst for us, you know, accordingly. But as Richard noted, they're still twice-nightly and clearly have expressed an interest and want to learn about FT-218. David Chambers- Okay.
spk15: That's helpful, Kohler. Thanks, guys. Kohler- Thanks, David.
spk13: David Chambers- Our next question will come from the line of Amin Fadia from Needham. You may begin.
spk10: Amin Fadia- Hi, good morning. Thanks for taking my question. Greg and team, congratulations on the update. I had one clarification on that. So it sounds like you got an update from the FDA that they will send you their comments on the label soon. Was this after a reasonably long gap since the last time in January that you communicated on the label? If you could clarify on that and If there was any other back and forth in the meantime, that would be helpful to know. And then I have some other follow-ups.
spk07: Yeah, Ami, thank you. There's definitely been back and forth, you know, over the period of time. But I will say that, you know, the last turn of the label was ours back in January. We haven't seen a label come back to us yet. But again, our edits at that point in time were fairly minor in nature and relative to just correcting some data tables and ensuring that everything was tied to the data in the NDA. But there's definitely been exchange going on, and again, this recent communication has occurred within the last week.
spk10: Okay, excellent. And in your meeting with the FDA and some senior management at the FDA earlier this year, they had given you some color on timelines Are we still within that timeline?
spk07: Yeah, they gave us some guidance in terms of how long they thought it may take them to complete the work that was remaining from that standpoint. And, you know, with us recognizing that there's nothing binding around that because we're not heading toward a specific action date. But at this stage, we haven't surpassed that, you know, that guidance we received.
spk10: Got it. Okay. Let me ask about just the launch. You talked about the REMS program. Can you give us some color around, you know, once everything is approved and you're ready to go, how long would it take you to enroll physicians into your REMS program? And tactically, how would you target patients that are already on a program Xyrem or Xyway product to seek conversion?
spk07: Yeah, thanks, Ami. Maybe Richard, if you want to start.
spk05: Yeah, Ami, great question. So the good news is under Jen's leadership, the REMS program built and also our overall distribution commercially has continued to progress. Obviously, we need the final components from the FDA to sort of get to that next sort of near finalization stage, but we've made really great progress to where we are, so we feel once we actually have the final details from the FDA, we will enter that next phase of building those programs out. Now, when you think about targeting the patients, so the good news is we know that this marketplace is very concentrated, both from a patient but also from a physician standpoint. We know that there's less than 6,000 overall oxidative prescribers. We know that there's less than 1,800 physicians who account for over 80% of the script volume. So we know who we have to go see as far as where those patients reside right now. So we think we have a very good targeted plan and obviously we'll be building our resources to make sure that we can support that network. But the other thing I would also add, as we see, is within the patient organizations, we think there is pretty good anticipation of having the potential option of a once-at-bedtime therapy to be available. So we will be approaching this both from our physician engagement that we have and also many of our targeted patient initiatives as well. So we feel really good about where we are right now, but clearly we need that approval to get to that next stage of our rollout as well.
spk10: Got it. Maybe a last question for me, if I may. Just with regards to switching patients from a low-sodium to a once-nightly product, the feedback that we've heard from patients and some of the other physicians is that patients that have, you know, a cardiovascular underlying disease or patients who are renal impaired were the ones that they see as most ideal for a low-sodium product. In your work and the feedback that you've received, would these patients also be interested in switching to a more convenient one-smiley dose? Thank you.
spk05: Well, I think, you know, go ahead, Richard. Great question. And, you know, so first and foremost, there are some patients that may benefit from a lower sodium version. You know, our perspective of that is it's relatively low, less than 10% of the overall potential marketplace with potentially more severe hypertension or on renal dialysis. The question fundamentally for a lot of those patients will still come down to the benefit of the salt versus the benefit of having a therapy that they only need to take once at bedtime. It's really the fundamental issue of treating the narcolepsy versus other conditions that the patient goes through as well. So we will not necessarily be the perfect option for everyone, but I think that's the fundamental choice in those more severe hypertensive and renal dialysis patients that may need to be considered.
spk10: Got it. Thank you.
spk07: Thanks, Ami.
spk13: Our next question will come from Mark Goodman from SVB. You may begin.
spk08: Thanks for taking my question. It's really out of line for Mark. So do you think payer pushback will be a hurdle for switching to 1-90 dosing after generic entry of Xyrem? And can you remind us what percentage of patients who tried and discontinued Xyrem or Zyvape, and how would you target these patients? Thanks. Yeah, go ahead, Richard.
spk05: Yeah, great question again. So as far as, you know, first and foremost, I'm really proud of the work that we've had of our PARA team engaging with the PARAs across the country as well. And I think the first thing that I would say is the clinical value proposition of once-at-bedtime FT218 has become very clear to all of our key stakeholders. Right now, as far as the pushback, I mean, obviously, the label will enable us to initiate the next wave of deeper conversations, but I think they're ready for another option to come into this overall class. There clearly haven't been a lot of options as far as they negotiate with industry within the Oxibate space. So I think that, along with our clear value proposition, puts us in quite a good position overall with the payers across the board. What I would say is they've been very engaged. We've been able to speak with all the major PBMs and GPOs that will cover the vast majority of patients that we look into. And they've been really good conversations. So we feel like we're in a really good spot there overall with our discussions. And then if you look at discontinuation rates, so some of our data tells us that after 30 days, after the first month, about 30% of patients have their twice-nightly oxibates. So now some of that may be for reasons that even a once at bedtime therapy may not be able to address. But for many of those, it may be an option. Some of the main reasons why people discontinue is lack of efficacy or they couldn't take their therapy. Now, we don't know if the lack of efficacy also ties into I couldn't take my second dose of treatment as well. But we do see that those people who have recently discontinued as a potential pool of patients, once again, maybe not all of them will consider that. But the fact that they took the steps to try to initiate an oxibate we see as a very good step. and the fact that there may be an option upon approval that there's a new once-at-bedtime opportunity, we think that bodes well for an opportunity for those patients to potentially benefit from an OxyBase treatment as well.
spk08: Thanks for the caller. It's very helpful.
spk13: Our next question will come from Paul Matias from Stifel. You may begin.
spk04: Thank you for taking my questions. I had a few related to orphan drug exclusivity. I know you've gotten this question from analysts and investors a bunch of times, but as hopefully the review process winds down here, do you think that some of the delay has been driven by vetting the implications of Zywave orphan drug exclusivity and what that means for FT218 freedom to operate? And then second, I was wondering if you could clarify the different scenarios here. So I guess for you to attain full approval, does that mean that FDA needs to essentially determine that once nightly provides a major contribution to patient care, thereby, I guess, breaking or transcending Zywaves ODE, or is that not the right way to think about it? Is there a tentative approval scenario? Maybe just expounding upon how this could kind of play out and how we should be thinking about it would be really helpful. Thank you.
spk07: Yeah, thanks, Paul. I won't speculate in terms of, you know, what all is kind of behind the scenes and has been described generally as administrative and internal to us by the FDA directly. But what I can say is we've had the opportunity during this recent period of time as we've met with the FDA. We went through every aspect of the NDA with them, every component, every section within the review, within the NDA, including orphan drug, seeking to clarify is there anything else needed, anything additional information required, anything we can do to help get to the decision more quickly that we believe is coming. And the answer to us was there's no questions, there's no information needed, there's no data needed from that standpoint. So we feel, again, we didn't learn anything in those conversations that compromised our belief in the full approvability of FT2 and 8, which is really the question. The second question is what do we think has to happen? We've always said and we firmly believe that, and we've communicated this directly to the FDA, that we believe we can have a full approval without an orphan drug exclusivity being granted. That being said, we believe we have provided a robust submission of data that clearly demonstrates, based on evidence, that once at bedtime dosing of FT218 is clinically superior to the current twice nightly options. based upon both safety and a major contribution to patient care. We believe that will be arbitrated and decided upon. Our expectation is in or around at the time of their final decision. That's our current expectation. But, again, we don't envision at this stage a scenario that wouldn't be other than a full approve.
spk08: All right. Thank you. Thanks.
spk13: Our next question will come from the line of Oren Livnet from AC Waymark. You may begin.
spk12: Thank you. I have a couple. You've clearly been investing heavily in the, you know, prelaunch activities, at least through Q4. I guess, first of all, could you comment on whether, you know, you've hit pause on some of that as you wait? But I guess more importantly, given all the progress you highlighted in the script, I is there potentially any shortening of the window post-approval to launch from, I think we've all been assuming, about six months, given, you know, all the progress you've made on setting up that REMS and also PBM interactions? And I guess just to make that a quintuple part question on the PBM issue, you know, how substantive have those conversations been? Should we hope for, you know, coverage right out the gate?
spk07: So on that last question, maybe I'll let Richard chime in.
spk05: Yeah, great question. So, you know, I've never been one to try to precisely predict a pair decision. But what I can say is, you know, obviously the time between an approval and the full launch, we intend to utilize every one of those days to our full extent. The conversation has gone very well, but, you know, until we actually have that full approval, it's hard to get the conversation to that really deep level. What I can say is we're definitely going to be working to have as much coverage as we can around the time of approval or shortly thereafter as well. And with where we are with conversations right now, I feel like we've made good progress. I think the next stage post triggered by the potential approval will really be the telltale sign around how quickly we can move things. So potentially at our upcoming calls, we can provide an update on sort of where we are with the payer engagement. And Greg, I'll turn the other questions over to you.
spk07: Yeah, again, we have a very disciplined approach to how we've built our pre-launch activities, recognizing that the approval is an important catalyst for us to really go to the next level. So I wouldn't say we've hit pause, but we've made progress in the areas where we can and need to make progress, where the lead times are even longer than what it may be relative to post-approval to a full launch. We certainly have made progress in certain areas and have made a decision to go forward, i.e., in building commercial supply and those sorts of things. And so we've been able to potentially, you know, draw back some time from that standpoint. I think the big, you know, kind of swing in that decision when we get our full approval will be what is our primary packaging look like? We don't believe and we don't expect there's going to be any changes to that artwork at this stage. But if there is, then we're going to have to reprint some of that primary packaging foil, which certainly doesn't allow the opportunity to shorten the time frame.
spk12: Okay. And then specifically, just on the sales force, I think you mentioned that 90% identified, maybe, I don't know if all of those have accepted offers, but just, you know, is that potential crew still ready to roll out post-approval with conditional offers, or given the delay, is there churn there? They need to work, right? So I guess post-approval, do you need to go back and sort of reinitiate some of that recruiting process? Yeah, go ahead, Richard.
spk05: Yeah, so the good thing that we've been doing is we've been trying to keep both our internal, our external, and our contingent sort of based upon approval potential candidates just engaged in the process. You know, it may sound cliche, but last year we rolled out new core values for the organization, and one of them is togetherness. And we've really tried to keep our candidates who are sort of triggered by the approval sort of up to date on where we are with things, showing them what we're doing. And so to be very candid, we probably lose a couple people, but we've built backup plans as well. It's been really impressive to really see the engagement of all these folks. I think wholeheartedly, They see the opportunity that we have with once-at-bedtime FC218 could potentially change a lot of patients' lives in a positive way. So my belief is we'll lose a couple, but we have backups. But holistically, people realize the opportunity that we have to make in the field of narcolepsy. And what I can say from my conversations is a lot of people want to be a part of this. So we think we have a special opportunity here, and it's been really great to see that reinforced by the people who want to join us on this journey at Avidel.
spk08: Great. Appreciate it.
spk13: Thanks, Jordan. The next question is from Matt Kaplan from Leidenberg.
spk14: Hi, good morning, guys. Thanks for taking the questions, and congrats on the recent feedback from the FDA. That's great. Just wanted to, most of my questions have been answered, but with respect to your comments earlier on the different formulation of the lower sodium content product, what's your sense in terms of what the development path of a product like that, is that just a simple PK, PD type of development, or will that entail a larger, more rigorous clinical trial?
spk07: Yeah, Matt, it's probably a bit premature to answer that question at this stage. We're still in the early stages of doing our work here, and that will be a discussion we clearly have with the FDA at the appropriate time in terms of what's the right development path forward, and that will be predicated on, you know, how our formulation advances and its relative bioequivalence to the current once-nightly formulation of FT218. So I think ultimately that will be, you know, that feedback or that data and that insight will help inform that recommendation and strategy that we'll go forward with, but I think it's a bit early at this stage.
spk14: Okay, fair enough. And then in terms of where you are in preparation for for launch, can you talk a little bit about your your drug supply and your manufacturing and and and where you have that in place ready, ready for launch?
spk07: Yeah, on the drug supply, we've initiated, you know, all of that effort and activity going back towards the end of last year from, you know, bulk API production and the beginning of manufacturing of what we'll just call bulk beads, right? So bulk supply of our immediate release microparticles and our controlled release microparticles. We'll go as far as making as much of that as we can in that bulk state through our primary match CMOs, but what we won't do at this stage is actually blend that product and put it inside of our daily stick pack until we have the final approved daily packaging labeling and artwork, right? We don't expect that to change, but the minute we put that product into a packaging in our primary packaging, And if that labeling would change, then that product we couldn't use. So we continue to make progress in building bulk, both active ingredient and immediate release and control release microparticles. Post an approval, those actions will expand to blending those two by strength in the proprietary ratio and then daily stick packing it, which is what will occur post the approvals. Great, great.
spk14: Thanks for the other details. Thanks.
spk13: Our next question comes from Robin Gardner from Craig Halem. You may begin.
spk01: Thank you for the additional information about your communications with the FDA and congratulations on that update. Some great questions already. Just one final question regarding your life cycle management. So in this case where you have an idiopathic hyperformulation with a different salt content, would this eventually overtake FT218 for use by narcolepsy patients, not just for idiopathic hypersomnia?
spk07: Well, again, I think as it relates to lifecycle management, it's a bit early to really speculate on anything specific. But we do, listen, let's be clear, we've demonstrated we have a technology that can deliver oxibate-related products in a once-at-bedtime dosing, and we certainly want to make sure all patients have the opportunity to be treated with a once-at-nightly bedtime option. As it relates to indication, the most likely approach for us I would say, you know, based upon our thinking, is that we certainly would pursue this formulation in narcolepsy. And what we do beyond that in terms of indication is work we're still evaluating, whether that's idiopathic hypersomnia or some other rare sleep disorder that could be a potential life cycle extension. But clearly, you know, there's a lot of interest and work being done in IH today, and that's, again, something we're evaluating closely.
spk01: Okay, thank you. Looking forward to future updates.
spk13: The next question comes from Adam Everts from LifeSci Capital. You may begin.
spk02: Great. Good morning. Thanks for the update. I know it's getting late here. On the topic of market expansion, I was hoping for a clarification on the types of patients who might not be in the OxyBait market today. You know, are these patients who are unwilling to take a twice-nightly product, patients whose physician hasn't been receptive to Oxibate products thus far, something else, a combination, just a little more info there.
spk07: Yeah, thanks, Adam. Richard, do you want to take that?
spk05: Yeah, hey, Adam, great question. So I think it's all of the above from what you've mentioned there. You know, even within the existing Oxibate prescribers today, there are patients that are just not getting an Oxibate treatment anymore. Some of that may be coming from the physician themselves, but oftentimes it's coming from the patients not wanting to take a therapy twice at night. So, you know, within those sort of 6,000 current prescribers, there is, you know, we do see room to expand the utilization there. But as Greg mentioned earlier, with the product profile of once at bedtime FT2 and 8, we are definitely seeing the feedback from people who just, physicians who just didn't want to prescribe the current formulations. that our clinical profile may attract them to want to do this as well. Subsequently, on the patient side, it's very similar where those people who have been exposed, they've been offered, we have research that says up to around 40% of patients who have been offered aid twice at night, OxyBait, just didn't want to take it. So we have a little bit more to learn there, but we definitely sort of see both the potential growth opportunity within the physician offices and clearly directly within the patient base directly as well.
spk02: Fantastic. Appreciate that. And one more very quick one. You know, as we speak with physicians about high sodium versus low sodium, you know, there's certainly a minority of patients who might need a low salt version due to comorbidities. But it seems like perhaps a more common reason to switch is taste. High sodium obviously being salty and then low sodium actually being sweet because there's some sweetener added there. Maybe can you comment on the taste of FT-218 and how it might fit regarding this?
spk07: Richard, you want to – you've tried a placebo. You want to give your personal experience?
spk05: Well, Adam, I'm more of a pizza guy than a chocolate guy myself, so that's just me personally. But, you know, also we do know from the mix-off version it does have sucralose. It has a different taste. Whether or not that's perceived to be better or worse, it's probably more of an individual judgment here. So we think the taste itself hopefully doesn't become a deterrent. I think for a lot of the previous oxidative experience patients, the saltiness shouldn't be anything new. But we also have heard from patients who have tried the low salt, mixed salt version as well, that the taste was different. And for some that was different, okay. And for some that was different, I didn't like that either. So I think the field is going to continue to grow, but we don't necessarily see taste as a major deterrent or potentially at this stage a major opportunity. We think it's really going to come down to much more of our overall clinical profile, but once at bedtime therapy.
spk07: Yeah, I'll just add to that, Adam, that, you know, we've had over, you know, we've had, you know, hundreds of patients on our therapy. We've got a large number on in our open label extension now. And taste hasn't been a reason for discontinuation. And let's remember that You know, our formulation is a proprietary ratio of IR and CR, and those CR beads are coated. So we're not releasing sodium in the mouth, in the taste buds, for a portion of our formulation, depending upon the strength and the dose that's being administered. So our product, FT218, has a different texture because we do have, you know, micro particles in there. But I also, as someone who's also taken the placebo drug, It's certainly not as salty as I expected it to be, and I, too, am a pizza man over chocolate.
spk11: Hey, Greg, just one comment. If you look at the poster for the discrete choice experiment, which there's a link to in the press release, taste was included as one of the attributes that people were asked about, and as well as, you know, once nightly dosing being a stronger preference than... than being the strongest preference, stronger preference than low sodium in overall product choice. Taste came very, very low down on that list as a driver of preference.
spk07: Yeah, thanks, Doug. Great, great comment.
spk13: And our next question will come from the line of Jason Gerbery from Bank of America. You may begin.
spk03: Okay, guys, thanks for squeezing me in. I wanted to come back to the payer discussion, so I guess is it fair to say what underpins your confidence regarding outlook for access is that payers do understand the nuances of this jazz HICMA settlement agreement and that there won't really be a truly multisource generic price point offered in the marketplace. And as we fast forward to, say, next year, HICMA has some options, presumably, to launch its own true generic, although I think that's debatable. But nonetheless, is that really, in your minds, the critical negotiation point with payers? Are they still maybe holding out hope that there's going to be true kind of generic pricing in the market? Or do you think that this settlement dynamic is pretty appreciated on their end when you talk to them? Thanks. Richard, do you want to answer?
spk05: Yeah. You know, I think, Jason, as Greg said earlier, I think from what the payers have heard, what we've heard from the payers, what they understand of the agreement is, once again, this is being viewed as a little bit more like a line extension as opposed to a quote-unquote new generic coming into this marketplace. So we haven't really heard, you know, obviously it's not in the marketplace yet, but we really haven't heard of anything that would substantially sort of change the way step edits are actually processed as far as the Hikma product is concerned. Also, we are aware that they would likely be using the same sort of infrastructure for the REMS and everything from what Jazz has set up so far as well. So we don't really sort of see that as sort of the main driver. Clearly for us, upon approval and upon full launch, Our goal is to get as many of the appropriate patients onto once at bedtime after two and eight. So, you know, we believe that will probably be a good defense in regards to any other generic community marketplace. But also at the end of the day, we'll keep coming back to our fundamental value proposition that for patients and clinicians, this value proposition is quite different. And we believe that the payers will have to acknowledge the fact that, at least for those patients who are doing well on once-at-bedtime therapy, that it would be very difficult to disrupt those patients going forward as well. So, clearly, dynamics will change. But, you know, fundamentally, our clinical value proposition is what we believe will continue to differentiate us with the payers as well. Great. Thank you. Thanks, Jason.
spk13: And I'm not showing any further questions in the queue. I'll turn it over to Greg Davis for any closing remarks.
spk07: Yeah, thank you, everybody. Just a quick comment to thank you again for joining us today and appreciate all the time and spending over an hour with us. And have a great day, and we'll look forward to any follow-up as necessary. Take care. Thank you.
spk13: And this concludes today's conference call. Thank you for participating. You may now disconnect. Everyone have a great day.
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