DURECT Corporation

Q2 2022 Earnings Conference Call

8/4/2022

spk01: Greetings and welcome to the Durex Corporation second quarter 2022 earnings conference call. At this time, all participants are in a listen-only mode. A brief 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 your host, Tim Papp, Chief Financial Officer. Thank you, Mr. Papp. You may begin.
spk06: Good afternoon and welcome to our second quarter 2022 earnings conference call. This is Tim Papp, Chief Financial Officer of Direct Corporation. Before beginning, I would like to remind you of our safe harbor statement. During the course of this call, we may make forward-looking statements regarding Direct's product and development, expected product benefits, our development plans, future clinical trials, or projected financial results. These forward-looking statements involve risks and uncertainties that can cause actual results to differ materially from those in such forward-looking statements. Further information regarding these and other risks can be found in our SEC filings, including our 10-K and 10-Qs under the heading Risk Factors. Let me now turn to our second quarter financials. Total revenue in the second quarter of 2022 was $2.1 million compared to $2.3 million in the second quarter of 2021. R&D expense was $8.8 million as compared to $7.4 million for the prior year. The increase was primarily due to higher clinical trial expenses for a firm and higher employee benefit costs partially offset by lower contract manufacturing costs for Lursa-Costero. SG&A expenses were $4 million in the second quarter of 2022 as compared to $3.2 million for the prior year. primarily due to higher employee benefit costs, patent expenses, and consulting expenses. At June 30th, we had cash and investments of $54.3 million as compared to $70 million at December 31st, 2021, and our cash burn during Q2 was $10.1 million. With that, let me turn the call over to Jim for an update on certain of our programs.
spk05: Thank you, Tim. Hello, everyone. Thank you for joining us today for our second quarter 2022 update. I want to take this opportunity to welcome Tim to the DIRECT team. It's a pleasure having Tim on board, and he's hit the ground running since joining as our CFO just last month. Tim brings more than 20 years of investment banking experience to DIRECT, with more than 15 years of that focused in biotechnology. This has been a busy time for DIRECT. Our primary focus at DIRECT is on completing the Phase IIb Affirmed Clinical Trial of Larcicosterol for the treatment of alcohol-associated hepatitis, or AH. We continue to add sites and enroll patients at a strong pace in AFIRM and have now reached the milestone of enrolling 170 out of the 300 patients planned for the study. Through a Type C meeting, we proposed and the FDA agreed to a modified primary endpoint for the AFIRM trial that we believe better reflects the current state of care for AAH patients. Just this week, we were issued a new patent covering Posimer out to 2,000 and 41. Under our agreement with INICOL, this triggers an $8 million milestone from INICOL to DIRECT. As I mentioned, we're primarily focused on completing enrollment in our Phase 2b AFFIRM trial for losucosterol in hospitalized patients with severe AH. AFFIRM is a placebo-controlled, double-blind, multinational study targeting 300 patients with two dosing arms and a placebo arm of 100 patients each. We are pleased with our progress on enrollment and have now reached the milestone of 170 patients dosed. Our pace of enrollment continues to accelerate, with more patients being enrolled in the second quarter of this year than in any prior quarter. We currently have over 60 sites open, including leading hospitals in the United States, Australia, the EU, and the UK. We continue to expect to complete enrollment in the middle of 2023, which will allow us to report top-line data from a firm 2023. The FDA has granted our Larciclosterol AH program fast-track designation, and a positive result in a firm could support an NDA filing. With this in mind, Larciclosterol has the potential to be the first FDA-approved treatment for AH, where there is a substantial unmet need for patients. Today, we announced that following a Type C meeting, the FDA has concurred with Direct's proposal to modify our primary endpoint in the affirmed trial. The updated primary endpoint will be the difference in outcomes, either death or liver transplant, between the treated and control patients at day 90. We believe this primary endpoint, which now includes liver transplantation in addition to mortality, better reflects the ability of a therapeutic such as Lorsucosterol to improve upon the current state of care for AH patients. As a reminder, AH is a lethal and costly disease that represents an unmet medical need with no approved therapy. AH results in about 137,000 hospitalizations per year in the United States, and hospitalized patients have a 90-day mortality rate of approximately 30%. While AH patients who are fortunate enough to receive a liver transplant generally see a survival benefit, a liver transplant also represents a serious medical event for patients. Transplanted patients face significant health consequences, including the need for immunosuppressive drugs for the rest of their lives. Therefore, we believe that including transplanted patients as well as mortality in the primary endpoint better reflects the most severe outcomes for AH patients. As the landscape for liver transplant has evolved due in part to improved treatment of HCV reducing the need for liver transplantation in these patients, We have seen some impact on the state of care for severe AH patients in recent years to include more liver transplants. Even so, there are fewer than 10,000 liver transplants performed annually in the United States, with the majority going to patients with other ailments, meaning that only a small fraction of the 137,000 AH patients who are hospitalized annually in the United States are able to receive a liver transplant. These numbers starkingly highlight the continued unmet need for treatment options in AH. AH continues to represent a significant cost burden to both the patients and the healthcare system. For those selected few patients who receive a liver transplant, the average cost is $875,000 per transplant in the United States. For the vast majority not receiving a transplant, the average cost of treating a hospitalized AH patient can range from $53,000 to $147,000. With this in mind, Larcicosterol represents a potential multibillion-dollar opportunity in the United States, while simultaneously providing substantial cost savings to the overall healthcare system. Now let's review why we are so optimistic about the use of Larcicosterol in the treatment of patients with severe AH. As I mentioned before, AH patients face a 90-day mortality rate of approximately 30%, and there is no approved treatment for this disease. Therefore, we were excited by the data from our Phase 2A trial of Lerciclosterol in moderate to severe AH patients. All 19 patients, including the 12 severe AH patients, survived. Additionally, 14 of the 19 patients were discharged in less than four days after receiving only a single IV infusion of Lerciclosterol. The prognostic scores from AH patients in this trial, including LEAL and MELD scores, bilirubin, and other biomarkers were improved as compared to baseline. Narsuclosterol was also well tolerated by all of the patients at all of the doses evaluated in the Phase 2A trial. There were no serious drug-related adverse events reported in this trial. In addition to the clinical trial results, we've generated supporting preclinical data from numerous in vivo animal models that demonstrates LARCiclosterol's protection against multi-organ failure, which is the primary driver of mortality in AH patients. LARCiclosterol's mechanism of action as an endogenous epigenetic regulator helps us to better understand the remarkable results we've observed in its impact on AH patients. LARCiclosterol binds to and inhibits the activity of three DNA methyltransferases, DNMT1, 3A, and 3B. These three enzymes regulate the epigenome by adding methyl groups to DNA in a process called DNA methylation. In one example, stressed liver cells, whose DNA was hypermethylated, had methylation levels of the DNA return closer to those observed in healthy liver cells after being treated with acyclosterol. Furthermore, prior studies of AH patients published in the medical literature have demonstrated that these DNMTs are elevated in AH patients, suggesting a mechanistic path for leuciclosterols potential benefit for these patients. And now, I'd like to move on to POSMAR, one of our last remaining legacy products. Posimer is a novel, non-opioid, sustained-release local anesthetic that is FDA-approved to provide post-surgical analgesia for up to 72 hours following arthroscopic subacromial decompression. We announced last year that we licensed the development and commercialization rights of Posimer in the United States to Intercall Pharmaceuticals. We selected Intercall as our commercial partner because of their strong commercial team and because they are focused on post-surgical pain. On August 2nd, we were issued a new patent by the U.S. Patent Office, extending our U.S. patent coverage of POSMR to 2041. Under our agreement with Intercall, this triggers an $8 million milestone to direct. We also receive a $2 million payment upon first commercial sale, which we believe is approaching. Following these two payments and the initial $4 million upfront payment, Direct is eligible for up to $122 million in additional commercial, regulatory, and intellectual property milestone payments, as well as tiered low to mid double-digit royalties on net product sales in the United States. In summary, we continue to make great strides with Affirm and have reached the milestone of 170 patients dosed with over 60 clinical sites up and running. We are on track to complete dosing the last patient in Affirm in the middle of 2023, which would enable reporting of top-line results in the second half of 2023. Our confidence that the affirmed trial will be successful is driven by our compelling Phase IIa study data, the mechanism of action of LARCIGLOS-GERAL, which ties directly into the biology of AH, our multiple preclinical animal studies where we observed a profound survival benefit in multiple relevant acute organ injury models. We expect that if we achieve a positive outcome in the affirmed trial, this could support an NDA file. Beyond AH, the mechanism of action for leucocesterol provides further scientific rationale for developing leucocesterol for other acute and chronic diseases. With that, we'd now like to take any questions you may have.
spk01: 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 that your line is in the question queue. You may press star 2 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 key. One moment, please, while we poll for questions. Our first question comes from Kristin Kluska with Cantor Fitzgerald. Please proceed with your question.
spk03: Good afternoon. This is Rick on for Kristen. Thank you for taking our questions. Given all the KOL diligence your team has done in the AH space, what can you say about how the addition of liver transplant endpoint fits in with what you've heard from physicians in the space? How do you think physicians in the space think about the kind of relative importance of the mortality and liver transplant aspects?
spk05: That's a very good question. I appreciate that. And As you can imagine, we did spend a lot of time talking with our KOLs about that, and I think I'll just let Norman address that question.
spk02: Yes, thanks very much for that question. As Jim said, we spent a lot of time with a lot of KOLs discussing it. We eventually came to the conclusion that patients that are doing very poorly, say in either arm, who would then be rescued by a transplant, looking purely at 90-day mortality gives you an artificial sense of the value of the treatment. And so we were pleased when FDA agreed with that viewpoint. And I would say the majority of our KOLs agreed with it.
spk05: That was it. I would second that.
spk03: Understood. And maybe one more. On your last call, you talked about increasing the total site target in a firm to 70 sites from 60 to 65 was the original guidance, I believe, due to the availability of certain U.S. sites that were appearing to be available. Could you give us an update on these specific U.S. sites that you were talking about here, the decision to kind of look at those and kind of where are they in the process of being up and running?
spk05: Yeah, we definitely like these sites. They were part of the NIH consortium that had a trial, unfortunately, that failed. And we've been working with these sites, and we're getting close. But once again, I think, Norman, maybe you can speak to the specifics of bringing on these additional U.S. sites.
spk02: Yes, so they were all very high-visibility sites with excellent investigators. And we are – one of them, I would – I think is probably – a matter of weeks away. A few of the others will take a little bit longer. But they're going to come on board fairly soon, and we think that they'll be very productive sites.
spk05: Yeah, these are excellent sites. It's just, unfortunately, most of them are universities and larger centers, and, you know, the process, the legal process just takes some time.
spk03: Understood. Maybe just one more really quick one about the $8 million milestone from Anna Call. Should we be thinking about that as being recognized in 3Q?
spk05: Tim, I'd like you to address that.
spk06: Yeah, that's right. We would expect that to be included in our revenue for the third quarter.
spk03: Great. That's all we have. Thank you very much. Thank you.
spk01: Our next question comes from Francois Brisebois with Oppenheimer. Please proceed with your question.
spk04: Hi, thanks for taking the questions. This is Dan on for Frank Brisebois. Just a couple from me. Regarding the primary endpoint amendment, firstly, just based on your conversations with KOLs, is there any concern about whether these endpoints are correlated in any way? And as a follow-up, The question is, if the trial is successful and approvable, is it either or endpoints, or is it both endpoints have to hit for it to be successful and approvable?
spk05: Thanks. Both endpoints are the same. It's an event, and the event would be either death or transplant. So we're considering transplant to be equivalent to death, because if a patient is You know, if they're transplanted, then the probability of them living for the next year or so is very, very high, 99 or whatever it is, you know, very high percentage. And so we wanted to make sure we were able to capture that for patients who were transplanted who otherwise might have died. And I should also let you know that in the United States, it's a more common practice to do this transplant. You know, this trial we are conducting globally, and in Europe there are many fewer people of these patients transplant, even though in the United States, not many of these patients get transplant anyway, just because of the availability of livers and the cost of this thing. But maybe Norman, if you want to add anything to that?
spk02: No, I agree. We consider the two points equivalent. In other words, people don't usually get a transplant if they're doing better. So the typical practice is the patient comes in, is doing poorly, there's a serious concern that the patient will die and therefore gets transplanted. And so that's why I think transplant is equivalent to death. Just so you know, we will also analyze death as a single endpoint, as a secondary analysis. Okay, makes sense.
spk04: And just as one more question from my end, are you able to provide any update on the timeline of when the first commercial sale of POSMO will be?
spk05: We just know that it's getting close, but we can't provide anything more than that. So we would expect it soon, but that's all I can say at this point.
spk04: Okay. Thank you. Thanks for taking the questions.
spk05: Sure. Absolutely.
spk01: Our next question is from Ed Arsley. HC Wainwright, please proceed with your question.
spk07: Hi, everyone. This is Thomas Yip asking for questions for Ed. We're very happy to see the firm's steady, continual progress in the quarter. So first question, it was just updated today, in addition to death and liver transplant and SBLO, As we all know, Leo score improvement was shown with acuclosterol earlier. What are your thoughts on this potential use in regular progress, either with the FDA or with other agencies that are XUS, and perhaps what are some potential use for Leo score, perhaps as a pretreatment screening tool?
spk05: Well, you know, LEAL score is generated seven days after the patient is in the hospital. And so LEAL is a good prognostic indicator of the patient's potential for survival post-treatment, because by that time, if they remained in the hospital the whole time, they would have received two doses of larch cyclosterol. If they went home, they would have still received one dose of larch cyclosterol. So it's a good prognostic indicator, but for this trial where, you know, we're looking at 90-day survival or transplant and the difference between that event occurring versus, you know, between the treatment groups and the placebo group. So I think Leo will be something we look at, but it won't be a primary driver of approval. Got it. I don't know if that answers your question.
spk07: Yes, it does. Yeah. Clearly, liver transplant and death are first and foremost most useful indicators. So for Affirm, and as you outlined, with the continual addition of new sites globally, what are your thoughts on your commercial approach perhaps for the ex-U.S. markets? What are some top markets in your view for now?
spk05: You know, it's a very good question. There are, you know, a large number of patients in Europe, probably equivalent or maybe a bit larger than the U.S. So it certainly is a problem on a global basis. And so it's more likely than not we won't be commercializing ourselves outside the U.S. So we are exploring, you know, possibilities there as well as we get closer, certainly. Thank you.
spk07: Got it. Perhaps one final question from us. Can you give us an update on the U.S. launch? What's the latest progress by any call there? And when should we expect that revenue to be a meaningful contribution to your top line?
spk05: Well, I think the launch is getting close. As far as the contribution, you know, all pharmaceutical sales, at least most of them, I've had a couple products in my career that go fast, but most of them are kind of a hockey step kind of growth. And then there's a trailing return. So, you know, it'll start slow and then build over time. I don't know what more to add other than that. I think, Tim, would you add anything to that?
spk06: I would just add that we don't really have any guidance from in a call on what their revenue projections look like and and they are a private company. So I'm not sure we'll have a lot of visibility into their expected revenue and how that translates into our royalties.
spk05: But the nice thing is we do have milestones on sales, and we have royalties as well that start in the low double digits and go to the mid double digits. So as their sales increase, then our return will certainly be doing the same.
spk07: Right. Understood. It's certainly a good way to ask you guys to focus primarily on the firm for now. To that end, we look forward to the progress in coming months and with the complete enrollment in next year.
spk05: Thank you. Thank you so much. Thank you.
spk01: There are no further questions at this time. I would now like to turn the floor back over to Jim Brown for closing comments.
spk05: Okay, with that, I just want to thank you all for your time today. And as always, if you have any questions, please feel free to give us a call. Thank you all and take care.
spk01: This concludes today's teleconference. You may disconnect your lines at this time. Thank you for your participation.
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