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spk03: results, and business update conference call. All participants will be in listen-only mode. Should you need assistance, please signal a conference specialist by pressing the star key followed by zero. After today's presentation, there will be an opportunity to ask questions. To ask a question, you may press star then one on your telephone keypad. To withdraw your question, please press star then two. Please note this event is being recorded. I would now like to turn the conference over to Gordon Dunn, CFO. Please go ahead.
spk00: Thank you and good morning. We appreciate you joining us on today's conference call. With me on the call are Dr. Michael Myers, CEO, and Denise Carter, COO. We're pleased to provide an update on our progress for the second quarter of 2023, as well as discussing our Q2 2023 financial results. Please note that our operational and financial results press release is now available on COIN's website. In keeping with our normal procedure, to begin, Michael will begin a corporate, clinical, and operational update, following which I will review our financial results. I will then hand the call back to Michael for closing comments. We will also be pleased to answer any questions at the end of the call. Before we begin, I'd like to remind everyone that statements made during this conference call will include forward-looking statements within the meeting of the Private Securities Litigation Reform Act of 1995, which involve risks and uncertainties that can cause actual results to differ materially from the information expressed or implied by these forward-looking statements. For more information regarding such risks and uncertainties, please see the risk factors outlined in the company's filings with the SEC. Any forward-looking statements are made only as of today, and we describe any obligation to update these forward-looking statements other than is required by law. Please see the forward-looking statements section in our financial results release issued this morning for more information. It is now my pleasure to turn the call over to our CEO, Michael Myers. Michael.
spk02: Thank you, Gordon, and good morning, everyone. On our last call, I reported on the very strong start Coin has had to 2023. Today, I'm very pleased to announce that this momentum carried into and through the second quarter. On May 24th, we announced that our open-labeled Nethicine Syndrome clinical trial had achieved 50% recruitment, and additional subjects have been enrolled since then. You may recall that we only dosed the first subject in this study in March of this year, so we are very pleased with the pace of recruitment overall. Our other Nefferson Syndrome clinical study is on track to have recruited the majority of subjects this month, and it is extremely encouraging how high the interest level continues to be for participation in both studies. We announced today the availability of data from the first patient to complete our open-label clinical study, and I will provide more commentary on this later in the call. Also, during the quarter, we made substantial progress on our plans to initiate additional Nessensen syndrome clinical studies, including one in a pre-identified cohort of approximately 20 Nessensen patients in the Middle East, as well as an ex-US study in pediatric subjects. We believe these additional clinical trials will supplement and enhance the data package that is currently being generated by our ongoing studies here in the US. As a leading company in this space, we are fully committed to generating a compelling body of clinical evidence to support global regulatory approval for QRX003 as a safe and effective treatment for Nettleton syndrome. During the quarter, we also continued to engage actively with Queensland University of Technology, or QUT, in Australia. COIN has two ongoing research programs with QUT, one for scleroderma and the other for Nettleton syndrome. The active ingredient in the LASA program has a different mechanism of action to our own QRx003, and we believe this product potentially has complementary features to QRx003. As previously outlined, while both of these programs are still at an early stage of development, we continue to believe that they are important components of our product portfolio and we look forward to their advancement into clinical testing. Both research programs are being conducted in Australia, where COIN is able to take advantage of a 43.5% tax rebate from the Australian government, as well as a more rapid advancement into human clinical testing than is possible here in the US or in Europe. During the quarter, we made tangible progress on our plans to initiate clinical testing of QRX003 in additional indications beyond Nephitan syndrome. These indications include peeling skin syndrome, sham syndrome, and palmoplantar keratoderma, none of which there are any approved treatments for. We hope to provide further updates on our progress in the near future including timing of initiation of proof of concept clinical testing in patients. During our last call, I also updated everyone on our M&A strategy. As discussed, given our strong balance sheet and the potential access to additional capital, we are acutely focused on expanding our product portfolio via acquisition, licensing, or other means. We are primarily interested in late stage assets in the rare and often disease space that are underpinned by strong clinical data and highly favorable commercial opportunities based on readily identifiable competitive strengths. While there can be no guarantees that a transaction will be consummated, I can tell you that a number of discussions continued to advance significantly throughout the quarter and we are optimistic that COIN will enter into at least one such transaction by the end of the year. We look forward to keeping you updated on our progress during this very exciting time for COIN. Also on our last call, I noted that another company has filed an IND with the FDA and received a study may proceed letter to initiate the clinical development of their product as a potential treatment for Nessuson syndrome. As of this morning, following a review of the clinicaltrials.gov website, it appears that this study has not yet been initiated and COIN remains the only pharmaceutical company to be actively conducting clinical studies in Nessuson syndrome under an open investigational new drug application, or IND. We and our eight global commercial partners continue to be excited by the extent of the commercial opportunity that obtaining the first regulatory approval for a treatment for this disease represents. Turning now to our ongoing clinical studies in netizens syndrome patients. Today, we had the privilege of announcing the availability of clinical data from the first subject to complete testing in our open-label study. I'm very pleased to be able to tell you that this data is positive across all measured endpoints. You may recall that in this open-label study, subjects are currently receiving off-label systemic treatment and will continue to do so in conjunction with QRx003 for the duration of the study. It is worth noting that all subjects in the study have been treated with off-label systemic therapy for at least one year, and in a number of cases, for multiple years. Notwithstanding this ongoing and long-term systemic therapy treatment, all patients recruited into the study demonstrated clear symptoms of Nessuson syndrome, including compromised skin and poor rhesus or itch. As a result of this long-term systemic therapy, subjects recruited into the study provided a natural baseline for us to assess if treatments with QRX003 over a duration of 12 weeks would lead to any therapeutic improvement across a number of assessed clinical endpoints. And so it was for the first subject who completed testing in the study. Despite the long-term systemic therapy, the subject exhibited classic symptoms of Nathanson syndrome on entry into the study. However, I'm very pleased to say on completion of the study for this subject, the QRX003 treatment area was deemed to be fully clear both by the clinical investigator as well as by a well-recognized visual scoring system. Furthermore, the subject also expressed a very favorable impression of QRX003 across a number of important metrics. Importantly also, on completion of the study, this subject's pruritus, RH, at the QRX003 treatment area was deemed to be negligible. Bear in mind that one of the primary reasons for people with Nessuson syndrome to go on systemic therapy is for symptomatic relief from their pruritus. For this particular subject, despite having been on systemic therapy for well over a year, it was only after 12 weeks of treatment with QRX003 that their pruritus had decreased to a negligible level. Although this data represents an important milestone for COIN as it is the first clinical evidence of the potential efficacy of QRX003 in Nessuson syndrome, I do want to strongly caution that it is from a single patient only and care should be taken not to read too much into this data. As other patients in the study reach the same point, we plan to provide additional updates in due course. As mentioned earlier, our ongoing double-blindness study is now on track to have a majority of subjects recruited this month. We are frequently asked if the open-label study is cannibalizing subjects from the double-blindness study, and I can tell you this is not the case at all. Both studies are recruiting independently of each other, and feedback from the Nessington community is that the availability of two such distinct clinical trial options allows patients the flexibility to enroll in a study that is best suited to their own ongoing treatment regimen. I also want to highlight that in both studies to date and across all subjects tested, QRX003 is demonstrating an exemplary safety profile. Given the highly compromised skin that Nethicin patients have, this is very encouraging news and further bolsters our confidence in the potential for QRX003 to become the first product to safely and effectively treat this disease. With that on our operational progress, let me turn it over to Gordon now to discuss our second quarter financial results.
spk00: Thank you, Michael. As of June 30, our cash and marketable securities was approximately $15.4 million compared to $17 million as of March 31, which we continue to expect will be sufficient to fund our operations into late 2024. Our net loss for the second quarter was $2.1 million compared to $2.7 million for the second quarter of 2022. The decrease was primarily due to exceptionally high legal and professional fees in the second quarter of 2022. I'll now turn the call back to Michael to make some closing remarks and begin our Q&A. Michael.
spk02: Thanks, Gordon. Today marks an important milestone for COIN, and we hope for net-in-patient syndrome patients everywhere. We are extremely excited to announce the results of the very first clinical data for QRX003 in Nessus and patients. As outlined, we strongly caution, and I do want to emphasize this point, the data is just from a single subject only and may not be representative of findings from additional subjects. However, that being said, the positive nature of the data across all measured clinical endpoints is certainly encouraging. With both of our studies continuing to recruit subjects, we look forward to releasing additional data in due course. We remain diligently focused on pursuing M&A activities and are targeting executing at least one such transaction by year end. With that, operator, we are now ready for questions.
spk03: We will now begin the question and answer session. To ask a question, you may press star then one on your telephone keypad. If you are using a speakerphone, please pick up your handset before pressing the keys. To withdraw your question, please press star then two. At this time, we will pause momentarily to assemble our roster. The first question comes from Naz Rahman with Maxim Group. Please go ahead.
spk01: Hi everyone. Congratulations on the data and the progress. I'm going to talk a little more about the data you saw from the single patient. I just want to start on, can you talk a little more about the onset, the cadence, and I guess the timing of the benefits? Did you like see most of the benefits early in the trial or did you see more of the benefits towards the end of the trial? And was the timing of the different benefits or I guess improvements on the different endpoints consistent for the different endpoints, or did different endpoints sort of reach, I guess, like positive stats or positive results at different time points?
spk02: Morning, Naz. Thanks for the call, and thanks, as always, for participating. So really good questions. The benefit really started to become evident for this particular subject in the second half of the study. So, as you know, it's a 12-week dosing period, so it was really from six weeks on. Now, we're not assessing the patient every week, so it's hard to pinpoint exactly when, but certainly it was in the second half of the study, and the timing of onset of benefit was consistent across all of the endpoints. So once the benefits started to kick in, it became evident across each of the four endpoints that were being measured.
spk01: Gotcha. That was helpful. I know it's only been so long since you evaluated that first patient, but since the patient has been off therapy, did you guys conduct like a follow-up and has the patient seen any recurrence of symptoms or have you found durability?
spk02: So that's information I don't have, Naz. The way the study was set up is that, you know, the patient was enrolled in the study, featured for 12 weeks, and then, you know, they're gone. So there really isn't follow-up. Now we are looking to see if we can continue to treat patients further in an extended open label part of the study, but I don't have any information as to what the current situation with that patient is. I will tell you, though, that the product QRX003 is a competitive serine protease inhibitor, so once you discontinue treatment with it, then you know, it targets the chalocrines in the skin that are responsible for a lot of the issues that patients suffer from. Once you discontinue treatment, those chalocrines go back to being out of control. So I would strongly suspect that the treatment areas of the patient have now reversed back to the previous Nettleton syndrome symptoms that they had on entry into the study.
spk01: Got it. And my last question is somewhat of a two-parter. Once again, acknowledging that this is just data from a single patient, have these findings somewhat impacted your thinking on how you might refine or adjust, um, later studies. And also since not for 10 syndrome, um, is really like a full body disease. Are you considering potentially, um, having larger application or more application areas for, um, later studies?
spk02: Yeah. So these are really good questions. And, um, You know look we started this we went into complete uncharted territory Nobody had ever been there before and you know neither us nor the FDA had any Precedence so our thinking is starting to evolve now as Data starts to be generated first of all and importantly we're not seeing any safety signals So that that's really good news so that allows us to think you know holistically Should we increase the dose? Should we maybe look to dose twice a day? So these are discussions that we're having because now we feel like these options come into play. And with regard to full body dosing, that is something that we are talking about. I mentioned a potential study in the Middle East on pre-identified Nessus in patients. That's something that we're considering for that particular study. I don't feel like we will deviate too far from what we have agreed with the FDA for U.S. approval, but certainly for these other studies, there's a lot more scope to either increase the dose, increase the frequency, and increase the surface area that's been tested.
spk01: Once again, congratulations on the data and looking forward to your future readouts.
spk02: Thanks, guys.
spk03: The next question, and again, if you have a question, please press star then one. And the next question is from Jim Malloy, Alliance Global Partners. Please go ahead.
spk04: Hello, this is Laura calling in for Jim Malloy. Thank you for taking my questions. So with the enrollment for both the QRx003 trials moving along, what's the potential timing here for top-line data and, you know, subsequent Phase III program as well?
spk02: So we certainly will have top-line data from the open-label study by the end of the year. And I believe for the double-blinded study early next year, early in next year. So we were continuing to target regulatory approval in the early part of 2025. So we are in a position to move pretty quickly here. And as you know, the number of patients that we need for approval is quite limited. So I think those targets remain.
spk04: Got it. And then also, you mentioned the 20-patient study to be conducted in the Middle East. So just what are some of the other updates you have on the potential expansion of, you know, QRX or O3 outside the U.S., and maybe some of the timelines here as well?
spk02: Yeah, so, you know, as you know, we have established eight commercial partnerships outside of the U.S. that now span 60 countries. So our focus here is not just US and Europe, we are looking at this as a global opportunity. So the data that we're starting to generate now from these ongoing studies could be used by some of our partners to obtain approval earlier in their respective jurisdictions than, say, in the US and Europe, and also allow for entry into compassionate use or early access programs. So the generation of data now starts to open up a lot of opportunities for us, and as we continue to assess how do we build the strongest possible database for this product, we're looking at these additional studies. So we're taking a very broad-based look at this. We're in very close communication with our commercial partners, what are their needs, to obtain approval in their countries. So a lot of behind-the-scenes discussions happening, Laura, and stay tuned. We'll have further updates.
spk04: Got it. Thank you for taking the questions, and congratulations on the progress this quarter.
spk03: This concludes our question-and-answer session. I would like to turn the conference back over to Michael Myers for any closing remarks.
spk02: Thank you. Just to say I appreciate everybody turning up this morning. And as always, if you have any further questions or need any additional information, please feel free to reach out to us. We remain readily accessible. So have a great day and thank you very much.
spk03: The conference is now concluded. Thank you for attending today's presentation. You may now disconnect.
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