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13-05-2025
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Q1 2025 Mineralys Therapeutics Inc Earnings Call
Daniel Ferry; Investor Relations; LifeSci Advisors Jon Congleton; Chief Executive Officer, Director; Mineralys Therapeutics Inc David Rodman; Chief Medical Officer; Mineralys Therapeutics Inc Adam Levy; Chief Financial Officer, Company Secretary; Mineralys Therapeutics Inc Michael DiFiore; Analyst; Evercore Jin Law; Analyst; Goldman Sachs Seamus Fernandez; Analyst; Guggenheim Securities Rami Katkhuda; Analyst; LifeSci Capital Operator Good afternoon, ladies and gentlemen, and welcome to the Mineralys' first-quarter 2025 earnings conference call. (Operator Instructions) This call is being recorded on Monday, May 12, 2025. I would now like to turn the conference over to Dan Ferry. Please go ahead. Daniel Ferry Thank you, operator. We'd like to welcome everyone joining us today for our first quarter 2025 conference call. Earlier this afternoon, we issued a press release providing our first quarter 2025 financial results and business updates. A replay of today's call will be available on the Investors section of our website approximately 1 hour after its completion. After our prepared remarks, we will open the call for Q&A. Before we begin, I would like to remind everyone that this conference call and webcast will contain forward-looking statements about the company. Actual results could differ materially from those stated or implied by these forward-looking statements due to risks and uncertainties associated with the company's business. These forward-looking statements are qualified by the cautionary statements contained in today's press release and our SEC filings, including our annual report on Form 10-K and subsequent filings. Please note that these statements reflect our opinions only as of today, May 12, 2025, and as except as required by law, we specifically disclaim any obligation to update or revise these forward-looking statements in light of new information or future events. I would now like to turn the call over to Jon Congleton, Chief Executive Officer of Mineralys Therapeutics. Jon Congleton Thank you, Dan. Good afternoon, everyone, and welcome to our first quarter 2025 financial results and corporate update conference call. I'm joined today by Adam Levy, our Chief Financial Officer; and Dr. David Rodman, our Chief Medical Officer. I'll begin with an overview of the business, then Dave will discuss our clinical programs and recent milestones, followed by Adam to review our first quarter financial results before we open up the call for your questions. This has been an exciting past few months for Mineralys as our team delivered on several clinical milestones to make significant progress across our entitlement pipeline. The most highly anticipated of these accomplishments was the simultaneous announcement of positive top line data from the pivotal trials Launch-HTN in Advance-HTN, which is understand in uncontrolled and resistant hypertension subjects. We were pleased to announce in March that both trials successfully achieved statistical significance and were clinically meaningful in Emery efficacy end points and demonstrated a favorable safety and tolerability profile. Detailed results from the Advance-HTN trial were also published in the New England Journal of Medicine, and presented in a late-breaking presentation at the American Cardiology's ACC '25 meeting. The Launch-HTN data has been accepted for a late-breaking presentation at the European Society of Hypertension on May 24 with a planned future publication. Each of these exciting outcomes helps to underscore the strength of these clinical data and the potentially transformative nature of a to help people achieve their blood pressure goal and potentially reduce their cardiovascular risk. The positive efficacy, safety and tolerability data from these 2 pivotal trials, along with the data from our Target-HTN Phase II trial of lorundrostat are key elements of our planned new drug application to the FDA. We continue to believe that this regulated aldosterone is not adequately addressed with currently available RAS directed therapeutics, including mineral corticoid receptor antagonist. These results we have seen with lorundrostat reinforce the need for a new Aldostero-directed therapeutic approach. The Transform-HTN open-label extension trial is evaluating the safety and efficacy of lorundrostat long-term use will be an important aspect of lorundrostat's profile and a critical component of our new drug application. We anticipate discussing the results from the Advance, Launch, Target and Transform HTN trials as well as the Explore-CKD trial with the FDA at a pre-NDA meeting in the fourth quarter of 2025, during which forward for an NDA submission and potential approval of lorundrostat. We look forward to providing updates on this program throughout the remainder of 2025. We're very optimistic about the interest physicians have on lorundrostat overall clinical profile based on the pivotal data, especially given the double-digit absolute reduction in systolic blood pressure. Supporting our excitement around the market opportunity from lorundrostat are the data we collected in a survey fielded in March, which evaluated the data from the Launch-HTN and Advance-HTN trials with cardiologists and primary care physicians. The results from that survey showed if lorundrostat is approved, 95% of the physicians are likely to prescribe lorundrostat broadly for hypertension and specifically in the third and fourth line position. This intent to prescribe is based on the health care professionals interpretation of the efficacy data relative to what they currently have available for uncontrolled and resistant hypertension as well as lorundrostat safety and tolerability profile. The overall results speak to the desire for innovative solutions that physicians want in their treatment armamentarium to address uncontrolled and resistant hypertension. Our two ongoing proof of con trials address advanced chronic kidney disease, specifically those with uncontrolled hypertension as well as obstructive sleep apnea with nocturnal hypertension. These trials are designed to enhance and extend the lorundrostat profile in hypertension subjects with comorbid conditions, largely driven by inadequately controlled blood pressure in dysregulated aldosterone. We've made steady progress with both trials since the beginning of 2025, and anticipate announcing top line data from Explore-CKD trial later this quarter. We're also pleased to announce the appointment of Eric Warren as Chief Commercial Officer. Eric brings approximately [30] years of experience in the pharmaceutical industry, during which he has developed a breadth of commercial and partnering expertise, focusing primarily on cardiometabolic and acute care medicine. He started his career as a pharmacist and then joined Merck & Company where we went on to hold commercial positions of increasing responsibility for almost 2 decades. In addition, Eric has held commercial leadership roles at Sanofi and Nabriva and was most recently the Chief Commercial Officer of the Therapeutics. As the Chief Commercial Officer of Mineralys will lead our commercial strategy as we prepare for the potential FDA approval of lorundrostat and support our partnering ambitions in the U.S. and ex U.S. markets. In March, we completed a public equity financing that raised gross proceeds of approximately $201.2 million before deducting fees and expenses. This financing contributed meaningfully to the strength of our balance sheet. Now to provide more color on our clinical pipeline and recent milestones, I'll turn the call over to Dave. David Rodman Thank you, Jon, and good afternoon, everybody. As Jon mentioned, our team has been had an exciting few months with the advancement of our clinical programs. I'll start by summarizing the top line results of the pivotal Phase III Launch-HTN trial, which randomized 1,083 subjects in North America and Europe who had failed to achieve the U.S. guidelines specified blood pressure targets despite having been provided on multidrug antihypertensive regimen. The trial, which tested lorundrostat in a real-world clinical context met its primary and secondary endpoints with highly statistically significant, clinically meaningful placebo-adjusted reduction, installed blood pressure as well as in the observed change in blood pressure that is conventionally used by prescribing physicians to assess response to antihypertensive therapy. At week 6, the primary end point, the 50-milligram once daily lorundrostat arm demonstrated a 9.1-millimeter of mercury placebo-adjusted reduction in systolic blood pressure, and a 16.9 meter mercury reduction in observed systolic blood pressure. At week 12, the reduction in systolic BP was maintained with the point estimate being greater than that observed at week 6. 11.7 millimeters of mercury and 19 millimeters of mercury for placebo-adjusted and observed changes, respectively. Reductions in blood pressure and this magnitude is linked to significant reduction in overall cardiovascular risk and the incidence of major adverse cardiovascular events. The Launch-HTN trial confirmed expected modest on-target increase in serum potassium that accompanies the therapeutic benefit in individuals with inadequately controlled hypertension as well as an overall safe and well-tolerated profile. The incidence of any potential measurement over 6 millimole per liter in the Launch-HTN trial in (inaudible) milligram arm was 1.1% in placebo in active and 0.7% in placebo. The prespecified rate, excluding falsely elevated or factitious hyperkalemia was comparable to placebo with the demonstrated incidences being 0.6% and 0.4%, respectively. While quantitative comparisons between different clinical trials are difficult, the incidence of moderate or severe hyperkalemia of approximately one-half of 1% compares quite favorably with most prior reports of mineralocoid receptor antagonist tested in a similar clinical context. The Launch-HTN global pivotal trial is the largest aldosterone on synthase inhibitor trial reported to date and the benefit of risk profile compares quite favorably with previously reported smaller trials of the 3 other aldosterone synthase inhibitors that have been tested in hypertensive individuals. Now turning to the Advance-HTN trial. Here, we tested the effect of lorundrostat in the clinical context and hypertension intensive individuals who are the most refractory to current standard of care, and often referred to hypertension specialists. The trial used highly rigorous criteria for enrollment and randomized -- randomization designed to mirror best practice care provided in the most advanced hypertension referral centers, maximization of conventional best practice 2 and 3 drug treatment regimens along with active monitoring of compliance, we're used to document and confirm the existence of uncontrolled or resistant hypertension. The results from the trial in the 50-milligram once-daily lorundrostat arm were highly statistically significant. The 7.9 millimeter mercury reduction in placebo-adjusted systolic blood pressure and 15.4 millimeter mercury reduction in observed systolic blood pressure measured by 24-hour ambulatory blood pressure were observed at the prespecified 12-week visit. Lorundrostat demonstrated a favorable safety and tolerability profile with modest on-target changes in serum potassium, sodium and EGFR and a low discontinuation rate. This trial was designed and conducted in partnership with the comprehensive hypertension center at the Cleveland Clinic and their C5 research team. Results were presented by the Co-Director of the Cleveland Clinic Hypertension Clinic, Dr. Lu Lasan, in a late-breaking session at the American College of Cardiology's ACC '25 meeting and published in the New England Journal of Medicine on May 8. As was reported in the New England Journal of Medicine Paper, the Advance-HTN trial per protocol confirmed incidence of hyperkalemia over 6 millimole per liter in the 50-milligram arm was 2.1%. Given the high dose of olmesartan, a potent long-acting arm, which also elevates serum potassium, we feel that this incidence of serum potassium greater than 6 millimole per liter has an acceptable benefit risk profile appropriate for the use in these patients. Okay. Now turning to our other programs, Explore-CKD and Explore-OSA Phase II proof-of-concept trials. Both of these trials are designed to provide data that augments the anti-type pretensive protocol of lorundrostat by profiling the an efficacy of lorundrostat in these 2 special populations of hypertensive individuals. During the first quarter, we announced the completion of enrollment in the Explore-CKD Phase II trial. This trial evaluates the safety and efficacy of lorundrostat for treatment of hypertension in subjects with an eGFR from 30 to 90 and at least 200 milligrams of UACR despite receiving stable treatment with an ACE inhibitor or an ARB as well as an SGLT2 inhibitor. Hypertension and associated hypertensive nephropathy is a leading cause of kidney damage alone and in combination with other obesity associated comorbidities. This is another area with great unmet medical aldosterone synthase inhibition with lorundrostat has the potential for transformative benefit to patients. In this trial, the primary outcome measure is change in systolic blood pressure during a 4-week treatment period relative to that seen in a 4-week placebo treatment period in the same individuals. The key mechanism of kidney damage in hypertensive nephropathy is elevated blood pressure glomerular hyper perfusion, scarring and reduction of the number of glomeruli available to filter the blood. Changing prutinuria is being assessed in this trial as well. In contrast to CKD due to diabetes and metabolic syndrome, where proteinuria is a useful surrogate endpoint. Individuals with predominant hypertensive nephropathy tend to have modest levels of proteinuria, change in blood pressure, along with acute physiological reduction in eGFR and rather than changing proteinuria may be a more useful outcome measure for a Phase II trial in this population. In the first quarter of 2025, we announced initiation of the Explorer-OSA Phase II trial to evaluate the effect of lorundrostat in treatment of moderate to severe obstructive sleep apnea, blood pressure increases significantly as arterial oxygenation falls during upper airway obstruction at night. By dosing lorundostated bedtime, we believe we will suppress the majority of aldosterone produced during sleep, while maintaining 24-hour blood pressure control. Episodes of nocturnal hypertension are underdiagnosed and lack a demonstrated highly effective treatment. The current treatment armamentarium is limited to weight loss and the use of positive airway pressure. We believe that neither is sufficiently effective at minimizing the impact of OSA on major adverse clinical outcomes. In summary, we have now demonstrated the clinically meaningful benefit risk profile of lorundrostat in individuals with aldosterone mediated hypertension. We are focused both on moving lorundrostat towards an NDA submission as well as exploring its use in prevalent comorbidities such as OSA hypertensive nephropathy, for which normalizing aldosterone production may result in meaningful clinical benefits. I'll now turn the call over to Adam to review our financial results for the first quarter of 2025. Adam Levy Thank you, Dave. Good afternoon, everyone. Today, I will discuss elect portions of our first quarter 2025 financial results. Additional details can be found in our Form 10-Q which will be filed with the SEC today, May 12. We ended the quarter with cash, cash equivalents and investments of $343 million as of March 31, 2025, compared to $198.2 million as of December 31, 2024. We believe that our current cash, cash equivalents and investments will be sufficient to fund our planned clinical trials and regulatory activities as well as support corporate operations into 2027. R&D expenses for the quarter ended March 31, 2025, were $37.9 million, compared to $30.8 million for the quarter ended March 31, 2024. The increase in R&D expenses was primarily due to increases of $4.8 million in preclinical and preclinical costs and $2.8 million in compensation expense resulting from additions to headcount, increases in salaries and accrued bonuses and increased stock-based compensation, partially offset by $0.5 million in lower clinical supply, manufacturing and regulatory costs. G&A expenses were $6.6 million for the quarter ended March 31, 2025, compared to $4.6 million for the quarter ended March 31, 2024. The increase in G&A expenses was primarily due to $1.2 million of compensation expense resulting from additions to headcount, increase in accrued bonuses and increased stock-based compensation and $0.7 million in higher professional fees. Total other income net was $2.2 million for the quarter ended March 31, 2025, compared to $3.9 million for the quarter ended March 31, 2024. The decrease was primarily attributable to decreased interest earned on our investments in money market funds and U.S. treasuries. Net loss was $42.2 million for the quarter ended March 31, 2025, compared to $31.5 million for the quarter ended March 31, 2024. The increase was primarily attributable to the factors impacting the company's expenses described above. With that, I'll ask the operator to open the call for questions. Operator? Operator (Operator Instructions) Michael DiFiore, Evercore. Michael DiFiore Congrats on all the progress. Just two for me. With regards to the CKD trial, in the past, you said that these patients are so sick that physicians will readily accept some level of hyperkalemia if it means that largesat will improve their blood pressure. So I guess the question is, will it be the max level of Grade 2 hyperkalemia that would be acceptable if lorundrostat were to yield a high single-digit placebo-adjusted SBP reduction? And then I have a follow-up. Jon Congleton Yes, Mike, just quick response. So I don't know that we categorized what would be an acceptable level. I think what's key and critical is we talk to specialists and our advisers who are treating these patients with hypertension and more advanced kidney disease. They're really looking at providing a benefit to the BP as well as relieving or improving the kidney function overall. I think these specialists tend to be predominantly nephrologists are more comfortable with higher level of capacity meetings within these patients. They have a means to manage that. They've got tools to use that -- they're also more likely to modulate other background treatments. In other words, if they're getting to be production with lorundrostat, they may reduce the dose of ACE or arm. So the take with us is within export, you get a clear sense of the safety, characterize the efficacy with this drug and knowing the full well, they're providing the benefit on both BP as well as kidney function in these subjects is what the specialists who are treating these patients predominantly are looking for. Michael DiFiore Got it. That's helpful. And my final question is like despite cadrostats, shorter half-life and lesser selectivity for aldosterone synthase inhibition relative to rent. It still showed a high single-digit percent SBP reduction over 14 weeks in their Phase II CKD trial. So I guess, should we expect similar efficacy and safety with lorundrostat? Jon Congleton Yes, Mike, I think it's too hard to hedge what we expect to see. I think we would anticipate seeing the clinically meaningful reduction in BP. I think the profile for lorundrostat has been well characterized now with 3 successful studies from Target-HTN, Advance and Launch. But it's too early to hedge what we'd anticipate seeing, but I would anticipate certainly a clinically meaningful reduction and then we'll see how the data evolves as far for those other hemodynamic characteristics. Operator Richard Law, Goldman Sachs. Jin Law Great. And congrats on the process from me as well. So a couple of questions from me. Can you discuss how the overall, like the Explore-CKD study fit in the strategy for were submission with Launch and Advance. My understanding is that the study is important to provide clinical support for patients below EGFR 45. And it will be great to hear your latest thinking on this and if that has evolved. And I have a follow-up. Jon Congleton Yes, Rich, thanks for the question. We're certainly excited about the benefit risk profile that's emerged now with lorundrostat with the successful completion of the Advance study and the Launch study, seeing double-digit reduction in BP with a really acceptable safety tolerability profile. The submission of the renters NDA will be inclusive of all 3 of those studies, as I noted, as well as the transform open-label extension, and Explore-CKD will be a component of that. Really, the biggest driver of Explore-CKD was related to informed blood pressure response in subjects with an EGFR down to 30 as well as going with a lower dose of 25 milligrams QD. And so it will be a component. I think it's going to be part of the totality of evidence of lorundrostat that will go into the NDA. I don't know if I could opine at this point as far as the specific language that will be included in the label from Explorer, but it's certainly a part of the total package what we have in the dialogue with the agency on. Jin Law Great. Fantastic. And then -- so we saw in the New England Journal publication that the patients who have the potassium levels greater than 6 have a much lower average eGFR compared to the rest of the population. What is -- I mean, in your view, like what is the typical EGFR between like such study population in EXPLORE CKD study and dosed in a general hypertension study, like the 1 in your pivotal study program. like BI, the CKD study is that a good benchmark in terms of patient population? Or is this a different population from that? Jon Congleton Rich, I'm sorry to do this. Can you rephrase your question? I just want to make sure I'm answering what you're looking for? Jin Law Yes. So in your New England Journal publication, patients who have the higher potassium levels greater than 6, they all have like lower than average eGFR compared to the rest of the population. So the question here is that how do we think about sort of the differences between the EGFR in your Explore-CKD study, compared to the Advance and Launch? And like what would be a good benchmark terms of the type of patient that would -- that -- in terms of the eGFR level, for your CKD study? Jon Congleton All right. Thanks, Rich. I appreciate that. Yes, I think that's why we're doing the Explore-CKD study. We know the eGFR, eGFR launch was higher than that in advance. Advance was a more high-risk population truly uncontrolled, truly confirmed resistant hypertension. They had a lower eGFR. I think you're alluding to what Luke shared at the ACC about the subjects above 6 had a mean of about 58. As far as how tight is the correlation between eGFR and risk and hypercare, I think we need more data and more evidence, but it's part of why we're doing the Explore-CKD trial. Looking at subjects going down to an eGFR 30. We know they have the risk of potential more challenges in managing electrolyze that's why we're testing the 25-milligram QD that we believe is an effective dose of lorundrostat. But as far as the correlation, I think that's something that will continue to unfold Dave, if you've got some additional thoughts, please. David Rodman Rich, good question. And how are you doing -- the -- when you talk about studies like this, the outliers are, in some ways, more important than the means, right? So the mean was above 60, say, for the people who didn't have any good incidents. It was a little bit lower and they had it. In this trial, what we're really looking for those individuals who are in that 30 to 45 range, maybe on the lower side and saying, what happens to them. Not that this is an issue other than giving guidance to clinicians for who to keep an eye on and probably who to give a potassium binder, if needed? Or as Jon said, back off on the art and see if you can maintain the same blood pressure. So it's a guidance, it's what we call a special population profiling study, and we anticipate looking just as much an outer as we do about means in that trial. Jin Law Got it. Very helpful. And then just one last question. Similar to Explore-CKD, do you expect to include data from the Explore-OSA in your found package? Jon Congleton I think, Rich, it's fair question. I think it's too early to opine on that. We haven't guided on top line data. We're excited about that study to address a significant unmet need within that resistant hypertension OSA population, but it's too early to comment what or would that not be included in the discussions with the FDA. Operator Seamus Fernandez, Guggenheim. Seamus Fernandez Great. So Jon, I think on the last discussion call, you mentioned that as many as 47,000 physicians could actually be appropriate for promotion in the uncontrolled and resistant hypertension opportunity. And then you also at ACC emphasized that the opportunity may sit a little bit more initially in the sort of fourth line hypertension opportunity. Can you just help us understand how does the sort of intersection of that broad physician base intersect with your view of the needs of a partner in that context? And what are you really looking for in the context of either a partner or something perhaps more strategic or an opportunity to actually start advancing the opportunity to promote on your own? Jon Congleton Yes. The 47,000, Rich, those that maybe hadn't heard before. So we did a significant project about a year ago with IQVIA with about [1 million] prescription claims within that. And when you basically narrowed down, where does 50% of the prescribing come from for third line or later priming, there's about 47,000 doctors that account for about half of that prescribing and a significant portion of the influence on the other 50%. And from our standpoint, there's a very efficient commercial model, particularly with kind of clinical profile that runestat has now demonstrated to go out and target the 47,000 prescribers and generate significant value. But as we've talked about in the past, partnering for us is inclusive of U.S., but certainly global, looking for partners they can help optimize the opportunity of lorundrostat U.S. because we have no intentions of creating Mineralys commercial entities stand-alone outside of United States. So finding a partner can help maximize that opportunity ex U.S. but then really fully tap into the opportunity in the United States as well. And that would basically mean some level of overall with the targeted physicians that we've talked about, but certainly coverage of those outside of those 47,000 that we target. And in fact, that target may be a little bit smaller as we think about an initial launch of lorundrostat, fourth line is probably going to be the ideal place to go. That's where there's minimal benefit with existing treatments beyond aldosterone directed therapeutics. We know spironolactone is thought to be valuable there, but it's greatly underutilized. I think our clinical program to date where we've targeted those subjects failing to get to goal on to more med shows the value of an atosteron-directed treatment that physicians are going to want to work, patients are going to want to take and persist with. So we think there's significant opportunity there. We think we could tap into a significant portion of those prescribers, but having a partner clearly is going to help us maximize the value of the asset in the United States. Seamus Fernandez Great. And maybe just one follow-up. Can you just remind us what gating factors are to sort of finalizing and filing the NDA specifically? Jon Congleton Yes, happy to do that. First and foremost, recently very pleased with the benefit risk profile that we continue to see with this molecule. Now with the 2 active portions of the pivotal program completed. As we've stated before, the unlabeled extension is a critical aspect of that. If you think about when the last subjects enrolled and launched in advance that was at the end of October last year, we would anticipate also just completing the 52-week open label by Q1 of next year. Now we don't need to have all of those subjects to enable the filing. But we need the -- certainly, a majority of those subjects through 52 weeks before we'd be comfortable with the NDA. But that's part of what we'll have a dialogue with the FDA in Q4, as we've discussed in the pre-IND meeting. And so it will be both the pit programs for Advance and Launch will be part of the target data, the Explore-CKD data. And then a portion of that open-label extension will be informative for that pre-NDA meeting that will then have better guidance for timing of an NDA submission. Operator Tim Anderson, Bank of America. This is Alice on for Tim. I just want to check, can you hear me okay? Jon Congleton Yes, we can. Okay. Perfect. Just following on from Seamus' questions on partnering. Could you talk about any early discussions you may have had so far? And what are the limiting factors that a partner may be looking for? So we're going to have the full data from Launch and the top line CKD study very soon. But do potential partners need to wait for the outcome of the pre-NDA meeting, for example, as well as the AstraZeneca Baxters at for data? And then I have a follow-up. Jon Congleton Sure. So to date, we haven't given updates on our partnering discussion, but we do continue to believe that a partner or multiple partners will be a part of our story, and we'll keep you updated as appropriate. Okay. And then as to the economy references a $5 billion peak sales for baxdrostat. I'm curious how are you thinking that you can best leverage a partner in order to realize this sort of potential with lorundrostat. For example, does it involve developing fixed dose combinations or other indications and things like that? Jon Congleton Yes. Thanks, Alex. There's clearly great deal of unmet need in this space. We're focused exquisitely right now in hypertension, but we know there's utility for an ideal aldosterone-directed treatment beyond that. That's why we're looking at the adjacencies because there's such an overlap in all of these card renal metabolic syndromes that have either hypertension or diabetes kind of at the central point. And so we think there is significant unmet need. There's significant value to provide to patients to help reduce their BP, which is the leading modifiable risk factor for cardiovascular risk. But moving from hypertension into adjacencies, such substructive sleep apnea, hypertensive nephropathy. As you heard Dave speak about, we think basically generate significant value for us. As we have partnering dialogues, as I've spoken about in the past, part of that is partnering from a commercial perspective. But -- for those that have a shared vision, it also could be development partnerships as well. Looking at some of these adjacent areas, such as heart figure or CKD. Again, we know that aldosterone plays a role across the spectrum, and having what we believe to be a leading ASI gives us significant opportunity to tap into that value. Operator Annabel Samimy, Stifel. This is Jed on for Annabel. I have two questions. First is -- at what point do you think that guideline -- hypertension guidelines would in start including Launch and added HTN data? Is there any possibility that it could be updated before you guys would theoretically launch? Jon Congleton Yes, I appreciate the question. I don't know that we can opine on when the timing will be specifically. I think we can only look at historical precedents. And I think the various guideline committees when faced with new valued innovations have been responsive to try to guide their constituents on how they should think about and integrate these new innovations into their treatment paradigm. So it's too early to opine. But it's -- it's a fair question. That's why we went to the what we did in Advance-HTN because I think it fundamentally addresses the kind of questions that these guideline committees wish to have. And that is not only in maybe an existing background treatment, but when you get to truly high-risk patients like we tested in advance, what does the profile physicians could expect? And how would guideline committees inform their communication and their constituents. Got it. And my other question is related to Explore-CKD. What do you think is the primary if you're looking for here? Are you looking for safety in the CKD population with concomitant drugs, inhibitors and ARBs? And then do you expect efficacy to generally be in the line of what you saw in -- or are there some nuances with that patient population that we should know? Jon Congleton Yes. I'll just reiterate what Dave had said with a profiling the study like the safety is a key element of the analysis and what we expect from a clinical benefit standpoint would be clinically meaningful reduction in blood pressure. I think that's been well characterized in the 3 studies to date. That's what we would anticipate to see in this population, and then providing additional information about the 25-milligram QD dose. Operator Mohit Bansal, Wells Fargo. This is Fatima on for Mohit. And congrats on all the recent progress. So on the hypertension readout, you've previously mentioned plans for subgroup Can you elaborate on those plans for which subgroups you're focused on and the time line for presentation of that data? And can you talk about how it could potentially help physicians select patients for lorundrostat? And if it could also influence placement of lorundrostat into treatment guidelines? Jon Congleton Yes, thank you for the question. As you know, we've to prespecify analysis of populations that may be unique responders to lorundrostat. You saw some of that data within the Advance-HTN, ACC as well as Nigam presentation and publications, respectively. I would anticipate seeing something similar with Launch-HTN. I think to date, what we've seen and it's frankly beneficial for prescribers, whether failing to achieve goal on 2 meds or 3 meds on controller resistant hypertension, you're seeing a pretty profound reduction in regardless of gender, age, race, number of background medications. And so it creates a predictable response that physicians can anticipate when using lorundrostat. We're going to continue to investigate and dive into the data. I mean what we've shared to date has been very informative about the value from a clinical reduction and safety standpoint. But there's a great deal of data we're going to continue to dig into within launch and advance and eventually Explore-CKD to really continue to further inform -- All right, what is the ideal population to respond to this drug. But to date, we've seen great responses across a multitude of subsets. And then on the OSA trial, how are you thinking about this 4-week endpoint? How it aligns with expected time lines for improvements in the hypoxia index and nocturnal blood pressure? And what magnitude of action would you consider to be clinically meaningful and also competitive in the context of the data reported with GLPs, for example? David Rodman Really good questions. Let me try to take those one at a time. So the first question was 4 weeks. What might we see? So as far as APMEA popular index, the primary mechanism through which our drug will work is the diuretic effect and reducing the amount of salt and water overload. Because when you lay down at night, the excess salt water, the fluid shifts up. It's called roster cattle redistribution into your upper body and neck. That benefit is accrued within a few weeks. And so by 4 weeks, we would expect to see the benefit on Apnea Hypopnea Index. As you know, around a 50% reduction has been seen with the Lilly study similar with the APMEA study of a different mechanism we're powered down to about 30%, and these are small trials. So we would ultimately be observing where we are in that range. So let me just say something. Treating at the FDA hypopnea index is important, but the main risk for adverse outcomes is this extreme burst of hypertension, these spikes that you see at night when those things happen, and we're going to be doing the first trial using sub-1-second measurements -- blood pressure over the course of an entire night. So we'll be able to look at how well does this drug actually reduce the risk for adverse clinical outcomes. In many ways, that's a more important endpoint. However, Apnea Hyponea Index and patient reported performance metrics are the current guidance from the agency for approval. So we're going down both of those paths. This is on antihypertensive drug, and it's a sodium depleter. We expect to see benefits on both, but both are going to be meaningful. So I can't tell you for sure if apnea hypoxia index is 30, but we see a terrific impact on nighttime blood pressure, maybe restoration, nighttime dipping. We'll be the only ones with those data at that point will be reporting them. And I think that will be really an important milestone in studying this disease. Operator Rami Katkhuda, LifeSci Capital. Rami Katkhuda I guess I just wanted to confirm a statement that Dr. Robin made that patients with hypertensive nephropathy may have more modest levels of proteinuria. I guess, is the patient population in Explore-CKD similar to that of the Bohringer study? Or are there other key differences in enrollment criteria? And I guess is that 37% placebo-adjusted UACR reduction with monotherapy a fair bar here? David Rodman Okay. Those are good questions. And if we think about this, and this has happened in other diseases, Chronic kidney disease is a syndrome, right? It can be autoimmune and you want to use an anti IGA, which has been very effective. If you -- it's in the context of obesity and diabetes, it's from metabolic syndrome, and that is the one that's associated with a fair amount of proteinuria, even nephrotic syndrome, which is an extreme of that. What we're looking at -- some of these patients may have high levels of proteinuria, but we anticipate that will not be the majority in this trial. So a different subset and it's actually a different subset of CKD. These people have scarring of their external part of their -- the cortex of their kidney, loss of these glomeruli from this water hammer effect of the pounding of blood pressure. For these people, getting their blood pressure down to 125 or 130 is not all they need. They need lower blood pressures than those to truly protect the globe live at our left. And so we're going to be looking at that and continuing to explore the possibility of differentiating on that basis as we get into this clinic kidney disease space. It's not our primary objective per se because we are going after hypertension broadly. We're now since we've proven, that's a highly safe and effective drug for uncontrolled and resistant hypertension. Now we're starting to go to the very high unmet need subpopulations, which right now is we consider to be hypertensive for nephropathy and OSA. Jon Congleton And Rami, just to add to that, the distinctions between the studies. I think the baseline is like BP in that study was low that is our inclusion criteria. So to Dave's point, we really are retreating the with GFR hypertension -- think that does create a distinct population between the two studies. Operator There are no further questions at this time. I would hand over the call to Jon Congleton for closing remarks. Please go ahead. Jon Congleton Thank you, operator. Mineralys Therapeutics, we're committed to improving the lives of patients with cardiorenal metabolic diseases. Uncontrolled and resistant hypertension are significant unmet medical needs impacting more than 20 million patients in the U.S. alone. Our Launch in Advanced studies reinforce the differentiated clinical profile of lorundrostat versus agents that are typically used in the third and fourth-line treatment positions and the quantitative research that we've done supports the commercial potential. We're excited for key upcoming milestones and look forward to sharing updates with you in the coming quarters. Thank you all. Thank you for joining for joining us today. And with that, we'll close the call. Thank you, everyone. Operator Ladies and gentlemen, this concludes today's conference call. 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Yahoo
12-05-2025
- Business
- Yahoo
Mineralys Therapeutics Reports First Quarter 2025 Financial Results and Provides Corporate Update
– Pivotal Advance-HTN and Launch-HTN trials successfully achieved statistical significance in primary efficacy endpoints and demonstrated a favorable safety and tolerability profile – – Anticipate Explore-CKD Phase 2 trial to deliver topline data in Q2 2025 – – Initiated Explore-OSA Phase 2 Trial in Q1 2025 – – Conference call today at 4:30 p.m. ET – RADNOR, Pa., May 12, 2025 (GLOBE NEWSWIRE) -- Mineralys Therapeutics, Inc. (Nasdaq: MLYS), a clinical-stage biopharmaceutical company focused on developing medicines to target hypertension, chronic kidney disease (CKD), obstructive sleep apnea (OSA) and other diseases driven by dysregulated aldosterone, today announced financial results for the first quarter ended March 31, 2025, and provided a corporate update. 'We are pleased to have recently announced positive topline results from our pivotal trials, Launch-HTN and Advance-HTN, that evaluated lorundrostat's efficacy and safety as a potential treatment for patients with uncontrolled or resistant hypertension. The Advance-HTN late-breaking presentation at the American College of Cardiology meeting and the recent publication in the New England Journal of Medicine underscore the strength of our clinical data and the potentially transformative nature of lorundrostat. With the success of our two pivotal trials, we are working toward submitting our new drug application with a pre-NDA meeting with the FDA anticipated in the fourth quarter of 2025,' stated Jon Congleton, Chief Executive Officer of Mineralys Therapeutics. 'We are excited to announce the appointment of Eric Warren as our Chief Commercial Officer. Eric's proven leadership skills, extensive cardiovascular experience and track record of success, will be invaluable to Mineralys as we solidify our commercial and partnering strategy.' Recent Clinical Highlights and Upcoming Milestones Pivotal Launch-HTN Phase 3 Trial – The trial met its primary endpoint in evaluating the efficacy and safety of lorundrostat for the treatment of subjects with uncontrolled hypertension (uHTN) or resistant hypertension (rHTN) as add-on therapy, who fail to achieve blood pressure control on their existing medications. The trial reported that the lorundrostat 50 mg dose achieved a 16.9 mmHg reduction in systolic blood pressure, and a 9.1 mmHg placebo-adjusted reduction (p-value < 0.0001), as assessed by automated office blood pressure at week six. This benefit was sustained with potential further reduction through week 12, with a 19.0 mmHg reduction in automated office systolic blood pressure and an 11.7 mmHg placebo-adjusted reduction (p-value <0.0001). The clinical safety results, including adrenocorticotropic hormone (ACTH)-stimulated and serum cortisol, change in serum potassium, serum sodium, and estimated glomerular filtration rate (eGFR), as well as incidence of hypotension, from the trial support a favorable benefit-risk profile in a 'real world' setting. The incidence of hyperkalemia (serum potassium >6.0 mmol/L) at the scheduled study visit was 1.1% and 1.5% in the 50 mg and 50 to 100 mg arms, respectively. After exclusion of factitious test results, the incidence of confirmed hyperkalemia was 0.6% and 1.1%, respectively. The Launch-HTN trial has been accepted as a late-breaking presentation at the 2025 European Society of Hypertension Meeting on Hypertension and Cardiovascular Protection, which is being held in Milan, Italy on May 23-26, 2025. Pivotal Advance-HTN Trial – Detailed results from the Advance-HTN trial were recently presented in a late-breaking presentation at the American College of Cardiology's ACC.25 meeting and published on May 8th in the New England Journal of Medicine. The trial met its primary endpoints in evaluating the efficacy and safety of lorundrostat for the treatment of confirmed uHTN or rHTN. The trial reported that the lorundrostat 50 mg dose cohort achieved a 15.4 mmHg absolute reduction in systolic blood pressure and a 7.9 mmHg placebo-adjusted reduction (p-value = 0.001), as assessed by 24-hour ambulatory blood pressure monitoring at 12 weeks. Lorundrostat demonstrated a favorable safety and tolerability profile, with modest changes in serum potassium, serum sodium and eGFR, and a low discontinuation rate. The incidence of hyperkalemia (serum potassium >6.0 mmol/L) at the scheduled study visit was 5.3% and 7.4% in the 50 mg and 50 to 100 mg arms, respectively. After exclusion of factitious test results, the incidence of confirmed hyperkalemia was 2.1% and 3.2%, respectively. These results reinforce lorundrostat's favorable benefit-risk profile in a high-risk population that would typically be treated by specialists rather than general practitioners. Transform-HTN Open-Label Extension Trial – The Company's ongoing open-label extension trial allows subjects to continue to receive lorundrostat and the Company to obtain additional safety and efficacy data. Explore-CKD Phase 2 Trial – Enrollment has been completed and topline data are anticipated in the second quarter of 2025. The trial is designed to evaluate the safety and efficacy of lorundrostat when added to background treatment with an ACE inhibitor or ARB and a SGLT2 inhibitor for the treatment of hypertension in subjects with Stage 2 to 3b CKD (eGFR greater than or equal to 30 mL/min/1.73m2 ) and albuminuria. Explore-OSA Phase 2 Trial – The Company initiated the trial in the first quarter of 2025, which will evaluate the safety and efficacy of lorundrostat in the treatment of overweight and obese subjects with moderate-to-severe OSA and hypertension. Expanded Management Team – Appointed Eric Warren as Chief Commercial Officer. Mr. Warren brings more than three decades of commercial leadership experience across multiple healthcare segments with a heavy emphasis on cardiovascular disease. Mr. Warren will lead the Company's commercial strategy and support future partnering opportunities. Strengthened Balance Sheet – On March 18, 2025, the Company completed a public equity financing for gross proceeds of approximately $201.2 million, before deducting fees and expenses. The Company reported a total of $343.0 million of cash, cash equivalents and investments as of March 31, 2025. First Quarter 2025 Financial Highlights Cash, cash equivalents and investments were $343.0 million as of March 31, 2025, compared to $198.2 million as of December 31, 2024. The Company believes that its current cash, cash equivalents and investments will be sufficient to fund its planned clinical trials and regulatory activities, as well as support corporate operations, into 2027. Research and Development (R&D) expenses for the quarter ended March 31, 2025 were $37.9 million, compared to $30.8 million for the quarter ended March 31, 2024. The increase in R&D expenses was primarily due to increases of $4.8 million in preclinical and clinical costs and $2.8 million in compensation expense resulting from additions to headcount, increases in salaries and accrued bonuses and increased stock-based compensation, partially offset by $0.5 million in lower clinical supply, manufacturing and regulatory costs. General and Administrative (G&A) expenses were $6.6 million for the quarter ended March 31, 2025, compared to $4.6 million for the quarter ended March 31, 2024. The increase in G&A expenses was primarily due to $1.2 million in higher compensation expense resulting from additions to headcount, increases in salaries and accrued bonuses and increased stock-based compensation, and $0.7 million in higher professional fees. Total other income, net was $2.2 million for the quarter ended March 31, 2025, compared to $3.9 million for the quarter ended March 31, 2024. The decrease was primarily attributable to decreased interest earned on our investments in money market funds and U.S. treasuries. Net loss was $42.2 million for the quarter ended March 31, 2025, compared to $31.5 million for the quarter ended March 31, 2024. The increase was primarily attributable to the factors impacting the Company's expenses described above. Conference Call The Company's management team will host a conference call at 4:30 p.m. ET on Monday, May 12, 2025. To access the call, please dial 1-800-717-1738 in the United States or 1-646-307-1865 outside the United States. A live webcast of the conference call may be found here. A replay of the call will be available on the 'News & Events' page in the Investor Relations section of the Mineralys Therapeutics website (click here). About Hypertension Having sustained, elevated blood pressure (or hypertension) increases the risk of heart disease, heart attack and stroke, which are leading causes of death in the United States. In 2022, more than 685,000 deaths in the United States included hypertension as a primary or contributing cause. Hypertension and related health issues resulted in an average annual economic burden of about $219 billion in the United States in 2019. Less than 50% of hypertension patients achieve their blood pressure goal with currently available medications. Dysregulated aldosterone levels are a key factor in driving hypertension in approximately 30% of all hypertensive patients. About CKD CKD, which is characterized by the gradual loss of kidney function, is estimated to affect more than 10% of the global population and is one of the leading causes of mortality worldwide. According to the U.S. Centers for Disease Control and Prevention (CDC), an estimated 1-in-7 (15%) of U.S. adults have CKD. Diabetes and hypertension are responsible for approximately two-thirds of CKD cases. Early detection and treatment can often keep CKD from getting worse. When CKD progresses, it may eventually lead to kidney failure, which requires dialysis or a kidney transplant to maintain life. About OSA OSA is characterized by repetitive overnight hypoxic episodes and subsequent sleep fragmentation due to a complete or partial collapse of the upper airway. Moderate OSA is defined as having between 15 and 30 breathing pauses (apnea or hypopnea events) per hour of sleep, while severe OSA indicates more than 30 breathing pauses per hour. OSA impacts almost one billion people globally, including 425 million moderate-to-severe cases. Around 80% of adults with OSA are undiagnosed. As of 2015, undiagnosed OSA is estimated to cost the United States approximately $149.6 billion annually from comorbid disease, workplace accidents, motor vehicle accidents and loss of workplace productivity. Between 30-50% of adults with hypertension have OSA, and this number increases to between 70-80% in adults with rHTN. Additionally, untreated moderate-to-severe OSA increases the risk of rHTN. Along with hypertension, OSA is a major risk factor of cardiovascular disease, type-2 diabetes mellitus and stroke. About Lorundrostat Lorundrostat is a proprietary, orally administered, highly selective aldosterone synthase inhibitor being developed for the treatment of uHTN or rHTN, as well as CKD and OSA. Lorundrostat was designed to reduce aldosterone levels by inhibiting CYP11B2, the enzyme responsible for its production. Lorundrostat has 374-fold selectivity for aldosterone-synthase inhibition versus cortisol-synthase inhibition in vitro, an observed half-life of 10-12 hours and demonstrated a 40-70% reduction in plasma aldosterone concentration in hypertensive subjects. In a Phase 2, proof-of-concept trial (Target-HTN) in uncontrolled or resistant hypertensive subjects, once-daily lorundrostat demonstrated statistically significant and clinically meaningful systolic blood pressure reduction in both automated office systolic blood pressure measurement and 24-hour ambulatory systolic blood pressure monitoring. Adverse events observed were a modest increase in serum potassium, decrease in eGFR, urinary tract infection and hypertension, with one serious adverse event possibly related to study drug being hyponatremia. About Mineralys Mineralys Therapeutics is a clinical-stage biopharmaceutical company focused on developing medicines to target hypertension, CKD, OSA and other diseases driven by dysregulated aldosterone. Its initial product candidate, lorundrostat, is a proprietary, orally administered, highly selective aldosterone synthase inhibitor that Mineralys Therapeutics is developing for the treatment of cardiorenal conditions affected by dysregulated aldosterone, including hypertension, CKD and OSA. Mineralys is based in Radnor, Pennsylvania, and was founded by Catalys Pacific. For more information, please visit Follow Mineralys on LinkedIn and Twitter. Forward-Looking Statements Mineralys Therapeutics cautions you that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. The forward-looking statements are based on our current beliefs and expectations and include, but are not limited to, statements regarding: the potential therapeutic benefits of lorundrostat; the Company's expectation that aldosterone synthase inhibitors with an SGLT2 inhibitor may provide additive clinical benefits to patients; the Company's expectation that Advance-HTN and Launch-HTN may serve as pivotal trials in any submission of a new drug application (NDA) to the U.S. Food and Drug Administration (FDA); the Company's ability to evaluate lorundrostat as a potential treatment for CKD, OSA, uHTN or rHTN; the planned future clinical development of lorundrostat and the timing thereof; and the expected timing of commencement and enrollment of patients in clinical trials and topline results from clinical trials. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in our business, including, without limitation: topline results that we report are based on a preliminary analysis of key efficacy and safety data, and such data may change following a more comprehensive review of the data related to the clinical trial and such topline data may not accurately reflect the complete results of a clinical trial; our future performance is dependent entirely on the success of lorundrostat; potential delays in the commencement, enrollment and completion of clinical trials and nonclinical studies; later developments with the FDA may be inconsistent with the feedback from the completed end of Phase 2 meeting, including whether the proposed pivotal program will support registration of lorundrostat which is a review issue with the FDA upon submission of an NDA; the results of our clinical trials, including the Advance-HTN and Launch-HTN trials, may not be deemed sufficient by the FDA to serve as the basis for an NDA submission or regulatory approval of lorundrostat; our dependence on third parties in connection with manufacturing, research and clinical and nonclinical testing; unexpected adverse side effects or inadequate efficacy of lorundrostat that may limit its development, regulatory approval and/or commercialization; unfavorable results from clinical trials and nonclinical studies; results of prior clinical trials and studies of lorundrostat are not necessarily predictive of future results; macroeconomic trends and uncertainty with regard to high interest rates, elevated inflation, tariffs, and the potential for a local and/or global economic recession; our ability to maintain undisrupted business operations due to any pandemic or future public health concerns; regulatory developments in the United States and foreign countries; our reliance on our exclusive license with Mitsubishi Tanabe Pharma to provide us with intellectual property rights to develop and commercialize lorundrostat; and other risks described in our filings with the Securities and Exchange Commission (SEC), including under the heading 'Risk Factors' in our annual report on Form 10-K, and any subsequent filings with the SEC. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and we undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Contact:Investor Relationsinvestorrelations@ Media RelationsTom WeibleElixir Health Public RelationsPhone: (1) 515-707-9678Email: tweible@ Mineralys Therapeutics, Inc. Condensed Statements of Operations (in thousands, except share and per share data) (unaudited) Three Months Ended March 31, 2025 2024 Operating expenses: Research and development $ 37,879 $ 30,754 General and administrative 6,568 4,608 Total operating expenses 44,447 35,362 Loss from operations (44,447 ) (35,362 ) Interest income, net 2,239 3,853 Other income (expense) (3 ) 1 Total other income, net 2,236 3,854 Net loss $ (42,211 ) $ (31,508 ) Net loss per share attributable to common stockholders, basic and diluted $ (0.79 ) $ (0.70 ) Weighted-average shares used to compute net loss per share attributable to common stockholders, basic and diluted 53,163,551 44,900,755 Mineralys Therapeutics, Inc. Selected Financial Information Condensed Balance Sheet Data (amounts in thousands) (unaudited) March 31, December 31, 2025 2024 Cash, cash equivalents and investments $ 343,026 $ 198,187 Total assets $ 354,941 $ 205,903 Total liabilities $ 13,386 $ 14,646 Total stockholders' equity $ 341,555 $ 191,257
Yahoo
23-04-2025
- Business
- Yahoo
Mineralys Therapeutics Announces Publication of Pivotal Phase 2 Advance-HTN Results in the New England Journal of Medicine (NEJM)
– Significant blood pressure reductions among patients with uncontrolled or resistant hypertension treated with lorundrostat reinforce key role of dysregulated aldosterone in disease onset and progression – – Detailed results from the second pivotal Phase 3 Launch-HTN trial to be presented at an upcoming medical conference and published in a peer-reviewed publication – RADNOR, Pa., April 23, 2025 (GLOBE NEWSWIRE) -- Mineralys Therapeutics, Inc. (Nasdaq: MLYS), a clinical-stage biopharmaceutical company focused on developing medicines to target hypertension, chronic kidney disease (CKD), obstructive sleep apnea (OSA) and other diseases driven by dysregulated aldosterone, today announced that the New England Journal of Medicine (NEJM) published the detailed results from the Company's pivotal Phase 2 Advance-HTN trial, the first of two pivotal trials evaluating lorundrostat in patients with uncontrolled hypertension (uHTN) or resistant hypertension (rHTN). The full manuscript is titled, 'Lorundrostat Efficacy and Safety in Patients with Uncontrolled Hypertension,' and is featured in the April 23, 2025 issue of NEJM. The key data from the publication showed that lorundrostat 50 mg demonstrated a 15.4 mmHg absolute reduction and a 7.9 mmHg placebo-adjusted reduction (p=0.001), in 24-hour ambulatory blood pressure at week 12. Lorundrostat worked equally well in those taking two baseline medications and those taking three or more, and in both men and women as well as in white and black patients. Lorundrostat demonstrated a favorable safety and tolerability profile, with modest changes in potassium, sodium and eGFR. 'The publication of our Advance-HTN trial results in the New England Journal of Medicine is a significant milestone that underscores both the strength of our clinical data and the potentially transformative nature of this new class of medicines that could help address dysregulated aldosterone, an unaddressed, key driver of hypertension,' stated Jon Congleton, Chief Executive Officer of Mineralys Therapeutics. 'Prior studies have shown that even modest reductions in systolic blood pressure can lead to a substantial decrease in the incidence of major cardiovascular events. The blood pressure reductions with lorundrostat observed in the Advance-HTN trial are particularly meaningful given the well-established correlation between elevated blood pressure, dysregulated aldosterone production and cardiovascular risk.' 'The significant blood pressure lowering with lorundrostat 50 mg in the Advance-HTN trial was seen in patients treated by specialists who were taking an optimized standardized antihypertensive regimen – those patients with true uncontrolled or resistant hypertension that desperately need new options to lower their blood pressure,' stated Luke Laffin, M.D., co-director of the Center for Blood Pressure Disorders in the Heart, Vascular & Thoracic Institute at Cleveland Clinic and the study's lead author. 'Currently available therapies to treat hypertension do not decrease aldosterone production in the body, and we know aldosterone dysregulation is a driving factor in the blood pressure elevation of many of our patients. The findings reinforce the critical role of aldosterone in the pathogenesis of hypertension and the potential of lorundrostat to address unmet medical needs facing patients with uncontrolled or treatment-resistant disease.' The NEJM publication of the detailed Advance-HTN results follows a late-breaking presentation of the data at the American College of Cardiology's Annual Scientific Session & Expo (ACC.25) in Chicago on March 29, 2025, and the announcement of positive topline results from both Advance-HTN and Launch-HTN earlier in March. Mineralys plans to provide additional data from the pivotal Phase 3 Launch-HTN at an upcoming medical conference and in a peer-reviewed publication. Additionally, the ongoing Transform-HTN open-label extension trial allows subjects to continue to receive lorundrostat and obtain additional safety and efficacy data. About Hypertension Having sustained, elevated blood pressure (or hypertension) increases the risk of heart disease, heart attack and stroke, which are leading causes of death in the U.S. In 2020, more than 670,000 deaths in the U.S. included hypertension as a primary or contributing cause. Hypertension and related health issues resulted in an average annual economic burden of about $219 billion in the U.S. in 2019. Less than 50% of hypertension patients achieve their blood pressure goal with currently available medications. Dysregulated aldosterone levels are a key factor in driving hypertension in approximately 30% of all hypertensive patients. About Lorundrostat Lorundrostat is a proprietary, orally administered, highly selective aldosterone synthase inhibitor being developed for the treatment of uHTN or rHTN, as well as CKD and OSA. Lorundrostat was designed to reduce aldosterone levels by inhibiting CYP11B2, the enzyme responsible for its production. Lorundrostat has 374-fold selectivity for aldosterone-synthase inhibition versus cortisol-synthase inhibition in vitro, an observed half-life of 10-12 hours and demonstrated approximately a 70% reduction in plasma aldosterone concentration in hypertensive subjects. In a Phase 2, proof-of-concept trial (Target-HTN) in uncontrolled or resistant hypertensive subjects, once-daily lorundrostat demonstrated statistically significant and clinically meaningful blood pressure reduction in both automated office blood pressure measurement and 24-hour ambulatory blood pressure monitoring. Adverse events observed were a modest increase in serum potassium, decrease in estimated glomerular filtration rate, urinary tract infection and hypertension with one serious adverse event possibly related to study drug being hyponatremia. About Advance-HTN The Advance-HTN trial (NCT05769608) was a randomized, double-blind, placebo-controlled Phase 2 clinical trial that evaluated the efficacy and safety of lorundrostat for the treatment of uHTN or rHTN, when used as an add-on therapy to a standardized background treatment of two or three antihypertensive medications in adult subjects. Subjects who met screening criteria had their existing hypertension medications discontinued and started on a standard regimen of an angiotensin II receptor blocker (ARB) and a diuretic, if previously on two medications, or a standard regimen of ARB, diuretic and calcium channel blocker if previously on three to five medications. Subjects who remained hypertensive despite the standardized regimen were then randomized into three cohorts and treated for twelve weeks: lorundrostat 50 mg once-daily (QD), lorundrostat 50 mg QD and an option to titrate to 100 mg QD at week four based on defined criteria, or placebo. The trial's primary endpoint was the change in 24-hour ambulatory systolic blood pressure at week twelve from baseline for active cohorts versus placebo. About Mineralys Mineralys Therapeutics is a clinical-stage biopharmaceutical company focused on developing medicines to target hypertension, CKD, OSA and other diseases driven by dysregulated aldosterone. Its initial product candidate, lorundrostat, is a proprietary, orally administered, highly selective aldosterone synthase inhibitor that Mineralys Therapeutics is developing for the treatment of cardiorenal conditions affected by dysregulated aldosterone, including hypertension, CKD and OSA. Mineralys is based in Radnor, Pennsylvania, and was founded by Catalys Pacific. For more information, please visit Follow Mineralys on LinkedIn and Twitter. Forward Looking Statements Mineralys Therapeutics cautions you that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. The forward-looking statements are based on our current beliefs and expectations and include, but are not limited to, statements regarding: the potential therapeutic benefits of lorundrostat; the Company's expectation that Advance-HTN and Launch-HTN may serve as pivotal trials in any submission of a new drug application (NDA) to the United States Food and Drug Administration (FDA); the Company's ability to evaluate lorundrostat as a potential treatment for CKD, OSA, uHTN or rHTN; and the planned future clinical development of lorundrostat and the timing thereof. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in our business, including, without limitation: topline results that we report are based on a preliminary analysis of key efficacy and safety data, and such data may change following a more comprehensive review of the data related to the clinical trial and such topline data may not accurately reflect the complete results of a clinical trial; our future performance is dependent entirely on the success of lorundrostat; potential delays in the commencement, enrollment and completion of clinical trials and nonclinical studies; later developments with the FDA may be inconsistent with the feedback from the completed end of Phase 2 meeting, including whether the proposed pivotal program will support registration of lorundrostat which is a review issue with the FDA upon submission of an NDA; the results of our clinical trials, including the Advance-HTN and Launch-HTN trials, may not be deemed sufficient by the FDA to serve as the basis for an NDA submission or regulatory approval of lorundrostat; our dependence on third parties in connection with manufacturing, research and clinical and nonclinical testing; unexpected adverse side effects or inadequate efficacy of lorundrostat that may limit its development, regulatory approval and/or commercialization; unfavorable results from clinical trials and nonclinical studies; results of prior clinical trials and studies of lorundrostat are not necessarily predictive of future results; regulatory developments in the United States and foreign countries; our reliance on our exclusive license with Mitsubishi Tanabe Pharma to provide us with intellectual property rights to develop and commercialize lorundrostat; and other risks described in our filings with the Securities and Exchange Commission (SEC), including under the heading 'Risk Factors' in our annual report on Form 10-K, and any subsequent filings with the SEC. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and we undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Contact:Investor Relationsinvestorrelations@ Media RelationsTom WeibleElixir Health Public RelationsPhone: (1) 515-707-9678Email: tweible@ in to access your portfolio
Yahoo
10-03-2025
- Business
- Yahoo
Mineralys's stock soars as hypertension pill scores in two pivotal trials
Mineralys Therapeutics's oral hypertension therapy, lorundrostat, has met the primary endpoint in two pivotal studies, boosting the company's stock price. The Phase III Launch-HTN (NCT06153693) and Phase II Advance-HTN (NCT05769608) trials evaluated the efficacy and safety of lorundrostat in patients with uncontrolled hypertension (uHTN) or resistant hypertension (rHTN). The biopharma's stock, listed on the Nasdaq exchange, rose by 65.59% on the news from a 7 March market close of $10.52 to a 10 March market high of $17.42 (correct as of 11:45am EST). The Launch-HTN trial met its primary endpoint, demonstrating a 16.9mmHg reduction in systolic blood pressure, and a 9.1mmHg placebo-adjusted reduction, after six weeks. This rose to a 19.0mmHg reduction in systolic blood pressure, and an 11.7mmHg placebo-adjusted reduction, after 12 weeks. The randomised, double-blinded, placebo-controlled trial enrolled patients who failed to achieve their blood pressure goal despite being on two to five antihypertensive medications. The Advance-HTN trial met its primary endpoint with a 7.9mmHg placebo-adjusted reduction in systolic blood pressure, assessed with 24-hour average blood pressure measurement after 12 weeks in patients treated with 50mg of lorundrostat. The randomised, double-blind, placebo-controlled study evaluated the drug as an add-on therapy to background treatment of two or three antihypertensive medications. The therapy remained safe and well tolerated in both studies, with Mineralys reporting that 14 patients in the Launch-HTN trial suffered serious adverse events (SAEs). There was also some incidence of hyperkalemia (serum potassium above 6.0mmol/L). Mineralys Therapeutics CEO Jon Congleton said: 'We have now completed three successful clinical trials demonstrating the efficacy, safety and tolerability of lorundrostat and the importance of targeting dysregulated aldosterone. We believe the clinical profile observed for lorundrostat supports the potential regulatory approval of this novel agent and its significant commercial value.' Further data from the studies will be presented at upcoming medical conferences and in peer-reviewed publications. The US-headquartered company previously announced positive data from the Phase II Target-HTN study. Mineralys is also running the Transform-HTN open-label extension trial to allow patients to continue receiving lorundrostat while generating additional safety and efficacy data. According to GlobalData forecasts, sales of lorundrostat could reach $601m in 2030. GlobalData is the parent company of Clinical Trials Arena. In November 2024, MSD's Winrevair (sotatercept-csrk) met the primary endpoint in a Phase III trial in patients with pulmonary arterial hypertension (PAH). "Mineralys's stock soars as hypertension pill scores in two pivotal trials" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site.
Associated Press
10-03-2025
- Business
- Associated Press
Mineralys Therapeutics Announces Positive Topline Results from Launch-HTN and Advance-HTN Pivotal Trials of Lorundrostat for the Treatment of Uncontrolled or Resistant Hypertension
– Launch-HTN met its primary endpoint with lorundrostat 50 mg dose achieving a 16.9 mmHg reduction in systolic blood pressure, and a 9.1 mmHg placebo-adjusted reduction (p-value < 0.0001) assessed by automated office blood pressure at week 6 – – Launch-HTN met a predefined endpoint with lorundrostat 50 mg dose achieving a 19.0 mmHg reduction in systolic blood pressure, and an 11.7 mmHg placebo-adjusted reduction (p-value < 0.0001) assessed by automated office blood pressure at end of treatment, week 12 – – Advance-HTN met its primary endpoint with lorundrostat 50 mg dose achieving a highly statistically significant 7.9 mmHg placebo-adjusted reduction assessed by 24hr ABPM at end of treatment, week 12 – – Lorundrostat demonstrated a favorable safety and tolerability profile in both pivotal trials – – Full results from Advance-HTN to be presented on March 29, 2025, at the American College of Cardiology Scientific Sessions – – Conference call today at 8:00 a.m. ET – RADNOR, Pa., March 10, 2025 (GLOBE NEWSWIRE) -- Mineralys Therapeutics, Inc. (Nasdaq: MLYS), a clinical-stage biopharmaceutical company focused on developing medicines to target hypertension, chronic kidney disease (CKD), obstructive sleep apnea (OSA) and other diseases driven by dysregulated aldosterone, today announced positive topline data from its pivotal Launch-HTN Phase 3 and pivotal Advance-HTN Phase 2 trials evaluating the efficacy and safety of lorundrostat for the treatment of uncontrolled hypertension (uHTN) or resistant hypertension (rHTN). Both trials successfully achieved statistical significance and were clinically meaningful in their pre-specified primary efficacy endpoints and demonstrated a favorable safety and tolerability profile. 'The positive results and clinically meaningful reduction in blood pressure observed in the Launch-HTN and Advance-HTN trials show us that lorundrostat has the potential to be a transformative new therapy for the approximately 15 to 20 million patients with uncontrolled hypertension in the United States,' stated Jon Congleton, Chief Executive Officer of Mineralys Therapeutics. 'We have now completed three successful clinical trials demonstrating the efficacy, safety and tolerability of lorundrostat and the importance of targeting dysregulated aldosterone. We believe the clinical profile observed for lorundrostat supports the potential regulatory approval of this novel agent and its significant commercial value. We appreciate the commitment and hard work of the clinical investigators, site staff, the Mineralys and Cleveland Clinic research teams, and especially the trial subjects who volunteered to participate in our program.' Efficacy Results The Launch-HTN trial was a global, randomized, double-blinded, placebo-controlled Phase 3 trial, which enrolled eligible adult participants who failed to achieve their blood pressure goal despite being on two to five antihypertensive medications. Launch-HTN reflects the real-world setting for clinicians by utilizing automated office blood pressure (AOBP) measurement and allowing participants to stay on their existing medications. The trial met its endpoints demonstrating clinically meaningful, statistically significant mean reduction from baseline in placebo-adjusted systolic blood pressure at week six and the benefit was sustained with potential further reduction through week 12. Launch-HTN Phase 3 Trial (automated office systolic blood pressure measure, n=1,083) Week 6 (50 mg pooled) Week 12 Absolute Reduction Placebo-Adjusted Reduction Absolute Reduction Placebo-Adjusted Reduction 50 mg -16.9 mmHg -9.1 mmHg (p<0.0001)* -19.0 mmHg -11.7 mmHg (p<0.0001) 50 to100 mg -15.7 mmHg -8.4 mmHg (p=0.0016) * Primary endpoint The change in blood pressure in response to lorundrostat in subjects using two background antihypertensives (uncontrolled) or three to five (resistant) were similar, and both were statistically significantly different from the response in those taking placebo. The Advance-HTN trial was a randomized, double-blind, placebo-controlled Phase 2 pivotal trial that evaluated the efficacy and safety of lorundrostat for the treatment of confirmed uHTN or rHTN, when used as add-on therapy to an optimized background treatment of two or three antihypertensive medications in adult subjects. The trial met its primary endpoint, with placebo-adjusted reduction from baseline in systolic blood pressure assessed with 24-hour average blood pressure measurement at week 12 of -7.9 mmHg in subjects treated with 50 mg of lorundrostat. Other prespecified outcome measures, including measures of efficacy in the dose-escalation cohort, safety and tolerability, were consistent with those observed in the Launch-HTN trial. Additional details regarding the results from Advance-HTN are embargoed until presentation on March 29, 2025, at the American College of Cardiology Scientific Sessions. Safety and Tolerability Results We believe clinical safety findings, including hypotension, serum potassium, eGFR and serum cortisol, from both pivotal trials, support a favorable benefit-risk profile. In the Launch-HTN trial there were 12 subjects (2.2%) and two subjects (0.7%) with treatment-emergent serious adverse events (SAEs) in the 50 mg and 50 mg with optional dose escalation to 100 mg arms, respectively, compared with eight subjects (3.0%) in the placebo arm. There was only one subject (0.1%) in the trial with treatment-related SAEs that occurred in the 50 mg arm. The incidence of hyperkalemia (serum potassium above 6.0 mmol/L) in the 50 mg and 50mg to 100mg arms, respectively, was 1.1% and 1.5% in the Launch-HTN trial and 5.3% and 7.4% in the Advance-HTN trial. 'The Launch-HTN study evaluating novel drug lorundrostat is one of the largest blood pressure studies in recent times and demonstrates its benefit in lowering blood pressure and its safety in a diverse group of patients whose hypertension is not well controlled,' stated Manish Saxena MBBS, Hypertension Specialist from Barts Health NHS Trust. 'Uncontrolled and resistant hypertension remains a global health concern as it continues to be the leading cause of cardiovascular deaths, heart attacks and strokes. Given today's announcement, lorundrostat could be a good treatment option for millions of patients with high blood pressure.' Mineralys plans to provide additional data from these two pivotal trials at upcoming medical conferences and in peer-reviewed publications. The ongoing Transform-HTN open-label extension trial allows subjects to continue to receive lorundrostat and generate additional safety and efficacy data. Conference Call The Company's management team will host a conference call today, March 10, 2025, at 8:00 a.m. ET. To access the call, please dial 1-877-704-4453 in the U.S. or 1-201-389-0920 outside the U.S. A live webcast of the conference call may be found here. A replay of the call will be available on the 'News & Events' page in the Investor Relations section of the Mineralys Therapeutics website ( click here). About Hypertension Having sustained, elevated blood pressure (or hypertension) increases the risk of heart disease, heart attack and stroke, which are leading causes of death in the U.S. In 2020, more than 670,000 deaths in the U.S. included hypertension as a primary or contributing cause. Hypertension and related health issues resulted in an estimated annual economic burden of about $219 billion in the U.S. in 2019. Less than 50 percent of hypertension patients achieve their blood pressure goal with currently available medications. Dysregulated aldosterone levels are a key factor in driving hypertension in approximately 30 percent of all hypertensive patients. About Lorundrostat Lorundrostat is a proprietary, orally administered, highly selective aldosterone synthase inhibitor being developed for the treatment of uHTN and rHTN as well as CKD and OSA. Lorundrostat was designed to reduce aldosterone levels by inhibiting CYP11B2, the enzyme responsible for its production. Lorundrostat has 374-fold selectivity for aldosterone-synthase inhibition versus cortisol-synthase inhibition in vitro, an observed half-life of 10-12 hours and demonstrated approximately a 70% reduction in plasma aldosterone concentration in hypertensive subjects. In a Phase 2, proof-of-concept trial (Target-HTN) in uncontrolled or resistant hypertensive subjects, once-daily lorundrostat demonstrated statistically significant and clinically meaningful blood pressure reduction in both automated office blood pressure measurement and 24-hour ambulatory blood pressure monitoring. Adverse events observed were a modest increase in serum potassium, decrease in estimated glomerular filtration rate, urinary tract infection and hypertension with one serious adverse event possibly related to study drug being hyponatremia. About Launch-HTN The Launch-HTN trial (NCT06153693) was a global, randomized, double-blinded, placebo-controlled Phase 3 trial, which enrolled eligible adult participants who failed to achieve their blood pressure goal despite being on two to five background antihypertensive medications. Eligible subjects were randomized to one of three arms: placebo, lorundrostat 50 mg once daily (QD), and lorundrostat 50 mg QD and then titrated to 100 mg QD, as needed, at week six. The primary endpoint of the trial was the change from baseline in systolic blood pressure versus placebo after six weeks of treatment, as measured by automated office blood pressure monitoring. About Advance-HTN The Advance-HTN trial (NCT05769608) was a randomized, double-blind, placebo-controlled Phase 2 clinical trial that evaluated the efficacy and safety of lorundrostat for the treatment of uHTN or rHTN, when used as an add-on therapy to a standardized background treatment of two or three antihypertensive medications in adult subjects. Subjects who meet screening criteria had their existing hypertension medications discontinued and start on a standard regimen of an angiotensin II receptor blocker (ARB) and a diuretic, if previously on two medications, or a standard regimen of ARB, diuretic and calcium channel blocker if previously on three to five medications. Subjects who remained hypertensive despite the standardized regimen were then randomized into three cohorts and treated for twelve weeks: lorundrostat 50 mg QD, lorundrostat 50 mg QD, and an option to titrate to 100 mg QD at week four based on defined criteria or placebo. The trial's primary endpoint was the change in 24-hour ambulatory systolic blood pressure at week twelve from baseline for active cohorts versus placebo. About Mineralys Mineralys Therapeutics is a clinical-stage biopharmaceutical company focused on developing medicines to target hypertension, CKD, OSA and other diseases driven by dysregulated aldosterone. Its initial product candidate, lorundrostat, is a proprietary, orally administered, highly selective aldosterone synthase inhibitor that Mineralys Therapeutics is developing for the treatment of cardiorenal conditions affected by dysregulated aldosterone, including hypertension, CKD and OSA. Mineralys is based in Radnor, Pennsylvania, and was founded by Catalys Pacific. For more information, please visit Follow Mineralys on LinkedIn and Twitter. Forward Looking Statements Mineralys Therapeutics cautions you that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. The forward-looking statements are based on our current beliefs and expectations and include, but are not limited to, statements regarding: the potential therapeutic benefits of lorundrostat; the Company's expectation that Advance-HTN and Launch-HTN may serve as pivotal trials in any submission of a new drug application (NDA) to the United States Food and Drug Administration (FDA); the Company's ability to evaluate lorundrostat as a potential treatment for CKD, uHTN or rHTN; and the planned future clinical development of lorundrostat and the timing thereof. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in our business, including, without limitation: topline results that we report are based on a preliminary analysis of key efficacy and safety data, and such data may change following a more comprehensive review of the data related to the clinical trial and such topline data may not accurately reflect the complete results of a clinical trial; our future performance is dependent entirely on the success of lorundrostat; potential delays in the commencement, enrollment and completion of clinical trials and nonclinical studies; later developments with the FDA may be inconsistent with the feedback from the completed end of Phase 2 meeting, including whether the proposed pivotal program will support registration of lorundrostat which is a review issue with the FDA upon submission of an NDA; the results of our clinical trials, including the Advance-HTN and Launch-HTN trials, may not be deemed sufficient by the FDA to serve as the basis for an NDA submission or regulatory approval of lorundrostat; our dependence on third parties in connection with manufacturing, research and clinical and nonclinical testing; unexpected adverse side effects or inadequate efficacy of lorundrostat that may limit its development, regulatory approval and/or commercialization; unfavorable results from clinical trials and nonclinical studies; results of prior clinical trials and studies of lorundrostat are not necessarily predictive of future results; regulatory developments in the United States and foreign countries; our reliance on our exclusive license with Mitsubishi Tanabe Pharma to provide us with intellectual property rights to develop and commercialize lorundrostat; and other risks described in our filings with the Securities and Exchange Commission (SEC), including under the heading 'Risk Factors' in our annual report on Form 10-K, and any subsequent filings with the SEC. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and we undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Media Relations Tom Weible Elixir Health Public Relations
