Latest news with #Phase3
Yahoo
3 days ago
- Business
- Yahoo
Biodexa Pharmaceuticals sees cash runway into 1Q26
As of May 29, 2025, the Company had access to the following cash resources: Cash at bank $5.7M; Cash in escrow for eRapa Phase 3 program $4.4M; Undrawn CPRIT grant for eRapa Phase 3 program $11.9M; Debt $0.5M. The CPRIT grant, together with Company match means the eRapa Phase 3 program is substantially funded. Based on its latest projections, the Company has sufficient working capital to fund operations into the first quarter of 2026. Issued shares, total voting rights, market capitalization As of May 29, 2025, there were 50,506,308,922 ordinary shares, equivalent to 5,050,630 American Depositary Shares, outstanding. The Company holds zero shares in treasury and therefore the number of voting rights is the same as the number of ordinary shares outstanding. Based on the closing share price on May 29, 2025 of $1.06 per ADS, the Company's market capitalization was $ Phase 3 study of eRapa in FAP is in the final stages of implementation. It will be a double-blind placebo-controlled trial in 168 patients, randomized 2:1 drug / placebo. It is expected the study will be conducted in approximately 30 clinical sites across the US and Europe. The US component of the study will be conducted by LumaBridge, based in San Antonio, Texas and the European component will be conducted by Precision for Medicine. All planned US sites and the majority of European sites have been identified. Recruitment is expected to begin in the next few weeks. Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See Insiders' Hot Stocks on TipRanks >> Read More on BDRX: Disclaimer & DisclosureReport an Issue Biodexa Pharmaceuticals Releases 2024 Annual Report and Announces AGM Biodexa Pharmaceuticals Plans Share Capital Reorganization and New Share Allotment Biodexa Pharmaceuticals Secures Additional $3M Grant for eRapa Phase 3 Program Biodexa Pharmaceuticals announces award of additional $3M grant from CPRIT Biodexa Pharmaceuticals Lowers Warrant Exercise Price to Boost Capital
National Post
3 days ago
- Business
- National Post
BeOne Medicines Presents New SEQUOIA Study Results Reinforcing BRUKINSA's Differentiated Profile with or without Venetoclax in Frontline CLL at ASCO 2025
Article content BRUKINSA plus venetoclax demonstrated high response rates and a favorable safety profile across CLL patient types in SEQUOIA Arm D, including those with high-risk del(17p) mutational status Article content Article content At 5-year follow-up of SEQUOIA Arm C, BRUKINSA monotherapy showed sustained OS and PFS benefit in hard-to-treat del(17p) CLL patients versus historical data Article content SAN CARLOS, Calif. — BeOne Medicines Ltd. (NASDAQ: ONC; HKEX: 06160; SSE: 688235), a global oncology company, today will present new data from the Arm C and D cohorts of the pivotal, global Phase 3 SEQUOIA trial of BRUKINSA ® (zanubrutinib). The findings underscore the strong and consistent efficacy of BRUKINSA across CLL patient types, including high-risk mutation status. These data will be presented in two rapid oral presentations at the American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago, IL. Article content Data from the Arm D of SEQUOIA demonstrate that treatment with BRUKINSA plus venetoclax has the potential to drive progression-free survival and overall deep and durable responses across the frontline CLL patient spectrum, including patients with high-risk mutational status. The best undetectable minimal residual disease (uMRD) rate in peripheral blood at a sensitivity level 10 -4 was 59%. These efficacy responses observed in Arm D, despite the high proportion of high-risk patients enrolled, are in line with recent fixed-duration studies in fitter, healthier patient populations. Additionally, 11 patients in Arm D were able to discontinue treatment early due to meeting uMRD-guided stopping criteria, and 9 patients remain in ongoing clinical remission with sustained uMRD (1 patient discontinued study while in clinical remission), allowing them to remain treatment-free. In patients without del(17p) and TP53 mutations, 43% achieved uMRD by cycle 16 and 60% by cycle 28. These data were published today in the Journal of Clinical Oncology. Article content 'While many first-line CLL studies have excluded patient populations with high-risk disease features, BeOne included those patients in SEQUOIA,' said Lai Wang, Ph.D., Global Head of R&D at BeOne. 'Nearly 88% of patients with del(17p) and /or TP53 treated with BRUKINSA plus venetoclax remain progression-free at 36 months, which represents an unprecedented outcome for a doublet regimen in this difficult-to-treat patient population. These new SEQUOIA data reinforce BRUKINSA's versatility across the spectrum of CLL patients and reflect BeOne's commitment to progressing a pipeline built to meet unmet patient needs and elevate the standard of care.' Article content Arm D Highlights (Abstract 7009) SEQUOIA Arm D investigated BRUKINSA plus venetoclax in 114 patients with treatment-naïve (TN) CLL / small lymphocytic lymphoma (SLL) with or without del(17p) and/or TP53 high-risk mutations. At a median follow-up of 31.2 months, the combination induced a high 24-month progression-free survival (PFS) rate of 92% (95% CI, 85-96%) and an impressive overall response rate (ORR) of 97%. The 24-month overall survival (OS) rate was 96% (95% CI, 90%-98%). Of those patients with del(17p) and/or TP53 mutations, 94% were progression-free at 24 months and 87.6% were progression-free at 36 months. Article content The safety profile of BRUKINSA was consistent with the results of prior studies with no new safety signals identified. Article content 'The zanubrutinib and venetoclax combination achieved deep, durable responses across risk groups, including patients with TP53 mutations, with a generally manageable safety profile. Notably, several patients were able to discontinue treatment and maintain remission, highlighting the potential for time-limited therapy with meaningful disease control,' said Mazyar Shadman, M.D., M.P.H., Associate Professor and Innovators Network Endowed Chair, Medical Director, Cellular Immunotherapy and the Bezos Family Immunotherapy Clinic at Fred Hutch Cancer Center. 'Generating data to inform future CLL treatment strategies that allow for both continuous therapy and planned time off treatment is essential, particularly for high-risk patients who are the most likely to succumb to this disease.' Article content Arm C Highlights (Abstract 7011) Arm C of the SEQUOIA study investigated BRUKINSA monotherapy in patients with TN CLL / SLL and del(17p) mutations and is the largest prospective cohort of CLL/SLL patients with del(17p). At a median follow-up of over 5.5 years (65.8 months), most patients remained progression-free. Notably, at 60 months, 72.2% of patients who received BRUKINSA remained progression-free (95% CI, 62.4, 79.8). When adjusted for the impact of the COVID-19 pandemic, 73.0% of patients in the cohort remained progression-free (95% CI, 63.3, 80.6) at 60 months. The 60-month OS rate was 85.1% (95% CI, 76.9, 90.6) and 87.0% (95% CI, 79.0, 92.1) when adjusted for COVID-19. At the time of data cut-off, the ORR was 97.3%, and 62.2% of patients were still receiving treatment with BRUKINSA. Article content The safety profile of BRUKINSA was consistent with the results of prior studies with no new safety signals identified. Article content For additional information about our presence at the 2025 ASCO Annual Meeting, please visit our meeting hub: Article content About Chronic Lymphocytic Leukemia Chronic lymphocytic leukemia (CLL) is a life-threatening cancer of adults. It is a type of mature B-cell malignancy in which abnormal leukemic B lymphocytes (a type of white blood cells) arise from the bone marrow and flood peripheral blood, bone marrow, and lymphoid tissues. 1,2 CLL is the most common type of leukemia in adults, accounting for about one-third of new cases. 2,3 Approximately 20,700 new cases of CLL will be diagnosed in the U.S. in 2024. 3 Article content About 50% of CLL patients have high-risk genetic features – including del(17p), TP53 or unmutated IGHV – that may limit the effectiveness of some treatments (e.g. chemotherapy) and increase the likelihood of disease progression. 4,5 Article content About SEQUOIA SEQUOIA ( NCT03336333) is a randomized, multicenter, global Phase 3 trial designed to evaluate the efficacy and safety of BRUKINSA in patients with treatment-naïve (TN) chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). The trial consists of three cohorts: Article content Cohort 1 (n=479): randomized 1:1 to receive BRUKINSA (n=241) or bendamustine plus rituximab (n=238) until disease progression or unacceptable toxicity, in patients not harboring del(17p); data from this group comprise the primary endpoint; Cohort 2/Arm C (n=110): patients with del(17p) receiving BRUKINSA as a monotherapy; and Cohort 3/Arm D (n=114): 66 patients with del(17p) and/or pathogenic TP53 mutation and 47 patients without del(17p) or TP53 were enrolled, with 110 patients receiving BRUKINSA in combination with venetoclax. Article content The results of Cohort 1 of the SEQUOIA study led to the regulatory approval of BRUKINSA monotherapy in the treatment of TN CLL in many countries across the world, including approvals by the U.S. Food and Drug Administration and the European Medicines Agency. The primary endpoint of the trial is progression-free survival (PFS), as assessed by an independent review committee (IRC). Secondary endpoints include investigator-assessed PFS, IRC- and investigator-assessed overall response rate (ORR), overall survival (OS), and safety, as well as PFS and ORR in patients with del(17p). Article content About BRUKINSA ® (zanubrutinib) BRUKINSA is an orally available, small molecule inhibitor of Bruton's tyrosine kinase (BTK) designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared with other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease-relevant tissues. Article content BRUKINSA has the broadest label globally of any BTK inhibitor and is the only BTK inhibitor to provide the flexibility of once or twice daily dosing. Additionally, BRUKINSA is also the only BTK inhibitor to demonstrate superiority to another BTK inhibitor in a Phase 3 study. The global BRUKINSA clinical development program includes about 7,100 patients enrolled in 30 countries and regions across more than 35 trials. BRUKINSA is approved in more than 75 markets, and more than 200,000 patients have been treated globally. Article content Chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). Waldenström's macroglobulinemia (WM). Mantle cell lymphoma (MCL) who have received at least one prior therapy. Relapsed or refractory marginal zone lymphoma (MZL) who have received at least one anti-CD20-based regimen. Relapsed or refractory follicular lymphoma (FL), in combination with obinutuzumab, after two or more lines of systemic therapy. Article content The MCL, MZL and FL indications are approved under accelerated approval based on overall response rate and durability of response. Continued approval for these indications may be contingent upon verification and description of clinical benefit in confirmatory trials. Article content Hemorrhage Article content Fatal and serious hemorrhage has occurred in patients with hematological malignancies treated with BRUKINSA. Grade 3 or higher hemorrhage including intracranial and gastrointestinal hemorrhage, hematuria, and hemothorax was reported in 3.8% of patients treated with BRUKINSA in clinical trials, with fatalities occurring in 0.2% of patients. Bleeding of any grade, excluding purpura and petechiae, occurred in 32% of patients. Article content Bleeding has occurred in patients with and without concomitant antiplatelet or anticoagulation therapy. Coadministration of BRUKINSA with antiplatelet or anticoagulant medications may further increase the risk of hemorrhage. Article content Monitor for signs and symptoms of bleeding. Discontinue BRUKINSA if intracranial hemorrhage of any grade occurs. Consider the benefit-risk of withholding BRUKINSA for 3-7 days before and after surgery depending upon the type of surgery and the risk of bleeding. Article content Fatal and serious infections (including bacterial, viral, or fungal infections) and opportunistic infections have occurred in patients with hematological malignancies treated with BRUKINSA. Grade 3 or higher infections occurred in 26% of patients, most commonly pneumonia (7.9%), with fatal infections occurring in 3.2% of patients. Infections due to hepatitis B virus (HBV) reactivation have occurred. Article content Consider prophylaxis for herpes simplex virus, pneumocystis jirovecii pneumonia, and other infections according to standard of care in patients who are at increased risk for infections. Monitor and evaluate patients for fever or other signs and symptoms of infection and treat appropriately. Article content Cytopenias Article content Grade 3 or 4 cytopenias, including neutropenia (21%), thrombocytopenia (8%) and anemia (8%) based on laboratory measurements, developed in patients treated with BRUKINSA. Grade 4 neutropenia occurred in 10% of patients, and Grade 4 thrombocytopenia occurred in 2.5% of patients. Article content Monitor complete blood counts regularly during treatment and interrupt treatment, reduce the dose, or discontinue treatment as warranted. Treat using growth factor or transfusions, as needed. Article content Second primary malignancies, including non-skin carcinoma, have occurred in 14% of patients treated with BRUKINSA. The most frequent second primary malignancy was non-melanoma skin cancers (8%), followed by other solid tumors in 7% of the patients (including melanoma in 1% of patients) and hematologic malignancies (0.7%). Advise patients to use sun protection and monitor patients for the development of second primary malignancies. Article content Cardiac Arrhythmias Article content Serious cardiac arrhythmias have occurred in patients treated with BRUKINSA. Atrial fibrillation and atrial flutter were reported in 4.4% patients treated with BRUKINSA, including Grade 3 or higher cases in 1.9% of patients. Patients with cardiac risk factors, hypertension, and acute infections may be at increased risk. Grade 3 or higher ventricular arrhythmias were reported in 0.3% of patients. Article content Monitor for signs and symptoms of cardiac arrhythmias (e.g., palpitations, dizziness, syncope, dyspnea, chest discomfort), manage appropriately, and consider the risks and benefits of continued BRUKINSA treatment. Article content Hepatotoxicity, including severe, life-threatening, and potentially fatal cases of drug-induced liver injury (DILI), has occurred in patients treated with Bruton tyrosine kinase inhibitors, including BRUKINSA. Article content Evaluate bilirubin and transaminases at baseline and throughout treatment with BRUKINSA. For patients who develop abnormal liver tests after BRUKINSA, monitor more frequently for liver test abnormalities and clinical signs and symptoms of hepatic toxicity. If DILI is suspected, withhold BRUKINSA. Upon confirmation of DILI, discontinue BRUKINSA. Article content Embryo-Fetal Toxicity Article content Based on findings in animals, BRUKINSA can cause fetal harm when administered to a pregnant woman. Administration of zanubrutinib to pregnant rats during the period of organogenesis caused embryo-fetal toxicity, including malformations at exposures that were 5 times higher than those reported in patients at the recommended dose of 160 mg twice daily. Advise women to avoid becoming pregnant while taking BRUKINSA and for 1 week after the last dose. Advise men to avoid fathering a child during treatment and for 1 week after the last dose. If this drug is used during pregnancy, or if the patient becomes pregnant while taking this drug, the patient should be apprised of the potential hazard to a fetus. Article content Adverse Reactions Article content The most common adverse reactions (≥30%), including laboratory abnormalities, in patients who received BRUKINSA (N=1729) are decreased neutrophil count (51%), decreased platelet count (41%), upper respiratory tract infection (38%), hemorrhage (32%), and musculoskeletal pain (31%). Article content Drug Interactions Article content CYP3A Inducers: Avoid coadministration with strong or moderate CYP3A inducers. Dose adjustment may be recommended with moderate CYP3A inducers. Article content About BeOne BeOne Medicines is a global oncology company domiciled in Switzerland that is discovering and developing innovative treatments that are more affordable and accessible to cancer patients worldwide. With a portfolio spanning hematology and solid tumors, BeOne is expediting development of its diverse pipeline of novel therapeutics through its internal capabilities and collaborations. With a growing global team of more than 11,000 colleagues spanning six continents, the Company is committed to radically improving access to medicines for far more patients who need them. To learn more about BeOne, please visit and follow us on LinkedIn, X, Facebook and Instagram. Article content Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding BeOne's ability to deliver advanced and effective treatments for a broad range of cancer patients; BRUKINSA's role across CLL patients; the ability of BeOne's pipeline to meeting evolving patient needs and elevate the standard of care; and BeOne's plans, commitments, aspirations, and goals under the heading 'About BeOne.' Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including BeOne's ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing, and progress of clinical trials and marketing approval; BeOne's ability to achieve commercial success for its marketed medicines and drug candidates, if approved; BeOne's ability to obtain and maintain protection of intellectual property for its medicines and technology; BeOne's reliance on third parties to conduct drug development, manufacturing, commercialization, and other services; BeOne's limited experience in obtaining regulatory approvals and commercializing pharmaceutical products; BeOne's ability to obtain additional funding for operations and to complete the development of its drug candidates and maintain profitability; and those risks more fully discussed in the section entitled 'Risk Factors' in BeOne's most recent quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in BeOne's subsequent filings with the U.S. Securities and Exchange Commission. All information in this press release is as of the date of this press release, and BeOne undertakes no duty to update such information unless required by law. Article content _______________________________ 1 National Cancer Institute. Chronic Lymphocytic Leukemia Treatment (PDQ)–Patient Version. Accessed November 2024. 2 American Cancer Society. What is Chronic Lymphocytic Leukemia? Updated May 10, 2018. Accessed November 2024. 3 American Cancer Society. Key Statistics for Chronic Lymphocytic Leukemia. Updated July 1, 2024. Accessed November 2024. 4 Leukemia and Lymphoma Society. Chronic Lymphocytic Leukemia. Revised June 2021. Accessed November 5, 2024. Article content Article content Article content Article content Article content Contacts Article content Article content Article content
USA Today
6 days ago
- Sport
- USA Today
Tennessee Titans provide positive injury update on Lloyd Cushenberry
Tennessee Titans provide positive injury update on Lloyd Cushenberry Tennessee Titans head coach Brian Callahan addressed the media before the start of Wednesday's open practice and offered some insight on how things are going. One of the main topics was the health of veterans returning from injury. This was a topic of interest after wide receiver Treylon Burks was spotted running routes on the first day of Phase 3 activities. While Burks is working his way back, he will be limited to individual drills and will not be part of any team-wide drills. The major update was about the status of veteran center Lloyd Cushenberry, who is still rehabbing from the torn Achilles that ended his season in 2024. While Cushenberry is at the facility, like Burks, he is rehabbing and is on schedule to return. That's an excellent sign for the Titans, who have reconstructed their offensive line during the offseason and are counting on Cushenberry to return to anchor the unit. He is entering the second year of a four-year, $50 million contract with Tennessee and is a crucial veteran cog in the new-look offensive line. The Titans brought back center Corey Levin during the offseason to compete for a backup role behind Cushenberry, but lack experienced depth at the position behind them.
Yahoo
24-05-2025
- Business
- Yahoo
Mineralys Therapeutics Announces Late-Breaking Presentation of Data from the Launch-HTN Pivotal Trial of Lorundrostat in Uncontrolled or Resistant Hypertension at 34th European Meeting on Hypertension and Cardiovascular Protection (ESH 2025)
– Largest hypertension trial of an aldosterone synthase inhibitor to date demonstrated the efficacy of lorundrostat in over 1,000 participants with uncontrolled or resistant hypertension in a real-world setting – – Lorundrostat 50 mg dosed once daily demonstrated clinically meaningful and sustained reductions in systolic blood pressure, with a 16.9 mmHg reduction at Week 6 (-9.1 mmHg placebo adjusted) and a 19.0 mmHg reduction at Week 12 (-11.7mm placebo adjusted) – – Lorundrostat demonstrated a favorable safety and tolerability profile – RADNOR, Pa., May 24, 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 detailed results from the pivotal Phase 3 Launch-HTN trial in over 1,000 participants with uncontrolled hypertension (uHTN) or resistant hypertension (rHTN) who were taking two to five antihypertensive medications. When added to existing background treatment, lorundrostat 50 mg dosed once daily demonstrated clinically meaningful and sustained reductions in automatized office systolic blood pressure, with a 16.9 mmHg reduction at Week 6 (-9.1 mmHg placebo adjusted; p-value < 0.0001) and a 19 mmHg reduction at Week 12 (-11.7mm placebo adjusted; p-value < 0.0001). Additionally, lorundrostat demonstrated a favorable safety and tolerability profile. 'The detailed results from Launch-HTN, which was designed to reflect treatment in the real-world setting, mark a pivotal milestone in our mission to deliver the first targeted aldosterone synthase inhibitor to the millions of people suffering from uncontrolled or resistant hypertension,' stated Jon Congleton, Chief Executive Officer of Mineralys Therapeutics. 'With these findings in hand, we now have data from two pivotal trials in distinct-but-complementary populations that reinforce the promise of a new treatment approach for hypertension that directly addresses the dysregulated aldosterone pathway – a key driver of the condition in many patients.' 'The Launch-HTN trial provides substantial evidence supporting lorundrostat's potential as a well-tolerated, effective treatment for patients with uncontrolled or resistant hypertension, with consistent blood pressure reductions across a large and diverse patient population,' stated Manish Saxena MBBS, Deputy Clinical Co-Director of Queen Mary University of London's William Harvey Research Institute and Hypertension Specialist at Barts Health NHS Trust. 'The clinically meaningful and sustained reductions in systolic blood pressure observed with lorundrostat are especially important, as long-term control is key to lowering the risk of serious cardiovascular, renal, and metabolic complications. The consistency of results seen in the lorundrostat development program – which includes multiple trials across differentiated patient populations – supports its potential to have a broad role in future hypertension care.' Results from Launch-HTN were presented in a late-breaking session at the 34th European Meeting on Hypertension and Cardiovascular Protection (ESH 2025) on Saturday, May 24, 2025, at 10:00am CEST. Efficacy Results from Launch-HTN 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. Primary Endpoint 50 mg(n=808) Change in AOBP at Week 6 -16.9 mmHg absolute change -9.1 mmHg placebo-adjusted change(p < 0.0001) Pre-Defined Endpoint 50 mg(n=538) 50 to 100 mg(n=270) Change in AOBP at Week 12 -19.0 mmHg absolute change -15.7 mmHg absolute change -11.7 mmHg placebo-adjusted change(p < 0.0001) -8.4 mmHg placebo-adjusted change(p = 0.0016) Safety and Tolerability Results Lorundrostat demonstrated a favorable safety and tolerability profile in the Launch-HTN trial. The anticipated on-target effects on serum electrolytes, increased serum potassium and reduced serum sodium were modest and rapidly reversible upon discontinuation of lorundrostat. Suppression of cortisol production was not observed and there was a very low incidence of drug-related serious adverse events resulting in discontinuation or dose-adjustment of study medication. Treatment-emergent serious adverse events (SAEs) occurred in 12 participants (2.2%) and two participants (0.7%) in the 50 mg and 50 mg with optional dose escalation to 100 mg arms, respectively, compared with eight participants (3.0%) in the placebo arm. There was only one participant (0.1%) in the trial with treatment-related SAE that occurred in the 50 mg arm. 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 per-protocol exclusion of factitious results, the values for confirmed hyperkalemia were 0.6% and 1.1%, respectively. Launch-HTN was the second of two pivotal trials evaluating lorundrostat in participants with uHTN or rHTN. Detailed results from the first pivotal trial (Advance-HTN) in participants who would normally be treated by specialists were recently published in The New England Journal of Medicine (NEJM). Advance-HTN results were first presented at the American College of Cardiology's Annual Scientific Session & Expo (ACC.25) in March 2025. 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 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 estimated 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 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 participants, once-daily lorundrostat demonstrated statistically significant and clinically meaningful systolic blood pressure reduction in both AOBP and 24-hour ambulatory systolic 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 SAE 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 participants 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 AOBP 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 participants. Participants who meet 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. Participants 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 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@
Yahoo
24-05-2025
- Business
- Yahoo
Mineralys Therapeutics Announces Late-Breaking Presentation of Data from the Launch-HTN Pivotal Trial of Lorundrostat in Uncontrolled or Resistant Hypertension at 34th European Meeting on Hypertension and Cardiovascular Protection (ESH 2025)
– Largest hypertension trial of an aldosterone synthase inhibitor to date demonstrated the efficacy of lorundrostat in over 1,000 participants with uncontrolled or resistant hypertension in a real-world setting – – Lorundrostat 50 mg dosed once daily demonstrated clinically meaningful and sustained reductions in systolic blood pressure, with a 16.9 mmHg reduction at Week 6 (-9.1 mmHg placebo adjusted) and a 19.0 mmHg reduction at Week 12 (-11.7mm placebo adjusted) – – Lorundrostat demonstrated a favorable safety and tolerability profile – RADNOR, Pa., May 24, 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 detailed results from the pivotal Phase 3 Launch-HTN trial in over 1,000 participants with uncontrolled hypertension (uHTN) or resistant hypertension (rHTN) who were taking two to five antihypertensive medications. When added to existing background treatment, lorundrostat 50 mg dosed once daily demonstrated clinically meaningful and sustained reductions in automatized office systolic blood pressure, with a 16.9 mmHg reduction at Week 6 (-9.1 mmHg placebo adjusted; p-value < 0.0001) and a 19 mmHg reduction at Week 12 (-11.7mm placebo adjusted; p-value < 0.0001). Additionally, lorundrostat demonstrated a favorable safety and tolerability profile. 'The detailed results from Launch-HTN, which was designed to reflect treatment in the real-world setting, mark a pivotal milestone in our mission to deliver the first targeted aldosterone synthase inhibitor to the millions of people suffering from uncontrolled or resistant hypertension,' stated Jon Congleton, Chief Executive Officer of Mineralys Therapeutics. 'With these findings in hand, we now have data from two pivotal trials in distinct-but-complementary populations that reinforce the promise of a new treatment approach for hypertension that directly addresses the dysregulated aldosterone pathway – a key driver of the condition in many patients.' 'The Launch-HTN trial provides substantial evidence supporting lorundrostat's potential as a well-tolerated, effective treatment for patients with uncontrolled or resistant hypertension, with consistent blood pressure reductions across a large and diverse patient population,' stated Manish Saxena MBBS, Deputy Clinical Co-Director of Queen Mary University of London's William Harvey Research Institute and Hypertension Specialist at Barts Health NHS Trust. 'The clinically meaningful and sustained reductions in systolic blood pressure observed with lorundrostat are especially important, as long-term control is key to lowering the risk of serious cardiovascular, renal, and metabolic complications. The consistency of results seen in the lorundrostat development program – which includes multiple trials across differentiated patient populations – supports its potential to have a broad role in future hypertension care.' Results from Launch-HTN were presented in a late-breaking session at the 34th European Meeting on Hypertension and Cardiovascular Protection (ESH 2025) on Saturday, May 24, 2025, at 10:00am CEST. Efficacy Results from Launch-HTN 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. Primary Endpoint 50 mg(n=808) Change in AOBP at Week 6 -16.9 mmHg absolute change -9.1 mmHg placebo-adjusted change(p < 0.0001) Pre-Defined Endpoint 50 mg(n=538) 50 to 100 mg(n=270) Change in AOBP at Week 12 -19.0 mmHg absolute change -15.7 mmHg absolute change -11.7 mmHg placebo-adjusted change(p < 0.0001) -8.4 mmHg placebo-adjusted change(p = 0.0016) Safety and Tolerability Results Lorundrostat demonstrated a favorable safety and tolerability profile in the Launch-HTN trial. The anticipated on-target effects on serum electrolytes, increased serum potassium and reduced serum sodium were modest and rapidly reversible upon discontinuation of lorundrostat. Suppression of cortisol production was not observed and there was a very low incidence of drug-related serious adverse events resulting in discontinuation or dose-adjustment of study medication. Treatment-emergent serious adverse events (SAEs) occurred in 12 participants (2.2%) and two participants (0.7%) in the 50 mg and 50 mg with optional dose escalation to 100 mg arms, respectively, compared with eight participants (3.0%) in the placebo arm. There was only one participant (0.1%) in the trial with treatment-related SAE that occurred in the 50 mg arm. 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 per-protocol exclusion of factitious results, the values for confirmed hyperkalemia were 0.6% and 1.1%, respectively. Launch-HTN was the second of two pivotal trials evaluating lorundrostat in participants with uHTN or rHTN. Detailed results from the first pivotal trial (Advance-HTN) in participants who would normally be treated by specialists were recently published in The New England Journal of Medicine (NEJM). Advance-HTN results were first presented at the American College of Cardiology's Annual Scientific Session & Expo (ACC.25) in March 2025. 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 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 estimated 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 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 participants, once-daily lorundrostat demonstrated statistically significant and clinically meaningful systolic blood pressure reduction in both AOBP and 24-hour ambulatory systolic 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 SAE 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 participants 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 AOBP 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 participants. Participants who meet 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. Participants 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 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@
