Latest news with #MirumPharmaceuticals
Yahoo
4 days ago
- Business
- Yahoo
Why Is Mirum Pharmaceuticals (MIRM) Down 1.3% Since Last Earnings Report?
It has been about a month since the last earnings report for Mirum Pharmaceuticals, Inc. (MIRM). Shares have lost about 1.3% in that time frame, underperforming the S&P 500. Will the recent negative trend continue leading up to its next earnings release, or is Mirum Pharmaceuticals due for a breakout? Before we dive into how investors and analysts have reacted as of late, let's take a quick look at the most recent earnings report in order to get a better handle on the important catalysts. It turns out, estimates revision have trended upward during the past month. The consensus estimate has shifted 12.93% due to these changes. At this time, Mirum Pharmaceuticals has a great Growth Score of A, a grade with the same score on the momentum front. However, the stock was allocated a grade of D on the value side, putting it in the bottom 40% for this investment strategy. Overall, the stock has an aggregate VGM Score of A. If you aren't focused on one strategy, this score is the one you should be interested in. Estimates have been trending upward for the stock, and the magnitude of these revisions looks promising. Notably, Mirum Pharmaceuticals has a Zacks Rank #3 (Hold). We expect an in-line return from the stock in the next few months. Mirum Pharmaceuticals is part of the Zacks Medical - Biomedical and Genetics industry. Over the past month, Moderna (MRNA), a stock from the same industry, has gained 7.1%. The company reported its results for the quarter ended March 2025 more than a month ago. Moderna reported revenues of $108 million in the last reported quarter, representing a year-over-year change of -35.3%. EPS of -$2.52 for the same period compares with -$3.07 a year ago. Moderna is expected to post a loss of $2.97 per share for the current quarter, representing a year-over-year change of +10.8%. Over the last 30 days, the Zacks Consensus Estimate has changed -3.9%. Moderna has a Zacks Rank #3 (Hold) based on the overall direction and magnitude of estimate revisions. Additionally, the stock has a VGM Score of D. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Mirum Pharmaceuticals, Inc. (MIRM) : Free Stock Analysis Report Moderna, Inc. (MRNA) : Free Stock Analysis Report This article originally published on Zacks Investment Research ( Zacks Investment Research Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
19-05-2025
- Business
- Business Wire
Mirum Pharmaceuticals Appoints Doug Sheehy, JD, as Chief Legal Officer
FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced the appointment of Doug Sheehy, JD, as chief legal officer. Mr. Sheehy brings nearly two decades of experience leading global legal and compliance operations for biopharmaceutical companies. Mr. Sheehy was most recently chief legal officer and secretary at Sonoma Biotherapeutics, Inc., which is developing regulatory T cell therapies for autoimmune and inflammatory diseases. Prior to Sonoma, from 2016 to 2020, he served as general counsel and secretary for Aimmune Therapeutics, Inc., a biopharmaceutical company that specialized in the development and commercialization of treatments for life-threatening food allergies. Mr. Sheehy served at Aimmune until its acquisition by Nestle Health Science in 2020. Before Aimmune, from 2007-2016, he was executive vice president, chief administrative officer, general counsel, and secretary of Codexis, Inc., a company focused on the development of synthetic enzymes for the pharmaceutical and food industries. Earlier in his career, he served in several roles within the legal department at CV Therapeutics. Mr. Sheehy began his career as a corporate and securities lawyer in Silicon Valley representing emerging growth companies and venture funds. Mr. Sheehy received an A.B. in History from Dartmouth College and a J.D. from American University's Washington College of Law. 'Doug brings extensive global legal experience that will strengthen Mirum's capabilities as we continue to grow our commercial business and advance our rare disease pipeline,' said Chris Peetz, chief executive officer at Mirum. 'Doug will be a great addition to Mirum's leadership team, and I look forward to leveraging his experience in guiding transformative growth for the company.' 'Mirum is an impressive company that has established its place as a leader in rare disease, and I am excited to have the opportunity to join the leadership team and contribute to the exciting work underway,' said Doug Sheehy. 'The company has a focused growth strategy and is poised for continued success as it works to make a meaningful difference in the lives of people impacted by rare disease.' About Mirum Pharmaceuticals, Inc. Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution/LIVMARLI® (maralixibat) tablets, CHOLBAM® (cholic acid) capsules, and CTEXLI™ (chenodiol) tablets. LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum has initiated the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. CHOLBAM is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms of liver disease. CTEXLI is FDA-approved for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Mirum's late-stage pipeline includes two investigational treatments for several rare diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Mirum is also planning for a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder. To learn more about Mirum, visit and follow Mirum on Facebook, LinkedIn, Instagram and Twitter (X).
Yahoo
12-05-2025
- Health
- Yahoo
Chronic Pruritus Market is Anticipated to Expand at a CAGR of 8.6% During the Study Period (2020-2034)
The chronic pruritus market is expected to grow across the 7MM, driven by the introduction of innovative therapies like Dupilumab, Linerixibat, and Volixibat, among others. Furthermore, the rising prevalence of chronic pruritus, driven by factors such as an aging population, increased exposure to environmental irritants, a higher occurrence of chronic inflammatory and autoimmune conditions, and improved diagnostic awareness among healthcare professionals, will further bolster the chronic pruritus market growth. LAS VEGAS, May 12, 2025 /PRNewswire/ -- DelveInsight's Chronic Pruritus Market Insights report includes a comprehensive understanding of current treatment practices, chronic pruritus emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan]. Key Takeaways from the Chronic Pruritus Market Report According to DelveInsight's analysis, the market size for chronic pruritus was found to be USD 3.5 billion in the 7MM in 2024. Estimates suggest that dupilumab is expected to generate approximately USD 700 million in the 7MM by 2034. According to DelveInsight's estimates, there were approximately 98 million total prevalent cases of chronic pruritus in the 7MM in 2024. Among these, around 22 million were diagnosed cases. Leading chronic pruritus companies developing emerging therapies, such as Sanofi, Regeneron, GlaxoSmithKline, Galderma, Chugai Pharmaceutical, Mirum Pharmaceuticals, and others, are developing novel chronic pruritus drugs that can be available in the chronic pruritus market in the coming years. The promising chronic pruritus therapies in the pipeline include DUPIXENT (dupilumab), Linerixibat (GSK2330672), NEMLUVIO/MITCHGA (nemolizumab), Volixibat, and others. In November 2024, GSK reported positive results from the global Phase III GLISTEN trial of linerixibat in adults with cholestatic pruritus in PBC at The Liver Meeting, hosted by the American Association for the Study of Liver Diseases (AASLD). Moreover, the company anticipates regulatory decisions in the U.S. in 2025, and in Europe and Japan in 2026. In November 2024, Mirum Pharmaceuticals presented interim results from the Phase IIb VINTAGE trial of volixibat for cholestatic pruritus in PBC at the AASLD. In September 2024, the Phase III LIBERTY-CPUO-CHIC trial evaluating DUPIXENT in adults with uncontrolled, severe chronic pruritus of unknown origin did not meet statistical significance for its primary itch responder endpoint, though it showed favorable numerical trends. The study achieved nominally significant improvements across all other itch-related endpoints. The Phase III program includes Study A (LIBERTY-CPUO-CHIC) and the planned pivotal Study B. Discover which therapies are expected to grab the major chronic pruritus market share @ Chronic Pruritus Market Report Chronic Pruritus Overview Chronic pruritus, defined as itching that persists for more than six weeks, can severely affect a person's quality of life and is linked to a variety of skin-related and systemic conditions. Its underlying mechanisms involve complex interactions between the immune system and the nervous system, with elements such as cytokines and neuropeptides playing key roles in maintaining the itch. This complexity makes diagnosis challenging, necessitating detailed skin evaluations to distinguish between primary skin issues and secondary changes. Given its diverse origins, effective management requires individualized treatment plans that address the root cause. Diagnosing chronic pruritus demands a structured and thorough approach due to its broad range of possible etiologies. The diagnostic process begins with an in-depth medical history and physical examination to detect any skin abnormalities or signs of systemic illness. Routine lab tests are typically ordered to evaluate the function of the liver, kidneys, and thyroid, along with checks for iron levels and inflammatory markers. Additional procedures, such as skin scrapings or biopsies, may be necessary to exclude infections or underlying malignancies. Chronic Pruritus Epidemiology Segmentation The chronic pruritus epidemiology section provides insights into the historical and current chronic pruritus patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders. The chronic pruritus market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Prevalent Cases of Chronic Pruritus Total Diagnosed Prevalent Cases of Chronic Pruritus Gender-specific Diagnosed Prevalent Cases of Chronic Pruritus Age-specific Diagnosed Prevalent Cases of Chronic Pruritus Severity-specific Diagnosed Prevalent Cases of Chronic Pruritus Etiology-specific Diagnosed Prevalent Cases of Chronic Pruritus Chronic Pruritus Treatment Market Chronic itch demands a comprehensive treatment plan tailored to its specific cause and biological mechanisms. Successfully managing this condition depends on pinpointing the origin—whether it stems from a skin disorder, an internal disease, a nerve-related issue, or psychological factors—before applying targeted therapies to relieve discomfort. As scientific understanding grows, care is evolving from broad symptom control toward precision medicine, using new drugs that specifically interrupt the pathways driving itch. When itch accompanies skin diseases such as atopic dermatitis or psoriasis, topical corticosteroids remain the mainstay for reducing inflammation and easing itching. By dampening the immune response, these medications lessen redness, swelling, and discomfort. In areas where steroids aren't suitable or for patients who can't use them, calcineurin inhibitors offer a steroid-free alternative. These drugs block calcineurin—a key protein in T-cell activation—thereby curbing inflammation without the typical steroid side effects. In more serious cases of atopic dermatitis, patients often receive systemic biologic treatment such as dupilumab. Dupilumab is a monoclonal antibody that inhibits the IL-4 and IL-13 signaling pathways—major contributors to Type 2 inflammation—leading to symptom relief and strengthened skin barrier function. For chronic kidney disease–associated pruritus (CKD-aP), therapeutic choices now include difelikefalin, a selective kappa-opioid receptor agonist. Given by intravenous infusion, difelikefalin acts on peripheral sensory neurons and immune cells to reduce itching, while its minimal penetration into the central nervous system helps avoid typical opioid-related central side effects. The current treatment landscape for chronic pruritus therapeutics market is relatively sparse, with only a handful of approved options: KORSUVA/KAPRUVIA (Kappa opioid receptor agonist), LIVMARLI (IBAT inhibitor), and BYLVAY/KAYFANDA (IBAT inhibitor). Notably, in March 2025, Japan approved LIVMARLI as the first and so far only therapy for cholestatic itching in patients with Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC). To know more about chronic pruritus treatment guidelines, visit @ Chronic Pruritus Management Chronic Pruritus Pipeline Therapies and Key Companies DUPIXENT (dupilumab): Sanofi/Regeneron Linerixibat (GSK2330672): GlaxoSmithKline NEMLUVIO/MITCHGA (nemolizumab): Galderma/Chugai Pharmaceutical Volixibat: Mirum Pharmaceuticals Discover more about chronic pruritus drugs in development @ Chronic Pruritus Clinical Trials Chronic Pruritus Market Dynamics The chronic pruritus market dynamics are expected to change in the coming years. A multidisciplinary collaboration among dermatologists, neurologists, allergists, and psychologists enhances diagnostic accuracy and enables personalized treatment, ensuring a comprehensive approach to managing chronic pruritus, which can be effectively treated with peripheral Kappa-Opioid Receptor (KOR) agonists that reduce itch while minimizing central side effects and improving tolerability; moreover, the limited industry focus on chronic pruritus—compared to broader pruritus—highlights an opportunity for innovation in specialized therapies, while a greater emphasis on quality-of-life metrics supports the development of care models that address both the psychosocial burden and symptom control, ultimately improving overall patient outcomes. Furthermore, potential therapies are being investigated for the treatment of chronic pruritus, and it is safe to predict that the treatment space will significantly impact the chronic pruritus market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the chronic pruritus market in the 7MM. However, several factors may impede the growth of the chronic pruritus market. Chronic pruritus of unknown origin remains challenging to manage due to the absence of reliable biomarkers, the failure to effectively target neural sensitization, the dominance of traditional treatments like antihistamines and corticosteroids, and the diverse underlying etiologies—including systemic diseases—all of which hinder precise diagnosis, delay effective treatment, and create barriers to the adoption of newer, more innovative therapies. Moreover, chronic pruritus treatment poses a significant economic burden and disrupts patients' overall well-being and QOL. Furthermore, the chronic pruritus market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the chronic pruritus market growth. Chronic Pruritus Market Report Metrics Details Study Period 2020–2034 Coverage 7MM [The United States, the EU4 (Germany, France, Italy, and Spain) and The United Kingdom, and Japan]. Chronic Pruritus Market CAGR 8.6 % Chronic Pruritus Market Size in 2023 USD 3.5 Billion Key Chronic Pruritus Companies Sanofi, Regeneron, GlaxoSmithKline, Galderma, Chugai Pharmaceutical, Mirum Pharmaceuticals, and others Key Pipeline Chronic Pruritus Therapies DUPIXENT (dupilumab), Linerixibat (GSK2330672), NEMLUVIO/MITCHGA (nemolizumab), Volixibat, and others Scope of the Chronic Pruritus Market Report Therapeutic Assessment: Chronic Pruritus current marketed and emerging therapies Chronic Pruritus Market Dynamics: Key Market Forecast Assumptions of Emerging Chronic Pruritus Drugs and Market Outlook Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Chronic Pruritus Market Access and Reimbursement Download the report to understand which factors are driving chronic pruritus market trends @ Chronic Pruritus Market Trends Table of Contents 1 Key Insights 2 Report Introduction 3 Chronic Pruritus Market Overview at a Glance 3.1 Market Share (%) Distribution of Chronic Pruritus by Therapies in the 7MM in 2020 3.2 Market Share (%) Distribution of Chronic Pruritus by Therapies in the 7MM in 2034 4 Executive Summary 5 Key Events 6 Disease Background and Overview 6.1 Introduction 6.2 Etiology 6.3 Pathophysiology 6.4 Diagnosis 6.4.1 Diagnostic Algorithm 6.4.2 Diagnostic Guidelines 6.5 Treatment and Management 6.5.1 Treatment Algorithm 6.5.2 Treatment Guidelines 7 Methodology 8 Epidemiology and Patient Population 8.1 Key Findings 8.2 Assumptions and Rationale: The 7MM 8.2.1 Total Prevalent Cases of Chronic Pruritus 8.2.2 Total Diagnosed Prevalent Cases of Chronic Pruritus 8.2.3 Gender-specific Cases of Chronic Pruritus 8.2.4 Age-specific Cases of Chronic Pruritus 8.2.5 Severity-specific Cases of Chronic Pruritus 8.2.6 Etiology-specific Cases of Chronic Pruritus 8.3 Total Prevalent Cases of Chronic Pruritus in the 7MM 8.4 Total Diagnosed Prevalent Cases of Chronic Pruritus in the 7MM 8.5 The US 8.5.1 Total Prevalent Cases of Chronic Pruritus in the US 8.5.2 Total Diagnosed Prevalent Cases of Chronic Pruritus in the US 8.5.3 Gender-specific Diagnosed Prevalent Cases of Chronic Pruritus in the US 8.5.4 Age-specific Diagnosed Prevalent Cases of Chronic Pruritus in the US 8.5.5 Severity-specific Diagnosed Prevalent Cases of Chronic Pruritus in the US 8.5.6 Etiology-specific Diagnosed Prevalent Cases of Chronic Pruritus in the US 8.6 EU4 and the UK 8.6.1 Total Prevalent Cases of Chronic Pruritus in EU4 and the UK 8.6.2 Total Diagnosed Prevalent Cases of Chronic Pruritus in EU4 and the UK 8.6.3 Gender-specific Diagnosed Prevalent Cases of Chronic Pruritus in EU4 and the UK 8.6.4 Age-specific Diagnosed Prevalent Cases of Chronic Pruritus in EU4 and the UK 8.6.5 Severity-specific Diagnosed Prevalent Cases of Chronic Pruritus in EU4 and the UK 8.6.6 Etiology-specific Diagnosed Prevalent Cases of Chronic Pruritus in EU4 and the UK 8.7 Japan 8.7.1 Total Diagnosed Prevalent Cases of Chronic Pruritus in Japan 8.7.2 Gender-specific Diagnosed Prevalent Cases of Chronic Pruritus in Japan 8.7.3 Age-specific Diagnosed Prevalent Cases of Chronic Pruritus in Japan 8.7.4 Severity-specific Diagnosed Prevalent Cases of Chronic Pruritus in Japan 8.7.5 Etiology-specific Diagnosed Prevalent Cases of Chronic Pruritus in Japan 9 Patient Journey 10 Marketed Drugs 10.1 Key Cross Competition 10.2 KORSUVA/KAPRUVIA (difelikefalin): Cara Therapeutics/CSL/ Vifor/Maruishi 10.2.1 Product Description 10.2.2 Regulatory Milestones 10.2.3 Other Developmental Activities 10.2.4 Clinical Trials Information 10.2.5 Safety and Efficacy 10.3 LIVMARLI (maralixibat): Mirum Pharmaceuticals/Takeda 10.3.1 Product Description 10.3.2 Regulatory Milestones 10.3.3 Other Developmental Activities 10.3.4 Clinical Trials Information 10.3.5 Safety and Efficacy 10.4 BYLVAY/KAYFANDA (odevixibat): Ipsen/Jadeite Medicines 10.4.1 Product Description 10.4.2 Regulatory Milestones 10.4.3 Other Developmental Activities 10.4.4 Clinical Trials Information 10.4.5 Safety and Efficacy List to be continued in the final report. 11 Emerging Drugs 11.1 Key Cross Competition 11.2 DUPIXENT (dupilumab): Sanofi/Regeneron 11.2.1 Drug Description 11.2.2 Other Developmental Activities 11.2.3 Clinical Trials Information 11.2.4 Analysts' View 11.3 Linerixibat (GSK2330672): GlaxoSmithKline 11.3.1 Drug Description 11.3.2 Other Developmental Activities 11.3.3 Clinical Trials Information 11.3.4 Safety and Efficacy 11.3.5 Analysts' View 11.4 NEMLUVIO/MITCHGA (nemolizumab): Galderma/Chugai Pharmaceutical 11.4.1 Drug Description 11.4.2 Other Developmental Activities 11.4.3 Clinical Trials Information 11.4.4 Safety and Efficacy 11.4.5 Analysts' View 11.5 Volixibat: Mirum Pharmaceuticals 11.5.1 Drug Description 11.5.2 Other Developmental Activities 11.5.3 Clinical Trials Information 11.5.4 Safety and Efficacy 11.5.5 Analysts' View List to be continued in the final report. 12 Chronic Pruritus – 7MM Market Analysis 12.1 Key Findings 12.2 Key Market Forecast Assumptions 12.2.1 Cost Assumptions and Rebates 12.2.2 Pricing Trends 12.2.3 Analogue Assessment 12.2.4 Launch Year and Therapy Uptake 12.3 Market Outlook 12.4 Attribute Analysis 12.5 Total Market Size of Chronic Pruritus in the 7MM 12.6 Market Size of Chronic Pruritus by Therapies in the 7MM 12.7 The US Market Size 12.7.1 Total Market Size of Chronic Pruritus in the US 12.7.2 Market Size of Chronic Pruritus by Therapies in the US 12.8 EU4 and the UK Market Size 12.8.1 Total Market Size of Chronic Pruritus in EU4 and the UK 12.8.2 Market Size of Chronic Pruritus by Therapies in EU4 and the UK 12.9 Japan Market Size 12.9.1 Total Market Size of Chronic Pruritus in Japan 12.9.2 Market Size of Chronic Pruritus by Therapies in Japan 13 KOL Views 14 Unmet Needs 15 SWOT Analysis 16 Market Access and Reimbursement 16.1 The United States 16.1.1 CMS 16.2 EU4 and the UK 16.2.1 Germany 16.2.2 France 16.2.3 Italy 16.2.4 Spain 16.2.5 The United Kingdom 16.3 Japan 16.3.1 MHLW 17 Appendix 17.1 Acronyms and Abbreviations 17.2 Bibliography 17.3 Report Methodology 18 DelveInsight Capabilities 19 Disclaimer 20 About DelveInsight Related Reports Psoriasis Market Psoriasis Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key psoriasis companies including AnaptysBio, Nimbus Lakshmi, Takeda, MoonLake Immunotherapeutics, Bristol-Myers Squibb, Amgen, Dermavant Sciences, Inc., Janssen Research & Development, LLC, Sun Pharmaceutical Industries Limited, UCB Pharma, ACELYRIN Inc., Novartis, among others. 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Kymab, BiomX, LEO Pharma, GlaxoSmithKline, Arjil Pharmaceuticals, SCM Lifescience, Sun Pharmaceutical Industries Limited, Brickell Biotech Inc, AstraZeneca, Kyowa Kirin, UCB Biopharma, Arcutis Biotherapeutics, Vanda Pharmaceuticals, Kyowa Kirin, Sanofi, KeyMed Biosciences, Asana BioSciences, Bristol-Myers Squibb, RAPT Therapeutics, Allakos, Novartis, BioMimetix, Shanghai Hengrui Pharmaceutical Co, Connect Biopharma, Pfizer, Evommune, Inc., Fresh Tracks Therapeutics, Biosion, Chia Tai Tianqing Pharmaceutical, Reistone Biopharma Company Limited, JW Pharmaceutical, Oneness Biotech, Alphyn Biologics, selectION, UNION Therapeutics, Ichnos Scien, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve. Contact UsShruti Thakur info@ + Logo: View original content: SOURCE DelveInsight Business Research, LLP Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
09-05-2025
- Health
- Business Wire
Volixibat Data from Mirum's VANTAGE PBC Study Showcased at EASL
FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today presented new data from its Phase 2b VANTAGE study at the European Association for the Study of the Liver (EASL) meeting in Amsterdam, the Netherlands. The VANTAGE study is evaluating volixibat in patients with cholestatic pruritus caused by primary biliary cholangitis (PBC). 'The extended VANTAGE analysis provides encouraging insights into the potential impact that volixibat may have in treating the most severe and bothersome symptoms for patients living with PBC, including pruritus and fatigue,' said Joanne Quan, MD, chief medical officer at Mirum. 'We are also encouraged by the reductions seen in IL-31 in conjunction with sBA reductions, showing the potential for volixibat treatment to help alter the inflammatory condition present in PBC.' Data from the interim analysis of VANTAGE were highlighted with new analyses through week 28. Patients were randomized to receive volixibat 20mg BID, 80mg BID, or placebo BID. The primary endpoint of the study was mean change in weekly averaged daily itch score, as measured by the adult ItchRO scale, from baseline to Week 17-Week 28. Volixibat treatment led to rapid (as early as week 1) and sustained reductions in ItchRO with the volixibat combined dose group achieving a statistically significant 3.78-point reduction from Baseline (p<0.0001) and a placebo-adjusted response of 2.51 (p=0.0004). Each volixibat dose achieved statistically significant and similar responses (20mg BID: -2.4 [P=0.0039]; 80mg BID: -2.6 [P=0.0010]). The analysis also provided data across secondary endpoints showing reductions in serum bile acids (sBA) and quality of life (QoL) measures. Overall, 70% of patients who received volixibat achieved ≥50% reduction in sBA. Further, the data showed improvement from baseline in health-related quality of life, including fatigue and sleep, for patients treated with volixibat. Reductions from baseline were observed in inflammatory biomarker IL-31 in patients treated with volixibat. No new safety signals were observed, and adverse events were similar between the 20 mg and 80 mg treatment groups. The most common adverse event was diarrhea (77%) with all cases mild to moderate; one case resulted in discontinuation. Given the similar results with both efficacy and safety between the 20mg BID and 80mg doses BID, the 20mg BID dose was selected for the confirmatory portion of the VANTAGE study. To view the presentation, please visit the Publications & Presentations on Mirum's website. About the VANTAGE Study VANTAGE is a randomized, double-blind, placebo-controlled study evaluating the efficacy and safety of volixibat in patients with pruritus caused by primary biliary cholangitis (PBC). Patients were randomized to receive volixibat 20mg BID (n=10), 80mg BID (n=10), or placebo BID (n=11). The primary endpoint of the study was mean change in weekly averaged daily itch score, as measured by the adult ItchRO scale (an 11 point scale where 0 = no itch and 10 = worst itch imaginable), from baseline to Week 17-Week 28. VANTAGE is designed as a two-part study: the first part is intended to confirm dose (completed) and the second intended to be the confirmatory portion (currently enrolling patients). In the first part of the study, patients were randomized to receive either volixibat 20mg BID, 80mg BID, or placebo BID; the second part of the study randomized patients to receive either volixibat 20mg BID (the selected dose based on part one) or placebo BID. About Primary Biliary Cholangitis Primary Biliary Cholangitis (PBC) is a chronic, progressive, immune-mediated condition leading to the destruction of small intrahepatic bile ducts. The disease impacts an estimated 230,000 patients in the United States and the European Union, with women predominantly affected (a sex ratio of 9:1). Key clinical features and symptoms include cholestatic pruritus, fatigue, sicca syndrome, abdominal pain, cirrhosis, and hepatocellular carcinoma. Cholestatic pruritus (itch) and fatigue are two of the most debilitating symptoms and greatly affect overall health-related quality of life (HRQoL). Cholestatic pruritus can affect up to 80% of individuals and is thought to result in part from accumulation of toxic bile acids. About Volixibat Volixibat is an oral, minimally absorbed agent designed to selectively inhibit the ileal bile acid transporter (IBAT). Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids, through inhibition of IBAT, thereby reducing bile acids systemically and in the liver. Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (PSC) (VISTAS study), and primary biliary cholangitis (PBC) (VANTAGE study). In June, Mirum announced positive interim results from the Phase 2b VANTAGE study showing statistically significant improvement in pruritus as well as reductions in serum bile acids and improvements in fatigue for patients treated with volixibat. No new safety signals were observed, and the most common adverse event was diarrhea with all cases mild to moderate. Volixibat has been granted breakthrough therapy designation for the treatment of PBC. About LIVMARLI® (maralixibat) oral solution and LIVMARLI® (maralixibat) tablets LIVMARLI ® (maralixibat) is an orally administered, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for two pediatric cholestatic liver diseases, in both liquid and tablet formulations. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. three months of age and older and in Europe for patients two months of age and older. It is also approved in the U.S. for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) 12 months of age and older and in Europe for the treatment of PFIC in patients three months of age and older. For more information for U.S. residents, please visit LIVMARLI is currently being evaluated in the Phase 3 EXPAND study in additional settings of cholestatic pruritus. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum's clinical trials section on the company's website. U.S. IMPORTANT SAFETY INFORMATION Limitation of Use: LIVMARLI is not for use in PFIC type 2 patients who have a severe defect in the bile salt export pump (BSEP) protein. LIVMARLI can cause side effects, including: Liver injury. Changes in certain liver tests are common in patients with Alagille syndrome and PFIC but can worsen during treatment. These changes may be a sign of liver injury. In PFIC, this can be serious or may lead to liver transplant or death. Your healthcare provider should do blood tests and physical exams before starting and during treatment to check your liver function. Tell your healthcare provider right away if you get any signs or symptoms of liver problems, including nausea or vomiting, skin or the white part of the eye turns yellow, dark or brown urine, pain on the right side of the stomach (abdomen), bloating in your stomach area, loss of appetite or bleeding or bruising more easily than normal. Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea and stomach pain. Your healthcare provider may advise you to monitor for new or worsening stomach problems including stomach pain, diarrhea, blood in your stool or vomiting. Tell your healthcare provider right away if you have any of these symptoms more often or more severely than normal for you. A condition called Fat Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat is common in patients with Alagille syndrome and PFIC but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment and may monitor for bone fractures and bleeding which have been reported as common side effects. LIVMARLI is available in oral solution or tablet formulations and is taken by mouth 30 minutes before a meal. For Alagille syndrome, LIVMARLI is taken one time each day in the morning. For PFIC, LIVMARLI is taken two times each day. If you take the oral solution, use the oral dosing dispenser to measure your dose. About Mirum Pharmaceuticals, Inc. Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution/LIVMARLI® (maralixibat) tablets, CHOLBAM® (cholic acid) capsules, and CTEXLI™ (chenodiol) tablets. LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum has initiated the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. CHOLBAM is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms of liver disease. CTEXLI is FDA-approved for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Mirum's late-stage pipeline includes two investigational treatments for several rare diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Mirum is also planning for a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder. To learn more about Mirum, visit and follow Mirum on Facebook, LinkedIn, Instagram and Twitter (X). Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, data from Mirum's volixibat studies, including the VANTAGE study of volixibat in patients with pruritus due to PBC and such implications for patients in real world settings. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as 'will,' 'could,' 'can,' 'would,' 'potential,' 'hope,' 'opportunity,' and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum's business in general, the impact of macroeconomic and geopolitical developments, and the other risks described in Mirum's Annual Report on Form 10-K for the year ended December 31, 2024, filed with the U.S. Securities and Exchange Commission on February 26, 2025, and subsequent filings with the U.S. Securities and Exchange Commission, which are available at All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.
Yahoo
09-05-2025
- Business
- Yahoo
Mirum Pharmaceuticals First Quarter 2025 Earnings: Beats Expectations
Revenue: US$111.6m (up 61% from 1Q 2024). Net loss: US$14.7m (loss narrowed by 42% from 1Q 2024). US$0.30 loss per share (improved from US$0.54 loss in 1Q 2024). We've discovered 1 warning sign about Mirum Pharmaceuticals. View them for free. All figures shown in the chart above are for the trailing 12 month (TTM) period Revenue exceeded analyst estimates by 13%. Earnings per share (EPS) also surpassed analyst estimates by 8.9%. Looking ahead, revenue is forecast to grow 20% p.a. on average during the next 3 years, compared to a 17% growth forecast for the Biotechs industry in the US. Performance of the American Biotechs industry. The company's shares are up 4.2% from a week ago. You should always think about risks. Case in point, we've spotted 1 warning sign for Mirum Pharmaceuticals you should be aware of. Have feedback on this article? Concerned about the content? Get in touch with us directly. Alternatively, email editorial-team (at) article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
