Madrigal Pharmaceuticals Announces Publication of Positive Health-Related Quality of Life Results from the Phase 3 MAESTRO-NASH Trial of Rezdiffra™ (resmetirom)
Madrigal Pharmaceuticals (NASDAQ:MDGL) has announced the publication of positive patient-reported outcomes data from the Phase 3 MAESTRO-NASH trial of Rezdiffra™ (resmetirom) in Hepatology. The study showed that Rezdiffra, the first FDA-approved medication for NASH, improved health-related quality of life (HRQL) in patients with moderate to advanced fibrosis.
Key findings include:
- Clinically meaningful and statistically significant improvements in emotional well-being and health distress
- Positive tolerability profile of Rezdiffra
- Improvements in multiple HRQL domains, including Worry, Health Distress, and Stigma
- No worsening of HRQL related to potential side effects
Rezdiffra is a once-daily, oral, liver-directed thyroid hormone receptor (THR)-β agonist indicated for treating adults with noncirrhotic NASH with moderate to advanced liver fibrosis.
Madrigal Pharmaceuticals (NASDAQ:MDGL) ha annunciato la pubblicazione di dati positivi sui risultati riportati dai pazienti provenienti dallo studio di Fase 3 MAESTRO-NASH su Rezdiffra™ (resmetirom) in epatologia. Lo studio ha dimostrato che Rezdiffra, il primo farmaco approvato dalla FDA per la NASH, ha migliorato la qualità della vita correlata alla salute (HRQL) nei pazienti con fibrosi moderata o avanzata.
Le principali scoperte includono:
- Miglioramenti clinicamente significativi e statisticamente rilevanti nel benessere emotivo e nello stress legato alla salute
- Profilo di tollerabilità positivo di Rezdiffra
- Miglioramenti in molteplici domini HRQL, inclusi Preoccupazione, Stress Legato alla Salute e Stigma
- Nessun peggioramento di HRQL legato ai potenziali effetti collaterali
Rezdiffra è un agonista del recettore degli ormoni tiroidei (THR)-β, somministrato per via orale una volta al giorno, indicato per il trattamento di adulti con NASH non cirrotica e fibrosi epatica moderata o avanzata.
Madrigal Pharmaceuticals (NASDAQ:MDGL) ha anunciado la publicación de datos positivos sobre los resultados informados por los pacientes del ensayo de Fase 3 MAESTRO-NASH de Rezdiffra™ (resmetirom) en hepatología. El estudio mostró que Rezdiffra, el primer medicamento aprobado por la FDA para NASH, mejoró la calidad de vida relacionada con la salud (HRQL) en pacientes con fibrosis moderada a avanzada.
Los hallazgos clave incluyen:
- Mejoras clínicamente significativas y estadísticamente relevantes en el bienestar emocional y la angustia relacionada con la salud
- Perfil de tolerabilidad positivo de Rezdiffra
- Mejoras en múltiples dominios de HRQL, incluyendo Preocupación, Angustia por la Salud y Estigmas
- No se observó un deterioro de HRQL relacionado con posibles efectos secundarios
Rezdiffra es un agonista del receptor de hormonas tiroideas (THR)-β, que se toma por vía oral una vez al día, indicado para el tratamiento de adultos con NASH no cirrótica y fibrosis hepática moderada a avanzada.
마드리갈 제약(MADRIGAL PHARMACEUTICALS, NASDAQ:MDGL)은 Rezdiffra™ (레스메티롬)에 대한 제3상 MAESTRO-NASH 시험의 긍정적인 환자 보고 결과 데이터를 발표하였습니다. 이 연구는 Rezdiffra가 FDA가 승인한 첫 번째 NASH 치료제로서 중등도에서 고급 섬유화 환자의 건강 관련 삶의 질(HRQL)을 개선했음을 보여주었습니다.
주요 발견 사항은 다음과 같습니다:
- 정신적 웰빙 및 건강 스트레스에서의 임상적으로 의미 있는 통계적 유의미성
- Rezdiffra의 긍정적인 내약성 프로필
- 걱정, 건강 스트레스 및 낙인 등 다양한 HRQL 분야에서의 개선
- 잠재적인 부작용으로 인한 HRQL 악화 없음
Rezdiffra는 비간경변성 NASH 및 중등도에서 고급 간 섬유 증이 있는 성인 치료를 위해 하루에 한 번 복용하는 경구용 갑상선 호르몬 수용체(THR)-β 작용제입니다.
Madrigal Pharmaceuticals (NASDAQ:MDGL) a annoncé la publication de données positives sur les résultats rapportés par les patients de l'essai de phase 3 MAESTRO-NASH concernant Rezdiffra™ (resmetirom) en hépatologie. L'étude a montré que Rezdiffra, le premier médicament approuvé par la FDA pour la NASH, a amélioré la qualité de vie liée à la santé (HRQL) chez les patients atteints de fibrose modérée à avancée.
Les principales conclusions comprennent :
- Améliorations cliniquement significatives et statistiquement significatives du bien-être émotionnel et du stress lié à la santé
- Profil de tolérabilité positif de Rezdiffra
- Améliorations dans plusieurs domaines de HRQL, y compris l'inquiétude, le stress de santé et le stigma
- Aucune détérioration de la HRQL liée aux effets secondaires potentiels
Rezdiffra est un agoniste oral du récepteur des hormones thyroïdiennes (THR)-β, prenant une fois par jour, indiqué pour le traitement des adultes atteints de NASH non cirrhotiques avec fibrose hépatique modérée à avancée.
Madrigal Pharmaceuticals (NASDAQ:MDGL) hat die Veröffentlichung positiver patientenberichteter Ergebnisse aus der Phase-3-Studie MAESTRO-NASH zu Rezdiffra™ (resmetirom) in der Hepatologie bekanntgegeben. Die Studie zeigte, dass Rezdiffra, das erste von der FDA zugelassene Medikament bei NASH, die gesundheitsbezogene Lebensqualität (HRQL) bei Patienten mit moderater bis fortgeschrittener Fibrose verbesserte.
Wichtige Ergebnisse sind:
- Klinisch bedeutsame und statistisch signifikante Verbesserungen im emotionalen Wohlbefinden und Gesundheitsstress
- Positives Tolerabilitätsprofil von Rezdiffra
- Verbesserungen in mehreren HRQL-Domänen, einschließlich Sorge, Gesundheitsstress und Stigma
- Kein Verschlechterung der HRQL im Zusammenhang mit möglichen Nebenwirkungen
Rezdiffra ist ein einmal täglich einzunehmender, oral verabreichter, leberspezifischer Agonist des Schilddrüsenhormonrezeptors (THR)-β, der zur Behandlung von Erwachsenen mit nicht-zirrhotischer NASH und moderater bis fortgeschrittener Leberfibrose indiziert ist.
- Rezdiffra is the first FDA-approved medication for NASH treatment
- Positive patient-reported outcomes data showing improved health-related quality of life
- Clinically meaningful and statistically significant improvements in emotional well-being and health distress
- Positive tolerability profile of Rezdiffra
- Improvements in multiple HRQL domains, including Worry, Health Distress, and Stigma
- No worsening of HRQL related to potential side effects
- Continued approval for Rezdiffra may be contingent upon verification and description of clinical benefit in ongoing confirmatory trials
Insights
The publication of positive health-related quality of life (HRQL) results from the Phase 3 MAESTRO-NASH trial for Rezdiffra (resmetirom) is a significant development for Madrigal Pharmaceuticals and the NASH treatment landscape. Key findings include:
- Clinically meaningful and statistically significant improvements in emotional well-being and health distress for NASH patients treated with Rezdiffra
- Improvements in multiple HRQL domains, including Worry, Health Distress and Stigma, especially for patients achieving fibrosis improvement or NASH resolution
- No worsening of HRQL related to potential side effects, underscoring Rezdiffra's tolerability profile
These results complement Rezdiffra's efficacy in improving fibrosis and resolving NASH, as demonstrated in previous trials. As the first FDA-approved medication for NASH, Rezdiffra's ability to improve patients' quality of life could be a significant differentiator in the market and may contribute to better treatment adherence and outcomes.
This publication strengthens Madrigal Pharmaceuticals' market position in the NASH treatment space. Key financial implications include:
- Potential for increased adoption and market penetration of Rezdiffra, given its positive impact on patients' quality of life
- Enhanced competitive advantage as the first FDA-approved NASH therapy with demonstrated HRQL benefits
- Possible support for pricing and reimbursement negotiations, as improved quality of life can justify higher value-based pricing
- Potential for expanded indications or combination therapies based on the comprehensive benefits observed
With a market cap of
- Patients with MASH/NASH treated with Rezdiffra experienced clinically meaningful and statistically significant improvements in emotional well-being and health distress
- Study results underscore the positive tolerability profile of Rezdiffra
CONSHOHOCKEN, Pa., Sept. 30, 2024 (GLOBE NEWSWIRE) -- Madrigal Pharmaceuticals, Inc. (NASDAQ:MDGL), a biopharmaceutical company focused on delivering novel therapeutics for nonalcoholic steatohepatitis (NASH)/metabolic dysfunction-associated steatohepatitis (MASH), today announced the publication of positive patient-reported outcomes data demonstrating Rezdiffra (resmetirom) improved health-related quality of life (HRQL) in patients with NASH with moderate to advanced fibrosis. The results were published in the journal Hepatology.
Rezdiffra is a once-daily, oral, liver-directed thyroid hormone receptor (THR)-β agonist designed to target key underlying causes of NASH. It is the first FDA-approved medication for the treatment of NASH. In the pivotal Phase 3 MAESTRO-NASH biopsy trial, Rezdiffra achieved both fibrosis improvement and NASH resolution primary endpoints, and
Zobair M. Younossi, M.D., MPH, FACP, FACG, AGAF, FAASLD, Professor and Chairman of the Beatty Liver and Obesity Research Program, Inova Health System, the Chairman of the Global NASH Council and lead author of the HRQL analysis stated, “The serious burden of NASH on patient quality of life remains poorly understood and underappreciated by the health system. NASH is associated with significant impairment of HRQL related to fatigue, lack of stamina and other symptoms. Therefore, these results demonstrating that Rezdiffra helped patients achieve clinically meaningful and statistically significantly improvements in multiple domains of HRQL are highly encouraging. Additionally, we found no worsening of HRQL related to potential side effects of Rezdiffra in the MAESTRO-NASH study, which underscores the tolerability profile of the medication.”
In the study, changes in HRQL scores from baseline were evaluated in patients receiving Rezdiffra versus placebo and compared between patients with and without biopsy response. By weeks 24 and 52, patients receiving both doses of Rezdiffra experienced improvement of HRQL scores in the Worry domain of the Chronic Liver Disease Questionnaire-NASH. At week 52, Rezdiffra-treated patients who achieved fibrosis improvement or NASH resolution experienced improvement in several HRQL domains, including domains for Worry, Health Distress and Stigma. The improvement in HRQL among Rezdiffra biopsy responders was contrasted by no similar improvement in the placebo group. Biopsy responders with stage F3 fibrosis at baseline had similar or more pronounced improvements of HRQL in comparison to those responders with F2 or F1B fibrosis at baseline.
Bill Sibold, Chief Executive Officer of Madrigal, stated, “NASH is the leading cause of liver transplant among women and those who get listed for hepatocellular carcinoma in the U.S. and the unpredictable speed of NASH disease progression can cause serious emotional distress for people living with the disease. In addition to the health burden of NASH, patient advocates frequently tell us they worry about the impact of the disease on their families and plans for the future. The introduction of Rezdiffra as the first FDA-approved therapy for NASH has provided hope for patients, and we see this demonstrated in the improvements in health-related quality of life observed in the MAESTRO-NASH trial.”
About NASH
Nonalcoholic steatohepatitis (NASH) is a more advanced form of nonalcoholic fatty liver disease (NAFLD). NASH is a leading cause of liver-related mortality and an increasing burden on healthcare systems globally. Additionally, patients with NASH, especially those with more advanced metabolic risk factors (hypertension, concomitant type 2 diabetes), are at increased risk for adverse cardiovascular events and increased morbidity and mortality.
Once patients progress to NASH with moderate to advanced liver fibrosis (consistent with stages F2 to F3 fibrosis), the risk of adverse liver outcomes increases dramatically. NASH is rapidly becoming the leading cause of liver transplantation in the U.S. and is already the leading cause in women.
Madrigal estimates that approximately 1.5 million patients have been diagnosed with NASH in the U.S., of which approximately 525,000 have NASH with moderate to advanced liver fibrosis. Madrigal plans to focus on approximately 315,000 diagnosed patients with NASH with moderate to advanced liver fibrosis under the care of the liver specialist physicians during the launch of Rezdiffra.
NASH is also known as metabolic dysfunction associated steatohepatitis (MASH). In 2023, global liver disease medical societies and patient groups came together to rename the disease, with the goal of establishing an affirmative, non-stigmatizing name and diagnosis. Nonalcoholic fatty liver disease (NAFLD) was renamed metabolic dysfunction-associated steatotic liver disease (MASLD); NASH was renamed MASH; and an overarching term, steatotic liver disease (SLD), was established to capture multiple types of liver diseases associated with fat buildup in the liver. In addition to liver disease, patients with MASH have at least one related comorbid condition (e.g., obesity, hypertension, dyslipidemia, or type 2 diabetes).
About Rezdiffra
What is Rezdiffra?
Rezdiffra is a prescribed medicine used along with diet and exercise to treat adults with nonalcoholic steatohepatitis (NASH) with moderate to advanced liver scarring (fibrosis), but not with cirrhosis of the liver.
It is not known if Rezdiffra is safe and effective in children (under 18 years old).
This indication is approved based on improvement of NASH and liver scarring (fibrosis). There are ongoing studies to confirm the clinical benefit of Rezdiffra.
Before you take Rezdiffra, tell your healthcare provider about all of your medical conditions, including if you:
- have any liver problems other than NASH.
- have gallbladder problems or have been told you have gallbladder problems, including gallstones.
- are pregnant or plan to become pregnant. It is not known if Rezdiffra will harm your unborn baby.
- are breastfeeding or plan to breastfeed. It is not known if Rezdiffra passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby if you take Rezdiffra.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
- Rezdiffra and other medicines may affect each other, causing side effects. Rezdiffra may affect the way other medicines work, and other medicines may affect how Rezdiffra works.
- Especially tell your healthcare provider if you take medicines that contain gemfibrozil to help lower your triglycerides, or cyclosporine to suppress your immune system, because Rezdiffra is not recommended in patients taking these medicines.
- Tell your healthcare provider if you are taking medicines such as clopidogrel to thin your blood or statin medicines to help lower your cholesterol.
- Know the medicines you take. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine.
What are the possible side effects of Rezdiffra?
Rezdiffra may cause serious side effects, including:
- liver injury (hepatotoxicity). Stop taking Rezdiffra and call your healthcare provider right away if you develop the following signs or symptoms of hepatotoxicity: tiredness, nausea, vomiting, fever, rash, your skin or the white part of your eyes turns yellow (jaundice), pain or tenderness in the upper middle or upper right area of your stomach (abdomen).
- gallbladder problems. Gallbladder problems such as gallstones, inflammation of the gallbladder, or inflammation of the pancreas from gallstones can occur with NASH and may occur if you take Rezdiffra. Call your healthcare provider right away if you develop any signs or symptoms of these conditions including nausea, vomiting, fever, or pain in your stomach area (abdomen) that is severe and will not go away. The pain may be felt going from your abdomen to your back and the pain may happen with or without vomiting.
The most common side effects of Rezdiffra include: diarrhea, nausea, itching, stomach (abdominal) pain, vomiting, dizziness, constipation.
These are not all the possible side effects of Rezdiffra. For more information, ask your healthcare provider or pharmacist.
Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Madrigal at 1-800-905-0324.
Please see the full Prescribing Information, including Patient Information, for Rezdiffra.
About Madrigal Pharmaceuticals
Madrigal Pharmaceuticals, Inc. (Nasdaq: MDGL) is a biopharmaceutical company pursuing novel therapeutics for nonalcoholic steatohepatitis (NASH), a liver disease with high unmet medical need. Madrigal’s medication, Rezdiffra (resmetirom), is a once-daily, oral, liver-directed THR-β agonist designed to target key underlying causes of NASH. For more information, visit www.madrigalpharma.com.
Forward Looking Statements
This press release includes “forward-looking statements” made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, that are based on Madrigal’s beliefs and assumptions and on information currently available to it but are subject to factors beyond its control. Forward-looking statements reflect management’s current knowledge, assumptions, judgment and expectations regarding future performance or events. Forward-looking statements include all statements that are not historical facts; statements referenced by forward-looking statement identifiers; and statements regarding: Rezdiffra (resmetirom) and its expected use for treating NASH with moderate to advanced fibrosis.
Forward-looking statements can be identified by terms such as “accelerate,” “achieve,” “allow,” “anticipates,” “appear,” “be,” “believes,” “can,” “confidence,” “continue,” “could,” “demonstrates,” ”design,” “estimates,” “expectation,” “expects,” “forecasts,” “future,” “goal,” “help,” “hopeful,” “inform,” “intended,” “intends,” “may,” “might,” “on track,” “planned,” “planning,” “plans,” “positions,” “potential,” “powers,” “predicts,” ”predictive,” “projects,” “seeks,” “should,” “will,” “will achieve,” “will be,” “would”, “future” or similar expressions and the negatives of those terms.
Forward-looking statements are subject to a number of risks and uncertainties including, but not limited to: the assumptions underlying the forward-looking statements; risks of obtaining and maintaining regulatory approvals, including, but not limited to, potential regulatory delays or rejections; the challenges with the commercial launch of a new product, particularly for a company that does not have commercial experience; risks associated with meeting the objectives of Madrigal’s clinical studies, including, but not limited to Madrigal’s ability to achieve enrollment objectives concerning patient numbers (including an adequate safety database), outcomes objectives and/or timing objectives for Madrigal’s studies; any delays or failures in enrollment, and the occurrence of adverse safety events; risks related to the effects of Rezdiffra’s (resmetirom’s) mechanism of action; enrollment and trial conclusion uncertainties; market demand for and acceptance of our product; the potential inability to raise sufficient capital to fund ongoing operations as currently planned or to obtain financings on terms similar to those arranged in the past; the ability to service indebtedness and otherwise comply with debt covenants; outcomes or trends from competitive studies; future topline data timing or results; our ability to prevent and/or mitigate cyber-attacks; the timing and outcomes of clinical studies of Rezdiffra (resmetirom); the uncertainties inherent in clinical testing; and uncertainties concerning analyses or assessments outside of a controlled clinical trial. Undue reliance should not be placed on forward looking statements, which speak only as of the date they are made. Madrigal undertakes no obligation to update any forward-looking statements to reflect new information, events, or circumstances after the date they are made, or to reflect the occurrence of unanticipated events. Please refer to Madrigal’s submissions filed with the U.S. Securities and Exchange Commission, or SEC, for more detailed information regarding these risks and uncertainties and other factors that may cause actual results to differ materially from those expressed or implied. Madrigal specifically discusses these risks and uncertainties in greater detail in the sections appearing in Part I, Item 1A of its Annual Report on Form 10-K for the year ended December 31, 2023, filed with the SEC on February 28, 2024, , and Part II, Item 1A of its Quarterly Report on Form 10-Q for the quarter ended June 30, 2024, filed with the SEC on August 7, 2024, and as updated from time to time by Madrigal’s other filings with the SEC.
Investor Contact
Tina Ventura, IR@madrigalpharma.com
Media Contact
Christopher Frates, media@madrigalpharma.com
FAQ
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