Comprehensive Case Challenges in MASH

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Learning Objectives

•Identify noninvasive tools that optimize diagnosis, staging, and monitoring of MASH

•Develop treatment plans that incorporate recently approved and available therapies that target MASH and comorbid metabolic conditions

•Describe recent advances and ongoing research in MASH diagnostics and pharmacologic treatment options

MANAGING ADVANCED FIBROSIS IN PATIENTS WITH MASH

Case 1: Cristina

•73-year-old Latina woman

•BMI, 26 kg/m2

•ALT, 60 U/L; AST, 45 U/L

•TE, 12 kPa

•FIB-4 score, 2.0

•LDL-C, 120 mg/dL; BP, 135/80 mm Hg

•Family history of cirrhosis

•Not currently taking any medications or have any comorbidities

•Abnormal liver enzymes documented for the past 3 years

Benefits of Early Treatment Initiation

Liver Fibrosis Associated With Mortality

All-Cause Mortality

Mortality

PYF, person-years of follow-up. Dulai PS, et al. Hepatology. 2017;65(5):1557-1565.

Patient Eligibility for Resmetirom

Resmetirom was approved by the FDA for the treatment of MASH in 2024.1

Step 12,3

Confirm MASLD and exclude other causes of liver disease

Step 2

Step 3

Identify patients with confirmed or likely F2 or F3 for treatment Exclude cirrhosis Consider treatment with resmetirom AND

MASH with F2 or F3 OR

• TE, 8-15 kPa

• MRE, 3.1-4.4 kPa

• ELF score, 9.2-9.7 (+ second NIT)

• ELF score, 9.8-11.3 (if in isolation)

• FAST, MAST, MEFIB

Cirrhosis on biopsy OR

• TE, ≥20 kPa; MRE, ≥5 kPa; ELF score, >11.3

• Platelets, ≤140,000/μL

• Evidence of PHTN, active thyroid disease or concomitant acute liver disease

• Excess alcohol use

F, fibrosis stage; FAST, FibroScan-AST; MASLD, metabolic dysfunction-associated steatotic liver disease; MAST, magnetic resonance imaging-AST; MEFIB, MRE and FIB-4; MRE, magnetic resonance elastography; PHTN, portal hypertension; US Food and Drug Administration (FDA). 1. FDA. https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/217785s000lbl.pdf; 2. Noureddin M, et al. Clin Gastroenterol Hepatol. 2024;22(12):2367-2377; 3. Chen VL, et al. Hepatology. 2025;81(1):312-320.

Head-to-Head Comparison of Composite Biomarkers for Detecting At-Risk MASH

aP=0.01; bP<0.001.

+ ≥F2 (centrally reviewed) Should cut-offs be adapted to T2D?

N=245 patients with T2D and suspected MASLD from the QUID-NASH study underwent TE, MRE, MRI-PDFF, routine laboratory tests, and liver biopsy.

AUROC, area under the receive operating characteristic; FNI, fibrotic NASH index; MRI-PDFF, MRI-PDFF, magnetic resonance imaging-derived proton density fat

T2D, type 2 diabetes.

Castera L, et al. J Hepatol. 2024;81:195-206.

Exploring Emerging NITs

Score Components Cutoff Notes

FAST1,2 TE +AST ≥0.67

Predictivity is largely driven by AST, and this cutoff may fail to identify many patients

If MRE is 4.4–4.9, additional caution needed to exclude the presence of cirrhosis

Positive MEFIB has a high PPV of >90%

Corrects for sex for miR-34a-5p values

LSM, liver stiffness measure; PPV, positive predictive value. 1. Noureddin M, et al. Clin Gastroenterol Hepatol. 2024:S1542-3565(24)00667-0; 2. Newsome PN, et al. Lancet Gastroenterol Hepatol. 2020;5(4):362-373; 3. Noureddin M, et al. J Hepatol. 2022;76(4):781-787; 4. Ajmera V, et al. Therap Adv Gastroenterol. 2022;15:17562848221093869; 5. Harrison SA, et al. J Hepatol. 2023;79(3):758-767.

Assessing Safety and Efficacy of Resmetirom

Start

resmetirom

• 80 mg if <100 kg

• 100 mg if >100 kg

Confirm safety

• Routine DILI parameters

• Tolerability

Treatment monitoring, including safety (thyroid function, lipid profile)

Assess Safety and Efficacy No response

• Worsening of NITs

• Consistent increase inALT

Benefit uncertain

• TE <25% or MRE <20% drop in LSM

• ALT: no significant improvement

• MRI-PDFF: <30 reduction

Beneficial response

• ALT: significant improvement or normalization

• TE: ≥25% or MRE: ≥20% drop in LSM

Stop

Consider continuing, addon, or alternate approach

3 Months

6 Months

12 Months

Continue

Case 1 Continuation

•Had PNPLA3 test performed

GG phenotype

Role of PNPLA3 in MASH and Its Heritability

•Conflicting data about relationship between PNPLA3 and occurrence of cardiometabolic conditions and outcomes1

– Some evidence that it is protective against T2D

– Association with a prothrombotic status

•Improvements seen with tirzepatide across all 3 PNPLA3 phenotypes2

– MASH resolution greater for CC genotype vs G-allele carriers

– Fibrosis improvement similar across genotypes

•Genetic risk scores can help predict fibrosis progression and cirrhosis development3

• PNPLA3 risk alleles did not affect response to resmetirom4

•Therapies targeting PNPLA3 are currently in development3 HCC, hepatocellular carcinoma; T2D, type 2 diabetes. 1. Petta S, et al. Liver Int. 2024. [Epub ahead of print]; 2. Sanyal A, et al. AASLD. November 15-19, 2024; San Diego, CA. Abstract 5014; 3. Tulone A, et al. United European Gastroenterol J. 2024;12(5):638-648; 4. Chalasani N, et al. AASLD. November 15-19, 2024; San Diego, CA. Abstract 0004.

MASH Therapies in Phase 3 Trials

Drug Trial Name Population

Resmetirom MAESTRO-NASH MASH with F1b, F2, or F3

Semaglutide ESSENCE MASH with F2 or F3

Lanifibranor NATiV3 MASH with F2 or F3

Efruxifermin Compensated cirrhosis due to MASH

Noninvasively diagnosed MASH or MASLD MASH with F2 or F3

Pegozafermin Compensated cirrhosis due to MASH

ENLIGHTEN-Fibrosis MASH with F2 or F3

Denifanstat FASCINATE-3 Mash with F2 or F3

FASCINIT MASLD and MASH

Survodutide LIVERAGE - Cirrhosis MASH with cirrhosis LIVERAGE MASH with F2 or F3

National Library of Medicine. www.clinicaltrials.gov.

MANAGING MASH AND ITS METABOLIC COMORBIDITIES

Case 2: Richard

•68-year-old White man

•BMI, 35 kg/m2

•7-year history of T2D; HbA1c, 8.1%

•eGFR, 57 mL/min/1.73 m2

•ALT, 72 U/L; AST, 65 U/L; GGT, 119 U/L

•9-year history of obstructive sleep apnea

•LDL-C, 85 mg/dL

•Liver biopsy 2 years ago, F2

•Has received vitamin E in the past but no effect on his AST or ALT

•Currently taking atorvastatin and metformin

Effect of GLP-1 RAs on MACE

Semaglutide Reduced MACE in Adults With Obesity and CVD1,a Tirzepatide Predicted to Reduce Future CVD Events2,b

Efficacy of GLP-1 RAs in Patients With

Semaglutide Significantly Reduced Body Weight at Week 681,a

Obesity

Tirzepatide Significantly Reduced Body Weight at Week 722,b a

Efficacy of Semaglutide in MASH ESSENCE Trial

Efficacy of a Dual GIP/GLP-1 RA in MASH SYNERGY-NASH Trial

Effect of Bariatric Surgery on Fibrosis in Patients With MASH

Improvement in Fibrosis Following Bariatric Surgery Among Patients With Advanced Fibrosis

Bariatric Surgery Resulted in Improved Liver Histology

The Future of Therapies to Target MASH and

Cardiometabolic Comorbidities

•Semaglutide + cilofexor/firsocostat1

– Phase 2 trial showed combination was well tolerated, with greater improvement in steatosis, fibrosis, and liver biochemistry vs semaglutide monotherapy

•Weight loss similar to semaglutide monotherapy

•Survodutide2,3

– Phase 2 trial showed greater rates of improvement in MASH, reduced liver fat, and improvement in fibrosis by ≥1 stage vs placebo

– Ongoing phase 3 trials

– Breakthrough therapy designation for noncirrhotic MASH

•Semaglutide + zalfermin4

– Phase 2 trial ongoing in patients with MASH and F2-F4

1. Alkhouri N, et al. J Hepatol. 2022;77(3):607-618; 2. Sanyal A, et al. N Engl J Med. 2024;391(4):311-319; 3. Boehringer Ingelheim. https://www.boehringer-ingelheim.com/humanhealth/metabolic-diseases/survodutide-us-fda-breakthrough-therapy-phase-3-trials-mash; 4. ClinicalTrials.gov. https://clinicaltrials.gov/study/NCT05016882.

Conclusions

•Resmetirom is the first FDA-approved therapy for MASH

–Eligible patients have MASH with F2/F3

•In addition to established NITs such as FIB-4, TE, and ELF, new NITs are emerging to help stratify fibrosis risk and stage

•There are genetic components that indicate risk for progression in patients with MASLD/MASH

•GLP-1 RAs continue to be a treatment option for patients with cardiometabolic comorbidities and have shown some efficacy in treating MASH

•Bariatric surgery can be considered for patients with MASH and have shown positive liver-related outcomes

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