Close-up of a scientist working in a lab to develop innovative therapies to treat liver and cardiometabolic diseases. Close-up of a scientist working in a lab to develop innovative therapies to treat liver and cardiometabolic diseases.

Advancing Into Clinical Development for Multiple Indications

Scientific Publications

Diagnostic Potential of FAST and AGILE3+ Scores for F2/F3 Fibrosis: An Analysis of the Phase 2 ENLIVEN Study

N. Alkhouri, A.J. Sanyal, G.D. Agollah, S. Feng, L. Tsai, N. Rash, M.D. Gottwald, M. Margalit, H. Mansbach, R. Loomba. Presented at The Liver Meeting AASLD, November 15-19, 2024.

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Diagnostic Potential of FAST and AGILE3+ Scores for F2/F3 Fibrosis: An Analysis of the Phase 2 ENLIVEN Study
Analysis and Integration of Machine Learning for Biomarker Assessments and Their Association With Treatment Response: Insights from the ENLIVEN Phase 2b Trial of Pegozafermin

O. Mansbach, A.J. Sanyal, R. Savic, L. Tseng, K. Balic, S. Feng, L. Tsai, M. Gottwald, M. Margalit, G.D. Agollah, H. Mansbach, Q. Anstee. Presented at The Liver Meeting AASLD, November 15-19, 2024.

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Analysis and Integration of Machine Learning for Biomarker Assessments and Their Association With Treatment Response: Insights from the ENLIVEN Phase 2b Trial of Pegozafermin
Pegozafermin Reduced Progression to Cirrhosis: A Post-Hoc Analysis From the Phase 2b ENLIVEN Study

J. Schattenberg, A.J. Sanyal, M.F. Abdelmalek, M. Margalit, G.D Agollah, M.D. Gottwald, S. Feng, L. Tseng, C.L. Hartsfield, H. Mansbach, R. Loomba. Presented at The Liver Meeting AASLD, November 15-19, 2024.

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Pegozafermin Reduced Progression to Cirrhosis: A Post-Hoc Analysis From the Phase 2b ENLIVEN Study
Biomarker Response in Metabolic Dysfunction-Associated Steatohepatitis (MASH) Patients With High-risk Baseline FAST Scores: Observations From the ENLIVEN Phase 2b Trial With Pegozafermin

N. Alkhouri, Q.M. Anstee, R. Loomba, M.F. Abdelmalek, K.V. Kowdley, M. Margalit, G.D. Agollah, S. Feng, C.L. Hartsfield, H. Mansbach, A.J. Sanyal. Presented at The Liver Meeting AASLD, November 15-19, 2024.

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Biomarker Response in Metabolic Dysfunction-Associated Steatohepatitis (MASH) Patients With High-risk Baseline FAST Scores: Observations From the ENLIVEN Phase 2b Trial With Pegozafermin
No Increase in the Risk of Liver or Muscle Toxicity in Pegozafermin-treated Patients with Metabolic Dysfunction-Associated Steatohepatitis (MASH) on Statin Background Therapy

O. Mansbach, L. Tseng, M. Margalit, H. Mansbach, K. Lichti-Kaiser, M. Gottwald, K. Balic. Presented at the American College of Clinical Pharmacology (ACCP) Annual Meeting, September 8–10, 2024.

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No Increase in the Risk of Liver or Muscle Toxicity in Pegozafermin-treated Patients with Metabolic Dysfunction-Associated Steatohepatitis (MASH) on Statin Background Therapy
Population Pharmacokinetics (PK) and Pharmacodynamics (PD) of Pegozafermin, a Novel GlycoPEGylated FGF21, in a Phase 2 Study in Severe Hypertriglyceridemia

L. Tseng, K. Balic, T. Parli, H. Mansbach, R.M. Savic. Presented at the American College of Clinical Pharmacology (ACCP) Annual Meeting, September 8–10, 2024.

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Population Pharmacokinetics (PK) and Pharmacodynamics (PD) of Pegozafermin, a Novel GlycoPEGylated FGF21, in a Phase 2 Study in Severe Hypertriglyceridemia
Week 48 results from the Phase 2b ENLIVEN extension study investigating pegozafermin for the treatment of metabolic dysfunction-associated steatohepatitis with fibrosis

R. Loomba, M.F. Abdelmalek, K.V. Kowdley, N. Alkhouri, J.M. Schattenberg, M.D. Gottwald, S. Feng, G.D. Agollah, C.L. Hartsfield, H. Mansbach, M. Margalit, A.J. Sanyal. Presented at the European Association for the Study of the Liver (EASL) Congress, June 5-8, 2024.

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Week 48 results from the Phase 2b ENLIVEN extension study investigating pegozafermin for the treatment of metabolic dysfunction-associated steatohepatitis with fibrosis
Pegozafermin added to background GLP-1 therapy in patients with metabolic dysfunction-associated steatohepatitis with F2/F3 fibrosis: ENLIVEN 48-week extension data

A.J. Sanyal, M.F. Abdelmalek, K.V. Kowdley, N. Alkhouri, M.D. Gottwald, S. Feng, G.D. Agollah, C.L. Hartsfield, H. Mansbach, M. Margalit, R. Loomba. Presented at the European Association for the Study of the Liver (EASL) Congress, June 5-8, 2024.

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Pegozafermin added to background GLP-1 therapy in patients with metabolic dysfunction-associated steatohepatitis with F2/F3 fibrosis: ENLIVEN 48-week extension data
Randomized, controlled trial of the FGF21 analogue pegozafermin in NASH

R. Loomba, A.J. Sanyal, K.V. Kowdley, D.L. Bhatt, N. Alkhouri, J.P. Frias, P. Bedossa, S.A. Harrison, D. Lazas, R. Barish, M.D. Gottwald, S. Feng, et al. N Engl J Med. 2023;389:998–1008.

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Fibrosis Improvement with Pegozafermin Treatment in MASH Patients with F4 Fibrosis
The FGF21 analog pegozafermin in severe hypertriglyceridemia: A randomized phase 2 trial

D.L. Bhatt, H.E. Bays, M. Miller, J.E. Cain III, K. Wasilewska, N.S. Andrawis, T. Parli, S. Feng, L. Sterling, L. Tseng, C.L. Hartsfield, G.D. Agollah, H. Mansbach, J.J.P. Kastelein & ENTRIGUE Principal Investigators. Nature Med. 2023;29:1782–1792.

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Fibrosis Improvement with Pegozafermin Treatment in MASH Patients with F4 Fibrosis
Fibrosis Improvement with Pegozafermin Treatment in MASH Patients with F4 Fibrosis

R. Loomba, A. J. Sanyal, K.V. Kowdley, N. Alkhouri, P. Bedossa, S.A. Harrison, M. Gottwald, S. Feng, G.D. Agollah, C.L. Hartsfield, H. Mansbach, M. Margalit, M.F. Abdelmalek. Presented at The Liver Meeting AASLD, November 10-14, 2023.

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Fibrosis Improvement with Pegozafermin Treatment in MASH Patients with F4 Fibrosis
Pegozafermin for the treatment of non-alcoholic steatohepatitis patients with F2/F3 fibrosis: A multi-center, randomized, double-blind, Placebo-controlled Phase 2b trial

R. Loomba, A. J. Sanyal, K. V. Kowdley, D. L. Bhatt, N. Alkhouri, J. P. Frias, P. Bedossa, S. A. Harrison, D. Lazas, R. Barish, M. Gottwald, S. Feng, G. D. Agollah, C. L. Hartsfield, H. Mansbach, M. Margalit, M. F. Abdelmalek. Presented at the EASL International Liver Congress, June 21-24, 2023.

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EASL 2023 presentation: Pegozafermin for NASH patients with F2/F3 fibrosis: Phase 2b ENLIVEN study.
Pegozafermin inhibits NASH-induced hepatocellular carcinoma in the STAM™ mouse model

M. Margalit, M. Rosenstock, L. Tseng, T. Hashiguchi, Y. Shirakata, H. Mansbach. Presented at the EASL International Liver Congress, June 21-24, 2023.

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EASL 2023 poster: Pegozafermin inhibits NASH-induced hepatocellular carcinoma in the STAM™ mouse model.
Pegozafermin Provides Beneficial Lipid Effects in Subjects with Severe Hypertriglyceridemia (SHTG) Regardless of Background Lipid Modifying Therapy Status: An Analysis of the Phase 2 ENTRIGUE study

D. L. Bhatt, H.E. Bays, M. Miller, T. Parli, S. Feng, L. Sterling, C. L. Hartsfield, G. D. Agollah, H. Mansbach, J. J. P. Kastelein. Presented at the American College of Cardiology, March 3-6, 2023.

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ACC 2023 poster: Pegozafermin has beneficial lipid effects in SHTG: Phase 2 ENTRIGUE study.
Safety, pharmacokinetics, and pharmacodynamics of pegozafermin in patients with non-alcoholic steatohepatitis: a randomised, double-blind, placebo-controlled, phase 1b/2a multiple-ascending-dose study

Rohit Loomba, Eric J Lawitz, Juan Pablo Frias, Grisell Ortiz-Lasanta, Lars Johansson, Bridgette Boggess Franey, Linda Morrow, Moti Rosenstock*, Cynthia L Hartsfield, Chao-Yin Chen, Leo Tseng, R Will Charlton, Hank Mansbach, Maya Margalit

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Lancet Gastroenterol Hepatol 2022: Pegozafermin in patients with NASH – Phase 1b/2a multiple-ascending-dose study.
Variability in Liver Biopsy Assessment: Data from the Pegozafermin Phase 1b/2a Study in Subjects with Non-Alcoholic Steatohepatitis (NASH)

R. Loomba, S. Feng, L. Tseng, G. D. Agollah, R.W. Charlton, J. S. Iyer, K. Wack, T. Kwan, P. Shin, H. Mansbach, M. Margalit. Presented at The Liver Meeting AASLD, November 4-8, 2022.

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AASLD 2023 poster: Variability in liver biopsy assessment: Data from pegozafermin Phase 1b/2a study in NASH.
Pegozafermin for the Treatment of Severe Hypertriglyceridemia: A Randomized, Double-blind, Placebo-controlled Phase 2 Study (ENTRIGUE STUDY)

D. Bhatt, H.E. Bays, M. Miller, T. Parli, S. Feng, L. Sterling, C.L. Hartsfield, G.D. Agollah, H. Mansbach, J.J.P. Kastelein. Presented at the Cardiometabolic Health Congress October 19-22, 2022.

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CMHC 2022 poster: Pegozafermin for SHTG: Phase 2 ENTRIGUE study.
Pegozafermin Led to Significant Metabolic Benefits, in Addition to Robust Beneficial Effects on the Liver, in an Open-label Cohort of a Phase 1b/2a Study in Subjects with Non-alcoholic Steatohepatitis (NASH)

N. Alkhouri, R. Loomba, J. P. Frias, L. Morrow, S. Feng, L. Tseng, G.D. Agollah, W.R Charlton, H. Mansbach, M. Margalit, S. Harrison. Presented at the Cardiometabolic Health Congress October 19-22, 2022.

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CMHC 2022 poster: Pegozafermin benefits: Phase 1b/2a study in subjects with NASH.
Pegozafermin Improved Liver Histology, Liver-Related Non-Invasive Tests (NITs) and Metabolic Profiles in an Open-Label Cohort of a Phase 1b/2a Study in Subjects with Non-Alcoholic Steatohepatitis

R. Loomba, N. Alkhouri, S. Feng, L. Tseng, G. D. Agollah, H. Mansbach, M. Margalit, C.L. Hartsfield, S.A. Harrison. Presented at the Cardiometabolic Health Congress October 19-22, 2022.

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CMHC 2022 poster: Pegozafermin improved multiple measures: Phase 1b/2a study in subjects with NASH.
Phase 2 Trial of Pegozafermin in Severe Hypertriglyceridemia

D. Bhatt, H. Bays, M. Miller, J.E. Cain III, K. Wasilewska, N. S. Andrawis, T. Parli, S. Feng, L. Sterliing, C.L. Hartsfield, G.D. Agollah, and H. Mansbach. Presented at the European Society of Cardiology (ESC) Congress 2022 Barcelona, Onsite & Online, August 26-29, 2022.

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ESC 2022 presentation: Phase 2 trial of pegozafermin in severe hypertriglyceridemia.
Pegozafermin Led to Significant Metabolic Benefits, in Addition to Robust Beneficial Effects on the Liver, in an Open label Cohort of a Phase 1b/2a Study in Subjects with Nonalcoholic Steatohepatitis (NASH)

N. Alkhouri, R. Loomba, J.P. Frias, L. Morrow, S. Feng, L. Tseng, G.D. Agollah, W.R. Charlton, H. Mansbach, M. Margalit, and S. Harrison. Poster presented at the EASL International Liver Congress, June 22-26, 2022.

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EASL 2022 poster: Pegozafermin metabolic and liver benefits: Phase 1b/2a study in NASH.
Pegozafermin Led to Improved Liver Histology, Liver-related Non-invasive Tests and Metabolic Profile, With Favorable Safety and Tolerability, in an Open label Cohort of a Phase 1b/2a Study in Subjects With Nonalcoholic Steatohepatitis

R. Loomba, N. Alkhouri, D. Lazas, P. Bedossa, L. Morrow, S. Feng, L. Tseng, G.D. Agollah, W.R. Charlton, H. Mansbach, M. Margalit, and S. Harrison. Poster presented at the EASL International Liver Congress, June 22-26, 2022.

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EASL 2022 poster: Pegozafermin efficacy and safety results: Phase 1b/2a study in subjects with NASH.
Pharmacokinetics (PK) and Pharmacodynamics (PD) of BIO89-100, a Novel GlycoPEGylated FGF21, in Nonalcoholic Steatohepatitis (NASH) Patients with Compensated Cirrhosis

N. Alkhouri, R.W. Charlton, H. Mansbach, M. Margalit, K. Balic, and L. Tseng. Poster presented at the EASL International Liver Congress, June 22-26, 2022.

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EASL 2022 poster: PK/PD of BIO89-100, a novel glycoPEGylated FGF21, in NASH patients with compensated cirrhosis.
Pegozafermin Treatment Ameliorates a Subclinical Increase in Spleen Volume, That Is Associated With a Worsening Metabolic Profile, in Non-cirrhotic NASH in Correlation With Change in Liver Fat and Inflammatory Markers in a Phase 1b/2a, Placebo-Controlled, Double-Blind Proof of Concept Study

J.P. Frias, L. Johansson, R.W. Charlton, S. Feng, G.D. Agollah, H. Mansbach, and M. Margalit. Poster presented at ENDO 2022, June 11-14, 2022.

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ENDO 2022 poster: Pegozafermin effects on spleen volume, liver fat, inflammatory markers: Phase 1b/2a study in NASH.

Posters

Bio89-100 Demonstrated Robust Reductions in Liver MRI-PDFF, Favorable Tolerability and Potential for every 2 Weeks Dosing in a Phase 1b/2a Placebo-Controlled, Double-Blind, Multiple Ascending Dose Study in NASH
Bio89-100 Demonstrated Robust Reductions in Liver MRI-PDFF, Favorable Tolerability and Potential for every 2 Weeks Dosing in a Phase 1b/2a Placebo-Controlled, Double-Blind, Multiple Ascending Dose Study in NASH
R. Loomba, E. J. Lawitz, B.B. Franey, L. Morrow, C. Chen, L. Tseng, H. Mansbach, M. Margalit. Poster presented at the Liver Meeting Digital Experience AASLD, November 13-16, 2020.
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BIO89-100, a novel glycoPEGylatedFibroblast Growth Factor 21 (FGF21) analog activates FGF receptors (FGFR) 1c, 2c and 3c but not FGFR4 in L6 cells transfected with the four different human FGFRs and beta-Klotho (KLB)
BIO89-100, a novel glycoPEGylatedFibroblast Growth Factor 21 (FGF21) analog activates FGF receptors (FGFR) 1c, 2c and 3c but not FGFR4 in L6 cells transfected with the four different human FGFRs and beta-Klotho (KLB)
M. Rosenstock, A. Pierce, A. Di Marco, G. Auciello, A. Bresciani, H. Mansbach, M.Margalit. Poster presented at the Liver Meeting Digital Experience AASLD, November 13-16, 2020.
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BIO89-100, a Novel Glycopegylated FGF21 Analogue, Demonstrates Robust Reduction in Serum Lipids and Long Half-life in a Phase 1 Randomized, Controlled Single Ascending Dose Trial in Healthy Subjects
BIO89-100, a Novel Glycopegylated FGF21 Analogue, Demonstrates Robust Reduction in Serum Lipids and Long Half-life in a Phase 1 Randomized, Controlled Single Ascending Dose Trial in Healthy Subjects
R. Loomba, H. Mansbach, L. Tseng, C. Chen, M. Rosenstock, G. Atiee and M. Margalit. Poster presented at the American Association for the Study of Liver Diseases (AASLD) Meeting, November 8-12, 2019 (Boston, MA).
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Weekly Subcutaneous Administration of BIO89-100, a Novel GlycoPEGylated-Fibroblast Growth Factor21 (FGF21) Analogue, Inhibits Sweetness Preference in Obese Cynomolgus Monkeys
Weekly Subcutaneous Administration of BIO89-100, a Novel GlycoPEGylated-Fibroblast Growth Factor21 (FGF21) Analogue, Inhibits Sweetness Preference in Obese Cynomolgus Monkeys
M. Rosenstock, Y. Liu, H. Mansbach and M. Margalit. Poster presented at the American Association for the Study of Liver Diseases (AASLD) Meeting, November 8-12, 2019 (Boston, MA).
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