Study Evaluating the Safety, Efficacy and Tolerability of BIO89-100 in Subjects With Biopsy-confirmed Nonalcoholic Steatohepatitis (NASH)

NCT04929483 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: BIO89-100-122
• Study Type: interventional
• Target: 222
• Locations: 2 countries
• Sites: 89

Brief Summary

This is a randomized, double-blind, placebo-controlled study that will evaluate the safety, efficacy, tolerability of BIO89-100 in patients with biopsy-confirmed fibrosis stages F2-F3 NASH.

Conditions

NASH - Nonalcoholic Steatohepatitis

Keywords

Liver Diseases; Fatty Liver; Digestive System Diseases; Non-alcoholic Fatty Liver Disease; Metabolic diseases; MASH MAFLD; NASH NAFLD

Outcome Measures

Primary Outcomes (2)

• Main Study: Number of Participants With Histological Resolution of Nonalcoholic Steatohepatitis (NASH) Without Worsening of Fibrosis

  Nonalcoholic fatty liver disease activity score (NAS) was the sum of the scores of steatosis, inflammation, and ballooning. NAS score ranged from 0 to 8, with higher scores indicating worse disease severity. Resolution of NASH was defined as the total absence of ballooning (score=0) and absent or mild inflammation (score=0 to 1). NASH clinical research system (CRN) fibrosis is staged on a 0-4 scale: 0 (none); 1 (perisinusoidal or periportal fibrosis); 2 (perisinusoidal and portal/periportal fibrosis); 3 (bridging fibrosis); 4 (cirrhosis). Worsening of fibrosis was defined as progression of fibrosis greater than or equal to (≥) 1 stage in NASH CRN fibrosis score.

  Week 24

• Main Study: Number of Participants Who Achieved Improvement of Fibrosis ≥1 Stage Without Worsening of NASH

  Worsening of NASH was defined as increase in nonalcoholic fatty liver disease activity score (NAS) for ballooning, inflammation, or steatosis. NAS was the sum of the scores of steatosis, inflammation, and ballooning. NAS score ranged from 0 to 8, with higher scores indicating worse disease severity. Fibrosis improvement was defined as ≥1-stage decrease in NASH CRN fibrosis score. NASH CRN fibrosis was staged on a 0-4 scale: 0 (none); 1 (perisinusoidal or periportal fibrosis); 2 (perisinusoidal and portal/periportal fibrosis); 3 (bridging fibrosis); 4 (cirrhosis).

  Week 24

Secondary Outcomes (18)

• Main Study: Number of Participants With at Least a 2-Point Improvement in NAS and no Worsening of Fibrosis

  Week 24

• Main Study: Number of Participants With NASH Resolution and Fibrosis Improvement ≥1 Stage

  Week 24

• Main Study: Number of Participants With at Least a 2-point Improvement in NAS Score and Are Magnetic Resonance Imaging - Proton Density Fat Fraction (MRI-PDFF) Responders and Alanine Aminotransferase (ALT) Responders

  Week 24

• Main Study: Percent Change From Baseline in Serum Triglycerides

  Baseline, Week 24

• Main Study: Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-c)

  Baseline, Week 24

Study Arms (6)

BIO89-100 - 15 mg once weekly (QW)

EXPERIMENTAL

Drug: BIO89-100

BIO89-100 - 30 mg QW

EXPERIMENTAL

Drug: BIO89-100

BIO89-100 - 44 mg once every 2 weeks (Q2W)

EXPERIMENTAL

Drug: BIO89-100

Placebo QW

PLACEBO COMPARATOR

Drug: Placebo

Placebo Q2W

PLACEBO COMPARATOR

Drug: Placebo

Placebo QW (Main study)/ BIO89-100 - 30 mg QW (Extension)

EXPERIMENTAL

Drug: BIO89-100; Drug: Placebo

Interventions

BIO89-100 DRUG

Subcutaneous injection

Also known as: Pegozafermin

BIO89-100 - 15 mg once weekly (QW); BIO89-100 - 30 mg QW; BIO89-100 - 44 mg once every 2 weeks (Q2W); Placebo QW (Main study)/ BIO89-100 - 30 mg QW (Extension)

Placebo DRUG

Subcutaneous injection

Placebo Q2W; Placebo QW; Placebo QW (Main study)/ BIO89-100 - 30 mg QW (Extension)

Eligibility Criteria

• Age: 21 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age 21 to 75
• Biopsy-confirmed NASH with fibrosis stage F2 or F3 per NASH CRN System and NAS ≥4, with a score of at least 1 in each of steatosis, ballooning degeneration, and lobular inflammation.
• Qualifying biopsy must be either within 6 months of screening visit or obtained during screening period

You may not qualify if:

• Have poorly controlled high blood pressure
• Have type 1 diabetes or poorly controlled type 2 diabetes.
• History of cirrhosis or evidence of cirrhosis by clinical, imaging, or liver biopsy evaluation
• Are planning to try to lose weight during the conduct of the study.
• Have a BMI \<25 kg/m2

Sponsors & Collaborators

• 89bio, Inc.: lead

Study Sites (89)

89bio Clinical Study Site

Birmingham, Alabama, 35209, United States

Location

89bio Clinical Study Site

Birmingham, Alabama, 35211, United States

Location

89bio Clinical Study Site

Dothan, Alabama, 36305, United States

Location

89bio Clinical Study Site

Guntersville, Alabama, 35976, United States

Location

89bio Clinical Study Site

Madison, Alabama, 35758, United States

Location

89bio Clinical Study Site

Chandler, Arizona, 85224, United States

Location

89bio Clinical Study Site

Glendale, Arizona, 85036, United States

Location

89bio Clinical Study Site

Peoria, Arizona, 85306, United States

Location

89bio Clinical Study Site

Tucson, Arizona, 85712-4044, United States

Location

89bio Clinical Study Site

Tucson, Arizona, 85712-4046, United States

Location

89bio Clinical Study Site

Tucson, Arizona, 85741, United States

Location

89bio Clinical Study Site

Little Rock, Arkansas, 72205-6414, United States

Location

89bio Clinical Study Site

Little Rock, Arkansas, 72205, United States

Location

89bio Clinical Study Site

Chula Vista, California, 91911, United States

Location

89bio Clinical Study Site

Huntington Park, California, 90255, United States

Location

89bio Clinical Study Site

Long Beach, California, 90808, United States

Location

89bio Clinical Study Site

Orange, California, 92866, United States

Location

89bio Clinical Study Site

Panorama City, California, 91402, United States

Location

89bio Clinical Study Site

Rialto, California, 92377, United States

Location

89bio Clinical Study Site

Santa Ana, California, 92704, United States

Location

89bio Clinical Study Site

Englewood, Colorado, 80113, United States

Location

89bio Clinical Study Site

Boynton Beach, Florida, 33472, United States

Location

89bio Clinical Study Site

Bradenton, Florida, 34208, United States

Location

89bio Clinical Study Site

Fort Myers, Florida, 33912, United States

Location

89bio Clinical Study Site

Lakeland, Florida, 33805, United States

Location

89bio Clinical Study Site

Maitland, Florida, 32127, United States

Location

89bio Clinical Study Site

Miami, Florida, 33014, United States

Location

89bio Clinical Study Site

Miami, Florida, 33147, United States

Location

89bio Clinical Study Site

Miami, Florida, 33157, United States

Location

89bio Clinical Study Site

Miami Lakes, Florida, 33016, United States

Location

89bio Clinical Study Site

Ocala, Florida, 34471, United States

Location

89bio Clinical Study Site

Palmetto Bay, Florida, 33157, United States

Location

89bio Clinical Study Site

Pinellas Park, Florida, 33781, United States

Location

89bio Clinical Study Site

Port Orange, Florida, 32127, United States

Location

89bio Clinical Study Site

Sarasota, Florida, 34240, United States

Location

89bio Clinical Study Site

Viera, Florida, 32940, United States

Location

89bio Clinical Study Site

Athens, Georgia, 30607, United States

Location

89bio Clinical Study Site

Sandy Springs, Georgia, 30328, United States

Location

89bio Clinical Study Site

New Albany, Indiana, 47150, United States

Location

89bio Clinical Study Site

South Bend, Indiana, 46635, United States

Location

89bio Clinical Study Site

Iowa City, Iowa, 52246, United States

Location

89bio Clinical Study Site

Topeka, Kansas, 66606, United States

Location

89bio Clinical Study Site

Marrero, Louisiana, 70072-3151, United States

Location

89bio Clinical Study Site

Marrero, Louisiana, 70072-3155, United States

Location

89bio Clinical Study Site

Monroe, Louisiana, 71201, United States

Location

89bio Clinical Study Site

Shreveport, Louisiana, 71103, United States

Location

89bio Clinical Study Site

Glen Burnie, Maryland, 21061, United States

Location

89bio Clinical Study Site

Greenbelt, Maryland, 20770, United States

Location

89bio Clinical Study Site

St Louis, Missouri, 63033, United States

Location

89bio Clinical Study Site

Las Vegas, Nevada, 89119, United States

Location

89bio Clinical Study Site

Las Vegas, Nevada, 89121, United States

Location

89bio Clinical Study Site

Florham Park, New Jersey, 07932, United States

Location

89bio Clinical Study Site

New York, New York, 10033, United States

Location

89bio Clinical Study Site

Concord, North Carolina, 28027, United States

Location

89bio Clinical Study Site

Columbus, Ohio, 43210, United States

Location

89bio Clinical Study Site

Dayton, Ohio, 45414, United States

Location

89bio Clinical Study Site

Springboro, Ohio, 45066, United States

Location

89bio Clinical Study Site

Westlake, Ohio, 44145, United States

Location

89bio Clinical Study Site

Greenwood, South Carolina, 29646, United States

Location

89bio Clinical Study Site

Summerville, South Carolina, 29485, United States

Location

89bio Clinical Study Site

Chattanooga, Tennessee, 37411, United States

Location

89bio Clinical Study Site

Chattanooga, Tennessee, 37421, United States

Location

89bio Clinical Study Site

Hermitage, Tennessee, 37076, United States

Location

89bio Clinical Study Site

Austin, Texas, 78757-8051, United States

Location

89bio Clinical Study Site

Austin, Texas, 78757-8059, United States

Location

89bio Clinical Study Site

Beaumont, Texas, 77702, United States

Location

89bio Clinical Study Site

Dallas, Texas, 75234, United States

Location

89bio Clinical Study Site

Dallas, Texas, 75246, United States

Location

89bio Clinical Study Site

Edinburg, Texas, 78539, United States

Location

89bio Clinical Study Site

Fort Worth, Texas, 76104, United States

Location

89bio Clinical Study Site

Garland, Texas, 75044, United States

Location

89bio Clinical Study Site

Houston, Texas, 77030, United States

Location

89bio Clinical Study Site

Houston, Texas, 77099, United States

Location

89bio Clinical Study Site

San Antonio, Texas, 78209, United States

Location

89bio Clinical Study Site

San Antonio, Texas, 78215, United States

Location

89bio Clinical Study Site

San Antonio, Texas, 78229-4801, United States

Location

89bio Clinical Study Site

San Antonio, Texas, 78229-5069, United States

Location

89bio Clinical Study Site

Waco, Texas, 76710, United States

Location

89bio Clinical Study Site

Waco, Texas, 76712, United States

Location

89bio Clinical Study Site

Wichita Falls, Texas, 76301, United States

Location

89bio Clinical Study Site

Ogden, Utah, 84405, United States

Location

89bio Clinical Study Site

Sandy City, Utah, 84092, United States

Location

89bio Clinical Study Site

Manassas, Virginia, 20110, United States

Location

89bio Clinical Study Site

Richmond, Virginia, 23235, United States

Location

89bio Clinical Study Site

Richmond, Virginia, 23249, United States

Location

89bio Clinical Study Site

Roanoke, Virginia, 24014, United States

Location

89bio Clinical Study Site

Seattle, Washington, 98105, United States

Location

89bio Clinical Study Site

Spokane, Washington, 99202, United States

Location

89bio Clinical Study Site

San Juan, 00927, Puerto Rico

Location

Related Publications (2)

• Tseng CL, Balic K, Charlton RW, Margalit M, Mansbach H, Savic RM. Population Pharmacokinetics and Pharmacodynamics of Pegozafermin in Patients with Nonalcoholic Steatohepatitis. Clin Pharmacol Ther. 2023 Dec;114(6):1323-1331. doi: 10.1002/cpt.3046. Epub 2023 Oct 2.

  PMID: 37696614 DERIVED

• Loomba R, Sanyal AJ, Kowdley KV, Bhatt DL, Alkhouri N, Frias JP, Bedossa P, Harrison SA, Lazas D, Barish R, Gottwald MD, Feng S, Agollah GD, Hartsfield CL, Mansbach H, Margalit M, Abdelmalek MF. Randomized, Controlled Trial of the FGF21 Analogue Pegozafermin in NASH. N Engl J Med. 2023 Sep 14;389(11):998-1008. doi: 10.1056/NEJMoa2304286. Epub 2023 Jun 24.

  PMID: 37356033 DERIVED

MeSH Terms

Conditions

Non-alcoholic Fatty Liver Disease; Liver Diseases; Fatty Liver; Digestive System Diseases; Metabolic Diseases

Condition Hierarchy (Ancestors)

Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Clinical Director
• Organization: 89bio, Inc.

bio89-100-122@89bio.com

1-858-261-8838

Study Officials

• Millie Gottwald, PharmD

  89bio, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 10, 2021
• First Posted: June 18, 2021
• Study Start: June 4, 2021
• Primary Completion: February 14, 2023
• Study Completion: October 8, 2024
• Last Updated: January 6, 2026
• Results First Posted: January 6, 2026

Record last verified: 2025-12

Locations

PR (1); US (88)