Study to Explore the Efficacy and Safety of BIO89-100 (Pegozafermin) in Participants With Severe Hypertriglyceridemia

NCT04541186 | Completed Phase 2 Results FDA Drug

• 1 other identifier: BIO89-100-221
• Study Type: interventional
• Target: 86
• Locations: 4 countries
• Sites: 48

Brief Summary

This study is designed to assess the efficacy, safety, and tolerability of different doses and dose regimens (once weekly \[QW\] or every 2 weeks \[Q2W\]), subcutaneous (SC) dosing of BIO89-100 (pegozafermin) compared to placebo in participants with severe hypertriglyceridemia (SHTG).

Conditions

Severe Hypertriglyceridemia

Keywords

Hypertriglyceridemia; Dyslipidemia; Lipid disorder; FGF21

Outcome Measures

Primary Outcomes (1)

• Percent Change From Baseline to Week 8 in Serum Triglyceride (TG)

  Baseline, Week 8

Secondary Outcomes (7)

• Number of Participants Who Achieved TG <500 mg/dL at Week 8

  Week 8

• Percent Change From Baseline to Week 8 in Non-high-density Lipoprotein Cholesterol (Non-HDL-C), Apolipoprotein B100 (ApoB), Low-density Lipoprotein Cholesterol (LDL-C), and High-density Lipoprotein Cholesterol (HDL-C)

  Baseline, Week 8

• Percent Change From Baseline to Week 8 in Very Low-density Lipoprotein Cholesterol (VLDL-C) and VLDL-TG

  Baseline, Week 8

• Percent Change in Baseline to Week 8 in Fasting Plasma Glucose, Adiponectin, and Body Weight

  Baseline, Week 8

• Percent Change From Baseline to Week 8 in Liver Fat as Assessed by Magnetic Resonance Imaging - Whole Liver Proton Density Fat Fraction (MRI-PDFF)

  Baseline, Week 8

Study Arms (5)

Pegozafermin 9 mg QW

EXPERIMENTAL

Participants received pegozafermin 9 mg QW as an SC injection for 8 weeks in the Main Study cohort.

Drug: Pegozafermin

Pegozafermin 18 mg QW

EXPERIMENTAL

Participants received pegozafermin 18 mg QW as an SC injection for 8 weeks in the Main Study cohort.

Drug: Pegozafermin

Pegozafermin 27 mg QW

EXPERIMENTAL

Participants received pegozafermin 27 mg QW as an SC injection for 8 weeks in the Main Study cohort or Fibrate Expansion cohort. Main Study and Fibrate Expansion pegozafermin 27 mg QW groups were pooled together due to low sample size in the expansion cohort.

Drug: Pegozafermin

Pegozafermin 36 mg Q2W

EXPERIMENTAL

Participants received pegozafermin 36 mg Q2W as an SC injection for 8 weeks in the Main Study cohort.

Drug: Pegozafermin

Placebo

PLACEBO COMPARATOR

Matching placebo was injected at matching frequency per assigned cohort for 8 weeks in the Main Study cohort or Fibrate Expansion cohort. Main Study and Fibrate Expansion placebo groups were pooled together due to low sample size in the expansion cohort.

Drug: Placebo

Interventions

Pegozafermin DRUG

Subcutaneous injection

Also known as: BIO89-100

Pegozafermin 18 mg QW; Pegozafermin 27 mg QW; Pegozafermin 36 mg Q2W; Pegozafermin 9 mg QW

Placebo DRUG

Matching placebo

Placebo

Eligibility Criteria

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

You may qualify if:

• Male or female age ≥21 to ≤75 years.
• Screening fasting triglyceride ≥500 milligrams (mg)/deciliters (dL) and ≤2000 mg/dL.
• Willing to maintain current eating and exercise habits from time of signing the informed consent and for the duration of the study.
• Participants could be taking statins and/or prescription fish oil as background therapy or not be taking any background therapy.
• Magnetic resonance imaging - whole liver proton density fat fraction (MRI-PDFF) of ≥6% for participants screened for the Fibrate Expansion cohort.

You may not qualify if:

• Uncontrolled or newly diagnosed hypertension.
• Body mass index \>45 kilograms (kg)/meters squared (m\^2).
• Receiving niacin, proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors, or supplements that could lower lipid levels.
• Type 1 diabetes mellitus.
• Diagnosis of Type 2 diabetes mellitus \<6 months prior to screening.
• History of malignancy within 5 years prior to screening.
• Participants with known lipoprotein lipase impairment or deficiency (Fredrickson Type 1), apolipoprotein C-II deficiency, or familial dysbetalipoproteinemia (Fredrickson Type 3).
• Clinically or otherwise documented cardiovascular or cerebrovascular disease.
• Weight change ≥5% in 3 months prior to first screening visit or weight change ≥5% during screening or planning to try to lose weight during conduct of study.

Sponsors & Collaborators

• 89bio, Inc.: lead

Study Sites (48)

89Bio Clinical Study Site

La Mesa, California, 91942, United States

Location

89Bio Clinical Study Site

San Francisco, California, 94158, United States

Location

89Bio Clinical Study Site

Clearwater, Florida, 33756, United States

Location

89Bio Clinical Study Site

Greenacres City, Florida, 33467, United States

Location

89Bio Clinical Study Site

Jacksonville, Florida, 32205, United States

Location

89Bio Clinical Study Site

Miami, Florida, 33125, United States

Location

89Bio Clinical Study Site

Miami, Florida, 33135, United States

Location

89Bio Clinical Study Site

Miami Lakes, Florida, 33014, United States

Location

89Bio Clinical Study Site

Miami Lakes, Florida, 33016, United States

Location

89Bio Clinical Study Site

North Miami Beach, Florida, 33162, United States

Location

89Bio Clinical Study Site

Orlando, Florida, 32825, United States

Location

89Bio Clinical Study Site

Tampa, Florida, 33606, United States

Location

89Bio Clinical Study Site

Lawrenceville, Georgia, 30044, United States

Location

89Bio Clinical Study Site

Quincy, Illinois, 62301, United States

Location

89Bio Clinical Study Site

Wauconda, Illinois, 60084, United States

Location

89Bio Clinical Study Site

West Des Moines, Iowa, 50266, United States

Location

89Bio Clinical Study Site

Louisville, Kentucky, 40213, United States

Location

89Bio Clinical Study Site

Olive Branch, Mississippi, 38654, United States

Location

89Bio Clinical Study Site

Albany, New York, 12203, United States

Location

89Bio Clinical Study Site

Greensboro, North Carolina, 27408, United States

Location

89Bio Clinical Study Site

Morganton, North Carolina, 28655, United States

Location

89Bio Clinical Study Site

Marion, Ohio, 43303, United States

Location

89Bio Clinical Study Site

Summerville, South Carolina, 29435, United States

Location

89Bio Clinical Study Site

Chattanooga, Tennessee, 37421, United States

Location

89Bio Clinical Study Site

Kingsport, Tennessee, 37660, United States

Location

89Bio Clinical Study Site

Austin, Texas, 78705, United States

Location

89Bio Clinical Study Site

Dallas, Texas, 75204, United States

Location

89Bio Clinical Study Site

Dallas, Texas, 77365, United States

Location

89Bio Clinical Study Site

Houston, Texas, 77089, United States

Location

89Bio Clinical Study Site

Lampasas, Texas, 76550, United States

Location

89Bio Clinical Study Site

Magnolia, Texas, 77355, United States

Location

89Bio Clinical Study Site

Manassas, Virginia, 20110, United States

Location

89Bio Clinical Study Site

Pardubice, 530 02, Czechia

Location

89Bio Clinical Study Site

Prague, 100 00, Czechia

Location

89Bio Clinical Study Site

Prague, 14021, Czechia

Location

89Bio Clinical Study Site

Prague, 150 00, Czechia

Location

89Bio Clinical Study Site

Prague, 158 00, Czechia

Location

89Bio Clinical Study Site

Miskolc, Borsod-Abauj Zemplen county, 3529, Hungary

Location

89Bio Clinical Study Site

Baja, Hungary

Location

89Bio Clinical Study Site

Békéscsaba, 5600, Hungary

Location

89Bio Clinical Study Site

Budapest, 1032, Hungary

Location

89Bio Clinical Study Site

Debrecen, 4032, Hungary

Location

89Bio Clinical Study Site

Bialystok, 15-351, Poland

Location

89Bio Clinical Study Site

Bialystok, 15-879, Poland

Location

89Bio Clinical Study Site

Lodz, 93-338, Poland

Location

89Bio Clinical Study Site

Oświęcim, 32-600, Poland

Location

89Bio Clinical Study Site

Rzeszów, 35-055, Poland

Location

89Bio Clinical Study Site

Torun, 87-100, Poland

Location

Related Publications (1)

• Bhatt DL, Bays HE, Miller M, Cain JE 3rd, Wasilewska K, Andrawis NS, Parli T, Feng S, Sterling L, Tseng L, Hartsfield CL, Agollah GD, Mansbach H, Kastelein JJP; ENTRIGUE Principal Investigators. The FGF21 analog pegozafermin in severe hypertriglyceridemia: a randomized phase 2 trial. Nat Med. 2023 Jul;29(7):1782-1792. doi: 10.1038/s41591-023-02427-z. Epub 2023 Jun 24.

  PMID: 37355760 RESULT

MeSH Terms

Conditions

Hypertriglyceridemia; Dyslipidemias; Lipid Metabolism Disorders

Condition Hierarchy (Ancestors)

Hyperlipidemias; Metabolic Diseases; Nutritional and Metabolic Diseases

Limitations and Caveats

The study did not separately evaluate the response to pegozafermin in participants who were on and who were not on concurrent fibrate therapy due to small sample size in the Fibrate Expansion cohort.

Results Point of Contact

• Title: Entrigue Study Team
• Organization: 89bio, Inc.

Ct.gov_SHTG@89bio.com

1-415-432-9270

Publication Agreements

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

Study Design

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

Study Record Dates

• First Submitted: August 14, 2020
• First Posted: September 9, 2020
• Study Start: September 1, 2020
• Primary Completion: May 19, 2022
• Study Completion: May 31, 2022
• Last Updated: July 25, 2024
• Results First Posted: July 25, 2024

Record last verified: 2024-06

Data Sharing

• IPD Sharing: Will not share

Locations

HU (5); PL (6); CZ (5); US (32)