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

Phase 2

Completed

• Conditions: Severe Hypertriglyceridemia
• Interventions: Drug: Pegozafermin; Drug: Placebo

• Registration Number: NCT04541186
• Lead Sponsor: 89bio, Inc.

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).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2020-08-14
• Study Start: 2020-09-01
• Study First Submitted QC: 2020-09-01
• Study First Posted: 2020-09-09
• Primary Completion: 2022-05-19
• Study Completion: 2022-05-31
• Results First Submitted: 2024-05-08
• Status Verified: 2024-06
• Results First Submitted QC: 2024-06-30
• Last Update Submitted: 2024-06-30
• Results First Posted: 2024-07-25
• Last Update Posted: 2024-07-25

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 86

Inclusion Criteria

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

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Exclusion Criteria

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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Pegozafermin 36 mg Q2W	Pegozafermin	Participants received pegozafermin 36 mg Q2W as an SC injection for 8 weeks in the Main Study cohort.
Pegozafermin 9 mg QW	Pegozafermin	Participants received pegozafermin 9 mg QW as an SC injection for 8 weeks in the Main Study cohort.
Pegozafermin 27 mg QW	Pegozafermin	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.
Pegozafermin 18 mg QW	Pegozafermin	Participants received pegozafermin 18 mg QW as an SC injection for 8 weeks in the Main Study cohort.
Placebo	Placebo	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.

Primary Outcome Measures

Name	Time	Method
Percent Change From Baseline to Week 8 in Serum Triglyceride (TG)	Baseline, Week 8

Secondary Outcome Measures

Name	Time	Method
Percent Change From Baseline to Week 8 in Very Low-density Lipoprotein Cholesterol (VLDL-C) and VLDL-TG	Baseline, Week 8
Percent Change From Baseline to Week 8 in High-sensitivity C-reactive Protein (hsCRP)	Baseline, Week 8
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	Least Squares Means were calculated using mixed-model repeated measures (MMRM).
Percent Change in Baseline to Week 8 in Fasting Plasma Glucose, Adiponectin, and Body Weight	Baseline, Week 8	Least Squares Mean was calculated using MMRM.
Percent Change From Baseline to Week 8 in Alanine Aminotransferase (ALT) and Aspartate Aminotransferase (AST)	Baseline, Week 8	Least Squares Mean was calculated using MMRM.
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	Least Squares Mean was calculated using analysis of covariance (ANCOVA).

Trial Locations

Locations (1)

89Bio Clinical Study Site; 🇵🇱 Toruń, Poland