Phase 2
N=80
Study to Explore the Efficacy and Safety of BIO89-100 (Pegozafermin) in Participants With Severe Hypertriglyceridemia
Severe Hypertriglyceridemia
Bottom Line
View on ClinicalTrials.gov: NCT04541186 ↗Enrolled (actual)
80
Serious AEs
1.2%
Results posted
Jul 2024
Primary outcome: Primary: Percent Change From Baseline to Week 8 in Serum Triglyceride (TG) — -57.33; -11.85 percent change from Baseline — p=<0.001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Pegozafermin (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 21+ yrs
- Sex
- All
- Sponsor
- 89bio, Inc.
- Primary completion
- May 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change From Baseline to Week 8 in Serum Triglyceride (TG) |
-57.33; -11.85 | <0.001 sig |
| SECONDARY Number of Participants Who Achieved TG <500 mg/dL at Week 8 |
51; 5 | <0.001 sig |
| SECONDARY 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) |
-18.29; -0.57; -10.51; 1.07; 10.44; 8.72 | 0.007 sig |
| SECONDARY Percent Change From Baseline to Week 8 in Very Low-density Lipoprotein Cholesterol (VLDL-C) and VLDL-TG |
-47.96; -0.41; -58.50; -0.85 | 0.006 sig |
| SECONDARY Percent Change in Baseline to Week 8 in Fasting Plasma Glucose, Adiponectin, and Body Weight |
-3.90; -2.67; 69.53; 5.70; -0.15; -0.14 | 0.809 |
| SECONDARY Percent Change From Baseline to Week 8 in Liver Fat as Assessed by Magnetic Resonance Imaging - Whole Liver Proton Density Fat Fraction (MRI-PDFF) |
-42.18; -8.26 | 0.012 sig |
| SECONDARY Percent Change From Baseline to Week 8 in High-sensitivity C-reactive Protein (hsCRP) |
-21.43; -1.10 | — |
| SECONDARY Percent Change From Baseline to Week 8 in Alanine Aminotransferase (ALT) and Aspartate Aminotransferase (AST) |
-4.28; 0.43; -11.50; 0.51 | — |
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).
Eligibility Criteria
Inclusion Criteria
- 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.
Exclusion Criteria
- 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.
Data sourced from ClinicalTrials.gov (NCT04541186). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.