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

89bio

Status and phase: Completed; Phase 2

Conditions: Severe Hypertriglyceridemia

Treatments: Drug: Pegozafermin; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT04541186

BIO89-100-221

Details and patient eligibility

About

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).

Enrollment: 86 patients

Sex: All

Ages: 21 to 75 years old

Volunteers: No Healthy Volunteers

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.

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

---

86 participants in 5 patient groups, including a placebo group

Pegozafermin 9 mg QW

Experimental group

Description:

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

Treatment:

Drug: Pegozafermin

Pegozafermin 18 mg QW

Experimental group

Description:

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

Treatment:

Drug: Pegozafermin

Pegozafermin 27 mg QW

Experimental group

Description:

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.

Treatment:

Drug: Pegozafermin

Pegozafermin 36 mg Q2W

Experimental group

Description:

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

Treatment:

Drug: Pegozafermin

Placebo

Placebo Comparator group

Description:

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.

Treatment:

Drug: Placebo