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ISIS 681257

Phase 2

Elevated Lipoprotein(a) | Small molecule | Cardiovascular |Ionis Pharmaceuticals, Inc.|Last Updated: Oct 30, 2020

Success Probability

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Trial Design

RandomizedDouble-BlindCONTROLLEDDMCBiomarker
Total Trials3
Total Enrollment322

FDA Designations

No designations recorded

Clinical trial landscape

ISIS 681257 · 4 trials · 4 indications

Phase 2 1Phase 1 3
NCT03070782Phase 2 Study of ISIS 681257 (AKCEA-APO(a)-LRx) in Participants With Hyperlipoproteinemia(a) and Cardiovascular DiseaseElevated Lipoprotein(a)
COMPLETED286 Analytics
PHASE2COMPLETED
Phase 2 Study of ISIS 681257 (AKCEA-APO(a)-LRx) in Participants With Hyperlipoproteinemia(a) and Cardiovascular Disease
Elevated Lipoprotein(a)Unlock trial analytics

Study Endpoints

Primary Endpoints

Percent Change From Baseline in Fasting Lipoprotein A [Lp(a)] at the Primary Analysis Time Point
Baseline and Month 6 (Week 25 for Cohorts A, B and C and Week 27 for Cohorts D and E)

An ANCOVA model was performed on the log ratio of Lp(a) value at the Primary Analysis Time Point to Lp(a) value at Baseline. The estimate of the log ratio was converted back to the original scale and percent change was calculated using formula: = (ratio of Lp(a) value at the Primary Analysis Time Point to Lp(a) value at Baseline - 1) × 100.

Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Up to 16 weeks post treatment period (up to approximately 1.3 years)

An adverse event (AE) was defined as any unfavorable and unintended sign (including a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE was considered related to the investigational drug product. TEAEs was defined as any AE with onset after the first administration of study medication through the end of the study, or any event that was present at baseline but worsened in intensity or was subsequently considered drug-related by the Investigator through the end of the study.

Number of Participants With TEAEs by Maximum Severity
Up to 16 weeks post treatment period (up to approximately 1.3 years)

An AE was defined as any unfavorable and unintended sign (including a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE was considered related to the investigational drug product. TEAEs was defined as any AE with onset after the first administration of study medication through the end of the study, or any event that was present at baseline but worsened in intensity or was subsequently considered drug-related by the Investigator through the end of the study. The severity of TEAEs was assessed based on the National Cancer Institute's (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.03. TEAEs were graded on a 5-point scale where 1 = Mild, 2 = Moderate, 3 = Severe, 4 = Potentially life-threatening and 5 = Death.

Number of Participants With TEAEs Leading to Study Discontinuation
Up to 16 weeks post treatment period (up to approximately 1.3 years)

An AE was defined as any unfavorable and unintended sign (including a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE was considered related to the investigational drug product. TEAE was defined as any AE with onset after the first administration of study medication through the end of the study, or any event that was present at baseline but worsened in intensity or was subsequently considered drug-related by the Investigator through the end of the study.

Evaluate the effect of ISIS 681257 following a single subcutaneous dose in subjects with impaired renal function relative to matched, healthy controls with normal renal function.
Day 31

The plasma concentrations of ISIS 681257 will be measured.

Evaluate the effect of multiple doses (2 doses) of ISIS 681257 40 mg subcutaneous injections on the PK of multiple oral doses of clopidogrel in healthy adult subjects
55 days

The plasma concentrations of Clopidogrel and ISIS 681257 will be measured at each individual time point.

The plasma concentrations of warfarin and ISIS 681257 will be measured at each individual time point.
Each day for days 1-10 and 15-45

To evaluate the effect of multiple doses of ISIS 681257 40 mg subcutaneous injections on the pharmacokinetics of a single oral dose of warfarin in healthy adult subjects.

Secondary Endpoints

Percent Change From Baseline in Fasting Low-Density Lipoprotein Cholesterol (LDL-C)
Baseline and Month 6 (Week 25 for Cohorts A, B and C and Week 27 for Cohorts D and E)
Percentage of Participants Who Achieved Plasma Lp(a) ≤ 125 Nanomoles Per Liter (Nmol/L) or ≤ 50 Milligrams Per Deciliter (mg/dL)
Baseline and Month 6 (Week 25 for Cohorts A, B and C and Week 27 for Cohorts D and E)
Percentage of Participants Who Achieved Plasma Lp(a) ≤ 75 Nmol/L or ≤ 30 mg/dL
Baseline and Month 6 (Week 25 for Cohorts A, B and C and Week 27 for Cohorts D and E)
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Study Design & Arms

AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Cohort A: ISIS 681257: 20 mg Q4WEXPERIMENTALCohort A participants received 20 milligrams (mg) ISIS 681257, subcutaneous (SC) injection, once every 4 weeks (Q4W), for up to 49 weeks and a maximum of 13 doses.
Cohort B: ISIS 681257: 40 mg Q4WEXPERIMENTALCohort B participants received 40 mg of ISIS 681257, SC injection, once Q4W, for up to 49 weeks and a maximum of 13 doses.
Cohort C: ISIS 681257: 60 mg Q4WEXPERIMENTALCohort C participants received 60 mg of ISIS 681257, SC injection, once Q4W, for up to 49 weeks and a maximum of 13 doses.
Cohort D: ISIS 681257: 20 mg Q2WEXPERIMENTALCohort D participants received 20 mg of ISIS 681257, SC injection, once every 2 weeks (Q2W), for up to 51 weeks and a maximum of 26 doses.
Cohort E: ISIS 681257: 20 mg QWEXPERIMENTALCohort E participants received 20 mg of ISIS 681257, SC injection, once weekly (QW), for up to 52 weeks and a maximum of 52 doses.
PlaceboPLACEBO_COMPARATORParticipants in each cohort were randomized to receive placebo at a dose-matched volume of study drug (ISIS 681257).
Normal Renal FunctionEXPERIMENTALSubjects with normal renal function will be matched by age (±10 years), weight (± 20%), and gender to the pooled mean values of subjects with the moderate renal impairment. Subjects will receive 1 dose of ISIS 681257.
Moderate Renal ImpairmentEXPERIMENTALPresence of moderate renal impairment (eGFR 30-59 mL/min/1.73m2). Subjects will receive 1 dose of ISIS 681257.
Clopidogrel DosingEXPERIMENTALMultiple doses of Clopidogrel to obtain pharmacokinetic information.
Clopidogrel in combination with ISIS 681257EXPERIMENTALMultiple doses of clopidogrel administered with 2 doses of ISIS 681257 at 2 individual timepoints to obtain pharmacokinetic information.
Single dose of warfarinEXPERIMENTALSingle dose of warfarin administered to obtain pharmacokinetic information.
Warfarin in combination with ISIS 681257EXPERIMENTALISIS 681257 administered and pharmacokinetic assessments are taken. Then ISIS 681257 is administered with warfarin and additional pharmacokinetic information is obtained.

Interventions

NameTypeDescription
ISIS 681257DRUGISIS 681257 solution for SC injection.
PlaceboDRUGSterile normal saline (0.9% NaCl)
ClopidogrelDRUG75mg tablet administered orally
WarfarinDRUG25mg tablet administered orally
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Eligibility Criteria

Age Range18 Years to 80 Years
SexALL
Healthy VolunteersNo
Study Sites32

Key Inclusion Criteria: * Clinical diagnosis of CVD defined as documented coronary artery disease, stroke, or peripheral artery disease * Lp(a) plasma level ≥ 60 mg/dL * Must be on standard-of-care preventative therapy for other than elevated Lp(a) CVD risk factors Key Exclusion Criteria: * Withi...

Countries:United StatesCanadaDenmarkGermanyNetherlands
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Frequently asked questions about ISIS 681257

What is ISIS 681257 used for?

ISIS 681257 is an investigational drug being studied for elevated lipoprotein(a) and cardiovascular disease, as well as for renal impairment. It is in Phase 2 clinical development for these conditions, with the primary study focusing on participants with hyperlipoproteinemia(a) and cardiovascular disease.

Who makes ISIS 681257?

ISIS 681257 is being developed by Ionis Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol IONS. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with elevated lipoprotein(a) and related cardiovascular conditions.

What phase is ISIS 681257 in?

ISIS 681257 is in Phase 2 clinical development. The most advanced trial is a Phase 2 study in participants with hyperlipoproteinemia(a) and cardiovascular disease, which has been completed. Earlier Phase 1 studies have also been completed to evaluate the drug's effects and interactions.

What clinical trials is ISIS 681257 in?

ISIS 681257 has completed three clinical trials. The main Phase 2 study (NCT03070782) enrolled 286 participants with elevated lipoprotein(a) and cardiovascular disease. Two Phase 1 drug-drug interaction studies evaluated effects on clopidogrel (NCT03392051) and warfarin (NCT03426033), plus a renal impairment study (NCT03506854).

Is ISIS 681257 the same as AKCEA-APO(a)-LRx?

Yes, ISIS 681257 is also known as AKCEA-APO(a)-LRx. The Phase 2 clinical trial NCT03070782 is titled 'Phase 2 Study of ISIS 681257 (AKCEA-APO(a)-LRx) in Participants With Hyperlipoproteinemia(a) and Cardiovascular Disease,' confirming that both names refer to the same investigational drug.

Is ISIS 681257 FDA approved?

ISIS 681257 is not FDA approved. It is an investigational drug currently in clinical development, with completed Phase 1 and Phase 2 trials. The drug has not yet received regulatory approval for any indication, and its safety and efficacy continue to be evaluated in clinical studies.