Biotechnology company · IONS

Ionis Pharmaceuticals
clinical trial
pipeline

Registry checked

Published

Olezarsen is linked to 4 distinct active studies, with 4 Phase 3. Donidalorsen is linked to 2 distinct active studies, with 2 Phase 3.

Phase 3 study NCT05392114 in Hereditary Angioedema lists estimated primary completion in December 2026. Phase 3 study NCT05681351 in Severe Hypertriglyceridemia lists estimated primary completion in March 2027. These are registry estimates of primary completion, not confirmed results announcements or overall study completion.

Active trial mix

Lead-sponsor scope

22active trials
as lead sponsor
  • Phase 12
  • Phase 25
  • Phase 312
  • Phase 40
  • Phase not recorded3

One highest explicitly recorded phase per active trial. Combined phases use the highest phase. Phase not recorded is shown separately.

Change history

Follow the changes behind the company’s clinical programme.

02 Oct 2026 – 31 Oct 2026 · UTC

Recorded changes

00 trials30 completed days

No eligible observations recorded in this period.

Recruitment statusPrimary completionEnrolmentFirst observedOther

Verified live registry-derived observations in completed UTC days. Recorded dates are observation dates, not necessarily registry amendment dates. Zero means no eligible observations recorded, not daily monitoring of every trial.

0 archive observations and 0 unverified observations excluded.

View exact daily values and type totals
Recorded observations in completed UTC days
Date (UTC)Recruitment statusPrimary completionEnrolmentFirst observedOtherTotalAffected trials
2026-10-020000000
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2026-10-080000000
2026-10-090000000
2026-10-100000000
2026-10-110000000
2026-10-120000000
2026-10-130000000
2026-10-140000000
2026-10-150000000
2026-10-160000000
2026-10-170000000
2026-10-180000000
2026-10-190000000
2026-10-200000000
2026-10-210000000
2026-10-220000000
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2026-10-250000000
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2026-10-300000000
2026-10-310000000
Type totals0000000 distinct

02 / Drugs & programmes

Explore the pipeline

6 selected programmes

Choose a drug to open its record in the tracker. Sign in to continue.

Olezarsen

TRYNGOLZA

4
Active trials
1
Recruiting trials
Trial phases
Highest: Phase 3
  • Phase 10
  • Phase 20
  • Phase 34
  • Phase 40
  • Phase not recorded0
Studied condition
Familial Chylomicronemia Syndrome · Severe Hypertriglyceridemia
Next primary completion
Mar 2027 · estimated
Open drug

Donidalorsen

DAWNZERA

2
Active trials
1
Recruiting trials
Trial phases
Highest: Phase 3
  • Phase 10
  • Phase 20
  • Phase 32
  • Phase 40
  • Phase not recorded0
Studied condition
Hereditary Angioedema · Hereditary Angioedema (HAE)
Next primary completion
Dec 2026 · estimated
Open drug

Eplontersen

wainua

2
Active trials
0
Recruiting trials
Trial phases
Highest: Phase 3
  • Phase 10
  • Phase 20
  • Phase 32
  • Phase 40
  • Phase not recorded0
Studied condition
Hereditary Transthyretin-Mediated Amyloid Polyneuropathy · Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
Next primary completion
Not reported
Open drug

Obudanersen Sodium

Canonical investigational asset

2
Active trials
0
Recruiting trials
Trial phases
Highest: Phase 3
  • Phase 10
  • Phase 21
  • Phase 31
  • Phase 40
  • Phase not recorded0
Studied condition
Angelman Syndrome
Next primary completion
Aug 2027 · estimated
Open drug

Ulefnersen

Canonical investigational asset

1
Active trials
0
Recruiting trials
Trial phases
Highest: Phase 3
  • Phase 10
  • Phase 20
  • Phase 31
  • Phase 40
  • Phase not recorded0
Studied condition
Amyotrophic Lateral Sclerosis
Next primary completion
Not reported
Open drug

zilganersen

Canonical investigational asset

1
Active trials
0
Recruiting trials
Trial phases
Highest: Phase 3
  • Phase 10
  • Phase 20
  • Phase 31
  • Phase 40
  • Phase not recorded0
Studied condition
Alexander Disease
Next primary completion
Not reported
Open drug

18 of 22 active trials have accepted investigational programme links. Trial context does not establish ownership or complete drug coverage.

6 selected programmes · accepted investigational links cover 18 of 22 active trials · brand names checked where available.

Accepted links do not establish complete intervention mapping or ownership. Programme populations overlap.

Up to six canonical assets identified through accepted investigational links in active trials. Counts describe this trial context and do not establish ownership or a complete corporate drug portfolio. Opening a record requires sign-in and does not start tracking.

03 / Clinical trial timeline

Ionis Pharmaceuticals trials in progress

Registry start → study completion

◇ Recorded observation│ Primary completion milestoneHatched ends retain month / year uncertaintyHover, focus or tap an observation
Trial / programme
6 selected Phase 3 / 2 trials
2021202220232024202520262027202820292030
A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)
Donidalorsen

NCT05392114

Phase 3 · Active, not recruiting

Trial source and qualifications

A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)

Duration ends at independently recorded study completion. Registry primary completion, not a confirmed results announcement. Observation markers describe recorded observations, not scheduled completion dates. Up to three newest eligible verified observations from a bounded trailing 24-month candidate window are shown, not full history. 3 inspected observations were not displayed.

3 candidate observations · 0 shown · 0 recorded after primary completion · 3 unverified · 0 invalid dates · 0 before supported start · 0 outside horizon · 0 excluded by display cap · source retrieval not capped.

ClinicalTrials.gov source

3 inspected observations omitted. At most three verified observations are shown, not full history.

    Start: 13 Jul 2022 · actual
    Primary completion: Dec 2026 · estimated
    Study completion: Mar 2027 · estimated

    2021202220232024202520262027202820292030
    CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG)
    Olezarsen

    NCT05681351

    Phase 3 · Active, not recruiting

    Trial source and qualifications

    CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG)

    Duration ends at independently recorded study completion. Registry primary completion, not a confirmed results announcement. Observation markers describe recorded observations, not scheduled completion dates. Up to three newest eligible verified observations from a bounded trailing 24-month candidate window are shown, not full history. 4 inspected observations were not displayed.

    4 candidate observations · 0 shown · 0 recorded after primary completion · 4 unverified · 0 invalid dates · 0 before supported start · 0 outside horizon · 0 excluded by display cap · source retrieval not capped.

    ClinicalTrials.gov source

    4 inspected observations omitted. At most three verified observations are shown, not full history.

      Start: 13 Dec 2022 · actual
      Primary completion: Mar 2027 · estimated
      Study completion: Mar 2027 · estimated

      2021202220232024202520262027202820292030
      PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717
      Erisonersen Sodium

      NCT06153966

      Phase 2 · Active, not recruiting

      Trial source and qualifications

      PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717

      Duration ends at independently recorded study completion. Registry primary completion, not a confirmed results announcement. Observation markers describe recorded observations, not scheduled completion dates. Up to three newest eligible verified observations from a bounded trailing 24-month candidate window are shown, not full history. 6 inspected observations were not displayed.

      9 candidate observations · 3 shown · 0 recorded after primary completion · 6 unverified · 0 invalid dates · 0 before supported start · 0 outside horizon · 0 excluded by display cap · source retrieval not capped.

      ClinicalTrials.gov source

      6 inspected observations omitted. At most three verified observations are shown, not full history.

      • status flip · Recorded 2026-08-20 · Verified live registry-derived observation. Recorded date is not necessarily the registry amendment date. Observations outside the duration bar do not extend the study.
      • enrollment change · Recorded 2026-08-20 · Verified live registry-derived observation. Recorded date is not necessarily the registry amendment date. Observations outside the duration bar do not extend the study.
      • pc date moved · Recorded 2026-09-15 · Verified live registry-derived observation. Recorded date is not necessarily the registry amendment date. Observations outside the duration bar do not extend the study.

      Start: 04 Jan 2024 · actual
      Primary completion: Apr 2027 · estimated
      Study completion: Aug 2030 · estimated

      2021202220232024202520262027202820292030
      A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen
      Olezarsen

      NCT05185843

      Phase 3 · Active, not recruiting

      Trial source and qualifications

      A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen

      Duration ends at independently recorded study completion. Registry primary completion, not a confirmed results announcement. Observation markers describe recorded observations, not scheduled completion dates. Up to three newest eligible verified observations from a bounded trailing 24-month candidate window are shown, not full history. 1 inspected observations were not displayed.

      1 candidate observations · 0 shown · 0 recorded after primary completion · 1 unverified · 0 invalid dates · 0 before supported start · 0 outside horizon · 0 excluded by display cap · source retrieval not capped.

      ClinicalTrials.gov source

      1 inspected observations omitted. At most three verified observations are shown, not full history.

        Start: 25 Feb 2022 · actual
        Primary completion: Jun 2027 · estimated
        Study completion: Jun 2027 · estimated

        2021202220232024202520262027202820292030
        REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome
        Obudanersen Sodium

        NCT06914609

        Phase 3 · Active, not recruiting

        Trial source and qualifications

        REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome

        Duration ends at independently recorded study completion. Registry primary completion, not a confirmed results announcement. Observation markers describe recorded observations, not scheduled completion dates. Up to three newest eligible verified observations from a bounded trailing 24-month candidate window are shown, not full history. 2 inspected observations were not displayed.

        4 candidate observations · 2 shown · 0 recorded after primary completion · 2 unverified · 0 invalid dates · 0 before supported start · 0 outside horizon · 0 excluded by display cap · source retrieval not capped.

        ClinicalTrials.gov source

        2 inspected observations omitted. At most three verified observations are shown, not full history.

        • enrollment change · Recorded 2026-09-30 · Verified live registry-derived observation. Recorded date is not necessarily the registry amendment date. Observations outside the duration bar do not extend the study.
        • status flip · Recorded 2026-09-30 · Verified live registry-derived observation. Recorded date is not necessarily the registry amendment date. Observations outside the duration bar do not extend the study.

        Start: 10 Jun 2025 · actual
        Primary completion: Aug 2027 · estimated
        Study completion: Apr 2030 · estimated

        2021202220232024202520262027202820292030
        ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)
        ION440

        NCT06430385

        Phase 2 · Recruiting

        Trial source and qualifications

        ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)

        Duration ends at independently recorded study completion. Registry primary completion, not a confirmed results announcement. Observation markers describe recorded observations, not scheduled completion dates. Up to three newest eligible verified observations from a bounded trailing 24-month candidate window are shown, not full history. 2 inspected observations were not displayed.

        2 candidate observations · 0 shown · 0 recorded after primary completion · 2 unverified · 0 invalid dates · 0 before supported start · 0 outside horizon · 0 excluded by display cap · source retrieval not capped.

        ClinicalTrials.gov source

        2 inspected observations omitted. At most three verified observations are shown, not full history.

          Start: 21 Oct 2024 · actual
          Primary completion: Sept 2027 · estimated
          Study completion: Apr 2030 · estimated

          2021202220232024202520262027202820292030

          Up to six active Phase 2/3 trials with upcoming estimated primary-completion intervals, ordered by nearest interval, highest phase and NCT. Historical dates never pad this selection. Ten calendar years are shown. Dates retain registry precision and independent date types.

          6 selected of 15 eligible trials · 0 without supported dates · 0 omitted for contradictory chronology. Excluded: 2 actual primary completions, 0 untyped primary completions, 0 past estimates, 0 outside the horizon, 0 contradictory dates.

          04 / Therapeutic focus

          Where the activity is

          4

          active trials in Genetic & Rare Disease

          Primary therapeutic groups are indexed enrichment. Unmapped trials and healthy volunteers remain in the active-trial denominator.

          Active trials by primary therapeutic area

          • Genetic & Rare Disease4 trials
          • Neurology3 trials
          • Immunology & Inflammation2 trials
          • Metabolic/Endocrine/Nutrition2 trials
          • Cardiovascular1 trials
          • Unmapped10 trials
          • Healthy volunteers0 trials

          Each active trial counts once. Full bar equals all active trials in this lead-sponsor scope. This grouping does not describe commercial strategy.

          06 / Related companies

          All companies
          A

          AstraZeneca

          Shared indexed programme context · 1 selected asset

          Based on the selected programmes and indexed lead-sponsor records, not a complete ranking or a competitive/ownership assessment.

          Sources, scope and methodology

          ClinicalTrials.gov lead-sponsor scope

          Canonical current-owner attribution covers 133 distinct NCT records attributed through the canonical sponsor map. Included canonical entities: Ionis Pharmaceuticals, Inc.. Phrases retrieve candidates only, then verbatim lead-sponsor attribution determines inclusion.

          ClinicalTrials.gov

          Attribution coverage

          133 distinct registry candidates produced 133 accepted studies. 0 mapped to other owners, 0 lacked a canonical mapping and 0 fell outside an operating subset. Accepted mappings include 0 unverified, 0 identity-only and 0 device-tagged trial memberships. Mapping flags describe evidence quality, not a separate ownership authority.

          Indexed enrichment

          122 scoped records have indexed enrichment and 11 do not. Missing enrichment does not remove a trial from cohort counts.

          Scope observation

          The successful attribution check completed at 2026-11-01T06:53:44.607Z. This is a dated canonical-attribution snapshot. Static pages normally regenerate after 24 hours, but cached HTML can outlive that interval when regeneration fails. Map changes affect the next successful regeneration. This timestamp is separate from individual registry-update dates. Snapshot 3e38de87afecfc045302dea814a3979b760ed7a16a8b48f6255bdd88090b78a5.

          Active means recruiting, not yet recruiting, active not recruiting or enrolling by invitation. Recruiting counts recruiting only. Trial counts describe lead sponsorship under the published canonical-attribution policy. Identified programmes and therapeutic groups depend on available enrichment. Drug, device and other clinical studies may be included. This is the mapped company scope, not an exhaustive corporate trial census.

          Dated canonical-attribution snapshot: 133 mapped lead-sponsored clinical studies across all statuses. 122 have indexed enrichment, 11 do not. Drug, device and other studies may be represented. Sponsor tags do not classify trial interventions. This is the mapped company scope, not an exhaustive corporate trial census. Recorded changes may reflect observation or archive ingestion rather than a new registry amendment.

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