Biotechnology company · PTCT

PTC Therapeutics
clinical trial
pipeline

Registry checked

Published

Neurology is the largest mapped primary therapeutic group, with 2 of 5 active trials. This ranking covers mapped groups only. Vatiquinone is linked to 2 distinct active studies, with 2 Phase 3. Sepiapterin is linked to 1 distinct active study, with 1 Phase 3.

Phase 2 study NCT07779616 in Rheumatoid Arthritis lists estimated primary completion on 30 September 2027. These are registry estimates of primary completion, not confirmed results announcements or overall study completion.

Active trial mix

Lead-sponsor scope

5active trials
as lead sponsor
  • Phase 10
  • Phase 22
  • Phase 33
  • Phase 40
  • Phase not recorded0

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

41 trials30 completed days
Recruitment statusPrimary completionEnrolmentFirst observedOther

Highlighted changes 02

Select an example to see what changed

01Recruitment status
CompletedTerminated

Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)

NCT01247207 · ClinicalTrials.gov
Recorded by ComplyRx
14 Oct 2026
Registry last updated
08 Oct 2026

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
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2026-10-100000000
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2026-10-141010241
2026-10-150000000
2026-10-160000000
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2026-10-190000000
2026-10-200000000
2026-10-210000000
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2026-10-300000000
2026-10-310000000
Type totals1010241 distinct

02 / Drugs & programmes

Explore the pipeline

3 selected programmes

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

Vatiquinone

Canonical investigational asset

2
Active trials
0
Recruiting trials
Trial phases
Highest: Phase 3
  • Phase 10
  • Phase 20
  • Phase 32
  • Phase 40
  • Phase not recorded0
Studied condition
Friedreich Ataxia · Friedreich's Ataxia
Next primary completion
Not reported
Open drug

Sepiapterin

sephience

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

Eladocagene Exuparvovec

kebilidi

1
Active trials
0
Recruiting trials
Trial phases
Highest: Phase 2
  • Phase 10
  • Phase 21
  • Phase 30
  • Phase 40
  • Phase not recorded0
Studied condition
AADC Deficiency
Next primary completion
Not reported
Open drug

4 of 5 active trials have accepted investigational programme links. Trial context does not establish ownership or complete drug coverage.

3 selected programmes · accepted investigational links cover 4 of 5 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

PTC Therapeutics trials in progress

Registry start → study completion

◇ Recorded observation│ Primary completion milestoneHatched ends retain month / year uncertaintyHover, focus or tap an observation
Trial / programme
3 selected Phase 3 / 2 trials
2021202220232024202520262027202820292030
Study of PTC844 in Participants With Active Rheumatoid Arthritis
Asset mapping not recorded

NCT07779616

Phase 2 · Not yet recruiting

Trial source and qualifications

Study of PTC844 in Participants With Active Rheumatoid Arthritis

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.

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

ClinicalTrials.gov source

No eligible observations recorded in the inspected period.

    Start: 31 Aug 2026 · estimated
    Primary completion: 30 Sept 2027 · estimated
    Study completion: 29 Oct 2027 · estimated

    2021202220232024202520262027202820292030
    A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
    Vatiquinone

    NCT05515536

    Phase 3 · Active, not recruiting

    Trial source and qualifications

    A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia

    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: 08 Dec 2022 · actual
      Primary completion: 31 Dec 2027 · estimated
      Study completion: 31 Dec 2027 · estimated

      2021202220232024202520262027202820292030
      PROVE-FA
      Vatiquinone

      NCT07681713

      Phase 3 · Not yet recruiting

      Trial source and qualifications

      Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)

      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.

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

      ClinicalTrials.gov source

      No eligible observations recorded in the inspected period.

        Start: 15 Aug 2026 · estimated
        Primary completion: 15 Mar 2029 · estimated
        Study completion: 15 Mar 2029 · 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.

        3 selected of 3 eligible trials · 0 without supported dates · 0 omitted for contradictory chronology. Excluded: 1 actual primary completions, 0 untyped primary completions, 0 past estimates, 1 outside the horizon, 0 contradictory dates.

        04 / Therapeutic focus

        Where the activity is

        2

        active trials in Neurology

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

        Active trials by primary therapeutic area

        • Neurology2 trials
        • Immunology & Inflammation1 trials
        • Metabolic/Endocrine/Nutrition1 trials
        • Unmapped1 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

        No supported overlap was identified in the selected programme context. 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 65 distinct NCT records attributed through the canonical sponsor map. Included canonical entities: PTC Therapeutics. Phrases retrieve candidates only, then verbatim lead-sponsor attribution determines inclusion.

        ClinicalTrials.gov

        Attribution coverage

        65 distinct registry candidates produced 65 accepted studies. 0 mapped to other owners, 0 lacked a canonical mapping and 0 fell outside an operating subset. Accepted mappings include 65 unverified, 0 identity-only and 0 device-tagged trial memberships. Mapping flags describe evidence quality, not a separate ownership authority.

        Indexed enrichment

        63 scoped records have indexed enrichment and 2 do not. Missing enrichment does not remove a trial from cohort counts.

        Scope observation

        The successful attribution check completed at 2026-11-01T03:18:11.169Z. 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 d68968bf1ac530f4801609c9991553d977b858590a23c1d2ec2591626e8f1f08.

        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: 65 mapped lead-sponsored clinical studies across all statuses. 63 have indexed enrichment, 2 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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