PTC Therapeutics, Inc.

Entry

FDA engagement on votoplam PIVOT-HD 24-month results in Huntington's disease · Company milestone

Drug

votoplam (PTC518)

For

Huntington's disease

Stage

Phase 3 · the last stage before a drug can be approved

When

H2 2026 · the company gave a window, not a day

Source note

Company plans to engage with FDA in 2H 2026 to discuss PIVOT-HD 24-month results; outcome frames a potential accelerated path for the Novartis-partnered program.

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• Source: prnewswire.com ↗ — where this entry was taken from
• SEC filings (8-K) ↗ — press releases are attached to 8-K filings
• ClinicalTrials.gov ↗ — trials of this drug
• PTCT headlines on this site

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Entry

PTC303 (MSH3 splicing) clinical entry · Company milestone

Drug

PTC303

For

somatic expansion disorders (Huntington's disease, myotonic dystrophy)

Stage

Phase 1 · an early safety study in a small group of people

When

2027 · the company gave a window, not a day

Source note

Company plans to enter the clinic in 2027 (year-level guidance only).

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• Source: prnewswire.com ↗ — where this entry was taken from
• SEC filings (8-K) ↗ — press releases are attached to 8-K filings
• ClinicalTrials.gov ↗ — trials of this drug
• PTCT headlines on this site

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Entry

PROVE-FA Phase 3 initiation for vatiquinone (post-CRL path) · Company milestone

Drug

vatiquinone

For

Friedreich ataxia

Stage

Phase 3 · the last stage before a drug can be approved

When

Q3 2026 · the company gave a window, not a day

Source note

PROVE-FA study to support NDA resubmission after the August 2025 CRL is "expected to initiate in Q3 2026" (global, ~120 patients ages 7-21). May have started just before the 8/21 window opens.

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• Source: prnewswire.com ↗ — where this entry was taken from
• SEC filings (8-K) ↗ — press releases are attached to 8-K filings
• ClinicalTrials.gov ↗ — trials of this drug
• PTCT headlines on this site

Plain-English summary generated from this entry’s own fields (drug, condition, stage, date). Glossary · Open this catalyst’s page

Entry

PTC844 (next-gen DHODH inhibitor) Phase 2a study initiation · Company milestone

Drug

PTC844

For

undisclosed (DHODH inhibitor)

Stage

Phase 2a

When

Q3 2026 · the company gave a window, not a day

Source note

Q2 2026 results PR (Jul 30, 2026). PTC612 NLRP3 Phase 1 already initiated in Q2.

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• Source: prnewswire.com ↗ — where this entry was taken from
• SEC filings (8-K) ↗ — press releases are attached to 8-K filings
• ClinicalTrials.gov ↗ — trials of this drug
• PTCT headlines on this site

Plain-English summary generated from this entry’s own fields (drug, condition, stage, date). Glossary · Open this catalyst’s page

Entry

FDA feedback sets new open-label vatiquinone registration study for Q3 2026 · Company milestone

Drug

vatiquinone

For

Friedreich's ataxia

Stage

Regulatory (NDA resubmission path)

When

May 7, 2026

Source note

PTC disclosed the outcome of an April 2026 Type C meeting with FDA on the path to an NDA resubmission for vatiquinone in Friedreich's ataxia: the company will run a new open-label study with a matched natural history control, starting Q3 2026, with mFARS change at 24 months as the primary endpoint.

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• Source: ir.ptcbio.com ↗ — where this entry was taken from
• SEC filings (8-K) ↗ — press releases are attached to 8-K filings
• ClinicalTrials.gov ↗ — trials of this drug
• PTCT headlines on this site

Plain-English summary generated from this entry’s own fields (drug, condition, stage, date). Glossary · Open this catalyst’s page

Entry

Positive Month 24 PIVOT-HD interim: votoplam slowed HD progression 52% · Clinical trial results (readout / topline data)

Drug

votoplam (PTC518)

For

Huntington's disease

Stage

Phase 2 · the stage that tests whether the drug actually works

When

Apr 28, 2026

Source note

PTC reported positive topline results from the 24-month interim analysis of the PIVOT-HD long-term extension, showing dose-dependent benefit on cUHDRS in Stage 2 Huntington's disease patients versus a matched natural history cohort, with 52% slowing at the 10 mg dose. Novartis has initiated the global Phase 3 INVEST-HD study.

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• Source: ir.ptcbio.com ↗ — where this entry was taken from
• SEC filings (8-K) ↗ — press releases are attached to 8-K filings
• ClinicalTrials.gov ↗ — trials of this drug
• PTCT headlines on this site

Plain-English summary generated from this entry’s own fields (drug, condition, stage, date). Glossary · Open this catalyst’s page