Drugs / Votoplam
Trials 3
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 22 trials · 1 met primary | ||||||
| Phase 2 | NCT06254482 | Aug 2023 → Jul 2029 expected | Huntington disease | Novartis Pharmaceuticals | Active not recruiting | No outcome recorded |
| Phase 2 | NCT05358717 | Jun 2022 → Feb 2025 | Huntington disease | Novartis Pharmaceuticals | Completed | Met primary |
| Phase 31 trial | ||||||
| Phase 3 | NCT07326709 | Mar 2026 → Apr 2030 expected | Huntington disease | Novartis Pharmaceuticals | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 7
| Date | Issuer | Release |
|---|---|---|
| 2026-04-28 | PTC Therapeutics | Results PTC Therapeutics Reports Positive Topline Results from Month 24 Interim Analysis of PIVOT-HD Extension Study of Votoplam ptcbio.com ↗
In particular, the evidence of dose-dependent slowing of progression on the cUHDRS disease rating scale in the Stage 2 study participants supports the Novartis-initiated Phase 3 INVEST-HD study. |
| 2025-05-05 | PTC Therapeutics | Results PTC518 PIVOT-HD Study Achieves Primary Endpoint ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced results from the Phase 2 PIVOT-HD study of PTC518 (votoplam) in Stage 2 and Stage 3 Huntington's disease (HD) patients. |
| 2024-09-26 | PTC Therapeutics | Regulatory FDA Grants Fast Track Designation to PTC518 Huntington's Disease Program ptcbio.com ↗
PTC Therapeutics (NASDAQ: PTCT) announced today that the FDA has granted Fast Track designation to the PTC518 program for the treatment of Huntington's disease. |
| 2024-06-20 | PTC Therapeutics | Results Interim PIVOT-HD Results Demonstrate Evidence of Favorable CNS Biomarker and Clinical Effects at Month 12 in Huntington's Disease Patients ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) today shared interim results from the Phase 2 PIVOT-HD study of PTC518 in Huntington's disease (HD) patients. |
| 2023-06-21 | PTC Therapeutics | Results PTC Therapeutics Shares Positive Interim Data from PIVOT-HD Clinical Trial in Huntington's Disease Patients ptcbio.com ↗
The study demonstrated dose-dependent lowering of Huntingtin (HTT) protein levels in peripheral blood cells, reaching a mean 30% reduction in mutant HTT levels at the 10mg dose level. |
| 2021-09-20 | PTC Therapeutics | Results PTC Therapeutics to Host Conference Call to Discuss Results of PTC518 Phase 1 Study for Huntington's Disease Program ptcbio.com ↗
The new results include HTT mRNA and protein lowering, as well as PTC518 exposure levels in the cerebrospinal fluid, consistent with the compound penetrating the blood-brain barrier and not being effluxed out of the brain. |
| 2021-05-04 | PTC Therapeutics | Results PTC Therapeutics Provides a Corporate Update and Reports First Quarter 2021 Financial Results ptcbio.com ↗
Preliminary results from the PTC518 Phase 1 healthy volunteer trial demonstrated dose-dependent reduction of Huntington mRNA beyond the 30-50% target. |
All press releases naming this drug 12 releases
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 4 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Votoplam | ClinicalTrials.gov intervention name — accepted as the source's own label NCT07326709 ↗ |
| Known as | PTC518 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05358717 ↗ |
| Modality | Small molecule | — CHEMBL4850295 ↗ |
| Route | Oral | “once daily orally” NCT05358717 ↗ |