Drugs / GTX-102
Regulatory milestones approvals, filings & regulatory actions · 1 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Clinical hold | US (FDA) | — | Angelman syndrome due to a point mutation | — | “Dosing was paused after the first SAE onset was observed and the study is on clinical hold.” ultragenyx.com ↗ |
Trials 5
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT05531890 | Sep → Nov 2022 | ataxia telangiectasia | Grace Therapeutics Inc. | Unknown | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT04259281 | Feb 2020 → Jan 2025 | Angelman syndrome | Ultragenyx Pharmaceutical Inc | Completed | No outcome recorded |
| Phase 21 trial | ||||||
| Phase 2 | NCT07157254 | Oct 2025 → Jan 2030 expected | Angelman syndrome | Ultragenyx Pharmaceutical Inc | Recruiting | No outcome recorded |
| Phase 32 trials · 1 missed | ||||||
| Phase 3 | NCT06617429 | Dec 2024 → Jul 2026 overdue | Angelman syndrome | Ultragenyx Pharmaceutical Inc | Active not recruiting | Missed primary |
| Phase 3 | NCT06415344 | Jul 2024 → Feb 2029 expected | Angelman syndrome | Ultragenyx Pharmaceutical Inc | Enrolling by invitation | No outcome recorded |
News releases announcing trial results or a regulatory action · 18
| Date | Issuer | Release |
|---|---|---|
| 2026-09-02 | Ultragenyx Pharmaceutical Inc | Results Ultragenyx Announces Phase 3 Aspire results in Angelman Syndrome ultragenyx.com ↗
The study did not achieve the primary endpoint of change from Baseline in Bayley-4 cognitive raw score nor the key secondary endpoint of net response in Multidomain Responder Index (MDRI). |
| 2025-08-05 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Reports Second Quarter 2025 Financial Results and Corporate Update ultragenyx.com ↗
GTX-102 for Angelman syndrome received Breakthrough Therapy Designation from FDA; |
| 2025-06-27 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Receives Breakthrough Therapy Designation for GTX-102 in Angelman Syndrome ultragenyx.com ↗
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that it has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for GTX-102 (apazunersen) as a treatment for Angelman syndrome. |
| 2024-11-09 | Ultragenyx Pharmaceutical Inc | Results Ultragenyx Presents Positive Update on GTX-102 Angelman Syndrome Program at FAST’s 17th Annual Global Science Summit ultragenyx.com ↗
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced Phase 1/2 data in support of the Phase 3 Aspire study for GTX-102, its investigational antisense oligonucleotide for Angelman syndrome, that will be presented at the 2024 Foundation for Angelman Syndrome Therapeutics (FAST) Global Science Summit in Orlando, Florida. |
| 2024-04-15 | Ultragenyx Pharmaceutical Inc | Results Ultragenyx Announces Positive Interim Phase 1/2 Data in Patients with Angelman Syndrome After Treatment with GTX-102 ultragenyx.com ↗
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced new data from the Phase 1/2 study of GTX-102 for the treatment of Angelman syndrome. |
| 2024-02-05 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Receives PRIME Designation from European Medicines Agency (EMA) for GTX-102 for the Treatment of Angelman Syndrome ultragenyx.com ↗ |
| 2023-10-16 | Ultragenyx Pharmaceutical Inc | Results Ultragenyx Announces Program and Pipeline Updates at Analyst Day Including Interim Data from Ongoing Studies in Osteogenesis Imperfecta (OI), Angelman Syndrome (AS) and Wilson Disease ultragenyx.com ↗
Quantitative data from the Phase 1/2 study of GTX-102 for AS show clinically meaningful improvements in multiple domains as compared to natural history |
| 2023-05-17 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Receives FDA Agreement to Expand Ongoing Global Phase 1/2 Trial Evaluating GTX-102 in Patients with Angelman Syndrome in the U.S. ultragenyx.com ↗
Agreement on the protocol amendment enables comparable dose ranges across all geographies and allows us to move forward rapidly to complete the study. |
| 2022-07-18 | Ultragenyx Pharmaceutical Inc | Results Ultragenyx and GeneTx Provide Program Update on GTX-102 for Angelman Syndrome Including Promising Interim Data from Phase 1/2 Study ultragenyx.com ↗
Interim results on 9 patients from the U.K./Canada arm and 2 patients from the U.S. arm of the Phase 1/2 study demonstrate a meaningful improvement in clinical disease and an acceptable safety profile. |
| 2022-01-05 | Ultragenyx Pharmaceutical Inc | Results GeneTx and Ultragenyx Provide Preliminary Update on Phase 1/2 Clinical Study of GTX-102 in Canada and U.K. Patients with Angelman Syndrome ultragenyx.com ↗
There have been no treatment-related serious adverse events of any type nor adverse events related to lower extremity weakness observed in these patients, and initial assessments have shown early signs of clinical activity. |
| 2021-09-27 | Ultragenyx Pharmaceutical Inc | Regulatory GeneTx and Ultragenyx Announce FDA has Removed Clinical Hold on Phase 1/2 Clinical Study of GTX-102 for the Treatment of Angelman Syndrome in the U.S. ultragenyx.com ↗
the U.S. Food and Drug Administration (FDA) has removed the clinical hold and GeneTx may begin dosing naïve patients in the Phase 1/2 study of GTX-102 in pediatric patients with Angelman syndrome. |
| 2021-06-10 | Ultragenyx Pharmaceutical Inc | Regulatory GeneTx and Ultragenyx Receive Approval from U.K. Regulatory Agency to Begin Clinical Study of GTX-102 for the Treatment of Angelman Syndrome ultragenyx.com ↗ |
| 2021-05-19 | Ultragenyx Pharmaceutical Inc | Regulatory GeneTx and Ultragenyx Receive Clearance from Health Canada to Begin Clinical Study of GTX-102 in Canada for the Treatment of Angelman Syndrome ultragenyx.com ↗ |
| 2020-12-05 | Ultragenyx Pharmaceutical Inc | Results GeneTx and Ultragenyx Announce Presentation of Phase 1/2 Data on Investigational GTX-102 in Patients with Angelman Syndrome ultragenyx.com ↗ |
| 2020-10-26 | Ultragenyx Pharmaceutical Inc | Results GeneTx and Ultragenyx Announce Positive Interim Phase 1/2 Data on Investigational GTX-102 Demonstrating Improvement in Patients with Angelman Syndrome ultragenyx.com ↗
GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), companies partnered in the development of intrathecally administered GTX-102, an investigational treatment for Angelman syndrome, today announced positive interim data from the Phase 1/2 study of GTX-102. |
| 2020-05-04 | Ultragenyx Pharmaceutical Inc | Regulatory GeneTx and Ultragenyx Announce GTX-102 Granted Fast Track Designation by U.S. FDA for Treatment of Angelman Syndrome ultragenyx.com ↗ |
| 2020-01-15 | Ultragenyx Pharmaceutical Inc | Regulatory GeneTx and Ultragenyx Announce Investigational New Drug (IND) Application Active for GTX-102 in Patients with Angelman Syndrome ultragenyx.com ↗
GeneTx’s Investigational New Drug (IND) Application for GTX-102, an experimental antisense oligonucleotide being evaluated for the treatment of Angelman syndrome (AS), filed with the U.S. Food and Drug Administration ( FDA ) is now active. |
| 2019-09-03 | Ultragenyx Pharmaceutical Inc | Regulatory GeneTx and Ultragenyx Announce Orphan Drug Designation and Rare Pediatric Disease Designation for GTX-102 ultragenyx.com ↗ |
All press releases naming this drug 27 releases
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 6 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | GTX-102 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06415344 ↗ |
| Known as | Betamethasone | “GTX-102 Betamethasone oral spray medium dose (0.05 mg/kg) administered in Period 1 and Period 2 fast” NCT05531890 ↗ |
| Action | Inhibit | “ASOs targeting this region precisely and efficiently repress the transcription of UBE3A-AS” PMID 36947593 ↗ Mar 2023 |
| Modality | Antisense oligonucleotide | “An ASO therapy for Angelman syndrome that targets an evolutionarily conserved region at the start of the UBE3A-AS transcript.” PMID 36947593 ↗ Mar 2023 |
| Route | Intrathecal | “GTX-102 administered by intrathecal (IT) injection” NCT04259281 ↗ |