Drugs / AAV5hFIX
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT02396342 | Jun 2015 → Apr 2021 | hemophilia B | CSL Behring | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 17
| Date | Issuer | Release |
|---|---|---|
| 2022-12-10 | CSL Behring | Results CSL's Novel Gene Therapy HEMGENIX® (etranacogene dezaparvovec-drlb) Demonstrates Durable Protection and Sustained Factor IX Activity Levels for People Living with Hemophilia B at 24-Months Post-Treatment csl.com ↗ |
| 2020-12-07 | UniQure Biopharma B.V. | Results uniQure Announces Two-Year Follow-Up Data from the Phase IIb Study of Etranacogene Dezaparvovec and Long-Term Follow-Up Data for AMT-060 in Patients with Hemophilia B gcs-web.com ↗ |
| 2019-12-08 | UniQure Biopharma B.V. | Results uniQure Announces One-Year Follow-Up Data from the Phase IIb Study of Etranacogene Dezaparvovec and Long-Term Follow-Up Data for AMT-060 in Patients with Hemophilia B gcs-web.com ↗ |
| 2019-07-06 | UniQure Biopharma B.V. | Results uniQure Announces 36 Weeks of Follow-Up Data from Phase IIb Study of AMT-061 and Long-Term Follow-Up Data for AMT-060 in Patients with Hemophilia B gcs-web.com ↗
Clinical Benefit and Tolerability of AMT-060 Maintained in All Patients Through up to 3.5 Years of Follow-up |
| 2018-12-03 | UniQure Biopharma B.V. | Results uniQure Announces Long-Term Clinical Data from Ongoing Phase I/II Trial of AMT-060 and Confirms Dose for AMT-061 Pivotal Study in Hemophilia B gcs-web.com ↗
All 10 patients sustained increases in FIX activity and improvements in their disease state as measured by reduced usage of FIX replacement therapy and decreased bleeding frequency. |
| 2018-05-21 | UniQure Biopharma B.V. | Results uniQure Presents New Data Demonstrating Clinical Benefit in Hemophilia B Patients with Pre-Existing Anti-AAV5 Neutralizing Antibodies gcs-web.com ↗
In a study re-analyzing pre-treatment sera samples of the ten patients in the Phase I/II clinical trial of AMT-060, no relationship was detected between the presence of pre-treatment anti-AAV5 NABs and clinical outcomes of AMT-060 in patients with hemophilia B. |
| 2017-12-18 | UniQure Biopharma B.V. | Results uniQure Announces Publication in the Journal Blood of Clinical Data from Phase I/II Trial of AMT-060 In Patients with Severe Hemophilia B gcs-web.com ↗
The published manuscript, entitled "Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B," shows that AAV5 liver-directed wildtype hFIX gene transfer was well tolerated and clinically effective in patients with severe and moderate-severe hemophilia B. |
| 2017-12-11 | UniQure Biopharma B.V. | Results uniQure Announces Updated, Long-Term Clinical Data from Ongoing Phase I/II Trial of AMT-060 In Patients with Severe Hemophilia B gcs-web.com ↗
Eighteen-month follow-up data from the second-dose cohort continue to show stable FIX activity with substantial improvement in disease state in all five patients, including the discontinuation of routine prophylactic FIX infusions in all patients that previously required chronic replacement therapy. |
| 2017-11-01 | UniQure Biopharma B.V. | Results uniQure Announces Presentations at the 59th American Society of Hematology (ASH) Annual Meeting gcs-web.com ↗
At equal doses, circulating vector DNA plasma levels, liver distribution, liver cell transduction and hFIX protein expression were comparable for both AMT-060 and AMT-061. |
| 2017-07-11 | UniQure Biopharma B.V. | Results uniQure Presents New Clinical Data in Hemophilia B Patients Demonstrating Therapeutic Efficacy of AAV5 Gene Therapy in the Presence of Pre-Existing Neutralizing Antibodies gcs-web.com ↗
Clinically meaningful factor IX (FIX) activity levels from the ongoing Phase I-II trial of AMT-060 were observed at NAB titers up to 1:341, determined as corresponding up to the 90 th percentile of a healthy control population. |
| 2017-07-10 | UniQure Biopharma B.V. | Results uniQure Announces Updated, Long-Term Clinical Data from Ongoing Phase I/II Trial of AMT-060 In Patients with Severe Hemophilia B gcs-web.com ↗
One-year follow-up data from the second-dose cohort continue to show a dose response with substantial improvement in disease state in all five patients, including the discontinuation of routine prophylactic FIX infusions in all patients that previously required chronic replacement therapy. |
| 2017-04-25 | UniQure Biopharma B.V. | Regulatory uniQure Receives European Medicines Agency Priority Medicines (PRIME) Designation for AMT-060 in Hemophilia B gcs-web.com ↗
LEXINGTON, Mass. and AMSTERDAM, the Netherlands, April 25, 2017 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that AMT-060, its proprietary, investigational gene therapy in patients with severe hemophilia B, has received PRIME designation by the European Medicines Agency (EMA). |
| 2017-01-30 | UniQure Biopharma B.V. | Regulatory uniQure Announces FDA Breakthrough Therapy Designation for AMT-060 in Hemophilia B gcs-web.com ↗
LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Jan. 30, 2017 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leader in human gene therapy, today announced that AMT-060, its proprietary, investigational gene therapy in patients with severe hemophilia B, has received Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA). |
| 2016-12-03 | UniQure Biopharma B.V. | Results uniQure Announces First Clinical Data From Second Dose Cohort of AMT-060 in Ongoing Phase I/II trial in Patients with Severe Hemophilia B gcs-web.com ↗
uniQure N.V. (Nasdaq:QURE), a leader in human gene therapy, today announced new and updated results from its ongoing, dose-ranging Phase I/II trial of AMT-060, its proprietary, investigational gene therapy in patients with severe hemophilia B. |
| 2016-07-27 | UniQure Biopharma B.V. | Results uniQure Presents Updated Clinical Data in Patients with Severe Hemophilia B Demonstrating up to 9 Months of Sustained Levels of gcs-web.com ↗
The updated data show that all patients in the low-dose cohort have sustained improvements in their disease phenotype and continue to maintain durable levels of Factor IX (FIX) gene activity for up to 39 weeks post treatment. |
| 2016-06-11 | UniQure Biopharma B.V. | Results uniQure Presents Clinical Data from Ongoing Phase I/II Study in Hemophilia B Demonstrating 6 Months of Sustained Increases in Fa gcs-web.com ↗
All patients in the low-dose cohort saw improvements in their disease phenotype and achieved sustained increases in FIX activity, with a median of 5.4% (expressed as % of normal) at 6 months post treatment. |
| 2016-01-07 | UniQure Biopharma B.V. | Results uniQure Announces Preliminary Topline Results from Low-Dose Cohort in Hemophilia B Phase I/II Gene Therapy Clinical Trial gcs-web.com ↗
uniQure N.V. (Nasdaq:QURE), a leader in human gene therapy, today announced preliminary topline results from the low-dose cohort of an ongoing Phase I/II clinical trial being conducted in adult hemophilia B patients treated with uniQure's novel AAV5/FIX gene therapy, AMT-060. |
All press releases naming this drug 19 releases
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | AAV5hFIX | “AAV5hFIX gene therapy” NCT02396342 ↗ |
| Known as | AMT-060 | “AMT-060 combines an adeno-associated virus-5 (AAV5) vector with a liver-specific promoter driving expression of a codon-optimized wild-type human FIX gene.” PMID 29246900 ↗ Dec 2017 |
| Modality | Gene therapy (AAV / viral vector) | “Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B.” PMID 29246900 ↗ Dec 2017 |
| Route | Intravenous | “AAV5-hFIX 5 × 10E12 gc/kg intravenous single infusion” NCT02396342 ↗ |