Drugs / sepofarsen
sepofarsen
Trials 4
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/22 trials | ||||||
| Phase 1/2 | NCT03913130 | May 2019 → Oct 2022 | Leber congenital amaurosis 10, blindness (disorder), hereditary neuro-ophthalmological disease | Laboratoires Thea | Terminated | No outcome recorded Stop: Business |
| Phase 1/2 | NCT03140969 | Oct 2017 → Oct 2019 | Leber congenital amaurosis | Laboratoires Thea | Completed | No outcome recorded |
| Phase 2/32 trials · 1 missed | ||||||
| Phase 2/3 | NCT04855045 | Mar 2021 → Dec 2023 | Leber congenital amaurosis 10, blindness (disorder) | ProQR Therapeutics | Unknown | No outcome recorded |
| Phase 2/3 | NCT03913143 | Apr 2019 → Jan 2022 overdue | Leber congenital amaurosis 10, blindness (disorder) | ProQR Therapeutics | Active not recruiting | Missed primary |
| Comparator or background therapy1 trial | ||||||
| Phase 3 | NCT06891443 Comparator | Jun 2025 → Nov 2027 expected | Leber congenital amaurosis 10, blindness (disorder), hereditary neuro-ophthalmological disease | Laboratoires Thea | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 19
| Date | Issuer | Release |
|---|---|---|
| 2022-08-11 | ProQR Therapeutics | Regulatory ProQR to Focus Exclusively on Axiomer RNA-editing Technology and Partner Ophthalmology Programs proqr.com ↗
Following the results from the sepofarsen Illuminate trial, the EMA has recommended an additional clinical trial be conducted for sepofarsen prior to submitting a Marketing Authorisation Application (MAA). |
| 2022-04-13 | ProQR Therapeutics | Results ProQR Announces Additional Sepofarsen Illuminate Trial Analyses and Provides Update on Company Strategy proqr.com ↗
Post-hoc analyses from Illuminate trial of sepofarsen demonstrate an encouraging efficacy signal when comparing active treatment and sham eyes to their corresponding contralateral eyes across multiple endpoints |
| 2022-02-11 | ProQR Therapeutics | Results ProQR Announces Top-Line Results from Phase 2/3 Illuminate Trial of Sepofarsen in CEP290-mediated LCA10 proqr.com ↗
ProQR Therapeutics N.V. (Nasdaq: PRQR) (the “Company”), a company dedicated to changing lives through the creation of transformative RNA therapies for genetic eye diseases, today announced its pivotal Phase 2/3 Illuminate trial of sepofarsen for the treatment of CEP290 -mediated Leber congenital amaurosis 10 (LCA10) did not meet its primary endpoint of Best Corrected Visual Acuity (BCVA) at Month 12. |
| 2021-04-13 | ProQR Therapeutics | Results ProQR Announces Publication in Nature Medicine for Sepofarsen in Leber Congenital Amaurosis 10 proqr.com ↗
Published results highlight a patient who is homozygous for the c.2991+1655A>G mutation in CEP290 and was part of a larger cohort in the Phase 1/2 clinical trial. |
| 2019-10-15 | ProQR Therapeutics | Regulatory ProQR Receives Rare Pediatric Disease Designation from FDA for Sepofarsen for the Treatment of LCA10 proqr.com ↗ |
| 2019-10-11 | ProQR Therapeutics | Results ProQR annonce des premiers résultats positifs pour l'essai de phase 1/2 du sepofarsen chez des patients souffrant d'ACL10 proqr.com ↗
Des améliorations rapides, significatives et durables de la vision à douze mois ont été rapportées |
| 2019-10-10 | ProQR Therapeutics | Results ProQR Announces Positive Top-Line Results from the Phase 1/2 Study of Sepofarsen in LCA10 Patients proqr.com ↗
We reported today that patients receiving sepofarsen had a clinically meaningful improvement in vision, and in some cases the patient’s vision improved to a level that could be deemed life changing. |
| 2019-07-29 | ProQR Therapeutics | Regulatory L’Agence européenne des médicaments accorde l’accès PRIME au Sepofarsen de ProQR pour l’amaurose congénitale de Leber 10 proqr.com ↗ |
| 2019-07-29 | ProQR Therapeutics | Regulatory European Medicines Agency Grants PRIME Access to ProQR’s Sepofarsen for Leber’s Congenital Amaurosis 10 proqr.com ↗ |
| 2019-01-07 | ProQR Therapeutics | Regulatory ProQR Reaches Agreement with the FDA on Design of Phase 2/3 Pivotal Trial for Sepofarsen (QR-110) for Leber’s Congenital Amaurosis 10 proqr.com ↗ |
| 2018-12-18 | ProQR Therapeutics | Results ProQR annonce la publication dans Nature Medicine des données du QR-110 pour l’amaurose congénitale de Leber 10 proqr.com ↗
Les résultats publiés détaillent une analyse intermédiaire planifiée de l’étude clinique PQ-110-001 en cours d’essai pour la première fois chez l’être humain, évaluant QR-110 chez des patients atteints de LCA10. |
| 2018-12-17 | ProQR Therapeutics | Results ProQR Announces Publication in Nature Medicine of QR-110 Data for Leber’s Congenital Amaurosis 10 proqr.com ↗ |
| 2018-09-05 | ProQR Therapeutics | Results ProQR Announces Positive Interim Results from Phase 1/2 Clinical Trial of QR-110 in LCA10 Patients, and Plans to Start a Phase 2/3 Pivotal Trial proqr.com ↗ |
| 2018-09-05 | ProQR Therapeutics | Results ProQR annonce des résultats intermédiaires positifs pour un essai clinique de phase 1/2 du QR-110 chez des patients souffrant de ACL10 et prévoit de commencer un essai pivot de phase 2/3 proqr.com ↗
Dans l’essai, le QR-110 a provoqué une amélioration rapide et soutenue de la vision chez les patients atteints d’ACL10, tant en termes d’acuité visuelle que de mobilité, et a été bien toléré, sans effet indésirable grave particulier. |
| 2018-08-01 | ProQR Therapeutics | Results ProQR Announces Publication on QR-110 for Leber’s Congenital Amaurosis 10 in the Journal Molecular Therapy - Nucleic Acids proqr.com ↗
In additional in vitro testing, QR-110 demonstrated a dose-dependent restoration of mRNA and functional CEP290 protein while in vivo testing showed that QR-110 was well-tolerated following intravitreal injection. |
| 2017-05-31 | ProQR Therapeutics | Regulatory ProQR Receives Fast Track Designation from the FDA for QR-110 for Leber’s Congenital Amaurosis Type 10 proqr.com ↗
ProQR Therapeutics N.V. (Nasdaq:PRQR) today announced that it received Fast Track designation from the U.S. Food and Drug Administration (FDA) for QR-110, the lead molecule in its ophthalmology pipeline. |
| 2017-04-27 | ProQR Therapeutics | Regulatory ProQR Announces Clearance of IND Application to Start Clinical Trial for QR-110 in Leber’s Congenital Amaurosis Type 10 Patients proqr.com ↗
With the clearance of the investigational new drug (IND) application by the U.S. Food and Drug Administration (FDA), ProQR can now start clinical development of QR-110 in Leber’s congenital amaurosis Type 10 (LCA 10) patients |
| 2016-06-01 | ProQR Therapeutics | Regulatory ProQR’s Drug Candidate QR-110 for Leber’s Congenital Amaurosis Type 10 Receives EMA and FDA Orphan Drug Designation proqr.com ↗
Obtaining orphan drug designations from EMA and FDA for QR-110 highlights the high unmet need in for this disease and are important milestones for our program. |
| 2016-04-25 | ProQR Therapeutics | Results ProQR Announces an Investor and Analyst Event and Pre-Clinical Data Presentations for QR-110 for LCA10 at the 2016 ARVO Annual Meeting proqr.com ↗
Following intravitreal injection in vivo , QR-110 is demonstrated to reach the outer nuclear layer of the retina, the target tissue. |
All press releases naming this drug 33 releases
Evidence & citations 7 cited values
Every value below carries the sentence it was read from. 7 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | sepofarsen | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03913143 ↗ |
| Known as | EW9I3PJC3R | ChEMBL registry synonym — accepted as the source's own label CHEMBL4594497 ↗ |
| Known as | QR-110 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03140969 ↗ |
| Known as | QR-110 FREE ACID | ChEMBL registry synonym — accepted as the source's own label CHEMBL4594497 ↗ |
| Action | Restore | “with intravitreal injections of an antisense oligonucleotide to restore correct splicing.” PMID 30559420 ↗ Dec 2018 |
| Modality | Antisense oligonucleotide | “RNA antisense oligonucleotide for intravitreal injection” NCT03140969 ↗ |
| Route | Intravitreal | “administered via intravitreal injection” NCT03140969 ↗ |