Update: 30 June 2021
Data presented at EHA2021 from the phase 2a clinical trial for the safety and efficacy of IMR-687 in 93 SCD patients showed that:
- IMR-687 was well-tolerated as a monotherapy and in combination with hydroxyurea
- Lower and less frequent rates of VOC: Patients treated with IMR-687 reported at least 169 days between the onset of treatment and their first VOC (compared to 87 days reported by the placebo group).
Update: 08 January 2021
Two case reports from the ongoing IMR-687 Phase 2a open label extension (OLE) clinical trial in adult patients with sickle cell disease (SCD) were presented at ASH2020:
- Patients were treated for more than 6 months, demonstrating higher HbF percentage and F-cell increases compared to baseline when treated with IMR-687, as a monotherapy or in combination with stable dose hydroxyurea (HU).
- Treatment with IMR-687 was also associated with improvements in clinical outcomes and red cell markers, including hemoglobin (Hb) levels and measures of haemolysis in both patients.
- IMR-687 was well tolerated by both patients.
Update: 05 November 2020
- The European Commission has granted orphan drug designation to IMR-687 for SCD.
- IMR-687 had already received the designations of orphan drug, fast track, and rare pediatric diseasefrom the S. Food and Drug Administration for SCD.
Update: 25 August 2020
Data from the ongoing Phase 2a clinical trial of IMR-687 in adult patients with sickle cell disease (SCD) were presented at the 25th European Hematology Association (EHA) Annual Congress. The data from demonstrated that IMR-687, an oral, once-a-day treatment was safe and well tolerated as a monotherapy and in combination with hydroxyurea (HU). In the higher dose cohort, IMR-687 monotherapy showed an increase in fetal hemoglobin (HbF), as well as a dose-dependent increase in HbF levels in adult patients with SCD.
Ardent IMR-687 Phase 2b Clinical Trial: A global, randomized, double-blind, placebo-controlled, multicenter Ardent Phase 2b clinical trial will enroll approximately 99 adult patients with sickle cell disease (SCD). The planned primary efficacy objective is to evaluate the proportion of all patients with fetal hemoglobin (HbF) response, defined as an increase of 3% in HbF.
- First patient dosed.
Update: 31 May 2020
- Interim data from the ongoing Phase 2a study of IMR-687 in patients with SCD will be presented at EHA25 Congress to be held in June.
- Enrolment in the trial was completed in January 2020.
- Final data will be announced in the fourth quarter of 2020.
Update: 31 March 2020
Based on the interim Phase 2a data, the initiation of two Phase 2b studies in the first half of 2020 are expected. One in in sickle cell disease and the other in beta thalassemia. Preliminary data from both studies are expected in the first half of 2021.