Clinical trial updates

Reblozyl

 

‘BELIEVE’ Study in Adults with TDT β-thalassaemia

 

Update: 30 June 2021

  • Health Canada has approved REBLOZYL® (luspatercept for injection) for the treatment of adult patients with transfusion-dependent anemia requiring at least two RBC red blood cell (RBC) units over 8 weeks.

Long term safety and efficacy results on REBLOZYL were presented at EHA2021:

  • Individuals who participated in the phase 3 BELIEVE trial for TDT continue to be monitored. Their follow-up data has been presented, and indicates a sustained reduction of transfusions for over 30 months.
  • Results of the Phase 2 BELIEVE trial in NTDT patients were presented. Data showed that 78% of NTDT patients (N=27) achieved a mean Hb increase of ≥ 1.0g/dl, coupled with decreased mean serum ferritin levels. These results were maintained for up to 4.8 years.
  • TDT patients of the BELIEVE trial who were classified as ‘non-responders’, meaning that their transfusion burden reduction did not reach 33% (the study’s primary endpoint) still demonstrated a clinical benefit. These patients had fewer transfusions, visited the hospital less, and saw a reduction in serum ferritin levels.

Source: https://investor.acceleronpharma.com/news/news-details/2021/Health-Canada-Approves-REBLOZYL-luspatercept-New-Class-of-Treatment-for-Adult-Patients-Living-With-Myelodysplastic-Syndromes/default.aspx

EHA2021 :

  • Long-term efficacy and safety outcomes in the Phase 2 study of luspatercept in beta-thalassemia
  • Fewer red blood cell transfusion units and visits across baseline transfusion burden levels in patients with beta-thalassemia treated with luspatercept in the Phase 3 BELIEVE trial
  • Benefit of continuing therapy with luspatercept in patients with beta-thalassemia who do not achieve ≥33% reduction in red blood cell (RBC) transfusion burden (TB) in weeks 13-24 in the BELIEVE trial

 

Update: 08 January 2021

Long term safety and efficacy results on REBLOZYL were presented at ASH2020 and demonstrated that a higher proportion of luspatercept-treated patients showed decreasing serum ferritin (SF), LIC, and myocardial iron levels during the first 48 wks. Long-term luspatercept treatment led to an increasing proportion of patients with SF < 1,000 μg/L and decreasing iron chelation therapy.

Source: https://ash.confex.com/ash/2020/webprogram/Paper136517.html

 

Update: 05 November 2020

Long term safety and efficacy results on REBLOZYL will be presented at the 62nd ASH Meeting in December 2020 focusing on:

  1. Health-related Quality of Life Outcomes
  2. Effect on Iron Overload and Iron Chelation Therapy
  3. Sustained Reductions of RBC Transfusion Burden

Source: http://investor.acceleronpharma.com/news-releases/news-release-details/acceleron-announces-reblozylr-luspatercept-aamt-virtual

 

Update: 25 August 2020

  • Long term safety and efficacy results from the phase III BELIEVE trial were presented at the EHA25 Congress in June, showing
    1. More than 33% reduction of required transfusions for:
      • 2% of β00 patients
      • 39% of β0+ patients
      • 6% of β++ patients
      • 7% of HbE/β-thalassaemia patients
    2. More than 50% reductions of required transfusion for:
      • 3% of β00 patients
      • 3% of β0+ patients
      • 2% of β++ patients
      • 8% of HbE/β-thalassaemia patients

 The European Commission (EC) has approved Reblozyl (luspatercept) for the treatment of adult patients with transfusion-dependent anaemia associated with beta thalassemia.

Sources: https://news.bms.com/press-release/corporatefinancial-news/european-commission-approves-reblozyl-luspatercept-treatment-t

https://www.ema.europa.eu/en/medicines/human/EPAR/reblozyl

https://library.ehaweb.org/eha/2020/eha25th/295114/maria.domenica.cappellini.assessment.of.response.to.luspatercept.by.-globin.html?f=listing%3D0%2Abrowseby%3D8%2Asortby%3D1%2Asearch%3Ds295

 

Update: 31 May 2020

  • The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has issued a positive opinion, recommending the approval of Reblozyl (luspatercept) for the treatment of adult patients with transfusion-dependent anemia associated with beta thalassemia.
  • This CHMP recommendation will now be reviewed by the European Commission (EC), which has the authority to approve medicines for the European Union (EU). If approved, Reblozyl would be the first erythroid maturation agent approved in the EU, representing a new class of therapy for eligible patients.
  • Long term safety and efficacy results from the phase III BELIEVE trial will be presented at the EHA25 Congress in June.

Sources: https://news.bms.com/press-release/corporatefinancial-news/reblozyl-luspatercept-receives-positive-chmp-opinion-treatment

http://investor.acceleronpharma.com/news-releases/news-release-details/acceleron-announces-presentations-reblozylr-luspatercept-aamt

https://news.bms.com/press-release/corporatefinancial-news/bristol-myers-squibb-research-eha-2020-demonstrates-continued- https://www.ema.europa.eu/en/medicines/human/summaries-opinion/reblozyl

 

‘BEYOND’ Study in Adults with NTDT β-thalassaemia

 

Update: 30 June 2021

Data from the BEYOND trial were presented at EHA2021.  BEYOND is a Phase 2, randomized, double-blind, placebo-controlled multi-center study to determine the efficacy and safety of Reblozyl in non-transfusion dependent (NTD) beta-thalassemia.

  • 145 eligible patients over 18 years of age with beta-thalassemia or hemoglobin (Hb) E beta-thalassemia and who received ≤5 red blood cell (RBC) units in the 24 weeks prior to the trial with mean baseline Hb ≤10.0 g/dL participated.
  • The primary endpoint was an achievement of ≥1.0 g/dL mean Hb increase from baseline over a continuous 12-week interval, sustained without RBC transfusions.
  • 1% of participants achieved an increase in haemoglobin compared to 0% patients on placebo. Of these, 72.7% of patients had a mean baseline Hb of <8.5 g/dL.
  • 6% of patients treated with Reblozyl remained transfusion-free for at least 24 weeks vs. 67.3% of patients in the placebo arm.

Source: EHA2021: Presidential Symposium:  The BEYOND Study: Results of a Phase 2, double-blind, randomized, placebo-controlled multi-center study of luspatercept in adult patients with non-transfusion dependent beta-thalassemia

https://news.bms.com/news/corporate-financial/2021/Bristol-Myers-Squibb-and-Acceleron-Present-First-Results-from-Phase-2-BEYOND-Study-of-Reblozylluspatercept-aamt-in-Adults-with-Non-Transfusion-DependentNTDBeta-Thalassemia/default.aspx

 

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