Clinical trial updates

IMR-687

IMR-687

Imara (IMR-687)   Update: 25 August 2020 Imara recently initiated a Phase 2b clinical trial of IMR-687 in adult patients with beta-thalassaemia and expects to dose the first patient in…
Rapamycin

Rapamycin

Update: 25 August 2020 No update available.   Update: 31 May 2020 No update available.   Update: 31 March 2020 No update available.   Update: 31 January 2020 No update…
Mitapivat (AG-348)

Mitapivat (AG-348)

Mitapivat (AG-348)   Update: 25 August 2020 FDA Orphan Drug Designation Granted to Mitapivat for Treatment of Thalassemia The ongoing, open-label Phase 2 study is evaluating the efficacy, safety, pharmacokinetics…
Sickle Cell Disease

Sickle Cell Disease

Mitapivat   Update: 25 August 2020 The ongoing Phase 1 study, which can enroll up to 25 patients, is evaluating the efficacy, safety, pharmacokinetics and pharmacodynamics of treatment with mitapivat…
Apotransferrin

Apotransferrin

Update: 25 August 2020 No update available.   Update: 31 May 2020 No update available.   Update: 31 March 2020 No update available.   Update: 31 January 2020 No update…
Ferroportin inhibitors

Ferroportin inhibitors

VIT-2763   Update: 25 August 2020 Data presented at the EHA Congress in June indicate that treatment with the oral ferroportin inhibitor VIT-2763 alone or in combination with DFX significantly…
SLN-124

SLN-124

SLN-124   Update: 25 August 2020 No update available   Update: 31 May 2020 Enrolment paused due to COVID-19 pandemic. Interim results expected to be published in early 2021. FDA…
Denosumab

Denosumab

Update: 25 August 2020 No update available.   Update: 31 May 2020 No update available.   Update: 31 March 2020 No update available.   Update: 31 January 2020 No update…
Reblozyl

Reblozyl

  ‘BELIEVE’ Study in Adults with TDT β-thalassaemia   Update: 25 August 2020 Long term safety and efficacy results from the phase III BELIEVE trial were presented at the EHA25…
Gene Therapy

Gene Therapy

ZYNTEGLO (Lentiglobin) for patients with TDT  Update: 25 August 2020 New data from ongoing Phase 3 studies show pediatric, adolescent and adult patients with a range of genotypes of transfusion-dependent…
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