Sickle cell disease (SCD) global products/pipeline

Last Update: Aug 18, 2021

Approved therapies

Company Product MoA Indication Approval
Novartis Adakveo (crizanlizumab) Anti-P-selectin mAb SCD add-on or monotherapy for prevention of VOCs ages 16+ Oct 2019 (EU), Nov 2019 (US)
Global Blood Therapeutics Oxbryta (voxelotor) HbS stabiliser/polymerisation inhibitor SCD ages 12+ (Accelerated approval) Nov 2019 (US)
Emmaus Life Sciences Endari (L-glutamine) Antioxidant/unclear Reduce acute complications of SCD July 2017 (US)
Multiple Hydroxyurea/ Hydroxycarbamide Increase HbF production, immunomodulation Reduce painful crises and blood transfusions in pts w/ SCA w/recurrent mod-severe painful crises 1998 (US)
- Allogeneic HSCT Replace beta globin-defective stem cells Severe SCD - eligible patients -

Global pipeline, disease modifying agents & curative therapies

Company Candidate MoA Indication Phase
Global Blood Therapeutics Inclacumab Anti P-selectin mAb SCD - reduce frequency of VOCs III1
Agios Pharma Mitapivat PKR activator PK deficiency, SCD, thalessemias II/III2
Forma Therapeutics FT-4202 PKR activator SCD - Hb+VOCs II/III3
Imara IMR-687 PDE9 inhibitor SCD IIb4
EpiDestiny/Novo Nordisk EPI01 DNMT1 inhibitor / increase expression of HbF SCD II
Agios Pharma AG-946 PKR activator SCD, hemolytic anemias I
Global Blood Therapeutics GBT6015 Next gen HbS polymerisation inhibitor SCD I
Fulcrum Therapeutics FTX-60588 EED inhibitor / increase expression of HbF SCD I
Potential cures
Bluebird bio LentiGlobin BB3056 Ex vivo autologous HSC lentiviral therapy delivering functional HBB SCD cure III, I/II
Vertex/CRISPR CTX001 Ex vivo autologous CRISPR/Cas9 BCL11A edited HSC Severe hemoglobinopathies (SCD) cure I/II
Sangamo/Bioverativ (Sanofi) BIVV-003 ZFN-modified autologous stem cells w/ disrupted BCL11A gene SCD cure, B-thalessemia I/II
Novartis/Intellia OTQ923 Ex vivo HSC gene therapy to increase HbF expression Severe SCD I/II
Aruvant Sciences RVT-1801 Ex vivo autologous HSC lentiviral therapy delivering gamma-globin gene SCD cure, beta-thalessemia I
Bluebird bio BCH-BB694 shRNA miR lentiviral vector to knockdown BCL11A expression Severe SCD I
CSL/Calimmune CSL200 (CAL-H) Ex vivo HSC gene therapy delivering gamma-globin gene SCD cure, B-thalessemia I
Novartis ADPT03 Ex vivo CRISPR SCD cure I
Graphite Bio GPH1019 Autologous HSCT Severe SCD cure I/II
Discontinued/Failed
Pfizer/Glycomimetics Rivipansel Pan-selectin antagonist SCD - reduce VOCs III
Modus Therapeutics Sevuparin Polysaccharide, anti-inflammatory SCD - reduce VOCs II
Merck Montelukast CysLT1 receptor inhibitor SCD II7
1. Inclacumab - two global Ph3 trials initiating in 2021.
2. Mitapivat - NDA accepted by FDA for PK deficiency (Aug 17 2021) PDUFA: Feb 17 2022, Ph2 for Thalassemia. Plan to initiate III in SCD in 2021
3. FT-4202 - initiating Ph2/3 in Q1 2021 measuring Hb response and VOC rate
4. IMR-687 - Ph2a results reported Jan 2021. Press release.
5. GBT601 - Ph1 in healthy volunteers initiated Dec 2020. Ph1 in SCD patients expected to begin in 2021.
6. All LentiGlobin trials have been on clinical hold since Feb 16 2021.
7. Montelukast - Ph2 results reported in Blood
8. FTX-6058 - reported positive ph1 interim results Aug 10 2021. Mean 4.5-fold induction in HBG mRNA after 14 days once-daily dosing. Ph1b initiating Q421. Press release
9. GPH101 - IND cleared 2021.

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