Sickle cell disease (SCD) global products/pipeline

Last Update: Aug 3, 2022

Approved therapies

Company Product MoA Indication Approval
Novartis Adakveo (crizanlizumab) Anti-P-selectin mAb Add-on or monotherapy to reduce the frequency of VOCs in SCD patients aged 16 and older. Nov 2019 (US), Oct 2020 (EU), Oct 2021 (UK)
Global Blood Therapeutics Oxbryta (voxelotor) HbS stabiliser/polymerisation inhibitor Treatment of SCD in adults and pediatric patients 4 years of age and older. Ages 12+ in UK. Nov 2019 (US), Feb 2022 (EU) July 2022 (UK)
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 -
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Global pipeline, disease modifying agents & curative therapies

Company Candidate MoA Indication Phase
Global Blood Therapeutics Inclacumab1 Anti P-selectin mAb SCD - reduce frequency of VOCs III
Forma Therapeutics Etavopivat (FT-4202)2 PKR activator SCD - Hb+VOCs II/III
Agios Pharma Mitapivat3 PKR activator SCD, thalessemias II/III
Global Blood Therapeutics GBT6015 Next gen HbS polymerisation inhibitor SCD - disease modifying II/III
EpiDestiny/Novo Nordisk EPI01 DNMT1 inhibitor / increase expression of HbF SCD II
Fulcrum Therapeutics FTX-60588 EED inhibitor / increase expression of HbF SCD Ib
Agios Pharma AG-946 PKR activator SCD, hemolytic anemias I
Potential cures
Bluebird bio LentiGlobin BB305 (bb1111)6 Ex vivo autologous HSC lentiviral therapy delivering functional HBB SCD cure III, I/II
Vertex/CRISPR CTX00110 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 HbA CRISPR-corrected HSCT Severe SCD cure I/II
Discontinued/Failed
Pfizer/Glycomimetics Rivipansel Pan-selectin antagonist SCD - reduce VOCs III
Imara Tovinontrine (IMR-687)4 PDE9 inhibitor SCD IIb
Modus Therapeutics Sevuparin Polysaccharide, anti-inflammatory SCD - reduce VOCs II
Merck Montelukast7 CysLT1 receptor inhibitor SCD II
1. Inclacumab - two global Ph3 trials initiating in 2021.
2. Etavopivat - Ph2/3 trial HIBISCUS initiated Q1 2021 measuring Hb response and VOC rate. First interim analysis by end of 2022.
3. Mitapivat - Approved by FDA on Feb 17 2022 for hemolytic anemia in adults with PK deficiency, Ph2 for Thalassemia. Ph2/3 trial RISE UP NCT05031780 in patients with SCD initiated Jan 2022.
4. IMR-687 - Ph2a results reported Jan 2021. Press release. Discontinued in Apr 2022 after no significant clinical benefit vs placebo in interim analyses of Ph2b studies for SCD and beta-thalassemia.
5. GBT601 - Ph2/3 trial initiated June 29 2022. Press release.
6. All LentiGlobin trials were put on clinical hold on Feb 16 2021. Clinical hold lifted June 7 2021. Ph3 trial for SCD currently recruiting NCT04293185 as of Q322.
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 initiated Q421. Press release. Initial Ph1b data reported June 2022, reporting 2.1-6.3% HbF induction (n=3), no serious TEAEs. Press release. Registrational trial to initiate in 2023.
9. GPH101 - IND cleared 2021.
10. CTX001 - EHA 2021 data presentation from ongoing Ph1/2 trial can be accessed here.

VOCs = Vaso-occlusive crisis. HSC = Hematopoietic stem cell. HBB = Hemoglobin subunit beta gene. ZFN = Zinc finger nuclease. HSCT = Hematopoietic stem cell transplantation.

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