Global Blood makes second sickle cell bet

Global Blood Therapeutics is upping its stake in sickle cell disease, but believes that there is room on the market for its new project inclacumab as well as its lead candidate, voxelotor. Perhaps inclacumab was too much of a bargain to resist – abandoned by Roche, it cost GBT just $2m up front. However, GBT’s stock sank 3% yesterday, perhaps on fears that the deal was a vote of no confidence for voxelotor. GBT plans to test the anti-P-selectin MAb in vaso-occlusive crises in sickle cell disease patients, making it a slightly different proposition to voxelotor, which the company plans to submit for accelerated approval in the wider sickle cell indication based on part A of the phase III Hope trial. Still, Hope did not find a significant improvement in vaso-occlusive crises, which might be one reason why GBT is hedging its bets. Roche had been studying inclacumab in coronary artery disease, but there is a rationale for evaluating the project in sickle cell disease: Novartis is studying a similarly acting candidate, crizanlizumab, in vaso-occlusive crises. Still, even if GBT benefits from its rival’s groundwork, inclacumab would likely be a distant second to market, especially as the company does not plan to file an IND until 2021. 

The sickle cell landscape
Project Company Mechanism Trial(s) Primary completion
Phase III
Voxelotor Global Blood Therapeutics Sickle haemoglobin polymerisation inhibitor Hope, NCT03036813 Part A data reported Jun 2018
Rivipansel* Pfizer/ Glycomimetics P-selectin inhibitor NCT02433158; NCT02187003 Oct 2018; May 2019
SEG101/ crizanlizumab* Novartis Anti-P-selectin antibody NCT03264989 Sep 2020
Phase II
Sevuparin* Modus Therapeutics/ Ergomed Cell adhesion molecule inhibitor NCT02515838 Oct 2018
CordIn Gamida Cell Hematopoietic cell therapy NCT02504619 Dec 2018
NiCord Gamida Cell Hematopoietic cell replacement NCT01590628 Jan 2019
LentiGlobin Bluebird Bio Beta-globin gene therapy NCT02151526 Feb 2019
IMR-687 Imara PDE9 inhibitor NCT03401112 Jun 2019
IW-1701 Ironwood Pharmaceuticals Soluble guanylate cyclase stimulant Strong-SCD, NCT03285178 Jul 2019
Canakinumab Novartis IL-1B antibody NCT02961218 Jun 2020
*Trials in vaso-occlusive crises only; Source: EvaluatePharma.      

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