Pfizer bets $5.4bn against gene therapies in sickle cell

Hopefully the acquisition of Global Blood Therapeutics will go better than Pfizer’s last foray into sickle cell deal-making.

Deploying a tiny fraction of its Covid bounty, Pfizer has gone from a minor player in sickle cell disease to the market leader. Its $5.4bn acquisition of Global Blood Therapeutics, announced today, enables it to leapfrog companies including Crispr/Vertex and Bluebird Bio in the race to dominate this lucrative niche. 

Or at least it will if the deal gets past antitrust regulators. While Pfizer and Global Blood are by no means the only companies contesting the sickle cell space, the current regulatory climate means that the deal could draw scrutiny from the FTC. 

The $68.50 per share all-cash transaction brings Pfizer Oxbryta, Global Blood’s sickle haemoglobin polymerisation inhibitor, which was approved three years ago and is forecast to sell around $1.5bn in 2028, according to Evaluate Pharma consensus. Pfizer also gets inclacumab, currently in pivotal trials, and a mid-stage asset, GBT021601, which has been touted as a potentially disease-modifying oral option. 

Pfizer, for its part, reckons that Global Blood’s sickle cell franchise could achieve combined worldwide peak annual sales of more than $3bn. If correct, this ought to help Pfizer towards its stated goal of using M&A to add $25bn in revenue by 2030.

Whether the big pharma will continue development of its in-house sickle cell agent, the E-selectin antagonist PF-07209326, is unclear, though the asset is still listed in Pfizer’s latest pipeline. 

Global Blood's sickle cell franchise…
Project Details Status
Oxbryta  Oral sickle haemoglobin polymerisation inhibitor  Approved in the US in 2019 for patients over 4; also approved in EU, United Arab Emirates, Oman & UK; in Hope-Kids-1-2 trials in younger patients 
Inclacumab Fully human monoclonal antibody targeting P-selectin, intended for quarterly administration In two ph3 trials: 131 in reduction of VOCs132 in reduction of hospital readmission rates due to VOCs
GBT021601, aka GBT601 Oral, once-daily sickle haemoglobin polymerisation inhibitor In ph2 portion of Ph2/3 021 study
… and Pfizer's
PF-07209326 E-selectin antagonist Ph1 trial ongoing
VOCs = vaso-occlusive crises. Source: Evaluate Pharma & company release. 

Neither is it entirely clear what this deal means for the gene therapy and editing space, spearheaded by Crispr Therapeutics and Vertex. Their ex-vivo gene-edited therapy exa-cel posted highly encouraging mid-stage data in June, and if durability holds up the asset could start to look like a functional cure (EHA 2022 – Crispr still looks bloody good, June 13, 2022).

The partners intend to file exa-cel later this year, and should they get to market Pfizer represents a more formidable foe than Global Blood. There are also questions about how big the market for gene edited therapies could be, given the harsh pre-conditioning regimens needed, not to mention the cost.

Other companies with late-stage sickle cell projects include Forma Therapeutics and Agios, both of which are developing oral pyruvate kinase R activators.

Investors might be unwise to count on that $3bn peak revenue prediction coming true. But they can at least hope that the Global Blood deal can wipe away the stain of Pfizer’s last foray into sickle cell deal-making. That ended with rivipansel, the pan-selectin antagonist Pfizer had licensed from Glycomimetics, failing in phase 3 in 2019.

What investors can rely on is Pfizer's appetite for deal making. The pharma giant has pledged almost $18bn on M&A and licensing deals in 2022, with Biohaven the biggest takeout of the year so far. With biotech valuations still under stress, the cash rich company surely has other targets in its sights. 

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