- Global Blood Therapeutics and Syros Pharmaceuticals are teaming up to use the latter company's technology to find new treatments for sickle cell disease and beta thalassemia.
- The aim is that Syros' gene control platform will be able to switch on production of fetal hemoglobin, a form of the red blood cell protein that carries oxygen throughout the body. The DNA responsible for making fetal hemoglobin goes dormant a few months after birth — giving way to the production of adult hemoglobin, which binds less tightly to oxygen.
- Both sickle cell and beta thalassemia start presenting symptoms after fetal hemoglobin production stops. Keeping that production going is at the center of Global Blood's new deal, as well as another biotech collaboration between Vertex and CRISPR Therapeutics. Early results of that pairing's gene-editing drug showed one sickle cell patient and one beta thalassemia patient seeming to respond well months after treatment.
Global Blood is fresh off a victory in sickle cell, having received approval in late November for a new medication called Oxbryta (voxelotor). There isn't much time to celebrate, however, given how quickly competition is forming.
While sickle cell patients for decades had just one treatment option, three have come to market since 2017. More may be on the way too, as Bluebird bio gears up for a late-stage study of its LentiGlobin gene therapy and Vertex and CRISPR forge ahead with their gene-editing approach.
The Syros deal suggests Global Blood is already preparing for a scenario in which Oxbryta must compete with newer, and potentially more effective, therapies.
Per deal terms, Global Blood is paying $20 million upfront plus another $40 million for preclinical research over three years. For any drug candidate the company decides to option, Syros could take home up to $315 million in various milestone payments. Syros is also eligible for sales royalties in the mid- to high-single digits for any products the deal creates, as well as co-promotional rights in the U.S. for the first product to market.
It will take years before Global Blood finds out whether this bet is fruitful. Still, fetal hemoglobin appears to be a promising target. Novo Nordisk, for example, put $400 million on the table in 2018 to license an EpiDestiny drug that raised levels of the protein in early clinical testing. Novo said it would study the drug for sickle cell and beta thalassaemia.
Vertex and CRISPR are furthest along in this approach. Their therapy edits a gene known as BCL11A in order to reactivate fetal hemoglobin production. So far, the two patients for whom data were disclosed each achieved close to normal levels of hemoglobin in the months after treatment.
"A little bit of fetal hemoglobin can go a long way — not just because you're replacing things, but because you're preventing the sickle [cells] from agglutinating together," said Charles Abrams, director of the Blood Center for Patient Care and Discovery at Penn Medicine, in an interview with BioPharma Dive last month at the American Society of Hematology's annual meeting.
Syros was also at the ASH meeting, where one of the company's studies found a novel suppressor to fetal hemoglobin production that's different than the BCL11A gene.
In a statement, Global Blood noted how Syros had identified a couple targets that "could serve as potential targets to switch on" the fetal hemoglobin gene.
"We believe that Syros' approach to inducing fetal hemoglobin is one of the most promising ways to identify the next generation of therapies to treat sickle cell disease and beta thalassemia at a fundamental level — upstream of serious complications such as organ damage, organ failure and early death," Global Blood CEO Ted Love said in the statement.
Global Blood's share price was down more than 5% in late morning trading Wednesday, while Syros was up 20%.
Ned Pagliarulo contributed reporting.