As Global Blood Therapeutics works to bring its sickle cell disease drug, Oxbryta, to younger patients, the company is bolstering its pipeline with a pair of early-stage programs from Sanofi.
In exchange for the global rights to the assets, Sanofi will receive up to $353 million, including a $2.25 million upfront fee and potential development, regulatory and commercial milestone payments down the line, according to a securities filing.
The programs, licensed from Sanofi’s Bioverativ unit, work differently than Oxbryta, which stops the polymerization, or sickling, of hemoglobin S, an abnormal type of the oxygen-carrying protein found in people with sickle cell disease.
One of the Sanofi programs uses a different anti-sickling mechanism than Oxbryta, while the other uses a new approach to reduce inflammation and oxidative stress. These mechanisms could be complementary to that of Oxbryta, GBT said in a statement, suggesting a potential for combinations.
“We envision a future in which sickle cell disease is a well-managed condition with the potential for a functional cure in the form of patient-friendly oral therapies,” said Jung Choi, GBT’s chief business and strategy officer, in the statement. “As we work toward this vision and our goal to transform the treatment and care of people living with this devastating disease, we are advancing our robust internal research programs with disease-modifying potential while continually exploring partnership opportunities across a variety of mechanisms.”
The FDA greenlighted Oxbryta for patients aged 12 and older in November 2019. It was a landmark approval in a field where doctors focused on managing symptoms, such as infections, pain throughout the body and fatigue. The company hopes to get an approval in younger children and has two studies ongoing in children and adolescents.
In August 2018, GBT brought in a discontinued heart disease drug from Roche with hopes that it could make a dent in sickle cell. The company paid $2 million upfront and promised about $125 million in milestones for inclacumab, an antibody that targets P-selectin, a cell adhesion protein found lining the walls of blood vessels and on the surface of activated platelets, helping them act as a coagulant in cell-cell interactions.
The company tapped Syros Pharmaceuticals and its gene control platform in December 2019 to develop new treatments for sickle cell and beta thalassemia. Under the deal, Syros is using its technology to identify targets and discover drugs that induce fetal hemoglobin. GBT has the option to license any drugs that come out of the partnership for development and commercialization.
Editor’s note: This story was updated to include financial terms of the agreement with Sanofi.