Global Blood Therapeutics (GBT) has been granted exclusive global rights to two small-molecule research programs for sickle cell disease (SCD) as part of a newly announced agreement between GBT and Sanofi.
The two early stage programs, from Sanofi’s subsidiary Bioverativ, will explore new mechanisms to prevent or reverse red blood cell sickling. They aim to develop a new therapeutic approach to reduce inflammation and oxidative stress, both of which contribute to the damaging effects of SCD. Of note, oxidative stress refers to cellular damage caused by the buildup of oxidant molecules inside cells.
“These novel discovery programs represent promising approaches that we believe may have the potential to lead to meaningful improvements for patients,” Jung E. Choi, chief business and strategy officer of GBT, said in a press release.
The programs will supplement GBT’s existing SCD pipeline, which includes the oral therapy Oxbryta (voxelotor), designed to prevent red blood cell sickling, and the investigational antibody inclacumab for the treatment of painful SCD crises.
Under the terms of the agreement, GBT will be responsible for all research and development, as well as global regulatory and marketing activities. In turn, Sanofi will be entitled to an upfront payment and payments of up to approximately $353 million upon the achievement of certain developmental, regulatory, and commercial milestones.
“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,” Choi said. “We are advancing our robust internal research programs with disease-modifying potential while continually exploring partnership opportunities across a variety of mechanisms.”
The new anti-sickling approaches are distinct from those of Oxbryta, which was approved by the U.S. Food and Drug Administration in 2019 for the treatment of SCD in patients ages 12 and older. It is the first approved treatment targeting red blood cell sickling, the root cause of SCD.
The European Medicines Agency is currently reviewing an application submitted by GBT requesting the approval of Oxbryta for the treatment of SCD in patients ages 12 and older in the EU.
SCD, also known as sickle cell anemia, is a genetic blood disease caused by a mutation in the HBB gene that encodes part of the oxygen-carrying protein hemoglobin. Red blood cells with mutant hemoglobin become rigid and sickle-shaped, obstructing blood vessels, causing inflammation, and eventually pain crises. Potential treatments for SCD usually focus on reducing red blood cell sickling, inflammation, and pain crises.
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