GBT plans to investigate inclacumab as a treatment for painful blockages of blood vessels in patients with sickle cell disease (SCD).
The technology transfer process from Roche to a contract manufacturing organization has already started.
Inclacumab is a human monoclonal antibody specifically made to bind to the protein P-selectin, which is found on the surface of the cells lining the inner walls of blood vessels.
In sickle cell anemia, P-selectin supports the adhesion of sickle-shape red blood cells to the blood vessel walls, thus blocking blood flow in smaller vessels. Blocked blood flow can then cause inflammation and pain crises.
An anti-P-selectin antibody holds the therapeutic potential of helping to maintain normal blood flow and avoiding the common painful complication of sickle cell anemia, called vaso-occlusive crises.
Under the exclusive global licensing agreement, GBT will oversee the global development, manufacturing, and commercialization of inclacumab.
According to the contract, Global Blood Therapeutics will pay Roche $2 million upfront and $125 million down the line in milestone payments, plus royalties, if inclacumab is approved to treat vaso-occlusive crises in sickle cell disease patients.
“We have been working diligently to diversify our product pipeline through both internal research and external business development efforts and are excited to have entered into this agreement for inclacumab,” Ted W. Love, MD, president and CEO of GBT, said in a news release.
“We are committed to developing transformative treatments for the SCD community and becoming the preeminent SCD company,” Love said. “Inclacumab is an ideal complement to voxelotor, our lead investigational oral, once-daily therapy, in Phase 3 clinical development for SCD.
“Like voxelotor, inclacumab has a strong scientific rationale and has the potential to provide significant clinical benefit for SCD patients,” he added.
GBT plans to submit an investigational new drug (IND) application to the U.S. Food and Drug Administration for inclacumab in 2021.
Roche’s technology was developed for people with coronary heart disease. The company completed a randomized, multicenter, double-blind, placebo-controlled Phase 2 trial (NCT01327183) to evaluate the effectiveness and safety of inclacumab in patients with a specific type of myocardial infarction (known as non-STEMI) undergoing a nonsurgical procedure to treat inadequate blood flow to the heart.
A total of 532 patients were randomly assigned to receive a 5 mg/kg, 20 mg/kg, or placebo infusion, administered between one and 24 hours before the medical procedure. They were then followed for four months.
Results showed that inclacumab at a 20 mg/kg dose significantly reduced heart damage after the nonsurgical intervention in non-STEMI patients, and benefits were more pronounced when the infusion was administered less than three hours before the procedure.
Roche discontinued the inclacumab program after this study.
Global Blood Therapeutics will make use of the safety data generated by Roche’s Phase 2 trial to advance the therapy’s development in sickle cell disease.