Björn Mellgård, MD, PhD: The First Clinical Study of TAK-755 in SCD Patients

The first clinical study of a recombinant ADAMTS13 in patients with sickle cell disease was presented at the 2021 American Society of Hematology (ASH) Annual Meeting & Exposition.

In an interview with HCPLive®, Björn Mellgård, MD, PhD, Vice President, Global Program Lead, Rare Genetics and Hematology, Takeda, discussed 2 studies that Takeda conducted on TAK-755, a recombinant enzyme called ADAMTS13 that’s involved in the regulation of clotting.

Of the 2 studies, the first examined the recombinant ADAMTS13 for patients with severe congenital thrombotic thrombocytopenic Purpura (cTTP), and the second study looked at patients with sickle cell disease (SCD).

The function of ADAMTS13, or TAK-755, is to reduce the size of the Von Willebrand factor (VWF), a protein involved with blood clotting.

Mellgård explained that when a patient has a deficiency of ADAMTS13 they could be at risk of spontaneous clotting because they accumulate long multimers of Von Willebrand factor.

"What this factor is doing is it's sort of attaching platelets, the small platelets, or thrombocytes, to the site of injury, and acting as a bridge between site of injury and the platelets to form a plug there," Mellgård said.

Patients with sickle cell disease can experience a vaso-occlusive crisis (VOC), also known as a pain crisis, at any time. In addition to being a very painful event, a pain crisis can damage the organs.

According to this research, investigators believe that during a pain crisis there's an increase of Von Willebrand factor. This can contribute to the formation of small blood clots in the organs and therefore to the signs and symptoms of a pain crisis.

"And if we give them TAK-755, we will reduce that tendency to formation of these small blood clots," Mellgård explained, "and reduce the tendency for ischemia or reduced blood flow in in critical organs."