Takeda recently announced favorable results from a phase 3 study of TAK-755, the company’s investigational enzyme replacement therapy for an ultra-rare blood disorder known as congenital thrombotic thrombocytopenic purpura (cTTP).
cTTP is a subtype of thrombotic thrombocytopenic purpura (TTP), a chronic, and debilitating blood clotting disorder linked to mutations in the ADAMTS13 gene. It is characterized by abnormal clotting in the small blood vessels of the body. Signs and symptoms typically arise in infancy/early childhood and include low platelet levels (thrombocytopenia), neurological dysfunction, and hemolytic anemia, a condition in which red blood cells are destroyed faster than they can be generated.
cTTP has been linked to stroke and cardiovascular disease. Affected individuals will often experience a significantly decreased quality of life, along with a shortened lifespan, as compared to the general population. Left untreated, the condition can be lethal. Existing treatments include plasmapheresis for acute episodes, and prophylactic plasma therapy for those with chronic disease.
The phase 3 study evaluated TAK-755 via a comparison with existing plasma-based therapies, which are the current standard of care (SoC). According to a Takeda release, TAK-755 is the first and only recombinant ADAMTS13 protein in development. It provides targeted therapy to address an unmet medical need in patients with TTP, by replacing the missing or deficient ADAMTS13 enzyme.
The interim results from the randomized cross over study showed that TAK-755 reduced the incidence of thrombocytopenia events by 60%, an important marker of disease activity in cTTP, as compared to SoC. The proportion of subjects experiencing adverse events determined to be related to the treatment was substantially lower among subjects during treatment with TAK-755 (8.9%) compared to that while receiving SoC therapy (47.7%).
“We are committed to advancing treatment options for those living with cTTP, who currently have no therapies approved specifically to manage their condition,” said Daniel Curran, MD, Head, Rare Genetics & Hematology Therapeutic Area Unit at Takeda. “The results of the trial are very encouraging, and we look forward to continuing to engage with global regulatory bodies with the aim of bringing TAK-755 to patients as rapidly as possible.”
The company plans to submit the results of this interim analysis for presentation at an upcoming scientific meeting.
Source: Takeda press release dated January 5, 2023