Takeda touts interim PhIII data of enzyme replacement therapy for rare blood disease
Takeda says in an interim Phase III analysis that its enzyme replacement therapy reduced instances of low blood platelet count for patients with a rare blood clot disorder known as congenital thrombotic thrombocytopenic purpura (cTTP) compared to standard of care plasma-based therapies.
Dan CurranNext, it plans to file for authorization for the drug later this year “broadly — so not just in the US and EMA but also other jurisdictions globally,” Takeda head of rare genetics and hematology Dan Curran told Endpoints News.
According to a press release Thursday morning, Takeda’s ERT, dubbed TAK-755, reduced low platelet count events by 60% compared to standard of care at the time of interim analysis. The study included around 60 patients, and the interim analysis used data on adolescents and adult patients, but not children under 12. Curran also noted that the ERT reduced instances of other symptoms associated with the disease, which manifests in a multitude of ways, but said that data would be presented at a future medical conference.
TTP comes in two forms — congenital and immune, in which patients develop an immune response later in life that triggers the disease. TTP patients lack a protein that cleaves Von Willebrand factor, the glue that sticks platelets together to form clots and stop bleeding. Takeda’s ERT is a recombinant version of that protein called ADAMTS13.
cTTP is exceedingly rare. Curran estimated that there are around 300 to 400 cases in the US. Patients with the disease experience bleeding in or under the skin, jaundice, extreme fatigue, vomiting, or other symptoms, and in the worst cases, stroke or seizures.
In the study, patients who got Takeda’s ERT also experienced fewer side effects — 8.9% had adverse events on Takeda’s treatment compared to 47.7% on plasma-based infusions. “Many patients discontinue supplementation with plasma-derived products because of adverse events,” Curran noted, “so it’s incredibly important that you have a safe drug that will be used by patients continually.”
He later added that, especially in the case of cTTP, in which many are diagnosed as children, it was important that patients could use the drug over their entire lifetime. Like other ERTs, Takeda’s ERT is dosed once every two weeks.
Takeda acquired TAK-755 from its 2019 buyout of Shire (which acquired the ERT from its 2016 merger with Baxalta).
Takeda is also testing the ERT in a Phase II study in iTTP and in a Phase Ib study in sickle cell disease, both of which Curran said will have data this year.
