Kiadis Pharma has completed the five-year follow-up of patients with high-risk malignancies from its Phase I/II clinical study that confirmed the long-term safety and efficacy of Atir over a broad dose range.
Atir is designed to allow stem cell transplantations from partially mismatched (haploidentical) family donors for patients who do not have a standard of care stem cell donor available.
The company said the results showed that Atir infusion after a T-cell depleted haploidentical hematopoietic stem cell transplantation (HSCT) offers immune protection after the transplantation and also enhances long-term outcome in high-risk patients with very poor prognosis.
The follow-up results demonstrated Atir’s effectiveness as a curative treatment for patients that do not have a standard of care stem cell donor available.
The study also showed that the long term survival compared favourably with patients who have a standard of care matched unrelated donor available.
According to the company, the overall survival of patients with high-risk malignancies in the Phase I/II study who received an efficacious dose of Atir was 78% and 67% after one and five years respectively.
The data gathered from the Center for International Blood & Marrow Transplant Research (CIMBTR) reveals that the one and five year survival of patients with acute myeloid leukemia (AML) who have a standard of care matched unrelated donor available, differs from 65% and 35% in low-risk patients to 45% and 20% in high-risk patients respectively.
Nineteen high-risk leukemia patients were treated with escalating doses of Atir after a haploidentical HSCT in the Phase I/II study.
No transplant related mortalities and Grade III-IV (life-threatening) acute graft versus host disease (GvHD) was noticed in patients, according to the five-year follow-up data.
Kiadis Pharma CEO Manfred Ruediger said the results from this study, which spans five years, show that Atir might change the way patients with hematological malignancies will be treated.
"These data show that Atir not only prevents significant infections without eliciting life-threatening GvHD, but also strongly improves survival rates in patients with high-risk malignancies and very poor prognosis," Ruediger said.
"Our currently ongoing international Phase II clinical study is designed to confirm and extend these data to expedite moving ATIR towards regulatory approval."
ATIR, a cell-based medicinal product, can serve as an effective therapy for blood cancer patients as it is highly effective in minimising post-transplantation risks and improving overall survival.
The company is currently conducting a multi-centre Phase II study to confirm and extend the data from the Phase I/II programme.
The topline results of the first phase of the study involving patients with AML, acute lymphoblastic leukemia and myelodysplastic syndrome are expected in the first half of 2014.