Date: 2013-09-11
Type of
information: Results
phase: 1-2
Announcement: results of five year follow-up
Company: Kiadis Pharma (The Netherlands)
Product: ATIR™
Action
mechanism:
- ATIR™ is a cell based medicinal product candidate enabling stem cell transplantations from mismatched (haploidentical) family donors to patients suffering from blood cancer. Stem cell transplantation is the only potentially curative option for many patients but a matching donor is available for only half of the patients in need. ATIR™ thus has the potential to address this unmet need and to make stem cell transplantations available for patients worldwide. Those T-cells in a haploidentical graft which would cause Graft-versus-Host-Disease (GvHD) are selectively eliminated using proprietary technology to produce ATIR™. ATIR™ is administered as an adjunctive treatment on top of a haploidentical stem cell transplantation enhancing early immune reconstitution without causing GvHD.
Disease: patients with high-risk malignancies
Therapeutic
area: Cancer - Oncology
Country:
Trial
details:
Latest
news:
- • On September 11, 2013, Kiadis Pharma, a clinical stage biopharmaceutical company developing treatments for blood cancers, has announced that the five year follow-up of patients with high-risk malignancies from its Phase I/II clinical study confirms long-term safety and efficacy of ATIR™ over a broad dose range. The results demonstrate proof of concept and show that ATIR™ infusion after a T-cell depleted haploidentical hematopoietic stem cell transplantation (HSCT) provides immune protection shortly after the transplantation and improves long-term outcome in high-risk patients with very poor prognosis.
- Not only does the study confirm that ATIR™ provides an effective treatment for patients for whom a standard of care stem cell donor is not available, the long term survival even seems to compare favorably to patients who do have a standard of care matched unrelated donor available. 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. Data from the Center for International Blood & Marrow Transplant Research (CIMBTR) show that the one (and five) year survival of patients with acute myeloid leukemia (AML) who do have a standard of care matched unrelated donor available, varies from 65% (and 35%) in low-risk patients to 45% (and 20%) in high-risk patients, respectively.
- In this Phase I/II study, 19 high-risk leukemia patients were treated with escalating doses of ATIR™ after a haploidentical HSCT. The five-year follow-up data show no transplant related mortality in the nine patients who received an efficacious dose of ATIR™. In addition, no Grade III-IV (life-threatening) acute Graft versus Host Disease (GvHD) was observed at any dose, which again compares favorably to standard of care matched unrelated donor transplantations, where (according to data from the CIBMTR) incidence of life-threatening GvHD is 30%. The strong five-year survival data also suggest that immune cells responsible for the Graft versus Leukemia effect are retained in ATIR™.
- The results of the study allowed selecting the optimal ATIR™ dose for further development. Data from this five-year study will be published in due course in a peer-reviewed medical journal.
- An international multi-center Phase II study including patients with AML, acute lymphoblastic leukemia (ALL) and myelodysplastic syndrome (MDS), to confirm and extend the data from the Phase I/II study, is now ongoing with topline results of the first phase expected in H1 2014.
Is
general: Yes
