Blood and bone marrow expert Kiadis Pharma has announced their submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for its lead product, ATIR101™. The Company is seeking marketing approval in the European Union for ATIR101™ as an adjunctive treatment in haematopoietic stem cell transplantation (HSCT) for malignant disease.
The filing is based on Kiadis Pharma’s existing clinical data and follows positive interactions with the EMA Rapporteur and Co-Rapporteur which indicated support for the filing using the Company’s single dose Phase II trial with ATIR101™ as the pivotal study. The Company submitted the application under the European Union’s centralized procedure, which permits the agency to issue a single marketing authorization that is valid across all EU countries.
Arthur Lahr, Chief Executive Officer of Kiadis Pharma, commented: “Submission of this MAA to the EMA marks a pivotal point in Kiadis Pharma’s development and brings us a significant step closer towards commercializing our lead product ATIR101™ for the treatment of blood cancer, across the EU. The data generated thus far for ATIR101™ has been very positive and we are optimistic that we will receive a positive outcome from the EMA during the second half of 2018, allowing us to make ATIR101™ available to transplantation centers across the EU in 2019. It is a great achievement that Kiadis Pharma has reached this milestone on its own merit, which only a very few biotech companies accomplish, and it is testament to the strength of the unique data and of the Kiadis Pharma organization.”
Positive and significant results from Kiadis Pharma’s single dose Phase II trial with ATIR101™ were reported on December 5, 2016 at the Annual Meeting of the American Society of Hematology (ASH). The data showed that ATIR101™ significantly improved overall survival compared to a historical matched control. In addition, relapse and Graft-versus-Host-Disease (GVHD) rates were significantly lower than those reported for alternative approaches for HSCT. Specifically, ATIR101™ did not elicit acute grade III-IV GVHD in any patient.
ATIR101™ protection for transplant vulnerability
For patients suffering from blood cancers and inherited blood disorders, an allogeneic hematopoietic stem cell transplantation (HSCT) is generally regarded as a potentially curative approach. During an HSCT treatment, the patient’s diseased blood and immune system are destroyed and subsequently replaced by a healthy system from a donor. The treatment is, however, very risky as it usually takes the patient at least six to twelve months to recover to near-normal immune cell functions, making patients highly vulnerable to infections and disease relapse. Mature lymphocytes in the donor graft would provide immediate protection, but, depending on the level of genetic mismatch between patient and donor, may cause life threatening Graft-versus-Host-Disease (GVHD).
The Company estimates that approximately 35% of patients who are eligible and in urgent need of an HSCT will not find an adequately matched donor in time. A half-matched (haploidentical) parent or child, however, could serve as a donor for nearly all patients, yet would cause severe GVHD due to the infusion of half-matched mature lymphocytes. The therapy Kiadis Pharma is developing would enable the use of haploidentical transplants without the unacceptable risk of GVHD.
ATIR101™ (Allodepleted T-cell ImmunotheRapeutics) provides for a safe single dose donor lymphocyte infusion (DLI) with functional, mature immune cells from a haploidentical family member with minimal risk of causing severe GVHD. ATIR101™ will help fight infections and remaining tumor cells and thereby bridge the time until the patient’s immune system has fully re-grown from stem cells in the transplanted graft.