Kiadis Pharma N.V., a clinical stage biopharmaceutical company developing innovative T-cell immunotherapy treatments for blood cancers and inherited blood disorders, today announces that the Pediatric Committee (PDCO) of the European Medicines Agency (EMA) has accepted the Company’s Pediatric Investigation Plan (PIP) for ATIR101™ for the adjunctive treatment in hematopoietic stem cell transplantation (HSCT) for a malignant disease. In addition, the PDCO has agreed that the Company may defer conducting the studies defined in the PIP until after it files a Marketing Authorization Application (MAA) in Europe for the use of ATIR101™ for the treatment of blood cancers.
The Company’s PIP provides for a Phase II trial to evaluate the safety and efficacy of ATIR101™ as an adjunctive treatment on top of an HSCT in pediatric patients up to 18 years of age with a hematologic malignancy, who are eligible for an HSCT but without the availability of a fully matched donor. Patients will receive either an HSCT from a partially matched (haploidentical) related family donor with the adjuvant infusion of ATIR101™, or an HSCT using umbilical cord blood stem cells from an unrelated donor. The primary endpoint of the trial will be Graft-versus-Host-Disease-free, relapse-free survival (GRFS).
Manfred Rüdiger, PhD, Chief Executive Officer of Kiadis Pharma, commented: “The acceptance of our pediatric development program by EMA’s PDCO is an important regulatory milestone for ATIR101™. Furthermore, the PDCO’s decision to allow us to defer the initiation of our PIP means the Company can submit an MAA to EMA for the use of ATIR101™ in adult blood cancer patients substantially earlier than if it was required to complete the PIP beforehand. This is an important step forward in our efforts to bring ATIR101™ to the market and evidences the close and constructive interactions that Kiadis Pharma has established with the regulatory authorities in developing ATIR101™.
For patients suffering from blood cancers, an allogeneic hematopoietic stem cell transplantation (HSCT) is generally regarded as the most effective curative approach. During an HSCT treatment, the bone marrow, harboring the diseased cancer cells, is completely destroyed and subsequently replaced by stem cells in the graft from a healthy donor. After an HSCT treatment it usually takes the patient at least six to twelve months to recover to near-normal blood cell levels and immune cell functions. During this period, the patient is highly vulnerable to infections caused by bacteria, viruses and fungi but also to disease relapse.
ATIR101™ (Allodepleted T-cell ImmunotheRapeutics) provides for a safe donor lymphocyte infusion (DLI) from a partially matched (haploidentical) family member without the risk of causing severe Graft-versus-Host-Disease (GVHD). The T-cells in ATIR101™ will help fight infections and remaining tumor cells and thereby bridge the time until the immune system has fully re-grown from stem cells in the transplanted graft.
In ATIR101™, T-cells that would cause GVHD are eliminated from the donor lymphocytes using Kiadis Pharma’s photodepletion technology, minimizing the risk of GVHD and eliminating the need for prophylactic immune-suppression. At the same time, ATIR101™ contains potential cancer killing T-cells from the donor that could eliminate residual cancer cells and help prevent relapse of the disease, known as the Graft-versus-Leukemia (GVL) effect.
ATIR101™, administered as an adjunctive immuno-therapeutic on top of HSCT, provides the patient with functional, mature immune cells from a partially matched family donor that can fight infections and tumor cells but that do not cause GVHD. ATIR101™ thus has the potential to make curative HSCT a viable option to many more patients.
The Company estimates that approximately 35% of patients who are eligible and in urgent need of HSCT will not find a matching donor in time. A partially matched (haploidentical) family donor, however, will be available to over 95% of patients.
ATIR101™, consisting of donor T-cells that fight infections and residual tumor cells while not eliciting severe GVHD, is designed to result in low relapse rates and low rates of death due to infections, in the absence of severe acute GVHD.