AMSTERDAM--(BUSINESS WIRE)--Regulatory News:
Kiadis Pharma N.V. (“Kiadis Pharma” or the “Company”) (Euronext Amsterdam and Brussels: KDS), 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.
About Kiadis Pharma
Kiadis Pharma is focused on cell-based immunotherapy products for the treatment of blood cancers and inherited blood disorders. The Company’s products have the potential to address the risks and limitations connected with allogeneic hematopoietic stem cell transplantation (HSCT), namely Graft-versus-Host-Disease (GVHD), cancer relapse, opportunistic infections and limited matched donor availability. The Company believes that HSCT could become a first-choice treatment for blood cancers, inherited blood disorders and possibly autoimmune diseases and solid organ transplantations.
On December 5, 2016 at the Annual Meeting of the American Society of Hematology (ASH), the Company reported positive Phase II results with its lead product ATIR101™ in patients with blood cancer. The data showed that ATIR101™ significantly reduced Transplant Related Mortality and significantly improved Overall Survival. In addition, ATIR101™ did not elicit grade III-IV GVHD in any patient. Based on these positive results, a Phase III clinical trial has been initiated. ATIR101™ has been granted Orphan Drug Designations both in the US and Europe.
The Company’s second product candidate, ATIR201™, addresses inherited blood disorders with an initial focus on thalassemia, a disease which results in destruction of red blood cells in patients. ATIR201™ Phase I/II clinical development has been initiated recently.
Kiadis Pharma, based in Amsterdam, The Netherlands, was granted an Advanced Therapy Medicinal Product (ATMP) certificate for manufacturing quality and non-clinical data by the European Medicines Agency (EMA). The Company’s shares are listed on Euronext Amsterdam and Euronext Brussels. For more information visit www.kiadis.com
Forward Looking Statements
Certain statements, beliefs and opinions in this press release are forward-looking, which reflect Kiadis Pharma’s or, as appropriate, Kiadis Pharma’s directors’ current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, Kiadis Pharma expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither Kiadis Pharma nor its advisers or representatives nor any of its subsidiary undertakings or any such person's officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this press release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release.