Top-Line Data From All Patients On The Primary Endpoint Of Kiadis Pharma’s Phase II Trial With ATIR101 Selected For Oral Presentation At 42nd Annual Meeting Of The European Society For Blood And Marrow Transplantation

Additional ATIR101™ data also selected for three poster presentations at EBMT

Amsterdam, The Netherlands, March 1, 2016, – 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 key data from the Company’s ongoing single-dose Phase II clinical trial (NCT01794299/EudraCT 2012-004461-41) with its lead product ATIR101™ has been selected for an oral presentation to discuss the top-line data at the 42nd Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT) on April 3-6, 2016 in Valencia, Spain. Three abstracts on additional ATIR101™ data and the ATIR101™ development program have also been accepted for poster presentations at EBMT.

The oral presentation entitled ‘Donor lymphocytes depleted of alloreactive T-cells (ATIR101) improve overall survival and reduce transplant related mortality in a T-cell depleted haploidentical HSCT: Results from a Phase 2 Trial in patients with AML and ALL’ will be delivered in Room B6 on Monday April 4, 2016 at 16:05 CET by Dr. Denis-Claude Roy, Professor of Medicine at the University of Montreal and principal investigator for the study. The presentation will include the full dataset on the primary endpoint of the study (Transplant Related Mortality at six months post transplantation) which will also be put into perspective with other current approaches in the literature for patients lacking a fully matched donor, such as the Baltimore approach using post-transplant cyclophosphamide.

Oral and poster presentations at EBMT:

Oral Presentation Monday April 4, 2016 at 16:05 CET (Room B6) Title: Donor lymphocytes depleted of alloreactive T-cells (ATIR101) improve overall survival and reduce transplant related mortality in a T-cell depleted haploidentical HSCT: Results from a Phase 2 Trial in patients with AML and ALL. Presenting author: Denis-Claude Roy

Poster Presentations
Monday April 4, 2016 from 17:15-19:00 CET, Poster Area
- P020: Title: Leukemia-associated antigen reactive T-cells in ATIR101, a recipient-specific allodepleted T-cell product facilitating haploidentical HSCT.
Presenting author: Jurjen Velthuis

Tuesday April 5, 2016 from 17:15-19:00 CET, Poster Area
- P442: Title: Effect of graft source on safety and efficacy in patients undergoing hematopoietic stem cell transplantation.
Presenting author: Stephan Mielke
- P462: Title: An exploratory, open-label, multicenter study to evaluate safety and efficacy of a two-dose regimen of ATIR101 in patients with a hematologic malignancy, who received a CD34-selected hematopoietic stem cell transplantation from a haploidentical donor.
Presenting author: Stephan Mielke

Full abstracts will be made available on the EBMT website (http://www.ebmt2016.org/abstracts.html) but data presented during the oral presentation will contain additional data that was not yet available at the time of abstract submission.

About 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, harbouring 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 tumour 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.

Therefore, 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 tumour 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 tumour 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 a clinical stage biopharmaceutical company focused on research, development and future commercialization of cell-based immunotherapy products for the treatment of blood cancers and inherited blood disorders. The Company believes that its innovative products have the potential to address the current risks and limitations connected with allogeneic hematopoietic stem cell transplantation (HSCT), being graft-versus-host disease (GVHD), cancer relapse, opportunistic infections and limited matched donor availability. HSCT is generally regarded as the most effective curative approach to blood cancers and certain inherited blood disorders and the Company expects that HSCT could become a first-choice treatment for blood cancers and inherited blood disorders once current risks and limitations are addressed, thereby meeting a significant unmet medical need with its products.

The Company’s product ATIR101™ is being tested using a single-dose regimen in an open-label fully enrolled Phase II trial in patients with blood cancer who have not found a matching donor and where a partially matched (haploidentical) family member is used as donor for HSCT. The primary endpoint for the final patient in this trial will be reached at the end of Q1, 2016 and top-line results will be announced in April 2016. Very encouraging and positive interim data of this trial was presented recently at ASH2015.

In addition, the Company is enrolling blood cancer patients in a further Phase II clinical trial to study the safety and efficacy of administrating a second dose of ATIR101™ to further improve the HSCT outcome.

The European Medicines Agency (EMA) has issued an Advanced Therapy Medicinal Product (ATMP) certificate for manufacturing quality and non-clinical data to the Company, and to date Kiadis Pharma is one of only five companies that have received such a certificate.

ATIR101™ has been granted Orphan Drug Designations both in the US and Europe.

ATIR201™ will be developed for inherited blood disorders with an initial focus on thalassaemia, an inherited blood disorder which results in improper oxygen transport and destruction of red blood cells in a patient. ATIR201™ is expected to enter Phase I/II clinical development for thalassaemia in the first quarter of 2016. Kiadis Pharma recently announced a collaboration with the Thalassaemia International Federation (TIF), an internationally renowned organisation that seeks to address the needs of patients, carers, healthcare professionals and the general public in the area of thalassaemia.

Kiadis Pharma is based in Amsterdam, the Netherlands and its shares are listed on Euronext Amsterdam and Euronext Brussels. Further information can be found at: www.kiadis.com

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