Clinical Trials

Date: 2017-04-03

Type of information: Clinical trial authorization

phase: 1-2

Announcement: clinical trial authorization

Company: Kiadis Pharma (The Netherlands)

Product: ATIR201™

Action mechanism:

• cell therapy/immunotherapy product. 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.

Disease: beta-thalassemia major

Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases

Country: UK

Trial details: https://www.clinicaltrialsregister.eu/ctr-search/trial/2016-002959-17/GB

Latest news:

• • On April 3, 2017, Kiadis Pharma announced that the company has received regulatory approval from the national authority in Germany (the PEI, the Paul-Ehrlich-Institute) to start its Phase I/II clinical trial with ATIR201™ for thalassemia (CR-BD-001). This follows after the previous announcement at the end of 2016 that it had initiated the Phase I/II trial with ATIR201™ after having obtained regulatory approval from the national authority in the United Kingdom.
• • On February 6, 2017, Kiadis Pharma provided a progress update on the clinical status of ATIR201™ for thalassemia (CR-BD-001). The trial has received regulatory approval from the Medicines and Healthcare products Regulatory Agency in the UK as well as approval from the Ethics Committees of the Royal Manchester Children’s Hospital and the Birmingham Children’s Hospital. In addition, approval from the Ethics Committee of the University of Regensburg in Germany has now been received, with regulatory approval from the national authority in Germany pending. Kiadis Pharma expects the first clinical trial data to become available in the second half of 2017.
• • On December 15, 2016, Kiadis Pharma announced that it has obtained regulatory approval from the national authority in the United Kingdom (the MHRA, the Medicines and Healthcare products Regulatory Agency), as well as approval from the Ethics Committees of the Royal Manchester Children’s Hospital and the Birmingham Children’s Hospital to start a Phase I/II clinical trial with its product ATIR201™ for thalassemia, according to schedule. In this new trial the safety and feasibility of using ATIR201™ in pediatric and adult patients suffering from beta-thalassemia major, the most severe form of the disease, will be studied. There is currently no approved curative medicine for beta-thalassemia major. The current standard of care treatment is purely symptomatic, requiring lifelong blood transfusions and iron chelators, with drug support to improve hematopoiesis. Replacing the diseased system and restoring the proper production of hemoglobin through an allogeneic hematopoietic stem cell transplantation (HSCT) from a healthy half-matched family donor could provide a cure for this disease. After an HSCT treatment, however, it usually takes the patient several months to recover to near-normal blood cell levels and immune cell functions, during which time the patient is vulnerable to infections caused by bacteria, viruses and fungi.
• The addition of ATIR201™, administered as an adjunctive immuno-therapeutic on top of an HSCT, is intended to provide the patient with functional, mature immune cells that can fight infections while not eliciting severe Graft-versus-Host-Disease (GVHD), thereby bridging the time until the immune system has fully re-grown from stem cells in the transplanted graft.
• The new trial will commence in the United Kingdom and the Company plans to expand the trial into Germany pending regulatory approval. A total of up to ten beta-thalassemia major patients will be enrolled. Kiadis Pharma expects first safety and efficacy results to become available in the second half of 2017.

 

Is general: Yes