Kiadis Pharma announces ini­ti­a­tion of a Phase I/II clin­i­cal tri­al with ATIR201™ for tha­lassemia

Amsterdam, The Netherlands, December 15, 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 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.
Thalassemia is an inher­it­ed blood dis­or­der which results in abnor­mal hemo­glo­bin for­ma­tion, lead­ing to improp­er oxy­gen trans­port and destruc­tion of red blood cells. 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.

Manfred Rüdiger, PhD, Chief Executive Officer of Kiadis Pharma, com­ment­ed: “We are excit­ed to ini­ti­ate this new Phase I/II study with ATIR201™ for tha­lassemia patients on track as planned. It is well estab­lished that a stem-cell trans­plant, once engraft­ed, can be func­tion­al for life and so the aim of our approach is to not just ame­lio­rate symp­toms or reduce the need for trans­fu­sions, but to pro­vide patients with an endur­ing, life-long cure. Our approach could pro­vide an alter­na­tive to var­i­ous gene-ther­a­py-based strate­gies which, for exam­ple, may suf­fer from gene silenc­ing or sub­op­ti­mal expres­sion lev­els over time. We believe ATIR201™ has the poten­tial to make cura­tive HSCT a viable option to many more patients suf­fer­ing from inborn dis­or­ders of the blood like tha­lassemia.

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