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Last updated on April 18, 2014 at 1:21 EDT

Kiadis Pharma Completes Five-year Follow-up of its Phase I/II Clinical Study with Blood Cancer Product ATIR(TM) and Study Meets Primary Objective

September 11, 2013

AMSTERDAM, The Netherlands, September 11, 2013 /PRNewswire/ –

~ 67% survival demonstrated after 5 years and no transplant related

mortalities ~

Manfred Ruediger, CEO of Kiadis Pharma, gives more information on
ATIR(TM)and the five-year follow-up data in a video available on http://www.kiadis.com

Kiadis Pharma B.V., a clinical stage biopharmaceutical company developing treatments
for blood cancers, announced today that the five-year follow-up of patients with high-risk
malignancies from its Phase I/II clinical study confirms long-term safety and efficacy of
ATIR(TM) over a broad dose range. ATIR(TM) is a cell-based product designed to enable stem
cell transplantations from partially mismatched (haploidentical) family donors for
patients who do not have a standard of care stem cell donor available. The results
demonstrate proof of concept and show that ATIR(TM) infusion after a T-cell depleted
haploidentical hematopoietic stem cell transplantation (HSCT) provides immune protection
shortly after the transplantation and improves long-term outcome in high-risk patients
with very poor prognosis.

Not only does the study confirm that ATIR(TM) provides an effective treatment for
patients for whom a standard of care stem cell donor is not available, the long term
survival even seems to compare favorably to patients who do have a standard of care
matched unrelated donor available. The overall survival of patients with high-risk
malignancies in the Phase I/II study who received an efficacious dose of ATIR(TM) was 78%
and 67% after one and five years, respectively. Data from the Center for International
Blood & Marrow Transplant Research (CIMBTR) show that the one (and five) year survival of
patients with acute myeloid leukemia (AML) who do have a standard of care matched
unrelated donor available, varies from 65% (and 35%) in low-risk patients to 45% (and 20%)
in high-risk patients, respectively.

In this Phase I/II study, 19 high-risk leukemia patients were treated with escalating
doses of ATIR(TM) after a haploidentical HSCT. The five-year follow-up data show no
transplant related mortality in the nine patients who received an efficacious dose of
ATIR(TM). In addition, no Grade III-IV (life-threatening) acute Graft versus Host Disease
(GvHD) was observed at any dose, which again compares favorably to standard of care
matched unrelated donor transplantations, where (according to data from the CIBMTR)
incidence of life-threatening GvHD is 30%. The strong five-year survival data also suggest
that immune cells responsible for the Graft versus Leukemia effect are retained in
ATIR(TM).

The results of the study allowed selecting the optimal ATIR(TM) dose for further
development. Data from this five-year study will be published in due course in a
peer-reviewed medical journal.

An international multi-center Phase II study including patients with AML, acute
lymphoblastic leukemia (ALL) and myelodysplastic syndrome (MDS), to confirm and extend the
data from the Phase I/II study, is now ongoing with topline results of the first phase
expected in H1 2014.

Manfred Ruediger, PhD, Chief Executive Officer of Kiadis Pharma, commented: “The
results from this study, which spans five years, show that ATIR(TM) might change the way
in which patients with hematological malignancies will be treated. These data show that
ATIR(TM) not only prevents significant infections without eliciting life-threatening GvHD,
but also strongly improves survival rates in patients with high-risk malignancies and very
poor prognosis. Our currently ongoing international Phase II clinical study is designed to
confirm and extend these data to expedite moving ATIR(TM) towards regulatory approval.”

Dr. Denis-Claude Roy, Professor of Medicine at the University of Montreal and
principal investigator for the study, added: “We are very excited about the long-term
effects of ATIR(TM). In demonstrating very strong efficacy in minimizing
post-transplantation risks and improving overall survival, these long-term data represent
a potential major advancement in providing patients for whom a suitable matched donor is
not available, with the opportunity to receive an HSCT from a mismatched family member
with ATIR(TM) added as an adjunctive treatment.”

About ATIR(TM)

ATIR(TM) is a cell-based medicinal product enabling stem cell transplantations using
partially mismatched (haploidentical) family members as donors for patients suffering from
blood cancer who do not have a standard of care stem cell donor available. An HSCT is the
only potentially curative treatment for many patients but a matching donor is available
for only half of the patients in need. ATIR(TM) thus has the potential to address this
unmet need and to make the HSCT available for all patients worldwide.

Those T-cells in a haploidentical graft which would cause life-threatening GvHD are
selectively eliminated using proprietary technology to produce ATIR(TM). ATIR(TM) is
administered as an adjunctive treatment after a haploidentical HSCT facilitating early
immune reconstitution without causing life-threatening (acute) GvHD.

ATIR(TM) is currently in Phase II clinical development and has been granted Orphan
Drug Designation both in the EU and the USA. Together, both regions represent a combined
primary market potential of more than EUR 1 billion per year.

About Kiadis Pharma

Kiadis Pharma B.V. is a private, clinical stage biopharmaceutical company focused on
the development of innovative and potentially life-saving therapies for patients with late
stage blood cancers and related disorders, an area of significant unmet medical need.

Kiadis Pharma’s lead product is ATIR(TM), a cell-based product designed to enable stem
cell transplantations from partially mismatched (haploidentical) family donors. ATIR(TM)
is currently being studied in an international Phase II study. Kiadis Pharma recently
obtained a GMP manufacturing license and GMP certificate for its Quality Control
laboratory.

Kiadis Pharma is supported by a strong group of leading international investors
including LSP, Alta Partners, DFJ Esprit, Quest for Growth, MedSciences Capital and NOM.
Kiadis Pharma is based in Amsterdam, The Netherlands. Further information can be found at:

http://www.kiadis.com

        Company Contact:
        Manfred Ruediger, CEO
        Kiadis Pharma
        Entrada 231-234
        1096 EG AMSTERDAM
        The Netherlands
        Tel. +31-20-314-02-50
        communication@kiadis.com

        Media and Investor Contact:
        Mary-Jane Elliott, Amber Bielecka, Lindsey Neville
        Consilium Strategic Communications
        Tel: +44-(0)207-920-2354
        kiadis@consilium-comms.com

SOURCE Kiadis Pharma


Source: PR Newswire