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EANS-News: DEWB Investment Holding NOXXON Initiates Phase IIa of Anti-Hepcidin Spiegelmer® NOX-H94

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Subtitle: Anemia of Chronic Disease Study is Fourth Phase II Initiated by NOXXON
in 2012

Company Information

Berlin, 04 December 2012 (euro adhoc) - NOXXON Pharma today announced the
treatment of the first patients in a Phase IIa clinical trial of its
anti-hepcidin Spiegelmer® NOX-H94 to treat anemia associated with chronic
disease. This Phase IIa study was initiated following the successful completion
of the clinical Phase I program, data from which will be presented at the
upcoming ASH (American Society of Hematology) meeting in Atlanta, Georgia, 8-11
Dec 2012. The Phase I program consisted of a comprehensive single and multiple
ascending dose study in healthy volunteers and a subsequent human
pharmacodynamic study to assess the ability of NOX-H94 to prevent
endotoxin-induced hypoferremia in healthy subjects. This endotoxemia study
delivered the first clinical evidence that NOX-H94 is capable of neutralizing
high levels of hepcidin in humans and maintaining higher serum iron
concentrations relative to subjects receiving placebo. 

NOX-H94 is the third Spiegelmer® to enter Phase II studies and this study is the
fourth Phase IIa trial that NOXXON has started this year. The other Phase IIa
studies initiated in 2012 include the NOX-E36 Phase IIa for the treatment of
diabetic nephropathy, the NOX-A12 Phase IIa for the treatment of Chronic
Lymphocytic Leukemia, and the NOX-A12 Phase IIa for the treatment of Multiple
Myeloma.

Excessive concentrations of the peptide hormone hepcidin, which is also called
the master regulator of iron homeostasis, occur in some chronic diseases such as
cancer, renal disease, or inflammatory diseases. These high hepcidin levels lead
to iron restriction, also known as functional iron deficiency: a condition in
which iron is blocked inside its cellular stores and is thus unavailable for
hemoglobin synthesis. This condition, over time, results in anemia of chronic
disease. NOX-H94 inhibits this pathological mechanism by binding and
inactivating hepcidin. 

The NOX-H94 Phase IIa study is being conducted to investigate the hypothesis
that inhibition of hepcidin can raise hemoglobin levels in patients with anemia
of chronic disease. The four-week repeated-dose multi-center study will be
conducted in Europe in anemic patients with cancer. An open-label pilot phase
will be followed by a 3-arm randomized, double-blind, placebo-controlled main
phase comparing two different dose-regimens of NOX-H94 with placebo. 

Hepcidin inhibitors such as NOX-H94 offer the potential to provide a targeted
treatment alternative for anemia of chronic disease and to avoid some of the
disadvantages of the existing unspecific therapies which are often given at
supra-physiological doses: erythropoiesis-stimulating agents (ESAs), i.v. iron,
and blood transfusions:
 
- the potential risks of ESAs in the treatment of patients with cancer and
chronic kidney disease are documented in the black box warning required by the
US FDA and include increased risk of tumor progression or recurrence;
 
- use of i.v. iron has increased in response to concerns with ESAs; but this
therapy is limited by the potential occurrence of iron overload, in addition
administration of i.v. iron leads to a counter-productive increase in hepcidin;

- blood transfusions also add iron to the body and in addition bring the risks
of transmissible diseases and immunosuppression.

NOX-H94 is the first hepcidin inhibitor to reach Phase II.

Based on information from the GLOBOCAN database and scientific publications on
rates and types of anemia in cancer and chronic kidney disease (CKD) patients,
NOXXON estimates that there are approximately 230,000 cancer patients and 3.6
million CKD patients requiring treatment for anemia of chronic disease every
year that could potentially benefit from a hepcidin inhibitor in the combined
markets of the EU-5 (France, Germany, Italy, Spain and the United Kingdom),
Japan and the United States.

About NOXXON Pharma AG 
NOXXON Pharma is a biopharmaceutical company pioneering the development of a new
class of proprietary therapeutics called Spiegelmers. Spiegelmers are the
chemically synthesized, non-immunogenic alternative to antibodies. NOXXON has a
diversified portfolio of clinical-stage Spiegelmer® therapeutics:

- NOX-E36, an anti-CCL2/MCP-1 (C-C chemokine ligand 2 / Monocyte Chemoattractant
Protein 1) Spiegelmer®, is currently in a Phase IIa study in patients with type
2 diabetes with albuminuria. CCL2 is a pro-inflammatory chemokine involved in
the recruitment of immune cells to inflamed tissues.

- NOX-A12, an anti-CXCL12/SDF-1 (CXC chemokine ligand 12 / Stromal Cell-Derived
Factor 1) Spiegelmer®, is currently in Phase IIa studies in two hematological
cancers, multiple myeloma (MM) and chronic lymphocytic leukemia (CLL). CXCL12 is
a chemokine mediator of tumor invasion, metastasis, and resistance to therapy.

- NOX-H94, an anti-hepcidin Spiegelmer®, is currently in a Phase IIa study in
cancer patients with anemia. Hepcidin is the key regulator of iron metabolism
and responsible for the iron restriction leading to anemia of chronic disease.

The Spiegelmer® platform provides the company with powerful and unique discovery
capabilities, which have generated a number of additional leads under
preclinical investigation. Located in Berlin, Germany, NOXXON is a well-financed
mature biotech company with a strong syndicate of international investors, and
approx. 60 employees. For more information, please visit: www.noxxon.com

About NOX-H94 & Anemia of Chronic Disease
NOX-H94 is a Spiegelmer® compound targeting the iron-regulating protein
hepcidin. Hepcidin is the master regulator of iron homeostasis via its effect on
ferroportin, the only known iron export protein. Cytokine-induced synthesis of
hepcidin plays a crucial role in macrophage iron retention, which underlies the
anemia of chronic disease by limiting the availability of iron for erythroid
progenitor cells. Patients with anemia of chronic disease display an impaired
response to erythropoietin (EPO). The NOX H94 compound is a 44-nucleotide L-RNA
oligonucleotide linked to 40 kDa PEG. Preclinical studies have demonstrated that
this compound inhibits IL-6 induced anemia in monkeys and has similar
pharmacokinetics to other Spiegelmer® compounds. The compound can be
administered intravenously or subcutaneously.

NOXXON received grant support within the program KMU-innovativ from the German
Federal Ministry of Education and Research (BMBF) for the preclinical and early
clinical development of NOX-H94.  

Further information about the ongoing NOX-H94 Phase IIa clinical trial is
available at ClinicalTrials.gov: ID: NCT01691040.

Contact:

NOXXON Pharma AG       
Emmanuelle Delabre 
T: +49-30-726247-100  
edelabre@noxxon.com       

College Hill Life Sciences
Melanie Toyne Sewell, Cora Kaiser
T: +49-89-57001806 
noxxon@collegehill.com

About DEWB 
Deutsche Effecten- und Wechsel-Beteiligungsgesellschaft AG (DEWB AG, ISIN:
DE0008041005) is a listed private equity house that specialises in young and
established medium-sized companies. The investment focus is on strong growth
companies from the areas of photonics and sensor systems as well as their fields
of application in automation and industrial engineering and related areas for
which DEWB provides support through shareholders' equity, expertise in corporate
development and its sector network. Since 1997 DEWB has invested more than 360
million Euros in 55 companies and realized more than 465 million Euros through
42 exits, eight of which were in the form of IPOs. The company is located in
Jena, one of the most successful technology and science regions in Germany, with
a long tradition in the field of optical technologies and one of the most
important European centres for photonics.


Further inquiry note:
Marco Scheidler
Tel.: +49 (0) 3641 3100030
E-Mail:  marco.scheidler@dewb.de

end of announcement                               euro adhoc 
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company:     Deutsche Effecten- und Wechsel-Beteiligungsges. AG
             Fraunhoferstraße 1
             D-07743 Jena
phone:       +49 (0)3641 3100030
FAX:         +49 (0)3641 3100040
mail:         ir@dewb.de
WWW:         http://www.dewb-vc.com
sector:      Financial & Business Services
ISIN:        DE0008041005
indexes:     
stockmarkets: free trade: Berlin, München, Stuttgart, Open Market / Entry
             Standard: Frankfurt 
language:   English

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