Two Posters and Two Oral Presentations on NOXXON Compounds at 2012 American Society of Hematology (ASH) Conference

Two Posters and Two Oral Presentations on NOXXON Compounds at 2012 American Society of Hematology (ASH) Conference

NOXXON Pharma AGEmmanuelle DelabreT: +49-30-726247-0orCollege Hill Life SciencesMelanie Toyne Sewell, Cora KaiserT: +49-89-57001806

NOXXON Pharma announced its participation at the 54 annual meeting of the American Society of Hematology (ASH) in Atlanta, USA from 8-11 December 2012.

Two posters will be presented based on the outcome of two independent Phase I clinical trials which assessed the safety of Spiegelmer NOX-H94 in a first-in-human study as well as its potential to prevent an inflammation induced drop in serum iron in a human experimental model of endotoxemia. NOX-H94 is a candidate for the treatment of anemia of chronic disease.

There will also be two oral presentations on the mode of action of the Spiegelmer NOX-A12 in two hematological cancers, chronic lymphocytic leukemia (CLL) and multiple myeloma (MM), which were studied in novel experimental settings.

The titles and contributors for the two NOX-H94 poster presentations at ASH are as follows:

Members of NOXXON’s drug development team and collaboration partners will be at the ASH conference to explain the mode of action and clinical potential of these innovative drug candidates.

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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:

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:

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 that is located on iron-storing cells, such as macrophages. Inflammation-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 polyethylene glycol (PEG). Preclinical studies have demonstrated that this compound inhibits IL-6 induced anemia in monkeys and has similar pharmacokinetics to other Spieglemer compounds.

Phase I clinical studies demonstrated that treatment with NOX-H94 was generally safe and well tolerated and that NOX-H94 could 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.

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: .

NOX-A12 specifically binds and neutralizes CXCL12/SDF-1 (CXC chemokine ligand 12 / Stromal Cell-Derived Factor-1), a chemokine which activates and attracts immune and non-immune cells including stem cells from the bone marrow. CXCL12 binds with high affinity to two chemokine receptors, CXCR4 and CXCR7. The CXCL12 / CXCR4 / CXCR7 axis has been shown to play a role in stem cell mobilization, vasculogenesis, tumor growth and metastasis. Inhibition of CXCL12 binding to its receptors sensitizes tumor cells to chemotherapy and in some solid tumors, prevents invasion and metastasis, suggesting that NOX-A12 in combination with therapy could be beneficial in the treatment of various cancers.

NOX-A12 has shown promising activity in models of both hematological and solid tumors in addition to models of stem cell mobilization. NOXXON’s collaborators have shown that in multiple myeloma models NOX-A12 detached myeloma cells from stromal cells and sensitized them to killing by Velcade/bortezomib both and . NOX-A12 has also been shown to inhibit chemotaxis of patient-derived primary CLL cells towards higher concentrations of CXCL12 and to have distinct properties from CXCR4 antagonists. In an animal model of glioblastoma, NOX-A12 treatment resulted in a significant extension of lifespan of animals when used in combination with radiation therapy.

In Phase I studies with healthy volunteers, single doses of NOX-A12 up to 10.8 mg/kg and daily doses up to 2 mg/kg for five days were found to be safe and well tolerated and resulted in dose-dependent mobilization of white blood cells and CD34 hematopoietic stem cells as predicted by preclinical studies.

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-A12.

Further information about the ongoing NOX-A12 Phase IIa clinical trials is available at ClinicalTrials.gov: relapsed MM (ID: NCT01521533) and relapsed CLL (ID: NCT01486797).

Roccaro AM (2011) ASH 53 Annual Meeting, oral presentation 887, Session 652

Hoellenriegel J (2011) ASH 53 Annual Meeting, poster 3878, Session 652

C. Liu (2012) AACR, Apr 3rd 2012 (abstract # 4382)

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