German Center for Lung Research (DZL) successfully completes investigator-initiated study evaluating SB010 in COPD patients

Marburg, Germany, 11 September 2017

  • Results presented today at the European Respiratory Society International Congress 2017 in Milan, Italy

sterna biologicals GmbH & Co. KG ("sterna biologicals") announced today that investigator Dr Timm Greulich from the Department of Medicine, Pulmonary and Critical Care Medicine, Philipps University Marburg, will present key results of the recently completed Phase II clinical trial of SB010 at the European Respiratory Society (ERS) Congress held in Milan, Italy, 9 to 13 September 2017.

The German Center for Lung Research (DZL) conducted the investigator-initiated trial (EudraCT 2013-002332-24) using sterna biologicals' drug candidate SB010 and coordinated by the Coordinating Centre for Clinical Trials of the Philipps University Marburg (KKS).

SB010 is an inhaled first-in-class GATA-3 antagonist that demonstrated strong efficacy and safety in a previous Phase IIa clinical trial in asthmatic patients (Krug et al, 2015 and Krug et al, 2017).

In this study, a total of 23 patients with moderate to severe chronic obstructive pulmonary disease (COPD) characterised by airway eosinophilia and using stable maintenance therapy, including inhaled corticosteroids, were randomised across three DZL study sites (Pulmonary Research Institute (PRI), Grosshansdorf; Fraunhofer Institute for Toxicology and Experimental Medicine (ITEM), Hannover; Universities of Giessen and Marburg Lung Center (UGMLC), Marburg) into parallel groups receiving SB010 (N=12) or placebo (N=11) twice-daily for 28 treatment days. The study met its primary endpoint, confirming that, as expected, between 10 and 25% of COPD patients have elevated eosinophils in the airways (in the study 26 of 130 screened patients, 20%).

Key secondary endpoints were also met, including a reduction in the relative sputum eosinophil count after SB010 treatment by 59% (10.7% to 4.4%, p=0.004) compared to 11% (7.2% to 6.4%, p=n.s.) in placebo-treated patients. Changes in eosinophils were accompanied by significant increase in blood interferon-gamma (p=0.02) and a trend to lower interleukin-5 levels. SB010 was safe and well-tolerated with no noteworthy differences in safety events between treatment groups.

Jonas Renz, Managing Director, commented: "We are extremely pleased by the pharmacological read-outs of this small study. In particular, the study confirmed for the first time that SB010 meaningfully reduces sputum eosinophils, a key marker of inflammation, on top of inhaled corticosteroid therapy and independent of an allergen-challenge protocol, in moderate to severe patients. These insights will inform study design for Phase IIb development."
The presentation by Dr Timm Greulich entitled "A GATA3-specific DNAzyme Attenuates Sputum Eosinophilia in Eosinophilic COPD patients" will take place today in session 348 (Update and New Perspectives in Airway Diseases), Room Red (North).

References
Krug N, Hohlfeld J, Kirsten A, Kornmann O, Beeh K, Kappeler D, Korn S, Ignatenko S, Timmer W, Rogon C, Zeitvogel J, Nan Z, Bille J, Homburg U, Turowska A, Bachert C, Werfel T, Buhl R, Renz J, Garn H, Renz H. "Allergen-Induced Asthmatic Responses Modified by a GATA-3 Specific DNAzyme." NEJM. 2015.

Krug N, Hohlfeld J, Buhl R, Renz J, Garn H, Renz H. "Blood Eosinophils Predict Therapeutic Effects of a GATA3-specific DNAzyme in Asthma Patients." Journal of Allergy and Clinical Immunology. 2017.

 
About SB012
sterna biologicals' drug candidate SB012 is an enema-applied DNAzyme-based GATA-3 antagonist for the treatment of ulcerative colitis. The GATA-3-specific DNAzyme is also contained in the drug candidate SB010 for the treatment of Th2-driven respiratory diseases and SB011 for the treatment of atopic dermatitis. GATA-3 is the master transcription factor in regulating inflammatory diseases associated with the Th2 pathway such as ulcerative colitis, allergies and allergic asthma. It is generally accepted that GATA-3 is necessary and sufficient for the production of key cytokines interleukin (IL)-4, IL- 5, and IL-13, which cause inflammation. In pre-clinical development, SB010 and SB012 significantly reduced expression of these cytokines and were safe and well-tolerated in toxicological studies with negligible side-effects. DNAzymes are single-stranded DNA molecules comprising a catalytic domain flanked by two binding domains. The binding domains attach to a specific sequence of targeted mRNA, in case of SB012 GATA-3 mRNA. After binding to the target, the catalytic domain then cleaves the mRNA, thereby inhibiting relevant cytokine expression.

About ulcerative colitis
Ulcerative colitis is characterised by a continuous mucosal inflammation which predominantly affects the large intestine. Clinical symptoms include persistent diarrhoea with severe faecal urgency and often incontinence, rectal bleeding, abdominal cramping, weight loss, and general malaise. It is estimated that the disease affects approximately 2 million people in the US, Europe, and Japan.