Marburg, Germany, 11 September 2012
SB010 was safe and well-tolerated across all dose levels
Phase IIa proof-of-concept trial filed with regulatory agencies
sterna biologicals GmbH & Co. KG (“sterna biologicals”) announced today that SB010, a DNAyzme-based GATA-3 antagonist for the treatment of moderate to severe Th2-driven asthma, successfully completed its comprehensive Phase I development programme.
In three clinical trials encompassing a total of 108 healthy subjects and mild asthmatics, safety, tolerability, and pharmacokinetics were assessed. SB010 was safe and well-tolerated across all dose levels.
The three clinical trials were:
- NCT01470911, a single ascending dose study in 48 healthy subjects,
- NCT01554319, a multiple ascending dose study in 36 healthy subjects, and
- NCT01577953, a single ascending dose study in 24 mild asthmatics with airway hyperreactivity.
Overall, no severe adverse events occurred and adverse events were limited and fully resolved at the end of the study periods. A 60 day follow-up visit performed in each of the trials did not reveal any medically relevant changes. Pharmacokinetics demonstrated rapid appearance of SB010 in plasma and subsequent swift reduction in systemic availability with no accumulation effects. Laboratory parameters, ECG, and vital signs were within the reference ranges.
Dr. Joachim Bille, Managing Director, commented: “These excellent clinical results once again confirm the expected good safety profile of our DNAzyme-based drug candidates. The combination of local application, highly specific mechanism of action, and endogenous enzymatic activity make DNAzymes a promising therapeutic class.”
Sterna biologicals is also pleased to announce that it has filed a Phase IIa proof-of-concept trial with regulatory agencies. This trial will assess safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of SB010 in asthmatic patients. The trial will be conducted in seven study centres in Germany and is expected to complete in H1 2013.
Dr. Joachim Bille added: “We look forward to our upcoming proof-of-concept trial, which marks the first ever evaluation of inhaled DNAzymes for efficacy in patients.”
sterna biologicals’ drug candidate SB010 is an inhaled DNAzyme-based GATA-3 antagonist. GATA-3 is the master transcription factor in regulating Th2-driven inflammatory diseases such as 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 significantly reduced expression of these cytokines and was 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 (antisense), in case of SB010 GATA-3 mRNA. After binding to the target, the catalytic domain then cleaves the mRNA, thereby inhibiting relevant cytokine expression.
Asthma is a major chronic inflammatory disease of the airways affecting an estimated 300 million people worldwide. In OECD countries, prevalence is around 10% and increasing, with greater than average prevalence amongst women, children, and the elderly.