Safety, Efficacy, PK, and PD Characteristics of Orally Inhaled SB010 in Male Patients With Mild Asthma (Multiple Dose)
Status:
Completed
Trial end date:
2013-12-01
Target enrollment:
Participant gender:
Summary
Asthma is a chronic inflammatory bronchial disorder with three distinct components: airway
hyper-responsiveness (respiratory hypersensitivity), airway inflammation, and intermittent
airway obstruction. One of the characteristics of the disease is an inflammatory reaction of
the immune system caused by cytokine production. A substantial number of asthma patients do
not satisfactorily respond to steroid therapy and consequently have an unmet medical need for
novel targeted therapies with improved specificity, tolerability, and compliance.
Novel therapeutic strategies for the treatment of chronic inflammatory diseases by targeting
early disease-causing mechanisms are a promising approach for the treatment of asthma. The
transcription factor GATA-3 plays a key role in mediating the asthmatic immune response and
has been shown to be necessary and sufficient for the production of cytokines interleukin
(IL)-4, IL-5, and IL-13. The active drug substance of the investigational medicinal product
SB010 is hgd40. SB010 belongs to a new class of antisense oligonucleotide therapeutics, the
10-23 DNA (deoxyribonucleic acid)zymes (antisense oligonucleotide).
DNAzymes are catalytically active nucleic acids that cleave complementary RNA (ribonucleic
acid) molecules. By cleaving GATA-3 mRNA, hgd40 reduces specific cytokine production and
thereby reduces key features of allergic airway inflammation. DNAzymes are generated
completely by chemical synthesis and can be produced under Good Manufacturing Practice (GMP)
controlled conditions. The DNAzymes are not biological drugs, i.e. they are not generated by
use of any living organism including cell culture or bacteria. The molecules are highly
water-soluble and will be applied as solution directly in their synthesized form.
This proof-of-concept study will evaluate the safety, tolerability, efficacy,
pharmacokinetics (PK), and pharmacodynamics (PD) of multiple doses of inhaled SB010 in male
patients with mild asthma.