OUR MISSION
SarcoMed USA was formed in 2017 to investigate if a novel DNase 1 compound (SM001) could influence the chronic pulmonary inflammation seen in Pulmonary Sarcoidosis patients.
Sarcoidosis is a systemic granulomatous disease characterized by a T-helper-1 response with accumulation of CD4+ lymphocytes and activated macrophages in the lungs and affected organs, resulting in characteristic granuloma formation. The lungs are involved in more than 90% of patients with sarcoidosis, thus abnormal pulmonary function is characteristic for many patients. Sarcoidosis affects approximately 200,000 people in the United States and at present corticosteroids are the only approved treatments.
A reduction in the use of steroids is a significant unmet need for sarcoidosis patients
OUR SCIENCE
The presence of microbial DNA (mDNA) from mycobacteria, propionibacteria and borrelia have been identified in sarcoidosis tissues suggesting their role in sarcoidosis granuloma formation
- Bacterial amyloid curli act as a carrier for such DNA to elicit an autoimmune response
- Serum amyloid A (SAA) derived from the host may participate in the granuloma formation by regulating inflammation through Toll-like receptor-2 (TLR2)
- SAA localized to macrophages and giant cells within sarcoidosis granulomas correlate with CD3(+) lymphocytes, linking expression to local Th1 responses
- Additional antigens implicated in initiating the inflammatory cascade leading to granuloma formation include beta-actin, intermediate filament vimentin and pathogen-associated molecular patterns (PAMPs)
The DNase activity of SM001 is expected to degrade the microbial DNA within the protein/DNA complex present in lungs of patients with pulmonary sarcoidosis (extracellular red X marks). The reduction of the burden of bacterial DNA could then reduce or eliminate the antigenic stimulation responsible for the persistent immunological stimulation causing granuloma formation (See intracellular orange X marks).
DEVELOPMENT
Lead product candidate, SM001, is a recombinant form of human deoxyribonuclease I (DNase I), with a proven mechanism of action and a differentiated clinical profile.
Regulatory Pathway
Clear development and regulatory pathway along with FDA Orphan status designation granted for the treatment of sarcoidosis.
Clinical Development
Phase 1 healthy subject clinical study completed (SAD, MAD with 18 subjects). SarcoMed is planning to initiate a Phase 1b Randomized, Double-Blind, Placebo-Controlled Multiple Ascending Dose Study of Inhalation SM001 in Patients with Pulmonary Sarcoidosis.
CONTACT US
Please contact us should you have an interest in learning more about our efforts.