The Synspira Therapeutics pipeline of orphan drugs is focused on overcoming persistent clinical challenges in rare diseases, with an initial focus on cystic fibrosis (CF).
Our lead compounds are first-in-class products with broad clinical utility that address key clinical challenges across a range of therapeutic areas.
SNSP113: A new class of inhaled non-absorbed glycopolymer therapeutics
SNSP113 (poly [acetyl, arginyl] glucosamine) is a novel glycopolymer therapeutic with a specific polydispersity and microheterogeneity that allows the molecule to exhibit a number of important biological and pharmacological activities. SNSP113 has the potential to treat the “Vicious Cycle” of progressive pulmonary disease and bronchiectasis by effectively targeting each of the key structural and functional challenges.
SNSP113 is designed to offer rapid improvement for acute recalcitrant life-threatening pulmonary infections and also as a chronic treatment for progressive pulmonary disease to manage pulmonary exacerbations, pulmonary function and mucus clearance.
SNSP113 Mechanism of Action
- Improving mucus structure and airway clearance: SNSP113 has been shown to reduce mucin viscosity and to normalize mucus structure and airway hydration correcting ciliary beat frequency as well as improving mucociliary and airway clearance. Increased mucus viscosity and delayed mucociliary clearance are linked to progressive pulmonary decline.
- Life-threatening recalcitrant pulmonary infections: SNSP113 has been shown in vitro to disrupt the bacterial cell wall of multiple bacterial species [(Pseudomonas aeruginosa, non-tuberculosis mycobacteria (NTM), Burkholderia cepacia complex, methicillin resistant Staphylococcus aureus (MRSA)] to increase membrane permeability, reduce bacteria viability and act synergistically with previously resistant antibiotics. In addition, SNSP113 has also been shown to disrupt the cohesion of the bacterial biofilm layer and eradicate recalcitrant persister cells protecting from re-infections and antibiotic resistance.
- Inflammation: SNSP113 has the potential to reduce the neutrophil cascade which degrades the healthy collagen matrix that provides the flexibility for lungs to expand and contract leading to functional impairment and scarring.
SNSP003: The evolution of oral enzyme replacement
SNSP003 is a non-porcine enzyme replacement therapy (ERT) designed to treat Malabsorption Syndrome and EPI with the opportunity to be the first significant advancement in 40 years. SNSP003 has the potential to be the only broad-spectrum non-porcine product that impacts all key macronutrients, delivering highly pure enzymes to hydrolyze fats to monoglycerides and free fatty acids, proteins into peptides and amino acids, and starches into dextrins and short chain sugars.
SNSP003 is expected to dramatically improve dosing convenience with reduced pill burden and a formulation compatible for pediatric and adult patients unable to swallow capsules.
Synspira brings an unmatched expertise in the development and delivery of oral enzyme therapies as well as the treatment of Malabsorption Syndromes.