Novel-class therapeutic for rare disease
Inhaled murepavadin: First targeted antibiotic being developed for treating Pseudomonas aeruginosa infections in people with cystic fibrosis
Inhaled Murepavadin is a precision antibiotic specifically targeting Pseudomonas aeruginosa for treating infections in cystic fibrosis, non-CF bronchiectasis, and more. As one of the first candidates emerging from the novel Outer Membrane Protein Targeting Antibiotics (OMPTA) class, it is highly potent against Pseudomonas aeruginosa (including multidrug resistant strains). It represents an attractive product candidate, having:
- Attractive market potential: New inhaled CF antibiotics have quickly exceeded $200M in annual sales. nCFBE represents a >$1B and growing market.
- External validation & substantial funding from the EU Innovative Medicines Initiative (IMI) for P1a & US CF Foundation for P2
- A high safety margin: 290 subjects have dosed, low systemic exposure upon inhalation
- Strong IP protection: Market exclusivity through about 2036 via COM/additional IP
In preclinical studies, Murepavadin was highly effective against Pseudomonas aeruginosa, without evidence of generating resistance.
- Murepavadin exerts a very potent anti‑pseudomonal activity and shows a good lung tissue penetration
- Is highly specific against Pseudomonas aeruginosa, including MDR (multidrug resistant) strains (precision medicine)
- Targets the essential outer membrane (OM) β-barrel transporter protein LptD, which translocates LPS from the periplasm to the OM and is essential for outer membrane biogenesis
- Is highly active in vitro (MIC90≤ 0.25µg/ml; >1,500 strains tested worldwide)
- Retains potent activity on Pseudomonas aeruginosa CF clinical isolates (strains MIC90= 2µg/ml; >400 strains tested)
- Is highly effective in several animal infection models with high exposure in the epithelial lung fluid (ELF)
- Showed no cross-resistance with current standard-of-care antibiotics
- Inhaled Murepavadin is expected to complete its preclinical program in the near future