Stopgap measures like antibiotics, anti-inflammatory drugs, chest percussion, and osmotic agents can help mitigate symptoms of cystic fibrosis, while CFTR modulators can help treat the root cause for about 90% of people with cystic fibrosis. But people who make little-to-no CFTR protein can’t benefit from modulators and are still awaiting meaningful relief.
At cystetic Medicines, we’re driving a new approach. Here’s how it works:
About 10% of people with cystic fibrosis make little or no CFTR protein, a critical anion channel that allows bicarbonate and other anions to be transported from inside cells to outside. The loss of bicarbonate secretion leads to airway surface liquid that is more acidic, less hydrated, and has decreased antimicrobial activity.
An alternative path to anion secretion
cystetic Medicines’ approach leverages amphotericin B, a naturally occurring small molecule that independently forms ion channels in cells. It can thus serve as a molecular prosthetic. The drug replicates the function of the missing protein, restoring anion transport.
cystetic Medicines’ approach further optimizes the behavior of the molecular prosthetic with state-of-the-art delivery technologies. Through established particle engineering methods, the aerodynamics of the drug are changed, allowing it to flow effectively into the lungs.
Even the most effective life-saving drug is useless if it can’t get to where it needs to be.
The result? A dry powder formulation that can be targeted with a portable inhaler to where it is needed in the bronchial airways.
At cystetic Medicines, we are laser-focused on making life-saving CF care accessible for the final 10%, as soon and as safely as possible.
Bringing hope to fruition
Having demonstrated the promise of the molecular prosthetics approach in a range of nonclinical studies and in a proof-of-concept clinical study in the noses of people with cystic fibrosis not on modulators, we have initiated phase 1 clinical studies supported by DevPro BioPharma with a new dry powder inhaler product, with phase 2 trials to begin as soon as possible thereafter.
As the background dry powder technologies have already been used in existing cystic fibrosis treatments, cystetic Medicines is already capable of producing the treatment at commercial scale. Upon approval, we will be able to produce as fast as the market demands.