DiscoveryBioMed, Inc. has announced the discovery of a novel, small molecule class of monotherapy that is already in the development process and may be able to impact the two major drug targets associated with the dysfunction and underlying pathophysiology of cystic fibrosis (CF). The normal functioning of target proteins is critical for proper salt and water transportation into the airways, facilitating hydration and mucus clearance.
The CFTR chloride ion channel, a protein product of the CF gene, and the epithelial sodium ion channel (ENaC) are affected by the life-threatening disease, making them function abnormally. Both the loss of CFTR and the over-activity of ENaC that affects CF patients cause the draining of salt and water from the patients’ airways, producing sticky mucus that leads to infected airways.
“Our single drug corrects and activates under basal conditions the most common mutant CFTR that is lost in CF. The same single drug inhibits the function of ENaC that is too active in CF,” stated Erik Schwiebert, the CEO and chief scientific officer at DiscoveryBioMed, in a press release. “This is the only class of compounds that we are aware of that affects both CFTR and ENaC as a single, small molecule drug.”
The company believes that their unique single molecular entity has the ability to provide breakthrough medical advancements, as well as improve the lives of patients suffering from the disease. DiscoveryBioMed’s optimism is based on exhaustive studies that have confirmed the dual action of the monotherapy, having received positive results from various independent labs.
“The fact that we have a single drug with dual effects on CFTR and ENaC and are focusing initially on inhaled delivery are two differentiating factors between our program and all other competitors in CFTR- and ENaC-specific medicines arena,” continued the investigator. “The competition is exploring combination medicines which is much more challenging to develop than the monotherapy strategy that DBM is able to conduct with this dual action drug series.”
“We intend to cover new ground with this novel, dual action drug series, and hope to provide the superior medical benefit to CF patients that they critically need,” added Schwiebert, who dedicated more than 20 years of his life to research on CF and was responsible for testing and optimizing DBM’s Humanized Drug Discovery approach as part of their CF Drug Discovery program. “We continue on this mission and are seeking a larger partner to help us finish it.”
The company has also submitted a new Fast Track SBIR application, seeking the support from the NHLBI, which may help them select a clinical development compound and initiate preclinical IND-enabling studies. In addition to CF, the drug series may also help patients with chronic obstructive pulmonary disease (COPD), asthma, lung edema and pulmonary hypertension.