DiscoveryBioMed Developing A New Small Molecule Monotherapy For Cystic Fibrosis Patients

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by Isaura Santos |

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DiscoveryBioMed, Inc. (DMB) has discovered and is currently developing a new class of small molecule monotherapy that utilizes the pathophysiological features of cystic fibrosis and works on two drug targets.

In Cystic Fibrosis, patients’ production of the CFTR protein and ENaC ion channel is abnormal, leading to a dysfunctional CFTR activity and over-activity of ENaC. The result is the loss of both salt and water from a patient’s airway surface in the lung, which is what leads to the production of thick, sticky mucus that can become easily infected.

What makes DMB’s experimental therapy unique is that it features a dual-action mechanism that improves CFTR production and decreases ENaC activity. “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. This is the only class of compounds that we are aware of that affects both CFTR and ENaC as a single, small molecule drug,” said Erik Schwiebert, CEO of DBM and its Chief Scientific Officer. This new, experimental therapy has the potential to become a breakthrough medicine and provide a much more efficient treatment option for cystic fibrosis patients.

The company recently submitted a new Fast Track application to be supported by the NHLBI. If the NHLBI chooses to fund the development of the drug, it will be selected as a clinical development compound and tested in pre-clinical studies. In addition to CF, the drug might be helpful for patients suffering from chronic obstructive pulmonary disease (COPD), asthma, pulmonary hypertension and lung edema.

“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. 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,” noted Dr. Schwiebert.

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“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. We continue on this mission and are seeking a larger partner to help us finish it,” Schwiebert concluded.

In other developments in cystic fibrosis, AffloVest, a wearable, self-contained oscillation vest device from International Biophysics that helps patients with Cystic Fibrosis breathe easier, was previously backed by numerous patient testimonies and is now proven in a new Clinical Study that it increased forced expiratory volume in one second (FEV1) an average of 11.5%. Five adolescent patients, all of whom had cystic fibrosis, used the AffloVest for three to five months each and saw an average FEV1 flow increase of 312 cc’s.