DiscoveryBioMed Is Developing A Novel “Dual Action” Small Molecule Monotherapy For All Cystic Fibrosis (CF) Patients

ST. LOUIS--(BUSINESS WIRE)--DiscoveryBioMed, Inc. (DBM, Inc.) has discovered and is developing a novel class of small molecule monotherapy that affects both major drug targets that are abnormal and underlie the pathophysiological features of CF. Proper function of the two target proteins that serve as transporters for salt and water into the airways is critical for hydration of the lung and airways and clearance of mucus and harmful inhaled materials. CFTR (the protein product of the CF gene, a chloride ion channel) and ENaC (the epithelial sodium ion channel) become very abnormal in this devastating disease of children and young adults. The loss of CFTR function and over-activity of ENaC in CF literally drains salt and water from the airway surface. Mucus becomes sticky and infected on the airway surface in CF, and the lung disease in affected patients becomes progressively worse.

“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.”

“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,” explained Dr. Erik Schwiebert, DBM’s CEO and Chief Scientific Officer. “This is the only class of compounds that we are aware of that affects both CFTR and ENaC as a single, small molecule drug.” This exciting and unique profile residing within a single molecular entity has the potential to be a breakthrough medicine, providing superior medical benefit than current treatments in most, if not all, CF patients. DBM was systematic in its examination of this ‘dual action’ and received confirmation of this unique profile from independent labs.

DBM has recently submitted a new Fast Track SBIR application to be supported by the NHLBI, if funded, to select a clinical development compound and initiate preclinical IND-enabling studies. This drug series also potentiates and activates wild-type CFTR, and therefore, may also help patients with chronic obstructive pulmonary disease (COPD) where CFTR is reduced in expression and function. This drug may also have utility in asthma, lung edema and pulmonary hypertension because of its novel inhibitory effects on ENaC. “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,” declared Dr. Schwiebert. “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.” Dr. Schwiebert has worked on CF for over 20 years and tested and optimized DBM’s Humanized Drug Discovery approach with this CF Drug Discovery program. “We continue on this mission and are seeking a larger partner to help us finish it.”

About Cystic Fibrosis (CF)

CF is a debilitating disease of children and young adults that affects primarily the respiratory and gastrointestinal tracts. It is an inherited genetic disorder that affects more than 30,000 patients in the US and more than 70,000 patients worldwide. While advances in genetic testing and new therapies have increased life expectancy to the third decade of life, there are few drugs that affect directly the root causes of the disease and provide benefit in the majority of CF patients, and no current treatment that provides disease-modifying benefit. Pancreatic insufficiency leads to poor digestion and nutrition, causing a failure to thrive. Recurrent infections of the nasal passages, airways and lungs of CF patients become progressively worse and difficult to treat. These abnormalities are caused by the abnormal function of two different salt channel proteins (CFTR and ENaC) in surface cells of the lungs and gut called epithelial cells. There is also hyperactive innate immunity and a loss of acquired immunity in CF. There remains a massive unmet need for the majority of CF patients for medicines to slow or reverse the progression of CF disease and all of its abnormal facets.

About DiscoveryBioMed, Inc.

This program is another example of DBM’s novel approach to drug discovery, namely Humanized Drug Discovery. This program used human CF airway epithelial cell platforms to discover and validate this novel ‘dual action’ CFTR and ENaC drug class. DBM is utilizing human cell platforms for all of its R&D programs in Oncologic, Renal, Respiratory and Metabolic areas. DBM’s complimentary CRO services business establishes human cell platforms for clients and it also uses such platforms to profile lead assets for clients. See the DBM website, www.discoverybiomed.com, for more information about the company, and its R&D programs, drug discovery and scientific strategies and technologies.

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