VEN100: Antibiotic-Associated Diarrhea

Antibiotic Associated Diarrhea

Ventria Bioscience's lead therapeutic candidate is VEN100 containing recombinant human lactoferrin (rHLF). Human lactoferrin is a principal protein component of human breast milk and contributes to the establishment of the healthy infant digestive tract. Its functional properties have been studied extensively, and it is known to have prebiotic, anti-inflammatory, and toxin-binding activity; to promote maintenance of normal intestinal permeability and repair of damaged intestinal cells; and to improve iron utilization during the formation of red blood cells (hematopoiesis). Its numerous beneficial properties are believed to be responsible for breast milk's known anti-diarrheal properties.

Today, an affordable and high quality supply of human lactoferrin limits its use in human therapeutics. Ventria Bioscience has developed the ability to produce therapeutic quantities of recombinant human lactoferrin (rHLF) protein and is currently studying its utility as a novel, orally administered, preventive treatment for antibiotic-associated diarrhea (AAD) in at-risk adults who are hospitalized or who are residents of skilled nursing facilities. AAD, which is diarrhea associated with the use of broad-spectrum antibiotics, is a common, growing health problem that costs the U.S. healthcare system between $6 billion and $7.5 billion annually. AAD disproportionately affects vulnerable populations such as the elderly, hospitalized, and immune-compromised patients. A significant number of AAD cases are caused by the opportunistic bacterial pathogen Clostridium difficile, which is difficult and expensive to treat, and which substantially increases mortality in hospitalized patients.

There are no safe, effective, commercially available products to prevent the occurrence of AAD. Treatment options are limited to discontinuation of antibiotic therapy or administration of additional antibiotics—practices that can increase the severity of infection, lead to unfavorable patient outcomes, and contribute to development of antibiotic-resistant bacterial strains.

Ventria Bioscience is developing VEN100 as a preventive therapy, to be administered in conjunction with necessary antibiotic treatments, for patients at increased risk of developing AAD. A phase 2 clinical study conducted at Johns Hopkins University demonstrated that VEN100, when administered concurrently with antibiotics in a long-term care setting, reduced the incidence of AAD by approximately 50% with no observed adverse effects. Ventria Bioscience has worked with the FDA to design a phase 2b dose-ranging clinical study to confirm the previous results and evaluate the effective dose, and expects to initiate this study in 2014.

Ventria Bioscience manufactures VEN100 (rHLF) as a cGMP drug substance. A chemistry and manufacturing controls (CMC) review of the manufacturing process was successfully completed with FDA in 2013 and confirmed Ventria Bioscience’s plans for the manufacturing of drug substance and drug product under cGMP. Topics reviewed with FDA included genetic stability, master and working seed bank establishment, assay methods to be used for drug substance and drug product release, and product specifications including identity, purity, impurities, stability and potency.