Shire, headquartered in the UK, has acquired the rights to AT-1001 outside of the US and Japan and will jointly fund development of the zonulin inhibitor after it has completed two Phase II trials for Celiac disease. At the turn of the millennium, a group of scientists including Dr Alessio Fasano at the University of Maryland were attempting to characterise the zonula occludens toxin secreted by the cholera bacteria Vibrio cholerae. They discovered that the toxin targets a specific signalling protein, which in turn regulates how permeable the so-called 'tight junctions' between epithelial and endothelial cells are. That protein target came to be called zonulin and was soon implicated in a variety of diseases where leakage occurs as molecules are moved between body compartments. These included several autoimmune conditions such as Type I diabetes, multiple sclerosis (MS) and, of course, celiac disease. Dr Fasano went on to co-found Alba - currently still the only company targeting zonulin - and just seven years later, the fruits of his research have led to this deal. Celiac disease is a T-cell mediated auto-immune disease that occurs in genetically susceptible individuals and is characterized by small intestinal inflammation triggered by gluten. There are no currently marketed pharmaceutical products for this disorder and the current treatment for Celiac is complete elimination of gluten from the diet. Alba will receive an initial licensing payment of $25m, followed by over $ 80m if certain clinical, regulatory and launch milestones are met for certain GI indications. Should the drug be used for other diseases, Shire will pay over $40m extra per new indication. As is typical with these deals, the bulk of the money is tied up in sales-based milestones ($220m), as well as tiered royalties. Dr Blake Paterson, Alba's CEO said: "The combination of Alba's barrier function technology and autoimmune development capabilities with Shire's proven track record in GI [gastrointestinal] drug development and commercialisation will greatly enhance our efforts to bring these novel therapies to patients." Shire already has a number of mesalamine-related gastrointestinal drugs, including Lialda/Mezavant and Pentasa.
US biopharma firm Alba Therapeutics has received a boost to its coffers after Shire agreed to pay up to $325m (€226m) to help bring its lead gastrointestinal drug candidate to market.