$54 million dependent upon the achievement of clinical and regulatory targets, plus up to 11% royalty on future sales of the combination of rHuA1AT with rHuPH20. The license provides Intrexon Corporation, a next generation synthetic biology company, with exclusivity to alpha 1-antitrypsin, for the indications resulting from A1AT deficiency. Additional terms of the transaction have not been disclosed.
Alpha 1-antitrypsin is a protease inhibitor that provides a protective effect from inflammatory cell proteases, especially neutrophil elastase. Intrexon is using its synthetic DNA platform for high-level expression of recombinant A1AT for the potential treatment of diseases resulting from genetic alpha 1-antitrypsin deficiency such as genetic emphysema. A1AT may also benefit patients with chronic obstructive pulmonary disease (COPD) and cystic fibrosis. Currently there is no A1AT recombinant protein available and, as a result, treatment for deficiency is limited and expensive. Intrexon will fund all development and commercialization expenses for the program, which is currently in the scale-up phase of process development.