Intrexon, Halozyme partner to develop subcutaneous alpha 1-antitrypsin for A1AT deficiency

Published on June 7, 2011 at 6:11 AM · No Comments
Halozyme Therapeutics, Inc. (Nasdaq: HALO) and Intrexon Corporation, today announced the signing of a worldwide exclusive licensing agreement for the use of rHuPH20 (recombinant human hyaluronidase) in the development of a subcutaneous (under the skin) injectable formulation of Intrexon Corporation's recombinant human alpha 1-antitrypsin (rHuA1AT). Under terms of the agreement, Halozyme may receive up to $63 million, commencing with an upfront payment of $9 million and total potential future milestone payments of $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.

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