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.
"We are pleased to collaborate with Intrexon on this exciting new endeavor for patients with A1AT deficiency," stated Gregory I. Frost, Ph.D., Halozyme's president and CEO. "The combination of Intrexon's synthetic DNA platform for high-level A1AT production with Halozyme's subcutaneous enzyme technology may enable the first recombinant human A1AT replacement therapy with a more patient-friendly administration profile."
"Halozyme's Enhanze technology is the perfect fit for our recombinant human alpha 1-antitrypsin program," stated Randal J. Kirk, CEO and chairman of the board of Intrexon and board member of Halozyme. Mr. Kirk's comment is seconded by Gerardo Zapata, Ph.D., president of Intrexon's Protein Production Division. "This collaboration allows us to utilize Halozyme's proprietary protein delivery technology with a subcutaneous recombinant human version of the A1AT protein, an innovative potential therapy for genetic emphysema, COPD, and other diseases caused by A1AT deficiency, and will facilitate our ability to bring a promising novel synthetic biologic treatment alternative to the currently available plasma-derived intravenous products," stated Zapata.
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