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Getting under our skin
February 2013
by Kelsey Kaustinen  |  Email the author


SAN DIEGO—In pursuit of easing medicine administration methods for patients, Pfizer Inc. and Halozyme Therapeutics Inc. have announced a worldwide collaboration and license agreement to develop and commercialize biologics for subcutaneous delivery. The partnership brings together proprietary biologics from Pfizer's collections with Halozyme's Enhanze technology, a proprietary drug delivery platform based on Halozyme's patented recombinant human hyaluronidase enzyme, rHuPH20).
Per the terms of the agreement, Pfizer has been granted a worldwide license to develop and commercialize products that combine its proprietary biologics, directed at up to six targets, with rHuPH20. The targets may be selected on an exclusive or non-exclusive basis. In return, Halozyme will receive an initial payment of $8 million, a sum that includes an upfront fee for exclusive licenses to two specified therapeutic targets in primary care and specialty care indications, as well as the right for Pfizer to select up to four additional targets upon the payment of additional fees. If certain development, regulatory and sales milestones are met, Halozyme stands to receive additional payments of up to $507 million, and is also eligible to receive royalty payments on net sales of any licensed products.  
"I am delighted about this opportunity, as it has the potential to enhance Pfizer's ability to optimize treatments for patients," Jose Carlos Gutierrez-Ramos, senior vice president of Pfizer BioTherapeutics R&D, said in a statement.  
Halozyme's Enhanze platform is based on rHuPH20. The enzyme removes the limitations on the volume of biologics that can be administered subcutaneously by "temporarily [degrading] hyaluronan, a structural component of the subcutaneous space that is just beneath the outside surface of the human skin," the company notes on its website. This creates a window of opportunity for the administration of larger injectable biologics such as monoclonal antibodies, allowing molecules as large as 200 nanometers to pass through the subcutaneous space, the company claims.  
"We look forward to working with Pfizer to apply Enhanze to these exciting targets," Dr. Gregory Frost, president and CEO of Halozyme, said in a press release. "Enhanze enables biologics to be delivered as a simple subcutaneous injection."  
Frost notes that following its acquisition of Wyeth, Pfizer has emerged as a strong partner in biologics. The company, he says, is one known for its strength in oral medications and small molecules, but in recent years, Pfizer's work with recombinant proteins has been growing as well.  
"When you think of some of the first targets that we're collaborating on, which are primary care and specialty markets, Pfizer has historically been a leader in these sorts of areas as well, from the commercial perspective. So the collection of those two really made for an exciting partnership for us and a really good fit," says Frost.
The Pfizer collaboration is one of several Halozyme has established based on its Enhanze platform—with other partners like Roche, Baxter, ViroPharma and Intrexon—and Frost says he considers the approach a "win-win" for patients in seeking to simplify medicine administration. Great medicines and advancements have been made, he notes, and trying to turn those medicines into easy-to-use products for patients is very important, with the added benefit of reducing healthcare costs.  
"Biologics, things like monoclonal antibodies today, they're great medicines. We've made, as an industry, so much progress on them. But they are not proteins that you can take as a simple pill. We would all agree that a pill you take once per month would be nirvana—unfortunately, science is just not there," says Frost. "Oral medications still have a very strong place in the industry, but when you start developing things like monoclonal antibodies, there's been a tremendous shift away from the classic intravenous infusion towards more simplified methods of administration. So self-administration for patients is something which is a trend that the industry is very much going towards."

Pfizer licenses spinal muscular atrophy program from Repligen  
WALTHAM, Mass.—Pfizer also recently entered into an exclusive worldwide licensing agreement with Repligen Corp. to advance Repligen's program for the treatment of spinal muscular atrophy (SMA), an orphan neurodegenerative genetic disease that presents early in life.  
The program includes RG3039, a small-molecule drug candidate in clinical development for SMA, as well as backup compounds and enabling technologies. Under the terms of the agreement, Repligen is entitled to receive up to $70 million from Pfizer, starting with an upfront payment of $5 million and total potential future milestone payments of up to $65 million, well as royalties on any future sales of SMA compounds developed under the agreement.
Under the terms of the agreement, Repligen is responsible for completing the first two cohorts of an active Phase I trial evaluating RG3039 in healthy volunteers, which it anticipates will occur during the first quarter. Repligen will also provide certain technology transfer services to Pfizer, which will then assume full responsibility for the SMA program moving forward, including the conduct of any registration trials necessary for product approval. Repligen has previously received U.S. Orphan Drug and Fast Track designations for RG3039 for the treatment of SMA, as well as Orphan Medicinal Product designation in Europe.  
"There is a critical need to expedite potential treatment solutions for rare diseases such as spinal muscular atrophy, where patients have such limited options," said Jose Carlos Gutierrez-Ramos, senior vice president of Pfizer BioTherapeutics R&D, in a statement. "This partnership will combine our expert capabilities in advancing molecules for genetic diseases with Repligen's leading SMA program."  
The SMA program was originally in-licensed from Families of SMA.
Code: E021302



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