Pfizer's sickle cell anemia candidate enters trials, to the delight of delivery partner Halozyme

The first subject in a Phase I study of Pfizer's ($PFE) subcutaneous sickle cell anemia candidate has been dosed, triggering $1 million milestone payment to Halozyme ($HALO), developer of the enhanze drug delivery platform.  

The drug, dubbed Rivipansel, is in Phase III as an intravenous formulation. It's specifically for a severe complication of the disease. So-called vaso-occlusive crisis occurs when sickle-shaped, adhesive red blood cells occlude small blood vessels. The condition can cause acute ischemic tissue injury, inflammation and pain that can be even worse than what's experienced during child birth, according to the NIH.

Halozyme says sickle cell anemia affects 100,000 people in the U.S. alone and millions across the world.

"The advancement of the first product candidate under our Pfizer collaboration represents further progress and validates the potential of our Enhanze platform," said Halozyme CEO Dr. Helen Torley in a statement. "The growing number of products co-formulated with Enhanze that are entering the clinic or already marketed represent, I believe, just the beginning of the potential that exists to help an even broader population of partners and patients."

The Enhanze enzyme (dubbed rHuPH20) enables subcutaneous delivery by degrading hyaluronan, a component of the subcutaneous space located just under the skin. Halozyme says on its website that Enhanze facilitates the passage of molecules as large as 200 nanometers through the subcutaneous space. And the degradation is temporary because hyaluronan reestablishes itself after several days.

In addition, Halozyme touts the benefits of converting an intravenous drug to a subcutaneous one, saying doing so can reduce costs, improve efficacy and extend a product's lifecycle.

Halozyme outlicensed rHuPH20 to Pfizer in 2012, for use in up to 6 Pfizer biologic candidates on an exclusive or non-exclusive basis in return for an upfront payment of $8 million, milestone payments and royalties on the sale of commercialized products. So far, four exclusive targets have been selected by the Big Pharma, according to a release.  

Halozyme says Roche ($RHHBY) has the rights to apply Enhanze to up to 8 targets, and has commercialize a subcutaneous formulation of two products in Europe. Meanwhile, Janssen, Baxalta ($BXLT) and AbbVie ($ABBV) are also in drug delivery partnerships with the company.  

Pfizer's milestone is also good news for Rivipansel developer GlycoMimetics ($GLYC), which outlicensed the compound to Pfizer in 2011.

- read the release from Halozyme
- here's the release from GlycoMimetics

Special Report: FierceDrugDelivery's 10 biggest partnerships in drug delivery - Johnson & Johnson - Halozyme

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