The Third Rock-launched Global Blood Therapeutics has rounded up $48 million to push into the clinic with an experimental therapy for sickle cell disease and set the stage for an IPO later this year--once they have proof-of-concept data for their drug in hand.
The first clinical trial is being started by Quintiles ($Q) and Guy's Hospital in London, says CEO Ted Love, the former R&D chief at Onyx who stepped in to helm the South San Francisco-based biotech last summer. Their compound was invented in GBT's lab, he adds, where the chemists were working with targets "where there is a lot of biology," inspiring "a high degree of confidence that if we can hit it, we'll see a positive outcome with the disease."
Sickle cell disease is rare in the U.S., with 100,000 cases to treat. But the CEO is looking at this globally, with millions of lethal cases that loom large.
"This is a big deal," adds Love. "There are 25 million people in the world with this problem. It's a huge, huge health problem globally. And we think this is a beautiful opportunity to do something dramatic."
Their drug, GBT440, is an oral therapy designed to increase hemoglobin's affinity for oxygen. The treatment blocks polymerization of sickle hemoglobin (HbS) and the sickling that results, which promises to halt the progression of the disease. And investigators at the growing biotech are also focused on two preclinical programs, an oral drug for hereditary angioedema, currently treated with IV therapies, and a second program for a therapy aimed at providing adequate oxygen in the blood.
That's a story that Love and GBT's backers believe will sell well on Wall Street. The Phase I/II study will begin early next year, giving them data to take to investors in the second half of 2015, provided the IPO window remains open for another year.
In the meantime, says Love, the staff of 37 will grow to about 50 as they gear up new work, including two preclinical projects. The Series B mezzanine round came from Wellington Management, RA Capital, Deerfield Management, Sabby Capital, Perceptive Life Sciences and an undisclosed investment fund.
Charles Homcy and Craig Muir of Third Rock are among the group of founders, which also includes David Phillips and three scientific researchers at the University of California, San Francisco: Matthew Jacobson, Andrej Sali, and Jack Taunton.
- here's the release