Several years ago, Genzyme developed a drug called plerixafor (Mozobil) that’s used to help with bone marrow transplants. Now a group of folks that helped push that drug forward have reunited for a new effort—to use a similar type of therapy for solid tumors and rare diseases.
As Xconomy reported earlier today, X4 Pharmaceuticals was started up in 2012 and is led by Paula Ragan (president and CEO), who spent six years at Genzyme. She confirmed today that the company has indeed raised a $37.5 million Series A, adding to roughly $1.5 million in seed funding. While the seed dollars came from people such as former Genzyme CEO Henri Termeer (pictured above) and former Cubist Pharmaceuticals leader Mike Bonney, among others, Ragan declined to disclose who has provided the Series A cash.
The plan, Ragan says, is to develop oral, small molecule drugs that target a receptor called CXC chemokine receptor type 4, or CXCR4, which is implicated in tumor progression and is overexpressed in several cancers. Ragan is leveraging the past experience of several Genzyme staffers with plerixafor, which is also a CXCR4 blocker. Renato Skerlj, for instance, was one of plerixafor’s inventors, and is a founder of X4. Chief operating officer Alison Lawton was part of the regulatory team that helped get the drug approved in 2008.
“We’re reconstituting a team of very experienced people who understand the target space very well based on their historical experience with [plerixafor],” Ragan says.
X4 has licensed a group of these CXCR4 blockers from a “top 10 global pharmaceutical company.” Ragan declined to say which company that is. As we reported earlier, Sanofi, which bought Genzyme in 2011, was trying to sell rights to a group of oral CXCR4 drugs in 2013. It hasn’t been confirmed whether the Sanofi assets were the ones licensed to X4, and neither Sanofi nor Genzyme have responded to an e-mail query.
Ragan says that CXCR4 has been “underexploited” as a drug target. It is involved in a variety of functions related to cancer, like metastasis, blood supply, and the trafficking of immune cells in and around tumors. Ragan points to a number of recent publications showing that blocking CXCR4 with a drug might help recruit T cells, the foot soldiers of the immune system, to recognize and fight a tumor. So in theory, a CXCR4-blocking drug might help “checkpoint inhibitor” drugs like pembrolizumab (Keytruda) or nivolumab (Opdivo) to be more potent, Ragan says.
“[I] have always been a fan of this pathway—[it’s] important in fibrosis as well,” says Michael Gilman, the CEO of Padlock Therapeutics and an X4 board member. “I was really grabbed by the therapeutic hypothesis in cancer—[CXCR4’s] role in establishing (or re-establishing in a relapsing tumor) a favorable microenvironment for tumor (re)growth—and the quality of folks of involved.”
X4’s two lead drugs are X4P-001 and X4P-002. Ragan says the first, X4P-001, should begin its first clinical testing by the end of the year—a Phase 1/2 study in either a kidney or ovarian cancer. The second drug, which would be for lethal brain cancers like glioblastoma multiforme, is a bit further behind. X4 is also going to start testing a CXCR4-blocking drug for an unspecified rare disease. The goal is to have three clinical trials underway next year in three different diseases, she says.
The company is currently based out of the Cambridge Innovation Center with less than a handful of full-time employees. But Ragan says that the plan is to grow X4 to 15 to 20 full-timers within the next six to 12 months.
“We’re basically transitioning from primarily consultants with a small number of employees to building up the management team and creating a core group of folks,” she says.
Some of the other folks already involved: independent observer and board member Richard Peters, currently Genzyme’s strategy development officer; board member Alan Walts (Advent Venture Partners); and Robert Arbeit (formerly of Idera Pharmaceuticals).
For more on the company, check out this story from earlier today.
