Grant Funding Helps Karyopharm Speed Lead Cancer Drug to Clinic

3/22/12Follow @arleneweintraub

In January 2011, Natick, MA-based Karyopharm’s 12 employees learned that the Multiple Myeloma Research Foundation would be awarding them $1 million in research funding—a giant sum considering most grants from non-profits are in the low- to mid-six-figure range. “One of the reasons it was such a substantial amount is that the grant program is focused on making products,” as opposed to just doing basic research, says Karypharm CEO Michael Kauffman. “To make products, you need to invest a lot. This was a very important grant for us.”

During a panel discussion on alternative funding sources at Xconomy’s April 4 forum, New England’s Emerging Biotech Stars, Kauffman will tell the story of how Karyopharm went about winning the grant. He’ll be joined on the panel by Louise Perkins, chief scientific officer of the Multiple Myeloma Research Foundation (MMRF), which has awarded $11 million to biotech companies since it launched its Biotech Investment Awards program in 2006. The panel will also include Julia Greenstein, assistant vice president at the Juvenile Diabetes Research Foundation, and Rick Winneker, senior vice president of the Leukemia & Lymphoma Society. The discussion, which will be moderated by Jens Eckstein of SR One, will focus on the increasingly important role of non-profit foundations in funding early-stage research.

Karyopharm was founded in December 2008 with the goal of combating cancer by focusing on a specific cellular process that hadn’t yet been addressed by available therapies. The company is developing compounds that inhibit CRM1—a protein that prevents the body’s natural tumor-fighting mechanisms from working properly. In healthy people, tumor-suppressor proteins live in cells and prevent them from becoming cancerous. But if CRM1 forces those proteins out of the center of the cell, known as the nucleus, they can no longer do their job. “We’re trying to get the tumor-suppressor proteins back into the nucleus by preventing them from getting carried out by CRM1,” Kauffman explains. By mid-year Karyopharm expects to begin testing its lead compound, KPT-330, in patients with some blood cancers, including multiple myeloma, and in patients with advanced solid tumors.

Karyopharm has raised $32 million from an undisclosed group of venture capitalists and private investors, but the contribution from MMRF is particularly meaningful, Kauffman says. MMRF, which was founded by patient Kathy Giusti, has committed to advancing drug-development programs and has chalked up some successes, including a molecule being developed by San Diego-based Intellikine that’s now in clinical trials. The organization’s Biotech Investment Awards program also handed $1 million to two other Boston-area companies last year: Constellation Pharmaceuticals and Epizyme. One advantage of getting funding from organizations that are focused on serving the needs of patients, Kauffman says, is that they’re not afraid to support completely novel approaches. “Venture capitalists are a bit of the opposite,” he says. “They’re much more nervous these days about novelty.”

That said, winning the MMRF grant was far from easy for Karyopharm. Kauffman says the company’s application was put through “a very intense review process,” which included two levels of scientific review facilitated by folks inside the MMRF.

One piece of advice Kauffman has for other entrepreneurs interested in obtaining grant funding is to establish early ties with physicians and scientists in academia. “The MMRF and others will always go directly to the people handling your drug to ask them how it compares to others they’ve seen,” he says. “You have to get your drugs into the hands of the people who are experts in the biology of the disease.” Karyopharm has more than 70 academic collaborations worldwide, he says.

Kauffman will pass along other tips during the panel discussion on alternative funding at Xconomy Forum: New England’s Emerging Biotech Stars. The event will be held at Biogen-Idec in Cambridge, MA starting at 1:30. We’ll also hear from five local biotech CEOs, Mark Levin of Third Rock Ventures, and top executives from Biogen. Check out the full agenda, and register today to get special deals for students and startups. You can follow all the action on Twitter at #XCbiostars. We look forward to seeing you at what’s sure to be a stimulating afternoon.

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