Even though many venture firms are scrimping, there’s hope for biotech startups in search of capital to fund bold new areas of biological research. NKT Therapeutics, which revealed its $8 million first-round financing in March, has rallied venture capitalists to back its efforts to develop drugs that target lesser-known immune cells that potentially play key roles in asthma, cancer, and a bevy of other major illnesses. Robert Mashal, CEO of the Newton, MA-based startup, filled me on how the firm is channeling its funds into the novel science.
NKT is developing biological treatments to interact with natural killer T cells (or NKT cells), a class of white blood cells, which have a growing following in both scientific and industry circles for their potential to lead to the discovery of new drugs. Though there are drugs in testing that hope to stimulate NKT cells as part of a multi-pronged immune response, no drug that exclusively targets NKT immune cells is in clinical trials or has a track record with the FDA, Mashal says. Thus, NKT Therapeutics has embarked on a biotech journey with both great challenges and great possibilities.
Despite the hurdles ahead, the startup has a deep understanding of NKT cell biology that comes from its founding researchers—Steve Balk, of Beth Israel Deaconess Medical Center, Mark Exley, of Harvard Medical School, and Brian Wilson, at Massachusetts General Hospital—who are experts in the field (they’ve also been research collaborators for years prior to founding the startup in early 2008.) Specifically, the startup has an antibody that is designed to home in on a cellular receptor—a type of protein marker on the surface of cells—it believes is unique to many NKT cells.
The firm plans to initially develop the antibody to block the function of NKT cells in patients with asthma. Research has found that removing NKT cells from genetically engineered mice quells their asthma, Mashal says, and mice or monkeys do have asthmatic responses when NKT cells are activated in their lungs. Yet the firm is about a year and a half to two years from testing the antibody in human clinical trials, he adds.
“If this therapeutic does in humans what it does in animals, then we’ve got a good drug,” Mashal says. “I think that’s the biggest ‘if’ in the industry, and until you do the trials to test that, you never really know.”
The firm’s biotech drug could be especially useful for severe cases of asthma. Mike Ross, a venture capitalist who invested in NKT on behalf of his firm SV Life Sciences, says that there are limits on the allowed dosage and effectiveness of one drug on the market for severe asthma,omalizumab (Xolair).That creates an opportunity to develop a superior treatment. Xolair, which marketed by South San Francisco-based biotech powerhouse Genentech (now owned by Roche) and Swiss drug giant Novartis, generated 2008 sales of $728 million between the two companies.
Mashal says that NKT’s treatment for moderate to severe asthma could gain acceptance among regulators, however, if the drug shows better effectiveness in reducing symptoms than existing drugs and provides improved safety for patients with the respiratory ailment. There also appears to be few competitors in the NKT field. Other firms focused on NKT cell biology include Innate Immune, a biotech startup with technology from Stanford University, and Immunox Therapeutics. There was little information I could glean on either of the two competitors on the Web, but what I did find indicates that Innate Immune is interested in treating asthma too.
NKT Therapeutics offers a big enough market opportunity to garner funding from SV Life Sciences, which was joined by British drug-maker AstraZeneca’s biotech venture group MedImmune Ventures in the startup’s first round of financing. Ross tells me that SV makes fewer investments in young startups based on bold biological discoveries than in more mature life sciences companies with products in clinical trials. “This is starting a bit earlier” for our firm, Ross says, “but that’s because we think the biology is so exciting.”
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