Tolerx and Glaxo Diabetes Drug Disappoints in Late-Stage Study

3/11/11

There’s some bad news from Cambridge, MA-based Tolerx today. The venture-backed biotech firm and its partner GlaxoSmithKline (NYSE:GSK) reported that their experimental drug for Type 1 diabetes failed to meet the main clinical goal of a late-stage study, called Defend-1.

The 272-patient Defend-1 study was designed to measure how well newly diagnosed adult patients were able to improve production of insulin a year after an 8-day course of the treatment, a monoclonal antibody called otelixizumab. The main goal or endpoint of the study was for patients treated with the eight-day course of the drug to show a change in a measure of insulin production called C-Peptide, but the results showed that that goal was not met. And while the companies are reviewing the results of the study, they have suspended recruitment and dosing of a separate late-stage trial of the treatment, called Defend-2.

Otelixizumab, which is the most advanced drug in Tolerx’s pipeline, is supposed to protect insulin-producing cells in the pancreas from immune system attacks that destroy their function in patients with Type 1 diabetes, ultimately hampering the body’s natural ability to control blood sugar levels. In October 2007, the company and Glaxo formed an alliance that could be worth as much as $760 million to develop otelixizumab for Type 1 diabetes and other diseases. Tolerx has no approved drugs on the market. Yet the company has several other drugs in its pipeline for cancer and other diseases in which the immune system plays a major role.

“Clearly these are disappointing data, but we are committed to working with Tolerx to better understand the results of this study and determine the way forward,” Jackie Parkin, medicines development leader at Glaxo, said in a statement.

Tolerx, founded in 2000, had attracted more than $150 million in investments as of last March when my colleague Luke Timmerman profiled the company. The company’s venture investors include HealthCare Ventures, Skyline Ventures, and Sprout Group. And the biotech has received support from the Juvenile Diabetes Research Foundation and, of course, Glaxo, through its major collaboration with the London-based drugmaker.

There have been high hopes for otelixizumab, which was licensed from London-based drug developer BTG, to offer a new way to treat patients with Type 1 diabetes, which is also called insulin dependent and juvenile diabetes. (The disease, which is far less common than Type 2 diabetes, affects about 5 percent of the more than 20 million Americans who have diabetes, according to the American Diabetes Association.) The standard way to treat the patients today is regular injections of insulin and closely monitoring their blood sugar levels.

In October 2010, Eli Lilly and MacroGenics announced that their comparable experimental antibody for Type 1 diabetes also did not meet the primary endpoint of a late-stage trial.

Despite the bad news about the Defend-1 study, Tolerx co-founder and CEO Doug Ringler sounded upbeat about the other drugs in his company’s pipeline. “While we are disappointed in the Defend-1 results of otelixizumab, we remain committed to the development and commercialization of the candidates in our pipeline, each of which has a distinct mechanism and target for correcting abnormal immune responses,” Ringler said in a statement.

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