CHICAGO--Elcelyx Therapeutics has debuted midstage data for its lead diabetes drug candidate, a delayed-release version of the old warhorse metformin. With the data being presented Sunday at the American Diabetes Association annual meeting, the San Diego startup also aims to upend beliefs about how metformin works.
About 4 million diabetics in the U.S. have kidney problems and aren't supposed to take metformin, a first-line and foundational treatment for Type 2 diabetes, because of the lactic acidosis that the drug can trigger after it enters the bloodstream. Elcelyx's formulation of metformin, called NewMet, is designed to deliver metformin at the lower bowel, where active drug acts on enteroendocrine cells. This way less metformin hits the bloodstream, potentially making the treatment available to millions of diabetics with renal impairment.
In a 24-patient Phase IIa study, the startup gained some early evidence that NewMet works as advertised.
"This is our first coming-out party with respect to data disclosures," said Elcelyx CEO Dr. Alain Baron, in an interview with FierceBiotech. "The diabetes expert community will have to face with changes in how metformin has worked all these years."
The Phase IIa study showed 45% and 57% drops, respectively, in metformin in the bloodstream in the 19 patients randomized to take either 1000 mg or 500 mg of the company's delayed-release version of the drug for 5 days. All 24 patients showed similar drops in blood sugar during the short study, including those on generic metformin, supporting the company's hypothesis that delivery at the lower bowel does the job.
The company also came away with some early safety data favoring the delayed-release versions. Nausea and vomiting occurred in 9% of the patients on generic metformin and in none of the patients on either dose of NewMet. This is a very small data set to draw many conclusions about the gastrointestinal safety of the experimental drug, but it shows signs that NewMet could be used in patients who aren't able to take metformin because of gut side effects.
Elcelyx has wrapped up enrollment for a 240-patients Phase IIb study that will compare treatment on three separate daily doses of NewMet against two doses of extended-release metformin. The data from the larger trial should show whether or not it's worth embarking on a massive and expensive development program to bring NewMet to market.
"When we think about the marketplace for NewMet," Baron said, "it's not in competition with old [metformin]. Old [metformin] is obviously generic, it's very cheap, and if people are taking it and they are doing fine with it, that's fine. But [NewMet] is for those patients who can't take metformin."
An estimated 40% of the 11 million Americans on metformin take reduced doses of the drug because of its side effects, the CEO pointed out, and some of them could also benefit from NewMet.
Baron, who formerly served as SVP of R&D at Amylin, has steered Elcelyx on a dual path to develop NewMet like any experimental drug as well as a nutritional supplement called Lovidia as an over-the-counter product to help people lose weight. The company has scored $43 million from backers including veteran investor Rick Barry, Morgenthaler Ventures, Kleiner Perkins Caufield & Byers and Technology Partners.
Yet given the massive investment required to develop new diabetes drugs, Baron said, Elcelyx aims to sell NewMet to a larger player in early 2014.
"We do not want to develop this in Phase III or commercialize this," Baron said. "This is not the purview of biotech. That experiment's been done, and it's not a good one."
- here's the release
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