Most biotech startups wait seven years or more before entering late-stage clinical trials. But Alnara Pharmaceuticals, which is just six months removed from its $20 million first-round financing, has acquired a license to an enzyme-replacement pill in the final stage of development for patients with cystic fibrosis and chronic pancreatitis.
Cambridge, MA-based Alnara has licensed the drug, liprotamase, from a nonprofit affiliate of the Cystic Fibrosis Foundation, taking over development of a treatment in the latter half of a Phase III trial, which is intended to provide the clinical data needed to submit an application to the FDA for approval of the drug, says Alnara CEO Alexey Margolin. Liprotamase could become the first pancreatic enzyme supplement from non-animal sources to be approved, providing an alternative to existing treatments for pancreatic insufficiency that use enzymes derived from the pancreatic glands of pigs. The drug is made from genetically engineered copies of microbial enzymes, which are believed to be more reproducible to manufacture, and carry fewer risks of provoking an immune system reaction, than the enzymes from pig pancreata.
Alnara has seized control of the oral drug because of an interesting confluence of factors. Financial constraints prompted Cambridge-based Altus Pharmaceuticals (NASDAQ:ALTU), the original developer of liprotamase, to fork over rights to the drug to the CF Foundation as part of a restructuring plan revealed in January. The CF Foundation, a Bethesda, MD-based organization that has supported the development of the drug since 2001, was on the hunt for another biotech firm to continue to develop it. The drug fit Alnara’s strategic mission to develop orally delivered protein drugs directly to the gut. And Alnara CEO Margolin knew this program quite well. He’s the former chief scientist at Altus and had been in charge of the program to develop the drug. Financial details of the licensing agreement between Alnara and the CF Foundation weren’t revealed.
“When I left Altus, I never thought that I would work on [liprotamase] again,” Margolin says, “and I never thought that when we closed our Series A [round] that in five or six months we would have a Phase III program.”
Margolin says he was surprised to see Altus cede rights to the drug, which cleared an earlier late-stage clinical trial in 2008. The current trial is intended to test the safety of the drug and how well it helps CF and pancreatitis patients maintain weight, height, and other measurements of its nutritional benefits. The study is expected to wrap up by this summer, Margolin says, and afterward Alnara will begin steps to ask the FDA to approve the drug. Also, the CF Foundation has agreed to financially support the continued development of the treatment. (He declined to say when the application for approval would be submitted to the FDA.)
About 90 percent of people with cystic fibrosis take pancreatic enzyme supplements to help them absorb nutrients, Margolin says. Cystic fibrosis, which affects 30,000 Americans and 70,000 people globally, is a genetic disease that causes lethal lung infections and digestive problems. The current market for pancreatic enzyme supplements—made by firms such as Belgium-based drug firm Solvay—is around $350 million per year, Margolin says.
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