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Alnara delivered positive results from a late-stage clinical trial in October at the North American Cystic Fibrosis Conference in Minneapolis. The study, which involved 214 patients in 7 countries, tested liprotamase in 145 cystic fibrosis patients over a 12-month period. And the study showed that 96 percent of patients that took the treatment either maintained or gained weight, a key measurement of how well the treatment enables patients to stay healthy. Some patients in the study experienced adverse events related to their disease more than the treatment, such as diarrhea and respiratory infections, says Lee Brettman, Alnara’s chief medical officer.
The company also plans to develop a new formulation of liprotamase for children, which will be made in a tablet that is dissolved in a glass of water and taken like a dose of Alka-Seltzer, Margolin says. There are no such formulations of the pancreatic enzyme therapy for children with cystic fibrosis, he noted, yet there is a great need for a version of the treatment developed specifically for the pediatric market, because people are born with cystic fibrosis, and have to deal with the symptoms throughout childhood and adolescence. An estimated 30,000 people in the U.S. have cystic fibrosis, and they are generally expected to live to their late 30s.
While liprotamase is a relatively new asset for Alnara, Margolin’s experience with the product dates back several years to his tenure as a chief scientist at the former Cambridge-based biotech Altus Pharmaceuticals. Altus handed over its rights to the treatment to the Cystic Fibrosis Foundation, which had helped pay to develop the drug. Altus ran out of money before it could finish development of liprotamase. Altus eventually shut its doors for good last September.
The Cystic Fibrosis Foundation found a new home for the treatment at Alnara, which had been founded with $20 million in venture capital a few months earlier to develop protein drugs that could be taken orally. Margolin and the other founders of the company jumped on the opportunity to gain a late-stage product candidate, and the CF Foundation helped fund the late-stage clinical trial that the company reported in October. (Margolin and Gallotto wouldn’t disclose how much the foundation provided for the trial.) Alnara also boasts a board of high-profile industry veterans, including Rich Aldrich, founder of the Boston-based biotech hedge fund RA Capital Management, and Christoph Westphal, the CEO of Sirtris, a subsidiary of London-based drug giant GlaxoSmithKline.
In any case, the acquisition of liprotamase transformed Alnara from an early-stage startup to a developer of a late-stage drug candidate very quickly. The company has had to grow up fast, and in October they secured the Swiss drug manufacturing contractor Lonza to produce the enzymes for commercial supplies of liprotamase. We’ll see how fast, or if, they are able to evolve into a commercial organization.