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Alnara Bags $35M B Round, Plans to Seek FDA Approval for Cystic Fibrosis Drug

[Corrected 1/28/10, 3:15 pm. See below] Alnara Pharmaceuticals has received a big financial boost at a critical time. The Cambridge, MA-based biotech firm says it has nabbed $35 million in a Series B funding round as it puts the finishing touches on the paperwork it needs to complete before asking the FDA for permission to sell its first product, an enzyme-replacement drug for patients with cystic fibrosis.

The firm’s new investor, MPM Capital, led the second-round financing, which included contributions from Bessemer Venture Partners, Frazier Healthcare Ventures, and Third Rock Ventures. Ashley Dombkowski, a managing director at MPM, is joining the board at Alnara as part of the funding round. With the new capital, the company has now raised $55 million since it was founded in 2008.

Alnara has ambitious plans for how to use its latest infusion of cash. By the end of the first quarter, the firm plans to file a new drug application to get U.S. approval of liprotamase, a pancreatic enzyme-replacement therapy that helps patients with cystic fibrosis to digest fats, proteins, and carbohydrates, according to Alexey Margolin, the company’s co-founder and CEO. The money will be needed to complete the application and prepare for the potential commercial introduction of liprotamase, which could hit the U.S. market as early as the fourth quarter of this year.

Robert Gallotto, the chief operating officer of Alnara, said that the firm expects to rapidly capture a significant portion of the estimated $400 million annual market for pancreatic enzyme replacement therapies in the U.S.

Liprotamase could provide big benefits to both cystic fibrosis patients as well as Alnara’s investors. Pancreatic enzyme treatments have been used for decades by patients whose pancreases are unable to produce enough of certain enzymes that people need to break down and digest certain fats and proteins. Traditionally, the enzymes are harvested from pig pancreases, and patients swallow a few capsules with their meals. Alnara, on the other hand, makes liprotamase with microbial enzymes in a process that is similar to how synthetic insulin is made. [Editor’s note: The original version of this paragraph erroneously described liprotamase as a “genetically engineered copy of a natural human enzyme,” though it is a recombinant microbial enzyme that is not a copy of human enzyme, according to a spokeswoman for Alnara.]

Alnara believes its treatment has several advantages over those made with pig enzymes. For one, the engineered, or recombinant, enzymes don’t carry the same risk of harboring viruses that the pig enzymes do. (However, Alnara acknowledges this safety risk is largely theoretical, because there have been no reported viral contamination in the previous treatments with pig enzymes.) Also, liprotamase can be taken in much lower doses than existing treatments, because the treatment is designed to prevent the digestive system from degrading its enzymes before they are needed. The company says that its research shows that patients need about five capsules of liprotamase per day, compared with 20 capsules per day for people who take competing treatments. The dominant competitor in this market is Solvay Pharmaceuticals.

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.