Alnara Pharma Completes Key Cystic Fibrosis Drug Trial, “Extremely Encouraged” by Results

5/28/09

Alnara Pharmaceuticals won’t likely reveal the full results of its late-stage clinical trial of enzyme-replacement drug liprotamase in patients with cystic fibrosis until this fall, but the Cambridge, MA-based biotech firm has completed the trial in recent weeks and continues to find the effectiveness of the treatment “extremely encouraging,” Robert Gallotto, chief business office of Alnara, tells Xconomy.

Gallotto isn’t disclosing detailed results of the key trial, designed to form the basis of an application for approval of liprotamase, so it’s too early to say whether the study was a success. Yet he indicates that he and others at Alnara are pleased with the results of the trial, which ended several weeks ago when the last of 145 patients involved in the trial received a final examination. He expects the full results of the trial to be reported during the Annual North American Cystic Fibrosis Conference in October. (The drug already showed positive effects in cystic fibrosis patients in a separate late-stage study reported last year, and it wouldn’t be a big surprise to see similar results in the larger trial that was just completed.)

“We are extremely encouraged with what we’ve seen,” Gallotto says. “The data continues to be encouraging and we look forward to sharing it in its totality sometime in the fall.”

Liprotamase became the No. 1 drug in Alnara’s pipeline in March, when the young biotech startup gained control of the treatment in a licensing deal with a nonprofit affiliate of the Cystic Fibrosis Foundation, of Bethesda, MD. The drug replaces a pancreatic enzyme for absorbing nutrients that is lacking in patients with diseases such as cystic fibrosis, a genetic disorder that results in lung infections and digestive problems. Gallotto and Alnara CEO Alexey Margolin were also intimately involved in the development of liprotamase in their former jobs as executives at Cambridge, MA-based Altus Pharmaceuticals (NASDAQ:ALTU), which discovered the drug but ended its program to develop it in January due to financial constraints. That prompted the CF Foundation, which had provided funding for Altus to develop the drug, to take over the program from Altus.

The latest clinical trial of liprotamase—which previously showed promising results in studies involving patients with chronic pancreatitis and other pancreatic disorders—was designed to test the safety of the oral drug and compare the growth, height, weight, and vitamin absorption of CF patients with what are considered normal levels among healthy individuals, Gallotto says. The trial studied the effects of the drug in each patient over a 12-month period. When Altus was still developing the drug late last year, it reported positive results of an initial late-stage clinical trial to study its effects in CF patients. But this latest study was needed to seek approval of the drug.

Alnara is already in talks with the FDA about the trial results and is writing an application for approval of liprotamase, aiming to submit a New Drug Application (NDA) to regulators by the end of the year, Gallotto says. The company—which launched last year with a $20 million Series A round of venture capital—has enough funding to support it up until it submits the NDA, he adds.

Liprotamase is designed to offer more stable enzymes than existing treatments and may enable patients to take fewer pills than they do with the other enzyme drugs. Liprotamase is also made from engineered microbes, as compared with versions of the drug now on the market derived from pancreatic enzymes in pigs. Gallotto says that regulators have noted concerns about the potential risk of those other treatments carrying swine viruses. Still, the FDA earlier this month granted formal approval to one of these drugs, marketed by Solvay Pharmaceuticals under the brand name Creon, saying that the treatment met safety and efficacy standards.

The U.S. market for such enzyme therapies is about $300 million annually, Gallotto says. Alnara hopes to begin marketing liprotamase in the U.S. next year.

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