A cystic fibrosis drug that seemed destined for death when its company faced financial troubles (and eventually went belly up last year) is being revived by another company.

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A new company launched by a veteran of the defunct company (Altus Pharmaceuticals, which we profiled last year) plans to submit the drug, a pancreatic enzyme replacement therapy, to the US Food and Drug Administration for approval.

Most CF patients need to supplement enzymes that help their bodies absorb nutrients in food. Liprotamase, Altus's drug, was based on microbial enzymes rather than those extracted from pig pancreas, and could be taken in a single pill, rather than the five or so patients generally take with each meal. Its development had been the cornerstone program for Altus Pharmaceuticals, based in Waltham, MA; the company had gone public after promising Phase 2 results for the drug in 2006.

But Phase 3 results two years later were not quite optimal; although the trial met its statistical endpoint -- a significant improvement in fat absorption (a measure of CF patients' often-compromised ability to take in nutrients) -- the average improvement of 15 percent was less than the 20-30 percent the FDA had requested.

Altus researchers thought the assay might be at fault, but investors lost interest, and in a bid to save itself in the crumbling economy, the company shelved the CF program and instead turned to a human growth hormone product it thought would be more lucrative. "The irony was, we spent $150 to $250 million developing [liprotamase], but we didn't have the $30 million to finish it," Ken Attie, Altus's vice president of clinical development and medical affairs, told The Scientist last summer.

Altus gave rights to the drug to the Cystic Fibrosis Foundation in January 2009, but just a few months later, CFF licensed the drug to Alnara Pharmaceuticals, started in 2008 by Alexey Margolin. who left his post as Altus's chief scientific officer to become Alnara's CEO. "Ten years ago I wrote a small SBIR grant, and that's how the [CF] program started," Margolin told The Scientist today. "I left this program behind, I never thought I would work on it again. But it was a great opportunity and I took it."

Alnara hired 35 or so employees who had been laid off from Altus, according to Mass High Tech. Altus's Phase 3 trial had statistically shown the drug's efficacy, but the company was too broke to finish another Phase 3 safety trial looking at long-term outcomes. Last October, about a month after Altus said it would end operations, Alnara announced the trial's completion with positive results. "Now we have long-term data" showing positive clinical outcomes such as increased body weight and body mass index while taking the drug, Margolin said, adding that Alnara plans to submit the paperwork for FDA approval this month.

He believes the drug's potential was always strong. "It just was a kind of unfortunate situation when Altus ran out of money," he said, which created "a perfect storm" with the collapse of the economy.

Correction (March 8): The original version of this post incorrectly referred to Alnara as a spinout of Altus. The mistake has been corrected; The Scientist regrets the error.


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