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| Fred Eshelman, founding chairman of Furiex Pharmaceuticals |
Furiex Pharmaceuticals says it collected positive data from a pair of late-stage studies on its lead drug for irritable bowel syndrome, putting the biotech on a short path to a near-term marketing application in the U.S. with a later filing planned for Europe. And while Furiex says it's within the realm of possibility to launch this drug--eluxadoline--by themselves, the developer expects the data to spur interest in a buyout based on what it sees as "blockbuster" potential for its therapy.
Morrisville, NC-based Furiex saw its shares ($FURX) soar on the news, rocketing up 129%. The stock closed Monday at $45.97, giving the biotech a market cap at the time of $476 million.
Investigators recruited 2,428 patients with what's called diarrhea-predominant cases of IBS for the placebo-controlled Phase III studies. They set out to determine how many people in each of three groups responded, with improvements in pain and stool consistency in at least half of the days patients tracked their responses. In one 12-week study for the FDA the 100-mg drug arm recorded a response rate of 29.5%, with 28.9% in the 75-mg drug arm responding and 16.2% in the placebo group meeting the criteria. Over a 26-week period tracked for the EMA the results were 32.6%, 30.4% and 20.2%, respectively.
Pooled data from both studies demonstrated a statistically significant improvement in response for the patients taking the two doses, though the 75-mg dose fell short of statistical significance in the second study.
That's plenty good enough for an approval, founding Chairman Fred Eshelman tells FierceBiotech, especially if you consider how demanding the trial design was.
"If we don't get offers," Eshelman adds, "then with the data we would mount a commercial offer ourselves. We'd have to do some money-raising of one type or another; we don't have any commercial operations." Eshelman, who founded PPD, declined to say whether the company had already begun sales talks with potential buyers, but he allowed that he would be surprised if the data didn't spur discussions.
Furiex has been working on the drug under the FDA's fast-track status. The therapy is a mu opioid receptor agonist and delta opioid receptor antagonist which acts in the gut. The dual opioid activity is designed to treat diarrhea and pain symptoms of IBS-d, without causing the constipating side effects that can occur with unopposed mu opioid agonists.
There are a total of about 28 million people in the EU and U.S. with the disease, says Furiex President June Almenoff. Women with severe cases of IBS with diarrhea are often prescribed Lotronex, she adds, while men in the same category don't have a treatment option. If you narrow the market down to patients with a moderate to severe case, "the person who can't control activities of daily living because of running to bathroom," the core patient population could be around two to three million patients.
"I believe it's blockbuster size myself," asserts Eshelman.
Eluxadoline is the lead drug in the pipeline at Furiex, a biotech that was spun out of the CRO PPD back in 2000 with a crew of development executives who felt they could set a hot pace in development work. Running Phase I studies through a mid-stage program and the two Phase III trials for eluxadoline consumed only 4 years, a quick turnaround by today's biotech standards.
- here's the press release