West Coast Editor
Bad pre-holiday news from the Phase III trial with pexelizumab gave investors in Alexion Pharmaceuticals Inc. little cause to be thankful, and Wall Street took away more than a fourth of the stock's value.
Alexion's shares (NASDAQ:ALXN) closed Wednesday at $21.53, down $8.03, or 27.2 percent.
Results of the study show the monoclonal antibody fragment, discovered by Alexion and aimed at inhibiting complement-mediated tissue damage, reduced the primary endpoint - nonfatal heart attack or death through 30 days after coronary artery bypass graft (CABG) surgery, in moderate to high-risk patients - but did not hit statistical significance.
"We've only had the data for a day," said Leonard Bell, CEO of Alexion. "We've looked through the Casablanca' list of the usual suspects [to determine what went wrong] and haven't found anything yet. If we had, we'd say so."
Titled "Pexelizumab for Reduction of Infarction and Mortality in Coronary Artery Bypass Graft Surgery 2," the trial, conducted under a special protocol assessment from the FDA, included about 4,250 patients enrolled at about 250 sites. It compared the safety and efficacy of pexelizumab against placebo in reducing heart attack and death after CABG surgery, with or without valve surgery.
Alexion and partner Procter & Gamble Pharmaceuticals Inc., a unit of Cincinnati-based Procter & Gamble Co., sponsored the study, results from which are due at an upcoming scientific meeting, yet to be decided.
The pharma partner apparently is still on board, Bell told BioWorld Today.
"We discussed the data yesterday, and as far as we know, that's the case," he said.
The deal to develop pexelizumab dates back to early 1999. First valued at about $95 million, the arrangement since has been renegotiated, giving Alexion more responsibility in the U.S. and more profit potential. (See BioWorld Today, Jan. 27, 1999, and Dec. 13, 2001.)
Bell said Alexion is "reticent" about investing more in the compound for the CABG indication until the new results are sifted further.
"We're not going to describe the data until [they] get presented," he added.
In an earlier, similar pivotal Phase III CABG study, the endpoint was reduced but not to a statistically significant degree (p=0.069). Also, in the 2,000-patient, moderate to high-risk study population from that study - that is, people with more than one pre-specified risk factor - death or heart attack by day 30 was reduced with statistical significance from 16.3 percent with placebo to 11.7 percent with pexelizumab (for a relative reduction of 28 percent, p=0.003). (See BioWorld Today, Nov. 11, 2003.)
Results from the first study were reported in the May 19, 2004, issue of the Journal of the American Medical Association. What the latest outcome means for another, ongoing pivotal trial is unclear for now. That international Phase III study, known as APEX-AMI, is testing pexelizumab in heart-attack patients treated with primary percutaneous coronary intervention, or angioplasty. More than 3,000 patients have been enrolled at more than 300 sites in the U.S. and overseas.
Meanwhile, Cheshire, Conn.-based Alexion forges ahead with the pivotal Phase III trial evaluating another compound, eculizumab, against the blood disorder paroxysmal nocturnal hemoglobinuria (PNH), with top-line data expected in the first quarter of next year.
"Probably the projected revenues are greater in PNH than in CABG," Bell said, and Alexion owns all rights to that compound worldwide. Next year, the company plans to start work with the drug in two more indications, he said.
