Cell Therapeutics (CTIC) got a momentary reprieve from potentially bad news Friday as the US Food and Drug Administration postponed a meeting about pixantrone, an experimental treatment for refractory aggressive non-Hodgkin’s lymphoma, due to blustery weather. It's the company's best hope at generating any substantial revenue and an important bet to win for Cell Therapeutics and, among others, the two institutional investors that put a fresh $30 million into the company in January.
Doubt about the drug's approval was cast by an FDA brief questioning pixantrone's efficacy February 8. That sparked investors to drive Cell Therapeutics' shares off a cliff, denting their value by 40 percent, briefly. It also called into question the likelihood of Novartis (NVS) acting on its option to license the drug, an event that could deliver as much as $104 million in registration and sales milestones should it pan out.
The Oncologic Drugs Advisory Committee noted that Cell Therapeutics had enrolled just 140 of the 320 patients it had expected to enroll in its pivotal study. Such a small group left the statistical significance of the study's results vulnerable to small errors. The smaller than expected patient group also led the study to be ended early, leaving “a higher level of evidence is usually required in trials which discontinue prior to the final analysis,” according to reviewers.
Reviewers also expressed concern about “substantial hematologic and cardiac toxicity” after finding that deaths, significant adverse events and other bad outcomes were all more common in patients taking pixantrone instead of other cancer drugs.
Should the panel advise against approving pixantrone, the Seattle-based company will be left with no drugs near market and no other imminent paths to generating revenue.
Although the FDA doesn't have to follow the advice of its advisory panels, it often does.
The panel considering pixantrone, to be marketed as Pixuvri, has not yet been rescheduled.
Assuming the sky doesn't fall on pixantrone, Cell Therapeutics plans to file a marketing authorization application for the drug in Europe by mid-2010.
Disclosure: No position