A little more than two years after paying a modest $10 million in near-term cash to license in the experimental blood cancer drug tosedostat, Cell Therapeutics ($CTIC) today announced that the FDA had put a partial clinical hold on its study after the death of one of the patients in a clinical trial. The patient died of myocarditis--an inflammation of heart muscle.
The news sliced 10% off of the value of the biotech's shares, which were trading at $1.09 ahead of the bell.
"Recently, a patient, who was in their seventies and was being treated with Tosedostat in combination with 5-azacitine or cytarabine on an investigator sponsored trial in patients with relapsed or refractory acute myeloid leukemia or high risk myelodysplastic syndrome (MDS), died of myocarditis," the biotech reported in a filing with the SEC. "The FDA has requested additional data on patients treated with Tosedostat, including additional information about the patient that died, a detailed review of all grades of cardiac adverse events or cardiac-related investigations in patients treated with Tosedostat, as well as benefit-risk analysis based on the data presented. The company has begun work to comply with the FDA request and expects to be able to submit these data to the FDA in the coming weeks."
The last few years have provided some dramatic plot twists for the small drug developer, which has a market cap of only $130 million. Years before Aveo ($AVEO) became a cautionary tale about the wrath of FDA cancer czar Richard Pazdur, Cell Therapeutics experienced what it was like to incite his opposition. The biotech lost a unanimous vote by outside FDA experts after Pazdur laid into the program for Pixuvri (pixantrone), which failed to demonstrate a statistically significant improvement over the control arm. Pazdur added that only 140 of a planned 320 patients were recruited for the trial, with a mere 8 U.S. patients in the study.
Later, in early 2012, Cell Therapeutics would pull a sudden regulatory U-turn, yanking a New Drug Application on a second try for the drug after claiming that the agency declined to give Cell Therapeutics some added time to prepare for a return bout. But the company has also kept growing its pipeline. Last spring Cell Therapeutics added pacritinib to the pipeline after paying S*Bio $30 million in cash and stock for the myelofibrosis drug. And last fall, Cell Therapeutics started the cautious rollout of Pixuvri in Europe after winning a conditional approval.
The fits and starts at Cell Therapeutics have been watched closely by a host of analysts, many fascinated by the regular retreat of its share price and subsequent moves by CEO Jim Bianco to find a fresh supply of cash.
- here's the SEC filing