Cyclacel lead drug likely to fail trial, says safety board

(Reuters) - Cyclacel Pharmaceuticals Inc said an independent committee had determined that its lead drug would unlikely show a statistically significant improvement in the survival rates in patients with a form of blood cancer. The company's stock plummeted as much as 75 percent to hit a record low of 70 cents on Tuesday. Cyclacel's oral drug, sapacitabine, is being tested in a late-stage study in elderly patients with acute myeloid leukemia (AML) unfit for or who have refused intensive chemotherapy. However, the independent data and safety monitoring board recommended that the patients remain on the treatment as the interim data analysis showed no safety concerns. It would be pragmatic to continue the study because mature data could reveal, in the best case, a survival benefit, but more likely a trend, Chief Executive Spiro Rombotis told Reuters. Rombotis said Johnson & Johnson's decitabine, which was rejected by the U.S. Food and Drug administration for use in AML, was approved in Europe on the basis of data that did not show a significant improvement in survival but a trend. "Even a trend may give Cyclacel the business case to file for approval, at least in Europe," he said. The interim analysis was primarily driven by the deaths in the early stages of the trial. Of the 247 deaths in the study so far, 70 percent occurred in the first 6 months, the company said. "For a disease like this, where you have a lot of patients dying on either arm (of the trial) - because the disease is too far gone - if you have benefit you will only see it at the end," Rombotis said. Rombotis also said that the board had determined that the drug was not found disadvantageous to the patient population. The late-phase study, whose main goal is overall survival, is comparing a regimen of sapacitabine alternating with intravenous decitabine versus decitabine alone. Sapacitabine has an orphan drug designation in the United States and Europe for AML. Acute myeloid leukemia - a disease with unclear cause - is a type of cancer that originates in the bone marrow. Left untreated, it can quickly become fatal. Sapacitabine is also being evaluated for use in myelodysplastic syndromes and solid tumors. The Berkeley Heights, New Jersey-based company's stock was down nearly 74 percent in afternoon trading on the Nasdaq. Nearly 3 million shares changed hands, compared to their 50-day average of 93,500. (Reporting by Natalie Grover in Bengaluru; Editing by Saumyadeb Chakrabarty and Sriraj Kalluvila)