Drug Development

Cyclacel unveils a flop in leukemia 2 years after a call on futility, but still won’t give up

Cyclacel Pharmaceuticals $CYCC knew more than two years ago that its Phase III study of sapacitabine for newly diagnosed cases of acute myeloid leukemia in the elderly was a bust, unlikely to produce positive data. But with no sign that the drug was doing any harm, investigators carried on treating patients.

MD Anderson’s Hagop Kantarjian

And today they proved that the data and safety monitoring board that called the study for futility at the end of 2014 was absolutely right. The drug flopped against a placebo, unable to demonstrate a significant overall survival benefit. But there was a small sliver of sunshine as the drug did post an “improvement” in complete responses. And that was enough for the lead investigator to carry on with the data analysis and go past the top-line results discussed this morning.

MD Anderson’s Hagop Kantarjian said:

Although the experimental arm of alternating decitabine-sapacitabine did not reach statistically significant superiority in overall survival, it is remarkable that an improvement in complete remission rate was observed.  Additional analysis of stratified and exploratory subgroups is warranted to identify patients who are most likely to benefit from treatment with the experimental arm.

Cyclacel’s market cap has been steadily whittled away over the past few years. Once a high flyer, the market cap is now a mere $15 million, and shrinking. Its stock plunged 33% in pre-market trading this morning. But Cyclacel CEO Spiro Rombotis says the biotech has the cash on hand to see things through to the end of next year, adding that he still has hopes for what can be gleaned in subgroup analyses.

“In parallel with data analysis and regulatory discussions, we will reevaluate our continued investment in sapacitabine in hematological malignancies,” the CEO said in a statement. “Our clinical development strategy in oncology will now concentrate on our two ongoing, clinical programs in DNA damage response and transcriptional regulation, which include our area of historical expertise in CDK inhibitors. These programs target biomarker-selected patients, such as those with BRCA mutations or resistance to existing cancer therapies.”


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RAPS Regulatory Convergence 2017