Bristol-Myers Squibb has scored significant overall survival results in a phase 3 trial of its oral maintenance therapy CC-486 in acute myeloid leukaemia patients who have limited treatment options.

The QUAZAR AML-001 study investigated CC-486 in newly-diagnosed AML patients who have achieved remission with intensive induction chemotherapy, are at least aged 55 years and are stem-cell transplant ineligible.

The overall survival benefit was significant, with median OS after 41.2 months of follow-up reaching 24.7 months in the CC-486 treatment arm, compared to 14.8 months for placebo an almost ten month improvement.

During a presentation of the results at the annual ASH 2019 conference, Andrew Wei, from the Alfred Hospital and Monash University, Melbourne, Australia, said: Based on the results of the QUAZAR study, we are excited about the clinical development of CC-486 and the potential to establish maintenance therapy as a new treatment paradigm for patients with AML in first remission.

Relapse-free survival also improved in patients treated with CC-486 10.2 months in the treatment arm compared to 4.8 months for placebo.

The study could have a practice-changing impact on the treatment of older AML patients who have a poor prognosis, according to Robert Brodsky, a professor of medicine at Johns Hopkins University in the US.

BMS took on the candidate following its acquisition of Celgene, which had already been developing the drug.

CC-486 is an oral version of Celgenes already-marketed chemotherapy Vidaza (azacitidine), which is administered intravenously it had been a top-selling product for the company, before being hit by generic competition in the US and Europe over recent years.

Vidaza brought in over $600m a year at its peak, as a treatment for AML and myelodysplastic syndrome (MDS), but fell to only $2m last year thanks to being pretty much entirely replaced by generics.

The new formulation could allow BMS to reclaim market share by moving the drug beyond its use as a first-line induction chemotherapy thanks to its oral dosing.

We are extremely encouraged by the results of the QUAZAR AML-001 study as a part of our continuing commitment to both epigenetic research and myeloid diseases, said Samit Hariwat, chief medical officer of BMS.

We now look forward to taking the next steps to bring CC-486 to eligible AML patients in need, he added.

According to a statement issued by BMS, regulatory submissions for CC-486 are planned for the first half of 2020.

The drug is also being tested in MDS, non-Hodgkins lymphoma and other cancers, both alone and in combination with checkpoint inhibitor drugs.

See the article here:
BMS' oral maintenance therapy boosts survival in AML patients - PMLiVE