Positive Phase III results for Vyxeos (cytarabine + daunorubicin) liposome for injection to treat acute myeloid leukaemia- Celator Pharma

Celator Pharmaceuticals announced positive results from the Phase III trial of Vyxeos (cytarabine + daunorubicin) liposome for injection (also known as CPX-351) in patients with high-risk (secondary) acute myeloid leukaemia (AML) compared to the standard of care regimen of cytarabine and daunorubicin known as 7+3. The trial met its primary endpoint demonstrating a statistically significant improvement in overall survival.

Data will be submitted for presentation at the American Society of Clinical Oncology 2016 Annual Meeting. The median overall survival for patients treated with Vyxeos in the study was 9.56 months compared to 5.95 months for patients receiving 7+3, representing a 3.61 month improvement in favour of Vyxeos. The hazard ratio (HR) was 0.69 (p=0.005) which represents a 31% reduction in the risk of death versus 7+3. The proportion of patients alive 12 months after randomisation was 41.5% in the Vyxeos arm compared to 27.6% in the 7+3 arm. The proportion of patients alive 24 months after randomisation was 31.1% in the Vyxeos arm compared to 12.3% in the 7+3 arm.

Vyxeos also demonstrated a statistically significant improvement in induction response rate (CR+CRi of 47.7% versus 33.3%; p=0.016) and this significance was maintained for the analysis of CR alone (CR of 37.3% versus 25.6%, p=0.040). Sixty-day all-cause mortality was 13.7% versus 21.2%, in favour of patients treated with Vyxeos. No substantial difference in Grade 3 or higher adverse events was observed between Vyxeos and 7+3. In the intent-to-treat population, Grade 3 or higher haematologic adverse events were similar for overall infections, febrile neutropenia, and bleeding events. In the intent-to-treat population, Grade 3 or higher non-haematologic adverse events were similar across all organ systems, including cardiac, gastrointestinal, general systems, metabolic disorders, musculoskeletal, nervous system, respiratory, skin and renal.

Comment: A significant competitor is AG 221 from Celgene which has just started Phase III trials.

Comment: Based on these Phase III results Celator expects to submit a New Drug Application (NDA) for Vyxeos with the FDA later this year and submit a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) in the first quarter of 2017.