Celator Pharmaceuticals Inc (NASDAQ:CPXX) announced positive response rate results in AML patients with FLT3 (FMS-like tyrosine kinase-3) mutation were presented at the European Hematology Association (EHA) 21st Annual Congress. The results are from the Company’s Phase 3 trial of VYXEOS™ (cytarabine: daunorubicin) Liposome for Injection (also known as CPX-351) in patients with high-risk (secondary) acute myeloid leukemia (AML).
Data, independently validated, from AML patients with a FLT3 mutation demonstrated VYXEOS had a statistically significant improvement in induction response rate (CR+CRi of 68.2% versus 25.0%; p=0.007). A benefit in induction response rate was seen in both FLT3-ITD and FLT3-TKD patients.
Response Rate (responders/number of patients)
An improvement in overall survival was also observed in FLT3 mutated patients with median overall survival of 10.25 months in the VYXEOS arm compared to 4.55 months in the 7+3 arm. The Hazard Ratio (HR) was 0.57 (p-value=0.093). In addition, preliminary data on NPM1 and CEBPα mutations were presented showing an improvement in response rate and overall survival in patients with these mutations. Final data for NPM1 and CEBPα mutations is expected to be presented at an upcoming medical conference.
“The improved response and survival observed in this exploratory analysis for VYXEOS over 7+3 in molecularly-defined AML subsets from the Phase 3 trial are promising, particularly for mutated FLT3 patients who typically have a poor prognosis,” said Bruno C. Medeiros, M.D., Associate Professor of Medicine, Stanford University Medical Center. “These clinical data corroborate previous ex vivo results suggesting increased sensitivity of FLT3 mutated AML blasts to VYXEOS and supports ongoing and prospective clinical studies in a broader range of AML cohorts.”
Approximately 20-30% of AML patients harbor some form of FLT3 mutation. The significance of the mutation, in any given patient, varies according to the nature of the mutation and the context in which it occurs. The most common type of mutation is an internal tandem duplication (ITD) mutation localized to a membrane region of the receptor, while point mutations in the tyrosine kinase domain (TKD) are less common. AML patients with a FLT3 mutation are believed to have a poorer prognosis than the overall population diagnosed with AML.
“We continue to learn more from the Phase 3 trial about the benefit of VYXEOS in AML patients,” said Scott Jackson, Chief Executive Officer of Celator Pharmaceuticals. “The results seen in patients with the FLT3 mutation are encouraging and generated the hypothesis that FLT3 mutated AML may be more sensitive to VYXEOS treatment and lead to improved clinical benefit. Further clinical trials are warranted in this patient population.”
The company expects to submit a New Drug Application (NDA) for VYXEOS with the U.S. Food and Drug Administration (FDA) by the end of the third quarter of 2016 and submit a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) in the first quarter of 2017.
The clinical trial was conducted in partnership with The Leukemia & Lymphoma Society® (LLS) through its Therapy Acceleration Program (TAP), which has supported the clinical development of VYXEOS beginning in Phase 2.
Phase 3 Trial Design
The randomized, controlled, Phase 3 trial (Protocol NCT01696084), enrolled 309 patients at 39 sites in the United States and Canada, and compared VYXEOS to the conventional cytarabine and daunorubicin treatment regimen (commonly referred to as 7+3) as first-line therapy in older (60-75 years of age) patients with high-risk (secondary) AML. Patients were stratified for age (60 to 69 and 70 to 75 years of age) and AML type; treatment-related AML, AML with documented history of myelodysplastic syndrome (MDS) with prior treatment with hypomethylating agent therapy, AML with documented history of MDS without prior hypomethlyating agent therapy, AML with a documented history of chronic myelomonocytic leukemia (CMMoL), and de novo AML with a karyotype characteristic of MDS.
Patients were randomized 1:1 to receive either VYXEOS or 7+3. Patients could receive one or two inductions, and responding patients could receive one or two consolidations. First induction for VYXEOS was 100u/m2; days 1, 3, and 5 by 90-minute infusion and for the control arm was cytarabine 100mg/m2/day by continuous infusion for 7 days and daunorubicin 60mg/m2 on days 1, 2, and 3 (7+3). Second induction for VYXEOS-treated patients was 100u/m2 on days 1 and 3, and the control arm was cytarabine 100mg/m2/day by continuous infusion for 5 days and daunorubicin 60mg/m2 on days 1 and 2 (5+2).
Only patients with documented CR or CRi were eligible to receive chemotherapy consolidation. Consolidation for VYXEOS-treated patients was 65u/m2 on days 1 and 3 and the control arm was cytarabine 100mg/m2/day by continuous infusion for 5 days and daunorubicin 60mg/m2 on days 1 and 2 (5+2). (Original Source)
Shares of Celator Pharmaceuticals closed yesterday at $30.13, down $-0.11 or -0.36%. CPXX has a 1-year high of $30.37 and a 1-year low of $1.12. The stock’s 50-day moving average is $19.52 and its 200-day moving average is $8.43.
On the ratings front, Celator Pharma has been the subject of a number of recent research reports. In a report issued on June 1, Janney Montgomery Scott analyst Ken Trbovich downgraded CPXX to Hold. Separately, on May 31, Roth Capital’s Joseph Pantginis downgraded the stock to Hold and has a price target of $30.25.
According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Ken Trbovich and Joseph Pantginis have a total average return of 62.8% and 7.4% respectively. Trbovich has a success rate of 41.2% and is ranked #100 out of 3884 analysts, while Pantginis has a success rate of 34.7% and is ranked #283.
Overall, 3 research analysts have assigned a Hold rating and one research analyst has given a a Buy rating to the stock. When considering if perhaps the stock is under or overvalued, the average price target is $30.25 which is 0.4% above where the stock closed yesterday.
Celator Pharmaceuticals, Inc. is a clinical-stage company, which engages in the research and development of biopharmaceutical products. It intends to provide chemotherapies and molecularly targeted agents to deliver anti-cancer activity and enhance treatment outcomes for cancer patients. It operates under the CombiPlex platform. Its products include VYXEOS, CPX-1, and CPX-8. The company was founded by Lawrence David Mayer and Marcel B. Bally in 1999 and is headquartered in Ewing, NJ.