White Plains, NY, March 14, 2016 – An investigational therapy supported by The Leukemia & Lymphoma Society (LLS) performed significantly better than the standard therapy in a Phase 3 clinical trial for patients with high-risk (secondary) acute myeloid leukemia (AML), its developer announced today. AML remains the deadliest of all blood cancers, and there have been no new therapies for nearly 40 years.
LLS funded the Phase 3 clinical trial through its Therapy Acceleration Program® (TAP), which supports promising projects and clinical trials through collaborations with biotechnology companies. LLS began partnering with Celator Pharmaceuticals, Inc. in 2009 to develop the therapy, CPX-351 (VYXEOS TM), an innovative formulation of two existing chemotherapy drugs used to treat AML, when it was still in Phase 2 clinical trials.
“The vision for our innovative Therapy Acceleration Program is becoming a reality with this promising potential therapy for patients with AML, as we are getting closer to addressing this urgent unmet need for blood cancer patients,” said Louis J. DeGennaro, LLS’s president and CEO. “From the start, LLS recognized the potential of CPX-351, so we are very gratified with the results of this clinical trial, and we are hopeful that this positive news brings us a step closer to delivering better outcomes for patients with high-risk (secondary) AML.”
LLS first launched TAP in 2007, recognizing that therapies were not getting to patients fast enough. Through TAP, LLS supports applied research and forges collaborations with biotechnology companies to help bridge the gap between discoveries in the laboratory and successful drug development. If CPX-351 receives FDA approval, it would mark the first time that LLS’s direct investment in a biotechnology company has resulted in helping to bring a drug into the health care arena, a significant achievement for LLS’s venture philanthropy initiative.
As the world’s largest voluntary agency dedicated to blood cancers, LLS is leading the offensive to change the paradigm of treatment for patients with AML, which remains one of the most lethal blood cancers. More than 25 percent of LLS’s budget is dedicated to AML research, and with the launch of its Beat AML initiative in 2013, LLS is fostering collaboration among researchers at multiple institutions, regulators, pharmaceutical and biotechnology companies, primary healthcare physicians and patients, to better understand the underlying causes of the disease and develop more effective therapies to treat patients with AML.
More about the CPX-351 clinical trial
The patients in the Phase 3 trial for CPX-351 were 60 to 75 years of age, newly diagnosed with AML, which they developed as a secondary cancer, either as a result of treatment for or history of a myelodysplastic syndrome (MDS), or other rare forms of myeloid malignancies. Overall, the median age of an AML patient is 67. While those with AML under the age of 60 have better treatment outcomes, collectively, AML patients across all risk categories face an especially grim prognosis, as 75-80% of patients die within five years. Those patients with high risk (secondary) AML have a worse prognosis, thereby accentuating the need for new therapy options. The standard of care for patients with AML has not changed in nearly 40 years.
The clinical trial successfully met its primary endpoint, demonstrating a statistically significant improvement in overall survival as compared to the conventional treatment regimen known as 7+3, which involves two chemotherapy drugs, cytarabine and daunorubicin. CPX-351 is a liposomal formulation of cytarabine and daunorubicin, and is designed to deliver a more optimal ratio of the drugs with less toxicity.
The median overall survival for patients treated with CPX-351 in the multi-site study was 9.56 months compared to 5.95 months for patients receiving 7+3 (HR=0.69, p=0.005). This represents a 31 percent reduction in the risk of death for patients treated with CPX-351. At 12 months, 41.5% of the patients were alive on the CPX-351 arm compared to 27.6% on the 7+3 arm. At 24 months, 31.1% of the patients were alive on the CPX-351 arm compared to 12.3% on the 7+3 arm.
Thirty-four percent of patients treated with CPX-351 went on to receive a stem cell transplant compared to 25 percent of patients treated with 7+3.
Based on these results, Celator expects to file a New Drug Application (NDA) for CPX-351 with the U.S. Food and Drug Administration (FDA) by the end of the third quarter 2016.
Celator will host a conference call and live audio webcast on Tuesday, March 15, 2016 at 8:00 a.m. EDT to discuss the results of the Phase 3 trial. To participate in the conference call, please dial 877-303-6316 (domestic) or 650-521-5176 (international) and refer to conference ID 71930208. The live webcast of the call can be accessed in the Investors section of Celator's website at www.celatorpharma.com. An archived webcast will be available on Celator's website beginning approximately two hours after the event.
Celator will present the data at the American Society of Clinical Oncology 2016 Annual Meeting in June.
See Celator’s news release for more details about the trial and the therapy.
About The Leukemia & Lymphoma Society
The Leukemia & Lymphoma Society ® (LLS) is the world's largest voluntary health agency dedicated to blood cancer. The LLS mission: Cure leukemia, lymphoma, multiple myeloma, and improve the quality of life of patients and their families. LLS funds lifesaving blood cancer research around the world, provides free information and support services, and is the voice for all blood cancer patients seeking access to quality, affordable, coordinated care.
Founded in 1949 and headquartered in White Plains, NY, LLS has chapters throughout the United States and Canada. To learn more, visit www.LLS.org. Patients should contact the Information Resource Center at (800) 955-4572, Monday through Friday, 9 a.m. to 9 p.m. ET.