Phase 3 Trial Results for High-Risk AML Investigational Therapy Look Promising, LLS Announces

Phase 3 Trial Results for High-Risk AML Investigational Therapy Look Promising, LLS Announces

The Leukemia & Lymphoma Society (LLS) financially supported a Phase 3 clinical trial of an investigational therapy, CPX-351, for patients with high-risk acute myeloid leukemia (AML). The organization is now announcing the first results, which suggest that the novel therapy significantly outperformed the standard therapy.

AML is the most life-threatening of all blood cancers, with no new therapies in more than four decades of research. This trial was funded through the LLS’s Therapy Acceleration Program (TAP), which helps promising projects and clinical trials advance their projects through financial collaborations with several biotech companies.

In 2009, TAP supported Celator Pharmaceuticals in its development of CPX-351 (Vyxeos), an innovative formulation of two prior chemotherapy drugs combined for AML treatment. At the time, the drug development was still in Phase II clinical trials.

The Phase III trial for CPX-351, “Phase III Study of CPX-351 Versus 7+3 in Patients 60-75 Years Old With Untreated High Risk (Secondary) Acute Myeloid Leukemia (301),” included patients aged 60 to 75 who had recently received an AML diagnosis as a secondary cancer development – either as a result of a treatment for or history of a myelodysplastic syndrome (MDS), or other myeloid malignancies.

While AML patients under the age of 60 would have better treatment outcomes, all AML patients face a poor diagnosis, with only 20-25 percent of patients surviving past the first five years of diagnosis. When AML is secondary, the prognosis is even worse, which is why it is more urgent to find therapy options for these patients.

The trial was successful in meeting its primary endpoint, showing a significant improvement in overall survival rate compared to the standard 7+3 treatment regimen which involves two chemo drugs, cytarabine and daunorubicin.

The novel CPX-351 is a liposomal formulation of cytarabine and daunorubicin designed to deliver an optimal combination of the two drugs, with less toxicity.

The median overall survival for patients who received CPX-351 treatment was 9.56 months compared to 5.95 months for those who received 7+3. This is a 31 percent reduction in the risk of death in patients treated with the novel drug.

After one year, 41.5 percent  of CPX-351 treated patients were alive, compared to 27.6 percent of 7+3 treated patients. At two years, 31.1 percent of CPX-351 patients were alive, compared to 12.3 percent of 7+3 treated patients. And, 34 percent of patients who received CPX-351 treatment went on to receive a stem cell transplant compared to 25 percent of patients treated with the combo chemo drugs 7+3.

Based on these results, Celator is now preparing to file a U.S. FDA New Drug Application (NDA) for CPX-351 by the second half of 2016. If CPX-351 is granted FDA approval, it will be the first time that an investment made directly by LLS in a biotech will have resulted in the advancement of a new drug into the healthcare arena — a significant achievement for LLS’s venture philanthropy initiative.

The company will present data on these results at the American Society of Clinical Oncology 2016 Annual Meeting in June.

“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,” LLS President and CEO Louis J. DeGennaro said in a press release.

“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,” he said.

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