Helsinn and MEI Discontinue AML Phase III Trial

 

Helsinn Group, based in Lugano, Switzerland, and MEI Pharma, headquartered in San Diego, announced they were discontinuing their Phase III trial of pracinostat with azacytidine in Acute Myeloid Leukemia (AML). The decision was made after an interim futility analysis by an Independent Data Monitoring Committee determined the trial was unlikely to meet the primary endpoint of overall survival (OS) compared to the control group. There were no safety issues reported.

Pracinostat is an oral histone deacetylase inhibitor. Azacytidine is a chemotherapy drug marketed under the brand name Vidaza by Celgene, a Bristol Myers Squibb company.

The drug is also being studied in a Phase II trial in patients with high or very high-risk myelodysplastic syndromes (MDS). Both the U.S. Food and Drug Administration (FDA) and the European Medicine Agency (EMA) granted pracinostat in combination with azacytidine Orphan Drug Designation for newly diagnosed AML patients who are equal than or older than 75 years of age or not eligible for intensive chemotherapy. The FDA also granted the combination Breakthrough Therapy Designation.

Helsinn and MEI Pharma inked the exclusive license, development and commercialization deal in August 2016 for pracinostat in AML and other possible indications. There were almost $500 million in milestone payments hanging on the trial.

MEI and Tokyo-based Kyowa Kirin announced on May 14, 2020, updated Phase Ib data on ME-401 for follicular lymphoma and other B-cell malignancies. ME-401 is an investigational oral phosphatidylinositol 3-kinase delta inhibitor. They presented the data in a poster session at the Virtual Edition of the 25^th European Hematology Association (EHA) Annual Congress held June 11 to June 14.

A day earlier, on May 13, Helsinn and MEI provided new data from the Phase II trial of pracinostat and azacytidine in high/very-high risk myelodysplastic syndromes. The study showed an estimated median OS rate of 23.5 months with a one-year overall survival rate of 77%. The median follow-up was 17.6 months and the overall response rate (ORR) was 33%, with all being complete responses (CR). The clinical benefit rate CR, mCR plus hematologic improvement (HI), mCR with no HI, or HI with no mCR, was 77%. Twenty-seven percent of the patients went on to receive a stem cell transplant while on the study. Only 11% discontinued treatment because of adverse events.

“Patients with high and very-high risk MDS currently have limited treatment options and poor outcomes,” said Ehab Atallah, Study Chair, Professor of Medicine, Medical College of Wisconsin, in May. “These data are promising and I continue to be encouraged by my experience to date with the combination of pracinostat and azacytidine evaluated in this study. The potential to offer patients a new combination treatment option in MDS is exciting.”

The Phase II trial is an open label, multicenter study evaluating a 45 mg dose of pracinostat in combination with the standard 75 mg dose of azacitidine in high/very high-risk MDS patients who had not previously received treatment with hypomethylating agents.

Helsinn focuses on cancer therapies. On June 10, it donated $100,000 to the American Society of Clinical Oncology (ASCO) Foundation’s Conquer Cancer, the foundation’s COVID Impacts Cancer fund. The campaign is an emergency initiative driven by Conquer Cancer to suport cancer patients, physicians and their care teams by offering educational resources and information to help patients receive care during and after the COVID-9 pandemic.

“We are pleased to support Conquer Cancer’s COVID Impacts Cancer fund, a great initiative, which will benefit cancer patients and oncologists during these unprecedented times,” said Riccardo Braglia, vice chairman and chief executive officer of Helsinn and board member of Conquer Cancer. “The work Conquer Cancer are doing to help the whole cancer community is vital and the ASCO Registry, in particular, will be invaluable to help physicians learn about the impact of COVID-19 on cancer patients and ensure we are well placed to protect these vulnerable patients should any future pandemics occur.”