On May 13, 2020 Helsinn Group, a Swiss pharmaceutical group focused on building quality cancer care and rare disease products, and MEI Pharma, Inc. (NASDAQ: MEIP), a late-stage pharmaceutical company focused on advancing potential new therapies for cancer, reported new data from the Phase 2 study evaluating pracinostat, an oral pan-histone deacetylase inhibitor (HDACi), in combination with azacitidine in patients with high and very-high risk myelodysplastic syndromes previously untreated with hypomethylating agents (Press release, MEI Pharma, MAY 13, 2020, View Source [SID1234557923]). The study results will be featured in a poster at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 Virtual Scientific Program.
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The new data from the Phase 2 study (n=64) demonstrated an estimated median overall survival (OS) rate of 23.5 months with a 1-year OS rate of 77%. The median follow-up was 17.6 months (range, 15.7–18.8) and the overall response rate (ORR) was 33% (21/64), all of which are complete responses (CR). The clinical benefit rate (CR, mCR plus hematologic improvement [HI], mCR with no HI, or HI with no mCR) was 77% (49/64). Twenty seven percent of patients (17/64) proceeded to a stem cell transplant while on study. Eleven percent of patients discontinued treatment because of adverse events. The most common grade ≥3 treatment emergent adverse events were hematologic, and included decreased neutrophil count (50%), anemia (39%), febrile neutropenia (34%), decreased platelet count (33%), thrombocytopenia (27%), and decreased white blood cell count (20%).
"Patients with high and very-high risk MDS currently have limited treatment options and poor outcomes," stated Ehab Atallah, M.D., Study Chair, Professor of Medicine, Medical College of Wisconsin. "These data are promising and I continue to be encouraged by my experience to date with the combination of pracinostat and azacitidine evaluated in this study. The potential to offer patients a new combination treatment option in MDS is exciting."
The poster, titled "Phase 2 study of lower-dose pracinostat plus azacitidine safety and efficacy in patients with high/very high-risk myelodysplastic syndromes," will be included in a poster session at the ASCO (Free ASCO Whitepaper) Virtual Scientific Program and will be available for on-demand viewing online beginning on May 29, 2020 at 8:00 a.m. EDT at View Source The poster will also be available for download via the MEI Pharma website.
The Phase 2 Study
The Phase 2 study is an open label, multicenter trial investigating a 45 mg dose of pracinostat in combination with the standard 75 mg dose of azacitidine in patients with high and very high-risk MDS who are previously untreated with hypomethylating agents.
The primary endpoints were safety and tolerability of pracinostat in combination with azacitidine and ORR, defined as CR plus partial response (PR). Overall survival was a secondary endpoint. All efficacy evaluable patients have been followed for at least one year to evaluate safety and efficacy.
About Higher Risk MDS
Higher risk MDS (high and very high risk in the IPSS-R classification) is a serious medical condition, with median survival of less than 18 months. The only curative therapy is allogeneic stem cell transplantation (SCT), however most patients with MDS are not candidates for SCT given their typically advanced age, comorbidities, and lack of a suitable donor. Standard therapy with HMAs in higher risk MDS provides modest responses, though azacitidine has been shown to improve survival when compared to conventional care regimens. Patients who do not respond to HMAs or progress after therapy with HMAs have a very poor outcome, with a median survival of less than one year.
About Pracinostat
Pracinostat is an oral histone deacetylase ("HDAC") inhibitor that is in a pivotal Phase 3 PRIMULA study in combination with azacitidine for the treatment of adults with newly diagnosed acute myeloid leukemia ("AML") who are unfit for intensive chemotherapy. It is also being evaluated in a Phase 2 study in patients with high or very high-risk myelodysplastic syndrome ("MDS"). The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted Orphan Drug Designation for pracinostat in combination with azacitidine for the treatment of patients with newly diagnosed AML who are ≥75 years of age or unfit for intensive chemotherapy. In addition, the FDA has granted Breakthrough Therapy Designation to the combination treatment in AML.
In August 2016, Helsinn and MEI Pharma entered an exclusive license, development and commercialization agreement for pracinostat in AML and other potential indications. The agreement provides that Helsinn is primarily responsible for development and commercialization costs for pracinostat in AML and other indications, including MDS.
Berlin Chemie AG, the German company of the Menarini Group, has been granted exclusive licensing rights to commercialize pracinostat worldwide (excluding US, Canada, Japan and South America). Blanver has been granted the rights to register, promote, distribute and commercialize pracinostat in Brazil, while Varifarma, located in Buenos Aires, covers Argentina, Bolivia, Chile, Colombia, Ecuador, Paraguay, Peru, and Uruguay. Endo Ventures Limited and Paladin Labs Inc., an operating company of Endo, are responsible for the registration, distribution, sales, marketing, medical affairs, pricing and reimbursement activities in connection with pracinostat in Canada.
Pracinostat is an investigational agent and is not approved for commercial use in the U.S. or any other country worldwide.