Clinical Trials

Date: 2017-06-14

Type of information: Treatment of the first patient

phase: 2

Announcement: treatment of the first patient

Company: Mei Pharma (USA - CA) Helsinn Group (Switzerland)

Product: pracinostat

Action mechanism:

histone deacetylase inhibitor. Pracinostat is an oral histone deacetylase (HDAC) inhibitor that has been tested in a number of Phase I and Phase II clinical trials in advanced hematologic disorders and solid tumor indications in both adult and pediatric patients. Since August 2016, MEI Pharma has entered into an exclusive licensing, development and commercialization agreement with Helsinn for pracinostat for the treatment of acute myeloid leukemia and other potential indications.

Disease: myelodysplastic syndrome (MDS)

Therapeutic area: Cancer - Oncology

Country: USA

Trial details:

This two-stage, Phase 2 study will investigate a pracinostat dose of 45 mg, 25% lower than the dose used in study MEI-003, in combination with the standard dose of azacitidine to determine whether lowering the pracinostat dose in a higher risk patient population can improve the tolerance of the combination while achieving a clinically meaningful improvement in efficacy. The first stage of the study will be open-label, single arm in up to 40 patients to assess if the lower pracinostat dose will result in a discontinuation rate that approximates the rate that was observed with azacitidine alone in study MEI-003 (10%). If results from the first stage support expansion of enrollment, the second stage will be randomized and placebo-controlled to confirm the discontinuation rate in a blinded setting and to provide data on safety and efficacy. The primary endpoints of the study are 1) safety and tolerability and 2) overall response rate, defined as complete remission (CR), partial remission (PR) and marrow CR. Secondary endpoints include CR rate, overall hematologic improvement (HI) response rate, clinical benefit rate (defined as rate of CR + PR + HI + Marrow CR), rate of cytogenetic complete response/remission, duration of response, rate of leukemic transformation, event-free survival, progression-free survival and overall survival. (NCT03151304)

Latest news:

• On June 14, 2017, Helsinn and MEI Pharma announced that the first patient has been dosed in a Phase 2 dose-optimization study of pracinostat in combination with azacitidine in patients with higher risk myelodysplastic syndromes (MDS) who are previously untreated with hypomethylating agents (HMAs).
The two-stage study will be conducted at approximately 25 sites and is expected to enroll up to 120 patients with high and very high risk MDS per the Revised International Prognostic Scoring System (IPSS-R). The high and very high risk groups represent the highest unmet need in MDS, with median survival estimates of only 1.6 years and 0.8 years, respectively1. The cost of this study will be shared by Helsinn and MEI Pharma. Data from the first stage is expected in the first quarter of 2018.