Medigene AG

Interim analysis from ongoing Phase I/II clinical trial with DC vaccines in AML patients presented at EHA - Seite 2

Interim results: The vaccinations were well tolerated with no serious adverse events (SAEs) related to the treatment. The most common adverse events (AEs) were injection site related, accounting for 35% of all AEs and mild in nature (Grade 1). The feasibility of the manufacture of the dendritic cell vaccine in these chemotherapy-pretreated subjects was already presented at AACR in 2018 (Link to poster: https://www.medigene.de/fileadmin/download/ab-stracts/180414_AACR_2018 ...).

After a 12-months treatment period, the overall survival was 89% (18 of 20 patients, 95% confi-dence interval: 61 to 97%) and the progression free survival was 60% (12 of 20 patients, 95% confidence interval: 36 to 78%). Most relapses, 5 out of 8, occurred within the first 80 days after initiation of vaccination, suggesting a possible molecular relapse upon entering the study.

The presence of common mutations was analyzed in all patients via next generation sequencing and two of the five early relapses showed mutational load upon entering the study (IDH2 and DNMT3A mutation in one patient and multiple mutations in the second (RUNX1, KRAS, ETV6, BCOR, DNMT3A). Two of the later relapses also showed mutational load before vaccination, one patient with a KRAS and TET2 mutation and one patient with multiple mutations (RUNX1, IDH2, SRSF2 and DNMT3A).

Dr. Kai Pinkernell, CMO and CDO of Medigene AG, comments: " We are pleased that the interim results after one year of treatment show an excellent safety profile, a very good manufacturability and encouraging overall and progression free survival data. In addition, the mutational load analysis may also allow patients to be stratified in future clinical trials."

The completion of the ongoing trial is scheduled for the end of 2019, following a two-year treatment period.

About acute myeloid leukaemia (AML): Acute myeloid leukaemia is a malignant disease of the hematopoietic system, affecting mainly adults above 60 years of age. In Germany, about 3,600 cases are registered annually.

AML is caused by uncontrolled growth of dysfunctional hematopoietic precursor cells in the bone marrow. These cells prevent the generation of normal blood cells, causing a decrease in erythrocytes and platelets, for example. Typical symptoms of AML include anemia, fever, increased risk of infection, and bleeding. AML progresses rapidly and may be fatal within a few weeks or months, if untreated.