On December 19, 2024 Modiblast GmbH, a preclinical-stage biopharmaceutical company developing novel immunomodulatory cancer therapies, reported the publication of novel preclinical in vivo data and initial human data validating its therapeutic strategy in the peer-review International Journal of Molecular Sciences. The data sets demonstrate the therapeutic potential of the patented approach aimed at the in vivo reprogramming of myeloid leukemic blasts into antigen-presenting dendritic cells of leukemic origin (DCleu) to boost adaptive and innate anti-cancer immunity. The full publication is available for download via the IJMS website.
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"The preclinical data sets published today further substantiate our previous work and validate our novel approach from various angles. Even more so, the addition of first patient data from off-label use significantly derisks the further development path and provides us with strong guidance for designing the upcoming clinical trial," commented Prof. Dr. rer. nat. Helga Schmetzer, Managing Director and Founder of Modiblast. "Overall, we continue to make steady progress in advancing our lead program MB101 towards an IND-ready state and plan to accelerate the development towards the initiation of clinical trials once backed by a new financing round in 2025."
Modiblast’s therapeutic approach combines two synergistic factors to turn ‘foe’ into ‘friend’. A myeloid differentiation factor fosters the creation of dendritic cell progenitor cells and hematological recovery. The combination with a danger signal and maturation factor drives the formation of dendritic cells (DCs). Both signals combined trigger the reprogramming of cancer blast into DCleu’s and boost the creation of healthy DCs. The first program, MB101, aims for a fixed-dose combination of granulocyte-macrophage colony stimulation factor (GM-CSF), and prostaglandin E1 (PGE1) as a ‘danger signal’.
In the preclinical portion of the new publication, MB101’s therapeutic effects were evaluated in an established animal acute myeloid leukemia (AML) model. After 9 days of treatment, DCleu and memory-like T cells increased in the peripheral blood, whereas regulatory T cells and especially blasts decreased in treated as compared to untreated control animals. A significant reduction of blasts compared to the control group was seen in both spleen and blood following treatment with GM-CSF and PGE1.
Based on the confirmatory preclinical data package generated for MB101’s drug combination to date, two heavily pre-treated and refractory AML patients received MB101 treatment on an individualized basis over a 4-week period. A third, untreated patient with similar clinical parameters served as a control. The treatment was shown to be safe, leading to neutrophil recovery without increasing blast counts. At the same time, immune cells of the adaptive and innate lines were activated and gave rise to memory as well as to antileukemic cells. One of two patients treated showed a rapid recovery of platelets, a postulated synergistic effect of the combination of GM-CSF and PGE1, which made the patient independent of platelet transfusions.
The full publication "In vivo Induction of Leukemia Specific Adaptive and Innate Immune Cells By Treatment of AML-Diseased Rats and Therapy-Refractory AML-Patients with Blast Modulating Response Modifiers" can be downloaded on the IJMS website or via our Publications section.
(Press release, Modiblast, DEC 19, 2024, https://www.modiblast.com/modiblast-publishes-preclinical-in-vivo-data-and-initial-human-data-on-novel-cancer-cell-reprogramming-approach-in-aml/ [SID1234662150])