Novel Mast Cell Therapy Platform

NOVEL MAST CELL THERAPY PLATFORM

SCIENCE

Mast cells are the oldest and one the most crucial immune cells that are distributed in all tissues and organs including, for example, the gastrointestinal tract, brain, lungs, skin, and cardio-vascular system. Mast cells play a key role in orchestrating our immune system with more than 600 receptors and the storage, formation and selective (differential) release of more than 200 different mediators. Disorders of the mast cell function, especially excessive and abnormal activation (more and more driven by polygenetic and epigenetic dysregulation),
lead to severe, often chronic diseases of the human organism which are meanwhile recognized as the “Modern Epidemic of Chronic Illness“. Prominent examples are systemic mast cell activation disease (MCAD) including its variant rare disease systemic mastocytosis, irritable bowel syndrome (IBS), fibromyalgia, chronic fatigue syndrome, asthma, food allergies and fibrotic diseases.

Based on 25 years of research, MC Sciences has made a groundbreaking discovery to develop mast cell-targeted therapies. MC Science’s key asset is the discovery of a novel mast cell target that acts as a master-regulator to control mast cell activity and a corresponding clearly identified pharmacophore that inhibits activated mast cells representing a so far undescribed and already clinically validated mechanism of action (so far unpublished). MC Sciences development plan aims at establishing a novel therapy platform and creating a new class of small molecule substances that selectively target mast cells.
Currently, there is no adequate treatment available for mast cell-induced diseases leading to often disabling symptoms in patients. The key to an effective treatment would be the selective inhibition of the abnormal activated mast cells without causing immunosuppressive side effects, as well as, in addition reducing accumulated activated mast cells in a controlled manner. However, the past decades up of therapeutic treatments and current drug developments have not yet solved the key challenge: a clinically significant inhibition of mast cell activity via single receptors cannot be achieved because induced inhibitory effects are compensated by redundant pathways within the mast cell.

For the first time, MC Sciences has discovered a novel target that allows to deactivate activated mast cells completely in a dose-dependent manner – basically “pulling the plug” but only selectively on activated mast cells that cause the trouble.

ADVANTAGES

Our novel inhibition of abnormally activated mast cells possesses the following significant advantages:

  • Complete inhibition of mast cell activity – meaning inhibition of all signaling pathways and complete prevention of mast cell mediator release (no activation of redundant pathway possible)
  • Dose-dependent and therefore controlled deactivation of mast cell activity
  • Selectively targeting activated mast cells without immunosuppressive side effects: resting mast cells continue in normal function (“normalization of immune system”)
  • Rapid onset of therapeutic effect within 60 minutes
  • Effective on all tissue-specific mast cell types
  • In humans clinically proven mechanism of action (unpublished)
  • Easy oral application
  • Symptom-dependent individual dosing to meet individual patient’s needs
  • Expected very favorable safety profile – possible side effect might include, for example, a reduced mobility of the gastro-intestinal tract

FIRST-IN-CLASS NOVEL MAST CELL THERAPY PLATFORM

MC Sciences is worldwide in a unique position to secure a first-in-class novel mast cell therapy platform. Based on the novel mast cell target and corresponding pharmacophore to control mast cell activity, our aim is to develop the following small molecules:

MCS1 Series:
Controlled complete inhibition of mast cell activity.

MCS10XX:
Lead compound series as complete inhibitors of mast cell activity with high effectivity and safety profile. Expected higher affinity and potency with equal or improved safety profile compared with MCS1010.

MCS1010: 
Is a derivative of our lead compound as highly effective inhibitor of mast cell activity with a favorable safety profile potenially targeted for drug repurposing for new disease indications with potential for accelerated approval due to high unmet medical needs.

MCS2 Series:
Selective reduction of accumulated abnormal activated mast cells in controlled dose-dependent manner as temporary curative approach:

  • Option 1: via irreversible binding of MCS10XX resulting in natural apoptosis of accumulated activated mast cells
  • Option 2: as pro-drug using MCS1 as carrier for a specific compound to selectively induce apoptosis in activated mast cells

MCSX Series in Precision Medicine:
Precise delivery of substances to control the behavior of mast cells, e.g. in immuno-oncology.