The Immune System in Drug Development

Understanding the human immune system has become increasingly important to drug development in the biotech era, as immune-mediated diseases are targeted (e.g. autoimmune diseases and sepsis) and the immune system is harnessed to elicit a therapeutic effect (e.g. vaccines and cancer immunotherapy).

It has become clear that the immune system is involved in complex ways in the pathogenesis of all disease. This complexity highlights the shortcomings of commonly used animal models (such as murine models) to study immunopathogenesis and therapeutic modulation of the human immune system. A new solution is needed to translate research to therapy.

To help bridge the gap, Synexa has develop a proprietary approach called a ProtoTrial.

What is a ProtoTrial?

A ProtoTrials is an in vitro GCLP translational immunology study performed on samples from human patients and healthy volunteers. ProtoTrials are adaptive and collaborative in nature and typically assesses drugs targeting an immune mechanism.

ProtoTrials can be used to:

  • Allow early insight into the efficacy, safety and MOA of a drug.
  • Refine target indication.
  • Generate serendipitous discoveries of interesting aspects of drug performance.
  • Focus the clinical biomarker panel and analytical techniques.
  • Benchmark the candidate drug against other drug leads and commercially available competitor products.

Illustrative Case Studies:

  • Assessment of a product’s risk of stimulating a cytokine response syndrome.
  • Quantification of an immunomodulator's effect on nTreg and iTreg (Tr1, Tr35 & Th3) subsets in patients with rheumatoid arthritis, psoriasis and inflammatory bowel disease.
  • Evaluation of a selection of PI3Kδ inhibitors based upon their ability to inhibit Basophil degranulation.
  • Assessment of an anti-inflammatory’s ability to inhibit the Th17 pro-inflammatory response.
  • Cellular immune biomarker discovery in patients with Lupus Nephritis.
  • Discovery, development and validation of cellular immune biomarkers (expressed on monocytes) for a non-opioid treatment of chronic neuropathic pain.
  • Identification and stabilization of bronchoalveolar lavage derived monocyte biomarkers in idiopathic pulmonary fibrosis.
  • In vitro bioequivalence study of the anti-inflammatory and anti-bacterial potency of a generic formulation of Ciprodex.
  • Quantification of the relative risk for TB reactivation across a range of biosimilar TNF alpha inhibitors.