Lymphocyte Trafficking

(Calix)

Immune System

TRIAL PHASE
MECHANISMINDICATION(S)PRODUCT NAMEPreclinicalPhase 1Phase 2Phase 3
Oral Administration of Biologics – Milk-Derived Exosomes
Immune Disorders
Calix

Immune System

Oral Administration of Biologics – Milk-Derived Exosomes
INDICATION(S):Immune Disorders
NAME:Calix
STAGE:Preclinical
Oral Administration of Biologics – Milk-Derived Exosomes

We are developing an exosome-based technology for the oral administration of biologics, nucleic acids, and complex small molecules. Milk exosomes represent a significant opportunity to potentially resolve the long-standing challenge of oral bioavailability of macromolecules and complex small molecules.

Our novel milk exosome-based technology may be uniquely positioned to permit oral administration of oligonucleotide-based therapeutics and other nucleic acid-based therapeutics such as mRNA, siRNA, antisense oligonucleitids, CRISPR nucleic acids. By advancing our industry-leading exosome platform, we are developing a robust therapeutic pipeline for the oral administration of macromolecules.


  • Patient Need & Market Potential
    • Biologics, such as proteins, peptides and antibodies, are the fastest growing segment of the pharmaceutical business
    • Nucleic acid-based therapies, such as RNAi, antisense and mRNA, represent the next big wave of biologics. Yet despite decades of efforts, almost all biologics are still administered via intravenous or subcutaneous routes
    • Oral administration of biologics has remained elusive. Some of the challenges for oral administration of biologics include low permeability of epithelial cells lining the GI tract, low stability in the harsh stomach environment result in significant enzymatic and pH-dependent degradation of biologics in the stomach and intestine. 
    • None of the current drug administration approaches harness natural trafficking systems within the body to transport biologics through the GI tract and into systemic circulation. The current approaches all impose stress and toxicity on the system
    • These trafficking and safety limitations often in turn limit applications of biologics such as nucleic acids to a very small number of specific tissues or organs, such as the liver, and to specific modalities, such as mRNA vaccines
    • By contrast, nature has designed safe, systemically distributable packets of information for communication using exosomes
  • Our Approach to Solving the Problem
    • Milk-derived exosomes, in particular, represent a mechanism designed by nature to enable transmission of messages (proteins, nucleic acids, small molecules) specifically through the oral route to systemic circulation and key tissues
    • Our approach harnesses milk-derived exosome trafficking for oral administration of biologics, including nucleic acids, peptides and proteins, to different parts of the body beyond the liver. Key sites beyond the liver that can potentially be targeted by this technology include the GI system, kidneys, lungs, immune cells, cardiac tissues, and lymph nodes
    • We believe that harnessing the underlying biology of milk-derived exosomes can be disruptive and potentially enable us to treat serious diseases, such as autoimmune, inflammatory disorders, cancer, diabetes, liver diseases, cardiac diseases, and lung disorders in previously unachievable ways by enabling the safe oral administration of complex, biologic payloads
  • Collaborations
    • Our Lymphocyte Trafficking technology is based on the pioneering research of Ramesh Gupta, PhD, Founder of 3P Biotechnologies, and Professor and Agnes Brown Duggan Chair in Oncological Research, James Graham Brown Cancer Center, and Department of Pharmacology and Toxicology, University of Louisville.
  • Expected Milestones and Timing
    • Our pivotal ADHD study, which assesses the efficacy and safety of Project: EVO in patients with pediatric ADHD, is expected to read out in the second half of 2017, with potential subsequent FDA filings as a digital treatment for ADHD. The trial was designed, with significant input from the FDA, to serve as a registration trial 
    • We are currently planning on or conducting multiple additional clinical trials across a variety of patient populations including autism spectrum disorder, depression, Alzheimer’s disease, multiple sclerosis, Parkinson’s disease and traumatic brain injury

Exosomes (small vesicles released by cells in nature for cell-cell communication), which contain mixtures of lipids, proteins and nucleic acids, play a critical physiologic role in intercellular communication and the transport of macromolecules between cells and tissues. Mammalian-derived exosomes have attractive potential as vehicles for the administration of a variety of drug payloads, especially nucleic acids, since their natural composition will likely provide superior tolerability over the variety of synthetic polymers currently in use.