Microparticle Platform

Innate Immunotherapeutics' core technology is a unique therapeutic microparticle which induces the human immune system to fight certain cancers and infections, or turns off certain immune mechanisms which lead to autoimmune diseases such as Multiple Sclerosis (MS). This same technology can also be used in the design of better vaccines to potentially prevent or treat diseases such as influenza, cancer, malaria, or tuberculosis.

Background - The Immune System

Unlike most pharmaceutical agents (drugs or monoclonal antibodies), immunomodulators like MIS416 do not act directly on the target (cancer tumor, infectious agent, damaged nerve), but instead switch on powerful disease fighting mechanisms that form part of the human immune system. The immune system is a collection of biological barriers and processes that protects against disease by identifying and killing external threats such as infectious agents (bacteria, viruses, parasites, other pathogens) and internal threats such as cancer tumor cells. To function properly, the immune system needs to distinguish between threats and the body's own healthy cells. When this ability to distinguish between non-self and self breaks down, the immune system can attack healthy cells resulting in one of several autoimmune diseases.

The immune system comprises several layers of defense. The first line of defense consists of physical barriers such as the skin and mucous membranes that line the digestive, respiratory, and reproductive tracts. For infection to occur, pathogens must first breach this physical barrier. When such a breach does occur, the innate immune system is the next line of defense - 'innate' because all animals naturally possess it from birth. The surveillance cells of the innate immune system firstly recognize the signature (or pattern) of an invading pathogen and then activate appropriate attack cells or mechanisms to clean out the invader. This same surveillance and response process also works for cells that go bad, e.g. early stage cancer cells. If the innate system is overwhelmed, the adaptive immune system is triggered, providing the last, but often the most potent layer of immune defense. The cells that form part of the adaptive response (antibodies and killer T-cells) must be custom-made to match the pathogen and so the process is relatively slow, but once designed, these cells can be made in huge quantities to overwhelm the threat. Once the system is adapted to recognize and destroy a particular invader, it remembers that invader, and can then react more quickly next time the invader is encountered.

In Summary, the innate system is pivotal to the successful operation of the entire human immune system. It recognizes trouble, provides an immediate general (or non-specific) defense and if required, gives permission to the adaptive system to respond. Importantly, it also regulates the overall reaction to ensure that the level of response is appropriate to the degree of the threat.

By activating and/or regulating important innate system mechanisms, Innate Immunotherapeutics' microparticle immunomodulator technology presents a new and effective way to safely fight infections, certain cancers, and to treat select autoimmune diseases.

Technical Description

Innate Immunotherapeutics has designed, manufactured, and patented a cost-effective immunomodulator microparticle technology which comprises multiple pathogen-associated molecular pattern recognition receptor ligands cross-linked to a stable microparticle that act in a safe, well defined, and synergistic manner. The novel cross linking of these multiple immunostimulatory ligands to form a microparticle exploits cellular uptake mechanisms to achieve reliable delivery to those immune cells responsible for the induction of both innate and adaptive immunity.

The first clinical candidate manufactured using this technology is MIS416. When administered as a distinct agent, MIS416 is a potent activator of broad but well characterized innate immune responses.

Schematic of the lead drug candidate - MIS416
The immunomodulator microparticle (~0.5 x ~2.0 micron)
with cross linked NOD-2 ligands (muramyl dipeptide repeats -in green)
and covalently attached TLR9 ligands (protected bacterial nucleic acid - in blue)