Modern medicine emphasizes cholesterol when assessing obesity related disease progression. However, researchers are now focusing on triglycerides in these processes and its central regulatory protein called Apolipoprotein CIII.
An Orphan Drug is a specific designation that provides a drug sponsor with unique benefits for expedited review, access to additional government funding and an exclusivity period for the treatment of its designated Orphan Disease.
Precision Medicine ties the use of a therapeutic to a specific biological marker (genetic or protein), specifically associated with a disease. This significantly reduces developmental cost and improves program success.
ApoCIII is a small liver derived protein that is a component of multiple lipoprotein particles (fat transport particles in the blood stream). These particles shuttle dietary and liver produced fat around the body for use in hormone production, energy and storage. However, ApoCIII has some very specific functions that have recently brought it into the scientific spotlight as a key regulator of fat metabolism and disease progression.
Hyperlipidemia is simply the excess of fat in the blood. There are two types of hyperlipidemia:
1) Hypercholesterolemia - Excess blood cholesterol.
2) Hypertriglyceridemia - An excess of saturated fats in the blood.
Western medicine focuses on the cholesterol issue, however, hypertriglyceridemia is becoming understood as equally problematic.
The leading cause of hyperTG is poor diet and sedentary lifestyle. Closely tied to obesity, the risks associated with prolonged hyperTG have been largely overlooked. The current epidemiological data indicate that from 2010 – 2020 there will be a modest rise (3.8 Million) in new U.S. hypercholesterolemia cases, while new cases of U.S. hypertriglyceridemia will see a rise by 16 Million. These numbers warrant concern in the current treatment methods of hyperTG and hyperlipidemia as a whole.
An orphan disease is defined defined by the Food & Drug Administration (FDA) as a condition that affects fewer than 200,000 people nationwide.
The Orphan Drug Designation program provides orphan status to drugs and biologics which are intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that meet the Orphan Disease definition.
With the receipt of an orphan drug designation, there are several benefits afforded to the drug sponsor. These are:
Even though our developing lead candidate drug (iMBP-001) has much broader application potential, the iMBP Management Team has elected to develop iMBP-001 as an Orphan Disease Drug. For this, the Rare disease called Familial Chylomicronemia Syndrome (FCS) has been selected. This indication is defined as a rare genetic disorder (occurrence rate is 1:1,000,000) that results in the decreased expression of Lipoprotein Lipase (LPL). LPL is the main enzyme that digests Triglycerides in blood, but it is also inhibited by ApoCIII. Therefore, normal levels of ApoCIII in FCS patients results in significantly increased blood Triglyceride levels which can lead to premature cardio metabolic disease and the development of a whole host of debilitating conditions.
The tailoring of medical treatment based upon characteristics that can classify patients into subpopulations that differ in their susceptibility to a particular disease, prognosis of a developing disease, or response to a specific treatment. Precision Medicine allows for more precise and cost effective preventative and therapeutic interventions as efforts can be concentrated on those patients who will respond the best.
A therapeutic product for which the label includes guidance on decisions and/or procedures for their use in individual patients based upon specific biological markers, identified by diagnostic tools.
The use of PM in Drug Discovery and Development has significantly increased in prevalence over the past decade. With the repeated demonstration of success in gaining regulatory approval, there is now enough statistical evidence to clearly illustrate the benefit of using a PM approach. The net result is that the success rate of achieving regulatory approval from Phase 1 (first in human) clinical trials increases from a paltry 8.4% using conventional methods, to 25.9% by utilizing a PM drug development plan.
As the primary indication of iMBP-001 is Familial Chylomicronemia Syndrome, there are clear genetic markers that show the down regulation of functional LPL in that patient. It is these markers that will be used for patient selection with the goal of improved clinical outcome.