Type 1 Diabetes (T1D) represents a significant commercial opportunity for Mercia. There are more than 2 million patients with T1D in the U.S. with about a 3% annual increase in its incidence worldwide. T1D patients eligible for treatment with MER3001 can be categorized into the following three market segments:
1. Newly diagnosed T1D – This group has ~20% residual beta cell function at the time of diagnosis and are likely to show benefit from stabilization of pancreatic beta cell destruction. In the U.S., the incidence is approximately 35,000 new T1D patients each year, projected to increase by 3% annually. As treatment is expected to be life-long, the pool of patients eligible for treatment will expand annually by at least 35,000. Additionally, 30% of younger patients diagnosed with type 2 diabetes and 10% of persons over age 35 diagnosed with type 2 diabetes may have an underlying autoimmune component which could be addressed with the MER3001.
2. Established T1D – Even though these patients are on insulin therapy, they retain capacity for regenerating functional beta cell activity. In the active disease state, this potential is insufficient to overcome the ongoing loss of beta cells; however, immunotherapy would permit beta cell regeneration and concomitant restoration of clinically significant insulin production. In addition, it is also likely that T1D immunotherapy will be beneficial when used with patients who receive transplanted beta islet cells and islet cell regeneration therapies.
3. High risk individuals– The average risk of a child developing T1D is 6% if either of the child’s parents or siblings has the disease. This represents an additional market opportunity of 1.3 million individuals for all age groups in the U.S. in 2007. A strong case can be made for early screening, coupled with prophylactic immunotherapy, which would go a long way towards mitigating the risk for these individuals.
T1D results from a progressive destruction of insulin-producing pancreatic cells which come under autoimmune attack when an imbalance develops between the body’s own CD4+ and CD8+ T cells. Mercia’s MER3001 for T1D, which is based upon the insulin B chain, provides a robust autoantigen-specific stimulus to the patient’s immune system to rebalance the autoaggressive/regulatory equilibrium.
Pre-clinical and clinical studies demonstrate that MER3001 has the desired properties for a successful T1D therapy.
MER3001 induces an autoantigen specific Th2-type response including stimulation of regulatory T-cells and regulatory cytokines to downregulate the Th1 autoimmune response, promoting a targeted rebalance of the immune system. Compared with placebo control, MER3001 induced a robust immune response in all patients treated following a single dose.
Evidence of clinical efficacy has been established for MER3001 in newly diagnosed T1D patients.
C-peptide levels following mixed meal stimulation, a measure of endogenous insulin production, were maintained in some patients for up to 2 years during follow up. This result suggests that further destruction of pancreatic islet cells had been arrested in these patients following a single dose. The results of this Phase I clinical study with MER3001 protoype immunotherapeutic have been published in Orban et al., J. Autoimmunity 2010.