Program Status
Cebix has spent 2009 pursuing an appropriate technology to convert C-peptide from a product that would require 3-4 subcutaneous doses per day to a product that can be dosed weekly. In the spring of 2010, Cebix developed two distinct approaches that will deliver C-peptide in a once weekly form and is advancing the lead product into clinical trials. The Company has met with the FDA and discussed our development plans. Our next step is to file an IND by year-end to allow safety testing in humans.
Cebix is focused on the development of chronic replacement therapy based on human proinsulin C-peptide for the treatment of type 1 diabetic neuropathy. The role of C-peptide will also be explored in type 1 diabetic nephropathy and retinopathy.
Type 1 diabetes is characterized by the body's inability to produce proinsulin and consequently both insulin and C-peptide. We have identified C-peptide as a biologically active endogenous peptide hormone; it was originally considered to be only an inactive byproduct of insulin production. Based on extensive scientific data, we think that C-peptide deficiency in type 1 (insulin-dependent) diabetes patients is a contributing cause of many of the complications associated with long-term disease despite controlled insulin replacement therapy. It is estimated that 4 million people in North America and Europe have type 1 diabetes and about 80,000 Americans are diagnosed with type 1 diabetes every year. On a global scale, type 1 diabetes affects more than 8 million people and the incidence is rising.
Type 1 diabetes can over time lead to microvascular disease, which is damage to the small blood vessels. The primary microvascular complications associated with type 1 diabetes are:
• Neuropathy; resulting in reduced sensibility of the feet and lower legs, ulcer formation, gastrointestinal and sexual dysfunction, occurring in approximately 80% of type 1 patients after 15-20 years;
• Nephropathy; resulting in gradual loss of kidney function and eventually end-stage renal disease, occurring in up to 25% of type 1 patients after 15-20 years
• Retinopathy; resulting in retinal edema, hemorrhage, and loss of vision, occurring in 30-50% of type 1 patients after 15-20 years.
There are currently few treatment options for these complications of type 1 diabetes; specifically for diabetic neuropathy, there are no approved drugs to treat the microvascular damage. Therefore, if demonstrated to be effective and safe, C-peptide replacement therapy would represent a first-in-class treatment option for patients debilitated by diabetic neuropathy and other long-term complications of type 1 diabetes.
