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Kamada presents positive results for efficacy and safety in a clinical study involving Type-1 diabetes pediatric and young adult patients treated with its AAT product
Ness Ziona, Israel, November 18, 2012. Kamada Ltd. (TASE: KMDA), an Israeli biopharmaceutical company, specializing in development, manufacturing and marketing of specialty plasma bio-therapeutics by intravenous or inhaled route for life saving uses, reports positive results for efficacy and safety in a unique of its kind, phase 1/2 clinical study in pediatric and young adult patients suffering from T1D. The patients were treated with Kamada's Glassia product that contains the AAT protein and that was fully developed by Kamada. The drug product is currently marketed for tens of millions of dollars in the US, Israel and some other countries worldwide for the indication of Alpha-1 Antitrypsin Deficiency. Analysis of the study data shows that AAT may slow down the rate of disease progression by allowing continued functionality of insulin-secreting beta cells and better glycemic control. This effect may potentially reduce future severe disease complications. Kamada is now evaluating its next moves towards the subsequent stages of this drug development and the ways to pursue its regulatory approval for T1D.
The analysis of the level of C peptide, that reflects the self-production of insulin, in the blood of the study population, shows that the majority of the patients maintained levels of C peptide higher than the clinical trough levels defined (=>0.2pmol/mL) as clinically meaningful and considered as crucial for slowed-down disease progression (“responder patients”). The results demonstrated existing functionality of pancreatic beta cell beyond a period of one year from the patient's first diagnosis as diabetic.
Analysis ofHbA1c, which is a measure of glucose containing Hemoglobin, a parameter which is indicative of the glycemic control, it was evident that almost all patients reached glycemic control below 7.5%, the level that is considered clinically meaningful for T1D patients and that is known to correlate with reduced chances to develop disease complications in the future. The decrease in HbA1c was apparent in all patient groups and was statistically significant throughout the different study time periods and at the end of the study.
The patients' diary reports recorded in all study periods a reduction in insulin consumption ,in parallel to reduction in levels of specific T1D disease antibodies, a sign that may potentially indicate a diversion or re-modulation of the immune system from attacking the self pancreatic cells.
The drug showed high safety and tolerability profile. All patients completed the clinical trial with no serious adverse events. Other adverse events were mild in intensity and not related to the study drug. These safety results further strengthen the high safety profile of the Kamada's AAT product, as demonstrated before in all previous clinical trials and during marketing phase.
In the study that was performed at two clinical sites, the Schneider Children's Medical Center of Israel and Aassaf Harofeh Medical Center of Israel, participated pediatric and young adult patients aged 9-17 years. The patients were randomized into three treatment groups (40mg, 60mg, 80mg/kg/week). All patients received the study drug for a period of 28 weeks in three treatment intervals. All patients continued the regular disease treatment and were not deprived of insulin or any other drugs required for disease management. All patients completed the treatment in full and underwent a follow up period.
David Tsur, CEO of Kamada stated: ” We are happy and proud with this achievement. This is the first clinical evidence that may lead to an actual breakthrough in the treatment, and possibly even of curing, T1D. The quality of the study data prompts us to continued drug development and pursuing regulatory approval for this indication.”
Considering the successful results of the study and the high market potential of this indication, the Company plans to continue the development of this drug towards regulatory approval, thereby assuring its world leadership in finding a treatment for T1D, for which preservation of beta cells remains an unmet need.
Dr Eli Lewis, a Professor and scientist at the Ben Gurion University of Beer Sheba and Head of the Islet and Transplantation Laboratory said:” After a decade of research on AAT, this important molecule in Kamada's Glassia drug, there is no greater satisfaction other than witnessing the signs of disease remission, and without any safety concern. “
T1D is an autoimmune disease in which the pancreatic beta cells that are responsible for the insulin secretion are attacked by the immune system. In the absence of self-produced insulin and the concomitant glycemic control, there is a need to supply extraneous insulin, in order to regain glycemic control, and prevent future disease complications that include heart disease, blood vessels disease, nerve and eye disease, infections, hypoglycemic events and unfortunately many more ailments.
The scientific rationale of the Kamada drug is based, among others, on the fact that AAT has an adjunct anti inflammatory activity that may immune-modulate the immune system in a way that prevents it from attacking the pancreatic beta cells which would be destroyed by the autoimmune attack. Past studies have shown that despite having a normal serum level of AAT, the AAT of diabetic patients is inactive in this respect and, therefore, unable to cope with the developing inflammation in the beta cells. Additionally, in a number of studies in the recent years, the rationale for treating T1D during the “honeymoon” period, a period during which there are still some existing functional beta cells, had been further established. It was hypothesized that AAT may halt the pancreatic inflammation and thereby allow the survival of active and operating beta cells that secret insulin, a survival which may allow the patient to reduce dependence on external insulin and eventually decrease disease complications.
According to the US Center for Disease Control, there are more than 10 million diabetic type 1 patients worldwide, which are joined yearly by an additional 100,000 newly diagnosed patients.