KAMADA » CLINICAL PIPELINE » DIABETES TYPE 1

DIABETES TYPE 1

Type 1 Diabetes is an autoimmune disease in which the pancreatic beta cells responsible for secretion of insulin are attacked and destroyed by the immune system. According to
estimates by the U.S. Centers for Disease Control, more than 10 million people throughout the world suffer from Type-1 Diabetes with 100,000 new patients diagnosed annually. According to
estimates by the American Association for Type-1 Diabetes, approximately three million people in the United States suffer from Type-1 Diabetes, with 30,000 new patients diagnosed annually.

Studies have demonstrated that even though the level of AAT protein in Type-1 Diabetes patients may be normal, the activity of the AAT protein in these patients is significantly lower than in healthy people. Because AAT has proven anti-inflammatory responses, treatment by AAT protein in the initial stages after diagnosis of Type-1 Diabetes may prevent or may delay the inflammation that is caused by the autoimmune destruction of the pancreatic cells. As a result, AAT therapeutics may slow the progression of the development of newly diagnosed Type-1 Diabetes and improve prognosis.

Kamada reported topline results of a phase II clinical trial in pediatric and young adult patients suffering from Type-1 Diabetes (T1D). This double-blind, placebo-controlled, multi-center clinical trial was designed to evaluate the efficacy and safety of AAT IV in halting disease progression in pediatric and young adult patients newly diagnosed with T1D. Study endpoints included beta cell function assessment as measured by change in C-peptide parameters, glycemic
control represented by HbA1C levels and insulin daily dose.

Kamada’s AAT IV demonstrated a very good safety profile in T1D pediatric and young adult
patients treated in the Phase 2 study, similar to that observed with GLASSIA® for the treatment
of Alpha-1 Antitrypsin Deficiency.

Analysis of the study data for the 12-18-year-old patient subgroup treated, shows that AAT may have a beneficial effect on beta-cell function, HbA1c level and insulin daily dose intake. This effect may potentially reduce future severe disease complications.