Kamada Ltd. (NASDAQ:KMDA) (TASE:KMDA), a plasma-derived protein therapeutics company focused on orphan indications, announces the initiation of a Phase 2/3 clinical trial of Glassia®, the Company's proprietary human Alpha-1 Antitrypsin (AAT), to treat newly diagnosed pediatric patients with type 1 diabetes (T1D). In T1D, autoimmune attacks occur on pancreatic beta cells that secrete insulin, thereby compromising insulin level and glycemic control. Over time there is progressive deterioration of self-insulin secretion, poor capability to control glucose levels, and eventually, full external insulin dependence.
This double-blind, placebo-controlled, multicenter Phase 2/3 clinical trial of 190 pediatric patients with newly diagnosed T1D will evaluate the safety and efficacy of intravenous Glassia to halt disease progression and maintain the ability of the pancreas to produce insulin. By maintaining its ability to produce insulin, the body can independently control glucose levels and avoid diabetes complications that result from poor glycemic control (e.g., cardiovascular disease, kidney disease, eye and vision problems, neurological damage and more). This two-year study follows U.S. Food and Drug Administration and European Medicines Agency guidelines for clinical trials evaluating beta cell preservation and will measure C-peptide parameters (which represent self-insulin secretion), HbA1C, hypoglycemic events and insulin daily dose, among others. Interim data are expected after approximately 90 patients complete one year of treatment, which will be in approximately two years. Initially, the trial will be conducted at four leading pediatric T1D medical centers in Israel with plans to expand the scope of the trial to include centers in other countries.
Kamada previously reported positive preliminary data from the extension portion of its Phase 1/2 clinical trial of Glassia to treat pediatric patients newly diagnosed with T1D. That preliminary data showed that at approximately 20 months from diagnosis and approximately 10 months following the last Glassia infusion, 60% of study subjects who participated in the extension portion of the trial had peak C-peptide levels greater than 0.2 pmol/ml, which indicates a functioning beta cell capacity and is considered to be a higher percentage than would be expected without intervention.
In addition, patients continued to attain HbA1C targets according to International Society for Pediatric and Adolescent Diabetes with an average HbA1C of 7.5%, and 75% of patients presented HbA1C levels even lower than 7.5%, which is the clinically desired level for glycemic control in pediatric diabetic patients, who usually demonstrate a more severe or volatile form of disease compared with adults.
"This is a very exciting opportunity for Kamada to bring a promising therapy to newly diagnosed pediatric patients with type 1 diabetes. Data from our earlier studies give us great encouragement to move forward with this pivotal study," stated David Tsur, Co-founder and Chief Executive Officer of Kamada. "The complications of type 1 diabetes remain an unmet need that is inadequately addressed with existing therapies, which include insulin, diet and behavioral changes. Moreover, these complications have direct correlation with the extent of glycemic control the patient reaches during early disease phases. We believe Glassia can be a groundbreaking treatment for newly diagnosed type 1 diabetes in pediatric patients as it should demonstrate the ability to halt disease progression and allow the pancreas to produce its own insulin," added Mr. Tsur.