Sernova Receives FDA IND Allowance to Initiate a U.S. Clinical Trial of its Cell Pouch

Sernova Corp., a clinical stage company developing regenerative medicine technologies for the long-term treatment of diseases including diabetes and hemophilia, has received U.S. Food and Drug Administration (FDA) notice of allowance for its IND for a new human clinical trial with the Cell Pouch System (CPS) in the United States. 
 
Sernova plans to initiate the new clinical trial under this U.S. IND to investigate the Cell Pouch for treatment of type 1 diabetes (T1D) in individuals with hypoglycemia unawareness. The trial is a Phase I/II prospective single arm study of islets transplanted into the subcutaneously implanted Cell Pouch. The primary objective of the study is to demonstrate safety and tolerability of islet transplantation into the Cell Pouch and the secondary objective is to assess efficacy through a series of defined measures.
 
JDRF has previously committed to provide Sernova up to $2.45 million to support the clinical trial.
 
“Hypoglycemia unawareness is a serious consequence of type 1 diabetes,” said Derek Rapp, president and CEO, JDRF International. “We are excited to see progress in this and other potentially life-saving JDRF-funded research, which could help prevent people with hypoglycemia unawareness from experiencing dangerous lows, as we strive to achieve our vision of a world without T1D.”
 
The study is a Phase I/II single-site, single arm, company-sponsored trial. Following approval by the Institutional Review Board, patients with hypoglycemia unawareness will be enrolled into the study under informed consent. Patients will then be implanted with the Cell Pouch including sentinel devices. Following vascularized tissue development, a dose of purified islets under strict release criteria will be transplanted into the Cell Pouch and patients followed for safety and efficacy measures for approximately six months. At this point a decision will be made whether to transplant a second islet dose with subsequent safety and efficacy follow up. Patients will then be further followed for one year.
 
“Sernova’s FDA clearance to commence human clinical trials in the United States is an exciting step forward in diabetes research, initially focused to reduce the risk of hypoglycemia unawareness, a complication in which a patient is unaware of a deep drop in blood sugar that can have life threatening consequences,” said Dave Prowten, president and CEO of JDRF Canada. “This is also an example of the international collaboration fostered by JDRF-funded projects to accelerate transformative research to benefit the T1D community.”

The Cell Pouch is a novel, proprietary, scalable, implantable macroencapsulation device for the long-term survival and function of therapeutic cells (donor, stem cell derived cells and xenogeneic cells) which then release proteins and/or hormones as required to treat disease. The device is designed upon implantation to incorporate with tissue, forming highly vascularized tissue chambers for the transplantation and function of therapeutic cells. The device with therapeutic cells has been shown to provide long term safety and efficacy in small and large animal models of diabetes and has been proven to provide a biologically compatible environment for insulin producing cells in humans.

“We are extremely enthusiastic about the promise of Sernova’s regenerative medicine platform to provide a new therapeutic option for diabetes patients with hypoglycemia unawareness. We believe Sernova’s multiple advancing cell based therapies have the potential to deliver significant improvement in the quality of life of patients suffering from diabetes and other debilitating diseases,” said Dr. Philip Toleikis, Sernova’s president and CEO.
 
T1D is a life-threatening disease in which the body’s immune system mistakenly attacks and kills the pancreatic cells that produce insulin—a hormone that is essential for life because of its role to help the body use glucose. The existing standard of care for patients with TID is suboptimal. To date, there is no cure for T1D, and people living with the disease are dependent on exogenous insulin therapy to help keep their blood-sugar levels from spiking too high, which can lead to long-term complications such as kidney and heart diseases or an acute, potentially deadly health crisis. Present-day insulin therapy is, however, an imperfect treatment method that requires people with T1D to carefully monitor their blood sugar throughout the day and take multiple, calculated doses of insulin based on food intake, exercise, stress, illness and other factors. A miscalculation or unexpected variable leading to high or low blood sugar episodes are daily threats, and only a third of people with T1D achieve their long-term blood glucose targets, placing them at risk for T1D-related health complications.
 
Sernova Corp. is developing disruptive regenerative medical technologies using a medical device and immune protected therapeutic cells to improve the treatment and quality of life of people with chronic metabolic diseases such as insulin-dependent diabetes, blood disorders including hemophilia, and other diseases treated through replacement of proteins or hormones missing or in short supply within the body. Sernova’s device forms a natural environment promoting the long-term function of therapeutic cells.