Sernova Announces Positive Ongoing Interim Phase 1/2 Clinical Data for the Cell Pouch System™ for Type 1 Diabetes Trial at the 2023 IPITA, IXA, and CTRMS Joint Congress

• All six patients in the first cohort (Cohort A) were successfully implanted with the 8-channel Cell Pouch System with post-transplant follow-up periods ranging from 6 months to 3.5 years;
• 5 of 6 patients in Cohort A discontinued insulin therapy (insulin independent) following islet transplantation into the Cell Pouch and modest islet top-up via portal vein. All 6 patients achieved HbA1c values in the non-diabetic range (<6.5%);
• In Cohort B, the first 6 of 7 planned patients have received the higher capacity 10-channel Cell Pouch and 5 patients have received a first islet transplant;
• Stable fasting and stimulated serum C-peptide levels were observed following a single islet transplant into the 10-channel Cell Pouch in the first assessable Cohort B patient who subsequently achieved insulin independence with a modest portal vein top up.

LONDON, Ontario and WINDHAM COUNTY, Conn., Oct. 27, 2023 (GLOBE NEWSWIRE) -- Sernova Corp. (TSX:SVA) (OTCQB:SEOVF) (FSE/XETRA:PSH), a clinical-stage company and leader in cell therapeutics, today presented interim positive results from its ongoing Phase 1/2 clinical trial investigating islet allotransplantation into pre-vascularized Sernova Cell Pouch™ during an oral presentation at the 2023 IPITA, IXA, and CTRMS Joint Congress in San Diego, California. Enrollment in Cohort A, which utilizes the 8-channel Cell Pouch, is complete with post-transplant data available for periods of follow-up ranging from 6 months to 3.5 years. Enrollment in Cohort B, which utilizes the higher capacity Cell Pouch and a revised and better-tolerated immunosuppressive regimen, began in November 2022 and 6 of the 7 planned patients have now been successfully implanted.

The primary objective of the study is to investigate the safety and tolerability of islet transplantation into Cell Pouch in patients with T1D, impaired hypoglycemia awareness, and a history of severe hypoglycemic episodes. Secondary study objectives include establishment of islet release criteria predictive of outcomes from islet transplant into the Cell Pouch and optimal dose and concentration ranges for purified islets transplanted into the Cell Pouch.

Interim results from Cohort A demonstrated successful implantations of the 8-channel Cell Pouch in the 6 treated patients that were well tolerated with no seromas and no unexpected AEs (adverse events), chronic pain or discomfort. Data showed histological evidence of surviving and functional islets and positive fasting and stimulated serum C-peptide (a measure of islet insulin secretion) in patients who maintained optimal immunosuppression. All 6 patients eventually received supplemental, marginal-dose islet infusions via the portal vein with the first 5 having achieved sustained insulin independence. All 6 Cohort A patients achieved HbA1c values in the non-diabetic range (<6.5%) with persistent serum fasting and stimulated C-peptide levels for current durations out to 3.5 years.

In Cohort B, 6 of the planned 7 patients have been implanted with the higher capacity 10-channel Cell Pouch, without complications. Among the 6 patients that have been implanted, 5 have completed at least one of the two protocol-defined islet transplants to Cell Pouch.

The first assessable patient in Cohort B following the first Cell Pouch islet transplant showed persistent fasting and stimulated serum C-peptide, with stable BETA-2 scores (a measure of islet graft function) that continued at Day 180 following their first islet transplant to Cell Pouch. The same patient showed modest but favorable improvements in HbA1c from 7.5% at baseline to 6.9% also at Day 180.

The day following the second islet transplant to Cell Pouch, results from a sample of the islets taken from the donor pancreas on the day of transplant came back positive for the yeast, Candida albicans. Out of an abundance of caution, Cell Pouches containing the contaminated islets were immediately removed. The Cell Pouches that were previously transplanted with the first dose of uncontaminated, healthy islets were not removed and remained in place, continuing to function. Explantation of the Cell Pouches containing contaminated islets was completed without complications and the patient fully recovered without any wound or systemic blood infection, demonstrating the designed retrievability of the transplanted Cell Pouch. Following recovery, this patient received a modest intraportal islet transplant and remains insulin independent.

The revised immunosuppression protocol, used in Cohort B, continues to demonstrate favorable protection for the islet grafts with no donor islet rejection or donor specific antibodies observed under the new regimen.

“I am pleased with the overall patient outcomes and learnings from the first trial cohort. We have applied those learnings to the second patient cohort along with the introduction of the higher capacity 10-channel Cell Pouch,” commented Dr. Piotr Witkowski, Director of the Pancreatic and Islet Transplant Program at The University of Chicago, and principal investigator for the Sernova trial. “I am encouraged by the positive safety profile observed with Cell Pouch implants longer than 4 years, and early patient outcomes with the enhanced 10-channel device that we are using in the second cohort. Enrollment of the second cohort is nearly complete, and I look forward to reporting further results.”

“We are very encouraged by the results and our learnings from our trial to date,” said Cynthia Pussinen, Chief Executive Officer at Sernova. “Having recently advanced the trial into Cohort B, using our higher capacity 10-channel Cell Pouch, we are already seeing positive signals for both safety and efficacy. We look forward to sharing the next trial update, in the coming months.”

These results were presented by Piotr Witkowski, Professor of Surgery at the University of Chicago at the International Pancreas and Islet Transplant Association (IPITA), the International Xenotransplantation Association (IXA), and the Cell Transplant and Regenerative Medicine Society (CTRMS) Joint Congress, taking place from October 26 – 29 in San Diego, CA as an oral presentation entitled “Islet allotransplantation into pre-vascularized Sernova Cell Pouch - Lessons learned from the first patient cohort” (Abstract #105, Session: “Islet Transplantation: Engineering the Islet Site Session,” Thursday, October 26, 2023 2:45 p.m. to 3:45 p.m. PT)

For more information on the ongoing clinical study, go to clinicaltrials.gov ().

ABOUT SERNOVA CORP. AND THE CELL POUCH SYSTEM PLATFORM FOR CELL THERAPY

Sernova Corp. is a clinical-stage biotechnology company that is developing therapeutic cell technologies for chronic diseases, including insulin-dependent diabetes, thyroid disease, and blood disorders that include hemophilia A. Sernova is currently focused on developing a ‘functional cure’ for insulin-dependent diabetes with its lead asset, the Cell Pouch System, a novel implantable and scalable medical device with immune protected therapeutic cells. On implantation, The Cell Pouch forms a natural vascularized tissue environment in the body for long-term survival and function of therapeutic cells that release essential factors that are absent or deficient in the bodies of patients with certain chronic diseases. Sernova’s Cell Pouch System has demonstrated its potential to be a ‘functional cure’ for people with T1D in an ongoing Phase 1/2 clinical study at the University of Chicago. Sernova is also advancing a proprietary technology in collaboration with the University of Miami to shield therapeutic cells from immune system attack with the goal to eliminate the need for chronic, systemic immunosuppression. In May 2022, Sernova and Evotec entered into a global strategic partnership to develop an implantable off-the-shelf iPSC (induced pluripotent stem cells) based islet replacement therapy. This partnership provides Sernova a potentially unlimited supply of insulin-producing cells to treat millions of patients with insulin-dependent diabetes (type 1 and type 2). Sernova continues to progress two additional development programs that utilize its Cell Pouch System: a cell therapy for hypothyroid disease resulting from thyroid gland removal and an ex vivo lentiviral Factor VIII gene therapy for hemophilia A.

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