Sernova Receives Orphan Drug and Rare Pediatric Disease Designations for its Hemophilia A Program from FDA

LONDON, Ontario and WINDHAM COUNTY, Conn., Nov. 27, 2023 (GLOBE NEWSWIRE) -- Sernova Corp. (TSX:SVA) (OTCQB:SEOVF) (FSE/XETRA:PSH), a clinical-stage company and leader in cell therapeutics, today announced the U.S. Food and Drug Administration (FDA) has granted both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) for the company’s Hemophilia A program.

The FDA grants orphan designation, also referred to as orphan status, to therapies intended for the treatment of rare diseases that affect fewer than 200,000 people in the U.S. This designation provides certain benefits, including tax credits for qualified clinical testing, waiver or partial payment of FDA application fees and seven years of market exclusivity, if approved. Separately, rare pediatric disease designations are granted for rare diseases that primarily affect children under 18 years old with recipients of this designation being awarded a priority review voucher, upon approval. The priority review voucher may be redeemed, transferred, or sold.

“We are pleased with the FDA’s decision to grant these designations for our novel treatment for Hemophilia A, which uses the Cell Pouch™ in combination with cells corrected for the production of Factor VIII,” commented Cynthia Pussinen, Chief Executive Officer of Sernova. “Hemophilia A is a serious, life limiting condition and we are committed to advancing development of the program, with a hope to positively impact patients around the world who are waiting for improved treatments.”

About Sernova’s Hemophilia A Cell Pouch System Program
Sernova’s Hemophilia A program combines the Sernova Cell Pouch™ with a patient’s own cells and will not require the use of immunosuppression medications. This therapy is intended to replace Factor VIII (FVIII) - an essential blood-clotting protein that is deficient or absent in patients with Hemophilia A; this is accomplished by correcting the patient’s own Blood Outgrowth Endothelial Cells (BOECs) and subsequently returning them to the patient via the Cell Pouch™. These modified cells function to release FVIII into the bloodstream, restoring the patient’s ability for clotting during periods of bleeding.

Sernova and research partners, through a Horizon 2020 grant which is part of the EU's research and innovation funding program in proof-of-concept work, successfully corrected human blood cells from patients with Hemophilia A to produce Factor VIII using a novel first-in-class gene and cell therapy approach where the corrected cells were transplanted into the pre-implanted, vascularized Cell Pouch in a preclinical model of Hemophilia A. The work demonstrated an improvement in blood clotting using the combined technologies (Efficient and safe correction of hemophilia A by lentiviral vector-transduced BOECs in an implantable device; Molecular Therapy: Methods & Clinical Development Vol.23, Dec 2021).

Collaboration with the University of Piemonte Orientale
The company is pleased to announce a collaboration with the University of Piemonte Orientale, Italy under the direction of Antonia Follenzi MD, Ph.D. Professor of Histology and Cell and Gene therapy. Dr. Follenzi is a pioneer of cell and gene therapy approaches to cure Hemophilia A. Her laboratory has expertise in the generation of BOECs from Hemophilic patients and correcting the FVIII gene using Lentiviral vectors.

The purpose of the new collaboration is to optimize the technology using lentiviral vectors to drive the expression of FVIII transgene under the control of novel promoters into BOECs of Hemophilia patients to achieve optimal sustained production of FVIII using an optimized cell dose within the Cell Pouch in an animal model of Hemophilia A. The overall goal of the collaboration is to develop a product combination along with pre-clinical results that support advancing into clinical trials in patients with Hemophilia A.

Professor Follenzi stated, “I am pleased to be working with the Sernova team on these advanced technologies for a new and safe treatment of patients with Hemophilia A using a gene and cell therapy approach within the Cell Pouch. Our goal is to advance these new technologies to further maximize the release of FVIII into the bloodstream and to extend the duration of that release for a sufficient duration of time to eliminate the need for weekly infusions of FVIII and to significantly improve the lives of people with Hemophilia A.”

BENEFITS OF ODD AND RPDD
Combined benefits of these designations include exclusive marketing rights for a seven-year period, after marketing approval, a 25% federal tax credit for clinical research expenses incurred in the U.S. which is applicable for up to 20 years, waiver of Prescription Drug User Fee Act (PDUFA) fees for orphan drugs (currently worth >US$3 million), ability to qualify to compete for research grants from the Office of Orphan Products Development (OOPD) to support clinical studies for the orphan indication, and eligibility to receive regulatory assistance and guidance from FDA to design the development plan.

Furthermore, once the therapy is approved for marketing, it cannot be copied and sold in the U.S. for 7 years regardless of patent life and the sponsor will be granted a Priority Voucher which can be used to receive approximately 4 months reduction time of the standard FDA review period or sold.

ABOUT HEMOPHILIA A
Hemophilia encompasses a group of inherited disorders that alter blood coagulation. Classical Hemophilia, also known as Hemophilia A, is a hereditary hemorrhagic disorder resulting from a congenital deficit of FVIII that manifests as protracted and excessive bleeding either spontaneously or secondary to trauma. ¹ Hemophilia A is the most common form of Hemophilia and is a genetic disorder caused by missing or defective FVIII, a blood clotting protein. Severe Hemophilia A occurs in about 60% of cases where the deficiency of FVIII is less than 1% of normal blood concentration. While it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous change in the gene.

According to the U.S. Centers for Disease Control and Prevention, Hemophilia A occurs in about 1 in 5,000 births. Prolonged bleeding, in areas such as the brain, of a person with Hemophilia A, can be fatal. Prolonged bleeding in joints can cause inflammatory responses and permanent joint damage. Approximately 20,000 people in the United States, 2,500 in Canada and 10,000 in Europe have moderate to severe forms of Hemophilia A. Though there is no cure for the disease, Hemophilia A can be controlled with regular infusions of recombinant clotting FVIII. Treatment costs per patient are as high as US$200,000 or more each year, with an aggregate therapeutic cost of over US$ 10 billion per year.

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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FORWARD-LOOKING INFORMATION
This release contains statements that, to the extent they are not recitations of historical facts, may constitute “forward-looking statements” that involve various risks, uncertainties, and assumptions, including, without limitation, statements regarding the prospects, plans, and objectives of the company. Wherever possible, but not always, words such as "expects", "plans", "anticipates", "believes", "intends", "estimates", "projects", "potential for" and similar expressions, or that events or conditions "will", "would", "may", "could" or "should" occur are used to identify forward-looking statements. These statements reflect management’s beliefs with respect to future events and are based on information currently available to management on the date such statements were made. Many factors could cause Sernova’s actual results, performances or achievements to not be as anticipated, estimated or intended or to differ materially from those expressed or implied by the forward-looking statements contained in this news release. Such factors could include, but are not limited to, the company’s ability to secure additional financing and licensing arrangements on reasonable terms, or at all; ability to conduct all required preclinical and clinical studies for the company’s Cell Pouch System and or related technologies, including the timing and results of those trials; ability to obtain all necessary regulatory approvals, or on a timely basis; ability to in-license additional complementary technologies; ability to execute its business strategy and successfully compete in the market; and the inherent risks associated with the development of biotechnology combination products generally. Many of the factors are beyond our control, including those caused by, related to, or impacted by the novel coronavirus pandemic. Investors should consult the company’s quarterly and annual filings available on www.sedarplus.ca for additional information on risks and uncertainties relating to the forward-looking statements. Sernova expressly disclaims any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.