Press release Communiqué de presse
Press release Communiqué de presse
January 20, 2022 20 January, 2022
Sernova Announces Peer Reviewed Publication Demonstrating Safety and Efficacy of a Novel Cell Pouch Cell Therapy Approach for Treatment of Severe Hemophilia A
LONDON, ONTARIO – January 20, 2022 – Sernova Corp. (TSX-V:SVA) (OTCQB:SEOVF) (FSE/XETRA:PSH), a
clinical-stage regenerative medicine and cell therapy therapeutics company developing a potential ‘functional
cure’ for chronic diseases is pleased to announce a peer reviewed published scientific journal article
highlighting successful development of a novel cell therapy approach for treatment of severe hemophilia A , a
serious genetic bleeding disorder caused by missing factor VIII (FVIII) activity in the blood stream.
The journal article entitled “Efficient and Safe Correction of Hemophilia A by Lentiviral Vector-Transduced BOECs in an Implantable device (Sernova’s Cell Pouch™),” was published in the prestigious scientific journal, Molecular Therapy: Methods & Clinical Development, Volume 23, December 2021.
The therapeutic approach includes use of the patient’s own cells obtained from a blood sample, which are then modified using a lentiviral vector-mediated gene transfer procedure using the B-domain deleted form of FVIII under the control of an endothelial-specific promoter and subsequently transplanted within Sernova’s vascularized Cell Pouch into a mouse model of hemophilia A. These cells then provide a continuous therapeutic release of factor VIII into the bloodstream. The publication highlights that the HemAcure Consortium has now successfully demonstrated safety and long-term improvement in blood clotting in a hemophilia A mouse model.
It is estimated that 115,000 people live with hemophilia A in North America and Europe with a significant proportion who rely on regular infusions of FVIII. Treatment costs per patient are as high as US $200,000 each year, with an aggregate therapeutic cost of over US$ 10 billion per year.
“This novel ex vivo gene therapy approach is the first demonstration showing the safety and feasibility of transplantation of lentiviral-corrected blood outgrowth endothelial cells (BOECs) within an implantable medical device using GMP-like procedures for the long-term treatment of hemophilia A,” said the lead author Dr. Antonia Follenzi, MD, PhD, Professor, Department of Health Sciences, School of Medicine, Università del Piemonte Orientale. “If this therapy is successful in future clinical trials, it could become an important new therapeutic approach to improve the quality of life for people suffering with severe hemophilia A.”
Dr. Philip Toleikis, President and CEO Sernova Corp stated, “This publication represents approximately four years of dedicated work by the HemAcure consortium, from conceptualizing this novel treatment approach, through to validating its potential as a safe and long-term treatment option for people with hemophilia A. The Sernova Cell Pouch provides the required environment for transplanted cells to survive and function in the body, as demonstrated by the production of FVIII to improve blood clotting as reported by Dr. Follenzi and colleagues.” He continued, “We recognizes ex vivo gene therapy as a potential therapeutic option for people suffering from multiple rare diseases and we are proud that our technologies may contribute to the development and future delivery of functional cures for these ailments.”
The HemAcure consortium research was supported by funding from the European Commission’s Horizon 2020.
ABOUT HEMOPHILIA A
Hemophilia A is the most common form of hemophilia and is a genetic disorder caused by the reduced or absence of FVIII, a blood clotting protein. People with hemophilia A have prolonged abnormal bleeding as a result of trauma or surgery. While it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous change in the gene responsible for FVIII production. According to the US Centers for Disease Control and Prevention, hemophilia A occurs in about 1 in 5,000 male births. Prolonged bleeding in high-risk areas such as the brain can be catastrophic, while persistent bleeding in joints results in permanent damage making joint replacement surgery common and risky procedures among these patients. Approximately 115,000 people in North America and Europe have hemophilia A. Although there is no cure for the disease, hemophilia A can be controlled with medications to supplement or replace the missing FVIII. Annual worldwide costs for the treatment of the disease for each patient may range from $60,000 to $260,000 US for an aggregate therapeutic cost of approximately $15B per year.
ABOUT SERNOVA Sernova is developing regenerative medicine therapeutic solutions using a medical device (Cell Pouch) and immune protected therapeutic cells / tissues (i.e. human donor cells, corrected human cells and stem cell- derived 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 cellular production of proteins or hormones missing or in short supply within the body. For more information, please visit www.sernova.com.
ABOUT SERNOVA’S CELL POUCH SYSTEM
The Cell Pouch, as part of the Cell Pouch System, is a novel, proprietary, scalable, implantable macro- encapsulation device solution designed for the long-term survival and function of therapeutic cells. The device upon implantation is designed to incorporate with tissue, forming highly vascularized tissue chambers for the transplantation and function of therapeutic cells, which then release proteins and hormones as required to treat disease.
The Cell Pouch, along 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 in a Canadian first-in-human study. Sernova is currently conducting a Phase I/II study at the University of Chicago. Positive initial results have been presented at several international scientific conferences.
ABOUT HORIZON 2020 PROGRAM AND HEMACURE
Horizon 2020 was the biggest EU Research and Innovation program ever with nearly €80 billion of funding available over seven years (2014 to 2020). It promised more breakthroughs, discoveries and world-firsts by taking great ideas from the lab to the market. The project was funded as part of societal challenges “personalizing health and care” in a specific call about innovative treatments and technologies. The overall objective of the HemAcure project was to develop and refine the tools and technologies for a novel ex vivo prepared cell-based therapy to treat the bleeding disease, hemophilia A (caused by a genetic deficiency in clotting Factor VIII (FVIII)) that should ultimately lead to improved quality of life of the patients. The European Union's Horizon 2020 research and innovation program awarded approximately €5.5 million (CDN$8.5) to support the project. International consortium members included the University Hospital Würzburg (Coordinating Institute), Germany; IMS - Integrierte Management, Heppenheim, Germany; Università del Piemonte Orientale “Amedeo Avogadro,” Novara, Italy; Loughborough University, Loughborough, United Kingdom; ARTTIC International Management Services, Munich, Germany and Sernova Corp., London, Ontario, Canada. This project received funding from the European Union’s Horizon 2020 Research and Innovation Programme under grant agreement No 667421. The Consortium worked together to provide sets of design and manufacturing protocols, based on current European GMP regulations to prepare an Investigational Medicinal Product Dossier (IMPD) for an Investigational Medicinal Product (IMP), composed of therapeutic cells and an implantable medical device (Sernova’s Cell Pouch).
FOR FURTHER INFORMATION, PLEASE CONTACT:
Christopher Barnes VP, Investor Relations Sernova Corp. firstname.lastname@example.org Tel: 519-902-7923 www.sernova.com
Corey Davis, Ph.D. LifeScience Advisors, LLC email@example.com Tel: 212-915-2577
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.sedar.com 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.