A series of breakthroughs from the International HemAcure Consortium provides a novel cell-based treatment approach for people living with Hemophilia A which could reduce or eliminate the need for lifelong Factor VIII infusions
LONDON, ONTARIO / TheNewswire / November 20, 2018 - Sernova Corp. (TSX-V: SVA) (OTCQB: SEOVF) (FSE: PSH) a clinical-stage regenerative medicine company, highlights the achievements in developing a novel cell-based therapy in combination with Sernova's Cell Pouch(TM) for treatment of Hemophilia A.
The therapeutic significance of this work supports the HemAcure Consortium's concept that Factor VIII genetically corrected human cells from a blood sample of patients with Hemophilia A transplanted into the Cell Pouch can improve blood clotting.
"I am impressed with the achievements of this International Consortium within the limited time allowed. The technical teams worked closely together to complete this work in developing a first-in-world ex vivo gene therapy approach in an implanted vascularized medical device for the treatment of Hemophilia A." said Dr. David Lillicrap, Professor in the Department of Pathology and Molecular Medicine at Queen's University, member of the Medical Advisory Board of the World Federation of Hemophilia, and HemAcure Scientific Advisory Board member.
A summary of the Consortium's achievements are as follows:
In blood donated from patients with Hemophilia A, endothelial outgrowth cells to be corrected for the Factor VIII gene were isolated and grown successfully in a specialized Good Manufacturing Process (cGMP) compliant medium developed by the Consortium.
Using a human Factor VIII gene insertion technique, the cells were corrected and confirmed to produce Factor VIII.
A preliminary experiment showed these cells could release Factor VIII in the blood over time and improve blood clotting in an animal model of Hemophilia A, in preparation for transplant into the Cell Pouch.
The corrected cells were proven to be successfully replicated through a production scale-up process. Following amplification, these cells maintained their normal healthy behavior in producing Factor VIII. Additional safety metrics were achieved using established tests.
The cells were then cryopreserved and shipped from the European partners to Sernova in North America where they were shown to remain healthy through quality control testing in preparation for transplantation.
The Cell Pouch, manufactured under cGMP, and following implantation in the Hemophilia A animal model showed development of vascularized chambers suitable to receive the corrected cells.
Following transplantation into the Cell Pouch in a Hemophilia A animal model, the patient's Factor VIII corrected cells survived at three months (the duration of the study).
Initial results showed Factor VIII released from the cells in the Cell Pouch was detected in blood and notably, showed improved clotting when compared to the Hemophilia A animal control which did not receive human corrected cells.
The steps of the cell production process were documented towards development of the cGMP manufacturing process for the corrected cells for future clinical use. An Instructions-for-Use document was also developed for implantation of the cGMP Cell Pouch and transplantation of patient corrected Factor VIII producing cells applicable for future human testing in patients with Hemophilia A.
Sernova's President & CEO, Dr. Philip Toleikis added, "These series of breakthroughs demonstrate success in correcting a Hemophilia A patient's own cells to produce the missing Factor VIII protein and to improve blood clotting in an animal model of Hemophilia A in preparation for future human clinical trials."
Technical results will continue to be presented by HemAcure Consortium members at scientific conferences and disseminated in scientific publications. Furthermore, steps are to complete cGMP manufacturing of the cells using the tools and methods developed by the Consortium, optimize dosing of cells in the Cell Pouch to achieve optimal therapeutic blood levels of Factor VIII, and conduct final formal studies in preparation for a first-in-human clinical study.
HemAcure's webinar in which the consortium expert leaders present an overview of the findings is now available both on Sernova's website at www.sernova.com and the HemAcure project website at www.hemacure.eu
The overall objective of the HemAcure project is 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 has awarded approximately EUR5.5 million (CDN$8.5) to support the project. International consortium members include the University Hospital Wuerzburg (Coordinating Institute), Germany, IMS - Integrierte Management, Heppenheim, Germany, Universita 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 has received funding from the European Union's Horizon 2020 Research and Innovation Programme under grant agreement No 667421. The Consortium has been working 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 more information, visit www.hemacure.eu
About Hemophilia A
People with Hemophilia A have prolonged abnormal bleeding as a result of trauma. 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 US 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. Annual worldwide costs for the treatment of the disease for each patient may range from $60,000 to $260,000 US for a total cost of approximately $15B per year.
About Horizon 2020 Programme
Horizon 2020 is the biggest EU Research and Innovation program ever with nearly EUR80 billion of funding available over seven years (2014 to 2020). It promises more breakthroughs, discoveries and world-firsts by taking great ideas from the lab to the market. The project is funded as part of societal challenges "personalizing health and care" in a specific call about innovative treatments and technologies. New therapies, such as gene or cell therapies, often require technological innovation in the form of development of specific component tools and techniques such as isolation and multiplication of a cell or development of a scaffold, delivery of the therapy to the patient and for following-up the effect of the therapy in the patient. In particular, achieving therapeutic scale production and cGMP standards at reasonable cost is often underestimated. The European Union aims to improve the development of advanced methods and devices for targeted and controlled delivery, and to bring these innovative treatments to the patient.
About Sernova Corp
Sernova Corp is developing regenerative medicine therapeutic 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 A, and other diseases treated through replacement of proteins or hormones missing or in short supply within the body. For more information, please visit www.sernova.com
For further information contact:
Danny Matthews, Solebury Trout
Tel: (646) 378-2928
Dominic Gray, Corporate Communications
Tel: (519) 858-5126
Ray Matthews & Associates
Tel: (604) 818-7778
This release may contain forward-looking statements. Forward-looking statements are statements that are not historical facts and are generally, but not always, identified by the words "expects", "plans", "anticipates", "believes", "intends", "estimates", "projects", "potential" and similar expressions, or that events or conditions "will", "would", "may", "could" or "should" occur. Although Sernova believes the expectations expressed in such forward-looking statements are based on reasonable assumptions, such statements are not guarantees of future performance, and actual results may differ materially from those in forward-looking statements. Forward-looking statements are based on the beliefs, estimates and opinions of Sernova's management on the date such statements were made, which include our beliefs about the conduct and outcome of the Hemophilia A program. 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.