Digitalis portfolio company Scout Bio, a biotechnology company focused on revolutionizing pet medicine by delivering a pipeline of one-time therapeutics for major chronic pet health conditions, today announced preliminary results from a pilot study of SB-001 demonstrating statistically significant clinical outcomes for SB-001, a novel AAV gene therapy expressing feline erythropoietin (fEPO), indicated for chronic kidney disease (CKD)-associated anemia.
“This data presents a game-changing opportunity for the animal health industry, and to our knowledge, is the first multi-center AAV gene therapy study conducted in the U.S. in client owned animals,” says Mark Heffernan, Ph.D., CEO of Scout Bio. “Not only is this a major milestone for Scout Bio, but it also shifts the paradigm in the animal health industry for the delivery of therapeutic peptides and proteins that are effective and safe long-term. These results reinforce gene therapy’s potential for the treatment of acquired chronic disease and paves the way for Scout Bio’s evolving gene therapy pipeline to advance into other major chronic diseases, such as diabetes, chronic pain and atopic dermatitis.”
The study compared the change in Packed Cell Volume (PCV), an indicator of increased hematocrit or red blood cell (RBC) number, from baseline in sixteen client owned cats with end-stage kidney disease. The study was designed to identify an optimized dose and to run a minimum of 70 days and maximum of 126 days for each animal. All cats received the therapy and two dosing protocols were evaluated, with success defined as an increase in PCV into the normal range or ≥5 percentage points above baseline.
Protocol A and B combined resulted in a 71% success rate and a significant increase in mean PCV (p<0.05). The success rate in protocol B alone, which allowed an optional second dose, was 87%. The increase in PCV resulted in improved clinical signs and quality of life scores. A few cats were followed beyond Day 70 and maintained an increased PCV for up to 152 days. No cat had adverse clinical signs attributed to SB-001 treatment. The study is ongoing and a protocol C (optimized single dose) is being tested, with results expected in early 2021.
This interim study data strongly validates that SB-001’s expressed feline erythropoietin (fEPO) results in a prolonged therapeutic effect after a single treatment protocol, rather than the peaks and troughs of multiple injections with off-label human EPO treatment.
Anne Traas, DVM, MS, DACT, Chief Development Officer for Scout Bio and head of the clinical team leading the study, explains, “When a cat develops anemia associated with CKD, it can be life-disrupting for the cat and the entire family. With no approved treatments available, owners are limited in helping their beloved pets overcome the symptoms of anemia which include extreme lethargy, weakness, increased respiratory effort, which often leads to euthanasia. This data demonstrates that SB-001 has the ability to increase PVC into the normal range, resulting in an improved quality of life for the cat by reducing, or eliminating, the clinical effects of the anemia and presents an enormous advantage in convenience and improved compliance.”
James Wilson, M.D., Ph.D. Director of the Gene Therapy Program at the University of Pennsylvania and a co-founder of Scout Bio commented, “I am thrilled that our work in animal models of human diseases is being used to inform the development of one-time treatments for disorders that commonly affect dogs and cats. I got into this field 35 years ago to help patients with chronic and disabling diseases, which is exactly what Scout Bio hopes to do in companion animal populations.”
SB-001 has a unique and innovative adeno-associated viral (AAV) vector, containing the gene for feline EPO, for delivery into the muscle cell nucleus which will continually produce therapeutic levels of the hormone. The expressed feline EPO stimulates erythropoiesis, or red blood cell production. The U.S. Food and Drug Administration (FDA) CVM has confirmed that SB-001 is eligible for expanded conditional approval, potentially shortening the time until this product could be approved and made available.
About Chronic Kidney Disease (CKD)-Associated Anemia
Anemia associated with chronic kidney disease (CKD) in cats is a disease of high unmet medical need, with no FDA-approved therapies currently available. CKD is one of the most common diseases in senior cats and is an end-of-life disease that creates quality of life issues for cats and their owners. Up to 21% of cats with CKD have anemia and the only currently available therapeutic options are multiple blood transfusions or repeated injections of off-label human drugs. Compared to the use of human EPO drugs, SB-001 is expected to have a superior safety profile with respect to immune reactions and incidence of pure red cell aplasia (PRCA), a serious life-threatening condition.
About Scout Bio
Scout Bio is a biotechnology company focused on revolutionizing pet medicine by delivering a pipeline of one-time therapeutics for major chronic pet health conditions. Scout’s therapeutics are designed to induce long-term expression of therapeutic proteins in pet patients using AAV vector technology. Scout has an exclusive research and development collaboration with the University of Pennsylvania’s Gene Therapy Program. Scout Bio’s innovative partnerships build on a 20-year history with AAV leaders and is complemented by Scout Bio’s global leaders in gene therapy research and development. Scout Bio is a private company headquartered in Philadelphia, Pennsylvania.
Penn Financial Disclosure
Dr. Wilson is a Penn faculty member and also a scientific collaborator, consultant and co-founder of Scout Bio. As such, he holds an equity stake in the Company, receives sponsored research funding from Scout Bio, and as an inventor of the licensed technology he may receive additional financial benefits under the license in the future. The University of Pennsylvania also holds equity and licensing interests in Scout Bio.
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