Selecta Biosciences Announces Preclinical Data Showing Potential Benefit in Treatment of Pompe Disease

Selecta Biosciences, Inc. recently announced that results from preclinical studies involving the use of SVP-Rapamycin in combination with alglucosidase alfa (marketed as Myozyme and Lumizyme) to treat Pompe disease are being presented at the 13th Annual WORLD Symposium by a research team led by Priya Kishnani, MD. Dr. Kishnani is Chief of Medical Genetics at Duke University Medical Center and is one of the world’s leading experts in the treatment of Pompe disease.

“The preclinical data suggest that co-administration of an antigen-specific immune tolerance therapy could induce immune tolerance to the enzyme replacement therapy alglucosidase alfa,” said Dr. Kishnani. “We look forward to additional studies to determine whether this novel approach has the potential to improve clinical outcomes for patients.”

Pompe disease is an inherited and often fatal disorder caused by a deficiency of alpha-glucosidase, an enzyme that is required to break down a form of sugar called glycogen that is used by the cells of the body for energy. Impacting one in approximately 12,000 to 40,000 people in the US, this disease typically manifests within months of birth or early in childhood, causing an accumulation of glycogen in the heart, skeletal muscle, and other tissues and resulting in impaired motor skills, muscle weakness, breathing challenges, and reduced growth rates. If untreated, Pompe disease can lead to death from respiratory or heart failure.

While the availability of enzyme replacement therapies (ERT), such as alglucosidase alfa, have already significantly improved the overall survival and quality of life for patients with Pompe disease, these therapies are known to cause the development of anti-drug antibodies (ADAs) that can severely compromise their efficacy and safety for many patients. Data included in the prescribing information for alglucosidase alfa indicate that ADAs were detected in 89% of its clinical trial patients and adverse infusion reactions were reported in 51% of clinical trial patients.

Titled Immunomodulation to ERT with Tolerogenic Nanoparticles Containing Rapamycin for Pompe Disease, the presentation by Dr. Kishnani’s group described a recent Duke University Medical Center preclinical study demonstrating that SVP-Rapamycin induces antigen-specific immunological tolerance and mitigates ADAs to alglucosidase alfa in a mouse model of Pompe disease. Animals treated with alglucosidase alfa and SVP-Rapamycin had significantly increased glycogen clearance in skeletal muscles and improved motor function as compared to treatment with alglucosidase alfa alone or with the global immunosuppressant methotrexate.

“The preclinical data presented by Dr. Kishnani’s team further illustrates the potential use of Selecta’s SVP-Rapamycin to generate novel biologics that avoid unwanted immunogenicity,” said Takashi Kei Kishimoto, PhD, Chief Scientific Officer of Selecta. “We believe this would enhance the efficacy and safety of treatments, increase the number of treatable patients and enable entirely new novel biologic therapeutics. With our lead product candidate, SEL-212, in a Phase II trial for refractory gout and proprietary and licensed gene therapy programs underway, we are making progress in developing our pipeline in the field of antigen-specific immune tolerance.”

Selecta Biosciences, Inc. is a clinical-stage biopharmaceutical company focused on developing biologic therapies for rare and serious diseases that avoid the immune responses that compromise efficacy and lead to life-threatening complications. Selecta is applying its proprietary Synthetic Vaccine Particles (SVP) to a range of therapeutic areas in which immunogenicity is a key challenge. SEL-212, the company’s lead candidate in Phase II, is being developed to treat chronic refractory gout patients and reduce their debilitating symptoms, including flares and inflammatory arthritis. Further, Selecta’s two proprietary gene therapy product candidates have the unique potential to enable repeat administration, allowing for dose adjustment in patients and maintenance of therapeutic activity over time. The company is seeking to expand the use of its SVP platform in other areas, such as immuno-oncology, allergies, autoimmune diseases, and vaccines. Selecta is based in Watertown, MA. For more information, visit http://selectabio.com.