Manufacturing process development and investigational new drug application-enabling studies are currently underway for ARU-2801. Pre-clinical research has shown treatment with ARU-2801 results in sustained elevation of tissue non-specific alkaline phosphatase (TNAP), the missing enzyme in HPP, at levels that ameliorate disease symptoms.
"The data being presented at SfE BES 2021 support the development of ARU-2801 as a new, one-time treatment option for patients with HPP," said Will Chou, M.D., chief executive officer of Aruvant. "We continue to make progress with our plans to move this important investigation product forward to help address the unmet needs of patients with HPP."
The oral presentation by Dr. Tae Matsumoto on data from Dr. Koichi Miyake's laboratory at Nippon Medical School examines the use of ARU-2801 administered as a single injection in a murine model for severe infantile HPP and its ability to improve bone maturation and survival. The treated mice exhibited high plasma alkaline phosphatase activity, normal function and behavior throughout their lives and lived for up to 18 months after injection—the duration of the study. This data suggests that using ARU-2801, an AAV gene therapy, may provide durable clinical benefit to HPP patients after a single injection. The abstract is available here  and the oral presentation can be seen here .
About Hypophosphatasia (HPP)
HPP can be a devastating, ultra-orphan disorder with multi-organ damage and high mortality when left untreated. This rare disease is caused by mutations in the gene encoding the enzyme tissue nonspecific alkaline phosphatase (TNAP). HPP is remarkably wide-ranging in severity. Most often this genetic and chronic disease is characterized by defective bone mineralization that can lead to destruction and deformity of bones, profound muscle weakness, seizures, respiratory failure and premature death. There are five types of HPP including: perinatal, infantile, childhood, adult and odontohypophosphatasia. Gene therapy could be a potentially curative approach to addressing this genetic disease.
About Aruvant Sciences
Aruvant Sciences, part of the Roivant family of companies, is a clinical-stage biopharmaceutical company focused on developing and commercializing gene therapies for the treatment of rare diseases. The company has a talented team with extensive experience in the development, manufacturing and commercialization of gene therapy products. Aruvant has an active research program with a lead product candidate, ARU-1801, in development for individuals suffering from SCD. ARU-1801, an investigational lentiviral gene therapy, is being studied in a Phase 1/2 clinical trial, the MOMENTUM study, as a one-time potentially curative treatment for SCD. Preliminary clinical data demonstrate engraftment of ARU-1801 and amelioration of SCD is possible with one dose of reduced intensity chemotherapy. The company's second product candidate, ARU-2801, is in development to cure hypophosphatasia, a devastating, ultra-orphan disorder that affects multiple organ systems and leads to high morbidity and mortality when not treated. Data from pre-clinical studies with ARU-2801 shows durable improvement in disease biomarkers and increased survival. For more information on the ongoing ARU-1801 clinical study, please visit www.momentumtrials.com , and for more on the company, please visit www.aruvant.com . Follow Aruvant on Facebook , Twitter @AruvantSciences  and on Instagram @Aruvant_Sciences .
SOURCE Aruvant Sciences