ARU-2801 is a one-time, adeno-associated virus (AAV) gene therapy designed to deliver potentially curative efficacy to patients with HPP without the limitations of chronic administration. Preclinical research shows 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. Manufacturing process development and Investigational New Drug application-enabling studies are currently underway.
"The data being presented at ASBMR 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. "Given the chronic nature and injection site issues associated with the current standard of care, there is a substantial unmet need that we are focused on addressing with ARU-2801."
The preclinical study presented by Dr. Koichi Miyake's laboratory at Nippon Medical School examined 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 long-term survival. The treated mice exhibited high plasma alkaline phosphatase activity, normal function and behavior throughout their lives and lived for the duration of the study, which was up to 18 months after injection. The study conducted in the laboratory of Dr. José Luis Millán, professor in the Human Genetics Program at Sanford Children's Health Research Center, showed ARU-2801 extended the lifespan of the HPP mice and showed improved dentoalveolar phenotypes. Both studies showed no evidence of ectopic or vascular calcifications with the therapeutic doses used in treated HPP mice. Data from both studies suggests that using ARU-2801, an AAV gene therapy, may provide durable clinical benefit to HPP patients after a single injection.
Poster Title:Successful adeno-associated virus mediated neonatal gene therapy treatment of a murine model of infantile hypophosphatasia resulted in bone maturation and increased survival to at least 18 months.
Time: The data will be presented on Friday, October 1, from 5:00 PM to 6:30 PM PT. An additional live virtual chat will occur on Sunday, October 3, from 1:00 PM to 2:00 PM PT.
Abstract: The abstract (VPL-412) can be found here  in the program.
Poster Title:Adeno-associated virus TNAP-D10-mediated gene therapy improves skeletal and dentoalveolar phenotypes in Alpl-/- mice
Time: The data will be presented on Friday, October 1, from 5:00 PM to 6:30 PM PT.
Abstract: The abstract (VPL-410) can be found here  in the program.
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 sickle cell disease (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 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