NEW YORK and BASEL, Switzerland, June 14, 2021 /PRNewswire/ -- Aruvant Sciences ("Aruvant"), a private company focused on developing gene therapies for rare diseases, announced today two publications of data on ARU-2801, a potentially curative gene therapy for people living with hypophosphatasia (HPP), in Molecular Therapy: Methods & Clinical Development and the Journal of Bone and Mineral Research (JBMR). To educate the HPP community about gene therapy, Aruvant has formed a partnership with Soft Bones, Inc.: the U.S. Hypophosphatasia Foundation ("Soft Bones"), a leading advocacy group dedicated to the community of patients, caregivers and families living with HPP.
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. Aruvant acquired the license to ARU-2801 from Koichi Miyake, M.D., Ph.D., professor, the Department of Gene Therapy, Nippon Medical School in Tokyo, Japan.
"The data published in these journals 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 are looking forward to working closely with Soft Bones to better understand the journey and unmet needs of patients with HPP."
The preclinical study published by Dr. Koichi Miyake's laboratory at Nippon Medical School in Molecular Therapy: Methods & Clinical Development 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 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.
"We are looking forward to working with Aruvant to educate our community about the potential for gene therapy to play an important role in the treatment of HPP," said Deborah Fowler, founder and president of Soft Bones. "Our patients vary in severity and there remains a significant unmet need for those unable to receive treatment and those who experience administration challenges with the chronic therapy."
The JBMR publication was from a preclinical study that evaluated the efficacy of a single intramuscular injection of ARU-2801 to increase the lifespan and improve the skeletal and dentoalveolar phenotypes in a murine model for severe infantile HPP. The study was conducted in the laboratory of Dr. José Luis Millán, professor in the Human Genetics Program at Sanford Children's Health Research Center, Sanford Burnham Prebys, and funded through a grant from the National Institutes of Health. ARU-2801 extended the lifespan of the mice and no ectopic calcifications were observed in the kidneys, aorta, coronary arteries or brain of the treated mice.
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.
Roivant's mission is to improve the delivery of healthcare to patients by treating every inefficiency as an opportunity. Roivant develops transformative medicines faster by building technologies and developing talent in creative ways, leveraging the Roivant platform to launch Vants – nimble and focused biopharmaceutical and health technology companies. For more information, please visit www.roivant.com.
About Soft Bones
Soft Bones was founded in 2009 to provide information and to establish a forum to educate, empower, and connect patients living with hypophosphatasia, their families, and caregivers. The Foundation also promotes research of this rare metabolic disease through awareness and fundraising efforts. While based in the United States, Soft Bones advocates for patients around the world, through policy initiatives, research funding, collaborations, awareness and educational activities. For more information visit, softbones.org.
SOURCE Aruvant Sciences