Aruvant Announces ARU-2801 Data Publications and Advocacy Partnership
--ARU-2801 is a potentially curative gene therapy in development for individuals with hypophosphatasia (HPP)--
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)
About Aruvant Sciences
About Soft Bones
SOURCE Aruvant Sciences