New York, Basel, and Fargo, N.D., June 16, 2021 – Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, and Aldevron, the leading provider of high-quality plasmid DNA, mRNA and recombinant proteins necessary for vaccines, gene and cell therapy, gene editing and diagnostic applications, announced today that Aldevron will support the development of ARU-1801, Aruvant’s one-time investigational gene therapy for sickle cell disease (SCD), and ARU-2801, a one-time investigational gene therapy for hypophosphatasia (HPP). Aldevron will provide good manufacturing practice (GMP)-grade plasmid for Aruvant’s upcoming ARU-1801 pivotal trial.
“Partnering with Aldevron enables us to tap into their cutting-edge expertise in manufacturing GMP-grade plasmid, a key component of pipeline products,” said Palani Palaniappan, Aruvant’s chief technology officer. “This partnership with Aldevron marks an important milestone as we complete our ongoing SCD Phase 1/2 study and move toward our pivotal clinical trial, which we anticipate initiating next year.”
Aruvant has a hybrid supply model that is led by the company’s cell and gene therapy scientists and leverages specialized external suppliers. The company has expanded its internal technical and operational organization with experts in vector and cell manufacturing, analytics, quality, supply and regulatory affairs. To augment these internal capabilities, Aruvant is partnering with world-leading contract development manufacturing organizations. The partnership with Aldevron is a critical step to advancing the clinical development of both ARU-1801 for SCD patients and ARU-2801 for HPP patients.
“By working with Aruvant, we are able to contribute to helping develop potentially life-saving and curative therapies for patients living with devastating conditions like sickle cell disease and hypophosphatasia, which is incredibly rewarding to our whole team,” said Michelle Berg, president of Aldevron’s nucleic acids business unit. “With our continued effort to invest in our people, processes and facilities, we will bring Aruvant the best components required to develop its cutting-edge gene therapy.”
ARU-1801 is designed to address the limitations of current curative treatment options, such as low donor availability and the risk of graft-versus-host disease (GvHD) seen with allogeneic stem cell transplants. Unlike investigational gene therapies and gene editing approaches which require fully myeloablative conditioning, the unique characteristics of ARU-1801 allow it to be given with reduced intensity conditioning (“RIC”). Compared to myeloablative approaches, the lower dose chemotherapy regimen underlying RIC has the potential to reduce not only hospital length of stay, but also the risk of short- and long-term adverse events such as infection and infertility.
The MOMENTUM Study
Aruvant is conducting the MOMENTUM study, which is evaluating ARU-1801, a one-time potentially curative investigational gene therapy for patients with SCD. This Phase 1/2 study is currently enrolling participants, and information may be found at www.momentumtrials.com which includes a patient brochure, an eligibility questionnaire and information for healthcare providers.
About Hypophosphatasia (HPP)
HPP is 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 tissue non-specific alkaline phosphatase (TNSALP) enzyme. 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. HPP is remarkably wide-ranging in severity. There are five types of HPP including: perinatal, infantile, childhood, adult and odontohypophosphatasia.
ARU-2801 is a one-time, adeno-associated virus (AAV) gene therapy designed to overcome the dosing limitations of a commercially validated, chronic enzyme replacement therapy standard of care. Preclinical research shows treatment with ARU-2801 results in adequate TNSALP enzyme levels. Manufacturing process development and investigational new drug application-enabling studies are currently underway.
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.
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.
Aldevron is a premier manufacturing partner in the global genetic medicine field. Founded in 1998 by Michael Chambers and John Ballantyne, the company provides critical nucleic acids and proteins used to make gene and cell therapies, DNA and RNA vaccines, and gene editing technologies. Aldevron’s 600 employees support thousands of scientists who are developing revolutionary treatments for millions of people. For more information, visit www.aldevron.com.
E. Blair Clark-Schoeb
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