NEW YORK and BASEL, Switzerland, Nov. 4, 2021 /PRNewswire/ -- Aruvant Sciences ("Aruvant"), a private company focused on developing gene therapies for rare diseases, announced that an abstract demonstrating the clinical benefit of the company's lead product candidate ARU-1801 has been published online and will be the subject of a poster presentation at the 63rd American Society of Hematology (ASH) Annual Meeting and Exposition. The meeting will take place in Atlanta, Georgia from December 11 to 14, 2021. Punam Malik, M.D., Director of the Cincinnati Comprehensive Sickle Cell Center and Program Leader of the Hematology and Gene Therapy Program at the Cincinnati Children's Hospital Medical Center, will present the data at 6:00 to 8:00 PM EST on December 13, 2021.
"Clinical data from our ongoing MOMENTUM study has shown 100 percent resolution of vaso-oclusive events (VOE) in our recently treated SCD patients at 18 and 12 months of follow up," said Will Chou, MD, Aruvant chief executive officer. "In addition, we are excited to be sharing additional clinical data at ASH that demonstrates how the unique anti-sickling potency of ARU-1801 translates to these robust clinical outcomes."
Dr. Malik will present data from the ongoing MOMENTUM study, an open label Phase 1/2 clinical trial examining ARU-1801 as a one-time potentially curative gene therapy for individuals with sickle cell disease (SCD). The MOMENTUM study examines ARU-1801, an autologous lentiviral cell therapy with a modified, highly potent gamma globin payload, in individuals with severe SCD. Unlike investigational gene therapies that require fully myeloablative conditioning, ARU-1801 is given with reduced intensity conditioning (RIC), which is a lower dose chemotherapy. ARU-1801 is designed to address the limitations of current curative allogeneic transplant options, such as low donor availability, the risk of graft-versus-host disease and toxicity from myeloablative chemotherapy. The data to be presented at ASH highlights clinically meaningful reduction in participants' VOEs and the unique attributes that contribute to the potency of ARU-1801.
"The emerging clinical data shows that ARU-1801 holds promise for achieving durable responses in patients with severe SCD using only reduced intensity conditioning—a key differentiator from other investigational gene therapy regimens," said Dr. Malik. "Given the advantage for patients, providers and payers a reduced conditioning regimen offers, ARU-1801 has the potential to be an important option for SCD patients seeking gene therapy, including those in low resource settings."
Abstract and Poster Presentation Information
Title: Safety and Efficacy of ARU-1801 in Patients with Sickle Cell Disease: Early Results from the Phase 1/2 MOMENTUM Study of a Modified Gamma Globin Gene Therapy and Reduced Intensity Conditioning
Publication Number: 3970
Session Name: 801. Gene Therapies: Poster III
Date: Monday, December 13, 2021
Presentation Time: 6:00 to 8:00 PM EST
Location: Georgia World Congress Center, Hall B5 or online through the ASH website.
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