–Ongoing manufacturing improvements shows enhanced efficacy of ARU-1801 for the treatment of sickle cell disease–

New York, NY, Nov. 4, 2020 –Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, announced that an abstract demonstrating clinical benefit of the company’s lead product candidate ARU-1801 has been published online and will be the subject of an oral presentation at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition. The meeting will take place virtually from December 5 to 8. Michael S. Grimley, M.D., Professor of Clinical Pediatrics, Medical Director of the Division of Bone Marrow Transplantation and Immune Deficiency at the Cincinnati Children’s Hospital Medical Center, will present the data at 2:15 PM ET on December 7, 2020. Today the abstract was published online and will be published in the upcoming supplemental issue of Blood.

Data presented at ASH is from the 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, which continues to enroll patients, examines ARU-1801, an autologous cell therapy leveraging a modified gamma globin lentivirus vector, 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 treatment options, such as low donor availability and the risk of Graft-versus-Host Disease (GvHD) seen with allogeneic stem cell transplants. The data to be presented at ASH highlights participants dosed with product manufactured with both the original academic manufacturing process and the enhanced Process II.

“The clinical results thus far demonstrate that ARU-1801 holds significant promise for achieving durable responses with a reduced intensity conditioning approach to gene therapy,” said Dr. Grimley, a principal investigator in the MOMENTUM study. “Given the impact chemotherapy toxicity has on physician and patient decision making around treatment options, ARU-1801 has the potential to be a unique option for SCD patients seeking gene therapy.”

Abstract and Oral Presentation Information

Title: Early Results from a Phase 1/2 Study of ARU-1801 Gene Therapy for Sickle Cell Disease (SCD): Manufacturing Process Enhancements Improve Efficacy of a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant
Publication Number: 680
Session Name: 114. Hemoglobinopathies, Excluding Thalassemia—Clinical: Novel Treatments for Sickle Cell Disease
Date: Monday, December 7, 2020
Session Time: 1:30 PM – 3:00 PM
Presentation Time: 2:15 PM

About ARU-1801
ARU-1801 is a one-time potentially curative investigational gene therapy for individuals living with sickle cell disease. This product candidate was designed to address the limitations of current treatment options including chemotherapy toxicity, donor availability, and chronic administration, and replace it with a curative therapy. ARU-1801 incorporates a patented modified gamma-globin into autologous stem cells, with the aim of restoring normal red blood cell function through increased levels of fetal hemoglobin. The high potency of the modified gamma globin enables ARU-1801 engraftment with only reduced intensity conditioning (RIC). Preliminary clinical data from the MOMENTUM study, an ongoing Phase 1/2 trial in patients with sickle cell disease, demonstrate continuing durable reductions in disease burden.

The MOMENTUM Study
Aruvant is currently conducting the MOMENTUM study, which is evaluating ARU-1801, a one-time only potentially curative gene therapy for patients with SCD. This Phase 1/2 study currently is enrolling individuals with SCD, and information may be found at www.momentumtrials.com.

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 is an investigational lentiviral gene therapy currently in a clinical trial, the MOMENTUM study, as a potential one-time curative treatment for SCD. Preliminary clinical data from the ongoing Phase 1/2 study demonstrated engraftment of ARU-1801 and amelioration of SCD is possible with one dose of low intensity chemotherapy. For more information on the clinical study, please visit www.momentumtrials.com and for more on the company, please visit www.aruvant.com.

Aruvant Contact
Blair Clark-Schoeb
blair.clark-schoeb@aruvant.com