NEW YORK, Dec. 7, 2020 /PRNewswire/ -- Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, announced new data demonstrating the potentially curative effect of ARU-1801 as a one-time investigational gene therapy for sickle cell disease (SCD) during an oral presentation at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition. 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, is presenting the data today, December 7, 2020, at 2:15 PM PT.
"ARU-1801 demonstrated clinically meaningful long-term reductions in sickle cell disease burden," said Dr. Grimley. "The potentially curative efficacy seen from the new manufacturing process and three year durability demonstrated by the first treated patient show that ARU-1801, given with only reduced intensity conditioning, can be an important and more tolerable treatment option for patients with severe SCD."
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 CRISPR 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 conditioning 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 data being presented today highlights participants dosed with product manufactured with both the original academic manufacturing process (Process I) and the enhanced Process II. ARU-1801 incorporates a modified gamma-globin gene 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 to engraft using RIC.
Highlights of the data:
- The first patient dosed has shown durable engraftment and stable fetal hemoglobin levels for more than three years.
- Patient 3, the first patient treated with Process II manufacturing, has shown improved outcomes including:
- Fetal hemoglobin of 38 percent at nine months.
- F cells (red blood cells that contain fetal hemoglobin) were 71 percent at six months and are expected to rise as transfused blood recedes.
- F-retics (new red blood cells that contain fetal hemoglobin) were 92 percent at six months.
- Normalization of total hemoglobin levels and measures of hemolysis.
- No vaso-occlusive episodes ("VOEs") through nine months.
- All participants had between 85 and 100 percent reduction in VOEs.
- ARU-1801 demonstrated a favorable safety profile with no treatment-related adverse events and no chemotherapy related serious adverse events to date.
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.
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. For more information on the clinical study, please visit www.momentumtrials.com and for more on the company, please visit www.aruvant.com.
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.
SOURCE Aruvant Sciences