Aruvant Announces Data Presentation at European Hematology Association 2022 Hybrid Congress
13 Jun 2022
Clinical ResultGene TherapyCell Therapy
MILLBURN, N.J. and BASEL, Switzerland, June 10, 2022 /PRNewswire/ -- Aruvant Sciences ("Aruvant"), a private company focused on developing gene therapies for rare diseases, announced that Michael W. Kent, M.D., pediatric blood and marrow transplant physician at Atrium Health/Levine Children's Hospital, is presenting new data on ARU-1801, a one-time gene therapy for individuals with severe sickle cell disease (SCD), at the European Hematology Association (EHA) 2022 Hybrid Congress. The data is from the ongoing MOMENTUM Phase 1/2 study which is examining the potential for ARU-1801 to increase levels of fetal hemoglobin (HbF) to reduce or eliminate vaso-occlusive events (VOEs).
"The data shows that ARU-1801 offers potential long-term improvement of the primary symptoms seen with SCD without the toxicities and resource utilization associated with fully myeloblative chemotherapies," said Dr. Kent. "The strong engraftment of ARU-1801 in patients with severe SCD is particularly notable since individuals were only given a single low dose of chemotherapy."
The data from the ongoing MOMENTUM Phase 1/2 clinical trial examining the clinical benefit of ARU-1801 demonstrates:
Strong long-term hematopoietic stem cell (HSC) engraftment as demonstrated by high, stable ratios of perhiperhal blood (PB) to drug product (DP)VCN, achieved with only reduced intensity conditioning (RIC);
ARU-1801 can reach effective levels of anti-sickling hemoglobin at vector copy numbers (VCN) less than or equal to one, with approximately 40 percent HbFG16D expression per drug product VCN;
HbFG16D may have a more potent anti-sickling effect than endogenous HbF; and
The EHA 2022 Hybrid Congress is taking place in Vienna, Austria from June 9 to June 12, 2022. The poster presentation is abstract #: EHA-2237. For more information, please visit https://ehaweb.org/congress/eha2022-hybrid/eha2022-congress/.
About ARU-1801 and the MOMENTUM Study
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 conditioning chemotherapy. The data being presented highlights clinically meaningful reduction in participants' vaso-occulsive events, which are the painful crisis that are commonly experienced by SCD patients.
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 https://sicklecellstudies.com/, and for more on the company, please visit www.aruvant.com. Follow Aruvant on Facebook, LinkedIn, Twitter @AruvantSciences, Instagram @Aruvant_Sciences or LinkedIn @AruvantSciences.
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