-- Stable transduction of fetal hemoglobin seen in sickle cell disease patients given ARU-1801 gene therapy in Phase 1/2 trial --
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 myeloablative 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 peripheral 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
- ARU-1801 offers potential long-term amelioration of SCD phenotype without the toxicities and resource utilization required by other approaches that use myeloablative conditioning.
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/.
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.
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.
SOURCE Aruvant Sciences