- Data suggest individualized melphalan dosing prior to ARU-1801 infusion may improve patient outcomes
NEW YORK and BASEL, Switzerland, May 12, 2021 /PRNewswire/ -- Aruvant Sciences ("Aruvant"), a private company focused on developing gene therapies for rare diseases, today announced the selection of an abstract for presentation at the European Hematology Association (EHA) Virtual Congress which will take place June 9 to 17, 2021. The data is on ARU-1801, a one-time gene therapy designed to cure sickle cell disease (SCD). The abstract is now available on the EHA website at ehaweb.org and the poster presentation will be available on June 11, 2021.
"The data being presented at EHA demonstrate that it is possible to tailor the reduced intensity melphalan dose required prior to infusing ARU-1801, a potentially curative gene therapy for sickle cell disease patients," said Will Chou, M.D., chief executive officer of Aruvant. "Some patients with SCD process melphalan differently than others. The data emerging from our Phase 1/2 trial is important because it highlights an opportunity to individualize our reduced intensity conditioning dose of melphalan and maximize the potential for ARU-1801 to engraft. The unique promise of ARU-1801 is combining the favorable toxicity profile of RIC with the potential for a cure."
The poster presentation will review the potential of a model-based, individualized melphalan dosing strategy for ARU-1801 in SCD. Melphalan exposure (AUC) after a single dose of 140 mg/m2 conditioning was observed in three patients with SCD in the ongoing Phase 1/2 MOMENTUM study and compared to predictions from a population pharmacokinetics (PK) model, based on patient covariates collected prior to melphalan infusion. These data suggest that a simple algorithm for individualized melphalan dosing based on a few covariate values has potential to achieve optimal melphalan exposure and reduce variability, which may result in improved patient outcomes after treatment with ARU-1801.
Title: Towards Patient-Specific Dosing of Melphalan Conditioning for ARU-1801, A Novel Gene Therapy for Treatment of Sickle Cell Disease
Date and Time: Friday, June 11, 2021
Abstract: The abstract was published today at the EHA 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 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. The company's second product candidate, ARU-2801, is in development to cure a devastating, ultra-orphan disorder that affects multiple organ systems and leads to high 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.
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.