New York, NY, Feb. 3, 2021 –Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, today announced that the European Medicines Agency (EMA) granted Priority Medicines (PRIME) designation to ARU-1801, a one-time investigational gene therapy for sickle cell disease (SCD).
“PRIME designation from EMA highlights the importance of ARU-1801, administered with only reduced intensity conditioning, for the treatment of individuals with severe sickle cell disease,” said Will Chou, M.D., Aruvant chief executive officer. “With PRIME, we will be able to work closely with EMA on the development of ARU-1801, with the goal of rapidly bringing this potential cure to SCD patients in Europe.”
PRIME was created by the European Medicines Agency (EMA) to enhance support for the development of innovative medicines that target an unmet medical need and demonstrate the potential to achieve relevant clinical outcomes on morbidity, mortality or underlying disease progression. The PRIME designation offers enhanced early interaction with companies developing promising medicines, to optimize development plans and speed up evaluation. PRIME focuses on medicines that may offer a major therapeutic advantage over existing treatments, or that benefit patients without treatment options.
ARU-1801 was designated PRIME status based on clinical data from the MOMENTUM study, an ongoing Phase 1/2 trial of ARU-1801 in patients with severe sickle cell disease, that demonstrate meaningful, durable reductions in disease burden.
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 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 chemotherapy 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. Preliminary clinical data from the MOMENTUM study, an ongoing Phase 1/2 trial of ARU-1801 in patients with severe sickle cell disease, demonstrate continuing durable reductions in disease burden.
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 which includes a patient brochure, an eligibility questionnaire and information for healthcare providers.
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. 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.
E. Blair Clark-Schoeb Senior Vice President, Communications