Aruvant Announces the European Medicines Agency (EMA) Granted Priority Medicines (PRIME) Designation to ARU-1801 for the Treatment of Sickle Cell…

Posted: February 6, 2021 at 4:51 am

NEW YORK, Feb. 3, 2021 /PRNewswire/ -- 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 EMAhighlightsthe importance of ARU-1801, administeredwith only reduced intensity conditioning,for the treatment ofindividuals with severe sickle cell disease,"said Will Chou, M.D., Aruvant chief executiveofficer."With PRIME,we will be able to work closely with EMAon the development of ARU-1801, with the goal of rapidlybringingthispotential cure toSCD patients in Europe."

PRIME was created by the European Medicines Agency (EMA) to enhance support for the development of innovative medicinesthat target an unmet medical needand demonstratethe potential to achieve relevant clinical outcomes on morbidity, mortality or underlying disease progression. The PRIME designation offersenhanced early interaction with companiesdeveloping 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 MOMENTUMstudy, an ongoing Phase 1/2 trial of ARU-1801 in patients with severe sickle cell disease, that demonstrate meaningful,durable reductions in disease burden.

About ARU-1801ARU-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 MOMENTUMstudy, 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 StudyAruvant 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 http://www.momentumtrials.comwhich includes a patient brochure, an eligibility questionnaireand information for healthcare providers.

About Aruvant SciencesAruvant 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 talentedteamwith extensive experience in the development, manufacturing and commercialization of gene therapy products. Aruvant has an activeresearchprogram with a lead product candidate, ARU-1801, in development for individuals suffering fromsickle cell disease(SCD). ARU-1801, an investigational lentiviral gene therapy, is being studied in aPhase 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 http://www.momentumtrials.comand for more on the company, please visitwww.aruvant.com. Follow Aruvant on Facebook, Twitter @AruvantSciencesand on Instagram @Aruvant_Sciences.

About RoivantRoivant'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 http://www.roivant.com.

SOURCE Aruvant Sciences

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