ARU-1801 reduced vaso-occlusive events and days in the hospital for patients with severe sickle cell disease

Aruvant Sciences ("Aruvant"), a private company focused on developing gene therapies for rare diseases, announced that data demonstrating the clinical benefit of the company's lead product candidate ARU-1801 for severe sickle cell disease (SCD) will be presented today in an oral presentation at the American Society of Gene and Cell Therapy (ASGCT) 25^th annual meeting. Punam Malik, M.D., Director of the Cincinnati Comprehensive Sickle Cell Center and Program Leader of the Hematology and Gene Therapy Program at the Cincinnati Children's Hospital Medical Center, will deliver the oral presentation this afternoon at 1:30 PM EDT.

"The data demonstrate that the ARU-1801 gene therapy may not only be able to reduce severe vaso-occlusive events (VOEs) but also reduce days in the hospital for SCD patients which could provide a clinically meaningful benefit for patients and help reduce health care costs," said Dr. Malik. "ARU-1801 is the only gene therapy in development that has been shown to achieve durable responses in patients with severe SCD using only reduced intensity conditioning—a key differentiator from other investigational gene therapy and gene editing regimens."

Dr. Malik will be presenting data from the ongoing MOMENTUM Phase 1/2 clinical trial that is examining ARU-1801, an autologous lentiviral cell therapy with a modified, highly potent gamma globin payload, in individuals with severe SCD. 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 chemotherapy. Unlike investigational gene therapies that require fully myeloablative conditioning, ARU-1801 is given with reduced intensity conditioning (RIC), which is a lower dose chemotherapy associated with less chemotherapy side effects including shorter time in the hospital and less short- and long-term toxicity than myeloablative approaches.

The data being presented demonstrates:

• All five study participants who had sufficient follow-up have had significant reductions in severe VOEs, demonstrating clinically meaningful reductions in disease burden;
• Participants have experienced durable engraftment and improvement in SCD burden without the use of myeloablative chemotherapy; and
• Four of the five patients treated have experienced complete resolution of severe VOEs since they received the treatment.

The ASGCT annual meeting is taking place in Washington, D.C. and virtually starting today, May 16, and will go through to May 19, 2022. For more information about the conference, please visit https://annualmeeting.asgct.org/.

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, Twitter @AruvantSciences, Instagram @Aruvant_Sciences or LinkedIn @AruvantSciences.