ARU-2801

NEW YORK and BASEL, Switzerland, Sept. 20, 2021 /PRNewswire/ -- Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, today announced that data on ARU-2801, a potentially curative gene therapy for people living with hypophosphatasia (HPP), will be highlighted in two poster presentations at the American Society for Bone and Mineral Research (ASBMR) 2021 Annual Meeting which is taking place in person and virtually from October 1 to October 4, 2021. ARU-2801 is a one-time, adeno-associated virus (AAV) gene therapy designed to deliver potentially curative efficacy to patients with HPP without the limitations of chronic administration. Preclinical research shows treatment with ARU-2801 results in sustained elevation of tissue non-specific alkaline phosphatase (TNAP), the missing enzyme in HPP, at levels that ameliorate disease symptoms. Manufacturing process development and Investigational New Drug application-enabling studies are currently underway. "The data being presented at ASBMR support the development of ARU-2801 as a new, one-time treatment option for patients with HPP," said Will Chou, M.D., chief executive officer of Aruvant. "Given the chronic nature and injection site issues associated with the current standard of care, there is a substantial unmet need that we are focused on addressing with ARU-2801." The preclinical study presented by Dr. Koichi Miyake's laboratory at Nippon Medical School examined the use of ARU-2801 administered as a single injection in a murine model for severe infantile HPP and its ability to improve bone maturation and long-term survival. The treated mice exhibited high plasma alkaline phosphatase activity, normal function and behavior throughout their lives and lived for the duration of the study, which was up to 18 months after injection. The study conducted in the laboratory of Dr. José Luis Millán, professor in the Human Genetics Program at Sanford Children's Health Research Center, showed ARU-2801 extended the lifespan of the HPP mice and showed improved dentoalveolar phenotypes. Both studies showed no evidence of ectopic or vascular calcifications with the therapeutic doses used in treated HPP mice. Data from both studies suggests that using ARU-2801, an AAV gene therapy, may provide durable clinical benefit to HPP patients after a single injection. Presentation Information Poster Title:  Successful adeno-associated virus mediated neonatal gene therapy treatment of a murine model of infantile hypophosphatasia resulted in bone maturation and increased survival to at least 18 months. Time: The data will be presented on Friday, October 1, from 5:00 PM to 6:30 PM PT. An additional live virtual chat will occur on Sunday, October 3, from 1:00 PM to 2:00 PM PT. Abstract: The abstract (VPL-412) can be found here in the program. Poster Title: Adeno-associated virus TNAP-D10-mediated gene therapy improves skeletal and dentoalveolar phenotypes in Alpl-/- mice Time: The data will be presented on Friday, October 1, from 5:00 PM to 6:30 PM PT. Abstract: The abstract (VPL-410) can be found here in the program. 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 hypophosphatasia, 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 and for more on the company, please visit . Follow Aruvant on Facebook, Twitter @AruvantSciences and on Instagram @Aruvant_Sciences.

NEW YORK and BASEL, Switzerland, May 27, 2021 /PRNewswire/ -- Aruvant Sciences ("Aruvant"), a private company focused on developing gene therapies for rare diseases, today announced that data on ARU-1801, a potentially curative lentiviral gene therapy for sickle cell disease (SCD), will be delivered in an oral presentation at the 15th Annual Sickle Cell Disease Research & Educational Symposium & 44th National Sickle Cell Disease Scientific Meeting. Dr. Monika Asnani, professor of family medicine & epidemiology at the Caribbean Institute for Health Research, will present data from the ARU-1801 Phase 1/2 MOMENTUM clinical trial during the meeting which will take place virtually from May 27 to May 30, 2021. ARU-1801 is a lentiviral gene therapy that is administered once and designed to address the limitations of current curative treatment options, such as low donor availability and toxicity from conditioning chemotherapy. Dr. Asnani's oral presentation will review early safety and efficacy results from the first three patients in the ongoing MOMENTUM study of ARU-1801 with reduced intensity conditioning (RIC) in patients with SCD. "Dr. Asnani will be presenting preliminary data that shows the promise of ARU-1801 as a potentially curative gene therapy that can be delivered without exposing patients to high intensity chemotherapy," said Will Chou, M.D., chief executive officer of Aruvant. "Long-term results from the first two patients showed between 85 and 93 percent reduction in the number of vaso-occlusive events (VOEs) and complete resolution of VOEs at 12 months for the third patient, the first participant treated with a newer manufacturing process used to improve efficacy. These results indicate that it may be possible to successfully treat individuals with SCD using only reduced intensity conditioning with ARU-1801." Treatment with ARU-1801 has shown meaningful improvement in clinical outcomes. In the 24 months before treatment with ARU-1801, patients had 12 to 41 VOEs and were hospitalized for one to seven of those VOEs. In the 24 months after treatment, patients one and two have seen 93% and 85% reductions in the number of VOEs, respectively and patient three has had no VOEs through 12 months of follow-up, a 100% reduction. The corresponding total days in hospital associated with those VOEs had an average reduction of 93.8 percent. These results are an encouraging sign of the therapeutic benefit of ARU-1801 with RIC for patients with SCD. Presentation Information Presentation Title: Early Results from a Phase 1/2 Study of ARU-1801 Gene Therapy for Sickle Cell Disease Session: Abstract Breakout Session I Clinical Research Oral Presentations Date: Sunday, May 30, 2021 Time: 11:00 a.m. EDT Abstract: The agenda and abstract can be found here. About ARU-1801 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 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 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 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 and for more on the company, please visit . Follow Aruvant on Facebook, Twitter @AruvantSciences and on Instagram @Aruvant_Sciences. About Roivant 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 .