BASEL, Switzerland and CINCINNATI, Nov. 26, 2018 /PRNewswire/ -- Roivant Sciences and Cincinnati Children's Hospital Medical Center today announced the launch of Aruvant Sciences, a biopharmaceutical company focused on developing innovative gene therapies for hematological conditions, and the formation of a new nonprofit foundation with the mission of improving access to medical care for patients with sickle cell disease in the developing world. The launch of Aruvant builds on a partnership between Roivant and Innovation Ventures, the commercialization arm of Cincinnati Children's, dating back to early 2015.

The lead candidate in Aruvant's pipeline, RVT-1801, is an investigational gene therapy for sickle cell disease and β-thalassemia. RVT-1801 utilizes proprietary technology intended to increase functioning red blood cells by inserting a modified fetal hemoglobin gene into autologous stem cells through a lentiviral vector. Studies have indicated that sickle cell patients with elevated levels of fetal hemoglobin have fewer vaso-occlusive crises and hospitalizations, due in part to improved characteristics of fetal hemoglobin relative to adult hemoglobin. RVT-1801 is the only known clinical-stage gene therapy to deliver the gene encoding fetal hemoglobin, which has been modified to optimize oxygen carrying capacity and anti-sickling properties. Myrtle Potter, Vant Operating Chair at Roivant Pharma, will serve as Chairman of Aruvant.

RVT-1801 was developed in the laboratory of Dr. Punam Malik, Director of the Cincinnati Comprehensive Sickle Cell Center at Cincinnati Children's. Preliminary clinical data from an ongoing Phase 1/2 study of RVT-1801 in patients with sickle cell disease conducted using a reduced-intensity conditioning (RIC) regimen enabled by the unique properties of modified fetal hemoglobin will be presented by Dr. Malik in an oral presentation at 6:15pm PST next Monday, December 3^rd, at the Annual Meeting and Exposition of the American Society of Hematology in San Diego, CA. Additional information on the presentation is available here.

"Treating sickle cell anemia and β-thalassemia has been my passion since I first trained as a physician in India," said Dr. Malik. "I look forward to sharing important data next week at ASH on RVT-1801, which has been designed to improve patient experience and access through our RIC regimen. I am excited to work with the team at Roivant to rapidly advance the development of this potentially transformative therapy. A critical reason why we chose to work with Roivant on this program was their authentic commitment to patients globally."

"We are excited to develop this novel modified fetal hemoglobin gene therapy for sickle cell disease," said Dr. Mayukh Sukhatme, President of Roivant Pharma. "Our partnership with Cincinnati Children's represents Roivant's commitment to the development of novel, transformational therapies, including gene therapies that have the potential to shift treatment paradigms for serious diseases."

Under the terms of the agreement, Cincinnati Children's will receive an upfront payment and shares of Aruvant and will be eligible for milestone payments and tiered royalties. Specific terms were not disclosed.

Roivant Foundation

In addition to establishing Aruvant, Roivant has created a nonprofit organization, the Roivant Foundation, focused on improving access to therapies for sickle cell disease for patients in the developing world. Over 70% of individuals with sickle cell disease live in Sub-Saharan Africa.

"As a native of Cincinnati, I am particularly excited about Roivant's sustained partnership with Cincinnati Children's, which dates back to our first year of operations," said Vivek Ramaswamy, Founder and CEO of Roivant. "I am also proud of our creative efforts to improve access to care in the developing world through the work of the Roivant Foundation."

"Roivant has been a valued partner of Cincinnati Children's for several years, and we are delighted to work together on the launch of Aruvant, a new company which will have staff based right here in Cincinnati," said Dr. Peggy Hostetter, Chief Medical Officer of Cincinnati Children's and Director of the Cincinnati Children's Research Foundation. "An important goal of our research enterprise is to facilitate the translation of promising discoveries from bench to bedside, and the closing of this deal represents a significant step in ensuring this program has the potential to impact patients here in Cincinnati and around the world."

About Sickle Cell Disease and β-Thalassemia

Sickle cell disease is a progressively debilitating and life-threatening inherited red blood cell disorder that causes a patient's oxygen-carrying cells to be abnormally inflexible and sickle-shaped. This genetic defect leads to lowered oxygen-carrying capacity in the hemoglobin protein of these red blood cells. Sickle cell disease can cause inflammation of extremities, bacterial infections, stroke, anemia, and attacks of pain called sickle cell crises.

β-thalassemia is an inherited red blood cell disorder distinguished by reduced or nonexistent production of functional β-globin, a critical component of adult hemoglobin. Patients with the disorder suffer from anemia, which can cause weakness, fatigue, and more serious complications. Individuals with β-thalassemia are at an increased risk of developing abnormal blood clots.

About Aruvant Sciences

Aruvant Sciences is a clinical-stage gene therapy company focused on hematological conditions, with an emphasis on helping patients suffering from sickle cell disease and β-thalassemia. The company's lead candidate, RVT-1801, is a modified fetal hemoglobin gene therapy for sickle cell disease and related hemoglobinopathies.

About Roivant Pharma

Roivant Pharma is a business unit of Roivant Sciences. Roivant Pharma is focused on end-to-end biopharmaceutical company creation, launch, and oversight. Roivant Pharma companies include Axovant, Myovant, Dermavant, Enzyvant, Urovant, Metavant, Genevant, Altavant, Immunovant, Respivant, Aruvant, and Arbutus.

About Roivant Sciences

Roivant aims to improve health by rapidly delivering innovative medicines and technologies to patients. We do this by building Vants – nimble, entrepreneurial biotech and healthcare technology companies with a unique approach to sourcing talent, aligning incentives, and deploying technology to drive greater efficiency in R&D and commercialization.

For more information, please visit www.roivant.com.

Contact:

Roivant Sciences

Paul Davis

Head of Communications

Email: [email protected]

Cincinnati Children's Hospital

Mike Pistone

Director, Innovation Acceleration

Email: [email protected]

SOURCE Roivant Sciences

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