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NEW YORK and BASEL, Switzerland, Dec. 1, 2020 — Aruvant, a clinical-stage biopharmaceutical company focused on developing and commercializing transformative gene therapies for the treatment of rare diseases, today announced Will Chou, M.D., Aruvant chief executive officer, and other members of the management team will participate in SVB Leerink’s Biopharma Private Company Connect. For individuals interested in attending the conference, please visit SVB Leerink for more information. In addition, data on the company’s lead asset ARU-1801, a potentially curative gene therapy for sickle cell disease, will be presented December 7 at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition. For more information, please visit the ASH press release.

About Aruvant
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 is an investigational lentiviral gene therapy currently in a Phase 1/2 clinical trial, the MOMENTUM study, as a potential one-time curative treatment for SCD. Preliminary clinical data demonstrate engraftment of ARU-1801 and amelioration of SCD is possible with one dose of low 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.

About Roivant
Roivant aims to improve health by rapidly delivering innovative medicines and technologies to patients. Roivant does 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:
E. Blair Clark-Schoeb
SVP, Communications
blair.clark-schoeb@aruvant.com

NEW YORK and BASEL, Switzerland, Nov. 16, 2020 — Aruvant, a clinical-stage biopharmaceutical company focused on developing and commercializing transformative gene therapies for the treatment of rare diseases, today announced that the European Medicines Agency (EMA) has granted orphan designation for ARU-1801, an investigational lentiviral gene therapy. The U.S. Food and Drug Administration (FDA) previously granted Orphan Drug and Rare Pediatric Disease Designations to ARU-1801 for the treatment of sickle cell disease (SCD).

“The EMA orphan designation highlights the importance of ARU-1801, our potentially curative gene therapy for individuals living with severe SCD,” said Will Chou, M.D., chief executive officer of Aruvant. “We are encouraged by the preliminary clinical data from our ongoing Phase 1/2 study that demonstrates ARU-1801, administered with only reduced intensity conditioning, can achieve durable reductions in disease burden.”

Orphan drug designation in the European Union (EU) is granted by the European Commission based on a positive opinion issued by the EMA Committee for Orphan Medicinal Products. To qualify, an investigational medicine must be intended to treat a seriously debilitating or life-threatening condition that affects fewer than five in 10,000 people in the EU, and there must be sufficient non-clinical or clinical data to suggest the investigational medicine may produce clinically relevant outcomes. EMA orphan drug designation provides companies with certain benefits and incentives, including clinical protocol assistance, differentiated evaluation procedures for Health Technology Assessments in certain countries, access to a centralized marketing authorization procedure valid in all EU member states, reduced regulatory fees and 10 years of market exclusivity upon marketing authorization.

About ARU-1801
ARU-1801 is a one-time potentially curative investigational gene therapy for individuals living with sickle cell disease. This product candidate was designed to address the limitations of current treatment options including myeloablative chemotherapy toxicity, donor availability, and chronic administration, and replace it with a curative therapy. ARU-1801 incorporates a patented modified gamma-globin into autologous stem cells, with the aim of restoring normal red blood cell function through increased levels of fetal hemoglobin. The high potency of the modified gamma globin enables ARU-1801 engraftment with only reduced intensity conditioning (RIC). 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.

About Aruvant
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 is an investigational lentiviral gene therapy currently in a Phase 1/2 clinical trial, the MOMENTUM study, as a potential one-time curative treatment for SCD. Preliminary clinical data demonstrate engraftment of ARU-1801 and amelioration of SCD is possible with one dose of low 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.

About Roivant
Roivant aims to improve health by rapidly delivering innovative medicines and technologies to patients. Roivant does 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:
E. Blair Clark-Schoeb
SVP, Communications
blair.clark-schoeb@aruvant.com

–Ongoing manufacturing improvements shows enhanced efficacy of ARU-1801 for the treatment of sickle cell disease–

New York, NY, Nov. 4, 2020 –Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, announced that an abstract demonstrating clinical benefit of the company’s lead product candidate ARU-1801 has been published online and will be the subject of an oral presentation at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition. The meeting will take place virtually from December 5 to 8. Michael S. Grimley, M.D., Professor of Clinical Pediatrics, Medical Director of the Division of Bone Marrow Transplantation and Immune Deficiency at the Cincinnati Children’s Hospital Medical Center, will present the data at 2:15 PM ET on December 7, 2020. Today the abstract was published online and will be published in the upcoming supplemental issue of Blood.

Data presented at ASH is from the MOMENTUM study, an open label Phase 1/2 clinical trial examining ARU-1801 as a one-time potentially curative gene therapy for individuals with sickle cell disease (SCD). The MOMENTUM study, which continues to enroll patients, examines ARU-1801, an autologous cell therapy leveraging a modified gamma globin lentivirus vector, in individuals with severe SCD. Unlike investigational gene therapies that require fully myeloablative conditioning, ARU-1801 is given with reduced intensity conditioning (RIC), which is a lower dose chemotherapy. 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. The data to be presented at ASH highlights participants dosed with product manufactured with both the original academic manufacturing process and the enhanced Process II.

“The clinical results thus far demonstrate that ARU-1801 holds significant promise for achieving durable responses with a reduced intensity conditioning approach to gene therapy,” said Dr. Grimley, a principal investigator in the MOMENTUM study. “Given the impact chemotherapy toxicity has on physician and patient decision making around treatment options, ARU-1801 has the potential to be a unique option for SCD patients seeking gene therapy.”

Abstract and Oral Presentation Information

Title: Early Results from a Phase 1/2 Study of ARU-1801 Gene Therapy for Sickle Cell Disease (SCD): Manufacturing Process Enhancements Improve Efficacy of a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant
Publication Number: 680
Session Name: 114. Hemoglobinopathies, Excluding Thalassemia—Clinical: Novel Treatments for Sickle Cell Disease
Date: Monday, December 7, 2020
Session Time: 1:30 PM – 3:00 PM
Presentation Time: 2:15 PM

About ARU-1801
ARU-1801 is a one-time potentially curative investigational gene therapy for individuals living with sickle cell disease. This product candidate was designed to address the limitations of current treatment options including chemotherapy toxicity, donor availability, and chronic administration, and replace it with a curative therapy. ARU-1801 incorporates a patented modified gamma-globin into autologous stem cells, with the aim of restoring normal red blood cell function through increased levels of fetal hemoglobin. The high potency of the modified gamma globin enables ARU-1801 engraftment with only reduced intensity conditioning (RIC). Preliminary clinical data from the MOMENTUM study, an ongoing Phase 1/2 trial in patients with sickle cell disease, demonstrate continuing durable reductions in disease burden.

The MOMENTUM Study
Aruvant is currently conducting the MOMENTUM study, which is evaluating ARU-1801, a one-time only potentially curative gene therapy for patients with SCD. This Phase 1/2 study currently is enrolling individuals with SCD, and information may be found at www.momentumtrials.com.

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 is an investigational lentiviral gene therapy currently in a clinical trial, the MOMENTUM study, as a potential one-time curative treatment for SCD. Preliminary clinical data from the ongoing Phase 1/2 study demonstrated engraftment of ARU-1801 and amelioration of SCD is possible with one dose of low 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.

Aruvant Contact
Blair Clark-Schoeb
blair.clark-schoeb@aruvant.com

Hanover, MD, and New York, NY, October 12, 2020 – The Sickle Cell Disease Association of America (SCDAA) and Aruvant Sciences are proud to announce a new partnership to create educational programs to increase awareness of gene therapy as a potential curative treatment option for sickle cell disease patients. This collaboration will help SCDAA continue to deliver on its mission, while assisting Aruvant in learning more about the needs of sickle cell disease (SCD) patients. Under the agreement, Aruvant will collaborate with SCDAA to host local and national educational events and develop materials for a public-awareness campaign.

“In partnership with SCDAA, we are working to educate patients about gene therapy, while gaining critical insights from the patient community for our ARU-1801 SCD development program,” said Will Chou, M.D., chief executive officer (CEO) of Aruvant. “Now is a perfect time to work with SCDAA to educate the community about gene therapy since we have an open and enrolling phase 1/2 clinical trial for our potentially curative experimental gene therapy, ARU-1801.”

Sickle cell disease affects 100,000 individuals in the United States, disproportionately affecting African Americans with one in 500 African Americans suffering from the disease. This inherited disease affects the production of hemoglobin, a protein in red blood cells that carries oxygen throughout the body. The disease occurs when people inherit a mutation from each of their parents which causes people with SCD to not have normal, healthy adult hemoglobin in their red blood cells and instead have an abnormal hemoglobin called sickle hemoglobin. SCD can cause frequent episodes of severe pain, weakness and other serious complications. Fetal hemoglobin is an “anti-sickling” hemoglobin that is present before birth in the red blood cells. After birth, the gene that makes fetal hemoglobin turns off, which mostly stops the production of fetal hemoglobin. More fetal hemoglobin in the blood can mean fewer episodes of sickling and pain.

“In partnership with Aruvant, we can provide the critical education needed for our community to understand gene therapy and how these promising new treatments work to treat and maybe cure this genetic disease that impacts so many in our community,” said Beverley Francis-Gibson, SCDAA president and CEO. “Partnering with companies like Aruvant is critical to help us support the research that could change the lives of many sickle cell disease patients.”

Aruvant and SCDAA’s educational events will review gene therapy and ongoing research, including discussion around Aruvant’s MOMENTUM study. This clinical trial is examining a one-time investigational treatment, ARU-1801, to increase levels of fetal hemoglobin in patients with severe sickle cell disease, with the hope of fewer episodes of sickling and pain. Aruvant provided funding for SCDAA’s 48th Annual National Convention 2020 which begins tomorrow, October 13, and will continue through October 17. To register, please visit https://bit.ly/SCDAA2020Convention.

The MOMENTUM Study

Aruvant is currently conducting the MOMENTUM study, which is evaluating ARU-1801, a one-time only potentially curative gene therapy for patients with SCD. The MOMENTUM study is currently enrolling, and more information may be found at www.momentumtrials.com.


About Sickle Cell Disease Association of America

Sickle Cell Disease Association of America advocates for people affected by sickle cell conditions and empowers community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure. The association and more than 50 member organizations support sickle cell research, public and professional health education and patient and community services. Visit www.sicklecelldisease.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, with an initial focus on helping patients suffering from sickle cell disease. The company’s lead candidate, ARU-1801, is an investigational lentiviral gene therapy for sickle cell disease. ARU-1801 incorporates a patented modified gamma-globin into autologous stem cells, with the aim of restoring normal red blood cell function through increased levels of fetal hemoglobin. The high potency of the modified gamma globin enables ARU-1801 engraftment with only reduced intensity conditioning (RIC). Preliminary clinical data from an ongoing Phase 1/2 study in patients with sickle cell disease demonstrated continuing durable reductions in disease burden. For more information on the clinical study, please visit www.momentumtrials.com and for more on the company, please visit www.aruvant.com.

Aruvant Contact

Blair Clark-Schoeb
Blair.clark-schoeb@aruvant.com.

SCDAA Contact

Andrew Aldrich
andrew@bonnieheneson.com

NEW YORK and BASEL, Switzerland, Sept. 29, 2020 — Aruvant, a clinical-stage biopharmaceutical company focused on developing and commercializing transformative gene therapies for the treatment of rare diseases, today announced it will present at multiple conferences in October. Will Chou, M.D., Aruvant chief executive officer, will present and do one-on-one meetings at the following conferences:

  • Chardan 4th Annual Genetic Medicines Conference. Presentation will take place on October 6 at 11:00 a.m. ET. Please visit www.chardan.com/ for more information.
  • 2020 Cell & Gene Meeting on the Mesa. Virtual conference will take place October 12-16 with the Aruvant presentation available on demand. Visit www.meetingonthemesa.com for more information.
  • BIO Investor Forum Digital. The virtual conference will take place October 13-15 with the Aruvant presentation available on demand. Visit www.bio.org/events/bio-investor-forum-digital for more information.

About Aruvant
Aruvant Sciences, part of the Roivant family of companies, is a clinical-stage biopharmaceutical company focused on developing and commercializing transformative gene therapies for rare diseases, with a near-term focus on helping patients suffering from sickle cell disease and β-thalassemia. The company’s lead candidate, ARU-1801, is an investigational lentiviral gene therapy for sickle cell disease and transfusion-dependent β-thalassemia. ARU-1801 incorporates a patented modified gamma-globin into autologous stem cells, with the aim of restoring normal red blood cell function through increased levels of fetal hemoglobin. The high potency of the modified gamma globin enables ARU-1801 engraftment with only reduced intensity conditioning (RIC). Preliminary clinical data from an ongoing Phase 1/2 study in patients with sickle cell disease demonstrated continuing durable reductions in disease burden. For more information on the trial, please visit www.momentumtrials.com and for information on the company, please visit www.aruvant.com.

About Roivant
Roivant aims to improve health by rapidly delivering innovative medicines and technologies to patients. Roivant does 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:
E. Blair Clark-Schoeb
SVP, Communications
blair.clark-schoeb@aruvant.com

Posted on
  • Dr. Palaniappan joins Aruvant from Sarepta where he oversaw technical operations and was responsible for the development and registration of a portfolio of more than 30 compounds including gene therapies for rare diseases
  • Dr. Palaniappan will lead the manufacturing process optimization for product candidate ARU-1801, a potentially curative investigational gene therapy for patients with sickle cell disease

NEW YORK and BASEL, Switzerland, June 1, 2020 — Aruvant Sciences Inc. (Aruvant), a clinical-stage biopharmaceutical company focused on developing and commercializing transformative therapies for the treatment of severe blood disorders, today announced the appointment of V. “Palani” Palaniappan, Ph.D. as chief technology officer (CTO) effective immediately. In this role, Dr. Palaniappan will be charged with growing Aruvant’s technical and manufacturing capabilities for current and future programs. With 25 years of research and drug development experience, including extensive work in chemistry, manufacturing and controls, a critical part of drug development, Dr. Palaniappan will oversee the manufacturing of ARU-1801, a one-time, potentially curative investigational gene therapy for patients with sickle cell disease administered with only reduced intensity conditioning.

“Dr. Palaniappan joins the team at an important time as we transition from academic to commercial manufacturing for ARU-1801, our potentially curative product candidate for individuals with sickle cell disease,” said Will Chou, M.D., chief executive officer of Aruvant. “While we have already seen strong clinical results from ARU-1801 manufactured with our first-generation process, Dr. Palaniappan has the depth of experience to successfully lead us to an optimized commercial-ready process. His proven track record and extensive experience with vector-based products, recombinant biologics and other novel modalities will be valuable as we refine our approach with ARU-1801.”

Dr. Palaniappan joined Aruvant from Sarepta where he was senior vice president, head of global technical operations and head of the Andover and Burlington sites. He was responsible for all stages of development from research through commercial for a portfolio of over 30 compounds using three modalities: antisense RNAs, gene therapy and gene editing. Prior to Sarepta, Dr. Palaniappan worked at Takeda for over a decade, where he led teams based in the United States and Japan focused on CMC development and manufacturing of novel modality products, including recombinant biologics and cell-based and live microbe products. Prior to joining Takeda, he worked in various development and product manufacturing roles of increasing responsibility at Millennium Pharmaceuticals (which was acquired by Takeda), Biogen and Nexstar Pharmaceuticals (which was acquired by Gilead). Dr. Palaniappan received his bachelor’s and master’s degree from Annamalai University of India. He earned his Ph.D. in Chemistry from the Indian Institute of Technology, Kanpur.

“Given the importance of a sound CMC strategy and robust manufacturing, I am honored to join the Aruvant team to help guide ARU-1801 through the next stages of development and registration,” said Dr. Palaniappan. “To accelerate bringing this potentially curative product to patients, we will focus on building capabilities to support a robust, sustainable manufacturing process and a sound regulatory strategy for ARU-1801.”

About Aruvant
Aruvant Sciences, part of the Roivant family of companies, is a clinical-stage biopharmaceutical company focused on developing and commercializing transformative therapies for the treatment of severe blood disorders, with an emphasis on helping patients suffering from sickle cell disease and β-thalassemia. The company’s lead candidate, ARU-1801, is an investigational lentiviral gene therapy for sickle cell disease and transfusion-dependent β-thalassemia. ARU-1801 incorporates a patented modified gamma-globin into autologous stem cells, with the aim of restoring normal red blood cell function through increased levels of fetal hemoglobin. The high potency of the modified gamma globin enables ARU-1801 engraftment with only Reduced Intensity Conditioning (RIC). Preliminary clinical data from an ongoing Phase 1/2 study in patients with sickle cell disease demonstrated continuing durable reductions in disease burden and cessation of daily opioid use for over 18 months of follow-up. For more information, please visit www.aruvant.com.

About Roivant
Roivant aims to improve health by rapidly delivering innovative medicines and technologies to patients. Roivant does 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:
E. Blair Clark-Schoeb
blair.clark-schoeb@aruvant.com

Posted on
  • Dr. McIntosh joins to lead clinical development at Aruvant, including the program for ARU-1801, a one-time potentially curative gene therapy for patients with sickle cell disease administered with reduced intensity conditioning
  • Most recently as head of clinical development at PTC Therapeutics, he oversaw a broad portfolio of chemical and gene therapies across hematology, oncology and genetic rare diseases

NEW YORK and BASEL, Switzerland, May 11, 2020 — Aruvant, a clinical-stage biopharmaceutical company focused on developing and commercializing transformative therapies for the treatment of severe blood disorders, today announced the appointment of Joseph McIntosh, M.D. as chief medical officer effective immediately. With 16 years of drug development experience, including more than a decade in rare disease, Dr. McIntosh will oversee clinical development at Aruvant. His initial focus will be the clinical program for ARU-1801, a one-time, potentially curative gene therapy for patients with sickle cell disease and β-thalassemia.

“Dr. McIntosh’s extensive experience in rare disease and gene therapy will be invaluable as we advance the clinical development of our potentially curative gene therapy ARU-1801,” said Will Chou, M.D., Chief Executive Officer of Aruvant. “Given the durable efficacy we have seen in the first sickle cell disease patients treated with ARU-1801, we are excited to accelerate this program forward under Dr. McIntosh’s leadership.”

Dr. McIntosh joins Aruvant from PTC Therapeutics, where he was senior vice president, head of clinical development responsible for a portfolio of assets, including two gene therapies, across hematology, oncology and genetic rare diseases. Prior to this, Dr. McIntosh was the vice president of development at PTC where he facilitated the approval of Translarna, PTC’s Duchenne muscular dystrophy asset. Prior to joining PTC, he worked in various development roles of increasing responsibility at NPS Pharmaceuticals, Eisai and Pfizer. Dr. McIntosh received his bachelor’s and medical degrees from University of Witwatersrand in South Africa.

“I am thrilled for this exciting opportunity to work on ARU-1801 and to join such a talented team at Aruvant,” said Dr. McIntosh. “We have the important goal of providing patients with sickle cell disease a potential cure with a lower conditioning chemotherapy burden.”

About Aruvant

Aruvant Sciences, part of the Roivant family of companies, is a clinical-stage biopharmaceutical company focused on developing and commercializing transformative therapies for the treatment of severe blood disorders, with an emphasis on helping patients suffering from sickle cell disease and β-thalassemia. The company’s lead candidate, ARU-1801, is a modified fetal hemoglobin gene therapy for sickle cell disease and related hemoglobinopathies. For more information, please visit www.aruvant.com.

About Roivant

Roivant aims to improve health by rapidly delivering innovative medicines and technologies to patients. Roivant does 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:
E. Blair Clark-Schoeb
blair.clark-schoeb@aruvant.com

  • ARU-1801 is being developed as a potential one-time treatment for patients suffering from sickle cell disease and β-thalassemia
  • ARU-1801 has been designed to deliver a highly potent gene payload with enhanced anti-sickling properties, allowing for a Reduced Intensity Conditioning (RIC) regimen

NEW YORK and BASEL, Switzerland, Jan. 22, 2020 — Aruvant, a clinical-stage biopharmaceutical company focused on developing and commercializing transformative therapies for the treatment of severe blood disorders, today announced that the U.S. Food and Drug Administration has granted Orphan Drug designation to ARU-1801, Aruvant’s investigational therapy for the treatment of sickle cell disease.

The FDA grants Orphan Drug designation to novel therapies that treat diseases impacting fewer than 200,000 individuals in the United States. The benefits of orphan designation include a seven-year period of marketing exclusivity in the U.S. upon approval if received, waiver of certain FDA user-fees, and tax credits for qualified clinical trials.

“We are excited to build on the momentum afforded by both today’s announcement of Orphan Drug status as well as our recent announcement of Rare Pediatric Disease status for ARU-1801,” said Will Chou, M.D., Chief Executive Officer of Aruvant. “For patients suffering from sickle cell disease, we believe the ultimate promise of gene therapy is a one-time cure without the side effect profile of high intensity myeloablative conditioning. We are committed to providing patients with that option and look forward to presenting more data on our Reduced Intensity Conditioning (RIC) approach as patients continue to be treated in our ongoing Phase 1/2 study.”

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

Aruvant Sciences, part of the Roivant family of companies, is a clinical-stage biopharmaceutical company focused on developing and commercializing transformative therapies for the treatment of severe blood disorders, with an emphasis on helping patients suffering from sickle cell disease and β-thalassemia. The company’s lead candidate, ARU-1801, is a modified fetal hemoglobin gene therapy for sickle cell disease and related hemoglobinopathies. For more information, please visit www.aruvant.com.

About Roivant

Roivant aims to improve health by rapidly delivering innovative medicines and technologies to patients. Roivant does 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:
Andrew Bogorad
andrew.bogorad@roivant.com

Posted on
  • ARU-1801 is being developed as a potential one-time treatment for patients suffering from sickle cell disease and β-thalassemia
  • ARU-1801 has been designed to deliver a highly potent gene payload with enhanced anti-sickling properties, allowing for a Reduced Intensity Conditioning (RIC) regimen

NEW YORK and BASEL, Switzerland, Jan. 8, 2020 — Aruvant, a clinical-stage biopharmaceutical company focused on developing and commercializing transformative therapies for the treatment of severe blood disorders, today announced that the U.S. Food and Drug Administration has granted Rare Pediatric Disease designation to ARU-1801, Aruvant’s investigational therapy for the treatment of sickle cell disease.

The FDA defines a rare pediatric disease as a serious or life-threatening disease that impacts fewer than 200,000 individuals in the United States and whose serious or life-threatening manifestations primarily affect individuals 18 years or younger. With this designation, Aruvant will be eligible to receive a priority review voucher upon approval by the FDA of a Biologics License Application for ARU-1801.

“The FDA’s Rare Pediatric Disease designation for ARU-1801 highlights the large and unmet need currently experienced by patients suffering from sickle cell disease,” said Will Chou, M.D., Chief Executive Officer of Aruvant. “Other investigational gene therapies for sickle cell disease require the use of high intensity myeloablative conditioning regimens, which are associated with lengthy hospital stays and a host of possibly serious complications. Our Reduced Intensity Conditioning (RIC) approach aims to provide patients a cure with an improved risk-benefit profile, including a lower risk of infertility and fewer days in the hospital.”

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

Aruvant Sciences, part of the Roivant family of companies, is a clinical-stage biopharmaceutical company focused on developing and commercializing transformative therapies for the treatment of severe blood disorders, with an emphasis on helping patients suffering from sickle cell disease and β-thalassemia. The company’s lead candidate, ARU-1801, is a modified fetal hemoglobin gene therapy for sickle cell disease and related hemoglobinopathies. For more information, please visit www.aruvant.com.

About Roivant Sciences

Roivant aims to improve health by rapidly delivering innovative medicines and technologies to patients. Roivant does 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:

Andrew Bogorad

andrew.bogorad@roivant.com