Hepatitis B DNA vaccine(Genencor International, Inc.)

Keynote by Admiral Rachel Levine, M.D., Assistant Secretary for Health, U.S. Department of Health and Human ServicesSOUTH SAN FRANCISCO, Calif. and BALTIMORE, Sept. 08, 2022 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) and the Sickle Cell Disease Association of America, Inc. (SCDAA) will host the 11th Annual Sickle Cell Disease (SCD) Therapeutics Conference on Wednesday, September 14th. The virtual conference, which takes place during National Sickle Cell Awareness Month, is open to the public and will be live streamed on the SCD Therapeutics Conference website and the Facebook page of Sickle Cell Warriors, an SCD community organization. Admiral Rachel Levine, M.D., assistant secretary for health, U.S. Department of Health and Human Services (HHS), will provide remarks at the conference. In her role, Admiral Levine fights every day to improve the health and well-being of all Americans. She's working to help the nation overcome the COVID-19 pandemic and build a stronger foundation for a healthier future. “Each year, the SCD Therapeutics Conference highlights the growing interest and progress that is being made to improve the treatment of sickle cell disease, though more is needed to achieve equitable health and personal outcomes for this community,” said Kim Smith-Whitley, M.D., executive vice president and head of research and development at GBT. “We are honored to again co-host the conference with the SCDAA and livestream the event with the Sickle Cell Warriors in order to advance the dialogue around addressing the multitude of opportunities we have to improve the outlook for people living with sickle cell disease.” This year’s conference will touch on a number of important topics, including the impact of SCD on patients and caregivers and exploring the caregiver experience throughout the continuum of care, as well a discussion of the national SCD legislative agenda. “We are excited for the opportunity to bring together leading physicians, warriors, advocates, and policymakers to discuss the next frontiers in SCD and how we can continue to drive meaningful change in the community,” said Regina Hartfield, president and chief executive officer of SCDAA. “This year’s conference will allow today’s leaders in SCD the opportunity to discuss current unmet needs and recent trends in therapeutic development, all with the goal of building access to comprehensive care to enable a better future for people living with sickle cell.” In addition to Admiral Levine, the 11th Annual SCD Therapeutics Conference will feature the following speakers and panelists: The Honorable Chris Van Hollen, U.S. Senator, MarylandLewis Hsu, M.D., Ph.D., Chief Medical Officer, Director of the Sickle Cell Center and Professor of Pediatrics; Pediatric Hematologist, University of Illinois at ChicagoAlan Anderson, M.D., Assistant Professor of Clinical Pediatrics, University of South Carolina School of Medicine, Greenville, South Carolina; Medical Director, Comprehensive Sickle Cell Disease Program, Prisma HealthKofi Anie, MBE, Ph.D., AFBPsS, Psychology Service Lead, London North West University Healthcare NHS TrustRichard Drachtman, M.D., Clinical Section Chief, Pediatric Hematology/Oncology Division; Medical Director, Pediatric Hematology/Oncology Clinical Program; Professor of Pediatrics Rutgers Cancer Institute of New Jersey, Rutgers Robert Wood Johnson Medical SchoolMarsha Treadwell, Ph.D., Professor of Pediatrics, Division of Hematology, and Jordan Fund Endowed Chair at the University of California San Francisco (UCSF); Co-Chair of the Diversity, Equity and Inclusion Council for the UCSF Benioff Children’s HospitalsAyana L. Johnson, SCDAA National Teen AmbassadorHermionne Johnson, R.N., M.S.N., SCD CaregiverRonisha Edwards-Elliot, SCD Patient AdvocateRebecca Robbins, SCD CaregiverJennifer George, B.A. & Stephanie George, B.Sc., M.Sc., SCD Patient Advocates & CaregiversBiree Andemariam, M.D., Professor of Medicine, American Red Cross Endowed Chair in Transfusion Medicine, Division of Hematology/Oncology; Director, New England Sickle Cell Institute, University of Connecticut Health; Vice Chair of the Board, SCDAAChancellor Donald, M.D., Chair, American Society of Hematology (ASH) Committee on Practice; Assistant Professor, Tulane University School of Medicine Multiple companies that have and are developing treatments for patients with SCD will present at the conference, including Agios Pharmaceuticals, CSL Behring, Forma Therapeutics, GBT, Graphite Bio, Pfizer, and Vertex Pharmaceuticals/CRISPR Therapeutics. For more information about the conference or to register to attend, visit http://scdconference.com/. Follow the conversation on Twitter using the hashtag #SCDTC. About Sickle Cell DiseaseIt is estimated that more than 100,000 people in the United States,1 52,000 people in Europe,2 up to 100,000 people in Brazil,3 and millions of people throughout the world have sickle cell disease (SCD).1 SCD occurs particularly among those whose ancestors are from sub-Saharan Africa, though it also occurs in people of Hispanic, South Asian, Southern European and Middle Eastern ancestry.1 SCD is a lifelong inherited blood disorder that impacts hemoglobin, a protein carried by red blood cells that delivers oxygen to tissues and organs throughout the body.4 Due to a genetic mutation, individuals with SCD form abnormal hemoglobin known as sickle hemoglobin. When sickle hemoglobin becomes deoxygenated, it polymerizes to form rods, which deforms the red blood cells into sickled – crescent-shaped, rigid – cells.4-6 The recurrent sickling process causes destruction of the red blood cells, hemolysis and anemia (low hemoglobin due to red blood cell destruction), which drives vascular inflammation contributing to blockages in capillaries and small blood vessels (vaso-occlusion) that impede the flow of blood and oxygen delivery throughout the body. Episodes of painful vascular occlusions are commonly referred to as vaso-occlusive crises (VOCs). The diminished oxygen delivery to tissues and organs can lead to life-threatening complications, including stroke and irreversible organ damage.5-9 Complications of SCD begin in early childhood and can include neurocognitive impairment, acute chest syndrome, and silent and overt stroke, among other serious issues.10 Early intervention and treatment of SCD have shown potential to modify the course of this disease, reduce symptoms and events, prevent long-term organ damage, and extend life expectancy.5 About Global Blood TherapeuticsGlobal Blood Therapeutics, Inc. (GBT) is a biopharmaceutical company dedicated to the discovery, development and delivery of life-changing treatments that provide hope to underserved patient communities, starting with sickle cell disease (SCD). Founded in 2011, GBT is delivering on its goal to transform the treatment and care of SCD, a lifelong, devastating inherited blood disorder. The company has introduced Oxbryta® (voxelotor), the first FDA-approved medicine that directly inhibits sickle hemoglobin (HbS) polymerization, the root cause of red blood cell sickling in SCD. GBT is also advancing its pipeline program in SCD with inclacumab, a P-selectin inhibitor in Phase 3 development to address pain crises associated with the disease, and GBT021601 (GBT601), the company’s next generation HbS polymerization inhibitor. In addition, GBT’s drug discovery teams are working on new targets to develop the next generation of treatments for SCD. To learn more, please visit www.gbt.com and follow the company on Twitter @GBT_news. About SCDAASCDAA’s mission is: To advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure. Visit http://www.sicklecelldisease.org/. References Centers for Disease Control and Prevention. Sickle Cell Disease Data and Statistics (SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html. Accessed June 7, 2022.European Medicines Agency. https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125. Accessed June 12, 2022.Ministério da Saúde (Brasil), Protocolo Clínico e Diretrizes Terapêuticas da Doença Falciforme, Feb. 19, 2018National Heart, Lung, and Blood Institute. Sickle Cell Disease. https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease. Accessed February 23, 2022.Kato GJ, et al. Nat Rev Dis Primers. 2018;4:18010.Rees DC, et al. Lancet. 2010;376(9757):2018-2031.Kato GJ, et al. J Clin Invest. 2017;127(3):750-760. Caboot JB, et al. Paediatr Respir Rev. 2014;15(1):17-23.Nader E, et al. Front Immunol. 2020 Mar 13;11:454.Kanter J, et al. Blood Rev. 2013 Nov;27(6):279-87. Contact:Steven Immergut (media)+1 650-410-3258simmergut@gbt.com Courtney Roberts (investors)+1 650-351-7881croberts@gbt.com SCDAA Contact Information: Emma Dayeday@sicklecelldisease.org Kyri Jacobskjacobs@sicklecelldisease.org

Pfizer Inc. (NYSE: PFE) and Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced the companies have entered into a definitive agreement under which Pfizer will acquire GBT, a biopharmaceutical company dedicated to the discovery, development and delivery of life-changing treatments that provide hope to underserved patient communities, starting with sickle cell disease (SCD). The acquisition complements and further enhances Pfizers more than 30-year heritage in rare hematology and reinforces the companys commitment to SCD by bringing expertise and a leading portfolio and pipeline with the potential to address the full spectrum of critical needs in this underserved community. Pfizer intends to continue to build on the companies shared commitment to and engagement with the SCD community. Under the terms of the transaction, Pfizer will acquire all the outstanding shares of GBT for $68.50 per share in cash, for a total enterprise value of approximately $5.4 billion, including debt and net of cash acquired. The Boards of Directors of both companies have unanimously approved the transaction. SCD is a lifelong, devastating inherited blood disorder impacting millions of people worldwide, predominantly in populations of African, Middle Eastern and South Asian descent. GBT developed Oxbryta (voxelotor) tablets, a first-in-class medicine that directly targets the root cause of SCD. Oxbryta was approved in the United States in November 2019 and is also approved in the European Union, United Arab Emirates, Oman and Great Britain. Net sales for Oxbryta were approximately $195 million in 2021. Leveraging its global platform, Pfizer plans to accelerate distribution of GBTs innovative treatment to parts of the world most impacted by SCD. In addition, GBT is developing GBT021601 (GBT601), an oral, once-daily, next-generation sickle hemoglobin (HbS) polymerization inhibitor in the Phase 2 portion of a Phase 2/3 clinical study. GBT601 has the potential to be a best-in-class agent targeting improvement in both hemolysis and frequency of vaso-occlusive crisis (VOC). GBTs promising pipeline also includes inclacumab, a fully human monoclonal antibody targeting P-selectin which is being evaluated in two Phase 3 clinical trials as a potential quarterly treatment to reduce the frequency of VOCs and to reduce hospital readmission rates due to VOCs. Both GBT601 and inclacumab have received Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA). If approved, GBTs pipeline and Oxbryta have the potential for an SCD franchise that could achieve combined worldwide peak sales of more than $3 billion. "Sickle cell disease is the most common inherited blood disorder, and it disproportionately affects people of African descent. We are excited to welcome GBT colleagues into Pfizer and to work together to transform the lives of patients, as we have long sought to address the needs of this underserved community," said Albert Bourla, Chairman and Chief Executive Officer, Pfizer. "The deep market knowledge and scientific and clinical capabilities we have built over three decades in rare hematology will enable us to accelerate innovation for the sickle cell disease community and bring these treatments to patients as quickly as possible." "Today is an exciting milestone that accelerates GBT's mission to discover, develop and deliver life-changing treatments that provide hope to underserved patient communities," said Ted W. Love, M.D., President and Chief Executive Officer, GBT. "Pfizer will broaden and amplify our impact for patients and further propel much-needed innovation and resources for the care of people with sickle cell disease and other rare diseases, including populations in limited-resource countries. We look forward to working together with Pfizer to serve our communities and advance our shared goal of improving health equity and expanding access to life-changing treatments to create a healthier future for all." Pfizer expects to finance the transaction with existing cash on hand. The proposed transaction is subject to customary closing conditions, including receipt of regulatory approvals and approval by GBTs stockholders. Due to the proposed transaction, GBT will not hold its previously scheduled conference call to discuss its second quarter 2022 financial results. The company will file its quarterly report on Form 10-Q for the quarter ending June 30, 2022 with the U.S. Securities and Exchange Commission announcing those results on August 8, 2022. Pfizer's financial advisors for the transaction are Morgan Stanley & Co. LLC and Goldman Sachs & Co. LLC, with Wachtell, Lipton, Rosen & Katz acting as its legal advisor. GBTs financial advisors for the transaction are J.P. Morgan Securities LLC and Centerview Partners LLC, with Cravath, Swaine & Moore LLP and Goodwin Procter LLP acting as legal advisors. About Sickle Cell Disease Sickle cell disease (SCD) is a lifelong, debilitating inherited blood disorder characterized by hemolytic anemia, acute pain crises and progressive end organ damage. Acute pain crisis, or vaso-occlusive crisis (VOC), occurs when sickled red blood cells irritate the lining of blood vessels and cause an inflammatory response leading to vascular occlusion, tissue ischemia and pain. Complications of SCD begin in early childhood and are associated with shortened life expectancy. Early intervention and treatment of SCD have shown potential to modify the course of this disease, reduce symptoms and events, prevent long-term organ damage, and extend life expectancy. Historically, there has been a high unmet need for therapies that address the root cause of SCD and its acute and chronic complications. While rare in developed markets, there are 4.5 million people living with SCD globally and more than 45 million people living with the sickle cell trait. SCD occurs particularly among those whose ancestors are from sub-Saharan Africa, though it also occurs in people of Hispanic, South Asian, Southern European and Middle Eastern ancestry. About Oxbryta (voxelotor) Oxbryta (voxelotor) is an oral, once-daily therapy for patients with sickle cell disease (SCD). Oxbryta works by increasing hemoglobins affinity for oxygen. Since oxygenated sickle hemoglobin does not polymerize, Oxbryta inhibits sickle hemoglobin polymerization and the resultant sickling and destruction of red blood cells leading to hemolysis and hemolytic anemia, which are primary pathologies faced by every single person living with SCD. Through addressing hemolytic anemia and improving oxygen delivery throughout the body, GBT believes that Oxbryta has the potential to modify the course of SCD. In November 2019, the FDA granted accelerated approval for Oxbryta tablets for the treatment of SCD in adults and children 12 years of age and older, and in December 2021, the FDA expanded the approved use of Oxbryta for the treatment of SCD in patients 4 years of age and older in the United States. As a condition of accelerated approval for patients ages 4 and older in the United States, GBT will continue to study Oxbryta in the HOPE-KIDS 2 Study, a post-approval confirmatory study using transcranial Doppler (TCD) flow velocity to assess the ability of the therapy to decrease stroke risk in children 2 to 14 years of age. In recognition of the critical need for new SCD treatments, the FDA granted Oxbryta Breakthrough Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease designations for the treatment of patients with SCD. Additionally, Oxbryta received the prestigious 2021 Prix Galien USA award for Best Biotechnology Product from The Galien Foundation. Oxbryta has been granted Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), Oxbryta was designated by the European Commission (EC) as an orphan medicinal product for the treatment of patients with SCD, and Oxbryta was granted Promising Innovative Medicine (PIM) designation in the United Kingdom from the Medicines and Healthcare products Regulatory Agency (MHRA). In February 2022, the European Commission (EC) granted Marketing Authorization for Oxbryta for the treatment of hemolytic anemia due to SCD in adult and pediatric patients 12 years of age and older as monotherapy or in combination with hydroxycarbamide (hydroxyurea). The MHRA has granted Oxbryta marketing authorization in Great Britain for the treatment of hemolytic anemia due to SCD in adult and pediatric patients 12 years of age and older. In addition, the Ministry of Health and Prevention (MOHAP) in the United Arab Emirates (UAE) has granted marketing authorization for Oxbryta for the treatment of SCD in adults and children 12 years of age and older. About Pfizer: Breakthroughs That Change Patients' Lives At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 170 years, we have worked to make a difference for all who rely on us. About Global Blood Therapeutics Global Blood Therapeutics, Inc. (GBT) is a biopharmaceutical company dedicated to the discovery, development and delivery of life-changing treatments that provide hope to underserved patient communities, starting with sickle cell disease (SCD). Founded in 2011, GBT is delivering on its goal to transform the treatment and care of SCD, a lifelong, devastating inherited blood disorder. The company has introduced Oxbryta (voxelotor), the first FDA-approved medicine that directly inhibits sickle hemoglobin (HbS) polymerization, the root cause of red blood cell sickling in SCD. GBT is also advancing its pipeline program in SCD with inclacumab, a P-selectin inhibitor in Phase 3 development to address pain crises associated with the disease, and GBT021601 (GBT601), the companys next generation HbS polymerization inhibitor. In addition, GBTs drug discovery teams are working on new targets to develop the next generation of treatments for SCD.