Alpha 1-Antitrypsin Deficiency

DALLAS--( BUSINESS WIRE )--AIC, a division of AIS Healthcare, now offers GLASSIA and ARALAST NP, manufactured by Takeda, for the treatment of adult patients with clinically evident emphysema due to severe hereditary deficiency of Alpha 1 -PI (alpha 1 -antitrypsin deficiency). The nationally accredited home infusion teams at AIC collaborate closely with physicians to provide specialized infusion services to patients in their home or at an infusion suite setting. The addition of these therapy options expands AIC’s ability to better serve patients with alpha 1 -antitrypsin deficiency. “We’re excited and proud to be able to add Takeda’s Alpha 1 therapies to our growing portfolio of infusion therapies,” said Jud Hall, President at AIC. “The addition of GLASSIA and ARALAST NP can help us provide patients with more options to meet individual treatment needs.” Approved by the U.S. Food and Drug Administration (FDA) in 2010 and 2002, respectively, GLASSIA and ARALAST NP are Alpha 1 -Proteinase Inhibitors (Human) (Alpha 1 -PI) indicated for chronic augmentation therapy in adults with clinically evident emphysema due to severe hereditary deficiency of Alpha 1 -PI. ARALAST NP increases antigenic and functional (anti-neutrophil elastase capacity, ANEC) serum levels and antigenic lung epithelial lining fluid levels of Alpha 1 -PI. The effect of augmentation therapy with GLASSIA, ARALAST NP, or any Alpha 1 -PI product on pulmonary exacerbations and on the progression of emphysema in Alpha 1 -PI deficiency has not been conclusively demonstrated in randomized, controlled clinical trials. Clinical data demonstrating the long-term effects of chronic augmentation and maintenance therapy of individuals with GLASSIA or ARALAST NP are not available. GLASSIA and ARALAST NP are not indicated as therapies for lung disease in patients in whom severe Alpha 1 -PI deficiency has not been established. Both GLASSIA and ARALAST NP are now available at all AIC locations (Valdosta, Georgia; Birmingham, Alabama; Panama City, Florida; Dallas, Texas). Like all AIC patients, those with alpha 1 -antitrypsin deficiency will receive a high level of care from AIC, including 24/7 access to clinical and support staff. Every patient is assigned a dedicated team of professionals, including clinical pharmacists, home infusion nurses, RN clinical review specialists and patient onboarding specialists to provide support throughout the treatment process. For providers, AIC is a full-service care delivery partner. We also work directly with patients, insurance companies, and provider offices to secure all needed insurance authorizations and approvals. AIC is dually accredited by URAC and the Accreditation Commission for Health Care (ACHC), further setting it apart from other infusion services providers. Physicians interested in AIC care services should contact their AIC representatives to get started or visit us at www.aiscaregroup.com/our-divisions/infusion-care/ About AIS Healthcare Advancing quality. Improving lives. As the leading provider of targeted drug delivery and infusion care solutions, AIS Healthcare is committed to doing more of what matters. From pharmacies that put patient safety first to comprehensive services that enhance the entire care experience, we go beyond the expected in everything we do. Learn more at aiscaregroup.com . About Takeda Takeda is focused on creating better health for people and a brighter future for the world. We aim to discover and deliver life-transforming treatments in our core therapeutic and business areas, including gastrointestinal and inflammation, rare diseases, plasma-derived therapies, oncology, neuroscience and vaccines. Together with our partners, we aim to improve the patient experience and advance a new frontier of treatment options through our dynamic and diverse pipeline. As a leading values-based, R&D-driven biopharmaceutical company headquartered in Japan, we are guided by our commitment to patients, our people and the planet. Our employees in approximately 80 countries and regions are driven by our purpose and are grounded in the values that have defined us for more than two centuries. For more information, visit www.takeda.com .

− Pre-Specified Interim Analysis Shows Positive Safety and Efficacy Results; Mezagitamab Safe and Well Tolerated − Takeda Intends to Initiate a Global Phase 3 Trial of Mezagitamab in Patients with Primary Immune Thrombocytopenia in Fiscal Year 2024 − Late-Stage Pipeline Continues to Gain Momentum With Five New Molecular Entities in Phase 3 Development in Fiscal Year 2024 OSAKA, Japan & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Takeda (TSE:4502/NYSE:TAK) today announced positive topline results from a Phase 2, randomized, double-blind, placebo-controlled study evaluating the safety, tolerability and efficacy of mezagitamab (TAK-079) in patients with persistent or chronic primary immune thrombocytopenia (ITP). Mezagitamab is a fully human immunoglobulin IgG1 monoclonal antibody (mAb) with high affinity for CD38 expressing cells (including plasmablasts, plasma cells, natural killer cells) resulting in their depletion. The TAK-079-1004 trial (NCT04278924) evaluated three different doses of subcutaneous mezagitamab vs placebo, given once weekly for eight weeks in patients with chronic (more than one year in duration) or persistent (3-12 months in duration) primary ITP. An interim analysis of the ongoing Phase 2 study demonstrated positive safety and efficacy results. Mezagitamab has been generally safe and well tolerated across all three cohorts. All mezagitamab doses tested demonstrated a higher platelet response rate than placebo. The increases in platelet count were dose-dependent with the greatest platelet response observed at the highest dose tested. Platelet response in mezagitamab treated patients occurred rapidly and was maintained post-therapy. Based on these positive results, and following consultation with global health authorities, Takeda plans to initiate a global Phase 3 trial of mezagitamab in ITP in fiscal year 2024. When mezagitamab advances to Phase 3 in ITP Takeda will have five new molecular entities in Phase 3 development in fiscal year 2024 including TAK-279 for the treatment of psoriasis and psoriatic arthritis, TAK-861 for the treatment of narcolepsy type 1, soticlestat for the treatment of Lennox-Gastaut syndrome and Dravet syndrome, and fazirsiran for the treatment of α1-antitrypsin associated liver disease. ITP is a rare, IgG mediated autoimmune disease caused, in part, by the development of autoantibodies to platelets (and/or megakaryocytes), which are blood cells responsible for preventing or stopping bleeding. ITP is characterized by the accelerated destruction of platelets (with or without impaired production), resulting in a decreased platelet count and an increased risk of bleeding, which can be debilitating (including fatigue and impaired quality of life), and in severe cases may be life-threatening. The precedent for approval of new drugs in this indication requires that platelet counts be maintained at 50,000/uL or more for a sustained period. Approximately 20 percent of patients with ITP do not achieve a platelet count above 50,000/uL following treatment with available first- and second-line therapies creating significant patient burden and unmet need for a disease modifying treatment that is also tolerable.1,2 “These Phase 2 results demonstrate mezagitamab’s compelling disease modifying mechanism of action, which has the potential to achieve disease remission for people with ITP. There remains considerable unmet need among ITP patients who may not respond or have inadequate response to prior treatment. In addition, some patients who do respond to available therapies struggle to manage side effects or relapse despite treatment,” said Chinwe Ukomadu, Head of the Gastrointestinal & Inflammation Therapeutic Area Unit at Takeda. “We look forward to initiating the Phase 3 trial and presenting these data at an upcoming scientific congress.” Therapy with mezagitamab is designed to deliver rapid and sustained improvement in platelet response and generally rapidly restores platelet counts to functional levels. Mezagitamab previously received Orphan Drug Designation for the treatment of ITP from the U.S. Food and Drug Administration and the program recently received Fast Track Designation. Results from the Phase 2 trial have no impact on the full year consolidated reported forecast for the fiscal year ending March 31, 2024 (Fiscal Year 2023). About Takeda Takeda is focused on creating better health for people and a brighter future for the world. We aim to discover and deliver life-transforming treatments in our core therapeutic and business areas, including gastrointestinal and inflammation, rare diseases, plasma-derived therapies, oncology, neuroscience, and vaccines. Together with our partners, we aim to improve the patient experience and advance a new frontier of treatment options through our dynamic and diverse pipeline. As a leading values-based, R&D-driven biopharmaceutical company headquartered in Japan, we are guided by our commitment to patients, our people and the planet. Our employees in approximately 80 countries and regions are driven by our purpose and are grounded in the values that have defined us for more than two centuries. For more information, visit . 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Takeda does not undertake to update any of the forward-looking statements contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule. Past performance is not an indicator of future results and the results or statements of Takeda in this press release may not be indicative of, and are not an estimate, forecast, guarantee or projection of Takeda’s future results. Medical Information This press release contains information about products that may not be available in all countries, or may be available under different trademarks, for different indications, in different dosages, or in different strengths. Nothing contained herein should be considered a solicitation, promotion or advertisement for any prescription drugs including the ones under development. References Provan D, Donald A, et al. Blood Advances. 2019;26;3(22):3780-3817. Rodeghiero F. International Journal of Hematology. 2023;117:316–33.

SOUTH SAN FRANCISCO, Calif., March 13, 2024 (GLOBE NEWSWIRE) -- Epic Bio, a leading epigenetic editing company that plans to dose its first patients later this year, today announced that Amber Salzman, Ph.D., chief executive officer, will present at the Charles River Cell & Gene Therapy Summit at 2:00 p.m. PT, Tuesday, March 19, 2024, in South San Francisco, Calif. Institutional investors can learn more about this event and register to attend here. About Epic Bio Epic Bio is a leading epigenetic editing company, leveraging the power of CRISPR without cutting DNA. The company’s proprietary Gene Expression Modulation System (GEMS) includes the smallest Cas protein known to work in human cells, enabling in vivo or ex vivo delivery via a single viral vector. Epic plans to begin dosing patients in a clinical trial of its lead program — EPI-321 for the treatment of facioscapulohumeral muscular dystrophy (FSHD) — in 2024; additional programs seek to address alpha-1 antitrypsin deficiency (A1AD), heterozygous familial hypercholesterolemia (HeFH), and other indications. Visit for more information or follow us on Twitter and LinkedIn. Investor Contact Shawn M. Cox Epic Bio Manager, Investor Relations, and Corporate Communications shawn.cox@epic-bio.com Media Contact Lisa Raffensperger Ten Bridge Communications lisa@tenbridgecommunications.com (617) 903-8783