Mumps

After stealing the show at December’s American Society of Hematology (ASH) meeting with promising early data of lupus patients treated with CAR-T cell therapy, a team of German scientists has now published updated results in the NEJM that suggest the one-time treatment can lead to sustained remission for patients with several autoimmune diseases.At 29 months of follow-up, all eight patients with systemic lupus erythematosus (SLE) had no signs of disease activity. “Even though it is premature to judge whether these patients are indeed cured from their autoimmune disease, CD19 CAR T cells at least appear to be able to achieve sustained disease- and drug-free remission,” the authors wrote.While four CD19-directed CAR-T therapies have been approved for various haematological malignancies, Thursday’s study adds to a growing body of evidence that the modality could also be an effective tool to treat autoimmune diseases because they target the same underlying culprit: B-cells. But while the approved treatments attack malignant B-cells in patients with cancer, for those with an autoimmune disease, the goal is a deep depletion of autoreactive B-cells.The study authors note that antibody treatments targeting B-cells have had modest efficacy in autoimmune diseases, but they’ve yet to achieve long-lasting drug-free remission. The goal with CAR-T therapy is to unleash one powerful treatment that reduces B-cells to a level so low that it can essentially result in a reboot of the immune system. Resetting autoimmunityThe study enrolled 15 patients who had an inadequate response to at least two previous immune-suppressive treatments, including eight with SLE, three with idiopathic inflammatory myositis, and four with systemic sclerosis. Each received a single treatment with MB-CART19.1, an experimental CD19-directed CAR-T therapy from Miltenyi Biomedicine. No authors on the paper are affiliated with the German biotech.At ASH, the researchers announced that all 15 patients had discontinued immunosuppressive drugs at the 15-month follow-up. In Thursday’s paper, the authors revealed more details they say supports the long-term benefit of CAR-T to treat autoimmune diseases, including autoantibody depletion, B-cell reconstitution, and vaccination response.  All but one of the lupus patients were evaluable for a one-year post-infusion autoantibody test, which found that all of them exhibited a sustained absence of SLE-specific autoantibodies. Additionally, all eight patients showed normal levels of complement factor C3, and seven experienced complete resolution of proteinuria.Moreover, all patients experienced full B-cell reconstitution for as long as two years without relapse, suggesting a one-time CAR-T treatment can “reset” pathologic autoimmunity. The cell therapy’s potential to be an immune on/off switch was also supported by the appearance of a naive non–class-switched B-cell system, the disappearance of circulating plasmablasts, and the down-regulation of specific disease-associated heavy and light chains.“Although CAR T-cell therapy is directed to both pathogenic and nonpathogenic B cells, the rebooting of the B-cell system after the disappearance of CAR T cells obviously occurs in the absence of the pathogenic B cells that trigger autoantibody production,” the authors wrote.Finally, the researchers administered standard vaccines to each lupus patient, including tetanus, measles, mumps, rubella, varicella–zoster virus, and Epstein–Barr virus, and their protective IgG antibody responses remained stable.

BEIJING--(BUSINESS WIRE)-- Sinovac Biotech Ltd. (“Sinovac” or the “Company”) (NASDAQ: SVA), a leading provider of biopharmaceutical products in China, today announced that its board of directors has amended its shareholder rights plan. The amendment extends the expiration date of the plan from February 22, 2024 to February 22, 2025. About SINOVAC Sinovac Biotech Ltd. (SINOVAC) is a China-based biopharmaceutical company that focuses on the R&D, manufacturing, and commercialization of biomedical products that protect against human infectious diseases. SINOVAC’s product portfolio includes vaccines against COVID-19, enterovirus 71 (EV71) infected hand-foot-mouth disease (HFMD), hepatitis A, varicella, influenza, poliomyelitis, pneumococcal disease, mumps, etc. The COVID-19 vaccine, CoronaVac®, has been approved for use in more than 60 countries and regions worldwide. The hepatitis A vaccine, Healive®, passed WHO prequalification requirements in 2017. The EV71 vaccine, Inlive®, is an innovative vaccine under "Category 1 Preventative Biological Products" and commercialized in China in 2016. In 2022, SINOVAC’s Sabin-strain inactivated polio vaccine (sIPV) and varicella vaccine were prequalified by the WHO. SINOVAC was the first company to be granted approval for its H1N1 influenza vaccine Panflu.1®, which has supplied the Chinese government's vaccination campaign and stockpiling program. The Company is also the only supplier of the H5N1 pandemic influenza vaccine, Panflu®, to the Chinese government stockpiling program. SINOVAC continually dedicates itself to pipeline development including but not limited to new technology, new vaccines as well as other biomedical products. We will constantly explore global opportunities of strategic expansion. For more information, please visit the Company’s website at . View source version on businesswire.com: Contacts Sinovac Biotech Ltd. Helen Yang Tel: +86-10-8279 9720 Email: ir@sinovac.com Source: Sinovac Biotech Ltd. View this news release online at:

OSAKA, Japan & CAMBRIDGE, MA, USA I January 29, 2024 I Takeda ( TSE:4502/NYSE:TAK ) today announced that the U.S. Food and Drug Administration (FDA) has approved GAMMAGARD LIQUID ® [Immune Globulin Infusion (Human) 10% solution] as an intravenous immunoglobulin (IVIG) therapy to improve neuromuscular disability and impairment in adults with chronic inflammatory demyelinating polyneuropathy (CIDP). 1 It can be used as induction therapy, which includes an induction dose followed by maintenance doses. 1 For treatment of CIDP, GAMMAGARD LIQUID has not been studied in immunoglobulin-naive patients nor as maintenance therapy for periods longer than six months. 1 This milestone follows the recent FDA approval of HYQVIA ® [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase] for maintenance therapy to prevent the relapse of neuromuscular disability and impairment in adults with CIDP. 2 HYQVIA is the only combination of immunoglobulin (IG) and hyaluronidase, which makes it a facilitated subcutaneous IG infusion. 2 “The approval of GAMMAGARD LIQUID for treatment of CIDP is an encouraging validation of our decades-long commitment to advancing plasma-derived therapies on behalf of people living with rare neuromuscular disorders and bringing our portfolio of differentiated IG therapies to these patients,” said Richard Ascroft, senior vice president and head of Takeda’s U.S. Plasma-Derived Therapies Business Unit. “Together with the recent HYQVIA approval in the U.S., we can now offer induction and maintenance therapy options to adults living with CIDP that may accommodate their personal treatment needs.” The approval is based on results from a prospective, open-label, single-arm, multicenter clinical study (ADVANCE-CIDP 2) that evaluated the efficacy and safety of GAMMAGARD LIQUID in adults with CIDP who developed a relapse in the randomized, double-blinded, placebo-controlled study evaluating efficacy, safety and tolerability of HYQVIA (ADVANCE-CIDP 1) in adults with CIDP. Efficacy in ADVANCE-CIDP 2 was based on responder rate, where a responder was defined as a subject who demonstrated an improvement of functional disability. The responder rate was 94.4% (N=18, 95% CI: 74.2% to 99.0%). Improvement in grip strength and change in Rasch-built Overall Disability Scale (R-ODS) score were recorded across participants. 1 The most common adverse reactions observed in ≥5% of clinical study patients were headache, pyrexia, anemia, leukopenia, neutropenia, illness, blood creatinine increased, dizziness, migraine, somnolence, tremor, nasal dryness, abdominal pain upper, vomiting, chills, nasopharyngitis and pain in extremity. 1 CIDP is a rare, acquired, immune-mediated neuromuscular disorder affecting the peripheral nervous system. 3,4 It is characterized by progressive, symmetric symptoms such as weakness, tingling or loss of feeling in distal and proximal limbs, loss of reflexes and difficulty walking. 3 Because its symptoms may overlap with other rare, neuromuscular conditions, CIDP may be misdiagnosed. 5 The mechanism of action of immunoglobulins in the treatment of CIDP in adults has not been fully elucidated but may include immunomodulatory effects. 1 “As the standard of care for the treatment of CIDP, IG therapy is thought to help normalize compromised immune systems through immunomodulatory mechanisms,” said Dr. Mamatha Pasnoor, professor in the Department of Neurology at the University of Kansas Medical Center. “Because CIDP is a progressive and complex disease, multiple treatment options are needed, and clinicians now have an additional therapy that can help adults with CIDP manage their disease.” GAMMAGARD LIQUID is the only IVIG with multiple neuromuscular disorder indications in the U.S. since it is now approved for CIDP and it is the only FDA-approved IVIG to treat multifocal motor neuropathy as a maintenance therapy to improve muscle strength and disability in adults. 1 It is also indicated in the U.S. as a replacement therapy for people two years of age or older living with primary immunodeficiency. 1 About GAMMAGARD LIQUID GAMMAGARD LIQUID ® [Immune Globulin Infusion (Human) 10% solution] is an intravenous immunoglobulin (IVIG) that is infused into the veins. GAMMAGARD LIQUID is approved in the U.S. as an IG therapy to improve neuromuscular disability and impairment in adult patients with CIDP, as a replacement therapy for primary immunodeficiency (PI) in adult and pediatric patients two years of age and older, and as a maintenance therapy to improve muscle strength and disability in adult patients with multifocal motor neuropathy (MMN). Also known as KIOVIG outside the U.S. and Canada, it is approved in 66 countries worldwide. About HYQVIA HYQVIA ® [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase] is a liquid medicine containing Recombinant Human Hyaluronidase and immunoglobulin (IG) and is approved in the U.S. to treat adults and children two years of age and older with primary immunodeficiency (PI), and as maintenance therapy to prevent relapse of neuromuscular disability and impairment in adult patients with CIDP. It is also approved by the European Medicines Agency (EMA) as a replacement therapy in adults, children and adolescents with PI and with secondary immunodeficiency (SID) who suffer from severe or recurrent infections, ineffective antimicrobial treatment, and either proven specific antibody failure (PSAF) or serum IgG level of <4 g/L. HYQVIA is infused under the skin into the fatty subcutaneous tissue. HYQVIA contains IG collected from human plasma. IG are antibodies that maintain the body’s immune system. The hyaluronidase part of HYQVIA facilitates the dispersion and absorption of IG in the subcutaneous space between the skin and the muscle. HYQVIA is infused up to once a month (every two, three or four weeks for CIDP; every three or four weeks for PI). About ADVANCE-CIDP 2 ADVANCE-CIDP 2 was a prospective, open-label, single-arm, multicenter clinical study that evaluated the efficacy and safety of GAMMAGARD LIQUID in adults with CIDP who developed a relapse in the randomized, double-blinded, placebo-controlled study evaluating efficacy, safety and tolerability of HYQVIA (ADVANCE-CIDP 1) in adults with CIDP. The 18 patients who relapsed during ADVANCE-CIDP 1 were offered enrollment in ADVANCE-CIDP 2. GAMMAGARD LIQUID was administered at an induction dose of 2 g/kg body weight, followed by maintenance infusions at every three weeks for a period of six months. The dose of GAMMAGARD LIQUID treatment could be adjusted at the investigator's discretion. Adjustments to the dosing interval of every three weeks were not allowed. All subjects completed the study. All dosed subjects were analyzed for efficacy and safety. Efficacy of ADVANCE-CIDP 2 was based on responder rate, where a responder is defined as subjects who had at least a one-point decrease in the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score at the completion of the treatment period (six months). The responder rate was 94.4% (N=18, 95% CI: 74.2% to 99.0%). The INCAT score returned to baseline values prior to joining the study in 17 of the 18 subjects (94.4%) at six months. All subjects had improvement in functional ability as defined by a composite outcome metrics that included INCAT score, grip strength, or Rasch-built Overall Disability Scale (R-ODS) score. Further information about the ADVANCE-CIDP 2 study is available at ClinicalTrials.gov under study identifier NCT02549170 . INDICATIONS GAMMAGARD LIQUID is indicated as replacement therapy for Primary Humoral Immunodeficiency (PI) in adult and pediatric patients ≥2 years, as a maintenance therapy to improve muscle strength and disability in adult patients with Multifocal Motor Neuropathy (MMN), and as a therapy to improve neuromuscular disability and impairment in adult patients with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP). LIMITATIONS OF USE (CIDP): GAMMAGARD LIQUID has not been studied in immunoglobulin-naive patients with CIDP. GAMMAGARD LIQUID maintenance therapy in CIDP has not been studied for periods longer than 6 months. After responding during an initial treatment period, not all patients require indefinite maintenance therapy with GAMMAGARD LIQUID in order to remain free of CIDP symptoms. Individualize the duration of any treatment beyond 6 months based upon the patient’s response and demonstrated need for continued therapy. HYQVIA is indicated for the treatment of Primary Immunodeficiency (PI) in adults and pediatric patients two years of age and older and for Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) as maintenance therapy to prevent relapse of neuromuscular disability and impairment in adults. HYQVIA is for subcutaneous use only. GAMMAGARD LIQUID for PI is for intravenous or subcutaneous use. GAMMAGARD LIQUID for MMN and CIDP is for intravenous use only. IMPORTANT SAFETY INFORMATION WARNING: THROMBOSIS HYQVIA and GAMMAGARD LIQUID WARNING: RENAL DYSFUNCTION and ACUTE RENAL FAILURE GAMMAGARD LIQUID Contraindications Warnings and Precautions Hypersensitivity: Severe hypersensitivity reactions may occur, even in patients who have tolerated previous treatment with human IG. If a hypersensitivity reaction occurs, discontinue infusion immediately and institute appropriate treatment. IgA-deficient patients with antibodies to IgA are at greater risk of developing potentially severe hypersensitivity reactions, including anaphylaxis. Renal Dysfunction/Failure: Acute renal dysfunction/failure, acute tubular necrosis, proximal tubular nephropathy, osmotic nephrosis, and death may occur with IV use of IG products, especially those containing sucrose. Acute renal dysfunction/failure has been reported in association with infusions of GAMMAGARD LIQUID . Ensure patients are not volume depleted prior to infusion. In patients at risk due to pre-existing renal insufficiency or predisposition to acute renal failure, assess renal function before initiation and throughout treatment, and consider lower, more frequent dosing. If renal function deteriorates, consider discontinuation. Thrombosis: Has been reported to occur following treatment with IG products, including HYQVIA and in the absence of known risk factors. In patients at risk, administer at the minimum dose and infusion rate practicable. Ensure adequate hydration before administration. Monitor for signs and symptoms of thrombosis and assess blood viscosity in patients at risk for hyperviscosity. Aseptic Meningitis Syndrome: Has been reported with use of IG, including HYQVIA and may occur more frequently in females. Conduct a thorough neurological exam on patients exhibiting signs and symptoms, to rule out other causes of meningitis. Discontinuing IG treatment has resulted in remission within several days without sequelae. The syndrome usually begins within several hours to two days following IG treatment. Hemolysis: HYQVIA and GAMMAGARD LIQUID contain blood group antibodies, which may cause a positive direct antiglobulin reaction and hemolysis. Monitor patients for signs and symptoms of hemolysis and delayed hemolytic anemia and, if present, perform appropriate confirmatory lab testing. Transfusion-Related Acute Lung Injury: Non-cardiogenic pulmonary edema has been reported with IV-administered IG, including GAMMAGARD LIQUID . Monitor patients for pulmonary adverse reactions. If suspected, perform appropriate tests for presence of anti-neutrophil and anti-HLA antibodies in both product and patient serum. May be managed using oxygen therapy with adequate ventilatory support. Transmittable Infectious Agents: Because HYQVIA and GAMMAGARD LIQUID are made from human plasma, they may carry a risk of transmitting infectious agents (e.g., viruses, other pathogens). No confirmed cases of viral transmission of variant Creutzfeldt-Jakob disease (vCJD) have been associated with GAMMAGARD LIQUID , and no cases have been associated with HYQVIA . Interference with Lab Tests: False positive serological test results and certain assay readings, with the potential for misleading interpretation, may occur as the result of passively transferred antibodies. Additional Warnings and Precautions for HYQVIA Immunogenicity of Recombinant Human Hyaluronidase (rHuPH20 ): Non-neutralizing antibodies to the Recombinant Human Hyaluronidase component can develop. The clinical significance of these antibodies or whether they interfere with fertilization in humans is unknown. Spread of Localized Infection: Do not infuse HYQVIA into or around an infected area due to potential risk of spreading a localized infection. Additional Warnings and Precautions for GAMMAGARD LIQUID Hyperproteinemia, increased serum viscosity, and hyponatremia may occur. It is critical to distinguish true hyponatremia from a pseudohyponatremia because certain treatments may lead to volume depletion, a further increase in serum viscosity, and a predisposition to thromboembolic events. Adverse Reactions HYQVIA The most common adverse reactions observed in >5% of patients in the clinical trials were: PI: local adverse reactions including pain, erythema, edema, and pruritus, and systemic adverse reactions including, headache, antibody formation against Recombinant Human Hyaluronidase (rHuPH20), fatigue, nausea, pyrexia, and vomiting. CIDP: local reactions, headache, pyrexia, nausea, fatigue, erythema, pruritus, increased lipase, abdominal pain, back pain, and pain in extremity. GAMMAGARD LIQUID The most serious adverse reactions observed in clinical studies in PI was aseptic meningitis, and in MMN were pulmonary embolism and blurred vision. The most common adverse reactions observed in ≥5% of patients in the clinical trials were: IV administration for PI: Headache, fatigue, pyrexia, nausea, chills, rigors, pain in extremity, diarrhea, migraine, dizziness, vomiting, cough, urticaria, asthma, pharyngolaryngeal pain, rash, arthralgia, myalgia, oedema peripheral, pruritus, and cardiac murmur. Subcutaneous administration for PI: Infusion site (local) event (rash, erythema, edema, hemorrhage, and irritation), headache, fatigue, heart rate increased, pyrexia, abdominal pain upper, nausea, vomiting, asthma, blood pressure systolic increased, diarrhea, ear pain, aphthous stomatitis, migraine, oropharyngeal pain, and pain in extremity. IV administration for MMN: Headache, chest discomfort, muscle spasms, muscular weakness, nausea, oropharyngeal pain, and pain in extremity. IV administration for CIDP: Headache, pyrexia, anemia, leukopenia, neutropenia, illness, blood creatinine increased, dizziness, migraine, somnolence, tremor, nasal dryness, abdominal pain upper, vomiting, chills, nasopharyngitis, and pain in extremity. Drug Interactions Passive transfer of antibodies may transiently interfere with the immune responses to live attenuated virus vaccines (e.g., measles, mumps, rubella, and varicella). Use In Specific Populations Pregnancy: Limited human data are available on the use of HYQVIA during pregnancy. The effects of antibodies to the Recombinant Human Hyaluronidase on the human embryo or fetal development are unknown. It is not known whether HYQVIA can cause fetal harm when administered to a pregnant woman or if it can affect reproductive capacity. HYQVIA should be given to a pregnant woman only if clearly needed. For Full U.S. Prescribing Information for GAMMAGARD LIQUID, please visit: https://www.shirecontent.com/PI/PDFs/GAMLIQUID_USA_ENG.pdf For Full U.S. Prescribing Information for HYQVIA, please visit: https://www.shirecontent.com/PI/PDFs/HYQVIA_USA_ENG.pdf 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 . 1 GAMMAGARD LIQUID ® [Immune Globulin Infusion (Human) 10% solution] U.S. Prescribing Information. https://www.shirecontent.com/PI/PDFs/GAMLIQUID_USA_ENG.pdf 2 HYQVIA ® [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase] U.S. Prescribing Information. https://www.shirecontent.com/PI/PDFs/HYQVIA_USA_ENG.pdf 3 Dalakas MC. Nat Rev Neurol . 2011;7(9):507–17. 4 GBS CIDP Foundation International. Voice of the Patient Report. August 26, 2022. www.gbs-cidp.org . Accessed August 2022. 5 Broers MC, Bunschoten C, Nieboer D, Lingsma HF, Jacobs BC. Eur J Neurol . 2021;28(6):2065-2073. SOURCE: Takeda Pharmaceutical Co