Juvenile Idiopathic Arthritis

STOCKHOLM, Oct. 30, 2024 /PRNewswire/ -- Sobi® (STO: SOBI) today announced that new research showing the effect of emapalumab in patients with macrophage activation syndrome (MAS) in Still's disease, including systemic juvenile idiopathic arthritis (sJIA) and adult-onset still's disease (AOSD) who had an inadequate response to high-dose glucocorticoids (GCs) will be presented at the American College of Rheumatology (ACR) Convergence in Washington D.C. The data were pooled from two open-label, single-arm interventional studies, the phase 3 NI-0501-14 study [EMERALD; NCT05001737] and NI-0501-06 [NCT03311854], including 39 patients with MAS. Key results: Complete response at week 8: At week 8, 21 patients (53.8%) achieved a complete response (95% confidence interval (CI): 37.2–69.9%). Complete response at any time: 33 patients (85%) achieved a complete response at any time during the studies. Glucocorticoid tapering: Weekly mean glucocorticoid doses were reduced by 70.1% after 2 weeks of treatment. By week 8, glucocorticoids were tapered to a clinically meaningful dose of ≤1 mg/kg/day in 28 patients (72%) and to ≤0.5 mg/kg/day in 17 patients (44%). Safety: No new safety concerns were identified during the studies. "MAS in Still's disease is a severe condition characterised by intense hyperinflammation and multiple organ failure. Those affected, including young children, often experience high fevers, liver and spleen enlargement, severe cytopenias, and neurological symptoms," said Lydia Abad-Franch, MD, Head of Research & Development and Medical Affairs and Chief Medical Officer at Sobi. "The results to be presented at ACR indicate that emapalumab could potentially provide a new therapeutic option, offering effective control of MAS symptoms and possibly reducing the need for high-dose glucocorticoids. This represents an important development in the treatment landscape for MAS." Additional key results: Overall response (OR) by week 8: 32 patients (82.4%) achieved an overall response by week 8. OR was observed as early as day 5, with a median time to first OR of 2.3 weeks. Clinical MAS remission: 32 patients (82.1%) achieved investigator-assessed clinical MAS remission at any time , with a median time to clinical remission of 3.3 weeks. Survival rate: 37 patients (94.9%) were alive at week 8. Emapalumab is a monoclonal antibody that neutralises interferon gamma (IFNγ), a key cytokine which contributes to the inflammation and tissue damage seen in MAS. The purpose of these two prospective studies is to assess the efficacy and safety of emapalumab in controlling MAS in patients with Still's disease, including sJIA and AOSD, who had an inadequate response to high-dose glucocorticoids. In May 2024, the US FDA granted emapalumab fast track designation as a potential therapeutic option in patient with MAS. Sobi plans to submit an sBLA for this indication to the FDA in 2024. The data will be presented by Alexei A. Grom, MD, Professor of Paediatrics, Research Director Division of Rheumatology, Cincinnati Children's Hospital Medical Centre, in a session entitled "Efficacy and Safety of emapalumab in children and adults with macrophage activation syndrome (MAS) in Still's disease: Results from a phase 3 study and a pooled analysis of two prospective trials" on Tuesday, 19 November at ACR Convergence. About macrophage activation syndrome (MAS) Macrophage activation syndrome (MAS) is a severe complication of rheumatic diseases, most frequently systemic juvenile idiopathic arthritis (sJIA) – a rare systemic disorder of auto-inflammatory nature with common clinical manifestations such as daily spiking fever, typical transient cutaneous rash, arthritis, lymphadenopathy, hepatosplenomegaly and serositis. MAS is characterised by fever, hepatosplenomegaly, liver dysfunction, cytopenias, coagulation abnormalities and hyperferritinaemia, possibly progressing to multiple organ failure and death. MAS is classified as a secondary form of haemophagocytic lymphohistiocytosis (HLH). About emapalumab Emapalumab (Gamifant®) is an anti-interferon gamma (IFNγ) monoclonal antibody that binds to and neutralises IFNγ. In the USA, emapalumab is indicated for the treatment of adult and paediatric (newborn and older) patients with primary haemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. Primary HLH is a rare syndrome of hyperinflammation that usually occurs within the first year of life and can rapidly become fatal unless diagnosed and treated. The FDA approval is based on data from the phase 2/3 studies (NCT01818492 and NCT02069899). Gamifant is indicated for administration through intravenous infusion over one hour twice per week until haematopoietic stem cell transplantation (HSCT). About Sobi® Sobi® is a specialised international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of haematology, immunology, and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia, and Australia. In 2023, revenue amounted to SEK 22.1 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. This information was brought to you by Cision The following files are available for download: SOURCE Swedish Orphan Biovitrum AB WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Media Release First safety data anticipated before year end with assessment of PK/PD relationships to follow in H1’CY2025 SYDNEY, AUSTRALIA, Oct. 17, 2024 (GLOBE NEWSWIRE) -- Immutep Limited (ASX: IMM; NASDAQ: IMMP) (“Immutep” or “the Company”), a clinical-stage biotechnology company developing novel LAG-3 immunotherapies for cancer and autoimmune disease, today announces that the first part (Part A, single dose) of the placebo-controlled, double-blind first-in-human Phase I study evaluating IMP761 has been fully recruited and the drug has been administered with no safety issues. The trial being conducted by the Centre for Human Drug Research (CHDR) in Leiden, the Netherlands, has now progressed to the Part B dose escalation phase with single IMP761 dosing planned to move from 0.03 mg/kg up to 0.90 mg/kg. Both safety and pharmacokinetic/pharmacodynamic (PK/PD) modeling will be assessed in this second cohort of healthy volunteers (N=30). Pending no safety issues, the trial will then shift to the multiple ascending dose portion (Part C) in 14 subjects in which PK will be further evaluated. CHDR will implement its unique keyhole limpet haemocyanin (KLH) challenge model in Parts B and C of this Phase I study, allowing for the evaluation of IMP761’s pharmacodynamic activity at this early stage of clinical development. Immutep anticipates the first safety data from the trial to be available before year end with assessment of PK/PD relationships to follow in the first half of CY2025. For more information on the trial, please visit clinicaltrials.gov (NCT06637865). About IMP761IMP761, a first-in-class immunosuppressive LAG-3 agonist antibody, has the potential to address the root cause of many autoimmune diseases by specifically silencing autoimmune memory T cells that accumulate at disease sites and restoring balance to the immune system. As published in the Journal of Immunology, encouraging pre-clinical in vivo and in vitro studies show IMP761 inhibits peptide-induced T cell proliferation, activation of human primary T cells, and an antigen-specific delayed-type hypersensitivity (DTH) reaction. Additional preclinical data in oligoarticular juvenile idiopathic arthritis (o-JIA) published in Pediatric Research details how IMP761 led to a decrease in a broad spectrum of effector cytokines in just 48 hours. This study also showed children with o-JIA have a skewed LAG-3 metabolism and suggested they can benefit from agonistic LAG-3 activity. About ImmutepImmutep is a clinical-stage biotechnology company developing novel LAG-3 immunotherapy for cancer and autoimmune disease. We are pioneers in the understanding and advancement of therapeutics related to Lymphocyte Activation Gene-3 (LAG-3), and our diversified product portfolio harnesses its unique ability to stimulate or suppress the immune response. Immutep is dedicated to leveraging its expertise to bring innovative treatment options to patients in need and to maximise value for shareholders. For more information, please visit www.immutep.com. Australian Investors/Media:Catherine Strong, Sodali & Co.+61 (0)406 759 268; catherine.strong@sodali.com U.S. Media:Chris Basta, VP, Investor Relations and Corporate Communications+1 (631) 318 4000; chris.basta@immutep.com

– Findings from nine human factors studies, assessing design and user interface, suggest SelfJect functions as an effective delivery device when used as intended1 – – The SelfJect delivery device received the Arthritis Foundation's Ease of Use certification – DUBLIN, Sept. 17, 2024 /PRNewswire/ -- Mallinckrodt plc, a global specialty pharmaceutical company, today announced the publication of findings from seven formative and two validation human factors studies of recently launched Acthar Gel (repository corticotropin injection) Single-Dose Pre-filled SelfJect™ Injector ("SelfJect").2 The manuscript, describing the formative and human factor outcomes, was recently published online in Expert Opinion on Drug Delivery. Approved by the U.S. Food and Drug Administration (FDA) in February of 2024, SelfJect also received the Arthritis Foundation's Ease of Use® certification. This certification recognizes SelfJect for its easy-to-use design, based on testing by an independent third party. Acthar Gel is a naturally sourced complex mixture of adrenocorticotropic hormone (ACTH) analogs and other pituitary peptides.2 Acthar Gel is approved by the FDA for the treatment of several autoimmune disorders and medical conditions known to cause inflammation.2 SelfJect is available in 40 USP units/0.5 mL and 80 USP units/1.0 mL injectors and must be administered by people 18 years of age or older. SelfJect is not to be used for the treatment of infantile spasms. Please see Indications and Important Safety Information for Acthar Gel below. "We're proud of the extensive research and testing conducted to develop SelfJect, including these human factors studies which were critical to our application for FDA approval," said Lisa French, Executive Vice President & Chief Commercial Officer. "These results, and the Arthritis Foundation's Ease of Use certification, are a testament to our commitment to modernize Acthar Gel for our patient community. In keeping with our mission of listening for needs, SelfJect provides an administration option designed to better support patients, caregivers, and medical professionals in managing appropriate conditions." Arthritis Foundation President and CEO Steven Taylor said, "Ease of Use products and packaging are designed to help people with arthritis and those with physical limitations by making everyday tasks easier. We're excited to partner with companies like Mallinckrodt that understand the importance of developing products and packages with the needs of patients at the forefront." About the Studies Human factors studies were conducted utilizing SelfJect to assess the design of the injection device and its user interface, as well as mitigate potential use-related hazards, through observation of participants using the device and participant feedback.1 The published research includes results from nine studies, including seven formative studies, a validation study (with four prior pilot validation studies), and a supplemental validation study with participants including lay users, patients, caregivers, and healthcare providers.1 The formative studies tested and developed various components such as packaging and instructional material, whereas the validation study evaluated administration of the device with the intended-to-market user interface and representative users in representative simulated-use environments.1 Four pilot studies then generated additional protocol recommendations for the validation study.1 In the 160-participant validation study, 91% (n=146) of participants successfully administered their first injection, with 98% (n=156) cumulative success after the second trial.1 Use errors were rare with simulated-use (6.9% [n=194] of all evaluated tasks) and knowledge-based (1.6% [n=56] of all evaluated tasks) testing. The most common use errors were related to not appropriately warming the product before administration.1 Limitations of the studies include1: The controlled study setting may not have accurately replicated real-world complexities that play a role in the administration of Acthar Gel via SelfJect, potentially altering the number of study use errors and deviation. Injections were performed with placebo gel into an injection pad and not a patient. The feedback that a user receives when injecting into skin versus a surrogate injection pad may influence use behavior. A more realistic delivery container could have been used to cue the participant that this product would arrive cold and would need to be stored appropriately. Participant selection bias, reliance on self-reported and qualitative data, and small sample sizes in the formative and pilot studies may have impacted results. If a customer is interested in learning more about SelfJect, they can reach out to their local representative or visit ActharHCP.com. INDICATIONS Acthar Gel is indicated for: Inducing a diuresis or a remission of proteinuria in nephrotic syndrome without uremia of the idiopathic type or that due to lupus erythematosus Monotherapy for the treatment of infantile spasms in infants and children under 2 years of age Treatment of acute exacerbations of multiple sclerosis in adults. Controlled clinical trials have shown Acthar to be effective in speeding the resolution of acute exacerbations of multiple sclerosis. However, there is no evidence that it affects the ultimate outcome or natural history of the disease Severe acute and chronic allergic and inflammatory processes involving the eye and its adnexa such as: keratitis, iritis, iridocyclitis, diffuse posterior uveitis and choroiditis, optic neuritis, chorioretinitis, anterior segment inflammation Symptomatic sarcoidosis Treatment during an exacerbation or as maintenance therapy in selected cases of systemic lupus erythematosus Treatment during an exacerbation or as maintenance therapy in selected cases of systemic dermatomyositis (polymyositis) Adjunctive therapy for short-term administration (to tide the patient over an acute episode or exacerbation) in: psoriatic arthritis; rheumatoid arthritis, including juvenile rheumatoid arthritis (selected cases may require low-dose maintenance therapy); ankylosing spondylitis IMPORTANT SAFETY INFORMATION Contraindications Acthar is contraindicated: For intravenous administration In infants under 2 years of age who have suspected congenital infections With concomitant administration of live or live attenuated vaccines in patients receiving immunosuppressive doses of Acthar In patients with scleroderma, osteoporosis, systemic fungal infections, ocular herpes simplex, recent surgery, history of the presence of a peptic ulcer, congestive heart failure, uncontrolled hypertension, primary adrenocortical insufficiency, adrenocortical hyperfunction, or sensitivity to proteins of porcine origin Warnings and Precautions The adverse effects of Acthar are related primarily to its steroidogenic effects Acthar may increase susceptibility to new infection or reactivation of latent infections Suppression of the hypothalamic-pituitary-adrenal (HPA) axis may occur following prolonged therapy with the potential for adrenal insufficiency after withdrawal of the medication. Adrenal insufficiency may be minimized by tapering of the dose when discontinuing treatment. During recovery of the adrenal gland patients should be protected from the stress (e.g., trauma or surgery) by the use of corticosteroids. Monitor patients for effects of HPA axis suppression after stopping treatment Cushing's syndrome may occur during therapy but generally resolves after therapy is stopped. Monitor patients for signs and symptoms Acthar can cause elevation of blood pressure, salt and water retention, and hypokalemia. Monitor blood pressure and sodium and potassium levels Acthar often acts by masking symptoms of other diseases/disorders. Monitor patients carefully during and for a period following discontinuation of therapy Acthar can cause gastrointestinal (GI) bleeding and gastric ulcer. There is also an increased risk for perforation in patients with certain GI disorders. Monitor for signs of perforation and bleeding Acthar may be associated with central nervous system effects ranging from euphoria, insomnia, irritability, mood swings, personality changes, and severe depression to psychosis. Existing conditions may be aggravated Patients with comorbid disease may have that disease worsened. Caution should be used when prescribing Acthar in patients with diabetes and myasthenia gravis Prolonged use of Acthar may produce cataracts, glaucoma, and secondary ocular infections. Monitor for signs and symptoms Acthar is immunogenic and prolonged administration of Acthar may increase the risk of hypersensitivity reactions. Cases of anaphylaxis have been reported in the postmarketing setting. Neutralizing antibodies with chronic administration may lead to loss of endogenous ACTH and Acthar activity There may be an enhanced effect in patients with hypothyroidism and in those with cirrhosis of the liver Long-term use may have negative effects on growth and physical development in children. Monitor pediatric patients Decrease in bone density may occur. Bone density should be monitored in patients on long-term therapy Adverse Reactions Commonly reported postmarketing adverse reactions for Acthar include injection site reaction, asthenic conditions (including fatigue, malaise, asthenia, and lethargy), fluid retention (including peripheral swelling), insomnia, headache, and blood glucose increased The most common adverse reactions for the treatment of infantile spasms (IS) are increased risk of infections, convulsions, hypertension, irritability, and pyrexia. Some patients with IS progress to other forms of seizures; IS sometimes masks these seizures, which may become visible once the clinical spasms from IS resolve Pregnancy Acthar may cause fetal harm when administered to a pregnant woman Please see full Prescribing Information for additional Important Safety Information. ABOUT MALLINCKRODT Mallinckrodt is a global business consisting of multiple wholly owned subsidiaries that develop, manufacture, market and distribute specialty pharmaceutical products and therapies. The company's Specialty Brands reportable segment's areas of focus include autoimmune and rare diseases in specialty areas like neurology, rheumatology, hepatology, nephrology, pulmonology, ophthalmology, and oncology; immunotherapy and neonatal respiratory critical care therapies; analgesics; and gastrointestinal products. Its Specialty Generics reportable segment includes specialty generic drugs and active pharmaceutical ingredients. To learn more about Mallinckrodt, visit . CAUTIONARY STATEMENTS RELATED TO FORWARD-LOOKING STATEMENTS This release contains forward-looking statements, including with regard to Acthar Gel (repository corticotropin injection), the Acthar Gel Single-Dose Pre-filled SelfJect™ Injector, the potential of these products to improve health and treatment outcomes, their potential impact on patients and the availability of Acthar Gel Single-Dose Pre-filled SelfJect Injector in the U.S. in the future. The statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those in the forward-looking statements: the effects of Mallinckrodt's recent emergence from bankruptcy; satisfaction of, and compliance with, regulatory and other requirements; actions of regulatory bodies and other governmental authorities; changes in laws and regulations; changes in market demand; issues with product quality, manufacturing or supply, or patient safety issues or adverse side effects or adverse reactions associated with Acthar Gel and Acthar Gel Single-Dose Pre-filled SelfJect Injector; and other risks identified and described in more detail in the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of Mallinckrodt's most recent Annual Report on Form 10-K and other filings with the SEC, all of which are available on its website. The forward-looking statements made herein speak only as of the date hereof and Mallinckrodt does not assume any obligation to update or revise any forward-looking statement, whether as a result of new information, future events and developments or otherwise, except as required by law. CONTACT Media Inquiries Green Room Communications 908-577-4531 [email protected] Investor Relations Derek Belz Vice President, Investor Relations 314-654-3950 [email protected] Mallinckrodt, the "M" brand mark, SelfJect, and the Mallinckrodt Pharmaceuticals logo are trademarks of a Mallinckrodt company. Other brands are trademarks of a Mallinckrodt company or their respective owners. ©2024 Mallinckrodt. US-2400718 09/24 References 1 Linnane A, Lau M, Miranda P, Elliott S. Formative and Validation Human Factors Studies of a New Disposable Prefilled Injection Device for Subcutaneous Delivery of Acthar Gel (Repository Corticotropin Injection). Expert Opinion on Drug Delivery. 2024. . 2 Acthar® Gel (repository corticotropin injection) [prescribing information]. Bridgewater, NJ: Mallinckrodt ARD LLC. SOURCE Mallinckrodt plc WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED