Bohring Syndrome

– THERAKOS, the world's only fully integrated and validated Extracorporeal Photopheresis (ECP) system, 1 received this CE certificate under the EU's new and more rigorous certification process – DUBLIN, Sept. 3, 2024 /PRNewswire/ -- Mallinckrodt plc, a global specialty pharmaceutical company, announced that the THERAKOS™ CELLEX™ Photopheresis System has obtained CE Certification under the updated European Union Medical Device Regulation (EU MDR) 2017/745. The EU MDR 2017/745 is a rigorous regulatory certification program for medical devices intended to support innovation while maintaining safety. The process for THERAKOS to achieve this new CE certificate involved a quality management system audit of the Dublin site, a technical review, a microbiological review, and a thorough clinical assessment. The EU MDR application for THERAKOS was submitted in September 2022. Along with addressing the new requirements for medical devices, this submission also included two important design enhancements: The addition of the anticoagulant ACD-A to the current labelling and relative Software changes to ensure optimal patient and operator experience Material changes to remove DEHP (phthalate) and ensure compliance to latest Restriction of Hazardous Substances in Electrical and Electronic Equipment (RoHS) standards THERAKOS is approved for use in patients who require the administration of photopheresis. This includes patients older than 18 with Cutaneous T Cell Lymphoma or Solid Organ Transplant Rejection (heart, lung), and patients older than 3 with Acute and Chronic Graft versus Host Disease. "We are pleased THERAKOS has received the CE Certificate under the new EU MDR requirements that have been put in place to ensure the highest quality and safety standards for medical devices in the EU," said Christopher Hirt, MD, Vice President Hospital International. "Importantly, THERAKOS was granted this certificate after meeting the increased safety and performance requirements, which may give healthcare providers even greater confidence when treating patients." About THERAKOS™ CELLEX™ Photopheresis System The THERAKOS CELLEX Photopheresis System is the world's only fully integrated and validated ECP system.¹ THERAKOS performs ECP using patented technology that collects, separates and treats a small amount of white blood cells (immune cells) while the patient is connected to the instrument. The treated cells are then returned to the patient where they help to modify the immune response in a process called immunomodulation. It is used to treat a range of immune-mediated diseases. THERAKOS Systems are used by over 300 treatment centres in over 30 countries worldwide.² About Extracorporeal Photopheresis (ECP) Extracorporeal photopheresis (ECP) is an immunomodulatory therapy that has demonstrated efficacy in various T-cell and immune-mediated diseases.³ ECP is recommended by international and national guidelines for a spectrum of diseases, including cutaneous T-cell lymphoma (CTCL), acute and chronic graft-versus-host disease (aGvHD and cGvHD), chronic lung allograft dysfunction-bronchiolitis obliterans syndrome (CLAD-BOS) and after cardiac transplantation.4,5,6,7,8,9,10,11,12,13,14,15 IMPORTANT SAFETY INFORMATION FOR THE THERAKOS™ PHOTOPHERESIS PROCEDURE UNDER EU MDR Indications under EU MDR The THERAKOS™ CELLEX™ Photopheresis System is indicated for patients older than 18 years of age for the administration of photopheresis in the following: Cutaneous T Cell Lymphoma (CTCL) Solid Organ Transplant Rejection (SOT) (heart, lung) The THERAKOS™ CELLEX™ Photopheresis System is indicated in patients older than 3 years of age for the management of: Acute and Chronic Graft versus Host Disease (aGvHD, cGvHD) Contraindications THERAKOS™ Photopheresis is contraindicated in: Patients possessing a specific history of a light sensitive disease Patients who cannot tolerate extracorporeal volume loss or who have white blood cell counts greater than 25,000 / mm3 Patients who have coagulation disorders or who have previously had a splenectomy Warnings and Precautions THERAKOS™ Photopheresis treatments should always be performed in locations where standard medical emergency equipment is available. Volume replacement fluids and/or volume expanders should be readily available throughout the procedure. Do not expose the device to a magnetic resonance (MR) environment. The device may present a risk of projectile injury, and thermal injury and burns may occur. The device may generate artifacts in the MR image, or may not function properly. Thromboembolic events, including pulmonary embolism and deep vein thrombosis, have been reported in the treatment of Graft versus Host Disease (GvHD). Special attention to adequate anticoagulation is advised when treating patients with GvHD. When prescribing and administering THERAKOS Photopheresis for patients receiving concomitant therapy, exercise caution when changing treatment schedules to avoid increased disease activity that may be caused by abrupt withdrawal of previous therapy. Adverse Events Hypotension may occur during any treatment involving extracorporeal circulation. Closely monitor the patient during the entire treatment for hypotension. Transient pyretic reactions, 37.7–38.9°C (100–102°F), have been observed in some patients within six to eight hours of reinfusion of the photoactivated leukocyte-enriched blood. A temporary increase in erythroderma may accompany the pyretic reaction. Treatment frequency exceeding labelling recommendations may result in anaemia. Venous access carries a small risk of infection and pain. Please refer to the THERAKOS™ CELLEX™ Photopheresis System Operator Manual for a complete list of warnings and precautions. IMPORTANT SAFETY INFORMATION FOR METHOXSALEN USED IN CONJUNCTION WITH THERAKOS™ PHOTOPHERESIS Contraindications Methoxsalen is contraindicated in: Patients exhibiting idiosyncratic or hypersensitivity reactions to methoxsalen, psoralen compounds, or any of the excipients Patients with co-existing melanoma, basal cell or squamous cell skin carcinoma Patients who are pregnant, and sexually active men and women of childbearing potential unless adequate contraception is used during treatment Patients with aphakia because of the significantly increased risk of retinal damage due to the absence of a lens Warnings and Precautions Special care should be exercised in treating patients who are receiving concomitant therapy (either topically or systemically) with known photosensitizing agents. Oral administration of methoxsalen followed by cutaneous UVA exposure (PUVA therapy) is carcinogenic. Patients should be told emphatically to wear UVA absorbing, wrap-around sunglasses for twenty-four (24) hours after methoxsalen treatment. They should wear these glasses any time they are exposed to direct or indirect sunlight, whether they are outdoors or exposed through a window. Safety in children has not been established. Refer to the package insert for methoxsalen sterile solution (20 micrograms / mL) or the oral 8-methoxypsoralen dosage formulation for a list of all warnings and precautions. Please refer to the THERAKOS™ CELLEX™ Photopheresis System Operator Manual for a complete list of warnings and precautions and adverse events. 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 THERAKOS, its potential to improve health and treatment outcomes, and its potential impact on patients. 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; issues with product quality, manufacturing or supply, or patient safety issues or adverse side effects or adverse reactions associated with THERAKOS; 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, Quarterly Reports on Form 10-Q, 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, 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. EU-2400172 09/24 References 1 Knobler R, et al. J Eur Acad Dermatol Venereol. 2020;34(12):2693-2716. 2 Data on File – Ref-07615. Mallinckrodt Pharmaceuticals. 3 Hart JW, et al. Ther Adv Hematol. 2013;4:320-334. 4 Knobler R, et al. J Eur Acad Dermatol Venereol. 2020;34(12):2693-2716. 5 Trautinger F, et al. Eur J Cancer. 2017;77:57 74. 6 Padmanabhan A, et al. J Clin Apher. 2019;34(3):171-354. 7 Alfred A, et al. Br J Haematol. 2017;177:287-310. 8 Cho A, et al. Front Med (Lausanne). 2018;5:236. 9 Zeiser R. et al. Graft-versus-Host Erkrankung, akut. 2021. Available at: . Accessed August 2024. 10 Bredeson C, et al. Curr Oncol. 2014;21(2):e310-325. 11 Pierelli L, et al. Transfusion. 2013;53(10):2340-2352. 12 Wolff D, et al. Biol Blood Marrow Transplant. 2011;17:1-17. 13 Dignan FL, et al. Br J Haematol. 2012;158(1):30-45; 46-61. 14 Knobler R, et al. J Eur Acad Dermatol Venereol. 2021;25(1):27-49. 15 Costanzo MR, et al. J Heart Lung Transplant. 2010;29(8):914-956. Logo -

Key secondary endpoints demonstrated the mean total score improvement in quality of life (QOL) at day 28 of treatment was more than twice the minimal clinically important difference (MCID). Patients reported markedly improved QOL based on the total score and all three domains of the St. George’s Respiratory Questionnaire (SGRQ). Placebo did not meet the MCID for the SGRQ during the study. This improvement in health status was confirmed by the Chronic Airways Assessment Test (CAAT) scores at day 28 demonstrating symptom reduction that exceeded the proposed MCID at the end of the treatment period. Placebo did not meet the MCID for the CAAT during the study. Analysis of sputum samples from the ARINA-1 treatment arm demonstrated a reduction in mucus viscosity and neutrophil elastase (NE) relative to placebo, confirming ARINA-1 previously observed mechanism of action data and providing strong mechanistic support for the improvements in QOL. CLIMB met the primary endpoint of safety and tolerability in patients at 28 days of treatment with no treatment-related serious adverse events. Renovion plans to advance ARINA-1 for the treatment of NCFBE in a Phase 3 program. CHAPEL HILL, N.C.--(BUSINESS WIRE)-- Renovion Inc., a biopharmaceutical company focused on developing therapies to restore lung health in individuals with mucus-related lung diseases, announced topline results of the Phase 2 CLIMB study of ARINA-1 in individuals with non-CF bronchiectasis (NCFBE). ARINA-1 is a novel nebulized therapy in development for reducing mucus symptoms in individuals with NCFBE, chronic bronchitis, lung transplant, and other respiratory diseases with chronic mucus symptoms. The CLIMB study was a randomized double-blind placebo-controlled study to assess the safety and efficacy of ARINA-1 in NCFBE. The trial was conducted at nine sites across the US. Participants were randomized (7:3, treatment:placebo), and 40 participants completed the study, with 29 completing in the ARINA-1 arm and 11 in the placebo (0.9% saline) arm. Topline results from the intention-to-treat (ITT) population demonstrate that ARINA-1 is safe and improves quality of life (QOL) in individuals with NCFBE who experience mucus symptoms. "These results highlight the important role that ARINA-1 could play in the treatment of non-CF bronchiectasis and are extremely encouraging for patients," said Charles Daley, MD, Professor and Chief, Division of Mycobacterial & Respiratory Infections at National Jewish Health in Denver, Colorado and Principal Investigator of the CLIMB trial. "Today, many [NCFBE] patients suffer from chronic cough and sputum production which affects their QOL and may eventually lead to exacerbations, with no approved pharmaceutical therapies available. There is an urgent need for new effective therapies that can break the vicious cycle of excess mucus, inflammation, and infection for these patients." ARINA-1 was well-tolerated in individuals with NCFBE with mucus symptoms. The most common treatment-related AEs (TRAEs) (event rates (95%CI)) in the ARINA-1 arm (n=29) were cough (0.3, 95%CI 0.1-0.5), dyspnea (0.2, 95%CI 0.1-0.4), and hemoptysis (0.1, 95%CI 0.0-0.3), which are all commonly associated with nebulized therapies and an underlying diagnosis of NCFBE. Topline ITT efficacy results from the trial demonstrated that ARINA-1 improved quality of life in individuals with NCFBE at day 28. The mean change in SGRQ (MCID, -4) in the ARINA-1 arm from baseline to day 28 exceeded the MCID for the SGRQ in the total score (-8.5) and all three domains (symptoms, -9.6; activity, -8.5; impact, -7.9). This improvement persisted in the total score persisted to day 56 (-6.1), 28 days after stopping therapy. This demonstrates that ARINA-1 not only improved quality of life during treatment, but that it also had a durable effect in these patients after stopping ARINA-1. The placebo group did not achieve an MCID in the total score or any domain of the SGRQ at days 28 (total score, -2.6) or 56 (total score, -1.4). Consistent with the SGRQ, the CAAT (MCID, -2), which is a validated assessment specific for mucus and cough symptoms, also demonstrated MCID improvements in mean change from baseline to day 28 (-3.7). The placebo group did not reach MCID for the CAAT questionnaire (day 28: -1.3). The topline exploratory analysis of sputum data indicated that ARINA-1 demonstrated improved mucus viscosity and sputum neutrophil elastase (NE) compared to placebo. Further analyses on the mechanism of action and biomarker data are ongoing. "ARINA-1’s novel mechanism improved quality of life and also improved mucus biomarkers in people with bronchiectasis," said Carolyn Durham, PhD, Chief Scientific Officer of Renovion. " The quality of life data confirms that improving mucus biomarkers, such as viscosity and NE, can reduce symptoms and also improve patient outcomes in this underserved patient population. These data provide a strong rationale for the continued development of ARINA-1 for NCFBE patients, as well as other populations that suffer from mucus symptoms. We look forward to further analyzing the data and discussing next steps with regulatory authorities." "These results show that ARINA-1 can have a profound benefit for patients. There are currently no approved therapies for bronchiectasis, and to see such a clear impact on symptom reduction with our benign safety pro ARINA-1 as an excellent first line therapy for patients. Better SGRQ scores are associated with better outcomes, and we believe that a similar benefit will be seen in chronic bronchitis patients via a study that is already underway, as well as in COPD and asthma patients with mucus," stated Dan Copeland, CEO of Renovion. "Millions of patients with these diseases face the cycle of mucus, inflammation and infection, and we can change the course of disease for patients facing the daily challenges of mucus." Renovion will discuss study results at the American Thoracic Society Meeting (May 17-22) and present findings at the World Bronchiectasis Conference (July 4-6) in Dundee, Scotland. About CLIMB CLIMB (RVN-301) was a randomized, double-blind, placebo-controlled, multi-center, US Phase 2 study to assess the safety, efficacy, and tolerability of ARINA-1 administered twice daily for 28 days in patients with non-cystic fibrosis bronchiectasis (NCFBE). CLIMB was conducted at 9 sites with 40 adult patients diagnosed with NCFBE who indicated that they had mucus symptoms at the time of screening. Patients were randomized 7:3 to receive either ARINA-1 or matching placebo. The primary endpoint was safety at day 28. Secondary endpoints included quality of life measurements at days 28 and 56, and sputum viscosity and neutrophil elastase were measured at day 28. About Non-Cystic Fibrosis Bronchiectasis (NCFBE) NCFBE is a severe, chronic inflammatory airways disease characterized by a chronic cycle of mucus, inflammation, and infection that causes permanent bronchial dilation. Most individuals with NCFBE experience severe and life-altering mucus symptoms including shortness of breath, productive and chronic cough, and daily activity impairment, as well as frequent exacerbations that require aggressive antibiotic therapy and / or hospitalization. Individuals experience progressive health and clinical decline. To date, there are no approved treatments that directly target mucociliary clearance or mucus symptoms. There are approximately 500,000 individuals living with NCFBE in the US today. About Renovion, Inc. Renovion is a clinical-stage pharmaceutical company focused on developing ARINA-1, novel nebulized therapy delivered to the lungs to treat pulmonary diseases. ARINA-1 clears mucus, reduces damaging inflammation, and has antibacterial properties that have been shown to restore lung health. Renovion is also conducting clinical trials for ARINA-1 for the prevention of Bronchiolitis Obliterans Syndrome (BOS) progression in lung transplant patients in an ongoing Phase 3 trial with a primary endpoint of FEV1 improvement compared to standard of care. Renovion is also conducting an investigational study in patients with chronic bronchitis. The Renovion team has in-depth experience in drug development with extensive scientific expertise in chronic pulmonary conditions and inflammation. For more information about Renovion, visit or follow Renovion on LinkedIn (Renovion, Inc.) and Twitter (@RenovionInc).

Feb. 15, 2024 -- OrphAI Therapeutics Inc. (“OrphAI”), a clinical-stage biopharmaceutical company developing novel therapeutics for rare diseases, announced today that it has received Orphan Drug Designation (ODD) in the European Union (EU) for the treatment of amyotrophic lateral sclerosis (ALS). AIT-101 previously received ODD from the U.S. FDA in June 2023 for the treatment of ALS. “OrphAI Therapeutics now has orphan designation for AIT-101 both in the US and the EU for the treatment of ALS, an important step toward bringing the drug to patients suffering from this neurodegenerative disorder,” said Dr. Brigette Roberts, CEO and Director of OrphAI. “AIT-101 has demonstrated compelling activity in its ability to clear toxic aggregates, which are a hallmark of ALS and other neurodegenerative diseases. We look forward to moving AIT-101 into the next stage of clinical development." AIT-101 is a first-in-class, potent and highly selective inhibitor of the lipid kinase PIKfyve. Inhibition of PIKfyve leads to activation of the transcription factor TFEB which drives the increased clearance of toxic protein aggregates via the lysosomal autophagy pathway. AIT-101 has now been demonstrated to reduce aggregates in human subjects, to improve the survival of motor neurons in in vitro models of familial and sporadic ALS, and to ameliorate functional deficits in a mutant TDP-43 animal model of ALS. AIT-101 is the most clinically advanced PIKfyve inhibitor in development. OrphAI Therapeutics’ mission is to transform the lives of patients with rare diseases. The company is currently developing three investigational therapies across multiple orphan indications: AIT-101, a first-in-class PIKfyve inhibitor, which recently completed a Phase 2a clinical trial in amyotrophic lateral sclerosis (ALS); LAM-001, a proprietary inhaled form of rapamycin, currently in Phase 2 for Group 1 and 3 pulmonary hypertension and bronchiolitis obliterans syndrome (BOS); and AIT-102, a proprietary analogue of mithramycin, in preclinical development, for the treatment of SWI/SNF mutated or dysregulated tumors. The content above comes from the network. if any infringement, please contact us to modify.