Bohring Syndrome

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.

Seasoned leader in drug discovery and developmentGUILFORD, Conn., Jan. 24, 2024 (GLOBE NEWSWIRE) -- OrphAI Therapeutics Inc. (“OrphAI”), a clinical-stage biopharmaceutical company developing novel therapeutics for rare diseases, announced today the appointment of Jay S. Fine, Ph.D. to its Board of Directors. Dr. Fine has a strong track record of drug discovery and development leadership in respiratory diseases, oncology, immunology and fibrotic disease indications. His leadership has contributed to the progression of over 30 new molecular entities into clinical development, leading to the commercial launch of multiple products including Skyrizi™ for Plaque Psoriasis, Psoriatic Arthritis and Crohn’s Disease and Spevigo™ for Generalized Pustular Psoriasis. He has chaired various governance and steering committees at several global pharmaceutical companies. Dr. Fine currently serves as President of Research and Development at EvolveImmune Therapeutics, a development-stage company focused on advancing differentiated immunotherapeutic approaches for the treatment of cancer. Previously, he served as Senior Vice President and Global Therapeutic Area Head for Immunology and Respiratory Diseases Research at Boehringer Ingelheim and as the Discovery Research Site Head at their Ridgefield, CT location. Dr. Fine also served as Head of Translational Biology at Roche Pharmaceuticals, was a Director in the Inflammation department at Schering-Plough and worked in the Biologic Response Modifiers Program at the National Cancer Institute. He holds a B.S. degree from Cornell University and a Ph.D. from the University of Rochester School of Medicine. Dr. Fine has authored over 75 scientific publications and patents and has been the recipient of numerous honors and awards during his career. “Jay Fine represents an extraordinary resource both for the Board as well as for the OrphAI management team, given his years of experience and success in the operational leadership of drug discovery and development. I am personally excited to work with him in this capacity,” said Dr. Brigette Roberts, CEO and Director of OrphAI. “I am very pleased to join the OrphAI Therapeutics’ Board of Directors,” said Dr. Fine. “The company has established an exciting product pipeline which holds tremendous potential to meaningfully improve the lives of patients with rare diseases. I look forward to working closely with the OrphAI team and its board to accelerate the progress of its promising pipeline.” “We are excited to welcome Jay Fine to our Board of Directors,” said David Scheer, Chairman of the Board of Directors at OrphAI. “Jay’s extensive leadership experience and drug development insights will be especially valuable to OrphAI and our Board as we collectively embark on this next stage of the company’s growth.” About OrphAI Therapeutics 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 bronchiolitis obliterans syndrome (BOS) and pulmonary arterial hypertension (PAH); and AIT-102, a proprietary analogue of mithramycin, in preclinical development for the treatment of SWI/SNF mutated or dysregulated tumors. To learn more, please visit: OrphAI-Therapeutics.com

DUBLIN, Dec. 7, 2023 /PRNewswire/ -- The "Itacitinib Emerging Drug Insight and Market Forecast - 2032" report has been added to ResearchAndMarkets.com's offering. This report provides comprehensive insights about itacitinib for Bronchiolitis Obliterans Syndrome (BOS) in the seven major markets. A detailed picture of the itacitinib for bronchiolitis obliterans syndrome in the 7MM, i.e., the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan for the study period 2019 -2032 is provided in this report along with a detailed description of the itacitinib for bronchiolitis obliterans syndrome. The report provides insights about mechanism of action, dosage and administration, as well as research and development including regulatory milestones, along with other developmental activities. Further, it also consists of future market assessments inclusive of the itacitinib market forecast analysis for bronchiolitis obliterans syndrome in the 7MM, SWOT, analysts' views, comprehensive overview of market competitors, and brief about other emerging therapies in bronchiolitis obliterans syndrome. Drug Summary Itacitinib (also known as INCB-039110) is an orally bioavailable inhibitor of Janus-associated kinase 1 (JAK1) with potential antineoplastic and immunomodulating activities. Upon oral administration, itacitinib selectively inhibits JAK-1, thereby inhibiting the phosphorylation of signal transducer and activator of transcription (STAT) proteins and the production of pro-inflammatory factors induced by other cytokines, including interleukin-23 (IL-23) and interleukin-6 (IL-6). The JAK-STAT pathway plays a key role in the signaling of many cytokines and growth factors and is involved in cellular proliferation, growth, hematopoiesis, and the immune response; JAK kinases may be upregulated in inflammatory diseases, myeloproliferative disorders, and various malignancies. Itacitinib Analytical Perspective In-depth Itacitinib Market Assessment This report provides a detailed market assessment of itacitinib for bronchiolitis obliterans syndrome in the seven major markets, i.e., the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan. This segment of the report provides forecasted sales data from 2031 to 2032. Itacitinib Clinical Assessment The report provides the clinical trials information of itacitinib for bronchiolitis obliterans syndrome covering trial interventions, trial conditions, trial status, start and completion dates. Report Highlights In the coming years, the market scenario for bronchiolitis obliterans syndrome is set to change due to the extensive research and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market. The companies are developing therapies that focus on novel approaches to treat/improve the disease condition, assess challenges, and seek opportunities that could influence itacitinib dominance. Other emerging products for bronchiolitis obliterans syndrome are expected to give tough market competition to itacitinib and launch of late-stage emerging therapies in the near future will significantly impact the market. A detailed description of regulatory milestones, and developmental activities, provide the current development scenario of itacitinib in bronchiolitis obliterans syndrome. This in-depth analysis of the forecasted sales data of itacitinib from 2031 to 2032 will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the itacitinib in bronchiolitis obliterans syndrome. Key Topics Covered: 1. Report Introduction 2. Itacitinib Overview in Bronchiolitis Obliterans Syndrome 2.1. Product Detail 2.2. Clinical Development 2.2.1. Clinical studies 2.2.2. Clinical trials information 2.2.3. Safety and efficacy 2.3. Product Profile 3. Competitive Landscape (Late-stage Emerging Therapies) 4. Itacitinib Market Assessment 4.1. Market Outlook of Itacitinib in Bronchiolitis Obliterans Syndrome 4.2. 7MM Analysis 4.2.1. Market Size of Itacitinib in the 7MM for Bronchiolitis Obliterans Syndrome 4.3. Country-wise Market Analysis 4.3.1. Market Size of Itacitinib in the United States for Bronchiolitis Obliterans Syndrome 4.3.2. Market Size of Itacitinib in Germany for Bronchiolitis Obliterans Syndrome 4.3.3. Market Size of Itacitinib in France for Bronchiolitis Obliterans Syndrome 4.3.4. Market Size of Itacitinib in Italy for Bronchiolitis Obliterans Syndrome 4.3.5. Market Size of Itacitinib in Spain for Bronchiolitis Obliterans Syndrome 4.3.6. Market Size of Itacitinib in the United Kingdom for Bronchiolitis Obliterans Syndrome 4.3.7. Market Size of Itacitinib in Japan for Bronchiolitis Obliterans Syndrome 5. SWOT Analysis 6. Analysts' Views 7. Appendix For more information about this report visit About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. 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