Arrowhead Pharmaceuticals’ Post

It was a pleasure to co-author a new paper forthcoming in the Journal of cystic fibrosis. It is now up on their website, link below. In this paper, Heching et al. show that in people with #CF who carry the splicing, residual function CFTR variant 3849+10kB C>T, elextacaftor+tezacaftor+ivacaftor (ETI) treatment (although not approved for this variant) has benefits on top of single or double modulators. Switching to ETI improved lung function and sweat chloride concentration in nine Israeli patients carrying this variant. Link to publication: https://lnkd.in/e3tZnpSu Moshe Heching, Inbal Golan-Tripto, Galit Livnat-Levanon, Karin Yaacoby-Bianu Liora Boehm Cohen, Guy Hazan, Liora Slomianski, Dario Prais, Huda Georgy Mussaffi, Joel Weinberg Mordechai R Kramer

Today, we are thrilled to announce that we have initiated our MIST Study – a Phase 2b clinical trial for AP01, inhaled pirfenidone treatment for patients with progressive pulmonary fibrosis (PPF), a rare and life-threatening lung disease. Our vision at Avalyn is to bring innovative solutions to people with rare respiratory diseases, including pulmonary fibrosis and other interstitial lung diseases. We believe inhaled delivery of the medications that we know work has the potential to make those treatments both more effective and safer for patients. Our flagship candidate, AP01, represents not just medical progress, but hope – a chance to significantly improve the lives of patients grappling with PPF, a condition with limited treatment options today, and a high burden on both quality of life and life expectancy. We are driven by this mission and inspired to make this medicine available in a new clinical trial to patients worldwide that desperately need better therapeutic options. Read the press release here: https://lnkd.in/ejFXG3Qn For more information about the MIST Study, visit our website: MISTPPFStudy.com #PulmonaryFibrosis #ProgressivePulmonaryFibrosis #PFMonth #ClinicalTrials #BiotechInnovation #Biotech

Today, Ocugen announced positive preliminary efficacy and safety data from the Phase 1 dose-escalation portion of the Phase 1/2 OCU410 ArMaDa clinical trial for geographic atrophy.  https://bit.ly/4fWAeXF The preliminary OCU410 data, along with data from the Phase 1/2 OCU410ST GARDian clinical trial for Stargardt disease and data on Leber congenital amaurosis (LCA) from the Phase 1/2 OCU400 clinical trial were presented during last week’s clinical showcase, which is now available on the Ocugen website https://bit.ly/490Nmc0

It was very knowledgeable attending the #IFCC live webinar "Troponin assays with higher clinical specificity; a novel future option". This webinar described the clinical need and analytical possibilities for creating #troponin assays with improved specificity. the webinar objectives included; The current clinical utility and performance of #cardiactroponins for diagnosing #acutecardiacdisease; Suggestions how to improve the clinical specificity when acute cardiac disease is investigated; Discussion of future troponin assays measuring different molecular forms of troponins, and how these may improve the clinical specificity of troponin measurements.

Learn more about clinical trials in Europe for myeloma and AL amyloidosis patients by using our Navigator tool. You can find trials matching your criteria, from specific hospitals to disease stages. Explore the MPE Navigator now: https://lnkd.in/dDRHsv2Z #ClinicalTrials #myeloma #ALAmyloidosis #MPENavigator

Discover clinical trials in Europe for myeloma and AL amyloidosis patients using our Navigator tool. Locate trials matching your criteria, from specific hospitals to disease stages. Explore the MPE Navigator now: https://lnkd.in/dDRHsv2Z #ClinicalTrials #Research #MyelomaAwareness #AmyloidosisAwareness

👁️ Exciting news from our portfolio company, Oculis! They announced positive Phase 2b RELIEF trial results for Licaminlimab/OCS-02, a breakthrough in treating Dry Eye Disease with precision medicine. -Significant efficacy improvements -TNFR1 biomarker patients showed a 7x higher symptom response and 5x higher sign response, proving the power of personalised treatment. These results will help them to accelerate Licaminlimab/OCS-02 development. Read the full article in the comments 👇 #Oculis #DryEyeDisease #PrecisionMedicine #HealthcareInnovation

🌟 𝐒𝐞𝐩𝐭𝐞𝐦𝐛𝐞𝐫 𝐢𝐬 𝐏𝐮𝐥𝐦𝐨𝐧𝐚𝐫𝐲 𝐅𝐢𝐛𝐫𝐨𝐬𝐢𝐬 𝐀𝐰𝐚𝐫𝐞𝐧𝐞𝐬𝐬 𝐌𝐨𝐧𝐭𝐡 🌟 This is a great opportunity to spotlight Noxon, one of our innovative portfolio companies dedicated to changing the treatment landscape for idiopathic pulmonary fibrosis (IPF). Noxon is advancing the fight against IPF with their novel drug, 3-cabamoyl-proxyl (3-CP), which targets fibrotic lung diseases with its unique antioxidant properties. This promising treatment aims to reduce inflammation and fibrosis, offering new hope for patients who face a median survival of just 2-3 years post-diagnosis. Next week, Firas Younis, the CEO of Noxon, will be attending the European Respiratory Society (ERS) conference in Vienna, one of the largest conferences for respiratory diseases. This marks a pivotal milestone as Noxon is achieving efficacy in animal models using the currently developed formulation for the oral slow release of the 3-CP API product. Pulmonary Fibrosis Awareness Month helps raise awareness about this serious condition and supports the ongoing search for more effective treatments. Discover how Noxon is making strides in this crucial area. Learn more about Noxon and their pioneering efforts in the battle against pulmonary fibrosis> https://lnkd.in/dBnA9xBE #PulmonaryFibrosis #PFMonth #InnovativeTreatment #Healthcare

The Temple Lung Center leads the way in advancing IPF treatment. The Fibroneer clinical trial marks a significant milestone in the fight against idiopathic pulmonary fibrosis (IPF). As the first Phase III IPF trial to meet its primary endpoint—measuring lung function based on absolute change from baseline in Forced Vital Capacity (FVC) at 52 weeks—this achievement represents a breakthrough in treatment options for patients. Temple Health is proud to have been the #1 enroller in the United States for this critical trial, contributing to the progress of Nerandomilast, a potential life-changing therapy for those with IPF. Our involvement underscores the Temple Lung Center's dedication to advancing interstitial lung disease research and delivering new hope to patients. Learn more about the Temple Lung Center's role in this achievement and our ongoing commitment to innovation in pulmonary care. https://bit.ly/4fuL3zH

It's time for #MedicationEducation where we highlight different drugs and services #ContinuumRx uses to treat patients from home or in one of our conveniently located infusion clinics. This week, we're focusing on IV antibiotics for cystic fibrosis (CF). CF, a genetic disease, leads to persistent lung infections and breathing difficulties due to an abnormality in a protein called cystic fibrosis transmembrane conductance regulator (CFTR). Besides affecting the lungs, CF can impact other organs and bodily functions. To combat infections, CF patients often need IV antibiotics such as nafcillin, oxacillin, and piperacillin. While patients traditionally receive this treatment in a hospital or their doctor's office, choosing ContinuumRx allows them the convenience of receiving treatment at home or at one of our nearby infusion suites, saving both time and money. Contact us to learn more! https://bit.ly/42hLLtL #ContinuumRx #CysticFibrosis #CF #IVAntibiotics #PatientCare #InfusionTherapy #HomeInfusion #patientsfirst Cystic Fibrosis Foundation Boomer Esiason Foundation Cystic Fibrosis Research Institute CysticFibrosis.com