#BreakingNews: In one of the most highly anticipated #clinicaltrial readouts this year, an #RNAsilencing treatment developed by Alnylam Pharmaceuticals reduced the risk of death, heart-related hospital visits and hospitalizations by 28% compared to placebo in patients with a #heartmuscledisease. The results, announced Monday, pave the way for #Alnylam’s therapy #vutrisiran to become the first #RNAsilencingtherapy on the market for #ATTRamyloidosis with #cardiomyopathy, a condition in which misfolded proteins build up in the heart and can result in heart failure. If approved, Alnylam could enter a multibillion-dollar market currently dominated by Pfizer. Vutrisiran is already approved as Amvuttra for ATTR amyloidosis with polyneuropathy, where the misfolded proteins affect the nerves. Alnylam believes that it can potentially reach 10 times more patients in the expanded indication, as the heart manifestation of the disease has been estimated to impact as many as 500,000 globally. “We believe with the data we have in hand, this will become the new standard of care in patients with ATTR cardiomyopathy,” Alnylam CEO Dr. Yvonne Greenstreet told Endpoints News on Saturday. “This is really, really tremendous data and clearly a huge opportunity for Alnylam.” Read more from Endpoints News 👇🏼
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Today, AbbVie announced positive top-line results from their Phase 3 study, SELECT-GCA. Upadacitinib (RINVOQ, 15 mg, once daily), paired with a 26-week steroid taper regimen, achieved its primary endpoint of sustained remission from week 12 through week 52 in adults with giant cell arteritis (GCA) with the safety profile remaining consistent with that in approved indications. Key secondary endpoints were also met, with a larger percentage of patients receiving Upadacitinib with the 26-week steroid taper regiment achieving sustained complete remission compared to placebo, and a lower percentage of patients experienced at least one disease flare through week 52 than placebo. However, the study showed that Upadacitinib 7.5 mg did not meet the primary or any of the secondary endpoints. Read more: https://lnkd.in/ejmhxSRF #GiantCellArteritis #GCA #Rheumatology #AbbVie
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Today, we presented first-time clinical data from the Phase 1 study of trispecific TREAT™ antibody, ISB 2001, showing high overall response rate (ORR), with high response rate and favorable safety profile in patients with heavily pretreated relapsed or refractory multiple myeloma (RRMM). Notably, no dose-limited toxicities were observed at weekly subcutaneous doses up to 1.2 mg/kg, with mostly mild Cytokine Release Syndrome, no cases of neurotoxicity, and no discontinuations due to adverse events. Early results showed an impressive ORR of 83% in heavily pretreated patients treated at active doses (50 µg/kg and above). Read the full release: https://bit.ly/3OKDn1v #ASH24
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https://bit.ly/45sSPGQ AL Amyloidosis is a blood desease that is crucial the early diagnosis. Before those patients werent treated whith the same dugs as MM ( Myeloma Múltiple), and nowadays we can afford only one approuved drug, in some countries whith difficulties to get out. Prothena is leading for a drug in Pase III giving to us one more option for ouers patients. #AL #ClinicalTrials #Prothena #SomosAmilo www.amilo.es
AL Amyloidosis
https://www.prothena.com
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Delighted to share our first application of virtual twins with liquid biopsy for precision dosing! #CPT, read the article here: doi.org/10.1002/cpt.3291 👥 Virtual Twin models for individual patients generated by customizing base population PBPK models, utilizing Simcyp, to predict exposure (AUC) of oral midazolam in patients with different grades of renal impairment, enabled model-informed precision dosing, with significant consequences for dose selection/adjustment for individual patients at the point of care!
Dealing With Variable Drug Exposure Due to Variable Hepatic Metabolism: A Proof‐of‐Concept Application of Liquid Biopsy in Renal Impairment
ascpt.onlinelibrary.wiley.com
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Machine Learning Model Accurately Predicts Intravenous Immunoglobulin Non-Responders For Kawasaki Disease: By developing the Shizuoka score, which features a new predictive model using machine learning, patients with Kawasaki disease who are non-responders to intravenous immunoglobulin were able to be accurately identified. #finance #pharmacy #lifesciences
Machine Learning Model Accurately Predicts Intravenous Immunoglobulin Non-Responders For Kawasaki Disease
pharmacytimes.com
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In 1L G/GEJC, Osemitamab plus Nivolumab /Chemotherapy produced Claudin18.2 expression dependent increase in both objective response rate and medium progression free survival, regardless of PD-L1 status, as we presented at ASCO 2024 (abstract 4048). This is very encouraging news to first-line G/GEJC patients with Claudin18.2 expression!!
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Today we announced the publication of a paper in American Society for Microbiology's Antimicrobial Agents and Chemotherapy. The paper focuses on our Phase 1 #ClinicalTrial evaluating lung pharmacokinetics of our drug candidate, SPR720. SPR720 is an oral candidate for the treatment of nontuberculous mycobacterium pulmonary disease (#NTMPD). Learn more and access the publication here: https://bit.ly/3TSpEZe #InfectiousDiseases
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We are pleased to announce that we have activated clinical sites and are initiating enrollment for the Phase 1 trial of ADI-001 in autoimmune diseases! This follows the U.S. Food and Drug Administration’s (FDA) decision to grant Fast Track Designation to ADI-001 for the treatment of relapsed/refractory class III or class IV #lupus nephritis (LN) and clearance from FDA to develop ADI-001 in four #autoimmune indications including LN, systemic lupus erythematosus (#SLE), systemic #sclerosis (SSc) and anti-neutrophil cytoplasmic autoantibody (ANCA)-associated #vasculitis (AAV). With its safety profile, robust tissue trafficking and complete B cell depletion in peripheral blood and secondary lymphoid tissue (characteristics important in autoimmune treatment), ADI-001 is uniquely differentiated with the goal to bring durable remissions and provide an immune reset for various autoimmune conditions. We are inspired by the idea of bringing to #patients a potential paradigm shift in the treatment of autoimmune diseases. To learn more about participating in this #clinicaltrial, visit https://lnkd.in/esC4winc or email clinicaltrials@adicetbio.com. #CART #celltherapy #gammadelta #clinicaltrials #autoimmune #autoimmuneclinicaltrials #biotech #biotechinvesting #biotechresearch #allogeneic #offtheshelf
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MASH is the newest area of intervention in metabolic health. Earlier this year, Rezdiffra (resmetirom) was launched, but at $48K a year and with very minimal results, there is still a lot to be desired. #GLP1 are also under the microscope in this space. Lilly released information on their trial this past week- https://lnkd.in/gT88XYuD And more novel therapies to come: https://lnkd.in/gKKQ84h7 https://lnkd.in/gkCZAfkU Lots to watch in this space over the next few years! #Pharmacists #Pharmacybenefits #liver #newdrugapproval #GLP1
Lilly touts fibrosis win as it pads case for tirzepatide in MASH
fiercepharma.com
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#ACR24 Abstracts Now Available: Efficacy, safety, and pharmacokinetics of anti-CD40 antibody in patients with Rheumatoid Arthritis: A phase 2, randomized, placebo-controlled 12-week-treatment proof-of-concept study. View here: https://bit.ly/3YLmvNM
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