RA Capital Management’s Post

Did you know metabolic dysfunction-associated steatohepatitis (MASH) is caused by an excess of fat cells in the liver? Our drug candidate for MASH, entering Phase 2a clinical trials this year, has the potential to be best-in-class based on recent data we presented. Learn more here: https://brnw.ch/21wHuxk

We are evaluating the effectiveness of our investigational oral therapy for #ThyroidEyeDisease (TED) in a Phase 2b/3 clinical trial. Learn more about LIDS: https://lnkd.in/e4xE8anj

Phase III KEYNOTE-811 trial met dual primary endpoint of overall survival as first-line treatment in patients with HER2-positive advanced gastric or gastroesophageal junction adenocarcinoma - Merck https://lnkd.in/e57G-_XC

Novo Nordisk’s semaglutide has achieved positive outcomes in a Phase 3 trial for metabolic dysfunction-associated steatohepatitis (previously known as nonalcoholic steatohepatitis).   The Essence trial demonstrated significant improvements in liver fibrosis and resolution of steatohepatitis, successfully meeting primary endpoints. With this breakthrough, Novo Nordisk aims to file for regulatory approval in the U.S. and Europe by 2025.   These promising results highlight the expanding role of GLP-1 therapies in many indications, including this complex field, which has limited treatment options.   Read more here: https://lnkd.in/ed5bvzDk   Find the clinical evidence for semaglutide on DRUGDOCS®: https://lnkd.in/gjy4C8QW   #MASH #NAFLD #GLP1 #LiverDisease

Blood transfusions are a life-saving #treatment for a range of conditions, yet individuals who regularly need #bloodtransfusions may develop systemic transfusional iron overload (#TIO) due to the body's inability to remove excess #iron. SP-420, an #innovativedrug from a novel class of orally administered iron chelators known as desferrithiocins, has successfully completed three Phase 1 #clinicaltrials to establish safety, with promising results, including high tissue penetration in critical organs. This drug holds the potential to be a safer and more effective treatment alternative for #ironoverload due to its excellent oral availability and enhanced tolerance. Developed by the Denmark-based Pharmacosmos, SP-420 is being explored in a Phase II clinical trial (THAL-01) for adults with transfusion-dependent ß-#thalassemia who are at risk of iron overload. For further details on SP-420 and updates on the THAL-01 trial, please visit: https://lnkd.in/exUKPCvV

We are pleased to share positive interim results from our Phase 1 clinical study of VQ-101, a small molecule allosteric activator of GCase. In healthy volunteers in Phase 1a, VQ-101 demonstrated good tolerability, robust lysosomal GCase activation, and pharmacokinetics consistent with once daily dosing. The study, conducted at Centre for Human Drug Research, has advanced to the Phase 1b portion in patients with Parkinson’s disease. https://lnkd.in/eqRzbQct

Roche to Present Data from the P-III (OCARINA II) Clinical Evaluation of Ocrevus to Treat Progressive and Relapsing Multiple Sclerosis #roche #ocrevus #multiplesclerosis #clinicaltrial #phase3 #treatment #safety #efficacy #progressive #relapse #pharmacokinetics #usfda #ema #ocarina2

In an interview with The Clinical Trial Vanguard’s Moe Alsumidaie, Elysium Therapeutics, Inc. CEO Greg Sturmer discusses O2P, a groundbreaking path to #painrelief that’s designed to help reduce the risk of opioid misuse. #opioidoverdose #opioidepidemic #painmedication #opioidmisuse #elysiumtherapeutics #hydrocodone https://lnkd.in/eUnapmfJ

IGC PHARMA announces promising #preclinical data for TGR-63, our innovative treatment for #AlzheimersDisease. Preliminary analyses confirm TGR-63’s ability to cross the blood-brain barrier and its excellent safety profile, with no adverse effects observed in extended studies. This marks a significant step forward in our mission to combat #Alzheimer's. Key highlights: • TGR-63 penetrates the blood-brain barrier, a critical milestone in #Alzheimer’s #treatment. • Eight-month safety studies in mice show no toxicity or adverse effects. • Validated potential to reduce #amyloid plaque buildup. Read more at https://lnkd.in/ePcj3HfF   #AlzheimersResearch #IGCPharma #PreclinicalData #ClinicalTrials #BloodBrainBarrier #AlzheimersTreatment #TGR63

Each year in the United States, 185,000 individuals experience recurrent strokes. (1) 😱 Let's not just scroll past this number. Let's take action! Spread awareness, learn the warning signs, and prioritize preventive care. Every step counts in the fight against strokes! 💪💙 Find out how the rapid Genomadix CYP2C19 test can help optimize DAPT treatment to reduce recurrent stroke rates at www.genomadixcom (2,3)🧬#StrokeAwareness #PersonalizedMedicine #Pharmacogenetics 1. Kleindorfer DO, et al. Stroke. 2021;52:e364–e467. 2. Wang Y, et al. N Engl J Med. 2021;385:2520-2530. 3. Lee CR, et al. J Clinical Pharmacol Therapeutics. 2022;122(5):959-967.