Arrowhead Pharmaceuticals’ Post

Promising results from ASC4FIRST trial. #Asciminib showed superior efficacy and a favorable safety profile in patients with newly diagnosed chronic phase #CML In this phase 3 trial, Asciminib was compared to investigator choice #TKI as a de novo treatment or after imatinib. Major molecular response at 48 weeks were recorded and Asciminib showed higher rates (67% vs 49%, P <0.001). Safety profile was also significantly better. https://lnkd.in/dsG_7R9b

Will you be at #ARVO2024? Let’s connect!   Join me for one of the many poster presentations featuring new and updated data demonstrating the potential efficacy of our lead drug candidate for Meibomian Gland Dysfunction (MGD). Session details are in the link below. 

Ahead of #JPM24 Week, Celltelligence is publishing a series of 2 blasts with the most important #celltherapy-related news items that occurred during the holidays. In this second blast, the following news are covered: • On Thursday, December 21, 2023, #Legend shared that the FDA (SEC filing: https://bit.ly/3S7PMhl) approved a label update for #Carvykti (#JNJ / Legend’s #BCMA CAR-T) including longer-term safety and efficacy information from the Ph1/2 CARTITUDE-1 trial (https://bit.ly/490CkTh), which incorporated the risk of secondary hematological malignancies to the Boxed Warning • On the same day, #Gilead announced (press release: https://bit.ly/3vp4FUF) that the FDA approved a label update for #Yescarta (#CD19 CAR-T) including the significant OS benefit induced by the CAR-T in ≥2L LBCL in the Ph3 ZUMA-7 trial (https://bit.ly/3H64O1V) • On Tuesday, December 19, #Galapagos announced (press release: https://bit.ly/3H6gula) that the Ph1/2 #PAPILIO trial evaluating #GLPG5301 (BCMA CAR-T) in ≥3L #MM has started recruiting patients in Europe Celltelligence provides insights and context for each news story: https://bit.ly/CT-845902

In ERA 2024's best-ranked abstract, we presented 72-week eGFR stabilization from our Phase 2b clinical trial for #IgANephropathy. We also showed rapid hematuria improvement and significantly higher hematuria resolution compared to placebo during the 36-week randomized period. Read the press release here: https://lnkd.in/eRcDMqd5 #ERA24

New study uncovers how GPCRs, particularly the vasopressin V2 receptor (V2R), continue to signal from internalized compartments, highlighting the role of βarrestin (βarr) and Gβγ in non-canonical endosomal signaling. Discover how these mechanisms can pave the way for innovative therapeutic strategies. Check out the latest GPCR news in the Ecosystem today! You’ll need to register, but don’t worry, it’s Free! ➡️https://bit.ly/4d9yUQ3 #GPCR #DrGPCR

New Article by Cort Johnson, ‘Looking Back at 2023 in ME/CFS, Long COVID and FM- the Most, Best, Cleverest and Strangest,’ has highlighted two remarkable OMF initiatives. 🌍 OMF’s StudyME project, a global participant registry connecting those interested in participating in research studies with the researchers conducting them, has been named the ‘Cleverest Idea.’ 💊 The Life Improvement Trial (LIFT), a double-blind placebo trial that will investigate two drugs: Pyridostigmine (commonly known as Mestinon) and low-dose naltrexone (LDN) separately and together as a combination, has been recognized as the ‘Best New ME/CFS Clinical Trial.’ Read the full article here 👉 https://ow.ly/t3Os50QnBRe.

We're proud to share that JAMA Neurol has published the results of batoclimab (HBM9161) phase 3 clinical trial, which examined the efficacy and safety of batoclimab, a fully human anti-FcRn mAb, in patients with generalized myasthenia gravis (gMG).   In this randomized clinical trial, batoclimab demonstrated sustained efficacy evaluated by MG-ADL (Myasthenia Gravis Activities of Daily Living), QMG (Quantitative Myasthenia Gravis) and other outcome measures. The results also revealed that batoclimab was safe and well tolerated in gMG patients.   With these groundbreaking data, batoclimab is expected to set a new benchmark for the treatment of gMG and bring benefit to patients in need.   Read more: https://lnkd.in/g35nqKf4 #gMG #HarbourBioMed

New hope for MDM2 inibition in liposarcoma? An early-phase trial presented at #ESMOSarcomaAndRareCancers24 shows the efficacy of brigimadlin in dedifferentiated and well-differentiated liposarcomas, but confirmation from phase III results is still awaited.  To learn more about this topic, read the #ESMODailyReporter article 👉https://ow.ly/tKgi50QUcMG

Excited with the announcement of #deucrictibant’s Phase 2 results in the #prevention of #HAE attacks. These data indicate that #deucrictibant could become the first HAE treatment to combine:   - Injectable-like efficacy and - A favorable safety profile - With the convenience of an oral therapy   A sincere thank you to all study participants and sites to make this happen!   Based on the results of #RAPIDe1 last year and #CHAPTER1 today, #deucrictibant has the potential to become the preferred option for #peoplelivingwithHAE to #treatandprevent #HAE attacks.

Have you heard of the Joint Access Advisory Mechanism (JAAM)? In this interview, Sanjay Bhagani, JAAM Chair, and Jacques Demotes Mainard, ECRIN Director General, discuss the origins of this mechanism to prioritise the testing of therapeutics during the COVID-19 pandemic through to its evolution in 2023.  🙌 This mechanism unites various EU funded projects including RECOVER EUROPE (REMAP-CAP), EU-RESPONSE (DisCovERY & #EUSolidACT), Ecraid Prime, and now CoMeCT project.  🤝 👉 Learn about the JAAM and how it supports clinical research: https://lnkd.in/eS-gPy9N