Atsena Therapeutics’ Post

Phase 2 clinical trial update on NETs! 📊 Lenvatinib and pembrolizumab combo in advanced GI and thoracic NETs showed 10% partial response, with median PFS of 8 months. Also, 60% experienced grade 3 adverse events, leading to dose adjustments or discontinuation. The trial did not show sufficient response to be continued. More details in the article: https://ow.ly/hwrG50R0Vbj #MedEd

🔬 A recent study performed a review and meta-analysis of previously gathered clinical trial data to compare 1st line chemotherapy for advanced pancreatic ductal adenocarcinoma with other treatment options for #PDAC. 👉 Learn more about the efficacy and toxicity of various treatment options: https://buff.ly/40XY1SR #PancreaticCancerAwareness

Human Clinical Article: This phase IIA clinical trial reports the safety and therapeutic efficacy of autologous chondroprogenitor cells derived from the nasal septum for the treatment of patients with temporomandibular joint. https://lnkd.in/d6mUJAxq

5 reasons to get tested using Carci Reagent from CNEU MEDICAL s.r.o. 1. High Sensitivity and Specificity: The Carci Reagent test has demonstrated a sensitivity of 96.7% for detecting cancer in medically diagnosed patients and a specificity of 99.4%, providing reliable and accurate results. 2. Ease of Use: Designed for home use, the Carci Reagent test allows individuals to perform the test themselves with a simple procedure, using a fresh urine sample and observing the color change, which is then compared to a provided color scale. 3. Non-Invasive Method: Unlike some other diagnostic tests, the Carci Reagent test is non-invasive as it only requires a urine sample, making it a more comfortable and less intimidating option for patients. 4. Early Detection: Early detection of cancer can significantly increase the chances of successful treatment. The Carci Reagent test can help in identifying high levels of tyrosine metabolites in the urine, which are indicative of cancer, thus allowing for timely medical intervention. 5. Validated by Research: The test has undergone extensive clinical testing and validation, including compliance with European standards (EN 13612:2002), and has been tested on over 8,000 individuals, ensuring its reliability and effectiveness. #CarciReagent #InnovationInHealth #CancerDetection #StayHealthy #EarlyDetectionMatters www.carcireagent.com

🚀We are delighted to announce encouraging early efficacy trends in the extension phase of our 🌈RAINBOW Phase 2 safety/PK trial for GM2 gangliosidosis and Niemann-Pick disease type C, ⏩ paving the way to start our Phase 3 trial. The results were presented at the #SSIEM2024 conference last week.   💈 Our 🌈RAINBOW trial was designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of nizubaglustat in patients with GM2 and NPC 💈 Data showed that our compound had a positive safety profile and was well tolerated in the 13 study participants 💈The extension phase of 🌈RAINBOW and initial, unblinded data from 10 patients showed 8 had improved or stabilized SARA (Scale for the Assessment and Rating of Ataxia) scores. Additionally, a reduction in seizure frequency (daily at baseline) was reported 💈Data from the 🌈 RAINBOW study will be used to inform the target dose for our planned Phase 3 pivotal studies, 🎯 due to be initiated in 2025 💈At SSIEM, we also presented our 12-month follow-up data from our PRONTO trial – the largest prospective natural history study in late-infantile/juvenile GM1 and GM2 gangliosides   We are immensely thankful to everyone who contributed to both our 🌈 RAINBOW and PRONTO trials.   📢 To see the full announcement, go to our website: https://lnkd.in/e4Bbcud4   💬Azafaros CEO, Stefano Portolano, MD, said: “The RAINBOW trial results support the positive safety profile of nizubaglustat and provide us with encouraging early efficacy trends for the compound. We want to thank all the physicians, patients, patient families, and patient organisations who contributed to this important study.” #Biotech, #clinicaltrials, #rarediseases, #Ataxia, #GM1, #GM2, #NPC, #nizubaglustat, #azasugar

Surprising decision at FDA to appove a new drug dedicated to #Duchenne Musculary Distrophy #DMD which failed to validate its primary endpoint. In its announcement the FDA states: "In making this decision, the FDA considered the totality of the #evidence, including the potential risks associated with the product, the life-threatening and debilitating nature of the disease and the urgent medical #unmetneeds." This is in line with the recommandation of the guidance dedicated to this disease: " "Sponsors should engage FDA early during the selection and/or development of efficacy endpoints. The sponsor should include an assessment of #multipleefficacyendpoints, when feasible, to characterize the breadth of effects on dystrophin-related pathologies, including skeletal, respiratory, and cardiac muscle function, even if the primary endpoint is only one of these measures. " Innovative drug will face more and more the need to assess #multipleoutcomes and uncover their overall #nettreatmentbenefit for the benefit of the patients https://lnkd.in/emDTbqUd

A newly presented analysis of the phase 3 ADAPT-SC study (NCT04818671) revealed that treatment with subcutaneous efgartigimod PH20 (Vyvgart Hytrulo; argenx) was safe and efficacious in patients with seronegative generalized myasthenia gravis. https://lnkd.in/eHWH_C9v

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

Observational studies to date indicate that TAVR in selected patients with bicuspid valve disease performs well, but validation in a randomized clinical trial against the current gold standard SAVR is missing. – Stephan Windecker, MD Around 10% of TAVR procedures are performed on patients with bicuspid aortic valves, yet no clinical trial data exist comparing TAVR vs. SAVR. Read "Clinical Decision-Making in Aortic Stenosis in Patients With Bicuspid Valves" in this month's #CardiologyMag: https://bit.ly/3VVhu4l

NEW #feature: #FDA approves cilta-cel for adult patients with R/R #multiplemyeloma after at least one prior line of therapy 🗞️ 🩸 This marks the first #CARTcell product to be approved for patients who relapse following first-line treatment 💡 At the 2024 EBMT-EHA 6th European CAR T-cell Meeting, we caught up with Hermann Einsele, MD, FRCP, University of Würzburg, who shared his insights on the value of bringing #CARTtherapy to earlier lines in multiple #myeloma Prof. Einsele stated, “I think #CARTITUDE-4 and also #KarMMa-3 have nicely shown that with earlier treatment you can be even more effective, and in CARTITUDE-4 it was actually 99.4% of patients responding to CAR T-cells and 86% achieving a complete #remission. I think that’s a response quality and response rate we have never seen before in myeloma.” 🎥 🌟 Explore the feature 👉 https://lnkd.in/eqeKWGPu #VJHemOnc