Merus N.V.’s Post

On #BiotechHangout today Daphne Zohar, Brad Loncar, John Maraganore, Josh Schimmer, Bruce Booth & Yaron Werber, M.D., MBA cover $ABBV / $IMGN $10B acquisition, FDA’s CAR-T safety concerns, data setbacks & wins ($ROIV, $ARGX, $XENE, & $IVMT), $REGN & $SNY seek to expand Dupixent approval to COPD, $SWTX approval, plus JAMA Internal Medicine paper calls to eliminate orphan drug exemptions, $SLRN & $BIVI point to CRO errors in trial failures, X Prize $101M prize in aging financings/biotech sentiment Listen here: https://lnkd.in/ejhkm9BY

I joined a session today at #ESMO23 that discussed dose optimization in clinical trials which is part of FDA Project Optimus. It was an interesting scientific discussion about maximum tolerated dose (MTD) versus 'optimal dose'. What actually sticked with me the most was a talk from patient advocate Jill Feldman who gave a more patient-centered consideration for treatment-related adverse events (TRAEs or rather; side effects) in relation to drug benefit: How a side effect is perceived is subjective, and patients are the ones that can actually only say if something is severe and/or intolerable. We use grading to standardize side effects, but there might be cases where Grade 3 or higher TRAEs that last for a short time might be less of a patient burden than Grade 1 or 2 side effects that last for a longer period of time. Quality of life is everything, how can we incorporate this even more in treatment considerations and patient information to make an informed (patient-centered) choice? Food for thought when flying back home tomorrow! It's all about the optimal balance between benefit and risks.

Real-world studies – otherwise known as pragmatic RCTs – complement the important results from RCTs by providing information on relative drug effectiveness in everyday practice conditions. Uncover the solutions and discover how we can help with trial design, recruitment and patient monitoring: www.nweh.co.uk   #decentralisedtrials #clinicaltrialdesign #clinicaltrialrecruitment

Friday is a big day. Both #Carvykti and #Abecma have pivotal trial data and are trying to obtain FDA approval to move into "earlier lines of therapy" in #multiplemyeloma Both products were approved in salvage settings - but the true promise of #celltherapy is in earlier use where patients have the potential to achieve greater benefit through potentially curative one-time treatments. At this Friday's ODAC meeting, the FDA has posed the following questions to the committee for discussion: 1️⃣ Discuss whether the results of CARTITUDE-4 are sufficient to support a positive risk-benefit assessment of cilta-cel (Carvykti) for the proposed indication. 2️⃣ Is the risk of early death associated with cilta-cel treatment acceptable in the context of clinical benefit? 3️⃣ Discuss whether the results of KarMMa-3 are sufficient to support a positive risk-benefit assessment of ide-cel (Abecma) for the proposed indication. 4️⃣ Is the risk of early death associated with ide-cel treatment acceptable in the context of clinical benefit? The FDA is raising concerns about risk-benefit and concerns over "early deaths". Hard to believe that we are talking about the possibility of these #cartcelltherapy's replacing toxic combination chemotherapies that have been standard of care for years! ⭕ Follow me Christopher Leidli to stay informed of updates in #advancedtherapies and #celltherapy

Friday is a big day. Both #Carvykti and #Abecma have pivotal trial data and are trying to obtain FDA approval to move into "earlier lines of therapy" in #multiplemyeloma Both products were approved in salvage settings - but the true promise of #celltherapy is in earlier use where patients have the potential to achieve greater benefit through potentially curative one-time treatments. At this Friday's ODAC meeting, the FDA has posed the following questions to the committee for discussion: 1️⃣ Discuss whether the results of CARTITUDE-4 are sufficient to support a positive risk-benefit assessment of cilta-cel (Carvykti) for the proposed indication. 2️⃣ Is the risk of early death associated with cilta-cel treatment acceptable in the context of clinical benefit? 3️⃣ Discuss whether the results of KarMMa-3 are sufficient to support a positive risk-benefit assessment of ide-cel (Abecma) for the proposed indication. 4️⃣ Is the risk of early death associated with ide-cel treatment acceptable in the context of clinical benefit? The FDA is raising concerns about risk-benefit and concerns over "early deaths". Hard to believe that we are talking about the possibility of these #cartcelltherapy's replacing toxic combination chemotherapies that have been standard of care for years! ⭕ Follow me Christopher Leidli to stay informed of updates in #advancedtherapies and #celltherapy

#Clinicaldecisionsupport systems are commonly used in hospital settings to assist in medication ordering, but numerous low-yield alerts can lead to alarm fatigue and lack of benefit. In a randomized trial conducted in nine intensive care units (ICUs) in the Netherlands, ICU-tailored drug-drug interaction alerts reduced administrations of high-risk drug combinations relative to control. Tailoring alert systems to be relevant to the clinical setting may result in meaningful improvements in care. Read What's New in #hospitalmedicine: https://ow.ly/nEKf50QUF2F #ClinicalUpdates

Watch Fran Gregory, PharmD, MBA in the virtual event "March of the Biosimilars" with STAT as she and other experts discuss adalimumab biosimilars and their impact on the healthcare ecosystem over the past year. #CardinalHealth #Biosimilars

Meet DUOFAG® (if you have not met it before). DUOFAG® is a drug product with bacteriophages against S. aureus and P. aeruginosa. The clinical trials, phase I IIA, were approved in the Czech Republic in December 2022 and first patients were hired at the end of 2023. Finally, the clinical trials were updated here: https://lnkd.in/eMH3C3h3 We expect first results to be announced in summer 2024. #bacteriophages #clinicaltrials #phagetherapy #antibioticresistance

A paper published in the journal ‘Pharmaco Economics’, demonstrated that "A major driver of antibiotic use is patients consulting for respiratory infections in primary care"[18] where access is easier. "Inappropriately high levels of antibiotics are still prescribed in primary health care for RTIs"[8]. Put another way, whilst antibiotics are essential for a safe operation within a hospital setting, their misuse is being driven in large part by prescriptions given for more minor ailments such as RTIs by community healthcare professionals. “Reducing inappropriate antibiotic use while expanding essential access is a difficult challenge, especially in low- and middle-income countries.”[17] Understanding and addressing primary care consultation patterns and prescriber behaviours is central to contending with the problem of antibiotic misuse. Visit the Senzo website to request the full report... #AMR #AntimicrobialResistance #Superbugs #PostAntibioticEra #AntibioticStewardship #InfectionControl #GlobalHealth #AMRActionFund #Diagnostics #NewAntibiotics #InfectiousDiseases #DrugResistance #Healthcare #PublicHealth #Medicine #Pharma #Biotech #IDWeek #ASMmicrobe #ICAAC #ECCMID #microbiology