Margarida Duarte

Next‑generation NLRP3 Inhibitors: The Future GLP‑1 Blockbuster? New article out today. Bottom line for pharma leadership: * NLRP3 is emerging as a franchise‑scale “master switch” for chronic disease and healthy longevity—and a competitive * NLRP3 program is now a strategic necessity. What’s changed: • Human validation is unequivocal (CAPS GOF mutations → IL‑1β biology; downstream IL‑1 blockade already disease‑modifying). • Research/funding inflected: NIH projects mentioning “NLRP3” grew from 392 (FY2020) to 1,326 (FY2024). Publications exploded across neurology, cardiology, and metabolism over the last decade. • Big‑pharma votes of confidence: Roche acquired Inflazome (≈€380M upfront, 2020); Novartis acquired IFM Tre ($310M upfront + up to $1.265B, 2019); Novo Nordisk licensed Ventus’ peripherally‑restricted inhibitor (deal value up to $700M, 2022). • Chemistry is maturing beyond early scaffolds (e.g., MCC950/GDC‑2394 toxicity attributed to scaffold, not target). Multiple next‑gen series now showing cleaner early safety. Why executives should care: • Pipeline‑in‑a‑product: addressable indications span Alzheimer’s, Parkinson’s, gout, IBD, CKD, NASH, atherosclerosis, osteoarthritis, COPD/IPF, obesity, and aging biology itself (“inflammaging”). • Synergy with GLP‑1s: CNS‑penetrant NLRP3 inhibitors can reduce hypothalamic inflammation; preclinical data show additive/synergistic weight loss and durability—positioned as a must‑have combination for a $100B+ category. • Differentiation vectors that matter: brain penetration vs peripheral restriction, long‑term safety, selectivity vs other inflammasomes, scaffold liability, DDI profile, oxidation risk, and once‑daily oral convenience. • IP tail: several best‑in‑class candidates carry 2023–2024 priority‑date composition‑of‑matter claims—~16–17 years fresher than some first‑gen assets. Competitive landscape highlights (from the piece): • >20 companies and >25 inhibitors across stages; class de‑risked yet still wide open for genuine best‑in‑class chemistry. • Insilico Medicine: ISM8969 (AI‑designed, brain‑penetrant) built for high potency, selectivity, and long‑term safety; broad indication strategy. Operator checklist for BD/R&D: • Prioritize scaffolds with proven translatability, clear CNS/peripheral knobs, and clean liver/safety margins. • Optimize for durability, once‑daily oral dosing, and scalable manufacturing. If GLP‑1s defined the last decade, targeted anti‑inflammasome therapy can define the next. The companies that win will pair iron‑clad chemistry with disciplined clinical and IP strategy. Read the full analysis: “Next‑generation NLRP3 Inhibitors: The Future GLP‑1 Blockbuster? A Potential Longevity Therapeutic Targeting Inflammaging.”  https://lnkd.in/gTD_5DGd

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💡 Valuable input to the ongoing discussion on equitable access to medicines: Roche CEO Thomas Schinecker highlighted that it would be “very easy” to cut US drug prices by 50%—by removing intermediaries and enabling direct-to-patient distribution. According to Schinecker, half of supply chain earnings currently go to middlemen who take “zero risk” in the innovation process. This dialogue underscores a key challenge: ensuring broad access to life-saving therapies while preserving the necessary incentives for those who invest heavily and take the scientific and financial risks to innovate for patients in need. HealthcarePolicy #PharmaInnovation #DrugPricing #AccessToMedicine #Biopharma

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🤩 Delighted to share that, today, the European Medicines Agency has granted approval to Servier’s first targeted therapy for patients with glioma.  ✅ This approval is based on robust clinical data, demonstrating a significant step forward in the treatment of a rare brain cancer.  ✅ It’s also a strong recognition of our scientific capabilities and the unfailing work of our R&D teams, who are committed to advancing precision medicine in oncology.  Proud to see our research contribute to transforming patient outcomes.  #Oncology #Glioma #RnD #WeAreServier 

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What does patient safety actually mean? After our Patient Safety Day Daiichi Sankyo Europe GmbH, I am more convinced than ever: it is about seeing the whole person, not just the treatment. We were privileged to hear directly from Diana Dragu and Katharina Bauer about the realities shaping their lives during treatment – from fertility and lactation, to the safety of their families at home, to the need for honest conversations about future plans. These are not “side topics” – they are central to patient safety. Pharmacovigilance helps us capture these lived experiences and turn them into knowledge we can act on. Listening alone is not enough. It is our responsibility to ensure these insights lead to tangible changes – in how we design our studies, how we counsel patients, and how we make sure our approach to treatment considers real-world needs. I cannot emphasize enough that patient safety is truly a shared mission. From Medical Affairs to Pharmacovigilance, from clinical teams to affiliates, each of us plays an important role in making sure treatments are not only effective but also safe and supportive. To patients and families – what aspects of safety matter most to you during and after treatment? 

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🔬 Remarkable strides and urgent gaps in the fight against lung cancer   Over the last decade, we’ve witnessed a transformation in the lung cancer treatment landscape. But despite these advances, many patients and families are still left with unmet needs. Let´s have a look at it:   From one-size-fits-all to precision medicine Thanks to targeted therapies and immunotherapies, we are now routinely matching treatments to molecular profiles (EGFR, ALK, ROS1, KRAS, etc.). These “precision” approaches have significantly improved survival, even for metastatic non-small cell lung cancer. These advances underscore that lung cancer is no longer a monolithic disease - we are steadily turning it into a constellation of molecularly defined subtypes, each with its own therapeutic roadmap.   But the unmet need is still very high Studies show that ~80% of lung cancer patients report at least one unmet supportive care need, with psychological and informational needs being among the most common.  In short: even as we unlock more precise therapies, many patients and caregivers are left navigating emotional, social, and practical challenges - sometimes far beyond what the clinic can deliver.   💡 Bottom line (for me and, I hope, for us all): We must continue pushing the frontier of innovative therapies - but we cannot forget the human side. Research, policy, and care models need to evolve in parallel: to anticipate and deliver on what patients and families truly need.   If you’re working in lung cancer care, research, patient advocacy, or policy, I’m always eager to connect and learn: See you hopefully at ESMO congress in Berlin!   #LungCancer #MyCompany #PrecisionMedicine #Oncology #PatientCare #ESMO26 #CancerResearch #FlatWhiteBerlin

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