Olivia Grace Edwards’ Post

📣 🔬 📊 Disclosing our first clinical data! Our Chief Medical Officer Tom Lillie has been presenting at #ASCO, highlighting our first clinical data for our lead immuno-oncology candidate, GRWD5769. The initial data from our ongoing study are incredibly encouraging, with clear signs of target engagement provide compelling support for our antigen modulation strategy in immuno-oncology. A few key takeaways from the presentation: 👉 GRWD5769 was well tolerated, with predictable pharmacokinetics following repeat oral administration in all dose cohorts (up to 200 mg BID).   👉 Dose-dependent target engagement as evidenced by modulation of the immunopeptidome.   👉 Clinical proof-of-mechanism, confirming the expected mechanistic effects of ERAP1 inhibition and consistent with those seen in preclinical models.   👉 Initial, early efficacy assessments demonstrating stable disease achieved by a number of patients, some of whom have remained on treatment for nearly a year. The dose escalation portion of the trial continues, where we have recently initiated combination dosing with GRWD5769 and cemiplimab. With our recent Series B extension, we now have plans for further expansion cohorts in the near future. We are incredibly proud of the team and the rapid progress we have made to this point in development, to deliver our ERAP inhibitors to the patients who need them the most. Read more in the full data release here: https://lnkd.in/e54cJEXA #ASCO #immunotherapy #immunooncology #clinicaltrials #ERAP

Your clinical trial's success begins well before the project starts. With ERGOMED’s proactive approach, we develop a comprehensive strategy that is deeply rooted in patient and physician engagement, leveraging public and private data sources and the expert insights of our scientific/medical and operational teams. Our focus on building trust-based relationships with sponsors is characterized by transparent communication, regular progress updates, and a flexible approach that ensures we always meet your needs.   In a new Q&A published in Pharma's Almanac, “Optimizing Oncology and Rare Disease Clinical Trials by Focusing on Science and Aligning with Both Patients and Investigators,” Dave Selkirk, President of Ergomed Clinical Research, delves into: - Ergomed’s legacy of co-development and scientific excellence - Ergomed’s approach to providing patient-centric solutions specialized in oncology and rare disease clinical research - The nuances of conducting global clinical research - Further clinical transformations on the horizon Read the article to learn more: https://lnkd.in/gTRkYtpB #OncologyCRO #RareDiseaseCRO #ComplexClinicalTrials

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Check out our latest press release: https://bit.ly/4fPfWjV In the fast-paced world of healthcare, especially for rare diseases and oncology, every second counts. Prognos Health is thrilled to announce its groundbreaking daily HCP alerts solution. This innovative solution empowers pharmaceutical companies to engage with healthcare providers (HCPs) precisely when critical treatment decisions are being made. 🚀 Key Benefits: * Timely: Delivered within 48 hours of a patient event * Precise: Target clinically complex patient populations using lab and claims data * Actionable: Focus on specialists actively managing and treating patients 💡 Perfect for oncology, rare diseases, and other complex clinical areas where timely intervention is critical. "Our daily alerts leverage unique lab-based datasets to accelerate interventions, ensuring patients receive the most targeted and effective therapies sooner," says Dr. Jason Bhan, Chief Medical Officer at Prognos Health. 🎯 Rollout begins October 1st! #PrognosHealth #HCPalerts #PrecisionMedicine #Oncology #RareDisease #HealthcareInnovation

When is the right time to include biomarkers in clinical development? In the quest to personalize patient care, biomarkers stand as a compass by which we navigate the vast ocean of clinical development. Their ability to reveal the biological secrets held within our genes, proteins, and cells has transformed the landscape of treatment and drug development. Take the landmark case of Keytruda (pembrolizumab) versus Opdivo (nivolumab) in the immunotherapy space... Keytruda's sales success was propelled by the strategic use of the PD-L1 biomarker in early clinical trials to identify patients who would most likely benefit from the treatment. This precision targeting not only exemplifies biomarker-driven drug development but also underscores the value of biomarkers in enhancing drug efficacy and market position. However, the journey with biomarkers is not without its challenges. For instance, pharmacodynamic testing for levels of EGFR in skin in patients with EGFR mutated lung cancer threw up some interesting results only to be understood years later when we realized that the mutated form of EGFR responds differently to EGFR inhibitors than the wild type form of EGFR. This serves as a cautionary tale, reminding us that while biomarkers can lead to groundbreaking advancements, their application requires rigorous validation to ensure accuracy and reliability. As we harness the power of biomarkers, we are tasked with the responsibility to validate and standardize these indicators to avoid misdirection in clinical decision-making. By doing so, we ensure that biomarkers continue to serve as reliable signposts, leading to improved patient outcomes and more successful therapeutic strategies. The tale of biomarkers is one of promise and caution, of breakthroughs and learning curves. It's a narrative that's still being written, with each chapter promising a new horizon in clinical development. Therefore, the answer to when you begin including biomarkers in clinical development is when you start clinical development! In fact the journey of many biomarkers starts well before that. #ClinicalDevelopment #Biomarkers #PrecisionMedicine #HealthcareInnovation

Amgen has reported positive topline results from its randomized, double-blind, multicenter, placebo-controlled Phase 3 clinical trial (NCT04540497) evaluating the efficacy and safety of UPLIZNA® (inebilizumab-cdon) HCP (inebilizumab-cdon) for the treatment of Immunoglobulin G4-related disease (IgG4-RD). Uplizna, an antibody therapy already with FDA approval for neuromyelitis optica spectrum disorder, is based on technology licensed from Duke University, developed by the late Duke University School of Medicine Dr. Thomas Tedder and colleagues. MITIGATE was conducted at 80 sites in 22 countries. It is the first placebo-controlled trial providing class 1 evidence for treating IgG4-RD, a chronic, systemic, immune-mediated, fibroinflammatory disease that can affect almost any organ in the body, often involving multiple organs at a time, and can result in irreversible organ damage. The novel, steroid-sparing study design paves the way for a reduced toxicity treatment approach. Based on the MITIGATE primary analysis results, Amgen is planning to file for approval in the U.S. followed by other key markets. https://lnkd.in/enXafGUh #innovation #techtransfer #licensing #immune #IgG4RD #NMOSD #autoimmune #therapeutic

Three European projects in which EHA is a partner by consortium leaders SUPPLY With the rising demand of immunoglobulins (Ig) worldwide and high dependency on the U.S. market, Europe’s plasma supply chain faces constant shortages. Therefore, the SUPPLY project was established to increase and strengthen the resilience of plasma collection in Europe. Work Package (WP) 6 focused on demand and use of Ig to provide recommendations on the appropriate and prioritised use of Ig in Europe during baseline and times of crises. Using a five-step approach, our results contributed towards the creation of five recommendations regarding improved understanding of Ig usage, harmonisation of prioritisation and shortage management plans, and enhanced collaborations and linkages. T2EVOLVE T2EVOLVE is a new breakthrough alliance of academic and industry leaders in cancer immunotherapy under the European Union’s Innovative Medicines Initiative (IMI). The key objective of T2EVOLVE is to accelerate development and increase awareness and access of cancer patients to immunotherapy with immune cells that harbor a genetically engineered T-cell receptor (TCR) or synthetic chimeric antigen receptor (CAR). Simultaneously, T2EVOLVE aims to provide guidance on sustainable integration of these treatment into the EU healthcare system. GoCART GoCART is a multi-stakeholder coalition of patient representatives, health care professionals, pharmaceutical companies, regulators, Health Technology Assessment (HTA) bodies and reimbursement agencies, and medical organisations, collaborating to maximise the potential of cellular therapies manufactured from cells and tissues of hematopoietic origin. Our mission is to promote patient access to novel cellular therapies and to contribute to health and well-being through innovation by multi-stakeholder collaboration on clinical data, standards of care, centre qualification, education and policy https://lnkd.in/dVvY6DhU

𝙅𝙤𝙝𝙣𝙨𝙤𝙣 & 𝙅𝙤𝙝𝙣𝙨𝙤𝙣 𝙧𝙚𝙥𝙤𝙧𝙩𝙨 𝙥𝙤𝙨𝙞𝙩𝙞𝙫𝙚 𝙋𝙝𝙖𝙨𝙚 3 𝙧𝙚𝙨𝙪𝙡𝙩𝙨 𝙞𝙣 𝙈𝙮𝙖𝙨𝙩𝙝𝙚𝙣𝙞𝙖 𝙂𝙧𝙖𝙫𝙞𝙨 💊 Johnson & Johnson announced significant positive results from the Vivacity-MG3 𝗽𝗵𝗮𝘀𝗲 𝟯 𝗰𝗹𝗶𝗻𝗶𝗰𝗮𝗹 𝘁𝗿𝗶𝗮𝗹 of nipocalimab in patients with generalized myasthenia gravis (gMG). The study enrolled 199 patients over 24 weeks, showing a substantial improvement in daily living activities and muscle function. 📈 Positive trial results can lead to increased investor confidence and potentially drive up J&J's stock value. With the potential regulatory approval of nipocalimab, J&J could see a boost in its immunology segment revenues, further strengthening its financial position in the biopharma market. Potential $𝟲.𝟱 𝗯𝗶𝗹𝗹𝗶𝗼𝗻 𝗽𝗿𝗼𝘀𝗽𝗲𝗰𝘁. 💰 The 𝗴𝗠𝗚 𝗺𝗮𝗿𝗸𝗲𝘁 is projected to grow significantly, with a rising number of cases and demand for effective treatments. Successful commercialization of nipocalimab could capture a substantial market share, providing a new revenue stream for J&J. Despite these promising results, how will Johnson & Johnson navigate the potential regulatory and market access challenges to ensure nipocalimab reaches the patients who need it most? #headcountag #JNJ #clinicaltrial #phase3 #investment https://lnkd.in/ddWvp7tQ

FDA Rare Disease Innovation Hub to Enhance and Advance Outcomes for Patients While there have been important advances in treatments for rare diseases, more needs to be done. Many rare diseases lack treatment options and therefore many patients have high unmet medical needs. This requires new approaches to expedite development and approval of safe and effective drugs and biologics. We see huge potential in establishing a new model, within the FDA, to leverage cross-agency expertise and greatly enhance our intercenter connectivity to spur the development of treatments for rare diseases. To that end, FDA plans to establish a Rare Disease Innovation Hub (the Hub). The Hub will work across rare diseases but will especially focus on products intended for smaller populations or for diseases where the natural history is variable and not fully understood, as we recognize that development of therapies for these conditions can be particularly challenging. The Hub would have three primary functions: Serve as a single point of connection and engagement with the rare disease community, including patient and caregiver groups, trade organizations, and scientific/academic organizations, for matters that intersect CDER and CBER. The Hub will help the larger rare disease community navigate important intersections across the FDA that affect patients with rare diseases, such as medical devices, including diagnostic tests, and combination products.  Enhance intercenter collaboration to address common scientific, clinical and policy issues related to rare disease product development, including relevant cross-disciplinary approaches related to product review, and promote consistency across offices and Centers.  Advance regulatory science with dedicated workstreams for consideration of novel endpoints, biomarker development and assays, innovative trial design, real world evidence, and statistical methods. #fda #hub #rarediseases #drugdevelopment #statisticalmethods #crossdisciplinary #collaboration https://lnkd.in/g9-wEzs4

In a first for Johnson & Johnson Innovative Medicine, I’m excited to share that the U.S. FDA awarded us a competitive U01 grant, with the goal of addressing clinical trial challenges using #realworldevidence in the #multiplemyeloma space. #Clinicaltrials are necessary to develop new treatments for patients, yet they can often take years to complete. As technology advances and standards of care change for patients, the care patients receive in the real world can be different from the care in clinical trials. As a result, we’re left asking how these new medicines compare to standards of care today. Take multiple myeloma, a rare form of blood cancer. In clinical trials, one of the standard endpoints is progression-free survival (PFS), which indicates the amount of time during and after treatment in which a patient’s disease doesn’t worsen. Since patients in clinical trial are assessed more frequently than patients in the real world, PFS measurements are more accurate and standardized. However, this makes comparisons to the real world challenging, where PFS may be measured at irregular intervals. With this grant, Johnson & Johnson Innovative Medicine will be part of a consortium of leading academic and medical experts working to develop novel methods for more valid and direct comparisons of real-world PFS to clinical trial PFS. If successful, this could allow us to overcome measurement bias in real-world multiple myeloma data and unlock the value of #realworldevidence in the #oncology space. A huge congratulations to lead authors from the Data Science, Oncology, Regulatory, Stats teams, Ashita Batavia, MD, MS, Jennifer Hayden, Khaled Sarsour, Ryan Gan, Jocelyn Wang, Joel Greshock, Kevin Bellew, Craig Tendler, Benjamin Ackerman, Kelly Reid, Harindra Ranil Abeysinghe, Mark Wildgust for their work on this project. Learn more about the grant: