Open Medicine Foundation (OMF)’s Post

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. 

Imagine you are tasked with designing a phase II dose finding study to come up with a suitable dose for phase III. To accomplish this, the invited talk by Gabriele Bleckert from Staburo GmbH at the 9th German Pharm-Tox Summit in Munich on 14 March 2024 within the AGAH session gave good directions. Ten years ago, the Multiple Comparison Procedure and Modelling (MCP-Mod) Methodology was qualified by regulators as an efficient statistical methodology for model-based design and analysis of phase II dose finding studies under model uncertainty. The talk showcased two clinical trials in the indication Schizophrenia and Diabetes where MCP-Mod has been implemented successfully. MCP-Mod was motivated and key features of the methodology were explained with focus on MCP-Mod at analysis stage. Planning of MCP-Mod trials was elaborated in the subsequent talk by Sebastian Bossert from Boehringer Ingelheim. Many thanks to Ruwen Boehm and Barbara Schug from SocraTec R&D who chaired the AGAH (Arbeitsgemeinschaft für Angewandte Humanpharmakologie e. V.) session for inviting us to share our insights on MCP-Mod. Special thanks to Sebastian Bossert for the illuminating exchange on MCP-Mod. #datascience #biostatistics #datatransparency #bioinformatics #clinicalresearch #clinicaltrials

We have analyzed the innovative molecules reached Phase 3 clinical trial stage as per class and therapeutic targets. Enjoy the analysis and outcomes 🚀

Which molecules are reaching phase 3 trials? Understanding which molecule types are reaching phase 3 trials can guide pharmaceutical companies' decision-making, enabling them to explore candidates that are more likely to make it to a phase 3 trial. At the same time, this understanding can also give organisations insight into new areas of opportunity, by revealing underexplored molecule types. In an increasingly competitive industry, this can help give your company an edge. Take a look at our analysis below and get in touch to learn more! #PharmaceuticalInsights #MoleculeTypes #Phase3Trials #Pharmaceuticals

Cereno Scientific reports significant progress and a timeline adjustment in the Phase II study of CS1 in rare disease PAH. We are today sharing significant progress in the study, however, a slower recruitment pace than estimated during the last months and a longer start-up phase for two new clinics have affected the study timeline. The updated study timeline now expects study completion and top-line results during Q2 2024. Björn Dahlöf, CMO, comments: "We are highly encouraged by the remarkable findings in the reported Patient Case as well as the positive findings from the DQCR that our drug candidate CS1 might be able to document substantial clinical benefits in this fatal disease. We are working hard to complete the recruitment of 30 patients to randomize to treatment with CS1 in the study. We have seen significant progress in the last period, however, there are still some patients to recruit. We currently have a number of additional patients identified at our current sites and our two new sites to be activated and are optimistic about the execution of the study based on this updated timeline.” Read more via link https://lnkd.in/gARwGkd5

If you’re attending ISCTM in Washington, DC, this week, don’t miss Dr. Joseph Geraci and Dr. Larry Alphs from NetraMark, where they’ll be presenting a poster THURSDAY EVENING on evaluating drug efficacy in clinical trials by leveraging AI-generated insights from placebo response modeling using NetraAI.    The NetraAI platform allows scientists to examine small, multimodal datasets in CNS trials to uncover connections related to efficacy, toxicity and placebo response. This accelerates traditional drug development through improved understanding of the underlying disease and mechanism of action, improving clinical trial designs and reducing expensive failures.   #NetraAI #ClinicalResearch #CNS #ISCTM

The collaborative mission of HESI's #TPDS committee is to advance efficient and effective translational safety assessment to maximize the therapeutic potential of targeted protein degraders for patients by an international network of multi-partite, multi-stakeholder experts. Check out their 2023 highlights and what they are focusing on in 2024. https://lnkd.in/gQ4ziG9P

Last night, was one of those nights where I did something I rarely do. I watched a "soapy" just to switch off from my work day. Which one? Grey's Anatomy. I don't think I've ever seen a full episode of this before and it was pretty torrid but what I found interesting was that one of patients in this episode had cystic fibrosis. He was very pragmatic and said that he had lived a lot longer than anyone expected him to, so he felt lucky he'd made it to 26 years old. Any my brain went - wait! What??? That's not right..... And then I realised, this episode first aired in 2005 when the median survival for CF patients was less than 30 years. It would be another 7 years before Vertex' first product, Kalydeco, would be approved by the FDA and a few months later, in 2013, I would submit this registration application to the TGA in Australia. The median survival for cystic fibrosis patients has now almost doubled thanks to new treatments for this disease. We are all very focused on finding the next drug target, starting the next clinical trial, submitting the next registration application, and rightly so. But sometimes, it's good to reflect on how far we've come in the orphan drug space in recent years and the impact this has had on patients and their families and friends. Next week I'll be attending the ARCS Conference in Sydney and will be part of the new BioBeacon sessions. Companies will be presenting on their new products in development and panels of experts will provide opinions on some of the pitfalls they may face, questions to ask etc. I very much look forward to being a member of this panel and hearing about what may come to market, from the Australian biotech ecosystem, in another 10-20 years time. #ARCS #biotech #innovation Adjutor Healthcare Group

First-of-its-kind Human Trial to be revealed at ASCO 2024. First-in-human trials act as the bridge for advancing new, promising drug candidates and are primarily conducted to establish the safe dosage range for subsequent clinical development. Numerous stakeholders are actively engaged in discussing their first-in-human trial data findings. This data appears highly compelling, particularly in the context of the innovative class of therapies currently in development within the pipeline. For more captivating insights, visit: https://lnkd.in/dj6Za_QE #FirstInHuman #ClinicalTrials #DrugDevelopment #SafeDose #MedicalResearch #TherapeuticInnovation #PipelineProgress #ClinicalData #HealthcareAdvancement

#ASH23 Analysis 3: #Gilead (Kite) and #BMS presented real-world (RW) and clinical data updates in r/r LBCL. Celltelligence provides insights and context for key selected presentations. The following topics are covered below: • BMS presented: ◦ Final analysis of the Ph2 PILOT study (https://bit.ly/48djNSZ) evaluating #Breyanzi (#CD19 CAR-T) in transplant-ineligible (TI) patients in 2L LBCL ◦ Results from a multicenter real-world (RW) study of Breyanzi in r/r #LBCL conducted in the US • Gilead (Kite) reported results from a RW study assessing the impact of bridging therapy (BT) prior to #Yescarta (CD19 CAR-T) infusion in the outcomes of US patients with ≥3L LBCL Celltelligence provides insights and context for key selected presentations: https://bit.ly/CT-835750