CMT Research Foundation’s Post

At Pomona our primary focus lies in the pre-clinical characterization stage, a pivotal phase that sets the foundation for successful drug development. Learn more on your website: #pomona #reserch #preclinical #pipeline #drugdevelopment #monoclonalantibody

🌟 Webinar Reminder: “First-In-Human Dose Selection & Approaches” 🗓️ Date: June 19, 2024 ⏰ Time: 3:00 PM Join us as we explore best practices in first-in-human (FIH) studies and early clinical development. Our guest speaker, Antoine Deslandes (LinkedIn: Antoine Deslandes), will review the different approaches. 🔗 Register here: https://lnkd.in/eYy57fY7 Feel free to share with colleagues interested in clinical pharmacology and drug development. See you there! 🌐👩⚕️👨⚕️ #ClinicalDevelopment #Pharmacology #Webinar

Antisense Oligonucleotide (ASO) deals continue last week with Novo - Cardior Pharma €1.025bn deal. However, lets dive deeper into the preclinical drug & clinical trial landscape. 🔵 Preclinical Drug Landscape: 67 biopharma developers operating at this early stage, representing 68% of all ASO drugs at this early stage. A staggering 38 ASO drugs have been disclosed in 2024 so far. 🔵 Clinical Trial Landscape: 428 trials across all stages. 8 have been added in 2024. DMD & SMA are the most common indications with 13% & 6% respectively. Interesting to see 15% at PIII which should yield more approvals soon! Planning your drug and trial strategy is key in this competitive space, Beacon RNA can simplify this complexity. #RNA #ASO #Oligo

https://buff.ly/47EvlQf 🧬 𝐅𝐃𝐀 𝐂𝐚𝐧𝐜𝐞𝐥𝐬 𝐀𝐝𝐜𝐨𝐦𝐦 𝐟𝐨𝐫 𝐑𝐚𝐫𝐞 𝐃𝐢𝐬𝐞𝐚𝐬𝐞 𝐃𝐫𝐮𝐠, 𝐒𝐭𝐨𝐜𝐤 𝐒𝐨𝐚𝐫𝐬 65% (𝐍𝐀𝐒𝐃𝐀𝐐:𝐀𝐏𝐋𝐓) 📈 : 𝐒𝐡𝐢𝐟𝐭𝐢𝐧𝐠 𝐑𝐞𝐠𝐮𝐥𝐚𝐭𝐨𝐫𝐲 𝐋𝐚𝐧𝐝𝐬𝐜𝐚𝐩𝐞? The FDA's recent actions in the rare disease space are hinting at a regulatory evolution we've long hoped to see. Their decision to cancel Applied Therapeutics' adcomm for govorestat in galactosemia is particularly intriguing. Despite missing its primary endpoint, the drug showed promise in secondary measures - and it seems the FDA is taking note. We're cautiously optimistic that this signals a more flexible approach to evaluating efficacy for rare disease therapies. When dealing with small patient populations and limited treatment options, the traditional rigid criteria often fall short. A more nuanced, holistic view of drug benefits is exactly what's needed, especially in areas of high unmet need. The establishment of the new Genetic Metabolic Diseases Advisory Committee further fuels our optimism. It's a proactive step that acknowledges the complexity of these disorders and the incoming wave of novel therapies. We hope this marks the beginning of a broader paradigm shift in rare disease drug evaluation. As our understanding of these complex disorders grows, so should our regulatory framework. If the FDA continues down this path, it could pave the way for more breakthrough therapies to reach patients who desperately need them. The hope is sponsors can look forward to regulators across the globe "rethinking" what is actually feasible when it comes to trial designs and data presentation for rare diseases, focusing more on comprehensive benefit profiles rather than single endpoints - with real-world evidence and post-approval commitments helping to "fulfill" traditional licensing obligations and still provide robust safety monitoring oversight to this neglected patient population... #RareDiseaseResearch #ClinicalTrials #RegulatoryAffairs #DrugDevelopment #BiotechInnovation #PatientCentricTrials #OrphanDrugs

The webinar (June 19th) is getting closer. Mark your calendar and REGISTER below

Biotech and pharma companies - How do we determine the most efficient regulatory path for codeveloped companion diagnostic tests (CDx)? A CDx can significantly maximize return on investment in clinical trials, but transitioning from a research-grade to a clinical-grade assay can present resource management challenges, especially for emerging companies. Regulatory experts Mwango Kashoki and Sinan Sarac share how to minimize time and resources in our New Medicines, Novel Insights Oncology report: https://lnkd.in/e-iG-67V #PrecisionOncology #Regulatory #NovelInsights

Preclinical ADME prediction remains a significant challenge in drug development. Fowler et al. (2022) underscore the necessity of a multifaceted approach to accurately predict ADME properties for complex therapeutic candidates. Our latest article delves into the complexities of preclinical human ADME profiling, cited in peer-reviewed publications, and explores advanced human-relevant preclinical models that can enhance bioavailability estimations for improving drug safety and efficacy. 👉 Read the full article to learn more: https://lnkd.in/e7iE9Rpz #OrganOnAChip #ADME #DrugDevelopment

Biotech and pharma companies - How do we determine the most efficient regulatory path for codeveloped companion diagnostic tests (CDx)? A CDx can significantly maximize return on investment in clinical trials, but transitioning from a research-grade to a clinical-grade assay can present resource management challenges, especially for emerging companies. Regulatory experts Mwango Kashoki and Sinan Sarac share how to minimize time and resources in our New Medicines, Novel Insights Oncology report: https://lnkd.in/eYHpRUxA #PrecisionOncology #Regulatory #NovelInsights

#NMD Pharma A/S announces that it has dosed the first generalized myasthenia gravis patient in a Phase 2b clinical trial of NMD670, after receiving FDA IND clearance in March 2024 to conduct the study. NMD670 is a first-in-class small molecule inhibitor of the skeletal muscle specific chloride ion channel (CIC-1). NMD Pharma has demonstrated that CIC-1 inhibition enhances weakened neuromuscular transmission and restores skeletal muscle function, and this novel treatment approach has demonstrated compelling preclinical and clinical data in animal models of myasthenia gravis, spinal muscular atrophy and a range of other neuromuscular disorders. NMD670 has also been granted orphan-drug designation by the U.S. FDA for treatment of gMG. Chemenu has been working to develop more compounds for drug discovery. Here comes the building blocks we can provide: https://lnkd.in/gyW2_JdM #NMD670 #CIC-1Inhibitor #myastheniagravis #MG #buildingblocks