Hongene Biotech Corporation’s Post

Engaged in early oligonucleotide development? If yes, you should take note of this new FDA guidance “Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics”. In detail the guidance refers to the following clinical pharmacology topics: (1) characterizing the potential for QTc interval prolongation, (2) performing immunogenicity risk assessment, (3) characterizing the impact of hepatic and renal impairment, and (4) assessing the potential for drug-drug interactions. A series of oligonucleotides have been approved during the last years (fomivirsen, mipomersen, eteplirsen, nusinersen, inotersen, patisiran, givosiran, golodirsen, and vitolarsen), and the recent FDA guidance translates the experience with the approval of these drugs into this new Guidance for Industry. In this context reference is made also to a review paper published by Rogers et al in 2021 (Clin Transl Sci (2021) 14, 468–475; doi:10.1111/cts.12945) #oligonucleotide, #EarlyPhase, #ClinicalPharmacology

On June 17, 2024, the FDA announced the availability of a final guidance for industry entitled Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics, replacing the June 2022 draft guidance on this topic. Specifically, it provides the FDA’s recommendations for certain evaluations during development of oligonucleotide therapeutics, including: (1) characterizing the potential for QTc interval prolongation, (2) performing immunogenicity risk assessment, (3) characterizing the impact of hepatic and renal impairment, and (4) assessing the potential for drug-drug interactions. This guidance also provides recommendations on when to conduct these assessments and what types of assessments are suitable to address the topics listed above.  For more information, visit https://lnkd.in/esQ36YuU #PharmaPro #FDA #FinalGuidelines #DrugDevelopment #ConsultingServices #ClinicalPharmacology #OligonucleotideTherapeutics

We are excited to share non-clinical pharmacodynamics research for siRNA drugs. We explain the PK/PD challenges and solutions for siRNA drugs, and our expertise in using non-human primate models for preclinical studies. https://lnkd.in/dKexkXpD #prisysbiotech #siRNA #pharmacodynamics #nonhumanprimates #drugdevelopment

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

💊 𝐒𝐦𝐚𝐥𝐥 𝐦𝐨𝐥𝐞𝐜𝐮𝐥𝐞𝐬 𝐝𝐨𝐦𝐢𝐧𝐚𝐭𝐞 𝐚𝐦𝐨𝐧𝐠 𝐅𝐃𝐀 𝐧𝐨𝐯𝐞𝐥 𝐝𝐫𝐮𝐠 𝐚𝐩𝐩𝐫𝐨𝐯𝐚𝐥𝐬 𝐢𝐧 2023 Among the FDA & CDER 2023 novel drug approvals, oncology and haematology are the dominant therapeutic areas. 2023 brought more diversity in terms of modalities, especially bringing an increase in nucleotide-based therapeutics. According to 𝘕𝘢𝘵𝘶𝘳𝘦 𝘙𝘦𝘷𝘪𝘦𝘸𝘴 𝘋𝘳𝘶𝘨 𝘋𝘪𝘴𝘤𝘰𝘷𝘦𝘳𝘺, the FDA approved in 2023: ⚆ 34 small molecule products ⚆ 17 protein-based products ⚆ 4 oligonucleotides 𝘞𝘩𝘢𝘵 𝘵𝘳𝘦𝘯𝘥𝘴 𝘪𝘯 𝘵𝘦𝘳𝘮𝘴 𝘰𝘧 𝘮𝘰𝘥𝘢𝘭𝘪𝘵𝘪𝘦𝘴 𝘤𝘢𝘯 𝘸𝘦 𝘦𝘹𝘱𝘦𝘤𝘵 𝘪𝘯 2024? Source: https://lnkd.in/eT_j65eT #pharma #drugdevelopment #biotech #innovation #business #clinicaltrials #womeninbusiness #drjojo

Antisense oligonucleotides (ASOs) an emerging drug modality that is having clinical impact in neurological and rare diseases, including ALS/Motor Neuron Disease. In this webinar experts from Amylyx Pharmaceuticals and Charles River discuss design and screening of ASO, and considerations for designing successful in vivo efficacy studies. https://okt.to/tFBCK5 

New webinar available on-demand: Clinical Pharmacology & Model-Informed Drug Development (MIDD) Insights for Targeted Radiation Therapies (TRT). Hear from our team of experts to learn how MIDD can address the unique considerations and challenges in TRT development! Watch on-demand: https://ow.ly/zKZl50STpPz #ClinicalPharmacology #Pharmacometrics #QuantitativeSystemsPharmacology #Radiopharmaceuticals #TargetedRadiationTherapies #DrugDevelopment

Happy Thursday! 🚨 Here are Drugs to Watch in 2024 🚨 🔍 Clarivate's latest report unveils a shift in pharmaceutical interest. Keep an eye on these developments for 2024: Aflibercept (high dose) Budesonide Datopotamab deruxtecan Efanesoctocog alfa Ensifentrine Exagamglogene autotemcel and lovotibeglogene autotemcel Mirikizumab Niraparib + abiraterone RSVpreF and RSVpreF3 Talquetamab Zolbetuximab I'm really excited about Mirikizumab, a first-in-class therapy for ulcerative colitis approved by the EMA and the U.S. FDA that will likely be the third in the class approved for Crohn’s disease. A delayed U.S. launch due to manufacturing concerns by the U.S. FDA means that it remains a drug to watch for 2024. There's also one notable omission here which is KarXT for schizophrenia. What therapies are you excited about in 2024? 💊📉 #PharmaWatch #DrugDevelopment #HealthcareInnovation #MedicalInnovation #DrugApproval #HealthcareAdvancements https://lnkd.in/g7zsXkX3

📣 Today! Earn 2.0 CE hours October 9 with SBIA webinar: ICH M12 Drug-Drug Interaction Studies Final Guidance If your work relates to drug interactions, drug metabolism, pharmacokinetics, or clinical pharmacology, join us for this webinar which will discuss the full scope of the guidance, which is the first globally harmonized regulatory guidance on assessments of pharmacokinetic drug interactions mediated by metabolic enzymes and drug transporters. Log in early! ➡ https://lnkd.in/e6ygc4HH

📰 RNA updates last week: 28 new drugs, 3 trial updates, 1 new deal.  💊 Drugs 🔵 27 new drugs disclosed by Argo Biopharma! Focused on using siRNA targeting mainly cardiovascular diseases. https://lnkd.in/evj_8UbB 🔵 Innorna mRNA asset IN006 using LNP's as the delivery system. https://lnkd.in/eewnm4Sr   👨⚕️ Trials 🔵 OliX Pharmaceuticals evaluation of OLX10212 in patients With Neovascular Age-related Macular Degeneration. Change of estimated enrolment and outcomes measures.   💰 Deals 🔵 RinuaGene Biotech Received Pre-A+ Round of Financing $12.4M. https://lnkd.in/ejA-SQRW   As always, thanks to our Beacon researches for capturing all the insights!