PharmaShots’ Post

Honored to have participated in the 45th SPSR International Webinar on 'Preclinical Animal Models Studying Neurological Diseases', organized by the Society of Pharmaceutical Sciences and Research (SPSR). A big thank you to SPSR for hosting this insightful session, fostering knowledge, and advancing research in the field of neurological diseases. #PharmaceuticalResearch #NeurologicalDiseases #SPSR #ContinuousLearning

Today we reported second quarter financial results and provided a business update. It's been a consequential period for Chemomab--we reported positive results from our Phase 2 trial trial in PSC--the first substantial clinical proof of concept for first-in-class CM-101 in fibro-inflammatory diseases and a foundation for End-of-Phase 2 discussions with the FDA that we believe will provide guidance for the design of a PSC registrational study--a major milestone for both the company and the field targeted for early 2025. We also completed a successful financing with leading biotech investors that extends our cash runway through the beginning of 2026. We look forward to a 2nd milestone in early 2025 when we report data from our Phase 2 trial open label extension--exciting times! For more information see https://lnkd.in/ewjazvh8

🎉 Exciting News! I'm thrilled to share that Lmito Therapeutics has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for LMT801, our investigational treatment for Charcot-Marie-Tooth Disease Type 1A (CMT1A). CMT1A is a rare genetic disorder affecting approximately 1 in 2,500 people worldwide, with about 126,000 patients in the U.S. and 3 million globally. Currently, there are no approved treatments for this devastating condition. Key highlights about LMT801: - Demonstrated significant improvements in motor function, sensory function, and nerve conduction in CMT1A animal models - Showed excellent safety profile in long-term oral administration studies - Potential to be a first-in-class therapy for CMT1A   The ODD grant provides several development incentives, including: Tax credits for qualified clinical trials, Exemption from user fees, Potential 7-year market exclusivity upon approval. #RareDiseases #Biotech #DrugDevelopment #Innovation #Healthcare #FDA #OrphanDrug #CMT #Research

"Thrilled to share that I have received a Certificate of Participation for the 45th SPSR International Webinar on 'Preclinical Animal Models Studying Neurological Diseases.' Grateful for the opportunity to engage with experts in the field and expand my knowledge. Thank you to the Society of Pharmaceutical Sciences and Research for hosting such an insightful event! #ProfessionalDevelopment #NeurologicalResearch #SPSR"

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Marcos Galarza is here to explain what you need to know about the FDA’s recent draft guidance regarding the nonclinical safety assessment of oligonucleotide-based therapeutics (ONTs). Marcos’ key takeaways include: • Tailor your nonclinical studies to reflect the unique characteristics of oligonucleotides, including both hybridization-dependent and hybridization-independent effects. • Leverage 𝘪𝘯 𝘴𝘪𝘭𝘪𝘤𝘰 and 𝘪𝘯 𝘷𝘪𝘵𝘳𝘰 tools to identify potential off-target interactions before moving into animal studies. • Design customized pharmacokinetic and toxicity studies, focusing on oligonucleotide-specific ADME profiles and extended tissue half-lives. • Utilize existing class data for well-characterized ONT classes to streamline development and reduce costs. The draft guidance aims to standardize safety evaluations to ensure rigorous testing before human trials. And once finalized, these recommendations will support safer, more efficient clinical development of ONTs. Read the full draft guidance: https://lnkd.in/eUnCwZZ2   Do you have questions for Marcos about this FDA update or any other regulatory agency? Leave a comment! #Oligonucleotides #Preclinical #GeneTherapy #DrugDevelopment

BGV (BioGeneration Ventures) portfolio company Citryll, a clinical-stage biotech company developing first-in-class therapeutics to treat immune-mediated inflammatory diseases, today announces the successful completion of dosing in the repeat dose stage of its Phase 1 trial for its lead drug candidate CIT-013. The Phase 1 first-in-human trial is assessing the safety and tolerability of CIT-013. Part D of the Phase 1 trial is investigating the safety and tolerability of repeat dosing in 9 patient volunteers with rheumatoid arthritis (RA) and 3 healthy volunteers. All trial participants have been enrolled and successfully completed dosing. Results are anticipated in Q4 2024, which, if positive, would lay the groundwork for the initiation of Phase 2a trials. Click on the following link to learn more: https://lnkd.in/ebkkfhTD #portfoliocompanynews #biotech #lifesciences #BGV #researchanddevelopment Daniela Couto

Yesterday, I attended the "𝗣𝗿𝗲𝗰𝗶𝘀𝗶𝗼𝗻 𝗣𝗮𝘁𝗵𝘄𝗮𝘆𝘀: 𝗜𝗻𝘀𝗶𝗴𝗵𝘁𝘀 𝗮𝗻𝗱 𝗦𝘁𝗿𝗮𝘁𝗲𝗴𝗶𝗲𝘀 𝘁𝗼 𝗡𝗮𝘃𝗶𝗴𝗮𝘁𝗲 𝗥𝗲𝗴𝘂𝗹𝗮𝘁𝗼𝗿𝘆 𝗥𝗲𝗾𝘂𝗶𝗿𝗲𝗺𝗲𝗻𝘁𝘀 𝗳𝗼𝗿 𝗢𝗻𝗰𝗼𝗹𝗼𝗴𝘆 𝗗𝗶𝗮𝗴𝗻𝗼𝘀𝘁𝗶𝗰𝘀" webinar led by Hakan Sakul, Elaine Katrivanos and Lauren Silvis. Tempus AI The session was focused on the evolving FDA regulatory landscape within oncology diagnostics. We explored strategic approaches to regulatory submissions, new requirements, and how these pathways can accelerate diagnostic developments and precision medicine initiatives. A key takeaway was the emphasis on collaboration – Hakan emphasised that diagnostic providers, drug developers, and the FDA must work closely throughout the process. “𝗢𝗽𝗲𝗻 𝗰𝗼𝗻𝘃𝗲𝗿𝘀𝗮𝘁𝗶𝗼𝗻 𝗶𝘀 𝗯𝗲𝘀𝘁…𝘁𝗵𝗲𝘆 𝘀𝗵𝗼𝘂𝗹𝗱 𝗯𝗲 𝗰𝗼𝗻𝗻𝗲𝗰𝘁𝗲𝗱 𝗮𝘁 𝘁𝗵𝗲 𝗵𝗶𝗽𝘀”. This collaborative approach ensures that regulatory submissions are efficient and aligned with market needs. It was a truly engaging and informative session; thank you. If you have any thoughts or insights, I’d love to discuss these with you! 👇 #OncologyDiagnostics #PrecisionMedicine #RegulatoryAffairs #FDARegulations #DiagnosticsInnovation #HealthcareCollaboration

Submit your abstract for #IgNS2024 today!