Zevra Therapeutics’ Post

Unity makes strength, and strength is needed to serve patients! 💪🚀 #AmgenProud The five key takeaways: 1- Combined capabilities strengthen portfolio of innovative therapeutics that are first-in-class 2- Leverages Amgen's global footprint, world-class manufacturing, and experience with products early in their lifecycle 3- Reinforces Amgen's leadership expertise and long track record of developing and commercializing rare disease products in inflammation and nephrology 4- Enables a sustained investment in R&D innovation for long-term growth 5- Furthers our mission to serve patients living with serious illnesses, including rare diseases

The start of a new year is a time to reflect on past accomplishments and future ambitions. This recently published article in the Clinical Leader shares how the FSHD Society-Fulcrum Therapeutics partnership began, how it has evolved over time, and where we hope to go in the future. You can read the whole story here: https://lnkd.in/ekCbQnqP

Welcome back to 2024! This year is gearing up to be even bigger than 2023 for Argenica Therapeutics, with Phase 2 clinical trial in acute ischaemic stroke (AIS) patients right around the corner. If you missed it, last month we provided a company update that addressed the current status of the clinical trial. ⬇ You can find the announcement here ⬇ AGN investor announcement: https://bit.ly/3NVPdWu #ASX #AGN #investor #clinicaltrial #clinicaltrialupdate #stroke #strokeawareness

ARG-007 has reached a number of key clinical milestones, with Argenica currently preparing for Stage 2 clinical trials later this year. Here are just a few things that sets ARG-007 apart when it comes to stroke therapeutics. To learn more, download our most recent Investor Presentation: https://bit.ly/3EpPzPO #ASX #AGN #ARG007 #stroketheraputics #clinicaltrials

In his foreword to the #100DaysMission Implementation Report released last week, Sir Patrick Vallance listed four “essential priorities for 2024.”   Can you guess which one topped the list?   1.    Greater coordination and investment in the therapeutics pipeline, to operationalize the 100DM Therapeutics Roadmap.   READDI helped develop the Therapeutics Roadmap and is doing its part to fill the pipeline to give people everywhere a frontline defense against the next potentially disastrous viral outbreak.   Learn more: https://lnkd.in/eQYcNaRc   And download the roadmap here: https://lnkd.in/eu2AhnDh

Exciting News in Cardiovascular Research! Today marks a significant milestone as a groundbreaking small molecule drug, CardiolRx™ currently in Phase II trials for recurrent pericarditis, receives Orphan Drug Designation from the FDA. This achievement underscores its potential to transform the lives of patients. Kudos to the team at Cardiol Therapeutics Inc. for their dedication. 👇Learn more about this groundbreaking development and the ongoing MAvERIC-Pilot study in the comments below. #MedicalInnovation #healthcareadvancements #cardiovascularresearch

Beacon Therapeutics presented 12-month data from its Phase II SKYLINE trial of its lead asset, AGTC-501, in XLRP at the Annual Macula Society Meeting, demonstrating encouraging efficacy and safety.   The presentation highlighted encouraging efficacy shown by improvements in retinal sensitivity, the primary endpoint for the trial, with a 63% response rate in the higher dose cohort. Beacon expects to announce longer term 24-month data from the trial in H2 CY2024.   Read the full announcement here: https://lnkd.in/d4Qfpq6d   #ophthalmology #blindness

Entrada Therapeutics, which received early funding from CureDuchenne, reported positive data from a Phase 1 ENTR-601-44-101 trial in healthy volunteers. ENTR-601-44, which is designed to skip exon 44, was well-tolerated, and the six individuals in the highest dose cohort (6 mg/kg) demonstrated an average of 0.44% exon skipping after a single dose. This was statistically significant compared to placebo control. Entrada plans to submit regulatory applications in Q4 2024 to initiate separate global Phase 2 clinical trials of ENTR-601-44 for Duchenne amenable to skipping exon 44, and for ENTR-601-45 for Duchenne amenable to skipping exon 45. They also plan to submit regulatory applications in 2025 for a global Phase 2 for Duchenne amenable to skipping exon 50. Link to press release: https://lnkd.in/gnd7kvUN

On Thursday, March 14, Autolus Therapeutics held its Q4 2024 earnings call (press release: https://bit.ly/48YCpGz / presentation: https://bit.ly/49VcGQt) highlighting the recent submission of a Market Authorization Application (MAA) for obe-cel (#CD19 CAR-T) in r/r ALL, while noting that its Nucleus manufacturing facility has successfully passed its first GMP inspection enabling the commercial product supply for obe-cel. Additionally, the Ph1 #CARLYSLE confirmatory trial evaluating obe-cel in severe, refractory SLE, and the Ph1 #MAGNETO trial (https://bit.ly/43jTSba) studying #AUTO6NG (#GD2 CAR-T) in pediatric #glioblastoma have been initiated. Celltelligence provides insights on Autolus’s preparedness for obe-cel potential launch in 2024, while discussing its efforts to accelerate obe-cel’s evaluation in the #autoimmune disease space: https://bit.ly/CT-862354