Vigeo Therapeutics ’s Post

Namodenoson is a small orally bioavailable drug that binds with high affinity and selectivity to the A3 adenosine receptor (A3AR). Namodenoson was evaluated in Phase II trials for two indications, as a second line treatment for hepatocellular carcinoma, and as a treatment for non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH). Namodenoson granted Orphan Drug and Fast track status from the FDA. A3AR is highly expressed in diseased cells whereas low expression is found in normal cells. This differential effect accounts for the excellent safety profile of the drug. Chemenu has been working to develop more compounds for drug discovery. Here comes the building blocks we can provide: https://lnkd.in/gtC2PYBK #Namodenoson #2-CL-IB-MECA #A3AR #buildingblocks

We are pleased to share our latest article exploring the oral delivery of #oligonucleotide therapeutics targeted against gastrointestinal (GI) diseases and cancers authored by N4 Pharma CEO Nigel Theobald. A promising route to success in this field is via our engineered silica nanoparticle, Nuvec®, which has a unique irregular surface structure that effectively traps and protects #siRNA as it migrates to the target cells, making it well suited to successful oral administration. Read the article published in the latest issue - and featured on the cover - of ONdrugDelivery here: https://lnkd.in/eZCAA-5N

Two recent papers -- by Fojo and Olivier -- explained why Sotorasib, a KRAS G12C inhibitor, became a regulatory setback for NSCLC? They wrote: Sotorasib was approved on the basis of a single-arm, uncontrolled phase 1–2 study (CodeBreaK 100) that reported an objective response rate (ORR) of 37% and a 11·1 month median duration of response. By contrast, other molecular therapies for NSCLC have higher ORRs; osimertinib targets EGFR-mutated NSCLC and has an ORR of 90% and alectinib targets ALK-mutated NSCLC and has reported an ORR of 82·9%.

At Artax Biopharma, we are developing first-in-class Nck modulators to treat T cell mediated autoimmune disease such as #psoriasis, #atopicdermatitis, and more. We're excited by the progress we've been making in the development of our first novel mechanism, AX-158, as we rapidly approach phase 2 trials for psoriasis treatment with first results expected in Q4 2024. We are simultaneously developing additional Nck modulator candidates for #autoimmunediseases with AX-194 undergoing preclinical testing to enter Phase 1 trials. Learn more about our candidate pipeline on our recently updated website here: https://lnkd.in/eqq3eSTK #clinincaltrials #immunology

💫 FDA Approval 💫 🌟 U.S. Food and Drug Administration Approves Augtyro™ (repotrectinib), a Next-Generation Tyrosine Kinase Inhibitor (TKI), for the Treatment of Patients with NTRK-Positive Locally Advanced or Metastatic Solid Tumors 👉 Augtyro is the only FDA-approved treatment option for NTRK-positive tumors studied in both TKI-naïve and TKI-pretreated patients across solid tumors, demonstrating clinically meaningful response rates in the TRIDENT-1 trial 🔗 https://buff.ly/4cf7pEN #OncEd #ONCOnews #OncoAlert

Today, Halia Therapeutics announced the dosing of the first patient in Phase 2a clinical trial evaluating its lead candidate, HT-6184, for the treatment of lower-risk myelodysplastic syndromes (LR-MDS). HT-6184 is a selective and orally bioavailable first-in-class inhibitor of NLRP3/NEK7 inflammasome, which is a main driver of inflammatory disease, as well as hematologic and other malignancies. To read the full release, visit: https://prn.to/3tiy5TO #inflammasome #NLRP3 #clinicaltrial #clinicalstudy #myelodysplasiasyndrome #MDS #MDStreatment #transfusion

📢 Promising Drug Blocks Epstein Barr Virus Diseases 🌟 Hornet Therapeutics, a spin-out from the University of Basel, has published first data on its preclinical IDO1 blocker HTX-201. They aim to start clinical tests within the next 12 to 18 months to target post-transplant lymphoproliferative disease. 🏥 This small molecule drug has the potential to block a target used by the Epstein Barr virus to cause diseases. 🦠 #BioprocessUpdates #HornetTherapeutics #EBV #diseaseblocker #HTX201 #clinicaltests #posttransplantlymphoproliferativedisease #smallmoleculedrug #EpsteinBarrvirus #preclinicaldata #UniversityofBasel #virusrelateddiseases #antiviraltherapy #medicalresearch #transplantmedicine #innovationinmedicine ▷ Read the full article here: 📎 https://lnkd.in/dSTEbP3u

📰 REGUALTORY UPDATE: The US Food and Drug Administration (FDA) has approved amivantamab-vmjw (Rybrevant) plus lazertinib (Lazcluze) for the first-line treatment of adults with locally advanced or metastatic NSCLC with EGFR exon 19 deletions or exon 21 L858R substitution mutations, as detected by an FDA-approved test, according to an announcement from Johnson & Johnson. ➡ Learn more: https://lnkd.in/gF9Jrm56

💪 Novartis has had a strong start to 2024 with net sales of $11.8Bn (+10%) and net income increasing to $2.68Bn (+25%) compared to Q1 2023. Here is my financial breakdown: 📈 Key growth drivers included the cardiovascular drug Entresto (+34%), psoriasis medication Cosentyx (+23%), MS drug Kesimpta (+66%), breast cancer drug Kisqali (+51%), and radioconjugate Pluvicto (+47%). 📉However, this growth was partially offset by increased generic competition for Lucentis (-25%) and Gilenya (-25%). #Novartis #q1 #financials #visualisation #data #breakdown #pharma #basel

Yesterday's decision by the FDA to accept CSF heparan sulfate as an endpoint to support #AcceleratedApproval in #SanfilippoSyndrome marks a significant victory for patients. This pivotal moment follows our collaborative workshop in February 2024 with the Reagan-Udall Foundation for the FDA, focusing on "Developing Biomarkers to Support Accelerated Approval in Rare Diseases." Thank you to the Reagan-Udall Foundation for the FDA for spearheading the discussion, alongside the FDA, Cure Sanfilippo Foundation, National MPS Society, The Ryan Foundation, National Organization for Rare Disorders and the academics and co-sponsors, Denali Therapeutics, Orchard Therapeutics - U.S., Allievex Corporation, and REGENXBIO Inc., who joined us. We are optimistic that this is the beginning of rethinking the approval pathway for ultrarare diseases by using disease-causing biomarkers as primary endpoints to support accelerated approvals.  #RareDisease #GeneTherapy #MPS