Immutep Limited’s Post

First MYC inhibitor to demonstrate safety and anti-tumor activity in a phase I first-in-human clinical trial

An estimated 10,000+ rare diseases affect more than 30 million people – approximately one out of every 10 people – in the U.S., and about half of these people are children. Many rare conditions are life threatening, and most do not have approved treatments.    The FDA plans to establish a Rare Disease Innovation Hub (the Hub). The Hub will work across rare diseases but will especially focus on products intended for smaller populations or for diseases where the natural history is variable and not fully understood, since the development of therapies for these conditions can be particularly challenging.   Learn more about the Hub’s three primary functions from the FDA’s Patrizia Cavazzoni, MD, Director, Center for Drug Evaluation and Research and Peter Marks, MD, PhD, Director, Center for Biologics Evaluation and Research (CBER).   https://bit.ly/3WTK0TO | #RareDiseases #ClinicalResearch #DrugDevelopement #ClinicalTrials Answers Media Network

🏆 Insilico reports positive initial trial data for AI-designed #IPF drug, the latest from Longevity.Technology The 12-week Phase 2a clinical trial enrolled 71 patients across 21 sites in China, with participants receiving either a placebo or varying doses of the drug: 30 mg once or twice daily, or 60 mg once daily. The trial’s primary endpoint was safety, and secondary efficacy endpoints included changes in lung function. The results indicated that the drug was well-tolerated across all dose levels, with the highest dose group (60 mg once daily) showing the most significant improvement in FVC. “These results are very encouraging, particularly the dose-dependent response in FVC,” said trial investigator Dr Toby M Maher, an expert in interstitial lung disease. “IPF is a devastating disease, and seeing improvements in lung function over just 12 weeks of treatment is a promising indication that ISM001-055 may provide a new therapeutic option for patients.” Read the full article here: https://lnkd.in/eg45xee2 #GenAIPhase2 #Phase2AI

Chronic #HepatitisDelta is a devastating viral disease with no approved treatments in the United States. I'm energized to see today's preliminary results from Vir Biotechnology, Inc.'s Phase 2 SOLSTICE clinical trial, evaluating mono- and combination therapies in the treatment of #HepatitisDelta. It's days like today that make me even more excited to come to work tomorrow. #PatientsAreWaiting https://lnkd.in/gHHhgdVS

https://lnkd.in/gGXp3QcW New Therapeutic Strategies for Genital Herpes Request for Applications—proposed FY 2026 initiative Objective: To stimulate development of new treatments for genital herpes that suppress shedding and lesion formation and potentially reduce transmission.

For #RareDiseaseDay2024, here's a round-up of the latest breakthroughs in the treatment of rare disease from Drug Discovery World. #rarediseaseday #raredisease #drugdiscovery https://lnkd.in/exgtVJFz

The FDA has just approved donanemab (Kisunla), a monoclonal antibody developed by Eli Lilly and Company to slow the progression of early symptomatic Alzheimer’s disease. Donanemab helps remove amyloid plaque buildup in the brain, a key factor in Alzheimer’s. Clinical trials have shown significant results, with a 35% lower risk of disease progression over 18 months. While not a cure, donanemab marks the second entry into the first class of disease-modifying drugs for Alzheimer's. This incredible progress is the result of more than 20 years of biomarker research and highlights the importance of public-private collaboration in advancing such medical breakthroughs. The FNIH is committed to continuing its work in developing robust, shareable data sets and sharing knowledge, expertise, and resources to allow researchers to expand their thinking and add new knowledge to solve complex problems like Alzheimer’s and other neurodegenerative diseases. Link here: https://lnkd.in/gcdswNp7 #Alzheimers #AlzheimersResearch #Donanemab #NeurodegenerativeDisease #BiomarkerResearch #AMPAD #BiomarkersConsortium #FNIH

SciRhom GmbH has announced that the dosing of participants has commenced in the first clinical study evaluating the company’s most advanced development program, SR-878, which is a monoclonal antibody targeting inactive Rhomboid 2 (iRhom2). iRhom2 regulates the TACE-dependent release of TNF-alpha and other pro-inflammatory molecules from immune cells. The first-in-human, double-blind, placebo-controlled, single ascending dose study (NCT06607484) will assess the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of SR-878 in up to 48 healthy volunteers. #mabs https://lnkd.in/e9XXG7Fj

Vertex Pharmaceuticals to Acquire Alpine Immune Sciences for $4.9 Billion US-based Vertex Pharmaceuticals has agreed to pay approximately $4.9 billion in cash to acquire Alpine Immune Sciences, Inc., a compatriot #biotechnology company focused on discovering and developing innovative, protein-based #immunotherapies. The transaction was unanimously approved by the boards of directors of both companies and is expected to close later this quarter. Alpine’s lead molecule, povetacicept (ALPN-303), is a dual antagonist of BAFF (B cell activating factor) and APRIL (a proliferation inducing ligand). Through Phase 2 development, povetacicept has shown potential best-in-class efficacy in IgA nephropathy (IgAN), the companies said in a joint statement. IgAN is a serious, progressive, autoimmune disease of the kidney that can lead to end-stage-renal disease. So far, there are no approved therapies that target the underlying cause of IgAN. Povetacicept is on track to enter Phase 3 clinical development in the second half of 2024. Read more on #CMI online: https://lnkd.in/ebDN63QP

We explore recent developments and clinical applications, highlighting how innovative approaches like N-acetylgalactosamine (GAlNac), aptamers, antibodies, and cell-penetrating peptides (CPPs) are transforming the field. #NucleicAcidTherapeutics #Bioconjugation #ChemicalModifications #GeneticDisorders #BiomedicalResearch #AdvancedTherapeutic Free access to read the full article here: https://lnkd.in/gVr6AmPs