Foundation for the National Institutes of Health’s Post

🌟 Exciting Update! 🌟 We have received FDA approval for a new Phase 3 clinical trial! Thanks to the promising outcomes from our Phase 0/2 study in collaboration with GSK, we're fast-tracking the Gliofocus Study. This global trial will compare niraparib against temozolomide in treating newly diagnosed MGMT-unmethylated #glioblastoma. 🌍 Learn more: https://loom.ly/ckeho64

US Rare Disease Biotech leaders: A quick 5-minute read on leveraging HTA assessments to strengthen your US payer negotiations. https://lnkd.in/ewC8CKt7 Follow Partner Rare for more bite-sized insights. If you're approaching a new Rare Disease project, get in touch - no pressure, just helpful expertise. 💚 #raredisease #biotech #pharma #HTA #marketentry #FDA #EMA #PartnerRare

𝗚𝗮𝗶𝗻 𝗧𝗵𝗲𝗿𝗮𝗽𝗲𝘂𝘁𝗶𝗰𝘀 𝗮𝗻𝗻𝗼𝘂𝗻𝗰𝗲𝘀 𝗽𝗿𝗼𝗺𝗶𝘀𝗶𝗻𝗴 𝘁𝗼𝗽𝗹𝗶𝗻𝗲 𝗿𝗲𝘀𝘂𝗹𝘁𝘀 𝗳𝗿𝗼𝗺 𝗣𝗵𝗮𝘀𝗲 𝗜 𝗰𝗹𝗶𝗻𝗶𝗰𝗮𝗹 𝘁𝗿𝗶𝗮𝗹 𝗼𝗳 𝘁𝗵𝗲𝗶𝗿 𝗱𝗿𝘂𝗴 𝗰𝗮𝗻𝗱𝗶𝗱𝗮𝘁𝗲 𝗳𝗼𝗿 𝗣𝗮𝗿𝗸𝗶𝗻𝘀𝗼𝗻'𝘀 𝗱𝗶𝘀𝗲𝗮𝘀𝗲. The trial, involving 72 healthy volunteers, demonstrated that GT-02287 is safe and well-tolerated across all dose levels, with no serious adverse events reported. This brain-penetrant small molecule aims to address Parkinson’s symptoms by reducing neuroinflammation, and with positive safety data Gain Therapeutics plans to initiate a Phase II trial in Parkinson’s patients by Q4 2024, aiming to further validate the drug’s efficacy and mechanism. #Pharma #ClinicalTrials #ParkinsonsDisease #Innovation #DrugDevelopment #GainTherapeutics

Nearly 13 million adults in the U.S. suffer from #IBSD. In recognition of #IBSAwarenessMonth, we continue raising awareness of the need for better therapeutic options for #IBSD that are safe, effective & convenient to effectively manage this chronic and debilitating condition.      CinRx portfolio company CinPhloro Pharma is working to address this need with CIN-103, a program in Phase 2 clinical investigation for chronic IBS-D.     Learn more about CinPhloro at https://lnkd.in/gNfzEtTM and the ongoing IBS-D clinical trial at https://lnkd.in/dMNaA-cg  

Datapoint: FDA Approves Lilly’s New Alzheimer’s Drug: The FDA on July 2 granted full approval to Eli Lilly’s Kisunla (donanemab) for the treatment of Alzheimer’s disease. The new therapy joins Eisai and Biogen’s Leqembi (lecanemab) in the burgeoning Alzheimer’s market basket. The two drugs’ in... Read More #marketaccess #lifesciences #pbm

Groundbreaking research news: CurePSP has joined the Accelerating Medicines Partnership® in Parkinson’s Diseases and Related Disorders (AMP® PDRD). This initiative, led by the Foundation for the National Institutes of Health (FNIH), aims to better differentiate Parkinson’s disease from related neurodegenerative disorders, including progressive supranuclear palsy (PSP), multiple system atrophy (MSA) and corticobasal degeneration (CBD), to enable earlier diagnoses, more timely interventions and better outcomes. CurePSP is committed to ensuring that this focus on early and accurate diagnosis for PSP, MSA, and CBD drives meaningful progress in improving patient care. We join this effort alongside several other key partners, including Aligning Science Across Parkinson’s | ASAP, C2N Diagnostics, Denali Therapeutics, GSK, The Michael J. Fox Foundation for Parkinson's Research and Sanofi, all working together to push the boundaries of research to make a tangible impact.  #PSP #MSA #CBD #Parkinsons #Neurodegeneration #FNIH #CurePSP #EarlyDiagnosis #NIH

Adoption of legislation for joint EU HTA Assessment has initiated a new era, with the Joint Clinical Assessment (JCA) being implemented for cancer and Advanced Therapy Medicinal Products (ATMPs) from January 2025. Our MD Anand Jha is moderating a session on this topic on 7th October, 2024 at the Pharma Marcon conference in Berlin, with fellow panelists Sandeep Kiri Stephanie Dr. med. Rosenfeld Inka Heikkinen Thomas Grub to look at the challenges that the companies will face for access planning and how to address them. The discussion will cover multiple aspects that the industry and multiple stakeholder groups are grappling with... Anastasia Trubnikova #euhta #healthpolicy #patientaccess #eunethta

Innovation opens opportunities for patients and caregivers, especially with cell and gene therapies offering the chance to tackle intractable diseases…but what happens if we cannot match innovation in science with innovation in our healthcare systems? Let’s discuss at the Pharma MARCON.

H.C Weinwright analyst: #CardiolRx Poised for the Final Stretch; Initiating With a Buy Rating and $9 PT (1) CardiolRx is a Phase 2 stage, pharmaceutical-grade, proprietary cannabidiol oral solution in development as a treatment for recurrent #pericarditis (RP) and acute #myocarditis (AM); (2) Cardiol’s use of a deliberate, scientific evidence-based approach to CardiolRx clinical advancement creates a significant barrier to entry; and (3) CardiolRx targets RP and AM, heart disease indications with significant unmet medical need with high potential to make CardiolRx a blockbuster drug that we forecast could achieve >$1.3B in peak annual sales. #CRDL #Cardiol #HeartDisease #CVD

Topia’s First Repurposed Drug Study for Alzheimer’s: TOP-RPBE001 Read the full article: https://lnkd.in/dgj4_6eY   We are thrilled to announce a major milestone in our journey to combat Alzheimer’s disease. Topia's first repurposed drug study, TOP-RPBE001, has demonstrated promising results in our in-vitro studies. TOP-RPBE001 effectively blocks enzymatic hydrolysis and facilitates the transportation of acetylcholine (Ach), a crucial neurotransmitter in the brain. This advancement represents a significant step forward in our mission to repurpose existing drugs to provide innovative and effective treatments for Alzheimer's disease. At Topia, we are committed to pioneering research and innovative approaches to tackle some of the most challenging health issues of our time. The success of TOP-RPBE001 is a testament to the dedication and expertise of our incredible team of scientists and researchers. Stay tuned for more updates as we move forward with further studies and clinical trials. Together, we can make a difference in the fight against Alzheimer's.   #AlzheimersResearch #DrugRepurposing #TopiaLifesciences #InnovativeHealthcare #NeurologicalHealth #Acetylcholine #BiotechBreakthrough #PharmaceuticalResearch #HealthcareInnovation #CognitiveHealth