Tsung Tan’s Post

Thank you for highlighting our recent publication authored by Drs. Valentina Di Francesco, et al., in the Journal of Controlled Release. The Minimally Invasive Nasal Infusion (MINI) approach represents a significant advancement in the delivery of protein therapeutics to the central nervous system (CNS). This method addresses the challenges posed by the blood-brain barrier, which traditionally limits the effectiveness of CNS drug delivery. By utilizing a programmable infusion pump in a mouse model, the MINI approach allows for the precise administration of therapeutics, such as ovalbumin, directly to the CNS. This technique not only improves the bioavailability of the drugs but also minimizes the invasiveness of the procedure compared to other direct CNS administration methods. The potential of this approach is vast, offering a promising avenue for treating CNS diseases with large-molecule therapeutics that would otherwise be unable to cross the blood-brain barrier efficiently.

Microscopic hematuria has emerged as a biomarker of activity in patients with IgAN, independent of proteinuria, but even more so in the presence of proteinuria. It is time to incorporate this cheap and easy test in our routine assessment of and decision on immunosuppression in a IgAN patient. Hematuria should be included into clinical trials to test drugs targeting the inflammatory reaction elicited by immune pathway activation in IgAN. Clinical Kidney Journal, Volume 16, Issue Supplement_2, December 2023, Pages ii19–ii27 https://lnkd.in/d4NPja7v

ENYO Pharma is very pleased to announce the start of its Alport syndrome clinical trial “ALPESTRIA-1” with the first site opened for patient recruitment in Idaho (US). 📃 The ALPESTRIA-1 study is a Phase 2 clinical study evaluating the safety, tolerability and benefit of three dose levels of the investigational drug Vonafexor on renal function and biomarkers in patients with Alport syndrome. The study aims to recruit 20 patients in the US and EU (France, Spain and Germany). 💊 Vonafexor is a clinical-stage compound with strong fibrolytic & anti-inflammatory effects already shown in the Phase 2 LIVIFY study and in various preclinical studies in Alport syndrome and severe Chronic Kidney Disease (CKD). Vonafexor has been administered to over 300 individuals in 9 clinical studies. 👨⚕️ Alport syndrome is a rare kidney disease, a genetic disorder affecting 1 in 5,000-10,000 births. It is characterized by a progressive loss of kidney function. To date, no cure is available for patients suffering from Alport syndrome. ❓ For more information about the study, please visit: ENYO Pharma: https://lnkd.in/eUJkvbDa Clinicaltrialsgov: https://lnkd.in/ecGjCDUU ASF: https://lnkd.in/etE2h77r We deeply thank all the involved stakeholders for their great work and support. #alportsyndrome #clinicaltrial #kidneydisease #drugdevelopment

In our latest review we discuss nucleic acid-based/targeted therapeutics to treat dyslipidemia and arterial hypertension. From the well-known #PCSK9 to the emerging #ANGPTL3; from  #siRNA inclisiran, which is already used in clinical practice, to the base editor #Verve-102, which has just entered a human trial - we tried to summarize the latest evidence and news in this very exciting and rapidly developing field.  #Cardiovascular Research  https://lnkd.in/evG4YiRx

📢 #Lilly's Kisunla (donanemab-azbt) approved by the FDA for the treatment of early symptomatic Alzheimer's Disease! The U.S. Food and Drug Administration (#FDA) approved Eli Lilly’s #donanemab-azbt (350 mg/20 mL once-monthly injection for IV infusion), sold under the brand name #Kisunla for the treatment of early symptomatic Alzheimer's disease (AD) in adults, which includes people with #mildcognitiveimpairment (MCI) as well as people with #mild #dementia stage of AD, with confirmed amyloid pathology. Once-monthly Kisunla is the first and only amyloid plaque-targeting therapy with evidence to support stopping therapy when #amyloidplaques are removed, which can result in lower treatment costs and fewer infusions. In its phase 3 TRAILBLAZER-ALZ 2 study, Kisunla slowed cognitive and functional decline by up to 35% (in those with less advanced disease) and 22% (in the overall population) compared to placebo, at 18 months. In both the groups, Kinsula reduced participants' risk of progressing to the next clinical stage of disease by up to 39% vs. placebo. A key differentiating factor for donanemab is the drug's finite dosing, which allows patients to stop taking the treatment once brain scans no longer show amyloid plaques. Like #Eisai, and #Biogen rival drug #Leqembi, which was approved a year ago, donanemab is designed to clear an Alzheimer's-related protein called beta amyloid from the brain. According to an article from Reuters, Lilly priced its drug at $695.65 per vial, or about $32,000 for 12-months of treatment consisting of 13 infusions. That's slightly higher than Eisai's Leqembi, which costs $26,500 a year. Detailed news here: https://lnkd.in/gADVZ9jQ   Stay updated on the latest trends and news in the pharmaceutical industry. Follow #RASLSS: https://lnkd.in/de5zNWmK   #EliLilly #Kisunla #donanemab #Alzheimerdisease #Drugapproval #USFDA #raslss  

"#Endothelin peptides (ET-1, ET-2, and ET-3) and their receptors ETA and ETB are pivotal regulators of cardiovascular functions, essential for maintaining vascular tone and overall cardiovascular homeostasis. Therapeutically targeting endothelin receptors, particularly through ETA #Antagonists, has shown #Efficacy in treating pulmonary arterial #Hypertension (PAH) and other cardiovascular- and renal-related diseases making ETA an important #DrugTarget! The structural basis for ETA #Antagonism remains less understood, a gap that limits #DrugDesign of selective antagonists. In addition to #SmallMolecule antagonists, a monoclonal #Antibody (Fab301) specifically targeting ETA is currently in phase Ib clinical trials. The specificity of antibodies may address the selectivity issues associated with small molecule antagonists for ETRs. However, there is currently no structural information on these antibody– #Receptor complexes!" Do not miss this fantastic open-access article published in Nature Portfolio Cell Discovery two days ago, in which Zhe Luo, Tian Hua and Changhong Miao from Fudan University and ShanghaiTech University revealed the #CryoEM structures of ETA in complex with two PAH drugs, #Macitentan and #Ambrisentan, along with #Zibotentan, a selective ETA antagonist, and the monoclonal antibody Fab301: https://lnkd.in/e2SBdD2K These structures, together with the active-state structures of ET-1-bound ETA and ETB, and the agonist BQ3020-bound ETB, in complex with Gq, unveil the #MechanismOfAction (MoA) of agonist/antagonist binding modes that establish a robust theoretical framework for #RationalDesign of small molecule drugs and antibodies with selective activity against endothelin receptors! If you are interested in how Cryo-EM is revolutionizing #DrugDiscovery, please follow this weblink to gain some deeper insights from there: https://lnkd.in/eXW7K_U6

The Foundation for the National Institutes of Health (FNIH) announces #research effort to speed #Parkinsons diagnosis and enable earlier intervention. The Accelerating Medicines Partnership® in Parkinson’s Disease and Related Disorders (AMP® PDRD), managed by the FNIH, brings together the resources and expertise of its public sector partners such as the National Institute of Neurological Disorders and Stroke (NINDS), the National Institute on Aging (NIA) at the The National Institutes of Health and the FDA. Private sector partners include The Michael J. Fox Foundation for Parkinson's Research, Sanofi, GSK, Denali Therapeutics, CurePSP, C2N Diagnostics and Aligning Science Across Parkinson’s | ASAP. The $21M commitment aims to identify unique #biomarkers that distinguish #ParkinsonsDisease from other conditions with similar pathologies to help decrease a risk for misdiagnosis that could undermine #clinicaltrials. #collaboration #partnership 

The U.S. Food and Drug Administration (#FDA) granted full approval to #TIVDAK (tisotumab vedotin-tftv) on April 29, 2024. This endorsement is based on the pivotal #Phase 3 innovaTV 301 study, which showed a statistically significant improvement in overall survival for patients with recurrent or metastatic #cervical cancer who had previously received chemotherapy. Indication: #TIVDAK is approved for use in adults with cervical cancer that has recurred or spread and has worsened during or after chemotherapy treatment. Trial Outcomes: The #innovaTV 301 trial achieved its main goal, demonstrating a 30% decrease in mortality risk compared to the chemotherapy group. The average survival time for those receiving TIVDAK was 11.5 months, in contrast to 9.5 months for those undergoing #chemotherapy. #Dosage: TIVDAK is administered at a dose of 2 mg/kg (capped at 200 mg) through an intravenous infusion lasting 30 minutes, repeated every three weeks until the cancer progresses or side effects become too severe. #Safety Information: The prescribing information for TIVDAK carries a prominent warning about the risk of eye toxicity. Other significant risks include nerve damage, bleeding, lung inflammation, serious skin reactions, and potential harm to an unborn baby. As the first ADC to demonstrate a significant extension in survival times for this patient group, TIVDAK’s full approval marks a major milestone in treatment options for women with advanced cervical cancer. #oliveridleyadvisory #oncology #cancer #cervicalcancer #clinicaltrials #chemotherapy

Multiple Sclerosis Treatment Advances: Roche's Fenebrutinib Shows Promise in Suppressing Disease Activity and Progression Roche has presented promising data from its Phase II FENopta open-label extension study, showcasing the efficacy of its investigational Bruton’s tyrosine kinase inhibitor, fenebrutinib, in treating relapsing multiple sclerosis (RMS). The results, which will be shared at the 40th Congress of the European Committee for Treatment and Research in Multiple Sclerosis in 2024, revealed that 96% of patients remained relapse-free, with significant reductions in brain lesions and no progression in disability after 48 weeks. Roche's Chief Medical Officer, Dr. Levi Garraway, highlighted the potential of fenebrutinib to transform RMS treatment pending further validation from ongoing Phase III trials. For more details please click the link! https://lnkd.in/dQrAZEpR #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical 

A New Hope for Inflammatory Bowel Disease? AI-Created Drug Enters Human Testing After Showing Promise in Early Research- A new AI-designed drug for inflammatory bowel disease has entered human clinical trials, representing a milestone for computer-aided drug discovery. - Inflammatory bowel disease (IBD), which afflicts over 1.6 million Americans, may soon have a new treatment option thanks to artificial intelligence. This past week, Insilico Medicine announced that its AI-designed drug for IBD, called ISM5411, has entered Phase I clinical trials. If approved, th READ HERE https://lnkd.in/dWFueSbc #AI #clinicaltrials #drugdiscovery #IBD #inflammatoryboweldisease