Gain Therapeutics’ Post

Cardiol Therapeutics Inc. (@cardiolrx) recently announced positive topline results from its Phase II MAvERIC-Pilot study, investigating the impact of CardiolRx™ on patients with symptomatic recurrent pericarditis. The study enrolled 27 adult patients across prominent clinical sites in the U.S., including the Mayo Clinic, Cleveland Clinic, and Massachusetts General Hospital. The results demonstrated a significant reduction in pericarditis pain, with the mean pain score dropping from 5.8 at baseline to 2.1 (64%) after eight weeks of treatment. Additionally, 80% of patients with elevated baseline C-reactive protein (CRP) levels achieved normalization, indicating a substantial reduction in inflammation. 'We are delighted to share the exceptional primary endpoint data from the MAvERIC-Pilot study, which demonstrated that oral administration of our small molecule CardiolRx™ led to marked reductions in pericarditis pain and inflammation,' said David Elsley, President & CEO of Cardiol Therapeutics. With an estimated 38,000 patients in the U.S. experiencing recurrent pericarditis annually, the need for effective treatments is critical. Cardiol Therapeutics aims to provide an accessible and non-immunosuppressive therapeutic option, potentially surpassing existing therapies. The positive results set the stage for a forthcoming Phase III trial. #RecurrentPericarditis #CardiolTherapeutics #CardiolRx #ClinicalTrials #Biotechnology #Healthcare @cardiolrx @mayoclinic @clevelandclinic @massgeneralnews

Sirius begins patient dosing in phase 1 trial of next─generation, long─acting factor XI siRNA anticoagulant to treat thromboembolic disorders

Hemab Therapeutics Presents Positive Phase 1 Results for HMB-001 in Glanzmann Thrombasthenia at 2024 EAHAD Annual Congress "The first clinical data for HMB-001 in Glanzmann suggest that the demonstrated mechanism is suitable as a new prophylactic treatment for people with neglected blood clotting disorders who face severe, potentially life-threatening bleeds every day," said Joe Vogel, Senior Director and Program Lead for HMB-001 at Hemab. "With Phase 2 already underway, we are committed to advancing clinical evaluation of HMB-001 toward bringing life-changing preventative treatments to improve patients' quality of life." VINODKUMAR VORA Nimish Dudhatra https://lnkd.in/d8dfjXwh

Another day, another life-changing scientific break through. Parkinson's particularly hits a raw nerve. Anyone who's seen a senior citizen suffering from this terrible affliction has got to feel some joy today. The bizarre paradox of America: The world's best medical innovation ecosystem coupled with the world's worst care delivery system. And "system" is being generous. The better word is probably "situation." #Biotech #Biotechnology #Bigpharma #Pharmaceutical #Parkinsons #Neurology

Exciting to be progressing to Phase 2 for Diabetic Foot Ulcers (DFU) with our Purified Exosome Product (PEP) #diabeticfootulcer #woundhealing #exosomes #biologics #regenerativemedicine #clinicaltrials

We are proud to announce the initiation of our Phase 2A study, which aims to assess the efficacy and safety of our novel regenerative exosome therapeutic, Purified Exosome Product™ (PEP™), in managing Diabetic Foot Ulcers (DFU). This significant milestone highlights our commitment to revolutionizing regenerative medicine and addressing critical unmet healthcare needs in chronic diseases like DFUs.   Building upon our Phase 1B study's success at Mayo Clinic, RION's Phase 2A trial marks a pivotal advancement in creating regenerative biologics for Diabetic Foot Ulcers. This rigorous, randomized, multi-center study is set to enroll 40 participants throughout the United States, carefully stratified into two groups to compare the standard of care against the innovative topical treatment with PEP™ exosomes. The findings will be instrumental, shaping the trajectory for future clinical research and laying the groundwork for a Biologics License Application (BLA)—a critical step in meeting the urgent need for new therapeutic options in diabetic wound care.   For more information about our trial and deeper insight into our breakthrough regenerative exosome technology, please read our press release by clicking the link below.   https://lnkd.in/ervYnYQU #Rion #RegenerativeMedicine #Exosomes #DiabeticFootUlcer #Diabetes

In one of the most highly anticipated clinical trial readouts this year, an #RNA silencing treatment developed by Alnylam Pharmaceuticals reduced the risk of death, heart-related hospital visits and hospitalizations by 28% compared to placebo in patients with a heart muscle disease. The results, announced Monday, pave the way for Alnylam’s therapy vutrisiran to become the first RNA silencing therapy on the market for ATTR amyloidosis with cardiomyopathy, a condition in which misfolded proteins build up in the heart and can result in heart failure. If approved, Alnylam could enter a multibillion-dollar market currently dominated by Pfizer. #clinicaltrials #clinicalresults #readout #RNAsilencing #heartmuscledisease #vutrisiran #heartdisease #cardiology #cardio #ATTRcardiomyopathy #patients #PhaseIII #clinicaldata

New, Emerging Treatment Options for Patients With Multiple Myeloma: In addition to emerging treatment options, the speakers addressed the importance of individualized decision-making when treating patients with multiple myeloma. #finance #pharmacy #lifesciences

Alentis Therapeutics is moving forward with new clinical advancements!   Our portfolio company today announced the first patient dosed in a Phase 2 clinical trial of lixudebart (formerly named ALE.F02), a Claudin-1 targeting investigational antibody for the treatment of organ fibrosis.   The study evaluates the drug’s safety, tolerability, pharmacokinetics, and how well it protects against the loss of kidney function in patients with Antineutrophil Cytoplasmic Antibody (ANCA) associated vasculitis with Rapidly Progressive Glomerulonephritis (RPGN).   It is the second clinical trial investigating lixudebart in patients after the initiation of a clinical study in advanced liver fibrosis patients earlier this year.   Read more 👉 https://lnkd.in/eiHUTCnE   #fasterforthepatient #clinicalstudy #fibrosis #biopharma

This is a great example of immunophenotyping on clinical trials. This is from Candel Therapeutics's June 2024 presentation (https://lnkd.in/g9DmSu2i), parts of which were shown at ASCO this weekend. For the uninitiated, in the first slide, you can see the injections generate an immune response at the right times. In the second slide, using multi-parameter cytometry, you can see the detail. The second slide compares changes in immune cells between the first and second CAN-2409 injections in patients who survived less than 24 months versus those who survived 24 months or more. Using multi-parameter cytometry, you can see that patients with a stronger immune activation after the second injection (the top right quadrant) tended to live longer.