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“Transforming Lives of Cystic Fibrosis Patients: A New Era for Orphan Drugs 🌟” “Cystic fibrosis is one of the rare diseases that has challenged the medical community for decades. However, with the introduction of breakthrough treatments like Trikafta, hope is now closer than ever. This revolutionary therapy addresses the root cause of the disease, leading to significant improvements in lung function and quality of life for over 90% of eligible patients. Advancements in orphan drugs are not just scientific milestones but also messages of hope for millions of patients worldwide who silently endure the challenges of rare diseases. How can we encourage more investment in scientific research to provide innovative solutions for these underserved patient populations? Let’s discuss!” #CysticFibrosis #OrphanDrugs #HealthcareInnovation #ScientificResearch# vertex pharmaceutical

Pathalys pharma secures $105 million series B financing. Oversubscribed financing paves path to NDA submission and pre-commercialization efforts for upacicalcet.  Upacicalcet is a novel calcimimetic that can be delivered intravenously at the end of the hemodialysis session via pre-filled syringe and has been demonstrated to effectively reduce levels of parathyroid hormone(PTH) and may have a better tolerability profile for patients. Upacicalcet can be used in secondary hyperparathyroidism (SHPT) to address an unmet need in the management of advanced chronic kidney disease (CKD). Chemenu has been working to develop more compounds for drug discovery. Here comes the building blocks we can provide: https://lnkd.in/gsg24G8S Keywords: Upacicalcet; CaSR; secondary hyperparathyroidism (SHPT); building blocks  

🌟 Advancing the Future of MASH Treatment 🌟 💊 Combination therapies are becoming more popular in the MASH treatment landscape, as these are expected to unlock new possibilities for patient care. 🔬 GLP-1 drugs—such as Eli Lilly and Company’s tirzepatide and Novo Nordisk’s semaglutide—are proving effective in addressing weight loss and inflammation, while MASH-specific therapies directly target disease progression. 🚀 Pioneers like Madrigal Pharmaceuticals, Akero Therapeutics, and Sagimet Biosciences are at the forefront, exploring synergistic potential between GLP-1s and MASH-targeted treatments. 💡 As these innovative combinations gain momentum, one critical question remains: Could this be the key to achieving superior patient outcomes? 📢 Comment your thoughts below 👇 and receive access to the full article! #AtacanaGroup #MASH #MASLD #PharmaTrends #Biopharma #HealthcareStrategy #GLP-1

💊 Clopidogrel ranks as one of the most prescribed antiplatelet medications in North America. (1) However, just 15% of the drug is metabolized by CYP450 enzymes into its active form. (1) Among these enzymes, CYP2C19 stands out with the most significant role in both stages of bioactivation. (1) Thus, clinical outcomes of patients treated with clopidogrel is impacted by CYP2C19 genotype (1). Discover how Genomadix can aid in CYP2C19 genotype-guided antiplatelet therapy for personalized treatment at www.genomadix.com  🧬 #Pharmacogenetics #PrecisionMedicine #PersonalizedMedicine #stroke #PCI   1.Lee CR, Luzum JA, Sangkuhl K, et al. Clinical Pharmacogenetics Implementation Consortium Guideline for CYP2C19 Genotype and Clopidogrel Therapy: 2022 Update. Clin Pharmacol Ther. 2022;112(5):959-967. doi:10.1002/cpt.2526

Precision Medicine Online, a leading pharmaceutical magazine, presented PolTREG to its U.S. readers in a broad-ranging interview with CEO Prof Piotr Trzonkowski. PolTREG’s investigational Treg therapies “could halt progression, or possibly even cure some patients with type 1 diabetes”, is how the magazine put it. Much of the interview focused on PolTREG’s Phase 2 clinical trial in presymptomatic diabetes patients, children for whom PolTREG hopes its treatment with its cellular therapy may mean they stay in clinical remission for the rest of their lives. But reporter Jessica Kim Cohen also talked about PolTREG’s future plans to develop therapies for auto-immune diseases like multiple sclerosis, rheumatoid arthritis, inflammatory bowel disease and amyotrophic lateral sclerosis. For a look at the story, click here (subscription required): https://lnkd.in/dZp5sBvm #T1D #biotech #biotechnology #celltherapy #cellandgenetherapy #autoimmunedisease #diabetes #type1diabetes #multiplesclerosis

PolTREG S.A. - Precision Medicine Online a leading pharmaceutical magazine, presented PolTREG to its U.S. readers in a broad-ranging interview with CEO Prof Piotr Trzonkowski. PolTREG’s investigational Treg therapies “could halt progression, or possibly even cure some patients with type 1 diabetes”, is how the magazine put it.

Nature’s Hidden Gems: Scorpion Venom & Vipera Lebetina Extracts Unlock groundbreaking possibilities in modern medicine with the extraordinary potential of Scorpion Venom and Vipera Lebetina extracts. These APIs are rich in bioactive components, offering innovations in cancer treatment, cardiovascular health, and more. With direct sourcing, unmatched quality, and a reliable supply chain, we prioritize trust, sustainability, and collaboration. 📩 Let’s connect! Message us to explore how these rare treasures can elevate your pharmaceutical innovations. Revolutionizing healthcare, one discovery at a time. #PharmaceuticalInnovation #ScorpionVenomAPI #ViperaLebetinaExtract #ActivePharmaceuticalIngredients #HealthcareSolutions #BiotechnologyResearch #ModernMedicine #R&DInnovation #PharmaExcellence #NatureInPharma #SustainableAPIs #MedicalBreakthroughs #DrugDevelopment #PharmaPartnerships #InnovativeHealthcare #i_daru #GlobalCollaboration #ModernMedicines

In the article below, KYBORA's Jean Chatellier, Dr. Nana Mainoo at Cleracs Consulting and Christian Girard at The PRV Fund Project, delve into the role of the FDA’s “not same active moiety” principle, and how it plays a key role in evaluating eligibility for Rare Pediatric Disease Designation (RPDD) and Priority Review Vouchers (PRV). Kindly click below to read and download the article. https://lnkd.in/dSNUj-56 #KYBORA #biotech #biotechnology #pharma #biopharma #pharmaindustry #lifesciences #PRV #PriorityReviewVoucher #Innovation #PediatricHealth #RegulatoryStrategy #RareDiseases #RPDD

Chronic pain is common, afflicting about 20% of the population. Yet, good biomarkers are lacking, pharmaceutical therapies only work to some degree in some patients, and novel analgesics in the pain field have a terrible track record. I believe these are signs that we need a better logical framework for how we think about pain disorders - and I also believe that the immune system offers an interesting way forward in this regard. I wrote a letter about this, go check it out: https://lnkd.in/dZu6XUYz

MSD and Eisai Co., Ltd. Report Latest Data from P-III (LEAP-015) Trial of Keytruda (pembrolizumab) and Lenvima (lenvatinib) Regimen to Treat Gastroesophageal Adenocarcinoma #merck #eisai #keytruda #lenvima #gastroesophagealadenocarcinoma #clinicaltrial #pharmashots