Congenital Hyperinsulinism International’s Post

𝐀𝐦𝐲𝐥𝐲𝐱 𝐏𝐡𝐚𝐫𝐦𝐚𝐜𝐞𝐮𝐭𝐢𝐜𝐚𝐥𝐬 𝐀𝐜𝐪𝐮𝐢𝐫𝐞𝐬 𝐀𝐯𝐞𝐱𝐢𝐭𝐢𝐝𝐞 𝐟𝐨𝐫 𝐓𝐫𝐞𝐚𝐭𝐦𝐞𝐧𝐭 𝐨𝐟 𝐇𝐲𝐩𝐞𝐫𝐢𝐧𝐬𝐮𝐥𝐢𝐧𝐞𝐦𝐢𝐜 𝐇𝐲𝐩𝐨𝐠𝐥𝐲𝐜𝐞𝐦𝐢𝐚 Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) Amylyx Pharmaceuticals today announced the acquisition of avexitide from Eiger BioPharmaceuticals, Inc. Avexitide, a novel GLP-1 receptor antagonist, has shown potential in treating hyperinsulinemic hypoglycemia, including post-bariatric hypoglycemia (PBH) and congenital hyperinsulinism (HI). The FDA has granted avexitide Breakthrough Therapy Designation for both indications. “Since Amylyx was founded, we have been guided by a rigorous approach to our science to bring potential treatments to communities with high unmet needs. When we reviewed all the compelling data supporting avexitide, it clearly aligned with our strategic scientific criteria, expertise, and community values, and we are excited to build upon the important work done to date to study this asset,” said Joshua Cohen Joshua Cohen and Justin Klee Justin Klee, Co-CEOs of Amylyx. The Phase 3 program for avexitide in PBH is expected to begin in Q1 2025, with data readout anticipated in 2026. Data from two Phase 2 studies of avexitide in PBH demonstrated highly significant reductions in severe hypoglycemic events. Avexitide was generally well tolerated, with a favorable safety profile replicated across five clinical trials in PBH. Avexitide works by binding to the GLP-1 receptor on pancreatic islet beta cells, decreasing insulin secretion and stabilizing glucose levels. Previous studies showed significant reductions in hypoglycemic events and favorable safety profiles. “PBH is a debilitating condition with no approved treatment options, and we look forward to advancing this critical work with avexitide into Phase 3 for individuals with PBH based on the totality of data from five clinical trials,” said Camille L. Bedrosian, MD Camille Bedrosian, Chief Medical Officer of Amylyx. #AmylyxPharmaceuticals #Avexitide #HyperinsulinemicHypoglycemia #PostBariatricHypoglycemia #CongenitalHyperinsulinism #GLP1ReceptorAntagonist #ClinicalTrials #Biotech #FDA

1. Q1 FY25 is described as a "breakthrough" quarter for Island Pharmaceuticals by CEO Dr. David Foster. 2. A Letter of Intent was signed with BioCryst for a potential acquisition of the antiviral molecule galidesivir. 3. The US FDA approved an amendment to the Phase 2 clinical trial protocol for ISLA-101 aimed at treating dengue fever. 4. Island Pharmaceuticals achieved several key milestones during the quarter, including updates on the ISLA-101 clinical program. 5. The Phase 2a/b clinical study for ISLA-101 has begun screening for subject enrollment following IRB approval. 6. Data from the Phase 2a prophylactic arm is expected by the end of the calendar year, with analyses of various factors planned. 7. The company announced board changes, including the appointment of Chris Ntoumenopoulos and the retirement of Dr. David Brookes. 8. A $3.5 million funding placement was completed post-quarter, enhancing financial stability for upcoming clinical milestones and pipeline development.

Intrigued about the recent developments on #denosumab, #ustekinumab, #ranibizumab, and #adalimumab? Look no further than the Pearce IP #BioBlast which reports on global #BioPharma developments weekly. The complex made simple!

🤔 What is the #CompetitiveLandscape for Thalassemia? Agios Pharmaceuticals posting reductions in blood transfusions for patients with #betathalassemia; from results of a PIII clinical trial of its oral, mitapivat. 💉 The nearest competitor from Bristol Myers Squibb, sc Reblozyl, have not had a H2H eval. 🆚 Others in the transfusion-dependent b-thalassemia space include GTs Zynteglo (bluebird bio) and Casgevy (Vertex Pharmaceuticals and CRISPR Therapeutics). 💊 Convenience of oral dosing will be a given... What's next? 📅 US filing; potential approval by EO24 https://lnkd.in/eXwjBwvr #Agios #RareDisease #CompetitiveIntelligence #MarketIntelligence #CompetitiveLandscape #MedicalAffairs #GeneTherapy #RegulatoryAffairs #Pharma #Healthcare

FDA approves Biocon Biologics' Yesintek, Bmab 1200 biosimilar to J&J's Stelara (ustekinumab) FDA has approved Yesintek (ustekinumab-kfce - Biocon Biologics), a biosimilar to Stelara (Johnson & Johnson). The monoclonal antibody is approved to treat Crohn disease, ulcerative colitis, plaque psoriasis, and psoriatic arthritis. Under a settlement and licensing agreement with Janssen Biotech, Janssen Sciences Ireland, and Johnson & Johnson that was announced in February, Biocon Biologics will commercialize Yesintek in the United States by February 22, 2025, following FDA approval. https://buff.ly/4fUIZCb

@Ainos, Inc., an innovative healthcare company focused on advanced artificial intelligence (AI)-driven point-of-care testing (POCT) and low-dose interferon therapeutics, has announced the signing of a strategically significant Memorandum of Understanding (MoU) with Taiwan Tanabe Seiyaku Co., a subsidiary of Mitsubishi Tanabe Pharma Corporation in Japan, bringing extensive pharmaceutical development and manufacturing expertise to the partnership. https://lnkd.in/dkWMSa64 Tanabe Seiyaku Co LTD #Healthcare #pharmaceuticalindustry #Healthtech

🔬 Factor XI in Thrombosis: Insights and Research Products Discover the pivotal role of Factor XI in thrombosis and hemostasis. This news item delves into the mechanisms of thrombin generation, the clinical implications of Factor XI deficiency, and the latest research and pharmaceutical developments. Explore how Prolytix's high-quality research products can advance your understanding and treatment of thrombotic conditions. 🧪 Key Highlights: - Mechanisms of thrombin generation and the role of Factor XI - Clinical insights into Factor XI deficiency and its implications - Cutting-edge research products from Prolytix, including human Factor XI, Factor XIa, and specialized antibodies

Ozempic demonstrates significant reduction in CKD risks🩸 Novo Nordisk Know complete story - https://lnkd.in/dxJkXZPz Zentalis Pharmaceuticals announces two key leadership appointments👩💼 Know complete story - https://lnkd.in/duNXjWPn Keros Therapeutics updates TROPOS trial with new dosing guidelines💊 Know complete story - https://lnkd.in/dsHfgS6d FDA grants MUMS status to Medicus Pharma Ltd. for a rare disease therapy✅ Know complete story - https://lnkd.in/dT8PPEQh #Ozempic #CKDRisks #Zentalis #Leadership #Keros #TROPOSTrial #MedicusPharma #FDAApproval

DelveInsight’s 2023 analysis reports that the inflammatory bowel disease (IBD) market includes ∼5 million diagnosed cases of ulcerative colitis and Crohn’s disease across the 7MM. The market is anticipated to grow significantly, driven by advancements in treatment options and increasing patient needs. Key companies making strides in the IBD space include Arena Pharmaceuticals, Inc., Pfizer, Abivax, The Janssen Pharmaceutical Companies of Johnson & Johnson, Celgene, and others. Notable emerging therapies are Etrasimod (APD334), obefazimod (ABX464), TREMFYA (guselkumab), and ZEPOSIA (ozanimod). These innovative treatments are set to enter the market soon and are expected to bring transformative changes to IBD management. Get More Detailed Information: https://lnkd.in/gKExmEBg #InflammatoryBowelDisease #IBD #HealthcareMarket #PharmaceuticalInnovation #Etrasimod #ABX464 #TREMFYA #ZEPOSIA #DelveInsight #MarketAnalysis #Biopharma

🔬 Factor XI in Thrombosis: Insights and Research Products Discover the pivotal role of Factor XI in thrombosis and hemostasis. This news item delves into the mechanisms of thrombin generation, the clinical implications of Factor XI deficiency, and the latest research and pharmaceutical developments. Explore how Prolytix's high-quality research products can advance your understanding and treatment of thrombotic conditions. 🧪 Key Highlights: - Mechanisms of thrombin generation and the role of Factor XI - Clinical insights into Factor XI deficiency and its implications - Cutting-edge research products from Prolytix, including human Factor XI, Factor XIa, and specialized antibodies