Pharmaceutical Business Review International’s Post

Vertex Pharmaceuticals and CRISPR Therapeutics Received Conditional Marketing Authorization for Casgevy by the United Kingdom MHRA to Treat Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia #vertex #crisprtherapeutics #casgevy #sicklecelldisease #transfusiondependentbetathalassemia #regulatory #clinicaltrial #unitedkingdom #mhra #medicinesandhealthcareproductsregulatoryagency #exagamglogeneautotemcel #conditionalmarketingauthorization #transfusionindependent #innovationpassport #innovativelicensingandaccesspathway

iRegene Receives IND approval from U.S. FDA to Start Clinical Trial for Parkinson’s Disease - https://lnkd.in/gbKS7Z8M On June 20 (UTC-4), the U.S. Food and Drug Administration (FDA) approved the IND application for NouvNeu001, a cell therapy product from iRegene Therapeutics targeting Parkinson’s Disease. This marks a significant milestone as NouvNeu001 becomes the world’s first chemically induced allogeneic cell therapy product to enter clinical stages for Parkinson’s disease in the US. Previously, the “Combined Phase I/II Clinical Study” of NouvNeu001 had already received approval from China NMPA in August 2023 and demonstrated positive safety and efficacy data in its Phase I trials. On June 20, 2024, iRegene further announced IND approval of NouvNeu001 by the U.S. FDA for the treatment of mid-to-late stage of Parkinson’s disease. Read more at: https://lnkd.in/gbKS7Z8M #parkinsonsdisease #regenerativemedicine #stemcell

iRegene Receives IND approval from U.S. FDA to Start Clinical Trial for Parkinson’s Disease - https://lnkd.in/g6vekNF6 On June 20 (UTC-4), the U.S. Food and Drug Administration (FDA) approved the IND application for NouvNeu001, a cell therapy product from iRegene Therapeutics targeting Parkinson’s Disease. This marks a significant milestone as NouvNeu001 becomes the world’s first chemically induced allogeneic cell therapy product to enter clinical stages for Parkinson’s disease in the US. Previously, the “Combined Phase I/II Clinical Study” of NouvNeu001 had already received approval from China NMPA in August 2023 and demonstrated positive safety and efficacy data in its Phase I trials. On June 20, 2024, iRegene further announced IND approval of NouvNeu001 by the U.S. FDA for the treatment of mid-to-late stage of Parkinson’s disease. Read more at: https://lnkd.in/g6vekNF6 #parkinsonsdisease #regenerativemedicine #stemcell

Longitude portfolio company Vera Therapeutics, Inc. shared exciting news at the recent European Renal Association (ERA) Congress: data presentations from its Phase 2b ORIGIN trial of atacicept in immunoglobulin A nephropathy (IgAN) showing that atacicept stabilized kidney function through 72 weeks and led to rapid improvements in hematuria. “For the first time in this field, we presented 72-week data from our Phase 2b ORIGIN trial showing stable kidney function over the duration of treatment. In addition, we presented data showing that atacicept leads to hematuria resolution in significantly more patients compared with placebo. The impact on hematuria was seen as early as 4 weeks after treatment initiation, which could have important implications for patients with acute kidney inflammation. The evolving atacicept data package supports our belief that atacicept may offer comprehensive disease modification to patients with IgAN,” said Vera Founder and CEO Marshall Fordyce, M.D. Explore more of the data: https://lnkd.in/eRcDMqd5 #biotech #ERA24

In a press release and call to investors today, March 19, 2024, Design Therapeutics, Inc. provided an update on the progress of GeneTAC™ program in FA. “Our lead program in FA has a new drug product, DT-216P2, that we have designed to have an improved pharmacokinetic and injection site safety profile, which positions us to resume clinical development for this debilitating, degenerative, neuro-muscular genetic disease. This work builds on encouraging data in FA patients from our previous clinical trial." - Pratik Shah, Ph.D., chairperson and chief executive officer of Design Therapeutics Design is advancing DT-216P2 for FA, with plans to complete GLP studies by year-end 2024 to start patient trials in 2025. Click here to read the full press release: https://bit.ly/4cmnatS or to access the presentation to investors: https://bit.ly/3IHQhdB

🗞️ Last week’s Pharma industry news in Germany and Switzerland 🗞️ Oculis Holding AG presents positive Phase 3 OPTIMIZE-1 trial results for OCS-01 at ASCRS. BioNTech SE presents promising three-year follow-up data for autogene cevumeran. Novartis announces results from the V-INITIATE trial, showing Leqvio significantly reduced LDL-C in ASCVD patients. ADC Therapeutics completes dose escalation in Phase 1b LOTIS-7 trial. Medigene AG secures European patent for its inducible Medigene T cell receptor (iM-TCR) technology. Basilea Pharmaceutica's ZEVTERA® (ceftobiprole medocaril) gains FDA approval for Staphylococcus aureus bloodstream infections, bacterial skin infections, and community-acquired pneumonia in adults and children. Addex Therapeutics and Perceptive Advisors establish Neurosterix, focusing on developing allosteric modulators for neurological disorders. #pharmaceuticalindustry #pharma #Monday_Pharma_News

🙌 Exciting news for liver disease, as Gilead Sciences acquires CymaBay Therapeutics! The acquisition gives Gilead ownership of Seladelpar, a drug to treat Primary Biliary Cholangitis (PBC), a disease which affects the bile ducts that run through the liver. The therapy is currently under review by the FDA, following a recent Phase III RESPONSE trial, which showed statistically significant results versus a placebo. As a fellow therapy developer in the liver disease space, this news is heartening – demonstrating that persistence, adaptability, and focused execution are key to advancing novel medicines for patients. Read more about the acquisition at STAT ⬇️ #biotech #genomics #LiverDisease

Navigating the complexities of drug development for inflammatory bowel disease - This new Perspective discusses challenges in IBD drug development and proposes novel strategies, including AI integration, organoid technology, and innovative trial designs. - Drug discovery and development for inflammatory bowel disease (IBD) is hampered by various challenges including the insufficient mechanistic understanding of IBD immunopathology, disease heterogeneity, inadequate preclinical models and clinical trial inefficiencies. - Despite the progress made over the past 25 years, rates of mucosal healing (a widely accepted treatment target in IBD correlating with improved longer-term outcomes) have reached a ceiling effect that does not exceed 30–40%. - Although the IBD therapeutic pipeline may appear rich and promising with more than 700 registered studies on ClinicalTrials.gov searching for participants, the cumulative probability of success of a compound entering phase I clinical trials through to approval is low at 15%. -  The future therapeutic landscape in IBD is likely to see a shift from clinician-driven treatment choices based on phenotypically similar but biologically heterogeneous populations to a machine learning guided approach that treats patients according to their molecular phenotype. https://lnkd.in/exAZK_UV #immunology #autoimmunity #IBD #drugdevelopment

China's NRDL 2023 marked a record for formulary expansion, including several new breakthroughs. Bruce Liu and colleagues discuss the developments in their new article. Learn more about the evolving value rating system as well as the impact on oncology and rare diseases here: https://lnkd.in/eh92SAvF #Healthcare #Pharma #Biotech #MarketStrategy #SimonKucher

👇 These are the top three news stories you need to read this week. 👇 #1: Gilead Sciences is bolstering its liver disease portfolio with the $4.3 billion acquisition of CymaBay Therapeutics, specifically for the primary biliary cholangitis (PBC) med seladelpar. #2: An experimental pill developed by KalVista Pharmaceuticals, Inc. succeeded in a Phase 3 trial, giving the drug a chance to become the first oral “on-demand” treatment for people with the rare swelling disorder hereditary angioedema. #3: The FDA has granted Fast Track designation to GSK’s novel antisense oligonucleotide (ASO) bepirovirsen to treat chronic hepatitis B (CHB).1 The drug was designed to specifically recognize the RNA used by hepatitis B virus (HBV) to replicate in infected liver cells. To read the full stories, please follow the links in the comments. #LifeSciencesNewsRoundUp #LifeSciences #ExecutiveSearch #FraserDove