Pharmaceutical Business Review International’s Post

We are excited to share that The Lancet published 12-month safety and efficacy data from our Phase I/II clinical trial evaluating ATSN-101 the first, investigational #GeneTherapy therapy to treat the #InheritedRetinalDisease #LeberCongenitalAmaurosis caused by biallelic mutations in GUCY2D (LCA1).   ATSN-101 demonstrated durable, clinically significant improvements in vision and was well-tolerated 12 months post-treatment. We are encouraged by these promising results, which support further evaluation in a Phase III trial. ATSN-101 has the potential to become the first-ever #GeneTherapy for LCA1, marking a significant advancement in reversing blindness that begins in childhood.   Read the full article published online in The Lancet. https://lnkd.in/eCsf8Khq   Image description: Text reads: Atsena Therapeutics Announces 12-Month Safety and Efficacy Data from Phase I/ll Clinical Trial of ATSN-101 in LCA1 Published in The Lancet. The Atsena Therapeutics logo is in the bottom right corner over a white bar stretching across the bottom.

The FDA has just approved donanemab (Kisunla), a monoclonal antibody developed by Eli Lilly and Company to slow the progression of early symptomatic Alzheimer’s disease. Donanemab helps remove amyloid plaque buildup in the brain, a key factor in Alzheimer’s. Clinical trials have shown significant results, with a 35% lower risk of disease progression over 18 months. While not a cure, donanemab marks the second entry into the first class of disease-modifying drugs for Alzheimer's. This incredible progress is the result of more than 20 years of biomarker research and highlights the importance of public-private collaboration in advancing such medical breakthroughs. The FNIH is committed to continuing its work in developing robust, shareable data sets and sharing knowledge, expertise, and resources to allow researchers to expand their thinking and add new knowledge to solve complex problems like Alzheimer’s and other neurodegenerative diseases. Link here: https://lnkd.in/gcdswNp7 #Alzheimers #AlzheimersResearch #Donanemab #NeurodegenerativeDisease #BiomarkerResearch #AMPAD #BiomarkersConsortium #FNIH

MaaT Pharma announced that its IASO Phase 1b clinical trial, evaluating the safety and tolerability of multiple doses of MaaT003 in amyotrophic lateral sclerosis (ALS), has met its primary endpoint 

Tiziana Life Sciences Ltd (NASDAQ:TLSA) announced that it has dosed the first patient with moderate Alzheimer’s disease with its lead investigational drug candidate intranasal foralumab. The company said this milestone took place at Brigham and Women’s Hospital in Boston as part of a study aimed at addressing neuroinflammation, a key factor in Alzheimer’s progression. Intranasal foralumab, a fully human anti-CD3 monoclonal antibody, offers a novel approach by activating regulatory T cells that cross the blood-brain barrier to reduce microglial inflammation. Unlike traditional therapies focused on beta-amyloid or tau protein, this strategy targets underlying... http://ow.ly/kNrZ105RTPa

🌎 LATE PHASE SUCCESS 🏥 Avance Clinical's late phase operations deliver multi-regional clinical excellence across key therapeutic areas including oncology, CNS, rare diseases and infectious diseases. Our global teams apply decades of drug development experience, and leverage the latest technology, including AI and gold standard systems and processes, across all functional areas to deliver an exceptional CRO experience for biotechs. As your drug development journey progresses, the Avance Clinical team can transition your clinical research from early to late phase multi-regional studies with the assurance that the global team brings the same expertise and professionalism you have come to expect. All the while saving time and costs and delivering rigorous globally accepted data. Learn more: https://lnkd.in/gsKzG5vv Request a Proposal now 👇 https://lnkd.in/gDgtaSqB #AvanceClinical #CRO #Biotech #LatePhaseSuccess #GlobalReadybyAvanceClinical #NorthAmerica #Asia #EU #ClinicalTrials #DrugDevelopment #BIO2024 #SanDiego #California #GloballyAcceptedData

⚖️ Novartis successfully defended the US patent for Entresto® on January 13, 2025, as affirmed by the US Court of Appeals. 💉 On December 30, 2024, Novartis reported that their Phase III study of Onasemnogene Abeparvovec met its primary endpoint, demonstrating significant patient improvement. The company plans to share these positive findings with regulatory agencies in 2025. 🧬 BioAge Labs announced a multi-year collaboration with Novartis on December 18, 2024. They aim to leverage their combined expertise to discover drug targets for age-related diseases and conditions. 🎗️ On December 10, 2024, Novartis Kisqali® demonstrated prolonged effectiveness in reducing distant recurrence in early breast cancer patients. 💊 Novartis' long-term data from December 8, 2024, showed Scemblix®'s superior efficacy and safety in treating adults newly diagnosed with CML. #DataPorium #Novartis #MedicalInnovation #PatientCare #BiotechResearch At DataPorium, we track the financial performance, product sales, market share, and social network analysis. We also track the trends of nearly 1,000 products every quarter. 🚀 Want to see more? Head over to https://lnkd.in/duDAwekv and explore! 🔗 Visit our LinkedIn page https://lnkd.in/gJ2iKqKw today for exclusive updates—completely free!

Another significant milestone for us as we confirm that the first patient has been dosed in our Phase 1b COMPOSER-1 trial of MB310, our precision microbiome medicine for ulcerative colitis (UC)! 30 patients with mild to moderate UC from sites in the UK, Austria, Bulgaria, Poland and Spain will be included in the trial.   MB310, an orally administered live biotherapeutic product, is a new treatment modality for UC patients, offering the potential of disease remission without immunosuppression. With patients now receiving our precision microbiome treatments in both the COMPOSER-1 trial for UC and the MELODY-1 trial for melanoma, we continue to advance new therapeutic possibilities for patients. Initial data readouts for both studies are expected by the end of 2025. 🔗 Read the press release: https://lnkd.in/gkzhmGCb 🔬www.microbiotica.com Walter Reinisch #biotech #pharma #ulcerativecolitis #microbiome #gutmicrobiome #clinicaltrials

At Artax Biopharma, we are developing first-in-class Nck modulators to treat T cell mediated autoimmune disease such as #psoriasis, #atopicdermatitis, and more. We're excited by the progress we've been making in the development of our first novel mechanism, AX-158, as we rapidly approach results from our phase 2 trials for psoriasis treatment with first results expected in December 2024. We are simultaneously developing additional Nck modulator candidates for #autoimmunediseases with AX-194 undergoing preclinical testing to enter Phase 1 trials. Learn more about our candidate pipeline here: https://lnkd.in/eqq3eSTK #clinincaltrials #immunology

The FDA and AACR collaborated to host a special workshop on Dihydropyrimidine Dehydrogenase (DPD) Deficiency Testing: Weighing Potential Harms. The workshop—which was chaired by Jennifer Gao, MD, associate director for education at the FDA Oncology Center of Excellence and AACR President Patricia LoRusso, DO, PhD (hc), FAACR—examined current evidence surrounding DPD deficiency testing, considered the potential clinical implications of requiring testing, and addressed the regulatory considerations for modifying FDA product labeling to require testing.