Greg Slabodkin’s Post

Vertex Pharmaceuticals moves #inaxaplin into Phase III trials for APOL-1 mediated kidney disease (AKMD), targeting the disease at its genetic roots. Phase II data showed a significant reduction in proteinuria, spotlighting inaxaplin's potential to transform AKMD treatment. Key Takeaways - 🔹 Impact - Offers new hope to ~100,000 affected patients in the U.S. and Europe. 🔹 Insight - Early and persistent efficacy in reducing proteinuria, with a safety profile that supports its use. 🔹 Discussion Point - How could inaxaplin’s approval change the landscape of kidney disease treatment? #KidneyDisease #ClinicalTrials #HealthcareInnovation

Vertex Pharmaceuticals to Acquire Alpine Immune Sciences for $4.9 Billion US-based Vertex Pharmaceuticals has agreed to pay approximately $4.9 billion in cash to acquire Alpine Immune Sciences, Inc., a compatriot #biotechnology company focused on discovering and developing innovative, protein-based #immunotherapies. The transaction was unanimously approved by the boards of directors of both companies and is expected to close later this quarter. Alpine’s lead molecule, povetacicept (ALPN-303), is a dual antagonist of BAFF (B cell activating factor) and APRIL (a proliferation inducing ligand). Through Phase 2 development, povetacicept has shown potential best-in-class efficacy in IgA nephropathy (IgAN), the companies said in a joint statement. IgAN is a serious, progressive, autoimmune disease of the kidney that can lead to end-stage-renal disease. So far, there are no approved therapies that target the underlying cause of IgAN. Povetacicept is on track to enter Phase 3 clinical development in the second half of 2024. Read more on #CMI online: https://lnkd.in/ebDN63QP

#Immunity | #Inflammation | #Therapy | Targeting TNF/TNFR superfamilies in immune-mediated inflammatory disease | Comprehensive Open Access Review from Pfizer scientists now online at Rockefeller University Press #JExpMed | Dysregulated signaling from TNF and TNFR proteins is implicated in several immune-mediated inflammatory diseases (IMIDs). This review* by Praveen Krishna Veerasubramanian, Thomas Wynn, Jie Quan & Fridrik Karlsson centers around seven IMIDs (rheumatoid arthritis, systemic lupus erythematosus, Crohn’s disease, ulcerative colitis, psoriasis, atopic dermatitis, and asthma) with substantial unmet medical needs and sheds light on the signaling mechanisms, disease relevance, and evolving drug development activities for five TNF/TNFR signaling axes that garner substantial drug development interest in these focus conditions. The review also explores the current landscape of therapeutics, emphasizing the limitations of the approved biologics, and the opportunities presented by small-molecule inhibitors and combination antagonists of TNF/TNFR signaling. *https://lnkd.in/e68mqnDQ Celentyx Ltd Professor Nicholas Barnes PhD, FBPhS Omar Qureshi Catherine Brady FIGURE | Molecular signaling mechanisms of the focus TNF/TNFR molecules and the anti-inflammatory benefits of their antagonism |

An estimated 10,000+ rare diseases affect more than 30 million people – approximately one out of every 10 people – in the U.S., and about half of these people are children. Many rare conditions are life threatening, and most do not have approved treatments.    The FDA plans to establish a Rare Disease Innovation Hub (the Hub). The Hub will work across rare diseases but will especially focus on products intended for smaller populations or for diseases where the natural history is variable and not fully understood, since the development of therapies for these conditions can be particularly challenging.   Learn more about the Hub’s three primary functions from the FDA’s Patrizia Cavazzoni, MD, Director, Center for Drug Evaluation and Research and Peter Marks, MD, PhD, Director, Center for Biologics Evaluation and Research (CBER).   https://bit.ly/3WTK0TO | #RareDiseases #ClinicalResearch #DrugDevelopement #ClinicalTrials Answers Media Network

Developing orphan drugs for rare diseases? At Quest Diagnostics, we understand the challenges of developing treatments for rare diseases. Traditional diagnostic methods have limitations, hindering patient selection and drug development timelines. Read "Navigating the Complexities of Rare Disease – A Quest Diagnostics Perspective" by Juha Koskenvuo to gain valuable insights for Pharma & researchers: https://lnkd.in/ePGwesT9

Yesterday, the FDA announced plans to launch a Rare Disease Innovation Hub that will utilise new and collaborative approaches to expedite the development and approval of safe and effective drugs and biologics that treat rare diseases. One out of every 10 people in the US are affected by a rare disease and around half of these people are children. Many rare conditions are life-threatening, and most do not have approved treatments. The new Hub will work across rare diseases with a focus on products intended for smaller populations or for diseases where the natural history is variable and not fully understood. It will serve as a single point of connection and engagement with the rare disease community; enhance agency collaboration to address common scientific, clinical and policy issues related to rare disease product development; and advance regulatory science. Having supported organisations across the globe with the development of medicines for rare diseases, Boyds warmly welcomes this promising initiative which ultimately aims to improve outcomes for people living with rare diseases by providing new therapeutic options. Find out more: https://lnkd.in/egJKYwvs #rarediseases #fda #innovationhub #medicine

Gene-editing therapy #Casgevy (exa-cel) has received early FDA approval to treat transfusion-dependent β-#thalassaemia (TDT). This comes less than six weeks after the US regulator approved Casgevy for treating #patients with #sicklecell disease (SCD). With the TDT approval, Casgevy becomes the first CRISPR-based gene-editing treatment in the US for this #blooddisorder. In 2022, Bluebird Bio also secured FDA-approval for their #genetherapy, #Zynteglo, to treat TDT. The FDA endorsement arrived over two months ahead of schedule, as a decision was initially expected on March 30. "Following the historic FDA decision on Casgevy for sickle cell disease, we're thrilled to secure approval for TDT well before the PDUFA date," said Reshma Kewalramani, MD FASN, Vertex Pharmaceuticals' CEO. Vertex announced the opening of nine authorized treatment centers (#ATCs) in the United States to administer Casgevy to #patients, covering both indications. Additional ATCs will be activated in the coming weeks. https://lnkd.in/dze-kDTn

📌 Vertex's recent acquisition of Alpine has raised our conviction on the potential of the immunoglobulin A Nephropathy (IgAN) market. With ~130K biopsy-confirmed IgAN patients in the USA, ~140K in the EU, ~130K in Japan, and ~5 million in China, it is the most common primary glomerulonephritis globally. 📌 Up to 50% of these patients will develop end-stage renal disease (ESRD) within 10-20 years from diagnosis, requiring dialysis or transplant. ESRD remains a major health economic burden and contributes to morbidity and mortality. 📌 Currently, Calliditas' TARPEYO and Travere Therapeutics' FILSPARI are FDA approved with disease-modifying potential. Several other drugs with distinct mechanisms of action are in late-stage development, with pivotal readouts expected in 2024. These programs include Chinook Therapeutics' atrasentan, Novartis' iptacopan, and Omeros' narsoplimab, among others. 📌 Despite rising competitive intensity, we hold an optimistic outlook on the IgAN market and are closely watching how the safety and efficacy stack up across programs. #kidneydisease hashtag #nephrology hashtag #ckd hashtag #strategy hashtag #marketintelligence hashtag #competitiveintelligence hashtag #eosintelligence hashtag #perspectives hashtag #management hashtag #consulting hashtag #pharmaceuticals hashtag #marketresearch hashtag #marketing hashtag #leadership hashtag #lifesciences hashtag #healthcare hashtag #pipeline hashtag #pharmainnovation hashtag #marketanalysis hashtag #biotech ----------------------------------------------------------------------------------- Ready to leverage the power of intelligence to drive Nephrology/Kidney Disease product development and commercialization strategy? At EOS Intelligence , we offer competitive intelligence monitoring services in key therapeutic areas to make you stay ahead of the curve. Let us know your perspectives & challenges and we will get you covered. Write us at bhabesh.panigrahi@eos-intelligence.com

FDA Rare Disease Innovation Hub FDA take another step to expedite development and approval of treatments for rare diseases. The Food and Drug Administration (FDA) announced the creation of a Rare Disease Innovation Hub to “expedite development and approval of safe and effective drugs and biologics” for rare diseases. What it will do, according to the FDA: ·   “Serve as a single point of connection and engagement with the rare disease community, including patient and caregiver groups, trade organizations, and scientific/academic organizations.” ·   “Enhance intercenter collaboration to address common scientific, clinical and policy issues related to rare disease product development.” ·   “Advance regulatory science with dedicated workstreams for consideration of novel endpoints, biomarker development and assays, innovative trial design, real world evidence, and statistical methods.” https://lnkd.in/d7fqn6tB