BioPharm International’s Post

Zai Lab (NASDAQ: ZLAB, SEHK: 9688) has achieved a significant milestone in the collaboration with Bristol Myers Squibb. The US FDA has granted accelerated approval for repotrectinib for the treatment of adult and pediatric patients aged 12 and older with NTRK gene fusion-positive solid tumors that are locally advanced, metastatic, or in cases where surgical resection would likely result in severe morbidity. This approval is based on promising results from the Phase 1/2 TRIDENT-1 study. #Biotech #Innovation #FDAApproval #DrugDiscovery #HealthcareInnovation #QimingPortfolio #QimingHealthcare Read More: https://lnkd.in/gmuv-Ysk

Do we witness the START of a new era in the assessment of novel therapies for rare diseases? FDA seems to be shifting gear and crush the gas pedal with START Program. The FDA has officially selected four participants for the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program, aimed at accelerating the development of novel therapies for rare diseases. The program, initially aiming to include six participants, will bring early-stage participants and relevant FDA staff closer together on clinical study design and patient population fine-tuning. Grace Science, co-founded by Nobel winner Carolyn Bertozzi, got accepted for its GS-100 gene therapy to treat NGLY1 Deficiency, a life-threatening rare disease with no approved therapy. Denali Therapeutics has also been selected for its investigational enzyme replacement therapy for MPS IIIA, while Larimar Therapeutics Inc. Therapeutics is evaluating nomlabofusp, a protein replacement therapy for Friedreich's ataxia. Neurogene Inc. has been selected for its NGN-401 gene therapy for Rett syndrome, a rare genetic mutation affecting brain development in girls. https://lnkd.in/ggzjiHmk #rarediseases #regulatoryaffairs #clinicaldevelopment #pharmaindustry #unmetmedicalneeds

Latest Healthcare News & Updates: 4D Molecular Therapeutics Presents Positive Interim Data from Randomized Phase II PRISM Clinical Trial of Intravitreal 4D-150 Demonstrating Favorable Tolerability & Clinical Activity in Wet AMD. Adaptimmune Announces FDA Acceptance of Biologics License Application for Afami-cel for the Treatment of Advanced Synovial Sarcoma with Priority Review. Astellas Pharma Submits Supplemental New Drug Application in Japan for PADCEV with KEYTRUDA for First-Line Treatment of Advanced Bladder Cancer. AnaMar AB Announces US and EU Orphan Drug Designation for AM1476 for Treating Systemic Sclerosis. BioSyngen Announces FDA Fast Track Designation for BST02 in Treatment of Liver Cancer. Acepodia Announces FDA Clearance of Investigational New Drug Application for ACE2016. Get more detailed insights, at: https://lnkd.in/g2jMdUXn #lifesciences #pharmaceuticals #marketresearch #marketanalysis #healthcare #markettrends #health #medical #news #healthcarenews #pharmanews #clinicaltrials #clinicalresearch

🚨 5 Key FDA Decisions to Watch in Q4 🚨 🫀 BridgeBio’s Acoramidis: A potential game-changer for a rare heart condition. Could it rival Pfizer’s multibillion-dollar Vyndamax? Decision by Nov. 29. 💨 AstraZeneca & Daiichi Sankyo’s Dato-Dxd: A lung cancer treatment aiming to replace chemo. Will it live up to expectations? Verdict by Dec. 20. 🫁 Vertex’s “Vanza Triple” for Cystic Fibrosis: New combo treatment that may offer patients more convenience. Decision by Jan. 2, 2025. 🧬 PTC’s Upstaza: Gene therapy for a rare nervous system disorder. Will the FDA finally greenlight it after delays? Expect an answer by Nov. 13. 💉 Bristol Myers’ Subcutaneous Opdivo: A more convenient version of their cancer immunotherapy. Can it protect their revenue as patents expire? Decision by Dec. 29. 🌟With billions on the line, these decisions could shift the landscape of biotech innovation. Stay tuned! #biopharma #FDA #drugapprovals #lifesciences

I would like to congratulate our team in Vancouver and our partner ReCode Therapeutics for a great achievement! ReCode Therapeutics has dosed their first healthy volunteer participants with a new mRNA-based genetic medicine for cystic fibrosis in a Phase I clinical study. Our team in Vancouver successfully manufactured clinical drug product in November 2023 – just four months from GMP production to the clinic!   The inhaled mRNA-based investigational therapy (RCT2100) developed by ReCode Therapeutics is for the treatment of the 10-13% of patients with cystic fibrosis who have Class I mutations in the specific gene known as CFTR. This type of cystic fibrosis affects approximately 130,000 people worldwide. A big thank you to the Evonik Health Care team in Vancouver for your excellent collaboration. We are thrilled to support ReCode Therapeutics in bringing their therapy closer to individuals with cystic fibrosis.   Read more about this news here: https://lnkd.in/egb9W4YP #pharmaceuticals #mRNA #cysticfibrosis #drugdelivery #clinicaltrial #Vancouver #geneticmedicine

🩸🤖 CAR-T Breakthrough: Transforming Lupus Treatment! 🧪 Insights Snapshot: 1. CAR-T Hope: University of Tennessee's CAR-T therapy delivers a game-changer for lupus. By targeting CD19, it wipes out problematic B cells, alleviating lupus symptoms and extending life in mice. A potential leap beyond monoclonal antibodies for a robust and lasting solution. 2. CAR-T Edge: Unlike frequent doses with antibodies, CAR-T cells show resilience, staying active for a year post-infusion. This promises a paradigm shift in lupus treatment, offering prolonged efficacy and potency. 3. Clinical Triumph: A compassionate-use CAR-T program in SLE patients achieves deep B cell depletion and rapid remission within three months. A testament to CAR-T's remarkable effectiveness and durability in treating autoimmune diseases. 🚀 Impact Burst: These studies herald a revolutionary era in lupus care. CAR-T therapies emerge as potent, enduring solutions, transforming the landscape of autoimmune disease treatment. #CAR-TBreakthrough #LupusInnovation #AutoimmuneRevolution 🧬💉 1. Mackensen A, Müller F, Mougiakakos D, et al. Anti-cd19 car T cell therapy for refractory systemic lupus erythematosus. Nature News.  2. Liu A. A car-T approach to lupus. Fierce Biotech. 

🤩 BPGbio, Inc. CEO Dr. Niven R. Narain Honored as PharmaVoice 100 Winner for Pioneering Work in AI-Driven Drug Discovery 🤩 Niven R Narain, Ph.D., President and CEO of BPGbio, Inc., a pioneering biology-first, AI-powered biopharma specializing in mitochondrial biology and protein homeostasis, has been named a PharmaVoice 100 winner, as announced today. This prestigious annual list recognizes the most inspiring leaders in the global life sciences industry. Under Dr. Narain’s leadership, BPGbio, Inc. has developed the NAi Interrogative Biology® Platform, a cutting-edge biology-first AI approach that has added the patient voice and intersection of math and biology to the drug discovery and development process. The platform has generated over 250 drug targets and diagnostic #biomarkers in huge unmet disease areas. Its hallmark achievements include a novel targeted protein degradation (TPD) program based on E2 enzymes, which were long considered undruggable, and a franchise drug candidate BPM31510 in topical, IV and oral formulations for multiple oncology and mitochondrial disorders. BPM31510 IV has recently received Rare Pediatric Disease designation from the U.S. Food and Drug Administration (#FDA) for its potential as a treatment for Primary CoQ10 Deficiency (PCQD) in addition to Orphan Drug Designation for Glioblastoma Multiforme (#GBM), Pancreatic Cancer, and Epidermolysis Bullosa (#EB). BPM31510 IV is also being studied in phase 2 clinical trials as a potential breakthrough therapy for aggressive cancers such as glioblastoma and pancreatic cancer while in planning phases for late-stage Phase 2/3 trials for EB and PCQD. “These honorees are inspirations. Their teams look to them for guidance, their patients for support, and their organizations for direction. Each winner represents progress toward a healthier future for everyone that is bolstered by science and technological advancements. We are excited to recognize their contributions through this annual list,” said Meagan Parrish, lead editor of PharmaVoice. For the full release, please visit: https://lnkd.in/gukTamAc #AI #drugdiscovery #recognition #NAiInterrogativeBiology #Mitochondrialbiology #Proteinhomeostasis #cancer #TPD #protac #E2notE3 #clinicaltrials #research #raredisease

🔍 FDA June 2024: Key Designations that Propel Clinical Advancements Forward 🚀 In June 2024, the FDA made significant strides in advancing treatments for various diseases. Here are some key takeaways and highlights: 1️⃣ Total 22 drugs and 3 devices received designations of which maximum designations are grabbed by cell & gene therapy i.e., 8 followed by 7 of small molecules. 2️⃣ A total of 5 P3 molecules have gotten different priorities which include BridgeBio (Ribitol), InnoCare Pharma (Tafasitamab), AstraZeneca (Tagrisso), Neurotech Pharmaceuticals, Inc. (NT-501) & Quince Therapeutics (EryDex). 3️⃣ Maximum designations have been fetched during P-1/2 stage for 9 molecules. 4️⃣ Intraveous ROA has bagged maximum designations with total 13 molecules. 5️⃣ US issued around 25 designations with maximum FTD's issued i.e., 8 followed by ODD i.e., 7 (6 US & 1EU). 6️⃣ Oncology bagged the maximum designation i.e. 13 followed by Neurology with 7. 7️⃣ 2 molecules received designations for Ovarian cancer Repare Therapeutics (Lunresertib + Camonsertib), Tubulis GmbH (TUB-040) followed by 2 molecules for Ataxia Telangiectasia ChromaDex (Nicotinamide Riboside Chloride), Quince Therapeutics (EryDex System) followed by . Additionally, This month, ChromaDex’s Nicotinamide Riboside Chloride received US FDA’s ODD & RPDD for Ataxia Telangiectasia and Aurion Biotech’s Neltependocel (AURN001) gained FDA’s BTD & RMAT for corneal endothelial disease . 🔬 At Octavus Consulting, we specialize in: ▶ Competitive Intelligence: Stay ahead of the curve with our expert analysis. ▶Primary Market Research: Understand market dynamics and customer needs. ▶Conference Coverage: Get insights from industry events. ▶Human Intelligence Gathering: Tap into valuable information sources. ▶Indication or Asset Prioritization: Optimize your portfolio. Let’s drive innovation together! 🌟 #HealthcareAdvancements #PharmaInnovations #OctavusConsulting #competitiveintelligence #intelligence #innovation #designations #approval #regulatory #launch

Great to see Bloomsbury's R&D strategy vindicated by the latest regulatory developments in the US! The stars are aligning to develop gene therapies much faster and in a more capital-efficient manner than what it took the first generation of treatments, and this strategy is essential to deliver the promise of gene therapy to more patients living with severe and rare/ultra-rare monogenic diseases in particular.

Boehringer Ingelheim announced a multi-target #collaboration agreement with Chinese biotech Suzhou Ribo Life Science Co., Ltd and its Swedish unit Ribocure Pharmaceuticals AB to develop #siRNA-based treatments for metabolic dysfunction-associated steatohepatitis (#MASH). Boehringer also partnered with MiNA Therapeutics in 2017 to develop a small activating RNA therapy for the same indication. Beyond MASH, Boehringer’s pipeline is also addressing #obesity with three Phase 3 trials for an investigational glucagon/GLP-1 receptor dual agonist survodutide. The late-stage studies follow promising Phase II data showing #weightloss by nearly 19% after 46 weeks. Survodutide is also being developed for MASH in a Phase 2 dose-ranging study. #cardiometabolic