Greg Slabodkin’s Post

Heading to SOT? Interested in discussing iPSCs, cardiotox and neurotox? Make sure to keep 12pm MDT on Tuesday 12th free in your diary! From the 10th to 14th March, we'll be in Salt Lake City for the Society of Toxicology 63rd Annual Meeting, discussing our work unlocking human iPSC technology for neuro and cardiac safety pharmacology. At 12pm MDT on Tuesday 12th March, we'll be joined by our friends from @innoVitro GmbH to our booth (#2032) to discuss iPSC technology, functional QC, and how we're helping researchers to build better in vitro cardiotoxicity models. You can also hear about our collaborative project with innoVitro testing the chamber-specific pharmacological responses of axoCells atrial and ventricular cardiomyocytes on the FLEXcyte 96 contractility system. Visit ToxExpo Exhibit Hall C on Wednesday 13th March to see poster number P544: "Contractility-based pharmacological characterization of hiPSC-derived atrial and ventricular cardiomyocytes for preclinical toxicity testing". If you're attending SOT, make sure to join us for an informal chat, and for your opportunity to ask any questions about how Axol Bioscience and innoVitro can help you to unlock the benefits of iPSC technology. #SOT #iPSCs #CiPA #cardiotoxicity #neurotoxicity #DrugDiscovery

Heading to SOT? Interested in discussing iPSCs, cardiotox and neurotox? Make sure to keep 12pm MDT on Tuesday 12th free in your diary! From the 10th to 14th March, we'll be in Salt Lake City for the Society of Toxicology 63rd Annual Meeting, discussing our work unlocking human iPSC technology for neuro and cardiac safety pharmacology. At 12pm MDT on Tuesday 12th March, we'll be joined by our friends from innoVitro to our booth (#2032) to discuss iPSC technology, functional QC, and how we're helping researchers to build better in vitro cardiotoxicity models. You can also hear about our collaborative project with innoVitro testing the chamber-specific pharmacological responses of axoCells atrial and ventricular cardiomyocytes on the FLEXcyte 96 contractility system. Visit ToxExpo Exhibit Hall C on Wednesday 13th March to see poster number P544: "Contractility-based pharmacological characterization of hiPSC-derived atrial and ventricular cardiomyocytes for preclinical toxicity testing". If you're attending SOT, make sure to join us for an informal chat, and for your opportunity to ask any questions about how Axol Bioscience and innoVitro can help you to unlock the benefits of iPSC technology. #SOT #iPSCs #CiPA #cardiotoxicity #neurotoxicity #DrugDiscovery

PhRMA: those darn 340B hospitals use more expensive drugs than others. Also PhRMA: check out this amazing new product priced at just $4.25 million… OK, Lenmeldy isn’t the best example to use here… And I’m not criticizing that drug in terms of benefit… Price benefit yes, but not clinical benefit… It’s revolutionary. All I’m saying is Pharma has a lot of nerve criticizing the hospitals that have the specialists that might order these drugs for…you know…ordering these drugs…,when they are the ones setting the price on them. #340B #drugcosts #hospitals #bigpharma #pharma #PhRMA #pharmacy #pharmacist https://lnkd.in/g9z9H4SR

Gene&Cell Therapy >> FDA sets late 2024 decision deadline for Pfizer’s 'pan-hemophilia' drug: #ASH23: SAN DIEGO — The FDA is expected to decide whether to approve Pfizer’s hemophilia A and B antibody in the fourth quarter of 2024, the New York pharma announced Monday afternoon. An approval of marstacimab could kickstart Pfizer’s challenge to Roche’s blockbuster Hemlibra in hemophilia A and provide a first weekly injection option for hemophilia B. In a clinical trial, 116 hemophilia patients received once-a-week treatment with Pfizer’s antibody marstacimab. Those who had previously been receiving regular infusions of factor VIII or IX — the clotting factors that patients with hemophilia A and B are missing — saw their annual rate of bleeding events drop by 35% on marstacimab. For the patients who had previously been getting on-demand infusions, annual bleeding events fell by 92% on marstacimab, which is delivered via a pre-filled pen. Detailed data were presented at the American Society of Hematology (ASH) annual meeting on Saturday. Greg Di Russo The FDA will decide whether to approve marstacimab for hemophilia A or hemophilia B patients who do not have neutralizing antibodies against clotting factors. Pfizer also announced that it expects a decision from European authorities by the first quarter of 2025. If approved, the antibody would challenge Roche’s hemophilia A drug Hemlibra, which in 2022 reeled in over $4 billion. “Marstacimab is really a pan-hemophilia opportunity for a broad range of patients,” Pfizer’s hematology development head Greg Di Russo told Endpoints News ahead of the ASH presentation. Pfizer is also developing gene therapies for hemophilia and is awaiting an FDA decision on its hemophilia B gene therapy in the second quarter of next year. Beyond the indication for which it has applied for approval, Pfizer is studying marstacimab in hemophilia patients who do have neutralizing antibodies against clotting factors. The presence of those antibodies makes current treatment options — factor VIII and IX infusions — less effective. Pfizer said that the arm of the trial has completed enrollment and that it expects data “as early as late 2024.” #lucidquest #genetherapy #celltherapy

Regulatory Update — Week of Jan. 29, 2024: Over the past week, the FDA issued a final rule to amend the device current good manufacturing practice (CGMP) requirements of the Quality System (QS) regulation. Final guidances were issued on chimeric antigen receptor T cell (CAR T) products, human gene therapy products and individual patient expanded access applications. Draft guidance was issued on collection of race and ethnicity data in clinical trials. #fda #financial #lifesciences

At Silo Pharma, we are developing treatments such as SPC-15 for those living with #PTSD. We recognize the urgency for effective treatment options, and we're actively pursuing preclinical work with our data that indicates that SPC-15 could have additive benefits for combating stress-induced pathophysiology, both at the behavioral and neural levels. We anticipate that our pre-IND submission will lead to valuable guidance from the FDA as we prepare for the first-in-human clinical trial of SPC-15 in PTSD. Stay tuned for further updates on our clinical roadmap. #PTSDResearch #MentalHealthMatters #Biopharmaceuticals

We've had a great time at SOT! If you're working in cardiac safety pharmacology, make sure to download our exciting concept discussion pieces. At Axol Bioscience, we've been looking into the key questions of utility and relevance for iPSC-derived cardiotoxicity models. We've therefore produced two discussion documents based on our research in this space: 1. Our ongoing work licensing an innovative protocol developed by a team at The Crick Institute, which aims to produce purer populations of left ventricular cardiomyocytes https://hubs.la/Q02prMJz0 2. The identification of a metabolic maturation media to enhance cardiomyocyte maturity https://hubs.la/Q02prFW30 We were delighted to see such a positive response to these documents at SOT, so we've made them available to download by clicking the links above. If you have any questions or would like to discuss how we could unlock iPSC technology for your cardiotoxicity projects, please get in touch as operations@axolbio.com #iPSCs #SOT #cardiotoxicity #neurotoxicity #CiPA #DrugDiscovery

📢Preventing, Predicting, and Mitigating ADC Toxicities! The 2nd ADC Toxicity Summit returns as the only conference bringing together 80+ experts in toxicology, pathology, translational sciences and more, to exclusively address challenges in minimizing ADC toxicity - https://ter.li/xa0lrc As toxicities continue to pose the most significant challenge in the progression of antibody-drug conjugates, join your peers to unlock first-hand insights into how to improve prediction and translation of ADC toxicities to drive more tolerable ADCs successfully into and through clinical development. Discover how to tackle ADC toxicities using in vivo models, enhancing the therapeutic index of ADCs, improving predictability, and much more – find out more here: https://ter.li/xa0lrc Don’t miss out on this unparalleled opportunity to collaborate with industry leaders to navigate the prevention, prediction, and mitigation of ADC toxicities, and make meaningful strides forward in developing safer ADCs. Register before midnight Friday, May 17 to save $850 – https://ter.li/lce30w

Exciting news!🎉 BVX001 has demonstrated a compelling in vivo safety profile, bringing it even closer to the clinic 🚀 We are thrilled to announce that BVX001, our first-in-class Bi-Cygni® ADC for Acute Myeloid Leukaemia (AML), has demonstrated a highly favourable safety profile in preclinical studies. The toxicological profile of BVX001 stands out against other FDA-approved ADCs using the same linker/cytotoxic payload. Some key highlights from the study include: 🌟 No observable severe pathological changes at all tested doses. 🌟 Ocular toxicity wasn't observed at mid to low doses; minimal effects at higher doses. 🌟 Data significantly strengthens our preclinical package and will help inform clinical dose selection. These findings pave the way for our final IND-enabling studies and move us closer to clinical studies. We look forward to presenting this data at the 2024 American Society of Haematology meeting! Read the full press release here: https://lnkd.in/ekt4TtaR And stay tuned as we continue to innovate and work towards more effective, less toxic treatments for AML. #BiVictriX #BVX001 #CancerTherapeutics #AML #Biotech #ClinicalTrials #DrugDiscovery #DrugDevelopment #Biotherapeutics

Gene&Cell Therapy >> Apellis expects EU to reject eye drug; Terray teams up with Bristol Myers; Vandria raises $20M: Plus, news on Inductive Bio, AbelZeta Pharma, GPCR Therapeutics, Bridge Biotherapeutics and NextCure. Apellis preempts EU rejection for eye drug: The company expects a negative opinion from the Committee for Medicinal Products for Human Use on pegcetacoplan, which is under review for the treatment of geographic atrophy secondary to age-related macular degeneration. The complement C5 inhibitor in February won US approval for the indication as Syfovre, but its launch has been plagued by real-world safety events. Apellis shares $APLS were down 20% on Thursday morning. — Ayisha Sharma Terray Therapeutics is partnering with Bristol Myers Squibb in a new drug discovery and development deal that aims to identify small molecules targeting “certain disease areas.” Financial terms of the deal, which includes an upfront and potential milestones, were not disclosed. — Jaimy Lee Swiss biotech debuts with $20.6M fundraise: Vandria plans to use the Series A proceeds to advance its portfolio of small molecule mitophagy inducers, including lead asset and central nervous system drug VNA-318. Mitophagy involves the selective removal and replacement of damaged mitochondria with anti-inflammatory effects. VNA-318 is set to enter the clinic in the second quarter of next year. — Ayisha Sharma Inductive Bio emerges from stealth: The startup’s $4.3 million seed round was led by Andreessen Horowitz Bio + Health and Lux Capital. Other investors include Character, Bessemer Venture Partners and Alleycorp. The company, which was founded by a pair of former Flatiron Health employees, is using machine learning to help chemists optimize compounds in the drug discovery process. — Jaimy Lee AbelZeta Pharma updated its licensing deal with Johnson & Johnson, which now has the option to get exclusive commercialization rights in China to two of the biopharma’s CAR-T treatment candidates. Both are being tested in patients with non-Hodgkin lymphoma. — Jaimy Lee GPCR Therapeutics, a Seoul-based biopharma, inked an out-licensing deal with Bridge Biotherapeutics for its CXCR4-LPA1 inhibitor combo. Bridge is paying $1.5 million upfront. — Jaimy Lee NextCure, a Maryland biopharma developing immunotherapies, is “actively” seeking partners to further progress its programs. — Jaimy Lee #lucidquest #genetherapy #celltherapy