Mike Davies, MD, FRCS

Our new episode it out discussing a very important issue - information clinical trials participants of their genetic testing results. Many companies opt-out and decide not to do it but is that always the best decision? You can learn more in the latest episode. #clinicaltrials #alzheimer

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Allogeneic CAR-T therapy drives autoimmune diseases into remission: Early reports suggest that chimeric antigen receptor (CAR)-T therapy has remarkable potential for treating autoimmune disease. Current approaches rely on autologous CAR-T cells, creating a bottleneck to the broad deployment of this therapy.  A recent publication in Cell reports the first use of allogeneic CAR-T cells in three patients with systemic autoimmune disease. This off-the-shelf cell product (TyU19) was manufactured by isolating T cells from a healthy donor’s peripheral blood mononuclear cells, transducing the T cells with a second-generation CD19 CAR, and then using CRISPR-Cas9 to target TRAC, HLA-A, HLA-B, CIITA, and PD-1 to reduce the risk of immune rejection and graft-versus-host disease (GvHD). These cells were then banked and infused into one patient with immune-mediated necrotizing myopathy and two patients with diffuse cutaneous systemic sclerosis. In all three patients, CAR-T cells engrafted and expanded, coinciding with a complete depletion of circulating B cells for at least 2 months. Impressively, all three patients showed significant clinical improvement via respective clinical indexes, molecular markers, and radiographic measures. Remarkably, fibrosis in the lungs and hearts of some patients showed improvement. Although follow-up has only been extended to 6 months, these patients have remained in remission. This scientific discovery holds great potential for future treatment of autoimmune diseases. Original publication: https://lnkd.in/ejPsikBa Further reading: https://lnkd.in/e9DssDqa https://lnkd.in/e2zB33zN https://lnkd.in/eH2aY-XG https://lnkd.in/egSa6gCH https://lnkd.in/eKKdWAWg https://lnkd.in/eFA4mcpN

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Hemogenyx Pharmaceuticals PLC (LSE:HEMO, OTC:HOPHF), the UK-based biotechnology firm, has secured £340,000 through a placing of 100,000 new shares at 340p each. The investment round was underwritten by a single institutional investor, who also received 50,000 warrants exercisable at 500p per share, valid for 12 months from March 1. The company stated that proceeds from the placing will be directed towards advancing clinical trials of its lead therapy, HG-CT-1, which targets relapsed or refractory acute myeloid leukaemia (AML) in adults. Hemogenyx noted that recruitment for the trial is underway, with the first patient undergoing eligibility screening. https://lnkd.in/e2UbdKVm

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Our proprietary algorithms analyzed over 1540 press releases between July 21-July 26 to identify the top 10 news stories impacting the market this week. Calliditas Therapeutics announces positive TRANSFORM Phase 2b topline data in primary biliary cholangitis https://lnkd.in/e-5eQnyQ Telix Pharmaceuticals Limited FDA Accepts Telix NDA for New Prostate Cancer Imaging Agent  https://lnkd.in/e54fUZkH A.forall A.forall Announces the Launch of Their Fifth Abbreviated New Drug Application (ANDA) and Second “First Cycle” FDA Approval https://lnkd.in/epnnVsM7 “First-Cycle”-FDA-Approval Merck Receives Positive EU CHMP Opinion for KEYTRUDA® (pembrolizumab) Plus Padcev® (enfortumab vedotin-ejfv) as First-Line Treatment for Patients With Unresectable or Metastatic Urothelial Carcinoma https://lnkd.in/esAiCPqA Tonix Pharmaceuticals Granted Fast Track Designation by FDA for Tonmya™ for Fibromyalgia https://lnkd.in/eWJ8tWV5 Ipsen receives CHMP positive opinions for Iqirvo® (elafibranor) in Primary Biliary Cholangitis and Kayfanda® (odevixibat) in Alagille Syndrome, two rare cholestatic liver diseases https://lnkd.in/eugi6nTw Chemomab Therapeutics Announces Positive Phase 2 Trial Results: CM-101 Achieves Primary and Secondary Endpoints Demonstrating Anti-Fibrotic, Anti-Inflammatory and Anti-Cholestatic Effects in Patients with Primary Sclerosing Cholangitis https://lnkd.in/eYhhkFbT Arcutis Biotherapeutics, Inc. (Nasdaq: ARQT) Arcutis Submits Supplemental New Drug Application for ZORYVE® (roflumilast) Foam to the FDA for the Treatment of Scalp and Body Psoriasis in Adults and Adolescents Ages 12 and Over https://lnkd.in/eafPFR_m SpyBiotech SpyBiotech Announces the Completion of Enrollment in Phase I Trial of SPYVLP01 Targeting Human Cytomegalovirus (HCMV) https://lnkd.in/e8xAvnvN Inimmune INIMMUNE ANNOUNCES FIRST CANCER PATIENT DOSED IN PHASE 1 CLINICAL STUDY USING THE IMMUNOTHERAPEUTIC INI-4001, A NOVEL TLR7/8 AGONIST https://lnkd.in/eWkma72Z

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🔉 Stay Informed on the Latest Advancements in ADCs for Oncology 💡 📜 As the ESMO conference approaches, take a look at the must-watch ADC developments for the event Exhibit: What to watch in ADC space at ESMO 2024 🔬 Clinical Update ➡Sino Biological launches FREE trial to enhance ADC evaluation, focusing on pharmacokinetics and drug distribution. Developing anti-DM1/DM4/SN-38/Dxd mAbs for evolving needs. ➡ABL Bio halts ABL501 (PD-L1 x LAG-3) after Phase 1 due to shifting focus. The stock fell nearly 10% following the announcement. 📋 Regulatory Update ➡European Commission approves Merck’s Keytruda (pembrolizumab) with Padcev (enfortumab vedotin) for first-line treatment of unresectable/metastatic urothelial carcinoma based on positive Phase 3 KEYNOTE-A39 (EV-302) results. 🤝 Deal Update ➡Avenzo Therapeutics announced clinical study collaboration with Gilead to evaluate AVZO-021 (CDK2 inhibitor) with Trodelvy (Trop2 ADC) in HR+/HER2- metastatic breast cancer. Trial to begin in Q4 2024. 🏢 Corporate Update ➡Jefferies boosts Daiichi Sankyo target. Raises price target to JPY7,600 from JPY6,600, maintains buy rating on Daiichi Sankyo, citing strong Q1 results and ADC developments. 📅 Conference Update ➡Eisai will present an open-label, multicenter Phase 2 study on BB-1701 (HER2 ADC) in previously treated HER2+ or HER2-low unresectable or metastatic breast cancer at ESMO, 13-17 Sep 2024 ➡Daiichi Sankyo's presentations in upcoming conferences: First-ever clinical data from Daiichi Sankyo’s second ADC platform will be presented at ESMO, 13-17 Sep 2024 Six late-breaking presentations on DXd ADCs in lung and breast cancers, including two Presidential Symposia featuring datopotamab deruxtecan (Trop2 ADC) at WCLC, 7-10 Sep 2024 ➡ Gilead will be presenting new Trodelvy (Trop2 ADC) data including updates from EVOKE-02, EVOKE-01, and TROPiCS-03 studies at the World Conference on Lung Cancer at WCLC, 7-10 Sep 2024

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The $ success of some precision medicine drugs makes them potential targets for government price control across the globe. However the underlying economics of precision medicine returns are more complex suggesting caution by governments not to disincentivise, what is widely recognized as a clincial success story for patients. A recent analysis showed that up to 40% of precision medicine therapies launched before 2020 did not achieve viable ROI. https://lnkd.in/eP857gvQ , for many reasons including an underinvestment in patient testing. On the industry side, precision medicine also enables a unique opportunity for leverage of the diagnostic pathways (upon which precision treatments depend) to revisit and reduce legacy commercialization costs . As economists and analysts better understand the underlying economics of a precision medicine future, there is scope for goal congruence among all stakeholders and continued incentive towards the transformation for patients which precision testing and treatment offers. In support of such informed analysis at Diaceutics, we shared an open source library of publications, including many of our own, with a multi stakeholder group we believe spotlighted the underlying economics in the space. The full results of this economic forum will be published soon, but the benefits of a shared and improved understanding of why precision medicine economics are different, led to more nuanced solutions. https://bit.ly/3Tm1R2L

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So... Lots going on re EMR analysis / AI for patient recruitment, but we still know that building relationships with potentially eligible patients / onboarding the right way is so important (shout out to the great study coordinators and PIs out there!). Is EMR analysis the main game? Is better patient onboarding the main game? Please check out (and like/promote) my latest column in Applied Clinical Trials that focuses on challenges / debates in clinical trial recruitment. Special thanks to Brett Kleger and Elisa Cascade for their contributions!

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🔊 Stay ahead with latest updates on ADC in Oncology 🚀 📅 Daiichi Sankyo US and AstraZeneca refiled their BLA for datopotamab deruxtecan (TROP2 ADC) targeting EGFR-mutated NSCLC based on updated TROPION-Lung05 data. 📅 Mersana Therapeutics advances XMT-1660 (B7-H4 ADC) in Phase 1 trials while XMT-2056 (HER2 ADC) shows promise in preclinical studies. 📅 Pyxis Oncology to present preliminary Phase 1 data for PYX-201 (EDB+FN ADC) in solid tumors on November 20, 2024. 🤝 Alphamab Oncology joins Shanghai’s pilot program for a 30-day phase 1/2 review of JSKN033 (HER2 ADC) in advanced tumors. 🎯 Innate Pharma to launch a Phase 1 trial of IPH4502 (Nectin-4 ADC) in Nectin-4 positive solid tumors by Q1 2025. 📅 GSK’s DREAMM-7 trial demonstrates improved survival for RRMM patients with Blenrep (ADC) + BorDex, with full results at ASH 2024. 🎯 ArriVent Biopharma and Aarvik Therapeutics Inc Therapeutics progress their ARR-002 ADC program for solid tumors, with IND studies slated for early 2025. 🎯 Mabwell’s 9MW2821 (Nectin-4 ADC) received IND approval in China for urothelial carcinoma and solid tumors. 🤝 Chime Biologics and Waterstone Pharmaceuticals partner to enhance ADCs, combining production and linker-payload expertise. 🧪 Lonza expands its bioconjugation facility by 2,000m² to meet rising ADC demand and future product approvals. 🤝 GlycoNex Inc. partners with Sterling for clinical trial production of GNX102 (NaPi2b ADC). 💰 Alentis Therapeutics raised $181.4M in Series D funding to advance ALE.P02 and ALE.P03 ADCs for CLDN1+ cancers and organ fibrosis. Fast track designation granted to ALE.P02. 💰 IPA (ImmunoPrecise Antibodies) targets next-gen ADCs for a $20B oncology market with high-impact antibody innovation. 🤝 Entero Therapeutics (Nasdaq: ENTO) merges with @Journey Therapeutics, advancing the NIC ADC cancer platform with 99% equity to Journey shareholders. 🤝 BioNTech SE acquires Biotheus Inc., gaining its bis ADC pipeline and proprietary technology for cancer and inflammatory diseases. 🤝 Telix Pharmaceuticals Limited partners with Eckert & Ziegler SE to enhance alpha therapy production, supporting TLX252 (CAIX radio ADC). 🤝 Akari Therapeutics, Plc and Peak Bio Inc. merge, securing $3.2M in PIPE financing and a $50M equity line for ADC advancements. 🎯 IDEAYA Biosciences selects IDE034 (B7H3/PTK7 Bs ADC) as a development candidate under an exclusive Biocytogen license. 🎯 Araris Biotech AG Biotech unveils new peptide linker technology, enabling more precise and versatile ADC creation.

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Thanks for sharing, Vincent Ling. It has been a journey. But there is still hope for cell therapy in solid tumors, including from so-called endogenous reprogramming approaches that are more akin to gene therapy since the modulation/modifications occur in the patient. Companies like Abintus Bio, Inc., Capstan Therapeutics, Interius BioTherapeutics, Nanocell Therapeutics, Strand Therapeutics and others are some of the more recent biotechs to advance these in vivo platforms, often based on RNA or viral vectors. #immunooncology #immunotherapy #cancer #oncology

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Crosswalk Therapeutics is pleased to share our plans to develop a novel, next-generation enzyme replacement therapy (ERT) for Fabry disease. Our discussions with physicians and patients at this year’s WORLDSymposium, MPS Society UK, here for those with rare diseases Fabry Matters conference, and the 8th Update on Fabry Disease event from @Kidneys for Life have further illuminated for us the unmet need in the patient community and the transformative potential of our investigational technology to improve the lives of people living with Fabry disease. Natalia Boukharov, our Head of Nonclinical Research, and John Chapin, our Head of Clinical Research, presented a poster at the 8th Update on Fabry Disease Conference in Hamburg, Germany. The aim of the poster presentation, “An analysis of clinical endpoints and industry-sponsored drug development in Fabry disease,” was to foster a conversation on the challenges of designing Fabry clinical trials. Crosswalk received invaluable feedback and insights from experts. It established new connections that will help facilitate the development of our novel investigational ERT to address the current unmet needs of Fabry patients. "Patients with Fabry disease have had marked improvement in the quality of their lives with the availability of enzyme replacement therapies and more recently chaperone therapy. It is very exciting for these patients, and their treaters, to see the second generation of therapies being developed. The current unmet clinical needs of these patients include the development of anti-drug antibodies, and tissue distribution and efficacy, especially for the cardiomyocytes. The present development efforts are directly targeting these issues and may well usher in an exciting new chapter in the treatment of Fabry disease."- David Warnock, MD, Professor of Medicine (Emeritus), University of Alabama at Birmingham. We are so excited to continue our momentum. Everyone at Crosswalk remains dedicated to the Fabry patient community (patients, caregivers, and loved ones) and we will continue to work tirelessly to develop technology and therapies intended to offer new hope and new options. Learn more about the exciting work we’re doing: https://lnkd.in/eXw_HBAK #raredisease #drugdevelopment #startup #lifesciences #biotech #healthinnovation #fabrydisease #healthcare #pharmaceuticals #healthtechnology #ERT #enzymereplacementtherapy

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I wish everyone a very successful 2025. Let's hope that all companies which have exciting new products will be able to secure funding in 2025. Our first video is out now. #clinicaltrials #UK #clinicalresearch

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90% of #therapies fail in #clinicaldevelopment before they reach the market. Great to arrange an interview between Brian K. Buntz, editor, Drug Discovery & Development and Ali Pashazadeh, CEO, Treehill Partners to chew the fat over this. Ali discusses why #therapies fail in #clinicaldevelopment, and how Treehill is actively helping a number of stakeholding #developers and companies across the #biopharma industry being therapies to both #market and #patients. https://lnkd.in/en6apJxg

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AstraZeneca advances its ambition to revolutionise cancer care with new data across its diverse, industry-leading portfolio and pipeline at the IASLC 2024 World Conference on Lung Cancer (WCLC) hosted by the International Association for the Study of Lung Cancer, 7 to 10 September 2024 and the ESMO - European Society for Medical Oncology Congress, 13 to 17 September 2024. Across the two meetings, more than 130 abstracts will feature 17 approved and potential new medicines from AstraZeneca including five Presidential Symposia and 41 oral presentations. Susan Galbraith, Executive Vice President, Oncology R&D, AstraZeneca, said: “The presentations across these two congresses advance our long-term strategy to revolutionise cancer care. We will show results from our computational pathology platform at WCLC which we are using across our antibody drug conjugate portfolio to develop predictive biomarkers to enhance patient selection and improve outcomes for patients. We will also share data on the use of our TROP2 antibody drug conjugate datopotamab deruxtecan in combination with Imfinzi in early-stage lung cancer, a promising first look at clinical activity from two of our own pipeline antibody drug conjugates and important progress for our next-generation immunotherapies.” David Fredrickson, Executive Vice President, Oncology Business Unit, AstraZeneca, said: “Our data featured at WCLC and ESMO will exhibit AstraZeneca’s progress in advancing several of the biggest trends transforming cancer treatment today and potentially in the future. Positive results for Imfinzi from the NIAGARA trial in muscle-invasive bladder cancer will show the importance of integrating perioperative immunotherapy in this setting, and data for datopotamab deruxtecan and Enhertu in several lung cancer settings will highlight how antibody drug conjugates have the potential to replace traditional chemotherapy approaches in many cancer settings.”   #cancertreatment #newdata #IASLC #WCLC #ESMO #Imfinzi #Enhertu #AstraZeneca #WhatScienceCanDo

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What are the challenges and misconceptions about Value Based Contracts (VBC's)? Why has the adoption of VBC's been historically slow? What can we learn from Cell & Gene Therapy VBC's to scale VBC's into Oncology, Obesity, and beyond? How is modern technology accelerating VBC trends? Find out in his thought piece by my colleague Heidi, and feel free to reach out to either one of us for more insights!

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A Comprehensive Overview of the Saudi and Arab Pharmaceutical Market: The Saudi and Arab pharmaceutical markets are transforming rapidly! Saudi Arabia alone surged from $8.6 billion in 2019 to $11.3 billion in 2023, leading regional growth driven by Vision 2030 initiatives, healthcare innovation, and robust investment. From enhanced research capabilities to manufacturing expansion, the sector aligns with national goals for economic diversification and global competitiveness, fueling unparalleled opportunities across the Arab world! Read more in this detailed overview from Makkah Newspaper: https://lnkd.in/dT4dDdXq

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Our recent industry meeting inspired me to share my experience with the clinical trials budget and contract process. As always I try to provide some recommendations on how we can improve the process. At the end of the day our patients will be the winners if we can set up clinical trials much quicker. It is always good to have that in mind! #clinicaltrials #budget #contract #UK

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Thank you Mike Rea for sharing IDEA Pharma | From R&D to RxD “Pharmaceutical Innovation and Invention Index” (2024 results). The read starts with the thought-provoking questions: 1. ‘if you give the same molecule to two companies in phase I, would they be equally successful?' 2. since the answer to 1. is 'no,' 'why'?' Intro excerpt: The Index is generated with information collected across the top 30 (by market cap) pharma/biotech companies on January 1st each year, to rank how they are doing on Invention (i.e., bringing ideas or technologies together in a novel way to create something that did not exist before) and Innovation (i.e., return of investment, creating meaningful value, from invention). No inclusion or exclusion rules, and no payments from the ranked orgs, applied.

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