Phil Murray

CD37 is a safe chimeric antigen receptor target to treat acute myeloid leukemia Highlights • CD37 is expressed on AML blasts • CD37 expression correlates with ELN 2017 risk stratification • CD37CAR T cells are efficient against AML in vitro and in vivo • CD37CAR T cells do not deplete myeloid progenitors Acute myeloid leukemia (AML) is characterized by the accumulation of immature myeloid cells in the bone marrow and the peripheral blood. Nearly half of the AML patients relapse after standard induction therapy, and new forms of therapy are urgently needed. Chimeric antigen receptor (CAR) T therapy has so far not been successful in AML due to lack of efficacy and safety. Indeed, the most attractive antigen targets are stem cell markers such as CD33 or CD123. We demonstrate that CD37, a mature B cell marker, is expressed in AML samples, and its presence correlates with the European LeukemiaNet (ELN) 2017 risk stratification. We repurpose the anti-lymphoma CD37CAR for the treatment of AML and show that CD37CAR T cells specifically kill AML cells, secrete proinflammatory cytokines, and control cancer progression in vivo. Importantly, CD37CAR T cells display no toxicity toward hematopoietic stem cells. Thus, CD37 is a promising and safe CAR T cell AML target. https://lnkd.in/esqup3tf

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More and more talk about Pb-212 being a realistic alternative to Ac-225 as an alpha emitter in RLT. Great to see!

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After a tough year, we will see a very different CGT landscape in 2025. As companies are striving for commercial viability, the points below make for interesting reading as to what some companies will need to consider along the way. What's your prediction for 2025? #CellandGeneTherapy #Biotech #Biopharma

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Mustang Bio Bio Announces Favorable Efficacy and Safety Data from Complete Waldenstrom Macroglobulinemia Cohort of Phase 1/2 Clinical Trial of MB-106, CD20-Targeted Autologous CAR-T Therapy WORCESTER, Mass., June 17, 2024 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (“Mustang” or the “Company”) (Nasdaq: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell therapies into potential cures for difficult-to-treat cancers, today announced that updated data from the ongoing Phase 1/2 clinical trial of MB-106, a CD20-targeted, autologous CAR T-cell therapy, show a favorable safety and efficacy profile in patients with Waldenstrom macroglobulinemia (“WM”), a rare form of blood cancer. MB-106 is being developed in a collaboration between Mustang and Fred Hutch Cancer Center (“Fred Hutch”) to treat patients with relapsed or refractory B-cell non-Hodgkin lymphomas (“B-NHLs”) and chronic lymphocytic leukemia (“CLL”). The updated results from the single-institution Phase 1/2 clinical trial were presented during a poster session at the European Hematology Association 2024 Hybrid Congress (“EHA2024”) by Brian Till, M.D., Associate Professor and physician at Fred Hutch and University of Washington. All ten patients in the study were previously treated with Bruton's tyrosine kinase inhibitors (“BTKi”), and their disease continued to progress while on BTKi. Overall, 90% (9/10) of the patients treated with MB-106 responded to treatment, including 3 complete responses, 2 very good partial responses and 4 partial responses. In addition, 1 patient experienced stable disease. One of the patients who achieved a complete response has remained in remission for 31 months, with an immunoglobulin M (IgM) level that decreased rapidly to the normal range after treatment with MB-106 and has remained normal since. Patients had a median of nine prior lines of therapy and only one patient has started additional anti-WM treatment after being treated with MB-106. https://lnkd.in/eRSu7Msk

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Interested in KRAS? Tempus is presenting Beyond G12: Comprehensive RAS Biomarker Strategy and Clinical Development at RAS-Targeted Drug Development Summit 2024 KRAS G12C is only the first druggable alteration in RAS genes. Tempus’ multimodal, de-identified data and certain assays can help identify a broad set of biomarkers in responsive patients including KRAS, HRAS, and NRAS amplifications, over-expression, and mutations, in addition to tumor immune microenvironment factors that may influence response to RAS neoantigen vaccines, revealing critical insights for targeted therapy development. Gain perspective on the significance of RAS alteration patterns to outcomes to standard of care and their potential to guide precision oncology, enhancing treatment efficacy and patient outcomes. Not attending? Let's connect! #PrecisionOncology #ClinicalDevelopment #BiomarkerStrategy #RAS #KRAS #NRAS #G12C

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Moderna exits gene editing deal worth up to $3B biobucks, leaving Metagenomi in the spotlight Earlier this year, Metagenomi had the spotlight with a $93.7 million IPO. Now, Moderna is exiting stage left on a partnership worth up to $3 billion in biobucks.  Back in 2021, Moderna entered the gene editing space via a four-year partnership with young Metagenomi. The research pact went after in vivo treatment options for serious genetic diseases, with Metagenomi offering access to its gene editing tools and Moderna sharing expertise in mRNA and lipid nanoparticle delivery technologies.   Now, both companies have come to the mutual decision to end the collaboration, Metagenomi CEO Brian Thomas, Ph.D., told Fierce Biotech in a May 1 interview. The move coincides with a strategic prioritization at Moderna. Requests for comment sent to Moderna were not returned as of publication.  Since Metagenomi has emerged on the Nasdaq, the terms of the deal have become public. Moderna handed over $70 million upfront in equity and cash and offered Metagenomi more than $3 billion in potential biobucks, according to Securities and Exchange Commission (SEC) documents.   https://lnkd.in/gCxgMc3Y

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Epoch 38: BIOTECH HEALTH - IS UP REALLY UP? 👉 Macro - rates and elections 👉 Deal valuation comps 👉 Pharma exit activity metrics https://lnkd.in/dEkBy2bY

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Novartis pays Schrödinger $150M, bets billions on the back end to form drug discovery pact Novartis is paying $150 million for a closer look at Schrödinger’s catalog of programs. The Big Pharma will work with Schrödinger to advance some of the computing-enabled R&D shop’s discovery programs and identify candidates against additional targets. Schrödinger made its name in the 1990s as a developer of R&D software before moving deeper into drug development, first through projects such as the spinout Nimbus Therapeutics and later by building out its own capabilities. The jury remains out on the success of the expansion—investors have sent the stock down 44% this year—but Novartis sees promise in some of the assets Schrödinger has identified. In return for $150 million upfront, Schrödinger has entered into a multiyear, multi-target collaboration and license agreement with Novartis. The partners will jointly advance several of Schrödinger’s existing, undisclosed non-cancer discovery programs and identify drug candidates against targets in Novartis’ core therapeutic areas. https://lnkd.in/e55ti9yn

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The biopharma industry is facing a $183 billion patent cliff as major drugs lose exclusivity through 2030, with companies like Amgen, BMS, and Merck most exposed. However, Morgan Stanley analysts highlight a $383 billion opportunity for M&A to address this revenue gap. With challenges like the patent expiration of Amgen’s Prolia, BMS’s Eliquis, and the elephant-in-the-room - Merck's Keytruda that generated $25 billion in sales last year - companies are leveraging acquisitions to secure their future pipelines. For example, Amgen’s $27.8 billion Horizon buyout and BMS’s acquisition spree of Karuna, Mirati, and RayzeBio underscore the role of M&A in mitigating risks. As competition for innovative assets intensifies, it is likely that the M&A will be more and more prominent. In fact Morgan Stanley's report estimates that Big Pharma has over $380 billion available for making deals. The evolving landscape highlights how strategic dealmaking remains critical for biopharma's ability to innovate and grow. Read more in the article in Fierce Biotech: https://lnkd.in/eykM7HPN #manda #pharma #patentcliff #innovation #merck #bms #amgen

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Proud to have fellow Biotechnology Innovation Organization board member and Arcutis Biotherapeutics, Inc. (Nasdaq: ARQT) CEO Frank Watanabe testify at the House Ways and Means Committee hearing on July 12th in Salt Lake City. Frank highlighted the contributions the biotech industry has made to human health and the economy: "In the last quarter century, American life expectancy has continued its steady increase upwards, with roughly one-third of that improvement directly attributable to biopharmaceutical innovations.” The big picture: The industry’s direct and indirect contribution to the U.S. GDP is anywhere from 2-7%, with an economic impact of $2.9 trillion in 2021. Biotech capabilities enhance national security by ensuring we can care for ourselves. Legislative proposals that would support innovation include: - The Ensuring Pathways to Innovative Cures (EPIC) Act, which would give small molecule drugs the same 13-year protection from price controls as biologics, and the ORPHAN Cures Act, which would exempt orphan drugs from price controls even if approved for more than one indication. - The Tax Relief for American Families and Workers Act (H.R. 7024), which was passed by the House earlier this year, to restore the R&D tax deduction. Allow for monetization of net operating losses (NOLs) and reform Section 382 on NOLs. - Improve the capital gains exemption under Section 1202 to encourage investment in small biotechs. - Ensure strong strong patent protections and reasonable regulation of M&A activity. Proud of the work BIO does to support the industry, and proud to be a part of community that works tirelessly to drive innovation and improve human and planetary health. #patientsfirst #biotechnology #innovation #humanhealth #sustainability

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As clinical research sites become overburdened with new technologies and standalone applications, sponsors are focusing more on how to be better partners to their sites. In my recent blog, I discuss how a top 20 biopharma streamlines study execution with a holistic site engagement strategy. By focusing on sites’ needs, they are able to:  📉 Reduce costs  💰 Use resources more efficiently  🏃‍♂️ Accelerate study start-up 🤝 Improve compliance and site collaboration Read the blog to learn more about their scalable blueprint to automate study training, simplify information sharing, and reduce site burden: https://bit.ly/3Yj6VJd

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Monica Ravindra Shah, MD, FACC, CTI Clinical Trial and Consulting Services's Chief Medical Officer, had the opportunity to host a forum at Alliance for Regenerative Medicine Cell and Gene Meeting on the Mesa. A few key takeaways for cell and gene therapies in cardiovascular disease (CVD): -Despite the global burden of CVD only a small fraction of advanced therapies are in CV indications -It is important to target patients at the optimal timing of disease progression -A clear understanding of the question you are trying to answer is a critical component of the clinical development plan -There is an opportunity for novel endpoints including biomarkers and intermediate endpoints CTI Clinical Trial and Consulting Services has a team of experts that can help navigate each of these points.

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An exciting to merge two technologies in one platform for next generation of in vivian CAR-T therapy. Nona Biosciences Enters into Collaboration Agreement with Umoja Biopharma Biopharma to Advance In Vivo CAR-T Cell Therapies CAMBRIDGE, Mass., Sept. 12, 2024 /PRNewswire/ -- Nona Biosciences, a global biotechnology company providing a total solution from "Idea to IND" (I to ITM), announced today that it has entered into a multi-target antibody discovery collaboration with Umoja Biopharma, a transformative immunotherapy company creating off-the-shelf treatments that aim to extend the reach and effectiveness of CAR-T cell therapies in oncology and autoimmunity. This collaboration leverages Nona's proprietary fully human heavy chain only antibody (HCAb) technology to produce novel in vivo generated CAR-T cell therapy drug candidates. Fully human HCAbs have the potential to significantly reduce immunogenicity and offer versatility in CAR design due to their compact size, simplified structure, and precisely calibrated binding properties. This partnership aims to combine Nona's HCAb Harbour Mice® platform and direct CAR-function-based HCAb library screening platform (NonaCarFx™) with Umoja's VivoVec™ platform to develop novel in vivo CAR-T cell therapies and expand the potential reach of this innovative delivery technology. https://lnkd.in/eEz8CQ4G

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Syndax Pharmaceuticals inks $350M deal with Royalty Pharma for funding to launch 2 new drugs With its first FDA approval coming in August and a second expected next month for another of its treatments, these are exhilarating days for 19-year-old Syndax Pharmaceuticals as it transforms into a commercial enterprise. To help foot the bill to launch its approved graft-versus-host disease drug Niktimvo (axatilimab) and the expected launch of its leukemia treatment revumenib, Syndax has struck a $350 million deal with Royalty Pharma. In exchange for the $350 million upfront, Syndax will pay a 13.8% royalty on U.S. sales of Niktimvo. The deal expires after Royalty gains 2.35 times its investment. “We expect this transaction to fund us through profitability, while ensuring that we continue to participate in the profits from Niktimvo and retain the upside of its future growth. With this significant infusion of capital, we are well positioned to successfully launch two first-in-class medicines and expand their opportunity with additional indications,” Michael Metzger, Syndax’s CEO, said in a release. With its strong profile as a third-line treatment for chronic graft-versus-host disease (cGVHD) and its potential to move into earlier lines, Niktimvo has been pegged with peak sales potential of $1 billion by Leerink Partners. Syndax plans to launch Niktimvo in early 2025. In 2016, Syndax gained exclusive rights to develop and commercialize axatilimab from UCB. Five years later, Incyte bought in to co-develop and co-commercialize axatilimab in cGVHD and future indications. https://lnkd.in/gnJxYJzZ

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DAC (Degrader Antibody Conjugate) is quickly growing. This big deal in DAC between Vertex Pharmaceuticals and Orum Therapeutics is the latest one after the following three major deals in DAC. 1. The deal between Nurix Therapeutics and Seagen in Sep., 2023; 2. The deal between Orum Therapeutics and Bristol Myers Squibb in Nov., 2023; 3. The deal between C4 Therapeutics, Inc. and Merck in Dec., 2023.

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@Eli Lilly to buy bowel disease drug developer Morphic Holding LLC for $3.2 bln July 8 (Reuters) - Eli Lilly (LLY.N), opens new tab  will acquire Morphic Holding (MORF.O), opens new tab  for $3.2 billion in cash, the companies said on Monday, giving the U.S. drugmaker access to an experimental drug for types of inflammatory bowel diseases. Shares of drug developer Morphic surged 76% to $56.15 premarket on Lilly's offer of $57 per share, which represents a 79% premium to stock's last closing price. Morphic's lead drug MORF-057 is an oral treatment that is being evaluated in two phase 2 studies in ulcerative colitis patients and one phase 2 study in Crohn's disease. Ulcerative colitis is a condition where abnormal reactions of the immune system cause inflammation and ulcers on the inner lining of the colon, which can lead to diarrhea, passing of blood with stool and abdominal pain. Last year, the U.S. Food and Drug Administration approved Lilly's Omvoh for treating adults with moderate-to-severe active ulcerative colitis. The drug is among Lilly's potential growth drivers for this decade alongside its diabetes and obesity drugs, Mounjaro and Zepbound. https://lnkd.in/eRk9dFRv

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