Todd Luman

BDRA Consulting LLC has been a member of AMDM since 2013. Come join AMDM for the entire story. Twice a year, members get to meet with the FDA to discuss upcoming regulatory and quality policies. The FDA provides industry updates. This year's meeting was held at Building 66, White Oak campus on May 21, 2024. Key Takeaways from the May 2024 AMDM Industry FDA Roundtable: 1. Cybersecurity for medical devices is a key issue. OHT7 and CDRH have increased focus on cybersecurity for medical devices. 2. The Oncology Diagnostic Pilot Program is nearly a year old, and no company has been accepted. This is primarily because drug manufacturers have not been able to provide validation data. 3. Reclassification plans for companion diagnostics are ongoing and will be completed by November 2027. - FDA is considering down-classifying three types of CDxʼs: i. Nucleic acid-based CDX ii. In situ hybridization-based CDX iii. Immuno-histochemistry-based CDX - Technologies will be grouped based on the intended use. 4. PCCP guidance for medical devices will be published before the end of the fiscal year 2024. A PCCP may be used to make changes to sample type changes, which may be acceptable in certain cases. 5. QSIT is being replaced by an entirely novel risk-based approach, which is currently being worked out. Manufacturers should be working through transition plans. #AMDM

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Check out this report from Drug Development & Delivery #Pharma #Biotech #CDMO #Formulation #DrugDevelopment #DrugManufacturing

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Genentech Wins FDA Approval for First Adjuvant Therapy for ALK-Positive, Early NSCLC The FDA on Thursday greenlit the use of Genentech’s Alecensa (alectinib) for the adjuvant treatment of patients with early-stage, anaplastic lymphoma kinase-positive non-small cell lung cancer who have undergone tumor resection. With the approval, Alecensa is now the “first and only” ALK inhibitor approved for this indication and patient population, according to Genentech. Thursday’s regulatory win is backed by data from the Phase III ALINA trial, a randomized, active-controlled and open-label study comparing adjuvant Alecensa with platinum-based chemotherapy. The study enrolled 257 patients with Stage IB to IIIA tumors who had undergone resection and tested positive for anaplastic lymphoma kinase (ALK) using an FDA-approved test. Results from ALINA, which the company released in October 2023, showed that Alecensa could cut the risk of disease recurrence or death by 76% versus platinum-based chemotherapy. This treatment effect was highly significant and yielded a p-value of less than 0.0001. The ALK blocker also led to a significant improvement in central nervous system-disease-free survival, according to an exploratory analysis of ALINA. Genentech CMO Levi Garraway in a statement on Thursday called ALINA’s disease-free survival data “unprecedented,” adding that “Alecensa significantly improves upon the standard of care for people with early-stage ALK-positive lung cancer.”

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The more we understand how #CellTherapies help patients, the better we will be able to develop safer, durable, and efficacious treatments! #TheMoreWeKnow "The immune system overreaction known as cytokine release syndrome (CRS), and neurologic toxicities are two well-known side effects of CAR-T treatment. Last week, regulators removed a requirement for clinicians to report serious cases of the side effects as part of its Risk Evaluation and Mitigation Strategies for Gilead’s treatments Yescarta and Tecartus." https://lnkd.in/efmXtYZ5

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Congratulations to Bristol Myers Squibb, BMS’ Augtyro Wins FDA Accelerated Approval for NTRK-Positive Solid Tumors Bristol Myers Squibb on Thursday announced that its tyrosine kinase inhibitor Augtyro (repotrectinib) was granted accelerated approval by the FDA for the treatment of NTRK-positive, locally advanced or metastatic solid tumors. Under the new indication, Augtyro can be used in patients aged 12 years and older whose disease has progressed after initial treatment. Augtyro can also be used in patients with no satisfactory alternative therapies or in those who are likely to suffer from “severe morbidity” due to surgical resection, according to the company. Under the FDA’s accelerated pathway and to keep Augtyro’s approval, BMS will need to run a confirmatory study to validate the clinical benefit of the drug in this indication and patient population. Nick Botwood, senior vice president of medical oncology at BMS, in a statement called Augtyro’s approval a “milestone” that “helps address this area of unmet need” and underscores the drug’s “clinical value for more people across multiple genetic markers.” “Previously, there was not an FDA-approved treatment option for NTRK-positive cancers that was studied in both TKI-naïve and TKI-pretreated patients across solid tumors,” Botwood said, noting that Augtyro can now provide these patients with an effective treatment option. The FDA’s approval of Augtyro is backed by data from the Phase I/II TRIDENT-1 trial, which enrolled patients who had been exposed to prior TKI treatment and those who were naïve to this drug class. A total of 15 different types of cancer were represented in the study. https://lnkd.in/e8eKHHvp

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This review proposes a set of modifications to existing regulatory requirements for #mRNA products, based on a platform perspective for quality, manufacturing, preclinical, and clinical data. The authors address development and potential regulatory requirements when the mRNA sequences and LNP composition vary in different products as well. In addition, they propose considerations for self-amplifying mRNA, individualized oncology mRNA products, and mRNA therapeutics.  

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Demonstrating the technical leadership of SK pharmteco! 🧬 Catch up with our team at ASGCT to discuss how SKPT can support your #cellandgene development!

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🌍 A Journey of Growth and Impact: Thermo Fisher Scientific 🌍 Discover the transformative journey of Alexis R. Director of EHS and Sustainability at Thermo Fisher Scientific. He touched on the opportunities for growth, the importance of compliance and safety, and the impact on making the world healthier, cleaner, and safer. Click on the link for the full article and be inspired by Thermo Fisher's mission. #LifeAtThermoFisher

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Umoja Biopharma Announces Publication in Blood Highlighting Successful Generation of In Vivo CAR T-cells in Non-Human Primate Modeling First landmark, peer-reviewed publication of successful in vivo CAR T-cell data in an immune competent NHP model Data validate the VivoVec™ platform in a translationally relevant model and support its transition into human clinical testing, offering a potential paradigm shift in the field of CAR T-cell therapies Key Study Findings VivoVec particles (VVPs) incorporating CD80 and CD58 costimulation proteins exhibit enhanced capacity for in vivo CAR T-cell generation and produce CAR T-cells with increased antitumor functionality compared to those produced from VVPs displaying anti-CD3 scFvs alone. Combining the anti-CD3 scFv together with the ligand binding domains of CD80 and CD58 into a single multi-domain fusion (MDF) protein markedly augments the particles’ ability to bind, activate, and transduce T-cells. VVPs incorporating MDF compatible with NHP T-cell activation and costimulation potently generate anti-CD20 CAR T-cells in vivo, comprising up to 65% https://lnkd.in/eqsSTKjf

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RNAi-based drug design: considerations and future directions More than 25 years after its discovery, the post-transcriptional gene regulation mechanism termed RNAi is now transforming pharmaceutical development, proved by the recent FDA approval of multiple small interfering RNA (siRNA) drugs that target the liver. Synthetic siRNAs that trigger RNAi have the potential to specifically silence virtually any therapeutic target with unprecedented potency and durability. Bringing this innovative class of medicines to patients, however, has been riddled with substantial challenges, with delivery issues at the forefront. Several classes of siRNA drug are under clinical evaluation, but their utility in treating extrahepatic diseases remains limited, demanding continued innovation. In this Review, we discuss principal considerations and future directions in the design of therapeutic siRNAs, with a particular emphasis on chemistry, the application of informatics, delivery strategies and the importance of careful target selection, which together influence therapeutic success. https://lnkd.in/ecB46Cfr

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The latest U.S. Biotechnology Instruments Market report unveils remarkable growth and new opportunities. Here are 10 essential takeaways for every biotech professional: 1️⃣ Market Growth Projections: The market is set to surge from USD 36.16 billion in 2024 to an impressive USD 49.11 billion by 2033, with a CAGR of 3.46%. 2️⃣ Dominance of Analytical Instruments: Analytical instruments, holding a 65.9% market share in 2023, play a vital role in biotechnological applications, from research to quality control. 3️⃣ Rapid Growth in Cell Culture Instruments: The cell culture instruments segment is poised for the fastest growth through 2033, driven by advancements in biopharmaceuticals, regenerative medicine, and personalized healthcare. 4️⃣ Pharmaceutical & Biotech Companies Lead in End-Use: Dominating the end-use segment with a 40.11% market share in 2023, these companies showcase the industry's dedication to drug discovery and genetic engineering. 5️⃣ Rising Investment in Precision Medicine: With expanding applications, precision medicine presents substantial opportunities, boosting demand for biotechnology instruments crucial for genomic sequencing and biomarker analysis. 6️⃣ Regional Innovation Hubs: California and Massachusetts remain strongholds of biotech innovation, with emerging clusters in Texas and North Carolina contributing significantly to industry growth. 7️⃣ Addressing Regulatory Challenges: Navigating regulatory complexities, especially around gene editing and stem cell research, is crucial for sustaining innovation and market growth. 8️⃣ Global Market Size Estimates: The global biotech instruments market is projected to grow from USD 89.85 billion in 2023 to USD 132.36 billion by 2033, at a CAGR of 3.95%. 9️⃣ Recent Product Innovations: 2024 saw notable advancements, including LICORbio's Odyssey® F biomolecular imaging systems and Waters Corporation’s Alliance iS Bio HPLC System, enhancing productivity and research capabilities. 🔟 Impact of Biopharmaceutical R&D: The expansion of biopharmaceutical R&D, focusing on biologic drugs like monoclonal antibodies and cell therapies, is driving increased demand for sophisticated biotechnology instruments. For those looking to stay ahead, these insights provide a roadmap to understanding market dynamics and seizing growth opportunities. Dive into the full report for an in-depth analysis. Procure Complete Report (220+ Pages PDF with Insights, Charts, Tables, and Figures) @ https://lnkd.in/dGry4wGJ #Biotech #MarketInsights #Innovation #PrecisionMedicine #Genomics #Biopharmaceuticals #BiotechInvestments #RegulatoryAffairs #BiotechResearch #MarketGrowth Let's continue to elevate the biotech industry together! 🚀

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What cancer clinical treatment cut the risk of death by more than half? A two months increase in life span is usually heralded. The combination of Kytruda (Merck) that enhances the immune response generally, and Padcev (Seattle Genetics), an antibody to nectin-4 that is over expressed in cancer cells, conjugated to a cell division disrupter, did just that for bladder cancer (Nature 623,p.231, 2023). The latter drug is a biotechnology Antibody Drug Conjugate (ADC). See also the blog https://lnkd.in/gaN_WB8F

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GMP-manufactured CRISPR/Cas9 technology as an advantageous tool to support cancer immunotherapy The latest advancements in GMP procedures are allowing an efficient improviement of personalized medicine. In particular immunotherapy is strongly taking advantage of clinical manufacturing platforms to cure patients who are refractory to previous therapy or who relapse upon a first period of remission. CRISPR/Cas9 technology has become the most widely used gene-editing tool in cancer immunotherapy favoring differentiation and persistence of genetically modified T-cells. The discovery of cancer-selected markers remains one of the principle obstacles while the use of allogenic CRISPR/Cas9-modified CAR T-cells is overcoming the difficulties to treat relapsing cancer cells showing an antigen different from that expressed at the onset of the disease. CAR T-cells generated against multiple tumor targets are preventing relapsing events. The manufacturing processes still comprised procedures performed manually even if supported by semi-automated manner, which result in product variability and very high cost. The development of a more controlled and cost-effective manufacturing process remains the pivotal aim to ensure CAR T-cells therapy for all patients. https://lnkd.in/eWkEGiNq

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We just released our 2023 Amgen ESG Report. Environmental Sustainability (Healthy Planet section) highlights are: 1. We remain on track to meet our 2027 Ambition to dramatically reduce carbon, water, and waste footprints. 2. We advanced Amgen EcovationTM — our approach to innovative and sustainable manufacturing — with our newest biomanufacturing facility in Ohio that was built to LEED Gold standard and includes solar arrays. 3. We formalized our expectations of engagement with suppliers to support their adoption of science-based targets. This is important as more than an estimated 90% of our greenhouse gas emissions come from our value chain. #mycompany #sustainability #innovation https://lnkd.in/g89fRurs

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Astellas Inks Second CAR-T Agreement with Poseida to Develop ‘Convertible’ Cancer Therapies Astellas Pharma on Wednesday entered into a collaboration and license agreement with Poseida Therapeutics to develop novel CAR-T therapies for solid tumors. Under the deal, Astellas will pay an upfront fee of $50 million and has pledged up to $550 million in potential development, sales and other contingent payments. Poseida will also be eligible for up to low-double digit tiered royalties on net sales. Astellas will conduct the transaction through its subsidiary Xyphos Biosciences. The collaboration is designed to combine “the innovative cell therapy platforms from each of the companies,” according to Wednesday’s announcement. Poseida will contribute its proprietary allogeneic CAR-T platform, while Xyphos will lend both its ACCEL and convertibleCAR technologies. The partnership’s goal is to produce one CAR-T construct, which in turn will yield two convertibleCAR product candidates for solid tumors. https://lnkd.in/gGZG4KKY

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Interesting trend: a federal judge on Monday just threw out BMS and J&J's challenges to the drug price negotiations outlined in Inflation Reduction Act. Given the 9 year patent protection for small molecules compared to the 13 year protection for biologics, it seems like investments for biologics will continue to gain traction. Biologics received 30% more funding than small molecules in 2022, and 50% more investment in 2023. Something to consider if you're looking for "stability" at your next employer in the Biotech/Pharma space.

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It’s difficult to navigate the balance phase-appropriate needs with commercial readiness during cell and gene therapy development. Partnering with a trusted and experienced viral vector #CDMO can help unlock unique insights and provide guidance to achieve the right balance for your therapeutic. Here are 4 recommendations to keep in mind during preclinical and clinical development:

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