Jeff Bockman

𝐑𝐄𝐆𝐔𝐋𝐀𝐓𝐎𝐑𝐘 𝐑𝐎𝐔𝐍𝐃𝐔𝐏 ⚛️ The FDA has released new draft guidance recommending best practices for multi-regional clinical trials (MRCTs) in oncology. Sponsors are encouraged to ensure that the outcomes of global oncology clinical development programs are understandable for US patients in support of marketing applications. 🌐 The FDA's final guidance on decentralized clinical trials (DCTs) clarifies several concerns raised by clinical experts and industry stakeholders regarding the earlier draft. This includes detailed requirements for maintaining a task log for healthcare providers, guidelines for physical inspections, and addressing challenges related to data variability in DCTs. 🤖 As the use of artificial intelligence (AI) tools, particularly generative AI, expands, experts remind regulatory professionals to prioritize the human aspect. Although these tools offer significant benefits, they necessitate human oversight and interpretation to be fully effective. During the opening plenary session, experts discussed how AI can be utilized across various sectors, the potential of this technology, and the hurdles in achieving its full benefits. Panelists included Ryan Kurtzman from the City of Long Beach, Bradley Thompson of Epstein Becker Green, Michelle Jump from MedSec, and Jay Mashburn from Recursion Pharmaceuticals, with moderation by Megha Iyer of Thermo Fisher Scientific. 🧬 As biosimilars gain market presence, they are generating competition with original products. However, patient adoption of biosimilars varies based on demographics, location, care setting, prescriber specialties, and types of insurance, according to two recent studies in Health Affairs. 🌐 The FDA has observed a rise in complete response letters (CRLs) issued to sponsors of biologics license applications (BLAs) over the last decade, with facility deficiencies being the most common reason cited, according to Leslie Rivera Rosado, a supervisory interdisciplinary scientist at the agency's Center for Drug Evaluation and Research (CDER). #Pharmaceuticals #Biotechnology #LifeSciences #PharmaNews #Biotech #HealthcareInnovation #DrugDevelopment #ClinicalResearch #Biopharma #PharmaTech #MedicalResearch #Healthcare #BioPharmaceuticals #PharmaIndustry #BiotechIndustry #PharmaCareers #MedicalInnovation #NewDrugs #Genomics #BiotechNews

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💊 #Wegovy Advances in #MASH Treatment but Faces Limits as a Long-Term Solution GLP-1s strike again... ❗ Wegovy’s results offer meaningful progress in addressing MASH, a complex and increasingly prevalent #liverdisease that currently lacks effective long-term treatments. However, while #GLP-1 receptor agonists like semaglutide show promise, they’re unlikely to revolutionize MASH management on their own, as evidenced by modest fibrosis improvement and the need for polypharmacy to address multifaceted disease pathways. This outcome aligns with broader trends in the field, where combination #therapies appear crucial to tackling the advanced stages of MASH. As Wegovy and similar drugs progress, their ultimate value may lie in complementary roles alongside other #treatments, reshaping MASH care without single-handedly transforming it.

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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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Led by Markus Renschler, MD, Light Horse Therapeutics has made significant progress in the oncology space with a $62M #SeriesA funding round led by MPM BioImpact and Omega Funds, alongside Novartis Venture Fund. This marks a big step for their work in developing LHT-001, a promising candidate targeting critical cancer pathways. In parallel, their partnership with Novartis could be worth up to $1B, combining Light Horse's innovative pipeline with Novartis' expertise in oncology drug development and commercialization. This collaboration has the potential to address unmet needs in cancer treatment and accelerate progress in this critical field. #Biotech #Oncology #Immunology #Innovation #LightHorseTherapeutics #Novartis #MPMBioImpact #OmegaFunds

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What an interesting read on the shift from traditional patient recruitment methods in rare disease! Learn more about how to optimize patient-driven site engagement in my team member’s blog: https://bit.ly/4eHoBn4

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🔍 ApoB: A Critical Marker in Cardiovascular Health 🔍 At UDS, we include ApoB in our advanced heart biomarker panel, paired with a comprehensive metabolic panel, during wellness visits for Firefighters and law enforcement officers. 🫀 Why ApoB Matters: ApoB reflects the number of atherogenic particles in the blood, making it a more accurate predictor of cardiovascular risk than LDL cholesterol alone. 👮‍♂️ Protecting Those Who Serve: Cardiovascular disease remains a leading health risk for first responders. By detecting it early through ApoB testing, we empower departments to take proactive steps for better health outcomes. ✅ Prevention starts with data—The action begins with awareness. #ApoB #CardiovascularHealth #WellnessScreening #LawEnforcement #EarlyDetection #UDS #HealthBiomarkers

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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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Excited to share the latest feature in our "Partnering Spotlight" series, where we share some of the connections we are building to foster scientific innovation in healthcare. In this edition, our very own Neha Kaushal sits down with Daniel Anderson, Professor of Chemical Engineering at MIT, to discuss the potential of innovative #mRNA applications in vaccines and novel treatments for genetic diseases. Check out the video to learn more about our collaboration! https://lnkd.in/eHwpyjeY #vaccines #immunoscience

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Another sizable licensing deal East-West. Interestingly, the licensor is not a big pharma. Showing that significant value creation could be captured by small/mid-size Western players. But also how crowded the space could become. #biotech #innovation

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Our recent webinar on the potential of targeting innate immunity in immunotherapy of cancer (https://lnkd.in/eSK5rBew) brought together a diverse group of experts to discuss the latest advancements and challenges in the field. The session began with a quick educational overview of the innate immune system, explaining its crucial role in detecting pathogens and tissue damage through pattern recognition receptors. The distinguished panelists -- Frank Borriello, Bjorn Frendeus, Anne Goubier, Arthur Krieg, Heidi LeBlanc, and Michael J. Newman, Ph.D. -- emphasized that while the adaptive immune system, particularly CD8+ T cells, has been the focus of cancer immunotherapy, the innate immune system offers additional pathways that can be harnessed for better therapeutic outcomes. One of the major themes discussed was the need for combination therapies that activate both innate and adaptive immune responses. Single-target approaches have shown limited success, and panelists highlighted the potential of combining multiple innate immune activators or integrating innate and adaptive strategies to enhance anti-tumor responses. This approach could help overcome resistance seen with current therapies, such as checkpoint inhibitors, and improve outcomes for a broader range of patients. The speakers noted that understanding the interplay between different immune cells and pathways is crucial for developing effective combination therapies. The discussion also touched on the challenges of translating preclinical findings into clinical success, particularly due to species differences in innate immunity. The panelists stressed the importance of human tumor biopsies and advanced technologies like spatial genomics to better understand and optimize immune responses in patients. Panelists debated the merits of systemic versus local administration of innate immune activators. Systemic administration could address metastatic disease by inducing a widespread immune response, while local administration might concentrate immune activation at the tumor site but could potentially lead to systemic effects ("abscopal") through draining lymph nodes. The role of myeloid cells, including dendritic cells and macrophages, was highlighted as crucial in transforming immune deserts (T cell-poor environments) into immune-active ones, thereby enhancing the effectiveness of immunotherapies. The concept of innate immune memory, also known as trained immunity, was discussed as an important mechanism that can provide lasting anti-tumor effects even in the absence of adaptive immune responses like T cells. In conclusion, the experts envisioned a future where innate immune activators could become a standard part of cancer treatment. #immunooncology #immunotherapy #cancer #oncology

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Immunovant 𝗶𝘀 𝘀𝗵𝗶𝗳𝘁𝗶𝗻𝗴 𝗶𝘁𝘀 𝗳𝗼𝗰𝘂𝘀 𝗳𝗿𝗼𝗺 𝗯𝗮𝘁𝗼𝗰𝗹𝗶𝗺𝗮𝗯 𝘁𝗼 𝗜𝗠𝗩𝗧-𝟭𝟰𝟬𝟮, 𝗮𝗻𝗼𝘁𝗵𝗲𝗿 𝗙𝗰𝗥𝗻 𝗶𝗻𝗵𝗶𝗯𝗶𝘁𝗼𝗿 𝘄𝗶𝘁𝗵 𝗽𝗿𝗼𝗺𝗶𝘀𝗶𝗻𝗴 𝗽𝗼𝘁𝗲𝗻𝘁𝗶𝗮𝗹 𝗶𝗻 𝘃𝗮𝗿𝗶𝗼𝘂𝘀 𝗮𝘂𝘁𝗼𝗶𝗺𝗺𝘂𝗻𝗲 𝗱𝗶𝘀𝗼𝗿𝗱𝗲𝗿𝘀, 𝗶𝗻𝗰𝗹𝘂𝗱𝗶𝗻𝗴 𝗠𝘆𝗮𝘀𝘁𝗲𝗻𝗶𝗮 𝗚𝗿𝗮𝘃𝗶𝘀. This decision gives argenx’s Vyvgart, a competing treatment for myasthenia gravis (MG), more market lead time. Immunovant is also a competitor of UCB in the treatment of myasthenia gravis (MG). Both companies are developing or marketing FcRn inhibitors for this autoimmune disorder. Immunovant's focus is on IMVT-1402 and batoclimab, while UCB is on rozanolixizumab, another FcRn inhibitor for MG. Yes, UCB has received European Commission (EC) authorization for rozanolixizumab, marketed as RYSTIGGO on January 2024. The competition in this therapeutic area is centered around offering effective treatments for MG, with each company aiming to demonstrate the efficacy and safety of their respective candidates. Immunovant plans to start several registrational studies for IMVT-1402 by March 2025 and additional trials by March 2026. Results for batoclimab in MG and Graves’ disease are expected soon, influencing its future development strategy. ------ I am Matteo and I support you in market knowledge and strategy. How? Since 2001, I have been involved in business, market, and competitive intelligence, helping companies define strategies in line with market and competitive scenarios. #marketintelligence #competitiveintelligence #strategy #marketsmind #pharmaceutical

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We started out Q1 with cautious optimism and the numbers continue to show the confidence was warranted. Biopharma M&A activity more than doubled nationally during the year’s first three months, coinciding with a rebound in venture capital deals as well as a flurry of IPOs. These are all good signs that the drug development cycle is normalizing and should translate into additional funding being available at the seed and Series A stages. Certain to be on the minds of funders and startups attending #AlignSummit24 on Thursday! https://lnkd.in/erEiJm6S  

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Tomorrow will be the first meeting of the FDA's newly formed, Genetic Metabolic Diseases Advisory Committee (GeMDAC). The GeMDAC will be discussing Zevra Therapeutics' NDA for arimoclomol for the treatment of Niemann-Pick Disease Type C (NPC). NPC is an ultra-rare neurodegenerative lysosomal storage disease which leads to neuronal death, uncontrolled seizures, and ultimately fatal dementia and early death by age 13 years for most patients. The unmet need for patients with NPC and their families is unimaginably urgent, with no FDA-approved treatments that can alter the course of the disease. In addition to a robust discussion of the specifics of the clinical data for arimoclomol to treat NPC, I am looking forward to observing how this new Committee works together to address the complex challenges of developing drugs for rare genetic metabolic diseases.

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Sharing some fresh survey results run by Daniel Erlanson, an industry thought leader in #fragment based drug discovery, who also keeps a blog on #practicalfragments. It is very interesting to know that X-ray crystallography is still the dominant working horse for fragment lead finding for innovative small molecule drug discovery, in the face of #AI and many other emerging tools. https://lnkd.in/gaMByTci #FBDD, #FBLD, #SBDD #XRay #crystallography #CryoEM #SPR #TSA (#DSF) #CADD, #MS (#ASMS), #FragmentLibrary #Screening #CrystalSoaking - all are flagship service areas at Viva Biotech

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Janux Therapeutics, a clinical-stage biopharmaceutical company, announced positive interim clinical results for its JANX007 program, a Tumor Activated T Cell Engager (TRACTr) therapy targeting metastatic castration-resistant prostate cancer (mCRPC). The data highlights substantial activity in heavily pre-treated patients, paving the way for expansion trials in earlier treatment lines. The Phase 1a clinical trial enrolled mCRPC patients who had received a median of four prior lines of therapy. As of November 15, 2024, sixteen pre-PLUVICTO® patients were treated with JANX007 once weekly at doses ranging from 2 mg to 9 mg. Across all doses, the therapy demonstrated significant prostate-specific antigen (PSA) response rates, with 100% of patients achieving at least a 50% PSA decline (PSA50), 63% achieving at least a 90% PSA decline (PSA90), and 31% achieving at least a 99% PSA decline (PSA99). These PSA declines were durable, with 75% of patients maintaining PSA50 declines for at least 12 weeks and 50% maintaining PSA90 declines for at least 12 weeks at doses of 2 mg or higher. The robust responses were observed regardless of resistance driver aberration status or prior exposure to taxane-based chemotherapy or androgen receptor pathway inhibitors (ARPi). In patients evaluable by RECIST criteria, anti-tumor activity was evident, with confirmed or unconfirmed partial responses seen in 50% (4 out of 8) of patients. JANX007 demonstrated a favorable safety profile. Cytokine release syndrome (CRS) and related adverse events were mild (grades 1 and 2) and generally occurred during the first treatment cycle. Other treatment-related adverse events were similarly mild and mostly confined to the initial cycle. Importantly, the maximum tolerable dose has not yet been determined. Based on these promising findings, Janux has selected two once-weekly step-dose regimens for Phase 1b expansion trials, which will focus on pre-PLUVICTO® second- and third-line patients. The company plans to provide additional updates on the JANX007 program in 2025. JANX007, which targets prostate-specific membrane antigen (PSMA), is the company’s first clinical candidate for prostate cancer. Janux is also advancing JANX008, its second clinical candidate targeting epidermal growth factor receptor (EGFR), currently in a Phase 1 trial for various solid tumors, including colorectal carcinoma, head and neck squamous cell carcinoma, non-small cell lung cancer, renal cell carcinoma, small cell lung cancer, pancreatic ductal adenocarcinoma, and triple-negative breast cancer. In addition, the company is progressing several preclinical programs focused on TRACTr and Tumor Activated Immunomodulator (TRACIr) therapeutics. 𝐅𝐨𝐫 𝐦𝐨𝐫𝐞 𝐝𝐞𝐭𝐚𝐢𝐥𝐬: https://lnkd.in/dbG-DbAk David Campbell Charles Winter Carolina Caffaro

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What could today's decision mean for biopharma? As former President Donald Trump returns to power The Associated Press projected his presidential victory Wednesday morning — the biopharma industry may see the door open wider for more dealmaking, with Lina Khan facing an exit at the Federal Trade Commission..... ...But Trump’s presumptive victory could also send shockwaves across the biopharma industry if his newly-appointed leaders at HHS or the FDA seek major reforms for their agencies under the “Make America Healthy Again” slogan.... https://lnkd.in/gsHjPa_t #Biopharma #HealthcarePolicy #FDA #LifeSciences #DrugPricing #PharmaDeals #BusinessDevelopment #RepublicanLeadership #USPolitics #FederalTradeCommission #InnovationEconomy #HealthcareReform #Investment #celltherapy #genetherapy #advancedtherapies #regenerativemedicine #biotech #biotechnology #lifescience #lifesciences #dealmaking #mergersandacquisitions #USHealthcare

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🚀 Weekly Biotech Roundup: Key Developments in R&D This week, the spotlight is on Bluebird Bio as the FDA investigates potential links between its gene therapy, elivaldogene autotemcel (Skysona), and blood cancers. This inquiry raises important questions about the safety of gene therapies. While Bluebird Bio collaborates with larger organizations, its challenges underscore the scrutiny early-stage biotechs face in this evolving landscape. 🔍 FDA Investigation The FDA's investigation into Bluebird's Skysona centers on reports of hematologic malignancies among patients treated with the therapy. This gene therapy, approved for treating cerebral adrenoleukodystrophy, is now under intense review. The findings could have significant implications for both Bluebird and the broader gene therapy sector. 🧬 CAR-T Cell Therapy In a positive development, Obe-Cel, the sole FDA-approved CAR-T cell therapy capable of split dosing, is making strides. This innovative approach offers flexibility in administration, particularly for patients with significant tumor burden. Its approval is a testament to the potential of targeted therapies in oncology, highlighting the importance of continued investment in novel treatment modalities. 🤝 Start-Up Collaborations While larger firms often dominate headlines, early-stage biotechs are essential to the industry’s progress. Companies like Obe-Cel and Bluebird Bio are examples of start-ups tackling complex medical challenges. Their partnerships with established organizations are crucial for scaling innovations and navigating regulatory landscapes. As we continue to monitor these developments, it’s clear that both challenges and opportunities abound for small biotechs. Staying informed and connected is vital for driving growth and fostering innovation in our sector. Let's support these pioneers as they navigate the complexities of biotech R&D!

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Enveda is making waves in #drugdiscovery by blending #AI with nature’s chemistry, and its recent $130 million #SeriesC raise underscores the potential of this approach. With 10 candidates in the pipeline and a lead compound (ENV-294) already in phase 1 trials for #atopicdermatitis and inflammatory conditions, the company is on an exciting trajectory. This funding, supported by renowned investors like Kinnevik and FPV Ventures, positions #Enveda to push more #nature-inspired drug candidates into #clinicaltrials over the next few years. It’s motivating to see how cutting-edge technology is accelerating the journey from discovery to treatment. #Biotech #NatureInspiredMedicine

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