Torben Colberg MD MBA

#Rezdiffra (Resmetirom) for NASH Rezdiffra, developed by #Madrigal Pharmaceuticals, is a once-daily oral selective agonist of the thyroid hormone receptor-beta (THR-β). THR-β is responsible for regulating metabolic pathways in the liver and is frequently impaired in NASH13. Patients with #NASH have reduced levels of thyroid hormone activity in the liver with resultant impaired hepatic function. The accelerated approval of Rezdiffra was based on results from the #Phase 3 #MAESTRO- NASH trial, which was recently published in the #New England Journal of Medicine. MAESTRO-NASH is an ongoing pivotal, multicenter, randomized, double-blind, placebo-controlled trial that enrolled 1,759 patients with biopsy-confirmed NASH. Results showed 25.9% of patients in the #resmetirom 80 mg group and 29.9% of patients in the 100 mg group achieved MASH resolution with no worsening of fibrosis compared with 9.7% of those in the placebo group.

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🚀 #GENETHERAPY IN OPHTHALMOLOGY 😍 🕮 Back in November 2021, one of my articles found its place in the pages of The Ophthalmologist , where I delved into the realm of gene and genetic therapies, highlighting the innovative approaches of five companies pushing the boundaries of treatment possibilities. ; Spark Therapeutics, Inc., Biogen, GenSight Biologics, ProQR Therapeutics Adverum Biotechnologies. Since then some those therapies weren't approved and some are still being studied but the work hasn't been for nothing as their research shed light on the promising advancements in the field and their potential to transform the lives of patients with various eye conditions. Now, as new developments emerge, we continue to track the progress of these ground-breaking therapies. 💉Ocugen has received FDA approval for an amendment to their investigational new drug (IND) application, paving the way for the launch of a phase 3 clinical trial of OCU400. This gene therapy candidate, designed by Ocugen for treating retinitis pigmentosa (RP), marks a significant advancement in the field. According to Dr. Shankar Musunuri, PhD, MBA, Chairman, CEO & Co-Founder of Ocugen, OCU400’s entry into phase 3 represents a milestone as the first gene therapy program with a broad RP indication. He highlights the previous scarcity of treatment options for RP, with only one marketed product targeting a fraction of associated gene mutations. This development offers newfound hope for all RP patients awaiting viable treatment options. 👁Meanwhile, REGENXBIO Inc. has announced promising results from their phase 1/2a study of ABBV-RGX-314 in The Lancet. This gene therapy, developed in collaboration with AbbVie, demonstrates effectiveness as a one-time treatment for wet age-related macular degeneration (AMD). Utilising the NAV AAV8 vector, the therapy inhibits vascular endothelial growth factor (VEGF), a pivotal factor in the progression of wet AMD and other chronic retinal conditions. ABBV-RGX-314 is currently undergoing evaluation in two pivotal trials, ATMOSPHERE and ASCENT, aimed at supporting global regulatory submissions. This breakthrough holds great potential for revolutionizing the treatment landscape for wet AMD and related retinal diseases. If either of these potential treatments work and receive approval the benefit to patients would be lifechanging so we wish the very best of luck to both companies 🙏

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FDA approves nogapendekin alfa inbakicept-pmln for BCG-unresponsive non-muscle invasive bladder cancer NEJM Group American Society of Clinical Oncology (ASCO) UCLA Health Sam S. Chang, MD MBA Gene Kramolowsky Mark Gonzalgo Jeffrey Bassett https://lnkd.in/dDdbhN8R #BladderCancer #Cancer #FDA #FDAApproval #Headlines #NonMuscleInvasiveBladderCancer #OncoDaily #Oncology #TransurethralResection #DrugDevelopment

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There are many reasons for women not being able to prioritize their own health or prevention. We developed solutions to remove some of the key barriers. This article equips you with knowledge and the latest innovative solutions for cervical cancer screening. I hope more women can be in charge of their own health.

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After learning about David Fajgenbaum, MD, MBA, MSc’s journey with a rare form of Castleman disease, Flatiron engineers utilized #machinelearning to better leverage Flatiron data, unlocking previously unreachable insights for a rare patient population to drive improved treatments and understanding of the disease. Read our #LearnTeachGrow article to gain a behind the scenes look at how their work using #ML enabled them to scale the identification of specific patient cohorts and ultimately inform treatment opportunities. https://lnkd.in/e2aWaJ3g

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Active times in the #PrivateEquity space! 📈 🚑 Alcanza Clinical Research is expanding its footprint! They've acquired FDI Clinical Research in Puerto Rico, bringing more capabilities under their belt. 🌐 Read more: https://axios.link/44cDQAk 🔐 Apax Digital makes a strategic investment in IANS, bolstering their position in the infosec advisory market. 🖥️ Explore: https://iansresearch.com 🚚 From UK, Astatine Investment Partners acquires NRG Riverside, a leader in specialist waste collection vehicles. 🇬🇧 Visit: https://nrgriverside.com 🖨️ Gamut Capital Management sets to acquire DEX Imaging from Staples. Another strategic play in managed print services! Details: https://deximaging.com 🚑 Big news from Stockholm as KKR plans to acquire Immedica Pharma AB. Impilo steps up to reinvest, becoming an equal owner. 💊 Learn more: https://axios.link/4aCZzDX 🏢 Masonite gears to divest its architectural segment to Industrial Opportunity Partners, LLC. 🚪 Check out: https://masonite.com 🛳️ Redwood Capital Investments LLC in talks to acquire Marquette Transportation Company, LLC, a key player in marine towing services. 📊 More info: https://axios.link/3UvbFJw ✈️ The Stephens Group, LLC invests in VisionSafe Corporation - EVAS, enhancing safety with innovative cockpit smoke displacement systems. Read more: https://axios.link/4deF1mQ 🏦 Apollo Global Management, Inc. reshuffles, selling a stake in ATLAS SP Partners to MassMutual. A strategic shift post acquisition from Credit Suisse. 📉 Discover: https://axios.link/3JvJxj8 📊 CoStar Group announces a massive deal to acquire Matterport for $2.1 billion, expanding its tech portfolio in spatial data. 🌍 Details: https://axios.link/49S2yHx 🚑 CVC Capital Partners contemplates the future of its stake in Recordati, a leading Italian drugmaker. 🇮🇹 More info: https://axios.link/4aPpMPH 💳 Thunes acquires Tilia, a next-gen payment platform for virtual worlds, after significant funding rounds. 🎮 Explore: https://axios.link/3Wb3hA7 #PrivateEquity #ValueCreation #TechInvestment #HealthcareInvestment #VentureCapital #Innovation #DigitalTransformation #InfoSec #MergersAndAcquisitions #StrategicInvestment #FinancialServices #CorporateGrowth #InvestmentNews #BusinessDevelopment

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𝗚𝗮𝘀𝘁𝗿𝗼𝗲𝗻𝘁𝗲𝗿𝗼𝗹𝗼𝗴𝘆 𝗮𝗻𝗱 𝗔𝗦𝗖𝘀: 5️⃣ 𝐌𝐎𝐑𝐄 𝘁𝗵𝗶𝗻𝗴𝘀 𝘆𝗼𝘂 𝘀𝗵𝗼𝘂𝗹𝗱 𝗸𝗻𝗼𝘄 1️⃣ In 2021 the U.S. Preventive Services Task Force lowered the recommended age for #colonoscopy screening from 50 to 45, creating 19 Million new patients. 2️⃣ The shift has led to significantly higher demand, and this has meant a huge increase in the number of procedures in outpatient settings such as #ASCs. 3️⃣ While the additional business to ASCs is advantageous, it could also exacerbate some struggles, such as the anticipated shortage of 1,630 #gastroenterologists in this next year, making it difficult for patients to get in for these procedures. 4️⃣ Another challenge is that the costs of a #colonoscopy to patients and #reimbursement rates for #physicians have not increased enough to battle inflation and rising procedural costs. 5️⃣ While new alternatives to in-office colonoscopies, like #GIcancer blood tests, are being developed, the American Gastroenterological Association (AGA) has warned that these tests are not yet ready to fully substitute a colonoscopy. Check out my original article: "𝗚𝗮𝘀𝘁𝗿𝗼𝗲𝗻𝘁𝗲𝗿𝗼𝗹𝗼𝗴𝘆 𝗮𝗻𝗱 𝗔𝗦𝗖𝘀: 5️⃣ 𝘁𝗵𝗶𝗻𝗴𝘀 𝘆𝗼𝘂 𝘀𝗵𝗼𝘂𝗹𝗱 𝗸𝗻𝗼𝘄" here ▶ https://lnkd.in/eB2kdrTQ Will you be attending #DDW in DC next month? Let's connect! ☕ #gi #gastroenterology #aga #gastrointestinal #healthcare #healthtech #medtech #medicaldevice #executivesearch #recruitment #ddw #ddw2024 #endoscopy #crc #colorectalcancer #cancerscreening

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Results of Tropion lung study

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Linda Rogers, MD, discusses a study regarding the use of an investigational oral Bruton's tyrosine kinase inhibitor as treatment for patients with poorly controlled asthma. Linda Rogers #AsthmaTreatment #ClinicalResearch #MedicalInnovation #Pulmonology #AsthmaManagement #BrutonsTyrosineKinase https://lnkd.in/eyKYqm8R

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FDA said that “the use of human- and animal-derived materials to manufacture CGT products and TEMPs raises several key issues to consider, including transmission of adventitious agents, material lot-to-lot consistency, and material identity, as well as general material qualification considerations.” For INDs, sponsors must provide FDA with a list of all materials used to manufacture products and a description of the quality and grade of these materials; these lists should be in a tabular format. Submissions should also adhere to the Common Technical Document (CTD)’s section 3.2.S.2.3 on control of materials and section 3.2.P.4 on control of excipients.

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#ADC #antibodydrugconjugate New Phase 3 data evaluating sacituzumab tirumotecan (sac-TMT), an investigational TROP2-directed antibody-drug conjugate, in previously treated locally recurrent or metastatic triple-negative breast cancer First presentation of Phase 2b three-year follow-up data evaluating mRNA-4157 (V940), an investigational individualized neoantigen therapy, in combination with KEYTRUDA® (pembrolizumab) in patients with high-risk stage III/IV melanoma following complete resection

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In this episode of The Syneos Health Podcast, our experts explore the evolution of genetic medicines, from the early days of clinical trials fraught with challenges to the recent surge in approvals and the promise of curative potential. Listen to the full episode: https://lnkd.in/esF9D_tE #SyneosHealth #WorkHereMattersEverywhere #SyneosHealthLife #ClinicalCareers

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Some Key Takeaways ASCO Tips - Aakash Desai, MD, MPH American Society of Clinical Oncology (ASCO) O'Neal Comprehensive Cancer Center at UAB #ASCO #ASCO24 #Cancer #OncoDaily #Oncology #ASCOMeetingApp

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🏥💰 Big News in Healthcare Pricing Transparency Attention #biotech and #pharma professionals! The Biden administration has made a surprising move in the ongoing battle for hospital price transparency. Key points: • No changes to current hospital price transparency regulations • Patient advocacy groups' pleas for stricter enforcement ignored • Concerns over hospitals' compliance and pricing accuracy remain unaddressed What this means for our industry: 1. Continued opacity in healthcare pricing 2. Potential impacts on drug pricing discussions and negotiations 3. Opportunities for innovative solutions in price transparency As leaders in biotech and pharma, we must consider: • How can we contribute to greater transparency in healthcare? • What role can our industry play in empowering patients with pricing information? • How might this decision affect our partnerships with hospitals and insurers? Let's discuss: What are your thoughts on this development? How do you see it impacting our sector? #HealthcareTransparency #Biotech #Pharma #HealthcarePolicy #PatientAdvocacy

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The number of biopharma M&A deals more than doubled in the first quarter of 2024 compared to the same period a year ago, exemplifying a top exit route for pre-commercial companies vying for validation and attention. 13 biopharma M&A deals in the first quarter of 2024 compared to six in the first quarter of 2023. 47 deals in the last twelve months through March 2024, compared to 42 through March 2023. #mergerandacquisition #investing #CEO #biopharma #wheretoinvest #druginnovation #artificialinyelligence

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