Luke Horrigan’s Post

🔎 Weekly Pharma Digest (Oct 5-11, 2024) 🎯 WINS REGULATORY 💼 🛆 Vertex Pharmaceuticals is on the brink of a significant milestone as it anticipates the FDA's decision on its innovative non-opioid pain medication, which could transform pain management practices. 🛆 Pfizer's Vyndamax, approved for treating ATTR-CM, successfully passed the reimbursement review panel after multiple unsuccessful attempts, potentially allowing patients to access the drug at a 10% co-payment rate 🛆 Roche's inavolisib, marketed as Itovebi, received FDA approval for first-line treatment of advanced breast cancer in combination with Ibrance and Faslodex, targeting PIK3CA-mutated, HR-positive, HER2-negative patients 🛆 FDA Advisory Committee Recommends Approval of Stealth BioTherapeutics' Elamipretide, to treat the ultra-rare and deadly condition Barth syndrome. CLINICAL 🔬 🛆 EU approval is being sought for D-VRd in newly diagnosed multiple myeloma, based on Phase 3 CEPHEUS trial results. D-VRd showed higher MRD-negativity and complete response rates compared to VRd. Primary endpoint was MRD negativity, while secondary endpoints were progression-free, and overall survival COMMERCIAL 💰 🛆 Sanofi Explores Strategic Sale of Opella Stake to Focus on Innovation 🛆 Mercy Places First Commercial Eversense 365: The World’s First and Only 365-Day Continuous Glucose Monitor 🛆 Immatics to Raise $150M in Public Stock Offering for R&D and Commercialization 🛑 CHALLENGES / SETBACKS 🛆 Gritstone bio Files for Bankruptcy to Preserve Clinical Research Amid Financial Struggles 🛆 Activist Investor Starboard Accuses Pfizer of Coercion and Unethical Behavior 🛆 Gilead Sciences Terminates MASH Drug Development Deal with Yuhan, Returning Rights 🛆 Regeneron Faces Setbacks with FDA Rejections for Bispecific Treatments in Oncology 🛆 Sanofi's Multiple Sclerosis Ph2 Trial Failure: A Major Setback in Denali Partnership ---- 💡 What are your thoughts on these developments? Don't forget to sign up for weekly updates within the pharma and biotech industry. #pharma #pharmaceuticals #biotech #news #competitiveintelligence #atacanagroup

Clinical Trial Supplies Market Worth $6.3 Billion Request Free Sample Report: https://lnkd.in/dbxzWTiE Clinical Trial Supplies Market by Services (Manufacturing, Packaging, Logistics), Phases, Type (Small Molecules, Biologics), Therapeutic Areas (Oncology, CVD, Infectious, Immunology), End User (Pharma, Biotech, CROs), & Region - Global Forecast to 2028 𝐓𝐡𝐞 𝐜𝐨𝐦𝐩𝐚𝐧𝐢𝐞𝐬 𝐟𝐞𝐚𝐭𝐮𝐫𝐞𝐝 𝐢𝐧 𝐭𝐡𝐢𝐬 𝐫𝐞𝐩𝐨𝐫𝐭 𝐢𝐧𝐜𝐥𝐮𝐝𝐞: Thermo Fisher Scientific, Catalent Pharma Solutions, Eurofins Scientific AG (Switzerland), Piramal Pharma Solutions, PRA Health Sciences, Marken (A Subsidiary of UPS) (US), Parexel, Biocair (UK), AlmacGroup (UK), Sharp Services, PCI Pharma Services, Nuvisan (Germany), Lonza Group (Switzerland) Other Companies that are Operating in this Market Globally: CTC Clinical Trial Consultants AB Clinical Research Malaysia Parexel Endpoint Clinical Fisher Clinical Services LMK Clinical Research Consulting DaVita Clinical Research Patheon Cytel WEP Clinical The Medical Affairs Company (TMAC) Q² Solutions DAVA Oncology Ephicacy Accelerated Enrollment Solutions Suvoda CBCC Global Research Katalyst HealthCares & Life Sciences Sharp Services Imperial Clinical Research Services Frontage Laboratories, Inc KlinEra Global Services Myonex #ClinicalTrialSupplies #ClinicalTrials #PharmaLogistics #DrugDevelopment #ClinicalResearch

FDA JUST RELEASED ‼️ Early Alzheimer’s Disease: Developing Drugs for Treatment Guidance for Industry The purpose of this guidance is to assist sponsors in the clinical development of #drugs for the treatment of the stages of sporadic Alzheimer’s disease (#AD) that occur before the onset of overt #dementia (i.e., Stages 1 through 3; discussed in section III). These stages are collectively referred to as “early AD” in this guidance; however, it is recognized that AD occurs on a continuum and patients in the last stage of early AD (i.e., late Stage 3) and patients with AD in the earliest stages of overt dementia (i.e., early Stage 4) may not differ significantly in clinical presentation. This guidance is intended to serve as a focus for continued discussions among representatives of the Office of Neuroscience in the Center for Drug Evaluation and Research or the Office of Therapeutic Products in the Center for Biologics Evaluation and Research, as appropriate, pharmaceutical sponsors, the scientific community, and the public about the development of drugs for the treatment of early AD. This guidance revises the draft guidance for industry Early #Alzheimer’s Disease: Developing Drugs for Treatment (February 2018). This revision, when finalized, will represent FDA’s current thinking regarding the selection of subjects with early AD for enrollment in clinical trials and the selection of endpoints for clinical trials in this population. If you like this post follow me on LinkedIn

Recent studies reveal a stark reality: only 1 in 10 drugs reach FDA approval, with even lower rates in vital areas like oncology. Failed trials cost an estimated $45B a year. QuantHealth’s drug simulation technology is the more efficient pathway to drug development, creating results that accelerate drug trial timelines and eliminate errors and discrepancies in drug development. By carefully improving how patients are categorized and how drugs are developed, we not only mitigate trial risks but also enhance success rates significantly. Additionally, the utilization of big data and diversified research models offers promising avenues for innovation at reduced costs. Read more about why 90% of clinical drug development fails and what can be done to bring FDA-approved drugs to the market quicker and with less cost in this The National Institutes of Health study here: https://lnkd.in/es6AKpZu   You can also learn more about what QuantHealth is doing to address these issues by visiting our LinkedIn page here! #DrugDevelopment #Innovation

Estimated Research and Development Investment Needed to Bring a New Medicine to Market, 2009-2018 Costs of Failed Trials Accurate information on costs of failures, ie, research and development outlays on candidates being developed by companies but not ultimately approved, is essential to estimating the costs of drug development. We accounted for failures using data on aggregate clinical trial success rates from a recent study by Wong et al (Table 1).18 Wong et al reported that the percentages of FDA approvals were 13.8% for therapeutic agents entering phase 1, 21.0% for those entering phase 2, and 59.0% for those entering phase 3.18 Subgroup Analyses by Therapeutic Area Median estimates by therapeutic area (for areas with ≥5 drugs), adjusted using area-specific rates and capitalized at 10.5% per year, ranged from $765.9 million (95% CI, $323.0 million-$1473.5 million) for nervous system agents to $2771.6 million (95% CI, $2051.8 million-$5366.2 million) for antineoplastic and immunomodulating agents. The corresponding mean estimates ranged from $1076.9 million (95% CI, $508.7 million-$1847.1 million) for nervous system agents to $4461.2 million (95% CI, $3114.0 million-$6001.3 million) for antineoplastic and immunomodulating agents (Table 4). The article: https://lnkd.in/gH7eTtVK

Want to save time and money on your clinical trials? Our latest blog post delves into how pharma and biotech companies can leverage PRS to: ⚪ Select the right participants for clinical trials ⚪ Reduce the number of participants ⚪ Accelerate clinical trials ⚪ Adjust for unmeasured confounders ⚪ Rescue clinical trials ⚪ Repurpose drugs You can read the full blog here: https://lnkd.in/dCy_uEZ5

In a new publication, my colleagues Gaurav, Harriet, Brandon, Valentina, Amy, and Jacob outline the increasing challenges in R&D productivity and present the argument that clinical trial patients (rather than scientific innovation or R&D funding) are now the biggest challenge for pharmaceutical productivity. The article identifies factors contributing to delays in trial recruitment including increasing demand for patients, rising burden on sites and patients, and challenging physician incentives. Finally, we present a set of potential actions that sponsors can take to address these challenges and drive development accelerations. 

Citeline’s influential clinical development success rate analyses have been foundational for supporting investment decisions in biopharma R&D. Our landmark paper in 2014 established the commonly cited 10% benchmark. However, this has steadily fallen over the last decade. This drop is both a concern and inevitable as competition intensifies and the barriers to market entry become higher. Click to read about why clinical development success rates are falling on the Norstella blog: https://ow.ly/4fh250RIMpu. #ClinicalDevelopment #DrugDevelopment #Biopharma #BiopharmaceuticalCompanies #BiopharmaTrends

How much does new drug development cost and here's why it's so expensive 1. In 2022, the estimated average cost of R&D expenses for a new molecular entity (NMEs) is $2,284 million. 2. The most expensive R&D is found in the anticancer drug category. 3. Compared to 2021, the R&D cost has increased from $1,986 million to $2,284 million in 2022, reflecting a 15 % increase. 4. It's not just the R&D cost that is rising, the cost of clinical trials and drug development are also increase due to clinical development represents 50-58% of the R&D expenses for each new medicine. 5. Factors of R&D cost are - Therapeutic area - Firm size - Funding source - Direct cash expenses - Increasing attrition rates - Duration of clinical trials - The future value at the time of launch - Technological advances. To make clinical trials more cost-effective and prevent excessive expenses, several strategies can be considered. 1. Enhancements in preclinical testing in some therapeutic areas have facilitated early discontinuation when warranted, preventing unnecessary clinical trial costs. 2. Advancements in technology have significantly expanded research possibilities in specific disease areas, increasing the number of possible compounds that can be tested. Reference Deloitte. (2023). Deloitte’s 13th Annual Pharmaceutical Innovation Report: Pharma R&D Return on Investment Falls in Post-Pandemic Market. Rertieve from https://lnkd.in/d8fVP-za Schlander, M., Villafuerte, H, K., Cheng, Y, C., Ferrandiz, M, J., Baumann, M. (2021). How Much Does It Cost to Research and Develop a New Drug? A Systematic Review and Assessment. PharmacoEconomics. Rertieve from https://lnkd.in/gTxWAF-k Simoens, S., Huys, I. (2021). R&D Costs of New Medicines: A Landscape Analysis. Rertieve from https://lnkd.in/gUiqBEeM

Clene Nanomedicine, Inc., a biopharmaceutical company focused on improving mitochondrial health, has announced an upcoming meeting with the U.S. Food and Drug Administration (FDA) leadership. The in-person meeting, scheduled before the end of November, will focus on the company's lead candidate CNM-Au8® and its biomarker, clinical, and survival data for amyotrophic lateral sclerosis (ALS). 'CNM-Au8 has been shown to restore and protect neurological function, offering hope for patients with neurodegenerative conditions,' the company stated. Clene is seeking an accelerated approval regulatory pathway for CNM-Au8® as a potential treatment for ALS. The meeting will be attended by top FDA officials, including the Director of the Office on New Drugs and the Director of the Office of Biostatistics, as well as key opinion leaders in ALS, biostatistics, and biomarkers. The FDA agreed to reevaluate Clene's submission, signaling a positive step towards potential approval. #ALSresearch #neurodegenerative #fdaregulation #biotechnology @cleneinc