Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.The FDA's accelerated approval pathway, established in 1992 to expedite the approval of drugs for serious conditions, has been under scrutiny due to recent market withdrawals and failed confirmatory trials. Despite these setbacks, the program has successfully brought over 200 new drugs to market, with more than half ultimately receiving full FDA approval. The accelerated approval pathway has been particularly successful in the cancer space, but is now expanding to include treatments for neurological, rare, and genetic diseases.Recent withdrawals of drugs like Pfizer's Oxbryta for sickle cell disease and Biogen's Aduhelm for Alzheimer's have raised concerns about the risk-benefit proposition of the accelerated approval pathway. To improve the program, experts suggest implementing tighter completion timelines for confirmatory trials and better understanding which biomarkers should be used as surrogate endpoints. Overall, the accelerated approval pathway has been effective in bringing innovative treatments to patients quickly, but there is a need for greater scrutiny and oversight to ensure the safety and efficacy of these drugs. ## A new study published in Nature Communications highlights the potential of CRISPR gene editing technology to treat genetic diseases. Researchers successfully used CRISPR to correct a mutation in blood stem cells from patients with sickle cell disease, demonstrating the potential for this technology to provide a cure for genetic disorders. The study paves the way for future clinical trials using CRISPR to treat a wide range of genetic diseases, offering hope for patients with conditions that currently have limited treatment options.The study published in Nature Communications showcases the promising potential of CRISPR gene editing technology in treating genetic diseases. Researchers were able to correct a mutation in blood stem cells from patients with sickle cell disease, indicating that this technology could potentially offer a cure for genetic disorders. This breakthrough opens up possibilities for future clinical trials utilizing CRISPR to address a variety of genetic diseases, providing optimism for individuals with conditions that are currently difficult to treat. ## The World Health Organization (WHO) recently announced that it will establish a new global hub for pandemic and epidemic intelligence in Berlin, Germany. The hub is intended to improve data sharing and analysis to help countries better prepare for future health emergencies. It will work closely with existing WHO partners and networks to strengthen global surveillance and response efforts.The establishment of this hub comes in response to lessons learned from the COVID-19 pandemic, highlighting the need for enhanced global collaboration and information sharing to effectively respond to health crises. By centralizing intelligence gathering and analysis, the new hub aims to facilitate early detection and response to emerging threats, ultimately reducing the impact of future pandemics and epidemics.The World Health Organization (WHO) has revealed plans to create a new global hub for pandemic and epidemic intelligence in Berlin, Germany. This initiative is designed to enhance data sharing and analysis in order to assist countries in better preparing for potential health emergencies. The hub will collaborate closely with existing WHO partners and networks to bolster worldwide surveillance and response endeavors. The establishment of this hub follows insights gained from the COVID-19 pandemic, underscoring the necessity for heightened international cooperation and information exchange to effectively address health crises. Through consolidating intelligence collection and analysis, the new hub seeks to streamline early detection and response to emerging threats, ultimately mitigating the impact of fort

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. Robert F. Kennedy Jr., known for his anti-vaccine views, has been chosen by President Trump to lead the Department of Health and Human Services. Kennedy has expressed plans to overhaul the FDA and reduce the NIH's headcount, raising concerns about the future of healthcare regulation. In other news, Celgene shareholders are suing Bristol-Myers Squibb for allegedly avoiding contingent value right payments related to their acquisition deal, while Eyenovia's stock plummets after dropping their lead program due to lack of efficacy. Bluebird Bio is facing a cash shortage with only two quarters of funding left, prompting executives to seek financing to reach breakeven point by 2025. Overall, the pharmaceutical industry had a tumultuous third quarter, with companies like Gilead and Kite cutting employees and closing sites. The industry is in a state of uncertainty as it navigates through these challenges.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.Merck has committed up to $3 billion in a deal with Lanova Medicines focused on PD-1/VEGF. The deal comes as Merck faces competition from Bristol Myers Squibb in leukemia, leading Allogene to end phase I enrollment. Analysts view Merck's move as conservative, despite recent excitement around PD-1/VEGF. In other news, GSK is making a case for Blenrep in multiple myeloma, while Adaptimmune plans to submit a rolling BLA for its sarcoma cell therapy. PTC has received the first FDA approval for direct-to-brain gene therapy. Additionally, Marinus Pharmaceuticals has laid off 45% of its employees. Transitioning to other topics, we also cover Biogen's stock performance, a significant deal in the CDMO sector, and recent developments in the pharmaceutical industry.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.Amgen's obesity candidate, maritide, faced new safety concerns related to bone density, causing a drop in the company's stock. Biogen's shares have also plummeted due to issues such as a slow launch for their Alzheimer's medicine and a lackluster pipeline. Meanwhile, the biotech company Metsera raised significant funds for their long-acting GLP-1 drug despite competition in the weight loss space. Additionally, Kezar's autoimmune drug faced a second FDA clinical hold, and Syros experienced a significant drop in stock value due to a late-stage MDS failure. Novo's deal with Catalent is seen as a significant moment for the CDMO sector. These developments indicate ongoing challenges and opportunities in the biotech industry.Biogen's shares have dropped by 36% in 2024 due to factors such as a slow launch for Alzheimer's medicine Leqembi and a lackluster pipeline. The Novo-Catalent deal is seen as a defining moment for the CDMO industry, with concerns about consolidation limiting competition but also providing opportunities for smaller-scale CDMDs to find new partners. Other news includes Amgen facing bone density safety concerns for Maritide, AbbVie shares plummeting due to a failed schizophrenia asset, and Bayer's CEO admitting that their earnings are not looking good. In the biopharma job market, positions such as Executive Director of Biologics Analytical Research and Development at Merck & Co. and Vice President of Global Program Head - Ophthalmology at Regeneron Pharmaceuticals are available.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.AstraZeneca reported strong Q3 sales and raised its 2024 outlook, while avoiding questions about China and announcing plans to invest $2 billion in the U.S. market. Bayer's earnings were described as "not pretty" by CEO Bill Anderson, leading to a decline in stock value. Roche signed a potential $1.8 billion deal with Flare for cancer treatment, while 23andMe cut its workforce by 40% and ceased drug discovery work. Johns Hopkins University is partnering with a design team to prepare undergraduates for success in the biotech industry. Other news includes updates on companies like NKarta, Intellia, and Cassava, as well as FDA approvals and developments in the biopharma industry.

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.AbbVie's shares dropped by 12% after its schizophrenia drug, emraclidine, failed in a phase II trial. This failure has raised concerns about the company's long-term neuroscience strategy. In the Huntington's disease space, companies like Prilenia and Wave Life Sciences are working on potential disease-modifying treatments, offering hope for a community that has faced disappointment in recent years. Rapt Therapeutics has crashed after liver injury concerns with its drug, and AstraZeneca and Amgen claim a late-stage win in chronic rhinosinusitis. The FDA has lifted a clinical hold on Novavax's COVID/flu vaccine candidates, while GSK has left the trade group BIO. Overall, the pharmaceutical industry is facing challenges and uncertainties as it continues to develop new treatments for various diseases.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.Eisai's sales of its Alzheimer's disease therapy Leqembi have disappointed analysts, leading the company to lower its fiscal 2024 guidance. The underwhelming sales are attributed to barriers such as coverage, infusion centers, and time to diagnosis. In other news, Pfizer has made a $1 billion commitment in China to boost innovation and target the Chinese market, while AstraZeneca's China head has been taken into custody by Chinese authorities for reasons that remain unclear. There is also a dispute between Lilly and Novo over Catalent's GLP-1 work, as regulators review Novo Holdings' buyout of Catalent and the work the contract drug manufacturer may or may not be performing for Eli Lilly remains contentious.Additionally, a gene therapy webinar on successful preparation for scaling gene therapy processes is highlighted. Other news includes the impact of a Trump victory on the biopharma industry, cell therapy biotechs shifting focus to autoimmune expertise, and updates on Lilly's Q3 performance and more.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. Moderna exceeded expectations for COVID-19 vaccine sales in the third quarter, but sales of their respiratory syncytial virus vaccine fell short. Gilead also beat expectations in Q3 due to strong sales of their HIV drug. Sarepta has halted development of a next-gen DMD drug, vesleteplirsen, and reported strong sales of elevidys. Lilly cited wholesalers as the reason for slow sales of their GLP-1 products, while Novo reported no supply issues with their rival medicines. The gene therapy sector is highlighted in an on-demand webinar. Teva reported strong demand for generics in Q3 and expects higher revenue in 2024. Viracta Therapeutics has cut staff as part of resource reprioritization. Cell therapy biotechs are seeking expertise in autoimmune diseases. Thank you for listening to Pharma and Biotech daily.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world. Analysts are expressing mixed reactions to Donald Trump's election victory. There are potential positives for the biopharma industry, such as a more friendly FTC for M&A deals. However, concerns have been raised about what role Robert F. Kennedy, Jr. might play in healthcare under Trump's administration. Moving on to recent news, Catalent missed fiscal Q1 revenue expectations and there is growing opposition to Novo Holdings' acquisition of Catalent. Novo Nordisk also missed overall Q3 revenue expectations, but exceeded sales forecasts for the weight loss drug Wegovy. Gilead and Arcellx are highlighting the best-in-class potential for CAR T therapy in multiple myeloma. Overall, Trump's win poses more questions than answers for the biopharma industry, with uncertainties about the future regulatory environment and potential impacts on healthcare policies.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.Beam Therapeutics reported a patient death in a gene editing trial for sickle cell disease, overshadowing otherwise positive data. Beam-101 was found to be competitive with approved treatments, highlighting the need for less-toxic preconditioning treatments. Sana Biotechnology is planning workforce cuts to reprioritize focus on its type 1 diabetes program and extend its cash runway. Astrazeneca has received promising early data for candidates in its obesity pipeline. Vertex Pharmaceuticals exceeded its Q3 forecast and raised full-year revenue guidance. Sana Biotechnology is gearing up for potential approvals for cystic fibrosis and non-opioid therapy. The FDA delayed a decision on Merus' bispecific antibody and lawsuits were filed by the family of Henrietta Lacks seeking a share of profits from HeLa cells.The impact of the presidential election on biopharma is being closely monitored.