Pharma and BioTech Daily

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.Roche has made a significant move in the pharmaceutical industry by acquiring 89bio for $3.5 billion, with a contingent value right of $6 per share. The acquisition includes pegozafermin, an analog in late-stage development for metabolic dysfunction-associated steatohepatitis. Meanwhile, former CDC director Susan Monarez spoke to the Senate about her firing, alleging that Health Secretary Robert F. Kennedy Jr. accused CDC employees of "killing children." In other news, Roivant and Pfizer's therapy shows promise in a rare inflammatory condition, BMS and Novo Nordisk have announced layoffs in New Jersey, and Sino Biological has developed a high-throughput platform for AI-driven antibody discovery. Additionally, Lexicon has backed phase III decision for a non-opioid pain medication, and Rosnilimab shows promise as a potential next-generation treatment for rheumatoid arthritis. The pharmaceutical industry is navigating uncertainty during turbulent times, with companies like Arsenal and X4 laying off staff to cut costs.The FDA is cracking down on pharmaceutical advertisements in a new regulatory initiative, targeting loopholes in direct-to-consumer ads. Experts believe the FDA is focusing on the wrong issues and should instead address the lack of regulations for compounder drug ads. Akeso, a biotech company in China, has seen success under CEO Michelle Xia's leadership. Other news in the pharmaceutical industry includes the rise of predictive care powered by consumers and AI, ongoing prescriptions of Sarepta's DMD gene therapy despite safety concerns, and companies like Sanofi pulling back from the UK market. Pharma companies are facing challenges in navigating uncertain times amidst layoffs and regulatory crackdowns. Stay tuned for more updates on the latest developments in the industry.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.Eli Lilly's oral weight loss pill, orfoglipron, has faced challenges in meeting expectations, possibly due to an over-representation of males and Hispanic patients in the phase III study. The company is now presenting detailed late-stage data to paint a competitive profile for the drug. Meanwhile, the FDA has targeted Lilly and Novo Nordisk for downplaying risks of their GLP-1 weight loss drugs in advertising, particularly during a prime time special with Oprah Winfrey. Lilly is also investing in a $5 billion manufacturing plant in Virginia, part of a larger $27 billion U.S. investment plan.In other news, GSK is committing $30 billion to boost R&D and manufacturing operations in the U.S., showing a strong commitment to innovation. Additionally, advancements in AI-driven antibody discovery technology by Sino Biological are revolutionizing the field of biotechnology. The pharmaceutical industry is facing tightening oversight and regulatory changes, as well as exploring predictive care powered by consumers and AI technology.This dynamic landscape highlights the importance of staying informed and adaptable in the ever-evolving world of Pharma and Biotech.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. Novo's amylin analog drug, cagrilintide, has shown a significant 11.8% weight loss in a phase III trial, with improved safety data to address past tolerability issues. The FDA is set to make a decision on the drug, with significant stakes for the company and the Barth Syndrome community. Additionally, Sarepta's DMD gene therapy is still being prescribed by doctors despite safety concerns, and new members have been appointed to the ACIP panel. Stealth Biotherapeutics is awaiting a verdict on their potential treatment for Barth Syndrome, while Sino Biological's high-throughput platform for AI-driven antibody discovery offers promise for advancing therapeutic candidates. Novo and Lilly are competing for market leadership in obesity treatment, while Sanofi and other pharma companies are pulling back from investments in the UK. The pharmaceutical industry is navigating uncertainty during turbulent times.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.Novartis and Monte Rosa have entered into their second molecular glue deal worth up to $5.7 billion, with Novartis putting $120 million upfront for more of the biotech's AI-discovered degraders. The myasthenia gravis market, once sparse, is now flourishing with new treatments approved and promising late-stage trial results from companies like Argenx and Regeneron. In other news, AstraZeneca has suspended its $270 million commitment in the UK, the FDA has flip-flopped on scrapping advisory committee meetings, and Sino Biological has developed a high-throughput platform for AI-driven antibody discovery. The myasthenia gravis space is heating up with targeted therapies, with several companies releasing promising late-stage trial results. Biogen is developing a pipeline for lupus, with investors showing interest in their programs. The FDA has several actions scheduled for September, including Merck's proposed subcutaneous formulation of Keytruda. Eli Lilly's obesity pill, Orforglipron, is in focus at the European Association for the Study of Diabetes meeting. In the cancer news, Merck's Keytruda challenger faces consistency problems, while other companies like Daiichi Sankyo and Biontech report positive data. Capsida reports a patient death in a gene therapy trial, while Alkermes shows promise in narcolepsy treatment. FDA is looking to streamline the development of non-opioid painkillers. Various webinars and events are upcoming in the pharma industry. Job opportunities are available at companies like Moderna, Abbvie, and Regeneron. Overall, the biopharma industry is seeing advancements and progress in various therapeutic areas.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.President Trump is considering exempting certain "non-patented" pharmaceuticals from tariffs, although specific guidelines have not yet been released. The Intercept has withdrawn a liver drug from the US market, and Ocaliva, approved for primary biliary cholangitis in 2016, did not receive full approval last year. Merck is cutting 125 employees in the UK as it ends R&D work. AbbVie has extended exclusivity for its drug Rinvoq until 2037 with a generics settlement. Novo's headcount has increased by 81% in five years as revenue climbed. The FDA is reportedly preparing an order restricting Chinese drug licensing deals. Capsida has reported a patient death in a gene therapy trial. Novartis' BD team is busy with bolt-on deal promises for 2025. That's all for today's news in the Pharma and Biotech world. Thank you for listening!

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.## Capsida has reported a patient death in a gene therapy trial for epilepsy, but the exact cause of death has not been disclosed. The patient had received the gene therapy cap-002. ## The FDA is looking to streamline the development of non-opioid painkillers, while Avidity has seen functional improvements in their DMD therapy. ## The White House is reportedly considering restrictions on Chinese licensing deals in the pharmaceutical industry. ## Lilly is seeking individuals willing to challenge the status quo of medicine. ## Merck has abandoned a $1.3 billion R&D center in London, resulting in layoffs. Novo has cut 9,000 employees, and New York City's life sciences scene is growing. Thank you for tuning in to today's episode of Pharma and Biotech daily. Stay informed and have a great day!

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.Novo Nordisk has cut 9,000 jobs worldwide and reduced its 2025 profit growth guidance. The new CEO is fulfilling a cost reallocation promise, leading to a decrease in operating profit growth projection from 10% to 16% down to 4% to 10%. The company's restructuring efforts are aimed at increasing efficiency and reducing costs. In other news, the ousted CDC director Susan Monarez will testify in front of the Senate, and a recent report from the Maha commission emphasizes chronic diseases and potential vaccine reform.Biotechs are using contingent value rights (CVRs) to close the gap between buyer and seller expectations in transactions amid a down market. Novartis is actively pursuing acquisitions, particularly in the cardiovascular space, while Novo Nordisk is restructuring and cutting costs, impacting profit growth projections. Amgen is looking beyond the scrutiny of one obesity asset to focus on a fuller portfolio. Trilink Biotechnologies offers process development services for nucleic acid therapeutic development. In other news, LB Neuroscience files for a $228 million IPO, Novartis acquires Tourmaline for $1.4 billion, and Hengrui licenses a heart disease drug to a US startup for up to $1 billion. The FDA debuts a consumer 'green list' instead of addressing the GLP-1 compounding fight. Thank you for tuning in to Pharma and Biotech daily.

Good morning from Pharma and Biotech Daily, the podcast that gives you only what's important to hear in the Pharma and Biotech world. Novartis recently acquired Tourmaline for $1.4 billion, continuing its investment in cardiovascular treatments. The company has also made other partnerships in the cardiovascular space this year. Biogen is preparing to release new data on its lupus treatments, with analysts praising the company's pipeline. Novartis has been active in mergers and acquisitions, putting over $17 billion into deals this year. The biotech industry is also seeing some activity, with LB filing for a $228 million IPO to support its phase III plans. Overall, companies like Novartis and Biogen are focusing on expanding their portfolios and developing innovative 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.A new data release shows that Summit's Keytruda competitor, Ivonescimab, had better overall survival rates in Asian patients compared to those in North America and Europe. Despite this, the drug narrowly missed the statistical significance threshold required by the FDA for approval in Western countries. Meanwhile, Rapport Therapeutics saw a 180% increase in its stock value after positive mid-stage data for its epilepsy pill, and the FDA opted for a consumer "green list" instead of addressing compounded versions of obesity drugs. The FDA's leadership turnover has led to confusion and uncertainty for small and medium biopharma companies. Lilly is seeking individuals who are willing to challenge the status quo in medicine.FDA expert panels are being criticized for lack of balance, with some meetings being one-sided. RFK Jr. faced bipartisan criticism for his anti-vaccine views during a Senate hearing. Meanwhile, Servier committed up to $530 million in an eye cancer deal and Gilead partnered with the US State Department to distribute a drug to low-income countries. The FDA released a new rare disease approval framework and dropped heavily redacted rejection letters. Amidst uncertainty in FDA leadership, small and medium biopharma companies are left without clear guidance for their therapies. Opinion pieces highlight the potential of small molecules in gene therapy for Duchenne muscular dystrophy and criticize the lack of balance in FDA expert panels. The newsletter also includes upcoming events, job listings, and a request for feedback on coverage topics.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. During a Senate hearing, Robert F. Kennedy Jr. faced criticism for spreading anti-vaccine views and breaking promises regarding vaccines. The FDA released rejection letters for companies like Lykos Therapeutics, Stealth Biotherapeutics, and Regeneron. Ousted CDC director Susan Monarez accused Kennedy of firing her for not supporting Covid-19 recommendations from an advisory panel with "antivaccine rhetoric." Hengrui Pharmaceuticals signed lucrative deals with Merck and GSK, while the FDA promised to release future Complete Response Letters promptly. In other news, Sanofi's anti-OX40 blocker failed in a Phase III study, Gilead partnered with the US State Department for low-income countries, and AC Immune announced workforce cuts. Kennedy was accused of lying during the hearing, and the FDA released a new rare disease approval framework.

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. faced the Senate Finance Committee in a tense hearing amidst calls for the removal of the health secretary. The committee questioned Kennedy on his recent actions, including the firing of CDC director Susan Monarez. Meanwhile, the FDA's new rare disease pathway received mixed reactions, with some questioning its tangible impacts. AC Immune downsized and refocused its pipeline, while Kennedy proposed adding seven new vaccine advisors to the ACIP. Other news included successful RNA editing in an AATD study, demands for YouTube to remove anti-vaccine videos, and a new framework for rare disease approvals by the FDA. Thank you for tuning in to today's episode of Pharma and Biotech daily.

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.Novartis has increased its commitment to its partnership with Argo BioPharma by an additional $5.2 billion, focusing on RNAi agreements targeting cardiovascular diseases. This highlights the ongoing advancements and challenges in the biopharmaceutical industry. Biotechs are turning to special purpose acquisition companies (SPACs) as a way to go public amid the IPO freeze. Gene therapy, with its potential to cure deadly diseases, is still facing challenges in terms of insurance coverage in the U.S. The industry is seeing a shift with some of the biggest biotech SPACs from the 2021 bubble no longer on the market. Meanwhile, Cytokinetics' cardiac myosin inhibitor, aficamten, has shown promising results in a phase III study for patients with obstructive hypertrophic cardiomyopathy. RFK Jr. has announced plans to reorganize chronic disease programs in the US to address high COVID-19 death rates. Companies like Novartis and Arrowhead are making significant commitments to various programs, while Trump's efforts to shore up the pharma supply chain with U.S. API are being questioned. Novartis continues its cutting spree with layoffs in New Jersey.These developments shed light on the evolving landscape of the biopharmaceutical industry.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. Novo Nordisk's drug Wegovy has successfully reduced cardiovascular risk by 57% compared to tirzepatide. Eli Lilly has terminated two mid-stage trials for a second obesity asset, while Vertex Pharmaceuticals and Enlaza Therapeutics have formed a potential $2 billion partnership to develop drugs for autoimmune diseases. Biogen and Eisai have received FDA approval for the subcutaneous maintenance formulation of Leqvembi. Arrowhead Pharmaceuticals has secured a commitment of up to $2 billion from Novartis for an siRNA Parkinson's program. The FDA's new radiopharma guidance is expected to accelerate the space, and Teva has launched the first generic version of Novo Nordisk's obesity drug Saxenda. The pharmaceutical industry is navigating uncertainty during turbulent times, with companies focusing on innovation and new partnerships to drive progress.The FDA has issued new radiopharma guidance, which former FDA Commissioner Stephen Hahn believes is crucial for cancer therapy. Despite recent investments in radiopharmaceutical therapeutics by big pharma, the FDA's approval of updated COVID-19 vaccines with restrictions contradicts the medical freedom promised by health secretary Robert F. Kennedy Jr. Rare diseases secured four FDA firsts in August, including a win for Novo Nordisk's glp-1 drug WeGovy. Investment in new ALS therapies signals progress after setbacks, with new biotechs and collaborative initiatives showing promise at Bio2025. Other news includes the closure of Appia, Senate summons of Kennedy, updates on COVID-19 vaccines, and Lilly's obesity pill heading to the FDA. 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 and Biotech world. ##FDA approves new drug for rare genetic disorderThe FDA has recently approved a new drug for the treatment of a rare genetic disorder. This drug has shown promising results in clinical trials and is expected to provide much-needed relief for patients suffering from this condition. ##Major pharmaceutical company announces mergerA major pharmaceutical company has announced plans to merge with another company in the industry. This merger is expected to create a powerhouse in the pharmaceutical world, with the potential for significant growth and innovation. ##Biotech startup secures funding for groundbreaking researchA biotech startup has recently secured a substantial amount of funding to support their groundbreaking research in a new area of biotechnology. This funding will allow the company to further develop their technology and bring it to market in the near future. ##FDA issues warning about potential side effects of popular medicationThe FDA has issued a warning about potential side effects of a popular medication that is commonly used to treat a variety of conditions. Patients are advised to speak with their healthcare provider if they have any concerns about taking this medication. ##Pharma company announces breakthrough in cancer treatmentA pharmaceutical company has announced a major breakthrough in the treatment of cancer. Their new therapy has shown promising results in clinical trials and has the potential to significantly improve outcomes for cancer patients. ##Biotech firm partners with university on new drug developmentA biotech firm has partnered with a university on a new drug development project. This collaboration brings together the expertise of both organizations to advance research and bring new treatments to market. ##FDA approves expanded use of existing medicationThe FDA has approved the expanded use of an existing medication for the treatment of a different condition. This decision is based on new research showing the effectiveness of this medication in treating this additional condition.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. Health Secretary Robert F. Kennedy Jr. is set to testify before the Senate Finance Committee on September 4. This comes after the removal of CDC Director Susan Monarez and the appointment of HHS Deputy Secretary Jim O'Neill as her interim replacement. Kennedy, along with FDA Commissioner Marty Makary and CBER Director Vinay Prasad, had previously been vocal against vaccine mandates during the COVID-19 pandemic, citing concerns about limiting medical choice. However, the FDA, under their leadership, has now approved updated COVID-19 vaccines with restrictions that go against this stance.RFK's vaccine restrictions are being viewed as restricting promised medical freedom. The FDA recently gave the green light to updated COVID-19 shots from Pfizer, Moderna, and Novavax but with certain limitations. This news comes as Teva introduces the first GLP-1 generic for obesity to the market, and J&J discontinues its rheumatoid arthritis program after disappointing mid-stage data. Kennedy's upcoming testimony highlights the ongoing turmoil within CDC leadership and raises questions about the conflicting messages surrounding vaccine mandates and medical freedom.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world. CDC Director Monarez was fired after less than a month due to internal unrest over new COVID-19 vaccine policies. Novo Nordisk is investing over $500 million in RNA obesity treatments. The FDA has approved updated COVID-19 shots from Pfizer, Moderna, and Novavax with restrictions, causing concern among some groups. Prothena's Alzheimer's drug, PRX012, has shown high rates of brain swelling in Phase I testing. Novo Nordisk is collaborating with other companies on cardiometabolic treatments. The pharma industry is facing challenges and changes, with layoffs and restructurings occurring at various companies. Massachusetts has seen a decline in R&D and biomanufacturing jobs in 2024. Various webinars and events are being held to discuss topics such as pharma manufacturing and data accessibility. Job opportunities in the pharmaceutical industry include positions in business analysis, quality engineering, and project engineering.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.Regeneron's experimental drug for myasthenia gravis, cemdisiran, has shown promising results in a Phase III trial and the company is aiming to file for FDA approval by 2026. Pharmaceutical spending in China has reached record levels, with more than $48 billion spent in the first half of the year. Former FDA Commissioner Scott Gottlieb has raised concerns about the US losing its edge in biopharma innovation to China. Additionally, a drug program for a rare neurodegenerative disorder has been discontinued by Amylyx, while pharma companies are increasingly focusing on scientific research and partnerships. Lilly's obesity pill is heading to the FDA for approval, AbbVie is investing in psychedelics, and HHS has announced more changes in the industry. The pharmaceutical landscape is rapidly evolving with new developments and challenges arising.Pharma CEOs are facing increasing pressure amid political turmoil and public distrust over drug pricing. The industry spent over $48 billion in China in the first half of the year, with expectations of increased deal volume in the future. Legal experts are working to determine ownership rights of AI creations in biotech. Obesity treatments are being compared for effectiveness, while biotech companies are making strides in AI-driven manufacturing. Job opportunities in gene therapy and biostatistics 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.Lilly's new oral obesity drug is progressing towards FDA approval in 2025 after positive results from the second phase III trial for Orfoglipron. Analysts are optimistic about the potential for a "double-digit billion dollar opportunity" due to a manufacturing advantage.Arginex is looking to expand the label for Vyvgart in myasthenia gravis, while drugmakers are seeing success in AI-driven manufacturing. The White House has denied rumors of a potential ban on COVID-19 vaccines, as the pharmaceutical industry continues to innovate and adapt to challenges.With ongoing developments in scientific research and drug development, the industry remains at the forefront of innovation.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.AbbVie has acquired Gilgamesh Pharmaceuticals for $1.2 billion, focusing on neurology and depression treatments. This move follows AbbVie's previous failure with Emraclidine in schizophrenia treatment. The biotech industry's IPO stalemate appears to be ending, with Lb Pharma planning an IPO to fund a phase III-ready schizophrenia asset. The FDA has pulled Valneva's chikungunya shot from the market due to safety concerns, and several obesity drugs have failed to make it to market. Meanwhile, Arnatar is working on advancing RNA therapies beyond silencing.In other news, the disappointment of oral obesity therapies is discussed, with Novo emerging as a leader in the field. Trials for Eli Lilly's Orforglipron and Viking Therapeutics' VK2735 were underwhelming, leading to increased interest in weight loss pills in low- and middle-income countries. Capricor Therapeutics plans to fight FDA rejection of its DMD cardiomyopathy treatment, while a study in JAMA contradicts claims of corruption in vaccine advisory committees. Updates on cancer, cell and gene therapy are also provided.Stay tuned for more updates and feel free to suggest topics for future coverage.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.##New drug approval for rare genetic disorderToday, the FDA announced the approval of a new drug for a rare genetic disorder that affects approximately 1 in every 50,000 people. This drug has shown promising results in clinical trials, with patients experiencing significant improvements in their symptoms. This approval is a major milestone for the company and for patients who have been waiting for a treatment for this debilitating condition.##Positive results from Phase 3 clinical trialIn other news, a pharmaceutical company has reported positive results from a Phase 3 clinical trial for a new cancer treatment. The trial showed that the treatment was effective in shrinking tumors and improving survival rates in patients with advanced cancer. This is great news for the company and for patients who are in need of new treatment options.##Collaboration between two biotech companiesTwo biotech companies have announced a collaboration to develop a new treatment for a common autoimmune disease. This collaboration brings together the expertise of both companies and has the potential to lead to significant advancements in the field of autoimmune disease treatments. This partnership is a great example of how collaboration between companies can lead to innovative new treatments for patients.##FDA warning about counterfeit drugsThe FDA has issued a warning about counterfeit drugs that are being sold online. These counterfeit drugs may contain harmful ingredients or may not be effective in treating the conditions they are intended for. It is important for consumers to be cautious when purchasing medications online and to only buy from reputable sources. The FDA is working to crack down on the sale of counterfeit drugs and protect consumers from potential harm.##Drug pricing controversyThere has been controversy surrounding the pricing of a new drug that was recently approved by the FDA. The high cost of the drug has raised concerns about access to treatment for patients who may not be able to afford it. The pharmaceutical company has defended the pricing of the drug, citing the high cost of research and development. This controversy highlights the ongoing debate over drug pricing and access to affordable healthcare.##Investment in gene editing technologyA biotech company has announced a major investment in gene editing technology, with the goal of developing new treatments for genetic disorders. Gene editing technology has the potential to revolutionize the field of medicine by allowing scientists to make precise changes to DNA. This investment represents a significant step forward in the development of gene editing therapies and could lead to groundbreaking new treatments for patients with genetic disorders.##That's all for today's episode. Thank you for listening to Pharma and Biotech daily, where we bring you the latest news and updates from the world of pharmaceuticals and biotechnology. Stay tuned for more episodes coming soon.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. HHS employees have accused RFK Jr. of spreading public mistrust against the CDC after a shooting at their headquarters. They have asked him to stop spreading health misinformation, especially about vaccines and infectious diseases. In other news, Johnson & Johnson has added $2 billion to their US manufacturing commitment amid Trump's tariff threats. Regeneron's Eylea HD decisions have been delayed, and Stealth Biotherapeutics' rejected Barth drug is returning to the FDA for review. Fedegari is now a one-stop solution provider for the pharmaceutical industry, offering highly customized solutions for sterile drug production. Additionally, the American pharma industry is facing challenges with tariffs and policy changes. Several upcoming webinars and job opportunities in the pharmaceutical industry are also highlighted in the newsletter.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.The FDA is pushing new guidelines for cancer trials, recommending overall survival as a primary endpoint. Capricor Therapeutics is asking the FDA to reconsider their rejection of a treatment for DMD cardiomyopathy with the return of Vinay Prasad. Rocket Pharmaceuticals' phase II trial has resumed after a patient death, and the Trump administration's tariffs and drug pricing policies are causing confusion in the pharmaceutical industry. Fedegari is now offering customized solutions for the bio/pharmaceutical industry. The American Academy of Pediatrics is recommending the COVID-19 vaccine for kids, and there are updates on various biotech developments. Opinion pieces discuss drug pricing and the viability of high-dose peptides.The American pharmaceutical industry has lost the trust of the people, with many viewing them as the 'bad guys' despite producing drugs in the U.S. for Americans. The industry faces challenges such as competition and changing regulations, with the Trump administration's trade policies adding to the confusion. Some companies are taking a platform strategy approach to drug development, using one molecule to target multiple diseases. Despite these challenges, there are opportunities for innovation and growth in the industry. Additionally, conflicts of interest in vaccine committees have been decreasing, and companies are making strategic moves such as forming partnerships and restructuring to adapt to the changing landscape. The industry continues to navigate uncertainty and seek ways to improve drug development and access for patients.

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.## Viking Therapeutics' oral obesity pill, VK2735, achieved over 12% weight loss in a clinical trial, causing their stock to plummet due to safety concerns. The pending approval of oral Wegovy has also brought attention to high-dose peptides.## Meanwhile, leaked information reveals a proposed overhaul of vaccine strategies and access to novel therapies. The FDA rejected PTC Therapeutics' drug for Friedreich's ataxia, and Novo Nordisk reassured investors of their capacity to supply oral semaglutide. Fedegari now offers customized solutions for the pharmaceutical industry, and Vantai is in talks with Halda for a potential proteomics partnership. ## Other news includes conflicts of interest in vaccine committees declining, RegenxBio facing a delay in their gene therapy approval, and Genentech ending a partnership with Adaptive Biotechnologies. Kriya raises $313 million for gene therapy, Merck KGaA invests in RNA-targeting technology, and Novo Nordisk receives approval for Wegovy.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. Novo Nordisk's drug Wegovy has received approval for label expansion, potentially boosting its market performance. Tonix has also secured approval for a new fibromyalgia medication. Additionally, Novo has teamed up with GoodRx to provide lower prices for its drugs. Merck KGaA has made a significant investment of $2 billion in RNA-targeting technology through a collaboration with Skyhawk Therapeutics. On the sterilization front, Fedegari now offers tailored solutions for the pharmaceutical sector. In other updates, Pfizer's sickle cell therapy did not succeed in Phase III trials, while gene therapy company Kriya has raised an impressive $313 million. Kennedy has shown support for mRNA vaccines in cancer treatment. Bausch Health has closed a facility, Precigen has received FDA approval for an immunotherapy drug, and Eli Lilly has partnered with Superluminal in the obesity pipeline. Stay tuned for more industry news and developments.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world. Eli Lilly plans to raise drug prices in Europe in response to Trump's Most Favored Nation policy. RFK Jr. has expressed support for mRNA cancer vaccines after cutting mRNA BARDA contracts. Precigen wins FDA approval for an immunotherapy for recurrent respiratory papillomatosis, while Kennedy revives a childhood vaccine safety group and suggests mRNA vaccines could be effective for cancer. Genscript announces a new era of innovation, Bausch Health closes a California site, and Eli Lilly partners with Superluminal for obesity treatments. Trump delays tariffs, and upcoming webinars focus on AI in research, integrated supply chain strategies, and data lakes. Job opportunities include positions at Utro Biopharma, BioMarin Pharmaceutical, and AbbVie.

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.Eli Lilly has invested $1.3 billion in a partnership with Superluminal, an AI/ML startup focusing on endocrine and cardiometabolic diseases to develop new small molecule obesity medications. Schrodinger has discontinued the development of an early-stage blood cancer drug after two patient deaths. Trump has delayed pharma tariffs citing other priorities. RFK Jr. has been criticized for canceling Barda contracts related to mRNA vaccine research, leading to more vaccine misinformation. Genscript announces a new era of innovation and trust. Other news includes Vedanta downsizing, Abata shutting down.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. Sarepta has released new safety data for their gene therapy Elevidys, showing zero ambulatory deaths in patients with Duchenne muscular dystrophy. This data confirms the therapy's positive risk/benefit profile. Meanwhile, Novo Nordisk's rapid rise and fall was driven by GLP-1s like Ozempic and Wegovy, leading to a plummet in stock value. Insmed has received FDA approval for the first bronchiectasis drug and DPP1 blocker, Brinsupri. Texas has accused Eli Lilly of "bribing" prescribers to push their drugs Mounjaro and Zepbound, leading to a new lawsuit. Genscript is entering a new era of innovation and trust, offering exclusive promos and events. Additionally, Tang Capital is on a buyout binge in the biotech industry, while a top ARPA-H official has departed in protest of cuts to mRNA funding. The biotech industry continues to see contractions with companies like AbSci, Fate, and Oric downsizing. In career advice, loyalty is seen as a flawed strategy that can cost individuals opportunities.Arrowhead, a biotech company, has emerged as a savior in the RNA interference (RNAi) pipeline after Sarepta faced troubles unrelated to Arrowhead's assets. Concentra Biosciences, a biotech shell company, has been on a buying spree, acquiring four biotechs in the past month and seven so far this year. President Trump's most favored nation drug pricing policy is seen as unfavorable for all parties involved. Novo Nordisk experienced a rapid rise and fall due to the success of ozempic and wegovy. Lilly is facing drug pricing pressure while defending its injectable GLP-1 empire. Novartis is rumored to be considering acquiring RNA specialist Avidity. Despite safety concerns, Sarepta beat Q2 estimates, but not due to sales of its product Elevidys. Various biotech companies are downsizing while others are receiving significant investments.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. ## Bayer has secured a $1.3 billion deal with Kumquat Biosciences for exclusive access to their small-molecule KRAS G12D blocker. Meanwhile, Arrowhead has navigated through the Sarepta storm and experienced a drop in stock prices, despite having no direct ties to Sarepta's issues. In other news, a medical journal stands firm in refusing to retract a vaccine study, HHS has put a freeze on Vaxart's oral COVID-19 vaccine, and GenScript is celebrating a new era of innovation.## Additionally, discussions surround obesity leaders defending their injectable GLP-1 empires, recent layoffs at Merck, and the hurdles faced by oral obesity drugs. Opinion pieces delve into the skepticism towards mRNA technology and the potential repercussions of dismissing the head maha implementor following an mRNA purge. Stay tuned for more updates on upcoming events and job opportunities in the industry.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.Vinay Prasad has returned to his position as director of the Center for Biologics Evaluation and Research at the FDA after a sudden exit less than two weeks ago. The FDA has restricted the patient pool for bluebird's Skysona gene therapy due to safety concerns, and Sarepta's AAV gene therapy platform has come under scrutiny following patient deaths from liver injuries. Genscript is celebrating a new era of innovation and trust.The text discusses the scrutiny faced by AAV gene therapy technology following patient deaths related to Sarepta Therapeutics' platform. There is a push for improvement in the technology, while some are looking to move on to other options. The article also highlights the importance of mRNA technology in cancer research and the need for continued investment in this area. Additionally, it mentions the FDA's decision on Biogen and Eisai's Alzheimer's drug, as well as five oral obesity drugs that could rival Lilly's Orforglipron. Other topics covered include cancer drug developments, gene therapy news, and upcoming industry events.Stay tuned for more updates on the latest news in the pharmaceutical and biotechnology industries.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.Vinay Prasad has returned to the FDA as the Chief of the Center for Biologics Evaluation and Research, just 10 days after his mysterious departure. His return comes after a short "vacation" in California, and it is believed that the regulator convinced him to come back. Some speculate that Prasad's time away may have helped him gain a better understanding of the complexities involved in decision-making at the FDA, especially when it comes to ensuring the safety of patients and meeting the needs of various stakeholders. This unexpected turn of events has generated interest and optimism among industry insiders.Gilead remains positive about the prospects of its HIV prevention drug, despite uncertainties surrounding recommendations from the U.S. Preventive Services Task Force. The FDA has launched a new program to help pharmaceutical companies lower regulatory barriers to manufacturing in the U.S. Other news includes Dewpoint Therapeutics slashing headcount, Novartis rumored to be taking over RNA specialist Avidity, and Trilink Biotechnologies offering process development services for nucleic acid therapeutic development. Lotte Biologics is expanding its global manufacturing bases, while other companies like Bicycle, Tune, and Iovance are downsizing to conserve cash. The FDA is in flux, Pfizer is discussing pricing with Trump, and Merck is cutting jobs. Vertex's next-generation pain drug failed in trials despite strong Q2 earnings. Thank you for listening to Pharma and Biotech daily: your source for quick updates on the latest news in the pharmaceutical and biotechnology industries.

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. Eli Lilly is facing significant drug pricing pressure, with issues ranging from the pricing of their weight loss pill Orfoglipron to market battles with competitors like Novo Nordisk. Despite the challenges, Lilly's executives see opportunities for their products in the real world. Jazz Pharmaceuticals received FDA approval for Dordaviprone, making it the first new treatment for an ultra-rare brain tumor. In other news, Sarepta beat Q2 estimates despite not holding an investor call, and Trilink Biotechnologies is offering process development services for nucleic acid therapeutic development. Lotte Biologics is expanding its global CDMO operations, while a big pharma-backed mRNA startup raised $153 million in funding. Novo Nordisk's new CEO is focusing on obesity and diabetes, Pfizer is in talks with Trump on most favored nation drug pricing, and Bayer expects more layoffs. These developments in the pharma and biotech world showcase the ongoing challenges and opportunities in the industry. Stay tuned for more updates on the latest news and trends in this rapidly evolving sector.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.Novo Nordisk's new CEO, Maziar Mike Doustdar, is focusing on developing more drugs for obesity and diabetes while trimming the company's pipeline. The Department of Health and Human Services is terminating around $500 million in contracts for mRNA vaccine development, affecting companies like Moderna and Pfizer. Chinese biotechs are turning to big pharma for partnerships to survive, while Trilink Biotechnologies offers process development services for nucleic acid therapeutic development. Other top stories include biopharma CEOs being pulled into policy discussions, Bayer expecting more layoffs, and Trump threatening high tariffs on pharmaceuticals. The industry is also seeing investments in future technologies and strategies, such as AI for real-world research and integrated supply chain strategies for pharma manufacturing.Big pharma is providing support to struggling Chinese biotech companies by out-licensing drugs to multinational corporations. The recent increase in deals highlights the potential for partnerships with biotechs in the region. Biopharma CEOs are facing policy questions from tariffs to drug pricing, impacting their second quarter earnings calls. AI can help pharmaceutical companies adapt to policy pressures in the regulatory environment. Big pharmas are investing in incubators and venture funds to uncover scientific trends and determine future focus points. Pfizer's CEO is in contact with the President over drug pricing policies, while Moderna is focused on R&D partnerships rather than mergers and acquisitions. Novo's new CEO is emphasizing execution in obesity and diabetes research while trimming their pipeline. Biontech is committing to infectious diseases amid concerns over vaccine demand. Trump's threat of high pharma tariffs, a surge in July layoffs, and legal battles between Moderna, Pfizer, and Biontech are also making headlines. Events on AI for real-world research and integrated supply chain strategies for pharma manufacturing are upcoming, along with job opportunities in biostatistics, manufacturing, and publications.

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.##Breaking News: Pfizer announces successful COVID-19 vaccine trialsIn a groundbreaking announcement, Pfizer revealed that their COVID-19 vaccine candidate has shown to be over 90% effective in preventing the virus. This news brings hope to the world as we continue to battle the global pandemic.##FDA approves new treatment for Alzheimer's diseaseThe FDA has approved a new treatment for Alzheimer's disease, marking a significant advancement in the fight against this debilitating condition. This approval could potentially change the lives of millions of patients and their families.##Johnson & Johnson recalls baby powder due to asbestos contaminationJohnson & Johnson has issued a voluntary recall of its baby powder products after trace amounts of asbestos were found in samples. This news has raised concerns about the safety of talc-based products and the potential risks they pose to consumers.##Novartis announces major breakthrough in cancer researchNovartis has made a significant breakthrough in cancer research with the development of a new targeted therapy that has shown promising results in clinical trials. This innovation has the potential to revolutionize cancer treatment and improve outcomes for patients.##Merck receives FDA approval for new diabetes drugMerck has received FDA approval for a new diabetes drug that offers another option for patients struggling to manage their condition. This approval expands treatment options and provides hope for those living with diabetes.##Roche acquires biotech company in multi-billion dollar dealRoche has announced the acquisition of a biotech company in a multi-billion dollar deal that will expand its portfolio and strengthen its position in the market. This strategic move demonstrates Roche's commitment to innovation and growth in the biotech sector.##Incyte collaborates with academic research center to develop new therapiesIncyte has formed a collaboration with an academic research center to develop new therapies for a range of diseases, including cancer and inflammatory conditions. This partnership brings together expertise from both sectors to accelerate the discovery and development of innovative treatments.##Overall, these recent developments in the pharmaceutical and biotech industry highlight the ongoing efforts to advance healthcare and improve patient outcomes. From groundbreaking vaccines to innovative therapies, these advancements are shaping the future of medicine and providing hope for patients worldwide.

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. BioNTech is focusing on infectious diseases as vaccine rates drop, with expectations that the US COVID-19 vaccination rate will decrease slightly. Glioblastoma research is seeing increased funding and interest from companies like Merck and Jazz Pharmaceuticals. The FDA's rejection of Replimmune's melanoma drug, despite staff consensus, has caused a drop in the company's stock. Sarepta and Capricor learned of regulatory decisions through media leaks, causing confusion for patients and families. Trilink Biotechnologies offers process development services for nucleic acid therapeutic development. Lotte Biologics is expanding globally, Allogene is discontinuing an immunosuppressive antibody after a patient death, and NIH funding cuts are impacting biotech. Pharmas are increasing investment in AI despite challenges in the industry. Trump urges pharma companies to address drug pricing, while Merck plans to cut 6,000 jobs to save $3 billion.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.Moderna CEO Stephane Bancel has stated that the company is not interested in pursuing mergers and acquisitions, preferring to focus on research and development partnerships instead. Former President Trump has threatened 17 big pharmaceutical companies with a deadline to lower drug prices or face government intervention. Regeneron faces regulatory issues affecting decision dates for high-dose Eylea, while Moderna has won a UK patent battle against Pfizer and BioNTech. After the removal of FDA chief Dr. Prasad, Dr. Makary is seeking better alignment between drug and biologic approvals at the agency. Alnylam's impressive sales of Amvuttra ATTR-CM have led to a surge in stock prices, while Merck is aiming to save $3 billion through job cuts. Moderna is also cutting 10% of its global workforce. Recent FDA approvals for myeloma and Alzheimer's treatments, as well as updates on layoffs and pipeline developments in the biotech industry are also making headlines.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. Biogen's sales of the partnered Alzheimer's drug Leqembi have climbed 20% in the second quarter, while CEO Chris Viehbacher dismisses rumors of disagreements with Eisai. Moderna has cut 10% of its global workforce as part of cost-cutting measures. AbbVie is celebrating the success of Skyrizi and Rinvoq, Lilly's Mounjaro shows promise in a cardiovascular trial, and there is optimism around Sarnas for therapeutic breakthroughs. Amidst industry challenges, many biopharma professionals are looking for new job opportunities.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.Vinay Prasad's departure from CBER has analysts anticipating a more traditional successor, while GSK streamlines its pipeline and pledges billions in US investments. Susan Monarez is now the confirmed CDC director, and the top biopharma venture capital raises of H1 2025 are summarized. Despite challenges like layoffs and market fluctuations, GSK remains committed to investing in the US despite tariffs. Other headlines include Madrigal's potential $2 billion investment, Biogen and Eisai's Alzheimer's drug update, and Novo Nordisk's new leadership. Trilink Biotechnologies is offering self-amplifying RNA constructs for potential therapeutic advancements.AI biotech companies have secured substantial funding in the first half of 2025, with continued investment pouring into startups within the industry. The top five biopharma venture capital raises of this period are highlighted. There is confusion surrounding Ira's definition of 'drug,' potentially hindering companies from pursuing approval for new formulations and indications. Vinay Prasad's departure from the FDA's Center for Biologics Evaluation and Research, following controversies related to Sarepta, has raised concerns among developers. Despite challenges, four biotech companies are successfully launching their products independently.Over 260 million people are impacted by rare diseases, emphasizing the need for faster evidence generation through global real-world data. GSK's commitment to investing in the US, Merck's cost-cutting measures to support its launch schedule, and Novo Nordisk's new leadership are also highlighted. The FDA has updated regulations regarding Sarepta's DMD gene therapy, making it available for ambulatory patients. Adaptimmune anticipates significant staff reductions following a cell therapy asset sale. Stay tuned for more updates on the latest 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 e Biotech world. Novo Nordisk has announced Maziar Mike Doustdar as its new CEO, along with a 5% reduction in its 2025 sales guidance. This decision comes as a response to the slowed growth of its semaglutide franchise in the U.S. Senate Democrats are currently looking into HHS Secretary RFK Jr.'s controversial move to dismiss all 17 members of the CDC's vaccine advisory committee. Astrazeneca CEO Pascal Soriot emphasized the importance of global collaboration in pharmaceutical research and development. Merck has allocated $3 billion to support upcoming product launches. In a surprising turn of events, the FDA has reversed its decision on Sarepta, now permitting DMD gene therapy for ambulatory patients.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world. GSK has entered a partnership with Hengrui worth up to $12 billion, focusing on the COPD candidate HRS-9821. The FDA's new voucher policy has caused confusion among experts, as it lacks clear definition and was announced without public input. The American Medical Association urges RFK Jr. to maintain the preventive task force, and Sarepta denies a patient death is linked to Elevidys as the FDA launches a probe. Biopharma companies are leaning towards holistic treatments for Alzheimer's, while Atai-partnered schizophrenia drug fails in a mid-stage trial. Boehringer partners with Irish startup Re-Vana in a $1 billion+ eye deal.As the Alzheimer's space becomes more competitive, biopharma companies are focusing on holistic treatments beyond disease-modifying drugs like Leqembi and Kisunla. Companies such as Bristol Myers Squibb, Acadia, Otsuka, and Lundbeck are renewing their search for symptomatic treatments. Five upcoming data drops could potentially lead to more effective therapies for Alzheimer's. Sarepta Therapeutics is facing challenges due to safety concerns surrounding its gene therapies, leading to a drop in stock value. The FDA's new voucher program aims to offer accelerated pathways for drugs meeting certain criteria, but experts criticize the lack of transparency and public input in the policy's announcement. Sarepta's future is uncertain as the FDA considers a new study for Elevidys, and the EU issues a negative opinion on the drug. Other news includes delays in the FDA decision on GSK's Blenrep, AstraZeneca's PIII win with nanobody treatment for myasthenia gravis, and the removal of thimerosal from influenza vaccines. George Tidmarsh has been appointed as the new chief of FDA's CDER.

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. The European Medicines Agency's CHMP did not recommend approving Elevidys for ambulatory patients with Duchenne muscular dystrophy, dealing a blow to Sarepta. FDA is rumored to request new data for Elevidys, leading to uncertainty as FDA considers a new study for the drug. In other news, Eli Lilly commits $856 million to Gate Bioscience for a new class of medicines, while Rocket trims headcount and pipeline focus. Roche also drops an early obesity asset as layoffs continue in the biopharma industry, with companies like Adicet optimizing their pipelines. Novartis makes a billion-dollar drug discovery deal with Matchpoint, and the FDA opens a pilot run of the commissioner voucher program.Stay tuned for more updates on the latest developments in the pharmaceutical and biotech world.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.Roche has dropped an early-stage obesity asset, CT-173, citing lack of competitiveness. Novartis has entered into a drug discovery deal with Matchpoint Therapeutics, acquiring global rights on all molecules for several inflammatory diseases. AstraZeneca claims a Phase III win with its nanobody treatment for myasthenia gravis. Second-quarter earnings season is approaching, and biotechs to watch include Sarepta and others facing challenges in the biopharma industry. Genentech downsizes as priorities shift, and GSK's comeback for Blenrep is on pause as the FDA delays its decision. The FDA's lack of transparency has tarnished Sarepta's reputation after patient deaths triggered an FDA battle. Opportunities in the industry include roles like Quality Specialist at CSL and Clinical Research Physician at Eli Lilly and Company.

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 has initiated a voucher program to address lower drug prices and boost domestic investment, with up to five companies being selected based on national priorities. Roche has halted shipments of Sarepta's Elevidys gene therapy after reports of patient deaths. Sarepta is under FDA scrutiny following a third patient death, while the agency welcomes a new top drug regulator and rejects multiple new drugs from entering the market. Insights from women in biopharma highlight the importance of resilience. Zhen Li, an engineer, is playing a crucial role in shaping the field of siRNA. Research suggests that GLP-1s may offer protection against neurodegenerative diseases. Dispatch Bio enters the scene with a substantial $216 million investment for universal CAR T therapy. Sarepta complies with an FDA request to suspend US shipments of Elevidys, raising concerns about transparency and impacting the company's reputation. Replimune experiences a significant drop in stock value following an FDA rejection, while AstraZeneca makes a bold $50 billion investment in US manufacturing to prepare for potential tariffs. Thank you for tuning in to Pharma and Biotech daily for the latest updates in the industry.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world. Sarepta Therapeutics has halted the distribution of Elevidys in the US after the FDA request, following two deaths. The company's transparency has been called into question, affecting its stock value. Replimmune also faced setbacks after the FDA rejected its melanoma treatment, causing a significant drop in shares. On a more positive note, AstraZeneca has pledged $50 billion for US manufacturing, focusing on drugs like Baxdrostat and oral glp-1 therapies. Sanofi has made a $1.6 billion vaccine acquisition, while Biogen commits $2 billion to expand US drug production. The pharmaceutical industry is seeing various developments and challenges, with companies adapting to regulatory demands and market pressures.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.George Tidmarsh, a biopharma veteran and adjunct professor at Stanford's medical school, has been appointed as the new head of the FDA's Center for Drug Evaluation and Research (CDER). His extensive industry experience will be valuable in his new government position. Data secrecy among cell and gene therapy developers is hindering progress in the field, causing fragmentation, stalling innovation, and delaying access to treatments. Meanwhile, Sarepta is facing challenges with its elevidys shipments and has lost platform designation for its technology. At the same time, Roche's phase III trial in COPD has failed, impacting the market path for astegolimab. Layoffs are happening at companies like GSK, Sail, and BioNTech. Experts are exploring new ways to overcome barriers in cell therapy production.Data secrecy among cell and gene therapy developers continues to hinder progress and access to treatments. Acadia has introduced a new team and pipeline with ambitious goals. Patients are fighting for access to Brainstorm's ALS drug after promising real-world data. Moderna's withdrawal of its flu vaccine has left combination flu/COVID-19 vaccines in limbo. In other news, Sarepta is facing challenges with its DMD gene therapy, Ultragenyx's gene therapy for Sanfilippo syndrome is rejected by the FDA, GSK's Blenrep loses an adcomm vote, and BMS' anemia drug Reblozyl fails a Phase III trial. The FDA is experiencing layoffs and employee turnover amid an overhaul. Vinay Prasad overruled reviewers on Moderna's COVID-19 shot for kids. Upcoming events include a webinar on AI for real-world research and job opportunities in the biopharma industry. Readers are encouraged to provide feedback and suggest topics for future coverage.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. Sarepta Therapeutics experienced a significant drop in shares as the FDA considers requesting a halt to shipments of the Duchenne muscular dystrophy therapy Elevidys. This decision comes after a third patient death linked to the underlying platform. The company's stock plummeted by 37% on Friday afternoon as reports of the potential shipment stop circulated in the media. This news adds to the already turbulent week for Sarepta, which has been facing challenges in its gene therapy and siRNA biotech developments. ## Novartis announced positive results from a phase 3 trial of its drug Cosentyx in treating axial spondyloarthritis. The study showed that patients treated with Cosentyx had significant improvements compared to those on a placebo. These results further solidify Novartis' position in the market for treatments of autoimmune diseases. The company plans to submit the data to regulatory authorities for potential approval of this indication.Novartis revealed encouraging outcomes from a phase 3 trial of its drug Cosentyx for treating axial spondyloarthritis. The study demonstrated that patients who received Cosentyx experienced notable enhancements compared to those who were given a placebo. These findings strengthen Novartis' standing in the autoimmune disease treatment market. The company intends to present the data to regulatory bodies for potential approval of this indication.## AstraZeneca faced setbacks as it announced delays in delivering its COVID-19 vaccine doses to the EU. The company cited production issues as the cause of the holdup, leading to frustration among European officials. This news comes at a time when vaccine distribution is crucial in combating the ongoing pandemic, highlighting the challenges faced by pharmaceutical companies in meeting global demand for vaccines.AstraZeneca encountered obstacles when it disclosed delays in distributing its COVID-19 vaccine doses to the EU. The company attributed production problems as the reason for the delay, causing frustration among European officials. This development occurs during a critical period in vaccine distribution to combat the current pandemic, underscoring the difficulties pharmaceutical companies encounter in meeting worldwide vaccine demands.## Pfizer and BioNTech announced plans to test a third dose of their COVID-19 vaccine to assess its effectiveness against new variants of the virus. The study will involve participants who have already received two doses of the vaccine and will evaluate the immune response generated by a booster shot. This initiative reflects ongoing efforts by pharmaceutical companies to adapt their vaccines to combat emerging strains of the virus.Pfizer and BioNTech unveiled intentions to examine a third dose of their COVID-19 vaccine to determine its efficacy against new virus variants. The research will include individuals who have already been administered two doses of the vaccine and will assess the immune response produced by an additional shot. This undertaking demonstrates continuous endeavors by pharmaceutical firms to modify their vaccines in response to evolving virus mutations.

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in the Pharma and Biotech world. Sarepta Therapeutics has recently experienced an 18% increase in its stock value following a significant business overhaul, which included staff layoffs and pipeline shifts. Analysts are cautiously optimistic about the company's future. At the same time, patients are advocating for access to Brainstorm Cell Therapeutics' ALS drug, Nurown, after promising results from an expanded access program. In other news, Novartis is in the process of reshoring its drug manufacturing operations in the US, a move that may take several years to complete. Additionally, a notable number of employees have departed from the FDA's Center for Drug Evaluation and Research amidst an overhaul by the Department of Health and Human Services. These developments underscore the continuous changes and challenges within the pharmaceutical industry. Sarepta Therapeutics recently announced a strategic overhaul, which involved cutting 500 staff members and shifting focus to sirna platform assets. This decision came after two patients passed away following treatment with its Duchenne muscular dystrophy gene therapy, Elevidys. The company has also added a black box warning for acute liver injury and failure to Elevidys as it pivots away from gene therapy programs.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. Johnson & Johnson reported second-quarter earnings of $23.7 billion, driven by cancer and neuroscience drugs, exceeding analyst expectations. CEO Joaquin Duato set a target of $50 billion in oncology sales by 2030. Despite challenges in the industry, Johnson & Johnson remains optimistic about its oncology sales target as the biotech industry continues to navigate the evolving landscape of drug development and funding.In a difficult investing environment where IPOs are not guaranteed, AI-focused biotech unicorns are facing challenges in securing funding. GSK's Blenrep is facing setbacks as the FDA questions its efficacy in treating multiple myeloma, while AstraZeneca's amyloidosis asset failed to improve survival in a late-stage trial. The industry is also seeing the rise of women leaders like Audrey Greenberg and the team at Acadia, who are making significant contributions to the field.Biospace is launching a new weekly newsletter focused on critical manufacturing issues in the biopharma industry, covering developments and impacts on companies such as Roche, Sanofi, and Johnson & Johnson. The newsletter aims to provide deep dives, analysis, and roundups of the biggest manufacturing stories of the week every Tuesday. Stay informed with weekly analysis and updates on the changing landscape of manufacturing in the U.S.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.The U.S. Department of Health and Human Services (HHS) has begun formally laying off thousands of employees following a Supreme Court ruling allowing the Trump administration to reorganize the agency. FDA Commissioner Marty Makary has proposed lowering industry user fees to make it easier for smaller companies and academics to participate in the regulatory process. Developments in HIV and prep treatments are being presented at the IAS 2025 conference, while a new dual glp-1/gip molecule from Hengrui and Kailera Therapeutics has shown promising weight loss results in Phase III trials. A new 96-channel pipettor from Rainin is also being promoted to streamline library preparation for next-generation sequencing. Updates on Acadia's pipeline, FDA rejections for Ultragenyx, advancements in narcolepsy treatments by Takeda, and the potential use of psychedelics in depression treatment are also discussed in this episode. Stay tuned for more updates on the latest news and developments in the pharmaceutical and healthcare industries.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world. Takeda has taken the lead in the race for a narcolepsy treatment with back-to-back phase III wins for their drug Oveporexton. Investors are eagerly awaiting breakthroughs in using psychedelics to treat depression. Ultragenyx faced a setback as the FDA rejected their gene therapy for Sanfilippo syndrome, citing manufacturing issues. The FDA is considering speeding up reviews for companies that promise to lower drug costs. Market reaction to recent readouts from Compass Pathways and Beckley Psytech/Atai in treatment-resistant depression shows the challenges psychedelic therapies must overcome for commercial viability. Rainin Micropro offers a solution to streamline NGS preparation with their 96-channel pipettor. The industry is also focused on precision diagnostics to support precision therapeutics in the future. AstraZeneca's Baxdrostat showed promising results in lowering blood pressure in a phase III trial. The ADA revealed R&D priorities for potential blockbuster obesity treatments. Relmada has abandoned development of a depression drug after three failed attempts.The challenges faced by psychedelic therapies in the treatment of depression are discussed, as recent readouts from Compass Pathways and Beckley Psytech/Atai in treatment-resistant depression have left investors wanting more. The market reaction highlights the hurdles psychedelic therapies must overcome to prove their commercial viability. Additionally, the importance of precision diagnostics in the development of next-generation precision oncology therapies is emphasized, stating that only with the adoption of digital imaging and AI-powered analysis will these therapies reach their full potential. The FDA has several important decisions lined up, including applications in lymphoma, rare diseases, and hormone deficiency, while the American Diabetes Association's annual meeting reveals R&D priorities for weight loss medicines. Topics discussed include Capricor's FDA rejection of a DMD cell therapy, the ALS community petitioning the FDA to reconsider Brainstorm's Nurown, and updates on COVID-19 vaccines and Alzheimer's drugs. Upcoming webinars and job opportunities in the biopharma industry are also included.

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.The FDA has rejected Capricor's therapy for Duchenne Muscular Dystrophy (DMD), causing shares to plunge due to insufficient evidence of effectiveness for cardiomyopathy associated with DMD. Lilly and Sarepta also received Complete Response Letters (CRLs) for their therapies. Moderna has won full approval for its COVID-19 vaccine for higher-risk kids, while Pacira has cut staff in San Diego. The FDA has released a cache of CRLs, providing insights into the journeys to market for various drugs. AbbVie has acquired Ichnos Glenmark's lead myeloma antibody, and Ultragenyx's stock plummeted after a late-stage trial disappointment. Kennedy postponed a preventive care panel meeting after a Supreme Court win. Transitioning from one piece of news to the next, let's move on to the webinar exploring digitization strategies for drug development. Stay tuned for more updates 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.The FDA has recently released over 200 complete response letters (CRLs) as part of its initiative to enhance transparency regarding the reasons behind the rejection of new drug and biologics applications. This effort reflects the agency's commitment to being more open with the public about its decision-making processes.In other news, AbbVie has made a significant acquisition by purchasing Ichnos Glenmark's lead myeloma antibody for nearly $2 billion, including a substantial upfront payment of $700 million. However, Ultragenyx and Mereo Biopharma faced challenges as their stocks declined following the announcement that the phase II/III study of their osteogenesis imperfecta candidate did not yield strong results during an interim analysis.Furthermore, there is growing concern surrounding the potential dismissal of the U.S. Preventive Services Task Force, following a Supreme Court ruling that affirmed Health Secretary Robert F. Kennedy Jr.'s authority to remove its members at his discretion. This development has raised questions about the future of preventive health initiatives in the country.For drug developers, finding the right balance between speed and quality is essential for achieving success in the industry. To optimize processes and leverage technology throughout the development journey, new digitization strategies are being explored. Embracing these advancements can help streamline operations and enhance overall efficiency in drug development efforts.