Evie Prescott’s Post

The largest pharmaceutical company in the world has tapped Swiss biotech HAYA Therapeutics to develop weight-loss drugs that target a specific type of RNA. Eli Lilly and Company—whose market capitalization currently hovers around $855 billion because of the commercial success of its weight-loss drugs Mounjaro and Zepbound—will collaborate with Haya to find new drug targets for chronic metabolic conditions and obesity. Lilly will pay Haya an undisclosed amount up front that includes an equity investment. Overall, the deal is worth up to $1 billion in potential milestone payments and royalties. “People are really starting to think about the large indications. We’ve seen this obviously in the cardiometabolic space and the obesity space, and I’ve sensed and felt a shift in priority from Big Pharma,” Haya CEO Samir Ounzain told C&EN earlier this year. “We believe the power of our platform can be applied to any cell state and any tissue. So absolutely, in the mid- to long term, I think the best way to demonstrate the potential of the regulatory genome is to find appropriate partners.” https://lnkd.in/djuWCDNv

A Record Year 2023 for Novel Drug Approvals by the FDA/CDER The estimated cost of developing a single FDA-approved drug is around $2.6 billion. The year 2023 has been a remarkable one for the pharmaceutical industry, as the FDA/CDER has approved 56 novel drugs, a 51% increase compared to 2022. #fda #drugapproval #innovation Here are some of the highlights and trends from the novel drug approvals in 2023: 🏆 Pfizer leads the pack with 6 approvals, followed by Biogen with 4, and AstraZeneca, Chiesi, and UCB with 3 each. #Pfizer #Biogen #AstraZeneca #Chiesi #UCB 🩺 Oncology remains the most popular therapeutic area, with 17 approvals, followed by neurology and autoimmune disorders, with 6 each. #Oncology #Neurology #Autoimmune #DrugApprovals 💊 Small molecules account for more than half of the approvals, with 31, followed by therapeutic monoclonal antibodies, with 12. #SmallMolecules #MonoclonalAntibodies #PharmaTrends 🧬 DNA/RNA based drugs, such as gene therapies and antisense oligonucleotides, are gaining traction, with 4 approvals. #GeneTherapy #RNA #Innovation 🌱 Other novel drugs include a natural extract, a radioactive diagnostic agent, and a fusion protein. #NaturalExtract #DiagnosticAgent #FusionProtein The novel drug approvals in 2023 reflect the diversity and innovation of the pharmaceutical industry, as well as the commitment and collaboration of the FDA/CDER to bring safe and effective treatments to the patients who need them. #pharmainnovation #patientcare #healthcareinnovation If you want to learn more about the novel drug approvals in 2023, you can check out this link: https://lnkd.in/gQ_kU7Tr 📰 #DrugApprovals #pharmanews #medicaladvancements 👉👉You can also check out this insightful analysis by Aaher Mullard in #NatureReviews, which covers the historical data of drug approval, the competition and innovation in the field, and the #DrugDiscovery expectations for 2024. You can find Aasher’s article on the following link: https://lnkd.in/ejHvaxT6

Otsuka Pharmaceutical Companies (U.S.) Pays Up to $1.1B to Buy Jnana Therapeutics, Adds Drug Discovery Capabilities With an upfront $800 million payment and $325 million in potential milestones, Otsuka Pharmaceutical is acquiring Jnana Therapeutics’ drug discovery platform and rare disease candidate, the companies announced Thursday. Otsuka Pharmaceutical has struck a deal to buy Jnana Therapeutics for $800 million upfront and a potential $325 million in milestone payments for its drug discovery platform and pipeline led by a clinical-phase rare disease candidate, the companies announced Thursday. Jnana discovers potential drug candidates by exposing protein targets to its library of chemical fragments inside living cells. The Boston-based biotech has designed the library to include a wide range of fragments that may bind and covalently label druggable pockets on the target protein. After finding a fragment that binds a target, Jnana screens a library of drug-like compounds to identify small molecules that engage the same part of the protein. Otsuka’s statement on the reasons for the acquisition discussed Jnana’s platform before mentioning the drug candidates it has generated. The Japanese drugmaker said the platform can address hard-to-drug target classes including solute carriers, transcription factors and signaling scaffold proteins. Otsuka sees the platform as complementary to the capabilities of its existing subsidiary Astex Pharmaceuticals. Jnana, like Astex, will operate as a wholly owned subsidiary of Otsuka and will continue its R&D. The biotech has used its platform to find small molecules that inhibit SLC6A19, a transporter responsible for intestinal absorption and renal reabsorption of phenylalanine (Phe). People with phenylketonuria (PKU) are unable to break down Phe. https://lnkd.in/ecgf2Yhs

𝗥𝗲𝘀𝗶𝗹𝗶𝗲𝗻𝗰𝗲 𝗘𝘅𝗽𝗮𝗻𝗱𝘀 𝗖𝗹𝗶𝗻𝗶𝗰𝗮𝗹 & 𝗖𝗼𝗺𝗺𝗲𝗿𝗰𝗶𝗮𝗹 𝗗𝗿𝘂𝗴 𝗣𝗿𝗼𝗱𝘂𝗰𝘁 𝗠𝗮𝗻𝘂𝗳𝗮𝗰𝘁𝘂𝗿𝗶𝗻𝗴 𝗖𝗮𝗽𝗮𝗯𝗶𝗹𝗶𝘁𝗶𝗲𝘀 Resilience Inc. a technology-focused biomanufacturing company dedicated to broadening access to complex medicines, has expanded its clinical and commercial drug product manufacturing capabilities with the goal of providing over 200 million units to its partners across its manufacturing network by 2025 in support of multiple modalities and therapeutic indications, including GLP-1.   This expansion aims to address the biomanufacturing industry’s growing demands within the drug product space, further responding to industry needs.   In Cincinnati, at least $225 million is planned to be invested to increase the site’s drug product capacity. The FDA-licensed site currently has three high speed fill lines for Vials, Cartridges and pre-filled syringes (PFS), with a fourth PFS fill line ready by 2025. The facility is also expanding to six device assembly and packaging suites by 2025 to meet the industry’s growing biomanufacturing demands.   Additionally, Resilience is expanding its drug product capabilities at the company’s RTP facility in North Carolina using a modular design within its segregated expansion space to offer drug product manufacturing for multiple modalities. The site has drug substance and drug product experience in support of Gene Therapy medicines. It also has 45,000 square feet of grey space available for additional expansion that will be leveraged for drug product operations. As part of the growth plans, a new Bausch+Ströbel SE + Co. KG Isolator filler will be installed to support Vials and PFS, providing flexibility and quality assurance with its barrier isolation technology.   “Strengthening our drug product manufacturing capacity across the Resilience network highlights our commitment to providing robust and scalable outsourcing options for our partners,” said Rahul Singhvi, Sc.D., CEO of Resilience. “As a highly experienced team already supporting a leading pharmaceutical company with their GLP-1 products, this expansion further supports Resilience’s mission to ensure adequate biomanufacturing capacity by addressing and overcoming historic manufacturing challenges throughout the industry.” https://lnkd.in/eTrgbCK2

📘 I recently dove into "Pharma and Profits" by John LaMattina, former President Pfizer Global Research & Development, and it's probably the best book I've read on the main debate that generally surrounds the pharmaceutical industry: drug pricing. Two insights 💡 from the book truly stood out: 1️⃣ FOCUS ON PRICE VS. VALUE: We often only see the price tag of new drugs, a $1000-pill here, a $475,000 gene therapy there, as these make for sensational headlines 📰 But as LaMattina outlines, this singular focus on price shows a disinterest to dig deeper to understand the current standard of care and value of a medicine. Fundamentally, we should always ask ourselves: 👉1/ How does a drug compare to existing treatments (including surgical and hospital based) in terms of efficacy and safety? 👉2/ How expensive are these other treatments? 👉3/ Without this drug, how many will die or have other adverse events? 👉4/ Without the drug, what is the ultimate cost to the healthcare system? In the case of the $1000-pill for example, which cures hepatitis C in more than 90% of cases in just 12 weeks (costing $84,000/patient), the answers would be: 👉1/ 90% cure rates vs. 40-80% for older treatments 👉2/ generally over $100,000 per treatment 👉3/ approx. 20% of people with chronic hepatitis C would progress to liver cirrhosis, liver cancer and may eventually experience liver failure 👉4/ with liver transplants alone costing around $300,000, and add to that all the post-transplant care and anti-rejection drugs ($40,000 per year per patient for several years), it becomes clear that the $1000-pill provides great value for money. The same exercise can be performed for the $475,000 gene therapy and other innovative medicines. 2️⃣ MEDICINE PRICES AREN’T WHAT’S DRIVING HEALTHCARE COSTS: High drug prices must be what drives healthcare costs, right? Not quite. In fact, as LaMattina outline for the US, pharmaceutical spending has been stable over past decades at around 13% of healthcare spending, while at the same time costs for hospital procedures have continuously been rising, outpacing the rate of inflation. But that’s the US, surely Europe is different? Nope, pharmaceutical expenditure in Europe has remained stable for the past 20 years at an average of around 15% of healthcare spending (https://lnkd.in/eB9ivDrK). This is despite an increase in the use and reliance on pharmaceuticals and the many breakthrough therapies that have emerged. ➡ There are many more insights like these in LaMattina's book. At only 97 pages, the book is short but as insights packed as it gets, and while focusing on the US, most of the principles and reasoning apply to the European context as well. #pharma #healthcare #medicines #healthcarecosts

🖇️Overcoming Bottlenecks in Drug Delivery by Innovative Proteolipid Vehicle 🔔🔔🔔🔔 Dr. Jailal Ablack kicked off the webinar by elaborating on challenges and recent technological breakthroughs in nucleic acid drugs. He noted that there are still bottlenecks to be overcome in terms of how researchers can seize the right timing to deliver therapeutic nucleic acids to the right cell types, which in turn leads to considerations of drug safety and redosability. 🎯🎯mRNA as the Fourth Pillar for Biopharmaceutical Innovation📢 Sophia Lugo analyzed the regulatory framework and approval process of nucleic acid drugs in the US FDA and European Medicines Agency (EMA) and shared valuable insights into current trends and challenges. “We are entering the age of RNA and mRNA can be called the fourth pillar for biopharmaceutical innovation, alongside small molecules, biologics, and cell and gene therapy,” said Lugo. Actually, the number of therapeutic mRNAs in the R&D stage has skyrocketed from about 100 to over 1,300 (as of June 2024) in just a few years compared to the pre-COVID-19 days. This, coupled with the success of mRNA COVID-19 vaccines and the progress of Moderna ’s cancer vaccine into Phase 3 clinical trials, highlights the tremendous possibilities of the mRNA market. 🔉🔊 Full article 🎮♟️ https://lnkd.in/dKGWmuWs #drugdelivery | #nucleicaciddrugs | #therapeutic |#BiopharmaceuticalInnovation

Resilience just announced the expansion of its clinical and commercial drug product manufacturing capabilities across its network. With a $225 million investment planned for its Cincinnati facility, which they acquired from AstraZeneca in 2022 and an expansion of fill/finish operations at its Research Triangle Park facility, which it acquired from bluebird bio for $110 million in 2021. By scaling its capacity, Resilience intends to provide over 200 million units to partners across its manufacturing network by 2025, supporting various modalities and therapeutic indications, including GLP-1. The expansion in Cincinnati will see investments to increase drug product capacity, including the addition of a fourth pre-filled syringe (PFS) fill line by 2025. The facility will also expand its device assembly and packaging suites to meet industry demands. In RTP, Resilience will leverage modular design within its segregated expansion space to offer drug product manufacturing for multiple modalities, including Gene Therapy medicines. A new Bausch & Strobel Isolator filler will be installed to support vials and PFS, enhancing flexibility and quality assurance. Rahul Singhvi, Chief Executive Officer of Resilience, stated, "Strengthening our drug product manufacturing capacity across the Resilience network highlights our commitment to providing robust and scalable outsourcing options for our partners. This expansion supports Resilience’s mission to ensure adequate biomanufacturing capacity by addressing and overcoming historic manufacturing challenges throughout the industry." Source: https://lnkd.in/g_emuzhM

Industry reports 88 deals worth $11.1Billion in July quater In July 2024, the pharma and healthcare industry reported 88 deals worth $11.1 billion, compared to the last 12-month (July 2023 to June 2024) average of 95 deals worth $18.7 billion Eli Lilly and Company, to acquire Morphic Holding, a biopharmaceutical company developing a portfolio of oral integrin therapies for the treatment of serious chronic diseases for a consideration of approximately $3.2 billion; Mankind Pharma, to acquire Bharat Serums and Vaccines, a biopharmaceutical company that develops and manufactures pharmaceutical and injectable biological products, for an enterprise value of approximately $1.6 billion; Boehringer Ingelheim International GmbH, to acquire Nerio Therapeutics, a biotechnology company for a consideration of approximately $1.3 billion. These three major deals contributed 55 per cent of the total deal value during July 2024. The lifesciences sector reported 73 venture capital (VC) deals worth $2.5 billion in July 2024, compared to the last 12 months (July 2023 to June 2024) an average of 105 deals worth $2.7 billion. The three major VC deals reported in July 2024 were Cardurion Pharmaceuticals, a clinical-stage biotechnology company, raising $260 million in series B financing to advance Its pipeline of innovative medicines for cardiovascular diseases; Beacon Therapeutics, an ophthalmic gene therapy company, raising $170 million in a series B round to support the continued clinical development of AGTC-501 for XLRP and to generate Phase I/II clinical trial data for its dAMD programme; and Third Arc Bio, a biotech company, raising $165 million in series A financing to deliver superior biologics for solid tumours and inflammatory and; immunology diseases.

Oral peptides may become the main stay for keeping the body healthy after protein vs organ/disease mapping is accomplished. Fine tuning the protein interactions utilizing peptide defining the stable biological state may be complex, however, it may most probably be solved within the near future. In a new study, scientists from the laboratory of Professor Christian Heinis at EPFL have achieved a significant milestone in drug development. Their research opens the door to a new class of orally available drugs, addressing a long-standing challenge in the pharmaceutical industry. "There are many diseases for which the targets were identified but drugs binding and reaching them could not be developed," says Heinis. "Most of them are types of cancer, and many targets in these cancers are protein-protein interactions that are important for the tumor growth but cannot be inhibited." The study focused on cyclic peptides, which are versatile molecules known for their high affinity and specificity in binding challenging disease targets. To generate cyclic peptides that can target thrombin and are sufficiently stable, the scientists developed a two-step combinatorial synthesis strategy to synthesize a vast library of cyclical peptides with thioether bonds, which enhance their metabolic stability when taken orally. "We have now succeeded in generating cyclic peptides that bind to a disease target of our choice and can also be administered orally," says Heinis. "To this end, we have developed a new method in which thousands of small cyclic peptides with random sequences are chemically synthesized on a nanoscale and examined in a high-throughput process." These researchers was able to generate a comprehensive library of 8,448 cyclic peptides with an average molecular mass of about 650 Daltons (Da), only slightly above the maximum limit of 500 Da recommended for orally-available small molecules. The cyclic peptides also showed a high affinity for thrombin. When tested on rats, the peptides showed oral bioavailability up to 18%, which means that when the cyclic peptide drug is taken orally, 18% of it successfully enters the bloodstream compared 2% that is currently possible. In the next step of this project, the researchers will target several intracellular protein-protein interaction targets for which it has been difficult to develop inhibitors based on classical small molecules. They are confident that orally applicable cyclic peptides can be developed for at least some of them. #healthcarecosts #healthyaging #oralpeptides #proteins

Exciting Developments in Drug Approvals in China and the US in 2023! 🌟 In 2023, the China National Medicines Pharmaceutical Administration (NMPA) approved 87 novel drugs, with targeted drugs making up 67% of the total. 🇨🇳 Meanwhile, the FDA gave the green light to 55 innovative drugs, emphasizing personalized treatment options. 🇺🇸 Key Highlights: 🌿 NMPA's approval included domestic innovations like CAR-T cell products and monoclonal antibodies. 🎯 FDA's focus on targeted therapies showcased advancements in treating various diseases, with cancer therapies leading the way. 💡 Emerging therapies such as gene therapies and bispecific antibodies are offering new hope for patients.🚀💊 Source: https://lnkd.in/eX4Am8AQ #cartcelltherapy #innovationinmedicine #personalizedmedicine #medicaladvancements #healthcareindustry