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There were 224 cancer drug approvals across 119 individual drugs, with a median annual cost of $196 000 (IQR, $170 000-$277 000). Gene and viral therapies were the most expensive (median, $448 000 [IQR, $448 000-$479 000]), followed by small molecule therapy (median, $244 000 [IQR, $203 000-$321 000), and biologics (median, $185 000 [IQR, $148 000-$195 000]). There was no significant difference in cost between first-in-class, next-in-class, and subsequent approvals of an already approved drug. The results of this study suggest that drug pricing cannot be explained by innovations alone

Shanghai Medicilonic Inc. (Medicilon) and #JiangsuHengruiPharmaceuticals (Hengrui Pharma Co.,Ltd) have reached a strategic #collaboration agreement. This partnership will focus on #preclinical evaluation of #ADCs, #SmallNucleicAcids, and #CGT (cell and gene therapy) drugs, aiming to advance #Chinese #PharmaceuticalInnovation on a global scale. Building on years of trust, Medicilon's #Preclinical R&D expertise will complement Hengrui Pharma's innovative strategies. Together, they plan to provide a range of preclinical services, focusing on #EfficacyTesting, #Pharmacokinetics, and #Toxicology studies, to achieve #breakthroughs in pharmaceutical therapies. During a recent visit to Medicilon's Pudong Chuansha Park, Hengrui Pharma's executives expressed confidence in Medicilon's robust innovation capabilities. This collaboration not only acknowledges past achievements but also explores future collaboration potential. #Medicilon and #HengruiPharma believe that their #Partnership will accelerate drug #RandD and contribute to global pharmaceutical innovation. #Pharmaceuticals #DrugDevelopment #Innovation #Collaboration #ADC #CellAndGeneTherapy #Medicilon #HengruiPharma #China #APAC #RASLifeScienceSolutions #MarketIntelligence #MarketResearch #CompetitiveIntelligence #StrategySupport #PortfolioOptimization  Detailed News: https://lnkd.in/gpg2whRp Follow our page for more industry updates: https://lnkd.in/de5zNWmK

📢 Otsuka Pharmaceutical Co., Ltd. has acquired Jnana Therapeutics🚀 This acquisition has taken place for an upfront $800 million, with an additional $325 million in milestone payments and includes Jnana’s innovative drug discovery platform and a promising rare disease candidate. 🔬 About Jnana Therapeutics: Boston-based Jnana uses a unique approach to discover new drugs by targeting proteins within living cells. Their advanced platform has the potential to tackle challenging protein targets, including those involved in rare diseases. 🌟 Key Highlights: Rare Disease Focus: Jnana's lead candidate targets PKU, a genetic disorder. Early trials show promising results, with a significant reduction in disease symptoms. Strategic Fit: Otsuka sees Jnana’s platform as a perfect match to enhance their existing capabilities, especially complementing their subsidiary, Astex Pharmaceuticals 🌐 What’s Next? Jnana will continue its research and development as a wholly owned subsidiary of Otsuka, moving forward with its groundbreaking work on small molecule therapies. This acquisition is a big step forward in the fight against rare diseases and highlights the power of innovative biotech platforms. How do you think this acquisition will impact the future of drug discovery in the biotech industry? #Biotech #Pharmaceuticals #DrugDiscovery #RareDisease #OtsukaPharmaceutical #JnanaTherapeutics #MedicalAdvancements #PKU

RNAi-based drug design: considerations and future directions More than 25 years after its discovery, the post-transcriptional gene regulation mechanism termed RNAi is now transforming pharmaceutical development, proved by the recent FDA approval of multiple small interfering RNA (siRNA) drugs that target the liver. Synthetic siRNAs that trigger RNAi have the potential to specifically silence virtually any therapeutic target with unprecedented potency and durability. Bringing this innovative class of medicines to patients, however, has been riddled with substantial challenges, with delivery issues at the forefront. Several classes of siRNA drug are under clinical evaluation, but their utility in treating extrahepatic diseases remains limited, demanding continued innovation. In this Review, we discuss principal considerations and future directions in the design of therapeutic siRNAs, with a particular emphasis on chemistry, the application of informatics, delivery strategies and the importance of careful target selection, which together influence therapeutic success. https://lnkd.in/ecB46Cfr

In business news, Vertex Pharmaceuticals Incorporated and Alpine Immune Sciences, Inc., a biotechnology company focused on discovering and developing innovative, protein-based immunotherapies, announced that the companies have entered into a definitive agreement under which Vertex will acquire Alpine for $65 per share or approximately $4.9 billion in cash. Based upon an engineered TACI (transmembrane activator and CAML interactor) domain, Alpine’s lead molecule, povetacicept (ALPN-303), is a highly potent and effective dual antagonist of BAFF (B cell activating factor) and APRIL (a proliferation inducing ligand). Through Phase 2 development, povetacicept has shown potential best-in-class efficacy in IgA nephropathy (IgAN). IgAN is a serious, progressive, autoimmune disease of the kidney that can lead to end-stage-renal disease. There are no approved therapies that target the underlying cause of IgAN. IgAN is the most common cause of primary (idiopathic) glomerulonephritis worldwide, affecting approximately 130,000 people in the U.S. Povetacicept is on track to enter Phase 3 clinical development in the second half of 2024. https://lnkd.in/eTssfCFp

In our recent Pharma's Almanac #roundtable, we asked experts from across the life sciences industry to reflect on 2023's trends, events, and results. Minjee KIm, Market Intelligence Specialist at Samsung Biologics, commented on the success of weightloss drugs this year. With increasing demands to supply these therapeutics to patients suffering from obesity – a core risk factor of various cardiovascular diseases and diabetes – the global pharmaceutical market may have to collaborate with CDMOs for help meeting high volumes. Read Minjee's full response and hear from other experts in a #2023reflection in the full article online from Pharma's Almanac.

📘 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

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

Gene&Cell Therapy >> Agios sells rights to glioma drug to Royalty Pharma; Cargo’s $110M placement: Plus, news about Turn Biotechnologies, HanAll Biopharma, Corcept Therapeutics, Actuate and Merck: Agios sells vorasidenib rights to Royalty Pharma: As part of the agreement, the biotech will get a $905 million payout upon FDA approval of the glioma drug, which has a PDUFA date of Aug. 20. Agios can also still receive a $200 million milestone payment upon approval from Servier, which purchased Agios’ oncology portfolio, including vorasidenib, in a 2020 deal for $1.8 billion upfront. Agios’ shares $AGIO rallied about 22% on Tuesday morning. — Ayisha Sharma Cargo Therapeutics enters $110M private placement: The cancer-focused cell therapy company plans to use the proceeds to prep for a potential BLA for its Phase 2 candidate, CRG-022, and support IND-enabling studies for CRG-023. The financing should extend its cash runway through 2026. — Ayisha Sharma Turn Biotechnologies, HanAll Biopharma ink new deal: The agreement, valued at $300 million or more, focuses on age-related eye and ear conditions. Turn, an mRNA medicines startup, will use its technologies to restore gene expression. — Kyle LaHucik Corcept Therapeutics touts Ph3 success for hypercortisolism drug: The biotech’s selective cortisol modulator, relacorilant, significantly improved loss of blood pressure control in patients with the endocrine disorder, which is also known as Cushing’s disease. The company’s shares $CORT were up around 12% on Tuesday morning. — Ayisha Sharma Cancer biotech files for IPO: Actuate, founded in 2015, plans to debut on the Nasdaq as “ACTU.” Its lead asset, elraglusib, is being tested in multiple adult and pediatric cancers. The biotech is also studying injectable and oral tablet formulations of the drug, which stems from research out of the University of Illinois Chicago and Northwestern University. Actuate joins Telix, Aprinoia and Rapport in seeking a Nasdaq listing this year. — Kyle LaHucik Merck’s Keytruda boosts breast cancer survival: The anti-PD-L1 drug combined with chemotherapy significantly improved overall survival in a Phase 3 trial of patients with high-risk early-stage triple-negative breast cancer. Merck plans to present the results at an upcoming medical meeting and share them with regulatory authorities. — Ayisha Sharma #lucidquest #genetherapy #celltherapy

siRNA all the way!!! Here are 8 #biotech companies that are leading the way in siRNA - pipeline info in the link below. 1. Alnylam Pharmaceuticals 2. Aro Biotherapeutics 3. Arrowhead Pharmaceuticals 4. Novo Nordisk (Dicerna Pharmaceuticals, Inc.) 5. DTx Pharma, A Novartis Company 6. Eleven Therapeutics 7. Switch Therapeutics 8. Wave Life Sciences The market for siRNA therapeutics is expected to grow from $12.7 billion to $39.2 billion by 2029. Here are eight companies contributing to the field of siRNA. https://lnkd.in/eHH-_UAs #siRNA #RNA #biopharma #vc #economics #drugdevelopment