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Sequana Medical NV has announced the US FDA approval of its Alfapump system for treating recurrent or refractory ascites due to liver cirrhosis.  This approval marks Alfapump as the first active implantable medical device for this condition in the US. Professor H.E. Vargas of the Mayo Clinic expressed the significance of this approval: “The FDA approval of alfapump opens the door to a breakthrough therapeutic option for patients with ascites. This technology not only provides effective treatment but also improves quality of life.” The company’s CEO, Ian Crosbie, also emphasised the milestone’s importance: “Today marks a huge milestone for the liver ascites community. Our vision is to transform the lives of these patients with a solution that allows them to take back their lives.” The Alfapump system is designed to automatically and continuously move ascites from the abdomen to the bladder, eliminating the need for therapeutic paracentesis.  The POSEIDON study showed significant improvements in patient quality of life with the Alfapump system. Sequana Medical plans to launch the Alfapump system commercially in the US in the second half of 2025, targeting liver transplant centers with a specialty sales force.  The company expects a market opportunity exceeding $2 billion by 2025, driven by conditions like NASH/MASH and alcoholic liver disease. Read more: https://lnkd.in/eaZJ7ER5 Stay in touch with all the leading stories, events and opportunities by subscribing to my weekly LinkedIn Newsletter here:  https://bit.ly/3KQs0mD

Amgen’s Imdyllytra (tarlatamab) has been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) to treat adults with small cell lung cancer (SCLC). The drug has been specifically authorised for use in patients whose disease has spread throughout the lungs, to other parts of the body, or both, and who have failed two other types of treatments. SCLC, one of the two main forms of primary lung cancer, accounts for approximately 15% of all lung cancer diagnoses and progresses rapidly. Imdyllytra is administered as an intravenous infusion and belongs to a class of drugs called antineoplastic agents, which are designed to kill cancer cells that rapidly divide. The MHRA’s decision was supported by positive results from the mid-stage DeLLphi-301 trial, which evaluated Amgen’s drug in patients with extensive-stage SCLC who had failed two or more prior lines of chemotherapy Imdyllytra demonstrated an overall response rate of 40% and a median duration of response of 9.7 months, which the UK regulator said was “encouraging in patients with advanced SCLC who have limited treatment options and for whom there is a high unmet need for effective treatment options”. Julian Beach, MHRA interim executive director of healthcare quality and access, said: “We’re assured that the appropriate regulatory standards of safety, quality and efficacy for the approval of [Imdyllytra] have been met. As with all products, we will keep its safety under close review.” The decision comes after the US Food and Drug Administration approved the drug for SCLC under the brand name Imdelltra in May. Both authorisations were made through Project Orbis, a global partnership between regulatory bodies to review and approve promising cancer drugs. Read more: https://lnkd.in/ec6DVcWS Stay in touch with all the leading stories, events and opportunities by subscribing to my weekly LinkedIn Newsletter here:  https://bit.ly/3KQs0mD

The U.S. Food and Drug Administration said on Friday that it has approved an injectable version of Bristol Myers Squibb's blockbuster cancer drug, Opdivo. Opdivo is part of a class of drugs called PD-1 inhibitors, which enhance the immune system's ability to fight cancer by removing its natural brakes. Like other PD-1 drugs such as Merck's Keytruda, it was previously available through infusions and patients received it via an intravenous drip in a health office. The new injectable form is expected to be more convenient for patients and could help shield the company from erosion of sales when the patent for the intravenous version expires later this decade. The injection, branded as Opdivo Qvantig, has been approved to treat all previously approved adult, solid tumor indications, either on its own, as maintenance therapy or in combination with chemotherapy. The drug will be available in early January, and will be priced at parity with the list price of the IV version, Adam Lenkowsky, Bristol's chief commercialization officer, told Reuters ahead of the approval. The IV version of the drug has a list price of $7,635 per infusion for two weeks for the lower dose and $15,269 per infusion for four weeks for the higher 480-milligram dose. The approval was based on data from a late-stage study, which showed that the subcutaneous form of the drug was not inferior to the intravenous formulation in patients with advanced kidney cancer who have received prior systemic therapy. The drugmaker is relying on newer treatments like Opdivo Qvantig to drive growth as patents on older drugs, such as cancer drug Revlimid and blood thinner Eliquis, expire later this decade. Read more: https://lnkd.in/eZTniYyk

Novo Nordisk plans to invest $1.2 billion to build a production facility in Odense, Denmark, focusing on therapies for rare diseases, including haemophilia. Plus other news and developments form the pharmaceutical and medical world this week.

MindMed has announced the dosing of the first patient in its phase 3 Voyage study of MM120 ODT, a pharmaceutically optimised form of lysergide D-tartrate (LSD) for treating Generalised Anxiety Disorder (GAD).  The study aims to evaluate the efficacy and safety of MM120 ODT compared to placebo, with approximately 200 participants expected to enrol in the United States. “Today marks a pivotal moment in our journey towards advancing a novel treatment option for the 20 million people in the US living with GAD,” said Dr Daniel R. Karlin, Chief Medical Officer of MindMed.  He emphasised that the phase 3 studies are built on rigorous phase 2b results, which showed efficacy exceeding current standards of care and a favourable tolerability profile. The 52-week Voyage study consists of two parts: a 12-week randomised, double-blind, placebo-controlled period, followed by a 40-week extension with open-label treatment based on symptom severity.  The primary endpoint will measure the change from baseline in the Hamilton Anxiety Rating Scale (HAM-A) at Week 12, consistent with the phase 2b study’s observed durable clinical effect. “It is critical to continue to develop new and effective treatment options for patients with GAD, a debilitating condition where there is an urgent need for transformational innovation,” said Dr David Feifel, an investigator in the Voyage study.  The study design includes best-in-class methodologies to mitigate unblinding and isolate the standalone drug effect of MM120 ODT. GAD affects around 20 million adults in the US, causing significant personal and societal burdens. Despite this, there has been little innovation in treatment over the past decades.  MindMed’s MM120 ODT offers a novel approach, leveraging rapid absorption and improved bioavailability to enhance therapeutic outcomes. Read more: https://lnkd.in/eDs8x_m9

AbbVie has shared promising top-line results from a phase 3 study of its investigational Parkinson’s disease (PD) candidate tavapadon in adults and said it is “on track” to submit a new drug application to the US Food and Drug Administration in 2025. The TEMPO-2 trial evaluated the D1/D5 dopamine receptor partial agonist as a flexible-dose monotherapy in patients with early stages of the neurodegenerative disorder. More than ten million people worldwide are living with PD, which is characterised by symptoms such as tremor, muscle rigidity, slowness of movement and difficulty with balance. TEMPO-2 met its primary endpoint, with tavapadon-treated patients experiencing a statistically significant improvement from baseline in the Movement Disorder Society – Unified Parkinson’s Disease Rating Scale parts two and three combined score compared to placebo at week 26. The study’s key secondary endpoint was also met, demonstrating a statistically significant and clinically meaningful improvement in motor aspects of experiences of daily living in the tavapadon group compared to placebo at week 26, and the drug’s safety profile was consistent with prior clinical trials. The update builds on positive top-line results from TEMPO-1, which evaluated two fixed doses of tavapadon, as well as the TEMPO-3 of the drug as an adjunctive therapy to levodopa, which is often viewed as the first-line drug for the management of PD motor symptoms. AbbVie is also conducting an open-label extension trial, TEMPO-4, to assess the long-term safety and tolerability of the candidate. Read more: https://lnkd.in/ey7CmS8F Stay in touch with all the leading stories, events and opportunities by subscribing to my weekly LinkedIn Newsletter here:  https://bit.ly/3KQs0mD

Intermittent fasting has proven benefits for metabolic health, but a new study shows that it could slow hair growth—at least in mice. Researchers report December 13 in the journal Cell that mice subjected to intermittent fasting regimes showed improved metabolic health but slower hair regeneration compared to mice with 24/7 access to food. A similar process might occur in humans, based on a small clinical trial that the team also conducted, but it's likely to be less severe since humans have a much slower metabolic rate and different hair growth patterns compared to mice. "We don't want to scare people away from practicing intermittent fasting because it is associated with a lot of beneficial effects—it's just important to be aware that it might have some unintended effects," says senior author and stem cell biologist Bing Zhang of Westlake University in Zhejiang, China. In addition to its metabolic benefits, previous studies have shown that fasting can improve the stress resistance of stem cells associated with blood, intestinal, and muscle tissue, but little is known about how it impacts peripheral tissues such as skin and hair. Zhang's team hypothesized that fasting might also be beneficial for skin tissue regeneration, the process by which old and damaged cells are replaced. To test this, they examined hair regrowth in mice that were shaved and then subjected to different intermittent fasting regimes. Some mice were fed on a time-restricted feeding (TRF) schedule that involved eight hours of food access and 16 hours of fasting each day, while other mice were subjected to alternate-day feeding (ADF). They were surprised to find that fasting inhibited hair regeneration. While control mice that had unlimited access to food had regrown most of their hair after 30 days, mice on both intermittent fasting regimes showed only partial hair regrowth after 96 days. The team showed that this inhibited hair growth occurs because hair follicle stem cells (HFSCs) are unable to cope with the oxidative stress associated with switching from using glucose to fat. HFSCs go through phases of activity and dormancy, and hair regrowth depends on these cells becoming active. While the control mice's HFSCs began to become activated around day 20 post-shaving and remained active until their hair had regrown, the intermittent fasting mice's activated HFSCs underwent apoptosis (programmed cell death) during extended fasting periods. Read more: https://lnkd.in/ggNAhbqD Stay in touch with all the leading stories, events and opportunities by subscribing to my weekly LinkedIn Newsletter here:  https://bit.ly/3KQs0mD

Eli Lilly has announced a $3bn expansion to the US manufacturing facility that it acquired from Nexus Pharmaceuticals earlier this year. The investment in Kenosha County, Wisconsin is aimed at extending the company’s global injectable product manufacturing network to help it meet the growing demand for its diabetes and obesity medicines, as well as future pipeline drugs across multiple therapeutic areas. The expanded facility, which brings Lilly’s total planned investment in Wisconsin to $4bn, will focus on manufacturing injectable medicines, device assembly and packaging. Lilly said it will use advanced automation, including guided vehicles, robotics and production equipment, throughout the site to accelerate processes and increase accuracy, which the company said will allow employees to “focus on making safe, high-quality medicines”. Construction on the expansion is expected to begin next year and, once complete, the facility is set to add 750 highly skilled jobs to the current 100-plus workforce at the Wisconsin location. The commitment represents Lilly’s single largest US manufacturing investment outside its home state of Indiana, explained Edgardo Hernandez, executive vice president and president of Lilly manufacturing operations. “We look forward to bringing high-wage, advanced manufacturing, engineering and science jobs to people in Wisconsin, a state that is becoming a critical geography in our global manufacturing operations,” he said. Lilly first announced its acquisition of the manufacturing facility from Nexus for an undisclosed sum in April. The site does not provide contract manufacturing services, meaning it is solely dedicated to Lilly’s manufacturing efforts. Read more: https://lnkd.in/gmFXdx92 Stay in touch with all the leading stories, events and opportunities by subscribing to my weekly LinkedIn Newsletter here:  https://bit.ly/3KQs0mD

Eli Lilly is taking an innovative step to combat competition from legal copycats of its weight-loss drug, Zepbound, by partnering with Ro, a telemedicine company. Zepbound, a form of tirzepatide, is part of the popular class of GLP-1 receptor agonists originally developed to treat type 2 diabetes but now widely used for weight loss. The immense demand for these medications has opened the door for compounding pharmacies, which legally create customized versions of the drugs, often at a significantly lower price. These compounded alternatives, while cheaper, do not always adhere to the same safety and efficacy standards as FDA-approved drugs. To address this competition, Eli Lilly has decided to distribute Zepbound through Ro’s online telemedicine platform. Patients can now obtain single-dose vials of the drug via prescriptions made through Ro, with the orders fulfilled by LillyDirect, Eli Lilly’s direct-to-consumer service. This move is designed to make Zepbound more accessible and competitively priced compared to its standard retail packaging, although it may still be more expensive than the compounded versions. The decision highlights the growing importance of telemedicine in pharmaceutical distribution, especially for high-demand drugs like GLP-1 agonists. It also reflects Eli Lilly’s strategic effort to secure its position in a market increasingly influenced by cheaper alternatives from compounding pharmacies. The competition between major pharmaceutical companies and these compounders is expected to intensify, potentially drawing further regulatory scrutiny on compounded drug practices. This development could set a precedent for how pharmaceutical companies address similar challenges in the future, emphasizing the importance of direct consumer access and competitive pricing strategies. As Eli Lilly expands its initiatives, the industry may see more partnerships and innovations aimed at navigating the complex dynamics of cost, accessibility, and safety in the pharmaceutical market. If desired, I can explore further aspects of this story, including the broader implications for the weight-loss drug market or the ethical debates surrounding compounding pharmacies.