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Høeg fired in latest FDA shakeup; 20 people die after taking Amgen drug

Today, a brief rundown of news involving the Food and Drug Administration and Amgen, as well as updates from Vincentage Pharma and Parabilis Medicines that you may have missed. The Food and Drug Administration has again shaken up its leadership ranks, as the a

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Today, a brief rundown of news involving the Food and Drug Administration and Amgen, as well as updates from Vincentage Pharma and Parabilis Medicines that you may have missed. The Food and Drug Administration has again shaken up its leadership ranks, as the acting directors of two main drug review offices, Tracy Beth Høeg and Katherine Szarama , have left the agency following the sudden resignation of commissioner Marty Makary . Høeg posted on the social media platform X that she was fired. According to Reuters , the move was part of a broader overhaul meant to place more traditional selections in top FDA positions. The regulator’s website now lists Michael Davis , a former agency deputy center director, as the acting head of the CDER office that regulates drugs and certain biologics. Karim Mikhail , the one-time leader of biotech Amarin , is now serving as the acting director of the CBER office in charge of vaccines and gene therapies. — Ben Fidler Twenty people in Japan have died after receiving Amgen’s rare disease drug Tavneos , the biotech’s partner Kissei Pharmaceutical revealed Friday . Kissei, which sells the medicine in Japan, said that the majority of the deaths were attributed to a condition known as vanishing bile duct syndrome . It’s warned doctors there to not prescribe Tavneos to new patients. The FDA has already asked Amgen to withdraw Tavneos in the U.S. due to safety concerns and an alleged manipulation of study data by the drug’s original developer, ChemoCentryx . It will remain on the market in the U.S. until Amgen chooses to remove it or is forced to do so by the FDA. — Ben Fidler An experimental oral obesity medicine from Vincentage Pharma succeeded in a late-stage study in China , positioning the company to seek approval there. According to Vincentage, treatment with its therapy VCT220 was associated with roughly 12% weight loss over a year, versus about a 1% loss for placebo recipients. Some 1.8% of participants receiving either low or high doses dropped out of the trial due to side effects, which Vincentage described as “generally mild to moderate” and consistent with what’s been seen with other GLP-1 medications. Startup Corxel Pharmaceuticals , which recently raised a $287 million venture financing , owns most rights to VCT220 and is running a global Phase 2 study. Results are expected by the end of 2026. — Ben Fidler Regeneron has inked a deal with privately held cancer drugmaker Parabilis Medicines to develop specialized medicines the startup refers to as “antibody-helicon conjugates.” Like antibody-drug conjugates, these medicines link a targeting molecule to a drug payload. But Parabilis’ therapies deliver a type of peptide the company claims can affect intracellular proteins, including those long viewed as “undruggable” by conventional methods. Regeneron is handing Parabilis $50 million up front, investing $75 million in the company’s next equity financing, and could shell out up to $2.2 billion in total downstream payments, according to a Monday announcement . — Gwendolyn Wu

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01What the Artificial Neuron Cannot Do

Hersam’s next goal is a small circuit — perhaps 10 artificial neurons — where each one fires differently, and together they accomplish what would require thousands of conventional transistors. “ Silicon achieves complexity by having billions of identical devices,” Hersam said. “The brain is the opposite. It’s heterogeneous. The complexity is at the device level.” But Gaudet sees a gap no circuit design can yet fill: Biological neurons grow new connections and prune old ones, strengthening pathways that are used and weakening those that aren’t. Hersam’s lab’s printed neurons — or any other neuromorphic technology that mimics neuronal dynamics — can’t achieve that level of complexity yet. Brown is careful about the distance remaining between these printed neurons and the real thing. “Neurons are just so flexible,” he said. “They can totally change what they’re doing based on whether they’ve learned something and based on your emotional state. There’s a lot of hidden mysteries.” Sangwan suspects the device has more to reveal. “It’s a nonlinear dynamical system,” he said. “We don’t fully know how many different variables you need to explain it. It’s just the beginning.” Hersam, Sangwan, Brown, Holla, and Gaudet reported having no relevant financial disclosures. Disclosure information for study authors is available in the original study publication.

Source: www.medscape.com ↗
02Why Muscle Cells Might Do Some Heavy Lifting

Brown was studying gene therapy in the 1990s when he designed a technology to turn mRNA expression on or off in different cells. For the new mouse study, published in Nature Biotechnology , he adapted the technology to turn off mRNA expression in dendritic cells, muscle cells, or liver cells. The researchers then vaccinated the mice with each version, delivering the vaccines both intravenously and intramuscularly. “The results were pretty stunning,” Brown said. When mRNA expression was turned off in muscle cells, T-cell response went down, suggesting muscle cells play a role in immunity. When expression was turned off in liver cells, T-cell expression tripled — indicating liver cells dampen immunity. Turning off expression in dendritic cells had no effect on T-cell activation, though it did reduce the number of killer T cells by as much as half. (Interestingly, no such reduction occurred when the antigen was SARS-CoV-2 spike. Brown is now investigating why different antigens had varying effects.) Knowing all this is crucial for designing effective mRNA vaccines and therapies. That’s because different mRNA therapies require different strategies. Cancer vaccines must boost tumor-fighting killer (CD8+) T cells. For genetic disease treatments, scientists want to avoid triggering the immune system to prevent killing the very cells the mRNA is meant to modify. “Understanding the immunology is extremely important for this class of drug,” Brown said. The finding doesn’t mean dendritic cells aren’t important for mRNA vaccines to work. “It just means that the mRNA doesn’t have to get into those cells to induce an immune response,” Brown said. Instead, the antigen can be transferred to those dendritic cells.

Source: www.medscape.com ↗
03China: Threat or opportunity?

One of the biggest biotech news stories of recent years is China’s continued rise as a biotech and life sciences powerhouse. China conducts a quarter of all clinical trials and drug development and has almost 1,500 new drugs in development.¹ Many China-based biotechs have benefitted from government funds, out-licencing deals with large pharmas and venture capital funding. However, policymakers in the US and EU have concerns about the possible threat to their region’s biosecurity and competitiveness as centres for health and life science research. Given China’s increased importance, ICON Biotech conducted the same biotech sector survey with 100 China-based biotech leaders. The results show that Chinese biotechs face many of the same challenges as biotechs located elsewhere. They share the same funding challenges and burdens associated with increasingly complex clinical trials and regulations.

Source: www.biopharmadive.com ↗
04What Comes Next

With data expected in the fourth quarter of 2026, we are prioritizing histology alongside patient-reported outcomes using the Celiac Disease Symptom Diary, one of only two instruments developed in line with U.S. Food and Drug Administration (FDA) guidance, to capture changes in symptoms such as abdominal pain and nausea. Ultimately, the broader aim is to give gastroenterologists and patients a therapeutic option for a disease that has long been managed without one. The future of drug development will not be defined by statistical significance alone, but by whether new therapies also improve the daily burden of living with celiac disease. “The first therapy to cross the line could change the field,” Geller concluded. “It would help establish celiac as a serious medical condition with options beyond a restrictive diet and open the door for what comes next.” Dr. Paul Lizzul is chief medical officer at First Tracks Biotherapeutics, a clinical ‑ stage biotechnology company advancing antibody therapeutics that modulate immune pathways implicated in autoimmune and inflammatory diseases. Marilyn Geller serves as an advisor to First Tracks Bio. Footnotes Abadie V, Jabri B. IL-15: a central regulator of celiac disease immunopathology. Immunol Rev . 2014;260(1):221-234. https://doi.org/10.1111/imr.12191. Yokoyama S, Watanabe N, Sato N, et al. Antibody-mediated blockade of IL-15 reverses the autoimmune intestinal damage in transgenic mice that overexpress IL-15 in enterocytes. Proc Natl Acad Sci U S A . 2009;106(37):15849-15854. https://doi/full/10.1073/pnas.0908834106. Anthony S, Schluns KS. Emerging roles for IL-15 in the activation and function of T-cells during immune stimulation. Research and Reports in Biology . 2015;6:25-37. https://doi.org/10.2147/RRB.S57685.

Source: www.biopharmadive.com ↗
05Lifestyle Matters: How do environmental and lifestyle factors influence Alzheimer’s disease?

Dr. Harrison and Finnish neuroscientist Dr. Miia Kivipelto explore the complex interplay between genetics and lifestyle in Alzheimer's development. Learn how the groundbreaking FINGER study demonstrates potential prevention strategies, and discover the latest evidence on how environmental factors, diet, and chronic conditions influence Alzheimer's risk.

Source: www.biopharmadive.com ↗
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Peptide Therapy Guide Editorial Team

Editorial team for Peptide Therapy Guide.

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