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AbbVie buys into PD-1/VEGF sweepstakes; Alnylam and Vertex set expectations

Today, a brief rundown of news announced ahead of the J.P. Morgan Healthcare Conference, including licensing deals from AbbVie and Novartis, earnings announcements from Vertex Pharmaceuticals and Alnylam Pharmaceuticals, and regulatory updates from Beam Therap

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Today, a brief rundown of news announced ahead of the J.P. Morgan Healthcare Conference, including licensing deals from AbbVie and Novartis, earnings announcements from Vertex Pharmaceuticals and Alnylam Pharmaceuticals, and regulatory updates from Beam Therapeutics and Atara Biotherapeutics. AbbVie is paying Yantai, China-based RemeGen $650 million up front , and potentially more than $5 billion overall, for a bispecific antibody drug targeting the proteins PD-1 and VEGF . The deal announced Monday hands AbbVie rights to develop, manufacture and commercialize RemeGen’s RC148 outside of China and nearby territories. The drug being evaluated in a Phase 1 lung cancer study and Phase 2 trials in other solid tumors and breast cancer. Some of those mid-stage studies are evaluating RC148 alongside an antibody-drug conjugate Pfizer gained rights to through a buyout of Seagen. That ADC is approved in China, as is an autoimmune drug RemeGen licensed to Vor Biopharma last year. — Jonathan Gardner Ahead of a Monday presentation at the J.P. Morgan Healthcare Conference, Alnylam Pharmaceuticals on Sunday pre-announced fourth quarter results and issued revenue guidance for the coming year. Alnylam said its closely watched transthyretin amyloidosis drug Amvuttra generated $827 million between October and December, which missed consensus estimates by more than $20 million. But the $4.4 billion to $4.7 billion in projected 2026 net revenue from its TTR drugs — which include Amvuttra as well as an older medication, Onpattro — came in ahead of analysts’ projections. The numbers were issued alongside a new five-year strategy that involves hitting at least a 25% average annual growth rate through the end of 2030. Shares ticked down about 8%. — Ben Fidler Vertex Pharmaceuticals also issued a business update that included insight into the recent sales performance for its pain drug Journavx . According to Vertex, more than 500,000 prescriptions have been filled since Journavx’s approval in January, and the company expects that number to more than triple in the coming year. Vertex has also secured coverage in acute pain with all of the large national pharmacy benefit managers. The updates suggest “steady growth” but imply fourth-quarter sales could come in slightly below consensus estimates of $33 million, wrote RBC Capital Markets analyst Brian Abrahams. Vertex also pre-reported $100 million in revenue for its gene editing treatment Casgevy. That number would come in slightly ahead of Wall Street projections, but the program “remains of low investor interest,” Abrahams wrote. — Ben Fidler Beam Therapeutics , meanwhile, said that it's aligned with U.S. regulators on a potential accelerated approval submission for one of its base editing therapies. Beam is testing the therapy in people with the rare lung disease alpha-1 antitrypsin deficiency and, last year, delivered early results suggesting the treatment could correct the disease-causing mutation underlying the condition. On Sunday, Beam said that the Food and Drug Administration could clear the drug, BEAM-302, based on positive effects on surrogate markers over the course of a year. Beam expects to enroll about 50 more patients in an ongoing trial and treat them with the "optimal" dose, and will report updated results, as well as next steps for the program, by the end of the first quarter. Beam additionally anticipates seeking approval of a sickle cell therapy, risto-cel, later this year. Company shares climbed more than 25%. — Ben Fidler Novartis is paying $165 million in upfront cash to license an experimental Alzheimer’s disease treatment from SciNeuro Pharmaceuticals . The drug is designed to remove toxic amyloid deposits from the brains of people with Alzheimer’s, but with the help of a “shuttle” technology that helps the medicine cross the blood-brain barrier. Mulitple other companies, including Roche , are evaluating similar approaches . SciNeuro, which recently raised $53 million in venture funding, could receive another $1.5 billion in the deal. — Jonathan Gardner The FDA has again rejected a cell therapy from Atara Biotherapeutics , the company said Monday. A year ago, the agency cited manufacturing issues in turning back Atara’s request to approve Ebvallo for a type of post-transplant disease associated with Epstein Barr virus infections. Now, however, Atara said that the agency has reversed its previous position and claimed that the single-arm study supporting the company’s application is no longer sufficient to support an approval. Atara had previously aligned with FDA leaders on the study’s design through “multiple, documented meetings” over the last five years. Partner Pierre Fabre Pharmaceuticals , which holds commercial rights to Ebvallo, intends to meet with the FDA to find a path forward. Atara shares fell by more than 50%. — Delilah Alvarado

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01Lifestyle 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 ↗
02What 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 ↗
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 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 ↗
05Why 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 ↗
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Peptide Therapy Guide Editorial Team

Editorial team for Peptide Therapy Guide.

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