Asthma

Hello and welcome back to another edition of Endpoints Weekly! We’ve got a full cadre of reporters in Berlin this weekend for the annual ESMO meeting, so make sure to keep checking back online for updates out of the conference. We’ll also have a rundown of the biggest stories in Monday’s newsletters. But let’s get into this week’s headlines first! Founding editor John Carroll took an in-depth look at the emerging trends from biotech’s third quarter. His two-part analysis (and subsequent appearance on Post-Hoc Live) showed how dealmaking seems to be making a comeback in the industry. And who were biopharma’s busiest BD teams? Paid subscribers can find out below. Elsewhere this week, we had a fascinating Q&A with Nobel Prize winner Fred Ramsdell, who made mainstream headlines for being on an off-the-grid vacation when the news broke. We also covered the launch of a new biotech’s radical plan for allergies, Jim Wilson’s outlook on rare disease gene therapy and a massive raise for a GLP-1 startup. I also took a look at the landscape for PD-1xVEGF bispecifics and whether they’re being tested in the right kinds of cancer. Take a read if you haven’t already , and have a good weekend! — Max Gelman 🤝 With the XBI hitting $100 for the first time in a while last month, John took a look at some of the forces driving the uptick in investor enthusiasm, with an assist from DealForma’s Chris Dokomajilar. Not only was there an increase in dealmaking and M&A, but venture investing also picked up in Q3 after a bleak second quarter. The first part of John’s analysis goes deeper into the “why” of it all. Later in the week, John examined which big companies were angling for deals and noted some surprises. For example, John writes, even though Merck and Bristol Myers Squibb get lots of attention for their deals, neither comes close to the top 5 in licensing deals among peer companies. Paid subscribers can take a closer look at the story being told by the numbers. John was also joined by Stifel’s Tim Opler on Post-Hoc Live to discuss whether or not the good times in biotech are really, truly back . You can watch the conversation on YouTube or listen to it on Spotify or Apple Podcasts. 🏔️ Fred Ramsdell was backpacking near Yellowstone National Park when he missed the phone call to tell him he won the Nobel Prize in Physiology or Medicine. From the snow-covered mountains of Montana, he spoke with Endpoints News’ Lei Lei Wu about winning the Nobel, cell therapy for autoimmune diseases, and the evolving state of basic science research. Ramsdell, Mary Brunkow and Shimon Sakaguchi won the prize for their research on regulatory T cells, the cells that keep the immune system under control. When asked about the current state of the cell therapy field, Ramsdell compared the innovation to cell phones , noting that “they’re too expensive, but no one’s going to make these things cheap and universally available — until they work.” “Think of the first cell phone, right? This thing that looked like a brick with a big antenna on it,” he said. “But without that, you’re not getting an iPhone.” Read the full conversation here. A new startup believes it’s found a way to revolutionize treatment for allergic and immunologic diseases. The company, called Excellergy, debuted this week with a $70 million Series A and a strategy to take on a longtime power player in those spaces: Roche and Novartis’ Xolair. Excellergy calls its drugs “effector cell response inhibitors” that are designed to interfere with the body’s allergic response and tamp down on inflammation. Read more from an exclusive interview we conducted with co-founder Alexander Eggel about how the company’s science works. Excellergy hasn’t yet decided which diseases it wants to test first for its lead program, but food allergy and chronic hives are currently the top two contenders. Other areas could include certain subtypes of asthma and additional immunologic diseases. 🧬Gene therapy pioneer Jim Wilson has struggled to convince investors to go in on treatments for ultra-rare genetic diseases. So his startup GEMMABio is pivoting to focus on several earlier-stage programs that target more common diseases. But that doesn’t mean Wilson is giving up on ultra-rare diseases. The company is separating three clinical programs for ultra-rare diseases and putting them into a new company called Rare Therapeutics. You can get the details here. “We repositioned GEMMA to focus on those three larger market indications,” he said. “That would then enable us to finance GEMMA. But that’s not why I got into GEMMA — I didn’t get into GEMMA to deprioritize ultra-orphan diseases.” Kailera Therapeutics, a closely watched private obesity biotech, raised $600 million to begin Phase 3 trials. The megaround ties Kailera with Isomorphic Labs for the largest biotech fundraise of the year. The Waltham, MA-based biotech said it will use the new funds, in part, to start two Phase 3 trials of KAI-9531, the injectable GLP-1/GIP receptor agonist it in-licensed from Hengrui Pharma.

By Siddhi Mahatole and Sneha S K Bengaluru: The U.S. Food and Drug Administration has approved Amgen and AstraZeneca 's drug for a type of chronic inflammatory sinus disease , the drugmakers said on Friday. The approval expands the use of the drug, Tezspire, as an add-on maintenance treatment of inadequately controlled chronic rhinosinusitis with nasal polyps in adult and pediatric patients aged 12 years and older. The condition causes the sinuses to stay inflamed for 12 weeks or more and soft, noncancerous growths called polyps to form in the nose. Symptoms include facial pain, reduced sense of smell and nasal congestion. Tezspire is already approved as a single-use pre-filled syringe in the U.S., EU and other countries for add-on maintenance treatment for severe asthma. The approval is based on the results from a late-stage study in which the drug showed clinically meaningful reduction in the size of nasal polyps and reduced nasal congestion compared to placebo. Tezspire also reduced the number of patients needing surgery for nasal polyps by 98% and reduced the need for oral steroids by 88%, the data showed. Data for Tezspire shows that it might be more effective than Sanofi and Regeneron's Dupixent for patients with this condition, William Blair analyst Matt Phipps said ahead of the approval. Tezspire, chemically known as tezepelumab-ekko, is a monoclonal antibody that blocks TSLP - a key protein involved in triggering and sustaining inflammation linked to severe asthma and related conditions. The approval "shows the versatility of TSLP inhibition beyond asthma and highlights both companies' commitment to take a really important scientific insight and way to treat patients into broad application that can make a real difference for patients," said Kate Chevlen, global commercial head of inflammation portfolio at Amgen. Other approved treatments for chronic rhinosinusitis include Dupixent, GSK's Nucala as well as Roche and Novartis' Xolair. (Reporting by Siddhi Mahatole and Sneha S K in Bengaluru; Editing by Sahal Muhammed and Shreya Biswas)

The FDA has approved Amgen and AstraZeneca’s Tezspire for patients age 12 and older with chronic rhinosinusitis with nasal polyps. Six years since the FDA blessed Sanofi and Regeneron’s Dupixent as the first biologic to treat chronic rhinosinusitis with nasal polyps (CRSwNP), a second biologic therapy has broken through with a nod in the indication.The U.S. regulator has endorsed Amgen and AstraZeneca’s Tezspire as an add-on maintenance treatment for patients age 12 and older. The first-in-class monoclonal antibody, which is injected monthly, inhibits the action of thymic stromal lymphopoietin (TSLP), a key epithelial cytokine which triggers inflammation.It’s this novel mechanism of action that keys the effectiveness of Tezspire, which has produced clinical results that suggest it could become the top product on the market for CRSwNP.“Tezspire is the only biologic that is able to target these cytokines upstream, next to the epithelium, and that gives you unique and broad coverage of that inflammation,” Pablo Panella, AstraZeneca’s senior vice president for global respiratory and immunology, said in an interview with Fierce Pharma.This is the second indication for Tezspire, which was originally approved in 2021 as an add-on maintenance treatment for severe asthma. It has had a major impact on the asthma market, achieving sales of $1.2 billion in 2024 and on its way to a significant boost in 2025, with sales reaching $826 million in the first half of the year.Although CRSwNP is not as prevalent a disorder as asthma, it still presents a substantial commercial opportunity because of the unmet need in the indication, according to Kate Tansey Chevlen, who heads up Amgen’s commercial efforts for its inflammation products.“It’s mostly managed with intranasal steroids and surgery, so there’s really an opportunity to offer patients more and to provide more lasting relief,” Chevlen said. “We think there’s a significant market for biologics in this space because patients need more than what they’re getting today.” Backing the label expansion were results from a phase 3 trial in which patients were treated for 52 weeks. The study achieved its two primary endpoints, showing significant improvements for Tezspire over placebo in total nasal polyp score (NCS) and nasal congestion score (NCS).On NPS, a metric with a range of 0 to 8, treatment with Tezspire led to a placebo-adjusted average improvement of 2.08 points. On NCS, which is scored between 0 and 3, Tezspire performed 1.04 points better than placebo.These figures compare favorably to those from a trial that facilitated Dupixent’s approval in the indication, with the usual caveat that cross-trial comparisons are often problematic because of design differences in the studies. Dupixent’s NPS improvement on placebo was an average of 1.8 points, and its difference versus placebo in NCS was 0.9.Tezspire also fared better than GSK’s investigative oral treatment depemokimab, which recorded a 0.7 difference versus placebo in NPS. Depemokimab is lined up for an FDA decision in the indication in December of this year. One key secondary endpoint from Tezspire’s trial was that it reduced the need for polyp surgery by 98%.“It’s a near complete elimination of surgery, which is a very aggressive procedure for these patients. It’s extremely encouraging,” Panella said. “And we think the unique mechanism of action is responsible for that.”CRSwNP affects roughly 320 million in the world, including 1 million to 2 million in the U.S. Of those, the companies believe there are roughly 300,000 patients who have need for a biologic treatment, but only about 40% of that group are currently taking them.