Stylus Medicine, Inc.

Welcome back to Endpoints Weekly! Before we get into this week’s headlines, here’s a holiday programming note: We won’t be sending our Saturday newsletter on Dec. 27 or Jan. 3. Our daily newsletters will also go dark between Dec. 24 and Jan. 1 — but our reporters will continue to bring you news on our website, so be sure to check in! It’s almost the end of the year, and that means it’s time for our newsroom to weigh in on the pharma industry’s highs and lows of 2025. Check out the team’s recap here , or watch our event for an inside look at how the list came together. We also reported this week on the Trump administration’s latest “most favored nation” deals, an AI biotech’s $130 million raise, and the rise of investigator-initiated trials in China. Our team will be on the ground at the JP Morgan conference next month. Whether you’re heading to San Francisco in person or tuning in remotely, you can sign up for our event here . Until then, happy holidays! Thanks so much for spending your Saturdays with us this year. See you in 2026! — Nicole DeFeudis 🤝Amgen, Boehringer Ingelheim, Bristol Myers Squibb, Genentech, GSK, Gilead, Merck, Novartis and Sanofi are the latest companies to announce drug pricing deals with the Trump administration. A senior administration official predicted major reductions in Medicaid prices as a result of the latest deals, which Zachary Brennan detailed here. And all of the companies have also agreed that any new product launches will come at the MFN-based prices. The White House plans to launch a new government site early next year, known as TrumpRx, to help patients purchase drugs directly at discounted rates. President Donald Trump sent letters to 17 drugmakers in July , calling for them to implement MFN pricing. Pfizer , Eli Lilly, Novo Nordisk , AstraZeneca and EMD Serono have also struck deals with the White House. The latest companies are expected to receive a three-year reprieve from tariffs, in line with other recently announced deals. 📋Investigator-initiated trials — or IITs — have grown increasingly popular among China researchers, and Western drugmakers working on genetic medicines want in, Endpoints’ Lei Lei Wu and Ryan Cross reported . The small, cheap, quick trials have played a major part in how China became a competitive force in cell and gene therapy. Unlike a traditional clinical trial, which requires approval from national regulators, IITs may only need clearance from the Chinese research hospital running the study. They also benefit from looser manufacturing requirements. And while drug regulators often require companies to monitor patients for long periods before ramping up to higher doses, the turnaround is shorter in an IIT. IITs can’t be used for approval. But they can help companies see whether their medicines are promising, or allow them to “fail fast,” Imviva Biotech CEO Lu Han told Endpoints. They can also be used to help attract new backing. EsoBiotec used an IIT to treat its first multiple myeloma patient in November 2024 and quickly showed that its therapy could keep the blood cancer at bay. Just months later, AstraZeneca said it planned to buy the company for $425 million. “Everybody in biotech is reevaluating how they work with China,” said Emile Nuwaysir, CEO of the recently launched Cambridge, MA-based genetic medicine startup Stylus Medicine. He said Stylus is considering a China IIT for its first clinical trial. How will the US respond? FDA Commissioner Marty Makary has expressed skepticism about trials conducted in China. He recently suggested that companies with early-stage studies outside the US might have to pay a higher user fee. Meanwhile, some US biotech investors have called for the FDA to follow China’s model, allowing for decentralized approvals for first-in-human trials. Read more here from Ryan and Lei Lei. ☕An ambitious AI biotech startup pulled in some new cash this week , effectively giving it unicorn status . Chai Discovery closed a $130 million Series B at a $1.3 billion valuation, continuing a furious fundraising pace since being founded last year. The round was co-led by Oak HC/FT and General Catalyst. Other investors included Thrive Capital, OpenAI and Dimension Capital. Chai publicly launched in 2024 with a $30 million seed round carrying a $150 million valuation. The company then closed a $70 million Series A round this August at a $550 million valuation and is now taking another sizable jump just four months later. Despite the speedy venture rounds, Chai has been slower in disclosing specifics on its business plans. In previous interviews with Endpoints News , its co-founders have declined to say if they plan to build an internal pipeline of drugs, or focus narrowly on selling software or services to the pharma industry. This week’s announcement suggests the former Endpoints 11 winner may be more focused on going the software and/or services route. Vivek Ramaswamy, the former Roivant CEO and presidential candidate, appeared on the board of a private chronic pain biotech this week. Ramaswamy, now running for governor of Ohio in next year’s election, is a co-founder, investor in and board member at Ambros Therapeutics. Ambros raised $125 million and hopes to begin a Phase 3 study in 2026 for its lead program, neridronate, in complex regional pain syndrome type 1, or CRPS-1. In somewhat classic Ramaswamy fashion, this isn’t neridronate’s first go-around at an approval. The drug has been approved for CRPS for more than a decade in Italy, but German drugmaker Grünenthal discontinued two Phase 3 studies in 2019. The new attempt harkens back to the business model Ramaswamy established at Roivant: taking older, less expensive drugs and running new, better studies for them. There are currently no FDA-approved treatments for CRPS. Biotech correspondent Kyle LaHucik reported this week that Areteia Therapeutics, one of the biotech industry’s biggest recent startup bets in respiratory illness, will wind down operations . Its top executives appear to have left the company, and three Phase 3 studies were listed as terminated on the federal clinical trials registry. Areteia had put together funding for as much as $425 million from investors such as Bain Capital Life Sciences, ARCH Venture Partners, Access Biotechnology, GV and Population Health Partners.

Earlier this year, the Belgian cell therapy startup EsoBiotec had only 12 employees and had raised €22 million to develop a cutting-edge infusion that turns a patient’s immune cells into destroyers of disease. But despite its meager resources, EsoBiotec managed to do something that its better-capitalized peers hadn’t: It became the first to report clinical data on a highly-anticipated cancer treatment called an in vivo CAR-T therapy. And it did so with help from China. EsoBiotec worked with Chinese doctors to run an investigator-initiated trial, known in the industry as an IIT. Unlike a traditional clinical trial that requires approval from national regulators, researchers just need clearance from the Chinese research hospital running the study to start an IIT. EsoBiotec treated its first multiple myeloma patient in November and quickly showed that its therapy could keep the blood cancer at bay. Just months later, AstraZeneca said it planned to buy the company for $425 million. “We’re all comfortable with the US being the dominant space for drug discovery and drug development, but I think that is definitely shifting,” AstraZeneca’s head of immuno-oncology and cell therapy Mark Cobbold told Endpoints News . IITs have grown increasingly popular among China researchers over the past decade, and have helped China’s fast-growing biotech sector catch up with rivals in the US and Europe. The very leniency that once made American and European companies view these Chinese studies with skepticism is now providing them a unique opportunity to cheaply and quickly assess their experimental medicines. While the small trials can’t be used for approval, they offer a nimble way to let companies see whether their medicines are promising — and help them attract new backing. That’s especially important for cell and gene therapy developers, where Chinese companies are beginning to pull even or ahead of Western biotech, prompting US policymakers to look at changes. According to one analysis conducted by Beijing-based research center Changping Laboratory, the number of Chinese IITs for cell and gene therapies grew 11-fold between 2015 and early 2024, totaling more than 1,000 studies — though other data sources suggest the number may be even higher. Because trials are local and there’s no centralized database to monitor them, it can be hard to know exactly how widespread they are. It’s also unknown how many of these studies have ties to companies outside of China. While biopharma companies have long conducted trials around the world, including in China, IITs have emerged as a new tactic. In conversations with more than a dozen biotech leaders working on cutting-edge cell and gene therapies, many US and Chinese drugmakers alike told Endpoints they are at least considering, if not already planning or currently running, an IIT in China. “Everybody in biotech is reevaluating how they work with China,” said Emile Nuwaysir, CEO of the recently launched Cambridge, MA-based genetic medicine startup Stylus Medicine . “Their model enables a much faster path to clinical validation, and for a startup, the most important thing you need is clinical validation. You’d have to be crazy not to be paying attention.” Nuwaysir said that Stylus is considering a China IIT for its first clinical trial, although he declined to share the company’s specific plans. And he expects more US biotechs to turn to China for IITs: “If they’re not doing it, they’re going to lose.” IITs played a major part in how China became a competitive force in cell and gene therapy. Legend Biotech, for instance, presented astonishing results in 2017 from an IIT study of its multiple myeloma CAR-T cell therapy. Those early data paid off. Johnson & Johnson struck a partnership with Legend later that year. Their cell therapy Carvykti surpassed $1 billion in sales in 2025. IITs must be run by researchers at a hospital or academic center. Close-knit relationships between Chinese biotech companies and physicians at top hospitals are key to the profluence of IITs and a high hurdle for Western drugmakers looking to break in. To run its IIT, EsoBiotec worked with Shenzhen-based Pregene Biopharma, which worked with two hospitals in Wuhan. For EsoBiotec, the advantages outweighed the challenges thanks to the most important feature of IITs in China: their regulatory flexibility. While physicians can run IITs in the US, they still need the FDA’s approval. In China, permission is granted by local authorities, such as ethics and science committees at research hospitals, which can make reviews faster and less stringent. “We gained probably two years,” EsoBiotec CEO JP Latere said. Another advantage is looser manufacturing requirements. In Phase 1 studies, companies have to adhere to Good Manufacturing Practice (GMP) standards, which require intense quality control and heavy documentation to ensure the process is replicable. But in IITs, companies can make smaller batches that are “GMP-like” or “halfway to GMP,” which saves time and money, several biotech leaders told Endpoints. And while drug regulators often require companies to monitor patients for long periods before ramping up to higher doses, the turnaround is shorter in an IIT. Companies work with the hospital directly to modify dosing, the study design and even the drug itself. Lu Han, CEO of Imviva Biotech (formerly known as Bioheng Therapeutics), said IITs can help a company “fail fast.” His company is using IITs to develop off-the-shelf cell therapies, an area where many US companies have struggled. “If you really want to crack this code of immune rejection, you might need a little more than just one or two trials – maybe 10 or even more,” Han said. New FDA leaders have acknowledged that early-stage work in the US needs to speed up. Lawmakers, too, are paying attention. “When the developers are looking outside our country for early-stage clinical work, we need to pick up our game,” Sen. Bill Cassidy (R-LA) said at a hearing on US biotech competitiveness. Some US biotech investors have called for the FDA to follow China’s model , allowing for decentralized approvals for first-in-man trials. Instead, many are going to China, where they can act now. Umoja Biopharma, one of the most well-funded in vivo CAR-T companies, is conducting its first clinical test in a China IIT. Gilead has also struck a deal with China’s Pregene to study cell therapies in human trials more quickly. Some new startups have told Endpoints they are banking on IITs to get their first clinical data with a modest amount of funding. Even so, FDA Commissioner Marty Makary has expressed skepticism about trials conducted in China. He recently suggested that companies with early-stage studies outside the US might have to pay a higher user fee. There are also lingering concerns around rigor and data trustworthiness. But those are no longer the roadblock they once were. Some experts believe China’s newly implemented standards for IITs could improve perceptions and scrupulousness of the studies. Many genetic medicine companies have already been going to countries like Australia and New Zealand to conduct their Phase 1 studies. Tests in China are just the next step. Nuwaysir, the Stylus CEO, said the US would benefit from “the elements of nimbleness” that China provides. “We’re going to need to replicate that paradigm here, or something like it, because there’s immense power in it,” Nuwaysir said. “There’s a lot for us to learn from what they’re doing. And frankly, if we don’t learn, there’s going to be more pain coming for biotech.”

Welcome back to Endpoints Weekly! This week, our team gathered in Boston to honor 11 of the most promising biotech startups of 2025. Be sure to check out this year’s list of companies chasing bold ideas and making big scientific bets. Our team also covered a group of closely-watched meetings this week: former CDC Director Susan Monarez’s testimony before the Senate HELP Committee, and a meeting of the CDC’s Advisory Committee on Immunization Practices. Read more below. Elizabeth Cairns has the latest on Eli Lilly’s obesity pill data, and Kyle LaHucik reported on another big deal for Novartis, as well as Genmab’s decision to end work on an ADC. We’ll be back in your inbox on Monday! — Nicole DeFeudis 🏆 Meet the most exciting biotech startups of 2025 with this year’s Endpoints 11. From superintelligent AI and in vivo CAR-T in solid tumors, to oncolytic viruses and biologics-in-a-pill, this year’s winners are backed by big names in science and investing. Founding editor John Carroll broke down the winners in-depth here . Our team of reporters also profiled each of the 11 companies that earned the honor. In alphabetical order, this year’s winners are: Averna Therapeutics , Candid Therapeutics , Chai Discovery , Dispatch Bio , Judo Bio , Lila Sciences , Orbis Medicines , Sironax , Stylus Medicine , Third Arc Bio and Umoja Biopharma . Each profile is linked to the company names, where you can read more about them. And if you missed the event, an on-demand recording will be available soon! Former CDC Director Susan Monarez told senators this week that she was ousted from the agency after she declined to pre-commit to approving recommendations from the CDC’s panel of vaccine advisors and refused to fire career officials responsible for vaccine policy without cause. She testified before the Senate HELP Committee, along with former CDC chief medical officer Debra Houry, who resigned shortly after Monarez left the agency. The Endpoints team live-blogged the event here . During the hearing, both Monarez and Houry brought up concerns about the composition of ACIP, an advisory committee that makes recommendations on vaccine use in the US. Monarez said HHS Secretary Robert F. Kennedy Jr. did not consult her before adding seven new members to the committee in June. Five additional members were announced this week . Monarez said she had not “prejudged” the committee’s meeting this week, but also said during her testimony that “there is real risk that recommendations could be made restricting access to vaccines for children and others in need without rigorous scientific review.” In a meeting the following day, ACIP voted not to recommend giving children under the age of 4 the combined measles, mumps, rubella and varicella vaccine. The committee voted that children in that age group should instead receive separate measles, mumps, and rubella vaccine and varicella vaccine. ACIP weighed the new MMRV guidance due to the slightly heightened risk of febrile seizures in kids 1 to 2 years old who receive the combo shot as their first dose of the regimen. A separate vote on whether to recommend delaying the hepatitis B birth dose if a pregnant woman tests negative for the virus was postponed. The committee also voted on Friday to recommend that all people 6 months and older receive consultation from a healthcare provider before getting a Covid-19 shot. 💊 Eli Lilly has outflanked Novo Nordisk in diabetes and obesity, but one area where Lilly’s drugs continue to lag is in the oral GLP-1 category. Additional Phase 3 results presented this week continue to underscore that advantage for Novo. In Lilly’s study, 55% of those given the pill, called orforglipron, had a body weight reduction of at least 10% when the dose was titrated up to 36 mg. That was a statistically significant improvement compared to the placebo group, where only 13% of participants achieved at least 10% weight reduction. But in a similar trial for Novo’s oral Wegovy, 63% of patients taking the drug saw at least the same level of weight reduction. Oral Wegovy also saw numerically greater benefits in the proportion of patients who lost at least 15% of their body weight: 50% who took oral Wegovy reached that marker, compared to 36% in the Lilly trial. Cross-trial comparisons like these are imperfect, but it may be years before oral Wegovy and orforglipron are pitted head-to-head in the same study, if ever. Researchers attributed some of the results to the gender differences between the studies. About 36% of patients in the Lilly trial were male, compared to 21% male in the Novo study. GLP-1 drugs are known to be more effective in women than men, study leader Sean Wharton said at a press conference in Vienna where the data were presented. Read more here from senior biopharma journalist Elizabeth Cairns, who has been following the GLP-1 space closely. 🤝 The deal comes with $120 million upfront, and is focused on an undisclosed discovery target and the option to license two preclinical immunology programs. It’s the second deal Novartis has made with Monte Rosa. Last October, Novartis signed a deal with the developer of molecular glue degraders that was worth $150 million upfront and $2.1 billion in milestones. The latest deal “allows us to do more with more,” Monte Rosa chief business and legal officer Phil Nickson said in an interview with Endpoints’ Kyle LaHucik. Novartis has been on a dealmaking spree the last couple months. The pharma company announced last week that it’s buying Tourmaline Bio for $1.4 billion. It also inked multibillion-dollar siRNA collaborations with Argo Biopharmaceutical and Arrowhead Pharmaceuticals, and a $772 million partnership with BioArctic, LaHucik reported. Genmab spent $1.8 billion to acquire ProfoundBio last year, but it’s already decided to cull some of the pipeline. In an announcement this week, Genmab said it would stop development of an early-stage antibody-drug conjugate from ProfoundBio. The drug, known as GEN1107 or PRO1107, was in a Phase 1/2 study for various solid tumors, including ovarian, endometrial, and urothelial cancers. GEN1107 was designed to target PTK7, which is of interest to a handful of other biopharma companies. Eli Lilly, Sichuan Kelun Pharmaceutical and Day One Biopharmaceuticals are also researching PTK7-targeted ADCs. GEN1107 was also one of three clinical-stage programs Genmab picked up in the ProfoundBio buyout.