Sanofi

Welcome back to Endpoints Weekly. It was a busy week, so let’s dive in. Endpoints’ Max Gelman covered uniQure’s dispute with the FDA over its experimental gene therapy for Huntington’s disease, which has raised broader questions about the use of placebo-controlled studies. We also have a feature story from Jared Whitlock on how Chinese companies are racing to develop cheaper versions of the world’s most expensive medicines. On Friday, Endpoints hosted a Post-Hoc Live session on competition from China and how policy change may have an impact — you can watch here . After a flow of new data from GLP-1 programs last week, the attention turned this week to amylin. You can find the details below. On Friday evening, Max Bayer reported that Vinay Prasad, head of the FDA’s vaccines and biologics division, will leave the agency. You can read that story here . — Nicole DeFeudis UniQure is looking for a path forward with its experimental gene therapy for Huntington’s disease. The company said the FDA has “strongly recommended” conducting a randomized and placebo-controlled study before submitting AMT-130 for approval. But in an earnings call Monday, company executives said a sham-controlled study may be difficult to enroll, if not outright impossible. “Some might even consider this trial design to be unethical,” chief medical officer Walid Abi-Saab said. UniQure’s stock got caught up in the chaos, plunging in the days after FDA Commissioner Marty Makary appeared to criticize uniQure’s Huntington’s program in a CNBC interview , despite not mentioning it by name. Analysts on uniQure’s earnings call asked for more information about how the FDA wants the placebo-controlled study to be conducted, but uniQure didn’t provide a clear answer. The company said it will need to follow up with the FDA in additional discussions, and executives are shooting for a second-quarter meeting. Under Makary, the agency has trumpeted efforts to accelerate rare disease drug approvals. In last week’s CNBC interview, Makary said he brings a “sense of urgency” for addressing rare diseases to his role at the agency. Following uniQure’s announcement, some were skeptical. “Whatever one thinks about the [uniQure] data package for AMT-130, broadly speaking it continues to seem like this FDA is not serious about exerting flexibility and accelerating treatments in rare disease,” Stifel analyst Paul Matteis wrote in a note to investors on Monday. UniQure CEO Matt Kapusta said the company is determined to bring AMT-130 forward. “If there is a study that we believe is feasible and ethical, we’re going to do everything we can,” he said on the earnings call. Read more here from Max. After a steady stream of GLP-1 data last week, the news kept flowing this week when Sciwind Biosciences got clearance from Chinese regulators for its GLP-1 injectable for obesity. Pfizer will commercialize the drug in that country following a deal last month. But the obesity attention this week was more focused on the amylin front. The mechanism, which some believe should lead to better quality weight loss than the megablockbuster GLP-1s, has caught the attention of many big pharma and biotech investors over the past few years. And it was one of those pharma deals — Roche’s up to $5.3 billion bet on Zealand Pharma’s petrelintide — that caught the industry by storm last year. Now, the Danish biotech’s stock is being wiped by investor displeasure in Phase 2 data that dropped Thursday evening. Phase 3 testing awaits for Zealand and its co-development partner later this year. But the European duo will have an uphill battle in what Zealand’s CEO has called the “weight loss Olympics.” Following the limited topline data disclosure, William Blair analyst Andy Hsieh wrote in a note to clients that the “real-world use-case for petrelintide will likely be materially reduced given the hypercompetitive nature of the obesity market.” In a global race to develop cheaper and potentially better alternatives to the world’s 10 most expensive medicines, China has pulled ahead, Endpoints’ Jared Whitlock wrote this week. Last April, Chinese regulators approved Belief BioMed’s gene therapy for the bleeding disorder hemophilia B. It’s the first hemophilia gene therapy in China to be developed and manufactured entirely by a domestic company. Belief priced the treatment at $350,000, one-tenth the cost of a rival therapy that sells for $3.5 million in the US. The company plans to dose its first commercial hemophilia B patient this month, and it’s working to secure approval in the US and additional countries. It’s also conducting clinical trials for gene therapies that would compete with two top-priced therapies for hemophilia A and the rare disorder Duchenne muscular dystrophy. Belief is joined by other Chinese companies that are chasing after the world’s most expensive drugs. An Endpoints News analysis of clinical trial databases shows that China accounts for 48 of the 77 programs targeting a group of ultra-expensive gene therapies. Even if only a fraction of these lower-cost alternatives reach the market, it could upend the economics of a gene therapy system built on multimillion-dollar prices, Jared wrote. Belief BioMed illustrates how China industrialized gene therapy development, as part of the country’s wider rise in biotech. You can get the full story from Jared here. One company’s mission to create individualized therapies has ended for now. EveryOne Medicines, which was a pioneering biotech in the fight to develop custom medicines for ultra-rare genetic diseases, is closing its doors, Ryan Cross and Jared Whitlock exclusively reported . EveryOne made the decision shortly after US health regulators outlined long-awaited guidance on the path for individualized therapies to gain FDA approval. A person familiar with the matter told Endpoints that the draft policy didn’t go as far as what EveryOne had hoped. Commercialization in the US wouldn’t be as feasible as initially anticipated. Despite the setback, EveryOne CEO Julia Vitarello said there’s still been plenty of progress in the past year. “Where we are today is a significantly better place,” she said in an interview. But “changing the system is hard. It is not linear,” she added. Backed by GV and Khosla Ventures, EveryOne had already treated a child with a rare neurological disease as part of a UK pilot program. Vitarello declined to answer questions about the future of that pilot program, which was expected to help nine additional children facing neurodegenerative conditions. The San Diego and Shanghai biotech , whose CEO has set a goal of proving whether T cell engagers can wipe out autoimmune diseases, will go public via a reverse merger . Rather than taking the traditional IPO path, which has gained some traction so far this year, Candid is going with an alternative route that sees an up-and-coming private biotech take the place of a struggling Nasdaq-listed company. Candid’s company of choice is Rallybio, a Connecticut-based drug developer that shifted plans over the past few years and had as recently as last month touted the potential for billions of dollars in peak sales for its C5 inhibition work. Going forward, the focus will be on Candid’s slate of TCEs that derived from China-based partners.

Thanks to Nemluvio, Galderma’s dermatology therapeutic category saw sales jump 50% year over year at constant currency, reaching $1.2 billion. Galderma has significantly raised its peak annual sales estimate for Nemluvio (nemolizumab) to more than $4 billion, doubling its previous projection of more than $2 billion. The update follows what CEO Flemming Ørnskov described as an “outstanding launch trajectory” for the inflammatory skin condition drug in its first full year on the market. Driven by strong adoption in its existing indications of atopic dermatitis and prurigo nodularis (PN), Nemluvio posted $452 million in 2025 sales. Growth accelerated sharply in the second half of the year, with the period contributing $321 million to the total. It comes as real-world experience with the IL-31 receptor inhibitor exceeded initial expectations, Ørnskov said on Galderma’s fourth-quarter earnings call Thursday.For now, Nemluvio’s sales are split almost 50-50 between the two indications, but atopic dermatitis, which is a market about 10 times bigger than PN, is quickly increasing its contribution, according to Galderma.In eczema, physicians view Nemluvio ahead of an unnamed “leading brand” on itch relief measures, Ørnskov said, citing a survey. That therapy is likely Dupixent, which generated 4.2 billion euros ($4.9 billion) in fourth-quarter sales across multiple immunology indications, Sanofi reported last month.Doctors place the highest importance on itch relief, where Nemluvio is differentiated and recognized as the therapy of choice, Ørnskov said. When Nemluvio scored its eczema approval from the FDA in December 2024, Jefferies analysts noted that the drug’s ability to rapidly reduce itch “may be particularly attractive to many patients.” Combined with a convenient every-eight-week maintenance dosing, Nemluvio “offers an attractive option for patients not controlled on Dupixent,” the analysts said in a note at the time. Back then, the Jefferies team put Nemluvio’s global peak sales potential at $2.1 billion, including $1.5 billion from atopic dermatitis.  In Galderma’s survey, among doctors who had limited experience with Nemluvio, perceptions of the drug came in significantly behind that leading brand on both achieving clear or almost-clear skin and “offering safe & well tolerated therapy,” the company reported. But those scores turned in favor of Nemluvio against the competitor among doctors who had used it a lot, highlighting the better-than-expected experience that drove the peak sales upgrade, Ørnskov said.Jefferies’ analysts did their own survey recently of 30 U.S. doctors, who reported expected use of Nemluvio in 13% of first-line and 15% of second-line patients with atopic dermatitis in five years’ time. Those figures were notably above the 6%-to-11% range that the team had previously reported. As a result, Jefferies' team dialed up its Nemluvio peak sales estimate to $4.2 billion in February.In the U.S., Nemluvio’s share in new patient starts was trending at about 35% in PN and about 8% in eczema around the end of 2025 and January 2026, with the majority of patients in both categories new to biologics, according to Ørnskov. About 85% of patients under commercial plans have coverage for Nemluvio as a first-line biologic treatment, and Galderma just secured its first—out of four—major Medicare payer agreements with first-line access.Before formal Medicare policies were in place, Nemluvio had a very high success rate when doctors applied for coverage under the medical exception process, which Ørnskov said, “means that the doctors really want the product for their patients.” While international sales only represent a small portion of the drug’s current haul, Nemluvio’s launch momentum outside the U.S. “has been even stronger.” In Germany, the first ex-U.S. market, Nemluvio has gained even greater market share in total prescriptions than in the U.S., and the U.K. is “significantly outperforming” sales forecasts, Ørnskov said.Some of the pricing discussions have been “a bit hard” for some international markets, such as Spain, Ørnskov said. “But as all of these countries accumulate, as their performance is really strong, as we learn from some of the best launches we’ve had, I think you will see continued very strong performance,” the CEO added.In 2025, Nemluvio remained a drag on Galderma’s bottom line, with a $153 million impact on the company’s profit, as it has invested to get the launch up and running, according to outgoing CFO Thomas Dittrich. But Galderma expects financials around Nemluvio to break even in 2026, one year ahead of initial expectations, according to Ørnskov.Dittrich is leaving Galderma to pursue another senior executive opportunity outside the company. Luigi La Corte, who recently served as CFO of Italian pharma Recordati, will take over effective May 1, Galderma announced last month. Thanks to Nemluvio, Galderma’s dermatology therapeutic category saw sales jump 50% year over year at constant exchange rates, reaching $1.2 billion. Net sales for the entire company surpassed $5 billion annually for the first time, as the $5.2 billion performance marked a 17.7% growth rate compared with 2024 sales.

Novo Nordisk confirmed that the expansion of its tablet facility in Athlone, Ireland, will cost €432 million ($502 million) for production of its Wegovy pill. Construction will be “finalized gradually” from the end of next year through 2028. EuroAPI’s net sales dropped 7% to €848.2 million ($984.5 million) last year as the company continues its multiyear restructuring . Its CDMO business declined by 9.6% in sales for 2025, reporting 39 manufacturing projects for the year, down from the 58 it reported the year prior. EuroAPI expects sales this year to decline by 10% due to the ongoing reorganization and “challenging business environment.” EuroAPI spun out of Sanofi in 2022. Also announced this week: Additional reporting by Reynald Castaneda