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FDA rejects Hunter gene therapy from Regenxbio

February 10, 2026
Biotech Correspondent

Morning! Today, we learn the NCI is planning to study ivermectin for cancer, despite significant scientific evidence suggesting it's a dead end. The FDA rejected a gene therapy from Regenexbio, and Novo Nordisk gets serious about prosecuting compounders.

Among other things, Novo maintains it's not easy to make copycat versions of its drugs easily and safely, which may be underscored by the fact that a compounder Hims & Hers acquired not long ago was apparently sanctioned by the FDA after its facilities were found to be harboring vermin. 

Europe

AstraZeneca CEO takes global view of obesity market

Earlier today, my colleague Andrew Joseph joined a group of reporters who huddled with AstraZeneca CEO Pascal Soriot at a London hotel to discuss the pharma giant's earnings report. Drew sent us his report: 

Soriot framed the company as a global player, touting its success not just in the standbys of Europe and the U.S. or even the surging market of China, but a range of emerging countries as well, from those in South America to the Middle East to South Asia.

And that, AZ executives suggested, could give the firm a lane when it eventually emerges onto the increasingly competitive obesity market. 

The company's lead drug candidate, an oral GLP-1 called elecoglipron, is only just moving to Phase 3 trials, AZ announced today as it reported 2025 earnings. It also has a range of other experimental drugs in earlier-stage studies — targeting not just GLP-1 but glucagon and amylin as well — that the company believes could stand apart from those that companies like Novo Nordisk, Eli Lilly, Pfizer, Roche, and so on are developing.

"We are working on new products that will either improve compliance — so for instance moving to monthly injections as opposed to weekly injections — but also products that will improve the quality of weight loss," meaning more fat loss and less muscle loss, Soriot said.

But the company's commercial operations globally could also come into play, executives indicated, noting there are billions of people around the world with obesity or who are overweight. A model could be Farxiga, the company's drug for diabetes and other conditions that last year earned more revenue outside of Europe and the U.S. than it did from either of those markets.

On obesity, AZ is focused on the U.S. and Europe, said Ruud Dobber, who leads the company's biopharmaceuticals business unit. "But equally over time of course," he added, "we will see hopefully the substantial utility in the international markets where there's a high unmet medical need."


Clinical trials

Eczema drug from Nektar Therapeutics maintains skin responses in one-year study

There is no reporter at STAT who enjoys digging deeply into clinical trial results more than my colleague Adam Feuerstein. He's on the case again this morning, covering Nektar Therapeutic's experimental treatment for eczema, called rezpeg.

From Adam: The big-picture takeaway from this morning's Nektar study readout: Monthly and quarterly injections of rezpeg enabled 71% and 83% of patients with moderate-to-severe eczema, respectively, to maintain stringent skin-lesion improvements after one year.

If that's confusing, here's what Nektar Chief Medical Officer Mary Tagliaferri told me. "When you compare our results competitively, we're showing better efficacy with less frequent injections." 

The results from Nektar's maintenance study met and in some cases exceeded investor expectations — positioning rezpeg as a treatment with the potential to compete strongly against Dupixent and Ebglyss, the two most commonly prescribed biologic drugs for eczema, also known as atopic dermatitis.

Read more here


gene therapy

FDA rebuffs Regenxbio gene therapy bid

The Food and Drug Administration has rejected Regenxbio's one-time gene therapy RGX-121 for Hunter syndrome, an ultra-rare rare condition, STAT's Adam Feuerstein writes. Regulators raised doubts about the surrogate biomarker that underpinned the company's bid for an accelerated approval, as well as the trial's eligibility criteria and the use of a natural history comparator arm. But the FDA's letter notably didn't cite any safety concerns.

"This decision is devastating for the families of boys living with this progressive, life-threatening disease," Regenxbio CEO Curran Simpson said in a statement. "We are concerned about FDA's feedback regarding the overall development path and evaluation of the data in the context of the urgent need for this irreversible ultra-rare disease."

The rejection adds to a growing list of cell and gene therapies for rare diseases that have stalled under the FDA center now led by Vinay Prasad. It also sent Regenxbio shares down, with trading halted after hours.

Read more.


glp-1 drugs

Novo: Hims lawsuit is a warning shot to compounders

Novo Nordisk is escalating its fight against compounded versions of Wegovy, warning telehealth companies and pharmacies that continued sales of copycat semaglutide put them "very, very much on notice," after the drugmaker sued Hims & Hers for patent infringement.

In an interview with STAT, John Kuckelman, Novo's chief counsel, said the tipping point was Hims' plan to sell a compounded version of Novo's new Wegovy pill — a product that, unlike injectable versions of semaglutide, was never in shortage. The lawsuit marks Novo's first patent action in its broader crackdown on compounded GLP-1s.

"It's like the old days of snake oil salesmen just selling things out on the road, telling people that, 'hey, take this, it'll do this for you,'" Kuckelman said, adding that Hims' announcement last week shows "that this is completely out of control at this point, where companies feel emboldened to put untested, unapproved medicines and represent them to the public."

Read more.

As it happens, STAT's Ed Silverman reported yesterday that the FDA recently warned MedisourceRx — a compounder acquired by Hims not long ago — after finding an "infestation" of rodents, birds, insects and other vermin in the buildings used to manufacture, process, or hold the medications. It also failed to report a serious adverse event, in which a patient who had taken a compounded version of semaglutide spent three nights in a hospital with "severe" stomach issues.

Read more.



CANCER

NCI studies ivermectin, worrying scientists

The National Cancer Institute is studying ivermectin as a potential cancer treatment, Director Anthony Letai said. Letai, a political appointee, said there's enough interest to justify a new preclinical look at whether the antiparasitic drug can kill cancer cells — even if no new evidence has emerged to suggest it would be effective. 

"We'll probably have those results in a few months," Letai said. "So we are taking it seriously."

The move has alarmed some career scientists, who say scarce research dollars are being diverted to chase a politically charged idea long hyped online without clinical support, reports Rachana Pradhan of KFF Health News. While ivermectin has shown anticancer effects in lab studies before, small human trials have not demonstrated benefit, and oncologists warn the renewed attention could fuel misinformation and lead patients to delay proven treatments.

Read more.


eczema

Evommune reports positive mid-stage data for eczema drug

From STAT's Jonathan Wosen: Here's a rare crossover between biotech and 18th-century English mathematician Thomas Bayes. This morning, Evommune, a publicly traded firm in Palo Alto, Calif., reported that its experimental drug succeeded in a mid-stage eczema trial. The 70-person, placebo-controlled study's primary endpoint was based on Bayesian statistics, an approach that updates the probability of a hypothesis based on new data.

The FDA issued guidance last month meant to encourage companies to use Bayesian methods, though Evommune planned its analysis before the agency's announcement. The company saw strong evidence that EASI, a common score used to measure disease extent and severity, improved by at least 8%. More traditional statistical analysis showed a placebo-adjusted improvement of 33% after 12 weeks, competitive with results for Dupixent, a blockbuster sold by Sanofi and Regeneron. "The data is so clear," Evommune CEO Luis Peña told STAT. "There's no ambiguity."

The biotech said the drug was safe and well tolerated, and it is now planning a trial to identify the optimal dose of the therapy, EVO301, and considering testing it in other conditions, including ulcerative colitis. Unlike Dupixent and many other eczema drugs in development, Evommune's therapy targets a different inflammatory pathway, IL-18.


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More need-to-know news this morning

  • Moderna and the Mexican government signed a strategic agreement to "enhance Mexico's health sovereignty and mRNA manufacturing resilience."

  • Incyte reported earnings.
  • Sutro Biopharma, a biotech zombie, raised $110 million from a gaggle of biotech hedge funds. Is Sutro back from the dead? What is going on? 

More reads

  • BMS and Evinova pen global development partnership, FierceBiotech

  • Iambic could earn almost $2 billion in new Takeda deal, C&EN

  • The true story behind Mariano Barbacid's 'first effective therapy against pancreatic cancer,' El Pais

Thanks for reading! Until tomorrow,


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