Sanofi research priorities in flux as new CEO logs in

From my colleague Andrew Joseph: One of the open questions about the future of Sanofi as Belén Garijo takes over as CEO this month is whether the company, which had upped its focus in immunology in recent years only to come up with lackluster results, will rebalance its priorities.

As the company reported first quarter earnings this morning, the answer seemed to be … maybe.

“We are fully committed to these therapeutic areas,” CFO François Roger told reporters, citing the company’s targeting of rare diseases and vaccines as well as immunology. But, he said: “Obviously the arrival of Belén will be an occasion as well to revisit if we want to expand into potentially other therapeutic areas." He added that pharmaceutical companies regularly review their pipeline strategies.

Garijo will at least be starting following a strong quarter. The French firm reported profits that beat analyst expectations.

Still, the quarter underscored the challenges that will greet the new CEO. Sales of the mega-blockbuster Dupixent, the immunology drug that Sanofi markets with Regeneron Pharmaceuticals, grew by nearly a third, to 4.2 billion euros, or $4.9 billion.

Garijo will be tasked with finding revenue replacements for such a heavyweight drug. The sense that the company has an uninspiring pipeline, on top of mixed or poor results in some trials, led to the departure of Paul Hudson as CEO earlier this year, even as he made a point of surging investment into the company’s R&D effort.

gene therapy

This gene therapy for deafness seems to be working

Three years after clinicians began administering an experimental gene therapy for congenital hearing loss to patients, it’s becoming clear that the therapy works.

“In some patients, the hearing improved so well that they can hear whispers,” Zheng-Yi Chen, the author of a new paper on the treatment and a Mass Eye and Ear otolaryngologist, told STAT’s O. Rose Broderick. “To reach a stage where you can hear normal conversation, it’s mind-boggling.”

About 90% of the participants in a China-based clinical trial reported significant improvement in their hearing after receiving an injection. The gene therapy was designed to reach the approximately 1% to 8% of people with hearing loss who have this congenital form.

The effect seems strongest in younger kids but it notably extends to adults, which hasn’t really been seen before. The work was a joint effort between investigators at Mass General Brigham and the Eye and ENT Hospital of Fudan University, though drugmakers like Eli Lilly and Regeneron are interested in bringing such a treatment to market.

telehealth

Cheap telehealth is a win for pharma. At what cost?

Drugmakers have found a neat workaround to get patients closer to their products: subsidize not just the drug, but the doctor visit, too. In the case of Addyi, a drug that treats low libido, that can mean a $10 telehealth consult, driven by pharma-linked platforms like Prescribery. Prescriptions often land after a quick, survey-based intake — an efficient move that expands access, especially for patients who might otherwise skip care.

But another view, as STAT's Katie Palmer lays out, is that these platforms are, essentially, a tightly engineered funnel nudging patients systematically toward specific branded drugs, with minimal exploration of alternatives or underlying causes. The same patient can end up with different prescriptions depending on which branded portal they click, raising concerns about overprescribing, fragmented care, and whether these arrangements skirt federal anti-kickback rules.

“Why would I not choose a $10 appointment versus a $30 appointment if I'm not aware of the differences in the telehealth companies or the differences in the therapeutic options?” said Sneha Dave, founder of Generation Patient, a nonprofit supporting young adults living with chronic medical conditions.

cancer

Can Erasca be biotech's next big thing? We'll see

Early buzz and a dollop of clinical data have shot Erasca toward a $7 billion valuation — on the promise that its pan-RAS inhibitor, ERAS-0015, might improve upon Revolution Medicines’ daraxonrasib, STAT’s Adam Feuerstein notes in his weekly Biotech Scorecard newsletter.

RevMed just reset expectations in pancreatic cancer with data that shows its drug doubled overall survival.

But Erasca is now positioning its drug as potentially more effective and better tolerated, with early hints of tumor responses and mild side effects fueling investor excitement. The real test comes in May, when more substantive data from dozens of patients drop.

opinion

RFK Jr. goes quiet on vaccines, but it seems strategic

Health secretary Robert F. Kennedy Jr.’s recent softening on vaccines — acknowledging, for example, that the MMR shot is safe for most people — looks less like a genuine shift and more like a tactical pause ahead of midterms, opines Will Walters, a public health policy and law expert.

Political pressure is mounting over infectious disease outbreaks and regulatory turmoil, triggering the shift, but behind the scenes HHS is still reshaping vaccine policy infrastructure, he says.

“We are likely to be in for a rude awakening once the political incentives to remain quiet disappear,” he writes. “The storm has not passed. After the midterm elections, Kennedy will be once again unfettered in his ability to chase destruction of vaccines.”

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