September 3, 2024

The need-to-know this morning

Dyne Therapeutics reported updated results from an early stage study of its experimental treatment for Duchenne muscular dystrophy amenable to exon 51 skipping. The company also announced the departures of its chief medical officer, chief operating officer, and chief business officer.
Vaxcyte reported results from a clinical trial of its experimental pneumococcal vaccine in adults.
Denali Therapeutics said it reached an agreement with the FDA for an accelerated approval filing in early 2025 of its treatment for Hunter syndrome.

cancer

How an exec-turned-patient helped revolutionize multiple myeloma treatment

In 1996, Kathy Giusti was diagnosed with multiple myeloma and given just three years to live. One reason she's still alive today: The former drug company executive founded the Multiple Myeloma Research Foundation in 1998, which has since raised more than $600 million for research, launched nearly 100 clinical trials, and helped bring more than 15 new drugs to market, STAT's Matthew Herper writes. Though her illness and work took a heavy toll on her family, the progress is stunning, given that multiple myeloma is a relatively rare cancer.

"We would not be where we are today in the treatment of multiple myeloma without Kathy's leadership," FDA oncology chief Richard Pazdur told STAT.

"That she took everything she knew about the pharmaceutical industry, having been part of it, and has really built a powerful advocacy for multiple myeloma patients, is remarkable," the former CEO of a myeloma-focused biotech separately told STAT.

Artificial intelligence

Recursion presents mixed data on lead drug candidate

Recursion Pharmaceuticals, the AI drug developer, announced this morning that its lead drug candidate achieved the primary goal of a year-long Phase 2 study, proving safe and tolerable for patients compared to a placebo, STAT's Allison DeAngelis reports. But the efficacy results were more ambiguous.

The drug, designed to treat cerebral cavernous malformation, or CCM, a potentially life-threatening brain condition, "showed a trend" relative to placebo toward reducing the size of lesions and rings of iron that surround CCM lesions as a result of pooling blood, the company said. The therapy, however, showed no improvements in three patient- and physician-reported outcome measures.

Recursion Communications Chief Ryan Kelly told STAT that company executives were encouraged by these results.

multiple sclerosis
Mixed success for Sanofi's MS drug
Sanofi's pill tolebrutinib hit its Phase 3 endpoint in non-relapsing secondary progressive multiple sclerosis — potentially paving the way for an FDA approval. But two other tolebrutinib studies focused on earlier stages of the disease had mixed results, confusing the trajectory for the experimental drug. Still, it's a win for Houman Ashrafian, the company's new R&D chief — and could help bolster investor confidence in Sanofi's ability to innovate.

"It's a meaningful but not big financial driver, but a big driver in terms of confidence," he told STAT's Matthew Herper. "So what does it mean for us? I think it drives a nail, positively, into the perspective that we can do R&D."

glp-1 drugs

Cardiologists can't stop talking about Wegovy

The annual meeting of the European Society of Cardiology was all abuzz over GLP-1s, thanks to their dramatic effects on heart health, diabetes, and obesity.

As Novo Nordisk and others explore how semaglutide works in conditions like Alzheimer's and immune disorders, researchers are eager to learn more about the underlying mechanisms of GLP-1 drugs, STAT's Andrew Joseph writes.

"As we speed toward an increased understanding of the many, sometimes unexpected, benefits" of GLP-1s, "what is becoming clear is that pathways beyond weight loss are playing a role," one Duke cardiologist wrote in an editorial about the conference.

genomics

Don't cut funding for All of Us

There's value yet in All of Us, the federal research initiative aimed at collecting the health, medical, and genetic information of one million U.S. volunteers. But the program is facing severe funding cuts from Congress, and that could jeopardize the country's efforts to maintain an edge in the life sciences, opines Pradeep Natarajan, a preventative cardiologist at Massachusetts General Hospital, in a new First Opinion.

"All of Us is only half complete, and a loss of stable funding threatens its timeliness and impact," Natarajan writes. "It is unclear how many more Americans will be able to sign up to contribute to this work; I worry that without the necessary support, recruitment levels will fall sharply and data generation will slow dramatically."

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