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How an immunological Trojan horse could treat disease

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Good morning, everyone. Damian here with some breathlessly anticipated news from Vertex Pharmaceuticals, an exit interview from an FDA lifer, and the debut of the $100 genome.


The need-to-know this morning

  • Pfizer reported fourth-quarter and 2023 earnings, and reaffirmed 2024 guidance.
  • Kura Oncology reported no cases of differentiation syndrome, a closely tracked side effect, in a new study that combines its experimental menin inhibitor, called ziftomenib, with chemotherapy in patients with acute myeloid leukemia. High rates of differentiation syndrome reported in previous ziftomenib monotherapy studies were a concern.
  • Regeneron Pharmaceuticals is spending $5 million to acquire 2Seventy Bio’s pipeline of cell therapies for cancer and other diseases. A smaller 2Seventy will carry on, supporting the marketing and further development of Abecma, a CAR-T for multiple myeloma partnered with Bristol Myers Squibb.
  • Gilead Sciences increased its ownership stake in Arcus Biosciences. The two companies are jointly developing an anti-TIGIT antibody for cancer.

Vertex’s pain drug hits in Phase 3

A novel pain medicine from Vertex Pharmaceuticals met its primary goals in three Phase 3 studies, setting the stage for an FDA application that could dramatically change the company’s fortunes.

As STAT’s Jonathan Wosen reports, Vertex’s drug, VX-548, significantly outperformed placebo in studies enrolling patients with acute pain following surgery. The drug missed its secondary goal of proving superior to a combination of acetaminophen and hydrocodone, a commonly prescribed opioid.

Vertex will submit the results, among 2024’s most hotly anticipated readouts, to the FDA by the middle of this year, the company said. If everything goes to plan, Vertex’s oral, non-addictive pain medicine could become a multibillion-dollar product, analysts have said, transforming the company in the process.


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How an immunological Trojan horse could treat disease

For millions of patients with autoimmune diseases, the body’s natural defenses errantly attack healthy tissues. One startup’s plan to intervene involves disguising drugs as bodily trash and tricking the immune system into dragging that Trojan horse to its biological targets.

As STAT’s Allison DeAngelis reports, the company is called Cour Pharmaceuticals, and it just raised a $105 million Series A round to flesh out its foundational idea. The technology involves using proprietary polymer nanoparticles to cloak molecules designed to sensitize the immune system. Once those nanoparticles reach the liver, the drug works like the inverse of a vaccine, telling the body to stop fighting against its own cells.

Cour is already in Phase 2 development with treatments for celiac disease and a rare liver disorder called primary biliary cholangitis. The company is also at work on treatments for type 1 diabetes and the neuromuscular disease myasthenia gravis.

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The Janet Woodcock exit interview

After more than three decades at the FDA, including a lengthy stint as its most powerful regulator of new drugs, Janet Woodcock is retiring from the agency and in the process ending an era.

Woodcock spoke to STAT’s Sarah Owermohle for a wide-ranging exit interview, addressing the FDA’s often tumultuous relationship with Congress, the agency’s future in a changing society, and, of course, the ongoing controversy over which drugs deserve accelerated approval and which do not.

“I tend to look at results,” Woodcock said. “What have been the results of that aggressive posture toward getting new cancer therapies — particularly targeted cancer therapies — out there to patients? Some people might say … there were some that didn’t work out. Well, that’s how accelerated approval is set up. Is that wrong? What’s the alternative? You hold back all of these until you have definitive evidence? Based on the results, you would have a lot of people who wouldn’t be alive. Was that worth it to have all that certainty?”

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The ‘dark horse’ startup with a $100 genome

More than a year after promising to roil the market for sequencing with a $100 genome, the privately held Ultima Genomics is launching a line of instruments meant to challenge Illumina’s near-monopoly.

As STAT’s Jonathan Wosen reports, the sequencer, called the UG 100, will cost $1.5 million and read up to 20,000 human genomes a year. Factoring in the cost of reagents, that works out to $100 per genome, according to Ultima, which plans to launch the sequencer at a scientific conference on Feb. 5.

The question for Ultima, and all of the many of the firms trying to unseat Illumina, is whether it can win over customers at the drug companies, hospitals, and research institutions that handle most of the world’s genomic sequencing. The company has raised more than $600 million in venture investment since its inception, tapping a syndicate that includes Andreessen Horowitz and Khosla Ventures.

Read more.

More reads

  • Can Wegovy treat depression as well as obesity? New research looks to GLP-1 drugs for mental illnesses, STAT
  • Pharma price cut proposals from US government could be steep, analysts say, Reuters
  • European regulators tout their accomplishments against pharma’s anticompetitive practices, STAT
  • Scientists document first-ever transmitted Alzheimer’s cases, tied to no-longer-used medical procedure, STAT
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