April 28, 2023

The need-to-know this morning

Johnson & Johnson is a diversified health care colossus, and as such, has long commanded the largest market value of any health care company on Wall Street. But remarkably, Eli Lilly is catching up, driven by the growth of its diabetes business and the mega-blockbuster potential of its medicines for obesity and Alzheimer's disease.

Today, the market-cap gap between J&J and Lilly stands at $74 billion, according to market data provider Koyfin. Call it a difference of one GSK. One year ago, the difference was $214 billion — basically one Pfizer.
ImmunoGen reported Elahere sales of $29.5 million in the March quarter — a strong launch for its ovarian cancer treatment. The drug received an FDA accelerated approval last November. ImmunoGen said today that a readout from the confirmatory study, dubbed MIRASOL, is coming in early May.
Last June, the cancer drug Enhertu was called a "game changer" for dramatically cutting the risk of death in women with advanced breast cancer. Today, its maker, Daiichi Sankyo, reported Enhertu global sales grew more than three times to $1.5 billion for its fiscal year that ended on March 31. Enhertu's torrid growth rate is forecast to continue this year, with global sales projected to reach $2.4 billion, the company said.

podcast

Is a specter haunting Europe?

Will Eli Lilly usurp Johnson & Johnson? And what does "Vowst" call to mind? We cover all that and more this week on "The Readout LOUD," STAT's biotech podcast.

Andrew Joseph, STAT's Europe correspondent, joins us to explain the EU's sweeping proposal to overhaul how new drugs are regulated on the continent — and why the pharmaceutical industry is fighting it. We also discuss the latest news in the life sciences, including Lilly's surging business, a pair of new drug approvals, and a novel idea in Alzheimer's disease.

Listen here.

Genetics

Sweeping study uncovers secrets of human DNA

So much of our DNA remains a mystery to us: The current estimate is that about one-tenth of the human genome is functional. Since 2015, scientists have been probing the genomes of animals to better understand what we have hidden in the genetic remainder. The Zoonomia Project compared the genomes of 240 mammals, ranging from bats to dolphins to lemurs to humans.

A set of 11 studies, published this week in Nature, shows that long stretches of code shared with these animals have remained largely unchanged over 100 million years of evolution — suggesting that the other 90% of supposedly non-functional human DNA could in fact be vitally important.

This code, called constrained DNA, doesn't serve as a template for protein production. But the new research suggests that mutations in this legacy DNA could play an important role in disease, such as certain brain cancers.

privacy

Illumina's sequencers pose cybersecurity risk

The FDA yesterday flagged a vulnerability in Illumina's cybersecurity: The sequencing giant's Universal Copy Service software could easily be hacked, regulators say. This, in turn, could impact the accuracy or privacy of genomic data results — which is used often for clinical diagnosis — that are spun out of many of Illumina's sequencing machines. That said, the FDA and Illumina haven't received any reports anything untoward has yet happened — and Illumina has developed a patch to protect against exploiting this vulnerability in the software.

The FDA suggests that users immediately download the software patch if they have an affected instrument. There are quite a few: This affects the MiSeqDx, NextSeq 550Dx, iScan, iSeq 100, MiniSeq, MiSeq, NextSeq 500, NextSeq 550, NextSeq 1000/2000, and NovaSeq 6000.

oncology

Tempest's drug may help fight liver cancer

When combined with standard-of-care drug regimen, an experimental small molecule from Tempest Therapeutics showed positive early results in a Phase 1b/2 trial of patients with unresectable or metastatic hepatocellular carcinoma. The drug, called TPST-1120, was combined with the antibody drugs atezolizumab and bevacizumab, which are used regularly in the first-line treatment of these liver cancers.

In the TPST-1120 arm, 30% of the patients had unconfirmed responses, and 17.5% had confirmed responses. In comparison, 17.2% of the control arm had unconfirmed responses, and 10.3% had confirmed responses. This suggests that TPST-1120 could indeed be active. Tempest said preclinical work showed the drug could kill tumor cells directly, and target suppressive immune pathways in the tumor microenvironment. A Phase 1 trial also showed that tumors shrank with the addition of TPST-1120.

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