June 5, 2023

The need-to-know this morning

The ongoing proxy battle between activist investor Alex Denner and Alkermes has intensified, with Denner's Sarissa Capital releasing another letter asking shareholders to vote for its slate of board nominees.
Alkeus Pharma raised $150 million in private funding to support the approval filing for a treatment for Stargardt disease, a genetic disorder that causes blindness in children. Vertex Pharma founder Joshua Boger joined Alkeus as its new board chairman.
Novo Nordisk is acquiring a French medical device company that has developed sensor technology for injection pens.
Happy ASCO stock-trading day!

ASCO

Lung cancer drug posts robust overall survival data

New overall survival data on Tagrisso, AstraZeneca's blockbuster lung cancer drug, is pretty remarkable: The EGFR inhibitor reduced the risk of death by 51% in patients who had their tumors removed, scientists reported at ASCO. Framed differently, this means that one in 10 patients receiving the drug will live another five years. The data will change the treatment paradigm for non-small cell lung cancer, researchers say.

"Now not only for this drug, but we'll see a sweeping change with all the targeted therapies being moved to earlier stages of disease," the study's lead author said. "I've been working in lung cancer for 30 years. I never cured anyone with an EGFR inhibitor; the disease always recurred at some point. But I believe we are curing the cancer by using these best drugs early."

The drug costs $17,000 per month, and is used broadly in patients with metastatic lung cancer — bringing in $5.3 billion in 2022 alone. But now that it's shown such value as an adjuvant therapy, meaning a medicine given after a tumor's resected, it could add another $1.8 billion to AstraZeneca's revenue.

liquid biopsies

Hundreds of Grail customers wrongly told they might have cancer

Misdiagnosis has always been the big risk of the new wave of multi-cancer blood tests: Sensitivity and specificity are tricky. But for Grail, the issue has become particularly awkward: The firm accidentally sent out more than 400 letters to patients saying that they might have cancer when they, in fact, did not, the Financial Times reports. Turns out, the letters were the result of a software glitch, sent in error by PWNHealth, a contractor that handles telemedicine for Grail.

Grail and PWNHealth reached out to its customers within 36 hours to tell them of the mistake. This has rattled insurers who are trialing Galleri, the company's $950 test that is designed to detect more than 50 types of cancers from a single blood draw.

asco

Targeted therapy slows glioma growth substantially

Vorasidenib, an experimental drug made by France's Servier Pharmaceuticals, dramatically slowed the growth of a certain type of brain tumor — potentially allowing a patient to undergo far less radiation and chemotherapy. Specifically, the drug reduced the growth of grade 2 gliomas by 61%: Progression took 11.1 months for those who took a placebo, compared to 27.7 months for those taking vorasidenib.

It was only effective in patients carrying certain mutations, but it still translates to potentially being useful for the 4,000 or so patients in the U.S. with this type of glioma. The results are "clinically very significant and important," one neuro-oncologist who was not involved in the study told STAT.

The drug was initially developed by Agios Pharmaceuticals, but was sold to Servier in December 2020 for $1.8 billion, plus future milestone payments and royalties. The data were reported at ASCO.

nash

Akero's NASH drug paired with GLP-1s lower liver fat

GLP-1 drugs like Ozempic and Wegovy are being studied for NASH, a difficult-to-treat form of fatty liver disease. But turns out, they're probably not enough on their own to treat a complex disease like NASH. An experimental medicine from Akero Therapeutics called efruxifermin, when combined with a GLP-1 drug, was better at reducing liver fat and scarring than the GLP-1 alone.

A small, 31-patient study of patients with both NASH and type 2 diabetes showed that 88% of those taking the combination treatment achieved normal levels of liver fat, compared to only 10% of the people taking only the GLP-1 drug.

"There has been speculation that maybe GLP-1 is so effective in diabetes and obesity that it's really going to be the key for NASH treatment as well," Akero COO Jonathon Young told STAT. "GLP-1 alone may not be adequate for the complex pathogenesis of NASH."