November 13, 2023

The need-to-know this morning

Forge Biologics, a privately held contract manufacturer of cell and gene therapies, is being acquired for $554 million by Ajinomoto, a Japanese multinational food and biotechnology conglomerate. Forge was created in 2020, backed by Perceptive Advisors' Xontogeny Venture Fund.
Aclaris Therapeutics said an experimental drug failed to reduce symptoms compared to a placebo in a mid-stage clinical trial of people with rheumatoid arthritis. The company is discontinuing development of the drug, called zunsemetinib, including an ongoing study in psoriasis.
Vir Biotechnology presented new data from mid-stage studies involving experimental treatments for chronic hepatitis delta and hepatitis B.

heart disease

Novo Nordisk wants to be a major cardiovascular player

Novo Nordisk's main focus has been the diabetes and obesity markets, but now it has its sights on becoming a major player in heart disease. Its drug Wegovy reduced the risk of heart attacks, strokes, or death by 20% in a key trial, the company said in August, leading to a surge in its stock. At the American Heart Association's conference this past weekend, the company presented full details from the study to a standing-room only crowd: The drug reduces the rate of heart attacks by 28%, strokes by 7%, and cardiovascular-related deaths by 15%.

"This is the first weight management therapy that we've proven in a rigorous trial to reduce the excess risk of cardiovascular events associated with overweight and obesity," the study's lead investigator told STAT. "This now establishes overweight and obesity as a new pathway, another modifiable risk factor, that we can treat in patients with cardiovascular disease."

heart disease

Verve's base editing treatment lowers LDL cholesterol

A CRISPR-based gene editing treatment from Verve Therapeutics successfully reduced levels of "bad" cholesterol in patients with an inherited form of high cholesterol in a small trial.

The 10 patients in this Phase 1 trial had heterozygous familial hypercholesterolemia, a genetic disease that causes high levels of bad cholesterol and advanced heart disease. Nine had heart procedures already, four had prior heart attacks, and all had been unable to control this condition with approved medicines.

When given to three patients, the therapy, which uses base editing, reduced LDL-C levels of 39%, 48%, and 55%. The latter patient's decline in high cholesterol lasted six months. Results were presented this past weekend at the American Heart Association conference.

"It's early, but we think these data open the door to a whole new way to treat coronary disease — a one-time therapy that durably lowers LDL," Verve CEO Sek Kathiresan told STAT.

heart disease

A chat with Eli Lilly's CSO

Eli Lilly has its own interest in the above studies: It has its own GLP-1 drug for obesity, Zepbound, which was just approved by the FDA last week. And it has partnered with Verve Therapeutics for its gene editing drug. The company's CSO, Dan Skovronsky, spoke with STAT, and shared some of his thoughts on its path forward in the cardiovascular space.

"When I think about the pharmaceutical industry and the 10 companies that together spend $50 billion to $100 billion on research and development every year, that's the major chunk of investment that's made in improving human health 10 or 20 years from now," Skovronsky told STAT. "We can make more and more powerful drugs, and sometimes there's a need for that… But other times we can look at an area like cardiovascular disease and say the major problem is not that the drugs aren't good enough, it's that the people stop taking them."

The way to solve this lack of patient adherence to more standard medications, in his opinion, is through technology in science.

infectious disease

UK panel recommends meningitis vaccine to prevent gonorrhea

A panel of immunization experts in the United Kingdom has recommend that a meningitis B vaccine could be used by people at high risk of contracting gonorrhea to bring down spiking rates of sexually transmitted infection.

"Introducing a MenB vaccination program to prevent gonorrhea in England would be a world first and should significantly help to reduce levels of gonorrhea, which are currently at a record high," the panel chair said in a statement.

Neisseria gonorrhoeae, the bacteria that causes the infection, is increasingly resistant to antibiotics. GSK's vaccine Bexsero protects against meningitis B, which is one of the serotypes of Neisseria meningitidis bacteria. The genetic sequences of the two bacteria are between 80% and 90% alike. This similarity suggests that the vaccine could offer at least some protection against gonorrhea.

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