September 11, 2023

The need-to-know this morning

Moderna and the German biotech Immatics announced a research partnership to jointly develop new cancer therapies based on Moderna's mRNA technology and Immatics' T-cell engineering. Under terms of the collaboration, Immatics will receive $120 million from Moderna, and is eligible for an additional $1.7 billion in future payments, based on success of the collaboration.
Roche executives are in London for an R&D day with investors and analysts. The slide presentations are here.
Madrigal Pharmaceuticals appointed Bill Sibold as its new CEO, succeeding current CEO Paul Friedman, who will remain on the company's board. Sibold was most recently a senior executive at Sanofi and a member of the pharma's executive committee.
Novartis discontinued the development of an experimental gene therapy for geographic atrophy, a common type of age-related eye disease. The decision was based on the recommendation of independent study monitors who reviewed data from a mid-stage study. The gene therapy, called GT005, was the centerpiece of Novartis' $800 million acquisition of Gyroscope Therapeutics in 2021.

rare disease

Acromegaly drug from Crinetics is effective

An experimental drug from Crinetics Pharmaceuticals successfully lowered growth hormone levels in patients with acromegaly, according to newly released data. People who have the rare and serious hormonal disorder produce too much growth hormone, usually from a benign pituitary tumor.

There are other competing treatments on the market for the condition — monthly injections made by Novartis and Ipsen that generate more than $1 billion a year apiece. But the drug from Crinetics, paltusotine, is a once-a-day pill, which would be much easier for patients to tolerate. There are about 26,000 people with acromegaly in the U.S.

regulatory

Alnylam's date with the FDA

On Wednesday, a panel of FDA advisers will weigh in on whether the agency should approve an Alnylam Pharmaceuticals treatment for an increasingly prevalent heart disease, a decision with hundreds of millions of dollars hanging in the balance.

At issue is patisiran, an intravenous drug, and its benefits for patients with a disease called ATTR-CM, in which misfolded proteins accumulate in the heart and cause life-threatening cardiovascular problems. In a pivotal study, patisiran met its goal by helping patients walk about 14.7 meters more over the course of six minutes compared to those who got placebo, a statistically significant difference suggesting the medicine is preventing the debilitating advance of ATTR-CM.

What remains to be seen is whether the FDA believes that benefit is reason enough to approve patisiran. The final decision, expected by Oct. 8, will determine whether Alnylam can compete in what has become a blockbuster market. An approved ATTR-CM treatment from Pfizer brings in about $2.5 billion a year, and a second from BridgeBio is expected to hit the market next year and, in time, generate billions of its own.

glp-1 drugs

Semaglutide helps people with type 1 diabetes wean from insulin

When given weekly, semaglutide helped seven out of 10 patients with newly diagnosed type 1 diabetes stop taking insulin entirely, a small study in NEJM shows. Researchers are now wondering if interventions with this blockbuster medicine could "change the whole natural history of type 1 diabetes," the study's lead researcher, Paresh Dandona, told CNN.

The study enrolled 10 patients between the ages of 21 and 39 who were taking both the fast-acting prandial insulin and the slower-acting basal insulin. After three months, all 10 patients were able to stop taking prandial insulin and after six months, seven patients had weaned from basal insulin. The patients were followed up to a year.

"Not only did we improve the diabetic control overall, get rid of insulin, but at the same time improve the capacity of the cells to produce insulin," said Dandona, a professor at the University at Buffalo.

politics

Nominee to lead NIH will finally get a hearing

The Senate confirmation hearing for Monica Bertagnolli, President Biden's nominee to run the NIH, will take place next month. Sen. Bernie Sanders had refused to allow a hearing until the Biden administration promised more drug pricing reform. But when HHS announced a deal with Regeneron on developing a new (reasonably priced) Covid-19 monoclonal antibody, Sanders stood down.

Sen. Elizabeth Warren had her own set of demands of Bertagnolli: She said the nominee should not join the biopharma industry or accept any payment from it until at least four years after leaving her post as NIH chief.