July 9, 2024

The need-to-know this morning

Pfizer Chief Scientific Officer and R&D Chief Mikael Dolsten is leaving the company after 15 years. A search for his successor is expected to last until early next year, the pharma giant said.
UniQure said its experimental gene therapy called AMT-130 slowed the progression of Huntington's disease by 80% compared to an external control group in a mid-stage clinical trial.

oncology

Interius to start first ever in-vivo CAR-T clinical trial

Interius Biotherapeutics will soon begin the world's first clinical trial of in-vivo CAR-T therapy. Patients will receive an IV infusion designed to transform their immune cells into cancer killers inside their bodies.

Traditional CAR-T therapy is "ex-vivo," meaning the immune cells are manipulated outside the body. It's a complicated and expensive process that involves extracting the cells from the patient, shipping them to a specialized facility to be worked on, and then reinfusing the cells back into the patient.

Traditional CAR-T is out of reach for many patients, and researchers hope that the new in-vivo approach could provide a cheaper and more scalable option.

politics

Pharma is losing friends on Capitol Hill

Pharma's position in Washington has already been weakening over the past few years, as underscored by the passage of the Inflation Reduction Act, which allowed Medicare to negotiate drug prices. The industry's influence is set to wane even more as some of its closest allies leave Capitol Hill.

My colleague Rachel Cohrs Zhang reports that at least six key pharma-friendly lawmakers are expected to have left their seats by the beginning of next year, and their likely replacements are less friendly to the industry and less interested in health care.

One example is Sen. Bob Menendez of New Jersey, home of pharma giants Johnson & Johnson, Merck, and Bristol Myers Squibb. Menendez is currently on trial for bribery charges, and the Democrat vying to replace him, Rep. Andy Kim, supports the party's more ambitious drug pricing plans.

Read more on the other allies pharma will be losing.

GLP-1s

Lilly's obesity drug looks more potent than Novo's in observational study

In its pivotal Phase 3 trial, Eli Lilly's tirzepatide (sold as Mounjaro/Zepbound) led to more weight loss than what was seen in the trial of Novo Nordisk's semaglutide (sold as Ozempic/Wegovy). It's been hard to directly compare the two drugs, though, since there haven't yet been results from any head-to-head trials, but a new observational study suggests Lilly's drug may indeed lead to greater weight loss.

The study, published yesterday in JAMA Internal Medicine, analyzed the health records of over 18,000 people and found that those on tirzepatide had about 15% weight loss at one year, while those on semaglutide had about 8%. Additionally, 42% of patients on tirzepatide achieved more than 15% weight loss, compared with 18% of patients on semaglutide.

Since it's a retrospective observational study, there are many limitations. For example, patients may have encountered shortages and may not have been able to consistently take their medications. Patients may have also been on different diets and exercise regimens.

We'll be watching for results of a randomized head-to-head trial that Lilly is running that's expected to complete in November this year.

Neurology

A private equity approach to investing in neuro drugs

Investors have long been reluctant to invest in drug programs for neurological disorders. There's been a history of failed studies and many trial endpoints are subjective and difficult to measure.

Bruce Leuchter, CEO of Neurvati Neurosciences, a Blackstone Life Sciences portfolio company, argues in a new opinion piece that investors should adopt a a "private equity model" to invest in neurology drug candidates.

This means looking at molecules later in the development lifecycle rather than at early-stage drugs that the classic venture model focuses on. While the costs to acquire a later-stage drug will be higher, investors would be able to better vet the program and confirm the rationale behind the drug's mechanism, Leuchter writes.