July 10, 2024

The need-to-know this morning

Flagship Pioneering raised a new $2.6 billion investment fund, plus an additional $1 billion for sector-specific side funds. This brings the total capital raised by Flagship into its funds since 2021 to $6.4 billion.
Abbvie named a new R&D chief. Roopal Thakkar takes over as chief scientific officer and executive vice president for R&D, replacing the retiring Thomas Hudson. Thakkar is currently Abbvie's chief medical officer.

job market

Laid-off biopharma workers vie for scarce openings

Layoffs have persisted in the biopharma industry for several years now, and workers seem to have fewer and fewer prospects. As an indication of how difficult the job market is, there have been more job cuts at large pharma companies this year than at small biotechs.

About 67% of workers laid off during the first half this year were at large pharma companies, according to data from the trade group BIO. That's a sharp jump from 2021 to 2023, when larger firms accounted for 47% of layoffs.

Many of these cuts have been announced as part of broader reorganizations by companies with top-selling products coming off patent. One employee who was laid off from Bristol Myers Squibb has applied to 60 openings so far to no avail. Another worker who was laid off from BMS decided to leave the biopharma industry altogether and accepted a job at Amazon.

stock moves

uniQure shares soar on Huntington's data

uniQure was the best-performing biopharma stock yesterday, with shares jumping 76% after the company reported positive data on its experimental gene therapy for Huntington's disease.

vTFET-uniqure-shares-jump-after-positive-huntington-s-data

Updated interim data from a Phase 1/2 trial showed that at two years, nine patients who received the high dose of the drug had a 0.2 point decrease on a measure of disease progression called cUHDRS. That compares with a 1 point decrease in an external control group based on natural history studies. These data suggest there was an 80% slowing of disease progression in the treated patients.

Analysts said the future of this drug, though, will depend largely on how uniQure's discussions with regulators go, particularly whether it will be okay to use a natural history control group. The company is slated to meet with the FDA in the second half of this year to discuss the possibility of accelerated approval.

biotech

Why Amylyx is betting on a GLP-1 antagonist

After pulling its ALS drug from the market following a failed trial, Amylyx has now acquired a GLP-1 receptor antagonist from Eiger BioPharmaceuticals. The company plans to start a Phase 3 program next year to test the drug in a condition called post-bariatric hypoglycemia, it said in a press release today. I chatted briefly with co-CEOs Justin Klee and Joshua Cohen about this decision.

First, to be clear, this drug is not in the same class as the highly popular diabetes and obesity drugs — those are GLP-1 receptor agonists. The drug that Amylyx has acquired, called avexitide, blocks GLP-1 receptors and has been tested in rare settings: post-bariatric hypoglycemia and congenital hyperinsulinism. In both conditions, patients's insulin levels are too high and they experience dangerously low blood sugar. The CEOs said those patients also have elevated levels of the GLP-1 hormone (which promotes insulin secretion), and avexitide is designed to help patients return to normal physiologic GLP-1 levels.

I asked Klee and Cohen why they opted for this drug, given Amylyx's historic focus on neurodegenerative diseases. The CEOs noted that they've long studied a candidate in Wolfram syndrome, a rare genetic disorder that causes childhood-onset diabetes, and the new drug expands on their work in endocrinology. So now, they see the two primary areas of focus for the company as neuro and endocrine conditions, they said.

In developing endocrine drugs, there are established outcomes to measure in trials, such as glucose and insulin levels, whereas for neurodegenerative conditions, there's still ongoing research on better biomarkers to measure disease, Cohen said.

people moves

Pfizer's chief scientific officer to step down

After serving as Pfizer's chief scientific officer for 15 years, through the tenures of three CEOs, Mikael Dolsten will be stepping down once he helps find his replacement.

My colleague Matt Herper looked back at the ups and downs of Dolsten's tenure. A key highlight was Pfizer's performance during the Covid-19 pandemic, when the company and its partner BioNTech beat Moderna to be the first to bring a Covid vaccine to market.

But more recently, Pfizer has failed to deliver on investors' expectations, with the company's shares now trading at about half where they were during their pandemic peak.

drug pricing

FTC goes after PBMs in new report

The FTC released a lengthy report yesterday that said pharmacy benefit managers wield "enormous power" that can affect which drugs are available, at what price, and which pharmacies patients can use to get medications.

The three largest PBMs — Caremark Rx, Express Scripts, and OptumRx —processed nearly 80% of the roughly 6.6 billion prescriptions dispensed by U.S. pharmacies in 2023, the FTC said.

Additionally, PBMs, which are now part of massive conglomerates that also consist of big insurers and pharmacy chains, impose unfair and harmful contractual terms on independent pharmacies that can put them out of business, according to the report.