September 24, 2024

The need-to-know this morning

Bluebird Bio said it will lay off 25% of its workforce as part of a restructuring plan needed to reduce the company's cash operating expenses by 20%. Bluebird has struggled to generate revenue from three approved gene therapies, including Lyfgenia for sickle cell disease.
Wave Life Sciences reported preliminary results from an early study of its RNA-based treatment for Duchenne muscular dystrophy amenable to exon 53 skipping.

obesity

Metsera's GLP-1 drug has potential for monthly dosing

This morning, Metsera, a new obesity biotech, reported that its GLP-1 drug led to substantial and durable weight loss in a Phase 1 study.

The highest dose of the drug tested, 1.2 mg, was given weekly five times, and then researchers monitored weight loss after the last dose. At 36 days, one week after the final dose, those on the highest dose had 7.5% weight loss. (That's quite rapid weight loss, if you consider that Wegovy led to 15% weight loss in a Phase 3 trial lasting over a year.)

Then, at 57 days, which was four weeks after the final dose, the weight loss extended to 8.1% — a finding that suggests the drug has a longer-lasting effect than currently available treatments, supporting the potential for monthly dosing. Metsera said the treatment has a half life of about 16 days.

The current GLP-1 drugs on the market, such as Wegovy and Zepbound, are dosed weekly, so a monthly option could be more convenient and attractive to patients. But Metsera is further behind many other companies developing next-generation obesity treatments, so it's not clear if its treatment would still be competitive if it eventually gets on the market.

Metsera said that gastrointestinal side effects were dose-related and mostly mild and transient, though it didn't report specific data. The researchers did not see any severe adverse events linked to the drug, and there were no treatment-related discontinuations.

The biotech plans to start a Phase 2b trial of the drug, called MET-097, in the fourth quarter of this year, with data expected in the first half of next year.

immunology

Biogen and UCB's risky bet seems to have paid off

Biogen and UCB said today that their experimental drug to treat systemic lupus reduced disease activity and symptoms, achieving the primary goal of a Phase 3 study.

The companies did not report detailed study results, so it's not yet known exactly how much patients benefitted from the drug, called dapirolizumab pegol. Still, the positive outcome is likely to be viewed favorably by investors given the drug's mixed history.

A Phase 2 study of the treatment in 2018 was negative, though the companies saw enough evidence of a treatment effect to keep developing the drug. That risky bet now appears to have paid off.

Patents

Regeneron's Eylea biosimilar fight heats up

From my colleague Adam Feuerstein: Shares of Regeneron fell 5% yesterday after the company's effort to block Amgen from launching a biosimilar version of its blockbuster eye drug Eylea hit a legal roadblock.

The judge overseeing a patent infringement case denied Regeneron's request for a temporary injunction that would have barred Amgen from immediately selling its Eylea copy, called Pavblu. The judge's ruling was a surprise, because the same judge has previously granted Regeneron's request for injunctions against other drugmakers seeking to launch Eylea biosimilars — seemingly supporting Amgen's assertion that Pavblu doesn't infringe Regeneron's patent. Regeneron filed an appeal.

Pavblu was approved in August, so technically, Amgen could bring the drug to the market "at risk," meaning before patent litigation is decided, although that decision, in itself, would be financially risky if Regeneron were to ultimately prevail in court.

Yesterday, Amgen, in a statement, acknowledged the judge's favorable ruling but only said launch plans would be disclosed "in due course."

Eylea is Regeneron's top-selling drug, with 2023 sales of $5.9 billion. Pavblu and other biosimilars would compete against a low-dose formulation of Eylea that comprises about 80% of the drug's sales. Regeneron has been trying to switch patients to a higher-dose formulation of the drug that has longer patent protection.

politics

Novo's CEO is in the hot seat today

Novo Nordisk CEO Lars Fruergaard Jørgensen will testify at a Senate health committee hearing today about the prices of the company's GLP-1 blockbusters Ozempic and Wegovy. For months, Bernie Sanders has bashed what he calls the "outrageous" prices of the treatments, launching an investigation and threatening to subpoena the CEO to get him to agree to testify.

What can we expect from the committee? Well, we can get some hints from a letter that several lawmakers (outside of the health committee) sent to the Biden administration yesterday. They pitched the use of a decades-old law to effectively let the government sidestep the patents on GLP-1 drugs and open the door to generics. Public Citizen, a non-profit that used to employ one of Sanders' top health staffers, also issued a letter last month arguing for the use of this authority. So we might see Sanders citing this law during the hearing as he argues for lower prices.

And what can we expect from Jørgensen? In written testimony submitted to the committee, the CEO argues that the diseases of diabetes and obesity pose significant costs to health systems and Novo's drugs would save money by preventing medical complications. He also emphasizes the amount of R&D that went into developing the drugs. And, he blames pharmacy benefit mangers for the high out-of-pocket costs for patients, noting that the net price of the treatments (the amount Novo actually gets after rebates to PBMs) has been decreasing.

pharma

Access to generic drugs is now under threat

In 1984, lawmakers enacted the Hatch-Waxman Act, the federal law that gave birth to the modern generic drug industry. Since then, millions of patients have gained access to more affordable treatments, collectively saving the U.S. health care system trillions of dollars.

But now, as the law turns 40 years old this month, experts fear that the generic drug industry is under threat. They cite three primary concerns: the fragility of the generics supply chain, quality concerns, and the difficulty of cheaply making generic versions of newer and costlier brand-name drugs.

Read more from Tradeoffs reporters Leslie Walker and Dan Gorenstein.

health tech

Novartis strikes deal with Flagship AI company

Generate:Biomedicines, a Flagship Pioneering company using AI to create new medicines, said today that it's struck a deal with Novartis to develop an undisclosed number of targets in various disease areas.

Generate is eligible to receive over $1 billion in milestone payments, in addition to royalties. The company, which uses AI combined with wet-lab validation to create new protein-based therapeutics, is one of numerous AI-driven companies fighting for attention from big pharmaceutical companies.