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A momentous ALS debate, Philip Morris' biotech aspirations, & the trouble with knock-off Wegovy

September 21, 2023
National Biotech Reporter
Good morning, all. Damian here with a preview of a momentous ALS meeting, an update on pivoting from cigarettes to biotech, and the legal whack-a-mole going on with obesity medicines.

The need-to-know this morning

  • Shares of Travere Therapeutics fell nearly 40% in premarket trading after its approved treatment for nephropathy narrowly missed its primary goal in a head-to-head trial with a commonly used generic medicine.
  • Aurinia Pharmaceuticals, under pressure to shake up its leadership, signed an agreement with MKT Capital, one of its displeased shareholders, and added the fund's nominee to its board.
  • BeiGene won European approval for the cancer immunotherapy Tevimbra, making it the first Chinese-developed treatment of its kind to become available in the western market.

ALS

Your guide to a massive day at the FDA

Next week, years of polarizing scientific discourse and bruising online debate will come to a head when advisers to the FDA weigh in on NurOwn, BrainStorm Cell Therapeutics' potential treatment for ALS that has bitterly divided physicians and patient advocates.

On Sept. 27, a panel of experts will consider the convoluted data supporting the potential approval of NurOwn, a stem-cell therapy the FDA previously refused to even consider. To some physicians, the drug's failure to show significant benefits in clinical studies leaves approval out of the question. Others, including the influential ALS Association, say NurOwn's manufacturer hasn't disclosed enough information to properly determine the medicine's value. 

Another group of patients and doctors argue that NurOwn's supporting evidence, while problematic, justifies making it available to the roughly 30,000 ALS patients in the U.S., people for whom there is currently no hope of arresting an incurable disease that gradually erodes motor function and leads to death within about five years of diagnosis.

Read more.



Financials

Biotech is harder than Philip Morris thought

Over the last two years, tobacco giant Philip Morris International has spent about $2 billion on a series of drug companies, conglomerating them into a pharmaceuticals division in a broader effort to diversify from cigarettes (which kill people) to medicines (which generally help keep them alive). 

Things don't seem to be going to plan. The Wall Street Journal reported yesterday that Philip Morris is now considering selling off a stake in its pharmaceutical unit or bringing in a partner with more experience in the field. The company has taken a nearly $700 million charge to reflect the declining value of its health business and recently postponed its prior revenue goals for the division.

Philip Morris raised a few eyebrows when it plotted a future in pharmaceuticals — "They're producing the poison and selling the cure to you later on," as one tobacco researcher put it — but early returns suggest the company might have misjudged just how difficult it is to make money in medicine.


Research

How do you treat a disease that hasn't yet manifested?

Despite years of scientific progress, the ALS field has had a blind spot: People who don't have the disease but have genetic mutations that predispose them to it have largely been left out of research and saddled with patchwork medical care.

As STAT's Jason Mast reports, a group called End The Legacy is out to change that, advocating on behalf of genetic carriers of ALS and frontotemporal dementia, or FTD, a neurodegenerative disease sometimes caused by the same mutations. 

Their demands are straightforward: Carriers need to be involved in decision-making on ALS policies and priorities; they need to be able to access routine medical care, so they can monitor their own health and be diagnosed as soon as symptoms appear; they should be able to access certain approved ALS drugs, even if those drugs have never been tested in carriers; and they need to be included in clinical trials for new ALS drugs.

Read more.


GLP-1

The boom for weight-loss drugs is benefitting lawyers, too

The explosion of demand for novel metabolic treatments Wegovy and Mounjaro has led scores of compounding pharmacies and wellness centers to offer dodgily regulated and questionably safe versions of those medicines, and now the manufacturers are mounting a legal challenge.

As STAT's Ed Silverman reports, Eli Lilly, maker of Mounjaro, filed a series of lawsuits accusing four compounders of selling unapproved medicines and several medical spas and wellness centers of trademark violations, false advertising, and unfair competition by selling drugs under the Mounjaro name. Earlier this year, Wegovy manufacturer Novo Nordisk filed a similar spate of its own lawsuits.

Their cases aren't as cut-and-dried as they might seem. Demand for Wegovy and Mounjaro has made both medicines intermittently impossible to get. The law allows for certain compounders to sell copies of FDA-approved medicines if they're subject to shortages, a legal loophole that could complicate matters for Lilly and Novo.

Read more.


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More reads

  • Novartis is scolded by U.K. trade group for the fourth time this year over Entresto promotions, STAT
  • Novo in supply deal with Ypsomed for auto-injector pens, Bloomberg
  • A breakthrough cystic fibrosis drug gave them the gift of time. But miracles come with complications, Boston Globe

Thanks for reading! Until tomorrow,


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