A setback for the Denali-Sanofi partnership

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This newsletter was penned in the company of some mastodon fossils in New York. They say good morning, too.

Today, we see an escalating battle between Eli Lilly and compounding pharmacies putting patients in the crossfire. Also, Sen. Elizabeth Warren is urging the FTC to probe the impending Catalent acquisition by Novo Holdings, and more.

The need-to-know this morning

• French drugmaker Sanofi confirmed negotiations are underway to sell a controlling 50% stake in Opella, its consumer health business. The prospective buyer for $16 billion is U.S. private equity firm Clayton Dubilier & Rice, according to media reports.
• Camp4 Therapeutics raised $75 million in an initial public offering priced at $11 per share. The company is developing RNA-based drugs for genetic diseases.
• Genentech, a unit of Roche, won FDA approval for a drug to treat breast cancer with a mutation in the PIK3CA gene. The drug will be marketed under the brand name Itovebi.

A fight over compounding puts patients in the middle

The battle is intensifying between Eli Lilly, whose weight loss and diabetes drugs are flying off shelves, and the compounding pharmacies that are producing off-brand versions of these medicines.

The FDA last week declared an end to the shortage of Lilly’s Mounjaro and Zepbound, and warned compounders to stop making copycat versions. But, as STAT’s Elaine Chen reports, many patients can’t afford the branded drugs, which carry a list price of more than $1,000 a month, and pharmacies are still having trouble keeping them in stock.

“A lot of people don’t understand how temporary those compounding allowances are,” one researcher tracking drug shortages told STAT. “It seems like people really started to rely on those products that were never — they were always kind of doomed.”

Read more.

FDA advisory panel recommends approval of drug for ultra-rare mitochondrial disease

An advisory committee to the Food and Drug Administration yesterday recommended the approval of a drug from Stealth Biotherapeutics to treat patients with Barth Syndrome, an ultra-rare and fatal mitochondrial disease.

By a 10-6 vote, members of the FDA’s Cardiovascular and Renal Drugs Advisory Committee concluded the Stealth drug, called elamipretide, was an effective treatment for Barth Syndrome, which causes muscle and cardiac weakness, fatigue, and delayed growth. The disease affects about 130 boys in the U.S.

During the meeting, FDA reviewers raised concerns that elamipretide’s clinical data were not sufficient to support approval. A randomized, placebo-controlled study that administered the drug to just 12 patients failed to achieve its efficacy goals. Stealth argued the drug was safe and that patients taking the drug in a long-term extension stage reported benefits. The panel members also heard testimony from Barth patients, caregivers, and physicians who said the drug should be approved.

The FDA is expected to make a decision on elamipretide by Jan. 29.

Sen. Warren asks FTC to probe Novo’s Catalent deal

Sen. Elizabeth Warren is urging the FTC to “closely scrutinize” the $16.5 billion acquisition of contract drug manufacturer Catalent by Novo Nordisk’s parent foundation. She argued that buying the company, which fills and packages syringes and injection pens for Novo Nordisk and other drugmakers — including its rival, Eli Lilly — would ultimately offer the company an unfair advantage.

“I am concerned that Novo Nordisk’s merger with Catalent will give Novo Nordisk unprecedented visibility into and control over its competitor’s production capacity, costs, and business practices, and the ability to preference its own products and obstruct its competitors’ use of catalent to produce” the drugs, she wrote.

Read more here, and for background on the issue read this.

Crypto for CRISPR and a Nobel for AI

How does John Maraganore decide which startups to get involved in? Why are longevity-minded tech bros getting involved with a controversial CRISPR scientist? And who would you rather hang out with at the STAT Summit next week, Adam or Allison?

We discuss all that and more on this week’s episode of “The Readout LOUD,” STAT’s biotech podcast. First, biotech veteran John Maraganore calls in to discuss his new startup, City Therapeutics, this week’s Nobel Prize awards, and the nerdy accolade he just received. Adam and Allison also talk to STAT’s Megan Molteni about her new article on “CRISPR baby” scientist He Jiankui and his new cryptocurrency financier.

Listen here.

And if you’re interested in a transcript of the conversation with Maraganore, here you go.

A setback for the Denali-Sanofi partnership

From STAT’s Jason Mast: Six years ago, Sanofi gave $125 million and promised over $1 billion in milestone payments to buzzy neuroscience startup Denali Therapeutics to go after a promising new target called RIPK1. The enzyme is thought to play a key role in cell death and the immune system — it operates on the same pathway as TNF, the target of an obscure drug called Humira — and the companies planned to study drugs in ALS, Alzheimer’s, and at least a couple immune-related diseases.

That partnership has not gone well. Although Denali is now a $3.7 billion public company, fueled by a technology it developed for shepherding proteins into the brain, the RIPK1 pact has mostly turned up duds. In 2020, the partners switched one of their lead drugs to a backup after initial studies. This past April, Sanofi announced the drug failed to slow ALS in a Phase 2 trial. And yesterday, Denali announced that Sanofi informed it that drug failed in multiple sclerosis as well.

At least one trial is still ongoing on a second drug that Sanofi is studying in ulcerative colitis. But the French drugmaker already scrapped a trial of that therapy in lupus. A couple other companies still appear to have interest. Eli Lilly has a partnership with Rigel Pharmaceuticals on RIPK1, although that was signed in 2021, before the series of setbacks.

Medical journal reviewers paid by industry

Nearly 60% of the experts reviewing manuscripts for four major journals — The BMJ, JAMA, The Lancet, and The New England Journal of Medicine — received substantial payments from industry during a recent three-year period, according to a new study. This new analysis highlights how fraught the world of peer review can be with conflict of interest.

BMJ executive editor Theodora Bloom told STAT that the journal “does, indeed, believe both scrutiny and transparency are necessary” and that authors, reviewers, and editors declare their interests. Other journal editors said similar.

Read more.

More reads

• Sarepta’s gene therapy is fully approved, but its stock has tanked. Here’s why, STAT
• How an accidental email tipped Pfizer off to an activist investor’s plan, Endpoints
• Gritstone files for bankruptcy in hope of keeping R&D afloat, FierceBiotech