November 7, 2023

The need-to-know this morning

Sage Therapeutics set the price of Zurzuvae, its recently approved treatment for postpartum depression, at $15,900 for a 14-day course of therapy. The once-daily pill will be launched in December, co-marketed with Biogen.
Eisai reported sales of 300 million yen ($2 million) for Leqembi, its treatment for Alzheimer's disease, in the company's fiscal second quarter. Approximately 800 people were receiving treatment with Leqembi as of Oct. 27, the company said.
In its third-quarter earnings report, Bluebird Bio said 16 patients with beta-thalassemia have started the process of cell collections required for treatment with its Zynteglo gene therapy, since its launch. That's up from 11 patients at the end of the second quarter.

Genome editing

A CRISPR milestone will have to wait

Vertex said yesterday it needed to conduct more lab and animal studies before beginning clinical trials on a treatment designed to use CRISPR genome editing to treat Duchenne muscular dystrophy.

The news, delivered in Vertex's third-quarter earnings (the company did $2.5 billion in sales and beat analyst estimates on profit), mark a setback for what researchers and advocates hope could be a breakthrough approach to treating the deadly muscle-wasting disease. The therapy could deliver the closest thing to a one-time fix, permanently restoring a nearly full-length copy of the gene broken in Duchenne. (A recently approved gene therapy from Sarepta only gives a partial gene and may fade over time). Vertex had hoped to ask the FDA this year to start a trial.

Vertex indicated the studies were needed for its "delivery system." That likely refers to AAVs, miniature viruses that can slide genes into patients' muscles but also come with potential side effects at high doses. Vertex's program would be among the first to deliver CRISPR with AAV, which outside scientists have said could raise new concerns.

Biotech

Watch what you call a 'landmark'

According to the Swiss biotech company MoonLake Immunotherapeutics, its recent Phase 2 trial was a "landmark" in the treatment of psoriatic arthritis, affirming the "potential best-in-class profile" of its investigational drug. And then the stock fell 30%.

That's because, as STAT's Adam Feuerstein writes, the finer print of MoonLake's announcement doesn't quite support those superlatives. In the study, the company's drug outperformed placebo at clearing patients' skin of psoriatic lesions but wasn't meaningfully better than Humira. And MoonLake's skin-clearance rate, adjusted for inflation, didn't stack up to previously disclosed data from rivals UCB and Acelyrin.

And yet the company was resolutely positive on a conference call yesterday, with CEO Jorge Santos da Silva saying the company was considering "strategic opportunities" with potential partners.

Read more.

R&D

A twist in biopharma's latest gold rush

TYK2 is supposed to be the next big thing in auto-immune medicine, a signaling protein that, if properly targeted, could be the key to treating psoriasis, arthritis, lupus, and other inflammatory diseases. But for Ventyx Biosciences, a company whose faith in TYK2 is right there in its name, things aren't going to plan.

Yesterday, Ventyx said its lead drug didn't meet expectations in a Phase 2 psoriasis study, leading the company to discontinue development in that disease as well as psoriatic arthritis. A study in Crohn's disease is ongoing with data expected early next year.

The news sent Ventyx's share price down nearly 80% in after-hours trading yesterday, as investors seemed to count the company out of the competitive TYK2 space. Bristol Myers Squibb won the first FDA approval for a TYK2 medicine in 2022. Takeda, Pfizer, and Galapagos are among the companies at work on rival treatments.

Policy

Why can't cell and gene therapies just be cheaper?

Cutting-edge cell and gene therapies have the potential to be one-time fixes for a range of deadly diseases. But these drugs are extraordinarily expensive and tricky to produce, which in turn pushes companies to charge multimillion-dollar price tags to recover their investments. Yesterday, experts at the Milken Institute's Future of Health Summit considered whether development costs could be brought down.

Panelists argued that more automated manufacturing approaches would be a big help, such as algorithms that can quickly assess whether cells are healthy enough to infuse into a patient. But they also hedged on the question of whether reducing the costs of making these therapies would translate into lower prices, reiterating a common industry refrain: drug pricing is complicated.

But Peter Marks, the Food and Drug Administration's director of the Center for Biologics Evaluation and Research, was clear about the urgency of the issue: "I don't think we have 20 years [to figure out cost]. I think if we don't get this right in the next 10 years, I think we're going to have a real problem of crisis proportions."

Regulatory

Vertex is back at odds with the U.K. over pricing

U.K. regulators have again taken issue with the cost-effectiveness of Vertex Pharmaceuticals' life-saving medicines for cystic fibrosis, a decision that could resurface a lengthy dispute between the two parties.

As STAT's Ed Silverman reports, the National Institute for Health and Care Excellence released draft guidance concluding that the undeniable benefits of Vertex's drugs didn't justify their prices.

It's a preliminary recommendation, and it doesn't affect U.K. patients who are already receiving the medicines. But it suggests Vertex's long-running issues with NICE might not be over. The company spent years disputing an earlier cost-effectiveness ruling, leading to a 2019 agreement that finally made its medicines available in the U.K. That four-year deal will soon expire, and if NICE's recommendation is affirmed, Vertex will be back where it started.

Read more.

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