Candor on gene therapy, Abiomed is warned, & BTK success in MS

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Good morning! Today, cell and gene therapy executives describe how they’re handling their sector’s contraction, we talk about how regulators are cracking down on clinical decision support software, and we look at some impressive BTK data in multiple sclerosis from Roche.

The need-to-know this morning

• Novo Nordisk said it is acquiring an experimental treatment for uncontrolled hypertension from KBP Biosciences, a Singapore-based drugmaker, for up to $1.3 billion. The drug, called ocedurenone, is being evaluated in a Phase 3 study for people with uncontrolled high blood pressure associated with chronic kidney disease.
• Omeros said its experimental drug narsoplimab failed to demonstrate a benefit over placebo in a Phase 3 study of participants with IgA nephropathy, a serious kidney disease.

Pfizer cuts costs as demand for Covid products slide

Pfizer said Friday that it will cut company-wide costs by $3.5 billion to account for reduced sales of its Covid-19 vaccine and antiviral treatment.

The pharma giant lowered revenue guidance for the remainder of the year to a range of $58-61 billion from $67-70 billion. Pfizer said it now expects sales of its Covid drug Paxlovid will be $7 billion lower than its previous forecast. Sales of the vaccine Cominarty will be $2 billion lower.

The reduced outlook for Covid-related products is the result of a revised Paxlovid supply agreement with the U.S. government, which is returning millions of unused treatment courses of the drug. Pfizer will begin selling Paxlovid commercially in January.  Overall demand for Covid products is also waning.

Pfizer said it expects sales of non-Covid products to grow 6-8% year over year.

Cell and gene therapy execs speak freely about industry contraction

Industry insiders can be buttoned-up about the state of the industry. But at a recent conference, top cell and gene therapy executives spoke with candor about how they’re coping, financially, during a difficult time.

The broader biotech sector is the midst of a downturn with no real end in sight — and cell and gene therapy companies have been hit particularly hard.

So how are they coping? Companies are now using smaller teams to work on fewer projects, partnering at times with outside contractors to accomplish what was once done in-house, they said during Meeting on the Mesa, an annual cell and gene therapy conference held in northern San Diego.

“There is no money out there for cell and gene therapy companies,” the CEO of one outfit told STAT. “Any cell therapy in a preclinical stage, if you do not deliver in the next two years, this field is going to shrink more and more. The onus is on us who are still surviving and thriving to deliver.”

Read more.

CMS lightens restrictions on PET scans for Alzheimer’s

CMS is lifting limits on reimbursement for amyloid PET scanning — which could ultimately increase not only the diagnosis of Alzheimer’s disease, but the uptake of the new wave of medicines in this space. PET scans for amyloid plaques were typically very difficult to access, only available to those in clinical trials or patient registries. They are, however, one of the most reliable tools to diagnose the disease.

Under the new policy, coverage decisions for amyloid PET scans will be made by local contractors for Medicare. Companies like Eisai and Eli Lilly, as well as patient advocacy groups, say the move to lift the barriers to amyloid PET scans will advance treatment for people with the neurodegenerative disease.

Read more.

FDA sends warning letter to J&J’s Abiomed over software concerns

Johnson & Johnson’s high-dollar acquisition, Abiomed, sells devices for heart failure patients, as well as software to monitor how they are performing. But last week, regulators posted a warning letter saying that Abiomed should have submitted its software for approval before selling it. It also took the company to task for not reporting a number of issues with its heart pumps.

Regulators announced last year that it would scrutinize health software tools more intently. Clinical decision support tools, which are often powered by artificial intelligence, are increasingly being used to inform patient care with drugs and devices. But patients can be harmed if these algorithms don’t work correctly, as a 2021 STAT investigation found. Companies are becoming reticent to challenge the FDA in court, but are becoming hesitant to go through the approval process for their software. This is meaning that fewer companies are pursuing clinical support software.

“What I’ve been doing is helping client after client water down their software to the point where it’s not innovative and it’s not for a medical purpose,” said an attorney that supports clinical decision software companies. “It’s for diet or exercise or whatever. It’s the consumer that’s losing.”

Read more.

Roche says its BTK inhibitor for MS has ‘holy grail’ potential

New Phase 2 data from Roche shows that its experimental multiple sclerosis drug fenebrutinib was able to successfully traverse the blood-brain barrier — reducing certain lesions by more than 90%. The drug, a BTK inhibitor, has the potential to serve as a “best in disease” medicine — with evidence of brain penetration being the “holy grail,” the Roche exec leading fenebrutinib development told FierceBiotech.

BTK inhibitors have been buzzed-about in the MS space for quite some time now, since they have potential to treat more stubborn lesions that tend to crop up later in life, or further along in the disease progression.

“What we’re hoping for is that with this dual mechanism of action — hitting B cells and myeloid cells — if you can get into the brain, then you could perhaps act on resident B cells within the central nervous system, and on microglia,” Alexandra Goodyear, Roche’s global development lead for fenebrutinib, said.

More reads

• How to pay for million-dollar gene therapy cures, Bloomberg
• FDA approves Velsipity, Pfizer’s inflammatory bowel disease drug, Reuters
• Eyeing Incyte’s Opzelura, Leo Pharma shares data for JAK cream in hand eczema, FiercePharma
• AstraZeneca chair tasked with finding replacement for boss Pascal Soriot, This Is Money
• EU delays approval of Novavax’s revised Covid jab, Financial Times