October 30, 2023

The need-to-know this morning

Novartis said an experimental drug achieved the goals of a Phase 3 study in participants with IgA nephropathy, a type of kidney disease. The Novartis drug, called atrasentan, was the centerpiece of its $3.2 billion acquisition of Chinook Therapeutics earlier this year.
Day One Pharmaceuticals said the FDA accepted its application for tovorafenib as a treatment for children with low-grade glioma, a type of brain cancer. The drug was granted a priority review with an approval decision expected on April 30, 2024.
United Therapeutics announced a deal to acquire Miromatrix Medical, the maker of an implantable, bioengineered kidney device, for $91 million.

rare disease

What Sarepta's new Duchenne data will mean

We'll soon learn whether Sarepta's gene therapy to treat Duchenne muscular dystrophy works to slow disease progression compared to placebo. The one-time treatment, Elevidys, secured an accelerated approval in June, with a stipulation that it be backed by confirmatory data. The new data will determine whether Elevidys stays on the market, and if older children can be eligible for treatment.

"We're all a little anxious and a little excited at the same time," one patient advocate told STAT.

Pfizer has its own Duchenne gene therapy in the works, and may have results this fall as well. But thanks to prior safety concerns, analysts believe that even if a Pfizer product becomes available, Elevidys could hold an edge over it. If Sarepta's product proves truly beneficial, the company could see its market value double or even triple.

c-suite

An exit interview with Bristol Myers Squibb's CEO

Bristol Myers Squibb CEO Giovanni Caforio will step down on Wednesday. He spoke with STAT in an exit interview, saying he thinks "it's important for the company to start a new chapter, with a new leader." The 59-year-old Caforio will be replaced by the company's chief operating officer, Christopher Boerner.

Caforio has led Bristol since 2015, when the company was in the midst of a heyday thanks to having brought cancer immunotherapies like Opdivo to market. But the company ceded control of the space when Keytruda edged ahead, becoming the world's best-selling medicine. Caforio led the deal to acquire Celgene in 2019 for $74 billion, in order to try and regain lost ground.

crispr

FDA concerns over CRISPR treatment for sickle cell

The FDA is concerned about the safety profile of a sickle cell therapy developed jointly by Vertex Pharmaceuticals and CRISPR Therapeutics. Documents released ahead of tomorrow's public hearing on the CRISPR-based treatment, exa-cel, show concern around the risk of off-target editing. That is, the agency wants to ensure the companies properly monitor whether the CRISPR treatment alters only the intended portions of genome, and doesn't make unintended alterations to a patient's DNA.

None of the concerns raised by the FDA suggest the agency is reluctant to approve the treatment.

If approved, exa-cel would be the first CRISPR-based treatment to be sold on the market. The FDA is expected to make a decision on it by Dec. 8.

markets

Why Sanofi's stock was eviscerated last week

Sanofi's stock dropped a stunning 18% on Friday, following news that the French drug giant would spin out its $5 billion consumer health division. One analyst dubbed the plunge as "the largest negative single-day stock move across Global Pharma in more than ten years." STAT's Matthew Herper weighed in on the decision, which would center on making drugs like Cialis and Tamiflu available over-the-counter.

The decline had less to do with investors irate with pharma's recent penchant to spin off its consumer health divisions. Instead, it likely had to do with Sanofi using a quarterly earnings report to announce a major strategic change, Matt writes. Sanofi also withdrew its earnings guidance for 2024 and 2025, and said it would substantially increase R&D investments in order to bulk up its pipeline over the longer term. But the move will lower earnings over the next few years — and investors were spooked.

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