September 25, 2023

The need-to-know this morning

Eisai and Biogen won approval in Japan for Leqembi, their treatment for Alzheimer's disease.
BrainStorm Cell Therapeutics, maker of the ALS treatment NurOwn, submitted "grossly deficient" manufacturing information to the FDA, according to agency documents disclosed ahead of a panel discussion scheduled for Wednesday.
BridgeBio Pharma raised $250 million in a private placement led by the Qatar Investment Authority, resolving some speculations on the company's finances as it seeks FDA approval for an investigational heart drug.

biotech market

We're still waiting for biotech to rebound

Where'd that so-called biotech resurgence go this year? New drugs and sparkly acquisitions were supposed to bolster a sector that's been kind of a mess the past two years. Biotech stocks are generally languishing, with the XBI biotech index falling more than 10% this year alone. In comparison, the S&P 500 rose about 13%. While the blockbuster weight loss drugs like Ozempic have offered a Pharma-specific boost, generalist investors are much more reticent about the riskier biotech stocks.

"Probably the biggest frustration of being a biotech analyst is that we as specialists are skating around this little pond and we don't know how thick or thin the ice will be at any given time because we're not in control of the overall climate," one analyst told STAT.

However, there are a few glimmers of hope: The Federal Reserve said interest rates would drop next year, and two biotechs went public in the past couple weeks. This could foreshadow other biotechs following suit in 2024.

drug shortages

Smaller practices hit worse by cancer drug scarcity

The cancer drug shortages have had a disproportionate impact on smaller practices. While large health systems have largely been able to maintain a consistent supply of medicines like carboplatin and cisplatin for their patients, smaller practices are struggling — widening inequities faced by rural clinics, or those that care for underserved communities.

One issue is that smaller practices can't order directly from a manufacturer, but have to work with intermediaries that essentially ration the drug from clinic to clinic. "What we were being allocated was dramatically lower than our typical usage," one oncologist working for a community practice said. And even though her practice employs more medical oncologists than many area hospitals, those places were able to get more drugs from distributors.

"I'm not sure how it's decided, but it feels really unfair," she said. "Our volume is higher, but we could only get one vial for a week when we needed seven."

cardiology

New drug combos to prevent heart disease

Colchicine is an ancient medicine — it's been used for millennia — that recently has been found useful in preventing cardiovascular inflammation. It's been standard practice to prevent heart attacks by using drugs like statins, but a newer approach is to target inflammation as well, using low doses of drugs like colchicine. It's "about combining therapies" that are "not in conflict, they're synergistic," one cardiology expert told the Wall Street Journal.

The idea now is that medicines could be used in tandem to lessen cardiovascular risk. It's a mix-and-match approach, making patient treatment more precise — for instance combining a non-statin drug that lowers cholesterol with another that lessens inflammation, while also weighing the benefits of the new wave of weight loss drugs. Monitoring the interactions of these new drug cocktails is of course critical, but "preventative cardiology is undergoing a renaissance," another cardiologist told WSJ. "We can do more than ever for our patients who have high risk."

rsv vaccines

CDC greenlights first maternal RSV vaccine

The CDC has endorsed Pfizer's RSV vaccine for women in the middle of their third trimester to prevent their babies from developing severe illness. An expert panel voted 11 to 1 to recommend that women between 32 and 36 weeks of pregnancy get the vaccine from September to January.

"This is another new tool we can use this fall and winter to help protect lives," CDC Director Mandy Cohen said in a statement.

Last month, the FDA approved Pfizer's RSV vaccine in expectant mothers, saying it could prevent lower respiratory infection and severe disease in infants until they're six months old. The CDC panelists said that getting the vaccine late in pregnancy could reduce the risk of preterm birth or other complications that might arise if the vaccine's taken earlier.

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