How long do we need gene therapy to last?

Biomarin Pharmaceuticals’ gene therapy for hemophilia A might work well enough to win approval, but the durability of its effects has led to a familiar debate for one-time treatments.

As STAT’s Adam Feuerstein and Matthew Herper write, the latest data are unquestionably positive by one metric: Patients had very few of the bleeding episodes that characterize hemophilia A. The issue is that the gene therapy’s effect on factor VIII, the clotting factor patients are missing, waned over time.

Biomarin believes the data it’s gathering will be enough to convince regulators to approve the therapy by the end of 2020. But just how much payers are willing to shell out for the one-time treatment will depend on how long it can keep hemophilia A patients out of the hospital and free from the need for other expensive drugs.

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A nail-biter for Celgene investors

Thanks to Bristol-Myers Squibb, every outstanding share of Celgene will soon be redeemed for one share of Bristol, $50 in cash, and one lottery ticket called a CVR. But unfortunately for Celgene shareholders, that lottery ticket is already looking a little shaky.

That’s because of ozanimod, a once-rejected treatment for multiple sclerosis. Celgene resubmitted it to the FDA back in March but hasn’t said a word about it since. And, as Mizuho analyst Salim Syed pointed out, the FDA’s customary 60-day review period has come and gone in that time.

That doesn't necessarily mean ozanimod's in trouble. The agency has the right to take an additional 14 days to review applications, and a Celgene spokeswoman said the company still expects to hear back from the agency within that timeframe.

But here's why the FDA silence could be alarming to Celgene shareholders: If three of the company’s drugs win approval on a specific timeline, each CVR transforms into $9 in cash. But if a single drug falls out, the CVR becomes worthless. For ozanimod, that means clearing the FDA by the end of 2020, a prospect that will get more difficult if the latest application needs amendments.

The drug industry’s income gap got wider

Last year, at the median, biopharma employees got a 6% raise over 2017. But their bosses in the C-suite saw total compensation increase by nearly 40%.

That’s according to a new analysis from BioPharma Dive, which dug through the annual filings of about 200 companies in the drug industry.

Interestingly, the increase in CEO pay had little correlation with company performance. Across the board, biopharmas lost about 8% of their share value, according to BioPharma Dive, and CEO pay grew more at the companies that declined than the ones that increased.

No one knows why cancer therapies cause diabetes

Checkpoint inhibitors for cancer have brought dramatic improvements to a fraction of cancer patients. But for a small percentage of people who get those immunotherapies, the benefits come with a mysterious and irreversible side effect.

About 1% of people who take drugs like Keytruda and Opdivo develop Type 1 diabetes, a lifelong metabolic disorder that requires regular doses of insulin. And, as Elie Dolgin writes for STAT, scientists are still scratching their heads as to why.

Now, with $10 million in research funding, groups including the Parker Institute for Cancer Immunotherapy are banding together to investigate the phenomenon and, ideally, find a way to extract all the benefits of checkpoint inhibitors without the risk of triggering diabetes.

“I’m pretty open-minded about what mechanistically can be happening,” Parker Institute president and CEO Jeff Bluestone said. “And I’m really optimistic that we’re going to learn a lot quickly.”

How long do we need gene therapy to last?

A nail-biter for Celgene investors

STAT webinar: Progress and pitfalls in the AI-aided search for novel drugs

The drug industry’s income gap got wider

No one knows why cancer therapies cause diabetes

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