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The Readout Damian Garde

An experimental drug delays Type 1 diabetes

It’s a watershed moment 33 years in the making: A single course of the experimental monoclonal antibody teplizumab has managed to delay the onset of Type 1 diabetes by about two years, scientists reported in NEJM and at the American Diabetes Association meeting over the weekend in San Francisco. This is, in essence, the first time a drug has been shown to stave off the autoimmune disease in humans.

The drug, under development by Provention Bio, was tested in 76 people — nearly three-quarters of whom were under 18. The participants had a high risk of developing Type 1 diabetes: Certain antibody markers in their blood indicated that their T cells were revving up to attack their pancreatic cells, which would ultimately cause the autoimmune disease. Teplizumab dissuades the T cells from spearheading an attack against a would-be diabetic’s pancreas. 

Many patients who develop Type 1 diabetes are quite young, which makes the constant blood sugar monitoring and insulin dosing onerous for both the child and the family. A drug that might delay the onset of this drug, even by a couple of years, could decrease the disease burden substantially, researchers say. 

Diabetes pill > diabetes injection

More exciting diabetes news out of ADA: Novo Nordisk may be gearing up for a win, having just revealed some promising data on an experimental oral diabetes drug.

Some of the more effective drugs for Type 2 diabetes belong to a class called glucagon-like peptide-one receptor agonists. These drugs, which include brands such as Eli Lilly’s Trulicity and Novo Nordisk’s Victoza, help lower both blood sugar and body weight — but are currently only approved as injections. 

A new Novo Nordisk-backed study, published in the Lancet, found that a GLP-1 receptor agonist taken in pill-form, dubbed oral semaglutide, is actually more effective than its injectable counterparts in lowering body weight — and no worse at lowering blood sugar. The drug was similar, in terms of tolerability, to its competitors. 

Pills are, of course, a bit more palatable to diabetes patients than daily injections — making these findings particularly promising for Novo Nordisk. It filed a new drug application with the FDA for oral semaglutide just this past March.

Sanofi's new CEO

Sanofi is bringing a new chief executive on board: Paul Hudson, the former pharma lead at Novartis. He’s stepping in for Olivier Brandicourt, who says he’s headed off for an early retirement. However, Brandicourt has faced plenty of investor chagrin in his four years as top dog at the French pharma giant: Sanofi’s pipeline is quite thin, and pricing pressures have taken its toll on the company’s value. 

The market’s taking to Hudson’s hire with some optimism, however, with shares jumping 5 percent in Europe after the Friday announcement. 

Pricing a new gene therapy

Bluebird Bio secured European approval for its beta thalassemia gene therapy earlier this month; this week, it will unveil pricing information. The company has said the treatment has an “intrinsic value” of $2.1 million; analysts, however, expect it’ll charge less. 

So, what do you think: How much less?

Less than Zolgensma, Novartis’ spinal muscular atrophy drug, which was priced at that $2.1 million.

Less than Glybera, a commercial flop in Europe at its $1 million price point and withdrawn from the market, meant to treat lipoprotein lipase deficiency.

Less than Spark's Luxturna, which treats a rare form of childhood blindness, and costs $850,000.

Less than Kymriah, the Novartis CAR-T drug that technically is indeed a gene therapy, and costs $450,000.
 

More reads

  • Lessons from a rare breed: Wall Street bears (Wall Street Journal)
  • Sarepta CEO says he welcomes Vertex's efforts to develop muscular dystrophy drugs. (Boston Globe

Thanks for reading! More tomorrow,

Damian

Monday, June 10, 2019

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