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Brainstorm Health: Sarepta Stock Spikes, Zika for Brain Cancer, Lasker Prize Winners

Greetings, readers! This is Sy.

Last year, Sarepta Therapeutics made waves as the first company to win (a controversial) Food and Drug Administration (FDA) approval for a Duchenne muscular dystrophy (DMD) treatment. This rare, genetic muscle-wasting disease leaves many of its victims dead by their 20s as patients don’t produce enough of a critical protein, called dystrophin, which helps keep muscles intact. Now, new data suggest the biotech may eventually be able to win another key FDA green light while confirming the effectiveness of its on-the-market product, sending the company’s stock up 14% in mid-day Wednesday trading.

Sarepta’s approved therapy is called Exondys 51 and has received plenty of scrutiny since its approval last fall. The reason? The FDA overruled the recommendations of its own scientists and advisers to clear the drug, arguing that a lack of options for DMD patients warranted approval. Exondys 51 didn’t show particularly impressive efficacy data in clinical trials, and some insurers have refused to cover the therapy barring more proof of a clinical benefit. Sarepta must conduct another study to confirm its efficacy.

But the new experimental treatment from Sarepta, golodirsen, may bolster the firm’s position if early results are any indication. In an early/mid-stage trial, golodirsen—which facilitates something called “exon 53 skipping,” as opposed to the “exon 51 skipping” mechanism at the heart of Exondys 51—proved successful in all 25 tested patients. (Exons are DNA portions involved in coding for protein production, and Duchenne patients have certain defective ones that prevent enough dystrophin from being created.) This resulted in a small bump in dystrophin production.

Some analysts said the new results should give the naysayers pause. “This… reduces any uncertainty around the efficacy of Exondys 51 and decreases any risk around the pending confirmatory study,” said Needham analyst Chad Messer.

Read on for the day’s news.

Sy Mukherjee


Could Zika be used to treat brain tumors? My colleague Laura Entis (who graciously covered for me while I was out of the country over Labor Day weekend) points to intriguing new research published in the Journal of Experimental Medicine finding that injecting mice with Zika reduced the size of cancerous brain tumors while leaving healthy cells intact. (Fortune)


Verastem soars on late-stage blood cancer trial data. Biotech Verastem saw its market value spike 30% after a phase 3 clinical study of its experimental blood cancer drug duvelisib showed the therapy to be far more effective than the current standard of care, pharma giant Novartis’s Arzerra. Duvelisib cut certain blood cancer patients’ risks for their disease getting worse or dying from their cancer by 48% compared to the Novartis treatment, according to the company. (Reuters)


Planned Parenthood, HPV vaccine developers win prestigious Lasker prizes. Three U.S. scientists and Planned Parenthood have been awarded the prestigious Lasker prizes for medicine. Two of the scientists were played key roles in developing the HPV vaccine, and Planned Parenthood was praised for the health care services it provides to both women and men. “We are thrilled to be honored,” said Planned Parenthood president Cecile Richards in a statement. “Planned Parenthood has substantially improved health outcomes for women—and we will keep fighting for our patients, no matter what.” (New York Times)

Senate HELP committee begins Obamacare hearings. A key Senate health committee has launched a series of bipartisan hearings on possible ways to shore up the Affordable Care Act’s individual insurance marketplaces. Insurance commissioners and other experts are testifying in these hearings, and suggestions have included more state flexibility on Obamacare regulations, a federal reinsurance program, and assured insurer subsidies to reduce consumers’ out-of-pocket medical costs.


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San Diego Declares Health Emergency Over Hepatitis A Outbreakby Jennifer Calfas

These Pictures of Hurricane Andrew’s Path Show Why Florida Is Tracking Irma So Closelyby John Patrick Pullen and Kacy Burdette

Produced by Sy Mukherjee

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