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August 13, 2019___

Today's Rundown

Featured Story

Novartis sells phase 3 rare disease drug to Pharming

Pharming has licensed late-phase rare genetic disease treatment leniolisib from Novartis for $20 million (€17.9 million) upfront. The immunomodulator could come to market in activated PI3K-delta syndrome in the second half of 2021. 

Top Stories

Deciphera soars as phase 3 cancer data tee up FDA filing

A phase 3 trial of Deciphera’s ripretinib in gastrointestinal stromal tumors has hit its primary endpoint. The data sparked a surge in the company's stock price, even though details of the results leave scope to question ripretinib’s prospects.

Regeneron, Ridgeback Ebola meds beat ZMapp, prompting early end for Congo study

Investigators ended a study testing Ebola treatments from Gilead, Regeneron and the NIH because the latter two drugs beat ZMapp, another unapproved drug that had been used in previous outbreaks.

Xontogeny's Landos Biopharma reels in $60M to ramp up IBD program

Landos Biopharma was the first biotech to come out of Xontogeny, the accelerator started by former Sarepta chief Chris Garabedian. Now, the company has picked up $60 million to propel its lead program into phase 2 for inflammatory bowel disease.

Boehringer, MD Anderson build out 'virtual R&D center' for cancer research

German pharma Boehringer Ingelheim and The University of Texas MD Anderson Cancer Center have joined forces to create a new “virtual” R&D hub so the pair can work on cancer drugs.

Datavant, PCORnet to build real-world data network spanning 60M patients

The People-Centered Research Foundation tapped Datavant to help it de-identify and coordinate the sharing of data across its national clinical research network covering over 60 million patients.

Novartis Form 483 puts into question who knew what, and when, about Zolgensma data manipulation 

Novartis, which last week was publicly shamed by the FDA for manipulating data tied to the application of its gene therapy Zolgensma, has said that it launched an investigation in March as soon as it learned of the issue. But records from the lab where the manipulation arose, suggest that the company’s investigation may not have been as deep or wide as the Swiss drugmaker has proclaimed.

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