Protocols

AveXis shares spike on more positive data for SMA gene therapy; Biogen stock tanks on fresh skepticism

→ AveXis $AVXS says that 12 of the 12 infants suffering from spinal muscular atrophy (SMA) who were treated with AVXS-101 reached 13.6 months of age event free. And 9 of 9 reached 20 months event free. The company says that only about one in four infants reaches 13.6 months without an event. The biotech is pushing ahead with a small pivotal trial of a gene therapy that is attracting a growing amount of attention. Their stock spiked 15% on the data.

→ Biogen shares tanked on Thursday, dropping more than 5% as key analysts raised questions about lackluster Spinraza sales and the long, risky road ahead in developing aducanumab for Alzheimer’s.

→ Pfizer once boasted of a PCSK9 inhibitor of its own — bococizumab — which looked like a prime contender to compete with the leading drugs on the market. But the drug proved erratic in trials, persuading the pharma giant to kill it. Today, at ACC, investigators said they believed the problem could be traced to the nonhuman elements in its antibody, which spurred the production of anti-drug antibodies that derailed results for a subset of patients.


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