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Signage is seen outside of the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, U.S., August 29, 2020. The FDA, in 2019, approved Pfizer's (PFE.N) oral drug tafamidis, sold under the brand names Vyndaqel and Vyndamax, to treat transthyretin amyloid cardiomyopathy in the United States. Alnylam said it will focus on another drug candidate, vutrisiran, an under-the-skin injection in late-stage trials to treat ATTR amyloidosis-related cardiomyopathy. Branded as Amvuttra, vutrisiran is also approved in the U.S. to treat nerve damage in adult patients with hereditary ATTR amyloidosis. William Blair analyst Myles Minter estimates peak U.S. sales of $2.2 billion for vutrisiran as an ATTR cardiomyopathy treatment.
Persons: Andrew Kelly, patisiran, ATTR amyloidosis, Alnylam, Luca Issi, ATTR, William Blair, Myles Minter, Mariam Sunny, Pooja Desai Organizations: Food and Drug Administration, FDA, REUTERS, Alnylam Pharmaceuticals, RBC, Thomson Locations: White Oak , Maryland, U.S, Massachusetts, United States, Bengaluru
This little-known pharma stock can rally 50%, Citi says
  + stars: | 2023-07-31 | by ( Brian Evans | ) www.cnbc.com   time to read: +1 min
It's time to buy shares of Ionis Pharmaceuticals , according to Citi. The firm upgraded the stock on Monday to buy from neutral and raised its price target to $60 from $36. "Though HAE is a competitive market, Phase II data hints that it could have an attractive product profile and be a meaningful player," Lebowitz said. Given current valuation, we view the risk-reward on Ionis shares as being skewed positive." Lebowitz also highlighted donidalorsen's phase 2 test performance, which underpinned the stock upgrade.
Persons: David Lebowitz, eplontersen, HAE, Lebowitz, donidalorsen, Ionis, Michael Bloom Organizations: Ionis Pharmaceuticals, Citi, AstraZeneca, donidalorsen, Ionis
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