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Top related persons:
Luca Issi - 2 Andrew Kelly - 1 patisiran - 1 ATTR amyloidosis - 1 Alnylam - 1 ATTR - 1 William Blair - 1 Myles Minter - 1
Top related locs:
U.S Bengaluru White Oak , Maryland Massachusetts United States Japan's Daiichi California Tyler Van Buren
Top related orgs:
Alnylam Pharmaceuticals - 2 Thomson - 2 AstraZeneca - 2 Food and Drug Administration - 1 FDA - 1 REUTERS - 1 RBC - 1 Jefferies - 1

Search resuls for: "transthyretin"


7 mentions found


☆ Alnylam gives up on expanded use of heart-disease drug in US after FDA declines approval
  + stars: | 2023-10-09 | by ( Mariam E Sunny | ) www.reuters.com   time to read: +2 min
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
☆ Jefferies upgrades this pharma stock, citing overlooked 'blockbuster' drug opportunities
  + stars: | 2023-09-25 | by ( Pia Singh | ) www.cnbc.com   time to read: +2 min
AstraZeneca's tozorakimab drug for chronic obstructive pulmonary disease also seems underappreciated, with a potential to reach $4.5 billion in peak sales, according to Jefferies. The firm also suggests up to $3.5 billion in sales for AstraZeneca's eplontersen , a drug aimed at treating hereditary transthyretin-mediated amyloid polyneuropathy. The pharmaceutical giant is also poised to be the frontrunner in breast cancer treatments. Another one of AstraZeneca's breast cancer therapies, also being developed with Daiichi Sankyo, came out with positive news Friday. Its experimental precision drug datopotamab deruxtecan demonstrated an improvement in slowing the progression of a common type of breast cancer in a late-stage trial.
Persons: Peter Welford, Welford, AstraZeneca's, Jefferies, AstraZeneca's eplontersen, transthyretin, deruxtecan, Pascal Soriot's, — CNBC's Michael Bloom Organizations: AstraZeneca, Jefferies, AstraZeneca's, Japan's, pharma Locations: Japan's Daiichi
☆ 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
☆ BridgeBio's heart disease drug meets late-stage study goal, shares soar
  + stars: | 2023-07-17 | by ( Khushi Mandowara | ) www.reuters.com   time to read: +2 min
July 17 (Reuters) - BridgeBio Pharma Inc's (BBIO.O) experimental drug for a rare heart disease showed significant improvement in patients in a late-stage study, sending its shares surging 65% on Monday. BridgeBio's drug improved survival by 81% in the study patients compared to 74% on the placebo, and also reduced frequency of heart disease-related hospitalization by 50%. The California-based company also provided additional data comparing it with Pfizer's drug. Acoramidis showed 92% improvement on heart failure test, compared to patients on placebo and Pfizer's drug at 30 months. The additional data suggests that BridgeBio's drug was better than tafamidis in controlling the disease, said TD Cowen analyst Tyler Van Buren.
Persons: acoramidis, Acoramidis, Cowen, Tyler Van, Luca Issi, BridgeBio, BridgeBio's, Khushi, Nivedita Bhattacharjee Organizations: BridgeBio Pharma, Alnylam Pharmaceuticals, Ionis Pharmaceutical, RBC Capital, U.S . Food, Drug Administration, Thomson Locations: U.S, California, Tyler Van Buren, Bengaluru
☆ Here are some of RBC's top stock picks for the second quarter
  + stars: | 2023-04-06 | by ( Hakyung Kim | Https | Www.Linkedin.Com In Hakyungkim | ) www.cnbc.com   time to read: +3 min
RBC Capital Markets anticipates that the next quarter could be choppy for stocks, but the firm shared its list of high-conviction names to navigate the volatility. With these concerns in mind, RBC shared a list of high-conviction stocks that it says are well-positioned to offer upside this quarter. Below are 10 of the names: Alnylam Pharmaceuticals and biotech engineering stock Boston Scientific are new additions to RBC's list of high-conviction names. RBC analyst Shagun Singh said Boston Scientific is "positioned to drive consistent double-digit EPS growth," with upcoming device launches and trial data readouts to act as catalysts. On the energy front, RBC named oil company Diamondback Energy as one of its top picks for the quarter.
☆ U.S. Supreme Court rebuffs Pfizer plan to help patients pay for heart medication
  + stars: | 2023-01-09 | by ( Andrew Chung | Industry Insight | Law Firm Pricing Professionals In | ) www.reuters.com   time to read: +2 min
The law targets fraud and abuse, such as bribery, in federal healthcare programs including Medicare, which provides coverage for the elderly and disabled. Pfizer contends the government's interpretation of the law was blocking access to critical medical care. New York-based Pfizer's medication, also known as tafamidis, treats a rare heart condition known as transthyretin amyloid cardiomyopathy that can lead to progressive heart failure. Given the drug's high price, patients would need to bear yearly co-payments of roughly $13,000, with Medicare covering the rest. Pfizer's proposal sought to assist "middle-income" patients who do not qualify for low-income co-pay support but might still be deterred by its cost.
☆ Gene-editing companies are growing thanks to fast-paced science, even as biotech faces a downturn
  + stars: | 2022-10-12 | by ( Andrew Dunn | ) www.businessinsider.com   time to read: +8 min
CRISPR companies are faring better by producing faster and more dramatic clinical results. A layoff tracker from Fierce Biotech counts at least 90 drug companies that have laid off staff in 2022. Gene-editing biotechs have avoided the brunt of the downturn because of fast clinical successIntellia Therapeutics is developing CRISPR-based gene-editing medicines that could cure diseases. Just in the past month, CRISPR Therapeutics moved into a swanky new headquarters in Boston that can house up to 1,000 people. BeamTo be sure, 2022 hasn't been smooth sailing for the CRISPR companies.
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