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☆ Stocks making the biggest moves midday: Dollar Tree, Amazon, FedEx and more
▼ + stars: | 2023-06-21 | by ( Alex Harring | ) www.cnbc.com time to read: +4 min

People walk by a Dollar Tree store on December 11, 2018 in the Brooklyn borough of New York City. FedEx — The delivery company fell 1.7% after quarterly revenue missed expectations and announced CFO Mike Lenz would retire on July 31. Adjusted earnings were better than expected at $4.94 per share against the anticipated $4.89, while forward guidance was around flat. Advanced Micro Devices — Shares of the chipmaker pulled back nearly 5%, on track for their biggest intraday loss in two weeks. Dollar Tree — Shares of Dollar Tree popped more than 3% after the discount retailer reiterated its fiscal second-quarter 2023 earnings guidance.

Persons: Mike Lenz, MicroStrategy, Tesla, Rivian, Goldman, OneSpaWorld, Walt Disney, Needham, Laura Martin, Bob Iger, Raymond James, Uniqure, hemophilia, Yun Li, Michelle Fox, Jesse Pound, Sarah Min, Brian Evans Organizations: Federal Trade Commission, Amazon, FedEx, Securities and Exchange Commission, Barclays, GlaxoSmithKline —, Petrobras —, Walt Disney —, AMD Locations: Brooklyn, New York City, bitcoin, Netherlands

☆ Pfizer's hemophilia B gene therapy succeeds in late-stage study
▼ + stars: | 2022-12-29 | by ( ) www.reuters.com time to read: +1 min

Dec 29 (Reuters) - U.S. drugmaker Pfizer Inc (PFE.N) said on Thursday its experimental gene therapy for the treatment of hemophilia B, a rare inherited blood disorder, met its main goal in a late-stage study. The drugmaker licensed its hemophilia B gene therapy from Roche's (ROG.S) Spark Therapeutics unit in 2014 for a $20-million upfront payment. Pfizer plans to discuss the late-stage data with regulatory authorities in Europe and the United States and share additional data for the experimental therapy at a scientific conference in early 2023. According to government data, the estimated prevalence of hemophilia in the United States is 12 cases per 100,000 males for hemophilia A and 3.7 cases per 100,000 males for hemophilia B. In November, the U.S. health regulator approved the first gene therapy, CSL Ltd and uniQure's Hemgenix, to treat hemophilia B.Pfizer is also testing other experimental gene therapies in late-stage trials as potential treatments for the bleeding disorder hemophilia A and muscular disorder Duchenne muscular dystrophy.

☆ Health-care stocks are looking good for 2023 and not just because the sector is a 'safe haven'
▼ + stars: | 2022-12-21 | by ( Christina Cheddar Berk | ) www.cnbc.com time to read: +9 min

Investors looking for cover this year would have done well if they sought a safe haven in large-cap pharmaceutical stocks, a trend that is likely to continue into 2023. The Inflation Reduction Act provided some clarity around drug pricing that should help health-care stocks. The average price target, according to FactSet, is $62, or nearly 40% above the stock's closing price on Tuesday. Gene therapies in focus Investors will be closely watching the progress of several gene therapies, according to Phipps. "Yes, these are expensive therapies," Phipps said.

☆ EU regulator backs gene therapy to treat rare bleeding disorder
▼ + stars: | 2022-12-16 | by ( ) www.reuters.com time to read: +1 min

Companies CSL Ltd FollowUniqure NV FollowDec 16 (Reuters) - The European Union's health regulator on Friday said it had recommended granting a conditional marketing authorisation for a gene therapy by Australian drugmaker CSL Ltd (CSL.AX) and partner uniQure N.V. , to treat haemophilia B, a rare bleeding disorder which is caused by genetic anomalies. About one in 40,000 people are affected by the inherited disorder, caused by a gene mutation that hampers the body's ability to make clotting protein factor IX. If approved, the treatment, branded Hemgenix, will be the first gene therapy in the European Union for the condition that is usually treated by regular injections of factor IX, the European Medicines Agency said. CSL had acquired exclusive global rights to Hemgenix in May last year from uniQure N.V. Reporting by Amna Karimi in Bengaluru; Editing by Nivedita BhattacharjeeOur Standards: The Thomson Reuters Trust Principles.

Nov 22 (Reuters) - Australian drugmaker CSL Ltd (CSL.AX) on Tuesday set the list price of its one-time gene therapy for hemophilia B at $3.5 million, making it the world's most expensive treatment, following its approval by the U.S. health regulator. The first gene therapy for the rare genetic blood clotting disorder offers a long-term solution for patients as against current treatments from Biogen (BIIB.O), Pfizer (PFE.N) and others that focus on regular infusions. The gene therapy consists of an engineered virus carrying a gene expressed in the liver to produce clotting factor IX. While Zynteglo was priced at $2.8 million, Skysona had a wholesale cost of $3.0 million. CSL shares were up 1% at A$300.62 on the Australian stock exchange, while UniQure stock edged up 0.9% in extended trading.

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