After reporting data from a phase 1/2 clinical trial, shares of ProQR Therapeutics N.V. (NASDAQ:PRQR), a clinical-stage biotech focused on RNA medicines that treat rare genetic diseases, rose 70% as of 12:13 p.m. EDT on Wednesday.
So whatProQR reported results from its phase 1/2 trial that is testing its compound called QR-110 as a hopeful treatment for LCA10, which is a genetic mutation that usually leads to childhood blindness.
Image source: Getty Images.
Here are the key takeaways from the study:
Use of QR-110 led to improvements in vision of the majority of patients when measured using visual acuity and the mobility course performance tests. Specifically, about 60% of patients showed a clinically meaningful response to the drug after three months of treatment. A secondary study also showed that the efficacy effects of the drug persisted beyond three months. The drug was well-tolerated by study participants, and no serious adverse events were reported. A pivotal phase 2/3 trial is set to start in the first half of 2019.Commenting on the study results, Dr. David Rodman, an executive vice president of R&D at ProQR, stated:
Hot Biotech Stocks To Buy For 2019: Alnylam Pharmaceuticals Inc.(ALNY)
Advisors' Opinion:- [By Logan Wallace]
Alnylam Pharmaceuticals (NASDAQ:ALNY) was downgraded by analysts at Zacks Investment Research from a hold rating to a sell rating. According to Zacks, “Although Alnylam has a broad and promising pipeline, we note that most candidates are in mid stages of development. These candidates still have a long way to go before hitting the market. The company relies highly on collaborators for funding. Any development/regulatory setback would be a negative for the company. However, Alnylam reported positive data from the ATLAS study in the first quarter which led to regulatory filings for its late-stage pipeline candidate patisiran and the FDA set an action date of Aug 11, 2018. The company along with its partners Sanofi and The Medicines Company, restarted fitusiran's ATLAS phase III study and advanced inclisiran in the ORION-9, -10, and -11 phase III studies, respectively, with results expected for both programs in 2019. Alnylam expects to achieve the profile of three marketed products by the end of 2020.”
- [By Joseph Griffin]
BidaskClub lowered shares of Alnylam Pharmaceuticals (NASDAQ:ALNY) from a strong-buy rating to a buy rating in a research report released on Monday.
- [By Keith Speights]
Speaking of competition, Ionis should have its hands full battling rivals for Tegsedi assuming the drug wins approval. Alnylam (NASDAQ:ALNY) anticipates winning FDA approval for its hATTR drug patisiran within a few weeks. Because the FDA delayed its decision on Tegsedi, Alnylam appears to be in position to reach the market first. In addition to its first-mover advantage, patisiran appears to have an edge over Tegsedi in efficacy and safety based on clinical data for the two drugs.
- [By Shane Hupp]
Get a free copy of the Zacks research report on Alnylam Pharmaceuticals (ALNY)
For more information about research offerings from Zacks Investment Research, visit Zacks.com
Hot Biotech Stocks To Buy For 2019: Amgen Inc.(AMGN)
Advisors' Opinion:- [By Logan Wallace]
Philadelphia Trust Co. decreased its holdings in Amgen, Inc. (NASDAQ:AMGN) by 8.0% during the 2nd quarter, according to the company in its most recent disclosure with the SEC. The institutional investor owned 74,676 shares of the medical research company’s stock after selling 6,501 shares during the quarter. Amgen makes up 1.2% of Philadelphia Trust Co.’s portfolio, making the stock its 22nd biggest position. Philadelphia Trust Co.’s holdings in Amgen were worth $13,784,000 as of its most recent SEC filing.
- [By Keith Speights]
Amgen Inc. (NASDAQ:AMGN) and Pfizer Inc. (NYSE:PFE) actually have quite a bit in common. They co-market one of the world's best-selling drugs, Enbrel. They both face some headwinds with declining sales of older drugs. Each company has promising new drugs. And both stocks are up by nearly the same percentage over the last 12 months.
- [By Chris Lange]
Amgen Inc. (NASDAQ: AMGN) saw its short interest fall to 9.62 million shares from the previous level of 9.79 million. Shares were last seen at $178.26, in a 52-week trading range of $153.56 to $201.23.
- [By Keith Speights]
Gilead Sciences (NASDAQ:GILD), Amgen (NASDAQ:AMGN), and Johnson & Johnson (NYSE:JNJ) rank as the three top biopharmaceutical companies when it comes to cash stockpiles. Here's what these drugmakers are most likely to do with all that money -- and whether or not you should consider investing some of your hard-earned cash to buy their stocks.
- [By Stephan Byrd]
Get a free copy of the Zacks research report on Amgen (AMGN)
For more information about research offerings from Zacks Investment Research, visit Zacks.com
Hot Biotech Stocks To Buy For 2019: ArQule Inc.(ARQL)
Advisors' Opinion:- [By Stephan Byrd]
Get a free copy of the Zacks research report on ArQule (ARQL)
For more information about research offerings from Zacks Investment Research, visit Zacks.com
- [By Logan Wallace]
ValuEngine downgraded shares of ArQule (NASDAQ:ARQL) from a strong-buy rating to a buy rating in a research report sent to investors on Saturday.
Several other brokerages also recently issued reports on ARQL. Zacks Investment Research upgraded shares of ArQule from a hold rating to a buy rating and set a $2.75 target price for the company in a research note on Tuesday, May 8th. B. Riley set a $4.00 target price on shares of ArQule and gave the company a buy rating in a research note on Monday, March 26th. Roth Capital raised their target price on shares of ArQule from $5.00 to $6.00 and gave the company a buy rating in a research note on Tuesday, April 17th. BidaskClub upgraded shares of ArQule from a hold rating to a buy rating in a research note on Saturday, May 19th. Finally, Leerink Swann upgraded shares of ArQule from a market perform rating to an outperform rating in a research note on Thursday, April 5th. One research analyst has rated the stock with a sell rating, six have issued a buy rating and one has issued a strong buy rating to the company. The company has an average rating of Buy and a consensus price target of $5.35.
- [By Joseph Griffin]
ArQule (NASDAQ:ARQL)‘s stock had its “buy” rating restated by equities researchers at Needham & Company LLC in a research report issued to clients and investors on Tuesday, Marketbeat Ratings reports. They currently have a $6.00 price target on the biotechnology company’s stock, up from their prior price target of $5.00. Needham & Company LLC’s price target suggests a potential upside of 134.38% from the company’s previous close.
Hot Biotech Stocks To Buy For 2019: Biogen Idec Inc(BIIB)
Advisors' Opinion:- [By Dan Caplinger]
The iShares biotech ETF has a structure that's familiar to anyone who invests regularly in exchange-traded funds. The ETF tracks an index of nearly 200 biotech and pharmaceutical stocks, with roughly 80% of assets dedicated to true biotechs and the rest split evenly between pharma and life sciences equipment and services providers. Top ETF holdings Biogen (NASDAQ:BIIB), Amgen (NASDAQ:AMGN), and Gilead Sciences (NASDAQ:GILD) make up a total of roughly 25% of the fund's assets.
- [By Dan Caplinger]
Wall Street continued its downward streak on Monday, with the Dow Jones Industrial Average falling more than 100 points. Most major benchmarks fell more modestly, with a few actually poking into positive territory on the day. Trade-sensitive stocks were among the weakest as investors focused on uncertainty related to tariff disputes between the U.S. and China. But for some other companies, bad news of a different sort was responsible for the drops in their shares. Biogen (NASDAQ:BIIB), Baytex Energy (NYSE:BTE), and Catalyst Biosciences (NASDAQ:CBIO) were among the worst performers on the day. Here's why they did so poorly.
- [By Chris Lange]
Ionis Pharmaceuticals Inc. (NASDAQ: IONS) shares made a handy gain on Friday after the firm announced an expanded strategic collaboration with Biogen Inc. (NASDAQ: BIIB). Through this partnership, these companies are planning to tackle and develop novel antisense drug candidates for a broad range of neurological diseases.
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