10 Best Life Sciences Stocks To Watch Right Now

10 Best Life Sciences Stocks To Watch Right Now: Sarepta Therapeutics Inc (SRPT)

Sarepta Therapeutics Inc., formerly AVI BioPharma, Inc., incorporated on July 22, 1980, biopharmaceutical company focused on the discovery and development of ribonucleic acid (RNA)-based therapeutics for the treatment of rare and infectious diseases. The Company's product candidates include Eteplirsen, AVI-6002, AVI-6003, and AVI-7100. As of December 31, 2011, the Company primarily focused on advancing the development of its Duchenne muscular dystrophy drug candidates, including its lead product candidate, eteplirsen, which is in a Phase IIb trial. The Company is also focused on developing therapeutics for the treatment of infectious diseases, including its lead infectious disease programs aimed at the development of drug candidates for the Ebola and Marburg hemorrhagic fever viruses. The Company's program focuses on the development of disease-modifying therapeutic candidates for Duchenne muscular dystrophy (DMD). The Company initiated a Phase IIb trial for eteplirsen in August 2011 with an objective of initiating a pivotal trial subsequent to 2011.

The Company is also leveraging the capabilities of its RNA-based technology platforms to develop therapeutics for the treatment of infectious diseases. The Company's RNA-based drug programs are clinically evaluated for the treatment of DMD and have also demonstrated anti-viral activity in infectious diseases such as Ebola, Marburg and H1N1 influenza in certain animal models. The Company's lead product candidates are at various stages of development.

Duchenne Muscular Dystrophy Program

The Company's lead program is designed to address specific gene mutations that result in DMD by forcing the genetic machinery to skip over an adjacent contiguous piece of RNA and, thus, restore the ability of the cell to express a new, truncated but! functional, dystrophin protein.

Eteplirsen is an antisense PMO-based therapeutic in clinical development for the treatmen t of individuals with DMD who have an error in the gene codi! ng for dystrophin that can be treated by skipping exon 51. Eteplirsen targets the frequent series of mutations that cause DMD. Eteplirsen has been granted orphan drug designation in the United States and European Union. In addition to the Company's lead product candidate, eteplirsen, the Company is actively pursues development of a product candidate that skips exon 45 through an IND-enabling collaboration.

Anti-Viral Programs

The Company is implementing its RNA-based technology platforms in its anti-viral programs for the development of therapeutics to treat viruses, such as Ebola, Marburg and influenza. The Company's arrangement with DoD supporting the development of the Company's Ebola and Marburg virus drug candidates provides funding for all clinical and licensure activities necessary to obtain approval of a New Drug Application (NDA), by the United States Food and Drug Administration (FDA), if DoD exercises all of its options under the arrange ment. During the year ended December 31, 2011, the Company paused its clinical development efforts on AVI-7100 and is exploring funding opportunities or partnerships with DHHS and industry collaborators to advance its development.

The Company's anti-viral therapeutic programs use the Company's translation suppression technology and applies its PMOplus chemistry backbone, an advanced generation of its base PMO chemistry backbone that selectively introduces positive backbone charges to improve selective interaction between the drug and its target. The Company's translation suppressing technology is based on Translation Suppressing Oligomers (TSOs), which are PMO-based compounds that stop or suppress the translation of a specific protein by binding to their specific target sequence in mRNA.

The Company is pursuing dev! elopment ! and regulatory approval of its Ebola and Marburg hemorrhagic fever virus product candidates under the FDA's Animal Rule. The Co mpany's lead product candidate against the Ebola virus infec! tion is A! VI-6002. For Marburg virus infection, the Company's lead product candidate has been AVI-6003. In February 2012, the Company announced that the Company received approval from the FDA to remove one of the two oligomers composing AVI-6003 and proceed with a single oligomer approach, AVI-7288, given that efficacy in non-human primates has been demonstrated to be attributable to this single oligomer. The Company is exploring the feasibility of alternate routes of administration of its Ebola and Marburg drug candidates, and at DoD's invitation, the Company is developing a proposal to be submitted for a study to demonstrates feasibility of the intramuscular route.

AVI-6002, which is a combination of AVI-7537 and AVI-7539, is designed for post-exposure prophylaxis after documented or suspected exposure to the Ebola virus. The Company is evaluating the feasibility of developing AVI-7537 as a single agent for the post-exposure prophylaxis after documented or suspected exp osure to Ebola virus. AVI-6003, which is a combination of AVI-7287 and AVI-7288, is designed for post-exposure prophylaxis after documented or suspected exposure to Marburg virus. In February 2012, the Company announced that the Company received approval from the FDA to proceed with AVI-7288 as a single agent against Marburg virus infection. The Company intends to proceed with dosing AVI-7288 in the Phase I multiple ascending dose studies and in non-human primate studies.

Influenza Program

The Company's anti-viral therapeutic programs are also focused on the development of the Company's product candidates designed to treat pandemic influenza viruses. AVI-7100 is the Company's lead product candidate for the treatment of influenza and employs its PMOplus technology. In June 2011, the Company initiated dosing! of AVI-7! 100 through intravenous infusion in single-ascending doses in up to 48 healthy adult volunteers. As of December 31, 2011, the Company pau sed its clinical development efforts on AVI-7100 and are exp! loring fu! nding opportunities or partnerships to advance its development.

The Company has developed three new phosphorodiamidate-linked morpholino oligomers (PMO)-based chemistry platforms in addition to its original PMO-based technology. The Company's PMO-based molecules are designed to sterically block the access of cellular machinery to pre-mRNA and mRNA without degrading the RNA. Through this selective targeting, two distinct biologic mechanisms of action can be initiated: modulation of pre-mRNA splicing and inhibition of mRNA translation.

The Company competes with GlaxoSmithKline plc, Toyama Chemical, Alnylam Pharmaceuticals, Inc., Tekmira Pharmaceuticals Corp., Isis Pharmaceuticals, Inc., Prosensa, and Santaris Pharma A/S.

Advisors' Opinion:

• [By Rich Bieglmeier]

  It's not every day that a stock is up 40% but still might have another 50%+ to go. However, that is the case with Sarepta Therapeutics Inc. (NASDAQ:SRPT).

• [By Ben Levisohn]

  Shares of Sarepta Therapeutics (SRPT) and Plug Power (PLUG) are falling in after-hours trading after the two companies announced plans to sell more shares.

  Sarepta Therapeutics said it plans to sell as much as $100 million in new shares and also granted underwriters a 30-day option to buy as much as $15 million more. Plug Power announced a sale, but said the amount would depend on “market and other conditions.”

  Shares of Sarepta Therapeutics have fallen 4.5% to $38.79 at 4:49 p.m. in after-hours trading, while Plug Power has tumbled 8.7% to $6.12.

• [By Ben Levisohn]

  Shares of Sarepta Therapeutics (SRPT) have surged more than 40% today after the biotech company said that the FDA would consider one of its drugs sooner than expected. The Wa! ll Street! Journal explains:

  Sarepta Therapeutics Inc. said Monday that it would submit a new drug application to the U.S. Food and Drug Administration by the end of the year for its experimental treatment for a fatal degenerative disease that afflicts children.

  Shares surged more than 50% in early trading as the news seemed to mark a reversal in the company’s dealings with the FDA.

  Late last year, the agency suggested that the company needed to do a larger, late-stage study for eteplirsen, which treats Duchenne muscular dystrophy treatment, a fatal disease that destroys muscle tissue in children and afflicts about one in every 3,500 boys born across the globe, according to Sarepta.

  Leerink’s Joseph Schwartz and Paul Matteis are “intrigued” by the FDA’s decision:

  We are intrigued that the FDA is allowing [Sarepta] to start a confirmatory eteplirsen study with an open-label design, although we are cautious that the FDA is also encouraging the company to start placebo-controlled studies for other exon-skippers as soon as possible in order to build the case for eteplirsen approval. We are cautious that the FDA will have the same concerns that were highlighted in today’s press release when it reviews eteplirsen in 2015, and may opt to continue to wait for more controlled data to be convinced. Importantly, the FDA believes it is not appropriate to exclude patients who lose ambulation while on drug from the analysis (modified intent-to- treat), which has been key for eteplirsen to compare favorably to historical control (which includes such patients). The FDA has shown a willingness to be patient and wait for more compelling/controlled data before approving eteplirsen so we see the risk that the FDA continues to do so beyo

source from Top Stocks For 2015:http://www.topstocksblog.com/10-best-life-sciences-stocks-to-watch-right-now.html