Top Information Technology Companies To Watch For 2015

Top In formation Technology Companies To Watch For 2015: 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 ne! w, 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 pursu! ing development 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-7100 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 Jayson Derrick]

  The FDA issued a statement related to Sarepta Therpaeutics (NASDAQ: SRPT) eteplirsen drug, noting that that the methods used in the study were not “adequately robust to support an NDA submission” and that it provided the company with recommendations on how to improve its analysis.” Shares gained 2.80 percent, closing at $16.17.

• [By Jayson Derrick]

  Analysts at Baird downgraded Sarepta Therapeutics (NASDAQ: SRPT) to Neutral form Outperform with a price target lowered to $21 from a previous $53. Shares lost 4.97 percent, closing at $15.12.

source from Top Stocks For 2015:http://www.topstocksblog.com/top-information-technology-companies-to-watch-for-2015.html