Top 5 Biotech Companies For 2014

Top 5 Biotech Companies For 2014: Pr osensa Holding NV (RNA)

Prosensa Holding N.V., formerly Prosensa Holding B.V., is a biotechnology company engaged in the discovery and development of ribonucleic acid-modulating (RNA)-modulating, therapeutics for the treatment of genetic disorders. The Company's primary focus is on rare neuromuscular and neurodegenerative disorders with a large unmet medical need, including Duchenne muscular dystrophy, myotonic dystrophy and Huntington's disease. The Company's clinical portfolio of RNA-based product candidates is focused on the treatment of Duchenne muscular dystrophy (DMD). The Company's platform technology allows the development of RNA-modulating therapeutics that either interferes with splicing (exon skipping, exon inclusion, or splice mutation correction), remove mutant RNA, or block RNA expression, for different indications.

DMD is a rare, severe muscle wasting disease that occurs in up to 1 in 3,500 male births. It is commonly diagnosed between the ages of three to five , when boys begin to show signs of impaired motor development. PRO044, the Company's product candidate, addresses a separate sub-population of DMD patients. The Company developed PRO044 using its exon-skipping technology to generate a product candidate with the same mechanism of action that is used by drisapersen.

Advisors' Opinion:

• [By Monica Gerson]

  Prosensa Holding NV (NASDAQ: RNA) soared 25.89% to $8.51 in the pre-market trading after the company reported 48-week data from Phase 2 placebo-controlled study of drisapersen in 51 DMD boys.

• [By Leo Sun]

  However, these three biotech stocks -- Amarin (NASDAQ: AMRN  ) , Dendreon (NASDAQ: DNDN  ) , Prosensa (NASDAQ: RNA  ) -- don't fit that category at all. Read on to understand why these dogs of the sector should never be mistaken as underdogs.

• [By Sean Williams]

  Shares of Sarepta climbed to a 52-week high of $55.61 in September shortly after an experimental rival drug, drisapersen, developed by Prosensa (NASDAQ: RNA  ) and GlaxoSmithKline (NYSE: GSK  ) , missed its primary end point by a mile in late-stage trials. However, Sarepta also tanked just weeks later after the Food and Drug Administration decided against supporting an accelerated drug approval for eteplirsen; the agency would not make the connection that increased dystrophin production led to its remarkable trial results. Also, given the recent failure of drisapersen in a phase 3 study, the FDA felt it pertinent that Sarepta engage in a broader study. Shares ultimately dipped as low as $12.12. 

• [By John Udovich]

  It should also be mentioned that earlier in the week, GlaxoSmithKline plc (NYSE: GSK) returned full rights of Duchenne muscular dystrophy drug drisapersen to their partner Prosensa Holding NV (NASDAQ: RNA) and that news sent shares down 12.48% on Monday. Moreover, Prosensa Holding also sank some 70% last fall when Drisapersen failed a phase 3 trial by missing a primary endpoint measuring for how far patients could walk over the course of six minutes (the improvement wasn't significantly better than the placebo). However, Prosensa Holding also rose 24.37% yesterday after announcing initial findings from further analyses from the aggregate data for drisapersen, with the CEO saying:

source from Top Stocks Blog:http://www.topstocksblog.com/top-5-biotech-companies-for-2014.html