Top Biotech Stocks To Watch Right Now

Amgen (AMGN) is among the hottest large-cap biotech stocks of this year. The stock has been following an upward trend since last April. Most recently, it rallied past the previous 52-week highs. After this run, the stock even surpassed the 1-year target estimates of many analysts. While it still looks cheap based on forward P/E ratio, there are growing concerns among the investors. Traders are becoming more apprehensive about a looming correction. I think a modest correction is possible. However, the company can be a good long-term investment. Here, I look at the company's financial profile and its business prospects to prove my point.

Stock Performance at a Glance

Amgen and its close peer Gilead (GILD) have followed similar trends this year. The price charts of both companies show that the bullish momentum started from the mid year of 2012. As companies followed a steady upward trend, a sharp correction is less likely to happen. Should the prices enter the correction stage the decrease will be gradual, as well.

5 Best Value Stocks For 2015: PTC Therapeutics Inc (PTCT)

PTC Therapeutics, Inc., incorporated on March 31, 1998 , is a biopharmaceutical company focused on the discovery and development of orally administered, small-molecule drugs that target post-transcriptional control processes. The Company�� lead product candidate includes ataluren, which is used for the treatment of patients with genetic disorders that arise from a type of genetic mutation known as a nonsense mutation. Ataluren is in late stage clinical development for the treatment of Duchenne muscular dystrophy caused by nonsense mutations (nmDMD) and cystic fibrosis caused by nonsense mutations (nmCF).

Ataluren is orally administered small-molecule compound that targets nonsense mutations. The Company is engaged in the development of ataluren for the treatment of genetic disorders in, which a nonsense mutation is the cause of the disease. Genetic tests are available for many genetic disorders, including Duchenne muscular dystrophy and cystic fibrosis, to determine if the underlying cause is a nonsense mutation. The EMA has designated ataluren as an orphan medicinal product for the treatment of nmDMD and nmCF. During the year ended December 31, 2012, the Phase III clinical trial completed. The Company�� Ataluren clinical trials in patients with nonsense mutation genetic disorders include Ataluren for nmDMD: Phase 2b clinical trial complete; Confirmatory Phase III clinical trial initiated, and Ataluren for nmCF: Phase III trial completed.

Advisors' Opinion:

• [By John Kell]

  PTC Therapeutics Inc.(PTCT) said the European Medicines Agency’s Committee for Medicinal Products for Human Use issued a negative opinion on the biopharmaceutical company’s marketing authorization application for its muscular dystrophy treatment. Shares slumped 21% to $20.60 in premarket trading.

• [By WWW.DAILYFINANCE.COM]
  Records fell on Wall Street Friday as another solid report on housing lifted the market for the second day in a row. There's a three-day weekend coming up -- something that often prompts investor caution -- but the gains were broad-based even though volume was fairly light. The VIX, which measures volatility, fell to its lowest level this year. The Dow Jones industrial average (^DJI) gained 63 points, the Nasdaq composite (^IXIC) rose 31, and the Standard & Poor's 500 index (^GPSC) added 8, topping the record high set last week. The Dow Transportation average also raced to an all-time high, lifted by airline stocks. United (UAL) soared more than 4 percent; Delta (DAL) gained more than 1 percent and Southwest (LUV) gained 2 percent. Southwest is at an all-time high, up 79 percent from a year ago. New home sales bounced back with a better than expected 6.4 percent increase last month. Lennar (LEN) and D.R. Horton (DHI) both rose 4 percent. Pulte (PHM), Beazer (BZH) and Hovnanian (HOV) also solid posted gains. Earnings continue to drive retail stocks. Gap (GPS) edged higher even though net fell. Foot Locker (FL) gained 1½ percent after topping expectations. GameStop (GME) rose 4 percent. Its net rose, helped by the rollout last year of new Xbox and PlayStation consoles. Zumiez (ZUMZ) rose 5½ percent on an earnings beat. But Aeropostale (ARO) tumbled 24 percent. Its loss widened and sales declined. The retailer continues to struggle with teen fashion trends. Also on the earnings front, TiVo (TIVO) rose 2 percent as it swung to a profit from a year ago loss. It also reported an increase in the number of subscribers. Hewlett-Packard (HPQ) rose 6 percent on news the company plans to eliminate up to 16,000 additional workers in an effort to cut costs. And several stocks extended big moves from yesterday. Best Buy (BBY) rose more than 3 percent for the second straight day after earnings beat expectations. Isis Pharmaceuticals (ISIS) jumped

Top Biotech Stocks To Watch Right Now: Ophthotech Corp (OPHT)

Ophthotech Corporation, incorporated on January 05, 2007, is a biopharmaceutical company specializing in the development of therapeutics to treat diseases of the eye. The Company�� advanced product candidate is Fovista, which the Company is developing for use in combination with anti-VEGF drugs that represent the current standard of care for the treatment of wet age-related macular degeneration (wet AMD). Wet AMD is a serious disease of the central portion of the retina, known as the macula, which is responsible for detailed central vision and color perception. It is characterized by abnormal new blood vessel formation and growth, referred to as neovascularization, which results in blood vessel leakage, retinal distortion and scar formation. If untreated, the progressive retinal damage results in rapid, irreversible and severe vision loss. Wet AMD is the cause of blindness in patients over the age of 55 in the United States and the European Union.

The anti-VEGF market for the treatment of wet AMD consists predominantly of two drugs that are approved for marketing and primarily prescribed for the treatment of wet AMD, Lucentis and Eylea, and off-label use of the cancer therapy Avastin. The use of anti-VEGF drugs has significantly improved visual outcomes for patients with wet AMD who have been treated with these drugs as compared to untreated patients.

Advisors' Opinion:

• [By John Udovich]

  The biotech sector has been pretty exciting this year�with small cap biotech stocks Prana Biotechnology Limited (NASDAQ: PRAN) and TNI BioTech (OTCMKTS: TNIB) having recently produced noteworthy news for investors�while Acceleron Pharma, Inc (NASDAQ: XLRN), Ophthotech (NASDAQ: OPHT) and BIND Therapeutics (NASDAQ: BIND) have just�set term sheets for their upcoming IPOs. Just consider all of the following recent news:

• [By Jake L'Ecuyer]

  Shares of Ophthotech (NASDAQ: OPHT) got a boost, shooting up 25.78 percent to $39.54 after the company reported that it has entered into an ex-US licensing and commercialization deal with Novartis Pharmaceuticals.

• [By John Udovich]

  If you have not been watching the biotech sector lately, you should start paying attention as the sector along with small cap biotech stocks like Cell Therapeutics Inc (NASDAQ: CTIC), BIND Therapeutics Inc (NASDAQ: BIND) and TNI BioTech (OTCMKTS: TNIB) continue to produce a steady stream of good news for investors thanks to positive industry trends. Moreover, Ophthotech Corp (NASDAQ: OPHT), Foundation Medicine Inc (NASDAQ: FMI), Evoke Pharma and Fate Therapeutics Inc (NASDAQ: FATE) are this week's biotech IPOs that will no doubt be watched closely by Wall Street and industry observers in general. With that in mind, consider the following biotech news or recent articles about the industry and the small cap players in it:

• [By John Udovich]

  Yesterday, small cap biotech Acceleron Pharma Inc (NASDAQ: XLRN) rose 9.76%�plus shares are up 183.6% for retail investors since its September IPO, meaning its worth taking a closer look at the stock along with the performance of other biotech IPOs like BIND Therapeutics Inc (NASDAQ: BIND), Ophthotech Corp (NASDAQ: OPHT) and Foundation Medicine Inc (NASDAQ: FMI) which also debuted at the same time.

Top Biotech Stocks To Watch Right Now: bluebird bio Inc (BLUE)

bluebird bio, Inc., incorporated on April 16, 1992, is a clinical-stage biotechnology company, the Company is focused on transforming the lives of patients with severe genetic and orphan diseases using gene therapy. Gene therapy seeks to introduce a functional copy of the defective gene into a patient�� own cells, a process called gene transfer. Through gene transfer, a functional copy of the mutated gene is delivered to the patient�� cells, thereby correcting the underlying genetic defect that causes aberrant gene expression. As of December 31, 2012, the Company is conducting a Phase I/II clinical study in France evaluating an earlier generation of its LentiGlobin vector for the treatment of �-thalassemia major and SCD. Initial proof-of-concept data from this study were published in Nature. During the year ended December 31, 2013, the Company plans to initiate an extension of this study under a revised protocol for LentiGlobin, which the Company refers to as the HGB-205 Study. The Company also plans o initiate a second Phase I/II clinical program in the United States for LentiGlobin, which the Company refers to as the HGB-204 Study, for �-thalassemia major. In March 2013, the Company entered into a strategic collaboration with Celgene Corporation, or Celgene, to discover, develop and commercialize, disease-altering gene therapies in oncology.

Its gene therapy platform is based on viral vectors that utilize a modified, non-replicating version of the Human Immunodeficiency Virus Type 1 (HIV-1) virus, that has been stripped of all of the components required for it to self-replicate and infect additional cells. The HIV-1 virus is part of the lentivirus family of viruses, as a result of which the Company refer to its vectors as lentiviral vectors. Its lentiviral vectors are used to introduce a functional copy of a gene to the patient�� own isolated blood stem cells, called hematopoietic stem cells (HSCs), which reside in a patient�� bone marrow and are capable of differentiating int! o a wide range of cell types. HSCs are dividing cells, thus its approach allows for sustained expression of the modified gene as the Company is able to take advantage of a lifetime of replication of the gene-modified HSCs. Additionally, the Company has developed a cell-based vector manufacturing process that is both reproducible and scalable.

Adrenoleukodystrophy

Adrenoleukodystrophy is a rare X-linked, inherited, neurological disorder that is often fatal. ALD is caused by mutations in the ABCD1 gene which encodes for a protein called the ALD protein (ALDP), which plays a critical role in the breakdown and metabolism of long-chain fatty acids (VLCFA). Without functional ALDP, VLCFA accumulate in cells including neural cells in which they cause damage to the myelin sheath, a protective and insulating membrane that surrounds nerve cells in the brain. This damage can result in decreased motor coordination and function, visual and hearing disturbances, the loss of cognitive function, dementia, seizures, adrenal dysfunction and other complications, including death. ALD is divided into various sub-segments with three main phenotypes that impact brain function: CCALD (Childhood cerebral adrenoleukodystrophy, AMN (Adrenomyeloneuropathy) and ACALD (Adult Cerebral ALD).

�-thalassemia

�-thalassemia is a rare hereditary blood disorder caused by a genetic abnormality of the �-globin gene resulting in defective red blood cells (RBCs). Genetic mutations cause the absence or reduced production of the beta chains of hemoglobin, or �-globin, thereby preventing the proper formation of hemoglobin A, which normally accounts for greater than 95% of the hemoglobin in the blood of adults. Hemoglobin is an iron-containing protein in the blood that carries oxygen from the respiratory organs to the rest of the body. Hemoglobin A consists of four chains-two chains each of a-globin and �-globin. Normally existing at an approximate 1:1 ratio, genetic mutations that impair t! he produc! tion of �-globin can lead to a relative excess of a-globin, premature death of red blood cells. The clinical implications of the a-globin/�-globin imbalance are two-fold: first, patients lack sufficient RBCs and hemoglobin to effectively transport oxygen throughout the body and can become severely anemic; and second, the shortened life span and ineffective production of RBCs can lead to other complications such as splenomegaly, marrow expansion, bone deformities, and iron overload in organs.

Sickle cell disease

Sickle cell disease (SCD) is a hereditary blood disorder resulting from a mutation in the �-globin gene that causes polymerization of hemoglobin proteins and abnormal red blood cell function. The disease is characterized by anemia, vaso-occlusive pain crisis (a common complication of SCD in which there is severe pain due to obstructed blood flow in the bones, joints, lungs, liver, spleen, kidney, eye, or central nervous system), infections, stroke, overall poor life and early death in a subset of patients. Under low-oxygen conditions, which are exacerbated by the red blood cell abnormalities, the mutant hemoglobin aggregates causing the RBCs to take on a sickle shape (sickle cells), which causes them to aggregate and obstruct small blood vessels, thereby restricting blood flow to organs resulting in pain, cell death and organ damage. If oxygen levels are restored, the hemoglobin can disaggregate and the RBCs return to their normal shape, but over time, the sickling damages the cell membrane and the cells fail to return to the normal shape even in high-oxygen conditions.

Advisors' Opinion:

• [By Garrett Cook]

  Bluebird Bio (NASDAQ: BLUE) shares shot up 34.74 percent to $35.15 following the presentation of positive data on LentiGlobin BB305 at the European Hematology Association (EHA).

Top Biotech Stocks To Watch Right Now: Agios Pharmaceuticals Inc (AGIO)

Agios Pharmaceuticals, Inc., incorporated on August 7, 2007, is a biopharmaceutical company. The Company is intend to apply its deep understanding of metabolism, coupled with the Company�� ability to create medicines that can inhibit or activate metabolic enzymes, to fundamentally change the way cancer and inborn errors of metabolism (IEMs) are treated. The Company has identified and validated novel and druggable targets in both cancer and IEMs. The Company�� two advanced cancer programs are targeting mutations in the enzymes isocitrate dehydrogenase 1 and 2, referred to as IDH1 and IDH2. The Company�� drug candidates are selective for the mutated forms of IDH1 and IDH2 found in cancer cells versus the normal forms of IDH1 and IDH2 found in all other cells.

The Company focused on developing medicines to address IEMs, with a novel approach to these orphan diseases for which no effective or disease-modifying therapy is available. The Company has also de-validated and terminated numerous programs, including many that have been reported in scientific journals. In the Company�� IEM portfolio, it uses an equally rigorous set of validation techniques.

Advisors' Opinion:

• [By Ben Levisohn]

  3. We see 23+ partnered pipeline assets and are most positive on Phase I/II MOR202 anti-CD38 for myeloma with data in 2015, and others such as Acceleron Pharma (XLRN), Agios Pharmaceuticals (AGIO), Epizyme (EPZM), Concert Pharmaceuticals (CNCE) (just started Phase I for next-gen Revlimid).