In the following video, Motley Fool health-care analyst David Williamson discusses Ariad Pharmaceuticals (NASDAQ: ARIA ) , as the company's stock reverses direction today, up 3% following yesterday's tumble. He notes the results of a boutique institutional research survey that found Ariad's new drug Iclusig to have toxicity concerns, and even more negative information coming from the FDA, citing blood clots and liver toxicity. David tells investors what the potential for this drug is, and whether it has a shot at success on the market despite these safety concerns.
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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).
- [By David Williamson]
In this video, health-care analyst David Williamson takes a look at the tremendous success of the�Bluebird Bio (NASDAQ: BLUE ) �IPO. The company increased the size of its initial public offering, and priced shares at $17 -- above the top end of its range -- but that still couldn't contain investor appetite for this stock. Shares shot up 50% on the opening day of trading, and have remained there.
- [By Garrett Cook]
Bluebird Bio (NASDAQ: BLUE) shares shot up 33.61 percent to $34.86 following the presentation of positive data on LentiGlobin BB305 at the European Hematology Association (EHA).
- [By Jay Silverman]
Some of the biggest leaders in that field, and there have been dozens in fields, if not more this year, such as Bluebird (BLUE) and Stemline Therapeutics (STML) and have all pulled back to significantly lower levels; even below, in Bluebird's case, the price that had actually opened up as an IPO, even though it's above its IPO price.
Top Biotech Stocks To Own Right Now: Genomic Health Inc (GHDX)
Genomic Health, Inc. (Genomic Health), incorporated in August 2000, is a molecular diagnostics company focused on the global development and commercialization of genomic-based clinical laboratory services that analyze the underlying biology of cancer allowing physicians and patients to make individualized treatment decisions. Its Oncotype DX platform utilizes quantitative genomic analysis known as reverse transcription polymerase chain reaction (RT-PCR), in standard tumor pathology specimens to provide tumor-specific information, or the oncotype of a tumor. As of February 2012, Oncotype DX was evaluated in invasive breast cancer in 13 clinical studies involving more than 4,000 breast cancer patients worldwide. Genomic Health offers its Oncotype DX tests as a clinical service, where it analyzes the expression levels of genes in tumor tissue samples and provide physicians with a quantitative gene expression profile expressed as a single quantitative score, which it calls a Recurrence Score, for invasive breast cancer and colon cancer and a DCIS Score for DCIS. Its Oncotype DX breast cancer test analyzes the expression levels of 21 genes and Oncotype DX colon cancer test analyzes the expression levels of 12 genes. In March 2012, the Company established a wholly owned subsidiary, InVitae Corporation.
Oncotype DX Platform
The Company�� Oncotype DX platform uses its RT-PCR approach to improve cancer treatment decisions. Its diagnostic approach correlates gene expression to clinical outcomes and provides an individualized analysis of each patient's tumor. The Company has built a diagnostic infrastructure that allows it to move from research into development through to processing actual patient samples in its clinical reference laboratory. The Company offers Oncotype DX tests as clinical laboratory services. Its technology allows the Company to analyze tumor tissue samples in its clinical reference laboratory and provide physicians with genomic information specific to the patient'! s tumor. It analyzes tissues that are handled, processed and stored under routine clinical pathology laboratory practices.
Oncotype DX Breast Cancer Test
To develop its Oncotype DX breast cancer test, the Company evaluated 250 genes in three independent clinical studies, which identified a 21-gene panel whose composite gene expression profile can be represented by a breast cancer Recurrence Score. The Company conducted studies of its Oncotype DX breast cancer test with clinical samples from postmenopausal women with invasive breast cancer who were treated with aromatase inhibitors. In March 2010, the Journal of Clinical Oncology published results from a European study using its test to analyze tumor samples from over 1,200 patients in the ATAC (Arimedix, Tamoxifen, Alone or in Combination) trial, which established the use of aromatase inhibitors for adjuvant treatment of postmenopausal women with hormone receptor-positive breast cancer. The study demonstrated that, along with other standard measures, such as tumor size, its Oncotype DX breast cancer test contributes independently to provide a more complete picture of prognosis for N- and N+ patients treated with aromatase inhibitors.
In December 2011, the Company presented positive results from the ECOG E5194 DCIS clinical validation study at SABCS. The study met its primary endpoint by demonstrating that a pre-specified Oncotype DX DCIS Score can predict the risk of local recurrence, defined as either the development of a new invasive breast cancer or the recurrence of DCIS in the same breast. In December 2011, the Company made Oncotype DX available for patients with ductal carcinoma in situ (DCIS), of the breast, a pre-invasive form of breast cancer. The launch of Oncotype DX for DCIS patients was based on positive results presented from a clinical validation study of Oncotype DX breast cancer test in patients with DCIS, conducted by the Eastern Cooperative Oncology Group (ECOG), a clinical trials cooperative! group su! pported by the National Cancer Institute.
Oncotype DX Colon Cancer Test
The Company developed its gene panel by identifying 761 cancer-related genes through review of existing research literature and computer analysis of genomic databases. The 761 candidate genes were also examined to determine whether they would be useful beyond other key variables including tumor stage, tumor grade, lymph nodes examined and MMR/MSI. It selected a final set of 12 genes, which were then independently evaluated in a validation study of over 1,400 stage II colon cancer patients from the Quick and Simple and Reliable (QUASAR), randomized study of adjuvant chemotherapy in the United Kingdom. This international, multi-center randomized trial examined the recurrence risk and the benefit associated with 5-fluorouracil/leucovorin, or 5FU/LV, adjuvant chemotherapy. Gene expression was quantified by RT-PCR from manually microdissected FPE primary colon cancer tissue, and recurrence-free interval, disease-free survival and overall survival were analyzed. In January 2012, the Company presented positive results of the first clinical decision making study of the Oncotype DX colon cancer test that shows that Recurrence Score result has a significant impact on treatment recommendations for stage II colon cancer patients.
The Company competes with General Electric Company, Hologic, Inc., Novartis AG, Myriad Genetics, Inc., Qiagen N.V., Response Genetics, Inc., Laboratory Corporation of America Holdings, Quest Diagnostics Incorporated, Roche Holding, Ltd, Siemens AG and Johnson & Johnson.
Advisors' Opinion: - [By Sean Williams]
What: Shares of Genomic Health (NASDAQ: GHDX ) , a provider of genome-based clinical diagnostic tests, popped as much as 10% following the release of its first-quarter results.
- [By John Udovich]
Small cap cancer diagnostic stock�Myriad Genetics, Inc (NASDAQ: MYGN) jumped to a five year high on Wednesday�thanks to good�news that the�government will be paying a heftier reimbursement for its main test than previously feared, meaning its worth taking a closer look at the stock along with small cap cancer diagnostic stocks like Rosetta Genomics Ltd (NASDAQ: ROSG) and�Genomic Health, Inc (NASDAQ: GHDX) plus mid cap diagnostic stock Quest Diagnostics Inc (NYSE: DGX). I should also mention that Myriad Genetics has been in our SmallCap Network Elite Opportunity (SCN EO) portfolio since early February and we are now up some 32.5% since then ��thanks in part to the shorts being put in a short squeeze.
- [By John Udovich]
Small cap genomics stocks Rosetta Genomics Ltd (NASDAQ: ROSG), Genomic Health, Inc (NASDAQ: GHDX) and CollabRx Inc (NASDAQ: CLRX) are at the forefront of genomics testing�or research. I should mention that Wikipedia defines genomics�as a�discipline in genetics that applies recombinant DNA, DNA sequencing methods and bioinformatics to sequence, assemble and analyze the function and structure of genomes (the complete set of DNA within a single cell of an organism). A 2013 Booz Allen Hamilton report on the sector noted that in�2011, the US genetic and genomic clinical testing market size was estimated to be $5.9 billion while the�number of available tests has grown substantially from 1,680 just 4 years ago to 2,886 in 2012.
- [By Keith Speights]
Genetic testing stands out as one current example of how scientific advances can help reduce medical costs. Genomic Health (NASDAQ: GHDX ) makes genetic diagnostic tests for�breast, colon, and prostate cancer. Half of the patients diagnosed with prostate cancer each year actually have a very low risk of the cancer progressing. However, 90% of these low-risk patients still undergo surgery or radiation -- at a cost of tens of thousands of dollars per patient. Genomic Health's genetic test helps identify which patients really need more extensive treatment and holds the potential to reduce overall costs.
Top Biotech Stocks To Own Right Now: Insys Therapeutics Inc (INSY)
Insys Therapeutics, Inc., incorporated on June 15, 1990, is a pharmaceutical company that develops and seeks to commercialize pharmaceutical products that target the unmet needs of cancer patients, with an initial focus on cancer-supportive care. The Company�� pharmaceuticals portfolio consists of one approved product and a number of product candidates targeting cancer-supportive care and cancer therapy. The Company�� product candidate includes Subsys, Dronabinol SG Capsule, Dronabinol RT Capsule, Dronabinol Oral Solution, Dronabinol Inhalation Device, and Dronabinol IV Solution. The Company is also developing cancer therapeutics, which is LEP-ETU, a formulation of paclitaxel, the active ingredient in the cancer drugs Taxol and Abraxane. On August 19, 2011, the Food & Drug Administration (FDA) approved its Dronabinol SG Capsule product, a generic equivalent to Marinol, for the treatment of chemotherapy induced nausea and vomiting (CINV), and anorexia associated with weight loss in patients with acquired immune deficiency syndrome (AIDS).
Subsys
The Company's Subsys is a single-use product that delivers fentanyl, an opioid analgesic, in seconds for transmucosal absorption underneath the tongue. Subsys is a transmucosal product to show pain relief when measuring the sum of pain intensity difference at five minutes in a Phase 3 breakthrough cancer pain (BTCP) clinical trial using fentanyl.
Dronabinol Product Family
The Company has an approved dronabinol product and is developing several dronabinol product candidates for the treatment of CINV and appetite stimulation in patients with AIDS, as well as other indications where dronabinol could have potential therapeutic benefits. Dronabinol, the active ingredient in Marinol, is a synthetic cannabinoid whose chemical name is delta-9-tetrahydrocannabinol (THC). Its portfolio consists of its Dronabinol SG Capsule product and Dronabinol RT Capsule product candidate, which are intended to be generic equi! valents to Marinol, in addition to three formulations, including Dronabinol Oral Solution. Dronabinol SG Capsule is a dronabinol soft gelatin capsule intended to be a generic equivalent to Marinol. Dronabinol RT Capsule is a dronabinol soft gel capsule that is stable at room temperature. Dronabinol Oral Solution is a ynthetic THC in an oral liquid formulation.
Cancer Therapeutics
In addition to its cancer-supportive care products, the Company intends to develop cancer therapeutics targeting limitations of existing commercial products. LEP-ETU, it advanced cancer therapeutic, is a NeoLipid liposomal, or microscopic membrane-like structure created from lipids, formulation that incorporates paclitaxel. LEP-ETU completed a Phase 2 clinical trial of 70 patients with metastatic breast cancer.
The Company competes with Cephalon, Inc., BioDelivery Sciences International, Inc., ProStrakan Group plc, Nycomed International Management GmbH, Archimedes Pharma Ltd., TEVA Pharmaceuticals USA, Watson Pharmaceuticals, Inc., AcelRx Pharmaceuticals, Inc., Akela Pharma Inc., Abbott Laboratories, Pharmaceutical International, Inc., Par Pharmaceutical Companies Inc., sanofi-aventis, Eisai Inc., Helsinn Group, Roche Holding AG, Par Pharmaceutical Companies Inc., GlaxoSmithKline plc, ProStrakan Group plc, Merck & Co, GW Pharmaceutical, A.P. Pharma, Inc., Aphios Corp., Roche Holding, Tesaro, Inc., Cornerstone Pharmaceutical, Inc., Bristol-Myers Squibb, Celgene Corporation, Laboratories, Amgen Inc., AstraZeneca PLC., Bayer AG, Biogen Idec Inc., Eisai Co., Ltd., F. Hoffmann- LaRoche Ltd., Johnson and Johnson, Merck and Co., Inc., Novartis AG, Onyx Pharmaceuticals Inc., Pfizer Inc., and Takeda Pharmaceutical Co. Ltd.
Advisors' Opinion: - [By David Zeiler]
2. Insys Therapeutics Inc. (Nasdaq: INSY): Insys is a biotech seeking to capitalize on the growing interest in medical marijuana by using a generic form of THC to create drugs to treat cancer pain. INSY had its IPO May 2 with an offer price of $8 a share. The stock rose 19.75% on its first day of trading. But investors really warmed up to Insys later; it currently trades at about $37.54, a 369.25% increase over the offer price.
- [By Louis Navellier]
INSYS Therapeutics (INSY) is a biotech company that focuses on developing pharmaceutical products that target the unmet needs of cancer patients, with a focus on cancer-supportive care. Their products include drugs that help patients deal with pain form the disease and help alleviate nausea and pain from chemotherapy. The company reported earnings this week and both profits and sales have exploded higher.
- [By Chris Preston]
INSYS Therapeutics (INSY) is one recent IPO that jumps out. The Arizona-based pharmaceutical company markets a synthetic marijuana drug to treat cancer pain. It went public in May at $8 per share. It opened at over $46 per share.
Top Biotech Stocks To Own Right Now: Amgen Inc.(AMGN)
Amgen Inc., a biotechnology medicines company, discovers, develops, manufactures, and markets human therapeutics based on advances in cellular and molecular biology for grievous illnesses primarily in the United States, Europe, and Canada. The company markets recombinant protein therapeutics in supportive cancer care, nephrology, and inflammation. Its principal products include Aranesp and EPOGEN erythropoietic-stimulating agents that stimulate the production of red blood cells; Neulasta and NEUPOGEN to stimulate the production of neutrophils, which is a type of white blood cell that helps the body to fight infections; and Enbrel, an inhibitor of tumor necrosis factor that plays a role in the body?s response to inflammatory diseases. The company also markets other products comprising Sensipar/Mimpara, a small molecule calcimimetic that lowers serum calcium levels; Vectibix, a monoclonal antibody that binds specifically to the epidermal growth factor receptor; and Nplate, a thrombopoietin (TPO) receptor agonist that mimics endogenous TPO, the primary driver of platelet production. In addition, it provides Denosumab, a human monoclonal antibody that targets RANKL, an essential regulator of osteoclasts. Further, the company offers product candidates in mid-to-late stage development in a variety of therapeutic areas, including oncology, hematology, inflammation, bone, nephrology, cardiovascular, and general medicine consisting of neurology. It markets its products to healthcare providers, including physicians or their clinics, dialysis centers, hospitals, and pharmacies; consumers; and wholesale distributors of pharmaceutical products. The company has various collaborative arrangements with Pfizer Inc.; GlaxoSmithKline plc; Takeda Pharmaceutical Company Limited; Daiichi Sankyo Company, Limited; Array BioPharma Inc.; Kyowa Hakko Kirin Co. Ltd.; and Cytokinetics, Inc. Amgen Inc. was founded in 1980 and is headquartered in Thousand Oaks, California.
Advisors' Opinion: - [By Wallace Witkowski]
Shares of Amgen Inc. (AMGN) �fell 0.7% to $119.90 on moderate volume even after the biotech reported adjusted earnings of $1.82 a share on revenue of $5.01 billion, while analysts expected earnings of $1.70 a share on revenue of $4.81 billion.
- [By Efsinvestment]
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.
- [By Jon C. Ogg]
Amgen Inc. (NASDAQ: AMGN)�was raised to Overweight from Neutral at Piper Jaffray.
Best Buy Co. Inc. (NYSE: BBY)�was reiterated as Hold as shares are perceived to be fully valued at Argus.
- [By RichardCox]
Another selection to consider is the Market Vectors Biotech ETF (BBH). The fund has seen returns of a massive 55% since its inception and has a net expense ratio of 0.35%. This fund is centered on fewer stock selections (in contrast to the SPDR S&P Biotech ETF), so there is an enhanced potential for price volatility. Fund holdings include Gilead (GILD), Celgene and Amgen (AMGN), which make up more than 2/3 of the fund. This essentially means investors will sacrifice some of the diversification that can be found in choices like the SPDR S&P Biotech ETF, but many of the individual selections are well-positioned for gains for the remainder of the year.
Top Biotech Stocks To Own Right Now: International Stem Cell Corp (ISCO)
International Stem Cell Corporation, incorporated on June 7, 2005 is a developmental-stage biotechnology company. The Company focused on therapeutic and biomedical product development. The Company�� wholly owned subsidiary Lifeline Cell Technology, LLC (LCT) develops, manufactures and commercializes over 130 human cell culture products, including frozen human primary cells and the reagents (media) needed to grow, maintain and differentiate the cells, in order to address this market opportunity. The Company�� scientists have used a technology called basal medium optimization to systematically produce optimized products designed to culture specific human cell types and to elicit specific cellular behaviors. The Company�� wholly owned subsidiary Lifeline Skin Care, Inc. (LSC) develops, manufactures and markets cosmetic skin care products using an extract derived from its pluripotent stem cells.
Therapeutic Product Candidates
Using the Company's technologies and know-how, the Company is exploring and creating a range of cell types that is useful in therapeutic treatments, which include Liver cells (hepatocytes) that may be used to treat a range of congenital and acquired liver diseases. Neuronal cells for potential treatment of Parkinson's disease and other central nervous system disorders, such as traumatic brain injury, stroke and Alzheimer�� disease. Three-dimensional eye structures to treat degenerative retinal diseases, corneal blindness, and to accelerate corneal healing.
Skin Care Products
The Company�� research scientists developed two skin care products, Defensive Day Serum and Recovery Night Serum, using an extract derived from human parthenogenic stem cells. Defensive Serum contains sunscreen, along with stem cell-derived ingredients. The day serum not only protects the skin from the aging effects of harsh light, but it continues to nurture the skin�� collagen and fibroblasts to give noticeably firmer, smoother, younger-looking ! skin. The Recovery Night Serum is a nighttime therapy that complements the Defensive Day Serum. The night serum nurtures the skin�� collagen and elastin and contains ingredients to defend against damaging free radicals, to help build firmer, smoother, younger and healthier-looking skin.
Research Products
The Company�� Lifeline subsidiary produces and sells over 250 human cell culture products. These products include frozen human cells and stem cells and the reagents needed to grow, maintain and differentiate the cells. These human cell-based products are used domestically and internationally by research scientists in pharmaceutical, academic and government research organizations to study human disease and basic cell biology.
The Company competes with Genzyme Corporation, StemCell, Advanced Cell Technology Inc., Aastrom Biosciences, ViaCyte, Obagi, SkinCeuticals, SkinMedica, Murad, Lonza, Chemicon, Life Technologies, StemCell Technologies, Merck, BioTime and Specialty Media.
Advisors' Opinion: - [By John Udovich]
The results of a recent Pew Center Poll regarding attitudes towards abortion and various forms of stem cell research could be a good sign for the stem cell industry along with small cap stem cell stocks like StemCells Inc (NASDAQ: STEM), NeoStem Inc (NASDAQ: NBS), Neuralstem, Inc (NYSEMKT: CUR),�International Stem Cell Corp (OTCMKTS: ISCO) and BioRestorative Therapies (OTCBB: BRTX). Basically, Americans think that having an abortion is a moral issue with 49% of American adults believing abortion is morally wrong, 23%�view it not as a moral issue and and 15% view it as morally acceptable. However and when Americans were asked about issues surrounding�human embryos, such as stem cell research or in vitro fertilization, as a matter of morality, their views were different.