Friday, October 25, 2013

Hot Biotech Companies To Invest In 2014

It's not easy to reinvent yourself in the biotechnology sector, but that's exactly the path that Alkermes (NASDAQ: ALKS  ) CEO Richard Pops has decided to take his company.

Source: Michael Chen, Flickr.

Over the last half-decade, we've witnessed a huge surge in the number of biotech companies that are geared toward treating orphan diseases (those that affect 200,000 or fewer people). There's absolutely nothing wrong with this given that people with rare diseases need treatment as well, and there can often be big profits in it for companies that put in the time and research dollars.

Alexion Pharmaceuticals (NASDAQ: ALXN  ) , for example, is currently worth more than $21 billion because of its focus on rare diseases. Its lone drug approved by the Food and Drug Administration, Soliris, is the most expensive drug in the world and is approved to treat a rare blood disorder known as paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome. This year alone, Soliris is expected to top $1.5 billion in sales.

Hot Biotech Companies To Invest In 2014: Algeta ASA (ALGETA.OL)

Algeta ASA is a Norway-based biotechnology company engaged in the development of targeted cancer therapies based on its alpha-pharmaceutical platform. The Company�� principal product is Alpharadin for the treatment of bone metastases resulting from castration-resistant prostate cancer. The Company�� pipeline also includes Alpharadin for the treatment of bone metastases resulting from breast cancer, a combination of Alpharadin with Taxotere for the treatment of bone metastases resulting from prostate cancer and Thorium-227 showing various cancer indications. The Company develops Alpharadin in a development and marketing cooperation with Bayer Schering Pharma. Algeta ASA is active through the two wholly owned subsidiaries, Algeta Innovations AS and Algeta UK Limited. On April 12, 2012, the Company announced that it estabilished a subsidiary active in the United States, Algeta US.

Hot Biotech Companies To Invest In 2014: Scancell Holdings PLC (SCLP.L)

Scancell Holdings PLC is a United Kingdom-based company. The Company�� principal activity of the consists of the discovery and development of monoclonal antibodies and vaccines for the treatment of cancer. In April 2012, the Company completed recruitment to the Phase 1 clinical trial of SCIBI. In May 2012, the Company commenced recruitment and treatment of the first patient in the second part of it Phase 1/2 clinical trial of SCIBI. The Phase 2 part of the trial is conducted in five United Kingdom centers in Nottingham, Manchester, Newcastle, Leeds, and Southampton. On August 15, 2012, the Company announced the development of a platform technology, Moditope.

Top 5 Clean Energy Companies For 2014: Neoprobe Corporation(NEOP)

Neoprobe Corporation, a biomedical company, engages in the development and commercialization of precision diagnostics that enhance patient care and improve patient benefit. The company is developing and commercializing targeted agents aimed at the identification of occult (undetected) disease. Neoprobe?s two lead radiopharmaceutical agent platforms, Lymphoseek and RIGScan are intended to help surgeons better identify and treat certain types of cancer. Lymphoseek is a diagnostic imaging agent intended for radiolabeling and administration in radiodetection and visualization of the lymphatic system draining the region of injection for delineation of the lymphatic tissue; and RIGScan is an intraoperative biologic targeting agent consisting of a radiolabeled murine monoclonal antibody. The company has a biopharmaceutical development and supply agreement with Laureate Biopharmaceutical Services, Inc. to support the initial evaluation of the viability of the CC49 master working c ell bank, as well as the initial steps in re-validating the commercial production process for the biologic agent used in RIGScan CR. The company was founded in 1983 and is based in Dublin, Ohio.

Hot Biotech Companies To Invest In 2014: Regeneron Pharmaceuticals Inc.(REGN)

Regeneron Pharmaceuticals, Inc., a biopharmaceutical company, discovers, develops, and commercializes pharmaceutical products for the treatment of serious medical conditions in the United States. The company?s commercial product includes ARCALYST (rilonacept) injection for subcutaneous use for the treatment of cryopyrin-associated periodic syndromes, including familial cold auto-inflammatory syndrome and muckle-wells syndrome in adults and children. Its products under Phase III clinical development stage consist of VEGF Trap-Eye, an aflibercept ophthalmic solution developed using intraocular delivery for the treatment of serious eye diseases; ARCALYST for the prevention of gout flares in patients initiating uric acid-lowering treatment; and Aflibercept (VEGF Trap), which is developed in oncology. The company?s earlier stage clinical programs include various human antibodies, such as REGN727 for low-density lipoprotein cholesterol reduction, REGN88 for rheumatoid arthritis and ankylosing spondylitis; REGN668 for atopic dermatitis and asthma; REGN421 and REGN910 for oncology; REGN475 for the treatment of pain; and REGN728 and REGN846. It also conducts preclinical research programs in the areas of oncology and angiogenesis, ophthalmology, metabolic and related diseases, muscle diseases and disorders, inflammation and immune diseases, bone and cartilage, pain, cardiovascular diseases, and infectious diseases. The company distributes its products through third party service providers. It has strategic collaboration with sanofi-aventis Group to discover, develop, and commercialize human monoclonal antibodies; and Bayer HealthCare LLC to develop and commercialize VEGF Trap. Regeneron Pharmaceuticals, Inc. was founded in 1988 and is based in Tarrytown, New York.

Advisors' Opinion:
  • [By Dan Carroll]

    Fellow major biotech stock Regeneron (NASDAQ: REGN  ) lost 5% during the week, part of a sustained 15% slump over the past month. Like Biogen, however, Regeneron's a company with a drug preparing for a strong future. The company's asthma therapy dupilumab, made jointly with Big Pharma's Sanofi, absolutely crushed a mid-stage clinical trial by paring asthmatic episodes back 87%. While the drug will need to proceed through later-stage trials before reaching the approval stage, things are looking up for this potentially high-flying asthma therapy in a highly lucrative market.

  • [By Sue Chang and Polya Lesova]

    Regeneron Pharmaceuticals Inc. (REGN) �shares extended losses for a third session to shed 3.8%. The shares have been very active in recent weeks due to strong interest in its cholesterol drug alirocumab.

  • [By Ben Levisohn]

    Gilead has gained 0.5% to $60.58 today, while Celgene has risen 1.5% to $142.13 and Biogen has advanced 3.8% to $221.1. Medivation is up 1.9% to $57.60. Biogen and Regeneron (REGN), which has gained 6% to $256.71, are two of the three-best performers in the S&P 500 today.

  • [By John Divine]

    Lastly, shares of Regeneron Pharmaceuticals (NASDAQ: REGN  ) shed 4.1% Friday. Shares in the biopharmaceutical company have more than doubled in the last year, and have been an absolute dream for longer-term shareholders, having been a 10-bagger in the past three years, and trading at more than 15 times their value five years ago. Right now, Wall Street is wondering whether Regeneron's still a bargain at 37 times forward earnings, and today's ruling was decisively negative on a day the health-care sector ended was the second-worst performing area of the markets.

Hot Biotech Companies To Invest In 2014: EntreMed Inc (ENMD.PH)

EntreMed, Inc. (EntreMed), incorporated in 1991, is a clinical-stage pharmaceutical company. EntreMed's drug candidate is ENMD-2076, an Aurora A and angiogenic kinase inhibitor for the treatment of cancer. ENMD-2076 has completed Phase I studies in patients with advanced solid tumors, multiple myeloma and leukemia and is completing data for a multi-center Phase II study in patients with platinum resistant ovarian cancer. The Company�� other product candidates have includes MKC-1, ENMD-1198 and 2-methoxyestrdiol (2ME2, Panzem) for treatment of rheumatoid arthritis.

ENMD-2076 is a novel orally-active, Aurora A/angiogenic kinase inhibitor with potent activity against Aurora A and multiple tyrosine kinases linked to cancer and inflammatory diseases. ENMD-2076 is relatively selective for the Aurora A isoform in comparison to Aurora B. Aurora kinases are key regulators of the process of mitosis, or cell division, and are often over-expressed in human cancers. E NMD-2076 exerts its effects through multiple mechanisms of action, including anti-proliferative activity and the inhibition of angiogenesis. ENMD-2076 has demonstrated significant, dose-dependent preclinical activity as a single agent, including tumor regression, in multiple xenograft models (such as breast, colon, leukemia), as well as activity towards ex vivo-treated human leukemia patient cells.

Hot Biotech Companies To Invest In 2014: Exelixis Inc.(EXEL)

Exelixis, Inc., a biotechnology company, develops small molecule therapies for the treatment of cancer. It focuses on developing Cabozantinib, an inhibitor of tumor growth, metastasis, and angiogenesis that target MET, VEGFR2, and RET, which are key kinases involved in the development and progression of various cancers. The cabozantinib is in Phase III clinical trial for the treatment for medullary thyroid cancer. The company also engages in various clinical programs for cabozantinib focused on the treatment of metastatic castration-resistant prostate cancer, ovarian cancer, breast cancer, renal cell carcinoma, non-small cell lung cancer, hepatocellular cancer, and melanoma. In addition, Exelixis, Inc. involves in developing a portfolio of other novel compounds to address serious unmet medical needs through collaborations with various pharmaceutical and biotechnology companies, including Bristol-Myers Squibb Company, sanofi-aventis, Genentech, Inc., Boehringer Ingelheim Gm bH, and GlaxoSmithKline and Daiichi Sankyo Company Limited. Its products under development through collaborations include XL475, XL281, XL139, and XL413 inhibitors; ROR antagonists; therapies targeted against LXR, a nuclear hormone receptor implicated in various cardiovascular and metabolic disorders; XL147, XL765, and isoform-selective PI3K inhibitors; XL518, a small-molecule inhibitor of MEK; sphingosine-1-phosphate type 1 receptor; XL880 inhibitor; and therapies targeted against the mineralocorticoid receptor, a nuclear hormone receptor implicated in various cardiovascular and metabolic diseases. The company was formerly known as Exelixis Pharmaceuticals, Inc. and changed its name to Exelixis, Inc. in February 2000. Exelixis, Inc. was founded in 1994 and is headquartered in South San Francisco, California.

Advisors' Opinion:
  • [By Selena Maranjian]

    The biggest new holdings are The Finish Line�and Aeropostale. Other new holdings of interest include biotech company Exelixis (NASDAQ: EXEL  ) , which received FDA approval last year for its thyroid cancer drug, Cometriq. The drug may also get approved to treat prostate cancer, and the company is looking at treating as many as nine different cancers with it. On the other hand, Cometriq is expensive, and the company's debt has been growing, along with its share count.

  • [By Sean Williams]

    Exelixis (NASDAQ: EXEL  )
    Yesterday was a big day for Exelixis shareholders, as it gave them their first glimpse of Cometriq sales. Designed for the treatment of metastatic medullary thyroid cancer, or MTC -- a rare but particularly aggressive form of thyroid cancer -- Cometriq, which is Exelixis' first drug approved by the Food and Drug Administration, nearly tripled progression-free survival in trials relative to AstraZeneca's (NYSE: AZN  ) Caprelsa, the current standard of treatment (11.2 months compared to four months). Based on those stats alone, I expect Cometriq to take practically all sales from AstraZeneca in MTC.

  • [By Sean Williams]

    Where investment dollars are headed
    Thyroid cancer is treated in nearly every case with a full or partial thyroid removal since the majority of thyroid cancers aren't aggressive. However, in those rare cases where surgery isn't an option or the disease has metastasized to other parts of the body, there are two drugs approved by the Food and Drug Administration to choose from.

    Caprelsa: AstraZeneca's (NYSE: AZN  ) Caprelsa was approved to treat unresectable, locally advanced, or metastatic medullary thyroid cancer in April 2011. In trials, AstraZeneca's pill increased progression-free survival over the placebo and delivered an overall response rate of 44%, compared with just 1% for the placebo -- although it should be noted that all responses were partial. However, Caprelsa also comes with a laundry list of side effects that range from something as simple as rash, nausea, and hypertension, to having resulted in death from respiratory arrest and cardiac failure with arrhythmia.� Cometriq: Exelixis' (NASDAQ: EXEL  ) Cometriq was approved last November to treat progressive metastatic medullary thyroid cancer. The capsules work by inhibiting multiple tyrosine kinases, which are crucial to blood vessel growth in solid and metastasizing tumors. In late-stage trials, patients receiving Cometriq demonstrated an astounding 11.2 months of progression-free survival compared with just four months for the placebo. Further, the objective response rate was 27% in the Cometriq arm and a goose egg for the placebo arm. Similar to AstraZeneca's Caprelsa, severe adverse reactions tended to increase for Cometriq users relative to the placebo.

    Just as we've witnessed with every previous cancer in this series, not every drug trial proves successful. Pfizer's (NYSE: PFE  ) Sutent, for instance, is a very successful treatment for kidney cancer, gastrointestinal stromal tumors, and pancreatic endocrine tumors, but it didn't fare as wel

  • [By Selena Maranjian]

    Finally, SAC Capital's biggest closed positions included Coach�and Dover. Other closed positions of interest include Exelixis (NASDAQ: EXEL  ) and, also,�Tronox Ltd. (NYSE: TROX  ) . Biotech company Exelixis recently reported non-blowout early sales of its thyroid cancer drug, Cometriq. Some are waiting to see if the drug gets approved to treat prostate cancer, too, and the company is looking at treating as many as nine different�conditions with it, such as bone tumors. On the other hand, Cometriq is expensive, and the company's debt has been growing, along with its share count.

Hot Biotech Companies To Invest In 2014: RXi Pharmaceuticals Corp (RXII)

RXi Pharmaceuticals Corporation (RXi), incorporated on September 8, 2011, is a development-stage company. The Company is a biotechnology company focused on discovering, developing and commercializing therapies addressing medical needs using RNA interference (RNAi)-targeted technologies. As of July 12, 2012, RXi was focusing on its internal therapeutic development efforts in fibrosis. RXI-109 is its RNAi product candidate, which is a dermal anti-scarring therapy that targets connective tissue growth factor (CTGF). The Company�� therapeutic platform consists of two main components: RNAi Compounds (rxRNA) and Advanced Delivery Technologies. RNAi compounds include rxRNAori, rxRNAsolo and sd-rxRNA, or self-delivering RNA. On April 26, 2012, it completed the spin-off transaction from Galena Biopharma, Inc. (Galena).

In January 2011, the Company announced research results in collaboration with Generex Biotechnology Corporation, and RXi�� wholly owned subsidiary Antigen Express, Inc., in developing vaccine formulations for immunotherapy. In January 2011, it announced initial results as part of its collaboration with miRagen Therapeutics, Inc. in creating microRNA mimics, or artificial copies of microRNAs, using the Company�� sd-rxRNA technology. In February 2011, it announced the initiation of RXi�� development program for RXI-109.

Hot Biotech Companies To Invest In 2014: Prima BioMed Ltd (PRR)

Prima BioMed Ltd is a biotechnology company is engaged in the development and commercialization of medical therapies with a focus on oncology. Its product candidates in development include Cvac, an autologous dendritic cell vaccine for ovarian cancer, monoclonal antibodies for multiple tumour types, and an oral formulation for the human papilloma virus (HPV), vaccine. Its product candidate Cvac is a dendritic cell therapy, for which it is conducting a Phase IIb trial for the treatment of ovarian cancer. Cvac is designed to target the tumour antigen mucin-1, which is expressed at high levels on different tumour types. It also has two preclinical product development programs. In May 2011, Prima BioMed GmbH, a 100 % owned subsidiary of Prima BioMed Ltd, was incorporated in Germany. In May 2011, Prima BioMed Middle East FZLLC, a 100 % owned subsidiary of Prima BioMed Ltd, was incorporated in the United Arab Emirates.

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