Top 10 Biotech Companies To Watch For 2014

LONDON — After two sessions of losses in a row, the FTSE 100 (FTSEINDICES: ^FTSE  ) is taking back some ground today, up 0.7% to 6,571 as of 8:10 a.m. EDT. Today it seems the markets are feeling more confident about worldwide economic-stimulus measures — though, in the long term, the economy will have to be left to its own devices, so Foolish investors should be looking beyond such things.

With such a modest start to the day, which shares look like they may beat the FTSE today? Here are two candidates from the various indexes.

Thomas Cook (LSE: TCG  )
Thomas Cook Group shares have picked up 0.6% to 144 pence after a vote at yesterday’s AGM supported the travel firm’s refinancing plans, and this morning the company announced that its capital reorganization, as announced on May 16, is complete. There is no change to the number of ordinary shares into which outstanding warrants may be converted, and there will be a further announcement regarding a recently announced placement in due course.

Top 10 Biotech Companies To Watch For 2014: Synageva BioPharma Corp (GEVA)

Synageva BioPharma Corp., incorporated in 1993, is a clinical-stage biopharmaceutical company. The Company is focused on the discovery, development and commercialization of therapeutic products. It has several protein therapeutics in development, including two enzyme replacement therapies for lysosomal storage disorders and two programs for life-threatening genetic conditions. Its lead program, SBC-102, recombinant human lysosomal acid lipase (LAL), is its advanced pipeline program in clinical development for LAL Deficiency. This enzyme is responsible for the metabolism of cholesteryl esters and triglycerides that are delivered to lysosomes by a variety of routes, including low-density lipoprotein receptor mediated endocytosis. On November 2, 2012, Trimeris, Inc. merged with Synageva BioPharma Corp.

SBC-102 is produced by recombinant deoxyribonucleic acid (DNA) technology in egg white using its protein manufacturing platform. The Company has initiated natural history studies in approximately 20 countries. In addition to SBC-102, it is progressing protein therapeutic programs for other rare diseases, which are at different stages of preclinical development. These include two enzyme replacement therapies for other lysosomal storage disorders and two programs for other rare life-threatening conditions. As of December 31, 2011, its product candidate pipeline included SBC-102, SBC-103, SBC-104, SBC-105 and SBC-106.

SBC-104 is an extracellular protein that targets a severe, rare genetic condition. SBC-105 is an enzyme replacement therapy being developed to treat a severe, rare metabolic disorder. SBC-106 is a protein therapy that targets a severe and rare genetic condition.

Advisors’ Opinion:

  • [By Ben Levisohn]

    Somaiya and team named Gilead and Neurocrine Biosciences (NBIX) their top picks, hile putting Buy ratings on Celgene, Biogen Idec, Alexion (ALXN), Incyte (INCY), Pharmacyclics (PCYC) and Synageva (GEVA). BioMarin (BMRN), Infinity Pharmaceuticals (INFI) and Amgen (AMGN) earned Neutral ratings.

Top 10 Biotech Companies To Watch For 2014: Intellicell Biosciences Inc (SVFC)

Intellicell Biosciences, Inc., formerly Media Exchange Group, Inc., incorporated on March 8, 1999, is engaged in regenerative medicine company focused on the expanding regenerative medical markets using a process to separate adult autologous vascular cells (AAVC’s) from blood vessels in adult adipose (fat) tissue. The Company is also exploring and undertaking, either on its own or in collaboration with a third party, providing a service for the collection, processing and storage of autologous cells for future use. As of December 31, 2011, the Company has developed technologies that allow reproducible separation of stromal vascular fraction (IntelliCell) containing adipose stem cells that can be performed in tissue processing centers and in doctors’ offices. On June 3, 2011, the Company completed the acquisition of Intellicell Biosciences, Inc. The Company formed a wholly-owned subsidiary, ICBS Research, Inc.

The Company’s process involves the application of ultrasonic cavitation (sound waves) to the extracted adipose tissue, which results in the separation AAVC’s from the blood vessels in adult adipose (fat) tissue. This AAVC, or stromal vascular fraction (IntelliCells), are removed from the patient at the point of care, and separated at the point of care under the supervision of its certified technicians following current good manufacturing practices (cGMPs) and current good tissue practices (cGTPs), and the cells are then returned to the medical professionals at the point of care for use a patient’s own body (autologous treatment), by way of a same-day clinical procedure for homologous use of these cells.

Intellicell Biosciences, Inc., formerly Media Exchange Group, Inc., incorporated on March 8, 1999, is engaged in regenerative medicine company focused on the expanding regenerative medical markets using a process to separate adult autologous vascular cells (AAVC’s) from blood vessels in adult adipose (fat) tissu e. The Company is also exploring and undertaking, either on ! its own or in collaboration with a third party, providing a service for the collection, processing and storage of autologous cells for future use. As of December 31, 2011, the Company has developed technologies that allow reproducible separation of stromal vascular fraction (IntelliCell) containing adipose stem cells that can be performed in tissue processing centers and in doctors’ offices. On June 3, 2011, the Company completed the acquisition of Intellicell Biosciences, Inc. The Company formed a wholly-owned subsidiary, ICBS Research, Inc.

The Company’s process involves the application of ultrasonic cavitation (sound waves) to the extracted adipose tissue, which results in the separation AAVC’s from the blood vessels in adult adipose (fat) tissue. This AAVC, or stromal vascular fraction (IntelliCells), are removed from the patient at the point of care, and separated at the point of care under the supervision of its certified technicians following current go od manufacturing practices (cGMPs) and current good tissue practices (cGTPs), and the cells are then returned to the medical professionals at the point of care for use a patient’s own body (autologous treatment), by way of a same-day clinical procedure for homologous use of these cells.

The Company competes with Cytori Therapeutics, Stem Cell Assurance, Inc., Osiris, Aastrom Biosciences, Aldagen, BioTime, Baxter International, Celgene, Geron, Harvest Technologies, Mesoblast, Regenexx, NeoStem, X-Cell Center, Stem Cells, Athersys, and Tissue Genesis, Life Technologies, Asterand, pacific biosciences of california inc. and AllCells, LLC.

Advisors’ Opinion:

  • [By Bryan Murphy]

    Well, though I give myself a C for timing, it looks like I’m going to be able to give myself an A for stock-picking. Back on January 27th I deemed IntelliCell BioSciences, Inc. (OTCMKTS:SVFC) was a compelling buy, and though SVFC was stagnant for a month after that, it looks like the stock’s finally getting on its horse. If you didn’t get it then, you may want to get in now.

  • [By Bryan Murphy]

    To say that shares of IntelliCell BioSciences, Inc. (OTCMKTS:SVFC) has been disappointing since 2011 would be an understatement. SVFC has been an outright disaster since 2011, falling from a peak of $19.00 to a low of, well, just a few pennies as of late last year. In fact, there are those who are understandably wondering how the company is still alive, only producing about a half a million dollars in revenue in 2012, and then dialing that figure back down to nothing for the last few quarters. Yet, there’s just something about a company that refuses to go away…. something compelling now.

Top 10 Biotech Companies To Watch For 2014: Organovo Holdings Inc (ONVO)

Organovo Holdings, Inc. (Organovo), formerly Real Estate Restoration & Rental, Inc., incorporated in 2007, is a development-stage company. The Company has developed and is commercializing a platform technology for the generation of three-dimensional (3D) human tissues that can be employed in drug discovery and development, biological research, and as therapeutic implants for the treatment of damaged or degenerating tissues and organs. On December 28, 2011, Real Estate Restoration and Rental, Inc.’s (RERR) entered into an Agreement and Plan of Merger, pursuant to which RERR merged with its, wholly owned subsidiary, Organovo (Merger Sub). On February 8, 2012, the Company merged with and into Organovo Acquisition Corp. (Acquisition Corp.), a wholly owned subsidiary of Organovo, with the Company surviving the merger as a wholly owned subsidiary of Organovo Holdings (the Merger). As a result of the Merger, Organovo acquired the business of Organovo, Inc.

The Comp any has collaborative research agreements with Pfizer, Inc. (Pfizer) and United Therapeutic Corporation (Unither). As of March 31, 2012, it has five federal grants, including Small Business Innovation Research grants and developed the NovoGen MMX Bioprinter (its first-generation 3D bioprinter). The Company is engaged in the development of specific 3D human tissues to aid Pfizer in discovery of therapies in two areas of interest. In addition, in October 2011, it entered into a research agreement with Unither to establish and conduct a research program to discover treatments for pulmonary hypertension using its NovoGen MMX Bioprinter technology. Additionally, under the research agreement with Unither, the Company granted Unither an option to acquire from the Company a worldwide, royalty-bearing license in certain intellectual property created under the research agreement solely for use in the treatment or prevention of pulmonary hypertension and all other lung diseases.

The Company’s NovoGen MMX Bioprinter is an automate! d device that enables the fabrication of three-dimensional (3D) living tissues comprised of mammalian cells. A custom graphic user interface (GUI) facilitates the 3D design and execution of scripts that direct precision movement of the dispensing heads to deposit cellular building blocks (bio-ink) or supporting hydrogel. The Company is using a third party manufacturer, Invetech Pty., of Melbourne, Australia, to manufacture its NovoGen MMX Bioprinter. Its bioprinting technology and surrounding intellectual property and commercial rights serve as a platform for product generation across multiple markets that employ cell- and tissue-based products and services.

The Company competes with Organogenesis, Advanced BioHealing, Tengion, Genzyme, HumaCyte and Cytograft Tissue Engineering.

Advisors’ Opinion:

  • [By Paul Ausick]

    A unique 3D printing stock is Organovo Holdings Inc. (NYSEMKT: ONVO). Shares are down more than 15% in 2014 after rising about 325% in 2013. The company is developing a 3D bioprinting technology that can create functional human tissues on demand for research and medical use. Organovo announced a first delivery of liver tissue in late January, well ahead of schedule, and the stock gained back some of its year-to-date losses. The company has been publicly traded for about a year and a half, and the stock hit a post-IPO high of $13.65 in November. It closed on Friday at $9.35. There is no price target on the stock, and the company is not expected to post positive earnings at least through 2015. In terms of market cap, though, Organovo ranks fourth among the publicly traded 3D printing companies with a market value of nearly $728 million.

  • [By MONEYMORNING]

    During my July interview, Michael enthusiastically recommended 3D Systems Corp. (NYSE: DDD) and Organovo Holdings Inc. (NYSEMKT: ONVO). But he urged readers to take a wait-and-see approach with ExOne, saying the stock had just corrected.

Top 10 Biotech Companies To Watch For 2014: Medizone International Inc (MZEI)

Medizone International, Inc. (Medizone), incorporated in January 31, 1986, is a development-stage company. The Company is engaged in research into the medical uses of ozone. Medizone focuses in the field of hospital sterilization. It is a research and development company engaged in developing its AsepticSure. The Company is developing an ozone-based technology (AsepticSure) for decontaminating and sterilizing hospital surgical suites, emergency rooms, and intensive care units.

The Company started hospital beta-testing of a prototype system utilizing the original technology. The first round of in-hospital beta-testing for this AsepticSure hospital disinfection system was completed at a Hotel Dieu hospital in Kingston, Ontario, Canada. In addition to the hospital disinfection system, it employs an ozone-destruct unit which is used following disinfection of the treated infrastructure to reverse the O3 gas in the space, and turn it back into O2 in a short period o f time. The Company’s subsidiaries include Medizone Canada, Ltd. (MedCan). As of December 31, 2011, the Company had not generated any revenues.

Advisors’ Opinion:

  • [By CRWE]

    Today, MZEI surged (+5.64%) up +0.0048 at $.0899 with 21,900 shares in play thus far (ref. google finance Delayed: 1:32PM EDT October 16, 2013).

    Medizone International, Inc. previously reported that its WHO award-winning green infection control technology, AsepticSure has been Granted a patent by the United States Patent and Trademark office. (US 61/223,219) titled “Healthcare Facility Disinfecting System”. The AsepticSure infection control system has repeatedly demonstrated 100% microbial kill rates when used to decontaminate hospital rooms of the causative agents of HAI (hospital acquired infections).

    “With patent protection now established in the United States, Canada and Singapore and pending applications in process for the 37 member countries of the EU as well as Korea, Japan, China, India, Brazil and Mexico, our patent momentum is clearly gaining strength,” stated Edwin Marshall, Medizone’s CEO.

Top 10 Biotech Companies To Watch For 2014: BioTime Inc (BTX)

BioTime, Inc., incorporated on November 30, 1990, is a biotechnology company focused on the emerging field of regenerative medicine. Its commercial strategy is focused on its line of research products, such as ACTCellerate cell lines and associated ESpan culture media, HyStem hydrogels, human embryonic stem (hES) cell lines, and royalties from Hextend. Its focus is to provide regenerative therapies for age-related degenerative diseases. In January 2011, the Company acquired the assets of Cell Targeting, Inc. (CTI), a biotechnology company focused on methods of painting molecules on the surface of cells, which in turn causes the cells to adhere to particular tissues, such as those afflicted with disease. On March 21, 2011, the Company acquired Glycosan BioSystems, Inc. (Glycosan) through a merger of Glycosan with OrthoCyte Corporation (OrthoCyte). Through the merger, OrthoCyte acquired all of Glycosan’s assets, including Glycosan’s Hystem hydrogel product line. In March 2011, the Company entered into an agreement with XenneX, Inc., pursuant to which it organized LifeMap Sciences, Inc. (LifeMap), a subsidiary formed to advance the development and commercialization of its human embryonic stem (hES) cell data base. In August 2011, four hES cell lines (ESI-035, ESI-049, ESI-051 and ESI-053) developed by its subsidiary ES Cell International (ESI) were approved by the National Institutes of Health (NIH) for inclusion in the NIH Human Embryonic Stem Cell Registry. In May 2012, the Company acquired XenneX, Inc., through a merger of XenneX into LifeMap Sciences. In September 2012, the Company formed a wholly owned subsidiary, BioTime Acquisition Corporation. In October 2013, Geron Corporation announced the closing of the transaction to divest the Company’s stem cell assets to Asterias Biotherapeutics, Inc., a subsidiary of BioTime, Inc.

HyStem is a biomaterial, which mimics the human extracellular matrix, which is the network of molec ules surrounding cells in organs and tissues which is essent! ial to cellular function. Its HyStem hydrogels may have other applications when combined with the diverse and scalable cell types its scientists have isolated from hES cells. HyStem-Rx is a clinical grade formulation of HyStem-C, a biocompatible, implantable hyaluronan and collagen-based matrix for cell delivery in human clinical applications. Its subsidiary, OncoCyte Corporation, is developing PanC-Dx, a non-invasive blood-based cancer screening test designed to detect the presence of various human cancers, including cancers of the breast, lung, bladder, uterus, stomach, and colon, during routine check -ups. BioTime Asia, Limited (BioTime Asia) sells products for research use and may develop therapies to treat cancer and neurological and orthopedic diseases in Asia. OrthoCyte Corporation (OrthoCyte) is developing therapies to treat orthopedic disorders, diseases and injuries. ReCyte Therapeutics, Inc. (ReCyte Therapeutics) is developing therapies for age-related cardiovascu lar and blood disorders. Cell Cure Neurosciences Ltd. (Cell Cure Neurosciences), is a biotechnology company focused on developing stem cell-based therapies for retinal and other neurological disorders, including the development of retinal pigment epithelial (RPE) cells for the treatment of age-related macular degeneration. LifeMap Sciences, Inc. (LifeMap) is advancing the development and commercialization of our embryonic stem cell database and plans to make the database available for the marketing of research products and for use by stem cell researchers at pharmaceutical and biotechnology companies and other institutions via paid subscriptions or on a fee per use basis.

The Company’s blood plasma expander product, Hextend, is a physiologically balanced intravenous solution used in the treatment of hypovolemia, a condition caused by low blood volume, often from blood loss during surgery or injury. Hextend maintains circulatory system fluid volume and blood pr essure, and keeps vital organs perfused during surgery and t! rauma car! e. Hextend is manufactured and distributed in the United States by Hospira, Inc., and in South Korea by CJ CheilJedang (CJ), under license from it.

Human Embryonic Stem Cell Lines for Research Use

Combined with the Company’s ACTCellerate technology, it derives of a range of markets human embryonic progenitor cells (hEPCs). ESI’s six current good manufacturing practices (cGMP) hES cell lines have been approved by the NIH for inclusion in the Human Embryonic Stem Cell Registry, which renders those cell lines eligible for use in federally funded research. The ESI hES cell lines are available for purchase through www.biotimeinc.com. It also markets hEPCs developed using ACTCellerate technology. Its hEPCs are also available for sale through www.biotimeinc.com. It derived the complete genome sequence of five of the ESI hES cell lines to facilitate the development of products derived from these cell lines. It has made these GMP-grade cell lines, along with certain documentation and complete genomic deoxyribonucleic acid (DNA) sequence information, available for sale.

The Company offers human stem cell products and technology that can be used by researchers at universities and at companies in the bioscience and biopharmaceutical industries. It has developed research and clinical grade hES cell lines, which it markets for both basic research and therapeutic product development. It also markets human embryonic progenitor cell (hEPCs) developed using ACTCellerate technology. These hEPCs are purified lineages of cells, which are intermediate in the developmental process between embryonic stem cells and fully differentiated cells. The ACTCellerate cell lines are also available for purchase through http://www.biotimeinc.com.

Human Embryonic Progenitor Cells

Through the Company’s subsidiary ReCyte Therapeutics, it had a license from Advanced Cell Technology, Inc. (ACT) to use ACTCellerat e technology, and the rights to market more than 200 human c! ell types! made using that process. ACTCellerate allows the rapid isolation of hEPCs, which are cells that are intermediate in the developmental process between embryonic stem cells and fully differentiated cells.

Through an agreement with ReCyte Therapeutics, Millipore Corporation became a global distributor of ACTCellerate hEPC lines. In addition to the products, which it is co-marketing with Millipore, it offers 92 other ACTCellerate hEPC lines for purchase at www.biotimeinc.com. Through its subsidiary LifeMap, it provides online biomedical database services to increase awareness of molecular markers and diverse cell types comprising its ACTCellerate hEPC lines. It also focuses to market additional cell types manufactured with its PureStem technology. PureStem cell lines are produced by the exogenous expression of specific transcription factors, which regulate the differentiation of cell types from hES or induced pluripotent stem (iPS) cells. BioTime Asia has an agree ment with Shanghai Genext Medical Technology Co., Ltd. to sell ACTCellerate hEPC lines and related ESpan growth media to the medical and biological research communities in China, Taiwan, Hong Kong, and Macau on an exclusive basis. The marketing agreement includes provisions for an initial stocking inventory and annual milestones to maintain exclusivity. The Company’s California Institute of Regenerative Medicine (CIRM)-funded research addresses the need for industrial scale production of purified therapeutic cells.

hES Cells Carrying Genetic Diseases

The Company focuses to add to its product line muscle progenitor cells produced from five hES cell lines carrying genes for Duchenne muscular dystrophy, Emery-Dreifuss muscular dystrophy, spinal muscular atrophy Type I, facioscapulohumeral muscular dystrophy 1A, and Becker muscular dystrophy. It obtained the diseased hES cell lines from Reproductive Genetics Institute (RGI).

ESpan Cell G rowth Media

The Company is marketing a range of! cell-gro! wth media products called ESpan. These growth media are optimized for the growth of hEPC types. Cells need to be propagated in liquid media, in both the laboratory setting, where basic research on stem cells is performed, and in the commercial sector. Its ESpan products are marketed through Millipore and Genext.

ESpy Cell Lines

The Company focuses for development of ESpy cell lines. The ability of the ESpy cells to emit light will allow researchers to track the location and distribution of the cells in both in vitro and in vivo studies.

HyStem Hydrogel for Research and HyStem-Rx for Cell Delivery Medical Devices

HyStem is a patented biomaterial that mimics the ECM, the network of molecules surrounding cells in organs and tissues that is essential to cellular function. Its HyStem technology forms the foundation for stem cell delivery products in both the adult and embryonic stem cell marketplace, including products manufactu red using its ACTCellerate technology. As an injectable product, HyStem-Rx may address need in cosmetic and reconstructive surgeries and other procedures by transplanting adipose derived cells or other adult stem cells. The transfer of cells in HyStem-Rx localizes the transplanted cells at the intended site and by providing a three-dimensional scaffold upon which cells can rebuild normal tissue. HyStem-Rx may support other emerging cell and tissue transplant therapies, such as those derived from hES and iPS cells, in addition to its potential application in the treatment of a range of conditions such as osteoarthritis, brain tumors, stroke, bone fracture, and wounds.

Advisors’ Opinion:

  • [By Brian Pacampara]

    Based on the aggregated intelligence of 180,000-plus investors participating in Motley Fool CAPS, the Fool’s free investing community, biotechnology company BioTime (NYSEMKT: BTX  ) has received the dreaded one-star ranking.

  • [By John Udovich]

    Stem cell stocks have not exactly been the best performers lately in part because the controversy over their use has died down over the years while major breakthroughs have been few or far between, but the industry along with small cap stem cell stocks Pluristem Therapeutics Inc (NASDAQ: PSTI), BioTime, Inc (NYSEMKT: BTX) and BioRestorative Therapies (OTCBB: BRTX) are still quietly producing their share of news or minor breakthroughs worth taking note of. Just consider the following stem cell news or news from small cap players in the sector:

Top 10 Biotech Companies To Watch For 2014: Oncolytics Biotech Inc (ONCY)

Oncolytics Biotech Inc. (Oncolytics), incorporated on April 2, 1998, is a development-stage company. The Company is focused on its research and development of REOLYSIN, which is its cancer therapeutic. REOLYSIN is developed from the reovirus. This virus has been demonstrated in tumour cells bearing an activated Ras pathway. Oncolytics is directing a clinical trial program with the focus of developing REOLYSIN as a human cancer therapeutic. The clinical program includes clinical trials, which it sponsors directly along with Third Party Clinical Trials. Third Party Clinical Trials are clinical trials that are being sponsored by other institutions. As of December 31, 2011, the United States National Cancer Institute (NCI), the University of Leeds and the Cancer Therapy & Research Center at the University of Texas Health Center in San Antonio (CTRC) were sponsoring part of its clinical trial program.

The Company’s clinical trial program has included human trials using REOLYSIN alone, and in combination with radiation and chemotherapy, and delivered via local administration and/or intravenous administration. Oncolytics uses contract toll manufacturers to produce REOLYSIN. On December 31, 2011, the Company had two wholly owned subsidiaries, Oncolytics Biotech (Barbados) Inc. (OBB) and Valens Pharma Ltd. Oncolytics Biotech (US) Inc. and Oncolytics Biotech (U.K.) are wholly owned subsidiaries of OBB.

Advisors’ Opinion:

  • [By John Udovich]

    The biotech sector along with small cap biotech stocks Cardiome Pharma Corp (NASDAQ: CRME), Oncolytics Biotech, Inc (NASDAQ: ONCY), Vital Therapies Inc (NASDAQ: VTL) and TNI BioTech (OTCMKTS: TNIB) have all been producing their share of news this week for investors and traders alike to trade on. Moreover and while some 42 “life sciences” companies have gone public raising about $3 billion from investors so far this year, there are a growing number of biotechs pulling the plug on upcoming IPOs who are citing market conditions. With that in mind, here is a look at important news from the biotech sector and small cap biotech stocks this week:

  • [By Maxx Chatsko]

    T-VEC is not your traditional biologic drug. It is actually a bioengineered form of the herpes virus that, once injected into cancerous tumors, replicates, and produces an immune-stimulating protein that puts a bulls eye on cancer cells throughout the body. Despite its promise and intriguing mechanism of action, T-VEC is not in further development at Amgen. However, Oncolytics (NASDAQ: ONCY  ) has shown promising results for its bioengineered form of reovirus called Reolysin. Initial phase 3 results showed that 86% of patients taking the drug had reduced tumor mass or growth after six weeks of treatment.  

Top 10 Biotech Companies To Watch For 2014: 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’s 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 d etermine 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’s 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.

Top 10 Biotech Companies To Watch For 2014: Regulus Therapeutics Inc (RGLS)

Regulus Therapeutics Inc., incorporated on September 5, 2007, is a biopharmaceutical company focused on discovering and developing microRNAs to treat a range of diseases. microRNAs are naturally occurring ribonucleic acid (RNA), molecules that play a critical role in regulating key biological pathways. The Company uses its microRNA product platform to develop chemically modified, single-stranded oligonucleotides that the Company calls anti-miRs. As of December 31, 2012, the Company’s operations included acquiring and in-licensing intellectual property rights, developing its microRNA, undertaking basic research around microRNA targets and conducting preclinical studies for its initial programs.

The Company is developing RG-101 for the treatment of HCV and is advancing other microRNA therapeutics toward clinical development in several areas, including oncology, fibrosis and metabolic diseases. The Company intends to focus its resources on product opportunitie s in therapeutic areas where development and commercialization activities are appropriate for its size and financial resources, which the Company anticipates will include niche indications and orphan diseases.

The Company competes with Groove Biopharma, Inc., miRagen Therapeutics, Inc., Mirna Therapeutics, Inc., and Santaris Pharma A/S.

Advisors’ Opinion:

  • [By Sean Williams]

    A blast to the past
    It’s occasionally very difficult to value biotech stocks, because of the wide range of outcomes possible given their pipelines. Other times, as in the case with Regulus Pharmaceuticals (NASDAQ: RGLS  ) , I bang my head against a table and wonder what the heck investors are thinking.

  • [By Brian Orelli]

    Partners like this investment idea
    When Regulus Therapeutics (NASDAQ: RGLS  ) went public last year, investors weren’t the only ones buying the share offering; Regulus’ partners and founders clearly thought it was a good investment idea, because they bought more shares. Lots of them. AstraZeneca, Biogen Idec, Sanofi, GlaxoSmithKline and Isis combined to purchase more than 70% of the shares that raised nearly $81 million for the biotech.

  • [By Sean Williams]

    Regulus Therapeutics (NASDAQ: RGLS  )
    Since presenting at the Deutsche Bank Healthcare Conference two weeks ago, shares of Regulus Therapeutics have been absolutely on fire. A mixture of ASCO and IPO fever have revived the love for newer issues, and having big name pharmaceutical companies gobbling up a majority of its IPO’d shares certainly helps its cause.

Top 10 Biotech Companies To Watch For 2014: DiaMedica Inc (DMA)

DiaMedica Inc. (DiaMedica) is a development-stage company. The Company is a biopharmaceutical company engaged in the discovery and development of drugs for the treatment of diabetes and related diseases. DiaMedica’s compound, DM-199, is a recombinant human protein for the treatment of both Type I and Type II diabetes and their complications. DiaMedica is starting a Phase I/II clinical trial for DM-199. DM-199 is a recombinant human protein, which improves glucose control, protects beta cells through the expansion of a population of antigen-specific immunosuppressive cells (Tregs), and proliferates insulin producing beta cells through the activation of certain growth factors. The Company’s DM-204 is a G-protein-coupled receptor agonist (GPCR) monoclonal antibody to treat Type II diabetes and some of the associated complication’s. activating a receptor resulted in insulin sensitivity, insulin secretion and vasodilation. Advisors’ Opinion:

  • [By Richard Rhodes]

    Given this economic backdrop, and developing pressure on corporate revenues, margins, and earnings, we feel that risk is being misplaced at current levels.

    The 14-day and 40-day models are now overbought. Now, the 14-day and 40-day are peaking, which would certainly indicate a correction stands as the highest probability.

    The % of stocks above their 10-day moving average (dma) is at the 70%-level; still a major divergence with prices.

    The % of stocks above their 200-dma stands at 77%. The 87% level marked previous highs. The 50-dma/150-dma cross breakdown now confirms a larger correction. Bottoms form between 30%-40%.

    Overall, the risk-reward remains skewed to the downside, regardless of whether prices remain above trendline resistance, as our model group suggests a correction to the 110-day moving average, currently at S&P 1711.

    A clear breakdown at that level would accelerate the decline towards the wide 200-dma and 380-dma range, between 1657-1571.

Top 10 Biotech Companies To Watch For 2014: Cellular Dynamics International Inc (ICEL)

Cellular Dynamics International, Inc., incorporated on November 16, 2007, develops and manufactures fully functioning human cells in industrial quantities to precise specifications. The Company’s iCell Operating System (iCell O/S) includes true human cells in multiple cell types (iCell products), human induced pluripotent stem cells (iPSCs) and custom iPSCs and iCell products (MyCell products). Customers use its iCell O/S products, among other purposes, for drug discovery and screening; to test the safety and efficacy of their small molecule and biologic drug candidates; for stem cell banking; and in researching cellular therapeutics. The Company’s iCell product line includes four different cell types: cardiomyocytes, neurons, hepatocytes and endothelial cells. The Company is actively developing an additional seven different cell types. iCell products are a consumable designed to be used once and then reordered.

The Company manufactures its iCell products from its iPSCs. An iPSC is a cell that has the ability both to replicate indefinitely and to be transformed into any cell type in the human body. The Company’s iCell O/S consists of six products, which include iCell Cardiomyocytes, iCell Neurons, iCell Endothelial Cells, iCell Hepatocytes and MyCell.

Advisors’ Opinion:

  • [By John Udovich]

    Stem cells may not be in the news much as the sector has moved beyond the use of embryotic ones, but small cap stem cell stocks Cellular Dynamics International Inc (NASDAQ: ICEL), International Stem Cell Corp (OTCMKTS: ISCO) and BioRestorative Therapies (OTCBB: BRTX) have been fairly active over the past several trading days as ICEL went public, ISCO raised additional funding and BRTX grabbed more attention: