Best Biotech Companies To Watch In Right Now

Celgene (NASDAQ: CELG  ) shares have soared more than 46% so far in 2013. This booming biotech stock can't go much higher, right? Surely, it's time now to take profits. Actually, the company just announced two developments that add to the reasons to buy Celgene rather than sell.

Facing the giants
For a company known for its blood cancer drugs, Celgene has been quite excited about anti-inflammatory drug apremilast. Clinical results for apremilast have been positive, but some observers have been a little skeptical about the drug's potential in the face of stiff competition. Celgene today announced more results from a phase 3 study of apremilast that could melt some of that skepticism.

Patients with psoriatic arthritis who took 20mg doses of apremilast for 52 weeks showed ACR 20 scores of 63%. Patients taking 30mg doses demonstrated ACR scores of 55%. These ACR scores are measurements established by the American College of Rheumatology that reflect 20% improvement in tender or swollen joint counts as well as 20 percent improvement in three other criteria. Apremilast's 52-week results show significant improvement from earlier results.

Best Biotech Companies To Watch In Right Now: Sanofi(SNY)

sanofi-aventis engages in the discovery, development, and distribution of therapeutic solutions to improve the lives of everyone. The company offers a range of healthcare assets, including a broad-based product portfolio in prescription drugs, OTC/OTX, generics, vaccines, and animal health. It has a strategic alliance with Regulus Therapeutics Inc. to discover, develop, and commercialize micro-RNA therapeutics, initially in fibrosis. The company was founded in 1970 and is headquartered in Paris, France.

Advisors' Opinion:

• [By Ben Levisohn]

  The European pharmaceutical stocks that don’t reflect the potential of biologics yet include Novartis (NVS) and Sanofi (SNY), McGarry says.

  Novartis has dropped 1.3% to $74.15 today, while Sanofi has fallen 2.4% to $49.57.

• [By Paul Ausick]

  The U.S. division of Paris-based Sanofi (NYSE: SNY) announced on Monday that an iconic U.S. consumer product not named Twinkies is on its way back to store shelves. Over-the-counter antacid Rolaids is once again available after a gap of nearly three years.

• [By Kanak Kanti, De]

  Teva's competition
  Teva will still face competition in the colorectal cancer generic market. The company's version of Xeloda is expected to compete with cytotoxic agents such as oxaliplatin, which is a generic version of French drugmaker Sanofi's (NYSE: SNY  ) Eloxatin/Eloxatine. In 2012, sales of Eloxatin totaled $1.3 billion. Generic competition has had a major impact on Eloxatin, however, with sales falling to $159 million in the first half of 2013.

Best Biotech Companies To Watch In Right Now: Inovio Pharmaceuticals Inc (INO)

Inovio Pharmaceuticals, Inc., incorporated on June 29, 1983, is engaged in the development of a new generation of vaccines, called synthetic vaccines, focused on cancers and infectious diseases. The Company's SynCon technology enables the design of universal vaccines capable of providing cross-protection against existing or changing strains of pathogens, such as influenza and human immunodeficiency virus (HIV). The Company's electroporation delivery technology uses brief, controlled electrical pulses to increase cellular uptake of the vaccine. Its clinical programs include cervical dysplasia (therapeutic), avian influenza (preventive), prostate cancer (therapeutic), leukemia (therapeutic), hepatitis C virus (HCV) and HIV vaccines. It is advancing preclinical research and clinical development for a universal seasonal/pandemic influenza vaccine, as well as preclinical work for other products, including malaria and prostate cancer vaccines. Its partners and collaborators include University of Pennsylvania, Drexel University, National Microbiology Laboratory of the Public Health Agency of Canada, Program for Appropriate Technology in Health/Malaria Vaccine Initiative (PATH/MVI), National Institute of Allergy and Infectious Diseases (NIAID), Merck, ChronTech, University of Southampton, United States Military HIV Research Program (USMHRP), the United States Army Medical Research Institute of Infectious Diseases (USAMRIID) and HIV Vaccines Trial Network (HVTN). As of December 31, 2011 it owned 16.1% interest in VGX Int��.

Inovio�� Solution

The Company�� synthetic vaccine platform consists of its SynCon vaccine design process and electroporation delivery technology. It has developed a preclinical and clinical stage pipeline of vaccines. The Company�� synthetic vaccines are designed to prevent a disease (prophylactic vaccines) or treat an existing disease (therapeutic vaccines). Its synthetic vaccine consists of a deoxyribonucleic acid (DNA) plasmid encoding a selected antigen! (s), which is introduced into cells of humans or animals with the purpose of evoking an immune response to the encoded antigen. The Company�� synthetic vaccines are designed to generate specific antibody and/or T-cell responses.

The Company�� SynCon technology provides processes that employ bioinformatics, which combine extensive genetic data and sophisticated algorithms. Its design process uses the genetic make-up of a common antigen(s) from multiple strains of a virus within a viral sub-type or taxonomic group (family) of pathogens, such as HIV, hepatitis C virus (HCV), human papillomavirus (HPV), influenza and other diseases to synthetically create a new antigen for the desired pathogen target that does not exist in nature. Its synthetic vaccine candidates are being delivered into cells of the body using its electroporation (EP) DNA delivery technology.

Cancer Synthetic Vaccines

The Company has two broad types of cancer vaccines: preventive (or prophylactic) vaccines, which are intended to prevent cancer from developing in healthy people, and treatment (or therapeutic) vaccines, which are intended to treat an existing cancer by strengthening the body�� natural defenses against the cancer. Two types of cancer preventive vaccines are available in the United States. The United States Food and Drug Administration (the FDA) has approved two vaccines, Gardasil and Cervarix that protect against infection by the two types of HPV-types 16 and 18-that cause approximately 70% of all cases of cervical cancer worldwide. In addition, Gardasil protects against infection by two additional HPV types, 6 and 11, which are responsible for about 90% of all cases of genital warts in males and females but do not cause cervical cancer.

Cervarix manufactured by GlaxoSmithKline, is composed of virus-like particles (VLPs) made with proteins from HPV types 16 and 18. Cervarix is approved for use in females��ages 10 to 25 for the prevention of cervical cancer caused by! HPV type! s 16 and 18. Gardasil manufactured by Merck, is approved for use in females for the prevention of cervical cancer, and some vulvar and vaginal cancers, caused by HPV types 16 and 18 and for use in males and females for the prevention of genital warts caused by HPV types 6 and 11. The vaccine is approved for these uses in females and males ages 9 to 26. The FDA has also approved a cancer preventive vaccine that protects against hepatitis B virus (HBV) infection.

Inovio�� VGX-3100 is designed to raise immune responses against the E6 and E7 genes of HPV types 16 and 18 that are present in both pre-cancerous and cancerous cells transformed by these HPV types. E6 and E7 are oncogenes that play an integral role in transforming HPV-infected cells into cancerous cells. In March 2011, it initiated a randomized, double-blind Phase II study of VGX-3100 delivered using the CELLECTRA intramuscular electroporation device in women with HPV Type 16 or 18 and diagnosed with, but not yet treated for, cervical intraepithelial neoplasia (CIN) 2/3. The study is designed to enroll 148 subjects. In January 2011, it announced the publication of a scientific paper in the journal Human Vaccines detailing potent immune responses in a preclinical study of its SynCon vaccine for prostate cancer targeting two antigens, prostate specific antigen (PSA) and prostate specific membrane antigen (PSMA).

In January 2011, the Company announced the regulatory approval of a Phase II clinical trial (WIN Trial) to treat leukemia utilizing its new ELGEN 1000 automated vaccine delivery device. The single dose level, Phase II study, called WT1 immunity via DNA fusion gene vaccination in haematological malignancies by intramuscular injection followed by intramuscular electroporation. Cancer Vaccines encodes for hTERT, an antigen related to non-small cell lung, breast and prostate cancers. The vaccine is delivered using its electroporation delivery technology.

Infectious Disease Synthetic Vaccines

In Marc! h 2011, the Company announced the initiation of a follow-on open label, single dose Phase II clinical study in collaboration with ChronTech of the ChronVac-C HCV DNA vaccine delivered using its electroporation technology in treatment naive HCV infected individuals. Its HIV vaccines consist of candidates for HIV prevention, as well as therapy or treatment. PENNVAX-B is designed to target HIV clade B (most commonly found in the United States, North America, Australia and the European Union (EU). PENNVAX-G is designed to target HIV clades A, C and D, which are more commonly found in Asia, Africa, Russia and South America. This Phase I clinical study of PENNVAX-B (HVTN-080) vaccinated 48 healthy, HIV-negative volunteers to assess safety and levels of immune responses generated by Inovio�� PENNVAX-B vaccine delivered with its CELLECTRA electroporation device. PENNVAX-B is a SynCon vaccine that targets HIV gag, pol, and env proteins.

The Company�� VGX-3400X targets H5N1. The vaccine consists of three distinct DNA plasmids coded for a consensus hemagglutinin (HA) antigen derived from different H5N1 virus strains; a consensus neuraminidase (NA) antigen derived from different N1 sequences; and a consensus nucleoprotein (NP) fused to a small portion of the m2 protein (m2E) based on a broader cross-section of influenza viruses in addition to H5N1 and H1N1. Conventional vaccines are strain-specific and have limited ability to protect against genetic shifts in the influenza strains they target. They are therefore modified annually in anticipation of the next flu season�� new strain(s). It is focused on developing DNA-based influenza vaccines able to provide broad protection against known as well as newly emerging, unknown seasonal and pandemic influenza strains.

Animal Health/Veterinary

VGX Animal Health, Inc. (VGX AH), a majority-owned subsidiary, has licensed LifeTide, a plasmid-based growth hormone releasing hormone (GHRH) technology for swine. LifeTide is one of onl! y four DN! A-based treatments approved for use in animals and is the only DNA-based agent delivered using electroporation that has been granted marketing approval (Australia). VGX AH is also developing a GHRH-based treatment for cancer and anemia in dogs and cats. It is developing a synthetic vaccine for foot-and-mouth disease (FMD) administered by its vaccine delivery technology. The FMD virus is one of the most infectious diseases affecting farm animals, including cattle, swine, sheep and goats, and is a serious threat to global food safety.

The Company competes with Crucell N.V, Sanofi-Aventis, Novartis, Inc., GlaxoSmithKline plc, Merck, Pfizer, AstraZeneca, Inc., Novartis, Inc., MedImmune and CSL.

Hot High Tech Companies To Invest In 2014: Gentium SpA(GENT)

Gentium S.p.A., a biopharmaceutical company, focuses on the development and manufacture of its primary product candidate, defibrotide, an investigational drug based on a mixture of single-stranded and double-stranded DNA extracted from pig intestines. It develops defibrotide for the treatment and prevention of hepatic veno-occlusive disease (VOD), a condition that occurs when veins in the liver are blocked as a result of cancer treatments, such as chemotherapy or radiation, that are administered prior to stem cell transplantation. The company has completed a Phase III clinical trial of defibrotide for the treatment of severe VOD in the United States, Canada, and Israel; and a Phase II/III pediatric trial in Europe for the prevention of VOD. It also offers sulglicotide that is developed from swine duodenum, and has ulcer healing and gastrointestinal protective properties in South Korea; and urokinase, which is made from human urine to treat various vascular disorders, such as deep vein thrombosis and pulmonary embolisms. The company was formerly known as Pharma Research S.r.L. and changed its name to Gentium S.p.A. in July 2001. Gentium S.p.A. was founded in 1993 and is headquartered in Villa Guardia, Italy.

Best Biotech Companies To Watch In Right Now: RXi Pharmaceuticals Corp (RXII.PK)

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 subsidia ry 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.

Best Biotech Companies To Watch In Right Now: ViroPharma Incorporated(VPHM)

ViroPharma Incorporated, a biotechnology company, develops and commercializes therapeutic products that address serious diseases in the United States and internationally. It focuses on developing products used by physician specialists or in hospital settings. The company markets and sells Cinryze, a C1 esterase inhibitor therapy for the routine prophylaxis against angioedema attacks in adolescent and adult patients with hereditary angioedema, a life-threatening genetic disorder; and Vancocin HCl capsule, an oral capsule formulation for the treatment of C. difficile-associated diarrhea (CDAD) and to treat enterocolitis caused by staphylococcus aureus, including methicillin-resistant strains. It also offers Plenadren, an orphan drug for treatment of adrenal insufficiency in adults; Buccolam, a oromucosal solution for treatment of prolonged, acute, and convulsive seizures in infants, toddlers, children, and adolescents; and maribavir, an antiviral compound for the treatment o f CMV disease through a license agreement with GlaxoSmithKline. The company?s primary development programs include Cinryze, a C1 esterase inhibitor for management of hereditary angioedema; and VP 20621, a non-toxigenic strain of C. difficile. Its clinical stage drug candidate comprises VP-20629 for the treatment of Friedreich?s Ataxia. The company sells its products directly to wholesale drug distributors and specialty pharmacies/distributors. ViroPharma Incorporated was founded in 1994 and is headquartered in Exton, Pennsylvania.

Advisors' Opinion:

• [By Max Macaluso and David Williamson]

  At the end of last week, a Bloomberg article revealed that Shire (NASDAQ: SHPG  ) and pharmaceutical giant Sanofi� (NYSE: SNY  ) may be circling ViroPharma� (NASDAQ: VPHM  ) . The the following video, from The Motley Fool's health care show Market Checkup, analysts David Williamson and Max Macaluso take a close look at ViroPharma and discuss the recent interest in this small biotech company.

Best Biotech Companies To Watch In Right Now: Telik Inc (TELK.PH)

Telik, Inc. (Telik), incorporated in 1988, is a clinical-stage drug development company focused on discovering and developing small molecule drugs to treat cancer. The Company discovers its product candidates using the Company�� drug discovery technology, Target-Related Affinity Profiling (TRAP). TELINTRA, its principal drug product candidate in clinical development, is a small molecule glutathione analog inhibitor of the enzyme glutathione S-transferase P1-1 (GST P1-1). TELCYTA, its other product candidate, is a small molecule cancer drug product candidate designed to be activated in cancer cells.

Clinical Product Development

TELINTRA is the Company�� lead small molecule product candidate in clinical development for the treatment of blood disorders, including cancer. It has a mechanism of action and acts by inhibiting GST P1-1, an enzyme that is involved in the control of cellular growth and differentiation. Inhibition of GST P1-1 results in the activation of the signaling molecule Jun kinase, a regulator of the function of blood precursor cells. Preclinical tests show that TELINTRA is capable of causing the death or apoptosis of leukemic or malignant blood cells, while stimulating the growth and development of normal blood precursor cells. TELINTRA has been studied in Myelodysplastic Syndrome (MDS) using two formulations. A liposomal formulation was developed for intravenous administration of TELINTRA and was used in Phase I and Phase II studies in MDS patients. The results from the Phase II intravenous liposomal TELINTRA clinical trials demonstrated that TELINTRA treatment was associated with improvement in all three types of blood cell levels in patients with all types of MDS, including those in intermediate and high-risk groups. An oral dosage formulation (tablet) was subsequently developed and results from a Phase I study with TELINTRA tablets showed clinical activity and the formulation to be well tole rated. In June 2011, the Company initiated a Phase II clini! c! al trial to evaluate TELINTRA tablets. In October 2011, the Company initiated an additional Phase IIb clinical trial to evaluate TELINTRA tablets. '

The activity and safety profile of tablet formulation allowed the Company to complete a Phase II trial of TELINTRA tablets in MDS. The primary objective of the Phase II TELINTRA tablet study was to determine the efficacy of TELINTRA. A multivariate logistic regression analysis was conducted to identify MDS disease prognostic factors associated with erythroid improvement response rates, including prior MDS treatment, age, gender, the international prognostic scoring system (IPSS), risk, Eastern Cooperative Group performance status, years from MDS diagnosis, MDS World Health Organization subtypes, anemia only versus anemia plus other cytopenias, dose schedule and starting dose. Results from this study show that TELINTRA is the first GSTP1-1 enzyme inhibitor shown to cause clinically reductions in red blood cell transf usions, including transfusion independence in low to intermediate-1 risk MDS patients, as well as improvement in platelet count and white blood cell levels in certain patients. TELINTRA, administered orally twice daily, appeared to be convenient and flexible for chronic treatment administration.

TELCYTA is a small molecule drug product candidate that the Company is developed for the treatment of cancer. TELCYTA binds to GST. TELCYTA has been evaluated in multiple Phase II and Phase III clinical trials, including trials using TELCYTA as monotherapy and in combination regimens in ovarian, non-small cell lung, breast and colorectal cancer. Results from these clinical trials indicate that TELCYTA monotherapy was generally well-tolerated, with mostly mild to moderate side effects, particularly when compared to the side effects and toxicities of standard chemotherapeutic drugs. When TELCYTA was evaluated in combination with standard chemotherapeutic drugs, the tolera bility of the combinations was similar to that expected! of e! ac! h drug ! alone.

Clinical activity including objective tumor responses and/or disease stabilization was reported in the TELCYTA Phase II trials; however, TELCYTA did not meet its primary endpoints in the Phase III studies. Positive results from a Phase I-IIa multicenter, dose-ranging study of TELCYTA in combination with carboplatin and paclitaxel as first-line therapy for patients with non-small cell lung cancer, or NSCLC, were published in a peer reviewed publication. Clinical data demonstrated positive results of TELCYTA in combination with carboplatin and paclitaxel in the treatment of first-line lung cancer followed by TELCYTA maintenance therapy. As of December 31, 2011, the Company had an on-going investigator-led study at a single site of TELCYTA in patients with refractory or relapsed mantle cell lymphoma, diffuse B cell lymphoma, and multiple myeloma.

Preclinical Drug Product Development

The Company has a small molecule compound, TLK60 404, in preclinical development that inhibits both Aurora kinase and VEGFR kinase. Aurora kinase is a signaling enzyme whose function is required for cancer cell division, while VEGF plays a key role in tumor blood vessel formation, ensuring an adequate supply of nutrients to support tumor growth. These lead compounds prevented tumor growth in preclinical models of human colon cancer and human leukemia by inhibiting both Aurora kinase and VEGFR kinase. A development drug product candidate, TLK60404, has been selected.

The Company, using its TRAP technology has discovered TLK60357, a novel, potent small molecule inhibitor of cell division. TLK60357 inhibits the formation of microtubules that are necessary for cancer cell growth leading to persistent G2/M cancer cell cycle block and subsequent cell death. This compound demonstrates potent broad-spectrum anticancer activity against a number of human cancer cells. This compound also displays oral efficacy in multipl e, standard preclinical models of cancer. TLK6059! 6, a pote! nt! VGFR kin! ase inhibitor, blocks the formation of new blood vessels in tumors. Oral administration of TLK60596 to animal models of human colon cancer reduced tumor growth.

Best Biotech Companies To Watch In Right Now: Alnylam Pharmaceuticals Inc.(ALNY)

Alnylam Pharmaceuticals, Inc., a biopharmaceutical company, engages in discovering, developing, and commercializing novel therapeutics based on RNA interference (RNAi). Its core product programs under clinical or pre-clinical development include ALN-TTR, a Phase I clinical trial program for the treatment of transthyretin-mediated amyloidosis; ALN-APC, a Phase I clinical trial program for the treatment of hemophilia; ALN-PCS for the treatment of severe hypercholesterolemia; ALN-HPN, a pre-clinical development for the treatment of refractory anemia; and ALN-TMP, a pre-clinical development for the treatment of hemoglobinopathies, including beta-thalassemia and sickle cell anemia. The company?s partner-based programs comprise ALN-RSV01, a Phase II clinical trial program for the treatment of respiratory syncytial virus infection; ALN-VSP, a Phase I clinical trial completed program for the treatment of liver cancers; and ALN-HTT, a pre-clinical development for the treatment of Huntington?s disease. It has strategic alliances with Novartis Pharma AG; F. Hoffmann-La Roche Ltd; Takeda Pharmaceutical Company Limited; Isis Pharmaceuticals, Inc.; Medtronic Inc.; Kyowa Hakko Kirin Co., Ltd.; and Cubist Pharmaceuticals, Inc. The company was founded in 2002 and is headquartered in Cambridge, Massachusetts.

Best Biotech Companies To Watch In Right Now: InterMune Inc.(ITMN)

InterMune, Inc., a biopharmaceutical company, engages in the research, development, and commercialization of therapies in pulmonology and fibrotic diseases. In pulmonology, the company focuses on therapies for the treatment of idiopathic pulmonary fibrosis (IPF), a progressive and fatal lung disease. It markets pirfenidone, an orally active drug that inhibits the synthesis of TGF-beta under the Esbriet name in the European Union, as well as in a Phase III clinical trial in the United States. Pirfenidone is also approved for the treatment of IPF in Japan, where it is marketed by Shionogi & Co. Ltd. under the Pirespa trade name. The company?s research programs focus on the discovery of small-molecule therapeutics and biomarkers to treat and monitor serious pulmonary and fibrotic diseases. InterMune, Inc. was founded in 1998 and is headquartered in Brisbane, California.

Advisors' Opinion:

• [By Lee Jackson]

  InterMune Inc. (NASDAQ: ITMN) continues to soar on the strength of global sales of its top drug Esbriet. The company is still seeking FDA approval to sell Esbriet in the United States. Before approval can take place, the company is awaiting top-line results from the phase 3 ASCEND study. InterMune expects to release these results during the second quarter of 2014. This could be a huge catalyst. UBS has a $15 price target, which should rise soon, and the consensus is at $16.