Top Biotech Companies To Watch In Right Now

They're demanding. They take risks -- but only calculated ones. And they ask lots of penetrating questions to understand as much as possible before putting a dime on the line. Venture capitalists, or VCs, funded nearly 3,700 deals last year. Many of those deals will fail, but the ones that don't could produce astronomical gains.

Investing in biotech stocks can be fraught with risk, but thinking like a venture capitalist can help increase the odds of success. Here are three VC principles that biotech investors should keep in mind.

1. Look for a disruptive technology that addresses an unmet need
Venture capitalists look for companies that target an unmet need and provide an innovative way to meet that need. The good news for prospective biotech investors is that there are plenty of companies fulfilling that criterion.

Perhaps the most obvious example of addressing an unmet need lies in targeting rare orphan diseases. For example, cystic fibrosis, while widely known, affects only around 30,000 Americans. Although some treatments exist to help improve qualify of life, there is no cure for the disease.

Top Biotech Companies To Watch In Right Now: CEL-SCI Corp (CVM)

CEL-SCI Corporation (CEL-SCI), incorporated on March 22, 1983, is engaged in the business of Multikine cancer therapy; New cold fill manufacturing service to the pharmaceutical industry, and ligand epitope antigen presentation System (LEAPS) technology, with two products, hemagglutinin type 1 and neuraminidase type 1 (H1N1) swine flu treatment for H1N1 hospitalized patients and CEL-2000, a rheumatoid arthritis treatment vaccine.

Multikine

CEL-SCI's Multikine, is being developed for the treatment of cancer. It is a cancer immunotherapy drugs called Combination Immunotherapy because it combines active and passive immunity in one product. It is the only cancer immunotherapy that both kills cancer cells and activates the general immune system to destroy the cancer. Multikine target the tumor micro-metastases for treatment failure. Multikine is also applicable in many other solid tumors.

New Manufacturing Facility

CEL-SCI's facility manufactures Multikine for CEL-SCI's Phase III clinical trial. CEL-SCI offers the use of the facility as a service to pharmaceutical companies and others, particularly those that need to fill and finish their drugs in a cold environment. Fill and finish is the process of filling injectable drugs in a sterile manner.

LEAPS

CEL-SCI's patented T-cell Modulation Process uses heteroconjugates to direct the body to choose a specific immune response. The heteroconjugate technology, referred to as LEAPS, is intended to stimulate the human immune system to fight bacterial, viral and parasitic infections, as well as autoimmune, allergies, transplantation rejection and cancer. Administered like vaccines, LEAPS combines T-cell binding ligands with small, disease associated and peptide antigens.

Using the LEAPS technology, CEL-SCI has created a peptide treatment for H1N1 (swine flu) hospitalized patients. This LEAPS flu treatment is designed to focus on the conserved, non-changing epitopes of the di! fferent strains of Type A Influenza viruses, including swine, avian or bird, and Spanish Influenza. CEL-SCI's LEAPS flu treatment contains epitopes.

Top Biotech Companies To Watch In Right Now: Cell Therapeutics Inc (CTIC)

Cell Therapeutics, Inc. (CTI), incorporated in 1991, develops, acquires and commercializes treatments for cancer. The Company�� research, development, acquisition and in-licensing activities concentrate on identifying and developing new ways to treat cancer. As of December 31, 2011, CTI focused its efforts on Pixuvri (pixantrone dimaleate) (Pixuvri), OPAXIO (paclitaxel poliglumex) (OPAXIO), tosedostat, brostallicin and bisplatinates. As of December 31, 2011, it developed Pixuvri, an anthracycline derivative for the treatment of hematologic malignancies and solid tumors. Another late-stage drug candidate of the Company, OPAXIO, is being studied as a potential maintenance therapy for women with advanced stage ovarian cancer, who achieve a complete remission following first-line therapy with paclitaxel and carboplatin. As of December 31, 2011, it also developed tosedostat in collaboration with Chroma Therapeutics, Ltd. (Chroma). On May 31, 2012, CTI completed its acquisition gaining worldwide rights to S*BIO Pte Ltd.'s (S*BIO) pacritinib.

Pixuvri

As of December 31, 2011, the Company developed Pixuvri, an aza-anthracenedione derivative, for the treatment of non-Hodgkin�� lymphoma (NHL), and various other hematologic malignancies, and solid tumors. Pixuvri was studied in the Company�� EXTEND, or PIX301, clinical trial, which was a phase III single-agent trial of Pixuvri for patients with relapsed, refractory aggressive NHL who received two or more prior therapies and who were sensitive to treatment with anthracyclines. On September 28, 2011, CTI announced that a second independent radiology assessment of response and progression endpoint data from its PIX301 clinical trial of Pixuvri was achieved with statistical significance. The results of the EXTEND trial met its primary endpoint and showed that patients randomized to treatment with Pixuvri achieved a significantly higher rate of confirmed and unconfirmed complete response compared to patients treated with standard chem! otherapy had a significantly increased overall response rate and experienced a statistically significant improvement in median progression free survival. Pixuvri had predictable and manageable toxicities when administered at the proposed dose and schedule in the EXTEND clinical trial in heavily pre-treated patients. In March 2011, the Company initiated the PIX-R trial to study Pixuvri in combination with rituximab in patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL). Pixuvri has also been studied in patients with HER2-negative metastatic breast cancer who have tumor progression after at least two, but not more than three, prior chemotherapy regimens. In the second quarter of 2010, the NCCTG opened this phase II study for enrollment. The study is closed to accrual and results are expected to be reported by the NCCTG later in 2012.

OPAXIO

OPAXIO is the Company�� biologically-enhanced chemotherapeutic agent that links paclitaxel to a biodegradable polyglutamate polymer, resulting in a new chemical entity. As of December 31, 2011, the Company focused its development of OPAXIO on ovarian, brain, esophageal, head and neck cancer. OPAXIO was designed to improve the delivery of paclitaxel to tumor tissue while protecting normal tissue from toxic side effects. In November 2010, results were presented by the Brown University Oncology Group from a phase II trial of OPAXIO combined with temozolomide (TMZ), and radiotherapy in patients with newly-diagnosed, high-grade gliomas, a type of brain cancer. The trial demonstrated a high rate of complete and partial responses and a high rate of six month progression free survival (PFS). Based on these results, the Brown University Oncology Group has initiated a randomized, multicenter, phase II study of OPAXIO and standard radiotherapy versus TMZ and radiotherapy for newly diagnosed patients with glioblastoma with an active gene termed MGMT that reduces responsiveness to TMZ. A phase I/II study of OPAXIO combined with radi! otherapy ! and cisplatin was initiated by SUNY Upstate Medical University, in patients with locally advanced head and neck cancer.

Tosedostat

In March 2011, the Company entered into a co-development and license agreement with Chroma Therapeutics, Ltd. (Chroma), providing the Company with marketing and co-development rights to Chroma�� drug candidate, tosedostat, in North, Central and South America. Tosedostat is an oral, aminopeptidase inhibitor that has demonstrated anti-tumor responses in blood related cancers and solid tumors in phase I-II clinical trials. Interim results from the phase II OPAL study of tosedostat in elderly patients with relapsed or refractory acute myeloid leukemia (AML) showed that once-daily, oral doses of tosedostat had predictable and manageable toxicities and results demonstrated response rates, including a high-response rate among patients who received prior hypomethylating agents, which are used to treat myelodysplastic syndrome (MDS), a precursor of AML.

Brostallicin

As of December 31, 2011, the Company developed brostallicin through its wholly owned subsidiary, Systems Medicine LLC, which holds rights to use, develop, import and export brostallicin. Brostallicin is a synthetic deoxyribonucleic acid (DNA) minor groove binding agent that has demonstrated anti-tumor activity and a favorable safety profile in clinical trials, in which more than 230 patients have been treated as of December 31, 2011. The Company uses a genomic-based platform to guide the development of brostallicin. A phase II study of brostallicin in relapsed, refractory soft tissue sarcoma met its predefined activity and safety hurdles and resulted in a first-line phase II clinical trial study that was conducted by the European Organization for Research and Treatment of Cancer (EORTC).

The Company competes with Bristol-Myers Squibb Company, Sanofi-Aventis, Pfizer, Roche Group, Genentech, Inc., Astellas Pharma, Eli Lilly and Company, Celgene, Telik, I! nc., TEVA! Pharmaceuticals Industries Ltd. and PharmaMar.

Advisors' Opinion:

• [By Bryan Murphy]

  If you're reading this, then odds are you already know that the last two weeks (not even a full two weeks) have been more fruitful for Cell Therapeutics Inc. (NASDAQ:CTIC) shareholders than the prior two years have been - the stock's up 28% since last Thursday. And, odds are you already know why. The question most of you are asking now is, can CTIC actually keep climbing at this pace, or even keep climbing at any pace? The answer is "yes", though floating that answer almost inherently requires a deeper explanation.

Top 10 Growth Stocks To Buy Right Now: Nektar Therapeutics(NKTR)

Nektar Therapeutics, a clinical-stage biopharmaceutical company, engages in developing a pipeline of drug candidates that utilize its PEGylation and polymer conjugate technology platforms. The company?s product pipeline consists of drug candidates across various therapeutic areas, including oncology, pain, anti-infectives, anti-viral, and immunology. Its research and development activities involve small molecule drugs, peptides, and other potential biologic drug candidates. The company?s proprietary drug candidates in clinical development comprise NKTR-118, a peripheral opioid antagonist, which has completed Phase II clinical trail for the treatment of opioid-induced constipation; BAY41-6551 that has completed Phase II clinical trail to treat gram-negative pneumonias; NKTR-102, a topoisomerase I inhibitor-polymer conjugate, which is in Phase II clinical trail for multiple cancer indications, including breast, ovarian, and colorectal; and NKTR-105 that is in Phase I clinica l trail to treat solid tumors. Its preclinical products consists of NKTR-119 (Opioid/NKTR-118 combinations) for the treatment of pain; NKTR-181 (abuse deterrent, tamper-resistant opioid) to treat pain; NKTR-194 (non-scheduled opioid) for the treatment of mild to moderate pain; NKTR-171 (tricyclic antidepressant) to treat neuropathic pain; and NKTR-140 (protease inhibitor candidate) to treat HIV. The company has collaboration with Bayer Healthcare LLC to develop BAY41-6551 (NKTR-061, Amikacin Inhale), which is an inhaled solution of amikacin, an aminoglycoside antibiotic; and a license agreement with AstraZeneca AB for the development and commercialization of Oral NKTR-118 and NKTR-119. In addition, Nektar Therapeutics has various license, manufacturing, and supply agreements for its technology with biotechnology and pharmaceutical companies, such as Affymax, Amgen, Baxter, Roche, Merck, Pfizer, and UCB Pharma. The company was founded in 1990 and is headquartered in San Franc isco, California.

Advisors' Opinion:

• [By Sean Williams]

  Levadex's approval or rejection could also mean a good or bad day for Nektar Therapeutics (NASDAQ: NKTR  ) , which looks to gain from royalty rights based on its contributions to Levadex's development. While impossible to predict, I'm going to go out on a limb and project an approval for Allergan.

Top Biotech Companies To Watch In Right Now: Cannabis Science Inc (CBIS)

Cannabis Science, Inc., incorporated on May 4, 2007, is a development-stage company. The Company is engaged in the creation of cannabis-based medicines, both with and without psychoactive properties, to treats disease and the symptoms of disease, as well as for general health maintenance. On February 9, 2012, the Company acquired GGECO University, Inc. (GGECO). On March 21, 2012, the Company acquired Cannabis Consulting Inc. (CCI Group).

The Company is engaged in medical marijuana research and development. The Company works with world authorities on phytocannabinoid science targeting critical illnesses, and adheres to scientific methodologies to develop, produce, and commercialize phytocannabinoid-based pharmaceutical products.

Advisors' Opinion:

• [By John Udovich]

  Although its summer, there has been a steady stream of good news about medical marijuana even though important small cap marijuana stocks�Medical Marijuana Inc (OTCMKTS: MJNA) and Cannabis Science Inc (OTCMKTS: CBIS) have been fairly quietly lately while Growlife Inc (OTCBB: PHOT), a more indirect play on the spread of legalized marijuana, has produced�some news for investors:

Top Biotech Companies To Watch In Right Now: Galena Biopharma Inc (GALE.PH)

Galena Biopharma, Inc. (Galena), formerly RXi Pharmaceuticals Corporation, incorporated on April 3, 2006, is a biotechnology company focused on discovering, developing and commercializing therapies addressing unmet medical needs using targeted biotherapeutics. The Company is pursuing the development of cancer therapeutics using peptide-based immunotherapy products, including its main product candidate, NeuVaxTM (E75), for the treatment of breast cancer and other tumors. NeuVax is a peptide-based immunotherapy intended to reduce the recurrence of breast cancer in low-to-intermediate HER2-positive breast cancer patients not eligible for trastuzumab (Herceptin; Genentech/Roche). On January 19, 2012, the Company initiated enrollment in its Phase 3 PRESENT clinical trial for NeuVax (E75 peptide plus GM-CSF) vaccine in low-to-intermediate HER2 1+ and 2+ breast cancer patients in the adjuvant setting to prevent recurrence (Clinicaltrials.gov identifier NCT01479244). The Preven tion of Recurrence in Early-Stage, Node-Positive Breast Cancer with Low to Intermediate HER2 Expression with NeuVax Treatment study is a randomized, multicenter, multinational clinical trial that will enroll approximately 700 breast cancer patients. The Company�� Phase 2 trial of NeuVax achieved its primary endpoint of disease-free survival (DFS). On April 13, 2011, the Company completed its acquisition of Apthera, Inc.,(Apthera).

The Company focuses to start a Phase 2 trial comparing NeuVax in combination with trastuzumab (Herceptin) versus trastuzumab, alone, in a 300-patient, randomized study in the adjuvant breast cancer setting. The Company's second product candidate, Folate Binding Protein-E39 (FBP), is a vaccine, consisting of the peptides E39 and J65, aimed at preventing the recurrence of ovarian, endometrial, and breast cancers. On February 14, 2012, the Company announced the initiation of a Phase 1/2 clinical trial in two gynecological cancers: ovari an and endometrial adenocarcinomas. Folate binding protein! h! as very limited tissue distribution and expression in non-malignant tissue and is over-expressed in more than 90% of ovarian and endometrial cancers, as well as in 20% to 50% of breast, lung, colorectal and renal cell carcinomas.

In April 2011, the Company acquired Apthera Inc and its NeuVax product candidate. The Company focuses on developing a pipeline of immunotherapy product candidates for the treatment of various cancers based on the E75 peptide, the advanced of which is NeuVax, which is targeted at preventing the recurrence of breast cancer. NeuVax has had positive Phase 1/2 clinical trial results for the prevention of breast cancer recurrence in patients who have had breast cancer and received the standard of care treatment (surgery, chemotherapy, radiotherapy and hormonal therapy as indicated). The Company had also initiated its Phase 3 PRESENT clinical trial of NeuVax for the prevention of breast cancer recurrence in early-stage low-to-intermediate HER2 breast cancer patients. NeuVax directs killer T-cells to target and destroy cancer cells that express HER2/neu, a protein associated with epithelial tumors in breast, ovarian, pancreatic, colon, bladder and prostate cancers. NeuVax is comprised of a HER2/neu-derived peptide called E75. E75 is a nine-amino acid sequence that is immunogenic (produces an immune response) and GM-CSF is a commercially available protein that acts to stimulate and activate components of the immune system such as macrophages and dendritic cells.

The Company also develops novel applications for NeuVax based on preclinical studies and phases 2 clinical trials which suggest that combining NeuVax and trastuzumab (Herceptin; Genentech/Roche) can increase antigen presentation by tumor cells by promoting receptor internalization and subsequent proteosomal degradation of the HER2 protein. The Company also is pursuing additional therapeutic indications for NeuVax that are in Phase 1/2 clinical trials. RXI-109, is a dermal anti-scarring therapy that ! targ! ets! conne! ctive tissue growth factor (CTGF) and that may inhibit connective tissue formation in human fibrotic disease.

The Company competes with Roche Laboratories, Inc., Pfizer Inc., Bayer HealthCare AG, Sanofi-Aventis, US, LLC, Amgen, Inc., GlaxoSmithKline plc, Renovo Group plc, CoDa Therapeutics, Inc., Sirnaomics, Inc., FirstString Research, Inc., Merz Pharmaceuticals, LLC, Capstone Therapeutics, Halscion, Inc., Garnet Bio Therapeutics, Inc., AkPharma Inc., Promedior, Inc., Kissei Pharmaceutical Co., Ltd., Eyegene, Derma Sciences, Inc., Healthpoint Biotherapeutics, Pharmaxon, Excaliard Pharmaceuticals, Inc., Alnylam Pharmaceuticals, Inc., Marina Biotech, Inc., Tacere Therapeutics, Inc., Benitec Limited, OPKO Health, Inc., Silence Therapeutics plc, Quark Pharmaceuticals, Inc., Rosetta Genomics Ltd., Lorus Therapeutics, Inc., Tekmira Pharmaceuticals Corporation, Arrowhead Research Corporation, Regulus Therapeutics Inc. and Santaris.

Top Biotech Companies To Watch In Right Now: Neurocrine Biosciences Inc.(NBIX)

Neurocrine Biosciences, Inc. engages in the discovery, development, and commercialization of drugs for the treatment of neurological and endocrine-related diseases and disorders in the United States. It develops drugs for endometriosis, stress-related disorders, pain, tardive dyskinesia, uterine fibroids, diabetes, insomnia, and other neurological and endocrine-related diseases and disorders. The company?s products in clinical development include Elagolix, a Phase II drug for endometriosis; Vesicular Monoamine Transporter 2 Inhibitor (VMAT2), a Phase II drug for movement disorders; CRF2 Peptide Agonist, a Phase II drug for cardiovascular diseases; CRF1 Antagonist, a Phase II drug for stress-related disorders; and Elagolix, a Phase II drug for uterine fibroids. Its research programs comprise G Protein-Coupled Receptor 119 (GPR119) for type II diabetes; VMAT2 for schizophrenia; GnRH Antagonists for men?s and women?s health, and oncology; Antiepileptic Drugs for epilepsy, essential tremor, and pain; and G Protein-Coupled Receptors for other conditions. The company has collaborations with GlaxoSmithKline to develop and commercialize CRF antagonists for psychiatric, neurological, and gastrointestinal diseases; Dainippon Sumitomo Pharma Co. Ltd. to develop and commercialize Indiplon in Japan; Abbott International Luxembourg S.�r.l. to develop and commercialize elagolix and GnRH antagonists for women?s and men?s health indications; and Boehringer Ingelheim International GmbH to research, develop, and commercialize small molecule GPR119 agonists for the treatment of type II diabetes and other indications. Neurocrine Biosciences, Inc. was founded in 1992 and is headquartered in San Diego, California.

Advisors' Opinion:

• [By Roberto Pedone]

  Another stock that's starting to trend within range of triggering a major breakout trade is Neurocrine Biosciences (NBIX), which discovers, develops and commercializes drugs for the treatment of neurological and endocrine-related diseases and disorders. This stock has been a hot name with bulls so far in 2013, with shares up 57%.

  If you look at the chart for Neurocrine Biosciences, you'll notice that this stock recently gapped down sharply from $16.74 to below $11.50 a share with heavy downside volume flows. Following that gap down, shares of NBIX went on to continue its trend lower and the stock hit a new low of $10.42 a share. Shares of NBIX have started to rebound off that $10.42 low and it's now moving within range of triggering a major breakout trade.

  Traders should now look for long-biased trades in NBIX if it manages to break out above some near-term overhead resistance levels at its 200-day moving average of $11.92 a share and above its gap down day high of $12.17 a share with high volume. Look for a sustained move or close above those levels with volume that hits near or above its three-month average action of 894,145 shares. If that breakout triggers soon, then NBIX will set up to re-fill some of its previous gap down zone from September that started at $16.74 a share. Some possible upside targets for NBIX if it gets into that gap with volume are $14 to $15 a share.

  Traders can look to buy NBIX off any weakness to anticipate that breakout and simply use a stop that sits right below support at $11 a share, or below that recent low of $10.42 a share. One can also buy NBIX off strength once it takes out that breakout levels with volume and then simply use a stop that sits a comfortable percentage from your entry point.

Top 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.

Advisors' Opinion:

• [By Dan Carroll]

  Biotech's known for volatility, and one of this year's biggest booms suffered a hit on that end this week. Alnylam Pharmaceuticals (NASDAQ: ALNY  ) , which has seen shares explode for gains of more than 142% year to date in 2013, lost more than 11% this past week to rank as one of biotech's biggest busts over the last five days.

• [By Ben Levisohn]

  Markey rates the stock a Buy with an $11 price target. Shares of MannKind have jumped 14% to $7.85 today. The SPDR Biotech ETF (XBI) has gained 0.7% to $119.85 today, while Alnylam Pharmaceuticals (ALNY) has gained 1% to $49.05, NPS Pharmaceuticals (NPSP) has fallen 4.1% to $23.56, and InterMune (ITMN) has risen 2.6% to $14.89.

Top Biotech Companies To Watch In Right Now: Dyadic International Inc (DYAI)

Dyadic International, Inc. (Dyadic), incorporated in September 2002, is a holding company. The Company is a global biotechnology company. The Company has operations at the United States and the Netherlands. Dyadic uses its technologies to conduct research and development (R&D) and commercial activities for the discovery, development, manufacture and sale of enzymes and proteins for the bioenergy, industrial enzyme, and biopharmaceutical industries. The Company derives all of its revenues from the licensing of its technologies, the sale of its enzymes and conducting research and development (R&D) activities for third parties. The Company operates in two segments: the United States operations and The Netherlands operations. The United States segment includes a subsidiary in Poland.

The United States operating segment is a developer, manufacturer and distributor of enzyme products, proteins, peptides and other bio-molecules derived from genes and a collaborative licensor of enabling technologies for the development and manufacturing of biological products and use in R&D. The Netherlands operating segment is also a researcher and developer of enzyme products, proteins, peptides and other bio-molecules derived from genes and, to date, has mainly invested in R&D activities.

Dyadic�� R&D activities focus on its fungal strains and associated technologies. Dyadic uses its Trichoderma and C1 fungal strains in the production of its industrial enzymes. Dyadic manufactures and sells liquid and dry enzyme products to global customers for use within the animal feed, pulp and paper, starch and alcohol, food and brewing, textiles, and biofuels industries.

Dyadic also utilizes a technology platform based on its patented and C1 fungus (the C1 Platform Technology), which enables the development and manufacture of proteins and enzymes for diverse market opportunities. The C1 Platform Technology can also be used to screen for the discovery of novel genes and proteins. The C1 Platf! orm Technology also has the potential of developing and producing other biological products such as antibodies, vaccines, proteins and polypeptides for the biopharmaceutical industry.