Finally Delivering An Effective Therapy For Cystic Fibrosis
Although cystic fibrosis has long been used as an example of where gene therapy could eventually improve clinical outcomes, it turns out that more conventional chemistry can still play a meaningful role. Between Kalydeco and two clinical CTFR ��orrectors�� Vertex has made a major step forward in the treatment of a disease that while not as lethal as it once was (at least in the U.S. and EU), is still a very serious and difficult to manage chronic illness.
Best Life Sciences Stocks To Watch For 2015: 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]
Yet no stock in biotech had a week like Alnylam Pharmaceuticals (NASDAQ: ALNY ) , whose stock shot up more than 32% over the past five days. Company shares rocketed up 19% on Thursday alone, after the company released early-stage clinical results that showed Alnylam's developmental therapy ALN-TTRsc, a treatment for a rare genetic disorder, reduced levels of the disease, transthyretin (or TTR) amyloidosis, by 80%. Alnylam also noted that the drug had been well received by clinical subjects, a critical key for investors cautious about the drug's safety.
- [By Markus Aarnio]
2. Alnylam Pharmaceuticals (ALNY), a biopharmaceutical company, engages in discovering, developing, and commercializing novel therapeutics based on RNA interference [RNAi].
- [By Keith Speights]
Early validation
Alnylam Pharmaceuticals (NASDAQ: ALNY ) was another big winner this week, with shares jumping 32%. Good news from an early-stage study powered the stock's climb. - [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.
Top Biotech Companies To Own For 2014: Clovis Oncology Inc (CLVS)
Clovis Oncology, Inc. (Clovis) incorporated on April 20, 2009, is a Development-stage Company. The Company is a Biopharmaceutical Company. The Company focuses on acquiring, developing and commercializing anti-cancer agents in the United States, Europe and additional international markets. The Company also focuses on the treatment of specific subsets of cancer populations. During the year ended December 31, 2010, the Company was in the process of developing three product candidates for which it holds global marketing rights: CO-101, a lipid-conjugated form of the anti-cancer drug gemcitabine; CO-1686, an oral epidermal growth factor receptor (EGFR) mutant-selective inhibitor and CO-338, a poly ADP (Adenosine Diphosphate)-ribose polymerase (PARP) inhibitor. Effective November 19, 2013, Clovis Oncology Inc acquired the entire share capital of Ethical Oncology Science SpA.
CO-101 - a Lipid-Conjugated Form of the Anti-Cancer Drug Gemcitabine
CO-101 is designed to treat patients with pancreatic cancer whose tumors express low amounts of a membrane transporter protein on the surface of the cancer cell known as hENT1 and are thus expected to be resistant to standard gemcitabine-based therapy. Based on the published results of multiple studies assessing the correlation of hENT1 expression to survival outcomes in pancreatic cancer patients treated with gemcitabine, the Company estimates that approximately 40% to 50% of pancreatic patients express low levels of hENT1, and thus derive little or no benefit from gemcitabine therapy. CO-101 is in an international, randomized and controlled 360-patient study for the first-line treatment of metastatic pancreatic cancer. This open-label study compares CO-101 to gemcitabine as a first-line therapy in patients with metastatic pancreatic cancer. Clovis is partnered with Ventana Medical Systems for the development and commercialization of a companion diagnostic for the assessment of hENT1 levels.
CO-1686-an Oral EGFR Mutant-Selective ! Inhibitor
CO-1686 is an orally available, small molecule covalent inhibitor of the cancer-causing mutant forms of EGFR for the treatment of non-small cell lung cancer (NSCLC). CO-1686 targets both the initial activating EGFR mutations as well as the primary resistance mutation, T790M, it treats both first- and second-line NSCLC patients with EGFR mutations. Such initiating activating mutations occur in approximately 10% to 15% of NSCLC cases in Caucasian patients and approximately 30% to 35% of NSCLC cases in East Asian patients. Following treatment with approved NSCLC therapies, Tarceva (erlotinib) or Iressa (gefitinib), both known as tyrosine kinase inhibitors (TKIs), approximately half of these patients develop the T790M mutation.
The Company focuses on the development of CO-1686 as both a second-line therapy for EGFR-mutated NSCLC patients who become resistant to TKIs due to the emergence of the T790M secondary mutation and as a first-line treatment for EGFR-mutated NSCLC. Clovis is partnered with Roche Molecular Systems for the development and commercialization of a companion diagnostic for identification of EGFR mutations.
CO-338-a PARP Inhibitor
CO-338 is a small molecule PARP inhibitor that the Company focuses to develop as both monotherapy and in combination with chemotherapeutic agents for the treatment of selected cancer patients. CO-338 is in a Phase I clinical trial to determine the maximum tolerated dose of oral CO-338 that can be combined with intravenous, or IV, platinum chemotherapy in the treatment of solid tumors. This program is supplemented by two ongoing investigator-initiated trials, using the IV formulation of CO-338: a Phase I/II study in germ-line BRCA mutant breast and ovarian cancer and a Phase II study in the adjuvant treatment of germ-line BRCA mutant and triple-negative breast cancer. The Company also focuses on initiating a Phase I monotherapy study of the oral formulation, to determine an appropriate dose and schedule.!
The Company competes with Eli Lilly, Teva Pharmaceutical Industries, APP Pharmaceuticals, AB Science SA, Amgen Inc., Astellas Pharma, BioSante Pharmaceuticals, Inc., Celgene Corporation, Immunomedics, Inc., Lorus Therapeutics, Threshold Pharmaceuticals, Inc., Boehringer Ingelheim, Pfizer, Sanofi-Aventis, Astra Zeneca, Abbott, Merck, Eisai, Cephalon and Biomarin.
Advisors' Opinion:- [By James E. Brumley]
Cancer drug investors who have been disappointed in recent results from shares of Clovis Oncology Inc. (NASDAQ:CLVS) or Nuvilex Inc. (OTCMKTS:NVLX) lately may want to take a look at ZIOPHARM Oncology Inc. (NASDAQ:ZIOP) as a replacement for either of those first two stocks. CLVS is down about 16% for the week on a less-than-flattering write-up in a Bloomberg publication, and NVLX has moved under a pair of key moving averages this week because, well, for no specific reason, but broadly because the recent wave of compelling news is already losing its potency, with most of that upside already being priced into shares (and then some) before it became official.
Top Biotech Companies To Own For 2014: Redpoint Bio Corp (RPBC)
Redpoint Bio Corporation (Redpoint), incorporated in August 1995, is a development-stage biotechnology company. The Company leverages discoveries in the molecular biology of taste to discover and develop taste modulators for the food and beverage industries. Its food and beverage program has been focused on identifying flavor modifiers that improve the taste of ingredients. In June 2009, the Company announced that it had identified an all-natural sweetness enhancer, RP44. RP44 is Reb-C (rebaudioside C), a component of the stevia plant.
Sweetness Enhancer Program
Redpoint announced that it had identified RP44, an all-natural sweetness enhancer, in June 2009. In January 2010, it disclosed that RP44 is Reb-C (rebaudioside C), a component of the stevia plant. RP44 is derived from material found in the side stream of the Reb A production process. A purified component of stevia known as Reb A has received regulatory approval in the United States. Unlike Reb A, RP44 has a low intrinsic level of sweetness and therefore is not useful as a sweetener.
Diabetes and Obesity Drug Discovery Program
The Company�� initial programs focused on the modulation of the TRPm5 ion channel, a signaling element in taste sensation, in order to discover compounds that modulate the taste of food and beverage products. Redpoint initiated a program designed to leverage the research it had already conducted on the discovery of modulators of the TRPm5 ion channel to further explore opportunities for the discovery of diabetes or obesity therapeutics. TRPm5 modulators discovered at Redpoint have been shown to elicit the secretion of hormones known to play roles in metabolism in relevant model systems.
The Company competes with International Flavors & Fragrances Inc., Givaudan SA, Symrise, Firmenich, Senomyx, PureCircle Ltd, GLG Life Tech Corporation, Eli Lilly and Company, Amylin Pharmaceuticals, Merck & Co., Inc., Metabolex, Inc., OSI Pharmaceuticals, GlaxoSmithKline! , Arena Pharmaceuticals, Inc. and Johnson & Johnson.
Advisors' Opinion:- [By Peter Graham]
Small cap stocks Greenfield Farms Food Inc (OTCMKTS: GRAS), International Stem Cell Corp (OTCMKTS: ISCO) and Redpoint Bio Corporation (OTCMKTS: RPBC) have all been getting some extra attention lately in various investment newsletters. However, none of these small cap stocks appear to have been the subject or paid promotions or investor relations activities. So does that make any of them good bets for traders and investors alike? Here is a quick look and a reality check:
Top Biotech Companies To Own For 2014: TESARO Inc (TSRO)
TESARO, Inc. (TESARO), incorporated in March 2010, is a development-stage, oncology-focused biopharmaceutical company for cancer patients. The Company focuses on rolapitant and TSR-011 product. The Company�� marketed products and product candidates in development treat cancer through non-specific damage to cellular components or alter cell metabolism or internal repair mechanisms to the demise of cancer cells.
Rolapitant
Rolapitant is a potent and long-acting neurokinin-1, or NK-1, receptor antagonist is in Phase III clinical trials for the prevention of chemotherapy induced nausea and vomiting (CINV). It is in Phase III clinical trials. CINV, if not prevented by prophylaxis, has the potential to afflict up to 90% or more of cancer patients undergoing chemotherapy, depending upon the type of chemotherapy administered the dosing schedule of the chemotherapy, and the patients' age and gender, among other predisposing factors. Prolonged nausea and vomiting may result in unwanted weight loss, dehydration and malnutrition, as well as hospitalization. The Company has in-licensed the rights to rolapitant from OPKO Health, Inc.
TSR-011
TSR-011 is an orally available ALK inhibitor in preclinical development. ALK is known to be involved in certain types of cancers, including subsets of NSCLC, neuroblastoma and lymphoma. For patients in these subsets, the ALK gene is fused to an activating partner or contains point mutations, resulting in constitutive activation of ALK and the growth of cancer cells and tumor development. Inhibition of ALK in these cancer cells results in cell death and tumor growth inhibition or regression. In August 2011, the United States Food and Drug Administration approved the first ALK inhibitor, developed by Pfizer Inc., Xalkori (crizotinib), which was approved for the treatment of patients with locally advanced or metastatic NSCLC that are ALK positive.
The Company competes with GlaxoSmithKline plc, Roche Holding Ltd! . and Sanofi S.A.
Advisors' Opinion:- [By Jake L'Ecuyer]
Equities Trading UP
Tesaro (NASDAQ: TSRO) shares shot up 18.28 percent to $28.40 after the company reported successful primary and secondary endpoints in final Phase 3 trial for rolapitant. - [By Ben Levisohn]
Werber and Eckhard’s favorites include Gilead (GILD) and Celgene (CELG), and they find the “risk/reward…compelling” in Medivation (MDVN) and Tesaro (TSRO).
- [By Keith Speights]
Positive response
Clovis wasn't the only company to bring good news about an ovarian cancer drug to ASCO. TESARO� (NASDAQ: TSRO ) announced phase 1 results for�niraparib. Shares of the biotech jumped 16% for the week. - [By Brian Orelli]
Tesaro (NASDAQ: TSRO ) is also up today after announcing that it established a partnership with clinicians to run the phase 3 development of its PARP inhibitor, niraparib. The trial will enroll about 300 breast cancer patients with BRCA mutations, comparing niraparib to investigators' choice of other breast cancer treatments.
Top Biotech Companies To Own For 2014: Receptos Inc (RCPT)
Receptos, Inc. (Receptos), incorporated on June 9, 2008, is a biopharmaceutical company. The Company is focused on discovering, developing and commercializing therapeutics for immune disorders. The Company�� product candidates span three specialty disease areas. The Company�� lead asset, RPC1063, is being developed as an oral therapy for the treatment of relapsing multiple sclerosis (RMS) and inflammatory bowel disease (IBD). Its second asset, RPC4046, is being developed for the treatment of an allergic/immune-mediated disorder, eosinophilic esophagitis (EoE), which is an orphan disease. RPC1063 is an oral, once daily, selective and potent sphingosine 1-phosphate 1 receptor (S1P1R) modulator. RPC4046 is a monoclonal antibody selective to interleukin-13 (IL-13) and produced by recombinant deoxyribonucleic acid (DNA) technology.
As of December 31, 2012, RPC1063 was being tested in the Phase II portion of an accelerated design, randomized Phase II/III study for the treatment of RMS. In addition, Receptos has obtained special protocol assessment (SPA) agreement from the United States food and drug administration (FDA) on its clinical trial design for the planned Phase III portion of the Phase II/III study, as well as a second planned RMS Phase III study. RPC1063 is also being tested in a randomized Phase II study for the treatment of ulcerative colitis (UC), a gastrointestinal (GI) disea se.
As of December 31, 2012, the Company was enrolling a randomized Phase II study evaluating the ability of RPC1063 to induce clinical remission in patients with moderately-to-severely active UC called TOUCHSTONE. The Company�� second asset, RPC4046 for the treatment of EoE, builds upon its competencies in immunology and GI diseases. In-licensed from AbbVie Bahamas Ltd. and AbbVie Inc. (AbbVie) RPC4046 is a monoclonal antibody directed against the IL-13 target, which has been validated in Asthma, a predominantly allergic/immune-mediated disorder.
Advisors' Opinion:- [By RichardCox]
For those willing to take on some added volatility (and potential for increased risk based on less diversification), investors can look at Receptos Inc. (RCPT), to play this year's momentum in IPO entries. Enhanced expectations for the company's IPO drove the company to raise its initial share offering to 5.2 million shares. Receptos has reached late-stage development for its oral treatment for multiple sclerosis (MS). MS treatments are dominated by industry heavyweights but MS pills are expected to surge in the next few years because oral treatments are becoming more efficient than injected therapies, and can cater to a broader consumer base. Being a new company, the Receptos chart only offers limited incite, and will not be included here.
- [By Roberto Pedone]
Receptos (RCPT) is a biopharmaceutical company engaged in discovering, developing and commercializing therapeutics for immune disorders. This stock closed up 5% at $23.19 in Monday's trading session.
Monday's Volume: 69,000
Three-Month Average Volume: 55,356
Volume % Change: 50%From a technical perspective, RCPT ripped higher here right above some key near-term support at $21 with decent upside volume. This move is quickly pushing shares of RCPT within range of triggering a major breakout trade. That trade will hit if RCPT manages to take out its all-time high at $25 with high volume.
Traders should now look for long-biased trades in RCPT as long as it's trending above support at $21 and then once it sustains a move or close above its all-time high at $25 with volume that's near or above 55,356 shares. If that breakout hits soon, then RCPT will set up to enter new all-time-high territory, which is bullish technical price action. Some possible upside targets off that breakout are $30 to $35.
- [By John Udovich]
Small cap �biopharmaceutical stock Receptos Inc (NASDAQ: RCPT) was one of the many hot biotech IPOs of last year and its also up 72.4% since the start of this year alone, meaning its time to take a closer look at this small cap stock with potential treatments for Relapsing Multiple Sclerosis (RMS) and Inflammatory Bowel Disease (IBD) along with the performance of biotech ETF benchmarks like the iShares NASDAQ Biotechnology Index ETF (NASDAQ: IBB) and SPDR S&P Biotech ETF (NYSEARCA: XBI).
Top Biotech Companies To Own For 2014: Galectin Therapeutics Inc (GALT)
Galectin Therapeutics Inc., formerly Pro-Pharmaceuticals, Inc., incorporated on January 26, 2001, is a development-stage company. The Company is engaged in drug development to create therapies for cancer and fibrotic disease. As of December 31, 2011, the Company has two compounds in development, one is to be used in cancer therapy and the other intended to be used in the treatment of liver fibrosis and fatty liver disease. These two compounds are produced from different natural starting materials, both possessing the property, which lends itself to binding to and inhibiting galectin proteins. GM-CT-01, the Company's product candidate for cancer therapy, is a linear polysaccharide polymer consisted of mannose and galactose that has a defined chemical structure and is derived from a plant source. GR-MD-02, the Company's product for treatment of liver fibrosis and fatty liver disease with inflammation and fibrosis, is a polysaccharide polymer possessing both linear and globular structures, which also is derived from a plant source.
GM-CT-01 has in development for the therapy of colorectal cancer and is in a Phase I/II clinical trial as a combination therapy with a tumor vaccine in patients with advanced melanoma. Based on the completed Phase I and partially completed Phase II clinical trials, the Company is exploring two additional potential indicia for the use of GM-CT-01 in combination with cancer chemotherapy. There are two additional pathways for the development of GM-CT-01 for use in treatment of cancer. GM-CT-01 was found to be generally safe when studied in a Phase I clinical trial in end-stage cancer patients with multiple tumor types alone and in combination with 5-Fluorouracil (5-FU), which is an Food and Drug Administration (FDA)-approved chemotherapy used for treatment of various types of cancer.
Advisors' Opinion:- [By Roberto Pedone]
Galectin Therapeutics (GALT) offers drug research and development to create new therapies for fibrotic disease and cancer. This stock closed up 9.6% to $12.06 in Monday's trading session.
Monday's Volume: 674,000
Three-Month Average Volume: 222,171
Volume % Change: 149%Shares of GALT jumped higher on Monday after Ascendiant initiated coverage on the stock with a buy recommendation.
From a technical perspective, GALT spiked sharply higher here with strong upside volume. This stock has been uptrending for the last three months, with shares ripping higher from its low of $3.95 to its recent high of $13.21. During that move, shares of GALT have been making mostly higher lows and higher highs, which is bullish technical price action. That move has now pushed shares of GALT within range of triggering a near-term breakout trade. That trade will hit if GALT manages to take out Monday's high of $12.44 and then once it clears its 52-week high at $13.21 with high volume.
Traders should now look for long-biased trades in GALT as long as it's trending above some near-term support levels at $11 or at $10 and then once it sustains a move or close above those breakout levels with volume that hits near or above 222,171 shares. If that breakout hits soon, then GALT will set up to enter new 52-week-high territory above $13.21, which is bullish technical price action. Some possible upside targets off that breakout are $15 to $16.
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