The biotech industry is a marvel among traders and investors because it can double your investment, or halve it, based on a single clinical study. Millions, sometimes billions, of research dollars are poured into developing new drugs and bringing these therapies to market -- $49 billion throughout the entire industry last year, according to PhRMA. However, a vast majority of drugs discovered in the laboratory setting will never see the light of day.
Source: Idaho National Laboratory, Flickr.
Based on data from Medscape, only about one in every 5,000 to 10,000 researched drugs in preclinical trials will be approved by the Food and Drug Administration. This means there's quite the premium given to biotech stocks because of solid clinical data, and quite the haircut when it doesn't pan out.
Top 5 Bank Stocks To Watch For 2015: Merck & Company Inc.(MRK)
Merck & Co., Inc. provides various health solutions through its prescription medicines, vaccines, biologic therapies, animal health, and consumer care products. The company?s Pharmaceutical segment provides human health pharmaceutical products, such as therapeutic and preventive agents for the treatment of human disorders in the areas of bone, respiratory, immunology, dermatology, cardiovascular, diabetes and obesity, infectious diseases, neurosciences and ophthalmology, oncology, vaccines, and women's health and endocrine. This segment also offers human health vaccines, such as preventive pediatric, adolescent, and adult vaccines. Its Animal Health segment discovers, develops, manufactures, and markets animal health products. This segment offers antibiotics, anti-inflammatory products, vaccines, products for the treatment of fertility disorders, and parasiticides for cattle, swine, horses, poultry, dogs, cats, salmons, and fish. The Consumer Care segment develops, manufac tures, and markets over-the-counter, foot care, and sun care products. Its over-the-counter product line includes non-drowsy antihistamines; treatment for occasional constipation; decongestant-free cold/flu medicine for people with high blood pressure; nasal decongestant spray; and treatment for frequent heartburn. This segment?s foot care products comprise topical antifungal, and foot and sneaker odor/wetness products; and sun care products include sun care lotions, sprays and dry oils; and sunburn relief products. The company serves drug wholesalers and retailers, hospitals, government agencies, physicians, physician distributors, veterinarians, animal producers, and managed health care providers, as well as food chain and mass merchandiser outlets in the United States and Canada. Merck & Co., Inc. was founded in 1891 and is headquartered in Whitehouse Station, New Jersey.
Advisors' Opinion:- [By Matt Thalman]
Shares of Merck (NYSE: MRK ) rose by 0.5% after it received a complete response letter pertaining to its investigational medicine focused on treating insomnia called suvorexant. The letter from the FDA explained that it didn't feel it was safe to start any patient, no matter the age, on any dose of the drug higher than 10 mg. Merck had previously wanted to start the elderly on 15 mg and move up to 30 mg if everything went well, while starting the non-elderly at 20 mg and possibly bumping them to a 40 mg dose if needed. The decision to not allow Merck to start above 10 mg will likely push the drug's rollout date back, as the company said it will have to performer further testing on the 10 mg dosage of suvorexant. �
Top Biotech Stocks To Own 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�� 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 ��ife 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. �
- [By Sean Williams]
With this in mind, I feel it'd be prudent of biotech-savvy investors to give Oncolytics Biotech (NASDAQ: ONCY ) a closer look.
The big risks
I'm quite aware that there are a lot factors that'd raise a red flag with Oncolytics. Similar to Affymax, you could say that Oncolytics has put all of its eggs in one basket with its lead experimental drug, reolysin. According to Oncolytics' website, including its U.K., Canadian, and U.S. studies, reolysin as either a monotherapy or combination therapy is the basis for all 31 clinical trials! Obviously, if reolysin proves ineffective or unsafe, Oncolytics is going to be a world of hurt.
Top Biotech Stocks To Own For 2014: BIND Therapeutics Inc (BIND)
BIND Therapeutics, Inc., incorporated on May 19, 2006, is a clinical-stage nanomedicine platform company developing Accurins, its targeted and programmable therapeutics. Accurins are designed with specified physical and chemical characteristics to target specific cells or tissues and concentrate a therapeutic payload at the site of disease to enhance efficacy while minimizing adverse effects on healthy tissues. Its drug candidate, BIND-014, is in Phase II clinical trials for non-small cell lung cancer, or NSCLC, and metastatic castrate-resistant prostate cancer (mCRPC).
Accurins represent the evolution of targeted therapies and nanomedicine. Accurins are polymeric nanoparticles that incorporate a therapeutic payload and are designed to have prolonged circulation within the bloodstream, enable targeting of the diseased tissue or cells, and provide for the controlled and timely release of the therapeutic payload. The four components include Targeting ligands, Stealth and protective layer, Controlled-release polymer matrix and Therapeutic payload. The Company focuses to use its medicinal nanoengineering platform to develop Accurins in several therapeutic areas, with an initial focus on the treatment of various types of cancer. In addition, the Company entered into collaboration agreements with several biopharmaceutical companies to develop and commercialize Accurins that are based on its collaborators��therapeutic payloads. The Company�� programs include BIND-014, solid tumor accurin and hematologic cancer accurin.
Advisors' Opinion:- [By John Udovich]
The biotech sector has been pretty exciting this year�with small cap biotech stocks Prana Biotechnology Limited (NASDAQ: PRAN) and TNI BioTech (OTCMKTS: TNIB) having recently produced noteworthy news for investors�while Acceleron Pharma, Inc (NASDAQ: XLRN), Ophthotech (NASDAQ: OPHT) and BIND Therapeutics (NASDAQ: BIND) have just�set term sheets for their upcoming IPOs. Just consider all of the following recent news:
- [By Anna Prior]
Bind Therapeutics sa(BIND)id a collaboration with Amgen Inc.(AMGN) has been ended after both companies agreed not to pursue an option to jointly develop a molecularly targeted cancer therapy. Bind Therapeutics shares fell 11% to $11.50 premarket.
Top Biotech Stocks To Own For 2014: Stemline Therapeutics Inc (STML)
Stemline Therapeutics, Inc. (Stemline), incorporated on August 8, 2003, is a clinical-stage biopharmaceutical company focused on discovering, acquiring, developing and commercializing therapeutics that target both cancer stem cells (CSCs) and tumor bulk. The Company is developing two clinical-stage product candidates, SL-401 and SL-701, for which it holds global marketing rights. The indication for SL-401, a biologic-drug conjugate, is acute myeloid leukemia (AML). The indications for SL-701, a synthetic peptide vaccine, are pediatric and adult brain cancer. It has a platform, StemScreen, for the discovery of CSC-targeted compounds, from which it has discovered or validated several of its clinical and preclinical product candidates. Stemline�� StemScreen consists of StemScreen-1 and StemScreen-2 for the identification of CSC-directed compounds.
SL-301 is a small molecule gamma-secretase inhibitor that inhibits Notch, a pathway expressed by CSCs and tumor bulk of multiple cancer types. SL-101 is a monoclonal antibody-based (mAb -based) compound that targets CD123 and has shown in vitro activity against certain hematologic cancers. SL-201 is a small molecule active against certain hematologic and solid tumor types. SL-601 is a mAb-based compound that targets a cell surface marker on bladder CSCs, which is also expressed on a variety of other solid tumor types. It has also in-licensed certain intellectual property directed to mAb-based therapeutics to validated oncology targets, including Glypican-3, Tie-1, CD133, Frizzled, Smoothened and Patched.
SL-401 - An IL-3R-Directed Compound Targeting Cancer Stem Cells and Tumor Bulk
SL-401 is a clinically active biologic-drug conjugate consisting of human interleukin-3 (IL-3) genetically linked to a truncated version of diphtheria toxin. SL-401 targets the IL-3 receptor (IL-3R), which is overexpressed on both the CSCs and tumor bulk of multiple hematologic cancers, including AML. SL-401 has demonstrated preclinical in vit! ro and in vivo activity against both leukemia blasts (which includes tumor bulk) and CSCs of a range of human leukemia cell lines and primary leukemia cells from patients.
SL-701
SL-701 is a clinically active synthetic peptide vaccine that targets several epitopes on CSCs and tumor bulk of brain cancer. In two completed Phase 1/2 clinical trials, SL-701 demonstrated single agent anti-tumor activity in pediatric patients with newly diagnosed brainstem glioma (BSG) and other high-grade gliomas (HGGs) and in adult patients with refractory or recurrent GBM, and other HGGs.
StemScreen-1
StemScreen-1 is a drug discovery platform designed to identify CSC-targeted compounds based on the isolation of CSCs and evaluation of CSC gene expression profiles. CSCs are isolated from primary tumor tissue or cell lines, and then subjected to gene expression analysis using a variety of technologies, including microarray. A control tissue, such as normal bone marrow is analyzed as a comparator against the gene expression profile of the isolated CSCs. These data are then interfaced with an information base of compounds and their mechanisms of action (that is which gene products and pathways they impact). It has utilized StemScreen-1 to discover a number of its preclinical drug candidates. These include SL-201, SL-301, and SL-601. In addition, SL-401 demonstrated activity against CSCs as determined by both an in vitro colony formation and in vivo animal implantation assay, thereby validating certain StemScreen-1 anti-CSC assays.
StemScreen-2
StemScreen-2 is a high throughput drug discovery platform it is developing to discover anti-CSC compounds. StemScreen-2 utilizes a cell-based assay that can track and follow CSCs in their natural state during high throughput screening. In particular, StemScreen-2 utilizes a CSC-specific promoter linked to a reporter as a method for identifying and following CSCs in their native environment of surrounding tumor b! ulk. In t! his way, StemScreen-2 enables the identification of compound hits, in a high throughput manner, with anti-CSC activity.
The Company competes with Boston Biomedical, Inc., Eclipse Therapeutics, Inc., OncoMed Pharmaceuticals, Inc., Verastem, Inc., Astellas Pharma US, Inc., Boehringer Ingelheim GmbH, Dainippon Sumitomo Pharma Co. Ltd., Geron Corp., GlaxoSmithKline plc, ImmunoCellular Therapeutics, Ltd, Macrogenics Inc., Amgen, Inc., Pfizer Inc., Roche Holding AG, Sanofi U.S. LLC., Cyclacel Pharmaceuticals, Inc., Sunesis Pharmaceuticals Inc., Clavis Pharma ASA, Ambit Biosciences Corporation, Celgene Corporation, Eisai Co. Ltd., Celator Pharmaceuticals, Inc., Merck & Co., Inc., Eisai Co., Inc., Roche Holding AG, Novartis AG and Celldex Therapeutics, Inc.
Advisors' Opinion:- [By Keith Speights]
Best-performing biotech IPO
Stemline Therapeutics� (NASDAQ: STML ) has only traded publicly this year, but what a year it's been. The stock's performance ranks Stemline as the best-performing biotech IPO so far in 2013. This week has been pretty good also, with shares moving up by 28%. - [By David Zeiler]
3. Stemline Therapeutics Inc. (Nasdaq: STML): This biotech develops drugs that target cancer stem cells and tumors. Stemline went public January 29 at $10 a share and rose just 11.78% on its first day. But STML has climbed steadily since, and currently trades at $37.46, up 274.6% from its IPO price.
- [By Jay Silverman]
Some of the biggest leaders in that field, and there have been dozens in fields, if not more this year, such as Bluebird (BLUE) and Stemline Therapeutics (STML) and have all pulled back to significantly lower levels; even below, in Bluebird's case, the price that had actually opened up as an IPO, even though it's above its IPO price.
Top Biotech Stocks To Own For 2014: KYTHERA Biopharmaceuticals Inc (KYTH)
KYTHERA Biopharmaceuticals, Inc., incorporated in June 2004, is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of prescription products for the aesthetic medicine market. The Company�� initial focus is on the facial aesthetics market. The Company�� product candidate, ATX-101, is a injectable drug in Phase III clinical development for the reduction of submental fat, which commonly presents as an undesirable double chin. Based on clinical trials conducted, ATX-101 has exhibited results in the reduction of submental fat. ATX-101 contains a synthetic form of sodium deoxycholate. In the United States and Canada, the Company is conducting two pivotal Phase III trials of ATX-101 for the reduction of submental fat. The Company initiated this pivotal Phase III clinical program, with planned enrollment of 1,000 patients, in March 2012.
In Europe, Bayer, its collaborator outside the United States and Canada, recently completed two pivotal Phase III trials of ATX-101 for the reduction of submental fat. In these multi-center, randomized, double-blind, placebo-controlled pivotal trials involving 723 patients, ATX-101 resulted in a reduction in submental fat, as assessed by a validated clinician scale and a patient satisfaction scale.
ATX-101 contains a synthetic form of sodium deoxycholate. ATX-101 is designed to be a locally-injected drug that causes proximal, preferential destruction of adipocytes, or fat cells, with minimal effect on surrounding tissue. Upon subcutaneous injection under the skin, ATX-101 disrupts cell membranes in protein-poor tissues, such as fat, while being attenuated by interactions with protein-rich tissue, such as skin, muscle and blood vessels. This attenuation by protein-rich tissue results in the preferential destruction of adipocytes by ATX-101. The destruction of adipocytes, or adipocytolysis, elicits a natural response, in which macrophages are attracted to remove cellular debris and fat particles t! hrough the lymphatic system. The macrophages also emit low levels of chemical messengers, known as cytokines, which attract fibroblasts, another cell type, to the area. Fibroblasts produce collagen, and it is believed that new collagen production, or neocollagenesis, promotes retraction of the skin in the areas of fat reduction. The fat removal process with ATX-101 is incremental with each treatment, thereby allowing for control of the aesthetic outcome.
Advisors' Opinion:- [By Lisa Levin]
Kythera Biopharmaceuticals (NASDAQ: KYTH) jumped 24.43% to $41.72 after the company reported positive ATX-101 top line phase III trial results for the reduction of submental fat.
- [By John Udovich]
On Tuesday, small cap biotech stock Kythera Biopharmaceuticals Inc (NASDAQ: KYTH) surged around 25% after announcing that its ATX-101 REFINE-1 and REFINE-2 Phase III trials met all primary and secondary endpoints for the reduction of so-called double chins; but if investors missed out on that rally, small caps Zeltiq Aesthetics Inc (NASDAQ: ZLTQ), Solta Medical Inc (NASDAQ: SLTM) and Cynosure, Inc (NASDAQ: CYNO) each have a piece of the aesthetic market as well. In the case of Kythera Biopharmaceuticals, its ATX-101 can be injected to deal with double chins���meaning its less invasive than liposuction as the drug dissolves fat cells but leaves other tissue alone. JP Morgan has noted:
- [By Jake L'Ecuyer]
Equities Trading UP
Repros Therapeutics (NASDAQ: RPRX) shot up 29.82 percent to $27.60 after the company announced topline results from both the second pivotal efficacy study as well as the 6 month safety study of Androxal庐. Shares of Kythera Biopharmaceuticals (NASDAQ: KYTH) got a boost, shooting up 26.04 percent to $42.26 after the company reported positive ATX-101 top line phase III trial results for the reduction of submental fat. Aeropostale (NYSE: ARO) was also up, gaining 16.75 percent to $10.05 after private equity firm Sycamore Partners reported that it had bought a 7.96 percent stake in the company.
Top Biotech Stocks To Own For 2014: Protalix Biotherapeutics Inc (PLX)
Protalix BioTherapeutics, Inc. is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins based on its ProCellEx protein expression system, ProCellEx. Using its ProCellEx system, the Company is developing a pipeline of biosimilar or generic versions of recombinant therapeutic proteins based on its plant cell-based expression technology, which focuses pharmaceutical markets and that rely upon known biological mechanisms of action. ProCellEx protein expression system consists of a set of technologies and capabilities for the development of recombinant proteins, including advanced genetic engineering technology and plant cell-based protein expression methods. Its ProCellEx protein expression system is built on flexible custom-designed bioreactors made of polyethylene and optimized for the development of complex proteins in plant cell cultures. In June 2010, it had completed the preliminary phase I clinical trial of PRX-105.
Taliglucerase Alfa
Taliglucerase alfa is a plant cell expressed recombinant glucocerebrosidase enzyme (GCD) for the treatment of Gaucher disease. The Company has commenced pre-clinical studies of an oral form of taliglucerase alfa. Its oral taliglucerase alfa is a plant cell expressed form of GCD that is naturally encapsulated within carrot cells genetically engineered to express the GCD enzyme. Pre-clinical studies of oral taliglucerase alfa demonstrate the stability of the enzyme in the cell and the capacity of the cell�� cellulose wall to protect the enzyme against degradation in the digestive tract in an in-vitro model of the stomach and intestines. Additionally, rats fed with lyophilized carrot cells expressing GCD have accumulated the active enzyme in the target organs; the spleen and liver. As of December 31, 2010, the Company had completed Phase III Clinical Trial.
PRX-102
The Company is developing PRX-102, its plant cell expressed modified version of the recombinant ! alpha-GAL-A protein, a therapeutic enzyme for the treatment of Fabry disease. Fabry disease is a rare, hereditary, genetic lysosomal storage disorder in humans caused by an X-lined deficiency of the alpha-GAL-A enzyme. The Company is in the animal evaluation testing phase of the development of PRX-102, which tests are based on a mouse model for Fabry disease.
Acetylcholinesterase
Protalix Ltd. is a wholly owned subsidiary of the Company is licensed the rights to certain technology under a research and license agreement with Yissum Research and Development Company (Yissum) and the Boyce Thompson Institute, Inc. Pursuant to the agreement, the Company is developing PRX-105, a plant cell-based acetylcholinesterase (AChE) and its molecular variants for the use in several therapeutic and prophylactic indications, as well as in a biodefense program and an organophosphate-based pesticide treatment program.
As of December 31, 2010, its in-vitro experiments of PRX-105 have shown that the acetylcholinesterase enzyme in its ProCellEx protein expression system demonstrates biological activity on biochemical and cellular levels. In addition, early animal studies demonstrated that the acetylcholinesterase expressed in its ProCellEx protein expression system was able to treat animals exposed to the nerve gas agent analogues, both when injected with its acetylcholinesterase product candidate immediately before exposure or when injected after exposure. In March 2010, it initiated a preliminary phase I clinical trial of PRX-105, which the Company completed in June 2010.
pr-antiTNF
pr-antiTNF is a candidate for the treatment of certain autoimmune diseases such as rheumatoid arthritis, juvenile idiopathic arthritis, ankylosing, spondylitis, psoriatic arthritis and plaque psoriasis. The Company has designed the antiTNF as pr-antiTNF. pr-antiTNF is a plant cell-expressed recombinant fusion protein made from the soluble form of the human TNF receptor (TNFR), f! used to t! he Fc component of a human antibody domain. pr-antiTNF has an identical amino acid sequence to Enbrel and its in-vitro and preclinical animal studies have demonstrated that pr-antiTNF exhibits similar activity to Enbrel. Specifically, pr-antiTNF binds TNF thereby inhibiting it from binding to cellular surface TNF receptors and protects L929 cells from TNF-induced apoptosis in a dose-dependent manner.
The Company competes with Genzyme, Actelion, Crucell N.V., Biolex, Inc., Chlorogen, Inc., Greenovation Biotech GmbH, Symbiosys, Novartis AG/Sandoz Pharmaceuticals, BioGeneriX AG, Stada Arzneimittel AG, BioPartners GmbH and Teva.
Advisors' Opinion:- [By Maxx Chatsko]
Industrial biotech isn't the only industry headed to Brazil. Protalix (NYSEMKT: PLX ) entered into a technology transfer agreement with Brazil's Ministry of Health last week that will pay the company $280 million. The deal is big news for Protalix's first product, Elelyso/Uplyso, which was developed with partner Pfizer (NYSE: PFE ) . The treatment is approved as an enzyme replacement therapy, or ERT, for adults with type 1 Gaucher disease, and it marks a huge step forward for the future of biomanufacturing. In the following video, Fool contributor Maxx Chatsko explains what this means for the product's commercialization and the adoption of Protalix's novel plant cell-based expression system for therapeutic proteins.
- [By Keith Speights]
Other investors might wish that Pfizer would use some of its cash to acquire a few smaller companies. Protalix BioTherapeutics (NYSEMKT: PLX ) has been mentioned as one possible candidate. The two companies already partner together on Gaucher disease drug Elelyso. In February, Protalix spurred rumors that Pfizer could be interested in buying the company after it announced that it had engaged Citigroup to pursue a "broad array of strategic alternatives."
No comments:
Post a Comment