The FDA recently instituted the "breakthrough therapy designation" as a way to more quickly get drugs for serious, unmet medical needs to the market. Today, orphan drugmaker Alexion Pharmaceuticals (NASDAQ: ALXN ) �was the latest biotech to announce that its experimental hypophosphatasia drug has been granted this designation. In the following video, health-care analyst Max Macaluso discusses the importance of this news for both patients and investors.
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�Top New Companies To Watch In Right Now: 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 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.
- [By Brian Pacampara]
Based on the aggregated intelligence of 180,000-plus investors participating in Motley Fool CAPS, the Fool's free investing community, biopharmaceutical company TESARO (NASDAQ: TSRO ) has received the dreaded one-star ranking.
Top 10 Medical Stocks 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 10 Medical Stocks For 2014: Actelion Ltd (ATLN.VX)
Actelion Ltd is a Swiss biopharmaceutical holding company that focuses on the discovery, development and commercialization of small molecule drugs. The Company has four approved drugs on the market: Tracleer, an oral dual endothelin receptor antagonist; Veletri, a prostanoid vasodilator; Ventavis, an inhaled formulation of iloprost, and Zavesca, an oral treatment for type 1 Gaucher disease. Furthermore, the Company has a number of compounds various stages of development. The Company operates through a number of worldwide subsidiaries, including Actelion Registration Ltd, which holds marketing authorizations for products marketed in the European Union; Actelion Clinical Research, Inc, engaged in clinical development on behalf of the Company's group; Actelion Re SA, providing insurance solutions for the Company's group and Actelion US Holding Company, engaged in the holding activities of the Company's United States-based units. In September 2013, it acquired Ceptaris Therapeutics, Inc. Advisors' Opinion:- [By Victor Selva]
Forest Laboratories has a current ratio of 2.69% which is higher than the ones registered by Endo International Plc (ENDP), Valeant Pharmaceuticals International (VRX) and Cubist Pharmaceuticals Inc. (CBST). For investors looking for a higher ROE, Allergan Inc. (AGN) and Actelion Ltd. (ATLN.VX) are good options.
Top 10 Medical Stocks For 2014: PTC Therapeutics Inc (PTCT)
PTC Therapeutics, Inc., incorporated on March 31, 1998 , is a biopharmaceutical company focused on the discovery and development of orally administered, small-molecule drugs that target post-transcriptional control processes. The Company�� lead product candidate includes ataluren, which is used for the treatment of patients with genetic disorders that arise from a type of genetic mutation known as a nonsense mutation. Ataluren is in late stage clinical development for the treatment of Duchenne muscular dystrophy caused by nonsense mutations (nmDMD) and cystic fibrosis caused by nonsense mutations (nmCF).
Ataluren is orally administered small-molecule compound that targets nonsense mutations. The Company is engaged in the development of ataluren for the treatment of genetic disorders in, which a nonsense mutation is the cause of the disease. Genetic tests are available for many genetic disorders, including Duchenne muscular dystrophy and cystic fibrosis, to determine if the underlying cause is a nonsense mutation. The EMA has designated ataluren as an orphan medicinal product for the treatment of nmDMD and nmCF. During the year ended December 31, 2012, the Phase III clinical trial completed. The Company�� Ataluren clinical trials in patients with nonsense mutation genetic disorders include Ataluren for nmDMD: Phase 2b clinical trial complete; Confirmatory Phase III clinical trial initiated, and Ataluren for nmCF: Phase III trial completed.
Advisors' Opinion:- [By John Kell]
PTC Therapeutics Inc.(PTCT) said the European Medicines Agency’s Committee for Medicinal Products for Human Use issued a negative opinion on the biopharmaceutical company’s marketing authorization application for its muscular dystrophy treatment. Shares slumped 21% to $20.60 in premarket trading.
- [By WWW.DAILYFINANCE.COM] Records fell on Wall Street Friday as another solid report on housing lifted the market for the second day in a row. There's a three-day weekend coming up -- something that often prompts investor caution -- but the gains were broad-based even though volume was fairly light. The VIX, which measures volatility, fell to its lowest level this year. The Dow Jones industrial average (^DJI) gained 63 points, the Nasdaq composite (^IXIC) rose 31, and the Standard & Poor's 500 index (^GPSC) added 8, topping the record high set last week. The Dow Transportation average also raced to an all-time high, lifted by airline stocks. United (UAL) soared more than 4 percent; Delta (DAL) gained more than 1 percent and Southwest (LUV) gained 2 percent. Southwest is at an all-time high, up 79 percent from a year ago. New home sales bounced back with a better than expected 6.4 percent increase last month. Lennar (LEN) and D.R. Horton (DHI) both rose 4 percent. Pulte (PHM), Beazer (BZH) and Hovnanian (HOV) also solid posted gains. Earnings continue to drive retail stocks. Gap (GPS) edged higher even though net fell. Foot Locker (FL) gained 1½ percent after topping expectations. GameStop (GME) rose 4 percent. Its net rose, helped by the rollout last year of new Xbox and PlayStation consoles. Zumiez (ZUMZ) rose 5½ percent on an earnings beat. But Aeropostale (ARO) tumbled 24 percent. Its loss widened and sales declined. The retailer continues to struggle with teen fashion trends. Also on the earnings front, TiVo (TIVO) rose 2 percent as it swung to a profit from a year ago loss. It also reported an increase in the number of subscribers. Hewlett-Packard (HPQ) rose 6 percent on news the company plans to eliminate up to 16,000 additional workers in an effort to cut costs. And several stocks extended big moves from yesterday. Best Buy (BBY) rose more than 3 percent for the second straight day after earnings beat expectations. Isis Pharmaceuticals (ISIS) jumped
Top 10 Medical Stocks For 2014: Prothena Corporation PLC (PRTA)
Prothena Corporation PLC, incorporated on September 26, 2012, is an Ireland-based, clinical-stage biotechnology company. The Company is engaged in discovering and developing monoclonal antibodies that are directed towards misfolded proteins or improper cell adhesion. Its pipeline includes NEOD001, PRX002 and PRX003. The Company�� work in protein misfolding could result in therapies to treat several neurodegenerative diseases, including AL (primary) and AA (secondary) forms of amyloidosis (NEOD001), Parkinson's disease and related synucleinopathies (PRX002). Its cell adhesion development activities could generate new therapies to treat inflammatory diseases and metastatic cancers (PRX003). The Company�� program, NEOD001, is in Phase 1. In addition to antibodies directed to neo-epitope targets, it is developing antibodies directed to other targets. The Company has generated antibodies against cell adhesion targets expressed on certain pathogenic Th17 immune cells and tumor cells.
The Company�� pipeline also includes several late discovery stage programs for which it is testing antibodies in preclinical models of disease. It is also generating additional antibodies against other targets involved in protein misfolding and cell adhesion for characterization in vivo and in vitro.
NEOD001 for Amyloidosis
NEOD001 is a monoclonal antibody that targets the amyloid that accumulates in both AL and AA forms of amyloidosis. The antibody was designed to not react with normal serum amyloid A and only with the aberrant cleaved form of the protein (amyloid A).
PRX002 for Parkinson�� Disease
The Company has generated antibodies targeting alpha-synuclein that may slow or reduce the neurodegeneration associated with synuclein misfolding and/or transmission. It has tested these antibodies in various cellular and animal models of synuclein-related disease. In a transgenic mouse model of Parkinson�� disease, passive immunization with 9E4, a murine ver! sion of PRX002, reduced the appearance of synuclein pathology, protected synapses and improved performance by the mice in behavioral testing. The humanized antibody product candidate PRX002 has advanced into manufacturing and preclinical testing.
PRX003 for Inflammatory Diseases and Cancers
The Company is developing PRX003, a monoclonal antibody targeting MCAM for the potential treatment of inflammatory diseases and cancers. It has generated monoclonal antibodies that block MCAM-mediated cell adhesion and have been shown to delay relapse and severity of relapse in a mouse model of multiple sclerosis known as experimental autoimmune encephalomyelitis. The Company�� antibodies are being tested in animal models of inflammatory diseases and cancers. Based on early results from these studies, it has identified a clinical candidate, PRX003. It has advanced this antibody into manufacturing and intends to advance this antibody into preclinical testing.
Advisors' Opinion:- [By Jake L'Ecuyer]
Equities Trading UP
Prothena Corporation plc (NASDAQ: PRTA) shares shot up 21.60 percent to $45.19 after the company reported clinical data to be presented at International Symposium on Amyloidosis. Morgan Stanley raised the price target on the stock from $35.00 to $53.00. - [By Lisa Levin]
Prothena Corporation plc (NASDAQ: PRTA) shares surged 23.52% to touch a new 52-week high of $45.90 after the company reported clinical data to be presented at International Symposium on Amyloidosis. Morgan Stanley raised the price target on the stock from $35.00 to $53.00.
- [By Jake L'Ecuyer]
Shares of Prothena Corporation plc (NASDAQ: PRTA) got a boost, shooting up 6.99 percent to $32.00 after the company reported the successful start of a Phase 1 clinical trial of PRX002 for the treatment of Parkinson's disease. Citigroup initiated coverage on the stock with a Buy rating and a $50.00 price target.
Top 10 Medical Stocks For 2014: ImmunoCellular Therapeutics Ltd (IMUC)
ImmunoCellular Therapeutics, Ltd., incorporated on March 20, 1987, is a clinical-stage biotechnology company. The Company is engaged in developing immune-based therapies for the treatment of cancers, such as brain, ovarian and other solid tumors. Immunotherapy is an approach to treat cancer in which a patient�� own immune system is stimulated to target tumor antigens, which are molecular signals that the immune system uses to identify foreign bodies. The Company�� products include ICT-107, ICT-140, ICT-120, ICT-109 and DIAAD. ICT-107, the pipeline product, is a Phase II therapeutic dendritic cell (DC) vaccine for the treatment of glioblastoma multiforme (GBM), the common and lethal type of brain cancer. ICT-107 is designed to activate a patient�� immune system to target six different tumor-associated antigens. In February 2012, it acquired a world-wide license from the University of Pennsylvania and The John Hopkins University (JHU).
The Company in addition to ICT-107, is also developing other therapeutic DC vaccines: ICT-140 for ovarian cancer and ICT-121 for recurrent GBM. ICT-140 targets seven tumor-associated antigens expressed on ovarian cancer cells. ICT-107 is a DC vaccine that targets six different tumor-associated antigens that are found on patients��tumor cells; four of the six antigens are expressed on CSCs. The therapeutic vaccine is used subsequent to conventional therapy or concomitantly with chemotherapy in patients with newly diagnosed GBM. ICT-140 is a DC vaccine that targets seven different ovarian cancer antigens. ICT-121 is a DC vaccine that targets CD133 antigens; it has the potential to be a universal cancer vaccine because CD133 is widely expressed on CSCs from a majority of cancers. Data from this small study demonstrated that ICT-109 had a statistically ability to discriminate between cancerous and non-cancerous samples, suggesting the potential to detect pancreatic and lung cancer in plasma and serum study sets. The DIAAD (differential immunization for an! tigen and antibody discovery) platform it acquired from Molecular Discoveries utilizes immunological tolerization to accelerate the discovery of the molecular differences between diseased cells and their normal counterparts. The monoclonal antibodies produced by DIAAD provide the basis for the discovery and development of its potential diagnostic and therapeutic products.
The Company competes with Dendreon, Oncothyreon, Galena, Bavarian Nordic and Immunovaccine, Northwest Biotherapeutics, Prima Biomed, DCPrime, Roche/Genentech, Seattle Genetics, Bristol-Myers Squibb and Immunogen.
Advisors' Opinion:- [By Lauren Pollock]
ImmunoCellular Therapeutics Ltd.(IMUC) said its leading product candidate, a dendritic cell-based vaccine for brain cancer, didn’t show a statistically significant advantage in overall survival in a Phase II study. In a research note, Maxim Group said it is pushing its EU approval date target to 2019 and lowered its estimate on the stock to $12 a share from $18 a share. The stock plunged 62% to $1.04 premarket.
- [By Wallace Witkowski]
Shares of ImmunoCellular (IMUC) �jumped after the drug developer said data on a mid-stage brain cancer drug study were looking encouraging.
- [By Monica Gerson]
ImmunoCellular Therapeutics (NYSE: IMUC) dropped 11.05% to $1.3699. ImmunoCellular's trailing-twelve-month ROE is -59.80%.
Posted-In: market losersNews Movers & Shakers Intraday Update Markets
Top 10 Medical Stocks For 2014: MedeFile International Inc (MDFI)
Medefile International, Inc.(Medefile), incorporated on July 16, 1997, through its MedeFile, Inc. subsidiary, has developed a patient-centric, Internet-enabled Personal Health Record (iPHR) system for gathering, digitizing, maintaining, accessing and sharing an individual's actual medical records. MedeFile's products and services are designed to provide healthcare providers with the ability to reference their patient's actual past medical records.
MedeFile iPHR
MedeFile is a Business-to-Business and a Business-to-Consumer subscription service. MedeFile is designed to create a cradle to grave longitudinal record for each of its members by retrieving and consolidating copies of their medical records. When the records are received, the MedeFile system consolidates them into a single medically correct format. The records are then stored in Medefile's MedeVault, a secure repository that can be accessed by MedeFile members 24 hours a day, 7 days a week.
MedeFile's Emergency Medical Information (EMI) Card
Upon becoming a MedeFile member, each individual will receive a Membership / Emergency Medical Information (EMI) Card. It contains instructions on how to contact MedeFile in order to retrieve the member's medical records.
The Digital Health Profile (DHP)
A part of a member's MedeFile is their Digital Health Profile (DHP). This form is completed by the patient in order to provide a summary of the patient's healthcare history which assists healthcare providers in understanding the patient's course of medical treatment.
MedeDrive
The MedeDrive is an external universal serial bus (USB) drive, which stores all of a patient's Emergency Medical Information and their MedeFile which can be viewed on a personal computer. MedeDrive self loads its own viewer, so no special program or software is required.
MedeVault
The MedeVault is designed to serve as an electronic data and document repo! sitory that incorporates state-of-the-art security features in order to prevent unauthorized access to a patient's records. Access to the MedeVault is provided through an encrypted connection to a Web service run by MedeFile. This connection is provided by Secure Sockets Layer (SSL) technology.
MedeMinder
MedeMinder is MedeFile's reminder service. The member tells the Company when and where to call, and the Company automatically contacts the member day or night with an appropriate reminder, spoken by real people.
MedePro
During the year ended December 31, 2011, the Company introduced MedePro. MedePro is a medical record retrieval and document management solution created specifically by MedeFile for legal and insurance professionals.
SecurMed
SecurMed is designed to serve as an authentication process. SecurMed protects against any information being viewed by unauthorized persons.
The Company competes with GE Healthcare, Bio-Imaging Technologies, and Cyber Records.
Advisors' Opinion:- [By Peter Graham]
Small cap healthcare and lifestyle stocks Axxess Pharma Inc (OTCMKTS: AXXE), Medefile International Inc (OTCMKTS: MDFI) and Intelligent Living Inc (OTCMKTS: ILIV) have all been getting some extra attention lately thanks in part to a few disclosed paid promotions or investor relations type of activities. But just how healthy are these small cap stocks for investors and traders alike? Here is a quick reality check:
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