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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�Best Gas Stocks To Invest In Right Now: Portola Pharmaceuticals Inc (PTLA)
Portola Pharmaceuticals, Inc. (Portola), incorporated on September 2, 2003, is a biopharmaceutical company developing product candidates that have the potential to represent advances in the fields of thrombosis and hematology. The Company is advancing its three wholly owned programs using biomarker and genetic approaches.
Betrixaban
Portola's compound, betrixaban, is an oral, once-daily Factor Xa inhibitor being evaluated in the only biomarker-based Phase-III study for hospital to home prophylaxis of venous thromboembolism (VTE) in acute medically ill patients. Betrixaban's properties particularly suited to potentially demonstrate efficacy without increasing bleeding in this patient population.
Andexanet Alfa
Portola's second development candidate, andexanet alfa (PRT4445), has the potential to be a reversal agent to directly reverse the effects of Factor Xa inhibitors in patients who suffer a bleeding episode or who require emergency surgery. Portola has entered into clinical collaboration agreements with all of the manufacturers of direct Factor Xa inhibitors, including Bristol-Myers Squibb and Pfizer (Eliquis [apixaban]), Bayer HealthCare and Janssen Pharmaceuticals (XARELTO [rivaroxaban]), and Daiichi Sankyo (Savaysa [edoxaban]), while retaining all decision-making and commercial rights to the program.
Cerdulatinib (PRT2070) and PRT2607
Portola's third wholly owned product candidate, cerdulatinib (PRT2070), is an orally available kinase inhibitor that uniquely inhibits two validated tumor proliferation pathways - spleen tyrosine kinase (Syk) and janus kinase (JAK). It is being studied in patients with leukemias or lymphomas with a focus on genetically-defined subtypes, as well as in patients who have failed therapy due to relapse or acquired mutations. Portola's fourth program is partnered with Biogen Idec and is focused on the development of PRT2607, a selective Syk inhibitor.
Advisors' Opinion:- [By Todd Campbell]
That leads me to Medivation (NASDAQ: MDVN ) , Ophthotech (NASDAQ: OPHT ) , and Portola (NASDAQ: PTLA ) , three companies with important therapies that may very well be destined to become top sellers.
Best Biotech Stocks To Invest In Right Now: Savient Pharmaceuticals Inc(SVNT)
Savient Pharmaceuticals, Inc., a specialty biopharmaceutical company, focuses on developing KRYSTEXXA, a biologic PEGylated uricase in the United States. The KRYSTEXXA is being developed as a treatment for chronic gout in patients refractory to conventional therapy. The company also sells and distributes branded and generic versions of oxandrolone, a drug used to promote weight gain following involuntary weight loss. It sells its products directly to drug wholesalers. The company, formerly known as Bio-Technology General Corp. and changed its name to Savient Pharmaceuticals, Inc. in June 2003. Savient Pharmaceuticals, Inc. was founded in 1980 and is headquartered in East Brunswick, New Jersey.
Advisors' Opinion:- [By James E. Brumley]
Since 2008's implosion from the stock, the interest in Savient Pharmaceuticals Inc. (NASDAQ:SVNT) has been waning. There was a brief burst of bullishness in September of last year, which stirred the bullish pot a little. But, when SVNT started to fade in October of that year - just as quickly as it had perked up - what lingering hopes there were for the stock finally started to melt away. By the middle of this year, pretty much everyone had written Savient Pharmaceuticals off as a lost cause. Big mistake. Over the last few days, SVNT has almost wiggled its way buck into a bullish zone.
- [By James E. Brumley]
It's still too soon to say Savient Pharmaceuticals Inc. (NASDAQ:SVNT) is off and running. In fact, the stock's decidedly NOT off and running yet. But, it's not too soon to put SVNT on your watchlist of potential breakout candidates, as it's much closer to a breakout than most anyone can see.
Best Biotech Stocks To Invest 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 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.
Best Biotech Stocks To Invest In Right Now: Osiris Therapeutics Inc.(OSIR)
Osiris Therapeutics, Inc., a stem cell company, focuses on the development and marketing of therapeutic products to treat various medical conditions in the inflammatory, autoimmune, orthopedic, and cardiovascular areas. It operates in two business segments, Therapeutics and Biosurgery. The Therapeutics segment focuses on developing biologic stem cell drug candidates from a readily available and non-controversial source, adult bone marrow. The Biosurgery segment works to harness the ability of cells and novel constructs to promote the body's natural healing. This segment focuses on developing biologic products for use in surgical procedures. The company?s lead biologic drug candidate is Prochymal, which is in phase 2 and 3 clinical trails for various indications, including acute graft versus host disease (GvHD), Crohn's disease, acute myocardial infarction, type 1 diabetes, pulmonary disease, and gastrointestinal injury resulting from radiation exposure. Its biologic drug candidates also include Chondrogen, a preparation of adult mesenchymal stem cells that is in phase 2 clinical trials for osteoarthritis and cartilage protection. The company has collaboration agreements with Genzyme Corporation for the development and commercialization of Prochymal and Chondrogen in various countries except in the United States and Canada. It also has a partnership with Juvenile Diabetes Research Foundation for the development of Prochymal as a treatment for the preservation of insulin production in patients with newly diagnosed type 1 diabetes mellitus. Osiris Therapeutics, Inc. was founded in 1992 and is headquartered in Columbia, Maryland.
Advisors' Opinion:- [By Alexander Maxwell]
One of the companies attempting to develop a better treatment for chronic diabetic foot ulcers is Osiris Therapeutics� (NASDAQ: OSIR ) . Earlier this month, Osiris shares more than doubled as the company announced positive data for its CDFU drug Grafix. The study results were very impressive to say the least; the study was stopped early due to the overwhelming efficacy exhibited by the treatment. A main highlight is the fact that 62% of Grafix patients had their wound closed at 12 weeks, compared to only 21% of patients using conventional methods. Clearly, the efficacy in this endpoint was overwhelming. Grafix also achieved all of the secondary endpoints for the trial, and more importantly demonstrated a relatively benign safety record.�
- [By Maxx Chatsko]
Additionally, stem cell therapies have remained elusive as the industry's ultimate Holy Grail. Osiris (NASDAQ: OSIR ) received Canadian approval for the world's first stem cell drug, Prochymal, for children battling acute graft-versus-host disease, or GvHD, last year. The approval meant more symbolically than to the bottom line, but it definitely put the potential of stem cells front and center for investors.
- [By Lauren Pollock]
Osiris Therapeutics Inc.(OSIR) said Friday a proposed ruling from the Centers for Medicare and Medicaid Services won’t immediately affect reimbursements for its Grafix stem-cell product. The regenerative medicine company said Grafix will maintain its current reimbursement status — also called transitional pass-through status — potentially through late 2015.
Best Biotech Stocks To Invest In Right Now: Regulus Therapeutics Inc (RGLS)
Regulus Therapeutics Inc., incorporated on September 5, 2007, is a biopharmaceutical company focused on discovering and developing microRNAs to treat a range of diseases. microRNAs are naturally occurring ribonucleic acid (RNA), molecules that play a critical role in regulating key biological pathways. The Company uses its microRNA product platform to develop chemically modified, single-stranded oligonucleotides that the Company calls anti-miRs. As of December 31, 2012, the Company�� operations included acquiring and in-licensing intellectual property rights, developing its microRNA, undertaking basic research around microRNA targets and conducting preclinical studies for its initial programs.
The Company is developing RG-101 for the treatment of HCV and is advancing other microRNA therapeutics toward clinical development in several areas, including oncology, fibrosis and metabolic diseases. The Company intends to focus its resources on product opportunities in therapeutic areas where development and commercialization activities are appropriate for its size and financial resources, which the Company anticipates will include niche indications and orphan diseases.
The Company competes with Groove Biopharma, Inc., miRagen Therapeutics, Inc., Mirna Therapeutics, Inc., and Santaris Pharma A/S.
Advisors' Opinion:- [By Sean Williams]
A blast to the past
It's occasionally very difficult to value biotech stocks, because of the wide range of outcomes possible given their pipelines. Other times, as in the case with Regulus Pharmaceuticals (NASDAQ: RGLS ) , I bang my head against a table and wonder what the heck investors are thinking. - [By Brian Orelli]
Partners like this investment idea
When Regulus Therapeutics (NASDAQ: RGLS ) went public last year, investors weren't the only ones buying the share offering; Regulus' partners and founders clearly thought it was a good investment idea, because they bought more shares. Lots of them. AstraZeneca, Biogen Idec, Sanofi, GlaxoSmithKline�and Isis combined to purchase more than 70% of the shares that raised nearly $81 million for the biotech. - [By Sean Williams]
Regulus Therapeutics (NASDAQ: RGLS )
Since presenting at the Deutsche Bank Healthcare Conference two weeks ago, shares of Regulus Therapeutics have been absolutely on fire. A mixture of ASCO and IPO fever have revived the love for newer issues, and having big name pharmaceutical companies gobbling up a majority of its IPO'd shares certainly helps its cause.
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