From the impact of Obamacare to cutting-edge research, biotech buyouts to Big Pharma court battles, The Motley Fool's health-care team sits down each week�to discuss the most fascinating developments in health care and their implications for long-term investors. In this week's edition, the team talks about the biggest headlines, stocks that have both popped and dropped, and companies our analysts will be watching in the coming days.
In the following segment, health-care analyst David Williamson discusses the exciting news for seasonal allergy sufferers. Watch and find out how this brand-new drug is poised to help Merck, and investors, reap pollen profits.
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Top Performing Stocks To Own Right Now: Rexahn Pharmaceuticals Inc (RNN)
Rexahn Pharmaceuticals, Inc. (Rexahn) is a development-stage biopharmaceutical company. The Company focuses on the development of cures for cancer to patients worldwide. The Company�� pipeline features one drug candidate in Phase II clinical trials. The Company also has several other drug candidates in pre-clinical development. In addition, the Company has two renal cell carcinoma (CNS) candidates, Serdaxin, CNS Disorders drug for depression and neurodegenerative diseases and Zoraxel, which is a erectile dysfunction (ED) and sexual dysfunction drug that are in clinical stages and the Company is are exploring options for further development . The Company�� drug candidate, Archexin is an anticancer Akt inhibitor.
Archexin
Archexin is potent inhibitor of the Akt protein kinase (Akt) in cancer cells. Archexin has FDA orphan drug designations for five cancers (RCC, glioblastoma, and cancers of the ovary, stomach and pancreas). Multiple indications for other solid tumors can also be pursued. Archexin inhibit both activated and inactivated forms of Akt, and to reverse the drug resistance observed with the protein kinase inhibitors. Archexin is an antisense oligonucleotide (ASO) compound that is complementary to Akt mRNA, and selective for inhibiting mRNA expression and production of Akt protein. As of December 31, 2011, Archexin was in Phase II clinical trials for the treatment of pancreatic cancer with enrollment completed in September, 2011.
Serdaxin
Serdaxin is an extended release formulation of clavulanic acid, which is an ingredient present in antibiotics approved by the FDA. The Company had been developing Serdaxin for the treatment of depression and neurodegenerative disorders. From January to September, 2011, the Company conducted a randomized, double-blind, placebo-controlled study compared two doses of Serdaxin, 0.5 milligram and 5 milligram, to placebo over an eight-week treatment period for major depressive disorder (MDD) patients. As of Dec! ember 31, 2011, the Company had not made a determination of Serdaxin�� future paths or resource allocations to further develop Serdaxin to treat MDD.
Zoraxel
Zoraxel is an orally administered, on-demand tablet to treat sexual dysfunction. Zoraxel is a dual enhancer of neurotransmitters in the brain that play a key role in sexual activity phases of motivation and arousal, erection and release, and may be the ED drug to affect all three of these phases of sexual activity. As of December 31, 2011, the Company was evaluating how to proceed with the Phase IIb study of Zoraxel.
The Company�� Pre-clinical Pipeline Drug Candidates includes RX-1792, which is a small molecule anticancer EGFR inhibitor; RX-5902, which is a small molecule anticancer ribonucleic acid (RNA) helicase regulator; RX-3117, which is a Small molecule anticancer deoxyribonucleic acid (DNA) synthesis Inhibitor; RX-8243, which is a small molecule anticancer aurora kinase inhibitor; RX-0201-Nano, which is a nanoliposomal anticancer Akt inhibitor; RX-0047-Nano, which is an nanoliposomal anticancer HIF-1 alpha inhibitor and RX-21101, which is a nano-polymer Anticancer.
Advisors' Opinion:- [By James E. Brumley]
If you were lucky enough to step into Rexahn Pharmaceuticals, Inc. (NYSEMKT:RNN) when your truly suggested it was a budding buy back on December 23rd, then congratulations - you're now up a little more than 60% (assuming you bought into RNN after the break above a key resistance line on the 27th). Now get out. Instead, use your profits from the Rexahn to take on a stake in GTx, Inc. (NASDAQ:GTXI). No, GTXI may not look like much at first, but when you take a step back and look at a chart of GTx, Inc. through a longer-term lens, the upside potential becomes clear.
- [By James E. Brumley]
With just a quick glance at a chart of Rexahn Pharmaceuticals, Inc. (NYSEMKT:RNN), it would be easy to conclude it's nothing but a volatile mess. When you take a step back and look at a long-term weekly chart of RNN, however, it starts to become clear that this small cap biopharma name is on the verge of a monster-sized breakout. First things first, however.
Best Biotech Companies To Watch For 2014: Elan Corporation PLC (ELN)
Elan Corporation, plc (Elan), incorporated in December 1969, is a neuroscience-based biotechnology company. The Company is focused on discovering and developing advanced therapies in neurodegenerative and autoimmune diseases. Elan�� business focuses on neurodegenerative diseases, such as Alzheimer�� disease and Parkinson�� disease; autoimmune diseases, including MS and Crohn�� disease and neo-epitope based targets for treatments across a range of therapeutic indications. Tysabri is a treatment for MS and Crohn�� disease that the Company markets and distributes with Biogen Idec. On September 16, 2011, Elan sold its EDT business to Alkermes, Inc. In November 2011, Elan launched a collaboration with the University of Cambridge, England, the Cambridge-Elan Centre for Research Innovation and Drug Discovery (Cambridge-Elan Centre). On December 21, 2012, the Company completed the demerger of Prothena Corporation plc. In April 2013, it closed the TYSABRI (natalizumab) Collaboration Transaction with Biogen Idec.
Tysabri
Tysabri, which is an alpha-4 integrin inhibitor, is a therapy for MS, a neurological disorder involving central nervous system dysfunction among adults. Tysabri is approved in more than 65 countries. Tysabri is approved in the United States as a monotherapy for relapsing forms of MS, for patients who have had an inadequate response to, or are unable to tolerate, an alternative MS therapy. In the European Union, it is approved for relapsing-remitting MS (RRMS) in adult patients who have failed to respond to beta interferon or have rapidly evolving, severe RRMS. As of December 31, 2011, there were approximately 64,400 patients on Tysabri therapy worldwide.
In June 2011, the European Commission (EC) approved the inclusion of the anti-JCV antibody status as an additional factor in stratifying patients at risk for developing PML in the Summary of Product Characteristics��(SmPC) for Tysabri in the European Union. The Company has developed a two-step ! enzyme-linked immunosorbent assay (ELISA), STRATIFY JCV, with Biogen Idec. The assay detects anti-JCV antibodies in the blood of patients, and is commercially available in Europe. In January 2012, the FDA cleared the assay for commercial use in the United States. As of December 31, 2011, over 80,000 tests had been administered using the assay. Tysabri is marketed and distributed by Elan and Biogen Idec. The Company�� research group, Neotope, is focused on creating monoclonal antibodies based on neo-epitope targets for the treatment of a range of therapeutic indications.
Beta Amyloid Immunotherapies (AIP)
Beta amyloid immunotherapy includes the treatment of Alzheimer�� disease by inducing or enhancing the body�� immune response in order to clear toxic species of beta amyloid from the brain. The AIP includes bapineuzumab (intravenous and subcutaneous delivery) and ACC-001, as well as other compounds. Bapineuzumab is an experimental humanized monoclonal antibody delivered intravenously that is being studied as a treatment for mild to moderate Alzheimer�� disease. It is designed to provide antibodies to beta amyloid directly to the patient (passive immunotherapy).
ELND005, an A� Aggregation Inhibitor
The small molecule ELND005 (Scyllo-inositol) is a beta amyloid anti-aggregation agent. Preclinical data suggest that ELND005 may act through the mechanism of preventing and reversing the fibrilisation of beta amyloid (the aggregation of beta amyloid into clumps of insoluble oligomers). ELND005 may have additional applications in psychiatric indications, such as bipolar disorder. In November 2011, the Company entered into a manufacturing agreement for the supply of the active pharmaceutical ingredient for ELND005 with Lonza Group AG.
Neotope Biosciences Limited
Neotope Biosciences Limited (Neotope) is the Company�� wholly owned subsidiary that focuses on the discovery and development of antibodies to neo-epitope related targ! ets for t! he treatment of a range of indications. It includes amyloidosis, diabetes, cancer and macular degeneration. Neotope�� portfolio of targets includes alpha-synuclein for the potential treatment of synucleinopathies, such as Lewy body dementia and Parkinson�� disease, tau for Alzheimer�� disease and other tauopathies. It also has a program for type 2-diabetes.
Onclave Therapeutics Limited
Elan�� wholly owned subsidiary Onclave Therapeutics Limited (Onclave) was formed to develop assets originating from Elan that have application in oncology related diseases. Onclave�� program, NEOD001, which originated from Neotope, is being investigated for the treatment of AL amyloidosis, which is a fatal disease involving abnormal accumulation of amyloid in organs and tissue. During the year ended December 31, 2011, Onclave filed for orphan drug designation of NEOD001. Onclave�� pipeline includes additional compounds with relevance in diverse cancer indications.
The Company competes with Biogen Idec, Bayer Schering Pharma AG, Bayer Schering Pharma, Merck Serono, Pfizer, Teva Neurosciences, Inc., Sanofi-Aventis and Novartis AG.
Advisors' Opinion:- [By John Maxfield]
Of the dozens of trades alleged in the various documents, the one that best demonstrates this is SAC's bet on the pharmaceutical giant Elan (NYSE: ELN ) in anticipation of the latter's Phase II trial results for a drug to treat Alzheimer's disease. I've mapped out the trade in the chart below.
- [By Tim Brugger]
After consulting with outside financial advisors, in conjunction with the Elan (NYSE: ELN ) board of directors and executive management team, the Ireland-based biotechnology company has determined the recent $12.50-a-share buyout offer from Royalty Pharma undervalues select Elan assets by as much as $4.3 billion, the company announced Wednesday.
Best Biotech Companies To Watch 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 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 Lauren Pollock]
Roche Holding AG(ROG.VX) has entered a pact with Prothena Corp.(PRTA) PLC to develop and commercialize a treatment the clinical-stage biotechnology firm is developing for Parkinson’s disease. In after-hours trading, Prothena’s stock rose 8.6% to $29.75 premarket.
Best Biotech Companies To Watch For 2014: International Stem Cell Corp (ISCO)
International Stem Cell Corporation, incorporated on June 7, 2005 is a developmental-stage biotechnology company. The Company focused on therapeutic and biomedical product development. The Company�� wholly owned subsidiary Lifeline Cell Technology, LLC (LCT) develops, manufactures and commercializes over 130 human cell culture products, including frozen human primary cells and the reagents (media) needed to grow, maintain and differentiate the cells, in order to address this market opportunity. The Company�� scientists have used a technology called basal medium optimization to systematically produce optimized products designed to culture specific human cell types and to elicit specific cellular behaviors. The Company�� wholly owned subsidiary Lifeline Skin Care, Inc. (LSC) develops, manufactures and markets cosmetic skin care products using an extract derived from its pluripotent stem cells.
Therapeutic Product Candidates
Using the Company's technologies and know-how, the Company is exploring and creating a range of cell types that is useful in therapeutic treatments, which include Liver cells (hepatocytes) that may be used to treat a range of congenital and acquired liver diseases. Neuronal cells for potential treatment of Parkinson's disease and other central nervous system disorders, such as traumatic brain injury, stroke and Alzheimer�� disease. Three-dimensional eye structures to treat degenerative retinal diseases, corneal blindness, and to accelerate corneal healing.
Skin Care Products
The Company�� research scientists developed two skin care products, Defensive Day Serum and Recovery Night Serum, using an extract derived from human parthenogenic stem cells. Defensive Serum contains sunscreen, along with stem cell-derived ingredients. The day serum not only protects the skin from the aging effects of harsh light, but it continues to nurture the skin�� collagen and fibroblasts to give noticeably firmer, smoother, younger-looking ! skin. The Recovery Night Serum is a nighttime therapy that complements the Defensive Day Serum. The night serum nurtures the skin�� collagen and elastin and contains ingredients to defend against damaging free radicals, to help build firmer, smoother, younger and healthier-looking skin.
Research Products
The Company�� Lifeline subsidiary produces and sells over 250 human cell culture products. These products include frozen human cells and stem cells and the reagents needed to grow, maintain and differentiate the cells. These human cell-based products are used domestically and internationally by research scientists in pharmaceutical, academic and government research organizations to study human disease and basic cell biology.
The Company competes with Genzyme Corporation, StemCell, Advanced Cell Technology Inc., Aastrom Biosciences, ViaCyte, Obagi, SkinCeuticals, SkinMedica, Murad, Lonza, Chemicon, Life Technologies, StemCell Technologies, Merck, BioTime and Specialty Media.
Advisors' Opinion:- [By John Udovich]
The results of a recent Pew Center Poll regarding attitudes towards abortion and various forms of stem cell research could be a good sign for the stem cell industry along with small cap stem cell stocks like StemCells Inc (NASDAQ: STEM), NeoStem Inc (NASDAQ: NBS), Neuralstem, Inc (NYSEMKT: CUR),�International Stem Cell Corp (OTCMKTS: ISCO) and BioRestorative Therapies (OTCBB: BRTX). Basically, Americans think that having an abortion is a moral issue with 49% of American adults believing abortion is morally wrong, 23%�view it not as a moral issue and and 15% view it as morally acceptable. However and when Americans were asked about issues surrounding�human embryos, such as stem cell research or in vitro fertilization, as a matter of morality, their views were different.
- [By John Udovich]
Stem cells may not be in the news much�as the sector has moved beyond the use of embryotic�ones, but small cap stem cell stocks Cellular Dynamics International Inc (NASDAQ: ICEL), International Stem Cell Corp (OTCMKTS: ISCO) and BioRestorative Therapies (OTCBB: BRTX) have been fairly active over the past several trading days as ICEL went public, ISCO raised additional funding and BRTX grabbed more attention:
Best Biotech Companies To Watch For 2014: Redhill Biopharma Ltd (RDHL)
Redhill Biopharma Ltd. is an Israel-based biopharmaceutical company. The Company is focused on the development and acquisition of therapeutic candidates. The Company�� pipeline consists of six late clinical development therapeutic candidates, two of which have completed bioequivalence clinical trials subject to review and approval by the United States Food and Drug Administration and, in some cases, regulatory authorities in other countries. The Company�� six clinical stage therapeutic candidates include RHB-101, RHB-102, RHB-103, RHB-104, RHB-105 and RHB-106.
RHB-101
RHB-101 is a treatment of hypertension, heart failure and left ventricular dysfunction (following myocardial infraction) by means of controlled release of an active ingredient known as carvedilol, which is designed to be administered to patients on a once-daily basis. RHB-101 is based on a patented technology for the controlled release of drugs administered orally.
RHB-102
RHB-102 is a once-daily controlled release oral formulation of ondansetron. RHB-102 utilizes a technology called CDT that uses salts to provide a controlled release of ondansetron.
RHB-103
RHB-103 is an oral thin film formulation of rizatriptan intended for the treatment of acute migraine headaches. Migraine is a neurovascular disorder (related to nerves and blood vessels) characterized by recurrent headaches in one side or both sides of the head.
The product is based on a technology called VersaFilm.
RHB-104
RHB-104 is an antibiotic combination therapy for the treatment of Crohn's disease (with a PIII clinical study underway), as well as Multiple Sclerosis (with an ongoing PIIa clinical study) and Rheumatoid Arthritis. RHB-104 is a combination of clarithromycin, clofazimine and rifabutin, three generic antibiotic ingredients, in a single capsule.
RHB-105
RHB-105, an antibiotics and proton pump inhibitor drug targeting Helico! bacter Pylori infection. RHB-105 is a combination of three approved drug products omeprazole, which is a proton pump inhibitor (the natural body pump that produces the gastric acids used for digesting the food in the stomach), and amoxicillin and rifabutin which are antibiotics. Chronic infection with Helicobacter pylori irritates the mucosal lining of the stomach and small intestine.
RHB-106
RHB-106, is a tablet for the preparation and cleansing of the gastrointestinal tract prior to the performance of abdominal procedures. Its abdominal procedures include diagnostic tests, such as colonoscopy, barium enema or virtual colonoscopy, as well as surgical interventions, such as laparotomy.
The company competes with GlaxoSmithKline, Sanofi-Aventis Groupe, Hoffman-La Roche Ltd, Merck and Co., Inc, Ferring Pharmaceuticals and Salix Pharmaceuticals Inc.
Advisors' Opinion:- [By Monica Gerson]
Breaking news
Vitran Corporation (NASDAQ: VTNC) announced today that it has entered into a definitive arrangement agreement with TransForce pursuant to which TransForce has agreed to acquire all of the outstanding common shares of Vitran not already owned by TransForce for US$6.50 in cash per share, in accordance with TransForce's prior proposal. To read the full news, click here. ReneSola (NYSE: SOL) today announced it signed a Memorandum of Intent (MOI) to sell three utility-scale projects in Western China, with a total capacity of 60MW, to Jiangsu Akcome Solar Science & Technology Co on December 30, 2013. To read the full news, click here. Cooper Tire & Rubber Company (NYSE: CTB) today announced it has terminated the merger agreement with Apollo Tyres (NSE:ApolloTYRE). To read the full news, click here. RedHill Biopharma (NASDAQ: RDHL) today announced that it has entered into a definitive agreement with leading healthcare investor OrbiMed Israel Partners Limited Partnership, an affiliate of OrbiMed Advisors LLC, for the sale of RedHill's American Depository Shares and warrants in a private placement transactionor a total sum of $6.0 million. To read the full news, click here.Posted-In: Guggenheim US Stock FuturesNews Eurozone Futures Global Pre-Market Outlook Markets
Best Biotech Companies To Watch For 2014: Amgen Inc.(AMGN)
Amgen Inc., a biotechnology medicines company, discovers, develops, manufactures, and markets human therapeutics based on advances in cellular and molecular biology for grievous illnesses primarily in the United States, Europe, and Canada. The company markets recombinant protein therapeutics in supportive cancer care, nephrology, and inflammation. Its principal products include Aranesp and EPOGEN erythropoietic-stimulating agents that stimulate the production of red blood cells; Neulasta and NEUPOGEN to stimulate the production of neutrophils, which is a type of white blood cell that helps the body to fight infections; and Enbrel, an inhibitor of tumor necrosis factor that plays a role in the body?s response to inflammatory diseases. The company also markets other products comprising Sensipar/Mimpara, a small molecule calcimimetic that lowers serum calcium levels; Vectibix, a monoclonal antibody that binds specifically to the epidermal growth factor receptor; and Nplate, a thrombopoietin (TPO) receptor agonist that mimics endogenous TPO, the primary driver of platelet production. In addition, it provides Denosumab, a human monoclonal antibody that targets RANKL, an essential regulator of osteoclasts. Further, the company offers product candidates in mid-to-late stage development in a variety of therapeutic areas, including oncology, hematology, inflammation, bone, nephrology, cardiovascular, and general medicine consisting of neurology. It markets its products to healthcare providers, including physicians or their clinics, dialysis centers, hospitals, and pharmacies; consumers; and wholesale distributors of pharmaceutical products. The company has various collaborative arrangements with Pfizer Inc.; GlaxoSmithKline plc; Takeda Pharmaceutical Company Limited; Daiichi Sankyo Company, Limited; Array BioPharma Inc.; Kyowa Hakko Kirin Co. Ltd.; and Cytokinetics, Inc. Amgen Inc. was founded in 1980 and is headquartered in Thousand Oaks, California.
Advisors' Opinion:- [By John Kell var popups = dojo.query(".socialByline .popC"); popups.forEach(func]
Amgen Inc.(AMGN) said its first-quarter earnings fell 25% on higher costs that masked the biopharmaceutical company’s revenue growth. Shares dropped 2.6%to $116.15 premarket.
- [By Jon C. Ogg]
Any time you see a merger announcement, you see a myriad of press releases pertaining to law firms and lawyers investigating the terms of the deal. This is in one sense the new form of class action lawsuit of yesteryear, but sometimes these suits and investigations do have merit. Now that Amgen Inc. (NASDAQ: AMGN) has confirmed its $10.4 billion of Onyx Pharmaceuticals Inc. (NASDAQ: ONXX), the lawyers have been coming out of the woodwork.
- [By James Brumley]
If there’s one thing investors have relearned about pharmaceutical stocks in the wake of third quarter’s earnings results, it’s that no two of these are cut from the same cloth. Oh, Amgen (AMGN), Bristol-Myers Squibb (BMY), Eli Lilly (LLY), and all the other pharmaceutical stocks are in the same business — they all create and market drugs. But, these companies are as different from one another as their drug portfolios are. They’re best handicapped on a case-by-case basis, and Q3′s earnings reports underscore much of what traders need to know.
Best Biotech Companies To Watch For 2014: Pharmacyclics Inc (PCYC)
Pharmacyclics, Inc., incorporated on April 19, 1991, is a clinical-stage biopharmaceutical company focused on developing and commercializing small-molecule drugs for the treatment of cancer and immune mediated diseases. The Company's clinical development and product candidates are small-molecule enzyme inhibitors designed to target biochemical pathways involved in human diseases. As of June 30, 2011, it had three drug candidates under clinical development and a number of preclinical lead molecules. This includes an inhibitor of Bruton�� tyrosine kinase (Btk) (PCI-32765) in Phase II studies in hematologic malignancies; a Btk inhibitor lead optimization program targeting autoimmune indications, an inhibitor of Factor VIIa (PCI-27483) in a Phase II clinical trial in pancreatic cancer, and a histone deacetylase (HDAC) inhibitor (PCI-24781) in Phase I and II clinical trials in solid tumors and hematological malignancies as of June 30, 2012.
As of June 30, 2012, the Company developed ibrutinib, which has demonstrated clinical activity and tolerability in Phase I and Phase II clinical trials in a variety of B-cell malignancies, including chronic lymphocytic leukemia (CLL) and a number of non-Hodgkin�� lymphoma (NHL) subtypes. CLL, mantle cell lymphoma (MCL), follicular lymphoma (FL), diffuse B-cell lymphoma (DLBCL) and multiple myeloma (MM) are specific indications of its current or planned Phase Ib/II and Phase III development program. had development programs for B-cell malignancies and autoimmune diseases. For malignant indications it has developed PCI-32765, which has demonstrated clinical activity and tolerability in Phase I and Phase II clinical trials in a range of B-cell malignancies, including chronic lymphocytic leukemia (CLL) and a number of non-Hodgkin�� lymphoma (NHL) subtypes. CLL, mantle cell lymphoma (MCL), follicular lymphoma (FL), diffuse large B cell lymphoma (DLBCL) and multiple myeloma (MM) are specific indications of its Phase II development. It has developed an assay! to measure occupancy of Btk in PBMCs using a cell-permeable fluorescently-labeled derivative of PCI-32765.
Factor VII is an enzyme that becomes activated (FVIIa) by binding to the cell surface protein tissue factor (TF), a protein found in the body that helps to trigger the process of blood clotting in response to injury. TF is over expressed in many cancers including gastric, breast, colon, lung, prostate, ovarian and pancreatic cancers. In these tumors, the FVIIa/TF complex induces intracellular signaling pathways by activating protease activated receptor 2 (PAR-2), another cell-surface protein. This in turn increases the expression of interleukin-8 (IL-8), a protein produced by white blood cells and other immune cells in response to pathogenic stimulation, and vascular endothelial growth factor (VEGF), a signal protein produced by cells that stimulate the growth of blood vessels. Both proteins play an important role in tumor growth and metastases as well as angiogenesis (growth of new blood vessels). FVIIa/TF complex also initiates the coagulation (a process by which blood forms clots) processes implicated in the high incidence of thromboembolic (the process by which the blood clots within a blood vessel) complications seen in patients with TF-expressing cancers. Thromboembolic events are a cause of death in patients with cancer and anticoagulant treatment has been shown to improve survival in a variety of cancers (Klerk et al. JCO. 2005).
PCI-27483 Factor VIIa Inhibitor
The Company�� Factor VIIa inhibitor PCI-27483 is a first-in-human small molecule inhibitor that selectively targets FVIIa. As an inhibitor of FVIIa, PCI-27483 has two potential mechanisms of action: inhibition of intracellular signaling involved in tumor growth and metastases and inhibition of early coagulation processes associated with thromboembolism.
Factor VIIa PCI-27483 Clinical Development Update
A multicenter Phase I/II of PCI-27483 in patients with locally a! dvanced o! r metastatic pancreatic cancer that are either receiving or are planned to receive gemcitabine therapy has completed enrollment. The Phase II portion of the study randomized patients to receive either gemcitabine alone or gemcitabine plus PCI-27483 (1.2 mg/kg twice daily). The objectives are to assess the safety of FVIIa Inhibitor PCI-27483 at pharmacologically active dose levels, to assess potential inhibition of tumor progression and to obtain initial information of the effects on the incidence of thromboembolic events. Due to a paradigm shift away from the use of gemcitabine alone for the treatment of pancreatic cancer, enrolling patients in this randomized study has been challenging. PCYC is evaluating other alternatives for development of this agent.
A multicenter Phase I/II of PCI-27483 in patients with locally advanced or metastatic pancreatic cancer that are either receiving or are planned to receive gemcitabine therapy has completed enrollment. The Phase II portion of the study randomized patients to receive either gemcitabine alone or gemcitabine plus PCI-27483 (1.2 mg/kg twice daily). PCI-27483 is covered by United States patents and patent applications and counterpart patents and patent applications in fourteen ex-United States territories, including Europe, Canada, Mexico, Japan, China, India, South Korea, Australia and Brazil.
Advisors' Opinion:- [By Jay Silverman]
Over the past year, including the end of this year, we've had new blockbuster drugs for hepatitis C, which is Gilead's (GILD) drug, and also for B-cell lymphomas, which is Pharmacyclics' (PCYC) drug. Those are looking to be two of the biggest drugs ever.
- [By Ben Levisohn]
Somaiya and team named Gilead and�Neurocrine Biosciences (NBIX) their top picks, hile putting Buy ratings on Celgene, Biogen Idec, Alexion (ALXN), Incyte (INCY), Pharmacyclics (PCYC) and Synageva (GEVA). BioMarin (BMRN), Infinity Pharmaceuticals (INFI) and Amgen (AMGN) earned Neutral ratings.
- [By Keith Speights]
Pharmacyclics (NASDAQ: PCYC ) and development partner Janssen, a division of Johnson & Johnson (NYSE: JNJ ) , announced on Wednesday that a New Drug Application, or NDA, has been submitted for ibrutinib. The NDA filed with the U.S. Food and Drug Administration requests marketing approval for the drug in the treatment of chronic lymphocytic leukemia/small lymphocytic lymphoma and in the treatment of previously treated patients with mantle cell lymphoma.
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