The biotech industry is rife with small companies that spend millions developing new drugs that may or may not get the green light from the Food and Drug Administration. Many face costly setbacks, like clinical trial failures or regulatory delays, that can rapidly deplete their cash reserves. Even those that are ultimately successful typically don't have enough money to take a new drug all the way from the laboratory to the market.
So how can investors avoid getting burned by companies in this cash-hungry industry?
Follow the money
When analyzing a biotech stock, one of the first things investors need to calculate is how much money the company is spending. This number, called the "burn rate," is the total amount of cash that's spent on operations and capital expenditures. Let's run through an example and look at how much drug developer Keryx Biopharmaceuticals (NASDAQ: KERX ) has been spending.
Top Biotech Stocks To Buy Right Now: Transition Therapeutics Inc.(TTHI)
Transition Therapeutics Inc., a biopharmaceutical company, develops novel therapeutics for various disease indications primarily in Canada. Its lead products include ELND005 (AZD-103), a Phase II clinical trial product for the treatment of Alzheimer?s disease; TT-301 and TT-302, which are Phase I clinical trial products, for the treatment of rheumatoid arthritis, Alzheimer?s disease, traumatic brain injury, and intracerebral hemorrhage; and TT401/402, a preclinical stage product to treat diabetes. The company also has an emerging pipeline of preclinical and clinical drug candidates for the treatment of anti-inflammatory and metabolic indications. It has strategic collaborations with Elan Pharma International Limited to develop and commercialize ELND005 (AZD-103); and a licensing and collaboration agreement with Eli Lilly and Company to develop and commercialize gastrin based therapies, and the preclinical compound TT401/402. The company was formerly known as Transition T herapeutics and Diagnostics Inc. and changed its name to Transition Therapeutics Inc. on December 2000. Transition Therapeutics Inc. was founded in 1987 and is headquartered in Toronto, Canada.
Top Biotech Stocks To Buy Right Now: EntreMed Inc (ENMD)
EntreMed, Inc. (EntreMed), incorporated in 1991, is a clinical-stage pharmaceutical company. EntreMed's drug candidate is ENMD-2076, an Aurora A and angiogenic kinase inhibitor for the treatment of cancer. ENMD-2076 has completed Phase I studies in patients with advanced solid tumors, multiple myeloma and leukemia and is completing data for a multi-center Phase II study in patients with platinum resistant ovarian cancer. The Company�� other product candidates have includes MKC-1, ENMD-1198 and 2-methoxyestrdiol (2ME2, Panzem) for treatment of rheumatoid arthritis.
ENMD-2076 is a novel orally-active, Aurora A/angiogenic kinase inhibitor with potent activity against Aurora A and multiple tyrosine kinases linked to cancer and inflammatory diseases. ENMD-2076 is relatively selective for the Aurora A isoform in comparison to Aurora B. Aurora kinases are key regulators of the process of mitosis, or cell division, and are often over-expressed in human cancers. ENMD-2076 exerts its effects through multiple mechanisms of action, including anti-proliferative activity and the inhibition of angiogenesis. ENMD-2076 has demonstrated significant, dose-dependent preclinical activity as a single agent, including tumor regression, in multiple xenograft models (such as breast, colon, leukemia), as well as activity towards ex vivo-treated human leukemia patient cells.
Top 10 Performing Companies To Buy For 2014: Medivation Inc.(MDVN)
Medivation, Inc., a biopharmaceutical company, focuses on the development of small molecule drugs for the treatment of castration-resistant prostate cancer, Alzheimer?s disease, and Huntington disease. The company?s product candidates under clinical development include MDV3100, which is in Phase 3 development for the treatment of castration-resistant prostate cancer; and dimebon, which is in Phase 3 clinical trial for the treatment of Alzheimer?s disease and Huntington disease. It has collaboration agreements with Pfizer Inc. to develop and commercialize dimebon; and Astellas Pharma Inc. to develop and commercialize MDV3100. The company was founded in 2003 and is based in San Francisco, California.
Advisors' Opinion:- [By Ben Levisohn]
[The] sell-off in recent days was broad based and…affecting stocks in direct relationship to their volatility and expected duration of negative cash flow. Small and mid cap stocks were most affected (especially post-IPO stocks), and those with major uncertain events looming (MDVN) or with significant revenue upside already built into valuation (PCYC) were among the most severely affected. However, nothing changed in the environment to suggest that those events were any more or less likely to have positive outcomes yesterday, or to suggest that revenue potential was any more or less likely to be achieved than was previously expected.
- [By Sean Williams]
Friday brought equally good news for Medivation (NASDAQ: MDVN ) and its advanced prostate cancer drug Xtandi, which also received a positive opinion from the EMA's advisory panel. This really shouldn't come as a surprise as Medivation's oral prostate cancer treatment won FDA approval months early in the U.S., and the 4.8 months of extended median survival it delivered compared to the placebo is remarkable for such an advanced stage disease.
- [By Brian Orelli]
The largest impact to sales came from competition from new prostate cancer drugs Johnson & Johnson's (NYSE: JNJ ) Zytiga and Astellas Pharma and Medivation's (NASDAQ: MDVN ) Xtandi. In December, Zytiga was approved for use in patients who haven't received chemotherapy, the same patients that Provenge is approved to treat. Xtandi is still only approved as a second-line treatment after chemotherapy, but is listed as an earlier treatment in the National Comprehensive Cancer Network Compendium, the oncologist's bible for off-label treatment that insurers often use to make coverage decisions.
- [By Sean Williams]
What: Shares of biopharmaceutical company Medivation (NASDAQ: MDVN ) sank as much as 11% after rival Johnson & Johnson (NYSE: JNJ ) announced it was purchasing Aragon Pharmaceuticals to get ahold of its experimental advanced prostate cancer drug, ARN-509.
Top Biotech Stocks To Buy Right Now: 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 Brian Orelli]
Most recently Elan (NYSE: ELN ) spun out its drug discovery business into Prothena. Without the burden of a drug discovery unit, the parent company is now being pursued by Royalty Pharma, although Elan isn't keen on its current offer.
Top Biotech Stocks To Buy Right Now: StemCells Inc (STEM)
StemCells, Inc. (StemCells), incorporated in August 1988, is engaged in the research, development, and commercialization of stem cell therapeutics and related tools and technologies for academia and industry. The Company is focused on developing and commercializing stem and progenitor cells as the basis for therapeutics and therapies, and cells and related tools and technologies to enable stem cell-based research and drug discovery and development. The Company�� primary research and development efforts are focused on identifying and developing stem and progenitor cells as potential therapeutic agents. The Company has two therapeutic product development programs, including its CNS Program, which is developing applications for HuCNS-SC cells, its human neural stem cell product candidate, and its Liver Program, which is characterizing the Company�� human liver cells as a therapeutic product.
CNS Program
The Company in its CNS Program, is in clinical development with its HuCNS-SC cells for a range of disorders of the central nervous system. The CNS includes the brain, spinal cord and eye. In February 2012, the Company had completed a Phase I clinical trial in Pelizeaus-Merzbacher Disease (PMD), a fatal myelination disorder in the brain.
The Company�� CNS Program is focused on developing clinical applications, in which transplanting HuCNS-SC cells protect or restore organ function of the patient before such function is irreversibly damaged or lost due to disease progression. The Company�� initial target indications are PMD, and more generally, diseases in which deficient myelination plays a central role, such as cerebral palsy or multiple sclerosis; spinal cord injury, disorders in which retinal degeneration plays a central role, such as age-related macular degeneration or retinitis pigmentosa. The Company�� product candidate, HuCNS-SC cells, is a purified and expanded composition of normal human neural stem cells. Its HuCNS-SC cells can be directly transp! lanted.
Liver Program
Liver stem or progenitor cells offer an alternative treatment for liver diseases. A liver cellular therapy or cell-based therapeutic provide or support liver function in patients with liver disease. The Company held a portfolio of issued and allowed patents in the liver field, which cover the isolation and use of both hLEC cells and the isolated subset, as well as the composition of the cells themselves.
The Company�� range of cell culture products, which are sold under the SC Proven brand, includes iSTEM, GS1-R, GS2-M, RHB-A, RHB-Basal, NDiff N2, and NDiff N2B27. Its iSTEM is a serum-free, feeder-free medium that maintains mouse embryonic stem cells in their pluripotent ground state by using selective small molecule inhibitors to block the pathways, which induce differentiation. RHB-A is a defined, serum-free culture medium for the selective culture of human and mouse neural stem cells and their maintenance and expansion as adherent cell populations. RHB-Basal is a defined, serum-free basal medium. When supplemented with specific growth factors, this media is formulated for the propagation and differentiation of adherent neural stem cells. RHB-Basal can also be tailored to specific-cell type requirements by the addition of customer preferred supplements.
The Company�� NDiff N2 is a defined serum-free scell culture supplement for the derivation, maintenance, expansion and/or differentiation of human and mouse embryonic stem (ES) cells and tissue-derived neural stem cells supplement. Its NDiff N2-AF is a serum-free and animal component-free version of NDiff N2. Its NDiff N2B27 is a defined, serum-free medium for the differentiation of mouse embryonic stem cells to neural cell types. NDiff N27-AF is a serum-free and animal component-free version of NDiff N27. Its GS1-R is a serum-free media formulation shown to enable the derivation and long-term maintenance of true, germline competent rat embryonic stem cells without the add! ition of ! cytokines or growth factors. Its GS2-M is a defined, serum- and feeder-free medium for the derivation and long-term maintenance of true, germline competent mouse iPS cells.
The Company also markets a number of antibody reagents for use in cell detection, isolation and characterization. These reagents are also under the SC Proven brand and it includes STEM24, STEM101, STEM121 and STEM123. Its STEM24 is a human antibody that recognizes human CD24, also known as heat stable antigen (HSA), a glycoprotein expressed on the surface of many human cell types, including immature human hematopoietic cells, peripheral blood lymphocytes, erythrocytes and many human carcinomas. Its CD24 is also a marker of human neural differentiation. Its STEM101 is a human-specific mouse antibody that recognizes the Ku80 protein found in human nuclei. Its STEM121 is a human-specific mouse antibody that recognizes a cytoplasmic protein of human cells. Its STEM123 is a human-specific mouse antibody that recognizes human glial fibrillary acidic protein (GFAP).
The Company�� Other products marketed under SC Proven include total cell genomic DNA (gDNA), RNA and protein lysate reagents purified from homogenous stem cell populations for intra-comparative studies, such as Epigenetic fingerprinting, Southern, Western and Northern blots, PCR, RT-PCR and microarrays. This range of purified stem cell line lysates includes mouse embryonic stem (ES) cells propagated in SC Proven 2i inhibitor-based GS2-M media and mouse ES cell-derived and fetal tissue-derived neural stem (NS) cells propagated in SC Proven RHB-A media.
Advisors' Opinion:- [By James E. Brumley]
When an investor thinks of spinal-related stem cell stocks, usually a name like Neuralstem, Inc (NYSEMKT: CUR) or StemCells Inc (NASDAQ: STEM) comes to mind. And well they should. STEM has logged some amazing breakthroughs in the field of spinal cord repair, while CUR has done the same. Not all back problems are spinal cord related though. In fact, most back problems - and therefore the most opportunity - are bone and disc related problems. That's where a young gun like BioRestorative Therapies (OTCBB: BRTX) can step in and make stem cell waves. BRTX has developed an approach to rejuvenate and revive failing spinal discs, potentially ending pain for millions of back-pain sufferers, and circumventing expensive spinal surgeries that are in increasing burden on insurance companies.
- [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.
Top Biotech Stocks To Buy Right Now: Quintiles Transnational Holdings Inc (Q)
Quintiles Transnational Holdings Inc. is a provider of biopharmaceutical development services and commercial outsourcing services. The Company operates in two segments: Product Development and Integrated Healthcare Services. The Company�� Product Development segment operates as a contract research organization (CRO) focused primarily on Phase II-IV clinical trials and associated laboratory and analytical activities. The Company�� Integrated Healthcare Services segment is a global commercial pharmaceutical sales and service organizations and Integrated Healthcare Services provides a range of services, including commercial services, such as providing contract pharmaceutical sales forces in geographic markets, as well as healthcare business services for the healthcare sector, such as outcome-based and payer and provider services. In August 2012, it acquired Expression Analysis, Inc.
Product Development
Product Development provides services and that allow biopharmaceutical companies to outsource the clinical development process from first in man trials to post-launch monitoring. The Company�� service offering provides the support and functional necessary at each stage of development, as well as the systems and analytical capabilities. Product Development consists of clinical solutions and services and consulting. Clinical solutions and services provides services necessary to develop biopharmaceutical products, including project management and clinical monitoring functions for conducting multi-site trials (generally Phase II-IV) (core clinical) and clinical trial support services that improve clinical trial decision making and include global laboratories, data management, biostatistical, safety and pharmacovigilance, and early clinical development trials, and strategic planning and design services that improve decisions and performance. Consulting provides strategy and management consulting services based on life science and advanced analytics, as well as regulatory and comp! liance consulting services.
The Company competes with Covance, Inc., Pharmaceutical Product Development, Inc., PAREXEL International Corporation, ICON plc, inVentiv Health, Inc. (inVentive), INC Research and PRA International.
Integrated Healthcare Services
Integrated Healthcare Services provides the healthcare industry with both geographic presence and commercial capabilities. The Company�� commercialization services are designed to accelerate the commercial of biopharmaceutical and other health-related products. Service offerings include commercial services (sales representatives, strategy, marketing communications and other areas related to commercialization), outcome research (drug therapy analysis, real-world research and evidence-based medicine, including research studies to prove a drug�� value) and payer and provider services comparative and cost-effectiveness research capabilities, clinical management analytics, decision support services, medication adherence and health outcome optimization services, and Web-based systems for measuring quality improvement.
The Company competes with inVentiv, PDI, Inc., Publicis Selling Solutions, United Drug plc, EPS Corporation and CMIC HOLDINGS Co., Ltd.
Top Biotech Stocks To Buy Right Now: Johnson & Johnson(JNJ)
Johnson & Johnson engages in the research and development, manufacture, and sale of various products in the health care field worldwide. The company operates in three segments: Consumer, Pharmaceutical, and Medical Devices and Diagnostics. The Consumer segment provides products used in baby care, skin care, oral care, wound care, and women?s health care fields, as well as nutritional, over-the-counter pharmaceutical products, and wellness and prevention platforms under the brands of JOHNSON?S, AVEENO, CLEAN & CLEAR, JOHNSON?S Adult, NEUTROGENA, RoC, LUBRIDERM, DABAO, LISTERINE, REACH, BAND-AID, CAREFREE, STAYFREE, SPLENDA, TYLENOL, SUDAFED, ZYRTEC, MOTRIN IB, and PEPCID AC. The Pharmaceutical segment offers products in various therapeutic areas, such as anti-infective, antipsychotic, contraceptive, dermatology, gastrointestinal, hematology, immunology, neurology, oncology, pain management, and virology. Its principal products include REMICADE for the treatment of immune me diated inflammatory diseases; STELARA for the treatment of moderate to severe plaque psoriasis; SIMPONI, a treatment for adults with moderate to severe rheumatoid arthritis, psoriatic arthritis, and ankylosing spondylitis; VELCADE for the treatment of multiple myeloma; PREZISTA and INTELENCE for treating HIV/AIDS patients; NUCYNTA for moderate to severe acute pain; INVEGA SUSTENNAtm for the acute and maintenance treatment of schizophrenia in adults; RISPERDAL CONSTA for the management of bipolar I disorder and schizophrenia; and PROCRIT to stimulate red blood cell production. The Medical Devices and Diagnostics segment primarily offers circulatory disease management products; orthopaedic joint reconstruction, spinal care, and sports medicine products; surgical care, aesthetics, and women?s health products; blood glucose monitoring and insulin delivery products; professional diagnostic products; and disposable contact lenses. The company was founded in 1886 and is based in Ne w Brunswick, New Jersey.
Advisors' Opinion:- [By Maxx Chatsko]
Companies have had success in the past. Johnson & Johnson (NYSE: JNJ ) fielded a rare antibiotic blockbuster in Levaquin in the last decade, which recorded sales of $1.6 billion in 2007. The company still researches infectious medicine, but has enjoyed much more success in oncology and anti-inflammatory diseases. The field is heating up, though. Novo Nordisk (NYSE: NVO ) bought anti-infective therapy developer Xellia for $700 million this week. Talk about enthusiasm for market prospects. �
- [By Dan Caplinger]
Still, the big challenge for Abbott will come from the medical-device arena, where many of Abbott's peers are struggling. Johnson & Johnson (NYSE: JNJ ) has seen double-digit percentage sales declines in its cardiovascular equipment division, with growth coming solely from its major acquisition of Synthes. Meanwhile, Boston Scientific (NYSE: BSX ) has suffered even steeper declines in sales than Abbott, as its cardiac rhythm management business in particular has seen increased pricing pressure from competition. Abbott and Boston Scientific both compete in the drug-eluting stent niche, and even though Abbott has come out with recent advances, its stent sales were down 15% in the U.S. last quarter. Still, the company sees the area as having promise, as just this morning, Abbott announced it would acquire leg-stent maker Idev for $310 million.
- [By Brian Orelli]
The most advanced second-generation hepatitis C drugs, Johnson & Johnson's (NYSE: JNJ ) simeprevir and Gilead Sciences' (NASDAQ: GILD ) sofosbuvir, are already being reviewed for approval by the FDA. They both should be approved by the end of the year.
Top Biotech Stocks To Buy Right Now: Celgene Corp (CELG)
Celgene Corporation is a global biopharmaceutical company primarily engaged in the discovery, development and commercialization of therapies designed to treat cancer and immune-inflammatory related diseases. The Company is engaged in the research and development, which is designed to bring new therapies to market, and is engaged in research in several scientific areas that may deliver therapies, focusing areas, such as intracellular signaling pathways in cancer and immune cells, immunomodulation in cancer and autoimmune diseases, and therapeutic application of cell therapies. The Company�� primary commercial stage products include REVLIMID, VIDAZA, THALOMID, ABRAXANE and ISTODAX. Additional sources of revenue include a licensing agreement with Novartis, which entitles it to royalties on FOCALIN XR and the entire RITALIN family of drugs, the sale of services through its Cellular Therapeutics subsidiary and other miscellaneous licensing agreements. In March 2012, it acquired Avila Therapeutics.
The Company invests in research and development, and the drug candidates in its pipeline at various stages of preclinical and clinical development. These candidates include pomalidomide and apremilast, its oral anti-cancer and anti-inflammatory agents, PDA-001, its cellular therapy, oral azacitidine, CC-223 and CC-115 for hematological and solid tumor malignancies, CC-122, its anti-cancer pleiotropic pathway modifier, and ACE-011 and ACE-536 biological products for anemia in several clinical settings of unmet need. Celgene product candidates include Pomalidomide (CC-4047), Oral Anti-Inflammatory: Apremilast (CC-10004), CC-11050, Kinase Inhibitors:Tanzisertib (CC-930), Cellular Therapies: PDA-001, Activin Biology: Sotatercept (ACE-011) ACE-536, and Anti-tumor Agents: CC-22, CC-115, CC-122 and Oral Azacitidine. It owns and operates a manufacturing facility in Zofingen, Switzerland. The Company also owns and operates a drug product manufacturing facility in Boudry, Switzerland.
Commercial! Stage Products
REVLIMID (lenalidomide) is an oral immunomodulatory drug marketed in the United States and many international markets, in combination with dexamethasone, for treatment of patients with multiple myeloma who have received at least one prior therapy. It is also marketed in the United States and certain international markets for the treatment of transfusion-dependent anemia due to low- or intermediate-1-risk myelodysplastic syndromes (MDS) associated with a deletion 5q cytogenetic abnormality with or without additional cytogenetic abnormalities. REVLIMID is distributed in the United States through contracted pharmacies under the RevAssist program, which is a risk-management distribution program. Internationally, REVLIMID is distributed under mandatory risk-management distribution programs.
REVLIMID continues to be evaluated in numerous clinical trials worldwide either alone or in combination with one or more other therapies in the treatment of a range of hematological malignancies, including multiple myeloma (MDS) various lymphomas, chronic lymphocytic leukemia (CLL) other cancers and other diseases. VIDAZA (azacitidine for injection) is a pyrimidine nucleoside. VIDAZA is a Category 1 recommended treatment for patients with intermediate-2 and high-risk MDS and is marketed in the United States for the treatment of all subtypes of MDS. In Europe, VIDAZA is marketed for the treatment of intermediate-2 and high-risk MDS, as well as acute myeloid leukemia (AML) with 30% blasts and has been granted orphan drug designation for the treatment of MDS and AML.
THALOMID (thalidomide) is marketed for patients with newly diagnosed multiple myeloma and for the acute treatment of the cutaneous manifestations of moderate to severe erythema nodosum leprosum (ENL) an inflammatory complication of leprosy and as maintenance therapy for prevention and suppression of the cutaneous manifestation of ENL recurrence. THALOMID is distributed in the United States under its System f! or Thalid! omide Education and Prescribing Safety (S.T.E.P.S.) program. Internationally, THALOMID is also distributed under mandatory risk-management distribution programs. ABRAXANE (paclitaxel albumin-bound particles for injectable suspension) is a solvent-free chemotherapy treatment option for metastatic breast cancer, which was developed using its nab technology platform. This protein-bound chemotherapy agent combines paclitaxel with albumin. As of December 31, 2011, ABRAXANE was in various stages of investigation for the treatment of expanded applications for metastatic breast; non-small cell lung; malignant melanoma; pancreatic; bladder and ovarian.
ISTODAX (romidepsin) has received orphan drug designation for the treatment of non-Hodgkin's T-cell lymphomas, which includes CTCL and PTCL. The Company has licensed the worldwide rights (excluding Canada) regarding certain chirally pure forms of methylphenidate for FOCALIN and FOCALIN XR to Novartis. It also licensed to Novartis the rights related to long-acting formulations of methylphenidate and dex-methylphenidate products which are used in FOCALIN XR and RITALIN LA.
Preclinical and Clinical-Stage Pipeline
The product candidates in the Company�� pipeline are at various stages of preclinical and clinical development. Pomalidomide is a small molecule that is orally available and modulates the immune system and other biologically important targets. Pomalidomide is being evaluated in a phase III clinical trial for the treatment of myelofibrosis and a phase III clinical trial evaluating pomalidomide as a treatment for patients with relapsed/refractory multiple myeloma is accruing patients.
The Company is developing a product, ORAL ANTI-INFLAMMATORY AGENTS, which is orally available small molecules that target PDE4, an intracellular enzyme that modulates the production of multiple pro-inflammatory and anti-inflammatory mediators, including interleukin-2 (IL-2), IL-10, IL-12, IL-23, INF-gamma, TNF-a, leukotrienes,! and nitr! ic oxide synthase. Its investigational drug, apremilast (CC-10004), is used for the treatment of moderate to severe psoriasis and active psoriatic arthritis and is being evaluated in a phase II trial for rheumatoid arthritis and six phase III multi-center international clinical trials. In addition, it is investigating its oral PDE4 inhibitor, CC-11050, which is an anti-inflammatory compound that treat a variety of chronic inflammatory conditions, such as Cutaneous Lupus Erythematosus (CLE).
The Company�� oral kinase inhibitor platform includes inhibitors of the c-Jun N-terminal kinase (JNK) mTOR kinase, spleen tyrosine kinase (Syk) c-fms tyrosine kinase (c-FMS) and DNA-dependent protein kinase (DNAPK). Its oral Syk, c-FMS and DNAPK kinase inhibitors are being investigated in pre-clinical studies. The Company�� new second generation JNK inhibitor, tanzisertib (CC-930), is being evaluated in a phase II trial for the treatment of idiopathic pulmonary fibrosis and a phase II trial for the treatment of discoid lupus is accruing patients. Amrubicin is a third-generation fully synthetic anthracycline molecule with potent topoisomerase II inhibition.
At Celgene Cellular Therapeutics (CCT), it is researching stem cells derived from the human placenta, as well as from the umbilical cord. CCT is the Company�� research and development division. Stem cell based therapies provide disease-modifying outcomes for serious diseases, which lack adequate therapy. It has developed technology for collecting, processing and storing placental stem cells with broad therapeutic applications in cancer, auto-immune diseases, including Crohn's disease, multiple sclerosis, neurological disorders, including stroke and amyotrophic lateral sclerosis (ALS), graft-versus-host disease, and other immunological / anti-inflammatory, rheumatologic and bone disorders.
The Company has collaborated with Acceleron Pharma, Inc. (Acceleron) to develop sotatercept. Two phase I clinical studies have been co! mpleted. ! An additional phase II clinical study has been initiated and is ongoing related to treatments for end-stage renal anemia and to evaluate effects on red blood cell mass and plasma volume.
The Company competes with Abbott Laboratories, Amgen Inc. (Amgen), AstraZeneca PLC., Biogen Idec Inc., Bristol-Myers Squibb Co., Eisai Co., Ltd., F. Hoffmann-LaRoche Ltd., Johnson and Johnson, Merck and Co., Inc., Novartis AG, Pfizer, Sanofi and Takeda Pharmaceutical Co. Ltd. (Takeda).
Advisors' Opinion:- [By Sean Williams]
As I prefer to always start you off with the good news, let's turn our attention to Celgene (NASDAQ: CELG ) , which, on Friday, announced that the European Commission had approved its oral relapsed and refractory rare blood cancer drug pomalidomide in combination with dexamethasone. Celgene will be launching the drug in Europe under the trade name Imnovid, and it'll be used in cases where patients have tried at least two previous cancer therapies. In late-stage trials, Imnovid delivered progression-free survival of 15.7 weeks, which was a dramatic improvement over the placebo. Worldwide peak sales estimates for the drug are around $1 billion, so this is certainly a good start.
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