Top 10 Medical Companies To Watch For 2014

The cost of medical care continues to rise and to be the wild card in retirement plans. Reports and studies update the estimates of the cost of retirement medical care each year. They show the cost to be high and also very unpredictable for individual retirees and couples. The studies focus on average or median costs. In your planning you have to be aware that individual costs vary greatly because of differences in personal health, geography, and insurance coverage. Your retirement medical costs can be substantially higher or lower than the overall forecasts.

We��e talking about out-of-pocket costs, those expenses that aren�� covered by Medicare. People on average incur higher medical costs than these estimates, but Medicare picks up some of the costs. These estimates are of what you��l have to pay.

A couple retiring in 2013 and incurring median drug expenses during retirement would need to save $151,000 to have a 50% chance of covering their lifetime costs for prescription drugs only, according to the latest study from the Employee Benefit Research Institute. Those who incur among the highest medicine expenses are likely to need over $220,000. The good news in the report is that the prescription drug expense estimates are lower than last year�� because of a reduction in the rate of growth of medical and drug costs.

Top Healthcare Technology Companies To Buy Right Now: Tandem Diabetes Care Inc (TNDM)

Tandem Diabetes Care, Inc., incorporated on July 1, 2008, is a medical device company with an approach to the design, development and commercialization of products for people with insulin-dependent diabetes. The Company designed and commercialized its flagship product, the t:slim Insulin Delivery System, or t:slim, based on its technology platform and consumer-focused approach. Its technology platform features Micro-Delivery Technology, a miniaturized pumping mechanism, which draws insulin from a flexible bag within the pump�� cartridge rather than relying on a syringe and plunger mechanism. The Company also applies the science of human factors to its design and development process, which seeks to optimize its devices to the intended users, allowing users to successfully operate the devices in their intended environment.

The Company developed t:slim to offer the specific features that people with insulin-dependent diabetes seek in a next-generation insulin pump. The Company designed it to have the look and feel of a modern consumer electronic device, such as a smartphone. It is the insulin pump to feature a high resolution, color touchscreen. It is also the slimmest and smallest durable insulin pump on the market, and can easily and discreetly fit into a pocket, while still carrying a cartridge with 300 units of insulin.

The Company designed its flagship product, t:slim, to have the look and feel of a modern consumer electronic device, such as a smartphone. t:slim is the slimmest and smallest durable insulin pump on the market. With its narrow profile, which is similar to many smartphones, t:slim can easily and discreetly fit into a pocket. Its technology platform allows for the use of a vivid touchscreen and easy-to-navigate software architecture, which provide users simple access to the key functions of t:slim directly from the Home Screen. Insulin pump users can quickly learn how to efficiently navigate t:slim�� software, thereby enabling healthcare providers to spend! less time teaching a person how to use the pump and more time improving management of their diabetes. Its software also features numerous shortcuts, including a simple way to return to the Home Screen and view critical information for therapy management. Its technology is specifically designed to help prevent the unintentional delivery of insulin and reduce fear associated with using a pump.

Advisors' Opinion:

• [By Paul Ausick]

  Insulin pump maker Tandem Diabetes Care Inc. (NASDAQ: TNDM) sold 8 million shares on Thursday at an IPO price of $15. The stock began trading at around $19 before closing about flat to the opening bid and then shooting up more than 13% on Friday to close at $21.84.

Top 10 Medical Companies To Watch 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 Smith On Stocks]

  Using my assumptions, the common of Agenus might be worth about $7.00 per share in early 2014 based on the royalties from the Glaxo vaccines and the net operating loss carry forward even if every other asset in the company were valued at zero. However, there may be substantial value in Agenus' internal vaccine programs in recurrent and newly diagnosed glioblastoma and the genital herpes vaccine. Based on a comparison to peer companies like Northwest Biotherapeutics (NWBO) and ImmunoCellular Therapeutics (IMUC), I believe that the internal programs at Agenus currently may be worth $150 million or $4.45 per share. Adding this to the value of the MAGE A-3 vaccine could produce a stock price of $11.00 in early 2014.

• [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

• [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 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.

Top 10 Medical Companies To Watch For 2014: Integra LifeSciences Holdings Corp (IART)

Integra LifeSciences Holdings Corporation (Integra), incorporated on June 19, 1989, is an integrated medical technology company. Integra offers solutions in orthopedic extremity surgery, neurosurgery, spine surgery, and reconstructive and general surgery. The Company has developed numerous product lines for applications ranging from burn and deep tissue wounds to regeneration of dura mater in the brain and repair of nerve and tendon. It operates in five business segments: U.S. Neurosurgery, U.S. Extremities, U.S. Instruments, U.S. Spine and Other, and International. The International segment sells similar products to Europe, Middle East and Africa, and Central/South America, Asia-Pacific and Canada. On April 29, 2013, the Company launched the Integra Compact Cranial Closure System, which provides titanium implants for non-loadbearing (non-facial) operative cranial neurosurgical procedures. On April 30, 2013, it released its Hollywood VI intervertebral body fusion device (IBD) system. In January 2014, Integra LifeSciences Holdings Corp completed the acquisition of DuraSeal product lines from Covidien. In January 2014, Covidien PLC completed the sale of its Confluent Surgical product line to Integra LifeSciences Corporation.

U.S. Neurosurgery

The Company�� U.S. Neurosurgery sales organization sells a full line of products for neurosurgery and neuro critical care. It has products for each step of a cranial procedure and the care of the patient after surgery. Its key products include dural repair products, cerebral spinal fluid (CSF) management devices, tissue ablation equipment, intracranial monitoring equipment and cranial stabilization equipment. The Company sells equipment used in the neurosurgery operating room and neurosurgery intensive care unit (NICU).

U.S. Extremities

Extremity reconstruction includes the repair of soft tissue and the orthopedic reconstruction of bone in the foot, ankle and leg below the knee (Lower Extremity), and the hand, ! wrist, elbow and shoulder (Upper Extremity). Its key products include bone and joint fixation devices, implants and instruments for osteoarthritis, rheumatoid arthritis, wrist arthroplasty, carpel tunnel syndrome, and cubital tunnel syndrome. Other key products include its regenerative medicine devices for the treatment of acute and chronic wounds, peripheral nerve repair and protection and tendon repair, and bone graft substitutes.

U.S. Instruments

Integra�� U.S. Instruments business is a surgical instrument suppliers in the United States. Its portfolio includes over 60,000 instrument patterns and surgical products sold a range of users, including hospitals, surgery centers, and physician, dental and veterinary offices. In addition to selling hand-held instruments, it sells surgical headlight systems and table-mounted retractors. The Company�� brands include Jarit, Miltex, Padgett, Ruggles, Luxtec and Omni-Tract.

U.S. Spine and Other

Integra�� U.S. Spine and Other segment offer spinal fusion technologies that surgeons use along the full length of the spine, as well as a range of related orthobiologics. During the year ended December 31, 2012, its Spine business launched multiple new implants into the integrated interbody fusion device market and the deformity market. Its key spinal hardware products include integrated interbody fusion devices, minimally invasive solutions, and deformity correction. It markets and sells a complete line of orthobiologics, including demineralized bone products, collagen ceramic matrices and pure synthetic bone grafting solutions, to neurosurgeons, and spine, orthopedic, trauma, and foot and ankle surgeons. Integra sells its products through specialty distributors focused on its spine and orthopedic surgeon customers, as well as through some direct sales representatives. This segment also includes private-label sales of a set of its regenerative medicine technologies. Its customers are other large medical technology! companie! s that sell to end markets primarily in orthopedics and wound care.

The Company competes with Johnson & Johnson, Synthes, Inc., Stryker Corporation, Tornier, Inc., Wright Medical Group, Inc., Zimmer, Inc., Small Bone Innovations, Inc., Medtronic, Inc., Globus Medical Inc., NuVasive, Inc., Orthofix, Alphatec Spine, Inc., B. Braun Medical Inc., CareFusion and Symmetry Medical.

Advisors' Opinion:

• [By John Udovich]

  On Thursday, small cap medical device stock Integra Lifesciences Holdings Corp (NASDAQ: IART) jumped 9.90% after the FDA completed its inspection of the company's manufacturing facility which led to positive comments from analysts, meaning it might be time to take a look at its performance verses that of medical device ETFs like iShares Dow Jones US Medical Device ETF (NYSEARCA: IHI) and SPDR S&P Health Care Equipment ETF (NYSEARCA: XHE).

• [By Sean Williams]

  What: Shares of Integra LifeSciences (NASDAQ: IART  ) , a manufacturer of surgical instruments and medical implants, fell as much as 13% after the company announced a voluntary recall of certain products at its Anyasco manufacturing facility in Puerto Rico.

Top 10 Medical Companies To Watch For 2014: Receptos Inc (RCPT)

Receptos, Inc. (Receptos), incorporated on June 9, 2008, is a biopharmaceutical company. The Company is focused on discovering, developing and commercializing therapeutics for immune disorders. The Company�� product candidates span three specialty disease areas. The Company�� lead asset, RPC1063, is being developed as an oral therapy for the treatment of relapsing multiple sclerosis (RMS) and inflammatory bowel disease (IBD). Its second asset, RPC4046, is being developed for the treatment of an allergic/immune-mediated disorder, eosinophilic esophagitis (EoE), which is an orphan disease. RPC1063 is an oral, once daily, selective and potent sphingosine 1-phosphate 1 receptor (S1P1R) modulator. RPC4046 is a monoclonal antibody selective to interleukin-13 (IL-13) and produced by recombinant deoxyribonucleic acid (DNA) technology.

As of December 31, 2012, RPC1063 was being tested in the Phase II portion of an accelerated design, randomized Phase II/III study for the treatment of RMS. In addition, Receptos has obtained special protocol assessment (SPA) agreement from the United States food and drug administration (FDA) on its clinical trial design for the planned Phase III portion of the Phase II/III study, as well as a second planned RMS Phase III study. RPC1063 is also being tested in a randomized Phase II study for the treatment of ulcerative colitis (UC), a gastrointestinal (GI) disea se.

As of December 31, 2012, the Company was enrolling a randomized Phase II study evaluating the ability of RPC1063 to induce clinical remission in patients with moderately-to-severely active UC called TOUCHSTONE. The Company�� second asset, RPC4046 for the treatment of EoE, builds upon its competencies in immunology and GI diseases. In-licensed from AbbVie Bahamas Ltd. and AbbVie Inc. (AbbVie) RPC4046 is a monoclonal antibody directed against the IL-13 target, which has been validated in Asthma, a predominantly allergic/immune-mediated disorder.

Advisors' Opinion:

• [By John Udovich]

  Small cap �biopharmaceutical stock Receptos Inc (NASDAQ: RCPT) was one of the many hot biotech IPOs of last year and its also up 72.4% since the start of this year alone, meaning its time to take a closer look at this small cap stock with potential treatments for Relapsing Multiple Sclerosis (RMS) and Inflammatory Bowel Disease (IBD) along with the performance of biotech ETF benchmarks like the iShares NASDAQ Biotechnology Index ETF (NASDAQ: IBB) and SPDR S&P Biotech ETF (NYSEARCA: XBI).

Top 10 Medical Companies To Watch For 2014: 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]

  At first glance, Rexahn Pharmaceuticals, Inc. (NYSEMKT:RNN) doesn't look like anything particularly trade-worthy. The stock's now where it was in late October, and where it was in late September, and even where it was in late June. In other words, though RNN has been much higher and much lower than its current price of $0.46 over the course of the past several months, it doesn't look like it's going anywhere - on a net basis - anytime.

• [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.

• [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.

Top 10 Medical Companies To Watch For 2014: Protalix Biotherapeutics Inc (PLX)

Protalix BioTherapeutics, Inc. is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins based on its ProCellEx protein expression system, ProCellEx. Using its ProCellEx system, the Company is developing a pipeline of biosimilar or generic versions of recombinant therapeutic proteins based on its plant cell-based expression technology, which focuses pharmaceutical markets and that rely upon known biological mechanisms of action. ProCellEx protein expression system consists of a set of technologies and capabilities for the development of recombinant proteins, including advanced genetic engineering technology and plant cell-based protein expression methods. Its ProCellEx protein expression system is built on flexible custom-designed bioreactors made of polyethylene and optimized for the development of complex proteins in plant cell cultures. In June 2010, it had completed the preliminary phase I clinical trial of PRX-105.

Taliglucerase Alfa

Taliglucerase alfa is a plant cell expressed recombinant glucocerebrosidase enzyme (GCD) for the treatment of Gaucher disease. The Company has commenced pre-clinical studies of an oral form of taliglucerase alfa. Its oral taliglucerase alfa is a plant cell expressed form of GCD that is naturally encapsulated within carrot cells genetically engineered to express the GCD enzyme. Pre-clinical studies of oral taliglucerase alfa demonstrate the stability of the enzyme in the cell and the capacity of the cell�� cellulose wall to protect the enzyme against degradation in the digestive tract in an in-vitro model of the stomach and intestines. Additionally, rats fed with lyophilized carrot cells expressing GCD have accumulated the active enzyme in the target organs; the spleen and liver. As of December 31, 2010, the Company had completed Phase III Clinical Trial.

PRX-102

The Company is developing PRX-102, its plant cell expressed modified version of the recombinant ! alpha-GAL-A protein, a therapeutic enzyme for the treatment of Fabry disease. Fabry disease is a rare, hereditary, genetic lysosomal storage disorder in humans caused by an X-lined deficiency of the alpha-GAL-A enzyme. The Company is in the animal evaluation testing phase of the development of PRX-102, which tests are based on a mouse model for Fabry disease.

Acetylcholinesterase

Protalix Ltd. is a wholly owned subsidiary of the Company is licensed the rights to certain technology under a research and license agreement with Yissum Research and Development Company (Yissum) and the Boyce Thompson Institute, Inc. Pursuant to the agreement, the Company is developing PRX-105, a plant cell-based acetylcholinesterase (AChE) and its molecular variants for the use in several therapeutic and prophylactic indications, as well as in a biodefense program and an organophosphate-based pesticide treatment program.

As of December 31, 2010, its in-vitro experiments of PRX-105 have shown that the acetylcholinesterase enzyme in its ProCellEx protein expression system demonstrates biological activity on biochemical and cellular levels. In addition, early animal studies demonstrated that the acetylcholinesterase expressed in its ProCellEx protein expression system was able to treat animals exposed to the nerve gas agent analogues, both when injected with its acetylcholinesterase product candidate immediately before exposure or when injected after exposure. In March 2010, it initiated a preliminary phase I clinical trial of PRX-105, which the Company completed in June 2010.

pr-antiTNF

pr-antiTNF is a candidate for the treatment of certain autoimmune diseases such as rheumatoid arthritis, juvenile idiopathic arthritis, ankylosing, spondylitis, psoriatic arthritis and plaque psoriasis. The Company has designed the antiTNF as pr-antiTNF. pr-antiTNF is a plant cell-expressed recombinant fusion protein made from the soluble form of the human TNF receptor (TNFR), f! used to t! he Fc component of a human antibody domain. pr-antiTNF has an identical amino acid sequence to Enbrel and its in-vitro and preclinical animal studies have demonstrated that pr-antiTNF exhibits similar activity to Enbrel. Specifically, pr-antiTNF binds TNF thereby inhibiting it from binding to cellular surface TNF receptors and protects L929 cells from TNF-induced apoptosis in a dose-dependent manner.

The Company competes with Genzyme, Actelion, Crucell N.V., Biolex, Inc., Chlorogen, Inc., Greenovation Biotech GmbH, Symbiosys, Novartis AG/Sandoz Pharmaceuticals, BioGeneriX AG, Stada Arzneimittel AG, BioPartners GmbH and Teva.

Advisors' Opinion:

• [By Keith Speights]

  Other investors might wish that Pfizer would use some of its cash to acquire a few smaller companies. Protalix BioTherapeutics (NYSEMKT: PLX  ) has been mentioned as one possible candidate. The two companies already partner together on Gaucher disease drug Elelyso. In February, Protalix spurred rumors that Pfizer could be interested in buying the company after it announced that it had engaged Citigroup to pursue a "broad array of strategic alternatives."

• [By Maxx Chatsko]

  Industrial biotech isn't the only industry headed to Brazil. Protalix (NYSEMKT: PLX  ) entered into a technology transfer agreement with Brazil's Ministry of Health last week that will pay the company $280 million. The deal is big news for Protalix's first product, Elelyso/Uplyso, which was developed with partner Pfizer (NYSE: PFE  ) . The treatment is approved as an enzyme replacement therapy, or ERT, for adults with type 1 Gaucher disease, and it marks a huge step forward for the future of biomanufacturing. In the following video, Fool contributor Maxx Chatsko explains what this means for the product's commercialization and the adoption of Protalix's novel plant cell-based expression system for therapeutic proteins.

Top 10 Medical Companies To Watch For 2014: Stemline Therapeutics Inc (STML)

Stemline Therapeutics, Inc. (Stemline), incorporated on August 8, 2003, is a clinical-stage biopharmaceutical company focused on discovering, acquiring, developing and commercializing therapeutics that target both cancer stem cells (CSCs) and tumor bulk. The Company is developing two clinical-stage product candidates, SL-401 and SL-701, for which it holds global marketing rights. The indication for SL-401, a biologic-drug conjugate, is acute myeloid leukemia (AML). The indications for SL-701, a synthetic peptide vaccine, are pediatric and adult brain cancer. It has a platform, StemScreen, for the discovery of CSC-targeted compounds, from which it has discovered or validated several of its clinical and preclinical product candidates. Stemline�� StemScreen consists of StemScreen-1 and StemScreen-2 for the identification of CSC-directed compounds.

SL-301 is a small molecule gamma-secretase inhibitor that inhibits Notch, a pathway expressed by CSCs and tumor bulk of multiple cancer types. SL-101 is a monoclonal antibody-based (mAb -based) compound that targets CD123 and has shown in vitro activity against certain hematologic cancers. SL-201 is a small molecule active against certain hematologic and solid tumor types. SL-601 is a mAb-based compound that targets a cell surface marker on bladder CSCs, which is also expressed on a variety of other solid tumor types. It has also in-licensed certain intellectual property directed to mAb-based therapeutics to validated oncology targets, including Glypican-3, Tie-1, CD133, Frizzled, Smoothened and Patched.

SL-401 - An IL-3R-Directed Compound Targeting Cancer Stem Cells and Tumor Bulk

SL-401 is a clinically active biologic-drug conjugate consisting of human interleukin-3 (IL-3) genetically linked to a truncated version of diphtheria toxin. SL-401 targets the IL-3 receptor (IL-3R), which is overexpressed on both the CSCs and tumor bulk of multiple hematologic cancers, including AML. SL-401 has demonstrated preclinical in vit! ro and in vivo activity against both leukemia blasts (which includes tumor bulk) and CSCs of a range of human leukemia cell lines and primary leukemia cells from patients.

SL-701

SL-701 is a clinically active synthetic peptide vaccine that targets several epitopes on CSCs and tumor bulk of brain cancer. In two completed Phase 1/2 clinical trials, SL-701 demonstrated single agent anti-tumor activity in pediatric patients with newly diagnosed brainstem glioma (BSG) and other high-grade gliomas (HGGs) and in adult patients with refractory or recurrent GBM, and other HGGs.

StemScreen-1

StemScreen-1 is a drug discovery platform designed to identify CSC-targeted compounds based on the isolation of CSCs and evaluation of CSC gene expression profiles. CSCs are isolated from primary tumor tissue or cell lines, and then subjected to gene expression analysis using a variety of technologies, including microarray. A control tissue, such as normal bone marrow is analyzed as a comparator against the gene expression profile of the isolated CSCs. These data are then interfaced with an information base of compounds and their mechanisms of action (that is which gene products and pathways they impact). It has utilized StemScreen-1 to discover a number of its preclinical drug candidates. These include SL-201, SL-301, and SL-601. In addition, SL-401 demonstrated activity against CSCs as determined by both an in vitro colony formation and in vivo animal implantation assay, thereby validating certain StemScreen-1 anti-CSC assays.

StemScreen-2

StemScreen-2 is a high throughput drug discovery platform it is developing to discover anti-CSC compounds. StemScreen-2 utilizes a cell-based assay that can track and follow CSCs in their natural state during high throughput screening. In particular, StemScreen-2 utilizes a CSC-specific promoter linked to a reporter as a method for identifying and following CSCs in their native environment of surrounding tumor b! ulk. In t! his way, StemScreen-2 enables the identification of compound hits, in a high throughput manner, with anti-CSC activity.

The Company competes with Boston Biomedical, Inc., Eclipse Therapeutics, Inc., OncoMed Pharmaceuticals, Inc., Verastem, Inc., Astellas Pharma US, Inc., Boehringer Ingelheim GmbH, Dainippon Sumitomo Pharma Co. Ltd., Geron Corp., GlaxoSmithKline plc, ImmunoCellular Therapeutics, Ltd, Macrogenics Inc., Amgen, Inc., Pfizer Inc., Roche Holding AG, Sanofi U.S. LLC., Cyclacel Pharmaceuticals, Inc., Sunesis Pharmaceuticals Inc., Clavis Pharma ASA, Ambit Biosciences Corporation, Celgene Corporation, Eisai Co. Ltd., Celator Pharmaceuticals, Inc., Merck & Co., Inc., Eisai Co., Inc., Roche Holding AG, Novartis AG and Celldex Therapeutics, Inc.

Advisors' Opinion:

• [By Jay Silverman]

  Some of the biggest leaders in that field, and there have been dozens in fields, if not more this year, such as Bluebird (BLUE) and Stemline Therapeutics (STML) and have all pulled back to significantly lower levels; even below, in Bluebird's case, the price that had actually opened up as an IPO, even though it's above its IPO price.