With the�SPDR S&P Biotech Index�up 29% over the trailing-12-month period, it's evident that investment dollars are willingly flowing into the biotech sector. Keeping that in mind, let's have a look at some of the rulings, studies, and companies that made waves in the sector last week.
Clinically impressive
As we do at the conclusion of every week, let's lead off with some of the most positive stories within the sector. Perhaps none stands out as more intriguing than Gilead Sciences (NASDAQ: GILD ) late-stage study of idelalisib in previously treated chronic lymphocytic leukemia patients who aren't fit for chemotherapy. According to Gilead's press release on Wednesday, an independent data monitoring committee stopped the trial early because of, "highly statistically significant efficacy for the primary endpoint of progression-free survival in patients receiving idelalisib plus rituximab compared to those receiving rituximab alone." It's looking as if we can chalk up another successful late-stage study for Gilead.
Top Defense Stocks For 2015: Tonix Pharmaceuticals Holding Corp (TNXP)
Tonix Pharmaceuticals Holding Corp., incorporated on November 16, 2011, is a specialty pharmaceutical company focused on developing novel pharmaceutical products for disorders of the central nervous system (CNS). The Company develops TNX-102 sublingual tablet, or TNX-102 SL. TNX-102 SL is a novel dose and formulation of cyclobenzaprine (CBP), the active pharmaceutical ingredient of two prescribed muscle relaxant products, Flexeril and Amrix. TNX-102 SL is also intended for chronic use. It develops TNX-102 SL for the treatment of FM under a United States Investigational New Drug application (IND), and under three clinical trial applications (CTAs), filed in Canada. It also develops TNX-102 SL for the treatment of post-traumatic stress disorder (PTSD). As of January 25, 2013, the Company focuses on product development, and had not generated any revenues.
The Company searches for potential therapeutic solutions among known pharmaceutical agents that lack regulatory approval for the indications it seeks, but may be approved for use in other indications. It seeks to create new dose and formulation options that are tailored to the therapeutic uses to which it applies these agents. TNX-102 SL is a small, rapidly disintegrating tablet containing CBP for sublingual administration at bedtime. The Company designed TNX-102 SL for delivery of CBP to the systemic circulation through sublingual transmucosal absorption and to avoid first-pass liver metabolism.
CBP is the active pharmaceutical ingredient in its lead product candidate, TNX-102 SL. In addition to CBP, TNX-102 SL contains inactive ingredients, called excipients, which are approved for pharmaceutical use. In addition to TNX-102 SL, it develops other formulations of CBP, including TNX-102 promicellar gelatin capsule, or TNX-102 gelcap. It developed TNX-102 gelcap under an agreement with Lipocine, Inc. (Lipocine), a contract formulation developer and small-scale manufacturer. In addition it has other pipeline of other product cand! idates, including TNX-201 and TNX-301. TNX-201 is based on isometheptene mucate and is under development as a treatment for certain types of headaches.
Advisors' Opinion:- [By John Kell]
Tonix Pharmaceuticals Holding Corp.(TNXP) is planning to offer some shares to raise money to fund clinical trials and the continued development of the specialty pharmaceutical company’s treatments. Shares edged up 2.1% to $16.75 premarket.
- [By John Udovich]
It�� a new year and the first one and a half trading weeks of 2014 has not disappointed biotech investors as the sector and mid cap or small cap biotech or�pharma stocks like Intercept Pharmaceuticals Inc (NASDAQ: ICPT), Epizyme Inc (NASDAQ: EPZM), Tonix Pharmaceuticals Holding Corp (NASDAQ: TNXP) and TNI BioTech Inc (OTCQB: TNIB) either surging or producing some news plus there have been IPO filings for future listings for�Flexion Therapeutics (NASDAQ: FLXN), Aldexa Therapeutics (NASDAQ: ALDX), Retrophin (NASDAQ:�RTRX) and Dicerna Pharmaceuticals (NASDAQ: DRNA). Consider the following news so far this year:
Top 5 Biotech Companies To Watch For 2014: Prosensa Holding NV (RNA)
Prosensa Holding N.V., formerly Prosensa Holding B.V., is a biotechnology company engaged in the discovery and development of ribonucleic acid-modulating (RNA)-modulating, therapeutics for the treatment of genetic disorders. The Company�� primary focus is on rare neuromuscular and neurodegenerative disorders with a large unmet medical need, including Duchenne muscular dystrophy, myotonic dystrophy and Huntington�� disease. The Company�� clinical portfolio of RNA-based product candidates is focused on the treatment of Duchenne muscular dystrophy (DMD). The Company�� platform technology allows the development of RNA-modulating therapeutics that either interferes with splicing (exon skipping, exon inclusion, or splice mutation correction), remove mutant RNA, or block RNA expression, for different indications.
DMD is a rare, severe muscle wasting disease that occurs in up to 1 in 3,500 male births. It is commonly diagnosed between the ages of three to five, when boys begin to show signs of impaired motor development. PRO044, the Company�� product candidate, addresses a separate sub-population of DMD patients. The Company developed PRO044 using its exon-skipping technology to generate a product candidate with the same mechanism of action that is used by drisapersen.
Advisors' Opinion:- [By John Udovich]
It should also be mentioned that earlier in the week, GlaxoSmithKline plc (NYSE: GSK) returned full rights of Duchenne muscular dystrophy drug drisapersen to their partner�Prosensa Holding NV (NASDAQ: RNA) and that news sent shares down 12.48% on�Monday. Moreover,�Prosensa Holding also sank some 70% last fall when Drisapersen failed a phase 3 trial by missing a primary endpoint measuring for how far patients could walk over the course of six minutes (the improvement�wasn't significantly better than the placebo).�However, Prosensa Holding also rose 24.37% yesterday after announcing�initial findings from further analyses from the aggregate data for drisapersen, with the CEO saying:
- [By Keith Speights]
Successful launch
You couldn't even buy stock in Prosensa (NASDAQ: RNA ) just a few weeks ago. The biotech launched its IPO on June 28. To say that launch has gone successfully is an understatement. Shares are now more than double the IPO price and climbed 41% this week.
Top 5 Biotech 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 David Zeiler]
3. Stemline Therapeutics Inc. (Nasdaq: STML): This biotech develops drugs that target cancer stem cells and tumors. Stemline went public January 29 at $10 a share and rose just 11.78% on its first day. But STML has climbed steadily since, and currently trades at $37.46, up 274.6% from its IPO price.
Top 5 Biotech Companies To Watch For 2014: 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 David Schneider]
There are 3 companies in particular that fit these criteria extremely well. First let's take a look at Johnson & Johnson (JNJ). This healthcare developer, manufacturer, and seller has been increasing dividends for over 25 years at an over 5 percent pace. In each scenario, it is reasonable to assume a 5 percent average annual dividend growth rate as well as a conservative 5 percent increase in share price. We will take a long-term view and look at the affects over a 40-year time horizon that younger investors can fully take advantage of.
- [By Lauren Pollock]
Johnson & Johnson sa(JNJ)id its fourth-quarter earnings rose 37% as the company’s pharmaceutical segment continued to lead sales growth. For the year, the maker of products from Band-Aids to knee-replacement parts forecast per-share earnings mostly below consensus estimates.
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