The Food and Drug Administration (FDA) made a groundbreaking policy change last year that is having a large impact on the biotech industry and creating a big opportunity for investors.
To create a faster path to commercialization for potentially life-saving drugs and medicine, the FDA in July introduced a new "breakthrough" status for drugs that show "substantial improvement on existing therapies for clinically significant endpoints."
For an industry that is accustomed to a costly 10-year commercialization process, the breakthrough status is a welcome shift. An accelerated channel to commercialization could enable drug companies to shave years and billions of dollars from the cumbersome process.
Best Biotech Stocks To Watch For 2014: Nektar Therapeutics(NKTR)
Nektar Therapeutics, a clinical-stage biopharmaceutical company, engages in developing a pipeline of drug candidates that utilize its PEGylation and polymer conjugate technology platforms. The company?s product pipeline consists of drug candidates across various therapeutic areas, including oncology, pain, anti-infectives, anti-viral, and immunology. Its research and development activities involve small molecule drugs, peptides, and other potential biologic drug candidates. The company?s proprietary drug candidates in clinical development comprise NKTR-118, a peripheral opioid antagonist, which has completed Phase II clinical trail for the treatment of opioid-induced constipation; BAY41-6551 that has completed Phase II clinical trail to treat gram-negative pneumonias; NKTR-102, a topoisomerase I inhibitor-polymer conjugate, which is in Phase II clinical trail for multiple cancer indications, including breast, ovarian, and colorectal; and NKTR-105 that is in Phase I clinica l trail to treat solid tumors. Its preclinical products consists of NKTR-119 (Opioid/NKTR-118 combinations) for the treatment of pain; NKTR-181 (abuse deterrent, tamper-resistant opioid) to treat pain; NKTR-194 (non-scheduled opioid) for the treatment of mild to moderate pain; NKTR-171 (tricyclic antidepressant) to treat neuropathic pain; and NKTR-140 (protease inhibitor candidate) to treat HIV. The company has collaboration with Bayer Healthcare LLC to develop BAY41-6551 (NKTR-061, Amikacin Inhale), which is an inhaled solution of amikacin, an aminoglycoside antibiotic; and a license agreement with AstraZeneca AB for the development and commercialization of Oral NKTR-118 and NKTR-119. In addition, Nektar Therapeutics has various license, manufacturing, and supply agreements for its technology with biotechnology and pharmaceutical companies, such as Affymax, Amgen, Baxter, Roche, Merck, Pfizer, and UCB Pharma. The company was founded in 1990 and is headquartered in San Franc isco, California.
Advisors' Opinion:- [By Jay Silverman]
Steve Halpern: Another stock you follow closely is Nektar (NKTR). What do you see happening there?
Jay Silverman: Yes, Nektar is another platform company at the MTSL and they are about to release data on one of their compounds that is a pain reliever-unlike the very severe side effects of OxyContin, the opioid pain relievers that are out there, which have caused severe health and addictive concerns, leading to very, very troublesome side effects and death in some cases.
Best Biotech Stocks To Watch For 2014: Cell Therapeutics Inc (CTIC)
Cell Therapeutics, Inc. (CTI), incorporated in 1991, develops, acquires and commercializes treatments for cancer. The Company�� research, development, acquisition and in-licensing activities concentrate on identifying and developing new ways to treat cancer. As of December 31, 2011, CTI focused its efforts on Pixuvri (pixantrone dimaleate) (Pixuvri), OPAXIO (paclitaxel poliglumex) (OPAXIO), tosedostat, brostallicin and bisplatinates. As of December 31, 2011, it developed Pixuvri, an anthracycline derivative for the treatment of hematologic malignancies and solid tumors. Another late-stage drug candidate of the Company, OPAXIO, is being studied as a potential maintenance therapy for women with advanced stage ovarian cancer, who achieve a complete remission following first-line therapy with paclitaxel and carboplatin. As of December 31, 2011, it also developed tosedostat in collaboration with Chroma Therapeutics, Ltd. (Chroma). On May 31, 2012, CTI completed its acquisition gaining worldwide rights to S*BIO Pte Ltd.'s (S*BIO) pacritinib.
Pixuvri
As of December 31, 2011, the Company developed Pixuvri, an aza-anthracenedione derivative, for the treatment of non-Hodgkin�� lymphoma (NHL), and various other hematologic malignancies, and solid tumors. Pixuvri was studied in the Company�� EXTEND, or PIX301, clinical trial, which was a phase III single-agent trial of Pixuvri for patients with relapsed, refractory aggressive NHL who received two or more prior therapies and who were sensitive to treatment with anthracyclines. On September 28, 2011, CTI announced that a second independent radiology assessment of response and progression endpoint data from its PIX301 clinical trial of Pixuvri was achieved with statistical significance. The results of the EXTEND trial met its primary endpoint and showed that patients randomized to treatment with Pixuvri achieved a significantly higher rate of confirmed and unconfirmed complete response compared to patients treated with standard chem! otherapy had a significantly increased overall response rate and experienced a statistically significant improvement in median progression free survival. Pixuvri had predictable and manageable toxicities when administered at the proposed dose and schedule in the EXTEND clinical trial in heavily pre-treated patients. In March 2011, the Company initiated the PIX-R trial to study Pixuvri in combination with rituximab in patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL). Pixuvri has also been studied in patients with HER2-negative metastatic breast cancer who have tumor progression after at least two, but not more than three, prior chemotherapy regimens. In the second quarter of 2010, the NCCTG opened this phase II study for enrollment. The study is closed to accrual and results are expected to be reported by the NCCTG later in 2012.
OPAXIO
OPAXIO is the Company�� biologically-enhanced chemotherapeutic agent that links paclitaxel to a biodegradable polyglutamate polymer, resulting in a new chemical entity. As of December 31, 2011, the Company focused its development of OPAXIO on ovarian, brain, esophageal, head and neck cancer. OPAXIO was designed to improve the delivery of paclitaxel to tumor tissue while protecting normal tissue from toxic side effects. In November 2010, results were presented by the Brown University Oncology Group from a phase II trial of OPAXIO combined with temozolomide (TMZ), and radiotherapy in patients with newly-diagnosed, high-grade gliomas, a type of brain cancer. The trial demonstrated a high rate of complete and partial responses and a high rate of six month progression free survival (PFS). Based on these results, the Brown University Oncology Group has initiated a randomized, multicenter, phase II study of OPAXIO and standard radiotherapy versus TMZ and radiotherapy for newly diagnosed patients with glioblastoma with an active gene termed MGMT that reduces responsiveness to TMZ. A phase I/II study of OPAXIO combined with radi! otherapy ! and cisplatin was initiated by SUNY Upstate Medical University, in patients with locally advanced head and neck cancer.
Tosedostat
In March 2011, the Company entered into a co-development and license agreement with Chroma Therapeutics, Ltd. (Chroma), providing the Company with marketing and co-development rights to Chroma�� drug candidate, tosedostat, in North, Central and South America. Tosedostat is an oral, aminopeptidase inhibitor that has demonstrated anti-tumor responses in blood related cancers and solid tumors in phase I-II clinical trials. Interim results from the phase II OPAL study of tosedostat in elderly patients with relapsed or refractory acute myeloid leukemia (AML) showed that once-daily, oral doses of tosedostat had predictable and manageable toxicities and results demonstrated response rates, including a high-response rate among patients who received prior hypomethylating agents, which are used to treat myelodysplastic syndrome (MDS), a precursor of AML.
Brostallicin
As of December 31, 2011, the Company developed brostallicin through its wholly owned subsidiary, Systems Medicine LLC, which holds rights to use, develop, import and export brostallicin. Brostallicin is a synthetic deoxyribonucleic acid (DNA) minor groove binding agent that has demonstrated anti-tumor activity and a favorable safety profile in clinical trials, in which more than 230 patients have been treated as of December 31, 2011. The Company uses a genomic-based platform to guide the development of brostallicin. A phase II study of brostallicin in relapsed, refractory soft tissue sarcoma met its predefined activity and safety hurdles and resulted in a first-line phase II clinical trial study that was conducted by the European Organization for Research and Treatment of Cancer (EORTC).
The Company competes with Bristol-Myers Squibb Company, Sanofi-Aventis, Pfizer, Roche Group, Genentech, Inc., Astellas Pharma, Eli Lilly and Company, Celgene, Telik, I! nc., TEVA! Pharmaceuticals Industries Ltd. and PharmaMar.
Advisors' Opinion:- [By John Udovich]
If you have not been watching the biotech sector lately, you should start paying attention as the sector along with small cap biotech stocks like Cell Therapeutics Inc (NASDAQ: CTIC), BIND Therapeutics Inc (NASDAQ: BIND) and TNI BioTech (OTCMKTS: TNIB) continue to produce a steady stream of good news for investors thanks to positive industry trends. Moreover, Ophthotech Corp (NASDAQ: OPHT), Foundation Medicine Inc (NASDAQ: FMI), Evoke Pharma and Fate Therapeutics Inc (NASDAQ: FATE) are this week's biotech IPOs that will no doubt be watched closely by Wall Street and industry observers in general. With that in mind, consider the following biotech news or recent articles about the industry and the small cap players in it:
- [By Bryan Murphy]
If you're reading this, then odds are you already know that the last two weeks (not even a full two weeks) have been more fruitful for Cell Therapeutics Inc. (NASDAQ:CTIC) shareholders than the prior two years have been - the stock's up 28% since last Thursday. And, odds are you already know why. The question most of you are asking now is, can CTIC actually keep climbing at this pace, or even keep climbing at any pace? The answer is "yes", though floating that answer almost inherently requires a deeper explanation.
Top 5 Growth Stocks To Own For 2014: Osiris Therapeutics Inc.(OSIR)
Osiris Therapeutics, Inc., a stem cell company, focuses on the development and marketing of therapeutic products to treat various medical conditions in the inflammatory, autoimmune, orthopedic, and cardiovascular areas. It operates in two business segments, Therapeutics and Biosurgery. The Therapeutics segment focuses on developing biologic stem cell drug candidates from a readily available and non-controversial source, adult bone marrow. The Biosurgery segment works to harness the ability of cells and novel constructs to promote the body's natural healing. This segment focuses on developing biologic products for use in surgical procedures. The company?s lead biologic drug candidate is Prochymal, which is in phase 2 and 3 clinical trails for various indications, including acute graft versus host disease (GvHD), Crohn's disease, acute myocardial infarction, type 1 diabetes, pulmonary disease, and gastrointestinal injury resulting from radiation exposure. Its biologic drug candidates also include Chondrogen, a preparation of adult mesenchymal stem cells that is in phase 2 clinical trials for osteoarthritis and cartilage protection. The company has collaboration agreements with Genzyme Corporation for the development and commercialization of Prochymal and Chondrogen in various countries except in the United States and Canada. It also has a partnership with Juvenile Diabetes Research Foundation for the development of Prochymal as a treatment for the preservation of insulin production in patients with newly diagnosed type 1 diabetes mellitus. Osiris Therapeutics, Inc. was founded in 1992 and is headquartered in Columbia, Maryland.
Advisors' Opinion:- [By Alexander Maxwell]
One of the companies attempting to develop a better treatment for chronic diabetic foot ulcers is Osiris Therapeutics� (NASDAQ: OSIR ) . Earlier this month, Osiris shares more than doubled as the company announced positive data for its CDFU drug Grafix. The study results were very impressive to say the least; the study was stopped early due to the overwhelming efficacy exhibited by the treatment. A main highlight is the fact that 62% of Grafix patients had their wound closed at 12 weeks, compared to only 21% of patients using conventional methods. Clearly, the efficacy in this endpoint was overwhelming. Grafix also achieved all of the secondary endpoints for the trial, and more importantly demonstrated a relatively benign safety record.�
Best Biotech Stocks To Watch For 2014: Cannabis Science Inc (CBIS.PK)
Cannabis Science, Inc., incorporated on May 4, 2007, is a development-stage company. The Company is engaged in the creation of cannabis-based medicines, both with and without psychoactive properties, to treats disease and the symptoms of disease, as well as for general health maintenance. On February 9, 2012, the Company acquired GGECO University, Inc. (GGECO). On March 21, 2012, the Company acquired Cannabis Consulting Inc. (CCI Group).
The Company is engaged in medical marijuana research and development. The Company works with world authorities on phytocannabinoid science targeting critical illnesses, and adheres to scientific methodologies to develop, produce, and commercialize phytocannabinoid-based pharmaceutical products.
Best Biotech Stocks To Watch For 2014: Neurocrine Biosciences Inc.(NBIX)
Neurocrine Biosciences, Inc. engages in the discovery, development, and commercialization of drugs for the treatment of neurological and endocrine-related diseases and disorders in the United States. It develops drugs for endometriosis, stress-related disorders, pain, tardive dyskinesia, uterine fibroids, diabetes, insomnia, and other neurological and endocrine-related diseases and disorders. The company?s products in clinical development include Elagolix, a Phase II drug for endometriosis; Vesicular Monoamine Transporter 2 Inhibitor (VMAT2), a Phase II drug for movement disorders; CRF2 Peptide Agonist, a Phase II drug for cardiovascular diseases; CRF1 Antagonist, a Phase II drug for stress-related disorders; and Elagolix, a Phase II drug for uterine fibroids. Its research programs comprise G Protein-Coupled Receptor 119 (GPR119) for type II diabetes; VMAT2 for schizophrenia; GnRH Antagonists for men?s and women?s health, and oncology; Antiepileptic Drugs for epilepsy, essential tremor, and pain; and G Protein-Coupled Receptors for other conditions. The company has collaborations with GlaxoSmithKline to develop and commercialize CRF antagonists for psychiatric, neurological, and gastrointestinal diseases; Dainippon Sumitomo Pharma Co. Ltd. to develop and commercialize Indiplon in Japan; Abbott International Luxembourg S.�r.l. to develop and commercialize elagolix and GnRH antagonists for women?s and men?s health indications; and Boehringer Ingelheim International GmbH to research, develop, and commercialize small molecule GPR119 agonists for the treatment of type II diabetes and other indications. Neurocrine Biosciences, Inc. was founded in 1992 and is headquartered in San Diego, California.
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