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Live Webcast Scheduled September 26 at 9:30 a.m. ET (6:30 a.m. PT)
LOS ANGELES--(BUSINESS WIRE)--Sept. 20, 2006--CytRx Corporation (Nasdaq:CYTR - News) today announced that initial findings from data analysis of the Company's Phase IIa clinical trial with arimoclomol for the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease) will be presented by President and CEO Steven A. Kriegsman and Senior Vice President of Drug Development, Jack Barber, Ph.D., at the UBS Global Life Sciences Conference. The Company's presentation is scheduled for Tuesday, September 26, 2006 at 9:30 a.m. Eastern time (6:30 a.m. Pacific time) at the Grand Hyatt New York Hotel in New York City.
A live audio webcast of the presentation and accompanying slides will be available on the Company's Web site at www.cytrx.com. Replays will be available approximately three hours after the initial presentation.
Arimoclomol Phase II Clinical Program
In a 10-center, double-blind, placebo-controlled Phase IIa clinical trial, 84 ALS patients were evaluated while receiving either placebo (a capsule without drug) or one of three dose levels of orally administered arimoclomol three times daily for 12 weeks and during a four-week follow-up period without drug. The primary endpoints of this trial are safety and tolerability. Secondary endpoints include a preliminary evaluation of efficacy using two widely accepted surrogate markers, the revised ALS Functional Rating Scale (ALSFRS-R), which is used to determine a patient's capacity and independence in 13 functional activities, and Vital Capacity (VC), an assessment of lung capacity. The Phase IIa trial is powered to detect only extreme responses in these two categories.
About ALS
ALS is a progressive degeneration of the brain and spinal column nerve cells that control the muscles that allow movement. Over a period of months or years, ALS causes increasing muscle weakness, inability to control movement and problems with speaking, swallowing and breathing. According to the ALS Survival Guide, 50% of ALS patients die within 18 months of diagnosis and 80% die within five years. More than 120,000 people are living with ALS worldwide.
About CytRx Corporation
CytRx Corporation is a biopharmaceutical research and development company engaged in the development of high value human therapeutics. The Company owns three clinical-stage compounds based on its small molecule "molecular chaperone" co-induction technology. In September 2006, CytRx announced the receipt of $24.5 million in a non-dilutive agreement with the privately funded ALS Charitable Remainder Trust to fund continued arimoclomol development for the treatment for ALS in return for a one percent royalty payment from potential worldwide sales of arimoclomol for the treatment of ALS to The Greater Los Angeles Chapter of The ALS Association. Arimoclomol has received Orphan Drug status and Fast Track designation from the FDA.
CytRx has previously announced that a novel polyvalent HIV DNA + protein vaccine exclusively licensed to CytRx, developed by researchers at the University of Massachusetts Medical School (UMMS) and Advanced BioScience Laboratories, and funded by the National Institutes of Health, demonstrated very promising interim Phase I clinical trial results that indicate its ability to produce potent antibody responses with neutralizing activity against multiple HIV viral strains. CytRx also has a broad-based strategic alliance with UMMS to develop novel compounds in the areas of ALS, obesity, type 2 diabetes and cytomegalovirus (CMV) using RNAi technology. The Company has a research program with Massachusetts General Hospital, Harvard University's teaching hospital, to use RNAi technology to develop a drug for the treatment of ALS. CytRx Drug Discovery division, located in Worcester, Mass., focuses on the use of RNAi technologies to develop small molecule and RNAi therapeutics to treat obesity and type 2 diabetes. For more information, visit CytRx's Web site at www.cytrx.com.
Forward-Looking Statements
This press release may contain forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended. Examples of such statements include, but are not limited to, statements relating to the expected timing, scope and results of our clinical development and research programs, including the initiation of clinical trials, and statements regarding the potential benefits of our drug candidates and potential drug candidates. Such statements involve risks and uncertainties that could cause actual events or results to differ materially from the events or results described in the forward-looking statements, including risks or uncertainties related to the outcome and timing of CytRx's Phase II clinical trial for arimoclomol, including the open-label extension of that trial, uncertainties regarding regulatory approvals for future clinical testing of arimoclomol and the scope of the clinical testing that may be required by regulatory authorities for arimoclomol, uncertainties regarding the timing and amount of revenues, if any, that will be realized by CytRx from the commercialization of arimoclomol, the significant time and expense that will be incurred in developing any of the potential commercial applications for arimoclomol and the need for additional capital to fund the development of arimoclomol, risks relating to the enforceability of any patents covering CytRx's products and to the possible infringement of third party patents by those products, and the impact of third party reimbursement policies on the use of and pricing for CytRx's products. Additional uncertainties and risks are described in CytRx's most recently filed SEC documents, such as its most recent annual report on Form 10-K, all quarterly reports on Form 10-Q and any current reports on Form 8-K filed since the date of the last Form 10-K. All forward-looking statements are based upon information available to CytRx on the date the statements are first published. CytRx undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.
Contact:
CytRx Corporation
Ed Umali, 310-826-5648, ext. 309
eumali@cytrx.com
or
CEOcast, Inc.
Kevin Theiss/Cormac Glynn, 212-732-4300
ktheiss@ceocast.com
cglynn@ceocast.com
Craig C. Mello, Ph.D.
Known as a technological innovator in the field of molecular genetics, Craig C. Mello, Ph.D., along with Andrew Fire, Ph.D., of Stanford University, is a key inventor on the first approved patent for the use of double stranded RNA for gene silencing, a technology that has come to be known as RNAi. Dr. Mello is the Blais University Chair and Distinguished Professor of Molecular Medicine and a Howard Hughes Medical Institute Investigator at the University of Massachusetts Medical School, in Worcester, Massachusetts. In this capacity, Dr. Mello studies both the biochemical pathway responsible for RNAi and the regulation of gene expression; he has numerous publications credits including 16 in the prestigious journals Science, Nature, Cell, and the Proceedings of the National Academy of Sciences. Prior to joining the University of Massachusetts Medical School, Dr. Mello was a post-doctoral fellow in the lab of Dr. Jim Priess at the Fred Hutchinson Cancer Research Center in Seattle. Dr. Mello received a Ph.D. from Harvard University and a Sc.B. from Brown University. He also conducted graduate studies in the laboratory of David Hirsh, Ph.D., at the University of Colorado. In 2003, Dr. Mello shared the Wiley Foundation Prize and the prestigious National Academy of Sciences Award in Molecular Biology with Dr. Fire for their work on RNAi.
Quelle siehe : http://www.cytrx.com/scientific_advisory_board.html
NEW YORK REAGIERTE HEUTE MIT +7,87 %. DIESE AKTIE IST ERNST ZU NEHMEN !
DAS KÖNNTE EINE SEHR INTERESSANTE STORY IM BIOTECH-SEKTOR GEBEN .
Zellen mit falschen Kopien überlisten - das ist der Trick, für den Fire und Mello den Nobelpreis bekommen. Dank ihrer Forschung können heute einzelne Gene stummgeschaltet werden. Vielleicht arbeiten bald auch Medikamente so. Die Preisträger jedenfalls sind echte Nobel-Schnellstarter.
Einen Hinweis gab es für die Wettsüchtigen und Kaffeesatzleser, die auf Websiten hartes Geld darauf setzten, welche Namen am heutigen Montag in Stockholm bekannt gegeben würden: Die Medizin-Nobelpreisträger Fire und Mello hatten bereits im März den mit 100.000 Euro dotierten Paul-Ehrlich-und Ludwig-Darmstädter-Preis erhalten - immerhin den wichtigsten deutschen Medizinpreis.
"Die Preisträger dieses Jahres haben einen fundamentalen Mechanismus entdeckt, um die Weitergabe genetischer Informationen zu kontrollieren", begründete das Nobelkomitee des Karolinska Instituts in Stockholm seine Wahl.
Mit ihrer Arbeit über RNA-Interferenz ehrt die Nobelstiftung gleichzeitig mikrobiologische Grundlagenforschung und die Entwicklung eines potenten Werkzeugs für die biomedizinische Forschung. Ob es eines Tages auch zum klinischen Einsatz kommt und als Therapie Kranken helfen wird, muss sich noch zeigen.
Die Wissenschaft beschäftigt der geheimnisvolle Mechanismus seit rund 15 Jahren. Erstmals aufmerksam wurden Genforscher im Jahr 1990 auf den Stummschalter für Gene - auch wenn sie sich ihn damals noch nicht erklären konnten: Um die Blütenfarbe von Petunien zu verstärken, hatten Wissenschaftler ein bestimmtes Gen in die Blumen eingeschleust, von dem man wusste, dass es die Produktion von Blütenfarbstoffen anregte. Doch zur allgemeinen Überraschung blieben die genveränderten Blümchen beinahe weiß. Nicht nur hatten die eingeschleusten Gene versagt, auch hatten sich offenbar die natürlich vorhandenen weniger stark bemerkbar gemacht.
Diese plötzliche Unterdrückung blieb zunächst geheimnisvoll. In den nächsten Jahren wurde aber klar: Nicht nur beim Ablesen vom DNA-Doppelstrang können Gene abgeschaltet werden. Auch später noch können die RNA-Abschriften einzelner Gene in den Zellen abgebaut werden - normalerweise hingegen würden nach der Anleitung in diesen Kopien Proteine gefertigt.
Strickleiter-Molekül schaltet Gene aus
Wenn die Zelle eines der Gene abliest, fertigt sie dafür zunächst eine Abschrift der gewünschten Erbanlage, die Boten-RNA (auch mRNA von Messenger, englisch für Bote). Nur sie verlässt den Zellkern. Die Abfolge der chemischen Bausteine darauf bestimmt darüber, welches Protein nach dieser Vorlage entsteht und seine Wirkung im Organismus entfalten kann. Die Boten-RNA ist stets als einzelner Strang, während die DNA doppelsträngig vorliegt.
Im Jahr 1998 wurde das Erbgut des Fadenwurms Caenorhabditis elegans sequenziert. Und die US-Forscher Andrew Fire von der kalifornischen Stanford University und Craig Mello von der University of Massachussets sorgten mit einem Experiment für Aufsehen.
Mello und Fire spritzten den winzigen Fadenwürmern künstliche RNA-Moleküle. Im Gegensatz zum Original waren diese zweisträngig - ähnlich einer Strickleiter. Als Reaktion auf die ungewohnten RNA-Schnipsel bauten die Zellen der Würmer nicht nur das irreguläre doppelte, sondern auch das normale einsträngige RNA-Molekül ab. Daher wurde das Protein, das in der RNA beschrieben war, nicht produziert. Forscher sprechen hier auch vom Gen-Knockdown.
Die Medizinnobelpreisträger 2006 hatten also einen natürlichen Mechanismus als Verfahren für die Forschung nutzbar gemacht: Wer die Reihenfolge der Original-Bauvorlage kennt, kann die passenden RNA-Doppelstränge im Labor herstellen und in die Zelle spritzen. Jedes Gen oder gleich mehrere lassen sich so ausschalten. Ein - wesentlich komplizierterer - Eingriff in die Erbsubstanz der DNA im Zellkern ist nicht nötig.
In den folgenden Jahren fanden viele Gruppen heraus, auf welche Weise Zellen die doppelten RNAs und das einsträngige Original entdecken und vernichten. Dabei stellte sich heraus, dass viele Organismen dieses Verfahren auch natürlicherweise nutzen: Viren etwa bringen häufig doppelsträngige RNA-Moleküle mit, um die Verteidigung befallener Zellen zu schwächen.
Nobelpreis für junge Entdeckung - und junge Forscher
Mit sogenannter Mikro-RNA (miRNA) lassen sich auch beim Menschen gezielt die Abschriften einzelner Gene stummschalten. 2002 kürte das Wissenschaftsmagazin "Science" diese Erkenntnis zum "Durchbruch des Jahres".
"RNA-Interferenz eröffnet uns aufregende Möglichkeiten in der Gentechnik", sagte das Nobelkomitee am heutigen Montag. "Es gibt bereits Pläne, RNA-Stummschaltung als Therapie bei Virusinfektionen, Herzkreislaufkrankheiten, Krebs, hormonellen Störungen und einer Reihe anderer Krankheiten zu entwickeln."
Er habe es für möglich gehalten, den Preis für die Entdeckung der RNA-Interferenz zu gewinnen, sagte Craig Mello, "aber ich bin erst 45 Jahre alt, daher dachte ich, dass es in zehn oder zwanzig Jahren passieren würde." Sein Kollege Fire ist 47 Jahre alt.
Seit rund fünfzehn Jahre beschäftigen sich Wissenschaftler mit diesem Phänomen, vor acht Jahren veröffentlichten Fire und Mello ihren Aufsatz zum Fadenwurm-Experiment. Das ist an den Maßstäben des Nobel-Komitees gemessen ein relativ überschaubarer Zeitraum: Der Forscher Peyton Rous etwa musste mehr als 50 Jahre warten, bis ihm der Medizin-Nobelpreis für die Entdeckung Krebs-verursachender Viren zuteil wurde.
stx/AFP/AP/dpa/rtr
BILD: Andrew Fire (links) und Craig Mello
Tuesday October 3, 8:51 am ET
LOS ANGELES--(BUSINESS WIRE)--CytRx Corporation (Nasdaq:CYTR - News) today applauded the announcement that its Scientific Advisory Board member Craig C. Mello, Ph.D., together with Andrew Fire, Ph.D., was awarded the Nobel Prize in medicine for discovery of the use of double-stranded RNA for gene silencing, a technology that has come to be known as RNA interference (RNAi). Drs. Mello and Fire acted as key inventors on the first approved RNAi patent, which is licensed to CytRx on a non-exclusive basis.
As a member of the CytRx Scientific Advisory Board, Dr. Mello acts in an advisory capacity to assist the Company to develop RNAi therapeutics for specific diseases.
Dr. Mello is the Blais University Chair and Distinguished Professor of Molecular Medicine and a Howard Hughes Medical Institute Investigator at the University of Massachusetts Medical School, in Worcester, Mass. In this capacity, Dr. Mello studies both the biochemical pathway responsible for RNAi and the regulation of gene expression. He has numerous publication credits including 16 in the prestigious journals Science, Nature, Cell, and the Proceedings of the National Academy of Sciences.
"We extend our sincerest congratulations to Drs. Mello and Fire for receiving this celebrated award and are delighted that the Nobel committee has recognized the potential of this highly novel approach that has implications over a broad range of catastrophic diseases," said President and CEO Steven A. Kriegsman. "RNAi could offer an entirely new paradigm in disease treatment by regulating the effect of specific diseases, making it a valuable tool in drug discovery, as well as a direct therapeutic. At CytRx, we are involved in drug discovery using RNAi targeting for major global health concerns that include obesity and type 2 diabetes."
CytRx uses RNAi to help screen and identify classical, orally-available small molecule drugs. In its obesity and type 2 diabetes programs, CytRx is using its proprietary high throughput RNAi-based screening technology to screen the more than 2,000 candidate genes that may be involved in diabetes and obesity in order to rapidly validate numerous drug targets in the most critical pathways which regulate metabolism. During the past year, CytRx-sponsored research programs have discovered and validated approximately 30 new type 2 diabetes and obesity drug targets. CytRx also is exploring the utility of RNAi for direct therapeutic applications when technically feasible.
"We strongly believe in the value of RNAi technology, which has prompted us to pursue a plan, subject to obtaining necessary funding, to transfer all of our RNAi therapeutics assets into a newly-formed subsidiary to accelerate the development and commercialization of drugs based on this platform," added Mr. Kriegsman. "We plan to continue using RNAi gene silencing technology as a drug discovery tool to facilitate our small molecule drug discovery program."
RNAi Background
RNA is a constituent of all living cells and many viruses, consisting of a long, usually single-stranded chain of alternating phosphate and ribose units with the bases adenine, guanine, cytosine, and uracil bonded to the ribose. The structure and base sequence of RNA is used by the cell as a messenger (mRNA) to be used as an "instruction manual" for the synthesis of a specific protein. RNAi technology uses short double-stranded RNA, or dsRNA, molecules to silence targeted genes and, as a result, is commonly referred to as "gene silencing." RNAi has been shown to effectively silence targeted genes within living cells with great specificity and potency. The end result is the destruction of the specific mRNA, thus silencing that gene and the protein that the mRNA would normally be used to synthesize.
About CytRx Corporation
CytRx Corporation is a biopharmaceutical research and development company engaged in the development of high-value human therapeutics. The Company owns three clinical-stage compounds based on its small molecule "molecular chaperone" co-induction technology. In September 2006, CytRx reported reaching the primary endpoints of safety and tolerability from a Phase IIa trial with its lead small molecule product candidate arimoclomol for the treatment for amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease). Also in September 2006 CytRx announced receipt of $24.5 million in a non-dilutive agreement with the privately funded ALS Charitable Remainder Trust to fund continued arimoclomol development for the treatment for ALS in return for one percent royalty payment from potential worldwide sales of arimoclomol for the treatment of ALS to The Greater Los Angeles Chapter of The ALS Association. Arimoclomol has received Orphan Drug status and Fast Track designation from the U.S. Food and Drug Administration.
CytRx has previously announced that a novel polyvalent HIV DNA + protein vaccine exclusively licensed to CytRx, developed by researchers at the University of Massachusetts Medical School (UMMS) and Advanced BioScience Laboratories, and funded by the National Institutes of Health, demonstrated very promising interim Phase I clinical trial results that indicate its ability to produce potent antibody responses with neutralizing activity against multiple HIV viral strains. CytRx also has a broad-based strategic alliance with UMMS to develop novel compounds in the areas of ALS, obesity, type 2 diabetes and cytomegalovirus (CMV) using RNAi technology. The Company has a research program with Massachusetts General Hospital, Harvard University's teaching hospital, to use RNAi technology to develop a drug for the treatment of ALS. CytRx Drug Discovery division, located in Worcester, Mass., focuses on the use of RNAi technologies to develop small molecule and RNAi therapeutics to treat obesity and type 2 diabetes. For more information, visit CytRx's Web site at www.cytrx.com.
Forward-Looking Statements
This press release may contain forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended. Examples of such statements include, but are not limited to, statements relating to the expected timing, scope and results of our clinical development and research programs, including the initiation of clinical trials, and statements regarding the potential benefits of our drug candidates and potential drug candidates. Such statements involve risks and uncertainties that could cause actual events or results to differ materially from the events or results described in the forward-looking statements, including risks or uncertainties related to the early stage of CytRx's RNAi, diabetes, obesity, cytomegalovirus and ALS research, the need for future clinical testing of any RNAi-based product candidates and small molecules that may be developed by CytRx using RNAi screening methods, the significant time and expense that will be incurred in developing any of the potential commercial applications for CytRx's RNAi technology or small molecules, uncertainties related to regulatory approvals for clinical testing and the scope of the clinical testing that may be required by regulatory authorities for its molecular chaperone co-induction drug candidates, including arimoclomol, and other products, and the timing and outcomes of those tests, risks relating to the enforceability of any patents covering CytRx's products and to the possible infringement of third party patents by those products, and the impact of third party reimbursement policies on the use of and pricing for CytRx's products. Additional uncertainties and risks are described in CytRx's most recently filed SEC documents, such as its most recent annual report on Form 10-K, all quarterly reports on Form 10-Q and any current reports on Form 8-K filed since the date of the last Form 10-K. All forward-looking statements are based upon information available to CytRx on the date the statements are first published. CytRx undertakes no obligation to publicly update or revise any forward- looking statements, whether as a result of new information, future events or otherwise.
Contact:
CEOcast, Inc.
Kevin Theiss
ktheiss@ceocast.com
Cormac Glynn
cglynn@ceocast.com
212-732-4300
CytRx RNAi Subsidiary, RXi Pharmaceuticals, Announces Worldwide Research and Therapeutic RNAi Licensing Agreement with Cold Spring Harbor Laboratory
Monday March 19, 8:30 am ET
LOS ANGELES--(BUSINESS WIRE)--CytRx Corporation (NASDAQ:CYTR - News) today announced that its majority-owned subsidiary, RXi Pharmaceuticals Corporation (RXi), has secured a non-exclusive, worldwide research and therapeutic license from Cold Spring Harbor Laboratory to their short hairpin RNAi technology (shRNAi). The licensed technology potentially allows for the more efficient triggering of RNA interference (RNAi) and includes the use of short hairpin RNAis (shRNAis) either delivered as RNA compositions or encoded by DNA constructs. Patents covering this technology are currently pending. Financial terms were not disclosed for competitive reasons.
"With this license for all human therapeutic areas, we gain the well-validated advantage of shRNAi - which is reported to be up to 10- to 100-fold more potent compared with standard siRNA," said Tod Woolf, Ph.D., RXi's President and CEO. "This license supports our activities to accumulate a broad technology portfolio of RNA chemistry, and configuration and delivery technology, and marks another step in our construction of a world-class RNAi therapeutics company."
The technology licensed by RXi was developed in the laboratory of Gregory J. Hannon, Ph.D., a Howard Hughes Medical Institute Investigator at Cold Spring Harbor Laboratory. Dr. Hannon, who is a co-founder and member of RXi's scientific advisory board, is also a leading expert on oncogene pathways and was formerly an advisor to Alnylam Pharmaceuticals. Dr. Hannon's laboratory is credited with discovering the mechanism of RNAi in human cells (RISC/siRNA), as well as developing shRNAi technology.
REST WIE ÜBLICH.