arrowhead läuft - zu recht
Gruss Macos
Arrowhead Receives Orphan Drug Designation for ARC-AAT
PASADENA, Calif.--(BUSINESS WIRE)-- Arrowhead Research Corporation (NASDAQ: ARWR), a biopharmaceutical company developing targeted RNAi therapeutics, today announced the United States Food and Drug Administration (FDA) has granted ARC-AAT orphan drug designation. ARC-AAT is Arrowhead's RNAi-based therapeutic candidate being investigated for the treatment of liver disease associated with Alpha-1 Antitrypsin Deficiency (AATD), a rare genetic disease that severely damages the liver and lungs of affected children and adults. Arrowhead is currently conducting part B of a Phase 1 study of ARC-AAT in patients with PiZZ genotype AATD.
"Receiving orphan drug designation is an important milestone in the development of ARC-AAT, which we think is a very promising program aimed at providing a better option for patients with liver disease associated with alpha-1 antitrypsin deficiency," said Bruce D. Given, M.D., Arrowhead's Chief Operating Officer. "The Orphan Drug Act provides important incentives for sponsors to develop drugs that treat rare diseases and we look forward to more engagement with the FDA as the development of ARC-AAT progresses."
The ongoing Phase 1 trial of ARC-AAT is a multi-center, randomized, placebo-controlled, double-blind, single dose-escalation first-in-human study to evaluate the safety, tolerability and pharmacokinetics of ARC-AAT and the effect on circulating AAT levels. The study has been enrolling in dose cohorts of six participants each, with participants randomized at a ratio of 2:1 (active:placebo) to receive a single intravenous injection of either ARC-AAT or placebo (normal saline). The study consists of two parts; Part A in healthy volunteers, which has been completed, and Part B to be conducted in patients with PiZZ genotype AATD. The study evaluates participants for 28 days following dosing, with additional follow-up if needed every 2 weeks until AAT levels return to baseline.
The FDA Office of Orphan Products Development (OOPD) mission is to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. In fulfilling that task, OOPD evaluates scientific and clinical data submissions from sponsors to identify and designate products as promising for rare diseases and to further advance scientific development of such promising medical products. Orphan drug designation provides incentives for sponsors to develop products for rare diseases. These incentives include increased engagement with FDA on drug development activities, exemption from all future product-specific regulatory fees, the opportunity to apply for R&D funding, tax credits, an increased chance of priority review, and 7 years of orphan exclusivity at time of New Drug Application (NDA) approval.
http://ir.arrowheadresearch.com/releasedetail.cfm?ReleaseID=917403
Apr 30, 2015
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Arrowhead Publishes Data on Advances in Subcutaneous siRNA Delivery
PASADENA, Calif.--(BUSINESS WIRE)-- Arrowhead Research Corporation (NASDAQ: ARWR), a biopharmaceutical company developing targeted RNAi therapeutics, today announced the publication of new data on a subcutaneously administered formulation of its Dynamic Polyconjugate (DPC) delivery system. The company believes the new DPCs are highly potent and may represent a dramatic improvement in duration of activity over competing technologies.This new class of DPCs may also enable targeting of RNAi therapeutics to tissues outside of the liver. The manuscript entitled, "Protease-triggered siRNA delivery vehicles," by David B. Rozema et al, was made available online ahead of print in the Journal of Controlled Release 209 (2015) 57-66.
In the publication, Arrowhead scientists describe the development of protease-sensitive masking chemistries that are used in a class DPCs where the RNAi trigger molecule is conjugated directly to the polymer delivery vehicle. These new vehicles expand on the company's existing acid labile DPCs, where the polymer and RNAi trigger are two separate molecules and are co-injected. The protease-sensitive linkages appear to be more stable and have longer circulation times, which may allow for an increased range of targeting, and appear amenable to subcutaneous administration. The publication reports a high level of target gene knockdown and long duration of effect can be achieved after subcutaneous injection with these new DPC delivery vehicles in nonhuman primates. After a single subcutaneous injection of 0.5 mg/kg siRNA against Factor 7 (FVII), a liver expressed coagulation factor that is secreted into the bloodstream, a 99% reduction of FVII activity was observed in nonhuman primates. Further, this reduction was highly durable with maximal knockdown occurring 24 days after injection and measurable reduction in FVII activity appears to persist for up to 200 days.
"This publication speaks to the flexibility of the DPCs and the ability of our scientists to continually expand the platform to enable additional uses," said Christopher Anzalone, Ph.D., president and chief executive officer. "These data suggest that subcutaneously administered DPCs that employ the protease sensitive masking chemistries may be well suited to address chronic diseases of the liver that require long-term treatment and where long dosing intervals would be very attractive to physicians and patients."
http://ir.arrowheadresearch.com/releasedetail.cfm?ReleaseID=909779
Gruss Macos
Feb 29, 2012
Arrowhead Announces Patent Issuance Covering siRNA Inhibitors of HIF-2α (EPAS1)
http://ir.arrowheadresearch.com/releasedetail.cfm?ReleaseID=841048
Was für ein Kursspektakel wird sich abspielen ?
Meinungen bitte und Grüße
Gädda
Livekonferenz aus NY-City-und die ganze Welt wartet gespannt auf die HBV Daten.
wünsche allen investierten viel Glück
Und ab wann und wie schnell könnten sie in die Klinik? Hast du schon börsennotierte crisperunternehmen ausgemacht? Fließt ja ne Menge Kohle in den Sektor..
Gruss Macos
http://finance.yahoo.com/news/...r-earnings-conference-012918049.html
ja die Aussichten für ARWR sind einfach gewaltig - was aber zählt ist die Realität.
Der Kurs wird sicher seit einiger Zeit manipuliert durch die shorts - da versuchen halt welche bevor sie long gehen noch ARWR über eine zusätzliche Finanzierung billig an Aktien zu kommen.
Man wird sehen ob da noch mal eine Attacke kommen kann ich hoffe nicht.
Die veröffentlichten Zahlen waren so überaschend gut, daß man ja vermuten könnte die hätten schon mit einem Partner die ARC520 Versuche gemacht ?? Vielleicht steckt Novartis dahinter und die haben evtl. auch bei den Tests mit den 9 Schimpansen geholfen....
Ich kann mir nicht vorstellen, daß es am 24.09 keine großen Ergebnisse geben wird.
Wer trommelt die HBV Welt zusammen und bringt nichts auf den Tisch.
Die Crisper-cash9 Technik kenne ich nicht so gut. Ethik wird wohl aus dem Weg geräumt. Wenn es was medizinisch Sinnvolles zu machen gibt dann macht der Mesch das früher oder später.
Was aber ist mit der Sicherheit ? da gabs ja auch die große Delle bei RNAi und ohne hätte ARWR damals nicht Miros Bio von Roche übernommen - man wird sehen. Im übrigen glaube ich das der große Vorteil von ARWR die Verwendung von DPC und deren Teilchengröße ist - ALNY hat da ja gerade Probleme.
Welche Infoquellen hast du so, ich sehe ständig bei Yahoo und IV nach und bin ganz froh wenn man mal was in deutsch lesen kann.
Gruß
gädda
Gruß
21.11.2011 - 16:33 Uhr
Das Pharma- und Biotechnologieunternehmen Gilead Science übernimmt für rund elf Milliarden Dollar Pharmasset. Nach einem rasanten Kursanstieg in den letzten Monate packt die Pharmasset-Aktie heut noch mal 90 Prozent drauf. Die Gilead-Aktie verliert hingegen deutlich.
http://www.deraktionaer.de/aktie/...appt-sich-pharmasset-17414579.htm
Der HBV Markt soll 2,5 mal so groß sein????
Und HBV ist das 1te Ziehl aus der stetig wachsenden Pipline
Sonnst nur die CC..da sind ja immer eine Menge Infos drin..alles andere ist halt Speku und erwartung..
Gruss
Wer trommelt die HBV Welt zusammen und bringt nichts auf den Tisch.
sehe ich auch so..aber man weiß ja was manchmal passiert, wenn man zu hohe erwartungen hat. ich lass mich überraschen..
vielleicht tüfteln sie ja auch schon mit Gilead an einer Kur(Toll Like Rezeptor+ARC-520)
Spekulationen gibt es genug, mal sehn was kommt :-)))
Gruss
PASADENA, Calif.--(BUSINESS WIRE)-- Arrowhead Research Corporation (NASDAQ: ARWR), a biopharmaceutical company developing targeted RNAi therapeutics, today announced that it will host an analyst day in New York on September 24, 2015, with a presentation starting at 11:00 a.m. EDT to discuss ARC-520, its candidate for the treatment of chronic hepatitis B infection. During the event, Arrowhead management and a panel of key opinion leaders will discuss results of the Heparc-2001 Phase 2a study and results of a nonclinical study conducted in 9 chimpanzees chronically infected with hepatitis B virus.
Guest speakers at the event include:
Robert G. Gish, M.D.
Medical Director, Hepatitis B Foundation
Clinical Professor of Medicine, University of Nevada, Las Vegas
Senior Medical Director, St. Joseph's Hospital and Medical Center
Clinical Professor of Medicine (Consultant), Stanford Hospital and Medical Center
Stephen A. Locarnini, BSc(Hons), PhD, MBBS, FRC(Path)
Head of Research and Molecular Development, Victorian Infectious Diseases Reference Laboratory
Director of WHO Collaborating Centre for Virus Reference and Research
Robert E. Lanford, PhD
Director of Southwest National Primate Research Center (SNPRC)
Scientist, Virology and Immunology and SNPRC, Texas BioMedical Research Institute
A live and archived version of the webcast, including presentation slides, will be available on the events section of the Company's website at ir.arrowheadresearch.com/events.cfm. To access an audio only version of the live presentation, dial 855-215-6159 toll-free from the U.S. or 315-625-6887 for international callers and enter Conference ID 19541930.
http://ir.arrowheadresearch.com/releasedetail.cfm?ReleaseID=930973
Data to be presented September 27 at the European Cancer Conference 2015 in Vienna
PASADENA, Calif.--(BUSINESS WIRE)-- Arrowhead Research Corporation (NASDAQ: ARWR), a biopharmaceutical company developing targeted RNAi therapeutics, today announced that it has nominated ARC-HIF2 as its first therapeutic candidate delivered using a new Dynamic Polyconjugate™ (DPC™) designed to target tissues outside of the liver. Arrowhead believes that ARC-HIF2, which uses RNA interference to silence transcription factor hypoxia-inducible factor 2α (HIF-2α), is a promising new candidate for the treatment of clear cell renal cell carcinoma (ccRCC). The company will present preclinical data at the European Cancer Congress 2015 (ECC2015) in Vienna on September 27 in a session starting at 16:45 CEST. In a poster titled "HIF-2α targeting with a novel RNAi delivery platform as therapy for renal cell carcinoma," (abstract #353), Arrowhead scientists will show data suggesting that HIF-2α inhibition through RNA interference may significantly impact late stage ccRCC progression. The company is in the process of manufacturing scale up to allow for initiation of IND-enabling studies. Timing for anticipated regulatory submission will be announced in the future.
"Preclinical data using our new extrahepatic DPC™ delivery system has been very promising. We think the ability to target tissues outside of the liver, including tumors, opens additional opportunities for Arrowhead to develop differentiated RNAi-therapeutics that address numerous diseases without adequate treatment options," said Christopher Anzalone, Ph.D., Arrowhead's president and chief executive officer. "This is an important milestone for Arrowhead and we look forward to continued development of the DPC™ delivery platform and product candidates based on it."
ARC-HIF2 is designed to inhibit the production of HIF-2α, which has been linked to tumor progression and metastasis in ccRCC. ARC-HIF2 employs a novel extrahepatic-targeted DPC™ that comprises a membrane active polymer to promote RNAi trigger endosomal release, an active ligand that targets the DPC™ to tumor cells, reversible masking to prevent polymer activity prior to cellular uptake, and an RNAi trigger to HIF-2α conjugated directly to the DPC™.
Using ARC-HIF2 in a preclinical ccRCC tumor model, mice treated with weekly injections led to greater than 80% knockdown of HIF-2α mRNA in tumors. Furthermore, tumors from treated mice exhibited statistically significant reductions in size and weight, extensive tumor cell death, reduction in the tumor-expressed VEGF-A biomarker, and destruction of the blood vessels feeding the tumors.
Therapies for metastatic ccRCC including agents that target the VEGF/VEGFR or mTor signaling pathways, which are validated cancer targets, have become the standard-of-care and have improved patient outcomes. However, since emergence of resistance to these agents is common, novel therapies targeting alternative pathways are needed for patients with resistant tumors. Arrowhead believes that HIF2α is an attractive target for intervention because over 90% of ccRCC tumors express a mutant form of the Von Hippel-Landau protein that is unable to degrade HIF-2α, leading to its accumulation during tumor hypoxia and promoting tumor growth.
An abstract of the data to be presented at ECC2015 is available on the conference website at www.europeancancercongress.org/.
http://ir.arrowheadresearch.com/releasedetail.cfm?ReleaseID=931212
https://de.wikipedia.org/wiki/Bevacizumab
Using ARC-HIF2 in a preclinical ccRCC tumor model, mice treated with weekly injections led to greater than 80% knockdown of HIF-2α mRNA in tumors. Furthermore, tumors from treated mice exhibited statistically significant reductions in size and weight, extensive tumor cell death, reduction in the tumor-expressed VEGF-A biomarker, and destruction of the blood vessels feeding the tumors.
http://ir.arrowheadresearch.com/releasedetail.cfm?ReleaseID=931212
kommen eventuel Daten zum 14 Oktober???
http://www.investorvillage.com/...mn=4749&pt=msg&mid=15358234
ISIS hat zwei Kandidaten für dieses Ziel und wohl eine Vereinbarung mit Alnylam, das sie das Ziel nicht angehen???
Per the terms of the new agreement, Alnylam and Isis are forming an IP cross-license with reciprocal economic terms on four therapeutic targets, where each company obtains exclusive license rights to two therapeutic programs. Alnylam is granting Isis an exclusive, royalty-bearing license to its chemistry, RNA-targeting mechanism and target-specific IP for oligonucleotide therapeutics against two targets: Factor XI and apolipoprotein (a) - or Apo(a). Isis is currently developing an investigational antisense drug toward Factor XI for the prevention of thrombosis. ISIS-FXIRx is currently in Phase 2 clinical development. Isis is also currently developing an investigational antisense drug targeting Apo(a) to treat cardiovascular disease. ISIS-APO(a)Rx is currently in a Phase 2 clinical trial. In exchange, Isis is granting Alnylam an exclusive, royalty-bearing license to its chemistry, RNA-targeting mechanism and target-specific IP for oligonucleotide therapeutics against two targets: antithrombin (AT) and aminolevulinic acid synthase-1 (ALAS-1). Alnylam is currently developing an investigational RNAi therapeutic targeting AT for the treatment of hemophilia and rare bleeding disorders. ALN-AT3 is currently in a Phase 1 clinical trial enrolling hemophilia subjects. Alnylam is also currently developing an investigational RNAi therapeutic targeting ALAS-1 for the treatment of hepatic porphyrias, including acute intermittent porphyria (AIP); Alnylam has just filed a clinical trial application (CTA) to begin a Phase 1 clinical trial with ALN-AS1.
http://investors.alnylam.com/releasedetail.cfm?ReleaseID=890484
Gruss Macos
Arrowhead Presents Overview of Its Broad RNAi Delivery Platform and Introduces New Subcutaneously Administered Format
http://ir.arrowheadresearch.com/releasedetail.cfm?ReleaseID=936589
"Our ability to generate RNAi drugs for a wide variety of indications has expanded dramatically with our advances in delivery," said Chris Anzalone, Ph.D., Arrowhead's president and chief executive officer. "We currently have two drugs in the clinic, ARC-520 and ARC-AAT, that validate our capabilities in IV liver delivery and provide additional confidence in follow-on candidates ARC-F12 and ARC-521. We are now able to deliver to tumors, as exemplified by ARC-HIF2, and report data today on subcutaneous administration for liver delivery with ARC-LPA. These capabilities provide broad opportunities to fight diverse diseases and enable us to drive an aggressive pipeline."
Angehängte Grafik:
pipeline-102015-small.png (verkleinert auf 63%)
pipeline-102015-small.png (verkleinert auf 63%)