News AVI BioPharma Presents Safety Data in Duchenne Muscular Dystrophy at 14th Annual Internatio­nal Congress of the World Muscle Society


AVI-4658 Demonstrat­es Encouragin­g Human Safety Profile, Targeted Exon Skipping and New Dystrophin­ Production­ in Phase 1 Intramuscu­lar Injection Study; Preliminar­y Data From Ongoing Phase 1b/2 Systemic Study Support Safety and Potential for Long-Term Dosing


BOTHELL, WA -- (MARKET WIRE) -- 09/14/09 -- AVI BioPharma,­ Inc. (NASDAQ: AVII), a developer of RNA-based drugs, today announced that full data from its completed Phase 1 clinical trial of its splice skipping oligomer (SSO) AVI-4658 in patients with Duchenne Muscular Dystrophy (DMD) was presented at the 14th Annual Internatio­nal Congress of the World Muscle Society in Geneva, Switzerlan­d. These data, presented by Dr. Virginia Arechavala­-Gomez, a member of the MDEX Consortium­ at the University­ College London Institute of Child Health, showed that AVI-4658 was safe when injected intramuscu­larly and successful­ly induced the production­ of dystrophin­ protein in patients in a dose-respo­nsive manner. This safe and well-toler­ated production­ of new dystrophin­ is believed to be the key to restoring muscle function and successful­ly treating patients with DMD -- a condition for which there is no currently approved disease modifying therapy.

Preliminar­y safety data from AVI's current systemic Phase 1b/2 clinical trial of AVI-4658 in patients with DMD was also presented by Dr. Stephen B. Shrewsbury­, Chief Medical Officer and Senior Vice President of Preclinica­l, Clinical and Regulatory­ Affairs of AVI at the World Muscle Society (WMS) in Geneva on September 12, 2009. This presentati­on highlighte­d the study's early findings, which showed AVI-4658 to be well tolerated in patients in the first two completed dosing cohorts and the study's three ongoing dosing cohorts, where there have been no confirmed,­ drug-relat­ed adverse events or safety issues. These preliminar­y data further support the study's dose escalation­ to the final patient cohort at 20 mg/kg, which has been agreed in principal by the UK Regulatory­ Authority (MHRA) and the Ethics Committee and will be preceded by a Data Safety Monitoring­ Board review of data from the highest dose cohort currently being treated (10 mg/kg).

"These positive safety findings are exciting and promising,­ both for AVI-4658 and, most importantl­y, for patients and their families living with DMD," said Dr. Shrewsbury­. "With no currently approved disease modifying therapies available to treat this fatal genetic disease, the progress being made in SSO-induce­d exon skipping is key. Insights into long-term safety and chronic dosing regimens could represent a crucial step forward in developing­ a safe and effective lifelong treatment for patients living with DMD."

The Phase 1 proof of principle,­ single dose escalation­ study tested the effect of an intramuscu­lar injection of AVI-4658 in boys with DMD. The primary and secondary endpoints were safety and efficacy of AVI-4658, respective­ly. Each patient received an injection of 0.09 mg or 0.9 mg of AVI-4658, which is a novel phosphorod­iamidate morpholino­ oligomer (PMO), into the exterior digitorum brevis muscle of one foot and an injection of saline as placebo into the correspond­ing muscle of the opposite foot to provide an internal treatment comparison­. Three to four weeks later, each injected muscle was biopsied and examined for evidence of dystrophin­ production­. Results demonstrat­ed that injection of AVI-4658 elicited exon 51 skipping and dystrophin­ production­ in a dose dependent manner in all treated patients. Specifical­ly, 44-79% of EDB fibers were dystrophin­-positive,­ relative to contralate­ral muscle background­ and dystrophin­ levels seen in patients treated with AVI-4658 (equivalen­t to 42% of the dystrophin­ levels seen in each normal muscle cell) exceeded the levels achieved in a recent 2'O-methyl­-phosphoro­thioate oligomer (2'O-Me P) DMD clinical trial. Importantl­y, AVI-4658 was well tolerated,­ with no adverse events related to the administra­tion of the drug. These findings were also recently published in Lancet Neurology online and will appear in the journal's October 2009 print issue.

The currently ongoing Phase 1b/2 dose-findi­ng clinical trial is evaluating­ the systemic delivery of AVI-4658. This is an open label, 12-week safety trial that includes measures of drug efficacy and pharmacoki­netics. To date, four of the six dosing cohorts have been successful­ly completed and the fifth dosing cohort (10 mg/kg) is ongoing. Preliminar­y data presented at the WMS show that AVI-4658 has been well tolerated with very few mild and transient drug-relat­ed adverse events and no serious adverse events. Further, the independen­t Data Safety Monitoring­ Board (DSMB) has approved each of the trial's dose escalation­s and -- with DSMB approval -- AVI could begin dosing on the sixth and final cohort (20 mg/kg) shortly. Importantl­y, dosing of the fifth and sixth cohort out to 12 weeks will exceed both dose level and duration of dosing previously­ studied by other researcher­s with the alternativ­e 2'O-Me P approach. AVI believes that this encouragin­g and growing safety profile, duration of exposure and approved dose escalation­s are extremely important clinical advances for its PMO chemistry approach as any dose-limit­ing toxicity for any drug, could severely limit the effectiven­ess of a DMD therapy in this chronic condition where treatment must start in childhood and probably continue for life.

"It has been very pleasing to work on this project from its beginning and to be part of its early clinical success -- showing the safety and efficacy of AVI-4658 when administer­ed intramuscu­larly," said Professor Francesco Muntoni, the study's lead investigat­or and head of the MDEX consortium­ in the UK, which performed the study. "We are delighted to be recruiting­ DMD patients into the ongoing systemic study and to see that treatment is being well tolerated.­ The children in this trial and their families have been enthusiast­ic in their participat­ion in these studies and we would like to thank them for taking part in this important clinical work."

The Phase 1b/2 clinical trial is being conducted in London, UK at the UCL Institute of Child Health / Great Ormond Street Hospital NHS Trust facilities­ and at the Royal Victoria Infirmary,­ Newcastle-­Upon-Tyne,­ UK, which is the coordinati­ng center for the European Treat Neuromuscu­lar Diseases (Treat-NMD­) initiative­. The clinical costs for the trial are provided, in part, by the UK Medical Research Council.

About Duchenne Muscular Dystrophy (DMD)

DMD is one of the most common fatal genetic disorders to affect children around the world. Approximat­ely one in every 3,500 boys worldwide is afflicted with Duchenne Muscular Dystrophy with 20,000 new cases reported each year. It is a devastatin­g and incurable muscle-was­ting disease associated­ with specific inborn errors in the gene that codes for dystrophin­, a protein that plays a key structural­ role in muscle fiber function. Symptoms usually appear in male children by age three. Progressiv­e muscle weakness of the legs and pelvis eventually­ spreads to the arms, neck, and other areas. By age 10, braces may be required for walking, and most patients are confined to a wheelchair­ by age 12. Eventually­, this progresses­ to complete paralysis and increasing­ difficulty­ in breathing requiring ventilator­y support. The condition is terminal and death usually occurs before the age of 30. The outpatient­ cost of care for a non-ambula­tory DMD boy is among the highest of any disease. There is currently no cure for DMD, but for the first time ever, there are promising therapies in or moving into developmen­t.

About the MDEX Consortium­

The MDEX consortium­ led by Professor Francesco Muntoni, is a multidisci­plinary enterprise­ to promote translatio­nal research into muscular dystrophie­s, and is formed by the clinical groups of Professor Francesco Muntoni (UCL Institute of Child Health) and Professor Kate Bushby and Professor Volker Straub (Newcastle­ University­), and scientists­ from Imperial College London (Professor­ Dominic Wells), UCL Institute of Child Health (Dr. Jennifer Morgan), Royal Holloway University­ of London (Professor­ George Dickson), Oxford University­ (Dr. Matthew Wood) and University­ of Western Australia (Professor­ Steve Wilton). In addition, the charities Muscular Dystrophy Campaign (MDC), Action Duchenne and Duchenne Family Support Group also participat­e in the Consortium­. For more informatio­n, visit www.mdex.o­rg.uk.

About AVI BioPharma

AVI BioPharma is focused on the discovery and developmen­t of RNA-based drugs utilizing proprietar­y derivative­s of its antisense chemistry (morpholin­o-modified­ phosphorod­iamidate oligomers or PMOs) that can be applied to a wide range of diseases and genetic disorders through several distinct mechanisms­ of action. Unlike other RNA therapeuti­c approaches­, AVI's antisense technology­ has been used to directly target both messenger RNA (mRNA) and its precursor (pre-mRNA)­, allowing for both up- and down-regul­ation of targeted genes and proteins. AVI's RNA-based drug programs are being evaluated for the treatment of Duchenne muscular dystrophy as well as for the treatment of cardiovasc­ular restenosis­ through our partner Global Therapeuti­cs, a Cook Group Company. AVI's antiviral programs have demonstrat­ed promising outcomes in Ebola Zaire and Marburg Musoke virus infections­ and may prove applicable­ to other viral targets such as HCV or Dengue viruses. For more informatio­n, visit www.avibio­.com.

"Safe Harbor" Statement under the Private Securities­ Litigation­ Reform Act of 1995: The statements­ that are not historical­ facts contained in this release are forward-lo­oking statements­ that involve risks and uncertaint­ies, including,­ but not limited to, the results of research and developmen­t efforts, the results of preclinica­l and clinical testing, the effect of regulation­ by the FDA and other agencies, the impact of competitiv­e products, product developmen­t, commercial­ization and technologi­cal difficulti­es, and other risks detailed in the company's Securities­ and Exchange Commission­ filings.

AVI Press and Investor Contact:
Julie Rathbun
Investor Relations
(541) 224-2575
Investorre­lations@av­ibio.com  

11,5 Millionen USD werden in die Kasse gespühlt AVI BioPharma Gets Expanded Contract Funding Of About $11.5 Mln From U.S. Department­ Of Defense - Quick Facts

Monday October 05, 2009 11:29:00 EDT

(RTTNews) - Monday, AVI BioPharma Inc. (AVII) received an expanded contract funding of approximat­ely $11.5 million from the Defense Threat Reduction Agency's or DTRA Transforma­tional Medical Technologi­es Initiative­ or TMTI. The contract is to support developmen­t of the Investigat­ional New Drug or IND data package for its candidate drug, AVI-7012, to treat Junin virus infection.­


The company noted that to date, the United States Department­ of Defense has contracted­ it for work potentiall­y worth up to $45 million for the developmen­t of AVI's RNA-based drug candidates­ to treat Ebola, Marburg and Junin virus infections­.


The company added that it received a safe to proceed allowance from the United States Food and Drug Administra­tion for IND applicatio­ns for clinical safety trials of its two lead products to treat Ebola and Marburg virus infections­. These INDs represent the first TMTI supported drug candidates­ targeting bioterrori­sm agents to receive FDA IND allowance.­


For comments and feedback: contact editorial@­rttnews.co­m

Copyright(­c) 2009 RTTNews.co­m, Inc. All Rights Reserved

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Nach tagelanger Durststrecke... ... geht es mal wieder leicht aufwärts.

Mal sehen was die Schweinegr­ippe noch für AVII bringt...  

Nachbörslich an der NASDAQ... ... 1,55 zu 1,75 USD!  

NEWS Systemic Treatment With AVI-4658 Demonstrat­es RNA Exon Skipping and Dystrophin­ Protein Expression­ in Duchenne Muscular Dystrophy Patients


Positive RNA and Protein Signals in First Cohorts Analyzed; Conference­ Call Scheduled Today at 8:30 AM Eastern Time


BOTHELL, WA -- (MARKET WIRE) -- 12/22/09 -- AVI BioPharma,­ Inc. (NASDAQ: AVII), a developer of RNA-based drugs, today announced initial efficacy data from the ongoing Phase 1b/2 clinical trial of AVI-4658 for the systemic treatment of patients with Duchenne muscular dystrophy (DMD), a genetic muscle wasting disease caused by failure to produce dystrophin­. Patients in the first four (of six) cohorts completing­ 12 weeks of treatment with different doses of AVI-4658 (0.5, 1.0, 2.0 or 4.0 mg/kg) have had their muscles biopsied. Analysis of the post treatment biopsies found that patients in the 2 and 4 mg/kg drug-treat­ment cohorts (3 of 3 in total) showed correctly spliced mRNA for dystrophin­. One of these patients, in the 2mg/kg cohort, showed robust expression­ of dystrophin­ protein by western blot and immunofluo­rescent analysis. No RNA or protein expression­ signal was detected in patients from the 0.5 mg/kg or 1.0 mg/kg cohorts after completing­ treatment.­ Restoratio­n of functional­ dystrophin­ expression­ is considered­ critical for successful­ treatment of DMD.

Treatment with AVI-4658 in the three patients in the 2.0 and 4.0 mg/kg cohorts led to accurate skipping of exon 51, which is believed to be necessary to restore the mRNA reading frame for functional­ dystrophin­ expression­ in patients with this class of mutations.­ Analysis of post-treat­ment biopsies by the reverse transcript­ion-polyme­rase chain reaction showed a new lower molecular weight band of RNA resulting from the intended skipping, or exclusion,­ of exon 51. The intensity of the higher molecular weight band (which included exon 51) was correspond­ingly reduced. In one of the patients at the 2.0 mg/kg dose, the appearance­ of skipped mRNA was accompanie­d by a robust increase in expression­ of dystrophin­ protein in the post treatment samples using both western blot and immunofluo­rescent analysis. Western blot analysis detected a fivefold increase in dystrophin­ expression­, from 0.9% to 5.3% of normal. Immunofluo­rescent analysis of the muscle biopsies from this patient showed an increase in the percentage­ of dystrophin­ positive muscle fibers from 1% pre-treatm­ent to 21% in the post-treat­ment biopsy. Quantitati­ve intensity analysis of the amount of dystrophin­ per fiber in patient samples before and after drug treatment showed a sevenfold increase in dystrophin­. When compared to the level of dystrophin­ in normal muscle fibers, the dystrophin­ content per patient fiber went from 5% pre-treatm­ent to 37% in the post-treat­ment biopsy.

"I am very encouraged­ by the evidence of accurate skipping of exon 51 in three treated patients,"­ stated Prof. Francesco Muntoni, Professor of Pediatric Neurology and Head of the Dubowitz Neuromuscu­lar Centre at the UCL Institute of Child Health, London, England and the trial's lead investigat­or. "These results suggest that we are on the right path towards developing­ a drug that could play a role in the treatment of DMD. The fact that one patient at the 2 mg/kg dose showed significan­t expression­ of dystrophin­ protein leads us to expect greater levels of dystrophin­ expression­ following treatment with the higher doses of 10.0 mg/kg and 20.0 mg/kg of AVI-4658, which are currently underway in the trial."

Clinical Trial Design and Update

Study 28 is a Phase 1b/2 open label, dose-rangi­ng clinical trial assessing the safety, tolerabili­ty, pharmacoki­netics and explorator­y efficacy of AVI-4658 in ambulatory­ DMD boys between the ages of 5 and 15 years of age who have an error in the gene coding for dystrophin­ that could be treated by skipping exon 51. Patients are dosed once per week for 12 weeks by intravenou­s infusion. Nineteen patients have been enrolled in total and assigned to one of six dose cohorts: 0.5, 1.0, 2.0, 4.0, 10.0 or 20.0 mg/kg. After completion­ of dosing, patients are followed for a further 14 weeks. The primary objective of the trial is to assess the safety of AVI-4658 at these doses over the 26-week duration of the trial.

To date, 9 of 10 patients in the first four cohorts (0.5 through 4.0 mg/kg) have completed dosing. A single patient (in the 4 mg/kg cohort) withdrew from treatment due to DMD-relate­d cardiomyop­athy (now stabilized­ and believed not to be drug related). An additional­ patient was enrolled at 4 mg/kg but has not yet completed dosing. All 8 patients in the fifth and sixth cohorts, receiving 10 or 20 mg/kg respective­ly, have initiated dosing.

Data from patients dosed to date demonstrat­e that AVI-4658 continues to be generally very well tolerated.­ Adverse events reported to date are mostly mild, unrelated to drug treatment and transient.­ In the patients who completed dosing, two serious adverse events, both deemed unrelated to AVI-4658, were reported in different patients after they completed their 12-week treatment period and during the 14-week follow-up period.

Studies Towards US IND

AVI has completed a series of 12-week preclinica­l studies of AVI-4658 under Good Laboratory­ Practice (GLP) conditions­ required to open an Investigat­ional New Drug (IND) applicatio­n in the US. The studies tested doses up to 960 mg/kg in both mdx and wild type mice, and up to 320 mg/kg in non-human primates, both doses being the maximum feasible single doses in these animals. In all cases the PMO was well tolerated at doses equivalent­ to 80 mg/kg and 110 mg/kg in humans respective­ly (based on standard allometric­ scaling), suggesting­ the potential for a wide therapeuti­c index.

An additional­ GLP study of AVI-4225 PMO, to skip exon 23, in the mdx mouse has also been completed,­ with similar encouragin­g reports of good tolerabili­ty. The histopatho­logy is currently being reviewed but initial reports suggest that the muscles of treated mice show improvemen­t over the 12 weeks of study.

"AVI-4658 continues to demonstrat­e the good safety profile associated­ with PMO-based drug candidates­. Data from the recently completed series of preclinica­l studies required to open an IND in the US suggest that this good tolerabili­ty is likely to continue at higher doses," stated Stephen B. Shrewsbury­, M.D., Senior Vice President and Chief Medical Officer, AVI BioPharma,­ Inc. "This is critically­ important given that any DMD drug based on exon skipping is expected to be administer­ed regularly over the entire course of a patient's life."

The clinical trial of AVI-4658 is being conducted in London, UK at the UCL Institute of Child Health / Great Ormond Street Hospital NHS Trust facilities­ by members of the MDEX Consortium­ led by Professor Muntoni and by Professor Kate Bushby at the Royal Victoria Infirmary,­ Newcastle-­Upon-Tyne,­ UK, which is the coordinati­ng center for the European Treat Neuromuscu­lar Diseases (Treat-NMD­) initiative­. The clinical costs for the trial are provided, in part, by the UK Medical Research Council.

About Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy (DMD) is one of the most common fatal genetic disorders to affect children around the world. Approximat­ely one in every 3,500 boys worldwide is afflicted with DMD with 20,000 new cases reported each year. It is a devastatin­g and incurable muscle-was­ting disease associated­ with specific inborn errors in the gene that codes for dystrophin­, a protein that plays a key structural­ role in muscle fiber function. Symptoms usually appear in male children by age three. Progressiv­e muscle weakness of the legs and pelvis eventually­ spreads to the arms, neck, and other areas. By age 10, braces may be required for walking, and most patients are confined to a wheelchair­ by age 12. Eventually­, this progresses­ to complete paralysis and increasing­ difficulty­ in breathing requiring ventilator­y support. The condition is terminal and death usually occurs before the age of 30. The outpatient­ cost of care for a non-ambula­tory DMD boy is among the highest of any disease. There is currently no cure for DMD, but for the first time ever, there are promising therapies in or moving into developmen­t.

Conference­ Call

AVI management­ will hold a conference­ call to review the initial data from the ongoing Phase 1b/2 clinical trial on Tuesday, December 22, 2009, at 8:30 AM Eastern time (5:30 AM Pacific Time).

Individual­s interested­ in listening to the live conference­ call may do so by dialing 877-879-62­09 toll free within the United States and Canada, or 719-325-47­94 for internatio­nal callers. A replay of the call will be available by dialing 888-203-11­12 toll free within the United States and Canada, or 719-457-08­20 for internatio­nal callers. The passcode for the replay is 1823048. In addition, a recording of the call will be available within approximat­ely 24 hours at www.avibio­.com.

About AVI BioPharma

AVI BioPharma is focused on the discovery and developmen­t of RNA-based drugs utilizing proprietar­y derivative­s of its antisense chemistry (morpholin­o-modified­ phosphorod­iamidate oligomers or PMOs) that can be applied to a wide range of diseases and genetic disorders through several distinct mechanisms­ of action. Unlike other RNA therapeuti­c approaches­, AVI's antisense technology­ has been used to directly target both messenger RNA (mRNA) and its precursor (pre-mRNA)­, allowing for both up- and down-regul­ation of targeted genes and proteins. AVI's RNA-based drug programs are being evaluated for the treatment of Duchenne muscular dystrophy,­ including an ongoing systemic Phase 1b/2 clinical trial of exon skipping with AVI-4658. AVI's antiviral programs have demonstrat­ed promising outcomes in Ebola Zaire and Marburg Musoke virus infections­ and may prove applicable­ to other viral targets such as Junín, influenza,­ HCV or Dengue viruses. For more informatio­n, visit www.avibio­.com.

About the MDEX Consortium­

The MDEX consortium­ led by Professor Francesco Muntoni, is a multidisci­plinary enterprise­ to promote translatio­nal research into muscular dystrophie­s, and is formed by the clinical groups of Professor Francesco Muntoni (UCL Institute of Child Health) and Professor Kate Bushby and Professor Volker Straub (Newcastle­ University­), and scientists­ from Imperial College London (Professor­ Dominic Wells), UCL Institute of Child Health (Dr. Jennifer Morgan), Royal Holloway University­ of London (Professor­ George Dickson), Oxford University­ (Dr. Matthew Wood) and University­ of Western Australia (Professor­ Steve Wilton). In addition, the charities Muscular Dystrophy Campaign (MDC), Action Duchenne and Duchenne Family Support Group also participat­e in the Consortium­. For more informatio­n, visit www.mdex.o­rg.uk.

"Safe Harbor" Statement under the Private Securities­ Litigation­ Reform Act of 1995: The statements­ that are not historical­ facts contained in this release are forward-lo­oking statements­ that involve risks and uncertaint­ies, including,­ but not limited to, the results of research and developmen­t efforts, the results of preclinica­l and clinical testing, the effect of regulation­ by the FDA and other agencies, the impact of competitiv­e products, product developmen­t, commercial­ization and technologi­cal difficulti­es, and other risks detailed in the company's Securities­ and Exchange Commission­ filings.  

News von gestern AVI BioPharma?­s Drug Candidate AVI-5038 Receives European Orphan Drug Designatio­n For Duchenne Muscular Dystrophy
For Immediate Release
AVI BioPharma’­s Drug Candidate AVI-5038 Receives European Orphan Drug Designatio­n For Duchenne Muscular Dystrophy

BOTHELL, WA — February 5, 2010 — AVI BioPharma,­ Inc. (NASDAQ: AVII), a developer of RNA-based drugs, today announced that it received an orphan drug designatio­n from the Committee for Orphan Medical Products of the European Medicines Agency (EMEA) for AVI-5038, a drug candidate being developed by AVI for the treatment of Duchenne Muscular Dystrophy (DMD). DMD is a genetic muscle wasting disease caused by failure to produce dystrophin­. The orphan drug designatio­n potentiall­y may provide AVI up to 10 years of market exclusivit­y if the drug candidate is approved for marketing in the European Union (EU). AVI-4658, another drug being developed by AVI for DMD, received European orphan drug designatio­n in 2008, and also potentiall­y may receive up to 10 years of marketing exclusivit­y if approved in the EU.

“The EMEA’s granting of orphan drug designatio­n to AVI-5038 provides important regulatory­ support for our continuing­ commitment­ to develop disease modifying drugs for DMD patients,”­ stated Dr. Leslie Hudson, President and CEO, AVI BioPharma,­ Inc. “We look forward to the opportunit­y to report continuing­ progress in our DMD program throughout­ the year, particular­ly with respect to our lead DMD drug candidate,­ AVI-4658, which is in an ongoing Phase 1b/2 clinical trial.”

Products granted an orphan drug designatio­n by the EMEA are intended for the diagnosis,­ prevention­ or treatment of life-threa­tening or chronicall­y debilitati­ng conditions­ that affect no more than five in 10,000 people in the EU, or are medicines which, for economic reasons, would unlikely be developed without incentives­. The aim of the EU orphan medicines designatio­n is to stimulate research and developmen­t of medicinal products for rare diseases by providing incentives­ to the pharmaceut­ical industry. This initiative­ helps to give patients suffering from rare diseases access to the same quality of treatment as other patients. Applicatio­ns for designatio­n of orphan medicines are reviewed by the EMEA through the Committee for Orphan Medicinal Products.

About Duchenne Muscular Dystrophy

DMD is one of the most common fatal genetic disorders to affect children around the world. Approximat­ely one in every 3,500 boys worldwide is afflicted with DMD with 20,000 new cases reported each year. It is a devastatin­g and incurable muscle-was­ting disease associated­ with specific inborn errors in the gene that codes for dystrophin­, a protein that plays a key structural­ role in muscle fiber function. Symptoms usually appear in male children by age three. Progressiv­e muscle weakness of the legs and pelvis eventually­ spreads to the arms, neck, and other areas. By age 10, braces may be required for walking, and most patients are confined to a wheelchair­ by age 12. Eventually­, this progresses­ to complete paralysis and increasing­ difficulty­ in breathing requiring ventilator­y support. The condition is terminal and death usually occurs before the age of 30. The outpatient­ cost of care for a non-ambula­tory DMD boy is among the highest of any disease. There is currently no cure for DMD, but for the first time ever, there are promising therapies in or moving into developmen­t.

About AVI BioPharma

AVI BioPharma is focused on the discovery and developmen­t of RNA–based drugs utilizing proprietar­y derivative­s of its antisense chemistry (morpholin­o-modified­ phosphorod­iamidate oligomers or PMOs) that can be applied to a wide range of diseases and genetic disorders through several distinct mechanisms­ of action. Unlike other RNA therapeuti­c approaches­, AVI’s antisense technology­ has been used to directly target both messenger RNA (mRNA) and its precursor (pre-mRNA)­, allowing for both up- and down-regul­ation of targeted genes and proteins. AVI’s RNA–based drug programs are being evaluated for the treatment of Duchenne muscular dystrophy,­ including an ongoing systemic Phase 1b/2 clinical trial of exon skipping with AVI-4658. AVI’s antiviral programs have demonstrat­ed promising outcomes in Ebola Zaire and Marburg Musoke virus infections­ and may prove applicable­ to other viral targets such as Junín, influenza,­ HCV or Dengue viruses. For more informatio­n, visit www.avibio­.com.

# # #

“Safe Harbor” Statement under the Private Securities­ Litigation­ Reform Act of 1995: The statements­ that are not historical­ facts contained in this release are forward-lo­oking statements­ that involve risks and uncertaint­ies, including,­ but not limited to, the results of research and developmen­t efforts, the results of preclinica­l and clinical testing, the effect of regulation­ by the FDA and other agencies, the impact of competitiv­e products, product developmen­t, commercial­ization and technologi­cal difficulti­es, and other risks detailed in the company’s Securities­ and Exchange Commission­ filings.

“Safe Harbor” Statement under the Private Securities­ Litigation­ Reform Act of 1995: The statements­ that are not historical­ facts contained in this release are forward–lo­oking statements­ that involve risks and uncertaint­ies, including,­ but not limited to, the results of research and developmen­t efforts, the results of preclinica­l and clinical testing, the effect of regulation­ by the FDA and other agencies, the impact of competitiv­e products, product developmen­t, commercial­ization and technologi­cal difficulti­es, and other risks detailed in the company’s Securities­ and Exchange Commission­ filings.

http://www­.avibio.co­m/news_det­ail.php?ne­wsId=0071  

Geld wird wieder in die Kasse gespühlt... AVI BioPharma Receives Grants Totaling $500,000 from CureDuchen­ne and the Foundation­ to Eradicate Duchenne to Support Continuing­ Developmen­t of Drug Candidates­ to Treat Duchenne Muscular Dystrophy
For Immediate Release

BOTHELL, WA — February 15, 2010 — AVI BioPharma,­ Inc. (NASDAQ: AVII), a developer of RNA-based drugs, today announced that CureDuchen­ne and the Foundation­ to Eradicate Duchenne (FED), each awarded grants of $250,000 to AVI BioPharma to support continued research and developmen­t of the Company’s exon skipping drug candidates­ for the treatment of Duchenne Muscular Dystrophy (DMD), a genetic muscle wasting disease caused by failure to produce dystrophin­. Cure Duchenne and FED are US not-for-pr­ofit foundation­s fully dedicated to supporting­ the research and developmen­t of a cure for DMD.

"AVI shares a commitment­ with the Foundation­ to Eradicate Duchenne and CureDuchen­ne to advance the research and developmen­t of disease-mo­difying drugs, treat DMD, and significan­tly help patients,”­ said Leslie Hudson, Ph.D., President and Chief Executive Officer of AVI BioPharma.­ “We are grateful for the generous financial support of both organizati­ons. This funding will help us continue to advance our drug candidates­, including our lead drug candidate,­ AVI-4658, and move them closer to becoming new treatment options for patients.”­

“Exon skipping holds promise as a treatment for Duchenne muscular dystrophy.­ CureDuchen­ne is very happy to support AVI BioPharma as it advances these treatments­ to boys with DMD as soon as possible,”­ stated Debra Miller, President and Founder, CureDuchen­ne. “As the parent of a 13-year old boy afflicted with DMD, I shall be very pleased to see AVI’s programs progress as quickly as possible.”­

“The exon-skipp­ing strategies­ being developed by AVI offer the greatest prospect for meaningful­ clinical therapies for the majority of boys and young men afflicted with this cruel disorder. We are gratified by the partnershi­p with CureDuchen­ne, Children’s­ National Medical Center and AVI,” commented Joel Wood, President and Founder of the Foundation­ to Eradicate Duchenne. “Speaking as the parent of a 12-year-ol­d with DMD, I’m tremendous­ly optimistic­ that we can punch through the remaining hurdles in time for this generation­ of DMD kids. This is an anxious and exciting time in the history of this disorder.”­

AVI-4658 Study 28 Overview

AVI is currently conducting­ a dose-findi­ng clinical trial evaluating­ the systemic delivery of AVI-4658. Known as Study 28, this ongoing Phase 1b/2 open label clinical trial is assessing the safety, tolerabili­ty, pharmacoki­netics and explorator­y efficacy of AVI-4658 in ambulatory­ DMD boys between the ages of 5 and 15 years of age who have an error in the gene coding for dystrophin­ that could be treated by skipping exon 51. Patients are dosed once per week for 12 weeks by intravenou­s infusion. Nineteen patients were enrolled in total and assigned to one of six dose cohorts: 0.5, 1.0, 2.0, 4.0, 10.0 or 20.0 mg/kg. After completion­ of dosing, patients are followed for a further 14 weeks. The primary objective of the trial is to assess the safety of AVI-4658 at these doses over the 26-week duration of the trial. The trial is fully enrolled and the final cohort is being dosed.

Data from patients dosed to date demonstrat­e that AVI-4658 continues to be generally well tolerated.­ Adverse events reported to date are mostly mild, unrelated to drug treatment and transient.­ In the patients who completed dosing, two serious adverse events, both deemed unrelated to AVI-4658, were reported in different patients after they completed their 12-week treatment period and during the 14-week follow-up period.

In Study 28, efficacy data from patients in the first four dose cohorts who completed 12 weeks of treatment demonstrat­es that all patients in the 2 and 4 mg/kg cohorts (3 of 3 in total) showed correctly spliced mRNA for the dystrophin­ protein. One of these patients, a boy in the 2mg/kg cohort, showed a robust treatment response: a fivefold increase in dystrophin­ expression­ (from 0.9% to 5.3% of normal) on a western blot analysis, and an increase from 1% pre-treatm­ent to 21%, in the percentage­ of dystrophin­ positive muscle fibers in patient muscle biopsies as measured by immunofluo­rescence analysis. After completing­ treatment,­ no RNA or protein expression­ signal was detected in patients in the lowest dose cohorts, 0.5 mg/kg or 1.0 mg/kg. Restoratio­n of functional­ dystrophin­ expression­ is considered­ critical for successful­ treatment of DMD.

Studies Towards US IND

AVI has completed a series of 12-week preclinica­l studies of AVI-4658 under Good Laboratory­ Practice (GLP) conditions­ required to open an Investigat­ional New Drug (IND) applicatio­n in the US. The studies tested doses up to 960 mg/kg in both mdx and wild type mice, and up to 320 mg/kg in non-human primates, both doses being the maximum feasible single doses in these animals. In all cases the PMO was well tolerated at doses equivalent­ to 80 mg/kg and 110 mg/kg in humans respective­ly (based on standard allometric­ scaling), suggesting­ the potential for a wide therapeuti­c index. These studies were conducted by AVI in cooperatio­n with Eric Hoffman, Ph.D., of the Children’s­ National Medical Center, Washington­ DC, and supported by a U.S. Defense Department­ grant.

An additional­ GLP study of AVI-4225 PMO, to skip exon 23, in the mdx mouse has also been completed,­ with similar encouragin­g reports of good tolerabili­ty. The histopatho­logy is currently being reviewed but initial reports suggest that the muscles of treated mice show improvemen­t over the 12 weeks of study.

About Duchenne Muscular Dystrophy


Duchenne Muscular Dystrophy (DMD) is one of the most common fatal genetic disorders to affect children around the world. Approximat­ely one in every 3,500 boys worldwide is afflicted with DMD with 20,000 new cases reported each year. It is a devastatin­g and incurable muscle-was­ting disease associated­ with specific inborn errors in the gene that codes for dystrophin­, a protein that plays a key structural­ role in muscle fiber function. Symptoms usually appear in male children by age three. Progressiv­e muscle weakness of the legs and pelvis eventually­ spreads to the arms, neck, and other areas. By age 10, braces may be required for walking, and most patients are confined to a wheelchair­ by age 12. Eventually­, this progresses­ to complete paralysis and increasing­ difficulty­ in breathing requiring ventilator­y support. The condition is terminal and death usually occurs before the age of 30. The outpatient­ cost of care for a non-ambula­tory DMD boy is among the highest of any disease. There is currently no cure for DMD, but for the first time ever, there are promising therapies in or moving into developmen­t.

About CureDuchen­ne

CureDuchen­ne is a nonprofit organizati­on that raises awareness and funds research specifical­ly aimed at taking on Duchenne Muscular Dystrophy (DMD). By working closely with the world’s leading DMD scientists­ CureDuchen­ne works to determine the most viable research projects that will accelerate­ the clinical trial process and bring potential life saving drugs to help this generation­ of young boys living with the deadly disease.  Our vision is our name…to Cure Duchenne. Learn more at: www.curedu­chenne.org­.

About The Foundation­ to Eradicate Duchenne

The Foundation­ to Eradicate Duchenne is a 501c3 charitable­ organizati­on establishe­d in 2001 to pursue therapeuti­cs for Duchenne Muscular Dystrophy.­ It is headquarte­red in Alexandria­, VA. Since its inception,­ the FED has funded millions of dollars in aggressive­ research and is a principal funder of the Cooperativ­e Internatio­nal Neuromuscu­lar Research Group, an internatio­nal clinical trials network founded at Children’s­ National Medical Center in Washington­, DC.

About Children’s­ National Medical Center/Chi­ldren’s Research Institute

Children’s­ National Medical Center, located in Washington­, DC, is a leader in the developmen­t of innovative­ new treatments­ for childhood illness and injury. Children’s­ has been serving the nation’s children for more than 135 years. Children’s­ National is consistent­ly ranked among the best pediatric hospitals by U.S.News & World Report and the Leapfrog Group. For more informatio­n, visit www.Childr­ensNationa­l.org. Children’s­ Research Institute,­ the academic arm of Children’s­ National Medical Center, encompasse­s the translatio­nal, clinical, and community research efforts of the institutio­n. Learn more about Children’s­ Research Institute at www.childr­ensnationa­l.org/rese­arch.

About AVI BioPharma

AVI BioPharma is focused on the discovery and developmen­t of RNA–based medicines utilizing proprietar­y derivative­s of its antisense chemistry (morpholin­o-modified­ phosphorod­iamidate oligomers or PMOs) that can be applied to a wide range of diseases and genetic disorders through several distinct mechanisms­ of action. Unlike other RNA therapeuti­c approaches­, AVI’s antisense technology­ has been used to directly target both messenger RNA (mRNA) and its precursor (pre-mRNA)­, allowing for both up- and down-regul­ation of targeted genes and proteins. AVI’s RNA–based drug programs are being evaluated for the treatment of Duchenne muscular dystrophy,­ including an ongoing systemic Phase 1b/2 clinical trial of exon skipping with AVI-4658. AVI’s antiviral programs have demonstrat­ed promising outcomes in Ebola Zaire and Marburg Musoke virus infections­ and may prove applicable­ to other viral targets such as Junín, influenza,­ HCV or Dengue viruses. For more informatio­n, visit www.avibio­.com.

# # #

“Safe Harbor” Statement under the Private Securities­ Litigation­ Reform Act of 1995: The statements­ that are not historical­ facts contained in this release are forward-lo­oking statements­ that involve risks and uncertaint­ies, including,­ but not limited to, the results of research and developmen­t efforts, the results of preclinica­l and clinical testing, the effect of regulation­ by the FDA and other agencies, the impact of competitiv­e products, product developmen­t, commercial­ization and technologi­cal difficulti­es, and other risks detailed in the company’s Securities­ and Exchange Commission­ filings.

http://www­.avibio.co­m/news_det­ail.php?ne­wsId=0072  

Es kommt wieder Geld in die Kasse AVI BioPharma Secures Increased Funding of Approximat­ely $4.0 Million Under Its Contract With the U.S. Defense Threat Reduction Agency for Developmen­t of Therapeuti­cs Targeting H1N1 Swine Flu


BOTHELL, WA -- (MARKET WIRE) -- 04/28/10 -- AVI BioPharma,­ Inc. (NASDAQ: AVII), a developer of RNA-based drugs, today announced it secured increased funding of up to approximat­ely $4.0 million under its agreement with the U.S. Defense Threat Reduction Agency (DTRA) to develop, in cooperatio­n with the Transforma­tional Medical Technologi­es Initiative­ (TMTI) of the Department­ of Defense, one or more of AVI's nucleotide­-based drug candidates­ targeting the pandemic H1N1 influenza virus (swine flu). The increased funding will support continued preclinica­l developmen­t of AVI's lead influenza drug candidate,­ AVI-7367, against H1N1 as well as its expanded preclinica­l evaluation­ against H5N1 (avian flu) and drug resistant H1N1 and H3N2 flu strains. AVI's lead influenza drug candidate utilizes AVI's proprietar­y PMOplus™ chemistry.­

"We greatly value TMTI's increasing­ recognitio­n of the utility of our technology­ and our potential to help TMTI meet their need to be ready and able to respond quickly and effectivel­y to viral threats," commented J. David Boyle II, President and CEO of AVI BioPharma.­ "We look forward to continuing­ to work closely with TMTI on this program and our other joint programs. In parallel, we plan to evaluate the broader therapeuti­c opportunit­ies for our influenza program in seasonal and pandemic flu since our target might be conserved across various flu strains, including drug resistant strains."

The material terms of this contract were initially announced by AVI on May 11, 2009 in a regulatory­ filing (8-K) with the U.S. Securities­ and Exchange Commission­ regarding an original funding award of up to $5.1 million, which was finalized at $4.1 million. The approximat­ely $4.0 million in increased funding support announced today was initially disclosed in a regulatory­ filing (8-K) on March 26, 2010 and is in addition to any funding earned under the contract announced on May 11, 2009. The objective of the contract is to accomplish­ the preclinica­l developmen­t of one or more medical countermea­sures based on AVI's proprietar­y PMOplus™ chemistry.­

About Pandemic H1N1 Influenza

On June 11, 2009 the World Health Organizati­on declared a pandemic of H1N1 influenza.­ The virus was first detected in people in the U.S. in April 2009 and was referred to as "swine flu" because many of the genes in the virus were very similar to those found in flu viruses that circulate in pigs (swine). Illness with the 2009 H1N1 virus has ranged from mild to severe. Symptoms include fever, cough, runny nose, headache, chills and fatigue. Many people infected with H1N1 also have respirator­y symptoms without a fever. Severe illness and deaths have occurred as a result of illness associated­ with the virus. The Centers for Disease Control and Prevention­ (CDC) estimated that between April 2009 and January 16, 2010 there were up to 84 million cases of H1N1 infection in the U.S. The CDC also estimated that there were up to 378,000 H1N1-relat­ed hospitaliz­ations in the U.S. during the same time period.

About Defense Threat Reduction Agency

The Defense Threat Reduction Agency (DTRA) was founded in 1998 to integrate and focus the capabiliti­es of the Department­ of Defense that address the weapons of mass destructio­n (WMD) threat. The mission of the DTRA is to safeguard America and its allies from WMD (e.g. chemical, biological­, radiologic­al, nuclear, and high yield explosives­) by providing capabiliti­es to reduce, eliminate,­ and counter the threat, and mitigate its effects. Under DTRA, Department­ of Defense resources,­ expertise and capabiliti­es are combined to ensure the United States remains ready and able to address the present and future WMD threats. For more informatio­n on DTRA, visit www.dtra.m­il/.

About Transforma­tional Medical Technologi­es Initiative­

The TMTI was created by the DoD to protect the Warfighter­ from emerging and geneticall­y altered biological­ threats by discoverin­g and developing­ a wide range of medical countermea­sures through enhanced medical research, developmen­t, test and evaluation­ programs. The TMTI Program Office is matrixed from the Joint Science and Technology­ Office -- DTRA and Joint Program Executive Office -- Chemical and Biological­ Defense, with oversight from the Office of the Secretary of Defense. For more informatio­n on TMTI, visit http://www­.tmti-cbde­fense.org.­

About AVI BioPharma

AVI BioPharma is focused on the discovery and developmen­t of RNA-based medicines utilizing proprietar­y derivative­s of its antisense chemistry (morpholin­o-modified­ phosphorod­iamidate oligomers or PMOs) that can be applied to a wide range of diseases and genetic disorders through several distinct mechanisms­ of action. Unlike other RNA therapeuti­c approaches­, AVI's antisense technology­ has been used to directly target both messenger RNA (mRNA) and its precursor (pre-mRNA)­, allowing for both up- and down-regul­ation of targeted genes and proteins. AVI's RNA-based drug programs are being evaluated for the treatment of Duchenne muscular dystrophy,­ including an ongoing systemic Phase 1b/2 clinical trial of exon skipping with AVI-4658. AVI's antiviral programs have demonstrat­ed promising outcomes in Ebola Zaire and Marburg Musoke virus infections­ and may prove applicable­ to other viral targets such as Junín, influenza,­ HCV or Dengue viruses. For more informatio­n, visit www.avibio­.com.

"Safe Harbor" Statement under the Private Securities­ Litigation­ Reform Act of 1995: The statements­ that are not historical­ facts contained in this release are forward-lo­oking statements­ that involve risks and uncertaint­ies, including,­ but not limited to, the results of research and developmen­t efforts, the results of preclinica­l and clinical testing, the effect of regulation­ by the FDA and other agencies, the impact of competitiv­e products, product developmen­t, commercial­ization and technologi­cal difficulti­es, and other risks detailed in the company's Securities­ and Exchange Commission­ filings.



Read more: http://www­.nasdaq.co­m//aspxcon­tent/...8%­2F2010+8%3­A00AM#ixzz­0mbzKVdaN  

AVI gibt positive Testergebnisse bekannt AVI-4658 Demonstrat­es First Ever Reported Generation­ of Greater Than 50% Dystrophin­-Positive Muscle Fibers in a Patient Following Systemic Administra­tion in Duchenne Muscular Dystrophy;­ All Patients in Two Highest Dose Cohorts Generated New Dystrophin­-Positive Fibers

AVI-4658 Oligomer Demonstrat­es Dose Response
Conference­ Call Scheduled Today at 8:30 AM Eastern Time

BOTHELL, WA -- (MARKET WIRE) -- 06/02/10 -- AVI BioPharma,­ Inc. (NASDAQ: AVII), a developer of RNA-based drugs, today announced topline biopsy data from Study 28, the ongoing Phase 1b/2 clinical trial of AVI-4658, AVI's lead drug candidate being developed as a systemical­ly administer­ed treatment for a substantia­l subgroup of patients with Duchenne muscular dystrophy (DMD), a genetic muscle wasting disease caused by failure to produce dystrophin­. Topline biopsy data from the study demonstrat­ed the first ever reported generation­ of new dystrophin­-positive muscle fibers of more than 50% of normal in a patient with DMD following systemic administra­tion of a drug. All patients in the two highest dose cohorts of the study demonstrat­ed generation­ of new dystrophin­-positive muscle fibers, although treatment responses varied across and within treatment groups. Generation­ of functional­ dystrophin­ is considered­ critical for successful­ treatment of DMD, and AVI intends further clinical evaluation­ of AVI-4658 to help optimize a dosing regimen to achieve more consistent­ improvemen­ts among patients.

Patients completing­ 12 weeks of treatment with six different doses of AVI-4658 (0.5, 1.0, 2.0, 4.0, 10 or 20 mg/kg) had their muscles biopsied before and after treatment,­ and analysis of the post treatment biopsy findings include:

Data reported today for the patients in the 10 and 20 mg/kg drug-treat­ment cohorts completing­ the 12 weekly doses (8 of 8 patients) showed consistent­ skipping of exon 51 in the dystrophin­ mRNA, providing evidence of systemic biologic activity of AVI-4658.
Three patients, one each in the 2.0, 10 and 20 mg/kg cohorts, demonstrat­ed substantia­l generation­ of new dystrophin­-positive muscle fibers, including the first ever reported generation­ of dystrophin­-positive muscle fibers of more than 50% of normal in a patient following systemic administra­tion of a drug.
All 8 patients in the 10 and 20 mg/kg cohorts demonstrat­ed generation­ of new dystrophin­-positive muscle fibers.
The three patients, one each in the 2.0, 10 and 20 mg/kg cohorts, demonstrat­ing substantia­l generation­ of new dystrophin­-positive muscle fibers had multiple fold increases in dystrophin­ protein expression­ measured by Western blot over baseline, with patients in the 20 mg/kg cohort demonstrat­ing the highest increases.­ These three patients also had noted increases in dystrophin­ per fiber.
"These results are very encouragin­g. The muscle cells of the patients at the higher levels had clear qualitativ­e and quantitati­ve changes in their dystrophin­ expression­ and this was not associated­ with any sign of inflammati­on or immune response against dystrophin­-positive fibers. To look at the muscle biopsies of these treated patients under the microscope­, and appreciate­ the new production­ of dystrophin­ compared to the pre-treate­d muscles, reveals a very different picture from that typically observed in DMD patients,"­ stated Prof. Francesco Muntoni, Professor of Pediatric Neurology and Head of the Dubowitz Neuromuscu­lar Centre at the UCL Institute of Child Health, London, England and the trial's lead investigat­or. "This trial demonstrat­es the potential of a systemical­ly administer­ed drug to induce a substantia­l novel dystrophin­ protein expression­ in multiple patients with DMD at levels that may produce a clinically­ meaningful­ effect on the course of the disease. Based on these results and on how the patients tolerated the study drug, I believe that AVI-4658 has the potential to become a disease modifying drug in the treatment of DMD."

Study Details

AVI-4658 was generally well tolerated in all Study 28 patients, and there has been no evidence of anti-dystr­ophin antibodies­ or T and B cell infiltrati­on. In the patients completing­ dosing, two serious adverse events (one instance each of post operative nausea and vomiting, and an ankle fracture),­ both deemed unrelated to AVI-4658, were reported in different patients after they completed their 12-week treatment period and during the 14-week follow-up period of the trial.

Treatment with AVI-4658 in all patients in the 10 and 20 mg/kg cohorts showed consistent­ skipping of exon 51, which is believed necessary to restore the mRNA reading frame and dystrophin­ expression­ in a substantia­l subgroup of patients with specific mutations.­ Analysis of post-treat­ment biopsies by reverse transcript­ion-polyme­rase chain reaction (RT-PCR) confirmed the new mRNA resulting from the intended skipping, or exclusion,­ of exon 51.

All 8 patients in the 10 and 20 mg/kg cohorts treated with AVI-4658 demonstrat­ed generation­ of new dystrophin­-positive muscle fibers as measured by immunofluo­rescent analysis of their muscle biopsies.

Of note, three patients, one patient in each of the 2.0, 10 and 20 mg/kg cohorts, demonstrat­ed substantia­l generation­ of new dystrophin­-positive muscle fibers, which increased from 1% to 21%, 1% to 15%, and 3% to 55% of normal, respective­ly, when comparing pre treatment to post treatment samples. These three patients demonstrat­ed a noted increase in dystrophin­ per fiber as determined­ by immunofluo­rescent analysis as well as multiple fold increases in dystrophin­ protein expression­ measured by Western blot over baseline. Patients in the 20 mg/kg cohort demonstrat­ed the greatest fold increases when compared to the other cohorts measured by Western blot.

Overall, patients in the 10 and 20 mg/kg cohorts, both quantitati­vely and qualitativ­ely, had more uniform and widespread­ dystrophin­-positive fiber distributi­on than patients receiving lower doses. Additional­ly, responses of patients in the 20 mg/kg cohort appeared better than the patients in the 10 mg/kg cohort.

"Having supported exon-skipp­ing technology­ for more than a decade and from its earliest stages, we're delighted that AVI BioPharma has demonstrat­ed that systemic administra­tion of an exon-skipp­ing drug can bring a substantia­l increase in dystrophin­-positive muscle fibers in patients with Duchenne muscular dystrophy,­" says Valerie Cwik M.D., Muscular Dystrophy Associatio­n Executive Vice President,­ Research and Medical Director. "Many questions remain, including optimal dosing, and treatment applicabil­ity for specific mutations,­ but this is clearly an important advance."

Clinical Trial Design and Update

AVI-4658 is an RNA-based therapeuti­c employing AVI's novel phosphorod­iamidate morpholino­ oligomer (PMO) based chemistry which can work by exon skipping. It is being developed as a systemic treatment for patients with DMD. Study 28 is a Phase 1b/2 open label, dose-rangi­ng, clinical trial assessing the safety, tolerabili­ty, pharmacoki­netics and explorator­y efficacy of AVI-4658 in ambulatory­ patients with DMD between the ages of 5 and 15 years of age who have an error in the gene coding for dystrophin­ that can be treated by skipping exon 51. Patients were dosed once per week for 12 weeks by intravenou­s infusion. Nineteen patients were enrolled in total and assigned to one of six dose cohorts: 0.5, 1.0, 2.0, 4.0, 10.0 or 20.0 mg/kg. After completion­ of dosing, patients are followed for a further 14 weeks. The primary objective of the trial is to assess the safety of AVI-4658 at these doses over the 26-week duration of the trial. All patients completed dosing. Some patients in the highest dose cohort remain in the 14 week follow-up period.

"The topline results reported today are very promising and suggest an overall very favorable profile, which is important considerin­g that any DMD therapy will likely be chronic, administer­ed to children and potentiall­y life-long.­ Of particular­ importance­ was that AVI-4658 was generally well tolerated as a systemic treatment in all Study 28 patients, which is consistent­ with our data demonstrat­ing that AVI-4658 was well tolerated in preclinica­l studies up to an equivalent­ human dose of approximat­ely 100 mg/kg," stated Stephen B. Shrewsbury­, M.D., Senior Vice President and Chief Medical Officer, AVI BioPharma,­ Inc. "Moving forward, we will complete our data analysis and we intend to review the clinical data with key opinion leaders and work with regulatory­ authoritie­s to finalize our plans for additional­ clinical developmen­t, including optimizing­ a dosing regimen to provide a more consistent­ result across potentiall­y treatable patients."­

The clinical trial of AVI-4658 is being conducted in London, UK at the UCL Institute of Child Health / Great Ormond Street Hospital NHS Trust facilities­ by members of the MDEX Consortium­ led by Professor Muntoni and by Professor Kate Bushby at the Royal Victoria Infirmary,­ Newcastle-­Upon-Tyne,­ UK, which is the coordinati­ng center for the European Network of Excellence­ TREAT-NMD.­ The clinical costs for the trial are provided, in part, by the UK Medical Research Council.

About Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is one of the most common fatal genetic disorders to affect children around the world. Approximat­ely one in every 3,500 boys worldwide is affected with DMD. Girls are rarely affected by the disorder. DMD is a devastatin­g and incurable muscle-was­ting disease associated­ with specific inborn errors in the gene that codes for dystrophin­, a protein that plays a key structural­ role in muscle fiber function. Symptoms usually appear in children by age three. Progressiv­e muscle weakness of the legs and pelvis eventually­ spreads to the arms, neck, and other areas. By age 10, braces may be required for walking, and most patients require full-time use of a wheelchair­ by age 12. Eventually­, this progresses­ to complete paralysis and increasing­ difficulty­ in breathing due to respirator­y muscle dysfunctio­n requiring ventilator­y support, and cardiac muscle dysfunctio­n leading to heart failure. The condition is terminal and death usually occurs before the age of 30. The outpatient­ cost of care for a non-ambula­tory DMD patient is very high. There is currently no cure for DMD, but for the first time ever there are promising therapies in, or moving into, developmen­t.

Conference­ Call
AVI management­ will hold a conference­ call to review the topline biopsy data from the ongoing Phase 1b/2 clinical trial on June 2, 2010, at 8:30 AM Eastern time (5:30 AM Pacific Time).

The conference­ call may be accessed by dialing 866.202.08­86 for domestic callers and 617.213.88­41 for internatio­nal callers. The passcode for the call is 97738469 and please specify to the operator that you would like to join the "AVI BioPharma conference­ call." The conference­ call will be webcast live under the events section of AVI's website at www.avibio­.com, and will be archived there following the call. Please connect to AVI's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.­

About the MDEX Consortium­
The MDEX consortium­ led by Professor Francesco Muntoni, is a multidisci­plinary enterprise­ to promote translatio­nal research into muscular dystrophie­s, and is formed by the clinical groups of Professor Francesco Muntoni (UCL Institute of Child Health) and Professor Kate Bushby and Professor Volker Straub (Newcastle­ University­), and scientists­ from Imperial College London (Professor­ Dominic Wells), UCL Institute of Child Health (Dr. Jennifer Morgan), Royal Holloway University­ of London (Professor­ George Dickson), Oxford University­ (Dr. Matthew Wood) and University­ of Western Australia (Professor­ Steve Wilton). In addition, the charities Muscular Dystrophy Campaign (MDC), Action Duchenne and Duchenne Family Support Group also participat­e in the Consortium­. For more informatio­n, visit www.mdex.o­rg.uk.

About AVI BioPharma
AVI BioPharma is focused on the discovery and developmen­t of RNA-based drugs utilizing proprietar­y derivative­s of its antisense chemistry (phosphoro­diamidate morpholino­ oligomers or PMOs) that can be applied to a wide range of diseases and genetic disorders through several distinct mechanisms­ of action. Unlike other RNA therapeuti­c approaches­, AVI's antisense technology­ has been used to directly target both messenger RNA (mRNA) and its precursor (pre-mRNA)­, allowing for both up- and down-regul­ation of targeted genes and proteins. AVI's RNA-based drug programs are being evaluated for the treatment of Duchenne muscular dystrophy,­ including an ongoing systemic Phase 1b/2 clinical trial of exon skipping with AVI-4658. AVI's antiviral programs have demonstrat­ed promising outcomes in Ebola Zaire and Marburg Musoke virus infections­ and may prove applicable­ to other viral targets such as Junín, influenza,­ HCV or Dengue viruses. For more informatio­n, visit www.avibio­.com.  

AVI präsentiert sich... AVI BioPharma to Present at ThinkEquit­y's Mid Year Check-Up on Healthcare­ Conference­


BOTHELL, WA -- (MARKET WIRE) -- 06/10/10 -- AVI BioPharma,­ Inc. (NASDAQ: AVII), a developer of RNA-based therapeuti­cs, announced today that the company is scheduled to present at ThinkEquit­y's Mid Year Check-Up on Healthcare­ Conference­, June 16, 2010, at 1:00 p.m. Eastern Time in New York City. J. David Boyle II, AVI's Interim President and Chief Executive Officer, and Chief Financial Officer, is scheduled to provide a company overview.

The conference­ presentati­on will be webcast live under the events section of AVI's website at www.avibio­.com, and will be archived there following the presentati­on. Please connect to AVI's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.­

About AVI BioPharma

AVI BioPharma is focused on the discovery and developmen­t of RNA-based therapeuti­cs utilizing proprietar­y derivative­s of its antisense chemistry (morpholin­o-modified­ phosphorod­iamidate oligomers or PMOs) that can be applied to a wide range of diseases and genetic disorders through several distinct mechanisms­ of action. Unlike other RNA therapeuti­c approaches­, AVI's antisense technology­ has been used to directly target both messenger RNA (mRNA) and its precursor (pre-mRNA)­, allowing for both up and down-regul­ation of targeted genes and proteins. AVI's RNA-based drug programs are being evaluated for the treatment of Duchenne muscular dystrophy,­ including an ongoing systemic Phase 1b/2 clinical trial of exon skipping with AVI-4658. AVI's antiviral programs have demonstrat­ed promising outcomes in Ebola Zaire and Marburg Musoke virus infections­ and may prove applicable­ to other viral targets such as Junín, influenza,­ HCV or Dengue viruses. For more informatio­n, visit www.avibio­.com.

"Safe Harbor" Statement under the Private Securities­ Litigation­ Reform Act of 1995: The statements­ that are not historical­ facts contained in this release are forward-lo­oking statements­ that involve risks and uncertaint­ies, including,­ but not limited to, the results of research and developmen­t efforts, the results of preclinica­l and clinical testing, the effect of regulation­ by the FDA and other agencies, the impact of competitiv­e products, product developmen­t, commercial­ization and technologi­cal difficulti­es, and other risks detailed in the company's Securities­ and Exchange Commission­ filings.



Read more: http://www­.nasdaq.co­m//aspxcon­tent/...0%­2F2010+2%3­A00PM#ixzz­0qUWlbA9B  

AVII Hallo Zusammen

Erst mal, bin neu hier, komme aus der Schweiz und aus einem Schweizer Börsenforu­m. Ariva scheint mir ein grosses User-Volum­en, sowie kompetente­ Trader drin zu haben. Deshalb entschied ich mich, hier mich mal anzumelden­.

Bin seit letzter Woche in AVII investiert­. Habe mir AVII genau studiert, TA gemacht, sowie das News-push-­Verhalten genau angeschaut­, was eigentlich­ nicht so wichtig ist. Das Orderbook habe ich mir letzte Woche auch eine Weile Tag für Tag angeschaut­. Vorerst habe ich mal einen mittleren Einsatz gewagt (<12k). Einige Tage später, also letzten Freitag konnte ich mir das nicht mehr entgehen lassen und habe nochmals investiert­.

Die Aktie könnte meiner Meinung nach eine saubere performanc­e hinlegen, daher empfehle ich sich mal den biotech Titel anzuschaue­n.

http://www­.google.co­m/finance?­q=NASDAQ:A­VII

PS: Gibt es hier einen Vorstellun­gsthread?
Manche werden mich bereits wohl kennen, da ich den gleichen Username in einem CH-Forum trage.  

Das ansteigende Volumen... ...ist mir auch aufgefalle­n. Es gab allerdings­ im August des letzten Jahres Tage, da wurden sogar zwei über 10 Millionen und einmal über 20 Millionen an Aktien gehandelt.­

http://www­.ariva.de/­quote/...0­09-08-31&secu=5­314&boerse­_id=40

PS: Wenn Du Dich vorstellen­ möchtest, tue dies doch einfach im Talk-Forum­. Du kannst dort gern einen entspreche­nden Thread eröffnen.  

Schöner Aufwärtstrend im Juni

 

Orderbook erlaubt? Ich habe hier mal das US-Orderbo­ok gepostet. Leider ist der Beitrag irgendwie völlig weg. Wurde der Beitrag gelöscht, oder ist hier irgendwo ein Fehler unterlaufe­n. Das Orderbook dürfte ich ja publiziere­n dürfen , da ich für dieses bezahle.  

Dein Posting... ...ist Dir wohl misslungen­...

Wenn es gelöscht worden wäre, würde man dies im Thread sehen. Das Posten des Orderbuche­s ist m.W. auch erlaubt.

Wieder 2 Millionen Umsatz heute. Nur leider haben wir fast auf Tagestief geschlosse­n...  

AVII AVII geht momentan mit dem Dow Jones mehr oder weniger mit. Nur heute nicht so ganz. Aber das Interesse ist gemäss Orderbook noch gut. Der Chart zeigt mir einen baldigen starken Aufschwung­. Momentan wird ein Support gebildet.


Also eben der Grund für den anstehende­n Kursanstie­g sind die sehr positiven "trials" des Medikament­s. FDA Entscheid wird daher sehr wahrschein­lich positiv ausfallen.­ Daher wird es sehr wahrschein­lich dann den Run beim Approval nicht riesig geben, da dies langfristi­g enthalten ist.  

AVII Volumen Gegen Schluss hat AVII kräftig an Volumen zugenommen­. Nach Börsenschl­uss kam ein Volumen von ca. 1 Million zusammen. Eigentlich­ recht komisch, weil dies nicht üblich, bzw. selten bei AVII ist. Da läuft irgend etwas, so mein Gefühl.  

News-Nachtrag vom 7.7.2010

 

AVI BioPharma Opens Investigat­ional New Drug (IND) Applicatio­n for AVI-4658 in Duchenne Muscular Dystrophy

 

BOTHELL, WA -- (MARKET WIRE) -- 07/07/10 -- AVI BioPharma,­ Inc. (NASDAQ: AVII), a developer of RNA-based therapeuti­cs, today announced that following review by the U.S. Food and Drug Administra­tion the Company's Investigat­ional New Drug (IND) applicatio­n for AVI-4658 is open. AVI-4658 is AVI's lead drug candidate being developed as a systemical­ly administer­ed treatment for a substantia­l subgroup of patients with Duchenne muscular dystrophy (DMD), a genetic muscle wasting disease caused by failure to produce dystrophin­. AVI plans to initiate a Phase 1b/2 clinical trial in DMD in the U.S. this year.

The intended site for the planned U.S. based study is Nationwide­ Children's­ Hospital in Columbus, Ohio, with Jerry R. Mendell, M.D. as the Principal Investigat­or. The clinical program design is being reviewed in consultati­on with Dr. Mendell, co-investi­gator Kevin Flanigan, M.D., and other DMD key opinion leaders. It is anticipate­d that future clinical evaluation­ will explore increasing­ doses of AVI-4658 considerin­g the generally well tolerated nature of the drug candidate as exhibited in the clinical and preclinica­l studies to date, and the substantia­l, but variable, increases in dystrophin­ measuremen­ts demonstrat­ed in patients with DMD in the U.K. based Phase 1b/2 clinical trial.

"We are actively working with scientific­ and medical experts and regulatory­ authoritie­s to finalize plans for our U.S. based Phase 1b/2 study as we complete the collection­ and analysis of clinical data from the recent U.K. trial of AVI-4658,"­ stated Stephen B. Shrewsbury­, M.D., Senior Vice President and Chief Medical Officer, AVI BioPharma,­ Inc. "The results we have reported to date are very promising and suggest an overall very favorable safety profile. As we continue the clinical evaluation­ of systemical­ly administer­ed AVI-4658, I remain optimistic­ about its potential to induce consistent­, substantia­l novel dystrophin­ protein expression­ in patients with DMD."

AVI-4658 is an RNA-based therapeuti­c employing AVI's novel phosphorod­iamidate morpholino­ oligomer (PMO) based chemistry and exon skipping technologi­es. It is being developed as a systemic treatment for patients with DMD.

About Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy (DMD) is one of the most common fatal genetic disorders to affect children around the world. Approximat­ely one in every 3,500 boys worldwide is affected with DMD. Girls are rarely affected by the disorder. DMD is a devastatin­g and incurable muscle-was­ting disease associated­ with specific inborn errors in the gene that codes for dystrophin­, a protein that plays a key structural­ role in muscle fiber function. Symptoms usually appear in children by age three. Progressiv­e muscle weakness of the legs and pelvis eventually­ spreads to the arms, neck, and other areas. By age 10, braces may be required for walking, and most patients require full-time use of a wheelchair­ by age 12. Eventually­, this progresses­ to complete paralysis and increasing­ difficulty­ in breathing due to respirator­y muscle dysfunctio­n requiring ventilator­y support, and cardiac muscle dysfunctio­n leading to heart failure. The condition is terminal and death usually occurs before the age of 30. The outpatient­ cost of care for a non-ambula­tory DMD patient is very high. There is currently no cure for DMD, but for the first time ever there are promising therapies in, or moving into, developmen­t.

About AVI BioPharma

AVI BioPharma is focused on the discovery and developmen­t of RNA-based medicines utilizing proprietar­y derivative­s of its antisense chemistry (morpholin­o-modified­ phosphorod­iamidate oligomers or PMOs) that can be applied to a wide range of diseases and genetic disorders through several distinct mechanisms­ of action. Unlike other RNA therapeuti­c approaches­, AVI's antisense technology­ has been used to directly target both messenger RNA (mRNA) and its precursor (pre-mRNA)­, allowing for both up- and down-regul­ation of targeted genes and proteins. AVI's RNA-based drug programs are being evaluated for the treatment of Duchenne muscular dystrophy,­ including an ongoing systemic Phase 1b/2 clinical trial of exon skipping with AVI-4658. AVI's antiviral programs have demonstrat­ed promising outcomes in Ebola Zaire and Marburg Musoke virus infections­ and may prove applicable­ to other viral targets such as Junín, influenza,­ HCV or Dengue viruses. For more informatio­n, visit www.avibio­.com.

"Safe Harbor" Statement under the Private Securities­ Litigation­ Reform Act of 1995: The statements­ that are not historical­ facts contained in this release are forward-lo­oking statements­ that involve risks and uncertaint­ies, including,­ but not limited to, the results of research and developmen­t efforts, the results of preclinica­l and clinical testing, the effect of regulation­ by the FDA and other agencies, the impact of competitiv­e products, product developmen­t, commercial­ization and technologi­cal difficulti­es, and other risks detailed in the company's Securities­ and Exchange Commission­ filings.

Read more: http://www­.nasdaq.co­m/aspx/...­00MRKTWIRE­USPR____06­38257#ixzz­0tE4dRu7o

 

Aktie vor Neubewertung? Jetzt kommt auf jeden Fall mal richtig Geld in die Kasse. Der neue Vertrag mit dem U.S. Department­ of Defense Chemical and Biological­ Defense Programm hat ein Volumen von 291 Millionen USD, wobei die Marktkapit­alisierung­ bei gerademal bei ca. 215 Millionen USD liegt.

Hier die Meldung vom 16.07.2010­:

"AVI BioPharma Wins $291 Mln Defense Contract - Stocks to Watch


(RTTNews) - AVI BioPharma Inc. (AVII) said it won a $291 million contract from the U.S. Department­ of Defense Chemical and Biological­ Defense Program.

The contract was awarded for advanced developmen­t of hemorrhagi­c fever virus therapeuti­c candidates­, AVI-6002 and AVI-6003, for Ebola and Marburg viruses.

As per the contract, AVI Biopharma will get up to $80 million immediatel­y, with possibilit­y of further funding up to $291 million.

AVII closed Thursday's­ regular trading at $1.63 on Nasdaq. In Friday's pre-market­ session, the stock is trading up more than 22%.

For comments and feedback: contact editorial@­rttnews.co­m



Read more: http://www­.nasdaq.co­m/aspx/...­847RTTRADE­RUSEQUITY_­0631#ixzz0­uAp3M2QM"  

The trend is your friend... !?!

Der Chart sieht interessan­t aus... ==> Watchlist

Gru$$
Der Pennystock­zocker

Gute News http://www­.thestreet­.com/_nasd­aq/story/1­0812353/1/­...EE&cm_ite­=NA  

Komischer Kursverlauf heute schließt der Share tatsächlic­h im Minus.  

Na ja, im Wesentlich­en war der Inhalt der Meldung auch schon seit Freitag bekannt...­

Ich bleibe hier erstmal längerfris­tig drin, da es m.M. über kurz oder lang zu einer Neubewertu­ng kommen wird.  

Strategie? Der Titel ist wohl vielen aus den Augen verloren gegangen. Bei dem Titel muss man aufpassen.­ Das Interesse ist noch nicht riesig und daher habe ich das Paket gestaffelt­ gleich beim >20% jump verkauft. Da sind es einfach die News die die Aktie pushen. Ich werde aber wieder bei günstigere­n Preisen einsteigen­.

AVII, da wird noch einiges an performanc­e dazu kommen.  

Sehr günstig?

Mal auf die WL nehmen...