Hello,
I just got off the phone with Lindsay, the clinical trials coordinator for Dr. Mendell. She asked me to post that anyone whose son is between 5-15 who would benefit from skipping 51 who is interested in participating in a systemic trial should contact her at arnottl@ccri.net.

The trial will have a placebo arm. It will take place in Columbus. I don't know the dosage.

Take care, all,
Mindy

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I agree with you but I would like to add something important here.

Jerry Mendell is a really exceptional person. He is spending every day of his life trying to find a treatment to improve the lives of boys/young man with DMD and other forms of MD. He worked in the field for more than 30 years and will continue to do so no matter what comments are posted or questions are asked. He will support AVI with all they need in order to test these compounds and hopefully bring something to the market if proven therapeutic. He will not provide inaccurate information or make unnecessary comments.

Being in the filed for so long, meeting so many families going through this, he understands better than anyone parent's desperation and does all in his power to help.



Mindy said:
I completely agree, Kelly. I didn't want to offend anyone, but Lindsay is getting some pretty frustrating emails, and asked me to tell everyone to tone it down a bit. I feel the desperation, too, but we do need to be careful...
Is Lindsay using the "DuchenneConnect" database? Or are not enough people registered fitting the criteria?

That would have saved her getting a bunch of those emails....
AVI BioPharma Announces Department of Defense Funding to Accelerate Development of AVI-4658 for Duchenne Muscular Dystrophy

http://www.avibio.com/pr/pr416.php

For Immediate Release
PORTLAND, OR — May 6, 2009 — AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based drugs, today announced a $2.5 million contract with Children’s National Medical Center (CNMC) in Washington, D.C. to support preclinical studies in the development of AVI-4658 for treatment of Duchenne muscular dystrophy. The work will be conducted with CNMC collaborators Eric Hoffman, Ph.D., an authority on DMD and Professor of Pediatrics, and Edward Connor, M.D., Director, Office of Investigational Therapeutics and Professor of Pediatrics. AVI will serve as a subcontractor to a grant awarded to CNMC by the U.S. Department of Defense.

“We are pleased to collaborate with Dr. Hoffman, a distinguished researcher and expert in the field of DMD, and advance AVI’s efforts toward the development of therapeutics utilizing exon skipping for the treatment of DMD,” said Steve Shrewsbury, M.D., Chief Medical Officer and Senior Vice President of Clinical and Regulatory Affairs of AVI BioPharma. “Through this collaboration, AVI hopes to provide the additional data as requested by the FDA to allow clinical studies with AVI-4658 to begin in the U.S.”

The collaboration will support a series of GLP toxicology studies for AVI’s exon skipping drug candidates based on phosphorodiamidate morpholino oligomers (PMO) chemistry. The funding is part of the Department of Defense’s Congressionally Directed Medical Research Program to identify and pursue research with the most promise for treatment of DMD.

“Exon skipping represents one of the most promising investigational approaches for the treatment of Duchenne muscular dystrophy, and it is imperative to accelerate the clinical development of therapeutics that could improve the care and quality of life for boys with this disease,’ said Dr. Hoffman. “We are excited to be collaborating with AVI, both a pioneer and a leading developer of RNA-based drugs.”

“A strong pre-clinical GLP toxicology package is a critical part of a robust drug development program, and this new grant is designed to help enable both intravenous and subcutaneous clinical trials in DMD patients in the U.S.,” said Dr. Connor.

AVI is currently evaluating AVI-4658 in human clinical trials in Europe. The drug is designed to skip exon 51 of the dystrophin gene, allowing for restoration of the reading frame in the mRNA sequence. By skipping this exon, a truncated, yet potentially functional form of the dystrophin protein is produced, which could ameliorate the disease process and possibly prolong and improve the quality of life in these patients. Results from a Phase 1 proof-of-concept trial showed that injection of the drug into the muscles of a series of DMD boys successfully induced dystrophin production in a dose-responsive manner. Further, the drug was well tolerated, with no significant drug–related adverse events reported. The clinical trial was conducted in collaboration with the MDEX Consortium in London UK. AVI is currently sponsoring an ongoing clinical trial in the UK evaluating the systemic delivery of AVI-4658. This is an open label, 12 week safety trial, which includes measures of drug efficacy and pharmacokinetics, 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.

About Duchenne Muscular Dystrophy (DMD)
DMD is the most common fatal genetic disorder 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 before age six. 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. 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 in decades, 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 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.

About Children’s National Medical Center/Children’s Research Institute
Children's National Medical Center, located in Washington, D.C., is a proven 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 proudly ranked among the best pediatric hospitals in America by US 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 our research programs at www.childrensnational.org/research and www.wickerproject.org.
well, great. So the FDA has requested yet more toxicology studies. Add on another 6-12 months before trials even begin.
Mindy,

I am not sure if this is a new request from FDA (?). Have you heard something about that? I am assuming (and the key word is assuming) that they want to increase the dose (as compared with the UK trials) and optimize the compound to be able to deliver it subcutaneous also. The one used in the UK trial can be delivered with good results only using IV. I whish I could ask some of these researchers for clarification. We might get more answers during AVI's financial results conference call on Monday.

Ofelia

Mindy said:
well, great. So the FDA has requested yet more toxicology studies. Add on another 6-12 months before trials even begin.
The people from Columbus told me that they were meeting with the FDA shortly to discuss requirements before the trial. They were hopeful that there would be no studies requested before beginning. I'm assuming this means that they are indeed asking for more studies? I don't know, though. My biggest frustration is that no one seems to be talking to one another enough to give us the straight story on what is going on. I would just like to know what is going on. I'm so tired.
I feel the same way! You hear different things from different people... We'll see what AVI has to say on Monday... The big question is why do they need additional preclinical work. I will be going to Columbus Children's tomorrow for an interview about the newborn screening study and if I see Mendell I'll ask.


Mindy said:
The people from Columbus told me that they were meeting with the FDA shortly to discuss requirements before the trial. They were hopeful that there would be no studies requested before beginning. I'm assuming this means that they are indeed asking for more studies? I don't know, though. My biggest frustration is that no one seems to be talking to one another enough to give us the straight story on what is going on. I would just like to know what is going on. I'm so tired.
OK - please let me know what they say. Thanks.
Please let me know what they say. My son has a deletion of 48-50 and needs skipping 51.

Ofelia Marin said:
I feel the same way! You hear different things from different people... We'll see what AVI has to say on Monday... The big question is why do they need additional preclinical work. I will be going to Columbus Children's tomorrow for an interview about the newborn screening study and if I see Mendell I'll ask.


Mindy said:
The people from Columbus told me that they were meeting with the FDA shortly to discuss requirements before the trial. They were hopeful that there would be no studies requested before beginning. I'm assuming this means that they are indeed asking for more studies? I don't know, though. My biggest frustration is that no one seems to be talking to one another enough to give us the straight story on what is going on. I would just like to know what is going on. I'm so tired.
From what I see here, additional data was requested by the FDA:

http://www.tradingmarkets.com/.site/news/Stock%20News/2317570/

May 08, 2009 (Datamonitor via COMTEX) -- AVII | Quote | Chart | News | PowerRating -- AVI BioPharma, a developer of RNA-based drugs, has obtained a $2.5 million contract from Children's National Medical Center or CNMC in Washington, District of Columbia, to support preclinical studies in the development of AVI-4658 for treatment of Duchenne muscular dystrophy.
The work will be conducted with CNMC collaborators Eric Hoffman, professor of pediatrics, and Edward Connor, director of Office of Investigational Therapeutics and professor of Pediatrics. AVI will serve as a subcontractor to a grant awarded to CNMC by the US Department of Defense.

The collaboration will support a series of GLP toxicology studies for AVI's exon skipping drug candidates based on phosphorodiamidate morpholino oligomers chemistry. The funding is part of the Department of Defense's Congressionally Directed Medical Research Program to identify and pursue research with the most promise for treatment of Duchenne muscular dystrophy (DMD).

AVI is currently evaluating AVI-4658 in human clinical trials in Europe. The drug is designed to skip exon 51 of the dystrophin gene, allowing for restoration of the reading frame in the mRNA sequence. By skipping this exon, a truncated, yet potentially functional form of the dystrophin protein is produced, which could ameliorate the disease process and possibly prolong and improve the quality of life in these patients.

According to AVI, results from a Phase I proof-of-concept trial showed that injection of the drug into the muscles of a series of DMD boys successfully induced dystrophin production in a dose-responsive manner. Further, the drug was well tolerated, with no significant drug-related adverse events reported.

Steve Shrewsbury, chief medical officer and senior vice president of clinical and regulatory affairs at AVI BioPharma, said: "We are pleased to collaborate with Dr Hoffman, a distinguished researcher and expert in the field of DMD, and advance AVI's efforts toward the development of therapeutics utilizing exon skipping for the treatment of DMD. Through this collaboration, AVI hopes to provide the additional data as requested by the FDA to allow clinical studies with AVI-4658 to begin in the US."
Thanks Ofelia, you are really on your game getting this stuff so quickly.
I sure would like to know what else the FDA required and HOW LONG will this additional process take. Any indication of either?

Ofelia Marin said:
From what I see here, additional data was requested by the FDA:

http://www.tradingmarkets.com/.site/news/Stock%20News/2317570/

May 08, 2009 (Datamonitor via COMTEX) -- AVII | Quote | Chart | News | PowerRating -- AVI BioPharma, a developer of RNA-based drugs, has obtained a $2.5 million contract from Children's National Medical Center or CNMC in Washington, District of Columbia, to support preclinical studies in the development of AVI-4658 for treatment of Duchenne muscular dystrophy.
The work will be conducted with CNMC collaborators Eric Hoffman, professor of pediatrics, and Edward Connor, director of Office of Investigational Therapeutics and professor of Pediatrics. AVI will serve as a subcontractor to a grant awarded to CNMC by the US Department of Defense.

The collaboration will support a series of GLP toxicology studies for AVI's exon skipping drug candidates based on phosphorodiamidate morpholino oligomers chemistry. The funding is part of the Department of Defense's Congressionally Directed Medical Research Program to identify and pursue research with the most promise for treatment of Duchenne muscular dystrophy (DMD).

AVI is currently evaluating AVI-4658 in human clinical trials in Europe. The drug is designed to skip exon 51 of the dystrophin gene, allowing for restoration of the reading frame in the mRNA sequence. By skipping this exon, a truncated, yet potentially functional form of the dystrophin protein is produced, which could ameliorate the disease process and possibly prolong and improve the quality of life in these patients.

According to AVI, results from a Phase I proof-of-concept trial showed that injection of the drug into the muscles of a series of DMD boys successfully induced dystrophin production in a dose-responsive manner. Further, the drug was well tolerated, with no significant drug-related adverse events reported.

Steve Shrewsbury, chief medical officer and senior vice president of clinical and regulatory affairs at AVI BioPharma, said: "We are pleased to collaborate with Dr Hoffman, a distinguished researcher and expert in the field of DMD, and advance AVI's efforts toward the development of therapeutics utilizing exon skipping for the treatment of DMD. Through this collaboration, AVI hopes to provide the additional data as requested by the FDA to allow clinical studies with AVI-4658 to begin in the US."
Very good results obrained in the UK IM trial -- much better than Prosensa's. I will try to call in for today's CC, hopefully they provide more details about the FDA and start of US trials.

http://www.avibio.com/pr/pr417.php

Announced successful completion of a single injection, dose escalation Phase 1 trial of AVI–4658 for the treatment of DMD by exon skipping. AVI-4658 induced a robust expression of dystrophin following IM injection in a series of drug-treated patients such that up to 80 of fibers were positive for dystrophin expression, when one corrects for background expression in the other, saline-treated foot. Equally importantly, individual fibers showed new protein expression at a level up to 40 % of normal. There was no immune response to the protein and no serious, treatment-related side effects were observed.

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