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...
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.
From what I see here, additional data was requested by the FDA:
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."