ADH and AVGN
Randy,
See PGS post 21616 for a link to a PI. One partial response and two with normalized markers. But there was also indication in the AEs that the drug was perhaps impacting the cardivascular system or that there was some immune response.
Here's a puzzle unrelated to ADH:
AVGN has sold off its gene therapy business to GENZ to concentrate on small molecules.
Lots of assests now, with cash in excess of the market cap.
Lead molecule is AV411, which is undisclosed. They want to develop it for neuropathy, with the premise that it inteferes glial cell activity, which is responsible for neuropathy. This is off-patent and has been used in Asia for a different indication for 10 years, with proven safety.
A poster on the YMB (beachbottlemessenger post 4703 and 4704) has listed his notes on it, which I copy below. Another poster has claimed to identify the compound but refuses to disclose it. Anyone have any idea of name or composition of the drug?
Quoted text:
I collected some info about AV411 from recent conferences:
** approved for over 10 years in Japan, Korea, and some other Asian countries,
the composition matter has expired because the drug was patented in the mid 80's (no need to in-license now)
** Avigen does not want to reveal the identity of AV411 because it still works on some IP issues like manufacturing and
trying to find another AV411 in the literature (they are doing extensive searches and they have some interesting hits
they want to try), they have not even contacted the pharma company that makes the compound,
current use is completely unrelated to neuropathic pain or any neurological condition,
Avigen's new use is a complete deviation from what the drug is currently used for,
the patent for composition matter expired so there is a freedom to operate,
Avigen filed its own patents for the new use of the molecule, also dosing and formulations,
they plan to disclose its identity in q1 of 2006
** AV411 is a small molecule that inhibits interleukin, interferon, and TNF.
it was selected because of potent glial atteniuating activity in the literature but it has not
been tried/used for any neuro condition before
** it has an excellent safety record in over 3 million patients,
the product insert has data for 15000 patients, side effect profile from product insert is extremely clean,
it allows them to have a quick development and eliminates one major
component of development which is safety of molecule,
the indication it is used for is chronic so there is a lot of data, people take it for at least 6 months
which satisfies the chronic definition
** the literature showed administration at higher doses to be safe,
Avigen did safety study with doses 2x what is currently used for and they see nothing unusual in safety data,
they did sedation tests with positive results, rats show some sedation for the first 30 mins but later the sedation
goes away (sedation completely reversed after 2 hours, after 3 days of using it becomes completely non-sedative),
if it holds true in humans AV411 can become the first drug that is efficacious and non-sedative,
AV411 is relatively long lasting (at least through the night), not addictive, preserves normal sensation,
their studies compared to Gabopentin, Neurontin - their reversal significant but short lasting compared to AV411
** Avigen wants to initiate trial in 2006, they hope to start in Phase II and bypass Phase I if FDA agrees but with
the safety data they hope this is the case, possibly they will start in Europe first
** AV411 is the center of a much bigger product,
in investor packet there is a copy of Science article (several months ago) that claims that gia is responsible
not only for the pain but also Parkinson's and other conditions,
Avigen has 15 proposed analogs, they made two analogs and are in process of making two others,
it gives them ways to improve the molecule and file more patents on the composition matters of the new molecules,
they are also looking at some other indictions: morphine/opiate withdrawal (they have some inital data from work
with Linda Watkins) and neurodegenerative diseases (like Parkinson's - they should have data by the end of this year)
** they found some references in the literature that the AV411 was used in humans for non-neuro indications at doses
close to 2x (this is what Avigen targets) and there was no reports of sedation
** it is easy to manufacture,
Avigen manufactured 50 grams of AV411 in about a week, initial purity was 98.5%
there are several API manufacturers out there, the costs of goods are going to me exteremely low
** the next step is to obtain GMP material and probably go straight into phase II trial in 2006, it is likely that
they do it first in Europe, they will prepare in parallel an IND package for FDA (in 2006),
the plan is to have a proof principle from Europe before filing IND with FDA, so with the longer process in US
they will have data on efficacy first
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