It is about product developements. Epix is on the cusp
of good news in that vein. Here is an article giving a market heads up and I concur. Epix will only suffer short term as weak hands and short term trades flee.
The strength of epix current product developement will launch the pos (price of stock :)) to former highs.
I am holding no matter how hard the mm and shorts shake the trees eod. Adding on weakness.
Baux..my goal in life is to hop on a plane soon and go visit Dr.E(Entendance) on his glorious island in the Seychelles!
I'm retired now in Delaware so I have the time!
On a sadder note just heard from my sister regarding our long time neighbor back in Maryland who had been suffering from AD and just passed away.Another funeral on monday. His family had been terrific caregivers for well over 15 years. This man was hard working Scottish/American with hands like a bear and a grip like a vice. A stone mason by trade all his working life and like a second parent/mentor to me! Just trying to put a "face" on this ravaging disease which will hit the US,Europe and Asia like a tsunami! These are the big three aging society demographics we EPIX longs need to understand.
To my friends here at EPIX I have made a major investment in FOLD a small Bio working on genetic disorders(enzyme folding disorder diseases) called Fabry's,Gaucher's, and Pompe's. They will be going up against Genzyme if Amigal,Plicera, and AT-2220 pan out.I am not pumping you guys. Shares are hard to get anyways due to heavy insider,private and institutional ownership.I only mention it because we(EPIX LONGS) all do tremendous DD and if you see something on Amicus Therapeutics(FOLD) please let these old eyes know about it good,bad,or indifferent!
I have just placed some MLNM windfall yesterday into FOLD. I am looking to add to EPIX also!
Emerging Drug Developer: Epix Pharmaceuticals
April 14, 2008
By John Carroll
For most investors, the sweet spot in any biotech play is right around proof-of-concept data on a new therapy for a disease that afflicts a large population of patients. That’s the point when a big pharma company can size up the commercial opportunities along with late-stage development costs and step in and make an offer to partner up.
It’s also the reason why emerging drug developers like Epix focus on diseases like Alzheimer’s and obesity, where current therapies often don’t work very well and the unmet medical need and market potential is greatest.
But two years ago the Cystic Fibrosis Foundation made Kauffman an offer that made him disregard the traditional business model and move into a disease category that otherwise wouldn’t have made much economic sense. The foundation put up $12 million to fund a program at Epix that put the biotech’s technology to work exploring a new therapy for cystic fibrosis. Rather than look for a market payoff, the foundation’s primary objective is to advance the research far enough along that the commercial opportunity comes within reach -- and the biotech can advance on its own to commercial development.
Last week, after Epix hit its fourth milestone in that development pact, the foundation came back and added a very big carrot in the form of an additional $37 million in fresh research funds.
“This funding goes through Phase IIa, early stage clinical proof-of-concept,” says Epix CEO Michael Kauffman, M.D., Ph.D. “If we see an effect in the clinic by then, I would stand by this. We would be very excited about this mechanism.” The foundation, he adds, “wants to see this disease treated. They want to take it to a point of top priority. There’s a different late-stage focus for the foundations and nonprofits.”
Epix’s success in the field comes from its expertise in developing computer models of the 3-D structures of drug targets – in this case the cystic fibrosis transmembrane conductance regulator, or CFTR. The most common mutation of the CFTR gene is the Delta F508 mutation, which is responsible for key symptoms of CF in about 70 percent of the patients who suffer from the disease. The body eliminates the abnormal protein made from the mutated gene, and the reduced levels of CFTR protein spur the creation of a sticky mucous material that causes lung infections and disrupts digestion in people who suffer from the disease.
“What happens is that the Delta F508 protein is made just fine,” says Kauffman. But the correct 3-dimensional folding process that normally follows goes awry. The body in turn detects the misfolded CFTR proteins and discards them as it normally does to protect the body from abnormal proteins. “The problem is that less than five percent of the Delta F508 ever makes it to the surface, so people with CF have a level of CFTR maybe 20- to 50-fold lower than normal, which is why you get the disease. The little bit that gets to the surface also doesn’t function as well as the normal protein.”
Any new therapy doesn’t have to be 100 percent effective, he adds. Even a 20 percent to 25 percent increase in CFTR would probably trigger a considerable improvement in patients.
Understanding the structure of CFTR gives Epix an inside track in developing a therapy that can make the protein work properly, eliminating those symptoms.
“We create our own computer-derived model we think is right,” says Kauffman. “Essentially, we’re trying to replace X-ray crystallography.”
To illustrate what he’s talking about, Kauffman frequently uses an analogy of examining a lock – the drug target -- to come up with a key – the drug. “If you know the structure inside the lock, it’s far easier to make a key.” That’s a far different approach than high-throughput screening technology, where you try a few hundred thousand keys to see which ones best fit the lock.