It looks like they are both partnering with Alpha-1 (it's a reasearch foundation) to address the same illness (AAT Deficiency Liver Disease) using competing delivery platforms. I suppose it could also be a combination of both delivery platforms in one study.... someone call me out if I'm wrong here.
But it's probably competing platforms in two studies side by side, sharing a control group... otherwise why not mention some form of cooperation between the two companies in the pr's?
From the Anylam pr:
New data presented at The Liver Meeting are based on an AAT-targeted siRNA conjugated to an N-acetylgalactosamine (GalNAc) ligand, which is designed to achieve targeted delivery of RNAi therapeutics to hepatocytes through uptake by the asialoglycoprotein receptor. Studies were performed in transgenic mice overexpressing the human Z-AAT protein. Subcutaneous administration of a GalNAc-siRNA conjugate targeting Z-AAT led to dose-dependent knockdown of serum levels of human Z-AAT. A single dose of 3 mg/kg led to greater than 95% knockdown of Z-AAT protein that lasted for greater than two weeks. In a multi-dose experiment, twice-weekly dosing at 0.5 mg/kg led to greater than 90% knockdown.
From the arrowhead pr:
"ARC-AAT is designed to inhibit the production of mutant AAT protein in the liver and in preclinical studies it has shown high levels of knockdown with long duration of action. AATD patients express mutant AAT, which can accumulate in hepatocytes and damage the liver. Liver disease associated with Alpha-1 antitrypsin deficiency has no current treatment options and patients can progress to cirrhosis, hepatocellular carcinoma, and may require liver transplant." Arrowhead has developed a novel unlocked nucleobase analog (UNA)-containing RNAi molecule designed for systemic delivery using the Dynamic Polyconjugate (DPC) delivery system.
The stuff Alnylam and other companies like it are developing are so new there is no way to value the technology. I really think long-term this company may be huge. If they can remain independent, this stock could be a homerun. If....!!!!
ANLY has got the jack to buy ARWR???
ANLY is a $4 billion company. ARWR is potentially worth way more than that. Hmmm
FDA can be as suspect as they like. It is the trials that will prove or disprove safety, not the FDA. RNAi now on the cutting edge of therapies for most nearly all diseases. Medicine as we know it is about to change. GLTA
You won't see this stock make any big gains for a long time without money printing courtesy of the FED. Now that the taper is coming to an end, the money flow into the stock market is going to dwindle. Stocks will no products and no earnings are therefore at great risk. This, of course, is the category to which ALNY falls into.
I know why are are you telling this, because you sh...now you don't this st goock up..Th culuesis istjis.t to this stock has very good opportunity to go and will up to 250 like. It ha pipelines...watching blosssebu them next sk rotin
oct....ty itock ....ot will n mm very goody to. cover boufht theni recommend inh ...ypu will going up.s 15 pbyi up
RNAi has produced more drug failures that Carters has pills. Six billion market cap. What a joke.
In the 90s, Ribozyme Pharmaceuticals (used to trade as RZYM) tried to make a go of this technology and failed. Zero Drugs.
In the 00s, Sirna Therapuetics gave it a go and they failed. Zero drugs.
in the 10s, Merck tried and they failed. Zero drugs.
Merck then sold Sirna to a sucker for $175 million. That sucker was ALNY.
All ALNY will do with this technology is separate a fool from his money. The money will go to the ALNY employees and to the firms on Wall Street and the hedgies. They've driven this stock up to an extraordinary valuation. And as they make wild price predictions, like Leerink Swann did the other day, they'll get the retail fools to buy while they unload. And then they'll short it and will make money on the downside. It is just like the dotcom bubble.
Did you read the first sentence "Alnylam ALNY intends to file a clinical trial application". Oh boy, they INTEND to do it! When don't they just put out a press release saying that they intend to get a drug to market or that they intend to make some money some day or that they intend to control their burn rate carefully.
Good lord, now the whole world knows this company is run by idiots! This has to be a first. Every other company I know only puts out a press release when something actually happens or they actually accomplish something. Not ALNY!
I can only surmise that they did this stupid release to get the stock price up a couple more points on Friday by getting a few more fools to buy this snake oil. The technology has had so many failures that the company loses no opportunity to pump itself. And I thought I saw it all. You can't make this stuff up.
Alnylam says on its website that RNAi therapeutics is "breakthrough technology". Who are these people kidding?
This so-called breakthrough technology was developed by Ribozyme Pharmaceuticals in the early 90s. I know because I was there as a shareholder. Ribozyme NEVER got a drug to market. Back then it was also touted as a new breakthrough technology. And after it went NOWHERE, after years of trying, the company operated under a new name, Sirna Pharmacueticals, which too touted it as breakthrough. Merck then acquired Sirna, hoping they could make something of it, which they evidently couldn't since Merck sold Sirna to Alnylam for a fraction of what Merck paid. In fact the purchase of Sirna is written about on Alnylam's website.
In short, this so called "breakthrough technology" has been around for decades. The early patents have now expired. No drug has ever come to market from it by Rybozyme, Sirna or Alnylam. And if Alnylam ever did get a drug to market, it would be years from now. The only thing this company has going for it are a bunch of foolish funds, banks, and retail investors who put their money here, thinking that they are buying a "breakthrough technology". ALNY has a market cap of $6B with no drug near approval, and I'd bet that they never get a drug to market. Now PCYC, on the other hand, has one of the greatest oncology drugs ever produced, and it recently entered the market: Imbruvica. Imbruvica has years of patent protection to go. It will generate billions in sales, and PCYC has a market cap of only $9B. IMHO ALNY is the best bio short out there, and PCYC is the greatest bio long.
I am not saying this because I am short. I am short for the reasons I expressed. You should go short too because this company will never make any money. That's my opinion.
This company is on track to lose $200M a year. In three years, they'll be broke and not have a single drug on the market.
Oh they'll keep touting their "pipeline" to keep this thing afloat in a further secondary, but I doubt they'll ever finish P3 successfully in any clinical trial. If they do, people will learn of the failed technology first hand rather than through earlier precedent.
interesting article. "Using a miniature model of the human intestine, only a few millimeters in size and made from stem cells, the scientists deactivated a gene in the cells tied to metabolic regulation called FOXO1. Once disabled, the cells began producing insulin. " possibly up ALNYs alley?
"The method, described today in the journal Nature Communications, raises the possibility of replacing insulin-making pancreatic beta cells lost in diabetics by using a drug to retrain patients’ existing cells. While progress has been made in generating beta cells from stem cells, the method hasn’t yet produced ones with all the needed functions, said Domenico Accili, the study’s lead author. Plus, such cells would require transplantation. "
There are a lot of great pipelines out there. ALNY has no drugs and works it the hardest therapeutic arena
currently. If you can tell me what year you expect a drug to pass the FDA that Alnylam makes. then I can
assuage your sinking feeling.