What Katrina Was to FEMA, Swine Flu Deaths Are to FDA – Buy BioCryst

In contrast to their good job shepherding swine flu vaccines through the approval process, the FDA has failed for months to provide Intensive Care Unit doctors with an an effective intravenous antiviral to treat the sickest victims of H1N1. FDA began a Pre-Emergency Use Authorization process for BioCryst’s peramivir almost six months ago. The development of peramivir has been sponsored by HHS, and it has completed Phase I and II trials in the U.S. and Phase I, II and III trials in Japan. It is known to be safe and effective, yet Americans are dying daily in the ICU without timely access to the drug.

Why is an intravenous antiviral the FDA knows to be safe and effective languishing in their bureaucratic process while Intensive Care Unit doctors plead for it to be released before more victims die? The FDA approached BioCryst Pharmaceuticals of Birmingham, AL almost six months ago to begin the Pre-Emergency Authorization Use process for peramivir. The Phase I and II U.S. trials were sponsored by the Department of Health & Human Services under a $102.4 million contract. FDA had already reviewed Phase I safety data and approved the drug for Phase II studies. They then reviewed the Phase II safety and efficacy data and approved the drug for Phase III studies that may not be completed until 2011. In June, they gave the drug Emergency IND status, so a single doctor with an identified patient can fill out forms and ask the FDA to permit BioCryst to ship a dose of the drug. This shows again that the FDA knows the drug is safe and effective.

The Emergency Use Authorization (EUA) process is designed to allow drugs in development to be sold or stockpiled to treat an imminent health threat, especially when no other effective treatment is available. There is no intravenous antiviral for the H1N1 flu approved anywhere in the world, including the U.S. Peramivir is the closest to achieving approval, in both Japan and Korea. I expected the FDA’s EUA approval process to take no more than 60 days. It has now taken six months, with no end in sight, and many critically ill U.S. swine flu victims have died unnecessarily as a direct result of the delay.

Most people who contract the swine flu simply suffer through the typical symptoms of flu. But 10% to 20% of the victims, who are often otherwise-healthy children, young adults or pregnant women become sick enough to be hospitalized. Most of those can be treated with Tamiflu or Relenza, which are already in national stockpiles around the world. But a significant portion – as high as 15 per cent, according to the World Health Organization – end up in ICUs for many days or even weeks, hovering between life and death.

Dr. Paul Hebert, editor of the Canadian Medical Association Journal and an intensive care physician in Ottawa who has treated many of these sickest patients, said: “I’ve never seen this. As an ICU doctor, it’s very, very, very rare I can’t deliver enough oxygen to someone to keep him alive. They die of other things, right? They die because their organs fail. In this case, we can barely oxygenate them.”

Peramivir is a very effective intravenous antiviral that can deliver a large load of drug very quickly, as opposed to the five-day treatment regimen with Tamiflu. Because the H1N1 virus often goes deep into the lungs and replicates much faster than the seasonal flu, many critically ill patients cannot use inhaled Relenza.

If an ICU patient does not respond to or cannot take Tamiflu or Relenza, the ICU doctors have nowhere else to turn. Although peramivir is available on a case-by-case basis under the awkward, slow Emergency IND process, in most cases the drug cannot be transported to doctors in time to save a patient’s life.

In one recent case, desperate ICU doctors in Great Britain broke open a Relenza inhaler and mixed the contents into an IV bag, saving the life of a 22-year old woman. ICU doctors need an intravenous antiviral immediately, and peramivir is the only one that has completed Phase III clinical trials. Shionogi announced the positive results of their Phase III trials this summer, again showing that intravenous peramivir is a safe and effective drug.

As a result of the FDA’s intolerable delay in issuing the EUA and an order to stockpile the drug, hundreds of Americans have died unnecessarily, including 144 children in the flu season so far. Yet the logic for approval is simple:

1. Is the drug safe and effective? Yes, as shown by the FDA’s review of Phase I and Phase II results, the Japanese Ministry of Health review of Phase I and Phase II results, Shionogi’s Phase III results reported recently at the ICAAC Conference in San Francisco, and the FDA’s own granting of the Emergency IND status.

2. Is there a need for the drug? Yes, there is no other intravenous antiviral available for ICU doctors to use when Tamiflu and/or Relenza have failed or cannot be given to the patient.

3. Is the Emergency IND process sufficient to fill the need in ICUs? No, the cumbersome, slow process has resulted in less than 10 patients having access to peramivir since June, while hundreds have died.

I am waiting for a crusading journalist, columnist or politician to hold the FDA’s feet to the fire on this issue, and demand the Emergency Use Authorization and a substantial stockpiling order immediately. BioCryst has already signed sales reps in Mexico, Brazil, Israel and China, and I expect India and Europe to be next. These countries can place orders for peramivir with less bureaucracy than the FDA, and the drug might be saving lives overseas while Americans are still dying without it. To quote one well-known biotech columnist: “Murphy…seems to be the only person I hear slamming the FDA about its response to the flu, blaming regulators for dying kids. As if Margaret Hamburg has blood on her hands.”

My sentiments exactly.

I expect BCRX to book a large order for the U.S. stockpile, equally large orders from the BRIC countries, Mexico, Israel, Europe and the World Health Organization, and ongoing orders for many years to replenish the peramivir that is used in ICUs or emergency rooms. Thus, my disclosure.

Disclosure: Long BCRX, obviously

You can go to http://seekingalpha.com/instablog/128006-michael-murphy/28294-what-katrina-was-to-fema-swine-flu-deaths-are-to-fda-buy-biocryst to see the comments on this article.  I expect the full complement of BioCryst bashers.

This mornings, BioCryst (BCRX) announced that it has signed binding letters of intent with three partners to represent peramivir for flu stockpiling programs in Mexico, Brazil, China and Israel.  Mexico is where it all started, and the company says Mexico and Brazil together represent 75% of the Latin American market.  China is

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Rob Stein of the Washington Post wrote a story Friday titled: “As Swine Flu Threat Grows, Health Officials Retool Strategies.” It is mainly about the impending disaster at intensive care units, and government efforts to get people to self-diagnose the degree of illness of their kids, and not go to the hospital until they are seriously ill. Good luck with that.

BUT – this gives us the opening we need to light a fire under the FDA’s collective butt. In the article, on page 2, Rob quoted an FDA official: “This is occurring in, I think, a highly charged environment where people are very concerned right now about denial of access to health care,” Jesse Goodman, chief scientist and deputy commissioner of the Food and Drug Administration, said at an Institute of Medicine forum. “We have to do the right thing from the public health point of view, but we also have to consider this in the lens it’s going to be viewed in.”

So I posted this comment:

“I am stunned that Jesse Goodman, chief scientist and deputy commissioner of the Food and Drug Administration, would dare to say: “People are very concerned right now about denial of access to health care. We have to do the right thing from the public health point of view.” This is the agency that has denied terminally ill children access to the drug that could save them by delaying issuing an Emergency Use Authorization for intravenous peramivir, the ONLY antiviral with extensive safety and efficacy data for the sickest H1N1 patients. Peramivir has been through Phase I and II trials in the U.S., funded by Health & Human Services, and Phase I, II and III trials in Japan, funded by Shionogi. It has zero safety issues. The FDA began working on issuing an Emergency Use Authorization for peramivir in March, and still has not announced the EUA or a vitally-needed major purchase order for the National Stockpile. Meanwhile, dozens of children have died – suffocated – who might have been saved if IV peramivir had been available on a timely basis.
What Hurricane Katrina was to FEMA, this unconscionably delayed EUA for peramivir will be to the FDA – and possibly the whole Obama Administration.”

I am asking any of you who are registered at the Washington Post, or any who are willing to do so (it’s free and fairly painless) to log in, go to the Comments page at http://www.washingtonpost.com/wp-dyn/content/article/2009/09/11/AR2009091102779_Comments.html and click the “Recommend” button under my comment in the lower left. I have already emailed Rob Stein directly to write a story based on my last sentence, and if there are a flood of “Recommends” he can show his editors and go for it. Everyone in Washington politics reads the Washington Post. This is our chance to put incredible pressure on the FDA to get the EUA and stockpile order done. Let’s take it.  Thank you!

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I am leaving this Flash Alert unprotected so anyone can read it. It is a way for people to voice their disgust with the FDA’s process on the EUA for peramivir. The more, the better.  Feel free to post this on any message board or forum that might care.

The bear raid on BioCryst (BCRX) ran

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Dear New World Investor:

It is very unusual to have

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Dear New World Investor:

This week’s issue is one day early to allow for my travel schedule to the Money Show at the Marriott in San Fransisco.

As I said in the Flash Alert on Tuesday, I think the S&P 500 wants to do a test back down to 960, with a possible intraday spike down to 912. But time is running out, because this whole downturn should be over by Friday or Monday, Day 30 or 31 of the new 86-day cycle, with the next leg up to September 10 starting then. Many bears who missed the rally are now following Henry Blodget in claiming victory by having stayed out of this “sucker’s rally.” It won’t take much more than one more big drop into options expiration this Friday to get the weekend news stories talking about false rallies, Great Depression comparisons, W-shaped recoveries, increasing unemployment rates, increasing foreclosures – did I miss any other negatives that are widely-known and thoroughly discounted by the markets already?

After breaking 990-995, it is very typical to see a test back up to the breakdown area. The energy for this type of rebound

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