"Researchers at Massachusetts General Hospital are launching a 5-year clinical trial of a vaccine that may not only treat the disease, but actually reverse it. The trial will investigate whether the BCG vaccination - a drug that has been used to prevent tuberculosis for decades - can improve type 1 diabetes in adults.
The research team led by Dr. Denise Faustman has already completed a successful trial showing that the BCG vaccine reversed advanced type 1 diabetes in mice and a phase I human clinical trial establishing the safety and potential of the drug.
"We saw early signs that even at low doses of this vaccine the bad white blood cells that were killing the pancreas were killed, and also the good white blood cells that quiet down type 1 diabetes were up-regulated," Faustman told CBS Boston's Dr. Mallika Marshall. "
Well, if there are interims, and there should be and if they go as the earlier trials, it won't be anywhere near 5 years.
Today's news, not really new of course, is the trial of BCG to reverse type 1. Cheap, existing, drug. Could work for type 2. 5 year trial. Not run by Pharma, of course.
go read. the CTO allowed MNKD to redact the milestone specifics, and guess what? they aren't there.
"The remaining milestones are mostly sales oriented," Pfeffer said. "We're confident we'll get all those milestones. The question is how rapidly."
So, MNKD has eaten up all the "production" milestone payments?
-- Mannkind will be reimbursed for every last one of those Afrezza doses in the form of production milestone payments.
Last I recall, nothing was made public regarding the milestones: neither amounts nor triggers. Cite?
-- the science is still there and the science works
The problem is that any of the once a day pills are just as good, from a label point of view (which doesn't count any of the wonderful claims made by whoever), are just as effective and more convenient. Unless, and until, Afrezza gets a label with unique therapeutic benefit, it will be a niche product. Since MNKD got no up-front moolah, just a loan, turning the 35% (and, is that net profit or gross profit?) into enough to justify $2 billion will be a hard slog.
ummmm Clinton left the Damn Gummint in 2000. Bush and his trickle down cronies made you poor. Unless you're from the 1%, in which case you should be happy; you and your kind hold more wealth now than ever before. Not enough for you?
-- diabetics will never want to go back to old-school, slow insulin with needles!
And... what % of diabetics (T1 & T2) are needle dependent? Not much market there, and Al and MNKD know it. Getting a label stating therapeutic benefit (reduced co-morbidities, for example) unique to Afrezza is necessary. Without that, a pill a day for T2s will win.
-- So, what do you do with the diabetic population increasing from 387 to 600 million? Ignore it?
And, of course, all of that growth will be serviced by inhaling Afrezza rather than popping one a day of the increasing number of pills available. Ya think?
The only processes, linear or non-linear, which are predictably stable in progression are those following natural laws. Because the rules of the underlying process can't be changed. Human activities follow unpredictable trajectories just because some humans can change the rules at a whim. That's how we got the Great Recession. If one believed the data in 2006, then exponential growth, forever, in housing prices was obvious. Until it wasn't. Same here.
And... if SNY and MNKD saw exponential growth in the numbers, that's what they would have said, not that a slow control launch was slower and launchier than they had projected.
Well, unfortunately, compound growth isn't as sure as compound interest. Compound interest happens because that's the contract; the bank has to. Compound growth happens only because lots of events happen after not so much events. There is no cause and effect. Sorry.
-- In no way could anybody say the company didn't do what the FDA demanded.
Well, and there's that frivolous class action suit that MNKD decided wasn't worth defending and settled. The crux of the case was that MNKD lied about what FDA demanded and what MNKD said about the situation. Remember that? You can look it up, if you've forgotten.
-- The FDA pulled a fast one
Fact is, there's no way to know that. MNKD has never, unless you can produce an explicit cite, stated on the record that FDA screwed them. Nor has there been, ditto, FDA documents which demonstrate a rescinding of such an agreement.
We only have posturing from MNKD and Manniacs.
This is Al, reporting on the 2010 CRL:
"We are currently reviewing the Complete Response letter and fully expect that we will be able to respond to the FDA's requests in a timely manner," said Alfred Mann, Chairman and Chief Executive Officer. "We had always planned to follow the original NDA for AFREZZA with a regulatory submission for our next-generation inhaler rather than launch with the commercial version of the MedTone device. We will discuss with the FDA whether it is appropriate to use what would otherwise have been a supplemental NDA submission, which we had planned to make during the second quarter of this year, to address the agency's requests. If this approach is acceptable, we believe that this regulatory action will not have a significant impact on the timing of the commercial launch of AFREZZA. We will work closely with the FDA to answer quickly the agency's questions and satisfy the requirements. We are committed to working with the FDA to make AFREZZA available to patients as soon as possible."
So, who tried to sneak past the FDA, Al or Ed?
Sure. MNKD tried to sneak Medtone data for Dreamboat after they were told they couldn't do so. That's just one example.
"The principal issue raised by the FDA concerned the usage of in vitro performance data and clinical pharmacology data to bridge MannKind's next-generation inhaler to the phase 3 trials conducted using its MedTone(R) inhaler. "
That's the sanitized version they PR'ed 19 Jan. 2011.