Prana Biotechnology (PRAN)

[interestingtome]

Very good for Prana not good for Biogen neccessarily -- "small group of patients sharing certain genetic traits" for Biogen the worst SAEs were in ApoE4 positives. We know that with 77% of the IMAGINE population ApoE4 positive and no reports of ARIA-E and greater than 2.5 SUVR they saw significant reductions this approach would benefit PBT2! Very important distinction between Biogen drug and PBT2. Typically around 65% of the AD population is ApoE4 positive.

and, hmmm "refining them and obtaining regulatory approval for their sale in the EU," sounds like that might be a possibility with EMA approval around the corner likely by the end of May.

Rx for Patients and Big Pharma: Speeding Up the FDA
The 21st Century Cures Act would speed the drug-approval process and allow the desperately ill to try promising experimental cures.

By JIM MCTAGUE
May 8, 2015 7:35 p.m. ET
Congress is racing to produce a panacea for all that ails us—including nettlesome barriers to investment in the pharmaceutical and medical-device sector. The 21st Century Cures Act is rapidly moving through the House Energy and Commerce Committee with bipartisan support. It’s viewed by medical professionals as the most revolutionary change to the Food and Drug Administration’s approval process since the National Cancer Act in 1971, in which Congress and President Richard Nixon declared all-out war on that dread disease.
The intention is to mark up the Cures Act by May 30 for a floor vote in June. A Senate committee has taken up a complementary bill. The rosy scenario: The House and Senate pass a reform bill before the end of the summer.
But that would not be the end of it. An appropriations bill would have to pass in both chambers to fund the new measure. The House bill increases funding for the National Institutes of Health, from $30.3 billion now to $31.8 billion in 2016, $33.3 billion in 2017, and $34.8 billion in 2018. Even the most cockeyed optimist can anticipate resistance to a budget increase by the House’s vocal bloc of fiscal conservatives. But the bipartisan House coalition that supports the measure will no doubt roll them, with the bill arriving on the president’s desk for his signature by fall.
BOTH THE HOUSE AND SENATE MEASURES aim to streamline the discovery-development-delivery cycle to accelerate the pace of cures, without introducing recklessness into the process. Speeding research and approval, in turn, should significantly lower the costs and risks of developing medicines, former FDA Commissioner Andrew von Eschenbach told me last Wednesday at an event sponsored by the nonprofit Alliance for Health Reform.
A faster cycle, he said, should particularly appeal to venture investors, who have been moving away from the space because of skyrocketing development costs and lengthy wait times that often are a consequence of an outmoded FDA approval process. “When the business model makes sense, then the whole system benefits,” he said. Capital, as Barron’s readers certainly appreciate, makes civilization go round.
The pharmaceutical, biotech, and life-science industries, of course, have been among the stock market’s brightest stars this year—in part, I suspect, because of favorable prospects for this legislation. Although Republicans and Democrats disagree on almost everything, both parties and the president strongly support improvements to the plodding, overly cautious FDA approval process. Nearly as important, the House bill also has the vocal backing of patient groups and pharmaceutical and medical-device manufacturers. Credit this to the efforts of the bill’s sponsors—Fred Upton, the Michigan Republican who chairs Energy and Commerce, and Diana DeGette, a Colorado Democrat on the committee—for engaging all of the stakeholders last year.
We the patients, of course, are intended to be the primary beneficiaries of the legislation. But there are economic benefits for the nation, as well. Absent an overhaul of the FDA’s approval process, there’s compelling reason for pharmaceutical and medical-device makers to shift some research jobs from the U.S. to the European Union, which boasts a less burdensome approval regime.
In fact, von Eschenbach wrote in a 2012 Wall Street Journal op-ed piece that a shift was under way. He cited a 2012 California survey of life-science CEOs, which found that 80% of respondents didn’t consider the FDA’s approval process to be the world’s best and that 81% believed that by 2017, some other country could surpass the U.S. as the nation that bestows a sort of global Good Housekeeping seal, potentially engendering a mass exodus of research-and-development jobs.
An expert tells me that some clinical-device manufacturers are seriously discussing developing their products in the U.S., refining them and obtaining regulatory approval for their sale in the EU, and then manufacturing and hawking the final product in China, which has the globe’s fastest-growing middle class. The companies are reluctant to pull all of their R&D from the U.S. because we still lead the world in intellectual capital by virtue of our universities and cancer and research centers. No other country can match our current research infrastructure.
THE MOST IMPORTANT PROPOSAL in the House bill is the required emphasis on “biomarkers” by the FDA when it is determining the efficacy of a drug or therapy. This would encourage the agency to approve a product that works in a small group of patients sharing certain genetic traits. The current process requires medicines to work reliably among a larger, more general clinical-trial population in order to be approved.
The approach is a keystone of the smaller, $215 million Precision Medicine Initiative that President Barack Obama unveiled in his 2015 State of the Union address. A one-size-fits-all-approach to developing treatments, the Obama administration said, can be “very successful for some patients, but not for others.” And the administration added, “This is changing with the emergence of precision medicine, an innovative approach to disease prevention and treatment that takes into account individual differences in people’s genes, environments, and lifestyles.”
To aid in the discovery of breakthroughs, red tape that discourages NIH scientists from traveling to conferences to share ideas would be cut. And a private-public nonprofit corporation with NIH representation would be established to foster scientific collaboration among the research community. During the product-development phase, companies would interact with the FDA early, to ensure that their products were in line with regulatory expectations. The aim would be to reduce the multiple rounds of FDA reviews that often occur. And during the development cycle, desperately ill persons would have easier access to highly promising experimental drugs.
Upton told the New York Times in April that the bill is an opportunity for Congress to “do something big.” Make that “do something gigantic.”

http://online.barrons.com/articles/rx-for-patients-and-big-pharma-speeding-up-the-fda-1431128153