Skip to content

PTC Duchenne med application dismissed by FDA panel

PTC Therapeutics Inc has not supplied persuasive evidence that its experimental drug to treat a form of Duchenne muscular dystrophy is effective, a preliminary review by scientists at the U.S. Food and Drug Administration concluded.

The review, posted on the FDA’s website on Tuesday, said data to establish the effectiveness of the drug, ataluren, “are not persuasive.”

The FDA’s comments come two days ahead of a meeting of outside advisors who will discuss ataluren. The agency has asked its advisors to decide whether or not the drug is effective or whether the data is inconclusive.

The FDA is not obliged to follow the recommendation of its advisors but typically does.

https://yhoo.it/2yEIkAv

Advertisements

As with welfare reform, states can fix ObamaCare’s ills

Back in 1996, a Republican Congress and a Democratic president (Bill Clinton) enacted one of the most historic bipartisan policy victories in modern times: the Personal Responsibility and Work Opportunity Reconciliation Act for welfare reform.

The passage of that bill has so many parallels to the fight over the Graham-Cassidy ObamaCare-reform legislation now before Congress that it is worth recounting some of the lessons.

Just as with the Graham-Cassidy bill, the ’96 welfare reform bill gave block grants to the states so they could operate their own welfare systems. The basic federal provisions encouraged tough love: requiring recipients to begin working within two years of receiving benefits; capping benefits at a lifetime total of five years; and strengthening the enforcement of child support. Most importantly, an open-ended federal entitlement was capped, so states had incentives to run their programs cost-effectively.

When that bill passed, liberals argued, as they do now regarding Graham-Cassidy, that people would die as a result. Rep. Maxine Waters warned that the law would cause “hungry children (to) starve.”

But of course, welfare reform worked far better than even supporters expected. Ron Haskins, an architect of that bill and now a welfare expert at the Brookings Institution, found: “Welfare rolls plunged by over 60%, as many as two million mothers entered the labor force, earnings for females heading families increased while their income from welfare payments fell, and child poverty declined every year between 1993 and 2000.”

Jimmy Kimmel, are you listening? Does that sound like the sort of result to ruin the lives of poor people and cause the sick to go without treatments?

One reason welfare reform succeeded is that we allowed 50 governors to come up with innovative solutions to end a corrosive federal welfare system that had financially incentivized out-of-wedlock births and trapped so many millions of low-income families in a cycle of poverty. We learned that state legislators are not heartless Scrooges.

The Graham-Cassidy bill also turns a welfare program — Medicaid — over to the states. States will get a lump sum of money, and they will be required to set up programs to provide insurance coverage for their eligible low-income residents. Contrary to what the histrionic media might have you believe, the bill requires states to find ways to cover residents who have pre-existing conditions.

A similar experimental block grant waiver given to Rhode Island back in 2000 had spectacular successes. The state covered more people, improved care and rooted out tens of millions of dollars of wasteful spending. It is a shining example of how the Graham-Cassidy reforms could transform our Medicaid system to benefit everyone.

There is one other critical political lesson from the ’96 welfare-reform policy victory. From the very start, Republicans and moderate Democrats (they existed back then) adopted a smart strategy that proved decisive. Welfare reform was never sold as a way to save money. It was about saving lives. It was about lifting poor families out of poverty and life on public assistance and into the fulfillment, dignity and upward mobility of employment.

One strategic mistake of the Graham-Cassidy bill is the infatuation with cutting the Medicaid budget. The White House and GOP talking points boast that the proposed legislation cuts spending and the deficit by hundreds of billions of dollars. Focusing on this angle plays into the hands of liberal critics, who say that 10 million Americans will lose their health insurance and that children with pre-existing conditions will lose treatments. In actuality, no one needs to lose his or her insurance under the GOP plan.

We should plow all those savings into a fund for pre-existing conditions and reimbursing states that lose money in this transition.

This isn’t about saving money or cutting the deficit. It’s about creating a 21st century health care system that taps into the power of free markets, consumer choice and competition to expand coverage, improve care and make health insurance affordable to everyone. Hopefully, even Jimmy Kimmel couldn’t be against that.

http://bit.ly/2fpGACM

Equifax hack effects likely to hang around for years, like chronic pain

Like many of you, I still feel blindsided by the Equifax hack. Knowing my private information is now in the hands of criminals with long-term plans—identity sales on the dark web haven’t spiked following the theft—is unsettling.

It’s also infuriating. I never gave my information to Equifax. The company (like the other two big credit bureaus, TransUnion and Experian) gathered Social Security numbers, driver’s license numbers, dates of birth, employment histories, credit cards and other sensitive details without explicit permission from 143 million people, including me and, if you’re an American, probably you.

The cost for consumers probably won’t be known for a while. We talked with previous victims of identity theft in this week’s Decrypted podcast. While their experiences varied—thieves committed felonies, got cancer treatment and delivered a baby using fake names—they shared the common experience of enduring a string of awful incidents. One victim compared it with chronic pain.

And the thing that really stings? Equifax makes much of its $3 billion or so a year by selling personal information to financial institutions when they want to determine my and other people’s credit worthiness. Even more insulting was how thoroughly Equifax bungled its response, first putting up a WordPress site (rather than a link off its main page), then tweeting out a phishing link instead of the correct page for customers to find out whether their  information had been compromised. Equifax has since deleted the tweet, and the phishing site has been taken down (luckily it was a security expert making a point, and he didn’t use the information), but the missteps further erode trust.

So does the backstory. As Bloomberg reported, the company had been hacked as early as March by the same group responsible for the July hack that led to the massive breach. Several top executives sold stock totaling $1.7 million days before the hack was announced and the board appeared, let’s say, disengaged. (The company said the executives didn’t know about the hack.) I’m eager to see what the Consumer Financial Protection Bureau and the Federal Trade Commission (which are working with the FBI) uncover as well as investigations by nearly three dozen state attorneys general.

Equifax has lost about a quarter of its value since announcing the breach earlier this month, but the final cost will certainly be higher once fines are assessed, lawsuits settled and the damage to the company’s reputation takes hold.

Identity theft affects 16 million or so victims in the U.S. each year, according to national nonprofit Identity Theft Resource Center. Incidents emerge months, years and even decades later following the initial theft. Ensuring your information is secure requires monthly checking of credit reports and spending a lot of time and energy on the phone and in person if it’s not. That cost, to each of our peace of minds, is tough to quantify but will be inevitably higher.

https://bloom.bg/2yEdKHm

Axovant Alzheimer’s med fails late stage trial

Axovant Sciences said its Alzheimer’s medicine had failed a late-stage clinical trial, extending a 14-year losing streak for companies trying to develop new drugs for the devastating form of dementia. The New York-based company said there was “essentially no difference” between its drug, known as intepirdine, and the placebo in the study of 1,150 patients on tests that measured a person’s ability to carry out daily activities like dressing or bathing.

Nor did the drug have a significant effect on brain power.  Shares plummeted almost 70 per cent to $7.37 in pre-market trading, wiping $1.8bn from its market capitalisation.

“While we are deeply disappointed by these trial results, we also are saddened for the millions of patients and families impacted by Alzheimer’s disease,” said David Hung, chief executive officer of Axovant. “However, we believe that the fight against Alzheimer’s and other important areas of unmet need in neurology is too important to be derailed by this setback.”

In a note published before the announcement, Michael Yee, a Jefferies analysts, predicted the stock price could fall as low as $3 per share if the study failed to show a benefit in either brain power or daily activities.

The failure of the drug is the latest in a string of Alzheimer’s flops and follows high-profile setbacks in recent months for companies including Eli Lilly, Merck and Lundbeck.

Pharma companies have spent billions of dollars on a fruitless search to discover new medicines for Alzheimer’s, tempted by the economics of finding a drug for a disease that affects nearly 44m worldwide.

Just four medicines have been approved for Alzheimer’s patients by the US Food and Drug Administration and the last time a new drug was given a green light was in 2003.

Some doctors and analysts believe that drugmakers are pressing ahead with trials without fully understanding the science underpinning the disease.

Axovant bought the rights to intepirdine from GlaxoSmithKline in 2014 for a relatively small upfront payment of $5m before pulling off what was then a record-breaking initial public offering in 2015 with a valuation that almost touched $3bn.  Some investors questioned why the company was able to secure such a lofty valuation on the back of a drug that had consistently flopped in trials at GSK and warned the IPO was a sign of a “biotech bubble”.

GSK had shelved the medicine after it failed in a string of mid-stage clinical studies, but Axovant’s 32-year-old founder, Vivek Ramaswamy, believed he had spotted a glimmer of hope in one study that suggested the medicine might work when added to an existing therapy called Aricept.

Shares in Axovant rallied in April after it appointed Dr Hung, a respected biotech veteran, as its chief executive in a move that some shareholders interpreted as a sign of confidence that the medicine would succeed.

Analysts had said the medicine could have generated up to $4bn in sales a year if it had been given a green light by the US Food and Drug Administration.

Axovant is also testing intepirdine in patients with a condition known as lewy body dementia as well as patients whose dementia is affecting their balance and gait. It said those studies would continue, as would trials of its other medicines.

http://on.ft.com/2fw3KeA

Drug maker Alvogen’s owners eye strategic options

  • Shanghai Pharma holds on-and-off talks on potential purchase
  • Discussions center on generic pharma company’s U.S. operations

Alvogen’s private equity owners including CVC Capital Partners are exploring options for the generic pharmaceutical company, which could be valued at about $4 billion, according to people familiar with the matter.

The controlling shareholders of the drugmaker have held on-and-off talks with Shanghai Pharmaceuticals Holding Co. focusing on a sale of Alvogen’s U.S. business, the people said. Alvogen would keep its operations in Asia as well as central and eastern Europe, one of the people said, asking not to be identified because the information is private.

While they are not running a formal auction process, Alvogen’s owners may look to hold discussions with other international buyers, the people said. Deliberations are at an early stage, and there’s no certainty they will lead to a transaction, according to the people.

A consortium including CVC and Singapore state investment company Temasek Holdings Pte bought a controlling stake in Alvogen in 2015 for about $2 billion, people with knowledge of the matter said at the time.

Shanghai Pharma has stepped up its hunt for investments after missing out on the purchase of German generic drugmaker Stada Arzneimittel AG. The company is currently bidding for a stake in Arbor Pharmaceuticals LLC as well as Cardinal Health Inc.’s Chinese distribution business, people familiar with the matter have said.

Manufacturing Facilities

Representatives for CVC and Shanghai Pharma declined to comment. A spokesman for Alvogen didn’t answer a phone call and an email seeking comment outside of regular business hours. Temasek said in an emailed statement it doesn’t comment on rumors or speculation.

The biggest market for Alvogen is the U.S., where it sells generic pharmaceuticals and provides third-party services such as contract manufacturing and clinical research. It operates a manufacturing facility in Norwich, New York under the Norwich Pharmaceuticals brand.

Alvogen has 350 marketed products spanning a range of therapeutic areas including oncology, cardiology, respiratory and neurology, according to its website. It develops, manufactures and sells generic drugs, branded medicines, biosimilar products and over-the-counter products like cosmetics and food supplements.

The company also has a plant in Romania and a packaging center in Serbia. It has made three acquisitions since 2012 in Taiwan, where it operates under the Lotus Pharmaceuticals brand, and South Korea combined. Part of its Asian operations are listed as Seoul-traded Alvogen Korea Co., which has a market value of about $286 million.

https://bloom.bg/2ypxMEx

Lannett chief Bedrosian to step down

Lannett Company, Inc. (NYSE: LCI) today announced that its board of directors has begun a search for a new chief executive officer (CEO). As part of the unanimously approved search process, the board has retained a leading executive search firm to identify potential candidates to assume the role of CEO, succeeding Arthur Bedrosian, who has agreed to step down once a new CEO is appointed. During the search process, Bedrosian will remain a member of the board of directors. Following the appointment of a successor, it is expected that Bedrosian will remain with the company in a strategic advisory role, ensuring a seamless transition of leadership responsibilities.

 

The company also announced that it has filed a 505(b)(2) New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) for Cocaine Hydrochloride (HCl) Topical Solution, the company’s proprietary local anesthetic product. The filing is the company’s first 505(b)(2) NDA submission to include full clinical trial studies in its history. Cocaine HCl Topical Solution is a liquid formulation of cocaine hydrochloride, with a proposed indication for the introduction of local (topical) anesthesia for diagnostic procedures and surgeries on or through the accessible mucous membranes of the nasal cavities. The 505(b)(2) NDA submission is supported by two Phase III, randomized, double-blind, placebo-controlled, multicenter studies in several hundred patients, as well as a Phase I pharmacokinetic study.

“Today’s announcement regarding the filing of the NDA for C-Topical® is among the highlights of my tenure at Lannett,” said Bedrosian. “I believe the company is strong financially and operationally, and has excellent prospects for continued success. We have built a solid core business, exemplary R&D leadership and a regulatory department that has worked tirelessly to assemble the NDA for C-Topical®. In addition to the ANDAs on file with FDA, we have put in place multiple initiatives to further grow the company for years to come.”

Jeffrey Farber, Lannett’s chairman, said, “The Board extends its deep appreciation to Arthur for his outstanding leadership over the past 11 years as CEO, his 15 years as an officer and 13 years as a director. During his tenure, Arthur significantly increased the size of the company through organic growth and acquisitions, with revenues climbing to $637 million for fiscal 2017 from $25 million for fiscal 2002. As CEO and president, he formed key strategic alliances, established and advanced our pain management business, forged a robust product development program and oversaw an extraordinary record of regulatory compliance, as well as the company’s evolution into an industry leader. We look forward to benefiting from Arthur’s continued leadership and insights during the transition and beyond. The board’s search for Arthur’s successor is in the early stages.”

Bedrosian has served as Lannett’s CEO since 2006. He was named president of the company in 2002, after a short stint as its vice president of business development. Bedrosian has been a member of the Lannett Board of Directors since 2006; he also served as a director from 2000 to 2002.

http://bit.ly/2xHUXNh

Why Amgen Neulasta might not get FDA OK

Jefferies analyst, Michael Yee, reiterated his Buy rating on shares of Amgen (NASDAQ: AMGN) noting that 2018-2020 consensus may be too low if the Mylan biosimilar, Neulasta, is not approved next month. He believes this would set shares up for a higher multiple like ABBV.

The analyst does not cite evidence that it will not be approved but offers several reasons why it MIGHT not be:

1) no adcom panel for any of the Neulasta biosimilars

2) three prior ones were already rejected due to various issues from CHRS, Apotex, Sandoz

3) MYL has commented they don’t include it in their 2018 planning and had mixed outlook on it

4) MYL BioCon manufacturer has had known issues recently

5) MYL recently pulled their EMA application for Neulasta citing “cGMP clearance of the drug product manufacturing facility was not obtained within current clock-stop.”

No change to the price target of $200.

http://bit.ly/2xHjQZs