FDA to ramp up cell and gene therapy activity as tidal wave of new products approaches

FDA to ramp up cell and gene therapy activity as tidal wave of new products approaches

In a statement, Commissioner Scott Gottlieb said the agency anticipates more than 200 cell and gene therapy INDs per year by 2020, and 10-20 approvals annually by 2025.

The Food and Drug Administration plans to add 50 new staffers to its clinical review group for cell and gene therapies as it anticipates a surge in new products entering the clinic and the market over the next several years.

In a statement Tuesday, FDA Commissioner Scott Gottlieb said that by next year, the agency will receive on an annual basis more than 200 Investigational New Drug applications – used by companies to get regulatory approval to start clinical trials – and approving 10-20 cell and gene products per year by 2025.

“The activity reflects a turning point in the development of these technologies and their application to human health,” Gottlieb’s statement read. “It’s similar to the period marking an acceleration in the development of antibody drugs in the late 1990s, and the mainstreaming of monoclonal antibodies as the backbone of modern treatment regimens.”

That picture stands in stark contrast to the current roster of approved cell and gene therapies. Those consist of two CAR-T cell therapies for blood cancers – Novartis’s Kymriah (tisagenlecleucel) and Gilead Sciences’ Yescarta (axicabtagene ciloleucel) – and one gene therapy, Spark Therapeutics’ Luxturna (voretigene neparvovec-rzyl), for a rare, inherited form of blindness.

For the application review of Kymriah’s initial approval in August 2017, for acute lymphoblastic leukemia in children and young adults, the agency convened the Oncologic Drugs Advisory Committee, a panel of outside experts who offer advice on approvals when the agency requests it. But it did not do so for Yescarta’s approval two months later for diffuse large B-cell lymphoma in adults, nor did it for Kymriah in DLBCL – the relative ease of the latter approvals indicating the agency had quickly become more comfortable approving the then-unprecedented cell therapies.

But Gottlieb noted there are now more than 800 active INDs for cell and gene therapies on file with the FDA. In a panel discussion at last week’s Biotech Showcase in San Francisco, which coincides with the annual J.P. Morgan Healthcare Conference, Iovance Biotherapeutics CEO Maria Fardis noted that competition in CAR-T therapy is heating up at clinical trial centers, making it harder to recruit patients. She was speaking in the context of CAR-Ts for solid tumors, which remain a much less well-established area of the field than blood cancers. Other types of cell therapies are in development as well, including T-cell receptors and tumor-infiltrating lymphocytes, respectively also known as TCRs and TILs.

Of course, the announcement took place against the background of the ongoing government shutdown. As a result, the FDA is currently only able to perform activities covered by user fees paid before the impasse, but is unable to perform many activities for which user fees had not yet been paid.

Several cell and gene therapy products are expected to reach the market in the near term. Celgene anticipates two CAR-T filings: bb2121 in multiple myeloma; and lisocabtagene maraleucel, which it acquired when it bought Juno Therapeutics last year. Both therapies were touted as near-term opportunities in Bristol-Myers Squibb’s recent $74 billion acquisition of Celgene. Separately, bluebird also anticipates approval in the US by next year for LentiGlobin for transfusion-dependent beta-thalassemia. BioMarin Pharmaceutical also anticipates filing for approval of valoctocogene roxaparvovec in hemophilia A.

 

Med School Grads Go to Work for Hedge Funds

https://www.bloomberg.com/news/articles/2017-09-05/med-school-grads-go-to-work-for-hedge-funds

 

More are starting biotech companies or joining consulting or financial firms instead of practicing—all while the U.S. suffers a shortage of doctors.

Matthew Alkaitis, a third-year student at Harvard Medical School, is calm, friendly, and a good listener—the kind of qualities you’d want in a doctor. But though he spends 14 hours a day studying for his board exams, the 29-year-old isn’t sure how long he’ll be wearing a white coat. In September, Alkaitis, who also has a Ph.D. in biomedical sciences, will be starting a two-year fellowship at McKinsey & Co., where he’ll be advising clients in the health-care field. “I really hope that my career involves a period of dedicated time taking care of patients,” he says. “But I also have this competing goal to one day start or help build out a company that really adds something new and interesting and innovative to the medical system.”

Like Alkaitis, more people are coming out of medical school and choosing not to practice medicine. Instead, they’re going into business—starting biotech and medical device companies, working at private equity firms, or doing consulting. In a 2016 survey of more than 17,000 med school grads by the Physicians Foundation and health-care recruitment firm Merritt Hawkins, 13.5 percent said they planned to seek a nonclinical job within three years. That’s up from 9.9 percent in 2012. A separate Merritt Hawkins survey asks final-year residents: “If you were to begin your education again, would you study medicine or would you select another field?” In 2015, 25 percent answered “another field,” up from 8 percent in 2006. Among the reasons they cited: a lack of free time, educational debt, and the hassle of dealing with insurance companies and other third-party payers.

The trend is worrying, as the U.S. already suffers a shortage of doctors, especially in rural areas. “If you have a large number of people out training to see patients and taking care of people in our communities, then all of a sudden deciding not to, that’s a concern,” says Atul Grover, executive vice president of the Association of American Medical Colleges. The AAMC projects a nationwide deficit of as many as 100,000 doctors by 2030.

“I think that we are at a crossroads,” says Dr. Kevin Campbell, a cardiologist in Raleigh, N.C. “I trained in the early ’90s, and back then you definitely were thought of as a sellout or a second-class citizen if you weren’t going into clinical medicine.”

Medical students have more options nowadays. Medical and business schools are teaming up to offer joint degrees. There were 148 students enrolled in M.D.-MBA programs in 2016, up from 61 in 2003, according to the AAMC. At Harvard Medical School, in a class of about 160 students, about 14 will pursue the joint degree, and an additional 25 or 30 will do master’s in other areas, such as law and public policy. “We have some students who want to go back to the Midwest and practice in a community setting,” says Dr. Anthony D’Amico, a professor of radiation oncology at Harvard Medical School and an advisory dean. And then there are those “who want to implement skill sets they’ve been blessed with and apply them on a broader scale.”

Dr. Rodney Altman of San Francisco says the time he spends treating patients in the emergency room informs his work as a managing director at Spindletop Capital, a private equity firm that invests in health-care companies. “I really wanted to practice health care on a macro level,” says Altman. “For me the one-on-one interaction with patients, while important and rewarding, wouldn’t have been as rewarding as being able to impact a larger number of patients.”

Altman says his mentors and colleagues had mixed feelings when, after a decade of practicing full time, he decided to dial back his hours in the emergency room. “Most people were supportive, a lot were envious, and some appropriately cautioned me about the risks I would be taking,” he says. “Out in the business world, you’re subject to the whims of the capital markets and to a lot more that is out of one’s control. I think medicine is quite safe and secure in that way.”

Some consulting companies are also stepping up hiring of doctors. Steffi Langner, a spokeswoman for McKinsey, says her firm is actively recruiting doctors because the analytical skills necessary to be an M.D. are similar to the problem-solving skills a consultant needs.

Dr. Jon Bloom trained as an anesthesiologist and practiced for three months, then enrolled at Massachusetts Institute of Technology’s Sloan School of Management. He says he was inspired by other doctors he knew who were inventors and entrepreneurs. One reason more are choosing that path is that investors are willing to fund them. Figures compiled by the National Venture Capital Associationshow that investment in medical-related startups climbed from $9.4 billion in 2007 to $11.9 billion in 2016.

Bloom is co-founder and chief executive officer of Podimetrics, a startup in Somerville, Mass., that has developed a mat device that predicts and prevents diabetic foot ulcers. He says that even though his invention is now on the market after receiving approval from the U.S. Food and Drug Administration in 2015, he’s still living the startup life. “I definitely don’t make nearly as much as what a doctor makes. That wasn’t really important to me,” he says. “My friends who graduated residency many years ago, they have multiple cars, fabulous houses. They did OK. I still occasionally eat ramen noodles,” he chuckles.

BOTTOM LINE – A U.S. deficit of doctors may worsen as a growing minority of medical school grads are choosing other professions.

Theranos agrees not to operate labs for two years

http://www.sfgate.com/business/article/Theranos-agrees-not-to-operate-labs-for-two-years-11079274.php

The Theranos lab in Newark Calif., seen on April 12, 2015. The company announced a settlement Monday with the Centers for Medicare and Medicaid Services that resolves all legal and regulatory proceedings between the federal agency and the embattled Palo Alto blood diagnostics firm. Photo: CARLOS CHAVARRIA, NYT

Theranos has reached a settlement with the Centers for Medicare and Medicaid Services that resolves all legal and regulatory proceedings between the federal agency and the embattled Palo Alto blood diagnostics firm, the company announced Monday.

Theranos has agreed to pay a penalty of $30,000 and cannot operate a clinical laboratory for the next two years.

As part of the settlement, the federal agency, which regulates blood testing labs, has withdrawn its revocation of Theranos’ lab operating certification.

Theranos, founded in 2003 by CEO Elizabeth Holmes, had been a high-flying startup that promised to revolutionize blood testing before a Wall Street Journal investigation alleged that the company misled people about the accuracy of its blood testing technology.

It is unclear whether the settlement has any bearing on investigations into the company by the Department of Justice and U.S. Securities and Exchange Commission. The company faces lawsuits from investors and Walgreens, its ex-partner that had been using Theranos blood-testing technology in dozens of stores before terminating the relationship.

Soon-Shiong made ‘implicit threat’ to spur investment in NantHealth, media company says

Soon-Shiong made ‘implicit threat’ to spur investment in NantHealth, media company says

When Dr. Patrick Soon-Shiong invested in the troubled media company Tronc, he was greeted as a white knight. But a lawyer for Tronc is now accusing Soon-Shiong of making an “implicit threat” that he would abandon the company unless Tronc invested in his own medical diagnostics startup, NantHealth.

Soon-Shiong helped rescue Tronc from a hostile takeover attempt last May with an investment of more than $70 million. But he first tried to convince the publishing company to invest in the initial public offering of NantHealth, according to a letter from Tronc’s attorneys filed with the Securities and Exchange Commission.

“Tronc properly declined to invest in that company, since such an investment would have had no logical connection to its business operations in the publishing industry,” the attorneys wrote.

When the company declined, Soon-Shiong insisted that Michael Ferro, Tronc’s chairman, invest in NantHealth, either personally or through his private equity firm, Merrick Ventures, the attorneys wrote.

“The implicit threat was that, if Merrick did not invest in NantHealth, Dr. Soon-Shiong would not invest in Tronc,” the Tronc legal team wrote.

Ferro bought a $10 million stake in NantHealth “so that Tronc did not have to,” according to the attorneys. NantHealth went public last June, to considerable fanfare, but its stock price has since fallen by more than 60 percent.

NantHealth did not respond to a request for comment.

With NantHealth, Soon-Shiong has promised to revolutionize cancer diagnostics through a proprietary technology called GPS Cancer. But the company has struggled to gain traction, posting disappointing sales numbers and losing $184 million in 2016.

And Soon-Shiong’s management of the company has come under increasing scrutiny. Last month, a STAT investigation found that he used his highly promoted cancer moonshot initiative as a marketing vehicle for GPS Cancer. A second investigation described how Soon-Shiong used a $12 million charitable gift to funnel business into NantHealth.

The company’s stock price declined after each report, and NantHealth is now facing lawsuits from investors claiming Soon-Shiong misrepresented facts and violated securities law.

Discovery enables ‘mass produced blood’

http://www.bbc.com/news/health-39354627

Blood

Scientists say they have made a significant leap towards mass-producing red blood cells suitable for donation.

Red blood cells can already be made in the lab, but the problem is scale.

A team at the University of Bristol and NHS Blood and Transplant have developed a method to produce an unlimited supply.

The artificial blood will be far more expensive than conventional donation. So it is likely to be used for people with very rare blood types.

The old technique involved taking a type of stem cell that manufactures red blood cells in the body and coaxing it to do so in the lab.

However, each cell eventually burns out and produces no more than 50,000 red blood cells.

The trick developed by the Bristol team was to trap the stem cells at an early stage where they grow in number indefinitely.

It is known as making them immortal.

Once the researchers have this group of cells, they can just trigger them to become red blood cells.

Dr Jan Frayne, one of the researchers, said: “We have demonstrated a feasible way to sustainably manufacture red cells for clinical use.

“We’ve grown litres of it.”

How the world’s richest doctor gave away millions — then steered the cash back to his company

How the world’s richest doctor gave away millions — then steered the cash back to his company

Image result for biotech laundry

He was greeted like a star philanthropist.

The world’s richest doctor had just made a $12 million gift to the University of Utah. Members of the university community were urged to come thank him. And so, a crowd gathered.

For months, Dr. Patrick Soon-Shiong would continue to reap praise for his generosity in publicity put out by the university. Not mentioned in any of the tributes: $10 million of his donation would be sent right back to one of his companies. And the contract for his gift was worded in a way that left the University of Utah with no other choice.

The university health system did get free and valuable information for genetics research through the deal. But a STAT investigation has found that Soon-Shiong benefited even more from his charitable donation.

He got reams of patient data to help him build a new commercial product meant to assess patients’ risk of rare and inherited diseases. He got a stream of cash for one of his struggling companies.

And the deal made it possible for his company to inflate, by more than 50 percent, the number of test orders it reported to investors late last year while updating them on interest in a flagship product, a diagnostic tool known as GPS Cancer. Soon-Shiong’s team counted genetic sequencing ordered by the University of Utah in those order numbers — even though the work for the university did not have anything to do with diagnosing or recommending treatments for cancer patients.

Even in the world of academic donations, which the wealthy often use to burnish their image or advance pet causes, the arrangement stands out as highly unusual.

STAT has previously detailed how Soon-Shiong’s high-profile cancer moonshot initiative achieved little scientific progress in its first year, instead functioning primarily as a marketing tool for GPS Cancer.

The University of Utah deal — laid out in contracts obtained by STAT through a public records request — illustrates how Soon-Shiong boosted his business through his philanthropy. He has been accused of doing just that in at least two legal filings, but the Utah contracts offer the first concrete example, spelled out in black and white.

Four tax experts who reviewed the contracts at STAT’s request all agreed that the Utah deal was suspicious. Two said it appeared to violate federal tax rules governing certain charitable donations, amounting to indirect self-dealing by Soon-Shiong and his foundations.

“They’re laundering the funds through the University of Utah,” said Marc Owens, a tax lawyer with Loeb & Loeb. Owens, who said the contracts appeared to violate federal rules, previously spent a decade as head of the Internal Revenue Service’s tax-exempt division.

The other two legal experts said the contracts were cleverly worded in a way that would likely steer clear of self-dealing — but agreed that, at the very least, they raised serious questions about Soon-Shiong’s intent.

“We pretty clearly have an optics problem,” said Morey Ward, a tax lawyer with Ropes & Gray who represents tax-exempt organizations.

Soon-Shiong’s spokeswoman, Jen Hodson, did not answer a list of emailed questions or return calls from STAT. Soon-Shiong has denied STAT’s repeated requests for an interview dating back to last fall.

 

Biotech billionaire in talks with Trump about a senior health care role, sources say

Biotech billionaire in talks with Trump about a senior health care role, sources say

Dr. Patrick Soon-Shiong, an audacious biotech billionaire who has pledged to “solve health care,” has been in talks with the Trump administration about the possibility of serving in a senior role overseeing the US health care system, according to individuals familiar with the discussions.

Soon-Shiong, a trained surgeon, has met with President Trump and his advisers at least twice in recent weeks. During those discussions, he raised the possibility that he could serve as a “health care czar” with a broad portfolio in the administration as it seeks to reshape the health care system and replace the Affordable Care Act, according to two individuals, who spoke on the condition of anonymity.

Asked about the discussions, an adviser to Soon-Shiong told STAT that the word “czar” had not been used but did not dispute that the biotech mogul has discussed the possibility of taking on a senior role overseeing health care in the Trump administration.

Healthcare’s $40B day: 5 things to know about Thursday’s mega-mergers

http://www.beckershospitalreview.com/hospital-transactions-and-valuation/healthcare-s-40b-day-5-things-to-know-about-thursday-s-mega-mergers.html

Mergers_and_Acquisitions_Tablet_1920x960

Some of the deal activity among medical device makers and pharmaceutical companies can be explained by the belief that gaining scale and becoming a leading provider is essential for effectively negotiating business with hospitals and insurers, according to The New York Times. Some of the impetus to consolidate can also be explained by the desire to acquire emerging product lines and biotech firms to supplement the declining sales of older products.

Moody’s breaks down challenges facing healthcare organizations in 2016

http://www.healthcarefinancenews.com/slideshow/moodys-explores-challenges-facing-healthcare-organizations?mkt_tok=eyJpIjoiTjJZMU5UQXhNVFJsWWpSaSIsInQiOiJidFZ1RUN4NjdcLzdLK3VFQTVIeGdxSnNWZHZxdThQemM5a2RScVoxTGRtVmFYUjZkeTl0Rms1U1J0SFlDeW52WmUrWjcyODFvaFFXZkl6aUZZSFB2TGZcL3Y4a3ZrUVpwaFNOOUdjanhlNWZZPSJ9

 

America’s Secret Weapon

https://www.linkedin.com/pulse/americas-secret-weapon-bill-gates?trk=eml-b2_content_ecosystem_digest-hero-14-null&midToken=AQHcpzuK5TbmJA&fromEmail=fromEmail&ut=3lEZJsOEg1ATc1

America’s Secret Weapon

What is new is that more countries than ever are competing for global leadership, and they know the value of innovation. Since 2000, South Korea’s research and development spending (measured as a percentage of GDP) has gone up 90 percent. China’s has doubled. The United States’ has essentially flatlined. It’s great that the rest of the world is committing more, but if the United States is going to maintain its leading role, it needs to up its game.