Drug makers dismiss outrage over high prices as ‘abomination’

first_img And so it went at the drug industry’s biggest showcase, the J.P. Morgan Healthcare conference that’s running all week in the heart of this city’s financial district. As executives and investors shuttled from meeting to meeting, seeking deals, many dismissed public outrage at the industry as misguided.Public anger at drug companies is “an abomination,” Ron Cohen, chairman of the big industry group BIO, said at the Biotech Showcase.advertisement By Rebecca Robbins Jan. 12, 2016 Reprints BusinessDrug makers dismiss outrage over high prices as ‘abomination’ SAN FRANCISCO — The protesters carried handwritten signs accusing drug maker Gilead Sciences of greed for pricing its breakthrough hepatitis C drug at $84,000 per treatment.So it perhaps wasn’t surprising that during a panel discussion here Monday, a Gilead executive was asked how he lives with himself.Gregg Alton, the executive vice president for corporate and medical affairs, joked that he goes running. Then his tone turned serious as he talked about research, innovation, and the value of life-saving new drugs. “I sleep quite well,” he concluded.advertisement Related: Live Blog: Updates from JP Morgan Healthcare Conference But it was Gilead Sciences that was in the hot seat Monday. Outside the stately old hotel where the J.P. Morgan conference is being held, a handful of protesters marched with signs declaring “Gilead = Greed,” “Public Health Not Private Wealth,” and “Jail Gilead Drug Profiteers.” Gilead President John Milligan, however, said drug pricing is “more of a campaign issue than an actual issue,” the San Francisco Business Times reported.The company, based in Foster City, Calif., has drawn the ire of lawmakers, doctors, and patients for the the price of its hepatitis C drug Sovaldi. Gilead has made about $27 billion in sales for Sovaldi and a sister drug, Harvoni, since the former was introduced in 2013.Gilead defends the price by explaining that Sovaldi can cure patients of hepatitis C, preserving their health and eliminating the need for expensive treatments later in life, including liver transplants.The company was also keen on Monday to spotlight its work on other drugs.Milligan opened his presentation by talking about Gilead’s efforts to “help the globe” by developing and distributing medications for HIV, Ebola, and the parasitic disease visceral leishmaniasis, among other conditions.Gilead’s work on some of those drugs has drawn protests in the past, especially from AIDS activists concerned about access to the HIV medication. But Sovaldi has taken center stage lately. All the talk about pharma profiteering, Cohen said, is “a perversion of reality.”Many drug makers have raised prices in the past year.  And a slew of new drugs have hit the market with eye-popping price tags: cancer drugs at more than $11,000 a month; cholesterol drugs at more than $14,000 a year. Then there’s Martin Shkreli, the pharma executive who bought up a decades-old drug and hiked the price 5,000 percent, turning himself into a target of nationwide protests before he was arrested last month on securities fraud charges. Related: The price of the drug has become a burden not just for individual patients but also for the Medicaid system. Some states have begun restricting access to the drug to patients in the most dire condition. Last year the Kaiser Family Foundation estimated that Sovaldi had boosted Medicare’s annual drug costs by between $2 and $6.5 billion.In a poll conducted jointly this fall by STAT and and the Harvard T.H. Chan School of Public Health, more than 9 of 10 respondents said it was “unreasonable” to price a hypothetical hepatitis C drug in the manner that Gilead did. (That poll did not specifically name the company.)And a Senate investigation released last month found that Gilead knew its prices would make the drug inaccessible for many Americans. “If Gilead’s approach is the future of how blockbuster drugs are launched in America, it’s going to cost billions and billions of dollars to treat just a fraction of patients in America,” Senator Ron Wyden (D-Ore.) said at the time. Related: Activists protest the high price of Gilead drugs outside the J.P. Morgan Healthcare Conference in San Francisco. Rebecca Robbins/STAT STAT-Harvard poll: Dismayed by drug prices, public supports Democrats’ ideas Tags drug pricesJPM16pharmaceutical industry Gilead pricing for Sovaldi hepatitis C drug slammed by senators last_img read more

Pharmalot, Pharmalittle: Pfizer and Allergan call off their merger

first_img Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. Alex Hogan/STAT [email protected] @Pharmalot The New England Journal of Medicine is coming under attack, ProPublica writes. Some physicians are questioning whether the publication is losing relevancy and reputation. The journal and its top editor have resisted correcting errors and lag other journals in an industry-wide push for more openness in medical research. Some have also complained that the journal dismisses dissent with a “paternalistic arrogance.”A Florida jury ruled that Tyco Integrated Systems was not liable for the theft of more than $60 million worth of prescription drugs from an Eli Lilly warehouse in Connecticut six years ago, the Associated Press reports. Lilly’s insurer, the National Union Fire Insurance, alleged Tyco failed to adequately report security weaknesses at the warehouse. The insurer said thieves obtained information from a Tyco report and used it to break into the building.advertisement About the Author Reprints Ed Silvermancenter_img A Valeant Pharmaceuticals board committee did not find additional accounting issues that would require more restatements, Bloomberg News says, adding the drug maker plans to file its annual report on or before April 29. The board examined how the drug maker accounted for revenue through the Philidor Rx Services mail-order pharmacy, which Valeant allegedly used to boost prescriptions and circumvent some insurance reimbursements.FDA medical staffers supported the use of an Intercept Pharmaceuticals drug as a monotherapy in patients with liver disease who did not respond to standard treatment, Reuters reports. The drug is being reviewed for use in patients with primary biliary cirrhosis, a rare liver disease caused by an autoimmune destruction of the bile ducts. An FDA advisory panel meets Thursday to review the medication.The FDA is being warned to regulate medical device cybersecurity more firmly, according to Regulatory Focus. The warning comes after the US Department of Homeland Security issued an advisory about more than 1,400 cybersecurity vulnerabilities that affect versions of an automated supply cabinet used in hospitals and other health facilities to dispense medicines.Johnson & Johnson is making a renewed push in Africa to boost sales of new drugs to fight HIV/AIDS and other illnesses, The Wall Street Journal writes. And J&J will invest $50 million in Tesaro and license a potential prostate cancer drug from the company, the paper adds.The US Centers for Medicare & Medicaid Services will delay enforcement of a rule that changes the way state Medicaid agencies reimburse pharmacies for prescription medicines, Modern Healthcare reports.European regulators backed the use of an Eisai breast cancer drug called Halaven to treat liposarcoma, according to Pharma Times.The new FDA draft guidelines for labeling requirements for biosimilar medicines will become a burden for Indian generic drug makers, TwoFour Insight says.Samsung Bioepis and Biogen filed a lawsuit in the UK against AbbVie over its strategy of filing patents designed to block biosimilar versions of its Humira rheumatoid arthritis treatment, Reuters reports. Hello, everyone, and welcome to the middle of the week. And congratulations are in order for having made it this far. On that note, why not forge ahead? After all, consider the alternatives. This calls for a nice cup of stimulation and, as always, you are invited to join us. Remember, no prescription is required. And now, onward. Here are some tidbits to get you going. Hope you conquer the world and do drop us a line when you run across something fascinating …Pfizer and Allergan called off their $160 billion merger after the US Treasury Department this week issued new rules to thwart tax inversions. The deal was designed to lower their corporate tax rate from about 24 percent to 17 percent, and save roughly $35 billion in taxes. The decision is a big victory for the Obama administration, whose rules were more aggressive than many tax experts had expected. Pfizer will pay Allergan a $150 million breakup fee. The US Food and Drug Administration approved Inflectra, a biosimilar version of Johnson & Johnson’s Remicade, to treat Crohn’s disease, ulcerative colitis, rheumatoid arthritis, and psoriasis, Reuters writes. The new version is made by Celltrion and will be marketed by Pfizer’s Hospira unit. This is only the second biosimilar approved by the FDA. Analysts expect the drug to become available later this year.advertisement By Ed Silverman April 6, 2016 Reprints Tags AllerganJohnson & JohnsonPfizer PharmalotPharmalot, Pharmalittle: Pfizer and Allergan call off their merger last_img read more

An ex-con is taking his debt-ridden, cash-burning biotech public. Why are people investing?

first_img About the Author Reprints Related: Damian Garde Its founder did time in federal prison for his role in the insider trading scandal that put Martha Stewart in handcuffs. Its CEO has a decades-old drug trafficking charge on his résumé. The company is also laden with debt and burning through cash at a rate that threatens to bankrupt it by year’s end.Yet biotech startup Kadmon has convinced investors to back an initial public offering that values the company at more than $800 million. It’s expected to raise $100 million on the New York Stock Exchange this week.“It doesn’t make much sense to me,” said one longtime biotech investor who has steered clear of the company. “Are there really enough suckers out there?”advertisement By Damian Garde July 26, 2016 Reprints The company’s allure boils down to one figure: founder Sam Waksal. Friends and former colleagues describe him as an eloquent, charismatic polymath with an eye for groundbreaking science — but they also call him a deceitful, arrogant victim of his own hubris.‘A larger-than life character’Waksal, an immunologist by training, rose to fame amid biotech’s millennial bubble, when his previous company, ImClone, had a promising treatment for colorectal cancer moving through the pipeline. Bristol-Myers Squibb bought into the drug in what was then biotech’s biggest-ever deal, and Waksal became the sector’s most visible executive.“He was a larger-than-life character,” recalls one biotech analyst who has covered the industry for more than four decades.Waksal, who declined to talk to STAT, hosted Wildean salons and extravagant parties at his 5,800-square-foot SoHo loft, decorated with paintings by Mark Rothko and pieces of Etruscan pottery. He claims to have composed two novels, a memoir, and various works of poetry, and he dots his interviews with references to Sophocles, Omar Khayyam, and Marquis de Sade. Oliver Stone, who sought Waksal’s counsel before shooting the sequel to “Wall Street,” described him as “a dynamo” to the New York Times.Waksal, now 68, reached his socio-cultural zenith in late 2001, at a decadent Christmas party in his home. The CEO mingled with celebrity guests including close friend Stewart, Mick Jagger, and MTV’s Serena Altschul, as one former ImClone investor remembers, holding court between the columns in his living room and telling all who would listen that his company’s wonder drug, Erbitux, was a sure-fire blockbuster.And he was right. Erbitux eventually won approval to treat colorectal cancer and now brings in revenue of about $1.5 billion a year. Pharma giant Eli Lilly later acquired ImClone for $6.5 billion.But if Waksal celebrated those victories, he did so from federal prison, where he spent five years for his role in an insider trading scandal that tarnished his reputation and imperiled his company.Sam Waksal (center) with Martha Stewart and her daughter, Alexis, in 1990. Robin Platzer/Twin Images‘Running around in your underwear’Days after the glitz of that 2001 party, Waksal got some bad news: The Food and Drug Administration was about to reject Erbitux’s initial application for approval because ImClone had submitted insufficient data. Waksal knew the news would tank ImClone’s stock price. But it wouldn’t become public until two days later. So Waksal tipped his family members to begin unloading their shares. He tried to dump about $5 million of his own ImClone stock, too, but was thwarted by a pair of brokers who refused to execute the trades.It was sloppy stuff, and federal investigators quickly picked up on Waksal’s trail.“It was incredibly dumb, obviously, to just try and dump that much stock on the eve of an important decision,” then-US attorney James Comey told CBS in 2003. “It was like running around in your underwear, in broad daylight.” NewslettersSign up for The Readout Your daily guide to what’s happening in biotech. Leave this field empty if you’re human: According to another complaint awaiting trial, Waksal promised a pair of investors a 6 percent stake in Kadmon in exchange for helping the company raise money. They did their part, and then Waksal skated on the deal, the suit claims.“It seems there’s a pattern of him basically reaching out and finding people to help him in raising money for his ventures, and then he goes out of his way to try to screw them,” said Stuart Meissner, an attorney representing the plaintiff in the second suit. (Belesis’s lawyer, who previously represented Bernie Madoff, declined to comment.)Kadmon already expects to pay $10.4 million to a different investor to settle allegations that Waksal breached an agreement, documents show. The company says the latest two lawsuits are without merit.Though he’s turned Kadmon over to his brother, Waksal hasn’t completely left the spotlight. In June, he made an appearance on CNBC to tell a story about the value of transparency.Three years ago, Waksal said, a journalist “accosted” him and asked him about Theranos, the now-disgraced blood testing outfit that has been loath to share data on how its technology works.“And I said, ‘If someone says, “It’s a secret” when you ask a question, it’s a scam,’” Waksal said.Kadmon appears less sold on the need for transparency. STAT sent emails and calls inquiring about the company’s pipeline of drugs, financial structure, and plans for the future to multiple members of the Kadmon’s management team. No one responded. Headquartered in New York City, Kadmon is developing a trio of treatments it believes can treat cancer, autoimmune disease, and rare disorders. But a peek at its balance sheet reveals a leaky ship, hemorrhaging funds and beset by lawsuits that accuse the company of scamming investors.The biotech industry in general has been stumbling, forcing dozens of young drug companies to delay or abandon their plans to go public. So how is Kadmon, with its rap sheets and red ink, succeeding where so many have failed?advertisement Leave this field empty if you’re human: The ensuing months brought a locus of scrutiny down on Waksal, as former scientific colleagues came forward with tales of doctored test results, brash carelessness, and outright fabrication spanning three decades. In 2003, Waksal was convicted of securities fraud, bank fraud, obstruction of justice, and perjury. He admitted lying to his board, to his investors, and to the Securities and Exchange Commission.Waksal would spend five years in prison — a period he called his “interregnum” in New York Magazine. Now he’s getting a second chance with Kadmon.‘Burning cash like it’s 1999’Waksal founded Kadmon, which borrows its name from the Kabbalah concept of “primordial man,” in 2009 and has spent the ensuing years piecing together a patchwork drug company.First, Kadmon bought a private drug manufacturer for $100 million in 2010, and then it paid $14 million to license an investigational cancer therapy passed over by GlaxoSmithKline and shelved by the biotech Exelixis. Kadmon later picked up an anti-inflammatory treatment from a company called Surface Logix for just $900,000 plus a percentage of future sales. Its third drug is a generic called trientine, which the company hopes to reformulate and sell as a treatment for a rare kidney disease.Kadmon’s commercial business is a fraying operation that relies almost entirely on sales of ribavirin, an aging treatment for hepatitis C. The drug is rapidly becoming obsolete as far more effective therapies come on the market. Kadmon’s sales plummeted from $63.5 million in 2014 to $29.3 million last year, and, in documents filed with the SEC, Kadmon acknowledges that its current drugs will “contribute insignificantly to revenue in 2017 and beyond.”But despite dwindling returns, Kadmon spent about $140 million last year to keep the doors open, maintaining a staff of 138 people and paying its CEO more than $16 million in cash and stock. Meanwhile, the company has amassed more than $218 million in debt.“They’re burning cash like it’s 1999 here,” said Maxim Jacobs, director of healthcare research at the investment group Edison. At 31, she runs one of the hottest biotech companies in the country Please enter a valid email address. The idea, according to Kadmon’s federal filings, is to keep the commercial arm up and running until the company can win marketing approval for its pipeline treatments, at which point it will become a fully integrated drug maker.But Kadmon acknowledges that its IPO, which seeks to raise about $100 million, will only get the company through 2017. At that point, its top drug candidates will remain years away from approval.Kadmon’s first big bet, a lung cancer drug called tesevatinib, has already come up short twice in clinical trials, forcing the company to pivot.The treatment is designed to fight lung cancer by blocking a protein called EGFR, which plays a role in tumor growth. But there are eight other drugs on the market that already do that for various cancers, including Erbitux.Kadmon tested its drug in lung cancer patients who had relapsed after other treatments; just 1 in 41 of them saw tumors shrink.The company’s new plan: using tesevatinib to treat lung cancer that has spread to the brain after patients have tried other EGFR inhibitors. There’s no proof yet that it’ll work; Kadmon is trying to recruit patients for a clinical trials.Waksal (left) and an aide enter federal court in New York City for sentencing in June 2003. Chris Hondros/Getty Images‘I wouldn’t trust anything he would tell me’But Waksal won’t be steering those efforts from the board room — because, legally, he can’t.The terms of his settlement with the SEC include a permanent ban on ever serving as a director of a publicly traded company. So Waksal quietly stepped down from Kadmon in February — but not without compensation. He is promised a $3 million severance payment, and that sum could balloon to nearly $25 million over the next three years if the company succeeds. He also retains about 76,500 shares in Kadmon and a 12.7 percent stake in the shell corporation that is the company’s majority owner.Now serving as Kadmon’s CEO is his younger brother, Harlan, who worked alongside Sam at ImClone for 19 years.But despite the shakeup in the C-suite, insiders say Kadmon is by all means a Sam Waksal endeavor. As one longtime biotech investor put it: “This is sort of like if Elon Musk stepped down from Tesla and his brother became the CEO. Do you really think it’s no longer an Elon Musk company?”As such, Kadmon is viewed on Wall Street in the context of its founder, a brilliant entrepreneur with a well-established penchant for untruths. On the one hand, no one disputes his ingenuity in identifying Erbitux’s potential, and ImClone’s investors did quite well for themselves while Waksal was in prison. On the other hand, Waksal went to prison.“Here’s a guy who can pick the right targets, and he knows the right investigators to do the right trials, but if something were to go wrong, I wouldn’t trust anything he would tell me,” a member of the biotech investment community said. Like many others, the insider spoke on condition of anonymity to preserve relationships within the industry. Privacy Policycenter_img Privacy Policy BiotechAn ex-con is taking his debt-ridden, cash-burning biotech public. Why are people investing? Newsletters Sign up for Daily Recap A roundup of STAT’s top stories of the day. With a rapper alter ego, a ‘loud-mouthed’ doctor takes on the medical establishment Sam Waksal leaves federal court in New York City after pleading guilty to conspiracy and wire fraud in March 2003. Waksal admitted that he avoided paying $1.2 million in taxes on nine paintings he purchased. Adam Rountree/Getty Images National Biotech Reporter Damian covers biotech, is a co-writer of The Readout newsletter, and a co-host of “The Readout LOUD” podcast. [email protected] Please enter a valid email address. Related: And that’s the Waksal paradox, one longtime friend said. He is adept at identifying promising drugs that others would pass over, but the combination of his criminal record and hyperbolic cheerleading leads people to doubt whether he’s telling the truth.Others, however, say the stint in prison has tamped down some of Waksal’s dicier tendencies to embellish.“I think Sam has learned his lesson,” said Robert Schneider, a New York University associate dean who cofounded ImClone and serves as a scientific adviser to Kadmon. “His grandiosity is what gets him into trouble, but he’s really dialed that back.”And despite his sometimes misguided exuberance, Waksal is at worst a stretcher of the truth, “not a pathological liar,” a former colleague said.“He’s a good guy, but there’s a mishegas there,” the executive said with a laugh, using a Yiddish term whose definitions range from eccentric to crazy. “That word was created in Yiddish for Sam.”‘A dramatic mistake in judgment’Harlan, Sam’s stand-in in the Kadmon corner office, has a rap sheet of his own, though it’s not related to biotech.Back in 1981, Harlan was a physician training at Tufts Medical Center. As he waited in line at the Ft. Lauderdale airport for a trip back to Boston, a pair of federal agents spotted him nervously scanning the terminal and pulled him aside for a search. Digging through his bag, they were met with three bags of cocaine and an exclamation of “How did that get there?” In Harlan’s pants, the agents found two more baggies, affixed to his underwear.It was a kilogram in total, enough to convict Harlan of possession with intent to distribute, which came with a nine-year prison sentence.But looking nervous is not grounds for search and seizure, Harlan’s attorneys argued, and a federal court of appeals agreed. In 1983, the court ruled that the cocaine in Harlan’s luggage and trousers was inadmissible evidence, reversing his conviction and letting him walk.“It was a dramatic mistake in judgment,” Harlan told Barron’s in 1993. (Like his brother, he declined to talk to STAT.) “I did it as a favor for someone, and it’s a favor I have obviously regretted.”But it’s not a mistake that precludes him from running a biotech company, one analyst not involved in Kadmon’s IPO said — though it doesn’t look great in Google search results. It takes all types to develop new treatments for disease.“There are a lot of people in the drug business who are in the drug business, too,” the analyst said.Harlan Waksal (right) leaves federal court in June 2003 after his brother, Sam, was sentenced to seven years in prison and a $3 million fine for insider trading. Harlan is joined by his father, Jack, and Sam’s daughter Aliza. Stephen Chernin/Getty Images‘He goes out of his way to try to screw them’While Kadmon tries to focus on the future, both the company and its founder have been hit by lawsuits that could cost them.Walking out of prison in 2009, Sam Waksal had lost a lot of his industry friends, insiders said, so he had to go to considerable lengths to raise money in the early days of Kadmon. That included reaching out to Anastasios “Tommy” Belesis, the man behind a “Wolf of Wall Street”-style boiler room who has since been permanently barred from trading securities.That relationship soon went awry.Belesis, who ran the now-defunct John Thomas Financial, is suing Kadmon with claims that Waksal pulled off a bait-and-switch to defraud him. Belesis says Waksal owed him 1 million shares of Kadmon but handed over just 120,000. When Belesis pushed back, Waksal offered to pay him $15 million out of pocket to drop the matter, documents show, and Belesis agreed to those terms. But then Waksal’s line went dead, leaving Belesis with no shares and no cash, according to the lawsuit. @damiangarde Tags celebritiesdrug developmentFDASEClast_img read more

Scientists scour the globe for a drug to kill deadly brain-eating amoeba

first_img Related: The Naegleria fowleri amoeba seen under the microscope. Dr. Visvesvara/CDC “Even with the best drug combinations, the fatality rate is over 98 percent,” said Dennis Kyle, an infectious disease researcher at the University of South Florida. “People are dying from this disease all the time, and we really have nothing to treat it effectively.”But Kyle and his team are working to change that reality. Borrowing from techniques used to develop drugs for diseases like malaria, they have created the first high-volume screening setup to hunt for compounds that kill N. fowleri. With the help of collaborators, Kyle has amassed a collection of over 30,000 natural compounds gathered from far-flung corners of the globe, including microbes fished out of mangrove swamps, salty Antarctic oceans, and off sea sponges in the Gulf of Mexico.advertisement Kyle’s lab can screen about 7,500 compounds in 72 hours, an improvement over other methods that took scientists about a week to evaluate a handful of candidate molecules. The speed not only allows his team to examine more compounds, it also means they are screening for fast-acting compounds — an important consideration for an infection that can kill within days of initial symptoms. In test-tube studies, some of these fast-acting compounds are 20 times more potent than miltefosine. Pearce said the team has already identified hundreds of compounds that kill amoeba in the lab.A different tack to finding a treatment for N. fowleri infection is being pursued by a research group in Seattle. Rather than broadly screening a bunch of compounds in a bottom-up fashion, the Seattle Structural Genomics Center for Infectious Disease aims to design a drug from the top down. They are using X-rays to understand the structure of proteins that keep N. fowleri alive, and then using computer modeling to develop compounds to target them.Working alongside Kyle, the Seattle group is in the early stages of investigating 157 different N. fowleri proteins. The researchers hope their different approaches will meet in the middle, said Robin Stacy, senior project manager for the center, using computer modeling to tweak Kyle’s most promising compounds.Cedric Pearce stands in his culture room surrounded by a quarter of a million test tubes filled with fungi. David Wilson, Chris English/UNC GreenboroLast year Kyle’s group reported their first drug candidates, after screening about 150 synthetic antimicrobial chemicals. The team is now testing eight of those compounds in mice, hoping to identify versions that can cross the blood-brain barrier. At the same time, they continue to search for even more potent compounds from nature.Their work “looks quite promising,” said Elizabeth Winzeler, a developmental biologist who studies malaria at the University of California, San Diego, and who is not involved in the research. “We and others in the field have used the same kind of approach and it’s worked quite well” to develop treatments against malaria. She cautioned, though, that the steps in drug development are numerous and complex and that it’s not unusual for a decade to pass between the discovery of molecular candidates and when drugs make it to the shelf.A moving targetFinding a treatment may be more important than ever as unusual cases have startled US health officials in the last five years. Infections contracted in Minnesota lakes — 600 miles farther north than ever before — and the first deaths from treated tap water have led some experts to hypothesize that a changing climate is expanding the range and transmission routes of these amoebas.“It’s not hypothetical. It’s occurring,” said Michael Beach, associate director of the healthy water division of the CDC’s National Center for Emerging, Zoonotic, and Infectious Diseases. “We’ve seen first cases in Kansas, first cases in Indiana, first cases in Minnesota, and the first case since 1969 in Virginia.”Beach urges awareness but not fear. To reduce the already-tiny risk of infection, the CDC recommends people use distilled and sterile water for nasal rinses, not submerge their heads in warm and brackish water, and consider wearing nose clips during water sports. Listen: Episode 13: The superbugs are winning the battle against us In the LabScientists scour the globe for a drug to kill deadly brain-eating amoeba Funding for research, meanwhile, has been rising but is still sparse. The National Institutes of Health awarded less than $800,000 in grants to scientists studying N. fowleri for 2016 — and about two-thirds of that went to Kyle and Pearce’s group.One reason for the low dollar amount is the rarity of the disease relative to others, says Lee Hall, chief of the Parasitology and International Programs Branch of the NIH’s National Institute of Allergy and Infectious Diseases. Another is that few researchers have applied for grants to study this disease.Kyle and Pearce are planning to apply for a new grant in September, and this time they will be asking for “a lot more money,” said Pearce.“There’s a significant need for more research,” said Kyle. “Better awareness of this in the research community is as important as it is in the general community.”This article has been updated to correct the number of US deaths from N. fowleri. A radically simple idea may open the door to a new world of antibiotics Related: Tags amoebabrain The deaths hit the headlines every summer, sometimes five or six of them across the country. They’re newsworthy for their rarity and for how innocuous the events leading up to them are — it’s usually a young person who was swimming in a lake, got some water up their nose, and within days, was dead.The cause is an amoeba called Naegleria fowleri, which when it infects the brain, causes massive swelling that is almost always fatal. Over the past half-century, 135 people in the US have died of the infection.That rarity means that hardly any research money exists to find treatments. The best line of attack at present is a combination of drugs designed for other conditions.advertisement By Lindzi Wessel July 22, 2016 Reprints Adding this supply to their arsenal of synthetic compounds and already-approved FDA drugs, the team is optimistic that they will eventually find something that can enter the human brain and take the amoeba out.Chemical warfareThe amoebas, which are thought to thrive in the soil and scum layers of warm waters, enter the brain when water is forced up the nose. Once there, they feed on brain matter, leading to rapid inflammation. Of all US victims, only three have survived. The antimicrobial drug miltefosine is the most promising existing treatment, and is credited with helping save two children in 2013. But it’s considered an investigational treatment, and others treated with the drug have not survived.Kyle and his group hope that a systematic survey of natural compounds will turn up a better drug.“We think that microorganisms use chemical warfare —  if you like — to kill other things that might be fighting for the same resources,” said Cedric Pearce, one of Kyle’s collaborators and founder of Mycosynthetix, a small company which boasts a library of 55,000 fungal strains from all over the world.last_img read more

Most drug makers promise access for poor patients. There’s no evidence their plans work

first_img APStock [email protected] By Ed Silverman April 6, 2017 Reprints Tags drug pricinginfectious diseasepharmaceuticalspublic healthSTAT+ Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr. STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. About the Author Reprints A decade ago, United Nations officials implored the pharmaceutical industry to expand access to medicines to low- and middle-income countries, and many companies have heeded the call. But a new analysis finds that evidence to evaluate and report the effectiveness of these initiatives is lacking.First, the good news: The number of initiatives operated by 21 global drug makers increased to 102 in 2015 from just 17 in 2000. And of the 120 access to medicines programs that were identified, 48 percent donated pharmaceuticals and 44 percent relied on a strategy for reducing prices. Only 22 percent involved licensing to other companies. Ed Silvermancenter_img Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED GET STARTED Pharmalot What’s included? What is it? Log In | Learn More @Pharmalot Most drug makers promise access for poor patients. There’s no evidence their plans work last_img read more

How one small biotech exploits a loophole in a federal transparency law

first_img What is it? How one small biotech exploits a loophole in a federal transparency law Log In | Learn More A gene therapy for a rare disease is unlikely to come cheap, so one fledgling biotech recently reached out to experts for insights into winning over cost-conscious hospitals. And to lure these experts to a so-called advisory meeting, the company used an enticing pitch — each attendee would be given a $3,000 honorarium, along with paid airfare and lodging for two-day session in November.However, AveXis (AVXS) dangled something else: The compensation would not be reported to a federal database to which industry must submit payments made to physicians. Known as OpenPayments, the database was launched three years ago in response to concerns that medical practice and research may be unduly influenced by financial relationships between doctors and drug and device makers. What’s included? Pharmalot Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr. By Ed Silverman Aug. 22, 2017 Reprints @Pharmalot Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. About the Author Reprints Ed Silverman STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. GET STARTED [email protected] Tags pharmaceuticalsSTAT+last_img read more

California just passed a law to rein in drug prices. Here’s why it’s unlikely to make much difference

first_imgThe Pharmalot View STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. [email protected] What is it? Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr. Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. Log In | Learn More Over bitter protests from the pharmaceutical industry, California this month enacted a law that requires drug makers to explain and justify prices for some medicines.Predictably, those who backed the effort are hailing the move as a significant victory, because they hope this will become the first step toward reining in drug prices. @Pharmalot California Gov. Jerry Brown signed the bill into law this month. Rich Pedroncelli/APcenter_img California just passed a law to rein in drug prices. Here’s why it’s unlikely to make much difference Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED About the Author Reprints GET STARTED What’s included? By Ed Silverman Oct. 24, 2017 Reprints Ed Silverman Tags drug pricinglegalpharmaceuticalspolicystateslast_img read more

Keeping the register ringing: Many old drugs have plenty of new patents

first_img Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. Keeping the register ringing: Many old drugs have plenty of new patents Ed Silverman @Pharmalot STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr. By Ed Silverman Nov. 8, 2017 Reprints Toby Talbot/AP What is it? What’s included?center_img Log In | Learn More [email protected] GET STARTED Tags legalpharmaceuticalsSTAT+ Pharmalot About the Author Reprints To most people, “evergreen” refers to a tree that manages to keep its leaves no matter what. But the term has another, highly contentious meaning when discussing prescription drugs — the use of additional patents to extend the monopoly on a medicine and keep cash registers ringing.Drug makers have long argued their patent modifications reflect substantive enhancements, but the practice has prompted complaints that companies often make minor changes in order to thwart generic competition. And a new study shows the approach may be more pervasive than thought — at least 74 percent of medicines associated with new patents were already on the market. Unlock this article — plus daily coverage and analysis of the pharma industry — by subscribing to STAT+. First 30 days free. GET STARTEDlast_img read more

Scientist concedes his controversial MS therapy, once a source of great hope, is ‘largely ineffective’

first_imgMany of those same neurologists, though, saw their relationships with their patients fractured as belief in the liberation therapy took hold in the community of patients and their families in Canada, parts of the United States, and farther afield. Doctors advising caution against a procedure that hadn’t been proved to work or even to be safe were derided as standing in the way of innovation to protect their own practices.Dr. Jock Murray, an MS expert and retired professor from Dalhousie University in Halifax, Nova Scotia, said the history of MS is laden with incidences like the Zamboni episode — though he said this one lasted longer than most.advertisement By Helen Branswell Nov. 28, 2017 Reprints Italian physician Paolo Zamboni has publicly concluded that a therapy he developed and dubbed “the liberation treatment” does not cure or mitigate the symptoms of MS. Nathan Denette/The Canadian Press/AP What many hope will be the final chapter in an unfortunate saga in multiple sclerosis research appears to have been written by the scientist who started the affair in the first place.Italian physician Paolo Zamboni has publicly acknowledged that a therapy he developed and dubbed “the liberation treatment” does not cure or mitigate the symptoms of MS. A randomized controlled trial — the gold standard of medical research — he and other Italian researchers conducted concluded the procedure is a “largely ineffective technique” that should not be recommended for MS patients.The trial’s result comes as no surprise to neurologists, most of whom felt Zamboni’s theory lacked plausibility from the moment news of it exploded through the MS community in 2009.advertisement Senior Writer, Infectious Disease Helen covers issues broadly related to infectious diseases, including outbreaks, preparedness, research, and vaccine development. “We hope these findings, coming from a carefully controlled, ‘gold standard’ study, will persuade people with MS not to pursue liberation therapy, an invasive procedure that carries the risk of complications, as well as significant financial cost,” Traboulsee said at the time of presentation.And now, Zamboni’s paper showing the same has been published in JAMA Neurology.The liberation treatment is one in a long list of therapies that have been embraced by the MS community but were ultimately found to be ineffective, said Murray. Hyperbaric oxygen chambers, snake venom, anti-coagulants, histamine desensitization therapy — all have had their turn.The particular nature of MS — for a lot of patients, symptoms worsen, then ease before worsening again — makes it easy to appear as if a treatment has had a benefit, Murray noted.He worried about the impact these episodes have on the MS community. “To have one after another fail, of this kind of thing, is very disheartening. And then they don’t know who to believe.” Please enter a valid email address. Bold claim of multiple sclerosis gene discovery comes under withering attack Zamboni, who works at University of Ferrara Hospital, did not respond to STAT’s email inquiry about the trial.The vascular surgeon was moved to look for the cause of MS because his wife suffers from the disease. In 2009, Zamboni published a paper suggesting the neurological damage the condition causes was triggered by pooling of blood in the brain that was the result of inadequate drainage.He called the condition — which he said was common among MS patients — “chronic cerebrospinal venous insufficiency,” or CCSVI.Zamboni further suggested inserting stents — which are designed to hold open arteries — into veins in the neck would increase blood drainage and improve symptoms.His 2009 study appeared to show it worked, though skeptics were quick to note major flaws. There were no controls — a group that didn’t get the treatment to use as comparators — and both the patients and the doctors knew everyone was treated. A study of this kind is open to the placebo effect and to researcher bias — both of which can result in individuals seeing something they hoped to see.Zamboni’s recently published study is the type his critics would have liked him to have done the first time — a double-blind randomized controlled trial in which neither patients nor the researchers assessing whether there were improvements post-treatment knew which patients got the actual procedure and which received a sham therapy.The Italian researcher’s 2009 claim received some prominent and unchallenging coverage from a couple of major Canadian media outlets, and the clamoring for the procedure began.“These patients looked like they were miraculous. And that got played over and over and over. Within 72 hours, everybody in Canada — all the patients and their families — knew about this new procedure,” said Murray. “So there was a great demand then for everyone to seek it out.” Related: Leave this field empty if you’re human: Patients weren’t willing to wait for science to conclude if Zamboni was correct or if his treatment was safe. Clinics sprang up in a number of countries — Poland, Costa Rica, and India among them — offering the controversial procedure. Many came home rapturously claiming benefit, only to see what others suggested was a placebo effect wane over time. A few people who underwent the procedure died.Patients pressured the MS societies in the U.S. and Canada to advocate for access to the treatment, and in Canada, there were strident calls for the national health care system to pay for the procedure.Meanwhile, the scientific community scrambled to try to test Zamboni’s theory and the procedure. Study after study concluded his idea was wrong. In fact, a key study in 2014 concluded that the neck veins of MS patients were no different from those of people who don’t have the condition. But disproving a theory takes time. And hope dies reluctantly.Millions of dollars were spent trying to see if Zamboni was correct. Untold research hours were diverted from other lines of MS inquiry. Previously collaborative relationships between doctors and patients, as well as patients and the foundations set up to advocate for their needs, were damaged, Murray said. “It caused a lot of trouble.”Earlier this year, Canadian researchers reported on a large, randomized double-blinded study — the kind designed to protect against researcher bias and the placebo effect — they had conducted on the liberation treatment. The results, presented to the Society of Interventional Radiology’s annual scientific meeting in March, showed the technique didn’t work. The study is currently under review at a top-tier medical journal, lead author Dr. Anthony Traboulsee, an associate professor of neurology at the University of British Columbia, said Tuesday. HealthScientist concedes his controversial MS therapy, once a source of great hope, is ‘largely ineffective’ @HelenBranswell Related: Helen Branswell Privacy Policy “Belief always trumps evidence,” Murray told STAT in an interview Monday. Gut germs play role in multiple sclerosis, studies show. Are probiotics for MS next? About the Author Reprints Newsletters Sign up for Daily Recap A roundup of STAT’s top stories of the day. Tags neurologyresearchlast_img read more

Feds broaden the definition of ‘pharmacy’ in a bid to level playing field for startups

first_img The mail-order drug company PillPack prepackages consumers’ pills and ships them. Pat Greenhouse/The Boston Globe Business Log In | Learn More STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. Across the country, upstart pharmacies are turning the prescription drug business on its head, offering digitally enabled mail-order offerings and more convenient ways for customers to get, and take, their medications. Massachusetts company PillPack, for instance, is pre-sorting drugs into daily regimens and shipping them to customers nationwide; another business, Capsule, is filling prescriptions on demand and delivering them to homes and workplaces across New York City.But their ability to grow is largely controlled by existing competitors, including major pharmacy benefit managers who have been accused of twisting contract terms to block pharmacies’ access to their millions of U.S. customers. What is it? Tags MedicaidMedicarepharmaceuticals What’s included? [email protected] National Technology Correspondent Casey covers the use of artificial intelligence in medicine and its underlying questions of safety, fairness, and privacy. He is the co-author of the newsletter STAT Health Tech.center_img GET STARTED Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr. About the Author Reprints @caseymross By Casey Ross April 6, 2018 Reprints Unlock this article — plus daily market-moving biopharma analysis — by subscribing to STAT+. First 30 days free. GET STARTED Casey Ross Feds broaden the definition of ‘pharmacy’ in a bid to level playing field for startups last_img read more