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Saturday, May 19, 2018

Hotel pools linked to many disease outbreaks

Here is something to think about before jumping into the pool on your next vacation.  Hotel pools and hot tubs are breeding grounds for waterborne diseases.
A study from the Centers for Disease Control and Prevention find a third of all recreational waterborne disease outbreaks occurred in hotel pools or hot tubs and water play venues.
Between 2000 and 2014 the CDC recorded nearly 500 disease outbreaks related to recreational water use that resulted in more than 27,000 illnesses and eight deaths.  Hotels were the leading setting, associated with 157 of the outbreaks
Of the outbreaks with a confirmed cause, most were due to pathogens in the pool while the rest were caused by chemicals.  A parasite that causes diarrhea and other gastrointestinal issues caused most of the illnesses.
Outbreaks spiked during June, July and August but they were observed to take place all year long with March being another month with a spike in cases.
Chlorine is the primary barrier to the transmission of pathogens in treated recreational water. At CDC-recommended concentrations of at least 1 ppm, free available chlorine inactivates most pathogens within minutes.

Janus Henderson reports 11.9% stake in athenahealth, urges for sale process

In a regulatory filing, Janus Henderson disclosed an 11.9% stake in athenahealth and said that, on May 15, 2018, it engaged the company’s board of directors to express, among other things, that it believes initiating a formal sale process for the company is in the best interest of the its shareholders. Janus also expressed concerns over management’s execution of strategic initiatives. As such, Janus expressed its view that it is in the best interest of athenahealth’s shareholders to engage with third parties making offers for the company, and to open up a broader sales process.

Duke team IDs new target for fatty liver disease and prediabetes

The buildup of fat in the liver known as nonalcoholic fatty liver disease now affects up to 25% of Americans, according to the American Liver Foundation, leading many experts in metabolic disorders to dub it a pandemic. The rise of the disease tracks with increasing obesity rates, though nonobese people can develop it, too. People with prediabetes face a high risk of developing fatty liver disease, and there are no cures.
Scientists at Duke have identified a pathway in the liver that they believe could be manipulated to reduce the deposition of fat in the liver. It involves balancing two enzymes that work against each other to control fat metabolism. When they tried their method in rat models of obesity and metabolic disease, they found they could lower the amount of fat deposited in the liver and improve glucose regulation. They published their study in the journal Cell Metabolism.
The key to the discovery lies in branched-chain amino acid (BCAA), which is produced when protein is metabolized. Scientists have long known of an association between BCAA, obesity and insulin resistance, but they’ve struggled to figure out the mechanisms behind that link.
So researchers at the Duke Molecular Physiology Institute focused on two enzymes that control how BCAA is broken down in the body: one a kinase that inhibits the breakdown and the other a phosphatase that activates it.
First they took a kinase-inhibiting drug developed at the University of Texas Southwestern Medical Center and tested it in the rat model. Then they found a way to activate phosphatase. Both experiments produced the same result: less fat in the liver and better glucose control. And the improvements started to show within a week.
“This particular rat model (the Zucker fatty rat) is an extreme model of obesity and metabolic disease, so if you can get an improvement in a week, that’s really significant,” said lead author Phillip White, Ph.D., an assistant professor of medicine, in a statement.
It’s the latest example of how new insight into enzyme activity is advancing research in diabetes and related disorders. In March, a team at Columbia University demonstrated in rodents that if they shut down the production of the enzyme DPP4 in the liver, they could improve insulin resistance. And a team in Japan published research in January suggesting that Sir2 could be targeted to improve glucose uptake in the liver.
The Duke team is planning further experiments to better understand how BCCA can be targeted in fatty liver disease and prediabetes. But their observations in the Zucker fatty rat are already motivating some specialists to consider new strategies for treating metabolic disorders.
“There’s growing evidence to suggest that BCAA isn’t just a passive marker of diabetes, but may actually play a role in driving the disease,” said Robert Gerszten, M.D., director of clinical and translational research for the Massachusetts General Hospital Heart Center, in the statement. “It gives us the motivation to test whether changes in the amino acid intake in our diets would be worth exploring.”

DC Week: HHS Chief Says Docs Must Stop Buying Part B Drugs

Forget the practice of doctors buying Part B drugs, Health and Human Services Secretary Alex Azar said earlier this week. The Trump administration has a better idea. The White House also is stepping up its anti-abortion efforts. And a busy week also saw the president announce his latest pick to head the Department of Veterans Affairs.
Get Docs Out of Drug-Buying Business, HHS Chief Urges
Doctors should get out of the business of making money off of providing prescription drugs to Medicare beneficiaries, Health and Human Services Secretary Alex Azar said Monday.
Currently, physicians who administer drugs paid for under Medicare Part B — which covers drugs administered in the physician’s office — pay for the drug themselves up front and then are reimbursed at the average sales price plus a 6% markup. This system “gets into the notion of physicians or facilities making money off the arbitrage between acquisition price and reimbursement,” Azar explained at a briefing with reporters following a speech he gave on President Trump’s plan to lower the cost of prescription drugs.
Instead, the administration is proposing a “competitive acquisition program” for Part B drugs.”The concept of the competitive acquisition program is not to have physicians take title to the medications they’re administering, but rather, have a competitive program where those drugs would be purchased and the capital outlay would occur elsewhere,” so the doctor doesn’t have to buy the drug “and the physician is paid … a fair, appropriate level of reimbursement for their services in administering the drug,” Azar said.
Trump Picks Wilkie as VA Secretary
At an event Friday morning focusing on prison reform, President Trump surprised everyone by announcing that he would nominate Robert Wilkie to head the Department of Veterans Affairs.
Among those surprised: Wilkie, currently the department’s acting secretary as well as the Defense Department’s undersecretary for personnel and readiness. Trump said Wilkie didn’t know the announcement was coming, CNN reported.
Wilkie, a lawyer by training who spent some 20 years on Capitol Hill as an aide to various Republican congressmen, has been in the acting role at the VA since late March, when Trump fired David Shulkin, MD.
FDA Tries Shaming Drugmakers that Block Generic Competition
The FDA released a list Thursday of brand-name drug companies it says are blocking the development of generic drugs by refusing to give generic drugmakers access to samples of brand-name products for use in clinical trials needed for approval.
“We hope that this increased transparency will help reduce unnecessary hurdles to generic drug development and approval,” FDA Commissioner Scott Gottlieb, MD, said in a statement.
The list’s release was part of the Trump administration’s plan to lower prices for prescription drugs. “As stressed by the President and [Health and Human Services] Secretary [Alex] Azar last week, one of the Administration’s highest priorities is advancing policies that increase competition as a way to help make drugs more affordable and improve access,” Gottlieb said.
White House Proposes Restrictions on Family Planning Clinics
The Trump administration is reportedly moving closer to issuing a regulation that would require clinics that accept federal funds to maintain complete separation of facilities and finances related to abortion services from those providing other services supported by the federal monies.
The proposed rule, which the White House sent to the Office of Management and Budgetfor review on Thursday, would apply to clinics that receive money from the federal government’s $287 million Title X family planning program.
Officials at Planned Parenthood, which receives the largest share of Title X funds of any organization ($50 to $60 million, according to reports), said they wouldn’t comply with the physical separation requirement.
Senators Focus on Hospital Drug Discount Program
The 340B discount program for prescription drugs used by safety-net hospitals needs better government oversight, several watchdog agency officials said at a Senate hearing.
Although the Health Resources and Services Administration (HRSA), which oversees the program, has taken some important steps to improve oversight, “a number of important issues remain,” said Debra Draper, PhD, director of the healthcare team at the Government Accountability Office, during a hearing Tuesday of the Senate Health, Education, Labor, & Pensions (HELP) Committee.
These issues include “whether the intent of the program is still relevant today given the vastly changed healthcare landscape and the 340B program environment, and the continued lack of specificity in program guidance, most notably the definition of patient and hospital eligibility criteria,”said Draper, whose agency released a 2011 report on the program’s problems. “Until these issues are resolved, there will continue to be questions about the integrity of the 340B program and HRSA’s ability to provide effective oversight.”
Second Bundle of Opioid Bills Passes House Committee
Thirty-two bills aimed at targeting the opioid epidemic advanced through a House committee on Thursday after an 8-hour markup session.
The same House Energy and Commerce Committee passed 25 other opioid-related bills last week, bringing the total to 57 bills, which now advance to the House.
Many of the measures discussed on Thursday moved handily through the committee, but members wrestled with a few with controversial elements.
These included a bill to relax confidentiality standards around substance use disorders; a bill to temporarily reverse the ban on Medicaid paying for inpatient treatment in certain facilities; and a bill — rewritten as an amendment — which would have permanently let certain non-physicians such as nurse midwives prescribe medication-assisted treatment.
“It’s laudable to try to expand treatment, but we need to do that very carefully,” said Rep. Larry Bucshon, MD (R-Ind.) in response to the idea of nonphysicians prescribing MAT.
Next Week
On Monday, Research!America and the American Society for Microbiology will examine vaccine survey data.
On Tuesday, The Hill will host a briefing on preventing opioid addiction in young people.
The Senate Health, Education, Labor and Pensions Committee will discuss ways to improve the healthcare work force.
And The Atlantic will explore the issue of finding treatment for pediatric cancers.
On Wednesday The Atlantic will host a second briefing, this time, on patient access to care and affordability.
And the Senate HELP Committee will examine the Pandemic and All-Hazards Preparedness and Advancing Innovation Act of 2018.
On Thursday, the Senate Finance Committee will discuss opportunities and challenges for rural healthcare.

Solid Biosciences announces new preclinical data for Duchenne med

Solid Biosciences announced the presentation of new preclinical data from its gene therapy development programs for Duchenne muscular dystrophy. New data for SGT-001, the company’s lead microdystrophin gene transfer candidate, further demonstrate its potential to produce long-term and body-wide microdystrophin expression that correlates with significant improvements in muscle function. Additionally, the company presented data supporting the development of novel promoters and capsids as part of its next generation gene therapy discovery efforts. These data were presented this week at the 21st Annual Meeting of The American Society of Gene and Cell Therapy in Chicago

Friday, May 18, 2018

Agile: FDA has concerns on contraceptive

Agile Therapeutics, Inc., (Nasdaq: AGRX) announced the content of the official minutes from its Type A meeting with the U.S. Food and Drug Administration (FDA) held on April 16, 2018 to discuss the complete response letter (CRL) issued by the FDA on December 21, 2017 relating to the New Drug Application (NDA) for Twirla (AG200-15), the Company’s investigational low-dose, non-daily, combination hormonal contraceptive patch. In the CRL, the FDA informed the Company that the Twirla NDA could not be approved due to deficiencies related to the manufacturing process and facility for Twirla, and because of questions the FDA had on the in vivo adhesion properties of Twirla and their potential relationship to the Company’s Phase 3 clinical trial results.
In the official minutes, the FDA informed the Company that it continues to have significant concerns regarding the adhesion of Twirla, which the FDA believes cannot be addressed through the Company’s proposed patient compliance programs, and that the Company needed to address the Twirla adhesion properties by reformulating the transdermal system and conducting a formal adhesion study with the new formulation. The FDA also informed the Company that it would need to demonstrate bioequivalence to the data and information for the original formulation. The FDA advised the Company that after the Company satisfies the FDA’s questions on adhesion and adequately bridges to the findings in the SECURE Phase 3 trial, it anticipates discussing the safety and efficacy of Twirla at an advisory committee meeting to obtain input on whether the benefits outweigh the risks. In the absence of a finding of bioequivalence, the Company would need to conduct a new Phase 3 study with the new formulation. Finally, the FDA provided guidance on the path forward for addressing manufacturing issues related to Twirla, which path is largely based on the materials the Company had previously submitted in December 2017. To the extent that the Company reformulates Twirla, it may create the need for additional manufacturing work and review by the FDA.
“We believe we had a constructive meeting with the FDA, however, we disagree with the FDA’s conclusions on the adhesion of Twirla and our patient compliance programs. We believe we have demonstrated an adhesion profile for Twirla that supports approval based on extensive data from our Phase 2 studies, including an extreme conditions trial, and our three Phase 3 trials. We also believe that we have planned compliance and education programs that can address the issues raised by the FDA and will support patient use of the product once it is approved. While we will continue to evaluate all of our options on next steps, we expect we will pursue formal dispute resolution. We will provide an update when we move forward,” said Al Altomari, Chairman and Chief Executive Officer, Agile Therapeutics. “In light of the feedback from the FDA, we also are re-evaluating our business plan to identify ways to extend our ability to fund the Company’s operations,” concluded Mr. Altomari.
Company to Host Conference Call
Agile Therapeutics will host a conference call on May 18, 2018 at 8:00 a.m. Eastern Time to discuss the Company’s regulatory update. A question and answer session will follow Agile Therapeutics’ remarks. To participate on the live call, please dial (844) 413-1773 (domestic) or (678) 865-8976 (international), and provide the conference ID number: 5858819.
A live audio webcast of the call will be available via the “Investor Relations” page of the Agile Therapeutics website, http://www.agiletherapeutics.com. Please log on through Agile Therapeutics’ website approximately 10 minutes prior to the scheduled start time. A replay of the webcast will be archived on Agile Therapeutics’ website for 60 days following the call.

Sarepta target upped by Goldman: ‘Multiple shots on DMD’

Goldman Sachs raised its price target on Sarepta Therapeutics (NASDAQ: SRPT) to $127.00 (from $84.00) while maintaining a Buy rating.