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Tuesday, December 31, 2019

FDA to ban fruity e-cigarette pod flavors, but allow tank vaping systems

The Food and Drug Administration plans to ban the sale of fruity flavors in cartridge-based e-cigarettes, but the restriction won’t apply to tank vaping systems commonly found at vape shops, according to people familiar with the matter.
The action is seen as a compromise between Trump administration officials who want to address a rise in teen vaping and those concerned about the impact on small businesses and the possible political fallout for President Donald Trump, these people said. Polls commissioned by the vaping industry have shown an outright ban would be unpopular in key states for the 2020 election.
Federal officials are expected to announce the new plan as soon as Friday. Open-tank vaping devices, which allow users to mix their own nicotine liquids, aren’t popular among children or teenagers, who tend to use vaporizers with prefilled cartridges such as those made by Juul Labs Inc. Open tanks are typically found in vape shops and allow consumers to custom-mix flavors.
The new policy, intended to curb a surge in underage vaping, would apply only to pod-based vaporizers such as those made by Juul, NJOY Holdings Inc. and Reynolds American Inc.

Direct-to-consumer fertility tests confuse and mislead consumers

Direct-to-consumer hormone-based “fertility testing” for women is viewed by consumers as both an alternative, empowering tool for family planning, and a confusing and misleading one, according to the results of a new study from Penn Medicine. Findings from the small, first-of-its-kind ethnographic study reinforce the need for consumer education around the purpose and accuracy of the tests, which have seen increasing interest in recent years due to the low cost and widespread availability. The study was published in the journal of Social Science and Medicine.
Many DTC companies in the “FemTech” space offer a test for anti-Mullerian hormone (AMH), a marker that estimates the size of a woman’s egg supply or “ovarian reserve,” which have also been called the ‘egg timer’ or ‘biological clock test.’ Fertility products and testing is frequently marketed as an accessible, low-cost option for investigating fertility status, yet the tests are not predictive of natural conception. In fact, a 2019 policy statement by the American College of Obstetricians and Gynecologists discouraged testing in otherwise healthy women without a history of infertility.
Measuring AMH levels has primarily been used as a clinical diagnostic tool prior to ovarian stimulation for in vitro fertilization (IVF) in women with infertility, or before oocyte cryopreservation (i.e. egg freezing), in order to guide medication selection and anticipate success rates. However, recently more women have been engaging with DTC testing, due, in part, to barriers to care like strict guidelines for diagnosis of infertility, insurance coverage limitations, high costs, and rising concern about age-related fertility decline.
“Consumers continue to desire these tests, and they’re attractive, but they don’t deliver on their promise,” said Moira Kyweluk, PhD, MPH, a fellow in the department of Medical Ethics and Health Policy in the Perelman School of Medicine at the University of Pennsylvania, and the author of the paper. “I view DTC testing as an entry point into what I term the ‘new (in)fertility pipeline’ for women today. Because it is low cost and widely available, it’s reaching a larger demographic, people of diverse identities and backgrounds, and raising awareness of more advanced procedures and technologies like egg freezing.”
To better understand the decisions and experiences around DTC fertility testing, Kyweluk closely followed 21 participants pursuing fertility testing recruited in Chicago in 2018. The participants represented different ethnicities, socioeconomic statuses, sexual orientations, gender identifies, and ages. Through an online company, the women visited an accredited laboratory to have their blood drawn for analyses; however, many companies offer at-home kits for women to collect a sample of their own blood, which is sent in for hormone analysis.
Four major themes emerged from participant interviews, surveys, and observation of follow up consultations with a nurse practitioner from the selected DTC fertility testing company including: knowledge, empowerment, DTC fertility services as a viable alternative for family planning, and finally, participant feelings of varying degrees of uncertainty.
This last theme, Kyweluk says, is the most important, and reinforces the need for better education to consumers around the true purpose for these tests.
“Though there may be some benefits to consumers using DTC fertility testing, across the board participants were left with incorrect assumptions about the power of hormone testing to predict fertility,” Kyweluk said. “No test or medical procedure guarantees future fertility–including egg freezing–and these startups directly target women who are concerned about their reproductive futures.”
Overall, the findings suggest that DTC fertility testing may allow for a larger population of women to be exposed to a range of assisted reproductive technologies and other information around fertility to inform family planning at any age. Kyweluk calls for future, similar research to better understand consumer experiences and outcomes with these products, as the infertility care industry and DTC fertility market are expected to expand.
“This study offers one model that social scientists can use to investigate the impact of new medical technologies and online delivery systems as they emerge…and create a better understanding of the ethical, legal, and social impacts of these options,” she wrote.

Possible dementia vaccine closer after mice studies

A preventive treatment for dementia may proceed to clinical trials after successful animal testing.
The US-led research is looking to develop effective immunotherapy via a new vaccine to remove ‘brain plaque’ and tau protein aggregates linked to Alzheimer’s disease.
Recent success in bigenic mice models supports progression to human trials in years to come, the researchers say.
A new paper in the journal Alzheimer’s Research & Therapy paves the way for more work in 2020, with medical researchers at the Institute for Molecular Medicine and University of California, Irvine (UCI) working with a successful vaccine formulated on adjuvant developed by Flinders University Professor Nikolai Petrovsky in South Australia.
The latest research aims to come up with a new treatment to remove accumulated beta-amyloid (Aβ) plaques and neurofibrillary tangles composed of hyperphosphorylated tau, which together lead to neurodegeneration and cognitive decline in Alzheimer’s disease.
Alzheimer’s disease (AD) is the leading cause of age-related dementia, affecting about 5.7 million people in the US. Major challenges in AD include the lack of effective treatments, reliable biomarkers, or preventive strategies.
Professor of the Institute for Molecular Medicine Anahit Ghochikyan and colleagues, Associate Professors Hvat Davtyan and Mathew Blurton-Jones from UCI, and other co-authors tested the universal MultiTEP platform-based vaccines formulated in the adjuvant developed at Professor Petrovsky’s Australian lab.
The possible new therapies were tested in bigenic mice with mix Aβ and tau pathologies.
“Taken together, these findings warrant further development of this dual vaccination strategy based on the MultiTEP technology for ultimate testing in human Alzheimer’s disease,” the lead authors Professor Ghochikyan and Blurton-Jones conclude.
Professor Petrovsky says the Advax adjuvant method is a pivotal system to help take the combination MultiTEP-based Aβ/tau vaccines therapy, as well as separate vaccines targeting these pathological molecules, to clinical trials – perhaps within two years.
“Our approach is looking to cover all bases and get past previous roadblocks in finding a therapy to slow the accumulation of Aβ/tau molecules and delay AD progression in a the rising number of people around the world,” says Professor Petrovsky, who will work in the US for the next three months.
Several promising drug candidates have failed in clinical trials so the search for new preventions or therapies continues.
A recent report on human monoclonal antibody, aducanumab, showed that high dose of this antibody reduced clinical decline in patients with early AD as measured by primary and secondary endpoints.
However, it is obvious that it could not be used as a preventive measure in healthy subjects due to the need for frequent (monthly) administration of high concentrations of immunotherapeutic.
Professor Ghochikyan says there is a pressing need to keep searching for new preventive vaccine to delay AD and slow down progression of this devastating disease.
The new combined vaccination approach could potentially be used to induce strong immune responses to both of the hallmark pathologies of AD in a broad population base of vaccinated subjects with high MHC (major histocompatibility complex) class II gene polymorphisms, the new paper concludes.
The latest article, ‘Testing a MultiTEP-based combination vaccine to reduce Aβ and tau pathology in Tau22/5xFAD bigenic mice’ (2019) by H Davtyan, A Hovakimyan, SK Shabestari, T Antonyan, MA Coburn, K Zagorski, G Chailyan, I Petrushina, O Svystun, E Danhash, N Petrovsky, DH Cribbs, MG Agadjanvan, M Blurton-Jones and A Ghochikyan, has been published in Alzheimer’s Research & Therapy (BMC, Springer Nature). DOI: 10.1186/s13195-019-0556-2
Also see ‘A MultiTEP platform-based epitope vaccine targeting the phosphatase activating domain (PAD) of tau: therapeutic efficacy in PS19 mice’ (2019) in Scientific Reports
The research is funded by various R01 grants from the National Institutes for Health in the USA.

U.S. appeals court upholds risk payments to health insurers

A U.S. appeals court upheld the validity of a federal program governing the payment of billions of dollars to insurers under the Affordable Care Act, reversing a lower court ruling that had prompted the White House to temporarily suspend payments.
Tuesday’s 3-0 decision by the 10th U.S. Circuit Court of Appeals in Denver is a victory for insurers that feared the Feb. 2018 lower court ruling and payments suspension could drive up premium costs and cause market turmoil.
The appeals court said the Department of Health & Human Services did not act arbitrarily and capriciously in implementing its “risk adjustment” payments program.
That program is intended to create incentives for insurers to cover sicker patients, including those with pre-existing conditions, by paying them with money collected from insurers that enroll healthier patients.
Circuit Judge Scott Matheson said HHS acted reasonably in using a formula that relied on the “statewide average premium,” or average of applicable premiums that insureds pay health insurers in a state, to calculate collections from or payments to insurers.
He said HHS justified its approach over alternatives such as using the plans’ own premiums, which it feared could dissuade high-risk plans to lower prices, to make the program “budget-neutral,” where total charges would equal total payments.
“Courts cannot second guess an agency’s rulemaking decision when it provided reasons for its chosen course of action,” Matheson wrote.

Tuesday’s decision overturned a ruling by U.S. District Judge James Browning in Albuquerque, New Mexico that had set aside payment rules for 2014 through 2018.
The case had been brought by New Mexico Health Connections, which had said the rules were slanted to favor large insurers over “smaller competitors” such as itself.
Lawyers for the insurer did not immediately respond to requests for comment. HHS had no immediate comment.
The Trump administration announced the payments suspension on July 7, 2018, freezing $10.4 billion for the 2017 benefit year, saying Browning’s ruling made it necessary but that it was “disappointed” with the ruling.
While the administration has long criticized the Affordable Care Act, it ended the suspension 2-1/2 weeks later to “mitigate some of the uncertainty caused by the New Mexico litigation,” after insurers said the suspension could upset markets.
HHS later issued new risk adjustment rules for 2017 and 2018, with additional justifications that Matheson said mooted New Mexico Health Connections’ claims for those years.
The case is New Mexico Health Connections v U.S. Department of Health & Human Services et al, 10th U.S. Circuit Court of Appeals, No. 18-2186.

What Awaits Mereo BioPharma (MREO) In 2020

Mereo BioPharma Group plc (MREO) has gained nearly 300% in the last 4 trading days as it gears up for a couple of important clinical trial catalysts in the coming year.
The Company’s lead drug candidate is Setrusumab, a potential treatment for osteogenesis imperfecta, also known as brittle bone disease. Setrusumab was acquired from Novartis in 2015.
Osteogenesis imperfecta, or OI, is a rare genetic disorder characterized by bone fragility leading to fracture and bone deformity, chronic bone pain and reduced mobility. (Source: NCBI). An estimated 20,000 to 50,000 people in the United States have osteogenesis imperfecta, according to reports. To date, there are no approved treatments for OI.
In a phase IIb study, which evaluated Setrusumab in adults with Type I, III or IV osteogenesis imperfecta, dubbed ASTEROID, a trend of reduction in fractures was observed in the high dose cohort. The results of this study were reported last month.
The primary endpoint of the ASTEROID study was change in Trabecular Volumetric Bone Mineral Density (Tr vBMD) of the radius (wrist) over baseline after 12 months of treatment. However, this was not met but an increase in total vBMD at the wrist, a secondary endpoint of the study, was observed and reached statistical significance in the medium and high dose cohorts, according to the Company.
Despite the ASTEROID study not meeting the primary endpoint, the Company is confident of Setrusumab’s potential and plans to advance the compound to a pediatric pivotal trial for the treatment of Osteogenesis Imperfecta.
Mereo’s Paediatric Investigation Plan (PIP) has been approved by the European Medicines Agency and a study design has been agreed for a pivotal study in children with OI, based on a primary endpoint of fracture rate over a 12-month period.
The pivotal study will be conducted in approximately 165 children aged 5 to less than 18 years old, with OI, initially in EU and Canada, with potential expansion into the U.S. following planned discussions with the FDA. This pivotal trial has fractures and fracture reduction as its primary endpoint.
The other investigational drugs in the pipeline are:
— MPH-966, (Alvelestat) an oral neutrophil elastase inhibitor, being explored for the potential treatment of alpha-1 antitrypsin deficiency (AATD), under a phase II study. Mereo expects to report top-line data from this study in mid-2020 and if the results are positive, will seek regulatory advice on the design of a pivotal trial. This compound was acquired from AstraZeneca in 2017.
— Acumapimod, a phase III-ready compound, for Acute Exacerbations of Chronic Obstructive Pulmonary Disease, or AECOPD.
— BGS-649 (leflutrozole) that has completed phase IIb development as a first-line therapy for the treatment of obese men with hypogonadotropic hypogonadism.
— Navicixizumab (OMP-305B83), a phase II-ready compound, for Advanced Platinum-Resistant Ovarian Cancer.
— Etigilimab, which has completed a phase 1a/b trial in advanced solid tumors.
The Company is seeking partnering opportunities for its compounds. As of June 30, 2019, Mereo had cash of £36.1 million.
Mereo BioPharma is the result of the merger between NASDAQ-listed OncoMed and London Stock Exchange’s AIM market-listed Mereo BioPharma, the combination of which was completed in April 2019. The combined company is dual-listed on the NASDAQ and AIM market.

BioXcel Therapeutics Starts Pivotal Phase 3 Studies

BioXcel Therapeutics (“BTI” or “Company”) (Nasdaq: BTAI), a clinical-stage biopharmaceutical company utilizing artificial intelligence approaches to identify and advance the next wave of medicines in neuroscience and immuno-oncology, today announced the initiation of its SERENITY (Sub-Lingual DExmedetomidine in Agitation Associated With SchizophRENIa and Bipolar Disorder STudY) program, two Phase 3 studies of BXCL501 for the acute treatment of agitation in patients with schizophrenia and bipolar disorder. Topline data from both Phase 3 trials are expected in mid-2020.
“The initiation of the SERENITY trials represents a critical next step in our aim to provide a safe and effective therapy to millions of individuals who suffer from agitation associated with schizophrenia and bipolar disorder,” commented Vimal Mehta, Chief Executive Officer of BTI. “Managing agitation in patients with neuropsychiatric disorders is a major challenge for both physicians and caregivers. BXCL501, designed as a non-invasive, fast acting therapy, may be able to overcome the limitations associated with current standards of care, as we believe it provides calming without excessive sedation while protecting the caregiver patient relationship.”

Drugmakers to hike U.S. prices of over 200 meds

Reuters reports that pharmaceutical companies, including Pfizer (NYSE:PFE), GlaxoSmithKline (NYSE:GSK) and Sanofi (NASDAQ:SNY), are planning to raise prices on more than 200 medicines in the U.S. tomorrow.
The price hikes are expected to be relatively modest, almost all below 10% and half between 4 – 6%.
Pfizer intends to boost prices on more than 50 drugs, including breast cancer med Ibrance (palbociclib) and arthritis/UC med Xeljanz (tofacitinib). A spokesperson stated that the average increase will be 5.6%.
GSK plans to raise prices on more than 30 medicines by 1 – 5%.
Sanofi will follow suit on ~10 drugs by 1 – 5%.
According to healthcare research shop 3 Axis Advisors, Teva Pharmaceutical Industries (NYSE:TEVA) raised prices on more than 15 drugs by more than 6%.

Research Continues Into Shingles Vax for Certain Cancer Patients

In December 2018, we reported on a study that found higher risk of herpes zoster, or shingles, in certain cancer patients, and discussed the roles that vaccine development could potentially play in preventing these painful episodes. Here, we examine research on zoster vaccines in immunocompromised patient populations appearing after that initial study was published.
In November, Clinical Infectious Diseases published a systematic review of 34 studies that examined risk of herpes zoster among patients with hematopoietic cell transplants, hematologic and solid tumors, as well as solid tumor malignancies, and other immunocompromised populations, such as HIV. The authors found estimates of herpes zoster incidence ranging from 9 to 92 cases/1,000 patient-years. Similar to the December 2018 study, the CID authors found incidence was higher in patients with hematopoietic cell transplants, followed by solid organ transplant and solid tumor malignancies.
In February, The Lancet Infectious Diseases published a summary of guidelines from the most recent European Conference on Infections in Leukaemia about vaccination in patients with hematologic malignancies who did not have transplantations.
Examining patients with multiple myeloma, the authors recommended valacyclovir prophylaxis, an antiviral used to treat active infections including shingles. Zoster live attenuated vaccine is contraindicated in such patients, the authors noted, and there are no data in this population yet about an approved inactivated subunit vaccine, though it is “much awaited” for these patients.
The guidelines also observed that there are no data on either the live attenuated or the inactivated vaccine among patients with chronic lymphocytic leukemia, despite their elevated risk for zoster attacks.
“The inactivated zoster vaccines should be assessed in adult and child patients with [hematological] disorders,” the authors said.
Researchers continued to examine the role of inactivated vaccines against herpes zoster in patients with solid tumor malignancies. A study published in August found that an inactivated varicella zoster virus vaccine was effective, meeting pre-specified criteria for preventing confirmed herpes zoster infection in patients with solid tumor malignancies who were receiving chemotherapy. However, the vaccine was not effective in patients with hematologic malignancies.
But a second study found that the adjuvanted recombinant zoster vaccine, Shingrix, produced an immunologic response in a cohort of adult patients with hematological malignancies during or soon after they underwent cancer treatment compared to placebo.
Adding to the evidence for the effectiveness of Shingrix, a July study also found that patients undergoing autologous hematopoietic stem cell transplantation who received the vaccine had lower incidence of shingles versus patients who received placebo.
Alison Freifeld, MD, of the University of Nebraska Medical Center in Omaha, who was not involved in the research, told MedPage Today in July that transplant patients couldn’t receive the zoster vaccine until at least 2 years after transplantation, due to the potential risk of a patient developing disseminated vaccine strain live virus.
“[Shingrix] is a great advantage for autologous transplant patients because it permits the vaccine to be given much earlier than the previous live virus vaccine,” she said.
Jeanne Marrazzo, MD, of the University of Alabama at Birmingham, told MedPage Today at that time that “protection was less than that observed in immune-competent people, which the investigators attribute to a generally weaker immune system in the HSCT recipients.”
“Regardless, this outcome is considerably better than no vaccine, which was the earlier alternative,” Marrazzo added.
In addition to cancer patients, researchers continued to discuss the use of both live-attenuated vaccines and inactivated vaccines among immunocompromised populations, such as patients with autoimmune conditions.
And research presented at the American College of Rheumatology annual meeting in November provided reassuring evidence about the safety of live attenuated herpes zoster vaccine, Zostavax, among patients treated with tumor necrosis factor inhibitors. A randomized trial found that no patients developed varicella (chicken pox, the predecessor to shingles) infections or shingles attacks, and there was no difference in rates of rheumatologic disease flare between groups receiving vaccine or placebo.

AHA largely silent on suing patients for unpaid bills, as chair’s hospital partakes

The American Hospital Association has stayed relatively quiet on suing patients for overdue bills despite growing scrutiny around the controversial tactic, according to Kaiser Health News.
AHA, which represents about 5,000 hospitals, has published few guidelines on aggressive collection practices like suing, seizing homes or wages and pushing families into bankruptcy over unpaid medical bills. Morris Plains, N.J.-based Atlantic Health System, the hospital that the association’s board chair leads, has sued patients more than 8,000 times in 2019 for unpaid bills, according to KHN‘s review of court records.
In a statement to KHN, an Atlantic Health System spokesperson said its “billing policy complies with all state and federal guidelines. While we are willing to assist patients no matter their financial situation, those who can pay should do so.”
At the same time, Yale New Haven (Conn.) Health, whose CEO also sits on AHA’s board, rarely sues patients for bills, with one leader telling KHN they haven’t signed off on a legal action against patients since 2015.
Still, several health systems, like UVA Health System in Charlottesville and Richmond-based Virginia Commonwealth University Health System have faced criticism for suing patients. The AHA still has not issued an industry-wide standard on the practice.
When asked this month about the practice, AHA CEO Rick Pollack told KHN: “We are reevaluating the guidelines [for collections and financial assistance] to ensure they best serve the needs of patients.” AHA has previously said billing offices should “assist patients who cannot pay” and treat them with “dignity and respect.”
For the full article, click here.

Cancer misinformation leads popular fake health news on social media: NBC

In 2019, the most popular articles sharing unproven cures and other health misinformation collectively received more than 12 million shares, comments and reactions on social media, according to an NBC News analysis.
Researchers compiled a list of articles touting false information that received more than 25,000 engagements on social media by searching for keywords related to the most common diseases and causes of death, as well as oft-targeted health topics such as vaccines, fluoride and natural cures. Many on the resulting list of 80 viral news items claimed that governments and medical communities were conspiring to withhold healthcare information and presented “cures” not backed by scientific proof as an alternative to common medical treatment of life-threatening diseases.
According to the analysis, the most viral health misinformation of 2019 pertained to cancer, followed by unproven cures and vaccines. In some cases, these illegitimate stories completely overshadowed real news on their respective topics. For example, the cancer-related article that received the most social media engagement this year comprised several conspiracies, including one claiming physicians and federal health organizations are hiding a cure for cancer.
The majority of the articles’ online engagement came via Facebook. In a statement to NBC News, a company spokesperson said, “While we have made progress this year, we know there is more work to do. We hope to continue our partnership with health organizations to expand our work in this space.”
Read the full report here.

Med Student Accused of Attempt to Smuggle Cancer Research to China

A Harvard University medical student and cancer researcher who allegedly tried to smuggle stolen scientific material into China has been charged and will be held without bail, a federal judge in Boston ruled Tuesday.
The 29-year-old Chinese student, Zaosong Zheng, was found with 21 vials in his luggage as he prepared to board a flight at Logan International Airport bound for Beijing on Dec. 9, court documents stated. The vials were wrapped in a plastic bag and hidden inside a sock.
Zheng was a cancer researcher who earned an educational visa with Beth Israel Deaconess Medical Center last year, according to The Boston Globe. Beth Israel fired Zheng after he was arrested for allegedly making false statements about the items in his bags. His visa was also revoked.

The vials appeared to contain a brown liquid and were being tested to confirm whether they were stolen from the lab. If so, Zheng could face more charges.
“Zheng’s appointment to [Beth Israel] was not an accident; he was knowingly gathering and collecting intellectual property from [Beth Israel] possibly on behalf of the Chinese government,” FBI agent Kara Spice said in a court affidavit.
Some of the vials included a colleague’s work Zheng said he had replicated without the lab’s knowledge, according to court documents.
Zheng said he had intended to “immediately” head to his lab in China to work with the vials and publish a paper taking credit for the findings, according to the documents.
U.S. District Court Magistrate Judge David H. Hennessy granted a request from federal prosecutors to detain Zheng until he can be tried, acknowledging his connections to the Chinese government made him a flight risk, the Globe reported.

Hain Celestial target hiked to $30 from $25 by Jefferies

Maintains Buy

Sol-Gel target upped to $26 from $23 by Wainwright

Maintains Buy

Neovasc files U.S. application for Reducer

Thinly traded nano cap Neovasc (NASDAQ:NVCN) is up 66% premarket in reaction to the filing of a U.S. marketing application for its Neovasc Reducer for the treatment of refractory angina. The submission includes a request for an advisory committee meeting.
The device was CE Mark’d in November 2011.

‘Abuse of the system’: Health economist on the rising costs of combination drugs

Drugmakers for years have developed combination drugs, medications that combine two or more drugs into a single pill, in an effort to improve convenience for patients. However, the prices of these combination drugs usually cost more than the sum of their generic active ingredients, according to NBC News
One example is the generic acid reflux drug Zegerid, manufactured by Dr. Reddy’s Laboratory. Zegerid’s list price is $14,213 for a 90-day supply, significantly higher than the sum of the prices of its main active ingredients: omeprazole and sodium bicarbonate. With a coupon, the two main ingredients would cost just $34 for a 3-month period.
“We’re not talking about transformative new therapies,” Chana Sacks, MD, an internist and medical researcher at Boston-based Massachusetts General Hospital, told NBC when asked about combination drugs. “We’re talking about very small tweaks to medicines that we have been using for these very reasons for years.”
Dr. Sacks authored a study on combination drugs in 2018 in which she found that brand-name combination drugs cost Medicare $925 million more in 2016 than their generic components.
In addition, drugmakers have continued to increase the prices on these reformulated combination treatments.
Vimovo, a combination painkiller manufactured by Horizon, has increased by more than 1,600 percent since 2013, according to NBC News. 
Horizon purchased Vimovo from AstraZeneca in 2013. Its list price was $114 for a two-month supply. Within weeks, the list price was raised to $799, and nine price hikes later, the painkiller costs $2,482 for a two-month supply.
The price hikes have largely gone unnoticed by patients because drugmakers offer financial assistance programs to them, or patients only pay attention to their copay amounts, according to the report.
“It’s an abuse of the system,” Devon Herrick, a health economist who examined drugmakers who reformulate over-the-counter drugs into expensive combination drugs. “Their business model essentially involves gouging insurers and health plans, which ultimately costs consumers,” he wrote in an analysis.
Mr. Herrick sees the financial assistance programs as a way to work around pharmacies that might suggest less expensive options to patients.
“If a drug company has to go around the local pharmacy and sign up independent pharmacies, it’s a case of gaming the healthcare system,” Mr. Herrick told NBC. “They try to circumvent the local pharmacy from saying ‘that’s over the counter.'”
Horizon told NBC News that it works to offer patients low copays for its drugs through various programs, including HorizonCares. It works with specific pharmacies to dispense combination drugs and reduce patients’ out-of-pocket costs to as little as $10.
“Part of our commercial strategy to increase adoption and access to our medicines in the face of these incentives to use generic alternatives is to offer physicians the opportunity to have patients fill prescriptions through independent pharmacies participating in our HorizonCares patient access program, including shipment of prescriptions to patients,” a 2019 quarterly filing from Horizon reads.

Americans Need to Tackle Youth Obesity: U.S. Task Force

Childhood obesity is such a crisis in America that officials have been updating recommendations for how early intervention should begin.
Obesity now affects as many as 20% of the nation’s children.
The U.S. Preventive Services Task Force reviewed the literature on kids between 2 and 18 years of age and now recommends that kids over 6 be screened using a measure of body fat based on height and weight called Body Mass Index (BMI). The Task Force also recommends intensive behavioral therapy for those kids who are obese. Behavioral therapy was shown to reduce blood pressure, although it did not show any reduction in cholesterol.
While the Task Force saw no benefit to performing obesity screenings on kids younger than 6, this isn’t to say it’s not a problem. Children between the ages of 2 and 5 as a group showed a sharp increase in obesity rates over just the last few years, according to a report from the American Academy of Pediatrics (AAP).
The AAP’s 2018 report also found an upward trend for all definitions of overweight and obesity among 2- to 19-year-olds, most pronounced among adolescents. And some ethnic groups are at greater risk than others. White and Asian American children have significantly lower rates of obesity than black and Hispanic kids. The report added that, contrary to the notion that obesity in youth has remained stable or decreased in recent years, it found no evidence of a decline at any age.
Because of the lifelong negative health consequences of obesity, it’s important to talk to your child’s doctor about steps to take to stop weight gain as early as possible.
More information
The AAP has more on childhood obesity and how to prevent it.

Big Advances Made Against Diabetes in 2019

A new artificial pancreas system, drugs that help control blood sugar and protect the heart and the kidneys, a new medication that delays type 1 diabetes, and a new way to track blood sugar throughout the day — 2019 was a pretty big year in diabetes care.
“This has been a good year for patients who have diabetes. There have been a lot of changes and there are more options. These changes will have far-reaching benefits,” explained Dr. Akankasha Goyal, an endocrinologist at NYU Langone Health in New York City.
In case you missed some of the latest diabetes developments, here’s a look at some of what happened in 2019:
Delaying type 1 diabetes
An immunotherapy treatment called teplizumab delayed the onset of type 1 diabetes in people who were at high risk of developing the disease. The drug is also now being tested in a phase 3 trial in people who were recently diagnosed with type 1 diabetes.
Another drug — anti-thymocyte globulin (ATG) — was given to people with newly diagnosed type 1 diabetes. Two-year study results were released this year. The drug helped preserve the function of insulin-producing beta cells and lowered blood sugar levels.
“These were amazing results in a new-onset type 1 diabetes population,” said Sanjoy Dutta, vice president of research at JDRF (formerly the Juvenile Diabetes Research Foundation). In addition to teplizumab and ATG, there have been a number of other promising drug studies this year, Dutta said.
The buzz on diabetes technology
In mid-December, the U.S. Food and Drug Administration approved the Tandem Control-IQ, an algorithm that can be used with the Tandem t:slim X2 insulin pump and the Dexcom G6 continuous glucose monitoring system. The algorithm itself could potentially be used with other devices as well. The algorithm isn’t yet designed to be free of user input. People have to enter the amount of carbohydrates they eat into the machine so it can calculate the proper insulin dose.
Another system — the iLet by Beta Bionics — was given “Breakthrough Device” status by the FDA in December. That means the device can move through the approval process faster. This artificial pancreas system is designed to be fully automatic. The only input users need to provide is their weight.
A different type of advancement in technology came for people using continuous glucose monitors (CGM). In June, the FDA approved the Eversense CGM by Senseonics. Instead of a sensor worn outside of the body with a thin wire inserted under the skin, the Eversense sensor is implanted under the skin and is worn for 90 days. Other CGM sensors are typically worn for a week or two. Information from the sensor is sent to an app on the user’s phone via a transmitter worn on the body.
“We have patients who have an implantable sensor and they absolutely love it,” said Goyal. She said it’s been a challenge getting some insurance companies to pay for it, but she’s hoping that will improve in 2020 when Medicare starts paying for the implantable sensors.
Managing heart and kidney complications
Several new classes of medications have been introduced during the last decade, including drugs known as SGLT2s (Farxiga, Jardiance, Invokana) and GLP1s (Trulicity, Victoza, Ozempic, Bydureon, Byetta, Adlyxin). Researchers are learning the drugs not only help lower blood sugar levels, they also help protect the heart and kidneys.
A study published earlier in the year found that both SGLT2s and GLP1s reduced the risk of heart and blood vessel disease in people with a history of those problems. The research also found that SGLT2s could significantly lower the risk of heart failure for people with type 2 diabetes. Both classes of drugs also showed positive effects on kidney health, but SGLT2s appeared to offer more kidney protection.
“These new classes of drugs lower blood glucose levels and help reduce the risk of complications. We don’t see that with other medications,” Goyal said.
A GLP1 medication — liraglutide (Victoza) — was approved by the FDA in June 2019 to treat type 2 diabetes in children aged 10 and older. It’s the first drug besides metformin and insulin that’s been approved for treating pediatric type 2 diabetes.
Treating severe low blood sugar
Dutta also noted that two new versions of glucagon — a hormone used to treat severe low blood sugar episodes — were approved by the FDA. One is called Baqsimi, administered through the nose via a special device. It’s the first non-injectable type of glucagon. The second version approved is an auto-injector called GVOKE, designed to be easier for caregivers to use. Until this year, glucagon was only available in a kit that required users to mix a dry powder with sterile water, and then draw it up into a syringe before injecting.
More information
Read more about treating your diabetes from the U.S. National Institute of Diabetes and Digestive and Kidney Diseases.
SOURCES: Akankasha Goyal, M.D., endocrinologist, NYU Langone Health, New York City; Sanjoy Dutta, Ph.D., vice president, research, JDRF (formerly the Juvenile Diabetes Research Foundation)

10 Biotech M&A Targets Under The Scanner For 2020

Biopharma M&A activity scaled record highs in 2019, with a few mega mergers announced involving names such as Bristol-Myers Squibb Co BMYRoche Holdings AG Basel ADR RHHBY and AbbVie Inc ABBV.
It’s feared that the frenetic pace of M&A in the sector could slow down in the coming year.
Yet there are fundamental reasons to think otherwise: there could at least be many more bolt-on acquisitions as companies, especially cash-rich big pharma companies with an ageing pipeline, strive to reinvigorate slowing growth.

The upcoming year is likely to see many hostile deals, Evaluate Pharma reported, citing Steven Slaughter, managing director of Manulife Investment Management.
“There are enough interesting targets where management teams don’t want to sell, but in the large-cap world balance sheets are becoming so large they just have to deploy some capital,” Slaughter was quoted as saying at the Jefferies Healthcare Conference.
Increased scrutiny by regulators could serve as a dampener. In 2019, the FTC made several second requests, requiring additional information for vetting M&A transactions.

10 Biotech M&A Targets For 2020

The following are potential targets that could find themselves in the M&A mix in the coming year.
  • Gene editing company Crispr Therapeutics AG CRSP is speculated to be a potential target for Vertex Pharmaceuticals Incorporated VRTX, given the licensing agreement in place between the companies. Incidentally, Vertex itself is considered an M&A target.
  • Alnylam Pharmaceuticals, Inc. ALNY, which has the first-ever FDA-approved RNAi therapeutic in Onpattro, is also a potential M&A target. Analysts single out Sanofi SA SNY as the likely pursuer, given the stake it has in Alnylam. It should be noted here that Sanofi recently agreed to buy Synthorx Inc THOR for $2.5 billion.
  • Amarin Corporation’s AMRN attraction as an M&A target has increased following the FDA nod for an expanded indication for its fish oil pill Vascepa.
  • Incyte Corporation INCY, which has a blockbuster commercial product in Jakafi.
  • Global Blood Therapeutics Inc GBT, which recently received the FDA nod for its sickle cell disease treatment Oxbryta
Several gene therapy companies are discussed as M&A prospects. Notable among them are:
  • Uniqure NV QURE.
  • Sarepta Therapeutics Inc SRPT. Following the recent licensing agreement with Roche, the probability of an outright sale has fallen.
  • Regenxbio Inc RGNX
  • Solid Biosciences Inc SLDB
  • BioMarin Pharmaceutical Inc. BMRN

California pharmacies rarely take back unused opioids

Only 1 in 10 California pharmacies have programs to take back unused prescription opioids and just one in five give consumers accurate disposal information, according to a study that suggests drugstores could do more to help combat substance abuse.
For the “secret shopper”-style study, researchers called 898 pharmacies in California to inquire about the availability of take-back programs for leftover opioids and antibiotics, and find out how to safely dispose of these medicines at home.
“The danger of unused and unwanted prescription medication is substantial – from accidental childhood poisoning to pollution to intentional misuse,” said senior study author Dr. Hillary Copp of the University of California, San Francisco.
“The FDA recommends dropping off medications at a take-back site as the best option for disposal,” Copp said by email. “However, there are specific recommendations for medication disposal at home if the consumer does not have access to a take-back site.”
Just 19% of pharmacies correctly told callers they should bring unused opioids back to a drugstore or flush unused opioids down the toilet, the study found. Only 11% of pharmacies offered to take back unused opioids at their location.
With antibiotics, 47% of pharmacies correctly advised callers to return leftovers to a drugstore or to mix unused medicines with unpalatable substances like coffee grounds or kitty litter and place in a sealed container before tossing the drugs in the trash. Only 19% of pharmacies offered to take back unused antibiotics.
Tossing leftover antibiotics in the trash helps prevent people from taking them in the future for illnesses they can’t cure, which contributes to the rise of antibiotic-resistant superbugs that can’t be treated with available medicines, Copp said. Flushing antibiotics might get them into the water supply, also contributing to antibiotic resistance.

Flushing opioids, however, prevents them falling into the wrong hands and contributing to substance misuse, addiction and overdoses. Addicts might still take opioids they find in the trash, even mixed with dirt or kitty litter or other substances, Copp said.
All of the secret shoppers in the study posed as parents of children who recently had surgery. Callers asked pharmacies what to do with two leftover medications: the antibiotic Bactrim (sulfamethoxazole-trimethoprim), and liquid Hycet (hydrocodone-acetaminophen), a pain reliever containing an opioid compound.
Pharmacies gave callers the correct information about disposal of antibiotics and opioids far more often on weekdays than on weekends.
One limitation of the study is that results from California, where about 10% of the nation’s pharmacies are located, might not reflect what would happen elsewhere, the study team notes in the Annals of Internal Medicine.
Still, “the lack of consistent and clear information for patients is disturbing, particularly given risks for improper use of medications that are left in the home,” said Stacie Dusetzina, a researcher at Vanderbilt University School of Medicine in Nashville, Tennessee, who wasn’t involved in the study.
“Given the different strategies for disposal for different medicines, it seems that offering patients more opportunities to bring drugs back to pharmacies for proper disposal would help,” Dusetzina said by email.
The study results suggest that many pharmacies may be falling short as educators and as places for safe disposal, said Dr. Chana Sacks of Massachusetts General Hospital and Harvard Medical School in Boston.

“Patients may have other sources of information for questions about medication disposal – they might look it up on the internet before calling their pharmacy, for example,” Sacks, who wasn’t involved in the study, said by email. “But ideally pharmacies would be a place patients can count on to receive accurate information about all aspects of medications, including safe disposal.”
SOURCE: Annals of Internal Medicine, online December 30, 2019.