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.

https://www.marketwatch.com/story/fda-to-ban-fruity-e-cigarette-pod-flavors-but-allow-tank-vaping-systems-2019-12-31?siteid=rss&rss=1

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.

https://www.eurekalert.org/pub_releases/2019-12/uops-dft123019.php

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.

https://www.eurekalert.org/pub_releases/2019-12/fu-pdv122919.php

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.

https://www.reuters.com/article/us-usa-obamacare-risk-payments-decision/u-s-appeals-court-upholds-risk-payments-to-health-insurers-idUSKBN1YZ1BW

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.

https://www.nasdaq.com/articles/what-awaits-mereo-biopharma-mreo-in-2020-2019-12-29

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.”

https://www.biospace.com/article/releases/bioxcel-therapeutics-announces-initiation-of-pivotal-phase-3-studies-of-bxcl501-for-acute-treatment-of-agitation-in-patients-with-schizophrenia-and-bipolar-disordertopline-results-expected-in-mid-2020/

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%.

Selected tickers: BIB, THW, ARKG, BME, GRX, IXJ, BIS, CHNA, IDNA, GDNA, KMED, XBI, XLV, BMY, AMGN, BIIB, GILD, VRTX, MRK, LLY, AZN, AGN, JNJ, ABBV, TAK, NVS, OTCQX:RHHBY, NVO

https://seekingalpha.com/news/3529000-drugmakers-to-hike-u-s-prices-of-over-200-meds